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Noted This Week - 2019 Archive

Archive of cancer-related news briefs, by week, for 2019


December 2019

December 27–January 2

  • The FDA approved maintenance olaparib (Lynparza; AstraZeneca) in patients with BRCA-mutated metastatic pancreatic cancer whose disease has not worsened after 16 weeks of chemotherapy. The approval is based on the phase III POLO trial, in which patients treated with the PARP inhibitor had a median progression-free survival (PFS) of 7.4 months, compared with 3.8 months in patients who received a placebo. The FDA also approved the BRACAnalysis CDx (Myriad Genetics) companion diagnostic.
  • The Trump administration announced a policy to stop the sale of flavored electronic cigarette cartridges other than menthol and tobacco. According to the policy, companies must halt manufacturing, distribution, and sale of flavored cartridges within 30 days. “Our action today seeks to strike the right public health balance by maintaining e-cigarettes as a potential off-ramp for adults using combustible tobacco while ensuring these products don’t provide an on-ramp to nicotine addiction for our youth,” said Health and Human Services Secretary Alex Azar.
  • PD-1 inhibitors may have a greater survival benefit than PD-L1 inhibitors, according to a meta-analysis in JAMA Oncology. Researchers analyzed 19 randomized clinical trials involving the agents and found that PD-1 inhibitors led to better overall survival and PFS compared with PD-L1 inhibitors; side effects did not significantly differ between the groups.

Earlier This Year:

 ::  January  ::  February  ::  March  ::  April  ::  May  ::  June  ::  July  ::  August  ::  September  ::  October  ::  November


Noted This Week Archive:

 ::  2023  ::  2022  ::  2021  ::  2020  ::  2019  ::  2018  ::  2017  ::  2016  ::  2015  ::  2014  ::  2013  ::  2012  ::  2011

  • A study in Nature reported that an artificial intelligence tool may be better at diagnosing breast cancer than human experts. The tool was trained on breast cancer scans from 91,000 women in the United States and UK, and then tested on a new set of scans. Overall, it resulted in 6% fewer false positives and 9% fewer false negatives than standard clinical practice in the United States, and 1% fewer false positives and 3% fewer false negatives than standard practice in the UK.
  • Pharmaceutical companies will increase prices in the United States for more than 200 drugs, including more than 50 Pfizer drugs, more than 30 GlaxoSmithKline drugs, and more than 15 Teva drugs. The list includes cancer agents such as the CDK4/6 inhibitor palbociclib (Ibrance; Pfizer) and the PARP inhibitor niraparib (Zejula; GlaxoSmithKline).

December 20–26

  • The FDA granted accelerated approval to fam-trastuzumab deruxtecan-nxki (Enhertu; AstraZeneca/Daiichi Sankyo) for patients with inoperable or metastatic HER2-positive breast cancer who have received at least two other anti-HER2 drugs. The decision was based on the phase II DESTINY-Breast01 trial, in which patients had an overall response rate of 60.9% and a median progression-free survival of 16.4 months. The agent, also known as trastuzumab deruxtecan, is an antibody–drug conjugate consisting of the HER2-targeting monoclonal antibody trastuzumab attached to a topoisomerase I inhibitor.
  • As part of a broader spending package, President Donald Trump signed legislation prohibiting the sale of tobacco and electronic cigarettes to anyone under the age of 21. The Tobacco 21 measure, which amends the Federal Food, Drug, and Cosmetic Act, went into effect immediately.
  • The Trump administration is considering issuing an executive order requiring federally funded research to be freely available as soon as it is published. More than 125 organizations—including the American Association for Cancer Research, the American Cancer Society, and the American Society of Clinical Oncology—have signed a letter expressing concerns about the order, saying it “would jeopardize the intellectual property of American organizations engaged in the creation of high-quality peer-reviewed journals and research articles and would potentially delay the publication of new research results.”
  • Juvisé Pharmaceuticals paid AstraZeneca $181 million up front for the commercial rights to the aromatase inhibitor anastrozole (Arimidex) and the androgen-receptor inhibitor bicalutamide (Casodex). The deal, which could be worth up to an additional $17 million, will allow Juvisé to sell the drug in more than 50 countries, including Germany, France, and Switzerland.
  • Johnson & Johnson announced that it acquired Taris Biomedical for an undisclosed sum. Taris’s lead product is TAR-200, a drug delivery system designed to continuously release therapies into the bladder to treat cancer and other diseases. The platform is being tested with the chemotherapy agent gemcitabine in a phase I trial of muscle-invasive bladder cancer.

December 13–19

  • The U.S. Congress approved the federal budget for fiscal year (FY) 2020, which President Donald Trump is expected to sign. Overall, the NIH will receive $41.7 billion, an increase of $2.6 billion over FY2019. The NCI will receive a $296 million funding increase, bringing its total budget to $6.44 billion. Of that sum, $195 million has been allocated for the Cancer Moonshot, and more than $210 million has been earmarked for additional competing grants.
  • The FDA granted accelerated approval to enfortumab vedotin-ejfv (Padcev; Astellas/Seattle Genetics) for patients with locally advanced or metastatic urothelial cancer who have received prior PD-1–PD-L1 therapy and a platinum-containing chemotherapy. The decision was based on the phase II EV-201 trial, in which 44% of patients responded to the drug and 12% experienced complete responses. Enfortumab vedotin-ejfv is the first Nectin-4–directed antibody–drug conjugate to receive the agency’s approval.
  • The FDA also approved enzalutamide (Xtandi; Astellas/Pfizer) for metastatic, castration-sensitive prostate cancer. The approval was based on the phase III ARCHES trial, in which patients treated with the agent plus androgen-deprivation therapy did not reach median radiographic progression-free survival, compared with 19.4 months in patients who received androgen-deprivation therapy alone. Enzalutamide is already approved for certain forms of castration-resistant prostate cancer.
  • Belantamab mafodotin (GSK2857916; GlaxoSmithKline) could soon be another treatment for patients with multiple myeloma who have already received other treatments. In the phase II DREAMM-2 trial, patients had received a median of seven prior therapies, and 34% responded to the therapy. The results have led GlaxoSmithKline to file for FDA approval of the immunoconjugate drug, which targets B-cell maturation antigen.
  • BeiGene announced that zanubrutinib (Brukinsa) may not be effective in Waldenström macroglobulinemia. In the phase III ASPEN trial, the therapy did not significantly improve complete or partial response rates compared with ibrutinib (Imbruvica; Janssen). A Bruton tyrosine kinase inhibitor, zanubrutinib received an accelerated approval in November for mantle cell lymphoma.
  • Bristol-Myers Squibb (BMS) won $752 million in a patent dispute with Gilead. A jury in Los Angeles, CA, determined that Gilead subsidiary Kite Pharma—with its chimeric antigen receptor (CAR) T-cell therapy axicabtagene ciloleucel (Yescarta)— infringed on a CAR T-cell patent held by BMS subsidiary Juno Therapeutics.
  • The American Association for Cancer Research (AACR) and MPM Capital issued the first two grants to fund cancer research through the AACR-MPM Oncology Charitable Foundation Transformative Cancer Research Grants program. The grants, $400,000 each over 2 years, will support research on myeloproliferative neoplasms and ferroptosis signaling.

December 6–12

  • The U.S. Senate confirmed Stephen Hahn, MD, as commissioner of the FDA by a vote of 72–18.
  • Merck announced that it will buy ArQule for $2.7 billion just as the Burlington, MA–based company presented positive results of a phase I trial of ARQ 531 in B-cell malignancies at the 2019 American Society of Hematology (ASH) Annual Meeting in Orlando, FL. An inhibitor of wild-type and C481S-mutant BTK, ARQ 531 led to an overall response rate of 89% in evaluable patients with chronic lymphocytic leukemia and 50% of those with Richter transformation; one patient with follicular lymphoma and one with diffuse large B-cell lymphoma experienced partial responses. ArQule had struggled in recent years as it tried to develop the c-MET inhibitor tivantinib, which had disappointing results in multiple trials.
  • In a phase I/Ib trial, Genentech’s mosunetuzumab induced complete remissions in patients with poor-prognosis non-Hodgkin lymphoma, including 22% of those whose disease had advanced despite receiving chimeric antigen receptor (CAR) T-cell therapies, researchers reported at ASH. In addition, after a median follow-up of 6 months, 24 of 29 patients with slow-growing lymphomas and 17 of 24 patients with fast-growing lymphomas who experienced complete remission remained free of disease. An off-the-shelf synthetic bispecific antibody, mosunetuzumab targets CD3 on T cells and CD20 on B cells.
  • A novel CAR T-cell therapy bearing two molecules that bind BCMA, JNJ-4528 led to high response rates in patients with relapsed/refractory multiple myeloma, according to data from a phase Ib/II study presented at ASH. All of the first 29 patients enrolled in the trial had clinical responses to JNJ-4528 after a median follow-up of 6 months. In addition, 66% experienced a stringent complete response, meaning that no myeloma proteins or cells could be found in blood, urine, or bone marrow.
  • In preclinical studies, Fate Therapeutics’ cellular immunotherapy FT596 killed cancerous white blood cells as effectively as standard CAR T-cell therapy, researchers announced at ASH. An off-the-shelf CAR natural killer (NK) cell product, FT596 is created with human induced pluripotent stem cells and genetically engineered to target CD19 and carry CD16, which boosts the NK cells’ ability to kill malignant cells, and IL15, which stimulates proliferation of NK cells. FT596 eliminates the expensive, time-consuming, and complex manufacturing of traditional CAR T cells.
  • At the 2019 San Antonio Breast Cancer Symposium (SABCS) in Texas, researchers reported that adding the small-molecule tyrosine kinase inhibitor tucatinib to trastuzumab (Herceptin; Genentech) and chemotherapy may improve survival in patients with locally advanced, inoperable, or metastatic HER2-positive breast cancer who have received prior therapies. In the phase III HER2CLIMB trial, patients treated with the tucatinib combination had a median overall survival (OS) of 21.9 months and a median progression-free survival (PFS) of 7.6 months, compared with 17.4 months and 5.4 months, respectively, in patients who received only trastuzumab and chemotherapy. Findings were simultaneously published in The New England Journal of Medicine; Seattle Genetics announced topline results in October.
  • Pertuzumab (Perjeta; Genentech) plus trastuzumab and chemotherapy may be effective in early-stage, HER2-positive breast cancer, according to results presented at SABCS. After 6 years of follow-up in the phase III APHINITY trial, the pertuzumab combination reduced the risk of breast cancer recurrence by 24% compared with trastuzumab plus chemotherapy, an effect that was even more pronounced in patients whose disease had spread to their lymph nodes.
  • Long-term results of the IBIS-II trial, presented at SABCS and simultaneously published in The Lancet, indicate that the aromatase inhibitor anastrozole continues to protect high-risk women from developing breast cancer after they stop taking it. After median follow-up of 10.9 years, women who took 5 years of anastrozole were 50% less likely to have developed breast cancer than women who received a placebo.
  • A new analysis of the KEYNOTE-522 trial revealed that the PD-1 inhibitor pembrolizumab plus chemotherapy may be particularly effective in certain patients with early-stage triple-negative breast cancer, namely those whose cancer has spread to their lymph nodes and/or those with stage III disease. In the phase III trial, reported at SABCS, patients with lymph node–positive disease had a pathologic complete response (pCR) rate of 64.8%, compared with 44.1% in those who received chemotherapy alone; patients with stage IIIa and stage IIIb disease had pCR rates of 66.7% and 48.6%, respectively, compared with 42.1% and 23.1% in patients who received chemotherapy. Researchers reported overall results at the ESMO Congress 2019 in September.

November 27–December 5

  • The FDA approved the PD-L1 inhibitor atezolizumab (Tecentriq; Roche/Genentech) plus chemotherapy in newly diagnosed patients with metastatic nonsquamous non–small cell lung cancer who do not have EGFR or ALK mutations. The approval was based on the phase III IMpower130 trial, in which patients treated with the combination had a median progression-free survival (PFS) of 7.2 months and a median overall survival of 18.6 months, compared with 6.5 months and 13.9 months, respectively, in patients who received chemotherapy alone.
  • The agency also issued a final guidance on adaptive designs for clinical trials. The guidance describes when and how adaptive trials—those that are modified based on accumulating data—should be used to provide evidence of the effectiveness and safety of drugs and biologics. The document also outlines what information should be reported to the FDA to facilitate the agency’s evaluation of such trials.
  • Foundation Medicine announced that the FDA approved FoundationOne CDx as a companion diagnostic for alpelisib (Piqray; Novartis), a PI3Kα inhibitor used with fulvestrant to treat men and postmenopausal women with PIK3CA-mutant, HR-positive, HER2-negative advanced breast cancer whose disease has worsened with an aromatase inhibitor. FoundationOne CDx is a next generation–sequencing test that can detect various genomic alterations, including insertions and deletions, gene rearrangements, and microsatellite instability.
  • A study in The Lancet Oncology reported that Aveo’s VEGFR inhibitor tivozanib may be effective in patients with metastatic renal cell carcinoma who have already received at least two therapies, including a VEGFR inhibitor. In the phase III TIVO-3 trial, the agent extended median PFS by 1.7 months compared with standard sorafenib (Nexavar; Bayer), and was similarly well tolerated.
  • Eli Lilly will tap executives from Loxo Oncology as it reorganizes its cancer research division following its purchase of Loxo for $8 billion in January 2019. Loxo’s former CEO and two members of its biotech leadership team will head a group that combines the laboratories of the two companies. The new group will focus on the development of the RET inhibitor selpercatinib (LOXO-292) and the Bruton tyrosine kinase inhibitor LOXO-305, among other agents.
  • Novartis and Amgen provided data to the American Society of Hematology Research Collaborative Data Hub, making them the first pharmaceutical companies to do so. The companies contributed deidentified data from nearly 500 patients with sickle cell disease and more than 1,000 patients with multiple myeloma. Launched in 2018, the Data Hub contains data from more than 3,000 patients with sickle cell disease and 2,000 patients with multiple myeloma that’s available to researchers.

November 2019

November 22–26

  • Genentech’s atezolizumab (Tecentriq) plus bevacizumab (Avastin) may improve survival in patients with inoperable hepatocellular carcinoma, researchers reported at the ESMO Asia 2019 Congress in Singapore, China. In the phase III IMbrave 150 trial, patients treated with the PD-L1–VEGF inhibitor combination had an overall response rate of 33%, had a median progression-free survival (PFS) of 6.8 months, and had not yet reached the median overall survival (OS), compared with 13%, 4.3 months, and 13.2 months, respectively, in patients who received sorafenib (Nexavar; Bayer). The combination is FDA approved for certain types of nonsquamous non–small cell lung cancer (NSCLC).
  • Also at the ESMO Asia Congress, Takeda presented results suggesting that brigatinib (Alunbrig) is effective in patients with advanced ALK+ NSCLC who have not received a prior ALK inhibitor. In the phase III ALTA-1L trial, patients who received the ALK inhibitor had a median PFS of 24 months and an objective response rate of 74%, compared with 11 months and 62% in patients receiving crizotinib (Xalkori; Pfizer); the improvement was even more pronounced in patients with brain metastases.
  • At the 2019 Society for Neuro-Oncology Annual Meeting in Phoenix, AZ, researchers reported mixed results for Toca 511 & Toca FC, a two-part therapy that uses a retroviral vector to deliver chemotherapy to the brain. In the phase III Toca 5 trial, the therapy did not extend OS compared with standard treatment. However, it did improve OS in patients whose cancer recurred, providing the most benefit to patients with IDH1 mutations and anaplastic astrocytoma.
  • The first patient to receive treatment under “right to try” legislation died after his glioblastoma returned despite receiving the experimental immunotherapeutic vaccine ERC-1671 (Gliovac; ERC-USA). Another patient with glioblastoma has shown early signs of remission after being treated with the therapy. “Right to try” legislation allows terminally ill patients to seek experimental treatments directly from pharmaceutical companies, outside of the FDA’s expanded-access pathway.
  • Cancer patients are at a higher risk of dying from cardiovascular disease than the general U.S. population. Researchers compared 3.2 million patients diagnosed with cancer between 1973 and 2012 with the general population. They found that patients diagnosed with cancer before age 55 were more than 10 times more likely to die from cardiovascular disease than the general population, and across ages 11.3% of patients with cancer died from cardiovascular disease.
  • A consortium of healthcare, biotech, and biopharma institutions in the Boston area announced plans to create a $50 million nonprofit facility devoted to the development of cell- and gene-based therapies. The goal of the consortium is to streamline the process of developing and manufacturing these therapies by providing researchers with resources and other support. Participating institutions will include Harvard University and its affiliated hospitals, MIT, and General Electric Healthcare Life Sciences, among others.

November 15–21

  • The FDA approved the Bruton tyrosine kinase inhibitor acalabrutinib (Calquence; AstraZeneca) to treat chronic lymphocytic leukemia and small lymphocytic leukemia. The approval was based on the phase III ELEVATE-TN and ASCEND trials, in which the drug extended progression-free survival compared with standard therapies. The approval is the second to be granted through Project Orbis, a collaboration between the FDA, the Australian Therapeutic Goods Administration, and Health Canada through which drugs are simultaneously greenlighted in all three countries.
  • Bristol-Myers Squibb (BMS) completed its acquisition of Celgene. The $74 billion deal was finalized after the companies won U.S. antitrust approval for their merger, on the condition that they sell Celgene’s psoriasis drug apremilast (Otezla). Amgen purchased the drug for $13.4 billion.
  • AbbVie signed a deal with Harpoon Therapeutics worth up to $2.4 billion. The deal, which expands the existing partnership between the companies, allows AbbVie to license Harpoon’s BCMA-targeting agent HPN21, as well as up to six additional targets— including two novel TriTAC molecules designed to engage T cells in solid tumors.
  • The gonadotropin-releasing hormone antagonist relugolix (Relumina; Myovant Sciences) may successfully suppress testosterone in men with androgen-sensitive advanced prostate cancer. In the phase III HERO trial, 96.7% of men treated with the agent had sustained testosterone suppression (defined as 50 ng/dL or less), compared with 88.8% of men who received leuprolide acetate.
  • BMS announced that ipilimumab (Yervoy) plus nivolumab (Opdivo) may not improve recurrence-free survival in patients with operable high-risk melanoma whose tumors lack PD-L1, compared with nivolumab alone. The phase III CHECKMATE-915 trial assessed patients treated with adjuvant therapy after removal of stage III or IV disease. An independent data-monitoring committee recommended that the trial continue as planned to see if the CTLA4–PD-1 inhibitor combination shows a benefit in a broader population of patients.
  • The European Commission approved the PD-1 inhibitor pembrolizumab (Keytruda) as a monotherapy or in combination with chemotherapy in newly diagnosed patients with PD-L1–positive metastatic or inoperable, recurrent head and neck squamous cell carcinoma. The approval was based on the phase III KEYNOTE-048 trial, in which the agent, alone or in combination with chemotherapy, significantly extended overall survival compared with chemotherapy alone.
  • The Cancer Prevention Research Institute of Texas in Austin awarded $38 million in grants to recruit researchers to institutions in Texas. Grantees will use the money to study immunotherapy, treatments for gastrointestinal cancer, and skeletal complications associated with cancer and cancer therapies, among other subjects. Texans recently voted to continue to support the funding through state taxes.

November 8–14

  • The FDA granted an accelerated approval to zanubrutinib (Brukinsa; BeiGene) in patients with mantle cell lymphoma who have received at least one previous therapy. The approval is based on two single-arm studies in which the drug elicited an overall response rate (ORR) of 84%. A Bruton tyrosine kinase inhibitor, zanubrutinib is the first drug developed in China to be approved by the FDA.
  • Teva announced it will restart production of vincristine, a chemotherapy agent commonly used to treat pediatric cancers. A shortage of vincristine occurred after Teva stopped making the drug, and Pfizer, the sole remaining manufacturer, couldn’t keep up with a spike in demand. Teva said it will make the drug available as early as possible in 2020.
  • A second surgery followed by chemotherapy in recurrent ovarian cancer does not improve survival compared with chemotherapy alone, researchers reported in The New England Journal of Medicine. In a phase III trial, patients who had a second surgery and then chemotherapy had a median overall survival (OS) of 50.6 months and a median progression-free survival (PFS) of 18.9 months, compared with 64.7 months and 16.2 months, respectively, in patients who received only chemotherapy.
  • Aslan’s pan-HER2 inhibitor varlitinib plus capecitabine may not lead to better responses in patients with biliary tract cancer who have received prior therapy. In the phase III TreeTopp trial, the combination did not significantly improve median PFS or ORR compared with a placebo plus capecitabine. Aslan is also testing varlitinib in breast and colorectal cancers.
  • EMD Serono and Pfizer announced that the PD-1 inhibitor avelumab (Bavencio) may not be an effective maintenance therapy in patients with inoperable locally advanced or metastatic HER2-negative gastric or gastroesophageal cancers. In the phase III JAVELIN Gastric 100 trial, the therapy did not improve OS compared with chemotherapy or best supportive care. Avelumab is already approved for certain types of renal cell, urothelial, and Merkle cell carcinoma.
  • The American Lung Association released a report indicating that the 5-year survival rate in lung cancer has improved over the past decade, from 17.2% to 21.7%. According to the report, more than 228,000 people will be diagnosed with lung cancer in 2019; Kentucky has the highest rate of disease, and Utah has the lowest. Five-year survival rates ranged from 17% in Alabama to 26% in Connecticut.

November 1–7

  • President Donald Trump nominated Stephen Hahn, MD, of The University of Texas MD Anderson Cancer Center in Houston, to be the next FDA commissioner. Acting FDA commissioner Norman “Ned” Sharpless, MD, will immediately return to his previous position as director of the NCI, and Brett Giroir, MD, the assistant secretary for health at the Department of Health and Human Services, will lead the FDA until Hahn is confirmed by the Senate.
  • Three patients with blood cancers were safely treated with CRISPR gene-editing technology. Researchers removed immune cells from the patients’ blood, genetically altered the cells to recognize and fight cancer, and reinfused them. After 2 to 3 months, one patient’s cancer continued to worsen, another patient experienced stable disease, and results are not yet available for the third patient.
  • MD Anderson and Takeda entered into a collaboration to develop chimeric antigen receptor natural killer (CAR NK)–cell therapies enhanced with IL15. Takeda will gain access to MD Anderson’s CAR NK platform and the exclusive right to develop and commercialize up to four products, including therapies that target CD19 and B-cell maturation antigen.
  • Daiichi Sankyo filed a lawsuit against Seattle Genetics over linker technology used in antibody–drug conjugates (ADC). In 2008 Seattle Genetics granted Daiichi a license to use its ADC technology, an agreement that ended in 2015. Now, Seattle Genetics is laying claim to the ADC technology used in Daiichi’s HER2-targeting agent DS-8201, which it began developing during the term of the agreement— and has continued developing since.
  • The FDA announced that the Office of Hematology and Oncology Products is being reorganized and renamed as the Office of Oncologic Diseases. The new office, which still falls within the Center for Drug Evaluation and Research, will increase from three to six divisions, each responsible for reviewing products for a different category of cancers. A safety team has also been added to assess and communicate safety information during the review process.
  • The agency also approved pegfilgrastim-bmez (Ziextenzo; Novartis), a biosimilar of pegfilgrastim (Neulasta; Amgen), based on evidence that it has a similar safety and efficacy profile as the reference drug. Pegfilgrastim-bmez, which is the third approved biosimilar of pegfilgrastim, decreases the risk of infection in patients with nonmyeloid cancer who are receiving chemotherapy and have febrile neutropenia.
  • Halozyme’s experimental HA-degrading drug PEGPH20 plus chemotherapy failed to extend overall survival in patients with pancreatic cancer compared with chemotherapy alone. Consequently, the company will discontinue its development and cut 160 jobs—55% of its workforce.
  • An article in JAMA reported that 27.5% of high schoolers and 10.5% of middle schoolers classified themselves as current users of electronic cigarettes (e-cigarettes) in 2019. The analysis, based on the 2019 National Youth Tobacco Survey, estimated that 4.1 million high school students and 1.2 million middle school students used the devices. A separate study in JAMA found that mint and mango were the most popular e-cigarette flavors among youth, and tobacco-related flavors were the least popular. In response, Juul Labs announced that it will sell only menthol and tobacco flavors.

October 2019

October 25–31
This week: Special content from the 2019 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics and other news

  • Merus’s bispecific MCLA-128 (zenocutuzumab) showed activity in patients with metastatic pancreatic and non–small cell lung cancer (NSCLC) who have NRG1 fusions, researchers announced at the 2019 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in Boston, MA, October 26–30. As part of an expanded access protocol involving three patients, the HER2/HER3 antibody elicited a partial response in one patient with pancreatic cancer and stable disease in another; a third patient, who has NSCLC, experienced a partial response. Based on these preliminary data, a phase I/II basket trial has been launched.
  • At the Molecular Targets meeting, Mirati announced that its KRAS G12C inhibitor MRTX849 may be a promising therapy in solid cancers, including NSCLC, colorectal cancer, and appendix cancer. In a phase I trial, three of six patients with NSCLC and one of four patients with colorectal cancer responded to the drug; all of the other patients achieved stable disease.
  • Also at the meeting, Boehringer Ingelheim presented positive preclinical results for BI 1701963, which targets multiple KRAS mutations by binding to SOS1. The company is now testing the agent in phase I trials as a monotherapy and in combination with the MEK inhibitor trametinib (Mekinist; GlaxoSmithKline).
  • The farnesyl transferase inhibitor tipifarnib (Kura Oncology) may be effective in recurrent and metastatic head and neck cancers with HRAS mutations, researchers reported at the Molecular Targets meeting. In a phase II trial, eight of 15 patients with HRAS mutations in more than 20% of their tumor cells responded to the therapy, with a median progression-free survival (PFS) of 8.3 months.
  • AstraZeneca announced that in the phase III POSIEDON trial, the PD-L1 inhibitor durvalumab (Imfinzi) plus chemotherapy significantly prolonged PFS in patients with newly diagnosed metastatic NSCLC who have squamous or nonsquamous disease, compared with chemotherapy alone. A PFS benefit was also seen for durvalumab plus the CTLA4 inhibitor tremelimumab (AstraZeneca) and chemotherapy versus chemotherapy alone.
  • The NCI renewed a Comprehensive Partnerships to Advance Cancer Health Equity grant for The University of Texas MD Anderson Cancer Center in Houston and the University of Puerto Rico in San Juan. The $13 million grant will support collaborative research projects, community outreach, and joint education programming, including the Infection-Driven Malignancies Program for Advancing Careers and Translational Research, a genomic data center, and a community outreach center.
  • A former Juul Labs executive claims that the company knowingly sold at least 1 million contaminated nicotine pods for electronic cigarettes. In a lawsuit filed in the U.S. District Court for the Northern District of California, the executive also said that he was fired for whistle-blowing, and described a lack of interest in safety and quality control at the company. About 50 other lawsuits have been filed against Juul—primarily for vaping-related illnesses and allegedly false marketing claims.

October 18–24

  • The FDA approved niraparib (Zejula; Tesaro) for patients with advanced ovarian, fallopian tube, or primary peritoneal cancer who have been treated with at least three chemotherapies and whose tumors are positive for homologous recombination deficiency. The approval was based on the phase II QUADRA trial in which patients had an overall response rate of 24% and a median duration of response of 8.3 months. A PARP inhibitor, niraparib was previously approved as a maintenance therapy in patients who responded to chemotherapy.
  • Bristol-Myers Squibb announced that the PD-1 inhibitor nivolumab (Opdivo) plus the CTLA4 inhibitor ipilimumab (Yervoy) may improve outcomes in non–small cell lung cancer. In the phase III CheckMate-9LA trial, patients who received the combination plus chemotherapy as a first-line treatment had longer overall survival than patients who received chemotherapy alone. The combination has previously been approved for various cancers, including colorectal cancer, renal cell carcinoma, and melanoma.
  • A new CRISPR tool called prime editing may one day treat a broader range of diseases than earlier iterations of CRISPR-Cas9, scientists reported in Nature. Prime editing is a more precise editing technique where any DNA nucleotide can be changed to any other, and nucleotides can be added or removed from specific spots in the genome, even in non-dividing cells. This approach could allow scientists to repair point mutations responsible for many inherited genetic diseases.
  • Roche’s atezolizumab (Tecentriq) plus bevacizumab (Avastin) may improve prognoses for patients with inoperable hepatocellular carcinoma. In the phase III IMbrave 150 trial, patients treated with the PD-L1 inhibitor/angiogenesis inhibitor combination had longer overall and progression-free survival (PFS) than patients who received standard sorafenib (Nexavar; Bayer).
  • The tyrosine kinase inhibitor tucatinib may benefit patients with inoperable or metastatic HER2-positive breast cancer who previously received trastuzumab (Herceptin; Genentech), pertuzumab (Perjeta; Genentech) and ado-trastuzumab emtansine (Kadcyla; Genentech), according to topline results announced by Seattle Genetics. In the phase II HER2CLIMB trial, patients treated with tucatinib plus trastuzumab and capecitabine had a longer median PFS than patients who received trastuzumab plus capecitabine, a trend that was also seen in the 47% of patients with brain metastases.
  • The FDA authorized marketing of eight smokeless tobacco products called snus through its modified risk tobacco product pathway. Manufacturers can now market these products with the claim "Using General Snus instead of cigarettes puts you at lower risk of mouth cancer, heart disease, lung cancer, stroke, emphysema, and chronic bronchitis," but the products must still carry the warning statements required for all smokeless tobacco products. Also, this week, Juul Labs announced that it will suspend sales of its flavored electronic cigarette products except tobacco, mint, and menthol, and it will stop advertising to youth.

October 11–17

  • Cancer centers are experiencing a shortage of vincristine, a chemotherapeutic used to treat leukemias, lymphomas, and brain tumors, especially in children. The shortage came about after Teva stopped producing vincristine, and Pfizer, the only other manufacturer, encountered production issues. The Children’s Oncology Group has made recommendations for altering clinical trial protocols to use less of the drug, and oncologists may soon be forced to begin rationing it.
  • Eli Lilly announced that pegilodecakin plus chemotherapy may not be effective in pancreatic cancer. In the phase III SEQUOIA trial, the combination did not extend overall survival compared with chemotherapy alone in patients with metastatic disease who had already received a gemcitabine-containing therapy. The company will continue testing pegilodecakin, a PEGylated form of IL10, in combination with chemotherapy and immunotherapy in non–small cell lung cancer and renal cell carcinoma.
  • Exercise can reduce the likelihood of developing cancer and improve outcomes when people do develop cancer, according to multiple sources. The guidelines are based on research suggesting that physically active people reduce their risk of being diagnosed with certain malignancies—including colon, breast, endometrial, kidney, bladder, esophageal, and stomach cancers—by as much as 69% compared with those who are sedentary. Further exercise during or after cancer treatment is associated with longer life and improved mood and energy levels. The guidelines were issued by the American Cancer Society, the American College of Sports Medicine, and 15 other organizations.
  • Syros Pharmaceuticals announced that it will halt development of the intravenous CDK7 inhibitor SY-1365, but it will continue developing the oral CDK7 inhibitor SY-5609. So far, SY-5609 appears more selective and potent, with greater antitumor activity than SY-1365 in preclinical models. The company expects to launch a phase I trial of SY-5609 in solid tumors early next year.
  • The UK's National Institute for Health and Care Excellence approved rucaparib (Rubraca; Clovis Oncology) as a maintenance therapy in patients with relapsed ovarian, fallopian tube, or peritoneal cancer who responded to platinum-based chemotherapy. The decision was based on the phase III ARIEL3 trial, in which patients treated with the PARP inhibitor had a median progression-free survival of 10.8 months, compared with 5.4 months in patients who received a placebo. The drug will now be available to patients through England’s Cancer Drugs Fund.
  • Women speaking at cancer conferences are less likely to be addressed by their professional titles than men, according to an analysis on unconscious bias in the Journal of Clinical Oncology. Researchers examined how professional titles were used when speakers were introduced at the American Society of Clinical Oncology Annual Meetings in 2017 and 2018. They found that women were addressed by their professional titles 62% of the time, compared with 81% in men, a difference that was even more pronounced when men were doing the introductions (53% in women versus 80% in men).

October 4–10

  • Lilly Oncology announced that ramucirumab (Cyramza) plus erlotinib (Tarceva; Genentech) may improve outcomes in patients with newly diagnosed, EGFR-mutant non–small cell lung cancer. In the phase III RELAY trial, patients treated with the EGFR inhibitor/VEGFR2 inhibitor combination had a median progression-free survival of 19.4 months, compared with 12.4 months in patients who received a placebo plus erlotinib. Results were published in The Lancet Oncology.
  • GRAIL released data validating its multicancer early-detection test that analyzes cell-free DNA in blood samples. The company ran its test on 1,264 participants from the Circulating Cell-Free Genome Atlas and STRIVE studies, including 654 people with cancer. With 99.3% specificity, the test had an overall detection rate of 76% for 12 cancers that generally have poor outcomes, including detection rates of 39% and 69% for stage I and stage II disease, respectively.
  • Bristol-Myers Squibb’s nivolumab (Opdivo) plus bevacizumab (Avastin; Genentech) may be a promising therapy in patients with relapsed epithelial ovarian cancer, according to findings in JAMA Oncology. In a phase II trial of 38 women, 28.9% had an objective response to the drug, including 40% with platinum-sensitive disease and 16.7% with platinum-resistant disease. A PD-1 inhibitor, nivolumab is approved for a variety of cancers, including melanoma, lung cancer, and hepatocellular carcinoma.
  • At the Biopharma Congress in Washington, DC, FDA leaders expressed concern about the agency's ability to recruit and retain experts in cutting-edge scientific fields such as cell and gene therapies and oncology. “We’re dealing with the issue of a very competitive job market,” said Peter Marks, MD, PhD, director of the FDA Center for Biologics Evaluation and Review. The FDA is considering measures such as increasing salary caps and transitioning to a more academic model in which its medical oncologists are encouraged to develop expertise on a particular topic and take on responsibilities beyond day-to-day review work.
  • The FDA awarded more than $15 million in grants to 12 research groups developing therapies for rare diseases. Eight of the grants will fund clinical trials in rare cancers, including high-grade gliomas, squamous cell carcinoma, and pediatric malignant cerebellar brain tumors. The grants, awarded through the FDA’s Orphan Products Clinical Trials Grants Program, will be spread over 3 to 4 years.

September 2019

September 27–October 3
This week: Special content from the ESMO Congress 2019 and other news

  • The combination of the PD-1 inhibitor nivolumab (Opdivo; Bristol-Myers Squibb) and the CTLA4 inhibitor ipilimumab (Yervoy; Bristol-Myers Squibb) represents a new first-line treatment for advanced non–small cell lung cancer (NSCLC). In the CheckMate-227 trial, researchers found that the combination, which was compared with chemotherapy, significantly prolonged overall survival (OS)—regardless of PD-L1 expression. Median OS was 17.1 months in the combination arm vs. 14.9 months in the chemotherapy arm in patients with PD-L1 expression of at least 1%, and 17.2 months vs. 12.2 months, respectively, in patients with PD-L1 expression less than 1%. The results were presented at the ESMO Congress 2019 in Barcelona, Spain, and concurrently published in The New England Journal of Medicine (NEJM).
  • Also at the ESMO Congress 2019, the nivolumab–ipilimumab combination yielded longer 5-year OS for patients with advanced melanoma than either agent alone. In the CheckMate-067 trial, after a median follow-up of 60 months, the median OS was more than 60 months in the combination arm (median not reached), compared with 36.9 months for nivolumab alone and 19.9 months for ipilimumab alone. The results were concurrently published in NEJM.
  • New data from the phase I/II BFAST trial, presented at the ESMO Congress 2019, showed that patients with treatment-naïve NSCLC who received next-generation sequencing (NGS) of cell-free DNA responded just as well to targeted therapy as those who received a traditional tissue biopsy, researchers said. Overall, 2,188 patients received NGS results—119 had ALK+ disease, 87 of whom subsequently received the ALK inhibitor alectinib (Alcensa; Genentech). After a median follow-up of 12.6 months, the overall response rate was 87.4%, and the 12-month duration of response rate was 75.9%; median progression-free survival (PFS) was not reached.
  • Patients with metastatic urothelial carcinoma have longer PFS when initially treated with immunotherapy and chemotherapy instead of chemotherapy alone, according to results of the IMvigor130 trial presented at the ESMO Congress 2019. In the phase III study, patients who received the combination of the PD-L1 inhibitor atezolizumab (Tecentriq; Genentech/Roche) and chemotherapy experienced a statistically significant 2-month improvement in PFS compared with those who received only chemotherapy. There was a trend toward improved OS in the combination arm, but the data are not yet mature.
  • The chemotherapy cabazitaxel (Jevtana; Sanofi) improves survival in men with metastatic, castration-resistant prostate cancer following treatment with docetaxel and either abiraterone or enzalutamide, according to results reported at the ESMO Congress 2019, and concurrently published in NEJM. In the phase IV CARD trial of 225 patients, those treated with cabazitaxel had a median PFS of 8 months and a median OS of 13.6 months, compared with 3.7 months and 11 months in patients who received additional abiraterone or enzalutamide.
  • At the ESMO Congress 2019, researchers reported that nivolumab leads to better outcomes in patients with inoperable advanced or recurrent esophageal cancer who have stopped responding to or can’t tolerate fluoropyrimidine plus platinum-based drugs. In the phase III ATTRACTION-3 trial, patients treated with the PD-1 inhibitor had a median OS of 10.9 months, compared with 8.4 months in patients who received chemotherapy, a benefit seen regardless of PD-L1 levels. Objective response rates were similar in both arms, but patients in the nivolumab arm had a median duration of response of 6.9 months, compared with 3.9 months in the chemotherapy arm. Results were concurrently published in The Lancet Oncology.
  • A study in Blood reports that the Bruton tyrosine kinase inhibitor ibrutinib (Imbruvica; Janssen) may be linked to high blood pressure and other cardiovascular problems. Researchers analyzed medical records from 562 patients with B-cell cancers treated with ibrutinib between 2009 and 2016 and found that 71.6% developed hypertension, and 17.7% were classified as high-grade cases. Additionally, patients who developed new or worse hypertension while taking the drug were more likely to experience arrhythmias, myocardial infarction, stroke, and heart failure.

September 20–26

  • The FDA approved the anti-CD38 therapy daratumumab (Darzalex; Janssen) plus bortezomib (Velcade; Takeda), thalidomide (Thalomid; Celgene), and dexamethasone in patients with newly diagnosed multiple myeloma who are eligible for autologous stem-cell transplant. The decision was based on the phase III CASSIOPEIA trial, in which patients treated with daratumumab plus the drug triplet had a stringent complete response rate of 29% and an 18-month progression-free survival rate of 93%, compared with 20% and 85%, respectively, in patients who received bortezomib, thalidomide, and dexamethasone alone.
  • The agency also expanded approval of the colorectal cancer screening test Cologuard (Exact Sciences) to include people of average risk age 45 and older. The test, which analyzes stool samples for blood and DNA alterations that can be indicative of colon cancer, was originally approved in 2014 for average-risk individuals age 49 and older.
  • "The data indicate that the FDA must do more," Acting FDA Commissioner Norman "Ned" Sharpless, MD, said in a testimony before Congress about electronic cigarettes (e-cigarettes). Sharpless outlined steps the agency is taking to investigate a lung illness associated with the devices that has been reported in more than 800 people and caused 12 deaths. He also discussed efforts to prevent youth from using e-cigarettes, including a proposal to remove flavored products from the market until they undergo official review. One company, Juul Labs, announced that it will suspend U.S. advertising of its devices and will not lobby against a ban on flavored products.
  • Amgen’s blinatumomab (Blincyto) showed strong efficacy in children with relapsed B-cell acute lymphoblastic leukemia (ALL), prompting the company to halt enrollment in two phase III trials. In the Study 20120215 trial, the bispecific antibody improved event-free survival compared with chemotherapy, and in the AALL 1331 trial, the therapy trended toward improved disease-free survival and overall survival compared with chemotherapy.
  • Eli Lilly’s pegilodecakin plus pembrolizumab (Keytruda; Merck) or nivolumab (Opdivo; Bristol-Myers Squibb) may be a promising treatment for patients with previously treated solid cancers, according to results in The Lancet Oncology. In a phase Ib trial, the drug elicited objective responses in 12 of 28 patients with non–small cell lung cancer, three of 31 patients with melanoma, and 14 of 35 patients with renal cell carcinoma. Pegilodecakin is an IL10 receptor agonist that induces oligoclonal T-cell expansion.
  • Drugmakers are recalling ranitidine-based heartburn medications after the FDA announced that it found unacceptable levels of the carcinogen N-Nitroso-dimethylamine in the drug. The recall applies to generic versions of ranitidine made by Novartis subsidiary Sandoz, as well those made by Apotex, Walgreens, Walmart, and Rite Aid.
  • The NIH Clinical Center started the Blood and Immune Deficiency–Cellular Therapy Program to treat patients with inherited blood and immune-system diseases. The program spans the NCI, the National Institute of Allergy and Infectious Diseases, the National Human Genome Institute, and the National Heart, Lung, and Blood Institute; patients will be treated by interdisciplinary teams of clinicians and studied by basic researchers.
  • Europe’s Committee for Medicinal Products for Human Use recommended the approval of gilteritinib (Xospata; Astellas Pharma) in relapsed/refractory FLT3-mutant acute myeloid leukemia. The committee also recommended the approval of three generic cancer drugs: Accord’s version of arsenic trioxide for acute promyelocytic leukemia, Fresenius Kabi’s version of bortezomib for multiple myeloma and mantle cell lymphoma, and Orphelia Pharma’s version of clofarabine for pediatric ALL.

September 13–19

  • For the first time, the FDA, the Australian Therapeutic Goods Administration, and Health Canada simultaneously reviewed a cancer drug, granting an accelerated approval to pembrolizumab (Keytruda; Merck) plus lenvatinib (Lenvima; Eisai) in certain patients with advanced endometrial carcinoma. The review was conducted through Project Orbis, an initiative of the FDA Oncology Center of Excellence. The approval was based on the phase III KEYNOTE-146 trial, in which 38 previously treated patients with tumors that were not microsatellite instability–high or mismatch repair–deficient had an objective response rate of 38.3%.
  • The FDA issued a warning that certain CDK4/6 inhibitors can cause rare but serious lung inflammation. The warning applies to palbociclib (Ibrance; Pfizer), ribociclib (Kisqali; Novartis), and abemaciclib (Verzenio; Eli Lilly), which are approved for HR-positive, HER2-negative breast cancer. The FDA notes that the drugs’ benefits are still greater than the risks when they are used as prescribed.
  • The agency also approved the antiandrogen apalutamide (Erleada; Janssen) for patients with metastatic, castration-sensitive prostate cancer. The approval was based on the phase III TITAN trial, in which patients treated with the drug plus androgen-deprivation therapy had a 2-year overall survival rate of 82.4% and a 2-year radiographic progression-free survival rate of 68.2%, compared with 73.5% and 47.5%, respectively, in patients who received a placebo plus androgen-deprivation therapy.
  • Radiotherapy may reduce the risk of cytokine release syndrome in patients with non-Hodgkin lymphoma treated with chimeric antigen receptor (CAR) T-cell therapies, according to findings presented at the American Society for Radiation of Oncology Annual Meeting in Chicago, IL. In the study, none of five patients who received radiotherapy in the month before CAR T-cell infusion and one of seven patients who received earlier radiotherapy experienced grade 3 or higher cytokine release syndrome, compared with five of 19 patients who did not receive radiotherapy.
  • The FDA placed a clinical hold on Amgen’s phase I trial testing the MCL1 inhibitor AMG 397 in multiple myeloma, non-Hodgkin lymphoma, and acute myeloid leukemia. During the hold, the company will evaluate the risk of cardiac toxicity. Amgen also halted a phase I trial of another MCL1 inhibitor, AMG 176.
  • Janssen announced that the anti-CD38 therapy daratumumab (Darzalex) plus bortezomib (Velcade; Takeda), lenalidomide (Revlimid; Celgene), and dexamethasone may improve responses in patients with multiple myeloma who are eligible for an autologous stem-cell transplant. In the phase II GRIFFIN trial, patients treated with the combination had a stringent complete response rate of 42% and a minimal residual disease rate of 59%, compared with 32% and 24%, respectively, in patients who received bortezomib, lenalidomide, and dexamethasone alone.
  • Carcinogenic chemicals in U.S. drinking water could cause 100,000 lifetime cases of cancer, according to a report by the Environmental Working Group. Researchers analyzed 22 contaminants in 48,000 water systems between 2010 to 2017 and found that the national risk of cancer is two orders of magnitude higher than what would be considered insignificant. Most of the risk can be attributed to arsenic, by-products of disinfectants, and radioactive chemicals.
  • The American Association for Cancer Research (AACR) released its 2019 Cancer Progress Report, available at http://cancerprogressreport.org. The report includes the latest information on cancer survival, diagnosis, prevention, and treatment. The AACR also led the seventh annual Rally for Medical Research Hill Day, joining more than 300 other organizations to advocate for robust, sustained, and predictable annual funding increases for the NIH.

September 6–12

  • AMG 510 is a promising therapy in patients with KRAS G12C–mutant non–small cell lung cancer (NSCLC), according to findings presented at the 2019 World Conference on Lung Cancer (WCLC) in Barcelona, Spain. In a phase I trial, seven of 13 evaluable patients with NSCLC had a partial response to the small-molecule inhibitor, and six more achieved stable disease. The results build on initial data presented at the 2019 American Society of Clinical Oncology Annual Meeting.
  • Also at the WCLC, Loxo Oncology announced that the RET inhibitor selpercatinib (LOXO-292) may be effective in patients with RET fusion–positive NSCLC. In the LIBRETTO-001 trial, the drug elicited an objective response rate (ORR) of 68% in 105 previously treated patients, and an ORR of 85% in 34 newly diagnosed patients. The drug was also generally well tolerated.
  • AstraZeneca’s durvalumab (Imfinzi) may improve survival of patients with newly diagnosed extensive-stage small cell lung cancer (SCLC), researchers reported at the WCLC. In the phase III CASPIAN trial, patients treated with the PD-L1 inhibitor plus chemotherapy had a median overall survival (OS) of 13 months, and 33.9% were still alive after 18 months, compared with 10.3 months and 24.7% in patients who received chemotherapy alone. Durvalumab is FDA-approved for NSCLC.
  • In an update also announced at the WCLC, patients with previously untreated metastatic NSCLC who had STK11 or KEAP1 mutations did not respond as well to therapy as patients who lacked the mutations. In the phase III MYSTIC trial, patients were treated with durvalumab, durvalumab plus tremelimumab, or chemotherapy. Across treatment arms, patients with STK11 mutations had a median OS of 6.8 months compared with 12.6 months in patients with a wild-type gene, and those with KEAP1 mutations had a median OS of 7.4 months, compared with 12.9 months in patients with a wild-type gene.
  • The European Commission approved the PD-L1 inhibitor atezolizumab (Tecentriq; Roche/Genentech) plus chemotherapy in patients with metastatic nonsquamous NSCLC who do not have EGFR or ALK mutations, and in patients with newly diagnosed, extensive-stage SCLC. The approvals were based on the phase III IMpower130 and IMpower133 trials, in which the combination significantly extended OS and progression-free survival compared with chemotherapy alone.
  • BioNTech filed to raise $100 million in an initial public offering. The move comes after the company raised $270 million in initial funding plus $325 million in private financing. BioNTech plans to use the funding to develop three off-the-shelf shared-antigen immunotherapies in clinical trials in advanced melanoma, head and neck cancer, and triple-negative breast cancer. The company’s lead candidate is the individualized neoantigen vaccine BNT122.
  • Tocagen announced that Toca 511 & Toca FC may not be effective in patients with recurrent high-grade glioma. In the phase III Toca 5 trial, the therapy did not extend OS compared with standard treatment. Toca 511 & Toca FC is a two-part therapy that uses a retroviral vector to deliver high concentrations of chemotherapy to the brain.
  • The NCI awarded $28.7 million to the Mayo Clinic Cancer Center in Rochester, MN, and renewed the center’s designation as a comprehensive cancer center following its review, which happens every 5 years. Mayo, which treats more than 120,000 patients annually, is one of 51 NCI-designated comprehensive cancer centers and is the only such center with multiple sites—it also has locations in Phoenix, AZ, and Jacksonville, FL.

August 30–September 5

  • The FDA finalized a guidance on using placebos and blinding in randomized controlled trials of cancer therapies. The guidance states that due to ethical concerns, trials should include placebos only in certain situations—for example, in testing maintenance or adjuvant therapies when surveillance is the standard of care, when the placebo is added to standard therapy, or when no standard therapy is available. According to the guidance, trial sponsors should provide rationale for trial design and consider unblinding patients if their disease recurs or progresses, or if they experience serious side effects.
  • The agency also tentatively approved Mylan’s generic version of Alimta (pemetrexed; Eli Lilly). A recent court case determined that the patent on pemetrexed—a chemotherapeutic approved for certain types of pleural mesothelioma and non–small cell lung cancer—will not expire until 2022, at which point Mylan can begin marketing its generic.
  • The U.S. Preventive Services Task Force released a final statement recommending that clinicians offer risk-reducing medications to women at increased risk of developing breast cancer who have a low risk of side effects. The recommendation is similar to the one published in 2013, but it adds the aromatase inhibitors anastrozole and exemestane to the list of acceptable risk-reducing medications, which already includes the selective estrogen receptor modulators tamoxifen and raloxifene.
  • Bristol-Myers Squibb announced that nivolumab (Opdivo) may not be an effective first-line therapy for glioblastoma multiforme that is MGMT methylated. In the phase III CheckMate-548 trial, the PD-1 inhibitor plus standard temozolomide and radiotherapy did not extend progression-free survival (PFS) compared with temozolomide and radiotherapy alone. However, an independent data-monitoring committee recommended that the trial continue to gather overall survival (OS) data.
  • Cancer is now the leading cause of death for middle-aged adults in high-income countries. Researchers analyzed data from 162,534 participants ages 35 to 70 in 21 countries enrolled in the Prospective Urban and Rural Epidemiologic study between 2005 and 2016 and followed them for a median of 9.5 years. Overall, 40% of participants died from cardiovascular disease and 26% died from cancer, but in high-income countries, 55% of participants died from cancer and only 23% died from cardiovascular disease.
  • The European Commission approved the PD-1 inhibitor pembrolizumab (Keytruda; Merck) plus the multityrosine kinase inhibitor axitinib (Inlyta; Pfizer) for newly diagnosed advanced renal cell carcinoma. The approval was based on the phase III KEYNOTE-426 trial, in which the combination significantly improved OS and PFS compared with sunitinib (Sutent; Pfizer). The FDA approved the combination for the same indication earlier this year.

August 2019

August 23–29

  • The FDA released a draft guidance on developing drugs to treat breast cancer in men. The guidance makes recommendations such as expanding the eligibility criteria of clinical trials for breast cancer therapies and using single-arm trials and real-world evidence to expand indications for the use of certain drugs to men. The draft guidance is open for public comment until October 28 at https://www.regulations.gov/.
  • The European Commission (EC) approved the monoclonal antibody elotuzumab (Empliciti; Bristol-Myers Squibb/AbbVie) plus pomalidomide and low-dose dexamethasone for use in patients with relapsed/refractory multiple myeloma who have received at least two prior therapies, including lenalidomide and a proteasome inhibitor. The approval was based on the phase III ELOQUENT-3 trial, in which the triplet extended progression-free survival (PFS) and improved the overall response rate compared with pomalidomide plus low-dose dexamethasone.
  • The EC also approved the PD-L1 inhibitor atezolizumab (Tecentriq; Roche) in combination with nab-paclitaxel (Abraxane; Celgene) for PD-L1–positive, metastatic triple-negative breast cancer. In the phase III IMpassion130 trial, which formed the basis of the approval, patients treated with the combination had a longer PFS and overall survival (OS) than patients receiving nab-paclitaxel alone.
  • AbbVie will discontinue research and development of rovalpituzumab tesirine (Rova-T), an antibody–drug conjugate being tested in small cell lung cancer. The decision comes after an independent data monitoring committee recommended termination of the phase III MERU trial due to a lack of efficacy. Last year, AbbVie halted enrollment of the phase III TAHOE trial after patients treated with Rova-T had a shorter OS than patients receiving standard topotecan chemotherapy.
  • Celgene will pay Immatics $75 million up front to jointly develop three adoptive cell therapies for cancer and up to $505 million more in milestone payments for each therapy. As part of the deal, Immatics will handle the initial development and validation of the therapies, at which point Celgene will have the option to take over development, manufacturing, and commercialization.
  • A patient in Illinois is the first to die from a lung illness associated with vaping that causes breathing difficulties, chest pain, vomiting, and fatigue. The condition has been reported in at least 215 people in the United States; in the most severe cases, extensive lung damage requires treatment with oxygen on a ventilator. It is unclear whether patients were smoking marijuana-based products or electronic cigarettes, or if contamination or a defective device was involved.
  • Five pharmaceutical companies filed a complaint in Quebec’s Superior Court challenging new Canadian regulations created to lower prices of patented drugs. The regulations, which could save Canadians $10 billion over 10 years, expand the powers of the Patented Medicine Prices Review Board, allowing the agency to consider additional information when setting price caps, such as the cost-effectiveness of drugs. The complaint was filed by Merck, Janssen, Bayer, Boehringer Ingelheim, and Servier.

August 16–22

  • The FDA approved the JAK inhibitor fedratinib (Inrebic; Celgene) in intermediate- and high-risk myelofibrosis. The approval was based on the phase III JAKARTA trial, in which 37% of patients treated with the drug had at least a 35% reduction in spleen volume, and 40% experienced at least a 50% reduction in disease-related symptoms, compared with 1% and 9%, respectively, in patients receiving a placebo. Fedratinib is the second JAK inhibitor and the second therapy for myelofibrosis to be approved.
  • The U.S. Preventive Services Task Force released a final statement recommending risk-assessment screening in women who have a personal or family history of breast, ovarian, tubal, or peritoneal cancer or ancestry associated with BRCA1/2 mutations, to determine their likelihood of carrying harmful BRCA1/2 mutations. The recommendation is similar to one published in 2013, but it expands the group that should undergo risk-assessment screening to include women who have completed treatment for breast, ovarian, tubal, or peritoneal cancer and are cancer free, as well as women of certain ancestries.
  • The U.S. Court of Appeals for the Federal Circuit declined to stop the sale of bevacizumab-awwb (Mvasi; Amgen/Allergan), a biosimilar of the angiogenesis inhibitor bevacizumab (Avastin; Genentech). The decision came in response to Genentech’s injunction claiming that Amgen should be required to wait longer to market the biosimilar after adding supplemental information to its original FDA application. Bevacizumab-awwb costs about 12% less than bevacizumab.
  • AstraZeneca announced that durvalumab (Imfinzi) plus tremelimumab may not be effective in treating non–small cell lung cancer. In the phase III NEPTUNE trial of newly diagnosed patients with a tumor mutation burden of at least 20 mutations per megabase, the PD-L1 inhibitor/CTLA4 inhibitor combination did not improve overall survival in patients with metastatic disease compared with standard chemotherapy.
  • NuCana will halt enrollment of a trial testing NIC-1031 (Acelarin) in metastatic pancreatic cancer based on a recommendation from an independent data-monitoring committee. The decision was made after the committee determined that the chemotherapeutic was unlikely to improve survival compared with gemcitabine in the phase III ACELERATE trial.
  • China’s National Healthcare Security Administration added 148 drugs to the list of medicines covered by its basic medical insurance plan. It also removed 150 drugs determined to have low clinical value or that could be replaced by more effective alternatives, bringing the total number on the list to 2,643 therapies. Additionally, 128 drugs that are efficacious but expensive have been identified as potential additions to the list, depending on the outcome of negotiations with the companies that make them.
  • In the United States, about 34,800 cancers per year could be attributed to human papillomavirus between 2012 to 2016, according to Morbidity and Mortality Weekly Report. In total, 92% of these HPV-related cancers were types targeted by the HPV vaccine. Another report indicated that in 2018, 51.1% of adolescents ages 13 to 17 had completed the HPV vaccine series, compared with 48.6% in 2017—an increase due entirely to more boys being vaccinated.

August 9–15

  • The FDA approved the selective TRK inhibitor entrectinib (Rozlytrek; Genentech) in adults with ROS1-positive, metastatic non–small cell lung cancer. The agency also granted the drug an accelerated approval in adults and children ages 12 and older with NTRK-positive solid tumors whose cancer has progressed on other therapies, or who lack first-line treatment options. The approvals were based on four trials in which the therapy elicited high overall response rates (ORR) and durable responses.
  • The FDA issued a proposed rule that would require graphic health warnings on cigarette packaging and advertisements. The 13 warnings, which fulfill a requirement of the Family Smoking Prevention and Tobacco Control Act, would include photorealistic color images of lesser-known health risks of smoking, such as bladder cancer, diabetes, and erectile dysfunction. The rule is open for public comment until October 15 at https://www.federalregister.gov/.
  • Preliminary results in the Journal of Clinical Oncology suggest that AstraZeneca’s experimental WEE1 inhibitor adavosertib (AZD1775) may be a promising first-line treatment for pancreatic cancer. In a phase I trial, patients treated with the drug in combination with gemcitabine and radiation had a median overall survival (OS) of 21.7 months and a median progression-free survival (PFS) of 9.4 months. Researchers are planning a phase II trial in pancreatic cancer and testing adavosertib in other solid cancers, including breast and ovarian cancers.
  • RABL3 mutations increase the risk of hereditary pancreatic ductal adenocarcinoma, according to findings in Nature Genetics. Researchers identified the mutation by performing genomic sequencing on multiple members of the same family who developed pancreatic cancer. They then recreated the mutation in zebrafish to further investigate its mechanism and biological function.
  • Deciphera announced that the KIT and PDGFRα inhibitor ripretinib (DCC-2618) may be an effective fourth- or fifth-line treatment for advanced gastrointestinal stromal tumors. In the phase III INVICTUS trial, patients treated with ripretinib had a median PFS of 6.3 months, a median OS of 15.1 months, and an ORR of 9.4%, compared with 1 month, 6.6 months, and 0%, respectively, in patients who received a placebo.
  • The U.S. Court of Appeals for the Federal Circuit upheld Sanofi’s patents on cabazitaxel (Jevtana), a microtubule inhibitor approved for metastatic, hormone-refractory prostate cancer. The decision overturns a previous ruling by the U.S. District Court of New Jersey that deemed one of Sanofi’s patents invalid. The lawsuit began after drug companies applied to the FDA to make generic versions of the drug before its patents expire in 2031, prompting Sanofi to sue.
  • Healthcare company Synapse and the FDA Oncology Center for Excellence announced a multiyear collaboration on real-world evidence (RWE) to support regulatory decisions. The organizations will explore methods of gathering RWE from electronic health records and other sources, and will investigate the utility of real-world endpoints in oncology trials.
  • Black and Hispanic patients are underrepresented in clinical trials that lead to oncology drug approvals, according to a study in JAMA Oncology. Researchers analyzed 230 trials conducted between 2008 and 2018 and found that 3.1% of participants were black and 6.1% were Hispanic—22% and 44%, respectively, of the expected proportions. Overall, 63% of trials reported on at least one race, and just 7.8% documented the four major U.S. races (white, Asian, black, and Hispanic).

August 2–8

  • Medicare will begin covering chimeric antigen receptor T-cell therapies per a final rule issued by the Centers for Medicare & Medicaid Services (CMS). The coverage, which extends to FDA-approved therapies as well as CMS-approved off-label uses, includes tisagenlecleucel (Kymriah; Novartis) and axicabtagene ciloleucel (Yescarta; Gilead) for certain leukemias and lymphomas. The therapies can be given at health centers enrolled in an FDA-mandated safety program; pharmaceutical companies will be responsible for gathering post-market data.
  • The U.S. Preventive Services Task Force recommended against imaging-based screening for pancreatic cancer in adults without symptoms, as well as treatment of pancreatic cancer detected via such screening. The agency concluded that screening does not reduce mortality and is associated with only small benefits, but moderate harms. The recommendation has not changed since 2004, but it’s now supported by additional scientific evidence.
  • AstraZeneca and Merck announced that the PARP inhibitor olaparib (Lynparza) may be an effective treatment in patients with metastatic castration-resistant prostate cancer who have homologous recombination repair mutations and whose cancer has worsened on hormone therapy. In the phase III PROfound trial, patients who received olaparib had significantly longer radiographic progression-free survival than those who received standard enzalutamide (Xtandi; Astellas/Pfizer) or abiraterone (Zytiga; Janssen).
  • Transgene and SillaJen will halt a trial of first-line pexastimogene devacirepvec (Pexa-Vec/JX-594) in liver cancer following a review from an independent data-monitoring committee. The committee’s recommendation was based on the phase III PHOCUS trial, in which the therapy followed by sorafenib (Nexavar; Bayer/Amgen) seemed unlikely to extend overall survival compared with sorafenib alone. Pexastimogene devacirepvec is an oncolytic vaccinia virus modified to express GM-CSF.
  • The NCI awarded $13.8 million to the Indiana University Melvin and Bren Simon Cancer Center in Indianapolis and promoted it to a comprehensive cancer center, giving it an “outstanding” rating following its 5-year review. The center, now one of 51 NCI-designated comprehensive cancer centers, is known for establishing a more effective treatment for testicular cancer and for research on therapies for breast, gastrointestinal, and hematologic malignancies, among others.
  • Women lead just 17.9% of later-phase oncology clinical trials, according to a study in JAMA Oncology. Researchers analyzed 598 phase III oncology trials published between 2003 and 2018 and found that women were the corresponding authors on 14.4% of industry-funded trials and 30.1% of other trials. They were most likely to lead trials in head and neck cancers, and least likely to lead trials in genitourinary cancer (39.1% and 7.2%, respectively).

July 2019

July 26–August 1

  • The U.S. Senate passed a 2-year budget deal that would increase overall discretionary spending from $1.32 trillion in fiscal year (FY) 2019 to $1.375 trillion in FY 2021, and includes a $320 billion increase in military and domestic caps. The deal, which also suspends the debt ceiling through July 31, 2021, had already been passed in the U.S. House of Representatives, and President Donald Trump is expected to sign it.
  • Exact Sciences announced it will acquire Genomic Health for $2.8 billion. Exact Sciences makes the colon cancer test Cologuard. In the deal, it will gain Genomic Health’s Oncotype DX genomic tests, which can inform treatment of breast, prostate, and colon cancers.
  • The FDA approved the androgen-receptor inhibitor darolutamide (Nubeqa; Bayer) in nonmetastatic castration-resistant prostate cancer. The approval was based on the phase III ARAMIS trial, in which patients treated with the drug plus androgen-deprivation therapy had a median metastasis-free survival of 40.4 months, compared with 18.4 months in patients receiving androgen-deprivation therapy alone.
  • The Sylvester Comprehensive Cancer Center in Miami, FL, received its first NCI designation. The center, which is part of the University of Miami Leonard M. Miller School of Medicine, is now the 71st NCI-designated cancer center. It is known for its work to address higher cancer rates in underserved communities, and its long-term research on cancer risk in firefighters.
  • Eli Lilly removed a PD-L1/TIM3 bispecific antibody from its clinical pipeline. The drug, LY3415244, was being tested in a phase I trial of advanced solid tumors. The company will continue studying LY3321367, an anti-TIM3 antibody that is in phase I testing as a monotherapy and in combination with a PD-L1 inhibitor.
  • Merck announced that the FDA approved pembrolizumab (Keytruda) as a monotherapy for patients with recurrent locally advanced or metastatic esophageal squamous cell carcinoma whose tumors express PD-L1 and who have received at least one prior therapy. In the phase III KEYNOTE-181 trial, which formed the basis of the approval, patients treated with the PD-1 inhibitor had a median overall survival of 10.3 months and a media progression-free survival of 3.2 months, compared with 6.7 months and 2.3 months, respectively, in patients who received chemotherapy.
  • Globally, 11.5 million years of healthy life were lost due to childhood cancer in 2017, according to a study in The Lancet Oncology based on the Global Burden of Diseases, Injuries, and Risk Factors Study of 2017. Researchers also found that childhood cancer was the sixth leading cause of cancer burden and the ninth leading cause of childhood diseases, disorders, illnesses, and infections.

July 19–25

  • The first two cancer biosimilars are now being sold in the United States: bevacizumab-awwb (Mvasi; Amgen/Allergan), a biosimilar of the angiogenesis inhibitor bevacizumab (Avastin; Genentech), and trastuzumab-anns (Kanjinti; Amgen/Allergan), a biosimilar of the HER2-targeted agent trastuzumab (Herceptin; Genentech). The agents currently have an average price that’s 12% to 13% less than their reference products.
  • Pfizer announced that the FDA approved rituximab-pvvr (Ruxience), a biosimilar of the CD20-specific antibody rituximab (Rituxan; Genentech), for non-Hodgkin lymphoma (NHL) and chronic lymphocytic leukemia. The approval was based on results of the REFLECTIONS B3281006 trial, indicating that the efficacy and safety of the agent mimic that of rituximab. Rituximab-pvvr is the second biosimilar approved for rituximab; rituximab-abbs (CT-P10; Truxima) was approved in November.
  • Bristol-Myers Squibb reported that the PD-1 inhibitor nivolumab (Opdivo) plus chemotherapy may not be an effective first-line therapy for nonsquamous non–small cell lung cancer (NSCLC). In part 2 of the phase III CheckMate-227 trial, the combination did not extend overall survival (OS) compared with chemotherapy. However, nivolumab plus the CTLA4 inhibitor ipilimumab (Yervoy) may be effective as a first-line treatment for patients with PD-L1–positive NSCLC: In part 1a of the trial, patients treated with the combination had longer OS than those who received chemotherapy.
  • Janssen’s erdafitinib (Balversa) is a promising treatment for patients with locally advanced or metastatic urothelial carcinoma who have an FGFR3 or FGFR2 mutation and have not responded to platinum-containing chemotherapy, according to findings in The New England Journal of Medicine. In the phase II BLC2001 trial, 99 patients had an objective response rate of 40%, a median OS of 13.8 months, and a median progression-free survival of 5.5 months. A pan-FGFR inhibitor, erdafitinib received accelerated approval from the FDA in April.
  • A research letter in JAMA Oncology suggested that germline BRCA2 mutations may be associated with an increased risk of NHL in children and adolescents. Researchers analyzed whole-genome sequencing data from 1,380 survivors of childhood lymphoma and 59,345 healthy controls and found that the subgroup of 565 survivors of NHL were significantly more likely to have BRCA2 mutations than healthy controls.
  • Allergan will recall textured breast implants linked to a rare cancer, per a request from the FDA. The agency reported that the Allergan implants have been linked to 481 cases of anaplastic large cell lymphoma (ALCL) and 11 deaths worldwide. An FDA analysis concluded that the risk of ALCL is six times higher with Allergan’s textured implants than with textured implants from other U.S. manufacturers.

July 12–18

  • The Biden Cancer Initiative will suspend operations indefinitely in response to former Vice President Joe Biden’s presidential run. Biden started the nonprofit 2 years ago after the creation of the federally funded Beau Biden Cancer Moonshot; the organization’s goal was to facilitate collaboration among researchers, companies, and patient groups. The decision comes amidst concerns about conflicts of interest that the nonprofit creates for Biden regarding drug pricing and pharmaceutical and health care companies.
  • AbbVie announced that it acquired the biopharmaceutical company Mavupharma for an undisclosed amount. The deal will give AbbVie access to Mavupharma’s pipeline of cancer therapies that target the STING immune pathway. Its lead candidate is MAVU-104, a small-molecule inhibitor of ENPP1, an enzyme involved in STING pathway regulation.
  • GlaxoSmithKline announced that the PARP inhibitor niraparib (Zejula) may be an effective first-line maintenance therapy in patients with ovarian cancer who have responded to platinum-based chemotherapy. In the phase III PRIMA trial, women treated with the drug had longer progression-free survival than women who received a placebo regardless of biomarker status; full data will be presented at an upcoming medical meeting. Niraparib is already approved for women with ovarian, fallopian tube, or primary peritoneal cancer following at least two lines of platinum-based chemotherapy.
  • An artificial intelligence tool may be able to predict which pancreatic cysts will become cancerous, according to findings in Science Translational Medicine. The test incorporates measurements of molecular and clinical markers in cyst fluid along with radiologic and clinical information. Researchers “trained” the tool with data from 426 patients and then used it to assess 426 more patients. It accurately identified 60.4% of those who should be discharged, 48.6% of those who should be monitored, and 90.8% of those requiring surgery, compared with 18.9%, 34.3%, and 88.8%, respectively, identified with standard pathology assessments.
  • The UK’s National Institute for Health and Care Excellence (NICE) recommended the CDK4/6 inhibitor ribociclib (Kisqali; Novartis) in combination with fulvestrant for patients with advanced HR-positive, HER2-negative breast cancer following endocrine therapy. NICE previously recommended against the drug due to cost, but since then Novartis has submitted an improved plan for patient access to the therapy. National Health Service England will now make the therapy available to patients through the Cancer Drugs Fund.

July 4–11

  • The FDA granted accelerated approval to selinexor (Xpovio; Karyopharm) in combination with dexamethasone for patients with relapsed/refractory multiple myeloma who have received at least four prior therapies—and whose disease has stopped responding to at least two proteasome inhibitors, two immunomodulatory agents, and a CD38-targeted monoclonal antibody. The approval is based on an overall response rate of 25.3% in the phase IIb STORM trial and is contingent on results from a phase III trial. A selective inhibitor of nuclear export, selinexor inhibits exportin-1.
  • Unum Therapeutics announced that the FDA placed a clinical hold on a phase I trial testing the autologous T-cell therapy ACTR087 in combination with rituximab (Rituxan; Genentech) in patients with relapsed/refractory CD20+ B-cell non-Hodgkin lymphoma who have received chemotherapy. The decision was made after a patient in the trial experienced severe neurotoxicity, cytomegalovirus infection, and respiratory distress. Accumulating safety concerns have prompted Unum to move away from developing ACTR087 in favor of advancing another autologous T-cell therapy, ACTR707.
  • A federal judge ruled that pharmaceutical companies do not need to disclose drug prices in television ads. The decision blocks a rule issued by the Department of Health and Human Services set to go into effect this month that would have required companies to include list prices for any drug costing $35 or more for a 30-day supply or for a typical course of treatment. The rule spurred Amgen, Merck, and Eli Lilly to file a suit against the department.
  • The World Health Organization (WHO) added 12 cancer drugs to its list of essential medicines. For example, the list now includes the PD-1 inhibitors pembrolizumab (Keytruda; Merck) and nivolumab (Opdivo; Bristol-Myers Squibb) for advanced melanoma. In addition, the indications were expanded for some drugs that were already on the list. According to the WHO, more than 150 countries consult the slate of drugs, which now includes 460 medicines for a variety of conditions, “to guide decisions about which medicines represent the best value for money, based on evidence and health impact.”
  • In 2015, deaths from cancer in the United States resulted in $94.4 billion in lost earnings, according to a report in JAMA Oncology. Among the 492,146 people between the ages of 16 and 84 who died from cancer that year, lung cancer caused the greatest loss in earnings ($21.3 billion), followed by colorectal cancer ($9.4 billion), breast cancer ($6.2 billion), and pancreatic cancer ($6.1 billion); earnings lost per 100,000 people, when standardized for age, was highest in Kentucky ($35.3 million) and lowest in Utah ($19.6 million).
  • Following a recommendation from an independent monitoring committee, Boston Biomedical announced that it will discontinue the phase III CanSTEM111P trial testing napabucasin (BBI608) in combination with nab-paclitaxel (Abraxane; Celgene) and gemcitabine in patients with metastatic pancreatic ductal adenocarcinoma, due to a lack of efficacy. Napabucasin is a cancer stemness inhibitor that targets STAT3. A phase III trial of the agent in metastatic colorectal cancer, as well as earlier-stage trials for other solid tumors, will continue.

June 28–July 3

  • The FDA approved bevacizumab-bvzr (Zirabev; Pfizer), a biosimilar of the angiogenesis inhibitor bevacizumab (Avastin; Genentech), for five cancers: metastatic colorectal cancer; unresectable, advanced, metastatic, or recurrent non–small cell lung cancer; recurrent glioblastoma; advanced or metastatic renal cell carcinoma; and persistent, recurrent, or metastatic cervical cancer. Bevacizumab-bvzr is the second biosimilar approved for bevacizumab; bevacizumab-awwb (Mvasi; Amgen) was approved in 2017.
  • According to an investigative report in Science, the FDA’s compliance and enforcement actions have decreased dramatically since President Donald Trump took office. The agency issued 1,033 warning letters between Trump’s inauguration and May 22, 2019, compared with 1,532 such letters during an equivalent period in the Obama Administration. Other actions, including FDA injunctions and warning letters from the FDA Center for Devices and Radiological Health, have also dropped, the report states.
  • Europe’s Committee for Medicinal Products for Human Use (CHMP) recommended the approval of the PD-L1 inhibitor atezolizumab (Tecentriq; Roche) plus nab-paclitaxel (Abraxane; Celgene) as a first-line treatment for patients with PD-L1–positive, inoperable advanced or metastatic triple-negative breast cancer. The recommendation was based on the phase III IMpassion130 trial, in which the combination extended progression-free survival and overall survival compared with nab-paclitaxel alone. Last year, the FDA granted accelerated approval to the drug for the same indication.
  • The UK’s National Institute for Health and Care Excellence (NICE) recommended the PD-1 inhibitor cemiplimab (Libtayo; Regeneron/Sanofi) for patients with advanced cutaneous squamous cell carcinoma who can’t have surgery or radiation therapy. The decision follows a recommendation from CHMP and a conditional marketing authorization from the European Commission based on high response rates in the phase II EMPOWER-CSCC-1 trial. Sanofi has agreed to a confidential price reduction for the PD-1 inhibitor, which typically costs around $100,000 per year; National Health Service England will make the therapy available to patients via the Cancer Drugs Fund.
  • Ten states plus Washington, DC, are suing the Environmental Protection Agency (EPA) over asbestos regulations. The lawsuit comes after the EPA denied a petition from the states requesting that it collect more data from chemical companies on asbestos use and health. Asbestos is linked to mesothelioma, lung cancer, and other malignancies.

June 2019

June 21–27

  • The FDA declined to approve the FLT3 inhibitor quizartinib (Daiichi Sankyo) for relapsed/refractory FLT3-mutant acute myeloid leukemia. The decision follows the recommendation of an advisory panel, which in May voted 8–3 against approval due to limited efficacy in the phase III QuANTUM-R trial; the agency does not need to adhere to the panel’s recommendation but usually does so. Although the drug was recently approved in Japan, it has not been approved elsewhere.
  • San Francisco, CA, passed a bill to ban the sale of electronic cigarettes (e-cigarettes), becoming the first city in the United States to do so. Under the law, which will go into effect 30 days after the mayor signs it, stores will have 6 months to sell their remaining stock of e-cigarettes. The law does not apply to combustible cigarettes or cannabis.
  • The FDA approved daratumumab (Darzalex; Janssen) in combination with lenalidomide (Revlimid; Celgene) and dexamethasone for patients with newly diagnosed multiple myeloma who aren’t eligible for an autologous stem cell transplant. The decision was based on the phase III MAIA trial, in which patients treated with the triplet had an overall response rate of 93% and did not reach median progression-free survival (PFS), compared with 81% and 31.9 months in patients who received lenalidomide plus dexamethasone. Daratumumab, a CD38-targeting agent, is already approved for multiple myeloma in other combinations.
  • The FDA lifted a partial hold on the phase III CANOVA trial testing venetoclax (Venclexta; AbbVie, Roche) plus dexamethasone in certain patients with relapsed/refractory multiple myeloma. The hold was initially placed on all venetoclax trials based on the phase III BELLINI trial, in which 21.1% of patients treated with venetoclax plus bortezomib (Velcade; Takeda) and dexamethasone died, compared with 11.3% of patients receiving a placebo plus bortezomib and dexamethasone. It was lifted on the CANOVA trial after the companies revised the study protocol to add, among other things, new measures to reduce risk.
  • Cobimetinib (Cotellic; Exelixis) plus atezolizumab (Tecentriq; Genentech) may not be an effective treatment for BRAF V600 wild-type melanoma. In the phase III IMspire170 trial, first-line treatment with the MEK/PD-L1 inhibitor combination did not extend median PFS compared with the PD-1 inhibitor pembrolizumab (Keytruda; Merck) alone. Cobimetinib is approved in combination with the BRAF inhibitor vemurafenib (Zelboraf; Genentech) for metastatic BRAF V600E/K-mutant melanoma.
  • The human papillomavirus (HPV) vaccine has significantly decreased the rate of HPV infections and the incidence of precancerous cervical lesions. Researchers analyzed 65 studies conducted in 14 high-income countries and found that after 5 to 8 years of vaccination, the rates of HPV 16 and 18 decreased by 83%, and the rates of HPV 31, 33, and 35 decreased by 54%, among girls ages 13 to 19. Additionally, after 5 to 9 years of vaccination, the prevalence of high-grade cervical intraepithelial neoplasia grade 2 or worse decreased by 51% among girls ages 15 to 19, and 31% among women ages 20 to 24.
  • The European Commission approved the PARP inhibitor talazoparib (Talzenna; Pfizer) for patients with advanced or metastatic HER2-negative breast cancer and germline BRCA1/2 mutations. The decision was based on the phase III EMBRACA trial, in which the drug extended PFS compared with standard chemotherapy. Talazoparib received FDA approval for the same indication last year.

June 14–20

  • Merck announced that the FDA expanded the indication for pembrolizumab (Keytruda) to include patients with metastatic small cell lung cancer whose disease progressed on or after platinum-based chemotherapy and at least one other therapy. The approval was based on pooled data from two trials in which patients had an objective response rate of 19% and a partial response (PR) rate of 17%. Among 16 responding patients, 94% responded for at least 6 months; 63% responded for at least a year.
  • At the meeting of the European Hematology Association (EHA), Chinese biotech BeiGene touted encouraging response rates to two of its drugs: the BTK inhibitor zanubrutinib and the PD-1 inhibitor tislelizumab.
    • In a nonrandomized cohort of 26 patients with the MYD88WT genotype of Waldenström macroglobulinemia, five of whom were treatment-naïve, the overall response rate (ORR) to zanubrutinib was 80.8% after a median follow-up of 12.2 months, with 53.8% of patients experiencing a PR or better. Neither progression-free survival (PFS) or overall survival had been reached.
    • Tislelizumab demonstrated an ORR of 87% and a complete response (CR) of 63% among 70 patients with relapsed/refractory classic Hodgkin lymphoma who had an average of three previous therapies. PFS in the phase II trial was estimated at 73.8%, and median PFS had not been reached after a median of 13.9 months of follow-up. The results were slightly better than those presented at the American Society of Hematology Annual Meeting last December.
  • Also at the EHA meeting, Regeneron announced positive results from early-stage trials of REGN1979, a bispecific monoclonal antibody designed to kill cells by binding to both CD20 and CD3. Four of seven patients with relapsed/refractory diffuse large B-cell lymphoma treated with the drug experienced a CR, including two patients whose disease had progressed after receiving chimeric antigen receptor therapy. In addition, 13 of 14 patients with follicular lymphoma grades 1 to 3a responded to the drug, 10 of whom experienced a CR.
  • Sanofi announced that it is eliminating 466 jobs in France and Germany and dropping new, in-house research on cardiology drugs. The pharmaceutical giant said that it will commit more resources to cancer, immunology, rare diseases, and vaccines; biologics and gene therapy research will also get a boost. The company plans to keep the job cuts voluntary.
  • Fighting back against a rule that would mandate the inclusion of drug prices in television ads, Amgen, Merck, and Eli Lilly filed suit against the Department of Health and Human Services. Along with the Association of National Advertisers, the companies argue that consumers generally don’t pay full price for medication due to insurance coverage, so the figures would be misleading—and that the rule impinges on their right to free speech. Set to take effect in July, the rule requires noting list prices for any drug costing $35 or more for a 30-day supply or for a typical course of treatment.

June 7–13

  • GlaxoSmithKline (GSK) and the University of California inked a 5-year collaboration to establish the Laboratory for Genomics Research (LGR), which will explore how gene mutations cause disease and develop technologies using CRISPR to accelerate the discovery of new medicines. The LGR, which will receive funding of $67 million from GSK, will focus on immunology, oncology, and neuroscience.
  • With the patent on trastuzumab (Herceptin; Genentech/Roche) in the United States about to expire, Amgen and Allergan announced that the FDA has approved their biosimilar product, trastuzumab-anns (Kanjinti). The go-ahead from the agency covers all the same indications for which trastuzumab is currently used: as a single agent for the treatment of patients with HER2-overexpressing breast cancer who have received at least one chemotherapy regimen for metastatic disease; in combination with paclitaxel for the first-line treatment of these cancers; or in combination with chemotherapy for the treatment of patients with HER2-overexpressing metastatic gastric or gastroesophageal junction adenocarcinoma who have not received prior treatment for metastatic disease. Trastuzumab-anns is the fifth trastuzumab biosimilar greenlighted by the FDA.
  • The FDA finalized its guidance document for manufacturers submitting new tobacco product applications for electronic nicotine delivery systems (ENDS), such as electronic cigarettes and the nicotine-containing liquids used with the devices. The document says that, in order to market ENDS products, manufacturers and importers must demonstrate, among other things, that the marketing of the new tobacco product would be appropriate for the protection of the public health—for example, that it is more likely that the devices would assist current tobacco users in quitting than that nonusers, especially children, would become users.
  • According to an article published in The New England Journal of Medicine, chemotherapy plus radiation is no better than chemotherapy alone for locally advanced endometrial cancer. Previous studies have, the researchers wrote, “supported the feasibility and efficacy of a combined treatment strategy.” However, in the phase III trial, the estimated percentage of relapse-free patients after 5 years in the chemoradiotherapy group was 59%, compared with 58% in the chemotherapy-only group.
  • Merck’s pembrolizumab (Keytruda) earned FDA approval for yet another indication: first-line treatment of patients with metastatic or inoperable recurrent head and neck squamous cell carcinoma (HNSCC) in combination with platinum and fluorouracil, and as a single agent for patients whose tumors express PD-L1. The agency also expanded the uses of the PD-L1 IHC 22C3 pharmDx kit (Agilent) to include use as a companion diagnostic device to select patients with HNSCC for single-agent treatment with pembrolizumab.
  • NIH Director Francis S. Collins, MD, PhD, announced that he will no longer participate in all-male speaking panels, noting that women and members of other groups underrepresented in science are often not given marquee speaking slots at scientific conferences and meetings. “Starting now, when I consider speaking invitations, I will expect a level playing field, where scientists of all backgrounds are evaluated fairly for speaking opportunities,” he said. “If that attention to inclusiveness is not evident in the agenda, I will decline to take part.”

May 31–June 6
This week: Special content from the 2019 ASCO Annual Meeting and other news

  • The FDA issued a draft guidance on increasing diversity in clinical trials. The document recommends strategies for broadening eligibility criteria and increasing enrollment of underrepresented populations, such as eliminating exclusion criteria that aren’t relevant, offering reimbursement for clinical trial expenses, and running trials in geographic locations with more racial and ethnic minority patients. The draft guidance is open for public comment until August 8 at www.regulations.gov.
  • AstraZeneca announced topline data indicating that the Bruton tyrosine kinase inhibitor acalabrutinib (Calquence) may improve outcomes of patients with newly diagnosed chronic lymphocytic leukemia (CLL). In the phase III ELEVATE-TN trial, acalabrutinib plus obinutuzumab, as well as acalabrutinib alone, significantly extended median progression-free survival (PFS) compared with chlorambucil plus obinutuzumab (Gazyva; Genentech), leading the company to stop the trial early. In May, AstraZeneca halted the phase III ASCEND trial early because acalabrutinib monotherapy extended PFS in previously treated patients with CLL compared with idelalisib (Zydelig; Gilead) or bendamustine.
  • The NCI awarded an $11 million grant to fund cervical cancer prevention efforts. The initiative will focus on Appalachian families in Ohio, Kentucky, Virginia, and West Virginia, where cervical cancer incidence and death rates are high. Eleven health systems in these states will collaborate to target the main causes of the disease, including human papillomavirus (HPV) infection, low rates of HPV vaccination and cancer screening, and cigarette smoking.
  • The KRASG12C inhibitor AMG 510 may be effective in patients with certain solid cancers who have a KRASG12C mutation, according to preliminary results presented by Marwan Fakih, MD, of City of Hope Comprehensive Cancer Center in Duarte, CA, on June 3 at the American Society of Clinical Oncology (ASCO) Annual Meeting. In a phase I trial, five out of 10 patients with non–small cell lung cancer responded to the drug, and four additional patients had stable disease; 13 out of 18 patients with colorectal cancer had stable disease.
  • At the ASCO meeting on June 3, Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence, announced a new pilot program to assist oncologists seeking access to unapproved therapies for their patients. Under the program, dubbed Project Facilitate, a central FDA center will help physicians through the process to submit an Expanded Access request, follow up on requests, and gather data, such as how many patients received investigational products and their outcomes. Project Facilitate can be reached at 240-402-0004 and OncProjectFacilitate@fda.hhs.gov.
  • During the meeting’s June 2 plenary session, researchers shared negative results for the phase III ANNOUNCE trial, which randomly assigned 509 patients with advanced soft-tissue sarcomas to receive either olaratumab plus doxorubicin or a placebo plus doxorubicin. Although the olaratumab–doxorubicin combination showed a survival benefit in a phase II trial, median overall survival (OS) was similar in both arms of the new trial: 20.4 months versus 19.9 months, respectively.
  • After a median follow-up of 12.7 months, responses from 38 patients with melanoma continued to improve on bempegaldesleukin (NKTR-214; Nektar Therapeutics) and nivolumab (Opdivo; Bristol-Myers Squibb). In the phase II PIVOT-02 trial, the objective response rate was 53%, the same as reported last year, but at ASCO, researchers reported that the complete response rate was 34% (13 of 38 patients), up from 24% in 2018. Median duration of response and median progression-free survival have not yet been reached. Bempegaldesleukin is a CD122-preferential IL2 pathway agonist.
  • On June 1, researchers at the ASCO meeting announced findings from the phase III MONALEESA-7 trial: They found that adding the CDK4/6 inhibitor ribociclib (Kisqali; Novartis) to standard-of-care endocrine therapy significantly improved OS for premenopausal women with advanced HR-positive, HER2-negative breast cancer compared with endocrine therapy alone. After 42 months of follow-up, the survival rate was 70% for women who received the combination compared with 46% for women who received endocrine therapy alone.
  • Also on June 1, Josep Tabernero, MD, PhD, of Vall d’Hebron University Hospital and Institute of Oncology in Barcelona, Spain, reported that Merck’s pembrolizumab (Keytruda) offers comparable OS to chemotherapy for advanced gastric and gastroesophageal junction cancers. The phase III noninferiority randomized clinical trial demonstrated that such patients with HER2-negative tumors that expressed high levels of PD-L1 responded well to the PD-1 inhibitor: At 2 years, 39% of patients who received pembrolizumab were alive, compared with 22% of people who received chemotherapy. Tabernero said that researchers are still trying to tease out why patients who received a combination of pembrolizumab and chemotherapy lived no longer than those in the chemotherapy-alone group.
  • Data from nearly 15 million people living with cancer in the United States is contained in electronic health records (EHR), but many EHR systems format data differently and use different terms to describe the same data, limiting the ability of researchers to draw conclusions about patient outcomes across institutions. To advance data sharing, ASCO, CancerLinQ, and the MITRE Corporation announced the launch of mCode, a set of common cancer data standards and specifications that can be used across EHRs, which is being piloted at cancer centers around the country. Details are available at www.mCODEinitiative.org.

May 2019

May 24–30

  • The FDA approved the first PI3K inhibitor for breast cancer—alpelisib (Piqray; Novartis). Combined with fulvestrant, the drug treats men and postmenopausal women with HR-positive, HER2-negative, PIK3CA-mutated, advanced or metastatic breast cancer whose disease has worsened after an endocrine-based regimen. The approval was based on the phase III SOLAR-1 trial, in which patients who received the combination had a median progression-free survival (PFS) of 11 months, compared with 5.7 months in patients who received a placebo plus fulvestrant.
  • Celgene announced that the FDA approved lenalidomide (Revlimid) in combination with rituximab (Rituxan; Genentech) for previously treated follicular lymphoma and previously treated marginal zone lymphoma. The approval was based on the phase III AUGMENT and MAGNIFY trials, in which the combination extended median PFS and elicited high objective response rates in both diseases. Lenalidomide is an immunomodulatory agent, and rituximab is a CD20-specific antibody; both were previously approved for other blood cancers.
  • In addition, the FDA approved ruxolitinib (Jakafi; Incyte) to treat GVHD in adults and children age 12 and older who don’t respond to steroids. The approval was based on the phase II REACH1 trial, in which patients treated with the drug plus corticosteroids had an overall response rate of 57% and a complete response rate of 31% at day 28. Ruxolitinib is a JAK inhibitor that has been approved for myelofibrosis and polycythemia vera.
  • An article in the Annals of Oncology suggests that patients with non-small cell lung cancer (NSCLC) who have actionable tumor mutations may benefit from receiving targeted therapies and chemotherapy before immunotherapy. Researchers conducted a retrospective analysis of 551 patients with advanced NSCLC who had at least one targetable alteration and had received immune checkpoint inhibitors. They found that patients had a median PFS of 2.8 months, a median OS of 13.3 months, and a response rate of 19%.
  • Ibrutinib (Imbruvica; Janssen) plus venetoclax (Venclexta; AbbVie/Genentech) may be an effective first-line treatment for chronic lymphocytic leukemia (CLL). A phase II trial tested the Bruton tyrosine kinase inhibitor–BCL2-inhibitor combination in 80 patients with CLL who had either a chromosome 17p deletion, mutated TP53, a chromosome 11q deletion, unmutated IGHV, or were over age 65. After 12 cycles of treatment, 88% of patients had a complete remission (CR) or CR with partial hematologic recovery, and 61% had remission with undetectable residual disease.
  • Cancer drugs approved via the FDA accelerated pathway may not provide a survival benefit. Researchers reviewed confirmatory trials for 93 drugs granted accelerated approval by the FDA between 1992 and 2017. They found that just 20% of the agents resulted in an improvement in overall survival (OS).
  • U.S. cancer mortality rates are decreasing in men and women ages 20 to 49, according to an analysis in the Journal of the National Cancer Institute. Between 2012 and 2016, cancer mortality rates decreased by 1.8% in men and 1.4% in women. Additionally, from 2011 to 2015, cancer incidence dropped by 2.1% in men and remained stable in women.

May 17–23

  • Merck will buy Peloton Therapeutics for $1.05 billion up front in a deal that could earn Peloton up to $1.15 billion more in milestone payments. Merck will gain access to Peloton’s experimental molecular therapies including PT2977, a HIF2α inhibitor that has shown promise in treating metastatic renal cell carcinoma in early-stage trials.
  • The U.S. District Court in Boston, MA, ruled that two U.S. scientists should be listed as inventors on six cancer immunotherapy patents. The court determined that the patents, previously issued to Ono Pharmaceutical and Tasuku Honjo, MD, PhD, of Kyoto University in Japan, should also include Gordon Freeman, PhD, of Dana-Farber Cancer Institute in Boston, MA, and Clive Wood, PhD, of Boehringer Ingelheim. The patents, which describe the PD-1 pathway, were foundational to the development of the PD-1 inhibitor nivolumab (Opdivo; Bristol-Myers Squibb).
  • Array BioPharma announced that the BRAF/MEK inhibitor combination encorafenib (Braftovi)/binimetinib (Mektovi) plus the anti-EGFR antibody cetuximab (Erbitux; Eli Lilly) may improve survival in patients with BRAFV600E-mutant metastatic colorectal cancer who have not responded to other therapies. In the phase III BEACON CRC trial, patients treated with the triplet had an overall response rate (ORR) of 26.1% and a median overall survival (OS) of 9 months, compared with 1.9% and 5.4 months in patients who received cetuximab plus chemotherapy; patients treated with encorafenib plus cetuximab had an ORR of 20.4% and a median OS of 8.4 months.
  • Dynavax Technologies will discontinue development of its immune-oncology candidates—and lay off 82 employees, about 37% of its workforce—to focus on its vaccine business, which includes the hepatitis B vaccine Heplisav-B. The company has two toll-like receptor 9 cancer drugs in clinical development: SD-101, which is being tested in combination with the PD-1 inhibitor pembrolizumab (Keytruda; Merck) in various cancers, and DV281, which is being studied in combination with nivolumab in non–small cell lung cancer (NSCLC).
  • Pembrolizumab monotherapy may not be an effective second- or third-line treatment for metastatic triple-negative breast cancer. In the phase III KEYNOTE-119 trial, the drug did not extend OS compared with standard chemotherapy. Pembrolizumab is approved as a monotherapy for various other cancers, including melanoma and NSCLC.
  • Artificial intelligence may be able to detect malignant lung nodules as well as or better than radiologists, according to findings published in Nature Medicine. Researchers developed and trained a deep-learning model using 42,290 CT scans from 14,851 patients who were screened for lung cancer. They found that the algorithm was 94% accurate at detecting the nodules and had fewer false positives and negatives than radiologists when prior imaging was not available; the system performed as well as radiologists when there were prior imaging results.
  • The FDA approved the NovoTTF-100L System (Novocure) in combination with pemetrexed and platinum-based chemotherapy as a first-line therapy for patients with locally advanced or metastatic malignant pleural mesothelioma who can’t have surgery. The approval was based on the single-arm STELLAR trial, in which patients who received the therapy had a median OS of 18.2 months. The system uses tumor treating fields—low-intensity alternating electric fields that interfere with the division of cancer cells.

May 10–16

  • The FDA approved several drugs for new indications and in new combinations, including:

     

    • the VEGFR2 inhibitor ramucirumab (Cyramza; Eli Lilly) for patients with hepatocellular carcinoma who have an alpha fetoprotein level of at least 400 ng/mL, and who have previously been treated with the tyrosine kinase inhibitor sorafenib (Nexavar; Bayer/Onyx)
       
    • the PD-L1 inhibitor avelumab (Bavencio; EMD Serono) plus the VEGFR inhibitor axitinib (Inlyta; Pfizer) for first-line treatment of advanced renal cell carcinoma
       
    • the BCL2 inhibitor venetoclax (Venclexta; AbbVie) plus the anti-CD20 antibody obinutuzumab (Gazyva; Genentech) as a first-line therapy for chronic lymphocytic leukemia or small lymphocytic leukemia
  • The agency also granted an orphan drug designation to P-BCMA-101 (Poseida Therapeutics) for relapsed/refractory multiple myeloma. The investigational agent is an autologous CAR T therapy composed of self-renewing stem cell memory T cells that target B-cell maturation antigen (BCMA) on cancer cells. Researchers are testing the therapy in a phase II trial.
  • Celgene’s immunomodulatory agent lenalidomide (Revlimid) may reduce the risk of smoldering multiple myeloma developing into multiple myeloma, according to findings presented during a media preview for the 2019 American Society of Clinical Oncology Annual Meeting. In the phase III E3A06 trial, patients treated with the drug for 3 years had a progression-free survival rate of 91%, compared with 66% in patients who did not receive treatment. However, 51% of patients discontinued the drug due to side effects.
  • An FDA advisory panel voted 8–3 against the approval of quizartinib (Daiichi Sankyo), a FLT3 inhibitor, as a treatment for patients with relapsed/refractory FLT3-mutant acute myeloid leukemia. The new drug application was submitted based on the findings of the phase III QuANTUM-R trial, in which the drug extended median overall survival by 1.5 months compared with chemotherapy. The panel members said that the results were not strong enough to merit approval; the FDA will make a final decision by August 25, 2019.
  • Advaxis announced that the FDA lifted a partial hold on the cancer vaccine axalimogene filolisbac (Axal) after the company provided additional information about the vaccine’s chemistry, manufacturing, and control groups in the phase III AIM2CERV trial. The company can now continue enrolling patients in the trial, which is testing the vaccine in cervical cancer.
  • The U.S. Court of Appeals for the Federal Circuit voted to uphold a Novartis patent on everolimus, the active ingredient in the chemotherapy drug Afinitor. The decision follows a 2017 ruling against West-Ward Pharmaceuticals, now known as Hikma Pharmaceuticals USA, which claimed the patent was invalid. West-Ward was seeking approval to develop a generic version of the drug for advanced renal cell carcinoma.
  • The NCI awarded nearly $11 million to the Hollings Cancer Center of the Medical University of South Carolina in Charleston and renewed it as an NCI-designated cancer center following its 5-year review. Hollings is one of 70 NCI-designated cancer centers—and the only one in South Carolina. Over the past 5 years, the center has opened 392 research studies and enrolled 3,522 patients in clinical trials.

May 3–9

  • Television ads will be required to include prices for certain prescription drugs covered by Medicare or Medicaid. The rule, issued by the Centers for Medicare & Medicaid Services and set to go into effect in about 60 days, requires commercials to include prices for drugs that cost more than $35 for a month’s supply or for a typical course of therapy. Until now, drug companies have been required to include information on products’ side effects but not prices.
  • A growing number of people with cancer in the United States are eligible for and respond to immune checkpoint inhibitors. Researchers retrospectively analyzed treatment data for six checkpoint inhibitors approved between 2011 and 2018 and found that the percentage of patients eligible for the drugs increased from 1.54% to 43.63%; the percentage who responded to them increased from 0.14% to 12.46%.
  • The FDA approved the antibody–drug conjugate ado-trastuzumab emtansine (T-DM1/Kadcyla; Genentech) as an adjuvant treatment for patients with HER2-positive early breast cancer who have residual invasive disease after receiving neoadjuvant taxane- and trastuzumab (Herceptin; Genentech)-based treatment. The approval was based on the phase III KATHERINE trial, in which the drug significantly improved invasive disease–free survival compared with trastuzumab.
  • The Society of Breast Surgeons called for annual breast cancer screening for women 40 and older of average risk, and a formal risk assessment for women older than 25. In a position statement, the organization recommended more frequent screening than guidelines released by the U.S. Preventive Services Task Force, the American Cancer Society, and the American College of Radiology, citing the need to make clearer screening recommendations to women.
  • Bristol-Myers Squibb announced that the PD-1 inhibitor nivolumab (Opdivo) may not be an effective treatment for some cases of newly diagnosed glioblastoma multiforme. In the phase III CheckMate-498 trial, the drug, in combination with radiation, did not extend overall survival compared with temozolomide chemotherapy plus radiation.
  • The active ingredients in sunscreen may be absorbed into the bloodstream at higher concentrations than previously thought, according to preliminary findings in JAMA. The study randomly assigned 24 participants to one of four sunscreens—two sprays, a lotion, and a cream—that were applied four times a day for 4 days. By the fourth application on the first day, the active ingredients avobenzone, oxybenzone, octocrylene, and ecamsule in all four sunscreens had reached concentrations of more than 0.5 ng/mL in the blood plasma—the threshold the FDA used to waive some nonclinical toxicology studies for sunscreens. The researchers suggest that additional research is needed on the clinical impact of the findings.

April 2019

April 26–May 2

  • The FDA approved ivosidenib (Tibsovo; Agios) as a first-line monotherapy for acute myeloid leukemia (AML) in patients with an IDH1 mutation who are 75 and older or who aren’t eligible for intensive chemotherapy. The approval was based on findings from a phase I trial in which patients treated with the drug had a complete response rate of 28.6% and a complete response plus complete response with partial hematologic recovery rate of 42.9%. Ivosidenib was already approved for patients with relapsed/refractory IDH1-mutant AML.
  • According to findings in JAMA Oncology, many randomized clinical trials for cancer drugs may not use optimal control arms. Researchers analyzed 98 multiarm trials that led to 96 drug approvals between 2013 and 2018 and found that 16 approvals were based on trials with suboptimal control arms. Of these control groups, 10 used an inferior agent or didn’t allow combinations, and four limited the investigator’s choice.
  • Xencor announced that the FDA lifted a partial clinical hold on XmAb14045, a bispecific antibody that targets CD123 and CD3. The hold followed two patient deaths in a phase I trial testing the drug in AML and other blood cancers, including one death associated with cytokine release syndrome (CRS). The company has since amended the trial protocol to include additional guidance on monitoring and managing CRS.
  • The FDA announced that a type of breast implant that has been linked to a rare cancer will continue to be sold in the United States. The implants, which have a textured surface, have been associated with anaplastic large-cell lymphoma, leading to bans in France, Canada, and the Netherlands. Following a two-day public hearing, the FDA determined that there is insufficient data to support banning the implants, although it is considering a black-box warning.
  • Europe’s Committee for Medicinal Products for Human Use issued recommendations on four cancer drugs: It called for the approval of the PARP inhibitor talazoparib (Talzenna; Pfizer) for patients with advanced or metastatic HER2-negative breast cancer and germline BRCA1/2 mutations, and the conditional approval of the PD-1 inhibitor cemiplimab (Libtayo; Regeneron) for advanced cutaneous squamous cell carcinoma. The group also suggested expanding the indication for the PARP inhibitor olaparib (Lynparza; AstraZeneca) as a first-line maintenance therapy for patients with advanced high-grade epithelial ovarian, fallopian tube, or primary peritoneal cancer following first-line platinum-based chemotherapy. Finally, it called for the withdrawal of a conditional approval for the monoclonal antibody olaratumab (Lartruvo; Eli Lily) in combination with doxorubicin for soft-tissue sarcoma.
  • The FDA authorized the marketing of the IQOS tobacco device made by Philip Morris. The “heat not burn” device heats a paper-wrapped tobacco stick, producing a nicotine-containing vapor that’s inhaled. In its announcement, the agency emphasized its commitment to restricting youth access to the devices and noted that the products produce fewer toxins than combustible cigarettes.

April 19–25

  • The FDA approved the PD-1 inhibitor pembrolizumab (Keytruda; Merck) in combination with axitinib (Inlyta; Pfizer), a multi-tyrosine kinase inhibitor, as a first-line treatment for patients with advanced renal cell carcinoma. The approval is based on the phase III KEYNOTE-426 trial, in which patients treated with the combination had an 18-month overall survival rate of 82.3%, a median progression-free survival of 15.1 months, and a response rate of 59.3%, compared with 72.1%, 11.1 months, and 35.7%, respectively, in patients who received sunitinib, a common treatment.
  • Tagraxofusp-erzs (Elzonris; Stemline Therapeutics) may be an effective first-line therapy for blastic plasmacytoid dendritic-cell neoplasm (BPDCN), according to a study published in The New England Journal of Medicine. In an open-label trial of 29 patients, 90% responded to the therapy and 45% went on to receive a stem cell transplant; 52% of patients were still alive at 2 years.  A CD123-directed cytotoxin consisting of recombinant human IL3 fused to truncated diphtheria toxin, tagraxofusp-erzs, was approved for BPCDN last year.
  • Researchers have developed an immunocompromised zebrafish model for studying cancer therapies, as described in Cell. The model, which lacks T, B, and natural killer cells, is clear and can engraft and grow human cancers, allowing researchers to see characteristics and therapeutic responses of individual cancer cells. The model has already been used to investigate olaparib (Lynparza; AstraZeneca) and temozolomide as a potential combination therapy for rhabdomyosarcoma.
  • The Centers for Medicare and Medicaid Services proposed increasing Medicare hospital reimbursement for CAR T-cell therapies such as tisagenlecleucel (Kymriah; Novartis) and axicabtagene ciloleucel (Yescarta; Kite/Gilead). The change would increase the maximum “new technology add-on payment,” which includes CAR T cells, from $186,500 to $242,450 per patient. It would also create a separate reimbursement category for CAR T-cell therapies to better track costs.
  • Patients with blood cancers treated with high-intensity radiation prior to transplant may be more likely to develop subsequent cancers, according to findings in Blood. Researchers retrospectively analyzed 5,000 patients who underwent a bone marrow transplant between 1969 and 2014 and found that those who received high-dose unfractionated or very high-dose fractionated total-body irradiation (TBI) had a higher incidence of secondary cancers than those who received low-dose TBI; the cumulative incidence by 30 years posttransplant was 22%.
  • The number of cervical precancers in the United States is declining, as outlined in Morbidity and Mortality Weekly Report. Between 2008 and 2016, the number of women diagnosed with a high-grade cervical intraepithelial neoplasia of grade 2 or worse decreased from 216,000 to 196,000, with the most significant decreases in women ages 18 to 24. The decline is mostly attributable to immunization with the human papillomavirus vaccine.

April 12–18

  • Bristol-Myers Squibb (BMS) shareholders voted to approve the company’s acquisition of Celgene. The $74 billion merger, announced in January, had been in doubt for the past few months as large BMS shareholders such as Wellington Management, Starboard Value, and Dodge & Cox expressed opposition to the deal out of concern that investors would be accepting too much risk.
  • The FDA granted accelerated approval to the pan-FGFR inhibitor erdafitinib (Balversa; Janssen) for patients with locally advanced or metastatic urothelial carcinoma who have an FGFR3 or FGFR2 mutation and who have not responded to platinum-containing chemotherapy. The approval, the first for a targeted therapy for bladder cancer, was based on the phase II BLC2001 trial, in which 87 patients had an overall response rate of 32.2%. The FDA also approved Qiagen’s Therascreen FGFR RGQ RT-PCR Kit as a companion diagnostic.
  • NPR reported that the first U.S. study attempting to use CRISPR to treat cancer in people is under way. The gene-editing technique has been used on a patient with multiple myeloma and a patient with sarcoma in a clinical trial at the University of Pennsylvania in Philadelphia. CRISPR studies in a variety of diseases, including sickle cell disease, Leber congenital amaurosis, and other cancers, will soon launch in Europe, the United States, and Canada.
  • The FDA released a draft guidance on the development of bispecific antibodies to treat cancer. The document discusses considerations such as pharmacology, manufacturing, and preclinical and clinical testing of the therapies, and delves into similarities and differences in the development of bispecific and monoclonal antibodies. Currently, blinatumomab (Blincyto; Amgen) is the only FDA-approved bispecific antibody; others are in clinical trials. The agency is accepting comments on the draft through June 17 at www.regulations.gov.
  • Clovis Oncology will halt the phase II ATLAS trial testing the PARP inhibitor rucaparib (Rubraca) as a monotherapy in recurrent, metastatic bladder cancer based on an independent data monitoring committee’s determination that treatment is unlikely to show a clinical benefit. The company will continue to test the drug in combinations for bladder cancer, and as a monotherapy for ovarian and prostate cancers. Rucaparib is approved as a second-line and maintenance therapy for BRCA-mutated ovarian, fallopian tube, and primary peritoneal cancers.
  • Tracon Pharmaceuticals is stopping the phase III TAPPAS trial testing TRC105 in combination with pazopanib (Votrient; GlaxoSmithKline) chemotherapy in advanced or metastatic angiosarcoma after an independent data monitoring committee suggested that the combination is not effective in patients. TRC105 is an endoglin antibody that previously received orphan drug designation from the FDA in soft-tissue sarcoma.

April 5–11

  • The American College of Physicians issued guidelines recommending biennial breast cancer screening for women ages 50 to 74 at average risk. The statement, which is based on evidence that screening every other year does not significantly increase breast cancer mortality, aligns with guidelines published by the U.S. Preventive Services Task Force. However, guidelines from the American Cancer Society and the American College of Radiology recommend more frequent screening.
  • Expanded germline panel testing may capture more pathogenic risk variants in patients with cancer, according to an analysis from Invitae presented at the 2019 American College of Medical Genetics and Genomics Annual Meeting in Seattle, WA. Researchers focused on 113,107 individuals with a history of breast, ovarian, colorectal, pancreatic, or prostate cancer, comparing standard genetic testing with two to five gene panels to an expanded next-generation sequencing panel of 83 cancer-related genes. They found that the expanded panel detected clinically actionable risk variants in an additional 9,739, or 8.6% of patients.
  • The FDA expanded the approval of first-line pembrolizumab (Keytruda; Merck) monotherapy in patients with advanced and metastatic non-small cell lung cancer (NSCLC) to include those with a PD-L1 expression level of at least 1% who do not have EGFR or ALK mutations. The approval was based on a phase III trial in which the drug extended overall survival (OS) by 4.6 months compared with chemotherapy. The PD-1 inhibitor was previously approved for the same indication in patients with a PD-L1 expression level of at least 50%.
  • Presented at the European Lung Cancer Congress 2019 in Geneva, Switzerland, a pair of studies highlight the uncertainty of whether elderly patients with advanced NSCLC benefit from immunotherapy as much as younger patients. In a real-world retrospective analysis, patients 70 and older treated with immunotherapy had a significantly shorter median OS and progression-free survival than patients younger than 70. However, in a study that pooled data from three randomized trials of pembrolizumab monotherapy, both groups of patients with advanced PD-L1-positive disease had comparable 1-year survival rates.
  • Merrimack Pharmaceuticals announced that it is discontinuing development of MM-310, an antibody-directed nanotherapeutic under study in various solid tumors. The decision was made because patients treated with the drug in a phase I trial developed peripheral neuropathy. The company previously halted development of the anti-HER2 drug MM-121 and the IGF1R-HER3 bispecific antibody MM-141 due to a lack of efficacy.
  • Researchers have developed a new approach for assessing the carcinogenicity of chemicals. They exposed human cell lines to 330 chemicals known to be carcinogens or noncarcinogens, generating around 6,000 gene-expression profiles. Based on the profiles, a computer algorithm was then able to accurately predict whether the chemicals were carcinogenic. The results have been compiled into a toxicogenomics database available >online.

March 29–April 4
This week: Special content from the AACR Annual Meeting 2019 and other news

  • During the Opening Ceremony of the AACR Annual Meeting, outgoing NCI director Norman "Ned" Sharpless highlighted the institution’s focus on big data, including the NCI Genomic Data Commons, a data repository accessed by 2,500 visitors a day, that will contain data from more than 70,000 patients by the end of 2019. However, he emphasized that genomic data becomes more powerful when linked to other types of data, such as clinical features, patient outcomes, proteomics, radiology, and pathology. "This is our vision—moving from a unimodal genomic data commons to a multimodal cancer research data commons," he said.
  • Also during the Opening Ceremony, the NCI’s soon-to-be acting director, Doug Lowy, MD, discussed the agency’s new childhood cancer data initiative, which will establish more efficient ways to use and share data. The initiative will involve creating a protocol for the collection of pediatric cancer data and developing a database to integrate and store data that will be available to researchers. "If we can combine this currently siloed information from institutions, we would have the potential to chart a path that would change the course of cancer for all children," he said.
  • "We really are in uncharted waters," said Richard Marais, PhD, director of the Cancer Research UK Manchester Institute, during a panel discussion on Brexit’s potential impact on cancer research. Researchers, organizations, industry employees, academics, and others have been calling upon the UK government to safeguard funding for science: "We need to protect ourselves from the consequences of Brexit," he said. Asked what attendees could do to prevent a Brexit-like situation in the future, Marais simply stated, "Vote."
  • Mesothelin-targeted CAR T cells may be a promising therapy for solid tumors, according to preliminary data presented by Prasad Adusumilli, MD, of Memorial Sloan Kettering Cancer Center in New York, NY. In a phase I trial of 21 patients with mesothelioma or pleural metastatic lung or breast cancer, the T cells elicited responses in 10 patients, including eight of 11 who received a PD-1 inhibitor following CAR T-cell therapy. "This strongly supports pursuing a CAR T-cell therapy combined with anti–PD-1 strategies in solid tumors—so strategies to make these ‘cold’ tumors ‘hot’ by injecting CAR T cells first," Adusumilli said.
  • Updating meeting attendees on the progress of the Biden Cancer Initiative, Dr. Jill Biden, wife of former U.S. Vice President Joe Biden, pointed out that some advances that have reduced cancer mortality have not been shared by all segments of the population. "Progress is uneven, and rural, black, Latino, and Native American populations see far worse outcomes," she said. "Poverty draws a line between surviving and succumbing to this disease. It doesn’t have to be this way. We have to put a stop to these disparities."
  • The FDA’s Richard Pazdur, MD, and Marc Theoret, MD, moderated a discussion on inhibitors of the PD-1 axis. Pressed on whether the slew of drugs approved over the last 5 years comprise more "me-too" agents than not, a panel of industry leaders—from Bristol-Myers Squibb, Merck, AstraZeneca, Genentech, EMD Serono, and Regeneron—agreed that although the drugs have largely shown similar clinical activity, there have been clear differences in outcomes that are as yet poorly understood. With head-to-head studies being unlikely, the panelists opined that as trials of anti–PD-1 agents combined with other therapies increase in number, these data could shed light on any differences to be found in blocking the PD-1 receptor versus taking aim at one of its two ligands.
  • During a special session on April 3, researchers, clinicians, nonprofit executives, and patient advocates debated which potential conflicts of interest should be disclosed—and how—when scientific data are reported at meetings and in journals. Some argued in favor of disclosing all possible conflicts for the sake of transparency; others advocated a more limited approach in which only relevant conflicts of interest would be disclosed to prevent key items from getting "lost in the noise." Lewis Cantley, PhD, director of the Cancer Center of Weill Cornell Medical College and New York-Presbyterian Hospital in New York, NY, provided food for thought when he asked, "Who decides what’s relevant?"
  • EMD Serono’s TGFβ- and PD-L1–targeting bifunctional fusion protein bintrafusp alpha (M7824) may be a promising therapy for HPV-associated cancers, according to preliminary data presented by Julius Strauss, MD, of the NCI at a Clinical Trials Plenary. In a phase I trial, 34.9% of 43 patients with HPV-associated cancers responded to the drug, with a median overall survival (OS) of 16.2 months and a 12-month survival rate of 56.2%. "I think it’s the duration of the response and its impact on OS that I find most intriguing about this agent," said discussant Maura Gillison, MD, PhD, of The University of Texas MD Anderson Center in Houston.
  • Speaking about the increasing use of electronic cigarettes (e-cigarettes) in the United States, Brian King, PhD, MPH, of the Centers for Disease Control and Prevention, highlighted the rapid evolution of the e-cigarette landscape and the need to modernize tobacco control strategies to keep up. "We don’t need to reinvent the wheel, folks, we just need to grease that squeaky wheel to ensure that our policies address the diversity of products that the American public is using," King said. He also called for more research, including randomized clinical trials that assess the value of e-cigarettes for smoking cessation.
  • Martin Hutchings, MD, PhD, of Copenhagen University Hospital in Denmark, discussed ongoing research on novel T-cell bispecific (TCB) antibodies for hematologic malignancies and solid tumors. One such agent is RG6026 (Genentech BioOncology), a CD20-TCB that binds to CD20 on tumor cells and CD3 on T cells and has shown preliminary efficacy in a phase I trial of relapsed/refractory B-cell non-Hodgkin lymphomas. Another, cibisatamab (RG7802; Genentech BioOncology), is a CEA-TCB that binds to carcinoembryonic antigen and CD3 and has demonstrated promising activity against non–T-cell inflamed solid tumors—namely colorectal cancer—in a phase I trial.
  • The AACR has partnered with Cancer Research UK to support collaborations between early-career cancer researchers in the United States and UK through the new Transatlantic Fellowships program. The awards will provide $400,000/£300,000 over 4 years to recent PhD recipients and early-stage postdoctoral researchers to help U.S. investigators establish research programs in the UK, and vice versa. The organizations are accepting applications until July 25.

 

In other news:

  • AstraZeneca will pay Daiichi Sankyo $1.35 billion up front in a deal that could be worth up to $6.9 billion. Per the deal, AstraZeneca will work with Daiichi Sankyo to develop and commercialize trastuzumab deruxtecan (DS-8201), Daiichi’s HER2-targeting antibody–drug conjugate. The drug has shown efficacy in phase I trials of HER2-positive cancers and is currently being tested in phase I, II, and III trials.
  • The FDA expanded the indication of the CDK4/6 inhibitor palbociclib (Ibrance; Pfizer) to include men with HR-positive, HER2-negative advanced or metastatic breast cancer. The approval is based on real-world data from post-marketing reports and electronic health records showing that the drug, plus an aromatase inhibitor or fulvestrant (Faslodex; AstraZeneca), has a similar safety profile in men and women.

March 2019

March 22–28

  • The FDA introduced a proposal that would require mammogram centers to inform patients if they have dense breast tissue, which can mask tumors on scans. The proposal recommends that patients with dense breasts consult with a health care provider about additional steps to take. More than half of women in the United States over age 40 have dense breast tissue, which is a risk factor for developing breast cancer. The proposal is available for public comment until June 26.
  • First-line treatment with the endocrine therapy fulvestrant plus the aromatase inhibitor anastrozole may improve long-term survival in postmenopausal women with hormone receptor–positive metastatic breast cancer, according to findings in The New England Journal of Medicine. After 7 years of follow-up in the phase III SWOG S0226 trial, patients treated with the combination had an overall survival of 49.8 months, compared with 42 months in patients who received anastrozole alone. The researchers previously reported that the combination extended progression-free survival.
  • Agios announced that the FDA granted a breakthrough therapy designation to the IDH1 inhibitor ivosidenib (Tibsovo) in combination with azacitidine chemotherapy as a first-line therapy for acute myeloid leukemia (AML) in patients who have an IDH1 mutation and are not eligible for intensive chemotherapy. The designation is based on positive results in a phase I/IIb trial, in which the drug elicited an overall response rate of 78.3%. Ivosidenib is FDA-approved for relapsed/refractory IDH1-mutant AML.
  • The agency also granted an orphan drug designation to tinostamustine (EDO-S101; Imbrium Therapeutics) for T-cell prolymphocytic leukemia, a rare and aggressive form of T-cell leukemia. The investigational agent is an alkylating deacetylase inhibiting molecule that is being studied in early-phase clinical trials.
  • The National Comprehensive Cancer Network (NCCN) updated clinical practice guidelines for prostate cancer to recommend greater genetic testing. The updates focus on germline testing in patients with metastatic or high-risk localized nonmetastatic disease, as well as somatic testing in patients with lymph node or distant metastases, the results of which can inform treatment. The changes were introduced at the 2019 National NCCN Annual Meeting in Orlando, FL.
  • Guardant Health announced it will acquire Bellwether Bio, a startup developing a method for early detection of blood cancers based on epigenetic signals in circulating cell-free DNA. In the deal, researchers from Bellwether will join Guardant’s work on that company’s early-detection pipeline, which includes the LUNAR liquid biopsy test for early-stage cancer detection based on genomic alterations and epigenomic signatures.

March 15–21

  • The FDA approved atezolizumab (Tecentriq; Roche) in combination with carboplatin and etoposide chemotherapy as a first-line therapy for small cell lung cancer. The approval was based on the phase III IMpower133 trial, in which atezolizumab plus chemotherapy extended median overall survival (OS) by 2 months and median progression-free survival (PFS) by 0.9 months compared with a placebo plus chemotherapy. A PD-L1 inhibitor, atezolizumab was previously approved for certain forms of bladder, breast, and non–small cell lung cancer.
  • The FDA placed a partial hold on clinical trials testing venetoclax (Venclexta; AbbVie, Roche) in multiple myeloma, halting enrollment of new patients. The decision was based on data from the phase III BELLINI trial, in which 21.1% of patients treated with venetoclax plus bortezomib (Velcade; Takeda) and dexamethasone died, compared with 11.3% of patients receiving a placebo plus bortezomib and dexamethasone. Venetoclax is approved for other blood cancers, including chronic lymphocytic leukemia and acute myeloid leukemia.
  • The phase III JAVELIN Ovarian PARP 100 trial will be discontinued. The trial is testing the PD-L1 inhibitor avelumab (Bavencio; Pfizer, EMD Serono) in combination with chemotherapy followed by maintenance avelumab plus the PARP inhibitor talazoparib (Talzenna; Pfizer) in patients newly diagnosed with advanced or metastatic ovarian cancer. The announcement came after an independent panel determined that the trial would not meet its primary endpoint, PFS.
  • The National Comprehensive Cancer Network updated its clinical practice guidelines for colorectal cancer to include Array BioPharma’s BRAF/MEK inhibitor combination, encorafenib (Braftovi)/binimetinib (Mektovi), plus an EGFR inhibitor as a second-line treatment option for patients with advanced BRAF-mutant disease. The recommendation is based on the BEACON CRC trial, in which the triplet elicited an overall response rate of 48%, an estimated median PFS of 8 months, and an estimated median OS of 15.3 months.
  • CureMetrix announced that the FDA cleared its cmTriage mammogram screening software. The software uses artificial intelligence to help radiologists sort and prioritize mammogram screening results by flagging images showing abnormalities that require more urgent review. The company is also developing a computer program called cmAssist that marks irregular regions on mammograms and ranks the degree of abnormality.
  • Medicaid coverage of lung cancer screening for high-risk individuals varies widely by state, according to a report released by the American Lung Association. The report found that 31 states cover lung cancer screening through Medicaid and 12 states do not; seven others do not have publicly available policies. In total, 26.3% of those on Medicaid are current smokers, a key risk factor for developing lung cancer that makes them more likely to require screening.

March 8–14

  • NCI Director Norman E. “Ned” Sharpless, MD, will become acting commissioner of the FDA in early April, replacing Scott Gottlieb, MD, who resigned. Sharpless, a medical oncologist, has led the NCI since October 2017. Deputy NCI Director Doug Lowy, MD, will become acting director of the institute.
  • President Donald Trump proposed a $4.7 billion decrease in funding for the NIH as part of the White House budget plan for fiscal year (FY) 2020; the 12% cut would result in a total budget of $34.4 billion for the agency. The proposal would cut NCI funding by $897 million, or 14.5%, bringing its total budget to $5.247 billion. The NIH budget increased by $3 billion in FY 2018, and $2 billion in FY 2019.
  • The FDA granted an accelerated approval to atezolizumab (Tecentriq; Genentech) in combination with nab-paclitaxel (Abraxane; Celgene) for patients with inoperable advanced or metastatic triple-negative breast cancer whose tumors express PD-L1. The approval is based on the phase III IMpassion130 trial, in which patients treated with the combination had a median progression-free survival of 7.4 months, compared with 4.8 months in those who received a placebo plus nab-paclitaxel. A PD-L1 inhibitor, atezolizumab is the first immunotherapy approved for breast cancer.
  • The agency also approved trastuzumab-qyyp (Trazimera; Pfizer), a biosimilar of trastuzumab (Herceptin; Genentech) for HER2-positive breast cancer and HER2-positive metastatic gastric or gastroesophageal junction adenocarcinoma. Trastuzumab-qyyp is the third biosimilar approved for trastuzumab: Trastuzumab-dttb (Ontruzant; Samsung Bioepis), trastuzumab-pkrb (Herzuma; Celltrion), and trastuzumab-dkst (Ogivri; Mylan) were previously approved for similar indications.
  • Researchers developed an ALK-targeted antibody–drug conjugate for neuroblastoma, according to findings published in Science Translational Medicine. The drug, CDX-0125-TEI, combines a monoclonal antibody that recognizes ALK-expressing cells with the alkylating chemotherapy agent thienoindole. In the study, it eliminated neuroblastoma cells in mouse models and cell cultures, and did not damage healthy cells.
  • The FDA issued a series of guidance documents aimed at increasing and broadening participation in cancer clinical trials. Four draft guidances focus on incorporating into trials specific subsets of patients who are typically considered ineligible, including those with HIV or hepatitis B or C virus infections, brain metastases, organ dysfunction, and prior or concurrent malignancies, as well as pediatric patients. The agency also issued a final guidance outlining considerations for including adolescent patients in adult oncology clinical trials. The draft guidances are open for public comment until April 12 at https://www.regulations.gov/.
  • The FDA also released a draft policy to regulate flavored electronic cigarettes (e-cigarettes) and combustible cigars. If approved, the policy would move up by one year, to August 8, 2021, the date by which e-cigarette companies must submit premarket applications for products with flavors other than mint, menthol, or tobacco on the market as of 2007. The policy also outlines specific steps to limit sales of flavored e-cigarettes to minors and proposes a ban on flavored cigars. The draft is available for public comment until April 10.

March 1–7

  • FDA Commissioner Scott Gottlieb, MD, announced that he will resign in early April. Gottlieb was confirmed as head of the agency in May 2017, and during his tenure he has focused on regulating tobacco and electronic cigarettes, speeding up generic drug approvals, and combating the opioid epidemic.
  • Paige.AI announced that the FDA granted a breakthrough designation to an artificial intelligence (AI) tool designed to diagnose cancer, the first such technology to receive the designation for cancer diagnosis. Launched last year, the company is training its deep-learning AI tool on digitized cancer pathology slides—to date, the company has over one million slides, with another 4 million to be added across cancer subtypes. Paige.AI plans to first market its AI technology for prostate cancer.
  • The FDA released an updated draft guidance on the naming convention for biological products. Per the guidance, the FDA will not change the names of biologics already licensed or approved under the Public Health Service Act without an FDA-designated suffix. However, moving forward, the agency will give each biological product—whether an originator product or a biosimilar—a name that combines the core drug name with a unique four-letter suffix. The agency is accepting comments on the draft through May 7 at www.regulations.gov.
  • A novel anti-CD47 drug may be active in patients with advanced solid tumors and lymphomas, according to a study in The Journal of Clinical Oncology. In a phase I trial of 62 patients, most of whom had colorectal or ovarian cancer, Hu5F9-G4 was well tolerated and yielded some responses: Two patients with ovarian cancer had partial remissions, and one patient with diffuse large B-cell lymphoma had a mixed response. A previously reported phase Ib trial indicated that the experimental antibody, in combination with rituximab, may be effective in patients with non-Hodgkin lymphoma.
  • ImmunoGen announced that the antibody–drug conjugate mirvetuximab soravtansine (IMGN853) may not be an effective monotherapy for patients with folate receptor alpha–positive (FRα), platinum-resistant ovarian cancer. In the phase III FORWARD 1 trial, the drug did not significantly improve progression-free survival compared with standard chemotherapy. Designed to target FRα, mirvetuximab soravtansine is also being tested in combination with other therapies for ovarian, peritoneal, and fallopian tube cancers.
  • Women awarded grants as first-time principal investigators (PI) receive less NIH funding than their male counterparts, according to findings in the Journal of the American Medical Association. Researchers analyzed 53,903 grants awarded to first-time PIs between 2006 and 2017 and found that women received a median of $126,615, whereas men received a median of $165,721. This trend held true at Big Ten universities ($66,365 vs. $148,076), Ivy League universities ($52,190 vs. $71,703), and the top 50 NIH-funded institutions ($93,916 vs. $134,919).

February 2019

February 22–28

  • The future of the $74 million merger between Bristol-Myers Squibb (BMS) and Celgene is in doubt because large shareholders have expressed their opposition to the deal, which was announced in January. Wellington Management company, which owns an 8% share of the company, said that it will not support the move because investors will be accepting too much risk and that shares of BMS offered to Celgene stockholders have been priced too low. Other shareholders with a large stake in BMS, namely Starboard Value and Dodge & Cox, are also unhappy with the merger.
  • AstraZeneca and Merck announced that the PARP inhibitor olaparib (Lynparza) improved progression-free survival in patients with pancreatic cancer compared with a placebo. The drug’s efficacy as a maintenance therapy for those with germline BRCA-mutated metastatic disease that has not progressed on platinum-based chemotherapy has been under investigation in a phase III trial. The companies called the results “statistically significant and clinically meaningful,” adding that data will be presented at an upcoming medical meeting.
  • The FDA approved trifluridine/tipiracil tablets (Lonsurf; Taiho) for adults with metastatic gastric or gastroesophageal junction cancer previously treated with at least two chemotherapy regimens that included a fluoropyrimidine, a platinum, either a taxane or irinotecan, and, if appropriate, HER2/neu-targeted therapy. Approval was based on a phase III trial that included 507 patients; median overall survival was 5.7 months for those receiving trifluridine, a nucleoside metabolic inhibitor, and tipiracil, a thymidine phosphorylase inhibitor, compared with 3.6 months for those who received a placebo.
  • In an 8 to 5 vote, members of an FDA advisory committee recommended that the agency await additional data from a phase III trial before approving selinexor (Karyopharm Therapeutics) for the treatment of multiple myeloma, due to the drug’s toxicity. Karyopharm is seeking approval of the drug, a first-in-class compound that inhibits the nuclear export protein XPO1, for patients who are refractory to three types of drugs: an anti-CD38 monoclonal antibody, a proteasome inhibitor, and an immunomodulatory agent. Although the agency usually follows the panel’s advice, its recommendation is nonbinding.
  • The FDA issued a safety communication regarding the use of robotically assisted surgical devices for mastectomy and for the prevention or treatment of various cancers that primarily affect women. Although robotically assisted procedures have been widely adopted because they require smaller incisions and could result in speedier recovery—as well as greater surgical precision—the safety and efficacy of the robots has not been established for mastectomy, the treatment of cervical cancer, and other procedures. The agency emphasized that physicians and patients need to consider the risks and possible benefits of these types of procedures and consider alternatives.

February 15–21

  • Merck announced that the PD-1 inhibitor pembrolizumab (Keytruda) may not be an effective second-line therapy for advanced hepatocellular carcinoma. In the phase III KEYNOTE-240 trial, the drug did not extend overall survival (OS) or progression-free survival (PFS) compared with a placebo. However, the drug may be an effective first-line treatment for non–clear cell renal cell carcinoma, according to results presented at the 2019 Genitourinary Cancers Symposium in San Francisco, CA. In the phase II KEYNOTE-427 trial, patients treated with pembrolizumab had an overall response rate of 24.8%, a median PFS of 4.1 months, and a 12-month OS rate of 72%.
  • The FDA approved pembrolizumab as an adjuvant treatment for patients with high-risk stage III melanoma that involves the lymph nodes. The approval is based on the phase III EORTC 1325/KEYNOTE-054 trial, in which patients treated with the drug had an 18-month recurrence-free survival (RFS) rate of 71.4%, and the median RFS was not reached, compared with 53.2% and 20.4 months in patients who received a placebo.
  • Following Eli Lilly’s purchase of Loxo Oncology for $8 billion, Bayer announced that it has acquired full rights to Loxo’s TRK inhibitors larotrectinib (Vitrakvi) and LOXO-915. In 2017, Bayer made a $400 million deal with Loxo that included an option to take full control of the drugs if Loxo was bought. Larotrectinib is approved for patients of all ages who have solid tumors harboring NTRK1/2/3 fusions, regardless of tissue of origin.
  • The U.S. Preventive Services Task Force released a draft statement recommending screening for women with a family history of breast, ovarian, tubal, or peritoneal cancer or ethnicity or ancestry associated with BRCA1/2 mutations to identify whether these factors place them at increased risk of carrying harmful BRCA1/2 mutations. The recommendation is similar to one published in 2013, but it adds ethnicity and ancestry as reasons to screen and incorporates evidence from more recent studies. The draft statement is open for public comment until March 18.
  • The Centers for Medicare and Medicaid Services (CMS) proposed covering FDA-approved chimeric antigen receptor (CAR) T-cell therapies when they are offered through a CMS-approved registry or clinical trial where patients are monitored for at least 2 years after treatment. Currently, there is no national Medicare policy in place to cover approved CAR T-cell therapies. The proposal is open for public comment until March 17.
  • Merck announced it will acquire Immune Design for $300 million. Immune Design is using in vivo approaches to develop immunotherapies. Its lead candidate is G100, an intratumoral TLR4 agonist that is currently is being tested in follicular non-Hodgkin lymphoma.
  • The FDA placed a partial clinical hold on XmAb14045 (Xencor), a bispecific antibody that targets CD123 and CD3. The move came after two patients died in a phase I trial testing the drug in relapsed/refractory acute myeloid leukemia and other blood cancers that express CD123.
  • The Broad Institute launched the Gerstner Center for Cancer Diagnostics, which will be funded by a $15 million donation from Louis V. Gerstner, Jr., plus a $10 million endowment from the Eli and Edythe Broad Foundation. The center, in Cambridge, MA, will focus on developing liquid biopsy technologies that can track patients’ responses to therapies and monitor their levels of minimal residual disease. The center will also develop new diagnostic tests.

February 8–14

  • Johnson & Johnson agreed to buy surgical robotics company Auris Health for $3.4 billion in a deal that could earn Auris up to $2.35 billion more in milestone payments. Auris, which will become part of Johnson & Johnson’s medical-devices division, is developing a surgical robotic endoscope called Monarch for the diagnosis and treatment of lung cancer. The device was cleared by the FDA last year.
  • African American men with advanced prostate cancer may respond better to certain therapies than white men, according to a retrospective study presented at the 2018 Genitourinary Cancers Symposium in San Francisco, CA. Researchers analyzed data from 787 African American men and 2,123 white men with metastatic castration-resistant disease treated with abiraterone or enzalutamide (Xtandi; Astellas Pharma) between 2013 and 2018. They found that the median overall survival (OS) was 30 months for African American men, compared with 26 months for white men.
  • Also reported at the Symposium, the targeted radiation therapy lutetium Lu 177 PSMA 617 (Endocyte) may be an effective treatment for men with PSMA-positive metastatic castration-resistant prostate cancer whose disease has worsened on other therapies. In a phase II study, 50 men treated with the therapy had a median OS of 13.3 months, and 32 men with a PSA decline of 50% or greater had a median OS of 18 months. Related therapy Lutetium Lu 177 dotatate (Lutathera; Novartis, Advanced Accelerator Applications) was FDA approved in 2018 for somatostatin receptor–positive gastroenteropancreatic neuroendocrine tumors.
  • A study in CANCER estimates that since 1989, hundreds of thousands of deaths due to breast cancer may have been prevented by screening mammography and improved treatment. Researchers analyzed population and breast cancer mortality data from the Surveillance, Epidemiology, and End Results program using different background mortality assumptions, and determined that about 384,046 to 614,484 deaths were averted between 1989 and 2018.
  • The NCI awarded nearly $20 million to the Perlmutter Cancer Center, NYU Langone Health, in New York, NY, and promoted the center to a comprehensive cancer center, giving it an “outstanding” rating following its 5-year review. Researchers at Perlmutter are involved in research in a variety of areas, including developing therapies for bladder and lung cancers and melanoma. Perlmutter is one of 50 NCI-designated comprehensive cancer centers.
  • According to survey data from the Centers for Disease Control and Prevention, more than 3.6 million U.S. middle and high school students used electronic cigarettes (e-cigarettes) in 2018, an increase of 1.5 million over 2017. In total, 4.9 million students used tobacco products in 2018, a 38.3% increase largely driven by e-cigarette usage.

February 1–7

  • A study in The Lancet Public Health reported that obesity-related cancer rates are increasing in younger U.S. adults. Researchers analyzed data on 30 cancers in 25 states from 1995 to 2014 and found that the incidence increased for six of 12 cancers related to obesity, including multiple myeloma and colorectal, kidney, and pancreatic cancers, in successively younger birth cohorts. By contrast, incidence decreased or stabilized in 16 of 18 cancers not linked to obesity.
  • The U.S. Preventive Services Task Force released a draft statement recommending against pancreatic cancer screening in adults who don’t have symptoms. The recommendation has not changed since 2004, but it now incorporates additional evidence from more recent studies. The draft statement will be open for public comment until March 4.
  • Only about a third of biomedical studies include data on sex, according to a study in The Lancet. Researchers analyzed more than 11.5 million medical research papers published between 1980 and 2016 and found that sex-related reporting increased from 59% to 67% in clinical medicine, 36% to 69% in public health, and 28% to 31% in biomedical research. Papers with a female first or last author were more likely to report sex, and journals with high impact factors were less likely.
  • In his State of the Union address, President Donald Trump called for $500 million to fund pediatric cancer research over the next decade. However, Speaker of the House Nancy Pelosi criticized the plan, noting that Congress approved $1.8 billion over 7 years for the Beau Biden Cancer Moonshot. “$500 million over 10 years—are you kidding me?” Pelosi said during a closed-door conference meeting, as reported by Politico. “We’re talking about a moonshot. He’s talking about a trolley ride.”
  • Men with prostate cancer may benefit from more widespread germline genetic testing, according to results in JAMA Oncology. Researchers performed germline genetic testing on 3,607 men with prostate cancer and found that 17.2% had germline mutations, 30.7% of which were BRCA1/2 and 4.5% of which were HOXB13. However, only 63% of those with mutations would have been approved for genetic testing based on current National Comprehensive Cancer Network guidelines.
  • Also in JAMA Oncology, researchers documented a case of bone cancer in a 240-million-year-old turtle from the Triassic period. The shell-less stem turtle, found in present-day Germany, had an osteosarcoma on its femur similar in appearance to present-day periosteal osteosarcoma in humans. The case is the oldest known instance of cancer in a mammal, bird, or reptile, and points to the deep evolutionary roots of the disease.

January 2019

January 25–31

  • Electronic cigarettes (e-cigarettes) may be an effective tool for smoking cessation, according to a study in The New England Journal of Medicine. In a randomized trial of 886 cigarette smokers in the UK, researchers found that 18% of those assigned to switch to e-cigarettes were smoke-free after a year, compared with 9.9% of those assigned to nicotine replacement therapies such as patches and gum. However, of the participants who were smoke-free after a year, 80% of e-cigarette users continued to use the product, compared with 9% of those who tried nicotine replacement therapies.
  • The monoclonal antibody olaratumab (Lartruvo; Eli Lilly) should not be started in new patients, and patients taking the drug should consult with their clinicians about continuing treatment, according to the FDA. The agency’s assessment was based on a phase III trial in which patients with soft-tissue sarcoma who received the drug plus doxorubicin lived no longer than those taking doxorubicin alone. The European Medicines Agency made a similar announcement.
  • Janssen and Pharmacyclics announced that the FDA approved ibrutinib (Imbruvica) in combination with obinutuzumab (Gazvya; Genentech) as a first-line treatment for patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL). The approval was based on the phase III iLLUMINATE trial, in which the combination significantly extended progression-free survival compared with chlorambucil plus obinutuzumab. A Bruton tyrosine kinase inhibitor, ibrutinib is also approved to treat other B-cell cancers, such as mantle cell lymphoma.
  • A study in Nature explained how pancreatic cancer starts and grows. Using a 3-D, whole-organ imaging technique that can analyze individual cells, researchers found that pancreatic tumors are either endophytic, growing into the lumen of pancreatic ducts, or exophytic, growing outward. The type of tumor depended on the diameter of the ducts, with ducts narrower than 20 µm yielding exophytic tumors. The team found similar growth patterns in lung and liver cancers.
  • The UK’s National Institute for Health and Care Excellence (NICE) recommended tisagenlecleucel (Kymriah; Novartis) for patients with relapsed/refractory diffuse large B-cell lymphoma. National Health Service England will make the chimeric antigen receptor (CAR) T-cell therapy available to patients via the Cancer Drugs Fund. The drug, which was previously recommended by NICE for patients under age 25 with relapsed/refractory B-cell acute lymphoblastic leukemia, will be provided by Novartis at a discounted price.
  • Health and Human Services Secretary Alex Azar announced draft rules to increase drug-pricing transparency. Under the rules, drug manufacturers could offer discounted prices directly to consumers, but could not give rebates to pharmacy benefit managers, who serve as middlemen between companies and consumers. If finalized, the regulations would apply to Medicare and Medicaid drug plans.
  • Stand Up To Cancer (SU2C) awarded $8 million to researchers studying T-cell lymphoma. The so-called Dream Team will work on developing off-the-shelf CAR T-cell therapies for the disease and identify biomarkers to track their effectiveness. The American Association for Cancer Research is SU2C’s scientific partner.

January 18–24

  • The FDA approved 23andMe’s direct-to-consumer genetic test for a hereditary colorectal cancer syndrome. The MUTYH-Associated Polyposis Genetic Health Risk report tests for two MUTYH variants that can increase the risk of developing colorectal cancer by 43% to 100%. The FDA previously approved 23andMe’s test for three BRCA1/2 mutations most commonly seen in women of Ashkenazi Jewish descent.
  • The agency also approved trastuzumab-dttb (Ontruzant; Samsung Bioepis), a biosimilar of trastuzumab (Herceptin; Genentech) for HER2-positive metastatic breast cancer and gastric cancer or gastroesophageal junction adenocarcinoma, and as an adjuvant treatment for HER2-positive breast cancer. Trastuzumab-dttb is the third biosimilar approved for trastuzumab: Trastuzumab-pkrb (Herzuma; Celltrion) and trastuzumab-dkst (Ogivri; Mylan) were previously approved for similar indications.
  • Researchers may soon be able to determine which precancerous lung lesions will become malignant, according to findings in Nature Medicine. The team performed genomic testing on 129 samples of precancerous lung lesions from 85 patients and identified molecular features such as mutations and copy-number changes present in lesions that became cancerous during a 5-year follow-up period. The reseach could be used to improve early detection of lung cancer and inform the development of preventive therapies.
  • Cancer Research UK awarded $79 million to three research ventures through its Grand Challenge. The projects, which will each receive around $26 million over 5 years, are investigating cancer drivers in different tissues, novel treatments for inflammation-associated cancers, and the link between the microbiome and colorectal cancer.
  • Bristol-Myers Squibb withdrew its FDA application for a checkpoint inhibitor combination—nivolumab (Opdivo) plus ipilimumab (Yervoy)—as a first-line treatment for patients with advanced non–small cell lung cancer who have a high tumor mutational burden. The decision came after the FDA asked the company for more data on the relationship between tumor mutational burden and PD-L1 expression. The combination is being tested in the ongoing phase III Checkmate-227 trial.
  • The FDA placed a partial hold on the cancer vaccine axalimogene filolisbac (Axal; Advaxis), requesting additional information about the vaccine’s chemistry, manufacturing, and control groups in the phase III AIM2CERV trial. Although the company must halt recruitment for AIM2CERV, which is testing the vaccine in cervical cancer, patients already enrolled can continue to receive treatment.
  • AbbVie announced that ibrutinib (Imbruvica) in combination with chemotherapy did not improve outcomes for patients with metastatic pancreatic cancer. In the phase III RESOLVE trial, the combination did not significantly extend progression-free or overall survival compared with a placebo plus chemotherapy. A Bruton tyrosine kinase inhibitor, ibrutinib is FDA approved for various blood cancers, including chronic lymphocytic leukemia, mantle cell lymphoma, and Waldenström macroglobulinemia.

January 11–17

  • The FDA approved cabozantinib (Cabometyx; Exelixis) for patients with hepatocellular carcinoma who have previously received sorafenib (Nexavar; Bayer/Onyx). Approval was based on the phase III CELESTIAL trial, in which patients treated with the drug had a median overall survival (OS) of 10.2 months and a median progression-free survival of 5.2 months, compared with 8 months and 1.9 months, respectively, in patients who received a placebo. A tyrosine kinase inhibitor, cabozantinib was previously approved for certain forms of renal cell carcinoma.
  • The U.S. House of Representatives Committee on Oversight and Reform announced it will investigate the drug-pricing methods of pharmaceutical companies. The committee requested information from 12 companies about 18 drugs, including Celgene’s immunomodulatory agent lenalidomide (Revlimid), AbbVie/Johnson & Johnson’s Bruton tyrosine kinase inhibitor ibrutinib (Imbruvica), and Novartis’s tyrosine kinase inhibitor imatinib (Gleevec).
  • Eli Lilly reported that the monoclonal antibody olaratumab (Lartruvo) may not be effective in advanced or metastatic soft-tissue sarcoma. In the phase III ANNOUNCE trial, the drug plus doxorubicin did not extend OS compared with doxorubicin alone, nor did it provide a survival benefit in the leiomyosarcoma subgroup. The FDA previously granted olaratumab an accelerated approval based on results of a phase II trial.
  • Patients with comorbidities are less likely to participate in clinical trials, according to findings in JAMA Oncology. Researchers analyzed survey data from 5,499 patients with cancer and found that 37.2% of those with comorbid conditions discussed clinical trials with their clinicians, 15.7% were offered slots in trials, and 7.8% participated in trials, compared with 44.1%, 21.7%, and 11.3% of those without multiple health problems. Removing the comorbidity restrictions that are currently recommended by the American Society of Clinical Oncology would result in up to 6,317 additional patients being enrolled in trials each year.
  • In a study in Nature Genetics, researchers characterized new subtypes of B-cell acute lymphoblastic leukemia (B-ALL). The team performed an integrated genomic analysis on 1,988 cases of pediatric and adult B-ALL and identified 23 subtypes of the disease, including eight new subtypes. Two of the new subtypes have PAX5 alterations and account for 10% of previously uncategorized cases of the disease; a third, defined by a rearrangement of BCL2 with MYC or BCL6, is associated with poor outcomes in patients.
  • Scientists may have developed a new approach for pancreatic cancer screening, according to a study in Clinical Cancer Research. The approach combines two blood tests that detect molecules produced by pancreatic cancer cells: a new test that measures the sTRA glycan and an existing test that measures the cancer antigen 19-9. Together, the tests detected 65%–75% of pancreatic cancers with 95%–97% accuracy.
  • The Huntsman Cancer Institute in Salt Lake City, UT, received a donation of $30 million from the Huntsman Foundation. The donation will fund the Kathryn F. Kirk Center for Comprehensive Cancer Care and Women’s Cancers building. The institute has been an NCI-designated comprehensive cancer center since 2015.
  • In Cancer Cell, researchers described a likely molecular mechanism underlying cancerous facial tumors in Tasmanian devils, one of the only known transmissible cancers. In a series of experiments, researchers established that the cancer cells have increased activation of ERBB receptors, which may activate STAT3 proteins and suppress expression of MHC class I genes, thus driving tumor growth. They also found that targeting the ERBB–STAT3 axis inhibited tumor growth and restored expression of MHC class I genes.

January 4–10

  • Eli Lilly announced it will acquire Loxo Oncology for $8 billion. With the deal, Lilly will gain access to Loxo’s cancer drugs—including the TRK inhibitor larotrectinib (Vitrakvi), which was approved in November for patients with solid tumors harboring NTRK1/2/3 fusions, and the RET inhibitor LOXO-292, which is being tested in various solid tumors, including lung and thyroid cancers.
  • As the partial government shutdown continues, the FDA announced it will shift funds to prioritize drug safety surveillance over premarket drug review. Since the start of the shutdown, the FDA has recalled several drugs due to safety concerns, including an anesthesia companion injection and a blood pressure medication. Currently, about 40% of FDA employees are furloughed.
  • Artificial intelligence may identify cervical cancer and precancer, according to findings published in the Journal of the National Cancer Institute. Researchers developed and trained a deep-learning algorithm using more than 60,000 photos from cervical cancer screenings. They found that the algorithm was significantly more accurate than clinicians or conventional cytology at identifying cases of cervical cancer and precancer, achieving an area under curve of 0.91, compared with 0.69 and 0.71, respectively.
  • The BRCA Exchange, a large-scale database of information on BRCA genes, became publicly available. The database, which was created through the BRCA Challenge, contains more than 20,000 unique BRCA1 and BRCA2 variants, including 6,100 variants that have been classified by an expert panel and 3,700 that are known to cause disease. A paper describing the development of the database was published in PLOS Genetics.
  • The U.S. cancer death rate has decreased continuously for the past 25 years, according to a report published by the American Cancer Society. Overall, the cancer death rate dropped by 27% between 1991 and 2016, although cancer is still the second leading cause of death after heart disease. The report also estimates that there will be 1,762,450 new cancer cases and 606,880 cancer deaths in the United States in 2019.
  • At the JP Morgan Healthcare Conference in San Francisco, CA, Exact Sciences announced that sales of its Cologuard test had a 115% compound annual growth rate between 2014 and 2018, but the test has been adopted by only 4% of its potential market. Cologuard, which was FDA approved in 2014, is a diagnostic test for colon cancer that detects DNA markers shed by cancerous and precancerous cells in a patient’s stool. In late 2018, Exact Sciences made a deal with Pfizer to jointly promote the test through 2021.
  • Also at the JP Morgan Healthcare Conference, UroGen Pharma announced positive results from a phase III trial of UGN-101, a novel version of the chemotherapeutic mitomycin. In the OLYMPUS trial, UGN-101 elicited complete responses (CR) in 57% of 61 patients with low-grade upper-tract urothelial carcinoma, and all evaluable patients with a CR were disease free at 6 months.
  • A patient with glioblastoma was the first to receive treatment under “right to try” legislation. The patient, who failed to qualify for a clinical trial, was treated with the experimental immunotherapeutic vaccine ERC-1671 (ERC-USA) after ERC-USA informed the FDA that it would provide the therapy. Passed last year, the legislation allows terminally ill patients to seek experimental treatments directly from pharmaceutical companies, outside of the FDA’s expanded access pathway.
 
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