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Noted This Week - 2015 Archive

Archive of cancer-related news briefs, by week, for 2015

December 2015

December 18, 2015

  • Under an omnibus spending bill approved by the U.S. Congress, the NIH will receive a $2 billion budget increase for fiscal year (FY) 2016, bringing its total budget to $32.084 billion. Similarly, the NCI and FDA will receive budget increases of more than 5% over their FY2015 budgets. All three agencies have experienced flat funding or outright cuts for more than a decade.
  • The FDA approved alectinib (Alecensa; Genentech) to treat people with metastatic ALK-positive non–small cell lung cancer (NSCLC) whose disease has worsened after, or who could not tolerate treatment with, crizotinib (Xalkori; Pfizer). Alectinib is an oral medication that blocks ALK activity, which may prevent NSCLC cells from growing and spreading. In addition to shrinking lung tumors in some patients, alectinib caused shrinkage of tumors that had spread to the brain, a common site of metastasis in patients with ALK-positive NSCLC.
  • The FDA also approved uridine triacetate (Vistogard; Wellstat Therapeutics) for the emergency treatment of adults and children with cancer who receive an overdose of fluorouracil or capecitabine, or who develop certain severe or life-threatening side effects within 4 days of receiving these drugs. Although an overdose of or severe reactions to fluorouracil or capecitabine are rare, they can be fatal if not treated quickly.
  • The Princeton, NJ–based pharmaceutical firm Advaxis announced that the FDA had lifted the clinical hold on all three of the company’s investigational drugs—axalimogene filolisbac (formerly ADXS-HPV), ADXS-PSA, and ADXS-HER2—after the company revised a study’s design, agreed to additional monitoring procedures, and took additional steps to insure patient safety. The move allows Advaxis to resume all clinical trials of the immunotherapies, which are in development for HPV-related cancers, prostate cancer, and HER2-expressing cancers, respectively. Development of all three drugs was stopped after a patient with cervical cancer died while participating in a study of axalimogene filolisbac.

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  • According to an analysis by the online health and life-sciences publication STAT, 90% of academic and nonprofit institutions and 74% of drug and device makers do not submit clinical trial results to ClinicalTrials.gov within 1 year of a study’s completion, as mandated by law, findings that NIH Director Francis Collins, MD, PhD, called “very troubling.” Although the federal government can impose fines for failure to comply with the law, no such fines have been levied; the publication estimated that the government could have collected $25 billion from drug companies alone over the past 7 years.
  • The FDA proposed tanning-bed age restrictions and other measures to help protect minors and adults from skin cancer, which can be caused by radiation from the devices. One rule would prohibit the use of sun lamps by anyone under age 18; people 18 and over would have to sign a form acknowledging the health risks. A second rule would require sunlamp manufacturers and tanning facilities to take additional safety measures such as making warnings easier to read and requiring emergency shut-off switches.

November 26–December 10, 2015

  • At the annual meeting of the American Society of Hematology (ASH) in Orlando, FL, investigators reported a new use for rituximab (Rituxan; Genentech): the treatment of acute lymphocytic leukemia. In a 220-patient study, those who received rituximab in addition to standard chemotherapy had a lower incidence of relapse compared with those who received chemotherapy alone (18% vs. 30.5%), and were more likely to achieve 2-year "event-free" survival (65% vs. 52%). Rituximab, which targets CD20, is approved for patients with other malignancies, including B-cell non-Hodgkin lymphoma and chronic lymphocytic leukemia (CLL).
  • Also at the ASH meeting, investigators presented data from a phase III trial suggesting that ibrutinib (Imbruvica) should be the standard first-line therapy for CLL and small lymphocytic lymphoma. In the trial, 269 newly diagnosed patients received either the Pharmacyclics/Janssen drug ibrutinib or the chemotherapy drug chlorambucil for up to 12 cycles. Those who received ibrutinib had an 18-month progression-free survival rate of 93.9%, versus 44.8% in those treated with chlorambucil. At 2 years, overall survival was 97.8% in the ibrutinib group, versus 85.3% in the chlorambucil group.
  • Mumbai, India’s Sun Pharmaceutical Industries announced that one of its subsidiaries received FDA approval for a generic version of Novartis’s Gleevec (imatinib mesylate), used for the treatment of chronic myeloid leukemia. The company aims to launch the therapeutic equivalent of Gleevec in the United States on February 1, 2016, and is eligible for 6 months of marketing exclusivity.
  • Jacksonville, FL–based TapImmune received the FDA’s Orphan Drug Designation for its TPIV 200 cancer vaccine for the treatment of ovarian cancer. TPIV is a multi-epitope peptide vaccine that targets folate receptor alpha, which is overexpressed in more than 90% of ovarian cancer cells. The designation provides the company with several benefits, such as tax credits on clinical research and 7 years of marketing exclusivity if the vaccine is approved.
  • Despite some promising signs, phase III studies of evofosfamide (TH-302) for the treatment of soft-tissue sarcoma and pancreatic cancer did not meet their primary endpoints, prompting Merck KGaA, which had partnered with Threshold Pharmaceuticals, to stop its development. Evofosfamide was designed to be administered as a prodrug that is activated only under hypoxic conditions commonly found in the tumor microenvironment. Instead, Merck KGaA said it will focus on the development of avelumab and other high-profile oncology and immunotherapy products.
  • According to a study of 16 countries published in the Bulletin of the World Health Organization, people living in low-income countries (India, Pakistan, and Zimbabwe) are exposed to 81 times more tobacco advertisements than those living in high-income countries (Canada, Sweden, and the United Arab Emirates). Similarly, the number of outlets selling tobacco products was two and a half times greater in the low-income and lower middle–income countries (China, Colombia, and Iran) than in the affluent nations.
  • Writing in Lancet Oncology, researchers reported that the list price of several cancer drugs across 15 European countries differs by up to 92% and that the actual prices paid, after discounts, varies by up to 58%. Also published in the journal, a separate study of 31 cancer drugs in 16 European countries, Australia, and New Zealand, found that the difference in prices for particular drugs varies between 28% and 388%. In general, the drug prices in Greece and the United Kingdom are lowest and those in Switzerland, Germany, and Sweden are the highest.

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November 2016

November 25, 2015

  • Pfizer and Allergan announced that they will merge operations in a deal worth $160 billion, creating the world’s largest drugmaker. Because Pfizer, an American corporation, is combining with a company headquartered in Ireland, which has a lower tax rate, Pfizer could save millions of dollars in U.S. taxes annually. Pfizer manufactures several cancer drugs, including palbociclib (Ibrance), an inhibitor of CDK4 and 6; sunitinib (Sutent), a multikinase angiogenesis inhibitor; and temsirolimus (Torisel), an mTOR inhibitor.
  • Bristol-Myers Squibb’s immunotherapeutic nivolumab (Opdivo) received FDA approval for two more indications: the treatment of patients with metastatic renal cell carcinoma after they have received an antiangiogenic agent, and as a single agent for patients with previously untreated BRAF wild-type advanced melanoma. The PD-1 inhibitor was originally approved in December 2014 for the treatment of inoperable, metastatic melanoma that progressed after treatment with the company’s CTLA-4 inhibitor ipilimumab (Yervoy).
  • The FDA also approved Eli Lilly’s necitumumab (Portrazza) in combination with gemcitabine and cisplatin for first-line treatment of patients with metastatic squamous non–small cell lung cancer (NSCLC); the drug is not indicated for the treatment of nonsquamous NSCLC. Necitumumab is a recombinant human IgG1 monoclonal antibody that binds to EGFR, blocking the binding of EGFR to its ligands.
  • Beverly, MA–based Cellceutix Corporation announced that the FDA granted Orphan Drug Designation to thioureidobutyronitrile (Kevetrin) for the treatment of retinoblastoma, a rare form of eye cancer. The drug activates wild-type p53 and induces apoptosis. The FDA designation provides federal grants, tax credits, and other incentives to companies developing drugs for diseases or medical conditions affecting fewer than 200,000 people in the United States.
  • Copenhagen, Denmark’s Genmab announced that a phase III trial of ofatumumab (Arzerra) in follicular non-Hodgkin lymphoma will be stopped early because an interim analysis found that the drug was unlikely to show superiority over a standard rituximab regimen. Approved for the treatment of certain patients with chronic lymphocytic leukemia, ofatumumab is designed to target the CD20 molecule.
  • Hampton, NJ–based Celldex Therapeutics announced that a phase II study of the vaccine rindopepimut (Rintega) showed a marked benefit in patients with recurrent glioblastoma whose cancer is EGFRvIII-positive. The study was designed to determine if adding rindopepimut to standard-of-care bevacizumab (Avastin; Genentech) improves survival over bevacizumab alone. Nine out of 10 patients on the rindopepimut arm have survived, compared with just two of five patients in the control arm, since the company presented preliminary data in May; at 2 years, the survival rate for rindopepimut patients is 25% compared with 0% for control patients.
  • A report by Paul Nurse, PhD, outgoing president of the Royal Society in London, UK, suggests that the nation’s seven research-funding agencies be managed by a new umbrella organization called Research UK, which would be more powerful than the current Research Councils UK. In the report, Nurse said that the new body would provide strong management, reduce bureaucracy, and facilitate discussions with the government about shifting funding among the seven agencies. He also proposed creating a new fund to support interdisciplinary research.

November 19, 2015

  • Gilead Sciences announced that a phase III study evaluating the addition of idelalisib (Zydelig) to the standard therapy of bendamustine plus rituximab in patients with previously treated chronic lymphocytic leukemia will be unblinded early. An interim analysis indicated a statistically significant improvement in progression-free survival and overall survival in patients who received the three-drug regimen. Detailed study results will be presented at next month’s annual meeting of the American Society of Hematology.
  • Merck KGaA and Pfizer announced that the FDA granted Breakthrough Therapy designation to avelumab, an investigational, fully human anti–PD-L1 IgG1 monoclonal antibody for the treatment of patients with metastatic Merkel cell carcinoma (MCC) whose disease has progressed after at least one chemotherapy regimen. The designation is designed to accelerate the development and review of medicines for which preliminary evidence demonstrates a significant improvement over currently available therapies for a serious condition. No therapy is currently approved for metastatic MCC.
  • Cambridge, MA–based Momenta Pharmaceuticals announced a temporary halt in enrollment in the phase II portion of its phase I/II trial of necuparanib (M402) in combination with nab-paclitaxel (Abraxane; Celgene) and gemcitabine (Gemzar; Eli Lilly) in patients with metastatic pancreatic cancer. An independent data safety monitoring committee noted some specific toxicities—thrombocytopenia, risk of bleeding, and thromboembolic events—in trial participants. Momenta is amending its trial protocol to standardize the diagnosis and management of thrombocytopenia.
  • President Obama’s nominee to head the FDA, Robert Califf, MD, faced questions from a U.S. Senate committee about his background and previous receipt of funding from the drug industry. Califf, who was named the FDA’s deputy commissioner for medical products and tobacco by former FDA commissioner Margaret Hamburg before she left the agency earlier this year, explained that industry is one of the main funders of research in the United States and that receiving their financial support doesn’t necessarily indicate a conflict of interest. A date for a vote on his confirmation has not been set.
  • The Wall Street Journal reported that sales of electronic cigarettes (e-cigarettes) have dropped dramatically over the past few months. The market for the devices has grown by 114% annually over the past 5 years, but that rate is expected to decline to 54% next year. Dissatisfaction among consumers, new state laws, and questions about e-cigarette safety have contributed to the sales decline.
  • Two significant, just-announced philanthropic gifts will support cancer researchers in their quest for cures. A $30 million commitment to The University of Texas MD Anderson Cancer Center in Houston from New York businessman Andy Sabin, president of Sabin Metal Corporation, will fund up to eight 2-year research fellowships at $100,000 each in the first year. The Robert and Myra Kraft Family Foundation, headed by New England Patriots owner Robert Kraft, established a $20 million endowment at Harvard Business School in Boston, MA, to support research with the nearby Broad Institute and other activities, such as addressing the business challenges associated with precision medicine.

November 13, 2015

  • Genentech’s cobimetinib (Cotellic), in combination with vemurafenib (Zelboraf), received FDA approval for the treatment of patients with inoperable or metastatic melanoma with the BRAF V600E or V600K mutation. Cobimetinib is not indicated for treatment of patients with wild-type BRAF melanoma. The approval was based on improvements in progression-free survival and overall survival in a multicenter, double-blind, placebo-controlled phase III trial involving 495 patients with BRAF V600 mutation–positive melanoma, as detected using the cobas 4800 BRAF V600 mutation test.
  • AstraZeneca announced that the FDA granted accelerated approval to osimertinib (AZD9291; Tagrisso) for the treatment of patients with metastatic EGFR T790M mutation–positive non–small cell lung (NSCLC) cancer whose disease worsened following treatment with another EGFR-blocking therapy. The agency also approved the cobas EGFR Mutation Test v2 to detect the T790M point mutation. The safety and efficacy of the drug were demonstrated in two multicenter, single-arm studies involving a total of 411 patients with NSCLC harboring this mutation; 57% of patients in the first study and 61% of those in the second experienced a complete or partial reduction in tumor size.
  • Following intense negotiations between the U.S. Senate and the House of Representatives, President Obama signed the Bipartisan Budget Act of 2015 on November 2. The act lifts caps on the budget for the 2016 and 2017 fiscal years (FY), allowing an additional $50 billion in spending for FY2016 and $30 billion in FY2017, half of which is to be directed to nondefense areas of the budget each year. Exactly how that money will be allocated has not yet been determined, but the NIH will likely receive additional funds because both Democrats and Republicans have shown strong support for the agency.
  • Data from the 2014 National Health Interview Survey, reported by the Centers for Disease Control and Prevention (CDC), indicate that the prevalence of cigarette smoking among U.S. adults declined from 20.9% to 16.8% between 2005 and 2014, including a decline of 1% between 2013 and 2014. Another major finding was that the average number of cigarettes smoked per day among daily smokers dropped from 16.7 in 2005 to 13.8 in 2014. As in past years, the prevalence of smoking was higher among males than females—18.8% versus 14.8%, respectively.
  • Scientists from the CDC reported in The New England Journal of Medicine that cancer cells originating in a common tapeworm can take root in people with a weakened immune system, causing the development of cancer-like tumors. The discovery came nearly 3 years after doctors in Colombia asked the CDC to help diagnose an HIV-positive patient whose tumors looked somewhat like human cancer but contained much smaller cells and didn’t behave like human cancer. The report raises concern that similar cases, if they occur, may be misdiagnosed as human cancer—especially in less-developed countries where this tapeworm and immune system–suppressing illnesses like HIV are widespread.

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October 2015

October 30 2015, 2015

  • A 22-member panel of researchers for the International Agency for Research on Cancer, part of the World Health Organization, declared that processed meat products, such as salami, bacon, and hot dogs, are carcinogenic to humans. That puts these products in the highest risk category for cancer, along with tobacco smoke and diesel engine exhaust. Although the evidence isn’t as strong as for processed meat, the panel also concluded that fresh meat, such as steak and pork, probably causes cancer.
  • The FDA approved talimogene laherparepvec (Imlygic, T-vec; Amgen), the first oncolytic viral therapy in the United States, for the local treatment of inoperable cutaneous, subcutaneous, and nodal lesions in patients with melanoma that recurs following initial surgery. Talimogene laherparepvec is a genetically modified herpes simplex virus that’s injected directly into tumors, where it replicates and produces granulocyte-macrophage colony-stimulating factor (GM-CSF). The tumor then ruptures, causing the release of tumor-derived antigens, which along with GM-CSF may promote an antitumor immune response.
  • The FDA also expanded the approved use of the CTLA-4 inhibitor ipilimumab (Yervoy; Bristol-Myers Squibb) to include adjuvant therapy for patients with stage III melanoma, to lower the risk that melanoma will return after surgery. Ipilimumab, which may work by allowing the immune system to recognize and attack tumor cells, was originally approved in 2011 to treat late-stage melanoma that cannot be treated with surgery.
  • The U.S. Patent and Trademark Office agreed to hear a case that could invalidate Celgene Corporation’s patent on lenalidomide (Revlimid). Angered by dramatic price increases for the blood cancer drug, hedge-fund manager Kyle Bass is challenging the company’s patent “for a program to ensure the drug isn’t given to pregnant women,” Bloomberg Business reported. A similar program is already in place for Accutane, an acne medicine, potentially invalidating Celgene’s patent.
  • In three new policy statements aimed at protecting youth from the effects of tobacco and nicotine, the American Academy of Pediatrics “strongly recommends the minimum age to purchase tobacco products, including e-cigarettes, should be increased to age 21 nationwide.” In addition, the group urges the FDA to regulate electronic cigarettes, battery-operated devices that burn liquid nicotine, in the same way as other tobacco products. This would include age restrictions, taxes, bans on advertising to youth, and bans on flavored products that attract youth. The recommendations are available in the policy and advocacy section at www.aasp.org.

October 23, 2015

  • Under updated screening guidelines issued by the American Cancer Society, women with an average risk of breast cancer should have annual mammograms beginning at age 45 instead of age 40, although they can opt to start as early as age 40. Women age 55 and older should transition to screening every other year unless they choose to stick with the annual schedule. Published in The Journal of the American Medical Association, the guidelines no longer recommend routine clinical breast examination.
  • The FDA approved the irinotecan liposome injection (Onivyde; Merrimack Pharmaceuticals), in combination with fluorouracil and leucovorin, to treat patients with advanced pancreatic cancer who have previously received gemcitabine-based chemotherapy. In a randomized, open-label study of 417 patients whose cancer had grown despite previous treatment, those who received the combination of drugs lived an average of 6.1 months, compared with 4.2 months for those treated with only fluorouracil and leucovorin. There was no improvement in survival for those who received only the irinotecan liposome injection.
  • The investigational antibody–drug conjugate inotuzumab ozogamicin received Breakthrough Therapy designation from the FDA for the treatment of acute lymphoblastic leukemia. Inotuzumab ozogamicin links a monoclonal antibody that targets CD22, a surface antigen expressed on about 90% of B-cell malignancies, with the cytotoxic agent calicheamicin, which enters cancer cells and destroys them. The designation is intended to expedite the development and review of certain drugs.
  • In addition, the FDA granted Orphan Drug designation to STM 434 (Atara Biotherapeutics) for the treatment of ovarian cancer. STM 434 is a fusion protein that binds Activin A and other ligands of the ActR2B receptor involved in the growth and proliferation of ovarian cancer and other malignancies.
  • The American Thyroid Association issued new evidence-based guidelines to help clinicians determine whether thyroid nodules are malignant, interpret biopsy results and molecular marker studies, and initiate risk assessment and cancer screening. Published in the journal Thyroid, the guidelines also focus on the diagnosis and management of differentiated thyroid cancer.
  • According to a report published by Nature, under an amended Russian law, scientists should vet research manuscripts with the country’s Federal Security Service before findings are published or presented at conferences. President Putin’s decree, now being enforced at the A.N. Belozersky Institute of Physico-Chemical Biology at Lomonosov Moscow State University, expands the scope of an existing law obligating scientists in Russia to get permission from the security service before publishing any information that might have “military or industrial significance.”
  • Oregon Health & Science University in Portland released an iPhone app designed to advance melanoma research by giving users the ability to accurately measure and monitor moles, and to contribute photos of how the moles evolve over time. Called Mole Mapper, the app is free to download from the App Store. It was developed with Research Kit, an open-source framework developed by Apple.

October 16, 2015

  • The FDA approved expanding the use of nivolumab (Opdivo) to treat patients with non-squamous non–small cell lung cancer (NSCLC) whose disease progressed during or after platinum-based chemotherapy. Because an evaluation of tumors from a subgroup of patients suggests that the level of PD-L1 expression in NSCLC tumors may help identify patients who are more likely to benefit from the drug, the FDA also approved the PD-L1 IHC 28-8 pharmDx test (Dako; Glostrup, Denmark) to detect PD-L1 expression levels. The Bristol-Myers Squibb drug was previously approved for the treatment of squamous NSCLC.
  • Reuters reported that AstraZeneca has temporarily stopped clinical trials of AZD9291 and durvalumab (MEDI4736), two experimental drugs for the treatment of advanced NSCLC, “due to an increase in the incidence of interstitial lung disease–like reports.” AZD9291 is an EGFR inhibitor, and durvalumab, an anti–PD-L1 therapy.
  • Cancer Research UK (CRUK) launched Grand Challenges, a grant program that will dole out £100 million (about $155 million) to “overcome the greatest barriers standing in the way of beating cancer.” CRUK will invite scientists from around the world in academia, technology, and business to come up with innovative and ambitious plans to develop vaccines to prevent nonviral cancers; eradicate cancers caused by the Epstein-Barr virus; discover how unusual patterns of mutation are induced by cancer-causing events; distinguish lethal cancers from nonlethal ones; map tumors at the molecular and cellular level; develop approaches to target MYC; and deliver biologically active macromolecules to cells in the body. For more details, visit www.cruk.org/grandchallenge.
  • Eli Lilly and Company announced plans to add 30,000 square feet to its R&D facility at the Alexandria Center for Life Science in New York, NY, space that will include a translational immune-oncology “hub” and help the company strengthen its relationships with nearby academic medical institutions and prominent medical schools. Earlier this year, Lilly announced that R&D expansions would also take place in San Diego, CA, and in Cambridge, MA.
  • The FDA approved SonaCare Medical’s Sonablate 450 focused ultrasound system for the ablation of prostate tissue. Focused ultrasound enables treatment of organ-confined prostate cancer, as well as benign prostatic hyperplasia, while preserving surrounding healthy tissues, without the need for radiation or surgery.
  • According to a survey of 3,954 patients undergoing surgery for lung cancer or colorectal cancer, 80% of patients with lung cancer and nearly 90% of those with colorectal cancer believed that surgery would cure their cancer. Even the majority of patients diagnosed with stage IV lung or colorectal cancer responded that surgery was likely to be curative—about 57% and 80%, respectively. The findings were published in Cancer.
  • A study of 2,259 patients published in The New England Journal of Medicine found that taking vitamin D and/or calcium supplements after having precancerous colorectal adenomas removed does not reduce the risk of developing polyps or cancer in the future. Earlier studies in cells and in mice had shown that vitamin D slowed the growth of cancer cells by inhibiting the formation of new blood vessels to feed tumors and by directly killing cancer cells; calcium, too, had shown promise in preventing colorectal cancer.

October 8, 2015

  • The director of the NIH, Francis Collins, MD, PhD, warned a congressional subcommittee that the Precision Medicine Initiative, the Brain Research though Advancing Innovation Initiative, and other key NIH programs might need to be put on hold if the agency doesn’t receive additional funding in fiscal year (FY) 2016, which began on October 1. A budget for the year has yet to be approved, and the government is operating on a continuing resolution (CR), which will maintain spending at FY 2015 levels until December 11. Asked about the possibility of a year-long CR, Collins said the impact on the NIH “would be simply devastating.”
  • The FDA approved expanding the indication for a medical device called Optune to treat patients with newly diagnosed glioblastoma multiforme as part of initial standard treatment. Optune, made by Novocure (Portsmouth, NH), involves placing electrodes on the surface of a patient’s scalp to deliver low-intensity, alternating electrical fields called tumor treatment fields, which target rapidly dividing tumor cells. The device was originally approved in 2011 for patients with GBM that recurred or progressed following chemotherapy treatment.
  • The Centers for Disease Control and Prevention announced that it awarded $22.8 million in an effort to increase colorectal cancer screening among people ages 50 to 75. The funds, grants as part of a competitive process, were given to 24 state health departments, as well as six universities and an American Indian tribe.
  • The U.S. Preventive Services Task Force issued draft recommendations for colorectal cancer screening to update its 2008 recommendations. It continues to recommend screening with colonoscopy every 10 years, beginning at age 50, plus an annual fecal immunochemical test and an annual high-sensitivity guaiac-based fecal occult blood test. To read the full draft statement, and to submit comments by November 2, log on to www.uspreventiveservicestaskforce.org.
  • Eli Lilly announced that the FDA granted Breakthrough Therapy Designation to abemaciclib, a cyclin-dependent kinase 4 and 6 inhibitor, for patients with refractory hormone receptor–positive advanced or metastatic breast cancer. Cyclin-dependent kinases play a key role in regulating cell-cycle progression. The designation expedites the development and review of drugs intended to treat a serious condition, for which preliminary evidence demonstrates that they may offer substantial improvement over existing therapies.
  • The FDA also conferred special status on two other agents. Boulder, CO’s Clovis Oncology announced that the agency granted priority review to rociletinib for the treatment of advanced EGFR-mutant non–small cell lung cancer that harbors the EGFR T790M mutation; a decision is expected by March 30, 2016. In addition, Cambridge, MA’s Blueprint Medicines announced that it received Orphan Drug Designation for BLU-554 for the treatment of hepatocellular carcinoma (HCC). The drug inhibits FGFR4, which may be a key driver in up to 30% of patients with HCC.
  • Berkeley, CA–based XOMA Corporation announced that it has licensed the global development and commercialization rights to its anti-TGFβ antibody program exclusively to Novartis. XOMA will receive a $37 million up-front payment and up to $480 million for meeting various milestones. TGFβ is a potent immune-suppressive cytokine involved in many cellular processes.
  • The FDA announced the launch of a national public education campaign to prevent and reduce tobacco use among multicultural youth who identify with the hip-hop peer crowd, a group that is often hard to reach and frequently exposed to pro-tobacco images and messages. Research suggests that they are more likely to use tobacco than other youth. The ads will begin to air on October 13 during the BET Hip-Hop Awards.

October 2, 2015

  • The FDA granted accelerated approval to the PD-1 inhibitor pembrolizumab (Keytruda; Merck) to treat patients with metastatic non–small cell lung cancer (NSCLC) whose disease has progressed after other treatments and with tumors that express a protein called PD-L1. Pembrolizumab is approved for use with a companion diagnostic, the PD-L1 IHC 22C3 pharmDx test, the first test designed to detect PD-L1 expression in NSCLC. Nivolumab (Opdivo; Bristol-Myers Squibb) also targets the PD-1/PD-L1 pathway and was approved to treat the squamous subtype of NSCLC in March.
  • The FDA also granted accelerated approval to nivolumab in combination with ipilimumab (Yervoy; Bristol-Myers Squibb) for the treatment of patients with BRAF V600 wild-type, unresectable or metastatic melanoma. Both drugs were already approved as monotherapies for advanced melanoma, with nivolumab intended for use after treatment with ipilimumab.
  • According to a study published in the Journal of Clinical Oncology, women with epithelial ovarian cancer can safely take hormone replacement therapy (HRT) during treatment—and it might extend their survival. The study followed 150 women with the disease, 75 of whom received HRT for up to 5 years after the study began, and 75 who did not take HRT. After 22 years, 53 women who took HRT had died, compared with 68 women in the group that did not.
  • The Henry Ford Health System unveiled plans for a $110 million outpatient cancer center in Detroit, MI. The five-story, 144,000-square-foot facility will feature a rooftop garden and a skywalk that connects to the Henry Ford Hospital. Groundbreaking will occur in the spring, with completion slated for mid-2018.

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September 2015

September 24, 2015

  • The FDA approved Taiho Oncology’s Lonsurf, a combination of trifluridine and tipiracil, for patients with metastatic colorectal cancer who have previously been treated with chemotherapy and biologic therapy and no longer respond to them. The safety and efficacy of Lonsurf were evaluated in an international, randomized, double-blind study involving 800 patients. Those treated with Lonsurf lived an average of 7.1 months compared with 5.3 months for patients treated with placebo.
  • In updated guidelines for smoking cessation for adults, the U.S. Preventive Services Task Force (USPSTF) concluded that there isn’t enough evidence to determine whether electronic cigarettes are a safe or effective method to help smokers kick the habit. The USPSTF recommends that clinicians point patients to cessation methods that have proven effective, such as nicotine replacement therapy. The guidelines were published in the Annals of Internal Medicine.
  • More than 300 scientists, health professionals, and patient advocates participated in the Rally for Medical Research Hill Day, converging on Capitol Hill for meetings with congressmen and their staff to urge the U.S. Congress to support robust, sustained, and predictable funding increases for the NIH in 2016 and beyond.
  • In a report on the United Kingdom’s (UK) Cancer Drugs Fund, the country’s National Audit Office (NAO) said that nearly £1 billion (about $1.52 billion) has been spent on drugs not covered by the National Health Service (NHS) for 74,000 patients since the fund was established in 2010. In addition, over the last 2 years, the NHS overspent its budget for the drugs by 35%. However, the NAO noted, due to poor recordkeeping and a lack of data, no one knows whether the drugs paid for by the fund led to improvements in patient survival compared with standard care.
  • The FDA granted Priority Review to two drugs for cancer: ofatumumab (Arzerra; Genmab/Novartis) as a maintenance therapy for relapsed chronic lymphocytic leukemia, and carfilzomib (Kyprolis; Amgen) in combination with dexamethasone for patients with multiple myeloma who have received at least one prior therapy. The agency is expected to issue approval decisions on both drugs on or before January 22, 2016.
  • In addition, the FDA granted special designations to two other cancer drugs. Can-Fite BioPharma announced that its small-molecule drug CF 102, which binds to the A3 adenosine receptor, received Fast Track Designation as a second-line treatment for hepatocellular carcinoma. AB Science announced that its tyrosine kinase inhibitor masitinib was granted Orphan Drug Designation for the treatment of esophagogastric adenocarcinoma.
  • Reuters reported that AstraZeneca has decided to release preclinical data from more than 50 drugs as part of a crowd-sourcing initiative to help speed the search for drug combinations that would fight cancers synergistically. The drug data will be provided to researchers along with matched genomic information from the Wellcome Trust Sanger Institute in the UK.
  • Researchers at Uppsala University in Sweden are taking a different tack to find optimal drug combinations for cancer. They have developed a robot with the ability to test a dozen drugs simultaneously, but they aim to have it handle many more, preferably hundreds. The team also wants to build more knowledge into the guiding algorithm of the robot, such as information about drug targets and disease pathways.

September 17, 2015

  • According to a national survey of 1,000 American voters conducted on behalf of the American Association for Cancer Research (AACR), 74% favor increasing federal funding for cancer research. The survey, conducted by Hart Research Associates and Public Opinion Strategies, also found that five out of every six voters recognize that progress is being made against cancer. The survey was released in conjunction with the AACR’s fifth annual Cancer Progress Report, which calls for annual budget increases of at least 7% for the NIH, NCI, and FDA beginning in 2016.
  • The FDA ordered R.J. Reynolds Tobacco Company to stop the sale and distribution of four currently marketed cigarette products—Camel Crush Bold, Pall Mall Deep Set Recessed Filter, Pall Mall Deep Set Recessed Filter Menthol, and Vantage Tech 13—because they do not meet requirements of the Federal Food, Drug, and Cosmetic Act—namely that they must be substantially equivalent to their respective “predicate” products (products that were commercially marketed as of February 15, 2007). The FDA said the products have different characteristics, and that the manufacturer failed to show that the new products do not raise different questions of public health when compared with the predicate products.
  • San Diego, CA–based Pathway Genomics launched CancerIntercept, a noninvasive liquid biopsy designed to detect tumor DNA in high-risk but otherwise healthy patients, and to monitor treatment efficacy, disease progression, and recurrence in patients with active or previously diagnosed cancer. The tests analyze the presence of 96 frequently occurring DNA mutations in nine cancer genes.
  • The FDA granted Breakthrough Therapy Designation to nivolumab (Opdivo) for advanced or metastatic renal cell carcinoma (RCC), Bristol-Myers Squibb announced. The designation is based on a phase III trial that evaluated survival among 821 patients with previously treated clear-cell RCC who received nivolumab or everolimus (Afinitor; Novartis), a current standard of care. The trial was stopped in July, after an independent data monitoring committee concluded that nivolumab demonstrated superior overall survival; data will be presented at the European Cancer Congress later this month.
  • In addition, the FDA granted Fast Track Designation to BC-819, a potential treatment for non–muscle invasive bladder cancer in patients whose disease recurred after standard care as well as in those who are unresponsive or intolerant to standard care. Under development by BioCancell of Jerusalem, Israel, BC-819 is a double-stranded DNA plasmid construct that uses the H19 gene to activate the synthesis of diphtheria toxin after entering a cell in which H19 transcription factors exist, destroying only that cell.
  • The Pharmaceutical Research and Manufacturers of America reported that U.S. biopharmaceutical companies are developing 836 medicines and vaccines to treat cancer, all of which are in clinical trials or awaiting FDA review. That number includes 123 drugs for lung cancer, 106 for leukemias, and 82 for breast cancer. The company’s report is available at www.phrma.org.
  • Cancer Research UK and MedImmune opened a new laboratory in Cambridge, UK, where scientists from both organizations will share knowledge and expertise to discover and develop novel biologics to treat and diagnose cancer. The alliance brings together MedImmune’s human antibody phase display libraries and protein-engineering expertise with Cancer Research UK’s cancer biology expertise.
  • Australia’s Industry and Science Minister Ian Macfarlane announced that a 54-year-old man with a chest-wall sarcoma received a customized 3-D printed titanium sternum and several ribs to replace diseased bone. The Melbourne-based company Anatomics used high-resolution CT scans to develop the implant, which maintained the contour of the patient’s chest and precisely met the ends of remaining bone. The patient was discharged 12 days after surgery.

September 10, 2015

  • The Albert and Mary Lasker Foundation announced the winners of the 2015 Lasker Awards, two of which will go to cancer researchers: Evelyn M. Witkin, PhD, of Rutgers University, New Brunswick, NJ, and Stephen J. Elledge, PhD, of Brigham and Women’s Hospital in Boston, MA, will share the Albert Lasker Basic Medical Research Award for their discoveries concerning the DNA-damage response. James P. Allison, PhD, of The University of Texas MD Anderson Cancer Center in Houston will receive the Lasker-DeBakey Clinical Medical Research Award for discovering and developing the anti–CTLA-4 therapy ipilimumab (Yervoy; Bristol-Myers Squibb). The awards, which carry an honorarium of $250,000 for each category, will be presented on September 18.
  • The International Association for the Study of Lung Cancer issued a new statement on tobacco control and smoking cessation at the 16th World Conference on Lung Cancer in Denver, CO. The statement calls for higher taxes on tobacco products, comprehensive advertising and promotion bans on all tobacco products, and mandating graphic warning labels on cigarette packaging. The complete declaration is available at www.iaslc.org.
  • Also at the World Conference on Lung Cancer, Bristol-Myers Squibb presented longer-term survival data from two trials evaluating nivolumab (Opdivo) in previously treated patients with squamous non–small cell lung cancer. In both trials, the drug showed an estimated 18-month overall survival rate of 27% to 28%. The safety profile was consistent with previously reported trials.
  • In addition, Bristol-Myers Squibb said that combining the PD-1 inhibitor nivolumab with relatively low doses of its other immunotherapy, the CTLA-4 inhibitor ipilimumab, proved safe and effective in a phase I trial. About 39% of patients taking nivolumab plus ipilimumab experienced some tumor reduction, compared with 31% of patients who took ipilimumab alone.
  • NHS England announced that it will stop paying for 17 more cancer drugs, agents that had been covered through the nation’s Cancer Drugs Fund, beginning November 4. The government launched the fund in 2011 to help patients pay for new treatments that have been rejected for general use because they are so expensive and because most extend lives by only a few weeks. In March, NHS England cut 16 other cancer drugs for 35 different indications due to cost concerns, although patients currently using the medications can continue to receive them.
  • The FDA granted Priority Review to Janssen’s daratumumab as a treatment for patients with multiple myeloma who are refractory to both a proteasome inhibitor and an immunomodulatory agent. An investigational human anti-CD38 monoclonal antibody, daratumumab received Breakthrough Therapy designation for this patient population from the FDA in May 2013. The agency is expected to issue a decision by March 9, 2016.
  • Young people who smoke electronic cigarettes (e-cigarettes) are considerably more likely to start smoking traditional cigarettes within a year than their peers who do not use the devices, according to a study published in JAMA Pediatrics. Researchers analyzed survey data from nearly 700 nonsmokers ages 16 to 26 in 2012 and again a year later. During that time, 38% of e-cigarette users at baseline started smoking traditional cigarettes, compared with only 10% of those where were not e-cigarette users at baseline.

September 3, 2015

  • The FDA ordered three tobacco manufacturers to stop describing their products as “additive-free” and/or “natural,” claims that could lead consumers to believe that the products aren’t as harmful as traditional cigarettes. The action marks the first time that the FDA has used its authority under a 2009 law to pursue regulatory action against companies using such claims on product packaging without first submitting scientific evidence to support the claims. The companies are ITG Brands, which sells Winston cigarettes; Santa Fe Natural Tobacco Company, which sells Natural American Spirit cigarettes; and Sherman’s 1400 Broadway N.Y.C., which sells Nat Sherman cigarettes.
  • The Centers for Disease Control and Prevention and the NCI released a report that includes federal statistics on cancer incidence and mortality. Titled “1999–2012 United States Cancer Statistics: Incidence and Mortality Web-Based Report,” the document shows that in 2012, 1,529,078 Americans were diagnosed with cancer, and 582,607 Americans died of the disease, not counting in situ cancers or cases of basal and squamous cell skin cancers.
  • The FDA granted Orphan Drug Designation to afatinib (Gilotrif; Boehringer Ingelheim) for the treatment of advanced squamous cell carcinoma of the lung that progresses after first-line chemotherapy. The drug is currently approved for the treatment of specific types of EGFR mutation–positive non–small cell lung cancer (NSCLC).
  • In addition, the FDA granted a 6-month Priority Review to MCNA (Telesta Therapeutics), a biologic therapy designed to treat patients with high-risk non–muscle invasive bladder cancer who are refractory to or relapsing from first-line therapy. Derived from nonpathogenic bacteria, MCNA is believed to directly affect the cancer as well as stimulate the immune system.
  • In a bipartisan effort, three U.S. representatives launched the Lung Cancer Caucus, which will provide information to Congress on a variety of lung cancer topics. These include eliminating the stigma associated with its diagnosis, the state of lung cancer research, screening and treatment to improve survival, and the impact of the disease on women, minority communities, and members of the military.
  • Novartis announced that the European Commission has approved the combination of dabrafenib (Tafinlar) and trametinib (Mekinist) for the treatment of adults with unresectable or metastatic melanoma with a BRAF V600 mutation. The approval was based on two phase III trials of the combination therapy that demonstrated significantly improved overall survival compared with the current standard of care—BRAF inhibitor monotherapy.
  • Due to a sagging economy and growing debt, the government of Brazil has cut the 2015 budget of the Ministry of Science by 25%, Science reported. The budget of the Ministry of Education, which helps fund graduate students, was cut by 9%. In response, research organizations have delayed grant payments and have canceled or delayed new requests for proposals for scientific research.
  • The American Society of Clinical Oncology (ASCO) issued an updated policy statement on genetic and genomic testing for cancer susceptibility. Published in the Journal of Clinical Oncology, the statement explains how new technologies, such as next-generation sequencing, are transforming the assessment and identification of inherited cancer susceptibility, and makes recommendations for using the technologies.
  • ASCO also updated its guideline on systemic therapy for patients with stage IV NSCLC. The guideline, also published in the Journal of Clinical Oncology, provides evidence-based recommendations on the use of chemotherapy and targeted therapy in first-, second-, and third-line treatment.

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August 2015

August 27, 2015

  • The FDA released draft guidance for industry on the nonproprietary naming of biologic products, including biosimilars—a biologic product with only minor differences in clinically inactive components compared with a reference product. The proposed naming convention, which would apply to new and previously licensed agents, would include the nonproprietary name followed by a unique four-letter suffix to distinguish the products. The guidance document is available at www.fda.gov; comments can be submitted within 60 days to www.regulations.gov.
  • A federal judge in Indianapolis, IN, ruled that patents on Eli Lilly’s chemotherapy drug Alimta (pemetrexed) remain in force, blocking generic versions developed by Teva Pharmaceuticals and APP Pharmaceuticals from entering the U.S. market until 2022, Bloomberg News reported. The drug interferes with cells’ ability to use folic acid to fuel their growth and survival. It is approved to treat non–small cell lung cancer and mesothelioma.
  • However, generic-drug makers won a patent case against Takeda Pharmaceutical in U.S. District Court, District of Delaware. The judge declared a patent on Velcade (bortezomib) invalid, agreeing with drug makers who argued that the formulation of the drug is “the inherent result of an obvious process,” according to Bloomberg Business. The ruling means that proposed generic versions of Velcade from Allergan, Norvartis, and Accord Healthcare could hit the U.S. market after another patent expires in 2017.
  • In a deal with GlaxoSmithKline (GSK) that could be worth up to $1 billion, Novartis bought the rights to develop and commercialize the cancer drug ofatumumab (Arzerra) for the treatment of multiple sclerosis and any other medical conditions. Earlier this year, Novartis acquired the rights from GSK to sell the CD20-directed monoclonal antibody for the treatment of chronic lymphocytic leukemia.
  • The FDA granted Orphan Drug Designation to two experimental cancer therapies: MTG-201 (MTG Biotherapeutics), a biologic therapy that targets Dickkopf-3 defects, for the treatment of malignant mesothelioma; and Toca 511 & Toca FC (Tocagen), designed to program cancer cells to convert the prodrug F-FC into the anticancer drug 5-FA and activate the immune system, for the treatment of glioblastoma.
  • In addition, the FDA granted Breakthrough Therapy Designation to cabozantinib (Cometriq; Exelixis) for the treatment of patients with advanced renal cell carcinoma who have received at least one prior therapy. Already approved for the treatment of medullary thyroid cancer, cabozantinib inhibits MET, VEGF receptors, AXL, and RET, receptor tyrosine kinases involved in oncogenesis, metastasis, and tumor angiogenesis.
  • Medivation purchased all rights to the PARP inhibitor talazoparib (BMN 673) from BioMarin Pharmaceutical in a deal valued at up to $570 million. Talazoparib is currently being assessed in a phase III trial in patients with deleterious germline BRCA1 and BRCA2 mutations and locally advanced or metastatic breast cancer.
  • Regulators have suspended the enrollment of new patients in a phase III trial testing Northwest Biotherapeutics’ brain tumor vaccine DCVax-L. The company said it must “submit certain information from the trial for regulatory review” but did not disclose what prompted suspension of enrollment or offer specifics on the information that was requested. Patients currently enrolled in the trial can continue treatment.
  • In spite of protests from parents, seventh graders in Rhode Island will be required to be vaccinated against human papillomavirus (HPV) when school starts in September. Currently, only Virginia and Washington, DC, require students to be vaccinated against HPV, which can cause cervical cancer and other malignancies.

August 13, 2015

  • A proposed rule in Massachusetts would limit prices on drugs, especially very high-priced agents such as newer, targeted therapies for cancer. In addition, the law would “force biotechnology and pharmaceutical companies to justify their prices by disclosing how much they spend on research, production, and marketing,” The Boston Globe reported.
  • The Texas Tribune reported that Republican lawmakers added a provision to the state budget to prohibit clinics with ties to abortion providers from receiving taxpayer money to pay for cancer screenings, a move that would cut off state funding for Planned Parenthood. The paper reported that the organization received $1.2 million in fiscal year 2014, which covered 910 clinical breast exams, 278 mammograms, and 1,854 Pap tests. The new budget takes effect on September 1.
  • AbbVie announced that a phase II trial of venetoclax met its primary endpoint of overall response rate in patients with relapsed or refractory or previously untreated chronic lymphocytic leukemia with 17p deletion. The study evaluated the safety and efficacy of venetoclax, an inhibitor of the B-cell lymphoma-2 protein in development Roche and Genentech. Data from the trial, which will be submitted to the FDA as part of the drug-approval process, will be presented at an upcoming medical conference.
  • The NIH launched a website to provide additional information about its Genomic Data Sharing policy, which went into effect in January. The policy applies to all NIH-funded research that generates large-scale human or nonhuman genomic data, regardless of funding level, as well as to the use of these data for subsequent research. To access the site, visit https://gds.nih.gov.
  • AstraZeneca bought the rights to Inovio’s experimental immunotherapy INO-3112, which targets cancers caused by human papillomavirus types 16 and 18, for $27.5 million up front. Depending on the success of the drug, Inovio could receive up to $700 million more, Reuters reported. INO-3112, designed to generate a tumor-specific T-cell response, is in phase I/II clinical trials for cervical cancer and head and neck cancers.
  • Janssen Biotech announced an agreement with Sweden’s Alligator Bioscience in which it obtains the rights to develop and commercialize ADC-1013, an agonistic, fully human monoclonal antibody; financial terms were not disclosed. ADC-1013 targets CD40, an immune-stimulatory receptor found on antigen-presenting cells, leading to an increase in T cells to attack a tumor. Alligator Bioscience will complete the current phase I study, and Janssen will then handle all subsequent development work.
  • An NCI-designated cancer center since 2005, the University of New Mexico Cancer Center in Albuquerque received the NCI’s Comprehensive Cancer Center designation, identifying it as one of the best in the nation. Facilities carrying this designation must have multidisciplinary teams of specialists who deliver the highest-quality diagnosis and treatment of cancer. They must also provide access to new treatments through clinical trials, conduct world-class research, and educate and train cancer researchers and health care providers.

August 6, 2015

  • According to a report on a phase III trial published in The New England Journal of Medicine, patients with metastatic prostate cancer who underwent six cycles of docetaxel chemotherapy along with a hormone blocker survived for a median of 57.6 months, compared with a median of 44 months for those who received only the hormone blocker. The results will likely change standard practice, which has been to withhold chemotherapy until hormone blockers become ineffective.
  • The oral targeted CSF-1R inhibitor PLX3397 induced prolonged tumor regressions in 52% of patients with tenosynovial giant cell tumor, a rare, locally aggressive neoplasm of the joint or tendon sheath, according to Daiichi Sankyo and Plexxikon. In addition, 7 of the 23 patients (30%) in the phase I study had stable disease following treatment with the compound, now being evaluated in a phase III trial. The results were published in The New England Journal of Medicine.
  • The European Commission designated SGX301 as an orphan drug for the treatment of cutaneous T-cell lymphoma, Soligenix announced. SGX301 (synthetic hypericin) is a novel, first-in-class photodynamic therapy activated by visible light instead of exposure to ultraviolet light, which can cause secondary malignancies. The compound previously received both Orphan Drug and Fast Track designations from the FDA for treatment of the disease.
  • NanoSmart Pharmaceuticals announced that the FDA granted Orphan Drug Designation to a novel formulation of dactinomycin for the treatment of Ewing sarcoma. This new formulation of an existing cancer drug incorporates dactinomycin into a proprietary ANA-conjugated nanoemulsion. It is expected to deliver the drug to the tumor, increasing its effectiveness while limiting damage to healthy tissue.
  • Bristol-Myers Squibb announced the establishment of its Immuno-Oncology Rare Population Malignancy Program in the United States. The program is a multi-institutional initiative with academic-based cancer centers to investigate immunotherapies for the treatment of high-risk, poor-prognosis cancers, defined as a rare population malignancy. A rare population malignancy is a subpopulation within a higher-incident disease population, such as BRCA 1 and BRCA2 breast cancers.
  • Boehringer Ingelheim agreed to pay Hanmi Pharmaceuticals $50 million up front for the rights to HM61713, an oral EGFR inhibitor, which has shown promise in early-stage trials in non–small cell lung cancer (NSCLC). Hanmi can collect up to $680 million more if the drug meets certain clinical and commercial milestones. HM61713 is being assessed in a phase II trial in patients with T790M-mutated NSCLC who have developed resistance to other EGFR inhibitors.
  • In a bipartisan effort, seven congressional representatives introduced a bill to establish the nation’s first registry to track patients with mesothelioma. The registry would allow researchers, health care professionals, and patients to search information about diagnosis, disease trends, risk factors, treatment availability, and outcomes.
  • The United Kingdom’s National Institute for Health and Care Excellence (NICE) once again refused to pay for the PARP inhibitor olaparib (Lynparza; AstraZeneca) for women with ovarian cancer until after they have had at least three rounds of chemotherapy. NICE said that the cost of the drug could not be justified because evidence that it extends survival remains uncertain.
  • Aiming to reduce variations in clinical practice, NICE published its first guidelines for the treatment of melanoma. Available at www.nice.org.uk/guidance/ng14, the guidelines call for the use of dermoscopy or other techniques to assess skin lesions, make recommendations about the use of sentinel lymph node biopsy, and prescribe imaging of the brain for patients with stage 4 disease, along with other advice.

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July 2015

July 30, 2015

  • Sanofi said it will make an upfront payment of $640 million to Regeneron and that the companies will invest $1 billion in the discovery and development of a monotherapy and novel combinations of immuno-oncology antibody candidates. The deal includes the development of REGN2810, a PD-1 inhibitor currently in phase I testing.
  • Pennsylvania’s University of Pittsburgh Cancer Institute (UPCI) received a 5-year $25.6 million grant from the NCI in conjunction with its renewal as an NCI-designated comprehensive cancer center. UPCI has held the NCI distinction since 1990, when it was first named a comprehensive cancer center.
  • Two companies announced that they received the FDA’s Breakthrough Therapy designation for a drug: Eisai Inc. said that the distinction was granted to lenvatinib (Lenvima) for the treatment of patients with advanced or metastatic renal cell carcinoma who previously received VEGF-targeted therapy. Progenics Pharmaceuticals said that the label was given to Ultratrace iobenguane I 131 (Azedra) for the treatment of patients with metastatic or recurrent pheochromocytoma and paraganglioma. The designation expedites the development and FDA review of drugs.
  • Boston, MA–based Ziopharm Oncology said that the FDA granted Orphan Drug designation to Ad-RTS-hIL-12 plus veledimex (INXN-1001), an activator ligand, for the treatment of malignant gliomas. Ad-RTS-hIL-12 is a novel gene therapy candidate for the controlled expression of IL-12, a critical protein for stimulating an anticancer T-cell immune response. The designation supports the development of agents for rare disorders, speeds FDA review, and provides tax credits and extended market exclusivity.
  • Genzyme said it will pay AstraZeneca up to $300 million to obtain global rights to sell and further develop vandetanib (Caprelsa), currently approved for the treatment of aggressive, symptomatic medullary thyroid carcinoma.
  • The FDA approved carfilzomib (Kyprolis; Onyx Pharmaceuticals), in combination with lenalidomide and dexamethasone for the treatment of patients with relapsed multiple myeloma who have received one to three prior lines of therapy. The approval was based on improved progression-free survival among patients who received the three-drug combination compared with those who received a two-drug combination of lenalidomide and dexamethasone (26.3 vs. 17.6 months, respectively). The drug was previously approved as a monotherapy for a subset of patients with multiple myeloma.
  • The FDA granted Priority Review to the combination of dabrafenib (Tafinlar) and trametinib (Mekinist) for the treatment of adults with inoperable or metastatic melanoma with a BRAF V600 mutation. Already approved as monotherapies, the combination of the Novartis drugs has been shown to slow tumor growth more effectively than either drug alone.
  • A study published in JAMA Internal Medicine found that about 10% of serious and unexpected complications are not reported to the FDA by drug manufacturers within 15 days, as directed by federal regulations, a delay that may compromise the safety of other patients. Researchers analyzed 1.6 million reports of side effects filed with the FDA from 2004 to 2014.

July 23, 2015

  • The U.S. Court of Appeals for the Federal Circuit ruled that, under federal law, Sandoz must wait 6 months to market Zarxio, its version of the biologic drug Neupogen (filgrastim; Amgen), following FDA approval. That means that an injunction imposed on the marketing of Zarxio will remain in effect until September 2. Amgen filed a lawsuit against Sandoz last fall claiming that the biosimilar maker had infringed on its Neupogen patents.
  • Although few details were provided, Amgen announced positive results for its phase II, single-arm trial of blinatumomab (Blincyto) in adults with relapsed or refractory Philadelphia chromosome–positive B-cell precursor acute lymphoblastic leukemia (ALL). The company said that the drug, used as monotherapy, induced complete remission or complete remission with partial hematologic recovery within two cycles of treatment “in a clinically meaningful number of patients.” The FDA approved blinatumomab for Philadelphia chromosome–negative B-cell precursor ALL in December 2014.
  • Results from a phase III trial show that panitumumab (Vectibix) improves overall survival (OS) in chemorefractory wild-type KRAS (exon 2) metastatic colorectal cancer (mCRC) versus best supportive care, Amgen also announced. In addition, treatment with panitumumab showed statistical significance for key secondary endpoints, including OS in patients with wild-type RAS (absence of mutations in exons 2, 3, and 4 of KRAS and NRAS) mCRC. Final results will be presented at a future medical conference.
  • The European Commission (EC) approved the PD-1 inhibitor pembrolizumab (Keytruda; Merck) in previously treated and newly diagnosed patients with advanced melanoma. The approval, which covers all 28 European Union nations, was based on phase III data showing that the immunotherapeutic provided a statistically superior survival benefit compared with ipilimumab (Yervoy; Bristol-Myers Squibb), the current standard of care for the disease.
  • The EC also approved the PD-1 inhibitor nivolumab (Opdivo; Bristol-Myers Squibb) for the treatment of locally advanced or metastatic squamous non–small cell lung cancer in patients who have already received chemotherapy. The approval was based on a phase III trial in which median OS was 9.2 months for nivolumab compared with 6 months for docetaxel chemotherapy.
  • The FDA granted Orphan Drug Designation to two agents—Delcath Systems’ melphalan chemotherapy for the treatment of cholangiocarcinoma, and Novogen Limited’s anti-tropomyosin chemotherapy Anisina for the treatment of neuroblastoma. The designation confers benefits such as extended marketing exclusivity and tax credits to companies that develop drugs for orphan diseases, defined as those that affect fewer than 200,000 people in the United States.
  • The American Society of Clinical Oncology (ASCO) issued recommendations calling upon federal agencies and the cancer research community to broaden clinical trials to include older adults and redefine eligibility for clinical trials to better understand how to treat this patient population. For example, ASCO suggests increasing the FDA’s authority to provide incentives or require research on older adults with cancer. The recommendations were published in the Journal of Clinical Oncology.
  • The American College of Medical Genetics and Genomics issued a list of five recommendations for health care providers to consider prior to ordering specific genetic tests, such as not ordering exome or genome sequencing before obtaining informed consent that includes the possibility of secondary findings. The evidence-based recommendations are designed to encourage patients and clinicians to have thorough conversations about choices related to genetic testing. The list is available at www.acmg.net.

July 16, 2015

  • The U.S. House of Representatives passed the 21st Century Cures Act, legislation aimed at speeding the development of new medicines and medical devices. The bill calls for new funding for the NIH that would total $8.75 billion over 5 years, in addition to its annual appropriation, to establish an innovation fund for the study of biomarkers and precision medicine, as well as other research. It also authorizes an additional $550 million over 5 years for the FDA. The bill now heads to the Senate.
  • An amendment to the 21st Century Cures Act noted that "unique device identifiers" should be included in patients' electronic health records, so that any adverse effects related to particular brands or models of devices can be tracked. The amendment was inspired by laparoscopic power morcellators, tools that cut up uterine fibroids so that the tissue can be removed through a small incision, which have been found to spread undiagnosed uterine cancers.
  • Science reported that the only private funder of scientific research in Russia, the Dynasty Foundation, will shut down. In May, the foundation was told that it must register as a "foreign agent," a designation conferred upon organizations that accept funding from the West. Fined about $5,000 for refusing to do so, Dynasty’s founder, Dmitry Zimin, to decide to close.
  • Roche announced that the investigational anti–PD-L1 immunotherapeutic atezolizumab (MPDL3280A) shrank tumors in people with locally advanced or metastatic urothelial bladder cancer whose disease had progressed on initial therapy. In addition, high levels of PD-L1 expression correlated with increased response to the medicine. Results of the phase II study will be presented at an upcoming medical meeting.
  • GlaxoSmithKline announced that it will collaborate with the United Kingdom’s Francis Crick Institute, a biomedical research facility scheduled to open in London at the end of the year, to better understand the underlying biology of disease. The partners plan to focus initially on HIV, malaria, and cancer.
  • San Diego, CA's Tocagen Inc. announced that the FDA granted Fast Track designation it its lead immuno-oncology product, Toca 511 & Toca FC, for the treatment of recurrent high-grade gliomas. Toca 511 selectively delivers a gene for the enzyme cytosine deaminase to cancer cells, while Toca FC, a novel antifungal drug, is converted into the chemotherapy drug 5-fluorouracil. As a result, infected cancer cells are killed, prompting the immune system to recognize cancer cells while leaving healthy cells alone.
  • Based on an informal poll, most members of an FDA advisory committee indicated that they would support the approval of Eli Lilly's necitumumab for the treatment of patients with advanced squamous non–small cell lung cancer, despite its small survival benefit: In a clinical trial of 1,093 patients, those who received the drug with chemotherapy survived an average of 11.5 months compared with 9.9 months for those who received chemotherapy alone. Necitumumab is designed to block the ligand binding site of EGFR.
  • According to Nature, a California bill that would have regulated e-cigarettes like other tobacco products was withdrawn from consideration after it was changed it so significantly that the state senator who originally pushed it “said it had become pointless.” The bill would’ve made it illegal to use the devices in restaurants, bars, hospitals, and other workplaces.
  • Only Washington, DC, and two states—Rhode Island and Virginia—will require teens and preteens to have the vaccine against human papillomavirus (HPV) come August. A study published in The Journal of the American Medical Association notes that most, if not all, states require children to have other vaccinations, including those against chickenpox (all states), meningitis (29 states and DC), and hepatitis B (47 states and DC), which, like HPV, is sexually transmitted.

July 9, 2015

  • Scientists in Greece are worrying about the fate of their research, which has received funding from the European Union, after voters soundly rejected the terms of a financial bailout from the EU. They fear that dropping the euro and returning to the drachma would worsen the financial situation for many labs—and that it would mean leaving the EU, further compromising research funding. In addition, Nature reported that the internet portal that provides scientists with access to electronic journals has suspended service because the Greek government can't pay for it.
  • Seattle, WA–based Blaze Bioscience announced that it has received the FDA's Orphan Drug Designation for BLZ-100 to treat malignant brain tumors. BLZ-100 contains an optimized peptide that binds to and is internalized by cancer cells, and a fluorescent dye that emits light in the near-infrared range. This so-called "tumor paint" provides real-time, high-resolution intraoperative images of cancer cells, enabling more precise surgical removal of tumors.
  • Thanks to a philanthropic commitment of $25 million from Stanley and Fiona Druckenmiller, New York, NY's Memorial Sloan Kettering Cancer Center has created a new lung cancer research center dedicated to developing innovative and more-effective treatments for people with the disease.
  • Proton Partners International Limited began work to build the United Kingdom's first proton beam cancer treatment center, which will be located in Newport. The company plans to build two other centers in the UK—one in Northumberland and the other in London. A type of radiotherapy, proton beam therapy can target hard-to-reach cancers, such as spinal tumors, with a lower risk of damaging surrounding tissue.
  • Summit, NJ–based Celgene said it will take over the nearby biopharma campus formerly occupied by Merck. The campus includes 450,000 square feet of R&D space and 850,000 square feet of administrative space, FierceBiotech reported.
  • According to a survey of more than 59,000 adults, warning messages about the increased risk of skin cancer are working: The percentage of adults who engage in indoor tanning dropped from 5.5% in 2010 to slightly more than 4% in 2013. The findings were published in JAM Dermatology.
  • The American Society of Human Genetics issued a position statement on genetic testing in children and adolescents that discussed ethical and other issues. The statement says that genetic testing should be limited to a single-gene analysis or targeted gene panels in this population. In addition, it says that testing for adult-onset conditions should be avoided unless a childhood treatment exists, and that secondary findings should be disclosed only when there is "clear clinical utility."
  • According to a study from the Centers for Disease Control and Prevention published in the American Journal of Preventive Medicine, three out of four adults—and seven in 10 cigarette smokers—favor raising the minimum age to purchase tobacco products to 21 nationwide. In most states, the minimum age is 18. Currently, Hawaii is the only state to have adopted a law prohibiting the sale of tobacco products to anyone under age 21.
  • Although most of the United States has experienced large declines in colorectal cancer death rates in recent years, progress has lagged in three "hot spots": 94 counties in the lower Mississippi Delta, 107 counties in west central Appalachia, and 37 counties in eastern Virginia/North Carolina. According to a study published in Cancer Epidemiology, Biomarkers & Prevention, colorectal cancer death rates were 40% higher in the lower Mississippi Delta from 2009 to 2011 than in other areas of the country. Rates in west central Appalachia and eastern Virginia/North Carolina were 18% and 9% higher, respectively.
  • The Wall Street Journal reported that Lina Medical agreed to settle a lawsuit for an undisclosed sum over the death of a woman who allegedly had undiagnosed leiomyosarcoma spread throughout her body by a power morcellator manufactured by the company. Such devices cut up the uterus or uterine fibroids so that the tissue can be removed through a small incision, but their use has nearly come to a halt since the FDA issued a warning about the risk of spreading an undiagnosed cancer. About two dozen similar suits have been filed.

July 2, 2015

  • The Summit, NJ–based biotech company Celgene announced that it will pay about $1 billion to collaborate with Juno Therapeutics of Seattle, WA, on the development of treatments for patients with cancer and autoimmune diseases, with an initial focus on chimeric antigen receptor T-cell (CAR-T) therapies. Under the terms of the 10-year collaboration, Celgene has the option to be the commercialization partner for some of Juno's immunotherapy candidates, including its CD19- and CD22- directed CAR-T products.
  • Thanks to a fundraising frenzy, the Oregon Health and Science University's Knight Cancer Institute will receive $1 billion for cancer research. In 2013, Phil Knight, the CEO and cofounder of Nike, pledged to give $500 million to the center on the condition that the organization raise $500 million more within 2 years. Knight and Brian Druker, MD, the cancer institute's director, made the announcement on Good Morning America.
  • Bristol-Myers Squibb announced that it will open a new state-of-the-art research facility in Cambridge, MA, in 2018. Researchers there will focus on the company's ongoing discovery efforts in genetically defined disease and molecular discovery techniques. The company's Waltham, MA, site will close and employees there will move to Cambridge; additional scientists in the Cambridge area will be recruited.
  • According to statistics from Cancer Research UK, the number of cases of lung cancer in women has risen to 20,000 a year in the United Kingdom for the first time. Despite falling smoking rates, lung cancer rates have increased by 22%, from about 14,200 cases two decades ago, the organization said.
  • After being cancelled at the end of 2014 due to serious design flaws, $1 billion in expenses, and trouble recruiting 100,000 pregnant women whose babies would be followed until age 21, the NIH National Children's Study (NCS) may be resurrected, although in a different form. Appropriations committees in both the U.S. House and Senate approved spending bills for the 2016 fiscal year that include $165 million to form the National Children's Study Alternative. A report accompanying the House version of the bill directs the NIH "to work with pediatric groups to develop a series of alternative research activities that build on NCS data… to address the developmental origins of health and disease."
  • The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) recommended that two cancer drugs receive approval from the European Commission. Roche said the committee recommended the use of pertuzumab (Perjeta) in combination in trastuzumab (Herceptin; Genentech) and chemotherapy in women with HER2-positive, locally advanced, inflammatory, or early-stage breast cancer at high risk of recurrence. Novartis said that the committee also recommended approval of panobinostat (Farydak), a histone deacetylase inhibitor, for the treatment of adults with relapsed and/or refractory multiple myeloma.
  • The UK's Medicines and Healthcare Products Regulatory Agency approved nivolumab (Opdivo) for the treatment of locally advanced or metastatic squamous non–small cell lung cancer (NSCLC) after prior chemotherapy. The PD-1 checkpoint inhibitor was approved under the UK's Early Access to Medicine Scheme, designed to speed patients' access to life-saving drugs. The CHMP recommended approval of nivolumab for second-line treatment of NSCLC in May, but it has not yet given final approval.

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June 2015

June 25, 2015

  • Noted San Diego developer and philanthropist Conrad Prebys will donate $100 million to the Sanford Burnham Medical Research Institute in nearby La Jolla, CA, the organization announced. The gift will allow investigators to advance translational research, further laboratory discoveries, and move additional drug candidates through its pipeline. In recognition of Prebys's contribution, the Institute has changed its name to the Sanford Burnham Prebys Medical Discovery Institute.
  • Bristol-Myers Squibb announced that the European Commission approved nivolumab (Opdivo) for the treatment of advanced, inoperable melanoma in adults regardless of BRAF status. The approval allows the marketing of the PD-1 inhibitor in all 28 European Union member states. Nivolumab was approved by the FDA in December 2014 for the treatment of patients with metastatic, inoperable melanoma and disease progression following treatment with ipilimumab (Yervoy) and, if BRAF V600 mutation–positive, a BRAF inhibitor.
  • The NIH submitted interim corrective action plans to the FDA to address problems with sterile manufacturing and other deficiencies in its pharmacy that were announced earlier this month. As part of the plan, the NIH will hire a firm experienced in Current Good Manufacturing Practice regulations and procedures to evaluate the Pharmaceutical Development Section, make recommendations for improvement, and address all FDA concerns. The problems halted the manufacture of several agents used in clinical trials, including some cancer drugs.
  • Cambridge, MA–based Merrimack Pharmaceuticals and Baxalta Inc. of Deerfield, IL, announced that the FDA granted Priority Review status to MM-398 (irinotecan liposome injection) for the treatment of patients with metastatic adenocarcinoma of the pancreas who have previously been treated with gemcitabine-based therapy. In addition, the European Medicines Agency accepted an application for approval of the drug, marking the beginning of the review process for the drug in the European Union.
  • Hawaii's governor signed a bill raising the legal smoking age to 21 statewide, making Hawaii the first state to do so. The law will also ban the sale, purchase, and use of electronic cigarettes by anyone under 21. The law will take effect on January 1, 2016.
  • The Wall Street Journal reported that two U.S. lawmakers have introduced legislation that would prevent drugmakers from stalling efforts of other companies to produce generic rivals. In the last few years, generic drugmakers have said that they cannot access the brand-name drugs they need to conduct tests of their generic products—tests that are required for FDA approval. A trade group for the industry estimates that such delays cost the U.S. health care system $5.4 billion annually.
  • Danbury, CT–based IMS Health and Foundation Medicine of Cambridge, MA, announced that they will collaborate to advance the understanding and effectiveness of precision medicines for treating cancer. IMS Health, a global information and technology services company that works with the health care industry, will provide data on clinical outcomes and the cost of care, while Foundation Medicine will provide anonymized genomic profiles from patients. Together, they aim to "improve cancer care by better connecting patients to the right targeted therapies at the right time," the companies said.
  • In its Morbidity and Mortality Weekly Report, the U.S. Centers for Disease Control and Prevention (CDC) revealed that states "underinvested in their tobacco prevention and control programs in fiscal year 2011, spending just '658 million," or just 17.8% of the CDC's recommended spending amount. States that make larger investments in tobacco prevention and control programs have recorded larger declines in cigarette sales and the prevalence of smoking.

June 18, 2015

  • GlaxoSmithKline (GSK) announced that it will launch the Altius Institute for Biomedical Sciences, an independent, nonprofit research venture, in Seattle, WA. GSK and Altius—to be led by John Stamatoyannopoulos, MD, a professor of genome sciences and medicine at the University of Washington School of Medicine in Seattle, have signed a 10-year collaboration agreement; during the first 5 years, GSK will provide more than $95 million in cash and other resources to advance the institute’s work. Altius will pioneer new technologies and approaches for decoding how genes are controlled and how a cell’s “operating system” functions in health and disease in order to select and validate better drug targets.
  • Two companies announced that they each have received Orphan Drug Designation from the FDA for an investigational drug: Los Angeles, CA–based Lion Biotechnologies, which develops cancer immunotherapies based on tumor-infiltrating lymphocytes, received the designation for LN-144 for the treatment of stage 2b to stage 4 malignant melanoma. The status was also conferred upon imetelstat, a telomerase inhibitor discovered by Geron Corporation (Menlo Park, CA) and licensed to Janssen Biotech (Horsham, PA), for the treatment of myelofibrosis.
  • The Office of Cellular, Tissue, and Gene Therapies at the FDA issued a guidance document for industry to facilitate the development of early-phase clinical trials for cellular and gene therapy products. Given the distinctive nature of these products, the document notes, the design of early-phase trials “often differs from the design of clinical trials for other types of pharmaceutical products.” The document finalizes the draft guidance that was dated July 2013.
  • A member of the Roche Group, Ventana Medical Systems announced that the FDA approved its ALK assay as a companion diagnostic to aid in the identification of patients with non–small cell lung cancer likely to benefit from the targeted drug crizotinib (Xalkori; Pfizer).
  • Also at the EHA meeting, researchers reported that 84% of patients (41 of 49) with relapsed chronic lymphocytic leukemia experienced objective responses to the combination of rituximab (Rituxan; Genentech) and venetoclax (ABT-199; AbbVie) in a phase Ib trial, with three patients maintaining a response after a median of 12 months. Venetoclax is an investigational B-cell lymphoma 2 selective inhibitor.
  • Cambridge, MA–based Agios Pharmaceuticals announced new data from phase I trials of the IDH1 inhibitor AG-120 and the IHD2 inhibitor AG-221 in patients with advanced hematologic malignancies at the 20th Congress of the European Hematology Association (EHA) in Vienna, Austria. For AG-120, the study had an overall response rate (ORR) of 31% (16 of 52 evaluable patients) and a complete remission rate of 15%, with responding patients on treatment for up to 11 months. For AG-221, the study had an ORR of 40% (63 of 158 evaluable patients) and a complete remission rate of 16%, with responding patients on treatment for more than 15 months.
  • In a phase III trial involving 326 patients with relapsed acute lymphoblastic leukemia, the investigational antibody–drug conjugate inotuzumab–ozogamicin led to complete responses in 80% of patients compared with 33% of patients treated with standard care, usually chemotherapy. The findings were reported at the EHA meeting.
  • The FDA is teaming up with the online network PatientsLikeMe to better understand the side effects of 1,000 different drugs, including anticancer drugs, as reported by the site’s 350,000 members. Reporting adverse events to the FDA is mandatory for drug manufacturers, but voluntary for healthcare professionals and patients. Because the data are generated by patients, the information provides “real-time insights into the nuances inherent in patients’ experiences over time, including drug tolerance, adherence, and quality of life,” according to PatientsLikeMe.
  • According to a study published by JAMA Internal Medicine, 48.5% of the 345,962 deaths from 12 cancers among adults 35 and older in 2011 were attributable to cigarette smoking. The largest proportions of smoking-attributable deaths were for cancers of the lung, bronchus, and trachea (125,799; 80.2%) and larynx (2,856; 76.6%). About half of the deaths from cancers of the oral cavity, esophagus, and urinary bladder were attributable to cigarettes.

June 11, 2015

  • The NIH Clinical Center suspended production of sterile materials after an FDA inspection in May uncovered serious manufacturing problems and lack of compliance with standard operating procedures in its Pharmaceutical Development Section. The drug development unit makes agents for certain clinical studies conducted at the NIH hospital and other facilities, potentially affecting participants in 46 studies, including some examining treatments for cancer. In April, two vials of albumin, used for the administration of interleukin, were found to have fungal contamination.
  • According to a panel of 29 experts from 16 countries convened by the International Agency for Research on Cancer, mammography screening is effective in reducing breast cancer mortality for women ages 50–74, and the benefits of screening outweigh the risks in these women. However, the panel concluded that, for women in their 40s, evidence of reduced breast cancer mortality due to mammography is limited. Their special reporton breast cancer screening was published in The New England Journal of Medicine.
  • Bothell, WA–based Seattle Genetics announced that it will develop and commercialize novel antibody-coupled T-cell receptor (ACTR) therapies for cancer with Unum Therapeutics of Cambridge, MA. Unum’s ACTR technology enables the programming of a patient’s T cells to attack tumor cells when administered with tumor-specific therapeutic antibodies. Seattle Genetics has a large portfolio of cancer targets and tumor-specific monoclonal antibodies that could become part of the collaboration.
  • The Associated Press reported that 1,500 people took part in a rally in Moscow, Russia, to protest the country’s lack of funding for scientific research. The rally was sparked by a decision by Russian authorities to place the nongovernmental Dynasty Foundation on its list of “foreign agents” because it provides grants for research, which is deemed a political activity. Dmitry Zimin, whose personal wealth funds Dynasty, said the foundation will cease operations as a result.
  • Matched siblings are typically preferred over unrelated donors when it comes to stem cell transplants. However, a new study in the Journal of Clinical Oncology shows that older patients who received stem cells from younger, unrelated donors with higher numbers of CD8 cells had significantly reduced risk of disease relapse and improved survival compared with those who received stem cells from donors with low numbers of CD8 cells, including older matched siblings. The researchers suggest that screening for donor T-cell characteristics will optimize donor section and lead to more successful transplants in older patients.
  • New data released by Macmillan Cancer Support and Public Health England National Cancer Intelligence Network in the United Kingdom show that nearly 80,000 people in the UK who were diagnosed with breast, prostate, colorectal, or lung cancer before age 45 are still alive 20 years later. However, Macmillan’s Juliet Bouverie, director of Services and Influencing, says that the National Health Service (NHS) is not prepared to assist “a rapidly growing number of cancer survivors with the long-term impact of cancer and give them a decent quality of life in the years after their treatment ends.”
  • The National Institute for Health and Care Excellence (NICE), which advises the UK’s NHS on which treatments to fund, recommended that radium-223 dichloride (Xofigo; Bayer) be covered for patients with prostate cancer that has stopped responding to hormone therapy and metastasized. However, NICE recommended against covering degarelix depot (Firmagon; Ferring) to treat advanced, hormone-dependent prostate cancer, saying that it is not cost-effective compared with other available treatments.

June 4, 2015

  • The American Society of Clinical Oncology (ASCO) announced its first-ever clinical trial, which will offer patients with advanced cancer access to molecularly targeted drugs and collect data on their outcomes to better understand the best uses for the drugs outside of FDA-approved indications. Pharmaceutical companies will contribute targeted therapies to the prospective, non-randomized trial; they will be assessed in solid tumors, multiple myeloma, and non-Hodgkin lymphoma with a genomic variation known to be a drug target. For more information, visit www.asco.org/TAPUR.
  • Researchers said that IDH might be a prognostic marker for anaplastic astrocytoma, based on a study in which patients who had a mutated IDH gene lived an average of 7.9 years after diagnosis versus 2.8 years for patients with unaltered IDH. The study, reported at the ASCO Annual Meeting in Chicago, IL, involved 301 patients who received radiation therapy plus either temozolomide or nitrosourea. There was no difference in overall survival between the groups after 3.6 years, but patients with mutant IDH who received temozolomide tended to live longer.
  • Also at the ASCO meeting, researchers said that the anti-CD38 antibody daratumumab was effective in a phase II trial of patients with multiple myeloma whose disease had worsened after at least three prior treatments. After a median follow-up of 9.4 months, 29.2% of the study’s 106 patients responded to daratumumab, with three complete remissions; responses lasted 7.4 months on average. Overall, the median time to disease progression was 3.7 months, and the estimated overall survival rate at 1 year was 65%.
  • Regulators in the European Union have approved lenvatinib (Lenvima; Eisai) to treat the most common types of thyroid cancer. The approval was based on clinical data showing that patients who received the drug lived 14.7 months longer without disease progression than those who received a placebo. Lenvatinib was approved earlier this year in the United States and Japan.
  • In the U.S. Court of Appeals for the Federal Circuit, Amgen and Sandoz continued their legal battle over Sandoz’s right to sell the biosimilar filgrastim-sndz (Zarxio) for the treatment of neutropenia in cancer patients. Amgen is arguing that Sandoz, a unit of Novartis, did not follow FDA rules when it sought approval for its version of Amgen’s biologic drug Neupogen. Filgrastim-sndz was approved in March, but Sandoz has been prohibited from selling it until the court issues a ruling.
  • The FDA granted Orphan Drug Designation to APTO-253 for the treatment of acute myeloid leukemia (AML), according to the Aptose Biosciences of San Diego, CA, and Toronto, Canada. Epigenetic suppression of Krüppel-like factor 4 (KLF4) has been reported as a transforming event in AML; a first-in-class targeted inducer of the KLF4 tumor suppressor, APTO-253 has demonstrated a favorable safety profile with no evidence of normal bone marrow suppression. The drug is currently in a phase Ib trial in patients with relapsed or refractory hematologic malignancies.
  • According to the U.S. Centers for Disease Control and Prevention, the incidence of melanoma has doubled in the United States over the last three decades. The Centers’ researchers noted that there were 65,647 new cases of melanoma in 2011, or 19.7 new cases per 100,000 people; non-Latino whites had the highest incidence of the disease, with 24.6 cases per 100,000 people. The number of cases could rise to 112,000 by 2030 if the trend continues.

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May 2015

May 15–28, 2015

  • Cambridge, MA–based Cerulean Pharma announced that the FDA granted Orphan Drug Designation to CRLX101 for the treatment of relapsed ovarian cancer. CRLX101 is a nanoparticle–drug conjugate designed to concentrate in tumors and slowly release its anticancer payload, camptothecin, inside tumor cells. It inhibits topoisomerase 1 and hypoxia-inducible factor-1α.
  • The FDA also granted Orphan Drug Designation to Aura Biosciences' AU-011 for the treatment of uveal melanoma, also called ocular melanoma. There are currently no approved drug therapies for the disease. The Cambridge, MA, company's drug consists of viral-like nanoparticles that demonstrate highly selective targeting of solid tumors while leaving normal epithelium untouched.
  • Pfizer announced the launch of a competitive, peer-reviewed grants program to support clinical research projects investigating palbociclib (Ibrance) in advanced breast cancer. The multiyear program will award up to six grants totaling as much as $3 million to investigators in the United States. For specifics regarding eligible areas of research, visit www.aspireresearch.org.
  • Memorial Sloan Kettering Cancer Center announced that it has received a $150 million commitment from David H. Koch. The contribution, the largest in the institution's history, will help fund a 23-story, 750,000-square-foot outpatient medical building in New York, NY. The facility, which will bear Koch's name, is scheduled to open in 2019.
  • In a phase III trial, Amgen's viral immunotherapy vaccine talimogene laherparepvec, or T-VEC, showed significant benefit among patients with aggressive, inoperable malignant melanoma compared with a control immunotherapy. Of the patients who received T-VEC, 16.3% showed a durable treatment response of more than 6 months, compared with 2.1% given the control treatment. The findings were published in the Journal of Clinical Oncology.
  • The National Academy of Sciences and the National Academy of Medicine are launching an initiative to guide decision-making about research involving human gene editing and related technologies, such as CRISPR-Cas9. Researchers and other experts will convene this fall to explore issues associated with human gene–editing research, and an international committee will study the scientific underpinnings and clinical, ethical, legal, and social implications of human gene editing. The committee will then recommend standards, guidelines, and practices governing the use of gene-editing technologies in biomedical research and medicine.
  • Speaking at a meeting of the Friends of Cancer Research, Richard Pazdur, director of the Office of Hematology and Oncology Products at the FDA, questioned the value of lists of side effects of prescription drugs at the end of television advertisements. For example, Ɗ'Don’t use if you have MAE1,'" Pazdur said. "What patient knows what MAE1 is? It's just a waste of time." The FDA has been studying whether the ads may lead viewers to miss the most dangerous aspects of a medicine, Bloomberg Business reported.

May 14, 2015

  • According to the U.S. Centers for Disease Control and Prevention, screening rates for three cancers either dropped or showed no improvement in 2013, the latest year for which data are available. The report concludes that about 20% of women are not up to date with cervical cancer screening, about 25% of women are not up to date with breast cancer screening, and about 40% of adults are not up to date with colorectal cancer screening.
  • Roche announced that the FDA has approved the cobas KRAS Mutation Test for diagnostic use. The real-time PCR test is designed to identify KRAS mutations in tumor samples from patients with metastatic colorectal cancer and help oncologists determine an appropriate therapy. Treatment with cetuximab (Erbitux; Lilly and Bristol-Myers Squibb) or panitumumab (Vectibix; Amgen) may be effective if no KRAS mutation is present.
  • The World Health Organization (WHO) added 16 cancer treatments to its so-called list of essential medicines, including imatinib (Gleevec; Novartis) and trastuzumab (Herceptin; Roche/Genentech). Because these medicines could have a tremendous impact on public health globally, the WHO wants drug manufacturers and governments to ensure that the medicines are both available and affordable.
  • Threshold Pharmaceuticals announced that the FDA granted Fast Track designation to evofosfamide (TH-302), in combination with gemcitabine, for the treatment of previously untreated patients with metastatic or locally advanced, inoperable pancreatic cancer. Last year, the drug earned the same designation for the treatment of advanced soft-tissue sarcoma. Headquartered in South San Francisco, CA, Threshold and Merck KGaA (Darmstadt, Germany) are collaborating on the development of evofosfamide, which is under evaluation in two phase III trials.
  • Swiss drugmaker Novartis said that the European Commission approved ceritinib (Zykadia) to treat adults with ALK-positive non–small cell lung cancer previously treated with crizotinib (Xalkori; Pfizer). Outside the European Union, the drug is approved for this indication in 10 countries, including the United States.
  • IBM announced that it is collaborating with more than a dozen cancer institutes to accelerate oncologists' ability to identify and personalize treatment options for their patients. The institutes will use IBM's Watson to sift through individual patients' genetic profiles and electronic medical records, journal studies, and clinical trial information to find the best possible treatment options for each one. Currently, clinicians can spend weeks combing through such data, but Watson can do it in a few minutes, the company says.
  • The FDA granted Priority Review status to Novocure's Optune, in combination with temozolomide chemotherapy, as a first-line treatment for glioblastoma. According to the St. Helier, Jersey, company, Optune uses its Tumor Treating Fields technology to create low-intensity, alternating electric fields within a tumor that exert physical forces on electrically charged cellular components, preventing mitosis and causing apoptosis. In an interim analysis of data from a phase III trial, patients treated with the device and temozolomide had a significant reduction in the risk of disease progression or death compared with temozolomide alone (median progression-free survival of 7.1 months vs. 4.0 months).
  • The FDA denied a petition from R.J. Reynolds Tobacco Company and American Snuff Company to tone down language in one of four required smokeless tobacco warning statements. The companies asked to change "WARNING: This product is not a safe alternative to cigarettes" to "WARNING: No tobacco product is safe, but this product presents substantially lower risks to health than cigarettes" or, barring that, to "WARNING: No tobacco product is safe; however, exclusive use of smokeless tobacco products presents substantially less risk to health than cigarettes." The FDA said that there was "insufficient evidence to show that the current warning is not factual or misleading, or that either of the proposed warnings is effective in promoting greater public understanding of the risk associated with smokeless tobacco products."

May 7, 2015

  • AbbVie announced that the investigational medicine venetoclax has been granted Breakthrough Therapy Designation by the FDA for the treatment of chronic lymphocytic leukemia in previously treated patients with the 17p deletion. An oral B-cell lymphoma-2 inhibitor, venetoclax is being tested in several other cancers. AbbVie is collaborating on its development with Genentech and Roche.
  • Calgary, Canada’s Oncolytics Biotech announced that the FDA has granted Orphan Drug Designation to REOLYSIN for the treatment of gastric cancer. A few days earlier, the European Medicines Agency granted Orphan Drug Designation to REOLYSIN for the treatment of pancreatic cancer. An isolate of the reovirus, the REOLYSIN infects and selectively targets tumors with activating Ras pathway mutations and/or overexpression of Ras pathway elements, including EGFR, BRAF, and KRAS.
  • The most recent draft of the House Energy and Commerce Committee's 21st Century Cures bill would grant approximately $1.5 billion in additional funds to the NIH in each of the next 3 fiscal years. In addition, it would establish an "NIH Innovation Fund" that would provide an additional $2 billion each year for 5 years ($10 billion total) through fiscal year 2020 for precision medicine, emerging young scientists, and other innovative activities. The bill has garnered bipartisan support.
  • Woburn, MA–based ArQule and Beryllium Discovery Corp. (Boston, MA, and Seattle, WA) announced a collaborative research and development agreement to identify and unlock the therapeutic potential of small-molecule compounds by combining ArQule's chemistry and drug development expertise with Beryllium's discovery platforms. They plan to pursue compounds that target PD-1 and PD-L1.
  • In new screening guidelines, the American College of Physicians says that clinicians should screen women ages 21 to 30 who are at average risk of cervical cancer every 3 years, using the Pap test alone. The guidelines say that women younger than 30 should not be tested for human papillomavirus (HPV) as part of cancer screening because of the high prevalence of HPV infection in this age group. Screening average-risk women younger than 21 is not recommended, regardless of sexual history.
  • Headquartered in Santa Clara, CA, Agilent Technologies said that it will acquire Cartagenia for an undisclosed sum. With offices in Leuven, Belguim, and Cambridge, MA, Cartagenia provides software solutions for assessing, reporting, and sharing information on genomic variants from next-generation sequencing and microarray data. The acquisition will aid Agilent in offering software solutions for its clinical genetics and molecular oncology customers.
  • With the NYU School of Medicine, Johnson & Johnson has formed a panel of 10 experts to evaluate appeals from dying patients for access to experimental medications, which are often in limited supply and reserved for clinical trial participants. So-called compassionate-use appeals create a quandary for drug developers, who risk public relations challenges if they don't provide a medication as well as challenges from regulators if unexpected adverse events occur. The committee could create a policy that would serve as a model for other pharmaceutical companies.

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April 2015

April 30, 2015

  • Eli Lilly and Company received a fourth FDA approval for ramucirumab (Cyramza), an antiangiogenic therapy that blocks VEGFR2. It can now be used in combination with FOLFIRI chemotherapy for the treatment of patients with metastatic colorectal cancer whose disease progresses on or after therapy with bevacizumab (Avastin; Genentech/Roche), oxaliplatin, and a fluoropyrimidine. Ramucirumab is also approved for patients with advanced or metastatic stomach cancer, gastroesophageal junction adenocarcinoma, and non–small cell lung cancer.
  • Summit, NJ–based Celgene inked a $450 million deal with AstraZeneca to develop the PD-L1 inhibitor MEDI4736 for non-Hodgkin lymphoma, multiple myeloma, and other related malignancies. AstraZeneca will also pay $250 million to France’s Innate Pharma to license the immunotherapeutic IPH2201 both as a monotherapy and in combination with MEDI4736. Those deals came a day after AstraZeneca partnered with Juno Therapeutics to test that company’s CAR-T technology with MEDI4736 in blood cancers, Fierce Biotech reported.
  • Celgene also announced that it will acquire Quanticel Pharmaceuticals, a privately held biotechnology company headquartered in San Francisco, CA, focused on cancer drug discovery, for $100 million up front and as much as $385 million more in contingency payments. The deal gives Celgene access to Quanticel’s proprietary platform for the single-cell genomic analysis of human cancers.
  • An FDA advisory committee voted 22–1 to recommend approval of Amgen’s oncolytic immunotherapy talimogene laherparepvec, or T-VEC, for the treatment of metastatic melanoma. Injected into tumors, T-VEC replicates inside cancer cells, causing them to rupture and release tumor-derived antigens, along with granulocyte-macrophage colony-stimulating factor, which can prompt an immune response. The FDA usually follows the recommendations of its advisory panel.
  • Acting on a positive recommendation from the European Medicines Agency, the European Commission granted orphan drug designation to VentiRx Pharmaceuticals’ motolimod (VTX-2337) for the treatment of ovarian cancer. Used in combination with pegylated liposomal doxorubicin, motolimod is a novel Toll-like receptor 8 immunotherapeutic being assessed in a phase II randomized, placebo-controlled trial.
  • ImmunoGen disclosed that the antibody–drug conjugate it licensed to Sanofi for development, SAR3419, has been dropped from that company’s oncology pipeline. The agent was designed to treat diffuse large B-cell lymphoma (DLBCL) and other blood cancers. It had shown potential as a second-line therapy for DLBCL in a phase II study.
  • In a survey of 7,000 people, 98% said that they want to be informed if researchers using their genetic data stumble upon indicators of a life-threatening disease that could be prevented or treated. Fewer people were interested in knowing about less serious conditions or genetic data of “uncertain clinical significance.” Led by the UK’s Wellcome Trust Sanger Institute, the study was published in the European Journal of Human Genetics.
  • Originally established as the health arm of the National Academy of Sciences in 1970, the Institute of Medicine will change its name to the National Academy of Medicine on July 1. With the National Academy of Engineering, the three honorific societies advise the country on matters of science, technology, and health.

April 23, 2015

  • The American Association for Cancer Research (AACR) inaugurated José Baselga, MD, PhD, as its president for the next year during the AACR Annual Meeting 2015, held in Philadelphia, PA, April 18–22. An internationally recognized physician-scientist whose research focuses on the clinical development of novel molecularly targeted agents for the treatment of cancer, particularly breast cancer, Baselga is physician-in-chief and chief medical officer at Memorial Sloan Kettering Cancer Center in New York, NY. He replaces Carlos L. Arteaga, MD, professor of medicine and cancer biology at Vanderbilt University School of Medicine in Nashville, TN.
  • Bristol-Myers Squibb stopped a phase III study of nivolumab (Opdivo) early after it demonstrated superior overall survival compared with docetaxel in patients with advanced nonsquamous non–small cell lung cancer (NSCLC). A PD-1 inhibitor, nivolumab is FDA-approved for the treatment of metastatic melanoma after disease progression with platinum-based chemotherapy, and for squamous NSCLC following treatment with the CTLA-4 inhibitor ipilimumab (Yervoy).
  • Roche signed a deal worth up to $555 million with India’s Curadev Pharma to develop cancer immunotherapies that target IDO1 and TDO. Roche will pay Curadev $25 million initially, with an additional $530 million if Curadev meets certain milestones.
  • AstraZeneca announced that the FDA granted Orphan Drug Designation to its MEK inhibitor selumetinib for the treatment of uveal melanoma, a cancer of the eye for which there are no approved treatments once the disease spreads beyond the eye. Data from a phase III study evaluating selumetinib in combination with chemotherapy in patients with metastatic uveal melanoma are expected to be released later this year. Selumetinib is also being investigated in KRAS mutation–positive lung cancer and thyroid cancer, and in children with neurofibromatosis.
  • Cambridge, MA–based Foundation Medicine announced that it will collaborate with the NCI, Nationwide Children’s Hospital, and Baylor College of Medicine on the Exceptional Responders Initiative, which seeks to understand why some patients have unexpected and prolonged responses to a therapy. Tissue from exceptional responders will be collected and processed at Nationwide Children’s in Columbus, OH, and then analyzed using Foundation Medicine’s FoundationOne comprehensive genomic profile test and by whole-exome and transcriptome RNA sequencing through Baylor, in Houston, TX. The resulting data could accelerate the development of targeted therapies.
  • French drug maker Ipsen and its Swedish partner, Active Biotech, announced that they will discontinue all studies of tasquinimod in prostate cancer after the drug failed to show any survival benefit in a phase III trial in men with metastatic, castration-resistant disease. Tasquinimod is an oral quinoline-3-carboxamide derivative with immunomodulatory properties and antiangiogenic activity.
  • The use of electronic cigarettes, or e-cigarettes, by teens and adolescents tripled between 2013 and 2014, according to an annual report by the Centers for Disease Control and Prevention and the FDA’s Center for Tobacco Products. Findings from the National Youth Tobacco Survey show that current e-cigarette use (meaning use at least once in the past month) among high school students increased from 4.5% to 13.4%; among middle school students, use more than tripled from 1.1% to 3.9%. This is the first time since the survey started collecting data on e-cigarettes in 2011 that e-cigarette use has surpassed use of every other tobacco product overall, including conventional cigarettes.

April 16, 2015

  • Also, the FDA granted Fast Track Designation to cabozantinib (Cometriq) for the treatment of patients with advanced renal cell carcinoma who have received one prior therapy, according to the drug's developer, Exelixis, Inc. Cabozantinib inhibits the activity of multiple tyrosine kinases, including MET, VEGFRs, and RET. It was approved in 2012 for the treatment of progressive, metastatic medullary thyroid cancer.
  • Based on an assessment by an independent data-monitoring committee, the phase III PALOMA-3 trial for palbociclib (Ibrance; Pfizer) was stopped early because it met its primary endpoint of demonstrating progression-free survival; detailed results will be presented at an upcoming oncology conference. The trial was testing palbociclib plus fulvestrant versus fulvestrant plus placebo in women with HR-positive, HER2-negative metastatic breast cancer following disease progression during or after endocrine therapy. Palbociclib inhibits cyclin-dependent kinase 4/6.
  • A Canadian study published in Cancer indicates that vaccinating 12-year-old boys against human papillomavirus (HPV) may be a cost-effective strategy for preventing oropharyngeal squamous cell cancer. When investigators applied a statistical model to a population of 192,940 boys who were 12 in 2012, they found that HPV vaccination could save between C$8 million and C$28 million over the boys' lifetimes. Many countries have established HPV vaccination programs for girls but not for boys.
  • An FDA advisory committee considered a request from Swedish Match North American to modify the current warning label on a smokeless tobacco product called snus, small tea bag–like packets of tobacco placed under the upper lip. The company wanted to change "This product is not a safe alternative to cigarettes," to "Warning: No tobacco product is safe, but this product presents substantially lower risk to health than cigarettes." The committee unanimously agreed that the suggested wording was not sufficient; the FDA does not have to follow the advice of its advisory committee, but it usually does.
  • The FDA granted Orphan Drug Designation to the anti–CTLA-4 monoclonal antibody tremelimumab for the treatment of malignant mesothelioma, AstraZeneca announced. Mesothelioma is a rare, aggressive cancer that affects the lining of the lungs and abdomen.
  • Roche acquired CAPP Medical, a privately held genomics research company based in Palo Alto, CA, to advance development of technology for cancer screening and monitoring through the detection of circulating tumor DNA (ctDNA) in blood. CAPP Medical’s technology analyzes ctDNA using next-generation sequencing that can detect multiple mutations with a single assay. Terms of the deal were not disclosed.
  • Kansas City, MO's Children's Mercy Hospital announced that it has become a partner in the University of Kansas Cancer Center NCI Consortium, which also includes the nearby Stowers Institute for Medical Research and the University of Kansas (KU) in Kansas City, KS. Strengths of Children's Mercy include pediatrics, genomic medicine, orphan-drug disease indications, and drug discovery for cancer. Already an NCI-designated cancer center, the KU Cancer Center will be better positioned to earn the NCI's distinction as a comprehensive cancer center, a status achieved by only 41 U.S. institutions.
  • According to a study in The Journal of Pediatrics, the incidence of melanoma is falling among children in the United States. Using national cancer registry data, the researchers found that the number of new cases of melanoma among people younger than 20 fell nearly 12% per year from 2004 to 2010. The researchers noted that their results contradict other studies, which suggest that melanoma incidence is on the rise.

April 9, 2015

  • Clovis Oncology (Boulder, CO) announced that the FDA granted Breakthrough Therapy designation to its PARP inhibitor rucaparib as a monotherapy for advanced ovarian cancer in women with BRCA-mutated tumors who have received at least two lines of platinum-containing therapy. The designation was granted based on interim efficacy and safety results from two phase II studies. New clinical data will be presented at a medical meeting later this year.
  • The European Commission approved the use of panitumumab (Vectibix; Amgen) as a first-line therapy for wild-type RAS metastatic colorectal cancer (mCRC) in combination with FOLFIRI. About half of mCRC patients have wild-type RAS disease. An irinotecan-based chemotherapy regimen, FOLFIRI is frequently used in Europe as a first-line treatment for colorectal cancer.
  • Roche said that the European Commission also approved the use of bevacizumab (Avastin) in combination with chemotherapy to treat women with advanced cervical cancer. Bevacizumab is already approved for this indication in the United States.
  • Novartis said it will pay Seattle, WA–based Juno Therapeutics %12.25 million immediately to settle a patent dispute concerning chimeric antigen receptor T-cell (CAR-T) technology. Novartis will make additional payments in the future, including royalties, if its leading CAR-T product makes it to market. The costimulatory domain 4-1BB was at the center of the litigation, which began in July 2012.
  • In a study of 396 patients, men with castration-resistant prostate cancer who received enzalutamide (Xtandi) experienced a statistically significant increase in progression-free survival compared with those who received bicalutamide, Astellas Pharma and Medivation announced. Median PFS was 19.4 months in the enzalutamide group compared with 5.7 months in the bicalutamide group. Additional results from the trial will be submitted for presentation at upcoming medical meetings.
  • Merck KGaA and Pfizer said that they will jointly promote Pfizer's ALK inhibitor crizotinib (Xalkori) later this year in the United States, Canada, Japan, and five European countries. The agreement demonstrates the companies' commitment to establishing a combined oncology sales organization in preparation for the potential launch of avelumab-based treatment regimens in the future. Last fall, the companies agreed to work together to develop and commercialize avelumab, an investigational anti–PD-L1 monoclonal antibody.
  • Health Canada issued a marketing authorization to Novartis to sell ceritinib (Zykadia) for the treatment of patients with ALK-positive, locally advanced or metastatic non–small cell lung cancer who have progressed on, or were intolerant to, crizotinib. The authorization was based on a pivotal phase I trial that included 163 patients with the disease who had received prior treatment with crizotinib. The overall response rate among these patients was 54.6% and the median duration of response was 7.4 months.
  • Seeking to move its basic research discoveries into the clinic, New York's Cold Spring Harbor Laboratory announced an alliance with North Shore-LIJ Health System. The affiliation will benefit from an investment of more than $120 million, which will be used to advance cancer therapeutics research, develop a new clinical cancer research unit at the North Shore-LIJ Cancer Institute headquarters in Lake Success, NY, to support early-phase clinical studies of new therapies, and recruit and train more clinician-scientists in oncology.
  • A study published in the journal Health Affairs estimates that the United States spends $4 billion annually on medical tests due to false-positive mammograms and treatment of breast tumors that are unlikely to cause life-threatening disease. That estimate includes care for women ages 40–59 only.

April 3, 2015

  • Novartis entered into an alliance with Aduro Biotech to discover and develop cancer immunotherapies targeting the STING, or stimulator of interferon genes, pathway; when activated, the pathway initiates broad innate and adaptive immune responses in tumors. Under the terms of the agreement, Novartis will pay Berkeley, CA–based Aduro $200 million and make two $25 million equity investments in the company—one now and one at a future date—which will receive payments for meeting various milestones as well. Novartis also announced the launch of a new immune-oncology research group.
  • Merck and Syndax Pharmaceuticals of Waltham, MA, will collaborate on a clinical trial to evaluate the safety and efficacy of combining Merck's PD-1 inhibitor pembrolizumab (Keytruda) and Syndax's investigational epigenetic therapy entinostat in patients with either advanced non–small cell lung cancer or melanoma. Entinostat is an oral highly selective HDAC inhibitor that has been shown to reduce the number and function of host immune suppressor cells, enhancing antitumor activity. Financial terms were not disclosed.
  • Also on the immune-oncology front, Ziopharm Oncology and Intrexon Corporation will develop novel chimeric antigen receptor T-cell products for cancer with Merck Serono, the biopharmaceutical division of Merck KGaA (Darmstadt, Germany). Germantown, MD–based Intrexon will share an up-front payment of $115 million from Merck Serono, in addition to any milestone and royalty payments, with Boston, MA–based Ziopharm.
  • PBS aired a three-part documentary on cancer produced by Ken Burns, Cancer: The Emperor of All Maladies, based on the Pulitzer Prize–winning book by Siddhartha Mukherjee, MD, PhD. Full episodes of the film and other materials are available at Cancer: The Emperor of All Maladies.
  • Lebanon, NH's Norris Cotton Cancer Center at Dartmouth was redesignated as a Comprehensive Cancer Center by NCI. The designation renews the center's core grant funding for the next 5 years, although the exact amount of the funding has yet to be determined. There are only 41 NCI-designated Comprehensive Cancer Centers in the United States.
  • Genomics England announced that 10 companies will collaborate as part of its 100,000 Genomes Project to accelerate the development of new diagnostics and treatments for patients with cancer and rare diseases. The Genomics Expert Network for Enterprises, or GENE, Consortium includes AbbVie, Alexion Pharmaceuticals, AstraZeneca, Biogen, Dimension Therapeutics, GlaxoSmithKline, Helomics, Roche, Takeda, and UCB.
  • Amgen lost another battle in its attempt to block Sandoz from selling a biosimilar version of its drug filgrastim (Neupogen), which helps patients prevent infections during chemotherapy. Amgen petitioned the FDA to stop Sandoz's sale of Zarxio, arguing that Sandoz didn't supply its marketing application to Amgen within 20 days of submitting it to the FDA, violating federal law, but the FDA said it doesn't have the authority to enforce the exchange of marketing information, according to The Wall Street Journal.
  • The NIH announced a team of experts to lead the Precision Medicine Initiative announced by President Obama in January. The group will be headed by Richard Lifton, MD, PhD, from Yale University; Bray Patrick-Lake, MFS, from Duke University; and Kathy Hudson, PhD, from NIH. For a complete list of the group's members, visit the Precision Medicine Initiative.

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March 2015

March 19, 2015

  • Bloomberg Philanthropies and the Bill & Melinda Gates Foundation launched the Anti-Tobacco Trade Litigation Fund to help combat the tobacco industry's use of international trade agreements to threaten countries and prevent them from passing strong tobacco-control laws. The fund, established with $4 million from the organizations, will help low- and middle-income countries draft and defend tobacco-control laws by paying for high-quality legal assistance. The Washington, DC–based Campaign for Tobacco-Free Kids will administer the fund.
  • To meet the needs of patients who are smokers at the time of a cancer diagnosis, the National Comprehensive Cancer Network has published clinical practice guidelines for smoking cessation. The guidelines, available at www.nccn.org, recommend that treatment plans for all smokers with cancer include evidence-based pharmacotherapy, counseling, and close follow-up with retreatment, if needed. Smoking status should be documented at regular intervals, as relapse is common, the guidelines note.
  • The American Society of Clinical Oncology (ASCO) published "The State of Cancer Care in America: 2015" and presented its findings at a Congressional briefing. The report chronicles the current realities of the cancer care system and examines trends in the oncology workforce and in oncology practices that are affecting patient care and access. "With this knowledge and insight, we can identify ways to ensure that all patients with cancer receive high-quality care—and help oncology practices adapt, survive, and succeed in today's demanding healthcare environment," said ASCO President Peter P. Yu, MD.
  • According to new figures released by Cancer Research UK, obese women have about a 40% greater risk of developing at least seven types of cancer, including colon, breast, gallbladder, uterine, kidney, pancreatic, and esophageal cancers. That means that obese women have about a one in four risk of developing a cancer linked to weight in their lifetime.
  • The cancer immunotherapy company Aduro Biotech has filed with the Securities and Exchange Commission to raise $86 million through an initial public offering of stock. Based in Berkeley, CA, Aduro is testing a combination of CRS-207, engineered Listeria monocytogenes bacteria, and the GVAX cancer vaccine, for the treatment of pancreatic cancer. The company is also testing the combination with Bristol-Myers Squibb's checkpoint inhibitor nivolumab (Opdivo).
  • Amgen announced that about 300 employees at its subsidiary Onyx Pharmaceuticals will be let go and that the Onyx site in South San Francisco, CA, will be shuttered at the end of the year. Remaining workers will be absorbed by Amgen and work at its Thousand Oaks, CA, headquarters. Onyx is perhaps best known for its blood cancer drug carfilzomib (Kyprolis).
  • Two out of three people diagnosed with cancer will survive at least 5 years, according to a report published by the Centers for Disease Control and Prevention based on data reported during 2011. The most common cancers continue to be prostate, female breast, lung and bronchus, and colon and rectum, the report noted. The full report, "Invasive Cancer Incidence and Survival—United States, 2011" is available at www.cdc.gov/mmwr.
  • Cell Medica, of London, UK, announced that the FDA granted Orphan Drug Designation to CMD-003, an immunotherapy under development for Epstein-Barr virus–positive non-Hodgkin lymphomas. The designation is granted to promising drugs for conditions that affect 200,000 or fewer people in the United States; companies receive several benefits from the designation, including tax credits and additional market exclusivity.

March 12, 2015

  • In spite of a 2007 law requiring sponsors of clinical trials to report results on www.clinicaltrials.gov within 1 year of completion or termination, results summaries were posted for just 13.4% of trials after 12 months, according to a study published in The New England Journal of Medicine. Even after 5 years, only 38.3% of trials had reported results. At both time points, industry-funded trials were more likely to have reported results than trials supported by NIH, nongovernment agencies, and academia.
  • Seeking "an opportunity for more expeditious clearance of Ablatherm" in the United States, Lyon, France–based EDAP TMS SA announced that it will seek FDA clearance for the device through the agency's Direct De Novo application instead of the standard, more rigorous Pre-Market Approval (PMA) application. To qualify for a De Novo application, the device must be novel, meaning that there is no suitable predicate device, and it must present no more than moderate risk. Last year, an FDA advisory committee considering the PMA recommended against approving Ablatherm, which uses high-frequency ultrasound to ablate prostate tumors.
  • Breast cancer survivors are at increased risk of developing thyroid cancer, especially within 5 years of their breast cancer diagnosis, according to results of a study presented at the Endocrine Society's 97th annual meeting in San Diego, CA. "Recognition of this association between breast and thyroid cancer should prompt vigilant screening for thyroid cancer among breast cancer survivors," said lead investigator Jennifer Hong Kuo, MD, assistant professor of surgery at Columbia University in New York, NY.
  • Increasing colorectal cancer screening rates to 80% by 2018 would prevent an additional 21,000 colorectal cancer deaths per year through 2030, according to a new study in Cancer. The researchers used a computer model to show the effects of increasing screening rates from about 58% in 2013 to 80% in 2018, compared with a scenario in which the screening rate remained relatively constant.
  • With help from the University of California, Los Angeles, Jonsson Comprehensive Cancer Center; Penn Medicine; Dana-Farber Cancer Institute; and Sage Bionetworks, Apple launched a mobile app to track quality of life of breast cancer survivors over time. Data will be used to study why some survivors recover faster than others, why symptoms vary, and what can be done to improve problems such as fatigue, mood and cognitive changes, and sleep disturbances. Called "Share the Journey: Mind, Body, and Wellness after Breast Cancer," the app is part of Apple's ResearchKit, which is designed to expand participation in research studies.
  • Cambridge, MA–based AVEO Oncology announced that, in a phase II trial, a subset of patients with metastatic colorectal cancer who received tivozanib experienced longer progression-free survival (PFS) than patients who received bevacizumab (17.9 months vs. 11.2 months). Patients in this subset had low levels of neuropilin-1 (NRP-1), suggesting that NRP-1 may have value as a prognostic biomarker for angiogenesis inhibitors. AVEO discontinued the study last year after an interim futility analysis determined that tivozanib was unlikely to demonstrate superiority to bevacizumab in the intent-to-treat population.
  • Also at the Endocrine Society meeting, researchers presented results of a phase II trial of sunitinib (Sutent; Pfizer) showing that 19 of 24 patients with advanced-stage differentiated thyroid cancer experienced either significant tumor shrinkage or stable disease. The median PFS was 241 days. Sunitinib is approved for the treatment of renal cell carcinoma and imatinib-resistant gastrointestinal stromal tumors.

March 6, 2015

  • The biopharmaceutical company AbbVie announced it will acquire Sunnyvale, CA–based Pharmacyclics in a deal valued at approximately $21 billion. Pharmacyclics' lead drug is ibrutinib (Imbruvica), a Bruton’s tyrosine kinase inhibitor approved for use in four indications to treat three types of blood cancer: chronic lymphocytic leukemia, mantle cell lymphoma, and Waldenström macroglobulinemia. More than 50 clinical studies are evaluating ibrutinib as a treatment for other indications, including various solid tumors and graft-vs.-host disease.
  • NCI Director Harold Varmus, MD, announced that he will step down from his post, effective March 31. He will join the faculty of Weill Cornell Medical College in New York, NY, and establish a lab to study mutations that drive lung adenocarcinoma. Douglas Lowy, MD, who currently serves as deputy director, will become acting director on April 1.
  • The FDA approved the use of nivolumab (Opdivo; Bristol-Myers Squibb) to treat patients with metastatic squamous non–small cell lung cancer whose disease progresses on or after platinum-based chemotherapy. The drug's efficacy was established in a randomized trial of 272 participants, of whom 135 received nivolumab and 137 received docetaxel; on average, those who received nivolumab lived 3.2 months longer. A PD-1 inhibitor, nivolumab was approved in December to treat advanced melanoma.
  • Toronto, Canada's Princess Margaret Cancer Centre and Barcelona, Spain's Vall d’Hebron Institute of Oncology announced that they will collaborate to advance personalized, targeted therapies against cancer. The two centers will exchange information about patient care and research, as well as academic programs. They will also share best practices in clinical research and clinical trials and support innovation on cancer education and training programs.
  • Bristol-Myers Squibb also signed an agreement with Bavarian Nordic (Kvistgaard, Denmark) to license and commercialize Prostvac, the Danish company's investigational, phase III immunotherapeutic "vaccine" for the treatment of metastatic castration-resistant prostate cancer. Bavarian Nordic will receive up to $975 million, including an up-front payment of $60 million. Last week, the companies released early data from a study of 30 men with advanced prostate cancer showing that 20% of them were still alive 80 months after starting treatment with the most promising dose of Prostvac and the checkpoint inhibitor ipilimumab (Yervoy; Bristol-Myers Squibb).
  • Testifying before the House Appropriations Subcommittee on Labor, HHS, Education, and Related Agencies, NIH Director Francis Collins, MD, PhD, said that his agency's budget request of $31.31 billion for fiscal year (FY) 2016 would fund 1,227 more research grants than in FY2015. An increase of $1 billion or 3.3% over FY2015, the request also includes $200 million for the Precision Medicine Initiative. 1The budget request allocates resources to areas of the most extraordinary promise for biomedical research," Collins said, "while maintaining the flexibility to pursue unplanned scientific opportunities and address unforeseen health needs."
  • The FDA launched a mobile application designed to speed public access to information about drug shortages. The app identifies current shortages, resolved shortages, and discontinuations of drugs. Users can browse by a drug's generic name or active ingredient and by therapeutic category, as well as report a suspected shortage or supply issue to the FDA.

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February 2015

February 26, 2015

  • Celldex Therapeutics announced that the FDA granted Breakthrough Therapy designation to rindopepimut (Rintega) for the treatment of adults with EGFRvIII-positive glioblastoma. The Hampton, NJ, company said that the decision was based on data from phase II trials involving patients with recurrent and newly diagnosed disease. Rindopepimut is an investigational immunotherapy that targets the tumor-specific oncogene EGFRvIII.
  • Eisai Inc. announced that treatment with eribulin (Halaven) led to a statistically significant improvement in overall survival in patients with advanced soft-tissue sarcomas compared with dacarbazine in a phase III trial, meeting the primary endpoint. Approved for metastatic breast cancer in 2010, eribulin triggers apoptosis of cancer cells following prolonged mitotic blockade. Data from the trial will be presented at an upcoming medical meeting, the company said.
  • Boehringer Ingelheim and Pfizer both announced that they will trim staff in the United States. According to FierceBiotech, Boehringer Ingelheim will cut about 5% of the 2,700 employees at its U.S. headquarters in Ridgefield, CT, possibly reassigning some to other positions. The Boston Globe reported that Pfizer would make "select headcount reductions in certain areas of R&D" at its research center in Cambridge, MA, but declined to specify how many. Some employees, Pfizer said, would be transferred and new employees hired in growing areas such as immuno-oncology.
  • Bristol-Myers Squibb announced two deals that will expand its oncology pipeline. The company will acquire San Carlos, CA–based Flexus Biosciences, a privately held biotech business developing IDO and TDO inhibitors, in a deal worth up to $1.25 billion. Rigel Pharmaceuticals of South San Francisco, CA, announced that it will develop its portfolio of small-molecule TGFβ receptor kinase inhibitors in collaboration with Bristol-Myers Squibb for use in cancer, which could net Rigel more than $339 million.
  • In an ironic twist, a drop in smoking rates could lead to an increase in deaths from lung cancer, a study suggested. The researchers blame current screening guidelines, which recommend annual low-dose CT screening for adults ages 55 to 80 who have smoked for 30 pack-years (a pack a day for 30 years), and who currently smoke or stopped smoking within the last 15 years. With the passage of time, former smokers will become ineligible for screening, meaning that patients who are eventually diagnosed with lung cancer will be diagnosed at a later stage, when treatment may no longer be effective.
  • The Federal Trade Commission fined the makers of two smartphone apps—Mole Detective and MelApp—that claimed they could detect melanoma and calculate melanoma risk by analyzing a photo of a mole. Although the apps recommended that users see a physician, advertisements gave buyers the impression that the apps could be used as diagnostic tools. The apps sold for $4.99 or less and were on the market in 2011 and 2012, according to The Washington Post.

February 19, 2015

  • Calgary, Canada–based Oncolytics Biotech Inc. announced that the FDA granted Orphan Drug Designation to REOLYSIN for the treatment of pancreatic cancer and ovarian cancer. REOLYSIN is Oncolytics' proprietary isolate of the reovirus, or respiratory enteric orphan virus. It is designed to infect and selectively target tumors with activating Ras pathway mutations and/or overexpression of Ras pathway elements including EGFR, BRAF, and KRAS.
  • Genentech, of South San Francisco, CA, announced that the FDA granted priority review to the MEK inhibitor cobimetinib in combination with vemurafenib (Zelboraf) for the treatment of BRAF V600 mutation–positive advanced melanoma. The FDA will make an approval decision by August 11. Cobimetinib is also being investigated as a treatment for non–small cell lung cancer, colorectal cancer, triple-negative breast cancer, and melanoma.
  • The FDA also expanded the existing indication for lenalidomide (Revlimid) in combination with dexamethasone to include patients newly diagnosed with multiple myeloma, Summit, NJ–based Celgene announced. The combination was originally approved in 2006 to treat patients with multiple myeloma who have received at least one prior therapy.
  • Takeda terminated its phase III MONET-A study because the primary endpoint of progression-free survival was not met. The study was comparing the investigational agent motesanib in combination with paclitaxel and carboplatin to placebo in combination with paclitaxel and carboplatin. Motesanib is a small-molecule antagonist of VEGF receptors 1, 2, and 3, platelet-driven growth factor receptors, and stem cell factor receptors.
  • Valeant Pharmaceuticals International Inc. will buy bankrupt Seattle, WA–based Dendreon Corp. for $400 million in cash. The sale will give Valeant, of Laval, Canada, Dendreon's sipuleucel-T (Provenge) prostate cancer vaccine and other assets. Valeant said it expects to finalize the deal by the end of the month
  • According to the Boston Globe, French drugmaker Sanofi will eliminate about 100 jobs in Massachusetts related to cancer research and its R&D operations. The paper reported that the company has about 5,000 employees in the state and about 110,000 worldwide.
  • Speaking at the annual AAAS conference in San Jose, CA, Wilson Compton, deputy director of the National Institute on Drug Abuse, said that early data on smoking from the Monitoring the Future Study conducted in 2014 are concerning because they suggest that nonsmoking adolescents could become addicted to nicotine through electronic cigarettes. That could lead them to use traditional cigarettes, he said. The study showed that 8% of eighth graders, 16% of tenth graders, and 17% of twelfth graders had used the battery-operated devices in the previous month.
  • The American Association for Cancer Research (AACR) announced 11 new fellows of its AACR Academy, which recognizes and honors distinguished scientists who have made exceptional contributions to cancer research and/or cancer-related biomedical science. The AACR will formally induct its 2015 class of elected fellows of the AACR Academy at its annual meeting, which will be held in Philadelphia, PA, April 18–22.

February 13, 2015

  • After serving as the agency's commissioner for nearly 6 years, Margaret Hamburg, MD, announced that she will leave the FDA at the end of March. Stephen Ostroff, MD, the FDA's chief scientist, will serve as acting commissioner following Hamburg's departure.
  • The FDA approved the kinase inhibitor Lenvima (lenvatinib; Eisai) to treat patients with progressive, differentiated thyroid cancer whose disease progressed despite their receiving radioactive iodine therapy. In a phase III trial, 392 participants were randomly assigned to receive either Lenvima or a placebo. Lenvima-treated patients lived a median of 18.3 months without their disease progressing compared with 3.6 months for patients who received a placebo.
  • The FDA also approved Imbruvica (ibrutinib; Pharmacyclics) for the treatment of patients with Waldenström macroglobulinemia. Imbruvica was initially approved in November 2013 for the treatment of patients with mantle cell lymphoma, and it has since been approved for the treatment of patients with chronic lymphocytic leukemia.
  • Roche announced that it acquired Signature Diagnostics, a translational oncology and genomics company located in Potsdam, Germany, for an undisclosed sum. Signature Diagnostics develops large blood plasma and tissue biobanks in multiple cancers, including colorectal and lung, which are constructed from multicenter, prospective clinical studies. The company also develops next-generation sequencing assays using targeted gene panels.
  • Children's Hospital Los Angeles announced that it will commit $50 million in institutional funding to expand its Center for Personalized Medicine. The California hospital will seek an additional $50 million in philanthropic support to translate laboratory research into bedside care. The center will initially focus its work on pediatric cancers.
  • Although exposure rates dropped by half between 1999–2000 and 2011–2012, more than 58 million nonsmokers in the United States are still exposed to secondhand smoke, according to a report from the Centers for Disease Control and Prevention. Exposure is more common among children ages 3 to 11, blacks, people living below the poverty level, and those who rent housing, the report says.
  • A meta-analysis published in The Lancet found that women who undergo hormone replacement therapy (HRT) are 40% more likely to develop the two most common types of ovarian cancer—serous and endometrioid ovarian cancers. The findings were based on data from 52 epidemiologic studies involving 21,488 women with ovarian cancer. Although the risk declined after stopping treatment, women who used HRT for 5 years or longer still had an increased risk of the disease 10 years later.
  • The American Society of Clinical Oncology announced the launch of a new journal: The Journal of Global Oncology, an online-only, open-access journal, will focus on cancer care, research, and care delivery issues unique to countries and settings with limited health care resources. David Kerr, MD, DSc, will serve as founding editor-in-chief.

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January 2015

January 29, 2015

  • According to The New York Times, Myriad Genetics of Salt Lake City, UT, seems to have given up efforts to prevent other companies from offering tests for mutations in BRCA1 and BRCA2, bringing several lawsuits to an end. Myriad has reached settlements with LabCorp, Invitae, and Pathway Genomics. Settlement talks are under way with Ambry, Quest Diagnostics, GeneDx, and Counsyl. Myriad is developing a more comprehensive test to examine 25 genes linked to cancer risk.
  • The American Lung Association released its 13th annual "State of Tobacco Control" report, which concluded that very little happened on the state or federal levels in 2014 to reduce tobacco use. No state passed a comprehensive smoke-free law or significantly increased tobacco taxes, and no state earned an "A" grade for providing access to quit smoking treatments. Only two states funded their state tobacco prevention programs at levels recommended by the Centers for Disease Control and Prevention.
  • A bill was introduced in the California senate that would define electronic cigarettes (e-cigarettes) as tobacco products. If passed, use of e-cigarettes—battery-powered devices that vaporize liquid containing nicotine—would be banned everywhere that traditional cigarettes are.
  • Phoenix, AZ–based Insys Therapeutics announced that the FDA granted Orphan Drug Designation to its drug candidate liposomal encapsulated paclitaxel for the treatment of ovarian cancer. The designation, which can be awarded to products that treat rare diseases or conditions, provides drug developers with an additional 7 years of marketing exclusivity and financial incentives to support the drug's development.
  • An analysis published in The New England Journal of Medicine found that e-cigarette liquids vaporized at higher-than-normal voltage (5.0 V) produced significant amounts of formaldehyde-releasing agents. Assuming the risk of inhaling formaldehyde-releasing agents is the same as inhaling gaseous formaldehyde, the authors wrote, long-term heavy use of e-cigarettes, or "vaping," could mean that the lifetime risk of developing cancer is 5 to 15 times as high as the risk associated with long-term smoking.
  • Medivation and Astellas Pharma announced that men with metastatic castration-resistant prostate cancer who received enzalutamide experienced statistically significant increases in progression-free survival (PFS) compared with those who received bicalutamide. Median PFS in the phase II trial, dubbed TERRAIN, was 15.7 months in the enzalutamide group vs. 5.8 months in the bicalutamide group. Additional results will be presented at an upcoming medical conference.
  • An analysis published in Cancer found that many women with breast cancer lack knowledge of their disease's characteristics, including tumor stage, grade, and receptor status. Only 20% to 58% of 500 women surveyed reported the characteristics correctly. Black and Hispanic women were often less likely than white women to know their cancer's characteristics. Increased knowledge of tumor characteristics, the researchers said, may lead to more informed treatment decisions and greater adherence to treatment.

January 22, 2015

  • Ewing, NJ–based Celator Pharmaceuticals announced that the FDA granted Fast Track designation to CPX-351, a liposomal formulation of cytarabine and daunorubicin, for the treatment of elderly patients with secondary acute myeloid leukemia. The designation helps facilitate the development and review of drugs intended to treat serious or life-threatening conditions and that may address unmet medical needs.
  • CytRx announced that the FDA has lifted a partial hold on its late-stage cancer drug, aldoxorubicin. The Los Angeles, CA–based company had been ordered to stop enrollment in clinical trials of aldoxorubicin in November after a patient died while taking the drug under compassionate-use rules. With modified study protocols to minimize risks, CytRx will continue its studies in patients with soft-tissue sarcoma, glioblastoma, Kaposi sarcoma, and small cell lung cancer, among others.
  • Boehringer Ingelheim established a multiyear research alliance with Vanderbilt University (Nashville, TN) to research and develop small molecule inhibitors of oncogenic Ras. Financial considerations and other details were not disclosed. Found in 20% to 30% of all human cancers, mutations in the Ras family have proven especially difficult to target.
  • Human Longevity, Inc. (HLI) announced a multiyear agreement with Genentech to conduct whole-genome sequencing on tens of thousands of deidentified tissue samples. Based in La Jolla, CA, HLI will sequence the genomes to 30x coverage and analyze the data. HLI aims to build the world's most comprehensive, integrated human genotype and phenotype database.
  • The American Society of Clinical Oncology (ASCO) for the first time named its Advance of the Year: the transformation of treatment for adults with chronic lymphocytic leukemia. Thanks to the recent approvals of two immunotherapies—obinutuzumab and ofatumumab—and two molecularly targeted drugs—ibrutinib and idelalisib—the outlook for patients has improved dramatically. The Advance of the Year was announced as part of the release of Clinical Cancer Advances 2015: ASCO's Annual Report on Progress Against Cancer.
  • Having already joined forces to develop immunotherapies for cancer, two companies have agreed to license a novel chimeric antigen receptor T-cell therapy from The University of Texas MD Anderson Cancer Center in Houston for $100 million in stock. In addition, the companies—Ziopharm of Boston, MA, and Intrexon, of Germantown, MD—will contribute $15 million to $20 million a year for 3 years for researching and developing related technologies.
  • Speaking to The Wall Street Journal at the annual JP Morgan health care conference in San Francisco, CA, NIH Director Francis Collins, MD, PhD, noted that the agency used to fund one in three applications for research funding. Because NIH now funds just one in six applications, talented researchers could move to other countries, such as China, that are increasing spending on science. "That's the thing that wakes me up at night," Collins told the publication.

January 13, 2015

  • The research advocacy organization Research!America urged the U.S. Congress to act on five science-related priorities during the first 100 days of the legislative session in order to elevate research and innovation on the nation's agenda: end sequestration, increase funding for the nation's research agencies, advance the 21st Century Cures initiative, repeal the medical device tax, and enact a permanent and enhanced research and development (R&D) tax credit. For details, visit www.researchamerica.org.
  • Cambridge, MA–based Foundation Medicine announced that it will "enter into a broad strategic collaboration" with Roche, giving the Swiss pharmaceutical giant an ownership stake in the company of up to 56.3%. Accounting for additional investments in R&D, Roche's total investment in Foundation Medicine could exceed $1 billion. Foundation Medicine develops assays to determine molecular alterations in a patient's cancer and match them with targeted therapies and clinical trials.
  • Biogen Idec in Cambridge, MA, and Columbia University Medical Center in New York, NY, signed a $30 million agreement to establish a sequencing and analysis facility, as well as a shared postdoctoral program. Biogen Idec says the collaboration will enable it and Columbia to study the genomes of patients who have unusual responses to treatment and to "explore the connections among genes, pathways, and disease processes," information that could be used to find new drug targets and develop new treatments.
  • Cambridge, MA–based Aveo Oncology continues to downsize following the FDA's rejection of its kidney cancer drug, tivozanib, in 2013. The company announced that it is laying off 40 of its 60 remaining employees and eliminating its internal research functions.
  • Also based in Cambridge, MA, Mersana Therapeutics announced that it will expand its partnership with Takeda Pharmaceutical to create novel antibody–drug conjugates for the treatment of cancer. Depending on the success of the collaboration, as well as up-front payments, Mersana could net an additional $300 million.
  • Wilmington, DE–based Incyte announced an alliance with the immuno-oncology company Agenus of Lexington, MA, to develop four checkpoint modulator antibodies directed against GITR, OX40, LAG-3, and TIM-3. Agenus could receive upwards of $410 million as part of the licensing, development, and commercialization deal.
  • The FDA's Center for Drug Evaluation and Research launched the Office of Pharmaceutical Quality. The new office will focus on drug quality by taking steps such as establishing consistent, clinically relevant quality standards and clear expectations for industry, and encouraging the use of modern, more efficient manufacturing technologies.

January 8, 2015

  • An FDA advisory panel unanimously recommended approval of the investigational biosimilar filgrastim (Sandoz) for all of the same indications as its reference product, Neupogen (Amgen), a granulocyte colony-stimulating factor analog used in cancer treatment. This is the first time that the panel has recommended FDA approval of a generic biologic drug. Sandoz has proposed marketing the biosimilar as Zarxio in the United States if it is approved.
  • The American Association for Cancer Research and the American Society of Clinical Oncology issued a joint statement on electronic cigarettes (e-cigarettes) and other nicotine delivery devices to help guide policymakers who are trying to minimize the potential harms of their use without undermining their potential as a smoking cessation tool. According to the statement, tobacco use is responsible for 30% of all cancer deaths and associated with an increased risk of at least 18 types of cancer.
  • The American Cancer Society's annual cancer statistics report finds that a 22% drop in cancer mortality over two decades led to the avoidance of more than 1.5 million cancer deaths that would have occurred if peak rates persisted. Also, while cancer death rates have declined in every state, the report finds substantial variation in the magnitude of these declines, generally with the states in the south showing the smallest decline and in the northeast the largest decline.
  • Amgen and Kite Pharma announced that they will collaborate to develop and commercialize the next generation of novel chimeric antigen receptor T-cell immunotherapies based on Kite's engineered autologous cell therapy platform and Amgen's array of cancer targets. Kite, based in Santa Monica, CA, will receive an up-front payment of $60 million from Amgen and may receive up to $525 million more for meeting regulatory and commercial milestones; Amgen, based in Thousand Oaks, CA, may receive up to $525 million in milestone payments per Kite program.
  • Cambridge, MA–based Idera Pharmaceuticals announced that the FDA granted orphan drug designation to its IMO-8400, an antagonist of the endosomal Toll-like receptors 7, 8, and 9, for the treatment of Waldenström macroglobulinemia. The company is conducting a phase I/II trial of the agent in patients with the disease who have a history of relapse or who have failed to respond to at least one other therapy. The designation provides incentives to companies to develop drugs intended to treat rare diseases or conditions.
  • The NIH awarded grants totaling more than $28 million aimed at understanding how and when genes are turned off and on. The awards are part of the recently launched Genomics and Gene Regulation program of the National Human Genome Research Institute, which is part of NIH. Insights into the mechanisms controlling gene expression may lead to new avenues for developing treatment for diseases such as cancer that are linked to faulty gene regulation.
  • With 2.5 million people in the UK living with cancer, the organization Macmillan Cancer Support issued a warning to the UK's National Health Service (NHS) about the burden of caring for people with long-term side effects of treatment. "While it is great news that more people are surviving cancer or living longer with it, progress is a double-edged sword," said Lynda Thomas, the organization's chief executive, who called upon politicians to commit to investing in cancer care. "As numbers surge, the NHS will soon be unable to cope with the huge increase in demand for health services."
 
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