Bio Roundup: Migraine Data, Cancer Drug Combos, Azar Hearing & More

Xconomy National — 

A pair of migraine drugs made a splash in biotech news this week with published data suggesting that they can reduce the frequency of headaches.

These drugs still await an FDA decision, and their impact on patients over the long term are being watched closely. As many as 38 million Americans suffer from migraines, according to the Migraine Research Foundation. Migraine sufferers and their physicians and patients are looking for new treatment options.

In other pharma news this week, U.S. Senators grilled a former pharmaceutical industry executive, a cancer DNA test won a landmark approval, more immunotherapy data emerged, and a tiny biotech company raised $114 million to develop a cell therapy for diabetes. Let’s start the roundup in our nation’s capital.

D.C. DOINGS

—In a three-hour Senate committee hearing, Health and Human Services secretary nominee Alex Azar faced a barrage of questions about drug prices and his former role as an Eli Lilly (NYSE: LLY) executive.

—Republican efforts to reform taxes could also affect healthcare. The Senate’s tax bill includes a provision to cripple the Affordable Care Act’s individual mandate, the requirement that everyone have health insurance or pay a penalty. A Los Angeles Times analysis found that removing the mandate would disproportionately affect rural parts of the country, leaving those regions with fewer and more expensive health plans.

—In what could be an important step forward for the mainstream acceptance of broad cancer genetic tests, a product from Foundation Medicine (NASDAQ: FMI), the FoundationOne CDx, simultaneously won FDA approval and got a coverage determination from the Centers for Medicare & Medicaid Services.

DATA DISCUSSION

—An emerging group of drugs meant to prevent migraines, not just deal with their symptoms, took center stage in the New England Journal of Medicine this week as two studies were published outlining the Phase 3 trials of rival experimental therapies from Amgen (NASDAQ: AMGN) and Teva Pharmaceutical (NYSE: TEVA). Yet some doctors expressed concern that a potentially high price tag will make them unaffordable for many patients who desperately need them.

—While it didn’t disclose specific details, Roche’s Genentech division reported that its immunotherapy atezolizumab (Tecentriq), when combined with chemotherapy and another one of its drugs, bevacizumab (Avastin), helped keep the tumors of newly diagnosed lung cancer patients in check longer than bevacizumab and chemotherapy alone. The trial could shake up the already competitive, fast-changing landscape of treatments for lung cancer.

—The New York Times reported that gene therapy companies are struggling to find supplies of viral vectors, a crucial component of the cutting-edge drugs. Cambridge, MA-based Bluebird Bio (NASDAQ: BLUE) announced it has bought its own virus-making facility, a site in Durham, NC.

—Pfizer (NYSE: PFE) and partner Merck KGaA’s effort to best rival immunotherapies in gastric cancer fell short, as their checkpoint inhibitor avelumab failed to beat chemotherapy in a Phase 3 study.

—Regeneron Pharmaceuticals (NASDAQ: REGN) scrapped development of a combination treatment it had been developing for age-related macular degeneration. The failure is a blow to the company’s efforts to protect flagship drug aflibercept (Eylea), and the latest combo therapy to flunk a study in AMD.

—-Cytokinetics’ (NASDAQ: CYTK) lead experimental treatment for amyotrophic lateral sclerosis (ALS) failed a Phase 3 study, leaving the South San Francisco, CA, company to shift its focus to a second compound for the disease that’s still in mid-stage clinical trials.

DEALS AND DOLLARS

—Moving on from the clinical trial failure of its eye drug combo treatment, Regeneron entered a partnership with Decibel Therapeutics of Boston to discover and develop drugs that could restore hearing. Regeneron is granting Decibel access to its technology and providing cash to support its partners’ R&D, but specific financials were not disclosed.

Codiak BioSciences raised another $76.5 million in financing, the latest sign that investments in technology harnessing exosomes continue to pick up.

—PureTech Health-backed resTORbio, a biotech developing drugs for diseases of aging, raised a $40 million Series B round led by OrbiMed.

—Cambridge, MA, and Providence, RI-based Semma Therapeutics bagged a $114 million Series B round to continue developing a cell therapy meant to free Type 1 diabetes patients from daily insulin injections.

—Biogen (NASDAQ: BIIB) paid $28 million up front to grab rights to ALKS8700, a multiple sclerosis drug Alkermes (NASDAQ: ALKS) has been developing to one-up Biogen’s dimethyl fumarate (Tecfidera).

—Fractyl Laboratories raised $44 million in a Series D financing round that the Lexington, MA, will use to help fund clinical testing of the intestinal procedure it has developed to treat Type 2 diabetes.

—San Mateo, CA-based Medior Therapeutics raised $57 million in a Series B round that will support Phase 3 studies of MDR-101, a cellular immunotherapy meant to replace the immunosuppressive drugs given to kidney transplant patients.

—-The Medicines Co. (NASDAQ: MDCO) of Parsippany, NJ, sold three infectious disease drugs to Melinta Therapeutics for $215 million, which it plans to apply toward late-stage clinical trials of an experimental cholesterol-lowering drug inclisiran.

—In April, the FDA awarded BioMarin Pharmaceuticals (NASDAQ: BMRN) a priority review voucher after the San Rafael, CA, company won approval for its Batten disease drug cerliponase alfa (Brineura). This week, the company reached a deal to sell that voucher for $125 million.

BIOTECH MOVERS AND SHAKERS

—As CEO of Sarepta Therapeutics (NASDAQ: SRPT), Ed Kaye helped engineer one of the most controversial drug approvals in recent memory, the 2016 FDA approval of Duchenne muscular dystrophy treatment eteplirsen (Exondys 51). Now he’s resurfacing as the CEO of a stealthy startup, Stoke Therapeutics. Xconomy spoke with Kaye about his long journey up the ranks in the biotech industry, his reflections on the eteplirsen saga, and his new gig.

—Xconomy profiled the work of Biogen, Women in Bio, and Harvard Medical School’s Joan Reede building diverse and inclusive workforces, efforts that earned them recognition as the winners in the Commitment to Diversity category in our Xconomy Awards.

—The Boston Business Journal reported that Ensemble Therapeutics, a developer of mid-size drugs, has quietly shut down.

—Teva got some shaking of the unpleasant kind. New CEO Kare Schultz axed several top execs amid big layoffs as the Israeli giant tries to parry generic competition.

Ben Fidler and Alex Lash contributed to this report.

Photo by Flickr user tr0tt3r via a Creative Commons license.