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Bio Roundup: Drug Pricing, Cystic Fibrosis Showdown, Myovant & More

Xconomy National — 

The 2016 election is (thankfully) almost here, and as one biotech analyst noted this week, a possible Democratic victory is to blame for a big October sell-off in biotech stocks. Since Donald Trump’s infamous “Access Hollywood” tape was released on October 7, Leerink Partners analyst Geoffrey Porges wrote, biotech investors have lost $50 billion. The prospects of a Hillary Clinton presidency and Democrat-controlled senate have spooked investors wary of drug policies “tremendously damaging to the industry,” Porges wrote.

In the meantime, however, the news hasn’t been all bad for drugmakers. A major shift in the treatment of lung cancer—for some patients, anyway—has begun. And new treatments to expand the reach of cystic fibrosis therapies have begun clinical testing. Those headlines and much more below.

TOP STORY

—The FDA approved pembrolizumab (Keytruda) from Merck (NYSE: MRK) for certain lung cancer patients who have received no previous treatment. It’s the first time a cancer immunotherapy has been approved in a first-line setting.

CYSTIC FIBROSIS SHOWDOWN

—As the North American Cystic Fibrosis Conference kicked off in Orlando, FL, Vertex Pharmaceuticals (NASDAQ: VRTX) said a key part of its plan to defend its industry-leading cystic fibrosis franchise is moving forward. Two “triple” combination regimens for CF, which are meant to expand the reach of its CF drugs, will begin Phase 2 trials by the end of the year, and two additional new CF drugs will start clinical testing in 2017. The news came with some caveats, however, as TheStreet.com explains here.

—Two Vertex rivals touted early-stage data at the NACFC conference: Belgium-based Galapagos, which is developing its own triple combination regimen, and Netherlands-based ProQr Therapeutics (NASDAQ: PRQR), which is advancing an RNA-based CF drug sprayed into the lungs.

THIS WEEK IN DRUG PRICING…

—A coalition of investors pressed drug makers to justify price increases. The group filed shareholder resolutions with 11 companies and cited a Kaiser Family Foundation poll that cited Americans’ concerns over drug prices.

—Richmond, VA-based Kaleo Pharmaceuticals said it would bring its anti-allergy auto-injector Auvi-Q back next year. The drug system went off the market in 2015, leaving Mylan’s EpiPen without competition and setting the stage for the furor over Mylan’s price hikes.

—Mylan CEO Heather Bresch has blamed EpiPen costs on middlemen. The largest of those middlemen, Express Scripts (NYSE: ESRX), said this week it received a subpoena from the U.S. Department of Justice for more information about its role in the drug-buying ecosystem.

…AND DUCHENNE

—Aaron Kesselheim and Jerry Avorn, both professors of medicine at Harvard Medical School, penned an op-ed in the Journal of the American Medical Association warning of the implications of the FDA’s September decision to approve Sarepta Therapeutics’ Duchenne muscular dystrophy drug eteplirsen (Exondys 51).

—Separately, the insurer Humana became the latest to cover Sarepta’s drug, but said it would only do so for Duchenne patients who are currently able to walk.

—A coalition of non-profits, drug companies, and medical experts have formed the Collaborative Trajectory Analysis Project, an effort to better quantify how Duchenne patients progress without treatment.

IPO’S, BIG AND SMALL

—Brisbane, CA-based Myovant Sciences (NASDAQ: MYOV), another biotech founded by Roivant Sciences founder Vivek Ramaswany, raised $218 million in the largest biotech IPO of 2016. Ramaswany’s Axovant Sciences (NASDAQ: AXON) bagged $360 million last year in the largest biotech IPO ever. Here’s more from Forbes.

— Cambridge, MA-based Ra Pharmaceuticals (NASDAQ: RARX) raised $92 million in an IPO, partially through the help of existing shareholders.

DEALS AND FUNDINGS

—Unity Biotechnology of South San Francisco, CA, reeled in $116 million to push drugs for knee arthritis and eye disease into clinical trials. The company’s strategy is to kill dormant cells in the body that worsen what it calls “diseases of aging.”

—The Bill and Melinda Gates Foundation gave $210 million to the University of Washington, a record for the school, to build a center for the study of global population health.

—Allergan (NYSE: AGN) exercised its option to buy a stomach drug from Boston-based Rhythm Pharmaceuticals for $200 million.

—Warsaw, IN-based Zimmer Biomet (NYSE: ZBH) expanded into telehealth by acquiring startup RespondWell for an undisclosed sum.

AND IN OTHER NEWS…

—Merck (NYSE:MRK) got the FDA’s nod for bezlotoxumab (Zinplava), a treatment for C. difficile infection. The approval comes three months after a new approach to fight the deadly infection—a mix of bacterial spores to restore a healthy gut microbiome—failed a major test in the hands of Seres Therapeutics (NASDAQ: MCRB).

—An experimental treatment for sickle cell disease will soon enter Phase 3 trials. Global Blood Therapeutics (NASDAQ: GBT) said it has the FDA’s green light to start a trial that could produce results in 2019. Meanwhile, cutting-edge techniques to knock out or fix the defective gene that causes sickle cell disease are in motion but have yet to reach human trials.

—The FDA put a partial clinical hold on some of the studies being run by Berkeley, CA-based Aduro Biotech (NASDAQ: ADRO) after a single patient tested positive for the bacterium Listeria.

—Shares of New York-based Dipexium Pharmaceuticals (NASDAQ: DPRX) plummeted more than 80 percent after a cream it had been developing for patients with infections from diabetic foot ulcers failed two Phase 3 trials.

—Irvine, CA, and Bedminster, NJ-based Aerie Pharmaceuticals (NASDAQ: AERI) withdrew the FDA approval application for its glaucoma drug, netarsudil (Rhopressa), because of manufacturing issues. Aerie expects to re-file the application in January.

—Tucked into Biogen’s (NASDAQ: BIIB) earnings report this week was news that the company discontinued development of a drug called MT-1303. Biogen paid Mitsubishi Tanabe Pharma $60 million in September 2015 for rights to the drug, which the company aimed to develop for a variety of autoimmune diseases, including ulcerative colitis and Crohn’s disease. The drug was a possible competitor to ozanimod, which Celgene acquired when it bought Receptos for $7.2 billion last year.

—And if you missed our “Boston’s Life Science Disruptors” conference last week, here’s a slideshow look at some of the festivities.

Alex Lash contributed to this report