Bio Roundup: Mylan Grilled, Duchenne Kids Thrilled, Zuck-Chan & More

Xconomy National — 

Another biopharma executive went before Congress to explain drug price hikes. Unlike Martin Shkreli, Mylan CEO Heather Bresch actually answered questions. Taking heat over the EpiPen scandal, she tried to stick to her script, getting testy when legislators brought up a USA Today story about her mother helping market the life-saving anti-allergy injection to schools. Elsewhere in Washington, the NIH chief warned about Congress’s inability to agree on Zika funding, and Vice President Joe Biden talked with Stat about cancer research.

But the biggest Beltway life-sciences story of the week was the Food and Drug Administration’s stunning approval of the drug eteplirsen (Exondys51). It feels odd to say it was the FDA’s approval, when in fact it was one top administrator’s decision, overruling many others… but read on. Let’s start with more on eteplirsen, and round up the rest of the week’s biggest news.

—In a controversial decision with potential consequences for the future regulation of medical treatments, the FDA gave accelerated approval to the first drug for Duchenne muscular dystrophy, eteplirsen, from Cambridge, MA-based Sarepta Therapeutics (NASDAQ: SRPT). Shares of Sarepta boomed and the Duchenne patient community cheered. But documents released by the FDA showed that eteplirsen was only approved after Janet Woodcock, the FDA’s top drug evaluator, went against the recommendations of outside experts and the agency’s own scientists and reviewers. Her colleagues were critical of the drug’s data and fearful of the precedent it might set for drug approvals and the power of patient lobbying. Sarepta said it expects to launch eterlipsen at an average annual price of $300,000 per patient, a figure not likely to be challenged by insurers.

—Great Britain’s GlaxoSmithKline (NYSE: GSK) became the first big drug firm to appoint a woman as CEO. Emma Walmsley will succeed Andrew Witty after running GSK’s consumer healthcare business for five years.

—Irish-domiciled Allergan (NYSE: AGN) kept up its year-long shopping spree, placing bets on drugs to treat the liver disease nonalcoholic steatohepatitis—a severe type of fatty liver brought on by obesity, in large part. It’s a growing problem, prompting Allergan to commit up to $1.7 billion for Bay Area biotech Tobira Therapeutics (NASDAQ: TBRA) despite shaky Phase 2 data for Tobira’s most advanced NASH drug. Allergan also bought Akarna Therapeutics, another NASH drug developer, for a much smaller sum.

—Facebook founder Mark Zuckerberg and his wife Priscilla Chan, a pediatrician, said the Zuckerberg-Chan Initiative, a holding company for their Facebook fortune, would invest $3 billion to “cure, prevent, or manage all diseases” by the end of the century. The first concrete step is $600 million to establish a “biohub” in San Francisco that will bring together researchers from Stanford University, and the University of California campuses in San Francisco and Berkeley.

—Bill Gates, who has turned his own tech wealth into the world’s richest charitable foundation, was in San Francisco to cheer on Zuckerberg and Chan. His former company Microsoft made its own grand health pronouncement this week: a computational-intensive push to “solve” cancer in ten years, leading one participant to predict a “century free of cancer.”

—Privately held Tarveda Therapeutics, of Watertown, MA, could pay up to $163 million to Madrigal Pharmaceuticals (NASDAQ: MDGL) to acquire the old cancer work of Synta Pharmaceuticals, which merged with Madrigal in April after Synta’s lead drug failed in clinical trials.

—Israel’s Teva Pharmaceutical (NYSE: TEVA) paid Regeneron Pharmaceuticals (NASDAQ: REGN) of Tarrytown, NY, $250 million upfront for partial rights to fasinumab, an experimental drug for chronic pain. Teva and Regeneron are chasing a rival drug from Pfizer and Eli Lilly. Both represent an alternative to addictive opioids but have been dogged by safety problems.

—Roche’s Genentech division is paying Germany’s BioNTech $310 million in upfront and near-term milestones to co-develop cancer vaccines.

—Trials and tribulations: Gilead Sciences (NASDAQ: GILD) of Foster City, CA, is ending a Phase 2/3 trial of an ulcerative colitis treatment because it isn’t helping patients… Mast Therapeutics (NYSEMKT: MSTX) announced its sickle cell treatment vepoloxamer failed a Phase 3 trial… Shire wants to dump its interest in pacritinib, a failed myelofibrosis drug from Seattle’s CTI Biopharma (NASDAQ: CTIC), that it inherited when it bought Baxalta this year.

—Following the failure of its lead eye-disease drug, Cambridge-based Eleven Biotherapeutics (NASDAQ: EBIO) acquired Toronto’s Viventia Bio in an all-stock deal and rebranded as a cancer drug developer.

—Biotech build-outs: Cambridge-based Moderna Therapeutics will spend $110 million to build a 200,000 square foot messenger RNA drug manufacturing facility in Norwood, MA, that should open by early 2018… Nearby Alnylam Pharmaceuticals (NASDAQ: ALNY) just opened a 100-employee commercial hub in the U.K.

—The Medicines Co. (NASDAQ: MDCO), of Parsippany, NJ, was awarded up to $132 million from the U.S. Biomedical Advanced Research and Development Authority to develop antibiotics to fight drug-resistant bacteria.

—Swiss Alzheimer’s drug developer AC Immune (NASDAQ: ACIU) raised $66 million before fees in its Nasdaq IPO… Durham, N.C.-based skin drug developer Novan (NASDAQ: NOVN) raised $45 million in its IPO. Xconomy’s Frank Vinluan profiled the company and its IPO plans last month.

Ben Fidler contributed to this report.

Photo of the U.S. Capitol courtesy of Kevin Dooley via Creative Commons.