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Bio Roundup: Graham-Cassidy, Axovant Fail, SpringWorks Launches & More

Xconomy National — 

After drug pricing, count drug shortages among the hot button issues sparking patient ire. Last year, the FDA says, 23 drug shortages were reported, down from 26 the previous year (and 251 in 2011). But Hurricane Maria’s direct strike on Puerto Rico might put the progress made on reducing shortages to the test.

The Pharmaceutical Industry Association of Puerto Rico lists 22 manufacturing sites on the island, operated by companies such as Amgen (NASDAQ: AMGN), Pfizer (NYSE:PFE), Bristol-Myers Squibb (NYSE: BMY), and Johnson & Johnson (NYSE: JNJ). Companies have avoided sounding alarms; Amgen, for one, said its drug supply shouldn’t be affected because the company’s existing inventory can meet demand.

By some estimates, Puerto Rico’s power won’t be restored for months, projections that might be rousing more concern from the FDA. The agency said it is aware of “instances where we may soon face critical shortages if we don’t find a path for removal or ways to get production back up and running.”

In other life sciences news, Pfizer blazed a new path for shelved drugs, the biotech IPO window remains open, and a much hyped Alzheimer’s drug failed. Let’s round up this week’s news.


—The White House’s opioid epidemic commission, chaired by New Jersey governor Chris Christie, reached an agreement with the drug industry to limit painkiller prescriptions to a seven-day supply, and to fast-track the development of non-addictive alternatives. The Trump administration has yet to declare the crisis a national emergency, although the commission has recommended that action.

—The Republican quest to replace the Affordable Care Act, aka Obamacare, foundered yet again. For various reasons, several GOP senators opposed the Graham-Cassidy bill that aimed to turn federal funding for state Medicaid programs into block grants. The bill would have given states the option to allow higher insurance rates for people with pre-existing conditions and, according to a preliminary Congressional Budget Office scoring, would have booted “millions” of people off Medicaid.


—Axovant Sciences (NASDAQ: AXON) reported bad news for its experimental Alzheimer’s treatment intepirdine. Combined with generic memory booster donepezil (Aricept), it did no better at helping patients with cognition and other daily functions than donepezil did alone. It’s the latest in a string of Phase 3 Alzheimer’s drug failures.

—An FDA advisory panel voted 10 to 1 that the data were inconclusive as to whether PTC Therapeutics’ (NASDAQ: [[ticker:PTCT) drug ataluren (Translarna) can help treat Duchenne muscular dystrophy, and needs more testing. The FDA has twice rejected ataluren, but PTC used a rarely used regulatory tool to force another review.

—Dupilumab (Dupixent) from Sanofi (NYSE: SNY) and Regeneron Pharmaceuticals (NASDAQ: REGN) received European approval to treat eczema, adding to the drug’s U.S. approval earlier this year.

—The FDA approved Abbott Laboratories’ (NYSE: ABT) glucose monitoring system, the agency’s first approval for such a device that does not require a blood sample from a fingerstick.

—The FDA rejected Intarcia Therapeutics’ implantable drug-device for type 2 diabetes, ITCA650, citing manufacturing problems. The Boston company doesn’t believe it needs to run any new clinical studies to satisfy the FDA and win approval.

—A weight loss pill from Gelesis posted mixed results in a Phase 3 trial, hitting on one main trial goal and missing on another. Gelesis, a startup from Boston company creator PureTech Health, still plans to discuss potential approval filings with regulators.

—Eli Lilly (NYSE: LLY) won FDA approval for breast cancer drug abemaciclib (Verzenio), to be used with the endocrine therapy fulvestrant.


—With its cash reserves already dwindling, Genocea Biosciences (NASDAQ: GNCA) couldn’t find the financing or partnership needed to advance its experimental genital herpes vaccine, GEN-003, into Phase 3 testing. So Genocea will now seek “strategic alternatives” for GEN-003, cut its workforce by 40 percent, and focus on its preclinical cancer vaccine work instead. That series of moves didn’t stop its shares from falling more than 76 percent to $1.25 apiece, their lowest levels ever.

Analytics 4 Life raised $25 million in a Series B funding round. The startup, with locations in Toronto and Research Triangle Park, NC, has developed a medical device that reads the heart’s electrical activity in order to form an image.

SpringWorks Therapeutics launched with $103 million in funding and four experimental drugs whose development had been stalled at Pfizer. SpringWorks aims to develop experimental therapies that pharma companies leave on the cutting room floor, specifically for diseases with no approved therapies or little competition. Xconomy spoke with ex-Pfizer and Alexion Pharmaceuticals vets Lara Sullivan and Saqib Islam about the effort.

—In a regulatory filing, Acorda Therapeutics (NASDAQ: ACOR) said it can address the FDA’s refusal to review experimental Parkinson’s drug Inbrija, and file a new approval application, without scheduling a meeting with the agency or starting a new clinical trial.

—Waltham, MA, cancer drug developer Deciphera Pharmaceuticals (NASDAQ: DCPH) raised $128 million in an upsized IPO, one of three biotech IPOs this week.

—The Biomedical Advanced Research and Development Authority (BARDA) awarded South San Francisco biotech Achaogen (NASDAQ: AKAO) an $18 million contract to develop drugs that treat extended spectrum beta-lactamase (ESBL)-producing Enterobacteriaceae, a nasty pathogen deemed a critical priority by the World Health Organization because it can be deadly in people who have weakened immune systems.

—GRO Biosciences, the latest startup from the labs of Harvard University genetics professor George Church, raised $2.1 million in seed funding led by Digitalis Ventures. Boston-based GRO is developing genetically altered bacteria that are meant to help boost the potency and durability of protein drugs.


—Xconomy announced the winners of its inaugural Xconomy Awards for the Boston life sciences community. Congratulations to all!

Alex Lash and Ben Fidler contributed to this report. 

Photo by Flickr user Antti Lipponen via a Creative Commons license.