Bio Roundup: Trumpcare Flails, Spinraza Sales, Batten OK & More

Xconomy National — 

To judge the first 100 days of a new administration is, as many pundits have pointed out, an odd vestige of history, more convenient than significant. Which is why we’ll start this week’s roundup with our 99-day evaluation of the Trump administration—at least when it comes to healthcare and the life sciences.

In healthcare, nothing the administration has done is starker than its failure so far to repeal and replace the Affordable Care Act, a.k.a. Obamacare, perhaps the signature promise of Trump’s presidential campaign. The Republican House plan, the American Health Care Act, had Trump’s approval but died in the face of opposition from conservative and moderate Republicans before it could come to a vote. Moves were afoot this week to try again, with concessions to conservatives but not moderates. As of late Thursday, those efforts appear to be shelved, too.

Trump has followed his campaign rhetoric to let Medicare negotiate drug prices with harsh words for high costs. But it’s not clear how his administration intends to lower costs. His pick to run Medicare and Medicaid, Seema Verma, and her boss, the Health and Human Services secretary Tom Price, seem far more likely to pare back federal involvement in healthcare than to give the government broad new powers to negotiate lower costs with its vast purchasing leverage.

The soon-to-be FDA commissioner Scott Gottlieb could be more of a wild card. If the administration wants to drastically shrink the FDA in size and scope, as Trump has threatened, there were few indications in Gottlieb’s confirmation hearing that he would be a willing water-carrier—or that he, trained as a physician, had much patience with anti-vaccine theories that Trump has encouraged.

Scientists of all stripes see the administration as a threat not just to funding—as the White House’s budget proposals have made clear—but to their world view. People marched in hundreds of cities last Saturday (Earth Day) to protest, and organizers spent this week reminding people that marching is not enough.

When it comes to developing a new medicine, 100 days is the blink of an eye. Let’s start this week’s roundup with news from drug development programs that have been years in the making, or have years to go.


—Biogen (NASDAQ: BIIB) reported better-than-expected sales for its closely watched spinal muscular atrophy drug, nusinersen (Spinraza). But executives noted that it remains difficult for patients to gain access to the pricey drug. New data disclosed at the American Academy of Neurology meeting this week could help ease those bottlenecks.

—The nusinersen story, and the role that patients and their advocates have played, will be a big topic of discussion at our “What’s Hot in Boston Biotech” event on May 11. You can get your tickets here.

—Cholesterol-lowering drugs known as PCSK9 blockers have been commercial busts so far, but The Medicines Co. (NASDAQ: MDCO) and Alnylam Pharmaceuticals (NASDAQ: ALNY) are charging ahead into a big Phase 3 program with their own version. The two outlined the trials, which include a 14,000 patient cardiovascular “outcomes” study, this week.

—NGM Bio of South San Francisco, CA, joined the scrum of drug companies looking to treat the liver disease nonalcoholic steatohepatitis, or NASH. Its Phase 2 data showed a reversal in one factor of the disease and offered hints that other factors were reversed. Liver doctors were encouraged but cautious about the limited data.

—Other NASH news: Bristol-Myers Squibb (NASDAQ: BMY) also reported positive Phase 2 data; French firm Genfit is enrolling patients for its Phase 3 study at a slower clip than expected, according to FierceBiotech.

—Hammered by two study failures this month, Redwood City, CA, cancer drug developer OncoMed (NASDAQ: OMED) said it would lay off half its workforce and focus on earlier- stage compounds in its pipeline.


—The Senate Health, Environment, Labor & Pensions committee voted 14-9 to send Scott Gottlieb’s nomination for FDA commissioner to the full Senate for a final vote. The vote was along party lines save for two Democrats who supported Gottlieb.

—BioMarin Pharmaceutical (NASDAQ: BMRN) notched FDA approval for cerliponase alfa (Brineura), the first drug ever to treat the rare genetic Batten disease. BioMarin announced an annual list price of $702,000. European regulators have seemed favorable to the drug, as well, and will issue a final decision by the end of June.

—The FDA also approved the use of regorafenib (Stivarga) from Bayer HealthCare to treat advanced liver cancer. Regorafenib is already approved for stomach and colon cancer.


—-Becton Dickinson (NYSE: [[ticker: BD]]), the Franklin Lakes, NJ-based medical technologies company, announced it would acquire Murray Hill, NJ-based medical device maker C.R. Bard in a $24 billion deal intended to diversify its portfolio and expand its international footprint.

—Cambridge, MA-based Akebia Therapeutics (NASDAQ: AKBA) expanded its partnership with Otsuka Pharmaceutical that was originally signed last year. Otsuka is paying $208 million for expanded rights to the experimental anemia pill vadadustat in Europe, China, and other countries. Akebia could receive $865 million more in downstream payments. Its shares were up more than 33 percent.

—Amgen (NASDAQ: AMGN) reworked its alliance with Novartis (NYSE: NVS) on erenumab, an experimental drug that could soon be the first of a wave of new therapies meant to prevent migraines. Novartis now shares U.S. rights to the drug with Amgen, which as FierceBiotech explains here, analysts took as a sign that Amgen is hedging its bet on the drug’s market potential.

—Cambridge-based Agios Pharmaceuticals (NASDAQ: AGIO) licensed a group of potential cancer drugs from Aurigene Discovery Technologies for $3 million up front.


—The Bill & Melinda Gates Foundation led a $45.5 million Series D round for Waltham, MA-based Arsanis, developer of antibodies for dangerous bacterial and viral infections co-founded by serial entrepreneur Tillman Gerngross.

—The Gates Foundation also said it was working on a nonprofit medical research institute, to be based in Seattle and Boston, to work on treatments for diseases in the developing world.

—San Diego-based Forge Therapeutics raised a $15 million Series A round to bring into clinical trials a drug that could treat drug-resistant infections.

—Sienna Biopharmaceuticals of Westlake Village, CA, raised a $40 million B round to apply to its pipeline of drugs for psoriasis and other skin conditions.

—Bay Area firm BridgeBio Pharma, which creates companies mainly around single drug assets, rolled out its latest, Eidos Therapeutics, with $27 million in funding. Eidos is built around a drug for the rare transthyretin amyloidosis, in which misfolded proteins build up in the heart and nerves.


—After steering Sarepta Therapeutics (NASDAQ: SRPT) towards the controversial first-ever approval for a Duchenne muscular dystrophy drug, president and CEO Ed Kaye will resign on Sept. 20 “or some other future date,” according to a regulatory filing. Kaye will stay on as a board member and advisor to Sarepta afterwards.

—For the first time, Cambridge-based Moderna Therapeutics published data, in humans, for one of its messenger RNA drug therapies—a new and unproven type of drugmaking. The study, published in the journal Molecular Therapy, tested an mRNA vaccine for the flu virus in 23 human subjects. The Wall Street Journal has more on the results here.

Ben Fidler and Frank Vinluan contributed to this report