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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.

TOP STORIES

—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 … Next Page »

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