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Brainstorm Health: Sarepta Duchenne Therapy, CVS Drug Delivery, Trump’s New Obamacare Move

June 19, 2018, 8:51 PM UTC

Hello, readers—This is Sy.

Shares of biotech Sarepta Therapeutics shot off to the moon Tuesday after the company announced early data on its experimental gene therapy to treat Duchenne muscular dystrophy. Sarepta stock was up as much as 67% during trading, settling closer to a 35% bump later in the afternoon, or just under $140 per share.

The investor frenzy was galvanized by some promising, though very preliminary, data presented at the firm’s first-ever R&D day. Sarepta’s treatment, for now dubbed AAVrh74.MHCK7.micro-Dystrophin—yes, you read that right—was reportedly able to dramatically cut the levels of an enzyme called creatine kinase (CK) associated with muscle damage while also significantly boosting the production of micro-dystrophin, which assists basic movement functions, in the patients who took it. Duchenne muscular dystrophy is a nasty, muscle-wasting disease that usually claims its victim’s lives by their mid-20s, eating away at their muscles by barring the production of dystrophin.

“I have been waiting my entire 49-year career to find a therapy that dramatically reduces CK levels and creates significant levels of dystrophin,” said Dr. Jerry Mendell of Nationwide Children’s Hospital said in a statement announcing the early stage gene therapy clinical trial results. “Although the data are early and preliminary, these results, if they persist and are confirmed in additional patients, will represent an unprecedented advancement in the treatment of DMD.”

A number of biopharma analysts and medical experts were stunned by Sarepta’s preliminary results themselves. “In biotech sometimes we get to witness history unfolding. Man, how technologies are improving so quickly,” tweeted Brad Loncar, CEO of Loncar Investments and an avid biotech industry watcher. JPMorgan analyst Anupam Rama said the results “meaningfully beat even the highest of expectations.”

Mendell’s point about the results’ preliminary nature is also worth keeping in mind. This early gene therapy study involved just three patients—although, granted, trials for rare disorders like Duchenne muscular dystrophy have very limited patient pools. But Sarepta also has a bit of a controversial history at the Food and Drug Administration (FDA). Its pioneering approval for a different muscular dystrophy treatment called Exondys 51 drew plenty of blowback from critics (and was cleared by a razor thin margin despite the initial objections of the FDA’s own advisers) who argued it didn’t show significant benefit.

An initial reading of the tea leaves suggests some more optimism about Sarepta’s new Duchenne gene therapy. The question is whether or not the early results will persist.

Read on for the day’s news.

Sy Mukherjee


CVS launches nationwide drug delivery. On Tuesday, CVS announced it will launch prescription drug and medication delivery across the U.S. That means, in certain towns, same-day drug delivery; in others, customers will be able to get their orders within a day or two. One potential reason for the move: The (still hazy) specter of one "Amazon." (Fortune)

Social media's health... benefits? There have been countless studies about the mental dangers of excessive social media use. But what if all that browsing could actually have a positive effect? New NIH-funded research being presented Tuesday finds that more social media use (including texting and scrolling through Instagram) was associated with "increased physical activity, less family conflict, and fewer sleep problems," NPR reports. (NPR)


Roche snaps up the rest of Foundation Medicine. Swiss drug giant Roche is going all in on Foundation Medicine, the cancer genomic testing outfit the firm already had a considerable stake in. Roche will shell out $2.4 billion to buy the rest of the company, which it hopes will boost its aggressive recent efforts in personalized cancer care. Earlier this year, Roche purchased Flatiron Health, a cancer data collection and analytics company the company bets can improve its drug development supply chain in a big way. (Reuters)


Trump's latest blow to Obamacare: Association health plans. The Trump administration is finalizing rules for certain kinds of insurance plans that experts say will further undermine the Affordable Care Act, potentially leading to higher premiums. “For the first time ever, sole proprietors will be able to buy lower-cost group insurance instead of getting ripped off by this disaster we all know as Obamacare,” President Trump said during a speech at the National Federation of Independent Business (NFIB). In essence, the rules would allow small businesses to join forces with trade groups to buy insurance plans outside of Obamacare marketplaces; since the regulations on them would be less stringent than those imposed on ACA plans, critics say it could continue a trend of healthy, younger people flocking to other markets (and thereby raise the costs of the sick and needy in the Obamacare exchanges). (Politico)


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Produced by Sy Mukherjee

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