Where's the Sarepta data we were promised?

Back in 2016, when Sarepta Therapeutics won a controversial approval for a rare disease drug, it promised to run a study confirming the treatment’s benefits. Three years later, that trial is yet to treat a single patient.

As STAT’s Adam Feuerstein reports, Sarepta said it’s working as fast as possible. In the meantime, however, the company is reaping about $300 million a year from a Duchenne muscular dystrophy treatment whose clinical benefit has never been demonstrated.

One doctor who treats patients with the condition told STAT that some families have reported their children have received a benefit from Exondys 51; other families have not and have discontinued treatment.

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AveXis’s top scientists lost their jobs after Zolgensma data scandal

Yesterday, Novartis disclosed that it had replaced the top two scientists at AveXis, its gene therapy subsidiary, and a person with knowledge of the situation said their departure was tied to the data falsification scandal that became public last week.

The news only complicates the known timeline.

What we knew before was that AveXis became aware of data manipulation in March; Novartis executives learned in May; and the FDA found out in late June. What we just learned is that Novartis dismissed AveXis’s top scientists in May, meaning it knew enough then to make a personnel decision but, apparently, not enough to inform regulators about it.

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When one young-blood company closes, another opens

You may be familiar with the trials and tribulations of Ambrosia, the startup charging $8,000 for infusions of blood from young donors. That caught the attention of Stephen Colbert, and of the FDA, which issued a stern warning earlier this year that prompted the company to halt its operations. Now, Ambrosia appears to have shut down altogether.

That's according to a new story from Business Insider, which cited an email from the startup's founder, Jesse Karmazin. In its place, Karmazin reportedly wrote, he's started a new company to offer blood transfusions. Its name? Ivy Plasma, which could be easily mistaken for a college admissions consultancy.

This time around, the branding may focus less on blood that is youthful. Karmazin told STAT by email yesterday that Ivy Plasma won't be looking for donors of only specific ages to supply blood. "The age range is potentially anyone," he wrote.

Life comes at biotech startups fast

Last summer, Compass Therapeutics raised $132 million “to advance its next-generation antibody-based therapeutics into the clinic.” A year later, the company is dosing patients as planned, but it had to lay off 25% of its workforce along the way.

That’s one of many anecdotes in the latest iteration of STAT’s Something Ventured, a series that catches up with biotech startups after the gleam of their financing announcements has faded.

There’s also an update from a company out to build a better nasal spray, one trying to treat hearing loss, and another with a novel way of targeting proteins.

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Where's the Sarepta data we were promised?

AveXis’s top scientists lost their jobs after Zolgensma data scandal

In-depth biopharma, business, and policy analysis at your fingertips

When one young-blood company closes, another opens

Life comes at biotech startups fast

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