The Readout Damian Garde

What to make of this whole Sarepta situation

Sarepta Therapeutics’ shares are down about 15% since last week after a confusing entry in an FDA database snowballed into concerns that the company’s next big idea might get derailed by a safety problem.

STAT’s Adam Feuerstein sat down to unpack the knowns, unknowns, and unknown unknowns tied to Sarepta’s gene therapy for Duchenne muscular dystrophy. That means an explanation of the FDA angle, a discussion of who knew what when, and all-important context for a fast-moving space.

Some highlights: Yes, this is serious. No, it’s not damning. Yes, Sarepta found out when you did. No, this isn’t the work of a dastardly cabal of short-sellers bent on keeping a good company down.

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The under-the-radar Alzheimer’s company worth $800 million

Cortexyme spent the first five years of its life in stealth. Then, in 2019, the company’s scientific founders published a head-turning paper on the potential cause of Alzheimer’s disease and promptly raised $71 million in an IPO.

Now, as CEO Casey Lynch explained to STAT’s Sharon Begley, Cortexyme is spending its money on a clinical trial that will put its hypothesis to the test. 

The company is founded on the idea that P. gingivalis, a bacteria tied to tooth decay, plays a role in the development of Alzheimer’s. Cortexyme’s drug, COR388, is meant to block the bacteria’s effects on neurons, and the company is enrolling 570 patients with mild to moderate Alzheimer’s to see if the therapy can change the course of the disease.

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Shanghai's first biotech IPO brought a lot of zeroes

Yesterday, Shanghai’s newfangled STAR Market hosted its first biotech IPO, and it brought about some extraordinary numbers. Shenzhen Chipscreen Biosciences opened up more than 500%, according to BioCentury, reaching a $5.5 billion valuation by the close.

That’s good for Chipscreen’s private backers, who are looking at an exponential return on their investment. But it’s arguably bad for the Shanghai exchange. All the handwringing about biotech coming to markets in China stemmed from an assumption, perhaps unfair, that bankers, analysts, and investors there didn’t have the expertise needed to sort good ideas from bad and thus settle on rational valuations.

Chipscreen’s five-fold jump suggests that either the bankers didn’t know enough about the company to price the shares correctly or the investors didn’t know enough to not overpay for them. Either way, it doesn’t exactly bode well for the development of a stable marketplace.

Regeneron is good at making antibodies

Back in 2015, as an Ebola virus outbreak killed thousands in West Africa, Regeneron Pharmaceuticals tapped its vaunted antibody-discovery technology and rush-ordered a three-antibody cocktail that might treat infection. Yesterday, we found out it worked.

In preliminary results, more than 70% of Ebola patients who got Regeneron’s antibodies survived, compared to about 50% of those who got ZMapp, which has been in use since 2015, STAT’s Helen Branswell reports.

What’s interesting is the difference in tone between public health authorities, who were effusive about the promise of Regeneron’s therapy, and the analyst community that has found the company’s recent news to be underwhelming. The scientific engine behind that Ebola treatment once made Regeneron the toast of biotech, but recent years have been leaner, and now the company needs prove it has a second act “to justify the stock’s return to its former lofty perch in the sector,” as SVB Leerink analyst Geoffrey Porges wrote in a note to investors last week.

More reads

  • Novartis benefits from faster China drug approvals. (Financial Times)
  • Few Americans took obesity drugs thanks to doctor doubts and spotty insurance. (STAT Plus)
  • Better birth control could exist, but it wouldn’t pay for Big Pharma. (Bloomberg)

Thanks for reading! Until tomorrow,


Tuesday, August 13, 2019


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