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A biotech’s binary bet ends badly

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Good morning! Elaine Chen is away, so I’m here in her stead. Take it slow out there, Northeast-ers, the outside temps are about to hit “wicked hot.”


The need-to-know this morning

  • Zentalis Pharmaceuticals said the FDA placed a partial hold on clinical trials involving its experimental cancer medicine azenosertib following the deaths of two patients due to “presumed sepsis.”
  • Intra-Cellular Therapies reported positive results from a second, Phase 3 clinical trial seeking to expand the use of its antipsychotic medicine Caplyta to patients with major depressive disorder. Positive results from the first Phase 3 study were reported in April.
  • Taysha Gene Therapies reported preliminary results from a study of  its gene therapy for adults and children with Rett syndrome.

Ethan Weiss has raked in some cash 

Now we know what the former UCSF cardiologist and frequent Twitter/X contributor has been doing for the past few years: starting up a biotech company.

The 10-person outfit called Marea said this morning that it had raised $190 million to help develop a drug to lower a type of cholesterol — what scientists call “remnant cholesterol” — that could represent a new way of attacking cardiovascular disease. In addition to being a founder, Weiss is Marea’s chief scientific officer.

The story of that drug, the protein that it targets, and the gene that codes for that protein (both are called ANGPTL4) says a lot about why medicines are so tough to develop and why heart disease remains the biggest killer in the U.S., taking 700,000 lives every year. It also provides a freeze-frame photo of the way in which the biotech industry’s capitalist flywheel of boom and bust cycles provides glimmers of scientific hope, writes my STAT colleague Matt Herper.


Marea, which is about three years old, is still at the beginning of its scientific journey. Its drug, initially developed at Novartis and then licensed to the startup, is only about to enter Phase 2 trials.

Read more.

A biotech’s binary bet ends badly

Shares of Aerovate Therapeutics plunged 93% yesterday, for a straightforward reason. A biotech company that bets its entire future on a single drug doesn’t have much of a future when that drug turns out not to work.

In a mid-stage clinical trial, Aerovate said its lead and only drug candidate, called AV-101, failed to reduce blood pressure in the lungs compared to a placebo in patients with pulmonary arterial hypertension. Based on the negative results, which extended to secondary endpoints as well, Aerovate said it is halting enrollment and shutting down an ongoing Phase 3 study.

Aerovate was founded to develop AV-101, an inhaled formulation of the cancer medicine imatinib repurposed for the treatment of pulmonary arterial hypertension. An oral form of imatinib had shown promising efficacy in previous studies, but its development was blocked by intolerable side effects. Aerovate believed delivering imatinib directly into the lungs might be the way forward — but yesterday’s study outcome proved otherwise.

The bad news caused Aerovate’s stock price to plunge to $1.65 yesterday from its Friday close of just below $25. Its market value ($49 million) is now less than the $100 million on its balance sheet.

Merck wins approval for pneumonia vaccine

The Food and Drug Administration yesterday approved Merck’s new pneumococcal vaccine for adults 18 and older.

The vaccine, which will be sold under the name Capvaxive, is designed to protect against pneumococcal pneumonia, which hospitalizes about 150,000 adults in the United States every year and kills about one in 20 who develop it, according to the National Foundation for Infectious Diseases.

Capvaxive covers 21 different serotypes of Streptococcus pneumoniae — the bacteria that cause pneumococcal disease — including eight that are not targeted by any of the other available vaccines. Until now, the vaccine that protected against the most serotypes of the bacteria was Pfizer’s Prevnar 20.

Read more.

Gilead Sciences: Obesity stock?

We’ve reached the stage of the GLP-1 hype cycle where a research abstract describing modest changes in the weight of monkeys is sufficient to label a large-cap biotech company as having “exposure to obesity now.”

That’s how Jefferies biotech analyst Michael Yee touted Gilead in a note Friday, highlighting a preclinical metabolic drug called GS-4571 that the company has never mentioned publicly. The company is presenting monkey data on GS-4571, an oral GLP-1, at the upcoming meeting of the American Diabetes Association. The discovery of the research abstract on Friday by Yee and some other analysts was enough to cause Gilead’s stock price to close 2% higher.

But later, Gilead management clarified that there’s been no decision on whether to advance GS-4571 into Phase 1 studies, and that the drug, for now, might be under consideration as a potential treatment for the fatty liver disease known as MASH.

Tap those Gilead obesity brakes, for now.

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