September 12, 2023

The need-to-know this morning

2Seventy Bio CEO Nick Leschly is resigning as part of a broader restructuring plan that will also see 40% of the cancer biotech's workforce terminated and its research pipeline trimmed in order to reduce costs. The company was spun out of gene therapy maker Bluebird Bio in 2021.
Neurocrine Biosciences said its experimental drug called crinecerfont achieved the goals of a Phase 3 study in adult participants with congenital adrenal hyperplasia, a genetic disorder that affects the production of adrenal hormones.
Lyra Therapeutics said an experimental treatment for chronic rhinosinusitis achieved the goals of a Phase 2 study.
Biomarin Pharmaceuticals is holding an R&D day for investors and analysts.
Under a transfer agreement reached with Orchard Therapeutics, the Italian biomedical charity Fondazione Telethon will provide the gene therapy Strimvelis to eligible people with an ultra-rare immunodeficiency disorder. Strimvelis was approved by European regulators in 2016 but Orchard's commercial launch was unsuccessful, forcing the company to divest.

Biotech

An IPO starlet stumbles

Acelyrin, one of just six biotech companies to go public in 2023, won a roughly $2.5 billion valuation on the promise of a potential blockbuster treatment for autoimmune disease. The first major test of that idea ended in disappointment.

The company's drug, a subcutaneous treatment called izokibep, missed its primary endpoint in a study enrolling patients with the common skin disease hidradenitis suppurativa, an inflammatory disorder that leads to abscesses and scars. The issue, according to Acelyrin, could be related to patients dropping out early after seeing their skin lesions clear up, which, combined with unexpected placebo responses, might have skewed the data.

Izokibep is still being studied in a hidradenitis suppurativa Phase 3 trial, and Acelyrin is developing it for other autoimmune diseases including psoriatic arthritis, the spinal disorder axial spondyloarthritis, and the inflammatory ocular disease uveitis. But the first stumble, which sent the company's stock price down more than 60%, might remind investors why risky biotech IPOs fell out of favor in the first place.

Read more.

Obesity

There are more than 70 would-be Wegovys

And keeping tabs on all of them is becoming a pain.

Thankfully, you've got the STAT Obesity Drug Tracker, our just-launched tool for tracking the many next-generation weight-loss medicines that aim to mimic, complement, or even unseat Novo Nordisk's Wegovy and Eli Lilly's Mounjaro.

About two-thirds of them use similar biological mechanisms, targeting the hormones that police cravings, but some are pursuing entirely novel ideas that, if they work, will push the science of obesity forward.

Check it out.

Regulatory

You're not going to believe what happened to Novavax

Novavax, whose potent Covid-19 vaccine all but missed out on the peak of the pandemic, is staking its future on selling booster doses into a shrinking market. But the first step of the process — winning FDA approval for an updated vaccine — didn't quite go to plan.

As STAT's Helen Branswell and Matthew Herper report, the FDA approved reformulated vaccines from Moderna and partners Pfizer and BioNTech, based on data that they work against novel variants of the virus. Missing from the announcement was Novavax, whose booster was also up for approval. The company "is currently responding to the FDA's requests to facilitate final review, and timing is ultimately at the discretion of the FDA," Novavax said in a statement, adding that it still expects its booster to be "widely available this September for consumers."

The company's long-suffering shareholders can be forgiven for feeling like they've heard this one before. Throughout 2021, Novavax repeatedly made and broke promises to speedily commercialize what was an unquestionably effective vaccine. Now, facing the legitimate threat of bankruptcy, Novavax needs to capitalize on the market for boosters, and it has already missed one milestone.

Pharma

AstraZeneca vs. the Daily Mail

AstraZeneca has long been the envy of pharmaceutical giants, rising from an also-ran in oncology to a world-leading drugmaker getting standing ovations at cancer conferences. That success has sowed a new anxiety: When is Pascal Soriot, CEO for the last decade, going to leave the company?

The latest flashpoint came yesterday, when AstraZeneca's shares fell as much as 4% on a Daily Mail story reporting that Soriot has "privately told friends and trusted advisers" that he's looking to leave the company as early as next year, something he has not shared with the board. In a statement, AstraZeneca said it does not comment on market speculation but that if there were truth to a rumor that would affect its stock price, the company would be legally required to make an announcement.

One could read between the lines and conclude both that AstraZeneca is telling the truth — it has no knowledge of plans for Soriot to leave — and that the Mail is correct — he hasn't told his employers about those plans. Regardless of what Soriot decides, the stock reaction is a little curious, as it suggests he is such a good CEO that the company will be worth less without him, but also that he's not such a good CEO that he would have installed an infrastructure that can thrive after he leaves.

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