September 13, 2023

The need-to-know this morning

Moderna is holding its annual R&D Day for investors and analysts. Its flu vaccine candidate generated a better immune response than a currently available vaccine, meeting the goals of a Phase 3 study. The maker of mRNA-based medicines and vaccines said it expects to add $10-15 billion in annual sales five years after launching new products, starting in 2028. The company also reiterated its previous forecast of $8-15 billion in sales from Covid, flu and RSV vaccines by 2027.
Alnylam Pharmaceuticals is going before an FDA advisory panel today, arguing that its approved medicine can improve the lives of patients with an increasingly prevalent cardiovascular disease. You can watch it live here.

Vaccines

Covid boosters are a go

Thanks to a panel of advisers to the CDC, the latest updates to Covid-19 vaccines are slated to roll out across the U.S. later this week, launching a test of whether the market for boosters can endure in the fourth year of the pandemic.

As STAT's Jason Mast reports, the group voted 13-1 to recommend boosters from Moderna and partners Pfizer and BioNTech, both of which won FDA approval Monday. That same recommendation could apply to Novavax's updated shot, if and when the company wins an authorization of its own.

Meanwhile, Wall Street is still waiting to see whether the rapidly eroding business of selling Covid-19 vaccines can survive a shift in payers. Since 2021, the federal government has been the sole purchaser of doses in the U.S. Starting this winter, the onus will fall on private payers.

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Biotech

Neurocrine's diversification is paying off

While Neurocrine Biosciences made its name developing medicines for neurological diseases, the company's foray into rare genetic disorders has set in motion what could be its next approved drug.

As STAT's Jonathan Wosen reports, a Neurocrine medicine met its goal in a pivotal study enrolling patients with congenital adrenal hyperplasia, an umbrella term for a set of genetic mutations that halt the body's production of necessary hormones. In the 182-patient study, Neurocrine's drug significantly reduced the need for standard-of-care treatment compared to placebo.

The data are likely enough to win FDA approval, analysts said, and assuming the drug carries a list price of around $200,000 per patient, it could be a roughly $1 billion annual opportunity, TD Cowen analyst Phil Nadeau wrote in a note to clients. That would transform Neurocrine's business, which is almost entirely dependent on Ingrezza, a movement disorder treatment that brought in about $1.4 billion last year.

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Markets

Biotech can be a team sport

Two of yesterday's biggest movers on the biotech market added millions to their market caps without issuing so much as a press release about their in-development drugs.

Spruce Biosciences, a thinly traded firm, rose about 12% on the news of Neurocrine's aforementioned success in congenital adrenal hyperplasia, as the data left analysts from Leerink "increasingly encouraged" about the company's own investigational medicine in the same indication. And Moonlake Therapeutics jumped 10%, adding nearly $300 million to its valuation, thanks to Acelyrin's clinical setback in the inflammatory disorder hidradenitis suppurativa.

Financials

CAR-T might be a winner-take-all business

Consider Legend Biotech. The Chinese company commands a roughly $12 billion valuation thanks to an approved CAR-T medicine for multiple myeloma that has led to dramatic benefits. Then there's 2Seventy Bio, maker of a similar and similarly approved CAR-T treatment, which is struggling to stay afloat.

Yesterday, 2Seventy slashed sales projections for its CAR-T, cut about 40% of its staff, and parted ways with CEO Nick Leschly, who has led the company since its 2021 spinout from Bluebird Bio. The company is expecting lower revenue from its approved medicine, called Abecma, and is reducing spending on its pipeline in an effort to conserve enough cash to stay in business through 2026.

Part of 2Seventy's problem is that Legend's treatment, partnered with Johnson & Johnson, simply has stronger supporting evidence in favor. But the other is practical. Getting CAR-T — which requires harvesting healthy cells, off-site genetic engineering, and extensive pre-treatment — is not as simple as picking up a prescription or stopping by an infusion center, which makes it difficult for a smaller company to compete with a larger rival.

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