September 28, 2023

The need-to-know this morning

Karuna Therapeutics submitted its investigational schizophrenia treatment for FDA approval, a drug that would mark the first novel approach to the disease in more than 50 years.
Immunovant is raising $450 million in a private placement, suggesting the company, a recent fixture of M&A rumors, isn't up for sale.
A German court suspended CureVac's patent infringement lawsuit against BioNTech over the mRNA technology used in Covid-19 vaccines.

Regulatory

FDA advisers vote overwhelmingly against BrainStorm's ALS treatment

A panel of expert advisers to the FDA voted decisively against a polarizing potential treatment for ALS yesterday, concluding that the medicine's convoluted supporting data did not meet the standard for approval.

After a day-long meeting that included impassioned testimony from ALS patients, the agency's expert advisers voted 17-1 with one abstention that the clinical trial results for NurOwn, a treatment from BrainStorm Cell Therapeutics, did not live up to the compelling but ultimately unreliable anecdotal evidence.

"Patients and families need hope, but providing false hope can be ethically problematic," said panelist Lisa Lee, a bioethicist at Virginia Tech. "False hope is provided when the probability of a positive outcome is overestimated, and I think that seems to be the case here."

Read more.

Biotech

Biogen shutters digital health group

In 2021, Biogen launched a digital health division with a broad mission to work on technologies that might advance drug development and help drive outcomes for patients. Now, STAT's Mario Aguilar and Matthew Herper report, Biogen Digital Health is being shuttered.

Executives say the move — as well as a decision to end a clinical trial being conducted with Apple earlier than expected — doesn't signal an abandonment of digital health. But outsiders worry it could signal problems for health technology companies that are banking on big collaborations with pharmaceutical companies as a source of growth.

Among those affected by the decision is John Wilbanks, a well-known figure in the health tech world who was head of data at Biogen Digital Health. In a post on LinkedIn, he said in the future he'd have a lot to say about running data at a pharma firm. "The good news and the bad news are the same news, which is that it's cultural as much as it's technical," he wrote.

Venture

What is Novo doing with all that money?

It's a rather fortunate problem to have: One arm of your business is booming, and now, you are tasked with taking the influx of cash and investing it. But in this market, how do you best do that?

That's the question in front of the Venture Investments group at Novo Holdings, the parent company of pharmaceutical firm Novo Nordisk. Novo revenues are up some 30% — "probably the strongest growth in the history of the company," one executive said — thanks to Ozempic and Wegovy sales. The company is now worth more than the entire GDP of Denmark.

The influx of cash means more work for its investment team. Allison DeAngelis spoke to members of the venture team about the task ahead of them, and why they're wary of investing in more obesity companies.

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Don't call it 'warp speed'

The U.S. hasn't given up on next-gen Covid vaccines

The multibillion-dollar sprint to find a vaccine for Covid-19 showed the world just how powerful mRNA could be. The ensuing years of SARS-CoV-2 mutation have shed light on some of its limitations.

The U.S. government, which helped bankroll the development of Moderna's mRNA vaccine, is investing in what could be an evolutionary approach to preventing Covid-19. Through Project Nextgen, a federal effort to accelerate and streamline the rapid development of the new vaccines and treatments, the government is giving Gritstone Bio up to $433 million to conduct a 10,000-volunteer clinical trial testing a new approach to creating a vaccine.

Gritstone is using a different technology from its Covid-19 forebears, and while existing vaccines both use mRNA to make a part of the virus known as the spike protein, the company said it had identified other antigens that it thinks will make it more difficult for a viral strain to evade the immunity spurred by the vaccine.

Read more.

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