September 26, 2023

The need-to-know this morning

Intercept Pharmaceuticals agreed to sell itself to the Italian firm Alfasigma for about $850 million.
Immunovant rose about 50% in premarket trading after the company's subcutaneous autoimmune treatment met its goals in an early-stage trial. Roivant Sciences, the firm's largest shareholder, rose about 10%.
Ionis Pharmaceuticals' treatment for familial chylomicronemia syndrome, a rare and serious disease, succeeded in a Phase 3 study.

Chart of the day

Turns out it could get worse

D3 vis exported to PNG (71)

Last week brought rending of garments and gnashing of teeth around the sorry state of biotech stocks. This week, things got that much sorrier as the closely watched XBI index hit its lowest point in 2023, down 12% since the start of the year.

Across the industry yesterday, companies with no news to speak of saw their share prices tumble. Sarepta Therapeutics fell 4%, while Viking Therapeutics dropped 10%, and Verve Therapeutics slumped 5%.

Investors and analysts who have endured a few biotech booms and busts are always quick to point out that these things are cyclical. History suggests all it takes is a little generalist interest — whether brought on by genomics, CRISPR, or a full-fledged pandemic — to dramatically turn things around. But with a macro environment that remains unfriendly to risky propositions like developing new drugs, there's an ambient fear that the last biotech boom, like cartoon apes and the entire metaverse, might have been a zero interest-rate phenomenon.

Regulatory

Is there any way NurOwn wins FDA approval?

Before yesterday, we knew the FDA took such a dim view of NurOwn, BrainStorm Cell Therapeutics' investigational ALS treatment, that it previously refused to even consider approving it. Now, thanks to agency briefing documents, we know the FDA also has grave concerns about whether BrainStorm can safely and consistently manufacture the product.

With all that baggage, is there a path for BrainStorm and its many supporters to win over panelists at tomorrow's hearing and convince the FDA to approve NurOwn anyway?

Wall Street would seem to think not. Yesterday's document release sent BrainStorm shares down about 40% to below 50 cents, the lowest price in nearly a decade. And yet the company's dire financial situation could work in NurOwn's favor. Outright rejection would likely put BrainStorm on the path to insolvency, making it impossible for the company to conduct the follow-up clinical trial physicians and scientists say NurOwn merits. The specter of bankruptcy, combined with pressure from patients and advocates, could convince the FDA to find a way forward for BrainStorm's embattled medicine.

Personnel

Madrigal picked an awkward time to change leadership

Madrigal Pharmaceuticals, whose treatment for the liver disease NASH is under FDA review, departed with its second C-suite leader in as many weeks.

The company said yesterday it is parting ways with its chief commercial officer, who joined in 2020, in a case of "termination without cause." Earlier this month, Madrigal appointed a new CEO in the form of Sanofi veteran Bill Sibold, who replaced Paul Friedman after seven years on the job.

The timing seems less than ideal. Earlier this month, the FDA accepted Madrigal's approval application for resmetirom, the NASH drug, and the agency set a decision date of March 14. The company is potentially on the verge of launching its first product into what is expected to be a blockbuster market, which is a curious occasion to change leadership.

Pharma

Pfizer's tornado-damaged plant is back up

Pfizer said it has restarted the majority of its manufacturing at the North Carolina plant that was heavily damaged by a tornado in July.

The facility, which produces about a quarter of the sterile injectables Pfizer sells to U.S. hospitals, will resume shipments in the fourth quarter of this year, the company said. It won't have enough inventory to meet full supply requirements until at least mid-2024.

Restarting production after a 10-week hiatus is "a proud achievement" for the company, Pfizer said. The North Carolina plant produces about 50 medicines, including anesthesia and micronutrients, and its output accounted for roughly 8% of the country's total hospital supply.

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