June 29, 2023

The need-to-know this morning

BioXcel Therapeutics said its treatment for agitation in people with Alzheimer's disease achieved the primary goal of a Phase 3 clinical trial.
But in a concurrent filing with the SEC, BioXcel disclosed that the lead investigator for the study, who enrolled 40% of the participants, may have failed to follow proper study procedures and allegedly submitted fake emails to the FDA regarding patient safety reports.
Eli Lilly is acquiring Sigilon Therapeutics, developer a cell therapy for type 1 diabetes, for $35 million. Sigilon shareholders will also receive a contingent value right for $275 million in potential future payments, based on the achievement of three development milestones.
An acrimonious proxy contest between current Alkermes management and activist fund Sarissa Capital, run by investor Alex Denner, comes to a head today. At its annual meeting, shareholders will vote on competing slates of directors for the drugmaker's board.

Business

Catalent is making the wrong kind of news

If you operate in the multibillion-dollar industry of providing contract services to major pharma companies, the path to success is one of quiet reliability, leaving the volatile business of drug development to your paying clients while you handle the predictable stuff for a fee. Catalent, a pharma contractor that has lost more than 60% of its value in the last year, has not been following that rubric.

The latest embarrassment relates to Regeneron Pharmaceuticals, whose closely watched eye drug got rejected by the FDA this week because of what the company called "inspection findings at a third-party filler." That third-party filler was Catalent, Bloomberg reported yesterday, marking the latest high-profile embarrassment for a company whose business relies on reputation.

A problem at the same facility constrained the supply of Moderna's Covid-19 booster shot in the fall, and Catalent was recently linked to shortages of Novo Nordisk's in-demand obesity treatment. Earlier this year, the company delayed reporting its quarterly results three times in less than a month to account for its mounting issues, eventually reducing its revenue projections for the second time in 2023.

Chart of the day

Is the year's biggest pharma deal in trouble?

D3 vis exported to PNG (61)

Shares of Seagen have fallen about 3% this week and are trading near their lowest levels since Pfizer signed a deal to buy the company for $43 billion back in March, a sign investors are increasingly concerned the FTC will intervene in the merger.

As of yesterday, Seagen's stock is worth 15% less than Pfizer's binding offer to buy the company for $229 per share. In light of the FTC's lawsuit to block Amgen from buying Horizon Pharmaceuticals, analysts at TD Cowen surveyed 31 investors and found that about 40% expect the government to take a similar approach to Pfizer's acquisition of Seagen, but more than 80% predicted the deal would still close within 12 months.

We should find out soon enough. Earlier this month, Pfizer refiled its merger paperwork to give the FTC more information about the deal, starting a 30-day countdown clock for the agency to review the proposal. That means that by July 14, the FTC will either approve the merger or request yet more information, which would suggest a protracted process ahead.

Psychedelics

What happens when an MDMA evangelist has to turn a profit?

Rick Doblin, who founded the non-profit MAPS to prove the therapeutic potential of MDMA, is closer than ever to his decades-in-the-making goal of convincing the FDA to approve it as a treatment for post-traumatic stress disorder. But the immense cost of developing a medicine and getting it to market has led to changes in MAPS's counter-cultural ethos, pushing its charismatic founder into the background.

As STAT's Olivia Goldhill reports, MAPS profit-seeking arm, which was once 100% owned by the broader non-profit, recently started accepting private donations to help fund its drug development. And the group has seemingly abandoned its long-held aversion to patenting its discoveries.

Doblin, who remains a major figure in the world of therapeutic psychedelics, quietly gave up the role of executive director after holding it for 37 years. He now faces challenges over important decisions and must answer to investors for the first time.

Read more.

Vaccines

After a 100-year wait, a new tuberculosis vaccine is moving forward

The Gates Foundation is funding a massive trial of a GSK-invented vaccine for tuberculosis that could become the first preventive weapon against the deadly infection in more than a century.

As STAT's Jason Mast reports, the vaccine is called M72/AS01, and it pairs a tuberculosis protein with an adjuvant meant to prime an immune response. The Gates Medical Research Institute, an affiliate of the foundation, is plotting a 26,000-person, Phase 3 study to begin next year, testing whether the vaccine can prevent people with latent tuberculosis — an infection with no symptoms — from progressing to full-blown disease.

The Gates Foundation, which also funded early research into the vaccine, has committed $400 million to the trial, and the U.K.'s Wellcome Trust is committing up to an additional $150 million. The trial will take place across more than 50 sites in Africa and Asia and likely take four to six years to complete, the foundation said.

Read more.

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