Bristol Myers Squibb's gamble on Cobenfy

Bristol Myers Squibb is staking a big chunk of its future on Cobenfy, a resurrected neuroscience drug whose promise dates back to a deeply flawed Alzheimer’s trial from the 1990s. The drugmaker, STAT’s Damian Garde writes, is betting it can finally treat psychosis in Alzheimer’s disease, an area with no approved therapies and millions of patients in need.

The pill, acquired in Bristol’s $14 billion buyout of Karuna Therapeutics, already won approval in schizophrenia and now heads into pivotal Alzheimer’s readouts later this year. Success could deliver multibillion-dollar sales and badly needed momentum as Bristol stares down an enormous patent cliff.

But analysts warn that failure could intensify scrutiny of the drugmaker’s R&D culture as well as its M&A strategy after a string of late-stage disappointments.

“Cobenfy for us is the beginning,” Cristian Massacesi, Bristol’s chief medical officer, told Damian. “We want to raise the bar and try to really change the outcome of this disease, not only controlling the symptoms and slowing down the progression but being able to treat patients before they have symptoms.”

rare disease

Sanofi pushes forward with Gaucher disease drug

Sanofi said this morning that its experimental oral drug venglustat improved neurological symptoms in patients with type 3 Gaucher disease in a Phase 3 trial, STAT’s Andrew Joseph writes. The drug, which is meant to reduce the buildup of toxic lipids, is a potential breakthrough for a form of a rare lysosomal storage disorder that has long lacked effective treatment.

Unlike enzyme replacement therapies such as Cerezyme, which address only peripheral symptoms, venglustat was designed to cross the blood-brain barrier and showed benefits on neurological assessments after a year of treatment, though with higher rates of nausea, diarrhea, and spleen enlargement.

Sanofi plans to submit the drug to regulators, underscoring what executives call its continued commitment to rare diseases even as it doubles down on immunology and navigates mixed late-stage trial results elsewhere in its pipeline.

opinion

Why psychedelics researchers should be transparent about their use

Psychedelic drug trials pose a unique challenge to clinical research norms, because many investigators and therapists have personal experience with the substances they study. While this can build trust and empathy with participants, it also introduces expectancy bias and can weaken blinding, opines Ian Reardon, a student at Penn’s Wharton School, head of marketing at a veteran-focused psychedelic retreat company, and a writer at Tripwire, a news blog covering psychedelics.

Surveys suggest psychedelic use is common among researchers and trial therapists, yet evidence shows participants and observers may judge such scientists as less objective, and outcomes in psychedelic trials are already highly sensitive to expectations.

To manage this tension, researchers are increasingly arguing for “scoped transparency" — a middle-ground approach in which personal psychedelic use and advocacy are disclosed through institutional channels, ethics review, and role assignment, rather than casually to participants.

glp-1 drugs

A play takes on Novo's myth and money

A new Danish stage play, “The Golden Calf,” premiered this weekend near Copenhagen — focused on the origins and modern contradictions of GLP-1 behemoth Novo Nordisk.

The production, which is happening entirely without Novo’s involvement, begins with with founders August and Marie Krogh bringing insulin to Denmark in the 1920s. It then fast-forwards to Novo’s rise as Europe’s most valuable company, and its current battle with Eli Lilly in the obesity market.

"It's a piece of our common story about these two scientists who get insulin to Denmark and start this huge snowball that becomes Novo Nordisk," the play's director Nicolei Faber told Reuters, noting that the play probes the ethical tension at the heart of the company’s success. “Do they produce this medicine to earn money or to help people who are sick? And where's the balance between these two things?"

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