Compass' psilocybin therapy appears likely to meet FDA bar for approval

Compass Pathways said this morning that its psilocybin therapy, COMP360, hit the necessary endpoints in two Phase 3 trials for treatment-resistant depression, STAT’s Elaine Chen writes.

Patients saw about a 3.6- to 3.8-point greater drop on a standard clinical measure of depression called the MADRS scale versus control at six weeks — a result that “probably meets the bar for approval. It doesn't shout out to you that this is miraculous,” said Jerry Rosenbaum, director of Massachusetts General Hospital’s Center for the Neuroscience of Psychedelics, who was not involved with the study.

The drug appears to work quickly, with sharp early drops in depression scores that wane over time and may call for repeat dosing. But there were some concerning safety signals — including transient suicidal ideation in a handful of patients — that will draw scrutiny.

eye disease

Ocular's Axpaxli hits wet AMD targets, with caveats

Ocular Therapeutix delivered what it technically intended: Its experimental drug Axpaxli beat low-dose Eylea in a Phase 3 wet AMD trial, with 74% of patients maintaining vision at nine months and 66% at one year after a single injection — an edge that did achieve statistical superiority.

But investors expected better, STAT’s Adam Feuerstein writes, and Regeneron’s drug Eylea held its own in comparison. Axpaxli didn’t improve visual acuity, though it showed stronger retinal fluid control, which Ocular argues is the more clinically meaningful signal.

“If I had this drug tomorrow, I’d use it tomorrow,” CEO Pravin Dugel told STAT. “This is exactly what the wet AMD community has been looking for for the last 20 years — a drug that is safe, effective and predictably more durable.”

glp-1 drugs

China's GLP-1 market is getting crowded

China is becoming a competitive force in GLP-1 development — and Novo Nordisk may have just felt the first tremor. Ozempic sales in China fell 5% last year, the first decline since the drug's 2021 approval there, STAT contributor Brian Yang writes. This suggests that competition from Eli Lilly, as well as a wave of domestic entrants, may be gaining traction.

With more than 100 GLP-1 assets in development — roughly 40% of the global pipeline — China is rapidly shifting from “class-based to brand-based competition,” said Justin Wang, head of LEK Consulting’s Shanghai office. This is helped along by e-commerce channels that favor local competitors and regulatory quirks that may limit uptake of foreign obesity drugs.

rare disease

Disc's fast-track voucher gets an FDA reality check

The FDA has rejected bitopertin, a drug made by Disc Medicine for the rare blood disorder porphyria. It's the first novel therapy to be reviewed as part of Commissioner Marty Makary’s new fast-track voucher program.

In a rejection letter, the FDA cited “uncertainties” about whether the biomarker used in trials actually predicts meaningful clinical benefit, STAT’s Lizzy Lawrence and Adam Feuerstein write.

Behind the scenes, the agency's dermatology division reportedly strained to meet the compressed timeline. But the rejection reaffirms FDA officials' assertions that the voucher program, despite criticism, shouldn't be viewed as a free pass for drugmakers.

vaccines

Vaccine lawsuit tests Kennedy's policy overhaul

After hours of arguments in a packed Boston courtroom on Friday, Judge Brian Murphy declined to rule immediately on the American Academy of Pediatrics’ challenge to HHS Secretary Robert F. Kennedy Jr.’s sweeping changes to federal vaccine policy.

Instead, STAT’s Anil Oza writes, he asked the government for additional briefing before deciding whether the moves violated the Administrative Procedure Act and federal advisory committee rules.

The suit has grown to encompass Kennedy’s removal of Covid shots from the childhood schedule, the reshaping of ACIP, and a pared-down immunization schedule issued without career CDC input. These are steps the AAP calls “arbitrary and capricious,” and harmful to public health. But government lawyers framed the changes as reasonable policy judgments aimed at restoring trust — and that the AAP’s suit was “metastasizing” the issue at hand.

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