The need-to-know this morning
psychedelics
Positive Compass therapy disappoints Wall Street
Compass Pathways said its Phase 3 trial of psilocybin for treatment-resistant depression met its primary endpoint, with a 3.6-point improvement on the MADRS scale over placebo — just above the company's predefined threshold of success.
But the modest effect size spooked investors, halving the stock's value on Monday; they had been looking for results closer to a 4-point improvement, as was seen in earlier studies. Safety data were encouraging, with no imbalance in suicidal ideation. The trial, the largest of its kind, remains blinded for 26 weeks, with more data expected later this year, STAT's Olivia Goldhill writes.
"The key finding here is safety," Compass CMO Guy Goodwin told STAT. The results, he said, were similar to studies of Spravato, Janssen's esketamine product, which would be a competitor should the Compass treatment ultimately win approval.
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A targeted therapy advances
From STAT's Matthew Herper: The biotech firm Nuvalent said this morning that new data indicate its targeted drug zidesamtinib could help patients with a rare form of lung cancer that has failed other targeted treatments.
For 1% to 2% of patients with non-small cell lung cancer, tumors test positive for alterations in a gene called ROS1. ROS1-positive tumors often respond to one of five targeted therapies on the market. Hopes for zidesamtinib and another targeted cancer medicine have led Nuvalent to have a market capitalization of $5.5 billion.
Nuvalent says zidesamtinib was designed to target ROS1 more specifically, causing less toxicity. Among 117 patients that had been treated with the drug in its hybrid Phase 1/2 trial, there was an objective response rate of 44%, meaning 51 patients saw the volume of their tumors shrink by at least a third on scans. Side effects included peripheral edema, elevated levels of a blood test used to track kidney function, constipation, and fatigue.
More here.
vaccines
RFK Jr.'s fractured vaccine policy concerns experts
The U.S. vaccination landscape is splintering under HHS Secretary Robert F. Kennedy, Jr., who has fired members of the CDC's advisory panel and replaced them in some cases with vaccine skeptics. This is prompting fears of eroded trust, falling vaccination rates, and the resurgence of preventable disease, STAT's Helen Branswell writes.
Long-standing national guidelines risk being supplanted by competing recommendations from professional societies like the AAP and ACOG, with no guarantee that insurers or federal programs will continue covering vaccines removed from official CDC schedules. This growing confusion that stems from politicized science could lead to diminished protection from dangerous diseases — particularly for vulnerable populations, experts warn.
"And that's what the public has to understand: That these diseases are here. They've never gone away," a former director of the CDC's National Center for Immunization and Respiratory Diseases said. "These are diseases that will come back quickly if we stop vaccinating for them."
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REGULATION
Another notable departure at FDA
Jacqueline Corrigan-Curay, acting head of the FDA's drug center CDER, is retiring in July, making yet another high-level departure under Commissioner Marty Makary. In an email to staff, Corrigan-Curay she made the decision after a vacation and called it "difficult," citing staff support during "challenging times."
Her exit follows a string of forced resignations and early retirements, as Makary and newly empowered deputy Vinay Prasad consolidate influence. Career scientists say they fear a continued erosion of expertise and autonomy, particularly after top gene therapy regulator Nicole Verdun was fired. Makary is reportedly eyeing ex-Amarin CEO Karim Mikhail as a replacement, STAT's Lizzy Lawrence writes.
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