June 23, 2025

The need-to-know this morning

Novo Nordisk terminated its marketing relationship with Hims & Hers, accusing the telehealth company of deceptive marketing practices and selling "illegitimate" compounded versions of the weight-loss drug Wegovy.
Compass Pathways reported results from a Phase 3 study of its psilocybin-based drug for treatment-resistant depression.

fda

FDA ouster tied to Duchenne therapy clash

The sudden removal of Nicole Verdun, the FDA’s top cell and gene therapy regulator, followed a dispute with her boss, Vinay Prasad, over holding a public advisory committee meeting on a cell therapy for Duchenne muscular dystrophy made by Capricor Therapeutics, STAT reports. Prasad unilaterally canceled the meeting, escalating internal tensions and leading to Verdun and her deputy being placed on administrative leave and escorted out of FDA headquarters — an unusually harsh move that stunned agency staff and industry alike.

Though Verdun faced management complaints, she was widely respected for balancing urgency with rigor. The shakeup comes amid mounting concern over inconsistent regulatory standards for high-stakes gene therapies. Notably, multiple agency employees have told STAT that Prasad is uninterested in the administrative aspects of running CBER — meaning that more expert managers in the department are critical.

diabetes

Vertex stem cell therapy spurs insulin independence

In a small but striking Phase 1/2 study, an off-the-shelf stem cell therapy made by Vertex Pharmaceuticals enabled 10 of 12 people with type 1 diabetes to stop insulin injections for at least a year — while maintaining healthy blood glucose levels and avoiding severe hypoglycemia. The treatment, zimislecel, is derived from lab-grown islet cells and delivered with immunosuppression — mimicking the success of islet transplants but without donor constraints.

“This study has the potential to get us one step closer to a ‘functional cure’ for patients with type 1 diabetes,” the study’s co-author told STAT.

hiv/aids

The amazing R&D behind Gilead's newly approved HIV prevention shot

Last week the FDA approved Gilead’s lenacapavir, sold as Yeztugo, for HIV prevention — the first long-acting injectable PrEP that only needs to be taken twice a year. The drug, born of a decades-long scientific saga, targets the HIV capsid, a once-dismissed structure thought undruggable. Its unique mechanism blocks multiple stages of the virus lifecycle, and its exceptional stability means months-long efficacy from a single dose.

In an excerpt from his book, "Breakthrough: The Quest for Life-Changing Medicine," physician-scientist William Pao details the decades-long journey to approval. Gilead scientists overcame daunting chemical and biological hurdles to develop it, and public health advocates now hope the injectable will dramatically expand access to HIV prevention — especially for marginalized communities.

“I think it’s really going to help end the HIV epidemic for everyone, everywhere,” Moupali Das, head of clinical development for HIV prevention at Gilead, told Pao. “This really could be a game changer.”

Obesity

Novo's CagriSema matches Zepbound, disappoints investors

Full Phase 3 results for Novo Nordisk’s CagriSema show the obesity drug delivers roughly the same weight loss and safety profile as Eli Lilly’s Zepbound, with patients losing 20% of body weight over 68 weeks, but the rate of weight loss plateaued, raising doubts about its potential to outperform.

Though clinicians praised the results as meaningful, investors had hoped for more — especially with Zepbound already showing benefits in heart failure and sleep apnea.

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