Soleno's 'life-changing' drug for Prader-Willi approved

The FDA yesterday approved the first medicine to treat people with Prader-Willi syndrome, a rare genetic disease that causes an insatiable desire to eat. The drug, a once-daily pill called Vykat XR, was developed by Soleno Therapeutics.

Vykat is not a cure, but in clinical trials, the medicine was shown to reduce hyperphagia, the incessant desire to eat, and other related behaviors in people with Prader-Willi. One endocrinologist who has treated patients with the drug in clinical trials said it is "nothing less than life-changing."

Soleno has identified through claims data approximately 10,000 people with Prader-Willi in the U.S.

artificial intelligence

AI protein model to be trained on AbbVie, J&J data

Life sciences data company Apheris said this morning that it's collaborating with AbbVie and Johnson & Johnson in an effort to boost the capabilities of a protein AI model.

The model, called OpenFold3, is an open-source dupe of AlphaFold3, the model developed by Google's AI unit DeepMind. The model is currently limited in its ability to make useful predictions for drug discovery, though, since the publicly available data it's trained on do not have enough information on protein interactions with other proteins or with drug-like molecules.

Under the new collaboration, OpenFold will produce a fine-tuned version of its model that's trained on AbbVie and Johnson & Johnson's vast stores of proprietary data, with the hope of accelerating drug discovery. That model will only be available to those participating through the Apheris-run consortium.

politics

NIH director targets misinformation research as more turmoil rocks health agencies

On Jay Bhattacharya's first day leading the NIH yesterday, the agency told staff in an email to compile a list of grants and contracts related to "fighting misinformation or disinformation" — an issue he has highlighted. In recent weeks, this type of a step has preceded the termination of research funding.

The email asked staff to respond with information on any contract that "may be related to any form of censorship at all or directing people to believe one idea over another related to health outcomes." It lists examples including contracts to promote vaccine uptake, or public health messages about the "dangers of Covid or not wearing masks."

The email is consistent with the new director's previous views. "Jay Bhattacharya has portrayed himself as a victim of censorship," one physician said. Read more from STAT's Anil Oza.

We also report that NIH has placed the official who oversaw the creation of ARPA-H, the new "moonshot" science agency, on administrative leave. This marks third time a senior leader has departed the NIH since the President Trump took office. Read more from my colleague Lev Facher.

Meanwhile, the Trump administration has also abruptly canceled more than $12 billion in federal grants to states that were being used for health services like tracking infectious diseases and treating mental health and addiction, The New York Times reports.

biotech

CRISPR sees a string of leadership departures

From my colleague Jason Mast: CRISPR Therapeutics said yesterday that chief operating officer Julianne Bruno was leaving to "pursue external opportunities." The news would hardly be notable except that, by STAT's count, this is the seventh member of CRISPR's leadership team to leave the company since 2023.

The departure also comes as the company faces questions about its future. Its sickle cell therapy, while life-changing for patients, has not brought revenue into the company and might not for years to come. It has a pipeline of other gene-editing medicines in or near human trials, but it remains unclear which programs could bring in significant revenue or differentiate the company from the many gene-editing competitors out there now. In addition to the departure of top officers, the company has also quietly laid off staff twice since late 2023.

Still, the company is in a far better position than many of its competitors. Thanks to its sickle cell work, it has amassed nearly $2 billion in cash to ride out the next few years. The first data readouts from a new wave of programs are expected later this year.

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