Regeneron Pharmaceuticals Inc.’s phase III Optima trial with garetosmab in fibrodysplasia ossificans progressiva (FOP) met its primary endpoint. The Tarrytown, N.Y.-based firm said that at 56 weeks, both doses of garetosmab, 3 mg/kg and 10 mg/kg, were “highly efficacious” in reducing the number of new bone lesions vs. placebo, showing a 94% and 90% reduction, respectively. Garetosmab is a monoclonal antibody that neutralizes the activin A protein, critical in the development of FOP lesions. Separately, the company provided updated analyses of the phase II Courage trial, testing new combinations of the GLP-1 receptor agonist semaglutide plus the anti-GDF8/anti-myostatin compound trevogrumab, with or without garetosmab, for obesity. The complete 26-week results were consistent with interim data previously reported, Regeneron said, proving that the addition of trevogrumab, with or without garetosmab, could significantly reduce the loss of lean mass associated with semaglutide-induced weight loss.

Ollin eyes next-gen ophthalmology space, launches with $100M

To say the team at Ollin Biosciences Inc. has some experience in ophthalmology would be an understatement. The company, which emerged from stealth with $100 million in venture capital and two in-licensed assets with plans to add more, brings to the table years of expertise from work on blockbuster retinal disease drugs Lucentis (ranibizumab, Roche AG) and Vabysmo (faricimab, Roche AG). The small firm, which has so far in-licensed two programs in its asset-centric, ophthalmology-focused endeavor, looks to combine that expertise with well-validated biology to create next-generation therapies targeting retinal and other eye-related diseases.

ACIP meeting cause for consternation at US Senate hearing

Today’s U.S. Senate Health, Education, Labor and Pensions Committee hearing on the CDC is throwing even more shade on tomorrow’s meeting of the agency’s reconstituted Advisory Committee for Immunization Practices (ACIP), which is expected to recommend changes to the childhood vaccine schedule. Answering questions about why she was fired as CDC director less than a month after being confirmed, Susan Monarez said Health and Human Services Secretary Robert Kennedy met with her Aug. 25 asking her to either commit to a blanket approval of all recommendations coming out of this week’s ACIP meeting or resign. Debra Houry, who recently resigned as CDC’s chief medical officer, also cast doubt on the upcoming ACIP meeting as she pointed out that, contrary to normal practice, the panel’s work groups were not involved in the background work for two of the three presentations and the documents for the meeting have yet to be posted. Typically, they would have been posted by yesterday at the latest.

New AI model simultaneously predicts risk of getting 1,000 diseases

A new generative AI model trained on UK Biobank data can simultaneously forecast the risks and timing of developing over 1,000 different diseases a decade into the future. The developers applied similar algorithmic concepts to those used to develop large language models like ChatGPT and Gemini to build the model, using medical records from 402,799 participants in UK Biobank. Its accuracy was validated against almost 100,000 other participants in UK Biobank whose data was not used for training, and then tested in a live setting using data from 1.9 million people in the national patient registry in Denmark.

Cancer vaccines face collateral damage of mRNA funding cuts

When Robert Kennedy Jr. announced the cancellation of 22 projects related to mRNA vaccines and the end of new investments in that technology, the U.S. Secretary of Health only mentioned their use against respiratory viruses, without referring to other applications. Kennedy claimed that “HHS supports safe, effective vaccines for every American who wants them. That’s why we’re moving beyond the limitations of mRNA and investing in better solutions.” The CDC’s Advisory Committee on Immunization Practices was summarily dissolved in June. Its 17 members were replaced, including individuals openly opposed to vaccines who were personally selected by Kennedy Jr. The current panel will review all vaccines in the U.S. starting with its meeting on Sept. 18-19, 2025. Access to the latest COVID-19 vaccines has already been restricted.

Roivant and Priovant head for an NDA in rare disease

Positive late-stage study results in a rare autoimmune disease have Roivant Sciences Ltd. and Priovant Therapeutics Inc. saying they plan an NDA filing sometime in the first half of 2026. The phase III study to evaluate brepocitinib in treating the rare autoimmune disease dermatomyositis hit its primary endpoint and all of its nine key secondary endpoints. The dual, selective inhibitor of tyrosine kinase 2 and JAK1 produced clinically meaningful and statistically significant improvements in the placebo-controlled trial in measures of skin and muscle disease, plus speed of onset. Dermatomyositis causes inflammation that leads to muscle weakness that impacts daily living, such as walking and getting dressed. The company’s stock (NASDAQ:ROIV) was up 11% at midday, with shares at $15.80 each.

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