Drug Development
Analysts said the outcome is disappointing because there are no approved treatments for dyskinetic cerebral palsy, but the setback had little impact on Neurocrine’s valuation.
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In the midst of regulatory and political upheaval, biopharma’s R&D engine kept running, churning out highs and lows in equal parts. Here are some of this year’s most glorious clinical trial victories.
Every year in biopharma brings its share of grueling defeats, and 2025 was no different, especially for companies targeting neurological diseases. Some failures split up partners, and one particularly egregious case even led to the demise of an entire company.
The R&D pipeline for depression therapies faced a demoralizing 2025 as five high-profile candidates, including KOR antagonists by Johnson & Johnson and Neumora Therapeutics, flunked late-stage clinical trials, underscoring the persistent challenges of CNS drug development.
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To more effectively treat neurodegenerative conditions, we first need diagnostic tools that lend a more complete picture of protein aggregates in the brain.
At the intersection of radiation and precision, Novartis, Bayer, AstraZeneca and more hope to cash in on a radiopharmaceuticals market that could top $16 billion by 2033.
In this episode of Denatured BioSpace’s head of insights Lori Ellis discusses the public health consequences of vaccine hesitancy and the critical distinction between skepticism and cynicism with Paul Offit, MD, director of the Vaccine Education Center at the Children’s Hospital of Philadelphia.
Yes, according to leading vaccine physician Paul Offit, who denounced the new placebo-controlled trial requirements for vaccines and sought greater clarity: “I don’t know what they’re talking about.”
CRISPR Therapeutics’ partner Vertex reported that more than 65 treatment centers have been activated for the gene therapy Casgevy. While Vertex handles the market, CRISPR has been focused on its clinical program.
Keytruda is set to lose exclusivity in 2028, meaning Summit may face competition from cheaper biosimilars. Meanwhile, other branded drugmakers are also seeking to improve on the blockbuster checkpoint inhibitor.
The biotech’s Huntingtin-targeting molecule lowered blood levels of the protein and elicited functional improvements in earlier-stage patients, but results were not as robust in other biomarkers or with patients at later stages of the disease.
Beam Therapeutics and Verve Therapeutics have each built their lead candidates on a technique billed as a safer alternative to conventional CRISPR. Clinical results have so far been promising.
The new policy, revealed by the Department of Health and Human Services on Wednesday, would make vaccines “less available and less affordable,” according to vaccine expert Paul Offit. It could also further complicate the picture for companies like Novavax.
According to CEO Daniel Vitt, clinical and disability-related outcomes are more relevant than brain volume change for drug development in multiple sclerosis.