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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The American Association for Cancer Research’s annual conference featured updates from several companies on key candidates and assets, including Merck’s Keytruda and GSK’s Jemperli.
The drug, a small molecule protein inhibitor, brought in $132 million in the first quarter, missing consensus estimates by 17%.
While Vaxart can proceed with its study, fellow COVID-19 vaccine developers GeoVax and Novavax continue to be hamstrung by various regulatory roadblocks.
Pfizer’s sasanlimab, when used with standard of care, reduced the likelihood of disease recurrence or progression, death due to any cause or persistence of cancer cells by 32% in patients with high-risk non-muscle invasive bladder cancer.
The targeted drug release device TAR-200 shows promising response and disease-free survival rates in specific populations of patients with non-muscle-invasive bladder cancer.
The FDA is currently reviewing Merck’s sBLA for Keytruda in head and neck cancer, with a target action date of June 23.
With ivonescimab’s data coming solely from China, its prospects in the U.S., where Summit owns the rights, remain up in the air.
As tariffs, HHS workforce cuts and the ouster of CBER Director Peter Marks threaten the “lifeblood” of the cell and gene therapy space, experts express wariness over the unknowns and optimism that Marks’ legacy will carry on.
The hold, placed by the FDA on an early-stage clinical trial of an experimental BET inhibitor, stems from a recent observation of testicular toxicity in canines given the treatment.
Presentations at this year’s American Association for Cancer Research meeting could have a broad impact on the treatment landscape for head and neck and lung cancer, and implications for specific drug modalities like TIGIT and VEGF.