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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Led by alums from Takeda and Boston Pharmaceuticals, Hillstar Bio is working on treatments that remove harmful immune cells to relieve disease.
The Eylea franchise was in need of a win after an appellate court last week denied the pharma’s bid to block Amgen’s biosimilar from the U.S.
In attempt to keep R&D costs low, the vivarium business model has emerged as a crucial solution for drug developers.
It’s early days for xenotransplantation, but eGenesis, Eledon, United Therapeutics and more are working to develop solutions to make this approach a viable option and help ease the organ shortage crisis.
Since 2016, the FDA has approved three disease-modifying treatments for spinal muscular atrophy, with several companies—including Novartis, Scholar Rock and Biogen—progressing novel candidates through clinical trials.
Biosimilars are essential healthcare equalizers, but their regulation is overly complicated due to lobbying by makers of branded biologics looking to maintain blockbuster revenue.
The label expansion could help J&J establish Tremfya as a successor to Stelara, which is now facing a growing biosimilar challenge.
The FDA approval of Alnylam’s Amvuttra sets up a three-way race with Pfizer and BridgeBio, which both market transthyretin stabilizers for transthyretin amyloid cardiomyopathy.
After Sarepta reported the death of a patient who had recently taken the gene therapy Elevidys, patient advocacy group Parent Project Muscular Dystrophy stepped up—as they always do.
After bringing Zolgensma to market in 2019 as the first gene therapy for spinal muscular atrophy, Novartis is back with an intrathecal formulation intended for older patients.