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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Bo Wang is a renowned AI scientist at the University of Toronto. He’s bringing his open-source culture and computational biology to Xaira Therapeutics in June.
After some high-profile crashes, the one-time biotech darling is inching toward success with its Hunter syndrome treatment, which today began a rolling BLA for accelerated approval.
Merck continues to build the case for the pulmonary arterial hypertension drug that won FDA approval in 2024.
Despite a $400 million impairment charge, analysts say the removal of a drug-device combo from its portfolio is not a huge loss for Vertex given that the company has a more advanced type 1 diabetes candidate in zimislecel.
BMO Capital Markets analysts said the results potentially position lepodisiran as “one of the most durable assets in development to date” in the competitive Lp(a) space, where drugs are designed to lower the risk of cardiovascular events such as heart attack and stroke.
According to BMO Capital Markets, Rybelsus’ outcomes in SOUL were “inconsistent,” failing to significantly lower cardiovascular death and nonfatal stroke.
AI offers tremendous potential but there are critical and time-consuming flaws in black box AI predictions.
Werner held roles at Bristol Myers Squibb, AstraZeneca and Novartis before landing at Alltrna, where she works to develop tRNA-based treatments for a range of diseases.
While Novartis and Bayer got there first, AstraZeneca, Bristol Myers Squibb and Eli Lilly are all vying to bring their radiopharmaceutical assets to a market projected to be worth over $13 billion by 2033.
Milestone Pharmaceuticals hit another bump in the road in its quest to get Cardamyst approved for paroxysmal supraventricular tachycardia when the FDA issued a Complete Response Letter on Friday.