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 plethora of genes involved in obesity presents an intriguing opportunity for both gene silencing and ex vivo gene therapy approaches.
Moderna said Thursday it plans to talk to regulators about the next steps after showing its next-generation candidate is more efficacious in adults than the biotech’s existing Spikevax COVID-19 shot.
Results of a large Stanford Medicine study, published Wednesday in The New England Journal of Medicine, finds CAR-T therapies carry a low risk of secondary malignancies not related to the T cells.
Coherus BioSciences and Junshi Biosciences’ PD-1 inhibitor Loqtorzi significantly boosted progression-free and overall survival in a late-stage study of patients with advanced hepatocellular carcinoma.
Pfizer’s investigational Duchenne muscular dystrophy gene therapy, fordadistrogene movaparvovec, failed in a late-stage study to significantly improve motor function in patients versus placebo.
Building on last year’s potential $7 billion partnership with Flagship Pioneering, Pfizer and Flagship-founded ProFound Therapeutics will work on discovering new obesity candidates.
Seeking to target the lucrative obesity market, Merck is focusing its R&D efforts on next-generation GLP-1 therapies that offer additional benefits beyond weight loss, according to company executives.
Sen. Bernie Sanders (I-Vt.), chair of the Senate health committee, has proposed issuing a subpoena to Novo Nordisk President Doug Langa forcing him to testify regarding the company’s pricing for Wegovy and Ozempic.
While Sage Therapeutics’ drug candidate showed a slight difference compared to placebo in a mid-stage Huntington’s disease trial, William Blair analysts in a Tuesday note to investors said they “remain cautious” on dalzanemdor and “do not view the small numerical changes as definitive.”
Seeking to carve out its own niche in the obesity space, Syntis Bio launched on Tuesday to develop an oral weight-loss treatment that mimics the effects of gastric bypass surgery.