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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Mixed headlines have plagued the cell and gene therapy space of late. We believe that a renewed case of optimism is not only warranted but essential if these therapies are to reach their full potential.
While expressing disappointment, William Blair analysts were unsurprised by the Phase II failure, having assigned the VISTA study a high level of risk given the “mixed” performance of a similar drug in a prior multiple sclerosis study.
The CRO market in the APAC region is thriving, particularly in China, due to intense clinical trial and innovation development, with Western investors and pharma leaning in.
Merck has made a $9.2 billion play for Cidara, and there’s another bidding war afoot, this one for sleep biotech Avadel. Meanwhile, Rick Pazdur has taken the helm at CDER while tensions run high between FDA Commissioner Marty Makary and Health Secretary RFK Jr.
Winrevair yielded significant and meaningful clinical benefits for patients with combined post- and precapillary pulmonary hypertension, an indication that, according to BMO Capital Markets, has few treatment options.
With positive data from the Phase III HERIZON-GEA-01 study, Zymeworks and Jazz Pharmaceuticals will file an approval application for Ziihera in the first half of 2026 for the treatment of gastroesophageal adenocarcinoma.
Bristol Myers Squibb and Johnson & Johnson will continue to test the asset in stroke and atrial fibrillation.
Gilead’s investigational drug combo bic/len could help lower the pill burden in patients with virologically suppressed HIV who are on complex treatment regimens, according to BMO Capital Markets.
Through substantial leadership turnover and workforce cuts, the FDA has continued to support the advanced therapy sector, actively working to remove obstacles to innovation.
Korro Bio is moving back to square one as a preclinical biotech after the failure of KRRO-110 in alpha-1 antitrypsin deficiency. The company’s stock is down 80% on all the news.