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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If approved, Pfizer’s sasanlimab will distinguish itself from Merck’s blockbuster Keytruda as the first PD-1 inhibitor indicated in combination with BCG for high-risk non-muscle invasive bladder cancer patients who had not previously undergone BCG treatment.
Misses from amyotrophic lateral sclerosis hopefuls Denali Therapeutics and partners AbbVie and Calico Life Sciences mark the latest setbacks for the controversial platform trial, the results from which have largely mirrored the dismal success rate in ALS overall.
MaaT013 in 2022 was put under clinical hold by the FDA, which cited safety and efficacy concerns with the sample-pooling method used to produce the investigational therapy. The hold was lifted in April 2023.
By mid-2025, the biotech will split into two entities: a new, as-yet-unnamed innovative medicines specialist and a cell therapy company, the latter of which will inherit the Galapagos name.
BioSpace presents 25 noteworthy biopharma startups in ’25; analysts forecast stronger M&A as the J.P. Morgan Healthcare Conference kicks off next week; GLP-1s continue to expand their reach as Novo, Lilly fight against compounders; and a look ahead to five key FDA decisions in Q1.
Amid recent backlash stemming from market withdrawals and trial delays, the FDA seeks to further clarify its requirements for confirmatory trials for therapies seeking the shortened path to market.
Months after posting weight loss of 7.5% at 36 days for patients taking MET-097i, Metsera releases mid-stage results of just over 11% average body weight reduction at 12 weeks, with no plateau and a promising safety profile.
Denali’s failure on Monday continues biopharma’s losing streak against amyotrophic lateral sclerosis. PTC Therapeutics and Amylyx have seen similarly disappointing results.
From ADCs and radiopharmaceuticals to cell and gene therapies, eager young startups are betting on advances in biopharma’s most competitive therapeutic spaces—and attracting dollars from Big Pharma.
Eli Lilly and Company has invested more than $20 billion in its manufacturing capabilities since 2020 to help meet high demand for its medicines. Its recently announced Lilly Medicine Foundry—which will support research and development efforts—is just the latest example of the ability to research new ways of producing medicines, while also scaling up manufacturing of medicines for clinical trials.