Drug Development
After a spate of patient deaths in 2025 linked to the company’s Duchenne gene therapy, Sarepta shared new data showing benefits of the therapy three years after dosing.
FEATURED STORIES
As drug candidates discovered via AI move into later-stage clinical trials, the technology seems to be doing as promised: speeding drug development.
Following the hard-won success of early anti-amyloid drugs, a new generation of Alzheimer’s modalities—from tau-targeting gene silencers to blood-brain barrier delivery platforms—is entering the pipeline to anchor future combination therapies.
Three years after the accelerated approval of its anti-amyloid Alzheimer’s therapy, Biogen—neck and neck in the market with Eli Lilly and its Kisunla offering—is focused on a near-term FDA decision for a subcutaneous induction dose of Leqembi, a presymptomatic readout in 2028 and a clutch of next-generation candidates.
Subscribe to ClinicaSpace
Clinical trial results, research news, the latest in cancer and cell and gene therapy, in your inbox every Monday
THE LATEST
As Omicron continues to dominate globally, research is coming in every day on this highly contagious variant of SARS-CoV-2.
Sixty-three percent of patients met the primary endpoint of a 2-point or greater improvement in disease severity without worsening of fibrosis.
The FDA cited data demonstrating that both treatments are not likely to be potent against omicron and should not be authorized for use in any U.S. state, territory, and jurisdiction for the time being.
The company submitted its application for AP-188 in early 2021, covering dosage details, formulation, and method of stroke rehabilitation.
BioSpace sat down with Niko Andre, M.D., Ph.D., global franchise head for hematology and immuno-oncology at AstraZeneca.
MaaT Pharma decided today to end the Phase Ib CIMON study for MaaT03, the company’s second drug candidate and first oral formulation in Phase 1b clinical trial, following positive interim engraftment data of the drug.
Oncopeptides has discontinued the marketing of Pepaxto drug in the US. But, in a recent turnaround, they decided to withdraw this decision of stopping the production.
The U.S. Food and Drug Administration issued a Complete Response Letter to Merck & Co. for its New Drug Application for gefapixant for refractory chronic cough or unexplained chronic cough.
FDA rejected OPKO’s once-weekly human growth hormone, somatrogon, which it co-developed with Pfizer for the treatment of growth hormone deficiency (GHD) in pediatric patients.
Suliman anticipates filing an Investigational New Drug application with the FDA for the PCD asset this year, and next year filing one for the cystic fibrosis asset.