Topline results from LELANTOS-1 phase III clinical study of pamrevlumab in non-ambulatory patients with Duchenne muscular dystrophy
FibroGen, Inc.announced topline data from the Phase III LELANTOS-1 placebo-controlled trial of pamrevlumab for the treatment of non-ambulatory patients with Duchenne Muscular Dystrophy (DMD) on background corticosteroids
The study did not meet the primary endpoint of Performance of the Upper Limb 2.0 (PUL 2.0) score at week 52 compared to baseline. Pamrevlumab was generally safe and well tolerated and the majority of treatment emergent adverse events were mild or moderate.
FibroGen plans to present the complete results of the LELANTOS-1 study at an upcoming medical conference and to publish the full results. Topline data from the Phase III LELANTOS-2 clinical trial of pamrevlumab for the treatment of ambulatory patients with DMD is expected 3Q 2023.
“While disappointed with these results, we look forward to sharing the data at a future medical conference to contribute towards the understanding of this devastating disease,” said Enrique Conterno, Chief Executive Officer, FibroGen. “FibroGen would like to thank the patients, caregivers and clinical trial investigators for their dedication in participating in this study.”
Related news and insights
Karuna Therapeutics, Inc. a biopharmaceutical company driven to discover, develop, and deliver transformative medicines for people living with psychiatric and neurological conditions, announced the submission of a New Drug Application (NDA) to the FDA for KarXT (xanomeline-trospium) for the treatment of schizophrenia
BrainStorm Cell Therapeutics Inc. announced the outcome of the FDA's Cellular, Tissue and Gene Therapies Advisory Committee meeting to review the Biologics License Application (BLA) for NurOwn, an investigational mesenchymal stem cell therapy for the treatment of amyotrophic lateral sclerosis (ALS)
Merck Inc., known as MSD outside of the United States and Canada, announced that the FDA has accepted for priority review a new Biologics License Application (BLA) for sotatercept, Merck’s novel investigational activin signaling inhibitor, for the treatment of adult patients with pulmonary arterial hypertension (PAH) (WHO Group 1)