
Zydus, a global pharmaceutical leader, has received approval from the US FDA to begin a Phase II(b) clinical trial for Usnoflast, an oral NLRP3 inflammasome inhibitor, in patients with Amyotrophic Lateral Sclerosis (ALS). The trial, led by Prof. Merit Cudkowicz, MD, at Massachusetts General Hospital, will assess Usnoflast’s efficacy, safety, and pharmacokinetics in ALS patients.
This double-blind, placebo-controlled study will involve 210 adult ALS patients, testing doses of 50 mg and 75 mg of Usnoflast. Over 36 weeks of treatment, the primary endpoint will be the change in ALSFRS-R total score, while key secondary endpoints include changes in Slow Vital Capacity (SVC) and neurofilament levels in cerebrospinal fluid (CSF). Biomarkers such as IL-18, IL-6, NLRP3, and serum amyloid A (SAA) will also be studied.
ALS, a neurodegenerative disease affecting around 32,000 people in the U.S. and 75,000 in India, leads to rapid neurodegeneration, paralysis, and death. The disease has no cure, and patients typically survive only two to five years post-diagnosis.
Usnoflast has shown promise in preclinical studies for neuroinflammation-related diseases like Parkinson’s, Multiple Sclerosis, and Inflammatory Bowel Disease. It has already received Orphan Drug Designation from the USFDA for treating Cryopyrin Associated Periodic Syndrome (CAPS). Zydus aims to present Phase 2(a) ALS trial data at upcoming medical conferences.