Usnoflast is a novel oral NLRP3 inflammasome inhibitor in patients with Amyotrophic Lateral Sclerosis (ALS)

Zydus Lifesciences has received approval from US FDA to conduct Phase II(b) clinical trial for Usnoflast, a novel oral NLRP3 inflammasome inhibitor in patients with Amyotrophic Lateral Sclerosis (ALS).

Under the leadership of  Principal Investigator Prof Merit Cudkowicz, MD, Director of the Sean M. Healey & AMG Centre for ALS; Chair of Neurology, Massachusetts General Hospital; Julieanne Dorn Professor of Neurology, Harvard Medical School, the Phase 2(b), randomised, double-blind, placebo-controlled, parallel-group, multicentre study will evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of Usnoflast administered to adult subjects with ALS.

The study consists of a 36-week treatment phase, followed by a 16-week open-label extension. This study will enrol 210 ALS patients and study doses of 50 mg and 75 mg Usnoflast versus placebo. The change in ALSFRS-R total score from baseline through week 36 will be measured as the primary endpoint of this trial. The key secondary endpoints will include changes in SVC (Slow Vital Capacity), CSF levels of NfL (neurofilament). In addition, the biomarkers including high-sensitivity C-reactive protein, (hs-CRP), interleukin (IL)-18, IL-6, IL-1β, NLRP3 and serum amyloid A (SAA), will also be evaluated.

Speaking on the development, Chairman of Zydus Lifesciences, Pankaj Patel, said, “We are excited to report the approval from USFDA to initiate this randomised, double-blind, placebo-controlled Phase 2(b) clinical trial in ALS patients. Zydus is committed to unlocking new frontiers in neuroscience and developing transformative breakthrough medicines.”