Zydus Bags USFDA Nod for Phase II(b) Trial to Optimize Usnoflast Dosage

Indian pharmaceutical company Zydus has been granted approval by the USFDA to perform a Phase II(b) clinical trial aimed at identifying the ideal dosage strength of Usnoflast, an innovative oral NLRP3 inflammasome inhibitor for individuals with Amyotrophic Lateral Sclerosis (ALS). The randomized, double-blind, placebo-controlled, parallel-group, multicentre Phase 2(b) study will assess the efficacy, safety, pharmacokinetics, and pharmacodynamics of Usnoflast in 210 ALS patients, comparing Usnoflast doses of 50 mg and 75 mg against placebo, according to the release.

Usnoflast (ZYIL1) is a new, oral small molecule NLRP3 inhibitor that is presently in clinical development for four indications, including neuroinflammation, Parkinson’s disease, Inflammatory Bowel Disease (IBD), and Multiple Sclerosis (MS). Up to this point, the novel medication has been granted an 'Orphan Drug Designation' by the US regulator for the treatment of patients with Cryopyrin Associated Periodic Syndrome (CAPS), which is a rare auto-inflammatory condition.

According to an estimate from the data-analytics company GlobalData, the drug segment is projected to reach 1.3 billion by 2029 in the eight major markets. At present, Radicava, marketed by Sanofi, and Galsody from Biogen possess a significant portion of the global market.

In the upcoming study, the pharmaceutical company will assess the alteration in the ALSFRS-R total score from the baseline over 36 weeks as the main endpoint, while significant secondary endpoints will encompass changes in SVC (Slow Vital Capacity) and CSF levels of NfL (neurofilament).