PrimeC Shows Safety and Potential Benefit in ALS Phase 2b as FDA Authorizes Phase 3

Overview

• PARADIGM randomized 68 participants 2:1 to receive PrimeC—an extended-release celecoxib/ciprofloxacin pill—or placebo for six months, followed by a 12-month open-label extension.
• The trial met its primary safety endpoint, with drug-related adverse events in 20.0% on PrimeC versus 4.3% on placebo, mostly mild to moderate.
• Exploratory outcomes favored PrimeC on the ALSFRS-R, including a +2.23-point difference at six months and +7.92 points at 18 months for those initially assigned to treatment, particularly in speech and swallowing.
• Early, continuous treatment was associated with a 64% lower risk of hospitalization, respiratory failure or death, with follow-up suggesting longer median survival than for delayed starters.
• Biomarker findings showed lower ferritin and certain ALS-linked microRNAs without changes in neurofilament light, the pre-specified primary biomarker remains unreported, and the FDA has cleared a Phase 3 trial expected to enroll about 300 patients that has not yet begun recruiting.