CNM-Au8 - (HEALEY ALS)

August 10, 2026: Clene Inc. (NASDAQ: CLNN) reported post hoc, exploratory analyses for CNM-Au8 30 mg in ALS from the Phase 2 HEALEY ALS Platform Trial and RESCUE-ALS, intended to support a planned NDA for accelerated approval and address questions raised in a March 2026 FDA Type C meeting about neurofilament light chain (NfL) as a potential surrogate endpoint.

In HEALEY, CNM-Au8-treated patients whose NfL declined or stabilized had a 38% lower risk of death vs concurrently randomized controls over full follow-up (HR 0.621, 95% CI 0.397–0.972; p=0.037), with nearly six months (177 days) of survival gain at four years (p=0.012). They also showed better CAFS outcomes combining survival with ALSFRS-R (p=0.032) and SVC (p=0.003). An FDA-requested principal stratum analysis found a 41% lower risk of death in predicted NfL responders (HR 0.593; p=0.026). CNM-Au8 30 mg previously showed an NfL reduction during HEALEY’s 24-week double-blind period (LS mean difference -0.100 log scale; p=0.040).

In RESCUE-ALS, NfL responders had a 76% lower adjusted risk of death than NfL non-responders (HR 0.24; p=0.008) and a 69% lower risk than propensity-matched real-world controls (HR 0.31; p=0.011).

Clene said neither trial met its prespecified primary efficacy endpoint. The company plans to file its NDA in early Q4 2026 and said the Phase 3 RESTORE-ALS confirmatory trial will prospectively evaluate the NfL relationship.