Clene Nanomedicine reports 24-month data from Phase 2 RESCUE-ALS

Clene through its wholly owned subsidiary Clene Nanomedicine announced the 24-month long-term data cut from the Phase 2 RESCUE-ALS ongoing open-label extension as of July 2023, which showed a significant median survival benefit of 19.3 months using the rank-preserving structural failure time model and a significant 52% decreased risk of ALS clinical worsening events with CNM-Au8 treatment. RESCUE-ALS was a 36-week double-blind placebo-controlled Phase 2 trial that originally randomized 45 people living with ALS, who were an average of 16 months from symptom onset. Survival Improvement: Cross-over adjusted median survival: 19.3 month median survival benefit; 75% decreased risk of long-term all-cause mortality in participants originally randomized to treatment with CNM-Au8 compared to those originally randomized to placebo after adjusting for benefit received by placebo after switching to CNM-Au8; The RPSFTM analysis method estimates the survival gained by receiving active treatment using the data from all study participants and then subtracts the benefit from ex-placebo participants switched to CNM-Au8 during the OLE to provide a comparison of CNM-Au8 versus placebo across the entire study period. Unadjusted median survival: 10.1 month median survival benefit when not accounting for the improvement by ex-placebo treated participants who switched to CNM-Au8 at the start of the OLE; 46% decreased risk of all-cause mortality in participants originally randomized to treatment. Observed survival versus ALS historical placebo controls: 70% decreased risk of long-term mortality in participants originally randomized to treatment with CNM-Au8 compared to matched placebo participants derived from the PRO-ACT database; PRO-ACT contains approximately 12,000 ALS patient records from multiple completed clinical trials. Improved Time to Clinical Worsening: 52% decreased risk of ALS clinical worsening events in the participants originally randomized to CNM-Au8 treatment versus original placebo. Safety: No serious adverse events have been assessed as related to CNM-Au8 treatment; adverse events observed with CNM-Au8 have been characterized as transient and predominantly mild-to-moderate in severity.