Topline results demonstrating survival signal for CNM-Au8 in Healey ALS Platform trial foramyotrophic lateral sclerosis
Clene Inc. announced topline phase II study results showing a survival benefit in the Healey ALS Platform trial of CNM-Au8, an investigational gold nanocrystal suspension, in participants with amyotrophic lateral sclerosis (ALS)
The primary endpoint of slope of change in ALS Functional Rating Scale Revised (ALSFRS-R) scores adjusted for mortality was not significant (2% slowing, 95% CI: -20% to +19%) at 24 weeks. Secondary endpoints of Combined Assessment of Function and Survival (CAFS) and slow vital capacity (SVC) were also not met at 24 weeks across the combined 30 mg and 60 mg CNM-Au8 doses.
The prespecified exploratory analyses of the secondary survival endpoint demonstrated a >90% reduction in risk of death alone or in risk of death/permanently assisted ventilation at 24 weeks, when adjusted for baseline imbalances in risk (p=0.028 to p=0.075, unadjusted for multiple comparisons) with the CNM-Au8 30 mg dose. These survival results were statistically consistent for the 30 mg dose between the regimen only and full analysis sets, which included shared placebo from other regimens participating in the Healey ALS Platform trial (Regimens A, B, and D).
This survival signal is consistent with results previously reported by Clene in the Phase II RESCUE-ALS trial with CNM-Au8.
The full analyses, including data on biomarkers of neurodegeneration and exploratory efficacy results, are expected later in 2022. The open-label extension will continue to follow participants and provide data updates in the future. Clene is in discussions with the Healey & AMG ALS Center to offer a broader EAP of CNM-Au8 30 mg for eligible participants of closed regimens and others.
Based on these topline findings, Clene has selected the CNM-Au8 30 mg dose for continued development in ALS. The CNM-Au8 60 mg dose did not demonstrate a survival benefit. CNM-Au8 was well-tolerated, and there were no drug-related serious adverse events or significant safety findings reported.
“There remains a high unmet medical need for treatments for people living with ALS. The potential survival benefit with CNM-Au8 at 30 mg is encouraging. Additional pre-specified exploratory analyses of both the RCT and open-label extension part of the study will be shared once available,” said Merit Cudkowicz, M.D., MSc, principal investigator and sponsor of the Healey ALS Platform Trial, director of the Sean M. Healey & AMG Center for ALS, chief of the Department of Neurology at Massachusetts General Hospital, and the Julieanne Dorn Professor of Neurology at Harvard Medical School. “We are thankful to the many people who participated in this study. We will learn from these results and continue to use these data to inform future advances in ALS trial design,” she concluded.