Positive results reported in open label extension of novel ALS therapeutic
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A clinical-stage biopharmaceutical company has reported new positive data from a 12-month long-term open label extension of the CNM-Au8 treatment arm in the HEALEY ALS Platform Trial.
According to a release from Clene Inc., the perpetual multicenter, randomized, double-blind, placebo-controlled trial, which evaluated safety and efficacy of multiple investigational products in those with amyotrophic lateral sclerosis, yielded positive results with respect to plasma neurofilament light chain (NfL) and decreased risk of all-cause mortality.
A total of 161 participants were randomized to receive 30 mg or 60 mg CNM-Au8 or placebo as adjunctive care for a 24 weeks treatment period. Plasma NfL declined by 16% (95% CI: 2%-28%) from baseline to 76 weeks of treatment in the trial’s open label extension (OLE) in those given CNM-Au8 30 mg compared with placebo. CNM-Au8 was associated with a 10% relative reduction in plasma NfL over the 24-week double-blind treatment period, which is evidence of durability in the long term, per the release.
Additionally, Clene reported long-term survival analyses, which included prespecified rank-preserving structural failure time model to account for the effects of CNM-Au8, demonstrated that treatment with CNM-Au8 resulted in a 60% decreased risk of long-term all-cause mortality in those randomized to treatment with CNM-Au8 compared with those originally randomized to placebo, after adjusting for the estimated benefit after being switched to CNM-Au8 (Cox HR = 0.4; 95% CI: 0.19-0.85).
“The clinical correlation seen with plasma neurofilament change, as well as long-term survival using [rank-preserving structural failure time model], provides further independent evidence to strongly support CNM-Au8 as a potential treatment for ALS,” Benjamin Greenberg, MD, head of medical at Clene, said in the release. “The concordance of these long-term biomarker and survival results with previously reported clinical outcomes from two phase 2 ALS trials is encouraging.”
Additional data from the OLE has been collected for analysis, and Clene expects results will be released during the first quarter of this year, the company said in the release.