Clene Announces Publication of Phase 2 CNM-Au8 Clinical Data for the Treatment of ALS

Clene Inc. and its wholly owned subsidiary Clene Nanomedicine, Inc. recently announced The Lancet’s journal eClinicalMedicine has published combined detailed analyses of the Phase 2 RESCUE-ALS study and its open-label extension trial. The article can be accessed via https://authors.elsevier.com/sd/article/S2589537023002134.

“Results from RESCUE-ALS study were important because they provided the first insights about the clinical benefits of CNM-Au8 in ALS patients and the duration of treatment required to detect them,” said Professor Steve Vucic, PhD, DSc, Northcott Chair of Neurology, The University of Sydney, and a co-investigator of the RESCUE-ALS study. “The treatment effect demonstrated a clear survival signal associated with CNM-Au8 treatment and evidence for delayed clinical worsening morbidity events such as tracheostomy and initiation of feeding tube during the double-blind placebo-controlled period. Additional functional benefits such as decreased worsening of ALSFRS-R scores are emerging later through the long-term open-label extension.”

“These results corroborate the mechanism that energetic support of neurons may protect CNS health and slow neurodegenerative disease progression and are a basis for continued clinical evaluation of this promising drug candidate,” said Professor Matthew Kiernan, PhD, DSc, Bushell Chair of Neurology, University of Sydney, and one of the trial’s clinical advisors. “In addition, our trial explored the utility of a novel primary endpoint, MUNIX, which has advanced the state of ALS research and will help future studies of other investigational ALS treatments.”

“We are pleased that CNM-Au8 results in RESCUE-ALS were concordant with the larger Phase 2b HEALEY ALS Platform Trial,” said Ben Greenberg, MD FAAN, Head of Medical at Clene. “We plan to report long-term follow-up data from this program later this year and meet with the FDA in the third quarter to identify next steps for our ALS program.”

RESCUE-ALS was a Phase 2 multi-center, randomized, double-blind, parallel-group, placebo-controlled trial, investigating the efficacy, safety, pharmacokinetics and pharmacodynamics of CNM-Au8 in 45 participants (73% limb onset, 27% bulbar onset) with early ALS over a 36-week treatment period. The primary endpoint was the percent change in the summated motor unit index (MUNIX) scores to week 36. The primary blinded comparative period was followed by an open-label extension (OLE) in which all participants received 30 mg of CNM-Au8 once-daily, with 90% (36 of 45 patients) entering the OLE. Clene announced topline results for RESCUE-ALS on November 2, 2021.

CNM-Au8 is an oral suspension of gold nanocrystals developed to restore neuronal health and function by increasing energy production and utilization. The catalytically active nanocrystals of CNM-Au8 drive critical cellular energy producing reactions that enable neuroprotection and remyelination by increasing neuronal and glial resilience to disease-relevant stressors. CNM-Au8® is a federally registered trademark of Clene Nanomedicine, Inc.

Clene is a clinical-stage biopharmaceutical company focused on revolutionizing the treatment of neurodegenerative disease by targeting energetic failure, an underlying cause of many neurological diseases. The company is based in Salt Lake City, UT, with R&D and manufacturing operations in MD. For more information, visit www.clene.com.