Clene (NASDAQ: CLNN), a late clinical-stage biopharmaceutical company focused on improving mitochondrial health and protecting neuronal function to treat neurodegenerative diseases, announced positive results of two independently conducted Phase 2 clinical trials, RESCUE-ALS and HEALEY ALS Platform Trials, of CNM-Au8 for the treatment of amyotrophic lateral sclerosis (“ALS”) (https://ibn.fm/ktu4u).
The new CNM-Au8 biomarker and clinical efficacy data was submitted to the FDA to supplement original data submitted in late 2023 and is intended to guide the granted FDA Type C interaction expected in the third quarter of 2024 to discuss an accelerated approval regulatory pathway, according to a company press release.
One of the key biomarkers of ALS disease progression and mortality risk is the level of neurofilament light (“NfL”) in plasma. Participants in the HEALEY ALS Platform Trial with substantial NfL declines saw significant improvement in survival rates, functional status, and combined assessment of function and survival, compared to NfL non-responders.
Independent of NFL responder status, participants who underwent long-term treatment with CNM-Au8 at a dose of 30 mg were found to have improved survival rates, both in the RESCUE-ALS and the HEALEY ALS Platform Trials, using updated long-term follow-up of survival status, the CLENE announcement said.
The two clinical trials also revealed consistent nicotinamide adenine dinucleotide (“NAD”) and glutathione improvements with CNM-Au8 treatment, associated with participants who were NfL responders, supporting a dual mechanism of action of neuronal metabolic support and decreased oxidative stress, and indicating target engagement in ALS patients.
The strong safety profile of CNM-Au8, with NfL biomarker response now linked to survival evidence, and new information on mechanisms of action, support proceeding to a confirmatory Phase 3 clinical trial and regulatory discussions on approval pathways, according to Merit Cudkowicz, M.D., Chair, Neurology Department, Massachusetts General Hospital, Director, Sean M Healey & AMG Center for ALS, and the Principal Investigator of the HEALEY ALS Platform Trial.
With more than 650 patient years of safety data, CNM-Au8 continued to show a robust safety profile without any observed safety signals or any significant safety concerns or adverse effects identified by any investigators to date. “The risk-benefit assessment evidence of CNM-Au8 is strong. Our next step is discussing this new CNM-Au8 biomarker and efficacy data with the FDA, with the hope that ALS patients will benefit from this drug, sooner rather than later,” said CEO and President of Clene, Rob Etherington.
Clene also shared its latest findings and positive developments at the Canaccord Genuity 44th Annual Growth Conference & Private Company Showcase, on August 13. During the conference, the company presented its latest data and discussed its strategic plans with investors and also hosted one-on-one investor meetings. The conference provided Clene with an opportunity to engage with stakeholders and showcase its innovative approach to treating neurological disorders. The company emphasized its commitment to advancing CNM-Au8 through the clinical development pipeline and ultimately bringing it to market.
For more information, visit the company’s website at www.Clene.com.
NOTE TO INVESTORS: The latest news and updates relating to CLNN are available in the company’s newsroom at https://ibn.fm/CLNN
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