Clene Reports Significantly Decreased Mortality in RESCUE-ALS Long-Term Open Label Extension Trial

• As of the July 5, 2022, data cutoff, early CNM-Au8^® treatment resulted in a significant survival benefit (5 CNM-Au8 deaths vs. 14 placebo deaths, HR=0.301, p=0.0143)
• CNM-Au8 treatment was well-tolerated, and there were no significant safety findings reported during this open-label trial
• Top-line results from HEALEY ALS Platform Trial are expected in current quarter

SALT LAKE CITY, July 14, 2022 (GLOBE NEWSWIRE) — Clene Inc. (Nasdaq: CLNN) (along with its subsidiaries, “Clene”) and its wholly owned subsidiary Clene Nanomedicine Inc., a clinical-stage biopharmaceutical company focused on revolutionizing the treatment of neurodegenerative disease, today reported significantly improved survival in ALS patients initially treated with CNM-Au8 compared to initially randomized placebo treated participants during the long-term open-label extension of its RESCUE-ALS trial.

“We are very pleased to see these results and the apparent survival benefit that our investigational drug, CNM-Au8, appears to provide to people living with ALS,” said Rob Etherington, President and CEO of Clene. “At this point, we are awaiting top-line data from the HEALEY ALS Platform Trial, which focuses on endpoints measuring patient function, survival and breathing over a six-month period in a much larger cohort. Clene expects to announce these results this quarter. Based on the larger number of patients treated in the HEALEY trial and the higher dose of CNM-Au8 being tested, we are optimistic that we will be able to adequately characterize the effects of our drug on this devastating disease.”

Study participants in RESCUE-ALS were randomized 1:1 to receive 30 mg of CNM-Au8 or placebo daily for 36 weeks during the double-blind portion of the study, followed by an open-label period that extended treatment up to 130 weeks from randomization. The trial enrolled 45 participants (n=23 on active treatment, and n=22 receiving placebo), and survival was compared by treatment group from randomization through the latest vital status observation with a cut-off as of July 5, 2022, for this interim analysis. Participants in the placebo-treated group, who received either no treatment or a nine-month delay in treatment initiation with CNM-Au8, were compared to participants treated daily with CNM-Au8 from randomization.

Survival data were obtained for 43 of 45 study participants, and one participant in each group was lost to follow-up. There were five deaths in the group originally randomized to CNM-Au8 and 14 deaths in the group originally randomized to receive placebo. Median survival from randomization for the CNM-Au8 group was undefined due to insufficient mortality events, and median survival for the placebo group was 23.1 months. Unadjusted Kaplan-Meier survival analyses demonstrated a significant survival benefit with participants initially randomized to CNM-Au8 treatment versus those initially randomized to placebo, resulting in a 70% decreased risk of death, log-rank hazard ratio = 0.301 (95% CI: 0.122 to 0.742, p = 0.0143). Sensitivity analyses substituting death in place of lost to follow-up censoring resulted in concordant findings (Hazard Ratio: 0.338, 95% CI: 0.143 to 0.797, p = 0.0182). The treatment was well-tolerated, and there were no significant safety findings reported during the long-term open label trial period. These data will be reported at an upcoming scientific congress.

About Clene
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, Utah, with R&D and manufacturing operations in Maryland. For more information, please visit www.clene.com or follow us on Twitter, LinkedIn and Facebook.

About CNM-Au8^®
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.

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Source: Clene Inc.