Clene Nanomedicine Presents Updated Long-Term Survival Data from RESCUE-ALS Participants at 2022 AANEM Scientific Conference

• Treatment with CNM-Au8 significantly improved long-term survival with   approximately a 70% decreased risk of mortality vs. original placebo randomization
• Comparable survival benefits were also shown   compared to ENCALS predicted median survival
• C   NM-Au8 treatment was well-tolerated, and there were no significant safety findings reported
  

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 diseases, today announced presentation of updated survival results from the Phase 2 RESCUE-ALS trial open-label extension (OLE) at the 2022 American Association of Neuromuscular and Electrodiagnostic Medicine (AANEM) Clinical & Scientific Conference, taking place September 21-24 in Nashville, Tennessee.

The poster titled, " Evidence for a Potential Survival Benefit in Amyotrophic Lateral Sclerosis with CNM-Au8 Treatment: Interim Results from the RESCUE-ALS trial Long-Term Open Label Extension   ," provides ongoing evidence supporting the clinical benefits of Clene's lead drug candidate, CNM-Au8 ^® , a catalytically active gold nanocrystal suspension that holds promise as a disease-modifying therapy for amyotrophic lateral sclerosis (ALS). Specifically, the poster evaluated the survival benefit associated with long-term CNM-Au8 treatment. Updated interim analyses of all-cause mortality with up to 137 weeks of follow-up from randomization comparing participants originally randomized to CNM-Au8 to participants originally randomized to placebo demonstrated a significant survival benefit with CNM-Au8 treatment, resulting in approximately a 70% decreased risk of death (HR = 0.29, p=0.01). Sensitivity analyses of observed survival compared to predicted median survival derived from the published ENCALS prediction model based on each participant's baseline study characteristics, with a data cutoff of August 31, 2022, also demonstrated a significant survival benefit with CNM-Au8 treatment (HR = 0.36, p=0.003). CNM-Au8 treatment was well-tolerated, and there were no significant safety findings reported during the OLE .

"These clinical and survival data from RESCUE-ALS contribute to the growing body of evidence supporting the potential for CNM-Au8 as a disease-modifying therapy for amyotrophic lateral sclerosis (ALS)," said Robert Glanzman, MD, FAAN, Clene's Chief Medical Officer.

Rob Etherington, Clene's CEO, added, "The impressive ALS survival benefits seen with CNM-Au8 treatment corroborate our thesis – energetic support of neurons may protect CNS health and slow neurodegenerative disease progression. We look forward to upcoming results from the HEALEY ALS Platform Trial and advancing CNM-Au8 development in ALS and other neurodegenerative diseases, including multiple sclerosis and Parkinson's."

About Rescue-ALS
RESCUE-ALS, a Phase 2 multi-center, randomized, double-blind, parallel-group, placebo-controlled trial, examined 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% (36/45 patients) entering the OLE. The primary endpoint was not significant, driven by limited MUNIX decline in bulbar-onset participants. However, in a prespecified analyses of limb-onset participants who demonstrated the expected decline in MUNIX scores, there was a strong trend for reducing MUNIX scores (p=0.074). There was a significant reduction in ALS disease progression evident in CNM-Au8 treated participants at week 36 (p=0.0125), and the risk of experiencing significant (≥ 6-point) decline in ALSFRSR was significantly reduced in the CNM-Au8 treated patients (p=0.035). Furthermore, CNMAu8 treated participants demonstrated improved quality of life on the ALSSQOL-SF (p=0.018). Importantly, there were no significant CNM-Au8 related adverse effects reported.

About CNM-Au8 ^®
CNM-Au8 is Clene's lead asset in mid- and late-stage clinical development for the treatment of multiple sclerosis and amyotrophic lateral sclerosis. An oral suspension of gold nanocrystals, CNM-Au8 was developed to protect 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.

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.

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Source: Clene Inc.