- Analyzes of the long-term, open-label extension of the RESCUE-ALS trial indicate improved survival compared to predictions derived from the validated ENCALS risk model
- Interim Results Demonstrate an Approximately 70% Decreased Risk of Death for Participants Who Participated in the Long-Term Open-Label Extension of RESCUE-ALS
SALT LAKE CITY, March 14, 2022 (GLOBE NEWSWIRE) — Clene Inc. (NASDAQ: CLNN) together with its subsidiaries “Clene” and its wholly-owned subsidiary Clene Nanomedicine, Inc., a clinical-stage biopharmaceutical company focused on revolutionizing in the treatment of neurodegenerative diseases, today announced several presentations of updated clinical trial results from the RESCUE-ALS and REPAIR Phase 2 trials in addition to new mechanistic preclinical data in ALS at the Clinical and Scientific Conference MDA 2022, which will be held March 13-16 in Nashville.
The first poster, titled “Results of the RESCUE-ALS Trial: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study of CNM-Au8 to Slow Disease Progression in ALS», selected as an oral presentation, and the second poster, «Evidence of a Potential Survival Benefit with CNM-Au8 Treatment from the Long-Term Open-Label Extension of the RESCUE-ALS Trial,” further support Clene’s lead drug candidate, CNM-Au8®, a suspension of catalytically active gold nanocrystals, as a potential disease-modifying treatment for amyotrophic lateral sclerosis (ALS).
RESCUE-ALS, a Phase 2 multicenter, randomized, double-blind, parallel-group, placebo-controlled trial, investigated the efficacy, safety, pharmacokinetics, and pharmacodynamics of CNM-Au8 in 45 participants with ALS early over a 36-week treatment period. Over the 36-week blinded period, CNM-Au8 treatment showed significant benefits: slowing of ALS progression (p=0.0125), decrease in the proportion of participants with a 6-point reduction in ALS Functional Rating Scale Revised (ALSFRS-R) (p=0.035), and improvement in quality of life as measured by the ALSSQOL-SF (ALS Specific Quality of Life) questionnaire (p= 0.018).
The second poster presented updated evidence of survival benefit with CNM-Au8 treatment that was reported in the long-term, open-label extension of the RESCUE-ALS trial for both active and placebo groups. Intermediate analyzes of observed survival versus estimated median survival derived from the validated ENCALS prediction model significantly favored CNM-Au8 treatment with a hazard ratio of 0.3 for participants who entered the open extension (HR 0.3; p = 0.01, log-rank test). CNM-Au8 was shown to be well tolerated and no safety signals were identified over 96 weeks of treatment.
The third poster, titled “Evidence of Brain Energy Metabolic Support with CNM-Au8 Treatment: Results of the REPAIR Phase 2 Clinical Trial with CNM-Au8“, demonstrated enhanced cerebral energy metabolism assessed by high-resolution magnetic resonance spectroscopy (31P-MRS). CNM-Au8 treatment resulted in improved brain NAD+/NADH ratio (primary endpoint, paired t test, p=0.0371). This result is explained both by the increase in NAD+ and a decrease in NADH (secondary endpoint, paired t-test, p=0.0264). CNM-Au8 treatment also resulted in homeostatic effects on brain energy-related phosphorus-containing metabolites, including ATP. Study participants whose whole-brain ATP levels were below the baseline population average saw their ATP levels rise significantly, while patients whose baseline levels were above the baseline mean decreased to the population mean (r2 = 0.711, p
The fourth poster accepted for presentation, “CNM-Au8 gold nanocatalysis protects neurons against degeneration and death in several in vitro SLA models,“demonstrates the ability of CNM-Au8 to promote neuronal survival and function in multiple in vitro ALS models: (i) treatment of rat spine primary motor neurons improves survival, preserves neurite networks, and reduces accumulation of cytoplasmic TDP-43 aggregates after excitotoxic glutamate injury or exposure to beta-amyloid oligomers (Aβ 1-42); (ii) processing of spinal motor neurons from transgenic SOD1g93A rats protected motor neurons from death upon exposure to excitotoxic glutamate in a cAMP-dependent manner and reduced SOD1 protein accumulation in a cAMP-independent manner; (iii) treatment of human induced pluripotent stem cell (iPSC)-derived neurons from C9ORF72 patients prevented neuronal death in response to stressors; and (iv) the survival and growth of iPSC-derived human motor neuron neurites in co-culture with toxic SOD1A4V Astrocytes derived from ALS patients were significantly and dose-dependently enhanced with CNM-Au8 treatment.
“The preclinical and clinical data presented at MDA further support Clene’s lead drug candidate, CNM-Au8, as a potential disease-modifying treatment for amyotrophic lateral sclerosis,” said Dr. Robert Glanzman, MD FAAN, MD -chief of Clene. “We look forward to the continued advancement of the ALS clinical program with top results from the HEALEY ALS Platform Trial expected in the second half of the year.”
Rob Etherington, CEO of Clene, added: “This is an exciting time for Clene as we build a larger body of scientific and clinical evidence in support of our CNM-Au8. We will continue to further validate our findings on neurological function and survival pending the results of larger ongoing clinical studies.
About CNM-Au8®, a suspension of gold nanocrystals
CNM-Au8 is an oral suspension of gold nanocrystals developed to restore neuronal health and function by increasing energy production and utilization. Catalytically active CNM-Au8 nanocrystals drive critical cellular energy-producing responses that enable neuroprotection and remyelination by increasing neuronal and glial resilience to disease-related 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 diseases by targeting energy failures, 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 https://clene.com or follow us on Twitter, LinkedIn and Facebook.
This press release contains “forward-looking statements” which are intended to be covered by the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Clene’s actual results may differ from its expectations, estimates and projections. and, therefore, you should not rely on these forward-looking statements as predictions of future events. Words such as “expect”, “estimate”, “project”, “budget”, “expect”, “anticipate”, “intend”, “plan”, “may”, “will” , “may”, “should”, “believes”, “predicts”, “potential”, “could” and “continues”, and similar expressions are intended to identify such forward-looking statements. These forward-looking statements involve important known and unknown risks and uncertainties, many of which are beyond Clene’s control and could cause actual results to differ materially and adversely from expected results. Factors that could drive such differences include Clene’s ability to demonstrate the efficacy and safety of its drug candidates; the clinical results of its drug candidates, which may not warrant further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; Clene’s ability to achieve commercial success for its marketed products and drug candidates, if approved; Clene’s ability to obtain and maintain intellectual property protection for its technology and medicines; Clene’s reliance on third parties to carry out drug development, manufacturing and other services; Clene’s limited operating history and its ability to obtain additional financing for its operations and to complete the licensing or development and commercialization of its drug candidates; the impact of the COVID-19 pandemic on the clinical development, business and other operations of Clene, as well as the risks discussed in greater detail in the section entitled “Risk Factors” of Clene’s Annual Report on Form 10-K , as well as discussions of potential risks, uncertainties and other important factors in Clene’s subsequent filings with the United States Securities and Exchange Commission. Clene undertakes no obligation to publicly release updates or revisions to any forward-looking statement to reflect any change in its expectations or any change in events, conditions or circumstances on which any such statement is based, subject to law. applies. All information contained in this press release speaks as of the date of this press release. The information contained in any website referenced herein is not, and should not be considered, a part of or incorporated into this press release.
Managing Director, Westwicke
Erica Fiorini, Ph.D., or David Schull
Russo Partners, LLC