Release Details

• Late-breaker oral presentation by Professor Steve Vucic, University of Sydney Medical School and Northcott Chair of Neurology
• CNM-Au8®demonstrated significantly slowed disease progression, decreased risk of 6-point ALSFRS-R decline, and improved quality of life in early ALS patients in the RESCUE-ALS trial
• CNM-Au8 was well tolerated and safe with no drug-related serious adverse events or drug discontinuations

SALT LAKE CITY, Dec. 10, 2021 (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, presented data from the RESCUE-ALS Phase 2 study of CNM-Au8®, a catalytically active gold nanocrystal suspension, for the treatment of amyotrophic lateral sclerosis (ALS) in a late-breaking oral presentation at the 32nd International Symposium on ALS/MND, Dec. 10, 2021 at 1:05 pm ET. The presentation can be viewed after the presentation on Clene’s website at: https://invest.clene.com/overview/default.aspx.

At the late-breaking session, Professor Steve Vucic will present RESCUE-ALS study results that were supportive of CNM-Au8 efficacy for clinically meaningful functional endpoints in an early ALS population. Professor Vucic is Northcott Chair of Neurology, Director Brain and Nerve Research Center, Concord Clinical School, University of Sydney Department of Neurology, Concord Hospital, and one of the study’s expert advisors.

RESCUE-ALS, a 36-week randomized, placebo-controlled, Phase 2 clinical trial enrolled 45 patients with early ALS, demonstrated:

• Significant reduction in risk of disease progression in CNM-Au8 treated patients as defined by the occurrence of death, tracheostomy, or King’s Clinical Stage IV (non-invasive ventilation or gastrostomy).
• Significant reduction in risk of major functional decline in CNM-Au8 treated patients as measured by the proportion with ≥ 6-point reduction in ALSFRS-R from Baseline to Week 36.
• Significant improvement in quality of life as measured by ALS specific quality of life short form.
• Trend for a slower rate of decline in the summated motor unit index (MUNIX) score observed in in limb-onset ALS patients suggesting preservation of lower motor neurons.   The primary endpoint, percent change in MUNIX score from Baseline to week 36, was not met.
• Trend in preservation of respiratory function, which was more prominent in limb-onset patients.
• CNM-Au8 was well tolerated and safe with no drug related serious adverse events or drug discontinuations.

Professor Vucic commented, “CNM-Au8 therapy represents an entirely novel approach for treating neurodegenerative diseases. RESCUE-ALS showed that CNM-Au8 significantly slowed disease progression, functional decline, improved quality of life, and prolonged survival in ALS patients. Additionally, there was a trend in preservation of lower motor neuron and respiratory function, which was more prominent in limb-onset ALS patients. We recognize the potential for CNM-Au8 as a breakthrough treatment for ALS and look forward to upcoming results from larger clinical studies.”

Rob Etherington, Clene’s Chief Executive Officer, added, “The statistically significant RESCUE-ALS results in reducing the number of patients experiencing a 6-point decline in ALSFRS-R and the data suggesting improved survival are highly relevant to the fully-enrolled, Phase 2-3 Healey ALS Platform Trial. Our lead asset CNM-Au8 was one of the first therapies chosen for the HEALEY ALS Platform Trial, led by the Sean M Healey & AMG Center for ALS at Massachusetts General Hospital. The Healey-ALS Platform Trial has a primary endpoint of ALSFRS-R change, and involves significantly more subjects with more than six times the number of participants than were enrolled in RESCUE-ALS. We are grateful to our advisors, Professors Steve Vucic and Matthew Kiernan, their clinical teams, the FIGHTMND Association for substantially funding the trial, and especially to the ALS patients who participated and their families. We look forward to the topline results from the HEALEY ALS platform trial in the second half of 2022.”

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 patients with early amyotrophic lateral sclerosis (ALS). In the trial, 45 subjects were randomized 1:1 to receive either active treatment with CNM-Au8 (30 mg) or placebo in addition to their current standard of care over a 36-week treatment period. The objective of the trial was to assess the impact of CNM-Au8 on disease progression in patients with early-stage ALS through changes in motor unit index (MUNIX). MUNIX values were evaluated for four muscles in the hand, arm, and leg: the abductor digit minimi, abductor pollicis brevis, tibialis anterior, and biceps brachii from baseline through 36 weeks of treatment. CNM-Au8 was selected by FightMND of Australia, and Clene was provided a substantial grant to investigate efficacy in ALS utilizing novel neurophysiological endpoints including MUNIX at two clinical sites in Australia. For more information, please see ClinicalTrials.gov Identifier: NCT04098406.

RESCUE-ALS did not meet the primary endpoint, change in MUNIX score from Baseline to week 36. The study population that presented with bulbar-onset ALS (approximately 27%) of did not worsen in MUNIX as expected (< 10% placebo loss at Week 36). In the population with limb-onset ALS, who did worsen as expected (>40% placebo mean loss at Week 36), treatment with CNM-Au8 resulted in a 45% relative reduction in MUNIX score decline (p=0.0741), suggesting protection of lower motor neurons. Limb onset ALS accounts for approximately 70% of the overall ALS population. CNM-Au8 showed improvement for both limb and bulbar patients across important key clinical endpoints unrelated to MUNIX including the slowing the rate of ALS disease progression (p=0.0125), and the proportion of subjects with <6 point ALSFRS-R decline (p=0.035).

About CNM-Au8®, a gold nanocrystal suspension
Clene’s lead drug candidate, CNM-Au8, a catalytically active gold nanotherapeutic, is the result of a patented manufacturing breakthrough. The catalytically active nanocrystals of CNM-Au8 drive critical cellular energy producing reactions in the brain that enable neuroprotection and remyelination by increasing neuronal and glial resilience to disease-relevant stressors. CNM-Au8 crosses the blood-brain barrier and is not associated with the toxicities related to synthetic gold compounds or nanoparticles manufactured via alternative methods. CNM-Au8 is being evaluated in a Phase 3 registration trial for the treatment of amyotrophic lateral sclerosis (ALS). In the REPAIR Program Phase 2 open-label biomarker clinical trials, CNM-Au8 demonstrated target engagement in the treatment of Parkinson’s disease (PD) and multiple sclerosis (MS). REPAIR-PD has concluded, and REPAIR-MS will continue with the initiation of a second MS dosing cohort. Preclinical data, both published in peer-reviewed journals and presented at scientific congresses, demonstrate that treatment of neuronal cultures with CNM-Au8 improves survival of neurons, protects neurite networks, decreases intracellular levels of reactive oxygen species and improves mitochondrial capacity in response to cellular stresses induced by numerous disease-relevant neurotoxins. Oral treatment with CNM-Au8 improved functional behaviors in rodent models of ALS, MS and PD versus vehicle (placebo). 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 with potential first-in-class nanotherapeutics to treat energetic failure, an underlying cause of many neurological diseases. Our lead drug candidate, CNM-Au8, is an oral suspension of gold nanocrystals that drive critical cellular energetic metabolism in the central nervous system (CNS). CNM-Au8 increases energy production and utilization to accelerate neurorepair and improve neuroprotection. CNM-Au8 is currently being evaluated in a Phase 2/3 registration trial in amyotrophic lateral sclerosis (ALS) and a Phase 2 trial for the treatment of chronic optic neuropathy in patients with stable relapsing multiple sclerosis (MS). Clene has also advanced into the clinic an aqueous solution of ionic zinc and silver for anti-viral and anti-microbial uses. 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.

Forward-Looking Statements

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 consequently, you should not rely on these forward-looking statements as predictions of future events. Words such as “expect,” “estimate,” “project,” “budget,” “forecast,” “anticipate,” “intend,” “plan,” “may,” “will,” “could,” “should,” “believes,” “predicts,” “potential,” “might” and “continues,” and similar expressions are intended to identify such forward-looking statements. These forward-looking statements involve significant 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 may cause such differences include Clene’s ability to demonstrate the efficacy and safety of its drug candidates; the clinical results for its drug candidates, which may not support 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 protection of intellectual property for its technology and drugs; Clene’s reliance on third parties to conduct drug development, manufacturing and other services; Clene’s limited operating history and its ability to obtain additional funding for operations and to complete the licensing or development and commercialization of its drug candidates; the impact of the COVID-19 pandemic on Clene’s clinical development, commercial and other operations, as well as those risks more fully discussed in the section entitled “Risk Factors” in 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 U.S. Securities and Exchange Commission. Clene undertakes no obligation to release publicly any updates or revisions to any forward-looking statements to reflect any change in its expectations or any change in events, conditions or circumstances on which any such statement is based, subject to applicable law. All information in this press release is as of the date of this press release. The information contained in any website referenced herein is not, and shall not be deemed to be, part of or incorporated into this press release.

Media Contact
Maggie Beller
Russo Partners, LLC
Maggie.Beller@RussoPartnersLLC.com
+1-646-942-5631

Investor Contact
John Woolford
Managing Director, Westwicke
clene@westwicke.com
+1-443-213-0506

Source: Clene Inc.