Clene Nanomedicine Presents Updated Interim Data from Phase 2 Multiple Sclerosis Programs at ACTRIMS Forum 2021

Rhea-AI Summary

Clene Inc. (NASDAQ: CLNN) announced positive interim data from its studies, VISIONARY-MS and REPAIR-MS, presented at the ACTRIMS Forum 2021. Interim results demonstrate CNM-Au8's potential in improving neurological functions in multiple sclerosis (MS) patients. The VISIONARY-MS trial showed significant enhancements in MS Functional Composite (MSFC) scores compared to baseline (p<0.0001). The REPAIR-MS study indicated notable metabolic improvements in CNS markers, suggesting a homeostatic effect of CNM-Au8. Unblinded topline data for both studies are expected in 2022.

Positive

• Interim data from VISIONARY-MS show significant improvements in MSFC scores (p<0.0001).
• REPAIR-MS study demonstrates improvements in NAD+/NADH ratio and ATP levels.
• CNM-Au8 shows potential for meaningful neurological improvements in MS patients.

Negative

• None.

• Blinded interim data from VISIONARY-MS continue to support the potential of CNM-Au8 to drive meaningful neurological functional improvements in MS patients
  
• Interim REPAIR-MS results provide further evidence of CNM-Au8’s homeostatic effect on brain bioenergetics in MS patients

SALT LAKE CITY, Feb. 25, 2021 (GLOBE NEWSWIRE) -- Clene Inc. (NASDAQ: CLNN) (along with its subsidiaries, “Clene”) today announced that its wholly owned subsidiary Clene Nanomedicine, Inc., a clinical-stage biopharmaceutical company, will present blinded interim data from its VISIONARY-MS study and updated interim data from its REPAIR-MS study in on-demand presentations at the Americas Committee for Treatment and Research in Multiple Sclerosis (“ACTRIMS”) ACTRIMS Forum 2021 taking place virtually from February 25-27, 2021. Together, these complementary Phase 2 studies evaluate the efficacy, safety, and central nervous system (CNS) metabolic effects of Clene’s lead drug candidate CNM-Au8, a bioenergetic nanocatalyst, in multiple sclerosis (MS) patients.

Updated blinded interim data from VISIONARY-MS, a double-blind, placebo-controlled Phase 2 trial evaluating the efficacy and safety of CNM-Au8 as a remyelinating and neuro-reparative treatment in MS patients will be presented. The analyses compare changes in Multiple Sclerosis Functional Composite (MSFC) scores over the study treatment period to the baseline values of study participants with mild disease, as defined by Baseline Expanded Disability Status Scale (EDSS) scores of 1.5 or less. Notably, baseline scores for these participants demonstrate less neurological impairment than those of the overall study population, providing a valid comparator group. Changes in the four MSFC sub-scales (low contrast letter acuity, symbol digit modalities test, 9-hole peg test, and timed 25-foot walk) were compared to baseline scores of the comparator group with mild disease. These comparisons were performed at each study time-point (Weeks 12, 24, and 36). At each visit, the overall study population (randomized 2:1 active CNM-Au8 to placebo) showed notable increasing mean improvements in overall MSFC scores and key MSFC sub-scales compared to the comparator group (mixed-effects model; p<0.0001 vs. baseline). These data support CNM-Au8’s potential to drive meaningful neurological improvements in MS patients. Unblinded topline data are expected in the first half of 2022.

In a second presentation, interim results from REPAIR-MS, an ongoing Phase 2 study utilizing high-resolution magnetic resonance spectroscopy (³¹P-MRS) to evaluate the effects of orally administered CNM-Au8 on the metabolic profile of MS patient brains, demonstrate significant CNS target engagement of CNM-Au8. Data presented at ACTRIMS continue to show catalytic bioenergetic improvements in NAD+/NADH ratio and adenosine triphosphate (ATP) levels, two key CNS metabolic markers. Together, these data indicate that orally administered CNM-Au8 has a homeostatic effect on brain bioenergetics. Clene expects to complete REPAIR-MS and report topline data in the second half of 2021.

“As VISIONARY-MS and REPAIR-MS continue to progress, we see consistent and complementary results,” said Robert Glanzman, MD, FAAN, chief medical officer of Clene. “Interim data from the REPAIR-MS program provide further evidence for CNM-Au8 to enter the brain and reverse bioenergetic failure, a key driver in the pathophysiology of MS and other neurodegenerative diseases. These mechanistic results provide important support for the updated, blinded interim VISIONARY data analysis, which suggest that CNM-Au8 has the potential to drive clinically meaningful improvements in recognized MS functional endpoints when administered in addition to standard of care. We look forward to the continued advancement of these trials.”

Rob Etherington, president and chief executive officer of Clene, added, “The results we’ve seen from our MS clinical program to date continue to be very promising. They demonstrate CNM-Au8’s unique mechanism of action as well as its potential to address the unmet need for neuro-reparative MS therapies. Unlike currently approved treatments, CNM-Au8 acts to improve cellular bioenergetics and thus enhance processes that are fundamental to neuronal survival, repair and function. This mechanism is broadly applicable, and leaves CNM-Au8 well positioned to potentially improve the lives of patients living with neurodegenerative disease.”

Details on the poster presentations, which are available on the “Events and Presentations” section of the Clene website, are shown below.

Title: Effects of Nanocatalysis on CNS Bioenergetic Markers in Patients Treated with CNM-Au8: Interim Results from a Phase 2 ³¹Phosphorous Magnetic Resonance Imaging Study in Relapsing MS
Presenter: Robert Glanzman, MD, FAAN, chief medical officer of Clene

Title: Update to a Phase 2 Clinical Trial of Catalytic Gold Nanocrystals, CNM-Au8, for the Treatment of Chronic Optic Neuropathy
Presenter: Robert Glanzman, MD, FAAN, chief medical officer of Clene

About CNM-Au8

Clene’s lead drug candidate, CNM-Au8, a bioenergetic nanocatalyst, is a stable, aqueous suspension of catalytically active gold (Au) nanocrystals. In a patented breakthrough, clean surfaced nanocrystalline CNM-Au8 drives critical cellular bioenergetic reactions in the brain to increase cellular energy, accelerate neurorepair, and improve neuroprotection. CNM-Au8 crosses the blood-brain barrier and is not associated with the toxicities related to synthetic gold compounds or synthetic nanoparticle chemistry. CNM-Au8 is currently being evaluated in a Phase 3 registration trial in amyotrophic lateral sclerosis (ALS), a Phase 2 trial for disease progression in patients with ALS, a Phase 2 trial for the treatment of chronic optic neuropathy in patients with stable relapsing multiple sclerosis (MS), and a Phase 2 for brain target engagement study in Parkinson’s disease (PD) and MS. CNM-Au8 has demonstrated safety in Phase 1 studies in healthy volunteers and has shown both remyelination and neuroprotective effects in multiple preclinical (animal) models. 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 multiple disease-relevant neurotoxins. Oral treatment with CNM-Au8 improved functional behaviors in rodent models of ALS, MS, and PD versus vehicle (placebo).

About VISIONARY-MS

VISIONARY-MS is a Phase 2, multi-center, double-blind, randomized, placebo-controlled trial evaluating the efficacy and safety of CNM-Au8 as a remyelinating and neuro-reparative treatment in stable relapsing multiple sclerosis (MS) patients with chronic visual impairment. 150 participants are being enrolled through 10 expert MS clinical trial sites in Australia. Subjects are randomized 1:1:1 (high-dose:low-dose:placebo). The primary endpoint is improvement in low contrast letter acuity (LCLA) from baseline to week-24. Key secondary endpoints include improvements from baseline to week-24 in the remaining modified-Multiple Sclerosis Functional Composite (MSFC) subscales (symbol digit modalities test, 9-hole peg test, and timed 25-foot walk). Interim blinded data from the Phase 2 VISIONARY-MS trial presented at ACTRIMS Forum 2021 Meeting demonstrated statistically significant improvements in both LCLA scores versus baseline in the overall study population and across the averaged components of the modified MSFC scale (p<0.001). Full enrollment is expected by the end of 2021, subject to ongoing COVID-19 related research restrictions at MS clinical trial sites. For more information see ClinicalTrials.gov Identifier: NCT03536559.

About REPAIR-MS

REPAIR-MS is a Phase 2, single-center, active only, sequential group, investigator blinded study to assess the metabolic effects, safety, pharmacokinetics and pharmacodynamics of CNM-Au8 in patients diagnosed with multiple sclerosis (MS) within 15 years of screening. Investigators and participants are blinded to dose. Participants receive orally delivered CNM-Au8, the concentrated nanocrystalline gold (Au) suspension, daily each morning for at least 12 weeks. Participants undergo ³¹P-MRS brain imaging scans to semi-quantitatively compare bioenergetic metabolites at baseline, prior to and after CNM-Au8 administration. The objective of this study is to demonstrate target engagement for CNM-Au8 on CNS biomarkers related to bioenergetics and neuronal metabolism in patients with MS. The study is taking place at the University of Texas Southwestern Medical Center with a team of internationally recognized experts in brain imaging and the treatment of disorders of the CNS. Interim results from REPAIR-MS presented at the ACTRIMS Forum 2021 Meeting showed improvements across key CNS bioenergetic metabolites, including total nicotinamide adenine dinucleotide (NAD) levels, NAD+/NADH ratio (primary endpoint) and adenosine triphosphate (ATP) levels (secondary endpoint), indicating a homeostatic effect of CNM-Au8 on brain bioenergetics. Topline data are expected in 2H 2021. For more information see ClinicalTrials.gov Identifier: NCT03993171.

About Clene

Clene, a clinical-stage biopharmaceutical company focused on neurodegenerative disease, has innovated a novel nanotechnology platform to create a new class of drugs—bioenergetic nanocatalysts. Clene’s lead drug candidate, CNM-Au8, is a the concentrated nanocrystalline gold (Au) suspension that drives critical cellular bioenergetic reactions in the CNS. CNM-Au8 increases cellular energy to accelerate neurorepair and improve neuroprotection. Currently, CNM-Au8 is being investigated for efficacy and safety in a Phase 3 registration trial for ALS and in Phase 2 trials for Multiple Sclerosis and Parkinson’s Disease. 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.

Forward-Looking Statements

This press release contains "forward-looking statements" within the meaning of 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 recently filed registration statement on Form S-4, 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
Andrew Mielach
LifeSci Communications
(646) 876-5868
amielach@lifescicomms.com

Investor Contact
Bruce Mackle
LifeSci Advisors, LLC
(929) 469-3859
bmackle@lifesciadvisors.com

Source: Clene Inc.

FAQ

What are the results of Clene's VISIONARY-MS study?

The VISIONARY-MS study's interim results show significant improvements in MSFC scores compared to baseline, indicating the potential of CNM-Au8 in enhancing neurological functions in MS patients.

What does the REPAIR-MS study data indicate?

The REPAIR-MS study shows that CNM-Au8 improves key CNS metabolic markers, including NAD+/NADH ratio and ATP levels, suggesting a positive effect on brain bioenergetics.

When will Clene report topline data for CNM-Au8 studies?

Topline data from both the VISIONARY-MS and REPAIR-MS studies are expected to be released in 2022.

What is CNM-Au8 and its significance?

CNM-Au8 is Clene's lead drug candidate, a bioenergetic nanocatalyst aimed at improving cellular energy and neurorepair in patients with multiple sclerosis.