Clene Nanomedicine to Present RESCUE-ALS Phase 2 Data in Late-Breaking Session at 32nd International Symposium on ALS/MND

December 16, 2021

Clene Inc. 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.”

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