Interim Phase 2 data indicate that CNM-Au8 has a homeostatic effect on brain bioenergetics
Topline data from a Phase 2 ALS trial and two Phase 2 target engagement studies in MS and Parkinson’s disease expected in 2H 2021
Full enrollment in Phase 3 registrational trial in ALS expected in mid-2021
Cash of $59.3 million as of December 31, 2020
SALT LAKE CITY, March 29, 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 dedicated to revolutionizing the treatment of neurodegenerative disease using nanocatalysis, today reported its full year 2020 operating and financial results.
“We are thrilled to enter this new year as a public company following the recent close of our merger and concurrent financing, which was enabled by several key milestones achieved in 2020,” said Rob Etherington, president and chief executive officer of Clene. “Over the last year, we continued advancing our clinical Phase 2 studies and Phase 3 study of CNM-Au8, a novel bioenergetic nanocatalyst, in ALS, multiple sclerosis (MS), and Parkinson’s disease (PD) and are very pleased to have reported promising preliminary clinical results that support its neuroprotective and remyelinating properties. CNM-Au8’s potential as a neuroprotective and neuro-reparative nanocatalyst is further validated by its selection as one of the first three therapies to be evaluated in the Phase 3 HEALEY ALS Platform trial, the first-ever platform trial for ALS. We are thankful to our patients, their care-givers, the MGH Healey ALS Center, the Northeast ALS Consortium clinical sites, and the entire Clene team, whose dedication throughout the pandemic drove this sustained progress.”
Mr. Etherington continued, “Looking ahead, we are well-positioned to continue the momentum of our clinical programs. In 2021 we expect to report topline safety and efficacy data from our Phase 2 RESCUE-ALS trial, complete enrollment in the registrational HEALEY ALS Platform trial, and report topline data from our Phase 2 brain target engagement studies in MS and PD. We are grateful for the continued support of our investors, which has enabled Clene to fund these robust R&D efforts. It is with great energy and dedication that Clene will continue working towards its mission of developing bioenergetic nanocatalysts as a new class of drugs to treat significant unmet medical needs in neurodegenerative diseases while driving further value for its stakeholders.”
Full Year 2020 and Recent Highlights
CNM-Au8 for the treatment of amyotrophic lateral sclerosis (ALS):
Presented blinded interim data from the Phase 2 RESCUE-ALS trial at the 31st International Symposium on ALS/MND:RESCUE-ALS is a Phase 2, multi-center, randomized, double-blind, placebo-controlled study designed to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of CNM-Au8, a neuro-reparative nanocatalyst, in early symptomatic ALS patients. The study’s primary endpoint evaluates improvements in muscle function using a sophisticated electromyography technique called Motor Unit Number Index over four muscles, MUNIX(4), which quantitatively reflects loss or preservation of motor neurons in ALS. Interim results from the trial showed that more than 40% of enrolled patients (active CNM-Au8 or placebo) who completed week 12 experienced improvements in motor neuron function as assessed by MUNIX(4). These data, though blinded, suggest that CNM-Au8 may have neuro-reparative potential in ALS patients. Clene expects to report topline data from the RESCUE-ALS study in the second half of 2021.
Full enrollment in Phase 3 registrational trial in ALS expected in mid-2021:
The HEALEY ALS Platform trial is a multi-center, multi-regimen, placebo-controlled, Phase 3 / registrational clinical program evaluating the safety and efficacy of multiple investigational products for the treatment of ALS. This first-ever ALS platform trial is designed to reduce trial time, reduce costs, and increase patient participation in developing novel therapies for ALS. It includes substantial financial support from philanthropic donors and foundations and provides access to 54 expert ALS clinical trial sites across the U.S. CNM-Au8 was selected as one of the first drug regimens to be evaluated in the HEALEY ALS Platform Trial. In the first quarter of 2021, the trial reached 50% of its target enrollment. Full enrollment of 160 patients into the CNM-Au8 portion of the trial is anticipated mid-2021, with topline data expected in the first half of 2022.
CNM-Au8 for the treatment of multiple sclerosis (MS):
Presented blinded interim data from the Phase 2 VISIONARY-MS trial at ACTRIMS Forum 2021: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 MS patients with chronic visual impairment. Updated blinded interim data from VISIONARY-MS continue to support the potential of CNM-Au8 to drive meaningful neurological improvements in MS. Interim blinded data from all enrolled participants (randomized 2:1, active CNM-Au8 to placebo) showed clinically relevant mean improvements in overall Multiple Sclerosis Functional Composite (MSFC) scores and key MSFC sub-scales compared to a valid comparator group (EDSS ≤1.5) that presented with less neurological impairment than that of the overall study population at baseline (mixed-effects model; p