Full Press Release Details
Clene Reports Full Year 2020 Operating and Financial
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 - 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
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
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
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<0.0001 vs. baseline). These data support CNM-Au8's potential
to drive meaningful neurological improvements in MS patients. Subject to ongoing pandemic-related research restrictions at MS clinical
trial sites, enrollment will advance through 2021.
Presented interim data from the Phase 2 REPAIR-MS
trial at ACTRIMS Forum 2021:
REPAIR-MS is a single-center, active-only, sequential
group, investigator-blinded study to assess the central nervous system (CNS) metabolic effects, safety, pharmacokinetics, and pharmacodynamics
of CNM-Au8 in MS patients. The study utilizes high-resolution magnetic resonance spectroscopy (31P-MRS) to evaluate the effects
of orally administered CNM-Au8 on the metabolic profile of MS patient brains. Updated interim data demonstrate significant CNS target
engagement of CNM-Au8 with 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.
CNM-Au8 for the treatment of Parkinson's
Presented interim data from the Phase 2 REPAIR-PD
trial at MSVirtual2020 Meeting:
REPAIR-PD is a single-center, active-only, sequential
group, investigator-blinded study to assess the CNS metabolic effects, safety, pharmacokinetics and pharmacodynamics of CNM-Au8 in PD
patients. As described in the REPAIR-MS trial above, this study also utilizes high-resolution magnetic resonance spectroscopy (31P-MRS)
to evaluate the effects of orally administered CNM-Au8 on the metabolic profile within the brain. Preliminary data has demonstrated CNM-Au8-mediated
modulation of key brain bioenergetic metabolites in PD patients. Clene expects to report topline data from REPAIR-PD in the second half
of 2021 and to launch an additional Phase 2 PD efficacy trial (RESCUE-PD) by the end of 2021.
Awarded Michael J. Fox Foundation (MJFF) Grant
to accelerate the development of CNM-Au8 in PD
The MJFF funding will support preclinical studies
in two complementary models of PD that will be led by Dr. Karen Ho, head of translational medicine at Clene, in collaboration with Prof.
Michela Deleidi, Helmholtz Young Investigator at the German Center for Neurodegenerative Diseases (DZNE), and Dr. James Koprich, chief
scientific officer of Atuka, Inc. The project will further evaluate the effects of CNM-Au8 on the survival and bioenergetic profiles of
human PD patient dopaminergic neurons in the presence of PD-related neurotoxins and characterize the effects of CNM-Au8 on motor behaviors
and neuronal survival in an animal model of PD, both of which will facilitate the advancement of CNM-Au8 into Phase 2 efficacy trials
CNM-ZnAg for the treatment of infectious diseases,
Initiated a Phase 2 study of CNM-ZnAg for patients
with COVID-19 in Brazil:
CNM-ZnAg is Clene's second key asset intended
for broad anti-viral and anti-microbial use. Clene has received official ANVISA approval in Brazil to commence its multicenter, randomized,
double-blind, placebo-controlled study assessing the efficacy and safety of CNM-ZnAg liquid solution in a Phase 2 clinical study in acutely
symptomatic, non-hospitalized COVID-19 patients. The primary endpoint will evaluate the incidence of hospitalization at day 28, with secondary
endpoints assessing time to symptom resolution. The trial is expected to enroll approximately 276 patients randomized 1:1:2 to receive
either a low or high dose of CNM-ZnAg or placebo in a double-blind fashion, in addition to standard supportive care. Enrollment in the
trial is expected to be completed in mid-2021, with results anticipated in the second half of 2021.
Corporate Highlights:
In December 2020, Clene closed a merger with Tottenham
Acquisition I Limited, a special purpose acquisition company. Clene received proceeds of approximately $31.8 million from the transaction,
which included funds held in Tottenham's trust account and a concurrent private investment in public equity financing