Full Press Release Details
First Quarter 2021 Operating and Financial Highlights
Interim Phase 2 data indicate that CNM-Au8 has
a homeostatic effect on brain bioenergetics and support its potential to drive meaningful neurological functional improvements in MS patients
Awarded Michael J. Fox Foundation (MJFF) grant
to accelerate development of CNM-Au8 as a treatment for Parkinson's disease
Phase 2 RESCUE-ALS trial and Phase 2 target
engagement trials in MS and PD remain on track for topline data in 2H 2021
Cash of $48.0 million as of March 31, 2021
SALT LAKE CITY, May 11, 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 bioenergetic nanocatalysis, today reported its first quarter
2021 operating and financial results.
"Clene is off to a strong start in 2021, having made great progress
across its programs," said Rob Etherington, president and chief executive officer of Clene. "During the first quarter, we
announced compelling interim data in MS patients indicating that CNM-Au8 has a homeostatic effect on brain bioenergetics, which may allow
it to ultimately slow or halt disease progression. We also received important external validation for our Parkinson's Disease program
in the form of a grant from the Michael J. Fox Foundation that will enable us to accelerate CNM-Au8's development in this high unmet
need indication. Looking ahead, we are on track to achieve multiple key inflection points across our pipeline over the remainder of the
year, the most notable being the announcement of topline data from our placebo-controlled, Phase 2 RESCUE-ALS study in the second half
of 2021. With our strong cash position, we will continue to work expeditiously on our mission to change the treatment paradigm for patients
with neurodegenerative diseases."
First Quarter 2021 and Recent Highlights
CNM-Au8 for the treatment of amyotrophic lateral sclerosis (ALS):
Phase 3 registrational trial in ALS on track for full enrollment
in second half of 2021
In the first quarter of 2021, the HEALEY ALS Platform trial, 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, reached 50% of its target enrollment for the first three of the ongoing studies, including CNM-Au8.
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 more than
50 expert ALS clinical trial sites across the U.S. Full enrollment of 160 patients into the CNM-Au8 portion of the trial is anticipated
before the end of 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
Updated blinded interim data from VISIONARY-MS continue to support the potential of CNM-Au8 to drive meaningful neurological improvements
in people with MS. Interim blinded data from all enrolled participants (randomized 2:1, active CNM-Au8 to placebo) showed clinically relevant
mean improvements in key MSFC sub-scales, as well as a composite score including all of the MSFC sub-scales. The overall study population's
progress on study was compared to the mildest sub-population (EDSS 1.5) scores at Baseline (mixed-effects model; p<0.0001 vs. baseline).
These findings 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
REPAIR-MS utilizes high-field strength magnetic resonance spectroscopy (31P-MRS) to evaluate the effects of orally administered
CNM-Au8 on the brain metabolic profile in people with MS. Updated interim data from the trial show significant CNS target engagement
of CNM-Au8 with catalytic bioenergetic improvements in NAD+/NADH ratio and normalization of adenosine triphosphate (ATP) levels, two
key CNS metabolic markers. Together, these data indicate that orally administered CNM-Au8 has positive effects on brain bioenergetics
in MS patients. Clene expects to complete REPAIR-MS and report topline data in the second half of 2021.
CNM-Au8 for the treatment of Parkinson's disease (PD):
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 partnership with academic and CRO collaborators. 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 in PD patients.
CNM-ZnAg for the treatment of infectious diseases, including COVID-19:
Initiated a Phase 2 study of CNM-ZnAg for patients with COVID-19
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 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. Clene expects to report topline results from the trial in the second half of 2021.
Corporate Highlights:
Intellectual Property:
Clene recently received a Notice of Allowance from the U.S. Patent and Trademark Office (USPTO) for methods of using CNM-Au8 for the treatment
of patients with MS, as well as two additional Notice of Allowances from the USPTO for patent applications covering device and process
claims for its platform technology and advanced stage clean-surfaced nanocrystal therapeutic candidates. The resulting patents will add
to Clene's robust intellectual property portfolio in the field of clean-surfaced nanocrystal therapeutics, which includes over 130
issued and allowed patents and approximately 30 additional applications pending.
Quarter 2021 Financial Results
Clene's cash totaled approximately $48.0 million as of March
31, 2021, compared to approximately $59.3 million as of December 31, 2020. The decrease in cash during the first quarter ended March 31,
2021 was primarily due to approximately $9.2 million of net cash used in operating activities, $0.2 million of net cash used in investing
activities, and $1.9 million of net cash used in financing activities. We expect that our cash as of March 31, 2021 will be sufficient
to fund our operating expenses into mid-2022.
Research and development ("R&D") expenses were approximately
$6.3 million for the first quarter ended March 31, 2021, compared to $3.2 million for the same period in 2020. The year over year increase
is primarily attributable to (i) the progression of our drug candidates through the clinical development process, including increased
enrollment into the REPAIR-PD and the REPAIR-MS studies, and calendar payments due for our participation in the HEALEY-ALS Platform Trial;
and (ii) $1.3 million of share-based expense related to restricted stock unit ("RSU") awards included in R&D expenses.
General and administrative ("G&A") expenses were $5.4
million for the first quarter ended March 31, 2021, compared to $0.8 million for the same period in 2020. The year over year increase
is primarily attributable to (i) increased professional expenses, legal fees, accounting fees, tax fees, and insurance expenses as a result
of becoming a public company on December 30, 2020; and (ii) $1.9 million of share-based expense related to RSU awards included in G&A
Clene's loss from operations was $11.7 million and $4.0 million
for the quarters ended March 31, 2021 and 2020, respectively. Clene's net loss was $39.8 million, or $0.66 per share, for the first
quarter ended March 31, 2021, compared to a net loss of $3.9 million, or $0.23 per share, for the first quarter ended March 31, 2020.
Included in the net loss for the first quarter ended March 31, 2021 is an unrealized loss from the change in fair value of contingent
earn-out liabilities of $28.6 million.
RESCUE-ALS is a Phase 2 multi-center, randomized,
double-blind, parallel-group, placebo-controlled study examining the efficacy, safety, pharmacokinetics and pharmacodynamics of CNM-Au8
in patients with early amyotrophic lateral sclerosis (ALS). The trial completed enrollment in 2H 2020. 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 study is to assess the impact of improving neuronal bioenergetics, reducing reactive oxygen species, and
promoting protein homeostasis with CNM-Au8 on disease progression in patients with early ALS. CNM-Au8 was selected by FightMND of Australia
and Clene was provided a substantial grant to investigate efficacy in ALS utilizing novel electromyography endpoints at two expert sites
in Australia. Topline data are expected in 2H 2021. For more information, please see ClinicalTrials.gov Identifier: NCT04098406.
About the HEALEY ALS Platform Trial
The HEALEY ALS Platform trial is a perpetual multi-center, randomized,