Full Press Release Details
Clene Nanomedicine Announces Top-Line Results
Phase 2 RESCUE-ALS Clinical Trial
SALT LAKE CITY, Nov. 2, 2021 -- Clene Inc. (Nasdaq: CLNN) along with
its subsidiaries "Clene" and its wholly owned subsidiary Clene Nanomedicine Inc., a clinical-stage biopharmaceutical company
focused on revolutionizing the treatment of neurodegenerative disease with its potential first-in-class catalytically active nanocrystal
suspension, today announced top-line data from RESCUE-ALS, a Phase 2 clinical trial evaluating CNM-Au8 as a disease modifying treatment
for people with early amyotrophic lateral sclerosis (ALS).
The trial did not meet the primary or secondary endpoints - Motor
Unit Number Index (MUNIX) and forced vital capacity (FVC) - at week 36. However, an efficacy signal was observed for the MUNIX endpoint
at week 12 (p=0.057). Furthermore, in a pre-specified analysis in the subset of limb onset ALS, CNM-Au8 demonstrated a significant treatment
effect in MUNIX at week 12 (p=0.0385) and a trend for improvement at week 36 (p=0.0741). Limb onset ALS accounts for approximately 70%
of the ALS population. MUNIX is a neurophysiological biomarker that estimates the number of functioning lower motor neurons serving selected
Clinically relevant exploratory endpoints through trial week 36 demonstrated
significant benefits with CNM-Au8 treatment, including slowing ALS disease progression (p=0.0125), decreasing the proportion of participants
with an ALS Functional Rating Scale Revised (ALSFRS-R) 6-point decline (p=0.035), and improving quality of life as measured by the ALS
Specific Quality of Life (ALSSQOL-SF) (p=0.018). In addition, RESCUE-ALS showed evidence for a potential long-term survival benefit when
comparing the survival of the trial population to the validated ENCALS predictive model1.
"These data are very encouraging to us in the ALS research and
treatment community as they demonstrate clinical benefits with CNM-Au8 treatment in outcomes that matter to patients and provide evidence
for improved long-term survival," said Professor Matthew Kiernan, AM MBBS(Hons), PhD, DSc, FRACP, FAHMS, Bushell Chair of Neurology,
University of Sydney and one of the trial's clinical advisors. "RESCUE-ALS was a proof-of-concept trial intended to establish
that treatment of neuronal energetic failure can provide disease-modifying effects in ALS. I am pleased to see the potential effectiveness
of CNM-Au8 demonstrated in this trial, and it is important to confirm these results in a larger clinical trial."
Rob Etherington, Clene's Chief Executive Officer, stated, "We
believe these results show the potential of CNM-Au8 to bring meaningful benefit to people living with ALS. Befitting of Lou Gehrig, whose
legacy is intertwined with the disease, we swung for the fences and ended with a stand-up triple. In the second half of next year, we
expect to report results from the HEALEY ALS Platform Trial with the objective of confirming CNM-Au8 as an effective disease-modifying
therapy for people with ALS."
RESCUE-ALS was a 36-week randomized, placebo-controlled Phase 2 clinical
trial that enrolled 45 patients with early ALS, randomized 1:1 to treatment with CNM-Au8 at 30 mg daily or matching placebo on top of
standard of care. The primary endpoint of the trial was the percent change of the sum of MUNIX from baseline to week 36. Secondary endpoints
were the change in FVC and the absolute change in MUNIX values to week 36. Exploratory endpoints included multiple clinically relevant
measures of ALS: disease progression, ALSFRS-R decline, and ALSSQOL-SF.
CNM-Au8 was found to be well-tolerated through 36 weeks of oral daily
dosing. There were no reported serious adverse events (SAEs) related to CNM-Au8 treatment. Treatment-emergent adverse events were predominantly
mild-to-moderate in severity. The most frequently reported adverse events associated with CNM-Au8 treatment included aspiration pneumonia
(n=3) and transient gastrointestinal distress (n=2). Topline results from RESCUE-ALS are expected to be presented at the upcoming International
Symposium on ALS/MND, which will take place Dec. 8-10, 2021.
Robert Glanzman, MD, FAAN, Clene's Chief Medical Officer, concluded,
"The results of RESCUE-ALS add to our expanding body of evidence that cellular energetic failure is an important pathophysiological
mechanism in ALS. We thank the trial participants and their families for their willingness to engage in clinical research, the site investigators
for their research excellence and dedication to patients, and FightMND of Australia for substantially funding the trial."
Bec Sheean, Research Director of FightMND commented, "FightMND
is committed to finding new treatment options for motor neurone disease. We awarded Clene a grant in 2019 to support the conduct of this
Phase 2 clinical trial with CNM-Au8 in Australia. These clinical trial results are encouraging for people with ALS. By achieving clinically
relevant endpoints, this trial demonstrates the potential for CNM-Au8 to support neuronal health. We are excited to see CNM-Au8 continue
its advancement into the clinic so that we can bring this potentially transformative treatment to patients."
Conference Call and Webcast Details
Clene will host a conference call and
live audio webcast today at 8:00 a.m. ET to discuss the Phase 2 RESCUE-ALS topline clinical trial results. The live webcast
may be accessed from the Investors section of the Company's website at www.clene.com. Please connect to the website prior to the
start of the conference call to ensure adequate time for any software downloads that may be necessary. Individuals may participate in
the conference call by dialing +1 800 309 3488 in the U.S., or +1 929 517 9012 outside the U.S., and using conference
An archived version of the webcast will be
available for at least one week in the Investors section of the Company's website at www.clene.com.
About CNM-Au8 , a gold nanocrystal suspension
Clene's lead drug candidate, CNM-Au8, a catalytically active
gold nanotherapeutic, is the result of a patented manufacturing breakthrough. The catalytically active nanocrystals of CNM-Au8 drive critical
cellular energy producing reactions in the brain that enable neuroprotection and remyelination by increasing neuronal and glial resilience
to disease-relevant stressors. CNM-Au8 crosses the blood-brain barrier and is not associated with the toxicities related to synthetic
gold compounds or nanoparticles manufactured via alternative methods. CNM-Au8 is being evaluated in a Phase 3 registration trial for the
treatment of amyotrophic lateral sclerosis (ALS). In the REPAIR Program Phase 2 open-label biomarker clinical trials, CNM-Au8 demonstrated
target engagement in the treatment of Parkinson's disease (PD) and multiple sclerosis (MS). REPAIR-PD has concluded, and REPAIR-MS
will continue with the initiation of a second MS dosing cohort. 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 numerous disease-relevant neurotoxins. Oral treatment with CNM-Au8 improved functional behaviors in rodent models of ALS, MS
and PD versus vehicle (placebo). CNM-Au8 is a federally registered trademark of Clene Nanomedicine, Inc.
RESCUE-ALS is a Phase 2 multi-center, randomized, double-blind, parallel-group,
placebo-controlled trial examining the efficacy, safety, pharmacokinetics and pharmacodynamics of CNM-Au8 in patients with early amyotrophic
lateral sclerosis (ALS). The trial completed enrollment in the second half of 2020. In the trial, 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 trial is to assess the impact of CNM-Au8 on disease progression in patients with early-stage ALS through changes
in motor unit index MUNIX. MUNIX values were evaluated for four muscles in the hand, arm, and leg: the abductor digiti minimi, abductor
pollicis brevis, tibialis anterior and biceps brachii from baseline through 36 weeks of treatment. CNM-Au8 was selected by FightMND of
Australia, and Clene was provided a substantial grant to investigate efficacy in ALS utilizing novel neurophysiological endpoints at two
clinical sites in Australia. For more information, please see ClinicalTrials.gov Identifier: NCT04098406.
FightMND is a not-for-profit registered charity, founded in 2014. It
was established to raise the awareness of Motor Neurone Disease (MND) in Australia, to increase funding for research to find an effective
treatment and cure and to provide care equipment for MND patients. FightMND has a clear objective - to have a world free from
FightMND is Australia's largest independent MND foundation focused
on funding large-scale, collaborative research and clinical trials. The generous donations contributed by everyday Australians, right
across the country, has enabled FightMND to raise and commit millions to cure and care initiatives.
Clene is a clinical-stage biopharmaceutical
company focused on revolutionizing the treatment of neurodegenerative disease with potential first-in-class nanotherapeutics to treat
energetic failure, an underlying cause of many neurological diseases. Our lead drug candidate, CNM-Au8, is an oral suspension of
gold nanocrystals that drive critical cellular energetic metabolism in the central nervous system (CNS). CNM-Au8 increases energy production
and utilization to accelerate neurorepair and improve neuroprotection. CNM-Au8 is currently being evaluated in a Phase 3 registration
trial in amyotrophic lateral sclerosis (ALS) and a Phase 2 trial for the treatment of chronic optic neuropathy in patients with stable
relapsing multiple sclerosis (MS). 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
Forward-Looking Statements
This press release contains "forward-looking statements"
which are intended to be covered by 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