Full Press Release Details
Clene Reports Second Quarter 2021 Operating
and Financial Highlights
Phase 2 REPAIR program demonstrated statistically
significant improvements in brain energetic metabolism with CNM-Au8 , a gold nanocrystal suspension
Phase 2, placebo-controlled RESCUE-ALS trial
remains on track for top-line data in 2H 2021
Interim blinded RESCUE-ALS efficacy data suggest
CNM-Au8 , a gold nanocrystal suspension, may have neuro-reparative potential in people with amyotrophic lateral sclerosis
Cash of $63 million as of June 30, 2021
SALT LAKE CITY, August 10, 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 the treatment of neurodegenerative disease using nanotechnology to treat energetic failure, today
reported its second quarter 2021 operating and financial results.
"Our recent progress has substantially bolstered our clinical
data set and has us poised to achieve multiple milestones by the end of 2021, the most notable being the expected top-line data release
from the placebo-controlled RESCUE-ALS trial," said Rob Etherington, President and CEO of Clene. "Our Phase 2 REPAIR program
in multiple sclerosis and Parkinson's disease has shown that CNM-Au8 significantly improves energy metabolism in the brains of MS
and PD patients. This is an exciting finding, as it clinically demonstrates CNM-Au8's mechanism of action is broadly applicable
and strongly supports the entirety of our clinical pipeline. We also presented highly encouraging blinded results from RESCUE-ALS last
quarter, further adding to the robust set of clinical and preclinical data demonstrating CNM-Au8's potential to become a breakthrough
for patients with neurodegenerative disease."
Second Quarter 2021 and Recent Highlights
CNM-Au8 for the treatment of multiple sclerosis
(MS) and Parkinson's disease (PD)
Reported positive top-line results from
the Phase 2 REPAIR clinical trials
The objective of the REPAIR clinical trial program was to demonstrate
the effects of Clene's energy-enhancing nanotherapeutic, CNM-Au8, on brain energy metabolites in two sister studies of patients
with Parkinson's disease (REPAIR-PD) and multiple sclerosis (REPAIR-MS). Patients were imaged using 31phosphorous magnetic
resonance spectroscopy, an innovative non-invasive brain imaging technique, before and after 12 weeks of daily oral dosing with CNM-Au8.
The results for the primary endpoint, the mean change in the brain NAD+/NADH ratio (the ratio of the oxidized to reduced form
of nicotinamide adenine dinucleotide), demonstrated a statistically significant increase by an average of 0.589 units (10.4%) following
12-weeks of treatment with CNM-Au8 (p=0.037, paired t-test), in the pre-specified integrated analysis of the REPAIR-PD and REPAIR-MS studies.
Key secondary endpoints, mean change from baseline in the NAD+ fraction and NADH fraction of the total NAD pool, were concordant
with the primary endpoint, demonstrating the NAD+ fraction increased (p=0.026), while the NADH fraction decreased (p=0.026). The
individual results for these sister studies demonstrated consistent statistical trends toward improvement in the NAD+/NADH ratio with
results of p=0.11 and p=0.14, for REPAIR-PD and REPAIR-MS, respectively. Collectively, these results provide clinical proof-of-mechanism
and support the potential of CNM-Au8 to drive meaningful neurological functional improvements in the treatment of neurodegenerative disorders.
These data, together with concordant data on key secondary and exploratory
endpoints, provide clinical proof-of-mechanism and support the potential of CNM-Au8 to drive meaningful functional improvements in the
treatment of neurodegenerative diseases.
CNM-Au8 for the treatment of amyotrophic lateral
Presented interim blinded efficacy data
from the Phase 2 RESCUE-ALS study at the ENCALS 2021 Annual Meeting
RESCUE-ALS is a randomized, placebo-controlled
Phase 2 study evaluating CNM-Au8 in patients with early ALS. The study's primary endpoint utilizes Motor Unit Number Index (MUNIX).
MUNIX is an electrophysiology technique which measures the estimated number of functioning motor neurons serving specific muscles and
has been shown to be a sensitive predictor of clinical decline in ALS. In the overall study population (n = 45; randomized 1:1 active
CNM-Au8 30 mg daily to placebo), 34%, 26%, and 18% of patients who completed weeks 12, 24, and 36, respectively, showed increases (improvements)
in MUNIX(4)sum values (equal to the sum of MUNIX values for the abductor digiti minimi, abductor pollicis brevis, tibialis anterior,
and biceps brachii muscles) from baseline. This differs from the expected continuous decline seen in published data from prior
observational studies1. Additionally, the mean reduction in forced vital capacity (FVC) for the overall study population was
approximately 11% (absolute change of the % predicted) at Week 24 (n=42), which is generally less decline than anticipated based on published
data sets2. Though blinded, these data suggest that CNM-Au8 may have neuro-reparative potential in ALS patients.
Evaluation of CNM-Au8 in indications beyond
ALS, multiple sclerosis, and Parkinson's disease:
Received a Healthy Longevity Catalyst Award
from the U.S. National Academy of Medicine
The award will provide funding to support the
accelerated preclinical development of CNM-Au8 as a treatment for neuronal aging-related deficits and Alzheimer's disease. Preclinical
studies designed to identify key mechanisms by which CNM-Au8 may impact age-related neurogenerative diseases will be led by Dr. Karen
Ho, Head of Translational Medicine at Clene, in collaboration with Assistant Professor Jerome Mertens of the University of Innsbruck (Austria).
Corporate Highlights:
Intellectual Property
In April 2021, Clene received Notices of Allowance from the U.S. Patent
and Trademark Office (USPTO) for two patent applications covering device and process claims for its platform technology and advanced stage
clean-surfaced nanocrystal therapeutic candidates. These have since issued as patents, adding to Clene's robust intellectual property
portfolio that includes more than 130 patents issued and allowed and approximately 30 more applications pending.
In May 2021, Clene appointed David J. Matlin as
a Chairman of the Company's Board of Directors. Mr. Matlin has served as a Director of Clene since 2020 and is currently the Chief
Executive Officer of MatlinPatterson Global Advisers, a global private equity firm he co-founded in 2002. Mr. Matlin is succeeding Shalom
Jacobovitz, who stepped down from his prior role as Chairman but continues to serve on the Company's Board.
In August 2021, Clene appointed Vallerie V. McLaughlin,
MD, to its Board as its seventh independent director. Dr. McLaughlin is the Kim A. Eagle MD Endowed Professor of Cardiovascular Medicine,
Associate Chief Clinical Officer for Cardiovascular Services of the University of Michigan Medical Group, Associate Chief, Division of
Cardiovascular Medicine, and Director of the Pulmonary Hypertension Program at the University of Michigan in Ann Arbor.
Manufacturing Facility
Clene is currently in negotiations to lease an
approximately 75,000 square foot facility in Elkton, Maryland. Subject to the successful consummation of those negotiations, the facility
will be redeveloped to enable an increase in Clene's manufacturing capacity in preparation for the expected data release in H1 2022
from its Phase 3 registration trial evaluating CNM-Au8 as a treatment for ALS.
Financing Agreements
In connection with its planned efforts to expand
its manufacturing capacity, Clene added net proceeds of approximately $24 million to its cash position through a private placement (PIPE)
financing that resulted in gross proceeds of approximately $9.25 million, and a Loan and Security Agreement with Avenue Venture Opportunities
Fund, L.P., a fund of the Avenue Capital Group. The Loan Agreement provides for term loans in an aggregate principal amount up to $30
million, with up to $20 million committed between May 24, 2021, and December 31, 2021, and up to a further $10 million funded between
January 1, 2022, and June 30, 2022. To date, Clene has received $15 million of gross proceeds under the Loan Agreement.
FTSE Russell Indexes
As of the market open on June 28, 2021, Clene was included as a member of the U.S. small-cap Russell 2000
Index and the all-cap Russell 3000 Index. Clene's common stock was also added to the appropriate growth and value
Expert Perspectives Webinar
On July 14, 2021, Clene hosted an expert perspectives webinar entitled:
"Cellular Energetic Failure: Addressing Unmet Needs and a New Investigational Treatment for ALS and MS." The webinar featured
presentations by two experts: Professor of Neurology Matthew Kiernan, PhD, DSc, FRACP,
FAHMS, AM, MBBS and Professor of Neurology Benjamin Greenberg, MD, MHS, FANA, FAAN, CRND, who discussed the current treatment landscape
and unmet medical needs in ALS and multiple sclerosis. A replay of the presentation is available here.
Quarter 2021 and Financial Results
Clene's cash totaled approximately $63.0 million as of June 30,
2021, compared to approximately $59.3 million as of December 31, 2020. The increase in cash through the second quarter ended June 30,