Full Press Release Details
Clene Reports Third Quarter 2021 Operating and
Financial Highlights
SALT LAKE CITY, Nov. 9, 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 reported its third quarter 2021 operating and financial results.
"We believe that the top-line data from RESCUE-ALS, a Phase 2
proof-of-concept clinical trial evaluating CNM-Au8 as a disease modifying treatment for people with early ALS, indicate the potential
of CNM-Au8 to benefit patients living with this debilitating and deadly disease. The results also provide further support in the ability
of our nanotherapeutics to treat energetic failure, an underlying cause of many neurological diseases," said Rob Etherington, President
and CEO of Clene. "Based on these results, we look forward to reporting results from the Phase 2/3 HEALEY ALS Platform Trial, which
we expect in the second half of 2022."
Third Quarter 2021 and Recent Highlights
CNM-Au8 for the treatment of amyotrophic lateral sclerosis (ALS):
Reported top-line results from RESCUE-ALS Phase 2 trial
RESCUE-ALS is a Phase 2 clinical trial evaluating CNM-Au8 as a disease
modifying treatment for people with early amyotrophic lateral sclerosis (ALS). This 36-week randomized, placebo-controlled trial enrolled
45 patients, randomized 1:1 to treatment with CNM-Au8 at 30 mg daily or matching placebo on top of standard of care. As previously announced,
the trial did not meet the primary endpoint, change in MUNIX score at week 36. In this study, ALS patients with early bulbar-onset did
not worsen in MUNIX, which measures lower motor neurons located in the spinal cord. In a pre-specified subset analysis of limb onset patients,
who did progress in MUNIX as expected, CNM-Au8 demonstrated an absolute improvement from baseline at week 12 (p=0.0385) and a 45% relative
improvement at week 36 (p=0.0741). RESCUE-ALS is first study to use MUNIX as a primary endpoint in a phase 2 proof of concept trial in
ALS and these results suggest lower motor neuron protection in limb onset ALS, which accounts for approximately 70% of the ALS population.
Importantly, in this study there were statistically significant improvements in clinically relevant exploratory endpoints through week
36, 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 from CNM-Au8 when comparing the observed
survival of the trial population to the predicted survival, based on the validated ENCALS model. Overall, CNM-Au8 was found to be well-tolerated
through 36 weeks of oral daily dosing.
Launched Second FDA Expanded Access Program for People living with
A second U.S. Food and Drug Administration (FDA) expanded access program
(CNMAu8.EAP02) was launched with CNM-Au8 for people living with ALS. This expanded access program will be implemented in conjunction with
the Healey ALS Platform Trial and is designed to provide people with ALS who are not eligible to enroll in the Healey ALS Platform Trial
CNM-Au8 for the treatment of multiple sclerosis (MS) and Parkinson's
Reported blinded interim update from VISIONARY-MS
VISIONARY-MS is a double-blind, placebo-controlled Phase 2 clinical
trial evaluating the efficacy and safety of CNM-Au8 as a remyelinating and neuro-reparative treatment in stable relapsing MS (RMS) patients.
Blinded analyses compared changes in the overall study population's modified Multiple Sclerosis Functional Composite (mod-MSFC) values
over the 48-week treatment period to the baseline values of study participants with mild disease, as defined by pre-treatment Expanded
Disability Status Scale (EDSS) scores of 1.5 or less. Changes in the four modified-MSFC sub-scales (low contrast letter acuity (LCLA),
symbol digit modalities test (SDMT), 9-hole peg test (9HPT), and timed 25-foot walk test (T25FWT)) were compared to baseline scores of
the mild disease comparator group at each 12-week study time-point (Weeks 12, 24, 36, and 48). At each visit, the overall study population
(randomized 2:1, active CNM-Au8 to placebo) showed increasing improvements in mean standardized change for LCLA (primary endpoint, mixed-effects
model; p<0.0001 vs. baseline), average MSFC scores (secondary endpoint, mixed-effects model; p<0.0001 vs. baseline), and other MSFC
sub-scales. These data support CNM-Au8's potential to drive meaningful neurological improvements in stable RMS patients. Unblinded
topline data are anticipated in the first half of 2023.
Reported positive top-line results from the Phase 2 REPAIR clinical
The objective of the REPAIR clinical trial program was to demonstrate
the effects of Clene's catalytically-active 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 31-phosphorous 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. REPAIR-PD study results were subsequently
presented at the International Parkinson and Movement Disorders Society (MDS) Virtual Congress 2021 meeting in September 2021 and REPAIR-MS
results were subsequently presented at the 37th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS)
Corporate Highlights:
Manufacturing Facility
In August, Clene completed two lease agreements that will more than
quadruple the company's manufacturing capacity. The first agreement is a ten-year lease for approximately 75,000 sq. ft. of manufacturing
space in Elkton, Maryland, which will be redeveloped to support Clene's proprietary electrochemical processes and will materially
increase its 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
The second agreement is for a seven-year lease to further expand its
existing manufacturing capacity at its site in North East, Maryland to a total of approximately 33,000 sq. ft. This expansion enables
additional R&D and manufacturing capacity for CNM-Au8 and further positions Clene to address its potential long-term needs.
Financing Agreements
In September 2021, Clene borrowed an additional $5.0 million under
its 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 $20 million of
gross proceeds under the Loan Agreement.
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.
Anticipated 2021 Milestones:
Third Quarter 2021 and Financial Results
Clene's cash and restricted cash totaled approximately $60.6
million as of September 30, 2021, compared to approximately $59.3 million as of December 31, 2020. The increase in cash and restricted
cash through the third quarter ended September 30, 2021 was primarily due to approximately $25.0 million of net cash used in operating
activities; $0.7 million of net cash used in investing activities; and $27.1 million of net cash provided by financing activities. Included
in net cash provided by financing activities is approximately $9.3 million of net proceeds from a PIPE offering in May 2021 and approximately
$19.5 million of net proceeds to date from a venture loan agreement which we entered into in May 2021. Clene expects that its cash as
of September 30, 2021 will be sufficient to fund its operations for a period extending beyond twelve months from the date the September
30, 2021 condensed consolidated financial statements are issued.
Research and development ("R&D") expenses were approximately
$6.1 million for the third quarter ended September 30, 2021, compared to $4.0 million for the same period in 2020. The year-over-year
increase is primarily attributable to (i) the progression of Clene's drug candidates through the clinical development process, including
increased enrollment into the REPAIR-PD and the REPAIR-MS studies, and calendar payments due for Clene's participation in the HEALEY-ALS
Platform Trial; and (ii) $0.9 million of share-based expense related to stock options and restricted stock awards included in R&D
General and administrative ("G&A") expenses were approximately
$4.4 million for the third quarter ended September 30, 2021, compared to $1.8 million for the same period in 2020. The year-over-year
increase is primarily attributable to (i) increased professional expenses, public company expenses, legal fees, accounting fees, tax fees,