- Recently announced top-line results from RESCUE-ALS Phase 2 trial with CNM-Au8®, a gold nanocrystal suspension
- VISIONARY-MS blinded interim data show clinically relevant improvements in the modified MS Functional Composite for the study population through 48 weeks of treatment
- Phase 2 REPAIR program established central nervous system target engagement in people with MS and PD
- Results for Healey ALS Platform Trial expected in the second half of 2022
- Cash and restricted cash of $60.6 million as of September 30, 2021
SALT LAKE CITY, Nov. 09, 2021 (GLOBE NEWSWIRE) -- 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 ALS
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 access to CNM-Au8.
CNM-Au8 for the treatment of multiple sclerosis (MS) and Parkinson’s disease (PD)
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