- An innovative combination of insights from physics, chemistry, materials science, and biology positions the new nanomedicine CNM-Au8(R) to address neurodegenerative disease, such as ALS, from a new angle
- The FDA has offered guidance to Clene on CNM-Au8’s application, with discussions ongoing for the possibility of accelerated approval on the basis of improved survival, biomarker, and clinical efficacy data in people living with ALS
- With plans to move forward on many fronts for the clinical development of CNM-Au8, 2024 looks bright for Clene
Clene (NASDAQ: CLNN) is a late clinical-stage biopharmaceutical company focused on improving mitochondrial health and protecting neuronal function to treat neurodegenerative diseases, including amyotrophic lateral sclerosis (“ALS”), Parkinson’s disease, and multiple sclerosis (“MS”).
The company’s lead drug candidate is CNM-Au8, an innovative nanomedicine uniting the principles of physics, chemistry, materials science, and biology to address neurodegenerative diseases from a new angle. Clene uses catalytic boosting of energy metabolism to enable dying neurons to resist the multiple stressors associated with disease, allowing them not only to survive but also to function.
ALS is among the most aggressive neurodegenerative diseases, in which the progressive loss of motor neurons can lead to death between 2-5 years of diagnosis. Although there are now three FDA approved drugs for the treatment of ALS, there is still great need for a drug that can slow disease progression and significantly impact survival.
Benjamin Greenberg, M.D., Clene’s Head of Medical, said that, as the company continues to analyze data from its RESCUE-ALS and Healey-ALS Regimen C phase 2 clinical programs, the evidence supports that CNM-Au8(R) treatment improved survival in people living with ALS. “We are also encouraged that the recently disclosed long-term NfL biomarker decreases are consistent with delayed clinical time-to-event outcomes,” Greenberg added.
As Clene advances, it also announced long-term results from its Phase 2 VISIONARY trial in MS, demonstrating long-term benefits on visual acuity and cognition. Lead clinician and MS clinician-scientist Michael Barnett of Sydney noted the results as “unprecedented.”
The CNM-Au8 development pathway for ALS has included:
- REPAIR-MS and REPAIR PD, two Phase 2 brain imaging studies that featured oral administration of CNM-Au8 for 12 weeks
- Improvements in brain energy metabolites in participants with MS and PD
- Evidence of target engagement, using an oral therapy to change brain energy potential
RESCUE-ALS, a nine-month, Phase 2 study:
- Randomized and placebo-controlled
- Investigated CNM-Au8 for efficacy and safety in early-stage ALS participants
- Primary endpoint: electrophysiological measure for motor unit preservation
- An unanticipated discovery during the trial: stable motor unit function in bulbar-onset ALS patients early in their disease with no detectable motor unit compromise in arms and legs, resulting in underpowering of the trial for primary endpoint
- First evidence of survival benefit and slowing of clinical worsening with CNM-Au8 treatment
HEALEY-ALS Platform trial, a six-month, Phase 2 study
- CNM-Au8 selected alongside three other ALS drugs, comprising the first four regimens of the Platform trial
- Six-month Phase 2 study; Randomized and placebo-controlled
- Investigated CNM-Au8 for efficacy and safety in participants with ALS
- Primary endpoint: change in ALSFRS-R (an instrument for evaluating the functional status of patients with Amyotrophic Lateral Sclerosis, and used to monitor functional change in a patient over time)
- No drug regimen in the Healey Trial has met primary endpoint, to date
- CNM-Au8 met the secondary endpoint of survival
- Ongoing long-term analyses of CNM-Au8 show impressive survival benefit and clinical improvement
- Significant reductions in levels of key biomarker Neurofilament light (“NfL”) with CNM-Au8 treatment during both the six-month period and one-year long-term open label period; CNM-Au8 is the first ALS drug investigated in a heterogenous ALS population to reach significance on this important ALS biomarker
The company is continuing to evaluate emerging long-term data from the RESCUE and HEALEY long term extension and their expanded access protocols (“EAP”) in ALS. Clene has announced plans for its global Phase 3 trial in ALS and is opening a new NIH-funded EAP for participants with ALS who do not qualify to participate in clinical trials in 2024.
In terms of safety, the company has accrued data on over 500 years of human subject exposure to CNM-Au8 from its clinical programs and compassionate use early access programs (FDA-approved EAPs) to date, with no serious safety signals being associated with drug treatment.
During 2024, Clene will work with the U.S. FDA to consider the strength of Clene’s data, while exploring all potential pathways to commercialization, including the possibility of accelerated approval based upon a biomarker and/or survival signal. With the supportive efficacy data and additional biomarker data in the pipeline, the company believes that the development path for CNM-Au8 looks bright in 2024.
For more information, visit the company’s website at www.Clene.com.
NOTE TO INVESTORS: The latest news and updates relating to CLNN are available in the company’s newsroom at https://ibn.fm/CLNN
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