- Clene is a late clinical-stage biopharmaceutical company focused on improving mitochondrial health and protecting neuronal function to treat neurodegenerative diseases like ALS
- In 2019, Clene’s drug candidate, CNM-Au8(R), was selected for the HEALEY ALS Platform Trial, with enrollment commencing in the summer of 2020
- Over the years since, Clene has consistently released additional data from the Trial, most recently reporting significant long-term survival improvement among patients treated with CNM-Au8 compared to PRO-ACT historical controls
- The company also announced the publication of a peer-reviewed study in the nanotechnology-focused journal Small; the study describes CNM-Au8’s mechanism of action
- Clene received a $45.1 million NINDS grant to support an Expanded Access Protocol (“EAP”) program and study for CNM-Au8 in ALS
Amyotrophic lateral sclerosis (“ALS”), or Lou Gehrig’s disease, is a progressive and degenerative neuromuscular disease that destroys nerve cells known as motor neurons, which control voluntary muscle movement (https://ibn.fm/bgJGu). A fatal neurological disorder, ALS lowers the life expectancy of patients to two to five years post-diagnosis, although some patients may live longer, as was the case with the late Stephen Hawking, who lived with ALS for more than five decades (https://ibn.fm/v6LIz).
The Centers for Disease Control and Prevention (“CDC”) estimated that more than 31,000 patients were living with ALS in 2017 (https://ibn.fm/Vdee8), with a subsequent study calculating the 2017 prevalence of ALS in the adult U.S. population arriving at an age-adjusted prevalence of between 5.5 and 9.9 per 100,000 U.S. population. The study based its calculations on estimates of adult persons who met the National ALS Registry definition of ALS (https://ibn.fm/YhM3C).
Unfortunately, no cure has yet been identified, although the U.S. Food and Drug Administration (“FDA”) has approved several treatments that slow disease progression in certain patients. Driven by the need to find a cure or treatment for all patients, the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital launched a platform trial for ALS (“the HEALEY ALS Platform Trial” or “the Trial”) in 2019. The Trial sought to evaluate several “promising” candidate treatments from different developers, among them CNM-Au8(R), the lead drug candidate for Clene Nanomedicine, Inc., a wholly owned subsidiary of Clene (NASDAQ: CLNN) (https://ibn.fm/E4bma). The CNM-Au8 arm of the Trial, dubbed Regimen C, initiated patient enrollment in the summer of 2020.
The platform trial was designed to accelerate the development of breakthrough treatments for patients with ALS by testing multiple drugs using a shared placebo group and infrastructure. This, according to Sean M. Healey & AMG Center for ALS, reduced the cost of research by 30%, lowered the trial time by 50%, and increased patient participation by 67% (https://ibn.fm/d2dau).
Since the commencement of the HEALEY ALS Platform Trial, Clene has regularly updated the scientific community and its shareholders by releasing new insights uncovered from the Trial. Most recently, the company announced long-term follow-up data following an analysis in which 59 participants who were originally randomized and treated with CNM-Au8 30mg for up to 133 weeks in the HEALEY ALS Platform Trial were compared to matched placebo participants derived from the largest U.S. clinical database of previous ALS trials, known simply as PRO-ACT (https://ibn.fm/RghZL).
The data revealed that the 59 participants demonstrated a statistically significant (p=0.046) 49% decreased risk of death compared to matched placebo participants drawn from the PRO-ACT dataset. In its analysis, Clene also pooled data from the HEALEY ALS Platform Trial and the RESCUE-ALS Trial, a Phase 2 multicenter, randomized, double-blind, parallel-group, placebo-controlled group, investigating the efficacy, safety, pharmacokinetics, and pharmacodynamics of CNM-Au8 in patients with early ALS. The combined data showed that 82 participants originally randomized to CNM-Au8 demonstrated a statistically significant (p=0.004) 59% decreased risk of death compared to PRO-ACT matched placebo patients. (Improved survival status is an important measure of drug effect.) Moreover, Clene reported no serious adverse events associated with CNM-Au8.
“To show such profound survival improvement using the HEALEY ALS Platform Trial dataset alone and a pooled HEALEY and RESCUE dataset is remarkable, and helps confirm the survival benefit seen in the prespecified secondary endpoint,” commented Benjamin Greenberg, M.D., Head of Medical at Clene. “Clene is extremely gratified to see this consistent long-term survival data from the HEALEY ALS Platform Trial OLE, with a continued clean safety profile, adding to the totality of the survival evidence.”
CNM-Au8 is an orally-dosed, investigational drug that improves mitochondrial function by targeting the NAD+ metabolic pathway, leading to increased neuronal survival and function. The robust neuroprotective properties of the drug candidate can be attributed to its therapeutic catalytic activity, according to a peer-reviewed scientific paper describing CNM-Au8’s mechanism of action (https://ibn.fm/wSgRu).
When neurons were exposed to toxins (excitotoxic stressors or toxic peptides) that induce neuronal death, the study observes, CNM-Au8 treatment promoted the survival of cells and preservation of the neurite network. The study, titled “A Mechanism Underpinning the Bioenergetic Metabolism-Regulating Function of Gold Nanocatalysts,” is published in the nanotechnology-focused journal Small. It is co-authored by lead investigators at the University of South Carolina and Clene, among them Professor Hui Wang, Ph.D., from the Department of Chemistry and Biochemistry at the Columbia, South Carolina-based institution. The publication is available through Open Access at (https://ibn.fm/m5kc0).
“The insights gained from this research provide, for the first time, important guiding principles for the rational design of a new class of gold-based catalytic therapeutic agents with energy metabolism-regulating and neuroprotective function,” said Professor Wang.
Meanwhile, Clene’s efforts to improve mitochondrial health and protect neuronal function to treat neurodegenerative diseases received a significant boost following a recently awarded grant. The National Institute of Neurological Disorder and Stroke (“NINDS”), a division of the National Institutes of Health (“NIH”), awarded a four-year grant totaling $45.1 million to Clene, through its subsidiary in collaboration with Columbia University and Synapticure, to support an Expanded Access Protocol (“EAP”) program and study for CNM-Au8 in ALS. Through the EAP program, the study will enable ALS patients who do not meet the criteria to enroll in a clinical trial to try the drug candidate as a novel investigational therapy (https://ibn.fm/ciWbQ).
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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