Clene, Inc. released data that shows its add-on treatment, CNM-Au8, significantly improved survival in patients with amyotrophic lateral sclerosis (ALS) by 70%.
Clene President and CEO Rob Etherington/Courtesy Clene Inc.
Shares of Clene, Inc. surged Thursday after the company released “golden” data from an interim analysis of a Phase II study showing that its add-on treatment, CNM-Au8, improved survival in patients with amyotrophic lateral sclerosis (ALS) by 70%.
The interim survival data from Utah-based Clene’s RESCUE-ALS study showed that patients who received CNM-Au8 saw a significant survival benefit compared to patients who were given a placebo. Only five patients who were on the CNM-Au8 arm died, compared to 14 patients on placebo, the company said. CNM-Au8 treatment was well-tolerated, and there were no significant safety findings reported during this open-label trial. The study included 45 patients with ALS.
Clene noted that the survival data obtained in the RESCUE-ALS study was from 43 of the 45 patients. Using the Kaplan-Meier scale to analyze survival, Clene said the data showed those patients that received CNM-Au8 as part of their treatment regimen saw a 70% decreased risk of death. Median survival from randomization for the CNM-Au8 group was undefined due to insufficient mortality events, and median survival for the placebo group was 23.1 months, the company said.
Additional data from the analysis will be reported at an upcoming scientific meeting.
Rob Etherington, president and CEO of Clene, expressed his excitement at the data from the trial. He said it supports the company’s belief that CNM-Au8 provides an increased survival benefit for patients with ALS, a fatal neurodegenerative disease that affects neurons in the brain and the spinal cord.
Awaiting Critical Data from HEALEY Trial
With data from RESCUE-ALS in hand, Clene is now awaiting data from a larger cohort in the HEALEY ALS Platform trial, which is expected in the third quarter. In a previous interview with BioSpace, Etherington called the HEALEY study, which is assessing multiple ALS treatments at one time, “critical” for determining the efficacy of CNM-Au8.
When that data is available, he said it will provide a definitive understanding of whether or not CNM-Au8 can make a meaningful impact on ALS. The company has noted that if data from the registrational HEALEY-ALS study hits the mark, it plans to file a New Drug Application for CNM-Au8 in the first half of 2023.
“At this point, we are awaiting top-line data from the HEALEY-ALS Platform Trial, which focuses on endpoints measuring patient function, survival and breathing over a six-month period in a much larger cohort,” Etherington said in a statement released Thursday morning. “Based on the larger number of patients treated in the Healey trial and the higher dose of CNM-Au8 being tested, we are optimistic that we will be able to adequately characterize the effects of our drug on this devastating disease.”
CNM-Au8 is an investigational gold bioenergetic nanocatalyst that can be used as an add-on to care for multiple neurodegenerative diseases, including ALS, Parkinson’s and multiple sclerosis. The investigational asset is designed to catalyze bio cellular reactions to enable neuroprotection and remyelination by increasing neuronal and glial resilience to disease-relevant stressors.
Will Clene Follow Amylyx to the FDA?
Clene’s data comes at a time when another ALS drug is awaiting a regulatory decision. Amylyx Pharmaceuticals is anticipating the FDA to examine its experimental ALS drug, AMX0035. First, though, the agency’s Peripheral and Central Nervous System Drugs Advisory Committee plans to re-review the data after it initially voted 6 to 4 against the drug’s efficacy based on data from a single Phase II study.
The advisory committee will reconvene September 7, which will give both the adcomm, as well as the full FDA itself, a chance to review the information that includes a post hoc analysis of data announced in May that showed significant survival benefits for ALS patients who received the experimental drug. With the additional data, the FDA delayed its decision and extended the PDUFA date.
