BioElectron Technology Corporation (BioElectron), a clinical-stage platform biotechnology company, today announced positive results from its recently completed phase 2a clinical trial of EPI-589 in patients with Amyotrophic Lateral Sclerosis (ALS).
The phase 2a trial (NCT02460679) was designed to assess EPI-589's safety, tolerability, and pharmacology in a cohort of ALS patients already receiving medications for ALS. In addition, the trial sought to identify signals of central nervous system and blood-based biomarkers consistent with ALS, drug target, and mechanism of action.
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The study achieved its primary safety and tolerability endpoint without drug-related serious adverse events or dose-limiting toxicities. Drug treatment was also associated with a statistically significant improvement in cerebrospinal fluid (CSF) and plasma-based biomarkers known to be associated with neuroinflammation and ALS disease progression. A series of clinical assessments further suggested the drug treatment was associated with a slowing of disease progression on the validated ALS Functional Rating Scale, in addition to associated improvements in grip strength and swallowing.
"These data demonstrating drug safety, tolerability, and disease biomarker effect all provide a strong rationale for the continued development of EPI-589 for ALS," said BioElectron CEO Dr. Matthew Klein. "Furthermore, the biochemical evidence of EPI-589's effect on biomarkers of neuroinflammation and disease progression support the development of EPI-589 for other neurological disorders characterized by neuroinflammation."
The clinical trial was conducted at three leading ALS sites in the United States: California Pacific Medical Center (CPMC) in San Francisco; Cedars-Sinai Medical Center in Los Angeles; and Providence Portland Medical Center in Portland. A total of 21 subjects were enrolled in the trial for a 30-day run-in period to establish baseline biomarker and clinical status. The subjects were treated for 90 days with EPI-589 and followed for an additional three months after withdrawal of therapy.
"The results of this trial are very encouraging for the ALS community," said Robert Miller, MD, study principal investigator and director of the Forbes Norris ALS/MDA Center at CPMC. "A safe and effective orally administered medication would be a significant addition to the treatment of ALS patients."
EPI-589 is BioElectron's second compound in active clinical development. EPI-589 is a novel, orally administered, small molecule being developed for adult neurodegenerative diseases in collaboration with Sumitomo Dainippon Pharma, who holds licensing rights for the compound in Japan and North America. EPI-589 targets oxidoreductase enzymes known to be critical to the regulation of inflammation and programmed cell death. Accordingly, it is being developed for CNS diseases characterized by inflammation. BioElectron's lead clinical compound, EPI-743, is in late-stage clinical development for the treatment of mitochondrial disease.