BioElectron Technology has reported positive results from a Phase lla clinical trial evaluating the safety, tolerability, and pharmacology of EPI-589 in patients with Amyotrophic Lateral Sclerosis (ALS).
The open-label, single-group assignment trial enrolled 21 ALS patients who were on medication for the disease.
It included a 30-day run-in period to establish baseline biomarker and clinical status.
During the trial, the enrolled patients received EPI-589 for a period of 90 days and were followed for an additional three months after withdrawal of therapy.
BioElectron conducted the trial at three ALS sites in the US, namely California Pacific Medical Center (CPMC) in San Francisco; Cedars-Sinai Medical Center in Los Angeles; and Providence Portland Medical Center in Portland.
The trial met its primary safety and tolerability objective with no drug-related serious adverse events or dose-limiting toxicities.
A statistically significant improvement in cerebrospinal fluid (CSF) and plasma-based biomarkers related to neuroinflammation and ALS disease progression were also observed.
Results of a series of clinical evaluation further showed that EPI-589 was able to slow down disease progression as measured by ALS Functional Rating Scale, as well as improved grip strength and swallowing.
BioElectron CEO Dr Matthew Klein said: “This data demonstrating drug safety, tolerability, and disease biomarker effect provides a strong rationale for the continued development of EPI-589 for ALS.
“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 characterised by neuroinflammation.”
EPI-589 is a new, orally administered small molecule currently under development for adult neurodegenerative diseases.