According to a story from BioSpace, the biotechnology company BioElectron recently announced that the results from its Phase 2a clinical trial of its experimental product EPI-589 are looking positive. EPI-589 is in development for the treatment of the rare disease amyotrophic lateral sclerosis. The data suggests that EPI-589 could be a useful therapy for this disease.
About Amyotrophic Lateral Sclerosis
Amyotrophic lateral sclerosis, otherwise known as Lou Gehrig’s disease, is a rare, degenerative disease that causes the death of nerve cells associated with the voluntary muscles. Little is known about the origins of amyotrophic lateral sclerosis, with no definitive cause in about 95 percent of cases. The remaining five percent appear to inherit the disease from their parents. Symptoms initially include loss of coordination, muscle weakness and atrophy, muscle stiffness and cramping, and trouble speaking, breathing, or swallowing. These symptoms worsen steadily over time; most patients die because of respiratory complications. Treatment is mostly symptomatic and the medication riluzole can prolong life. Life expectancy after diagnosis ranges from two to four years, but some patients can survive for substantially longer. To learn more about amyotrophic lateral sclerosis, click here.
About The Trial
This Phase 2a trial was intended to assess the therapy’s safety, effectiveness, and tolerability in a group of patients that were already taking medications for their disease. The trial utilized previously identified biomarkers in order to assess the activity of the drugs in these patients. Thankfully, EPI-589 displayed a good safety and toleration profile, with no major adverse events or dose limiting toxic reactions reported in the trial. Biomarker response was also encouraging, with improvements to biomarkers that are linked to neuroinflammation and the progression of amyotrophic lateral sclerosis.
A New Hope
The drug also showed improvements in grip strength and swallowing ability for patients. The study involved 21 patients, who were treated for a period of 90 days and then monitored for three months afterward. Overall, the results of this trial should be a source of excitement for amyotrophic lateral sclerosis patients. There is an urgent need for better therapies for this illness, which is always lethal and has a serious lack of treatments that can effectively slow or halt disease progression.
