According to a story from Market Screener, the clinical stage biopharmaceutical company Kringle Pharma, Inc., has recently announced an update on the progress of its phase 2 clinical trial. The study is testing KP-100IT, its recombinant HGF, as a treatment for amyotrophic lateral sclerosis (ALS), a rare disease. A total of 46 patients are involved in the trial, all of whom are being treated within 30 months of diagnosis.
About Amyotrophic Lateral Sclerosis (ALS)
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 the disease, 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.
Trial Update
32 patients were treated with KP-100IT and rest received a placebo. The treatment failed to achieve either the primary endpoint, which was changes to the ALS Functional Rating Scale-Revised (ALSFRS-R) score, or secondary endpoints. Nevertheless, some patients that received the therapy saw slowed disease progression. Further analysis of these results will be necessary.
Safety findings were similar to the placebo group. The company plans to communicate with Tohoku University to decide on next steps in the evaluation of KP-100IT.
About the ALS Functional Rating Scale-Revised (ALSFRS-R)
This measurement tool evaluates 12 different aspects of daily physical functioning to determine a amyotrophic lateral sclerosis patient’s degree of ability and disease progression.
