ALS drug trial shows promising signs for Philly-area startup’s once-daily pill
February 24, 2026
Mitochon is betting that stabilizing mitochondria can slow progression in the incurable neurodegenerative disease.
For patients with ALS, even a small gain can be enormous. In an early-stage trial, a new once-a-day drug has shown promising signs.
Blue Bell-based Mitochon Pharmaceuticals completed the clinical study in Europe, involving 18 patients over two weeks, with the goal of testing whether its treatment can help slow the disease by stabilizing nerve cells. During that time, one person was able to button their shirt for the first time in months.
About Mitochon Pharmaceuticals
Mitochon was founded in 2014 with the mission to develop treatments for insidious diseases through the modulation of mitochondrial physiology, with applications to neurodegeneration, neuromuscular, and developmental diseases. Mitochon’s lead programs, MP101 and MP201, specifically harnesses the power of the mitochondria to provide broad neural protection. These compounds elicit mild increases in energy expenditure that result in strengthening cellular survival—similar to the positive effects seen with fasting and exercise. These compounds also induce an important neurotrophin, Brain Derived Neurotrophic Factor (BDNF), involved in cognition and neural growth. Mitochon is supported by Ben Franklin Technology Partners Southeastern PA, an initiative of the Pennsylvania Department of Community and Economic Development funded by the Ben Franklin Technology Development Authority.