BLUE BELL, PA — Mitochon Pharmaceuticals received a $1 million clinical trial award from the ALS Association to help fund a 24-week Phase 2 study of its experimental drug MP-101 in people with sporadic amyotrophic lateral sclerosis, advancing the Blue Bell biotechnology company’s lead mitochondrial therapy toward another clinical test in early 2027.
The Hoffman ALS Clinical Trial Award will support a study designed to build on recent clinical work in Europe, where Mitochon reported declines in neurofilament light, or NfL, among sporadic ALS participants receiving MP-101 capsules.
NfL is a biomarker associated with nerve-cell damage. The company did not provide detailed results from the European studies, including the magnitude of the reported decline, participant numbers or comparative data, in the announcement.
MP-101 is an oral, brain-penetrating small molecule designed to alter mitochondrial activity. Mitochon is developing the drug on the premise that targeting mitochondrial dysfunction, oxidative stress, calcium toxicity and impaired energy production could protect neurons affected by ALS.
The company describes the drug’s mechanism as mitochondrial proton therapy, an approach using mitochondrial uncoupling to alter cellular energy processes.
Mitochon Chief Scientific Officer and co-founder John Geisler said research by University of Florida scientist Lan Wei-LaPierre helped support the company’s focus on calcium toxicity as a potential early contributor to motor-neuron loss in ALS.
“ALS has been top of our priorities for many years,” Geisler said, adding that Wei-LaPierre’s research indicated MP-101’s pharmacology was consistent with that hypothesis.
Chief Executive Officer Robert Alonso said the award will help finance the Phase 2 study planned for early 2027.
The ALS Association uses its Hoffman ALS Clinical Trial Awards to support experimental therapies at the clinical-development stage.
“By funding programs at this critical stage, we are striving to accelerate the development of therapeutic candidates that can help make ALS a livable disease,” ALS Association Chief Scientist Kuldip Dave said.
ALS is a progressive neurodegenerative disease that damages the nerve cells controlling voluntary muscle movement. Mitochon plans to evaluate whether MP-101’s effects on mitochondrial function can translate into a therapeutic benefit for patients with sporadic disease.
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