EU agency OKs study of therapy to improve mitochondrial function

Trial of MP101 to enroll patients with MS, other neurodegenerative diseases

Andrea Lobo, PhD avatar

by Andrea Lobo, PhD |

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Mitochon Pharmaceuticals is launching a pilot clinical trial to evaluate MP101, its treatment candidate for improving mitochondrial function, in people with multiple sclerosis (MS) and other neurodegenerative diseases.

The announcement follows the trial’s clearance by the European Medicines Agency (EMA), which enables Mitochon to begin enrolling patients with neurodegenerative conditions in the biomarker study.

The Phase 1/2a trial will enroll people with MS, amyotrophic lateral sclerosis (ALS), Alzheimer’s disease, and Huntington’s disease, each of whom will receive the experimental therapy for 14 days. The goal is to demonstrate whether the therapy is safe in these populations and induces relevant changes in disease-related biomarkers.

“We are delighted for the opportunity to explore [the] provocative idea that most, if not all neurodegenerative diseases are rooted in mitochondrial dysfunction,” John G. Geisler, PhD, Mitochon’s co-founder and chief scientific officer, said in a company press release.

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MP101 designed to work as stimulator for mitochondrial function

Mitochondria are small cellular structures that produce most of the energy cells need to function — including the energy required for nerve cells to transmit electrical signals to other cells. They’re also involved in other essential cell functions.

However, research has shown that these cellular organelles are dysfunctional, or don’t work properly, in most neurodegenerative conditions, including in multiple sclerosis. Thus, impaired mitochondrial function is thought to play an important role in neuronal damage and disease progression.

MP101 is an investigational, brain-penetrant oral therapy that works as a mitochondrial stimulator, improving the function of these organelles across cells in the brain and spinal cord. This increases the amount of energy produced by cells and also induces the production of neuroprotective factors, which help protect cells from neurodegenerative disease processes.

We predict that chronic treatment with this unique platform, at micro-doses, will resolve mitochondrial issues and change important disease specific biomarkers in [MS, ALS, Huntington’s disease and Alzheimer’s disease] similarly.


In preclinical studies, researchers showed that MP101 was able to protect nerve cells from the demyelination that occurs in people with MS. It also reduced disease severity and improved mobility in animal models of the disease. Similar results were obtained with MP201, a similar candidate molecule.

The upcoming trial is expected to confirm the benefits of MP101 in neurodegenerative conditions. If positive, these findings are expected to support Phase 2b studies in people with primary progressive MS and secondary progressive MS, as well as ALS, Huntington’s, and Alzheimer’s.

“We predict that chronic treatment with this unique platform, at micro-doses, will resolve mitochondrial issues and change important disease specific biomarkers in all four indications similarly,” Geisler said.

No timeline for the trial was provided by Mitochon.