EU agency OKs study of therapy to improve mitochondrial function

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

by Andrea Lobo, PhD | January 16, 2024

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 1/2atrial 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.

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 incrases the amount of energy produced by cells and also induces the production of neuroprotective factors, which help protect cells from neurodegenerative disease processes.

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