The European Medicines Agency (EMA) has awarded approval to Mitochon Pharmaceuticals to initiate a Phase I/IIa biomarker study to treat patients with several neurogenerative diseases. 

The 14-day pilot study will investigate MP101, a small molecule orally administered drug which targets mitochondrial function. The clinical trial will enrol patients with amyotrophic lateral sclerosis (ALS), multiple sclerosis, Huntington’s disease, and Alzheimer’s disease, and evaluate the safety and potential changes in disease-specific biomarkers of the drug. 

Mitochon received orphan drug designation from the US Food and Drug Administration (FDA) for MP101 to treat Huntington’s disease and ALS, in 2019 and 2020, respectively. 

The candidate generated promising results in preclinical studies, with Mitochon reporting that MP101 indicated favourable pharmacological and biochemical responses. Data showed that MP-101 preserves brain volume, medium spiny neurons, general neurons and limb movement, as well as reducing seizure duration. 

According to GlobalData’s Pharma Intelligence Center, MP101 has been investigated in several clinical studies, including for optic neuritis, in normal healthy volunteers, and in Huntington’s disease patients. 

GlobalData is the parent company of Pharmaceutical Technology. 

In the announcement accompanying the approval, Mitochon’s co-founder and chief scientific officer (CSO) John Geisler said: “We are delighted for the opportunity to explore this provocative idea that most, if not all neurodegenerative diseases are rooted in mitochondrial dysfunction. 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.” 

According to GlobalData’s Pharma Intelligence Center, Mitochon has another drug in its pipeline, MP201. The therapy modulates mitochondrial function, protecting the neurons through mitochondrial uncoupling, which prevents excessive ROS formation and oxidative damage. The candidate is also under development for the treatment of Parkinson’s disease, amyotrophic lateral sclerosis, and traumatic brain injury. 

In July 2023, InSilicoTrials and Axoltis teamed up to develop disease-modifying drugs for neurological disorders. Precision Biosciences filed a patent in September 2023 on a mitochondria-targeting engineered meganuclease for modifying human mitochondrial DNA.