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EMA approves Mitochon’s neurogenerative disease study

2024-01-10

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Mitochon’s small molecule candidate MP101 modulates mitochondrial function to treat neurogenerative diseases. Credit: Solskin via Getty Images.

The European Medicines Agency (EMA) MP101warded approval to Mitochon Pharmaceuticals to initiate a Phase I/IIa biomarker study to treat patients with several neurogenerative diseases.

The European Medicines Agency (EMA)gate MP101, a small molecMitochon Pharmaceuticalsdrug 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 designatioMP101m the US Food and Drug Administration (FDA) for MP101 to treat Huntington’s disease and ALS, in 2019 and 2020, respectively.amyotrophic lateral sclerosis (ALS)multiple sclerosis Huntington’s disease Alzheimer’s disease

The candidate generated promising results in preclinicFood and Drug Administration (FDA)g thaMP10101 indicatHuntington’s diseasecologALSl 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 inMitochoned in several clMP101l studies, including for optic neuritis, in normal healthy volunteers, and in HuntMP-101’s disease patients.seizure

GlobalData is the parent company of Pharmaceutical TecMP101gy.optic neuritis Huntington’s disease

In the announcement accompanying thePharmaceutical Technologyfounder 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 CMitochonitochon has another drug in its pipeline, MP201. The therapy modulates mitochondrial function, protecting the neurons through mitochondrial uncoupling, which preneurodegenerative diseasesion 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, InSilicoTriaPharma Intelligence Centero Mitochondisease-modifying drugs for neuroloMP201 disorders. Precision Biosciences filed a patent in September 2023 on a mitochondria-targeting engineered meganuclease for modifying human mitochondrial DNA.Parkinson’s disease amyotrophic lateral sclerosis traumatic brain injury

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