Mitochon Pharmaceuticals, Inc.


Mitochon Pharmaceuticals is a clinical-stage biotech company focused on developing drugs that target mitochondrial dysfunction to treat serious neurodegenerative and neuromuscular diseases. Their mission is to create meaningful therapies that modulate mitochondrial physiology to slow disease progression and improve patient outcomes. Mitochon aims to open the door for a broad range of disease-modifying therapies by correcting mitochondrial dysfunctions linked to various diseases.

Industries

biopharma
biotechnology
pharmaceutical

Nr. of Employees

small (1-50)

Mitochon Pharmaceuticals, Inc.

970 Cross Lane, Blue Bell, PA 19422


Patents

Induced expression of brain derived neurotrophic factor (BDNF) for treatment of neuromuscular, neurodegenerative, autoimmune, developmental and/or metabolic diseases

US-11717497-B2

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DNP and DNP prodrug treatment of neuromuscular, neurodegenerative, autoimmune, developmental, traumatic brain injury, concussion, dry eye disease, hearing loss and/or metabolic diseases

US-11406642-B2

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Induced expression of brain derived neurotrophic factor (BDNF) for treatment of neuromuscular, neurodegenerative, autoimmune, developmental and/or metabolic diseases

US-10864173-B2

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DNP and DNP prodrug treatment of neuromuscular, neurodegenerative, autoimmune, developmental, traumatic brain injury, concussion, dry eye disease, hearing loss and/or metabolic diseases

US-10548900-B2

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Induced expression of brain derived neurotrophic factor (BDNF) for treatment of Neuromuscular, neurodegenerative, autoimmune, developmental and/or metabolic diseases

US-10220006-B2

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Induced expression of brain derived neurotrophic factor (BDNF) for treatment of neuromuscular, neurodegenerative, autoimmune, developmental and/or metabolic diseases

US-9974755-B2

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Products

Orally administered brain-penetrant mitochondrial uncoupler (small molecule)

A low-dose, orally delivered small-molecule mitochondrial uncoupler designed to provide neuroprotection in preclinical models through mild increases in mitochondrial energy expenditure.

Slow-release CNS-penetrant prodrug formulation

A prodrug formulation intended to provide sustained release of a mitochondrial-modulating agent to support tissue protection and cognitive/functional improvement in traumatic brain injury and other CNS models.

Peer-reviewed preclinical study demonstrating micro-dose mitochondrial uncoupler efficacy in ALS model

A published preclinical study reporting that micro-dose mitochondrial uncoupler treatment delayed disease onset, preserved motor coordination and muscle function, preserved neuromuscular junction morphology, reduced inflammation and protein oxidation, and produced a period of functional recovery in an ALS mouse model.


Services

Collaborative programs with academic, government and military institutions to advance preclinical efficacy, biomarker and translational studies, funded through grants and cooperative agreements.

Planning and preparatory services for early-phase biomarker-driven clinical studies including protocol design, biomarker selection and regulatory engagement to enable Phase I/IIa initiation.

Expertise Areas

  • Mitochondrial-targeted small-molecule therapeutics
  • Translational neurodegenerative disease research
  • Preclinical efficacy testing in multiple animal disease models
  • Behavioral and histological functional assessment in vivo
  • Show More (6)

Key Technologies

  • Mild mitochondrial uncoupling pharmacology (chemical uncouplers/protonophores)
  • Prodrug and controlled-release medicinal chemistry for CNS delivery
  • Preclinical rodent disease models (ALS SOD1, Huntington N171-82Q, EAE, cuprizone, 6-OHDA, cortical impact, blast TBI)
  • Behavioral testing platforms (rotarod, balance/tapered beam, longitudinal motor assays)
  • Show More (4)

News & Updates

Research published on November 18, 2024, demonstrating the efficacy of micro-doses of DNP in ALS models.

Announcement on January 9, 2024, regarding the initiation of a biomarker study for neurodegenerative diseases.

An article discussing the potential of Mitochon’s drugs in treating traumatic brain injuries, published on May 6, 2024.

An article published on April 4, 2023, discussing Mitochon’s approach to neurodegeneration and aging.

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