MitoChem Therapeutics

MitoChem Therapeutics, Inc. operates as a preclinical-stage biopharmaceutical entity focused on developing therapies for diseases linked to mitochondrial dysfunction. Founded in 2010 by Dr. Craig Beeson, a medicinal chemist, and Dr. Bärbel Rohrer, a neuroscientist, the company leverages the founders' combined expertise in mitochondrial science and drug discovery from their work at the Medical University of South Carolina. Their research targets the development of treatments for a range of neurodegenerative and metabolic diseases by protecting mitochondrial homeostasis.

The company's primary market is ophthalmic diseases, specifically those causing vision loss, such as retinitis pigmentosa (RP) and dry age-related macular degeneration (dry AMD). MitoChem's core technology revolves around a proprietary library of small-molecule compounds designed to protect mitochondria. Its lead candidate, MC16, is a first-in-class small molecule formulated as a pharmaceutical eyedrop that works by upregulating pro-survival and anti-oxidant pathways. Preclinical data in animal models have demonstrated that MC16 can preserve photoreceptors, improve cone function, and restore mitochondrial energy capacity in the retina under stress. In 2021, the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to MC16 for the treatment of RP.

MitoChem’s business model centers on advancing its compounds through preclinical stages and forming strategic partnerships for further clinical development and commercialization. The company actively seeks collaborations to explore non-ophthalmic indications for its technology, including Parkinson's, Alzheimer's, ALS, muscular dystrophies, and kidney diseases. The company has secured funding through various channels, including grants from the Foundation Fighting Blindness and the National Institutes of Health (NIH). Notable funding includes a $25,000 academic startup grant in 2019 and a subsequent $200,000 investment from SC Launch. In August 2022, MitoChem secured a $1.5 million seed investment from Ichor Life Sciences, a preclinical contract research organization, to advance its ocular disease program. This partnership also involved establishing a satellite lab at Clarkson University's Peyton Hall Biotechnology Incubator to support collaborative research.

Keywords: mitochondrial dysfunction, neurodegenerative diseases, ophthalmology, retinitis pigmentosa, age-related macular degeneration, dry AMD, preclinical therapeutics, drug discovery, vision loss treatment, MC16 compound, Orphan Drug Designation, biopharmaceutical, small molecule drugs, mitochondrial homeostasis, antioxidant pathways, photoreceptor preservation, ocular disease, SC Launch, Ichor Life Sciences, Foundation Fighting Blindness