Mitochondria play a crucial role in the energy production that is essential for the cell survival. Extraordinary endeavors have recently been made to investigate the impact of mitochondrial dysfunctions on the pathogenesis of Alzheimer’s disease (AD), such as oxidative stress by excessive reactive oxygen species (ROS), Ca2+ disturbance, depletion of adenosine triphosphate (ATP), and impaired mitochondrial dynamics.
Beta-amyloid and tau in AD afflict neuronal activities by interrupting the mitochondrial functions (energy supply and antioxidant response), eventually causing the synaptic dysfunction and neuronal death.
Recent research evidences suggest that therapeutic approaches targeting the mitochondrial dysfunction can set back the onset and slow down the progression of AD.
MitoImmune Therapeutics Inc. (hereafter ‘MitoImmune’ for short), headquartered in Seoul, is a biotech company pioneering new mitochondria-targeted therapeutics using its proprietary platform technology for the treatment of mitochondrial dysfunction-related inflammatory and necrotic diseases.
The company developed its innovative anti-inflammatory and anti-necrotic agent, named ‘MIT-001’, using its new Mitochondriotropic Indole-based Technology (MIT) Platform, which can scavenge the excessive ROS in mitochondria and inhibit the inflammation and necrosis.
MIT-001 was developed initially for the prevention of oral mucositis in cancer patients. However, the company has recently begun to expand the potential clinical indications of MT-001 beyond the inflammatory diseases to the field of neurodegenerative diseases like AD.
MitoImmune submitted the research proposal titled ‘The development of mitochondria-targeted candidate therapeutic molecule for dementia that improves the mitochondrial functions and neuroinflammation through the regulation of ROS and Ca2+ with the aim of entering the phase 1 clinical trial’, which was finally accepted by the Korea Dementia Research Center in August 2021.
With this preclinical study, the company will develop the oral formulation of MIT-001 that can cross the blood-brain barrier (BBB) with high efficiency for the treatment of AD.
In parallel with this, MitoImmune’s MIT-001 received the FDA approval for its phase 2 trial of oral mucositis prevention in the patients with head and neck squamous cell carcinoma who are getting concurrent chemoradiotherapy in January 2021.
In February this year, the company successfully raised additional $23 million in a round of Series B which was led by the Korea Development Bank. This new round of funding was added to $10.5 million secured through Series A in April 2019. This Series B funding will expedite its phase 2 clinical trial of MIT-001 for oral mucositis in the U.S.
In the meantime, to get listed on the KOSDAQ (Korea Securities Dealers Automated Quotation), the company is planning to apply for the technology evaluation and to file for its Initial Public Offering (IPO) in 2022.
Related Review Article
Misrani A, Tabassum S, Yang L. Mitochondrial Dysfunction and Oxidative Stress in Alzheimer's Disease. Front Aging Neurosci. 2021 Feb 18;13:617588.