Mitochon Pharmaceuticals announced the completion of a second year of financing, raising a total $1.6 million to advance its lead compound, MP101, a mitochondrial targeted neuro-protective agent, into clinical testing. The company is developing mitochondrial modulators for patients with neurodegenerative diseases ranging from Alzheimer’s and Parkinson’s to multiple sclerosis (MS).
A number of studies have shown an association between mitochondrial dysfunction and neurodegenerative diseases. Mitochondria generate the energy responsible for the maintenance of nerve cells and transmission of nerve signals, and defects in mitochondrial DNA — or changes in mitochondria function or structure — lead to nerve cell damage. Improving mitochondrial function would decrease the severity of a variety of symptoms in diseases characterized by nerve damage.
“We are pleased to close on this financing and continue our efforts in developing neuro-protective compounds for insidious neurodegenerative diseases with few treatment options, including: Huntington’s Disease, Rett Syndrome, Multiple Sclerosis, Parkinson’s and Alzheimer’s Disease,” the company’s co-founders Robert Alonso, CEO, and John Geisler, PhD, CSO, said in a press release. “Mitochon had a remarkable 2015, with a series of successful proof-of-concept studies, that have now paved the path for clinical development.”
Participating in the financing round were Ben Franklin Technology Partners and a number of private investors.
MP101 is a daily, oral therapy shown to protect cells from neurodegenerative processes caused by auto-immune responses, injuries, or genetic factors. It has demonstrated significant benefits in several disease models, including Huntington’s, Rett syndrome, Parkinson’s and MS. In MS, particularly, it has been shown to provide axon protection from demyelination.
The company expects to take MP101 into Phase 1 studies in healthy volunteers this year, and to begin studies with Huntington’s patients, where the agent has been shown to aid brain volume, in 2017.
Mitochon is also developing a mitochondria-targeted neuroprotector, MP201, and plans to soon file an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA). Similar to MP101, MP201 has been shown to elicit mild increases in energy production, improving nerve cell survival. Both drugs induce the production of brain-derived neurotrophic factor (BDNF), an important neurotropin for neural growth and cognition.