Mitochon Pharmaceuticals announced the completion of a second year of financing, with investments totaling $1.6 million that will advance the company’s lead compound MP101 — a neuroprotective drug targeting mitochondria — into clinical studies. The funds will also be used to further develop MP201 in preparation for the filing of an Investigational New Drug Application with the U.S. Food and Drug Administration.
The company, founded two years ago, has made notable progress with its lead mitochondrial drugs MP101 and MP201. While focusing on different conditions — Huntington’s disease, Duchenne muscular dystrophy, Battens disease, epilepsy, burn injuries, multiple sclerosis, stroke, Alzheimer’s and Parkinson’s — both drugs intend to provide neuroprotection by mild increases in energy consumption, resulting in improved cell survival. As part of their mechanisms, the drugs also trigger the Brain Derived Neurotrophic Factor (BDNF), a key molecule for the growth of neurons.
MP101, a compound that employs an entirely new mechanism to affect its target tissues, is intended as a once-daily oral treatment to protect cells from a range of neurodegenerative processes. It reduces damage induced by mechanisms as variable as genetic factors, autoimmune processes, and tissue injury, and has shown highly positive results in preclinical experiments modeling various diseases.
In Huntington’s, the MP101 drug could spare brain volume decrease; in multiple sclerosis, it might protect axons from demyelination. It also led to motor symptom improvement in Rett syndrome and Parkinson’s disease.
Mitochon plans to start a Phase 1 clinical trial of MP101 involving healthy volunteers this year, to investigate how the effects observed in animal models translate to human physiology. A trial in Huntington’s disease patients is expected to take place in 2017.
“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, 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.”
Investors included the Pennsylvania-based technology development program Ben Franklin Technology Partners, as well as private investors.