The U.S. Patent and Trade Mark Office (USPTO) sent a notice of allowance — meaning it intends to issue a patent — to Mitochon Pharmaceuticals regarding the clinical development of its investigative drug MP101 for the treatment of several mitochondrial-related diseases, including Huntington’s disease and Duchenne muscular dystrophy, among others.
This is Mitochon’s first patent allowance. It is for its U.S. Patent Application (No. 15/002,531) titled “Induced Expression Of Brain Derived Neurotrophic Factor (Bdnf) for Treatment of Neuromuscular, Neurodegenerative, Autoimmune, Developmental and/or Metabolic Disease,” and is designed to protect the method of treatment of certain diseases.
“We are delighted to receive our first patent allowance. It clearly demonstrates the novelty of our work in developing mitochondrial targeted therapies for neurodegeneration,” Robert Alonso, co-founder and CEO of Mitochon, said in a press release.
“Importantly, this Patent Allowance will help pave the way for human clinical development, with the hope of helping the millions of people with devastating, neurological diseases,” he said.
MP101 and MP201 are two investigative therapies designed to target mitochondrial dysfunction with a once-daily oral dosage. These drugs were engineered to prevent the production by mitochondria and accumulation in cells of harmful reactive oxygen species (ROS). In addition, they were also developed to promote the production of new mitochondria and the release of signaling molecules, which will support the growth and nourishment of nerve cells.
In preclinical studies these investigative drugs have shown potential in protecting nerve cells from degenerative damage in several disease models.
Administration of MP101 or MP201 preserved brain volume and improved functional motor performance in models of Huntington’s disease and multiple sclerosis.
These therapy candidates have also been shown to protect nerve cells from losing their myelin protective layer and prevent the death of nerve cells. In addition MP101 improved muscle response and strength in mice with Duchenne muscular dystrophy.
“By harnessing the power of the mitochondria, our compounds are able to protect cells from the destructive effects of degeneration,” Alonso said.
Mitochon is planning to initiate Phase 1 clinical studies to evaluate the safety of its drug candidates in healthy volunteers in 2018, and Phase 2 trials in 2019.