An Israeli biopharmaceutical company, Mitoconix Bio, has nabbed $20 million from investors to develop a promising drug for the treatment of Huntington’s disease and other neurodegenerative disorders.
There is no drug in existence that can slow or stop the progression of such diseases, said Dr. Eyal Neria, CEO of Mitoconix Bio. “This is the Holy Grail of drugs for neurodegeneration, or what’s called disease modifying agents,” he said.
The company was founded in August 2016 by Daria Mochly-Rosen, of Stanford University’s School of Medicine. Mochly-Rosen led a team of researchers that identified the molecular interactions that cause the mitochondria in brain cells to break down into smaller, dysfunctional versions of themselves. Mitochondria are tiny cellular structures (organelles) within cells that produce energy and contribute to other important functions, including regulation of cell growth.
Excessive fission, or splitting, of mitochondria is a central pathological process in human diseases, especially in neurodegeneration. The brain typically accounts for only two percent of a person’s body weight but 20 percent of the body’s energy consumption. As the energy-producing organelles break down, patients with Huntington’s disease experience subtle problems with mood regulation and mental abilities. A loss of physical coordination follows, which can worsen until any coordinated movement or speech become difficult. The condition often leads to dementia.
The Stanford team identified chemical inhibitors of excessive mitochondrial splitting, which slowed or stopped the disease in animal test subjects. That’s a breakthrough, since existing drugs only treat the symptoms of Huntington’s while the disease progresses “relentlessly,” according to Neria.
Existing drugs do “not really affect the underlying pathology,” he explained. “Here we think we have a drug that will slow or stop the progression of the underlying pathology.”
While the company is focusing on developing a drug specifically for Huntington’s disease, its success would lay the foundation for similar treatment advances for other neurodegenerative disorders, including Alzheimer’s and Parkinson’s, Neria said.
Mitoconix Bio licensed the inhibitor technology from Stanford University. The $20 million raised will fund preclinical and clinical trials in humans. Neria said he expects the drug’s clinical development to start in about two years.
“This is very significant funding for a Series A for a company in Israel,” he said.
Remiges Ventures, a US-based venture capital firm, led the round of funding in partnership with OrbiMed, Dementia Discovery Fund, Arix Bioscience and RMGP Bio-Pharma Investment Fund, the firm said.
Mitoconix Bio was established at the biotech incubator FutuRx in Israel, which is backed by Johnson & Johnson and the Israel Innovation Authority, among others.
Anya Eldan, general manager of the Israel Innovation Authority’s startup division, said her agency’s participation makes connections with experienced investors and management teams. “Government support reduces the risk and enables establishment of innovative early stage ventures such as Mitoconix,” she said.