MP101 Given FDA’s Orphan Drug Status for Potential to Treat Huntington’s Disease

The U.S. Food and Drug Administration has granted orphan drug designation to MP101, an investigational mitochondrial-targeted neuroprotective agent, to treat Huntington’s disease.

Mitochon Pharmaceuticals, the therapy’s developer, expects to begin a Phase 1 clinical trial in healthy volunteers in 2019 and move to Phase 2 studies in 2020.

“We are delighted to receive our first orphan drug designation for Huntington’s Disease, a devastating neurodegenerative disorder with no disease modifying therapies on the market today,” Robert Alonso, co-founder and CEO of Mitochon, said in a press release.

A number of studies have shown an association among mitochondrial dysfunction, oxidative stress, and neurodegenerative diseases, including Huntington’s disease. Mitochondria are small cellular organelles that provide energy to cells. Oxidative stress is caused by an imbalance between the body’s production of potentially harmful reactive oxygen species and its ability to contain them, causing nerve cell damage.

MP101 is one of Mitochon’s two leading therapeutic candidates. The compound is a daily, oral therapy that specifically targets the mitochondria to provide broad neural protection. MP101 elicits mild increases in energy expenditure, resulting in stronger cellular survival, and is similar to the positive effects observed with fasting and exercise.

MP101 and MP201 also induces an important neurotrophin called brain-derived neurotrophic factor (BDNF), which is involved in cognition and neural growth and also improves calcium handling.

Previous data using a mouse model of Huntington’s disease showed that treatment with MP101 improved animals’ motor function and reduced brain shrinkage, or atrophy. Moreover, MP101 reduced oxidative stress in the brain cortex — an area involved in information processing.

“By harnessing the power of the mitochondria, MP-101 has been shown to protect both spiny neurons and general neurons as well as minimize brain volume loss from the destructive effects of Huntington’s Disease. Together these effects have the potential to significantly change the progression of the disease in patients,” Alonso said.

“This designation is an important milestone for Mitochon and we are excited to move into patients and help the thousands of families with Huntington’s Disease,” he added.

In November 2017, Mitochon was granted a patent securing the development of MP101.

Orphan drug status is intended to encourage therapies for rare and serious diseases through benefits such as seven years of market exclusivity and exemption from FDA application fees.