Pharmather to Advance Ketamine as Potential Dyskinesia Treatment

Pharmather has entered into an exclusive licensing agreement with the University of Arizona, allowing the company to further develop ketamine as a potential treatment of dyskinesia — involuntary, jerky movements — associated with levodopa use in people with Parkinson’s disease.

The company plans to file an investigational new drug application with the U.S. Food and Drug Administration (FDA) to open a Phase 2 trial of ketamine, an approved anesthetic, later this year.

“We are very pleased to have an exclusive license agreement in place with the University of Arizona to allow Pharmather to advance the clinical program of ketamine in the treatment of Parkinson’s disease, specifically targeting levodopa-induced dyskinesia, a significant clinical unmet need,” Fabio Chianelli, CEO of Pharmather, said in a press release.

Evidence suggests that roughly 50% of Parkinson’s patients receiving levodopa, a mainstay of disease treatment, will develop dyskinesia within four or five years, and an estimated 80% will experience it after 10 to 12 years of levodopa therapy.

Ketamine has also been shown to reduce pain and symptoms of depression when given at lower, sub-anesthetic doses. Researchers at the University of Arizona originally intended to use ketamine as pain reliever for people with Parkinson’s.

When investigators treated five hospitalized patients with low-dose infusions of ketamine, they noticed an easing not only of their pain symptoms, but also their involuntary movements. One patient remained free of dyskinesia for several weeks post-treatment.

This observation led researchers to conduct additional preclinical studies that demonstrated ketamine, given at low doses, relieved abnormally jerky movements in a mouse model of levodopa-induced dyskinesia (LID).

Study data prompted the team to patent this treatment, a process that is ongoing, and to license it to Pharmather in order to advance its clinical development. If approved for dyskinesia, the treatment will also be marketed by the company.

As an FDA-approved treatment, ketamine has a known safety profile in humans, which may speed its potential repurposing as a treatment of LID in people with Parkinson’s.

Pharmather plans to possibly expand ketamine’s clinical development. In addition to being an anesthetic for surgeries or other medical procedures, ketamine is known to be effective in people with chronic pain and treatment-resistant depression.

“With promising results in preclinical models and in Parkinson’s disease patients, we aim to expeditiously advance the ketamine program in Parkinson’s disease to reduce dyskinesia associated with levodopa therapy,” and to advance it to potentially “treat depression and pain associated with Parkinson’s disease and other motor disorders that are prevalent in Multiple sclerosis, Alzheimer’s and Huntington’s disease,” Chianelli said.

A company goal is “ultimately creating a unique pharmaceutical-based ketamine franchise,” he added.