Dr. Colin Meyer, the chief medical officer and vice president of Reata Pharmaceuticals, spoke Friday in a taped interview with reporter Patricia Inacio about omaveloxolone, an oral therapy in line to possibly become the first FDA-approved treatment for Friedreich’s ataxia.
A Phase 2 clinical trial, called MOXIe (NCT02255435), is moving into a second and possibly pivotal part, testing the effectiveness of omaveloxolone against placebo in about 100 FA patients ages 16 to 40. All enrolled will be randomized to either omaveloxolone at 150 mg or a placebo. The trial’s main goal is to evaluate changes from baseline in the modified Friedreich Ataxia Rating Scale (mFARS).
Omaveloxolone, also known as omav, is designed to activate the Nrf2 protein within cells to restore mitochondrial energy production, which is depressed in FA, and to dampen inflammation in cells. “This translates,” Meyer said, “to improved organ function in the muscles, in the brain and elsewhere.”
Part one of MOXIe — a dose-escalating phase conducted in 69 FA patients — went well, Meyer said in the interview.
“We were able to show in patients with Friedreich’s ataxia that omav could induce Nrf2 … and improve markers of mitochondrial function. We also demonstrated the drug has activity on many important neurological parameters, such as the modified FARs,” he said, adding that omaveloxolone “was also well-tolerated with no discontinuations at the optimal dose.”
This trial is a “registrational,” meaning that it is like a Phase 3 trial and — if successful — could lead to a first FA treatment being approved worldwide, Meyer said.
But before that, he added, we have to enroll the patients.
MOXIe is currently recruiting FA patients with mFARS scores from 20 to 80 at study sites across the U.S., and in Austria, Australia and the U.K. More information, including on enrollment and eligibility, is available on its clinical trials.gov page.
The full interview with Dr. Meyer can be seen here: