The submission was prompted by results of a Phase 1/2 trial showing that the therapy was safe and exhibited early signs of effectiveness — an ability to improve patients’ physical functioning. A pivotal trial is one that can provide the evidence needed for regulatory approval. It is usually a Phase 2 or 3 study.
“The Phase 1b/2a trial provides an early signal that RT001 may be able to address one of the most important concerns of FA patients — namely, the ability to generate additional energy during exercise and avoid the profound fatigue in performing most tasks,” Peter G. Milner, Retrotope’s chief medical officer, said in a press release.
“Based on these findings and additional positive results from the trial, we intend to move RT001 forward in this disease and have submitted a pivotal study protocol to the US FDA for review,” he said.
The Phase 1/2 trial (NCT02445794) covered 18 Friedreich’s ataxia patients. They were randomized to receive either one of two oral doses of RT001 or a placebo for 28 days. Researchers said the trial met its main objectives of showing that the treatment was safe and that patients could tolerate it well.
While the study wasn’t designed to assess the therapy’s effectiveness, in just one month, patients’ physical functioning improved in three measures — peak workout ability, peak oxygen consumption, and stride speed — compared with controls.
Researchers published the trial results in the journal Movement Disorders in a study titled “Randomized, clinical trial of RT001: Early signals of efficacy in Friedreich’s ataxia.”
“These are the first findings published in a peer-reviewed journal demonstrating that a D-PUFA [deuterated polyunsaturated fatty acid] can show both safety and early indications of possible efficacy over a short treatment window of 28 days in patients with a progressive neurodegenerative disease such as FA,” said Theresa Zesiewicz, M.D., director of the University of South Florida’s Ataxia Research Center and principal investigator of the trial.
“While biological activity was not a primary goal of the study, we are encouraged by the study results and look forward to further progress of the program,” she added.