Rare pediatric disease designation is granted to product applications for diseases that affect fewer than 200,000 children, age 18 or younger, in the U.S.
Fast track is intended to accelerate a therapy’s development and expedite its approval by providing more frequent meetings with the FDA and discussions about the development plan.
FA participants, 18 and older, will be randomly assigned to a placebo or single ascending doses of CTI-1601, both administered subcutaneously (under the skin).
The study’s primary endpoint is to assess the therapy’s safety and tolerability. Additional (secondary) endpoints include its pharmacokinetics — the therapy’s absorption, distribution, metabolism, and excretion — and pharmacodynamics, the study of the therapy’s effect on the body and its mechanism of action.
Topline results of the Phase 1 clinical program are expected by the end of 2020.
“On behalf of FARA [Friedreich’s Ataxia Research Alliance] and the FA community, we are grateful to the Chondrial team for their hard work and dedication in advancing CTI-1601 to this important milestone for a debilitating, progressive disease that lacks adequate treatment options and is currently only treated symptomatically,” Jennifer Farmer, executive director of FARA, said in a press release.
“FARA is committed to assisting Chondrial with recruitment for this trial by reaching out to individuals with FA enrolled in the Friedreich’s Ataxia Global Patient Registry and other awareness efforts,” she added.
CTI-1601 uses a carrier protein to deliver frataxin (the protein missing in FA) to the mitochondria, where the protein is expected to mature and integrate in mitochondrial metabolism. By replenishing the levels of healthy frataxin, CTI-1601 may assist in minimizing and halting disease progression.
“CTI-1601 is designed to address the root cause of Friedreich’s ataxia — low levels of frataxin,” said Carole Ben-Maimon, MD, president and CEO of Chondrial Therapeutics.
Studies in a mouse model of FA showed that treatment with CTI-1601 (formerly called TAT Frataxin) enhanced the mean lifespan of mice by 53%, with improvements also shown in cardiac function. These findings supported the launching of the clinical program for CTI-1601.
“The initiation of our Phase 1 clinical program was supported by positive preclinical data and is an important step forward for patients affected by FA, a devastating and progressive disease for which there is no cure,” said Ben-Maimon.
“We are incredibly pleased to announce this proposed merger, as, once completed, we anticipate it will provide significant resources to advance CTI-1601, our novel therapeutic being developed for patients with Friedreich’s ataxia and expand our efforts on the development of additional potential treatments for other rare diseases,” said Ben-Maimon.