Larimar Therapeutics Reports Positive Preliminary Top-line Data from Phase 2 Study in FA

Rare Daily Staff

Larimar Therapeutics said preliminary top-line data from the 25 mg cohort of its phase 2, four-week, placebo-controlled, dose exploration trial of CTI-1601 in participants with Friedreich’s ataxia was generally well tolerated and showed increases in frataxin levels from baseline compared to placebo in all evaluated skin and buccal cells at day 14.

Friedreich’s ataxia (FA) is a rare, progressive, multi-symptom genetic disease that typically presents in mid-childhood and affects the functioning of multiple organs and systems. The most common inherited ataxia, FA is a neurodegenerative disease resulting in multiple symptoms including progressive neurologic and cardiac dysfunction—poor coordination of legs and arms, progressive loss of the ability to walk, generalized weakness, loss of sensation, scoliosis, diabetes and cardiomyopathy as well as impaired vision, hearing and speech. FA affects an estimated 4,000-5,000 individuals living in the United States and between 18,000 and 20,000 patients in the European Union. FA results from a deficiency of the mitochondrial protein, frataxin (FXN), which is found in cells throughout the body. To date, there are no medical treatment options approved for patients with FA.

CTI-1601 is a recombinant fusion protein intended to deliver human frataxin to the mitochondria of patients with Friedreich’s ataxia who are unable to produce enough of this essential protein. The U.S. Food and Drug Administration granted CTI-1601 Rare Pediatric Disease designation, Fast Track designation and Orphan Drug designation. The European Medicines Agency granted CTI-1601 Orphan Drug designation and PRIME designation.

Larimar’s phase 2 data and non-interventional study results follow phase 1 data that showed dose-dependent increases in frataxin levels in peripheral tissue with daily dosing of 50 and 100 mg of CTI-1601 for at least seven days, and no detectable increase in FXN levels with daily dosing of 25 mg of CTI-1601 for only four days.

Larimar has submitted the data from the trial’s 25 mg cohort to FDA and has a meeting scheduled with the agency for later this quarter to discuss the information needed to gain clearance to initiate a 50 mg cohort in the phase 2 trial.

“Our preliminary phase 2 data provide the first clinical indication that a 25 mg dose of CTI-1601 can increase frataxin levels in peripheral tissues, building upon our proof-of-concept phase 1 results,” said Carole Ben-Maimon, president and CEO of Larimar. “Importantly, the frataxin increases achieved with a relatively low 25 mg dose in our phase 2 trial suggest a continuous daily dosing regimen is preferred for maintaining increases achieved with 25 mg CTI-1601.”

Photo: Carole Ben-Maimon, president and CEO of Larimar