Larimar’s CTI-1601 Phase 2 Trial Now Enrolling in US
Trial results are anticipated to inform CTI-1601’s long-term dose regimen
A four-week Phase 2 clinical trial that’ll be testing CTI-1601, Larimar Therapeutics’ experimental therapy for Friedreich’s ataxia (FA), is now enrolling in the U.S., the company announced in a press release.
Patient recruitment began shortly after the U.S. Food and Drug Administration (FDA) lifted its full hold on the clinical program for CTI-1601 and cleared its return under a partial hold. The program was originally paused for safety concerns after the deaths of primates treated with a high dose and more data was requested.
The trial is designed to enroll up to 30 patients across two groups, with a first group of 12–15 patients receiving either a 25 mg dose of CTI-1601 or a placebo. Under the partial hold, the trial’s design stays the same, but the company is being asked to review data from the first group before escalating the dose in the second group, pending the FDA’s go-ahead.
With recruitment now underway at Clinilabs in Eatontown, New Jersey, the company can maintain its planned timeline. An update is expected in the second quarter of 2023 and top-line data in the second half of 2023. Those interested in enrolling may contact Amber Farwig, at 212-994-4567 (ext 66407) or via email at [email protected].
“This is an exciting time for Larimar, as we recently received regulatory clearance for CTI-1601’s return to the clinic under a partial hold,” said Carole Ben-Maimon, MD, Larimar’s president and CEO.
The company has recently secured a cash runway into the second half of 2024 and a patent for CTI-1601 that provides composition of matter protection until at least July 2040.
“These important accomplishments allowed us to begin enrolling patients in the first cohort of our Phase 2 trial in Friedreich’s ataxia patients, which continues to make progress,” Ben-Maimon said.
People with FA have a shortage of frataxin, a protein required for mitochondria, a cell’s energy-producing power plants, to properly function. Too little frataxin causes symptoms such as loss of balance and coordination, increasing muscle weakness, and vision and hearing loss.
CTI-1601 uses a peptide (small protein) as a carrier to deliver the missing frataxin protein into mitochondria. Once in, this carrier-bound frataxin can be processed to become mature frataxin. This is expected to restore frataxin levels and keep mitochondria healthy and working, which may help ease symptoms.
Results of a Phase 1 clinical trial (NCT04519567) showed CTI-1601 led to a dose-dependent increase in frataxin levels and was well tolerated. No serious side effects were reported.
The goal of the Phase 2 clinical trial (NCT05579691) is to test how safe and well tolerated CTI-1601 is versus a placebo in people ages 18 and up with a genetically confirmed diagnosis. CTI-1601 will be given as an under-the-skin (subcutaneous) injection over 28 days. The injections will be given once a day for 14 days and every other day for the rest of the study.
It will also be testing CTI-1601’s pharmacokinetics (its movement into, through, and out of the body) and pharmacodynamics (its effects in the body).
“The trial’s results are expected to inform CTI-1601’s long-term dose regimen, thereby building upon the clinical proof-of-concept data generated in our Phase 1 program,” Ben-Maimon said.
Among other criteria, eligible participants must be able to to cross 25 feet with or without an assistive device, such as a cane, walker, crutches, or self-propelled wheelchair. FA patients who participated in prior Phase 1 CTI-1601 studies may be eligible if they didn’t have a clinically significant side effect or a serious side effect related to treatment.
“We are grateful for the continued support we have received from the Friedreich’s ataxia community and our shareholders over these past months and look forward to CTI-1601’s continued development as a potentially disease-modifying therapy,” Ben-Maimon said.