US-based biotechnology company Larimar Therapeutics has reported preliminary top-line results from a trial of CTI-1601 for the treatment of Friedreich’s ataxia (FA).

The results were from the 25mg cohort of the Phase II clinical trial of CTI-1601.

The four-week, placebo-controlled, dose exploration study has enrolled 13 participants with FA.

Nine of these participants received subcutaneous injections of 25 mg CTI-1601 daily for 14 days and every other day thereafter until day 28, while the remaining patients were given placebo.

Participants who received CTI-1601 showed increases in levels of frataxin (FXN) from baseline against placebo in skin and buccal cells and were well-tolerated.

No serious adverse events or important medical events were reported during the study.

Larimar Therapeutics president and CEO Carole Ben-Maimon said: “Thirty-seven adults with FA have been dosed with CTI-1601 across our Phase I and II trials, with 35 completing treatment, one withdrawing due to an allergic reaction, and another withdrawing after a single 50mg dose in the multiple ascending dose trial due to mild to moderate nausea and vomiting.

“We believe the safety, pharmacokinetic, and pharmacodynamic data generated to date support evaluation of a 50mg dose of CTI-1601 in our Phase II trial and look forward to discussing our findings with the US Food and Drug Administration (FDA) at our meeting later this quarter.”

Larimar Therapeutics has submitted the data from the 25mg cohort of the trial to the FDA.

A meeting is scheduled for later this quarter to receive authorisation and start a 50mg cohort as part of the trial.

Based in Pennsylvania, Larimar Therapeutics is focused on developing treatments for complex rare diseases.

In addition to CTI-1601, the company plans to use its intracellular delivery platform to design other fusion proteins to target rare diseases characterised by deficiencies in intracellular bioactive compounds.