Participants in the open label extension study will receive subcutaneous injections of 25mg CTI-1601 daily. Credit: Marko Aliaksandr/Shutterstock.com.

Larimar Therapeutics has received clearance from the US Food and Drug Administration (FDA) to advance its Phase II trial of CTI-1601 to a 50mg cohort in patients with Friedreich’s ataxia (FA), a rare inherited disease causing damage to the nervous system and problems in movements.

The dose exploration, placebo-controlled trial will assess the pharmacokinetic (PK), pharmacodynamics (PD) and safety of the protein replacement therapy CTI-1601 in FA patients.

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They will receive a daily dose of the therapy for the first 14 days, and then every other day until day 28.

The approval is also to initiate an open-label extension (OLE) trial with 25mg CTI-1601. Participants in this study will receive subcutaneous injections daily.

Larimar Therapeutics president and CEO Carole Ben-Maimon said: “Gaining clearance to advance to a 50mg cohort in our Phase II trial and initiate the OLE trial are crucial steps in CTI-1601’s development as potentially the first therapy to increase frataxin levels (FXN) in patients with FA.

“Given the inability of current treatments to address the frataxin deficiency underlying Friedreich’s ataxia, we believe CTI-1601 has the potential to improve the treatment paradigm for this devastating disease.

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“We now look forward to data from our Phase II trial’s 50mg cohort in the first half of 2024, which will help us further characterise the safety and PK profiles of CTI-1601 and its ability to increase frataxin levels in a dose-dependent fashion as seen in our earlier Phase I studies.”

Approvals for both trials come after the FDA’s review of the company’s complete response to its partial clinical hold that featured unblinded safety, PK and PD data from the Phase II trial’s completed 25mg group. 

In the 25mg cohort of the Phase II trial, which included 13 patients, CTI-1601 was found to be well tolerated and showed an increase in FXN levels from baseline, as compared with placebo in all evaluated tissues (skin and buccal cells) at day 14.

Larimar stated that the dose escalation in the Phase II and OLE trials and commencement of further trials in the US are based on the FDA’s review of results from the Phase II trial’s 50mg cohort in line with a partial clinical hold. 

The OLE trial is anticipated to commence in the first quarter of next year while interim data is expected in the fourth quarter of next year.