Following clearance by the US drug regulatory body the Food and Drug Administration (FDA) in July 2023, the pharmaceutical company Larimar Therapeutics has completed full enrolment and dosing of the 50 mg cohort in their US Phase 2 dose exploration trial of the drug CTI-1601 (nomlabofusp) for Friedreich’s ataxia (FA). CTI-1601 is a protein replacement therapy designed to deliver frataxin protein to the cells of people with FA. Frataxin proteins are decreased in those with FA. This phase of the trial will involve a daily dose of 50 mg of CTI-1601 for the first 14 days and then every other day until day 28, and it includes 15 participants. There were no reported serious adverse effects in either the CTI-1601 or placebo groups during the dosing period.
This progress comes after data from the completed 25 mg cohort indicated that CTI-1601 was generally well tolerated and showed increases in frataxin protein levels from baseline compared to placebo at day 14 (the final day of daily dosing in the trial). The company hope to release further data on the both the 50 mg and 25 mg cohorts, including safety information, in the first quarter of 2024.
The FDA has also given approval for Larimar Therapeutics to begin an open label extension trial with a 25 mg daily dose of CTI-1601 for 28 days in the first quarter of 2024. Open label extension trials are those that take place after randomised drug trials if results show the drug is found to have potential benefit. This open label trial will look at the long-term effects of CTI-1601. Larimar Therapeutics plan to use the findings from the 50 mg dose exploration trial to inform the dose and dosing regimen for the open label trial.
You can read the full press release from Larimar Therapeutics here. To receive more updates about the trials and their results, sign up to our e-newsletter here and follow our ‘Research news’ section on the Ataxia UK website here.