FDA lifts hold on Larimar's trial of CTI-1601 in Friedreich's ataxia patients

Positive news from the medicines regulator yesterday saw US clinical-stage biotech Larimar Therapeutics’ (Nasdaq: LRMR) shares close up 15.5% at $3.64.

The US Food and Drug Administration (FDA) has cleared the initiation of the 25mg cohort of a Phase II four-week, placebo-controlled, dose exploration trial of CTI-1601 in Friedreich’s ataxia (FA) patients, said Larimar, which was formed in 2020 through the merger of Chondrial Therapeutics and Zafgen.

In a written communication to Larimar, the FDA indicated it was lifting its full clinical hold on the CTI-1601 program and imposing a partial hold. The design of the upcoming Phase II trial is identical to the design proposed by Larimar, with the exception of a requirement for the FDA to review data from the 25mg cohort prior to escalating the dose in the second cohort. Larimar expects to begin the Phase II trial in fourth-quarter 2022, with top-line data expected in second-half 2023.