Larimar Therapeutics reports Q1 EPS (27c), consensus (23c)

As of March 31, the company had cash, cash equivalents and marketable securities totaling $239M. “We have started 2024 off strong, achieving critical milestones that support late-stage advancement of our nomlabofusp clinical program. Positive Phase 2 dose exploration study data demonstrated that nomlabofusp appears to be generally well-tolerated, and observed dose dependent increases in tissue frataxin levels that have the potential to address the underlying frataxin deficiency that is the root cause of Friedreich’s ataxia (FA). In March, we dosed the first patient in our OLE study and continue to enroll patients and activate additional sites. We are on track to report interim data in the fourth quarter of the year which will inform on the long-term safety and tissue frataxin levels of nomlabofusp,” said CEO Carole Ben-Maimon. “Together, these datasets will help support our BLA submission which we are targeting for the second half of 2025. We are continuing our regulatory discussions with the FDA on the potential use of frataxin as a novel surrogate endpoint to support accelerated approval and are planning for a global double-blind placebo-controlled confirmatory study which we expect to initiate prior to BLA submission. With our recent capital infusion and runway extended through key registrational catalysts, we are well positioned to further advance the first potential therapy to increase frataxin levels in patients with FA.”