Larimar Therapeutics Announces Dosing of Adolescents in Nomlabofusp Pediatric Pharmacokinetic Run-In Study for Patients with Friedreich's Ataxia

Core Viewpoint - Larimar Therapeutics has initiated dosing of adolescents aged 12-17 in a pediatric pharmacokinetic (PK) run-in study for Friedreich's ataxia, marking a significant step in evaluating the safety and pharmacokinetics of their lead compound, nomlabofusp [1][2]. Group 1: Study Details - The PK run-in study involves a randomized 2:1 allocation of participants to receive either nomlabofusp or a placebo for seven days [2]. - Following the assessment of safety and PK data, participants will be eligible to transition into the ongoing open-label extension (OLE) study [2][7]. - A second cohort of children aged 2-11 years is planned to begin in the first half of 2025 [2][7]. Group 2: Future Expectations - Long-term data from the OLE study, including 50 mg data from adults and available data from adolescents, is expected to be reported in mid-2025 [2][7]. - The OLE study will evaluate safety, tolerability, pharmacokinetics, FXN levels, and exploratory pharmacodynamic markers following long-term administration of nomlabofusp [3]. Group 3: Company Overview - Larimar Therapeutics is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, with nomlabofusp as its lead compound for Friedreich's ataxia [4]. - The company aims to utilize its intracellular delivery platform to design additional fusion proteins targeting other rare diseases characterized by deficiencies in intracellular bioactive compounds [4].