Core Insights - Larimar Therapeutics Inc. is experiencing a decline in stock price following the release of data from its ongoing long-term open-label study of nomlabofusp for Friedreich's ataxia [1][7] Study Overview - The open-label study is assessing the safety, tolerability, pharmacokinetics, FXN levels in skin and buccal cells, exploratory pharmacodynamic markers, and clinical outcomes after long-term subcutaneous administration of nomlabofusp [2] - Initial dosing started at 25 mg daily, with an increase to 50 mg planned for the fourth quarter of 2024, and all new patients have been receiving the 50 mg dose since November 2024 [2] Patient Safety and Adverse Events - A total of 65 patients have received at least one dose of nomlabofusp across four completed studies and the ongoing open-label study [3] - Seven participants experienced anaphylaxis and were withdrawn from the study, with most events occurring on the first day of administration and within the first six weeks [3][4] - The company has modified its dosing regimen to include a 5 mg test dose followed by a 25 mg dose under observation [5] Clinical Outcomes - Trends towards improvement were noted in the modified Friedreich Ataxia Rating Scale (mFARS), FARS-Activities of Daily Living (ADL), 9 Hole Peg Test (9-HPT), and Modified Fatigue Impact Scale (MFIS) after one year of treatment [6] - Participants treated for one year showed a median improvement in mFARS score of 2.25, contrasting with a worsening of 1.00 in the FACOMS reference population [6] Future Plans - The company is on track to submit a marketing application for potential accelerated approval of nomlabofusp in Friedreich's ataxia in the second quarter of 2026 [7]