
Core Insights - Larimar Therapeutics, Inc. released initial data from a long-term study on nomlabofusp for Friedreich's Ataxia, showing significant increases in tissue FXN levels [1][2] - The data indicates a promising potential for nomlabofusp to transform treatment for this rare neurological disease, with analysts projecting over 200% upside for the stock [2] Group 1: Study Results - The study evaluated daily subcutaneous injections of 25 mg of nomlabofusp, resulting in a mean change of 1.32 pg/μg in buccal cells and 9.28 pg/μg in skin cells at Day 90 [1] - Tissue FXN levels increased from 15% of healthy volunteers at baseline to 30% in buccal cells and from 16% to 72% in skin cells by Day 90 [2] - FXN levels reached steady-state by Day 30 in buccal cells [2] Group 2: Analyst Perspectives - William Blair considers the biomarker data for the 25 mg dose to be stronger than expected, nearing the 50% FXN level seen in unaffected healthy individuals [3] - The analyst anticipates that escalating the dose to 50 mg will further enhance FXN levels to at least 30% of normal levels in approximately 90% of patients [3] Group 3: Safety and Stock Performance - Concerns regarding the safety of nomlabofusp have contributed to stock weakness, with four patients withdrawing from the study due to logistical challenges and serious adverse events [4] - Despite these concerns, William Blair confirmed no CNS-related adverse events in toxicology studies and maintained an Outperform rating for the stock [5] - LRMR stock experienced an 11.8% decline, trading at $4.262 [6]