Larimar Therapeutics' New Drug Could Transform Treatment For Rare Neurological Disease, Analyst Sees Over 200% Upside For Stock

Company Overview - Oppenheimer has initiated coverage on Larimar Therapeutics (LRMR), a clinical-stage biotechnology company focused on developing treatments for rare diseases [1] - The company's lead program, Nomlabofusp, is viewed as a differentiated, disease-modifying therapy targeting the key pathogenic mechanism of frataxin (FXN) deficiency in Friedreich's ataxia (FA) [1] Market Potential - Friedreich's ataxia is a rare neurodegenerative disease affecting approximately 20,000 patients globally [2] - Oppenheimer expects open-label extension (OLE) interim data in Q4 2024 and plans for Larimar to expand into the pediatric population, pursuing accelerated approval with a potential FDA marketing application submission in the second half of 2025 under the FDA's START Pilot Program [2] Financial Projections - Oppenheimer has set an Outperform rating with a price target of $26, assuming a 60% chance of accelerated approval, modeling a U.S. launch in 2027 and a European launch in 2028 for FA, estimating $1.2 billion in global revenues by 2030 [3] - Following the early 2023 approval of Skyclarys, developed by Reata and acquired by Biogen for $7.3 billion, it has generated approximately $278 million in global sales [3] Competitive Advantage - Nomlabofusp is distinguished from competitors due to its potential to modify the disease, with Larimar's strategy to include pediatric and adolescent FA patients in its Phase 2 OLE trial, addressing a larger patient demographic [4] - In contrast, Reata/Biogen's Skyclarys is approved for patients aged over 16, and PTC Therapeutics' vatiquinone is approved for patients over 7 [4] Stock Performance - LRMR stock is currently up 2.56% at $7.61 [5]