Larimar Therapeutics Inc. (NASDAQ:LRMR) has submitted the initial module of its Biologics License Application (BLA) to the U.S. Food and Drug Administration for Nomlabofusp, seeking accelerated approval. The company plans to file the remaining modules in the second half of 2026.
BLA Submission and Clinical Data
Larimar announced on June 29 that it had submitted the first module of its BLA for Nomlabofusp, a therapy targeting Friedreich's ataxia. The application is based on positive results from an open-label study in adult patients receiving daily injections. President and CEO Carole Ben-Maimon described the new data as a significant milestone in developing the treatment. The FDA has confirmed that existing data, which uses skin frataxin as a novel surrogate endpoint, is sufficient to support the application.
Analyst Reaction and Price Target
On June 30, Baird analyst Christopher Chen lowered his price target for Larimar shares from $7 to $5, while maintaining an Outperform rating. The revised target still implies over 53% upside from current levels. Chen's adjustment followed the company's Nomlabofusp updates, which showed clinical benefits but also raised some safety concerns. Larimar is a clinical-stage biotech focused on rare diseases using its cell-penetrating peptide platform, with CTI-1601 as its lead candidate in Phase 2 open-label extension trials for Friedreich's ataxia.