Larimar Therapeutics Inc

Larimar Therapeutics has submitted the first module of its rolling Biologics License Application seeking accelerated approval of nomlabofusp for Friedreich’s ataxia, with completion expected in the second half of 2026. The submission follows a successful Type B pre-BLA meeting with the FDA, which confirmed the existing data package appears capable of supporting review and agreed to consider frataxin as a novel surrogate endpoint. Longer-term open-label study data showed sustained increases in skin frataxin levels, with 100% of participants reaching levels above asymptomatic carriers at one year, and a 2.6-point benefit on the mFARS scale at one year relative to a natural history population. The company reported $156.3 million in cash, cash equivalents and marketable securities as of June 30, 2026, providing a projected cash runway into the third quarter of 2027. Dosing of the first patient in a global confirmatory Phase 3 study is expected in the third quarter of 2026, with a potential launch targeted for mid-2027 if approved.

Larimar Therapeutics has submitted the initial module of its Biologics License Application to the U.S. FDA seeking accelerated approval for its Nomlabofusp therapy. The company plans to submit the remaining modules during the second half of 2026. The therapy showed positive outcomes in an open-label study of adult patients receiving daily injections for Friedreich's ataxia, and the FDA confirmed that existing data based on the surrogate endpoint of skin frataxin is sufficient to support the application. Separately, Baird analyst Christopher Chen lowered his price target on the stock from $7 to $5 while maintaining an Outperform rating, citing therapy updates that indicate clinical benefits but also carry safety concerns.

Larimar Therapeutics announced it will host an investor event on Monday, June 29, 2026, to provide updates on its Nomlabofusp program for Friedreich's ataxia. The webcast is scheduled for 7:45 a.m. Eastern Time and will include a regulatory update and data from the ongoing Phase 2 long-term open-label study of Nomlabofusp. The company is developing Nomlabofusp as a potential treatment for both adults and children with the rare neurodegenerative disorder. Larimar also plans to use its intracellular delivery platform to design fusion proteins for other rare diseases. The stock closed Friday at $3.66, up 4.57%, and rose to $4.00 in after-hours trading, a gain of 9.29%.