On Monday, shares of Larimar Therapeutics fell significantly after a data update from the ongoing study into Friedreich’s ataxia treatment. The update included reports of serious adverse events (SAEs) in two patients, both of whom chose to withdraw from the study. Despite these events being resolved within 24 hours, the stock briefly fell as much as 51% in New York trading before recovering slightly to a 25% loss.
The company claimed in a premarket statement that daily subcutaneous injections of 25 mg of nomlabofusp were “generally well tolerated for up to 260 days” in the open-label extension study (OLE). Larimar remains committed to its goal of submitting a Biologics License Application (BLA) to U.S. regulators in the second half of 2025, aiming for possible accelerated approval for the treatment of the rare neurodegenerative disease.
According to a press release dated December 16, 2024, Larimar announced positive initial data from the OLE study showing that nomlabofusp increased and maintained tissue frataxin (FXN) levels. The company has also observed early trends indicating potential clinical benefits. Treatment appears to have reached steady state by day 30 and dose escalation to 50 mg has begun in six participants. In addition, screening in adolescents for a pediatric pharmacokinetic (PK) study is underway, with dosing expected in early 2025.
Despite the setbacks, analysts from Leerink Partners, Citi and Guggenheim shared their views on the stock’s potential. They acknowledged the adverse events, but highlighted the encouraging biomarker and functional data, the company’s strong balance sheet and the continued development of the nomlabofusp program. They also noted that the stock’s reaction to the safety news appeared overdone and recommended buying on the dip, reiterating confidence in the drug’s favorable risk-benefit profile.
Larimar ended the third quarter of 2024 with $203.7 million in cash and investments, extending its financial runway into the second quarter of 2026. The company is preparing for key upcoming milestones, including the launch of a global confirmation/registration study in mid-2025. initial data from the 50 mg dose in the OLE study and the targeted BLA submission for possible accelerated approval in the second half from 2025.
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