BALA CYNWYD, PA — Larimar Therapeutics, Inc. (Nasdaq: LRMR) has unveiled its operational and financial results for the first quarter of 2025, showcasing significant progress in its clinical development program for nomlabofusp, a potential disease-modifying therapy for Friedreich’s ataxia (FA). The company remains on track with its development timeline and has announced notable regulatory discussions and clinical milestones.
“Our strong execution across our nomlabofusp clinical development program continues, and we are focused on delivering the first potential disease-modifying therapy to patients with FA,” said Carole Ben-Maimon, MD, President and CEO of Larimar. “With the robust preclinical and clinical data package we have in hand and the additional clinical data readouts coming later this year, coupled with the positive FDA feedback and recommendations in line with our current approach, we are on track to submit our planned Biologics License Application (BLA) by the end of 2025 to seek accelerated approval.”
Key updates include completed adolescent dosing in a pharmacokinetic (PK) run-in study, with data expected as part of a program update in September 2025. The company is also moving forward with plans to initiate a global Phase 3 study in mid-2025 following regulatory feedback. Additionally, Larimar plans to transition to a lyophilized formulation of nomlabofusp to support future commercial use.
The FDA’s willingness to consider skin FXN concentration as a reasonably likely surrogate endpoint marks a critical regulatory milestone, paving the way for potential accelerated approval. The agency has indicated that additional clinical and pharmacodynamic data could strengthen the case for FXN as a surrogate biomarker.
From a financial perspective, Larimar ended the first quarter with $157.5 million in cash, cash equivalents, and marketable securities, projecting its cash runway through the second quarter of 2026. The company reported a net loss of $29.3 million for Q1 2025, up from $14.7 million in Q1 2024, driven primarily by increased research and development expenses tied to clinical trial costs and manufacturing investments.
Research and development expenses grew to $26.6 million, a rise attributed to higher manufacturing costs, clinical activities, and expanded staffing for the planned global Phase 3 study. General and administrative expenses also increased slightly to $4.6 million, reflecting additional personnel and consulting costs linked to commercial preparations.
Looking ahead, Larimar remains focused on key catalysts, including the launch of its global Phase 3 trial, clinical results from various studies, and the planned BLA submission by year’s end. With these efforts, the company aims to advance nomlabofusp as a groundbreaking treatment for FA while further strengthening its position in the rare disease space.
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