BALA CYNWYD, PA — Larimar Therapeutics, Inc. (Nasdaq: LRMR) has announced significant progress in its nomlabofusp program for Friedreich’s Ataxia (FA), reporting key regulatory advancements, ongoing clinical developments, and a robust financial position as of the end of 2024.
The company revealed encouraging news from the U.S. Food and Drug Administration (FDA), which has expressed openness to consider skin frataxin (FXN) concentrations as a reasonably likely surrogate endpoint for Larimar’s Biologics License Application (BLA), targeted for submission by the end of 2025. The FDA also acknowledged that initial findings suggest a meaningful relationship between increased skin FXN levels and tissues such as the heart and skeletal muscle.
“The strong clinical and regulatory progress across our nomlabofusp program reinforces the timing of our planned Biologics License Application (BLA) submission expected by the end of 2025 to seek accelerated approval,” said Carole Ben-Maimon, MD, President and Chief Executive Officer of Larimar. “Importantly, we are enthusiastic about the recent FDA interactions and their openness to consider skin frataxin (FXN) concentrations as a potential novel surrogate endpoint reasonably likely to predict clinical benefit in patients with Friedreich’s ataxia (FA). Acceptability of FXN as a novel surrogate endpoint will be based on review of the data by the Food and Drug Administration (FDA) in the future BLA. As we previously disclosed, we have been collecting skin FXN concentration data throughout the development program.”
Regulatory and Clinical Milestones
Larimar continues to advance its clinical trials and engage with both the FDA and international regulatory agencies. Key updates include plans to begin a global Phase 3 trial by mid-2025, which will include sites in the U.S., Europe, U.K., Canada, and Australia.
“Our open label extension (OLE) study continues to advance, with some participants on daily treatment for up to one year,” added Dr. Ben-Maimon. “We continue to actively enroll study participants, and all participants are currently receiving the 50 mg dose.” Larimar plans to present updates on the OLE study, including data from 30 to 40 participants, in September 2025.
Additionally, an adolescent cohort in the pediatric pharmacokinetic (PK) run-in study is progressing, with participants receiving weight-based doses expected to mimic adult PK profiles. The data from this cohort is also anticipated later this year.
The FDA has also approved transitioning to a lyophilized form of nomlabofusp, which Larimar intends to commercialize.
Financial Results
Larimar reported $183.5 million in cash, cash equivalents, and marketable securities as of December 31, 2024, projecting that this will sustain operations into the second quarter of 2026.
The company’s net loss for 2024 totaled $80.6 million, driven by increased research and development expenses, which rose to $73.3 million due to manufacturing scaling and clinical trial advancements.
Reflecting on the progress, Dr. Ben-Maimon stated, “We believe our near-term data package strongly positions us to bring the first potential disease modifying therapy to patients with FA.”
Larimar remains focused on advancing its clinical program and delivering a long-awaited breakthrough for patients with unmet medical needs. The company’s ongoing engagement with the FDA and global regulatory agencies reinforces confidence in progressing toward a potential therapy for Friedreich’s Ataxia.
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