BALA CYNWYD, PA — Larimar Therapeutics, Inc. (Nasdaq: LRMR) reported new long-term open-label (OL) data for nomlabofusp in Friedreich’s ataxia (FA), showing sustained increases in frataxin (FXN) protein and consistent directional improvements across four clinical outcomes, alongside a revised starting-dose regimen and an accelerated-approval BLA timeline for Q2 2026.
As of August 27, 2025, 39 participants in the OL study had received at least one dose; 14 had been treated for at least six months and eight for more than one year. Across four completed studies and the ongoing OL study, 65 individuals have received at least one dose.
Daily subcutaneous nomlabofusp increased tissue FXN: all 10 participants with six-month data achieved skin FXN levels over 50% of median levels in healthy volunteers. At one year, the study showed directional improvements in mFARS, FARS-ADL, 9-Hole Peg Test, and MFIS versus worsening in a matched reference cohort from the FACOMS natural-history study.
“Importantly, achieving tissue FXN levels equivalent to more than 50% of those found in healthy volunteers means participants are at levels found in asymptomatic carriers who do not develop the disease,” said Carole Ben-Maimon, MD, President and Chief Executive Officer of Larimar. “Long-term treatment with daily nomlabofusp, including 8 participants for over 1 year, was generally well-tolerated. Through the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program, Larimar has regularly updated the FDA regarding all aspects of the clinical development program, including the data presented in this press release. We continue to hear strong interest in the nomlabofusp clinical program directly from patients with FA and their parents. The long-term clinical data presented today reinforce our conviction in the potential of nomlabofusp to address the root cause of FA and be the first potential disease modifying therapy.”
Safety: seven OL participants experienced anaphylaxis, most on the initial day and all within the first six weeks; all recovered with standard treatment. Other common adverse events were mild-to-moderate injection-site reactions. Following the two most recent anaphylaxis cases, Larimar consulted experts and modified the starting regimen: a 5 mg test dose, then a 25 mg dose one hour later under observation; 25 mg once daily through Day 30; then 50 mg once daily thereafter. The FDA agreed with the approach.
PK: rapid absorption after subcutaneous dosing; exposure reached steady state by Day 30 at 25 mg and 50 mg, consistent with Phase 1/2 data. In an adolescent PK run-in (ages 12–17), exposure on a weight-based 50 mg equivalent for seven days (n=14, 5 placebo) was similar to adults on 50 mg; six adolescents are currently enrolled in the OL study.
Program updates and timeline: the OL protocol now includes adolescents and adults without prior nomlabofusp exposure, with plans to enroll children ages 2–11 directly into the OL study. Global Phase 3 sites have been identified in the U.S., Europe, U.K., Canada, and Australia. On CMC, Larimar and FDA aligned on analytical testing, including potency; process performance qualification of commercial-scale drug substance is planned for Q4 2025, with material expected to support initial launch supply. Key milestones: implement the new dosing regimen in Q4 2025; complete PPQ in Q4 2025; submit BLA seeking accelerated approval in Q2 2026.
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