Larimar Therapeutics (NASDAQ: LRMR) has announced positive 25 mg and 50 mg data from the ongoing long-term open label (OL) study evaluating daily subcutaneous injections of nomlabofusp self-administered or administered by a caregiver in participants with Friedreich’s ataxia (FA).
According to Larimar, the study—evaluating the safety and tolerability, pharmacokinetics, and FXN levels in skin and buccal cells, along with exploratory pharmacodynamic markers (lipid profiles) and clinical outcomes following long-term subcutaneous administration of nomlabofusp—has been amended to include both adolescent and adult patients with FA. Its Biologics License Application submission seeking accelerated approval is targeted in Q2 2026.
In a statement, Carole Ben-Maimon, MD, president, and CEO of Larimar, commented, “We are excited to announce the consistent directional improvements across 4 key clinical outcomes observed in the OL study relative to a Friedrich’s Ataxia Clinical Outcomes Measure Study (FACOMS) reference population and the observed increase in FXN levels. These new data, as well as the improvement in abnormal lipid profiles observed in prior completed studies, provide support that nomlabofusp increases FXN in patients with FA and that the strategy of FXN replacement has the potential to result in a clinical benefit. 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.”
Dr. Ben-Maimon added, “Long-term treatment with daily nomlabofusp, including eight participants for over one 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.”
