BALA CYNWYD, PA — Larimar Therapeutics, Inc. (Nasdaq: LRMR) has begun dosing adolescents aged 12-17 in its pediatric pharmacokinetic (PK) run-in study for Friedreich’s ataxia (FA), marking an important step in expanding the clinical evaluation of its investigational therapy, nomlabofusp, to younger patients.
Adolescents are receiving a weight-adjusted dose equivalent to the 50 mg dose used in adults. Upon completing the PK run-in study, eligible participants will transition into Larimar’s ongoing open-label extension (OLE) study, which evaluates safety, tolerability, and other measures under long-term subcutaneous administration.
Larimar intends to expand the study to include children aged 2-11 in the first half of 2025, forming a second cohort after collecting safety and PK data from the adolescent group. Interim findings from the adolescent PK run-in and long-term adult OLE data are expected to be reported in mid-2025.
The PK run-in study incorporates a randomized 2:1 design, where participants receive either nomlabofusp or placebo daily over seven days. Data from each cohort will be used to ensure safety, guiding transitions into the OLE study. The OLE study will investigate pharmacodynamic markers, including FXN levels in buccal and skin cells and exploratory indicators such as lipid profiles, gene expression, and clinical outcomes.
“We’re encouraged by this step toward understanding nomlabofusp’s potential in pediatric patients. This progression sets the stage for evaluating safety and exposure among younger age groups,” said Dr. Rusty Clayton, Chief Medical Officer of Larimar Therapeutics.
With its ongoing research, Larimar Therapeutics aims to advance therapeutic options for patients with Friedreich’s ataxia, expanding treatment opportunities to younger populations in the pursuit of improved outcomes.
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