Update from Larimar Therapeutics

January 23, 2025 — Larimar Therapeutics today announced that dosing of adolescents 12-17 years old has started in the nomlabofusp pediatric PK run-in study for patients with Friedreich’s ataxia (FA). Nomlabofusp is a protein replacement therapy designed to address the deficiency of frataxin (FXN) in FA.

Study participants in the PK run-in study are randomized 2:1 to receive either nomlabofusp or placebo daily for seven days. Following the assessment of safety and PK data of each successive cohort, participants will be eligible to screen for the OLE study. Larimar plans to initiate a cohort of children (2- 11 years old) in the first half of 2025.

The OLE study is evaluating the safety and tolerability, PK, and FXN levels in buccal and skin cells, along with exploratory pharmacodynamic markers (lipid profiles and gene expression) and clinical outcome measures, following long-term subcutaneous administration of nomlabofusp.

Larimar expects long-term 50 mg data from adult OLE study and available adolescent PK run-in data in mid-2025.

Recruitment information for the adolescent and pediatric study of nomlabofusp can be found here: A Study to Assess Nomlabofusp in Adolescents and Children with Friedreich’s Ataxia

Clinical trials related to this update

Frataxin Replacement, Stabilizers, or Enhancers
Clinical Trial | Ages 2-17

A Study to Assess Nomlabofusp in Adolescents and Children with Friedreich’s Ataxia

Larimar Therapeutics
Phase I | In-person
Frataxin Replacement, Stabilizers, or Enhancers
Clinical Trial | Ages 18+

An Open Label Extension Study of CTI-1601 in Subjects With Friedreich’s Ataxia (Jive)

Larimar Therapeutics
Phase II | In-person