Business
Larimar Therapeutics Announces Dosing of Adolescents in Nomlabofusp Pediatric Pharmacokinetic Run-In Study for Patients with Friedreich’s Ataxia
Adolescents receive a weight-based dose equivalent to the 50 mg adult dose Adolescents 12-17 years old who complete participation in the pharmacokinetic (PK)
About this update from Larimar Therapeutics, Inc.
[{"type":"text","content":"Adolescents receive a weight-based dose equivalent to the 50 mg adult dose Adolescents 12-17 years old who complete participation in the pharmacokinetic (PK) run-in study will be eligible to screen in the ongoing open label extension (OLE) study Larimar is continuing to enroll adolescent patients and plans to initiate a cohort of children 2-11 years old in 1H 2025 Long-term 50 mg data from adults in OLE study and available adolescent PK run-in data expected mid-2025 BALA CYNWYD, Pa., Jan. 23, 2025 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today announced that dosing of adolescents 12-17 years old has started in the Company’s pediatric PK run-in study for patients with Friedreich’s ataxia (FA). “Dosing adolescents is the first step in evaluating the safety and PK of nomlabofusp in pediatric patients with FA. We continue to enroll adolescents in our first cohort. This cohort will be followed by a second cohort of children 2-11 years old. We expect to transition both the adolescents and children into the ongoing OLE study after assessing safety and exposure data from each successive cohort,” said Dr. Rusty Clayton, Chief Medical Officer of Larimar. “We look forward to reporting long-term 50 mg data in adults from our OLE study, as well as available data from adolescents completing the pediatric PK run-in study, in mid- 2025.” Study participants in the PK run-in study are randomized 2:1 to receive either nomlabofusp or placebo daily for seven days. Following assessment of safety and PK data of each successive cohort, participants will be eligible to screen for the OLE study. A cohort of children (2- 11 years old) is planned to initiate 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. About Larimar TherapeuticsLarimar Therapeutics, Inc. (Nasdaq: LRMR), is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases. Larimar’s lead compound, nomlabofusp, is being developed as a potential treatment for Friedreich's at...