BALA CYNWYD, PA — Clinical-stage biotechnology company Larimar Therapeutics, Inc. (Nasdaq: LRMR) has announced the dosing of the first patient in an open label extension (OLE) study for its novel protein replacement therapy, nomlabofusp. Designed to treat Friedreich’s ataxia (FA), a rare genetic disease that causes difficulty walking, speech problems, and heart disease, nomlabofusp seeks to address the root cause of the condition by delivering a protein called frataxin to mitochondria, the energy-producing structures within cells.
The OLE study follows the successful completion of the company’s Phase 2 dose escalation study. It aims to evaluate the long-term safety profile of daily 25 mg subcutaneous injections of nomlabofusp, measure tissue frataxin levels, and provide preliminary data on patient or caregiver administration of the drug.
“We are pleased to further advance the nomlabofusp clinical program with the commencement of our OLE study,” said Carole Ben-Maimon, MD, President, and CEO of Larimar. “We expect to continue daily dosing throughout the study, based on our Phase 1 and Phase 2 findings.”
Patients who completed treatment in the recent Phase 2 dose exploration trial or a previous Phase 1 clinical trial of nomlabofusp could potentially be eligible for the OLE study.
In February, Larimar announced its intention to pursue frataxin as a potential novel surrogate endpoint for accelerated approval. Data from the OLE study, including frataxin levels, pharmacodynamics, clinical outcomes information, and safety data, along with additional nonclinical pharmacology information, will be used to support a potential Biologics License Application (BLA) submission for accelerated approval targeted for the second half of 2025.
The OLE study will also evaluate pharmacokinetics and exploratory pharmacodynamic markers, such as lipid profiles and gene expression data, following long-term subcutaneous administration of nomlabofusp. Clinical measures collected during the trial will be compared to data from a synthetic control arm derived from participants in the Friedreich’s Ataxia Clinical Outcome Measures Study (FACOMS) database.
Initial data from the OLE study is expected in the fourth quarter of 2024. The company’s progress marks a significant step forward in the development of treatments for Friedreich’s ataxia.
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