Larimar Therapeutics Begins Dosing Adolescents in New Study
Introduction to the Pediatric Pharmacokinetic Study
Larimar Therapeutics, Inc. (Nasdaq: LRMR), a pioneering biotechnology company focused on addressing complex rare diseases, has commenced the dosing of adolescents aged 12 to 17 in its pediatric pharmacokinetic (PK) run-in study targeting Friedreich’s ataxia (FA). This innovative study is designed to assess the safety and effectiveness of their lead compound, nomlabofusp, in younger patients.
Details of the Dosing Process
The dosing protocol involves providing adolescents a weight-based dose equivalent to the adult dosage of 50 mg. By integrating the adolescent cohort into this study, Larimar aims to gather essential data that contributes to a better understanding of how nomlabofusp works in the pediatric population.
Eligibility and Future Plans
Participants who complete this pharmacokinetic run-in study may qualify to advance into the ongoing open label extension (OLE) study. This staged approach allows researchers to closely monitor both the safety and the pharmacokinetic profiles as they progress from adolescents to younger children, ages 2 to 11, with plans to initiate this second cohort in the first half of 2025.
Importance of the Study
Dr. Rusty Clayton, Chief Medical Officer of Larimar, emphasized the significance of this initial dosing phase. He stated, “This cohort will lead to valuable insights into the pharmacokinetics and safety of nomlabofusp in a younger demographic.” The results are anticipated to reinforce the transition of both adolescents and subsequently enrolled children into the OLE study, enabling further exploration of long-term efficacy and safety data.
Research Objectives and Outcomes
The objective of the OLE study is to determine the safety, tolerability, and pharmacokinetic parameters along with assessing FXN levels in buccal and skin cells. The study also aims to evaluate exploratory pharmacodynamic markers, including lipid profiles and gene expression, which are crucial in understanding the drug’s overall impact on patient health and well-being.
Future Data Expectations
Larimar anticipates sharing long-term 50 mg data from adult participants in the OLE study, alongside the findings from the adolescent cohort undergoing the PK run-in study. Such data is vital for stakeholders and families invested in treatments for Friedreich's Ataxia, potentially impacting future therapy options.
About Larimar Therapeutics
Larimar Therapeutics, Inc. (Nasdaq: LRMR), stands at the forefront of biopharmaceutical innovation, particularly for rare diseases characterized by deficient intracellular bioactive compounds. The company is dedicated to evolving and utilizing its sophisticated delivery platform to engineer and develop various fusion proteins targeting an array of challenging medical conditions.
Frequently Asked Questions
What is the age range for adolescents participating in the study?
The study focuses on adolescents aged 12 to 17 years old.
What are the primary objectives of the pediatric PK run-in study?
The main objectives are to evaluate the safety, tolerability, and pharmacokinetic profile of nomlabofusp in adolescents with Friedreich's ataxia.
When does Larimar plan to initiate the cohort for younger children?
The cohort of children aged 2-11 years is expected to begin in the first half of 2025.
What will happen to participants after they complete the PK run-in study?
Participants who complete the PK run-in will be eligible to progress to the ongoing open label extension (OLE) study.
What is nomlabofusp?
Nomlabofusp is Larimar's lead compound, being developed as a treatment option for Friedreich’s ataxia.
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