A Treatment for Patients with Jansens Metaphyseal Chondrodysplasia (JMC)

National Center For Advancing Translational Sciences

Investigators

Abstract

TRND scientists initiated a preclinical development campaign to advance the PTH-IA candidate to clinical evaluation. The team standardized a process to manufacture PTH-IA drug substance and has produced a clinical-grade (Good Manufacturing Practice, GMP) formulation for toxicology testing and clinical trials. In-use testing and stability studies have been performed on the formulated drug product, and Good Laboratory Practice (GLP) toxicology studies in adult and juvenile rats have been completed to assess its safety for adult and pediatric patients. The validation and qualification of pharmacokinetic and anti-drug antibody testing methods for measuring clinical samples has been completed. Additional method optimization and qualification efforts for neutralizing antibody testing are ongoing. Long term stability testing of the drug substance and the drug product is ongoing. The team prepared and submitted an Investigational New Drug (IND) application, which received clearance from the FDA to proceed with a Phase 1/2 clinical trial in adult patients. Additionally, Orphan Drug and Rare Pediatric Disease Designations were granted by the FDA for PTH-IA and JMC, respectively. A natural history study in JMC patients is currently ongoing at the NIH Clinical Center, and preparations for the first-in-human clinical trial, planned to begin in late 2025 at the NIH CC, are underway.