EXTENDED STABILITY STUDIES OF PTH-IA

Investigators

Abstract

1. Background PTH-IA is being evaluated as a potential treatment for Jansen Metaphyseal Chondrodysplasia (JMC), a rare disease of skeletal development and mineral ion homeostasis. The NCATS Therapeutical Development Branch (TDB) is supporting late-stage pre-clinical development toward an Investigational New Drug (IND) application and clinical testing of PTH-IA. A GMP batch of PTH-IA (lot1000044350) has been manufactured at Bachem under an expired contract (HHSN271201700009I/75N95020F00001) and being used to support preclinical toxicology and Phase I clinical studies. The contractor (Bachem) has performed 3-year stability studies (the last report date in August 2024) on the API. Because of the slow progress on the clinical trial, PTH-IA project team in TDB identified it is necessary to extend our current stability study on PTh-IA (lot 1000044350) for another 2 years to cover the estimated clinical trial period. PTH-IA peptide structure: H-Leu-Leu-His-Asp-Leu-dTrp-Lys-Ser-Ile-Gln-Asp-Leu-Arg-Arg-Arg-Phe-Trp-Leu-His-His-Leu-Ile-Ala-Glu-Ile-His-Thr-Ala-Glu-Tyr-NH2 or LLHDLXKSIQDLRRRFWLHHLIAEIHTAEY; X = dTrp The counter ion: acetate Rare Diseases