Hoth Therapeutics’ HT-KIT granted FDA Orphan Drug Designation

Hoth Therapeutics (HOTH) highlighted FDA Orphan Drug Designation for HT-KIT and new preclinical data demonstrating greater than80% suppression of KIT expression and significant tumor-volume reduction by Day 8 in systemic mastocytosis and GIST models. HT-KIT, a precision antisense oligonucleotide – ASO – targeting KIT mRNA, also completed GLP-validated bioanalytical methods supporting IND-enabling studies; the Japan patent extends platform protection to 2039. KIT achieved greater than80% reduction of KIT mRNA/protein across in-vitro systems and in vivo models of systemic mastocytosis and GIST. In xenograft models, statistically significant tumor-volume reduction by Day 8 was observed, accompanied by apoptotic signaling consistent with KIT pathway knock-down. No dose-limiting toxicities were observed in the reported preclinical work to date. GLP-validated bioanalytical methods completed to support pharmacokinetic, biodistribution, and exposure-response analyses for IND. Planned next steps include complete GLP toxicology and CMC packages and submission of an IND. The company intends to initiate a Phase 1/2 dose-escalation/expansion study in advanced systemic mastocytosis and other KIT-driven tumors with translational biomarkers of target engagement and early efficacy readouts, and to continue regional IP expansion and evaluate strategic partnerships for development and commercialization.