Prnewswire·2025-10-21 12:23Core Insights - Hoth Therapeutics, Inc. has received FDA Orphan Drug Designation for HT-KIT, a precision antisense oligonucleotide targeting KIT mRNA, demonstrating over 80% suppression of KIT expression and significant tumor-volume reduction in systemic mastocytosis and GIST models [1][3][5] - The company has completed GLP-validated bioanalytical methods to support IND-enabling studies, with a Japan Patent extending platform protection to 2039 [1][3] Preclinical Data - HT-KIT achieved over 80% reduction of KIT mRNA/protein in both in vitro and in vivo models, with significant tumor-volume reduction observed by Day 8 in xenograft models [7] - The preclinical studies reported no dose-limiting toxicities, indicating a favorable tolerability profile [7] Mechanism of Action - HT-KIT operates at the transcript level, silencing both mutant and wild-type KIT, which may help bypass resistance pathways and reduce off-target effects compared to small-molecule TKIs [3][5] Next Steps - The company plans to complete GLP toxicology and CMC packages, submit an IND, and initiate a Phase 1/2 dose-escalation/expansion study in advanced systemic mastocytosis and other KIT-driven tumors [6][8] - The study will include translational biomarkers of target engagement and early efficacy readouts [6][8] Company Overview - Hoth Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative treatments to improve patient quality of life, collaborating with scientists and clinicians to advance early-stage pharmaceutical research [9]