Hoth Therapeutics(US:HOTH) Prnewswireยท2025-05-12 11:30Core Insights - Hoth Therapeutics has announced promising preclinical data for HT-KIT, an antisense oligonucleotide therapeutic targeting aberrant KIT gene expression linked to rare, treatment-resistant cancers [1][2] - HT-KIT is designed to selectively bind to mutant KIT mRNA transcripts, blocking their translation and preventing the production of the KIT protein, which drives tumor growth in various cancers [2][3] - The company plans to file an Investigational New Drug (IND) application with the FDA in early 2026, with Phase 1 human trials to follow shortly thereafter [4] Preclinical Data - HT-KIT has demonstrated over 80% reduction in KIT expression in vitro using cancer cell lines with activating KIT mutations [6][7] - Significant inhibition of tumor growth has been observed in preclinical models of gastrointestinal stromal tumors (GIST) and systemic mastocytosis following systemic administration of HT-KIT [6][7] - No observable off-target toxicity in liver, kidney, or bone marrow suggests a favorable safety profile for HT-KIT [6][7] Treatment Advantages - Current treatment options for KIT-driven cancers often rely on tyrosine kinase inhibitors (TKIs), which can lead to drug resistance and systemic side effects [3] - HT-KIT offers a targeted alternative by addressing the disease at the mRNA level, potentially avoiding resistance mechanisms associated with small-molecule therapies [3][5] - The company believes HT-KIT represents a first-in-class approach to treating KIT-mutated cancers at the genetic level, providing hope for patients who have exhausted traditional therapies [2][5] Company Overview - Hoth Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative treatments to improve patient quality of life [6] - The company utilizes a patient-centric approach and collaborates with scientists and key opinion leaders to explore therapeutics with significant potential [8]