Hoth Therapeutics reports preclinical data for HT-KIT

Hoth Therapeutics (HOTH) announced compelling preclinical data for HT-KIT, its proprietary antisense oligonucleotide, or ASO, therapeutic designed to target and silence aberrant KIT gene expression-implicated in a variety of rare, treatment-resistant cancers. HT-KIT is engineered to selectively bind to mutant KIT mRNA transcripts and block their translation, thereby preventing the production of the KIT protein, a critical driver of tumor growth in cancers such as gastrointestinal stromal tumors, or GIST, systemic mastocytosis, and certain acute leukemias. Preclinical milestones include: Over 80% reduction in KIT expression in vitro using cancer cell lines harboring activating KIT mutations; Significant tumor growth in GIST and mast cell tumor animal models following systemic administration of HT-KIT; No observable off-target toxicity in liver, kidney, or bone marrow, suggesting a favorable safety profile. Hoth Therapeutics expects to file an Investigational New Drug, or IND, application with the FDA in early 2026, with first-in-human Phase 1 trials planned shortly thereafter. The company is actively engaging regulatory advisors and contract research partners to accelerate clinical development.