Hoth Therapeutics Inc (NASDAQ: HOTH), a US-based biopharmaceutical company developing innovative therapies for unmet medical needs, on Monday announced preclinical data for HT-KIT, its proprietary antisense oligonucleotide (ASO) therapeutic designed to target and silence aberrant KIT gene expression, implicated in a variety of rare, treatment-resistant cancers.
The company says that 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 tumour growth in cancers such as gastrointestinal stromal tumours (GIST), systemic mastocytosis, and certain acute leukaemias.
According to the company, the product's preclinical milestones included: over 80% reduction in KIT expression in vitro using cancer cell lines harbouring activating KIT mutations; significant inhibition of tumour growth in GIST and mast cell tumour animal models following systemic administration of HT-KIT; and no observable off-target toxicity in liver, kidney, or bone marrow, suggesting a favourable safety profile.
Hoth Therapeutics expects to file an Investigational New Drug (IND) application with the FDA in early 2026, followed by first-in-human Phase 1 trials. The company is actively engaging regulatory advisors and contract research partners to accelerate clinical development.
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