Hoth Therapeutics Unveils Promising Data for KIT-Targeting Therapy

Hoth Therapeutics Inc, a clinical stage biopharmaceutical company, also dedicated to novel medical needs, has reported very promising data of preclinical efficacy for HT-KIT, its proprietary ASO (antisense oligonucleotide) therapeutic. Specifically aimed to block abnormal KIT gene expression, HT-KIT has a promise of becoming a next-gen therapy for rare and drug-resistant cancers, such as gastrointestinal stromal tumors (GIST), systemic mastocytosis and specific acute leukemias.

HT-KIT is beneficial because it selectively attaches to mutant KIT mRNA transcripts and inhibits their translation preventing the synthesis of the KIT protein that is an essential component of the tumor progression. HT-KIT in preclinical experiments had more than 80% suppression of KIT expression in vitro in a range of cancer cell lines with the stable D816V activating mutation. Also, in animal models of GIST and mast cell tumors, systemic administration resulted in significant tumor growth inhibition with no toxicity in the organism, in liver, kidney, or bone marrow—which implies the favorable safety profile.

In most cases, the current treatment tends to be limited when encountering resistance and systemic effects or development. HT-KIT by addressing things at the genetic level, is a more targeted and possibly long-lasting way, by interfering with disease-causing pathways prior to protein synthesis.

“Our data strongly advocate HT KIT’s potential for being a first on class precision oncology drug”, said Robb Knie CEO HOTH Therapeutics. “We are constantly pushing our technology closer and closer to IND submission and will be ready for human trial soon after”.

 The company hopes to submit an Investigational New Drug (IND) application to FDA in the first quarter of 2026 and is engaging regulatory experts and research co-leaders to expedite clinical development.