Hoth Therapeutics Announces Positive Preclinical Results for HT-KIT Targeting Rare KIT-Driven Cancers

Hoth Therapeutics, a biopharmaceutical company known for its innovative approaches to addressing unmet medical needs, has announced promising preclinical outcomes for its new therapeutic, HT-KIT. This treatment is a proprietary antisense oligonucleotide (ASO) designed to target and silence the KIT gene, which is often implicated in rare and treatment-resistant cancers.

The company highlights that HT-KIT has been engineered to specifically bind to mutant KIT mRNA transcripts, thereby blocking their translation. This action prevents the production of the KIT protein, a crucial element in the growth of tumors associated with cancers such as gastrointestinal stromal tumors (GIST), systemic mastocytosis, and some acute leukemias.

In preclinical trials, HT-KIT has shown a dramatic ability to suppress tumor growth. Using cancer cell lines with activating KIT mutations, the treatment achieved over an 80% reduction in KIT expression in vitro. In animal models, especially those simulating GIST and mast cell tumors, HT-KIT significantly inhibited tumor growth when administered systemically. Importantly, there were no signs of off-target toxicity in critical organs such as the liver, kidneys, or bone marrow, which suggests a favorable safety profile for the drug.

Robb Knie, CEO of Hoth Therapeutics, expressed optimism about HT-KIT’s potential, describing it as a pioneering approach at the genetic level to treat cancers driven by KIT mutations. The company is preparing for an Investigational New Drug (IND) application, anticipated for early 2026, with the intent to begin human trials soon after. This move could pave the way for first-in-human Phase 1 trials, marking a significant milestone in HT-KIT’s development journey.

Current treatments for cancers driven by KIT mutations typically involve tyrosine kinase inhibitors (TKIs). However, these can lead to challenges such as drug resistance and systemic side effects. HT-KIT offers a unique avenue by tackling the disease at the mRNA level, potentially circumventing the resistance mechanisms that are common with small-molecule therapies.

Hoth Therapeutics is actively engaging with regulatory advisors and contract research partners to expedite the clinical development process. The company holds an exclusive license for the proprietary gene-silencing technology used in developing HT-KIT, aiming to overcome the resistance often observed in patients previously treated with TKIs.

Hoth Therapeutics, a clinical-stage biopharmaceutical firm, focuses on developing groundbreaking therapies to enhance patient quality of life. The company acts as a catalyst in early-stage pharmaceutical research and development, transitioning promising drugs from the laboratory to preclinical and clinical testing stages. By collaborating with scientists, clinicians, and leading experts, Hoth seeks to explore and develop therapeutics with the potential to revolutionize treatment options for various medical conditions. Through its patient-centric approach, the company is committed to diversifying treatment alternatives and achieving significant breakthroughs in the field of medicine.