Hoth Therapeutics, Inc., a biopharmaceutical company focused on developing innovative therapies for unmet medical needs, recently announced promising preclinical data for HT-KIT, its proprietary antisense oligonucleotide (ASO) therapy aimed at targeting and silencing the aberrant expression of the KIT gene, which plays a role in various rare, treatment-resistant cancers.

HT-KIT is specifically engineered to bind to mutant KIT mRNA transcripts, blocking their translation and preventing the production of the KIT protein, a key driver of tumor growth in conditions like gastrointestinal stromal tumors (GIST), systemic mastocytosis, and certain acute leukemias.

Preclinical Milestones:

Over 80% reduction in KIT expression in vitro using cancer cell lines with activating KIT mutations.

Significant tumor growth inhibition in GIST and mast cell tumor animal models following systemic administration of HT-KIT.

No observed off-target toxicity in liver, kidney, or bone marrow, suggesting a favorable safety profile.

Robb Knie, CEO of Hoth Therapeutics, stated, "HT-KIT represents a pioneering approach to treating KIT-mutated cancers at the genetic level, providing new hope for patients who have exhausted conventional therapies. The robustness of our preclinical data positions HT-KIT as a strong candidate for precision oncology, and we are advancing swiftly toward IND submission with plans to begin human trials soon."

Current treatment options for KIT-driven cancers typically involve tyrosine kinase inhibitors (TKIs), which can result in drug resistance or systemic side effects. HT-KIT offers a more targeted alternative by addressing the disease at the mRNA level, potentially avoiding resistance mechanisms associated with small-molecule therapies.

Next Steps:
Hoth Therapeutics plans to submit an Investigational New Drug (IND) application to the FDA in early 2026, with first-in-human Phase 1 trials anticipated shortly after. The company is working closely with regulatory advisors and contract research partners to expedite clinical development.

Source: https://ir.hoththerapeutics.com