- HT-KIT demonstrates over 80% reduction in KIT expression, a promising breakthrough in cancer treatment.
- Significant tumor growth inhibition observed in preclinical models of GIST and mast cell tumors.
- Hoth Therapeutics plans IND application for FDA approval in early 2026, moving toward human trials.
Hoth Therapeutics, Inc. (HOTH), a biopharmaceutical company focused on developing innovative medical solutions, has announced promising preclinical results for their antisense oligonucleotide therapy, HT-KIT. The treatment, targeting KIT-driven cancers, achieved an over 80% reduction in KIT expression in vitro, alongside significant tumor growth inhibition in models of gastrointestinal stromal tumors (GIST) and mast cell tumors.
The preclinical studies reported success in targeting mutant KIT mRNA transcripts without any observable off-target toxicity in vital organs like the liver, kidney, or bone marrow. This highlights HT-KIT's favorable safety profile as a potential alternative to existing tyrosine kinase inhibitors (TKIs), which face challenges like drug resistance. HT-KIT's novel approach at the mRNA level provides a strategic advantage, potentially avoiding the resistance mechanisms encountered by current small-molecule therapies.
Hoth Therapeutics has set plans to file for an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) by early 2026. Following the IND submission, the company aims to initiate Phase 1 human trials, marking a significant step forward in addressing rare and aggressive KIT-driven cancers, including GIST, systemic mastocytosis, and certain acute leukemias.
HT-KIT's promising preclinical results position it as a compelling candidate in precision oncology, offering a new hope for patients with treatment-resistant cancers. Hoth Therapeutics continues to actively engage with regulatory advisors and partners to accelerate the clinical development of this groundbreaking therapy.
