Hoth Therapeutics announced that its precision oncology candidate HT-KIT has received Orphan Drug Designation from the FDA for the treatment of rare c-KIT-driven malignancies, including systemic mastocytosis and gastrointestinal stromal tumors (GIST). The company also reported compelling preclinical data demonstrating potent gene-level activity and tumor reduction effects.
In multiple preclinical studies, HT-KIT, an antisense oligonucleotide designed to silence the KIT gene, achieved more than 80% suppression of KIT mRNA and protein expression. The therapy produced statistically significant tumor-volume reductions by Day 8 in xenograft models of systemic mastocytosis and GIST. No dose-limiting toxicities or off-target effects were observed, and GLP-validated bioanalytical assays have been completed in preparation for Investigational New Drug (IND) submission.
Patent protection for HT-KIT was strengthened with the issuance of Japan Patent No. 7677628, extending intellectual property coverage through 2039. The company plans to finalize GLP toxicology and chemistry, manufacturing, and controls (CMC) packages before submitting its IND filing. A Phase 1/2 dose-escalation and expansion trial is planned to evaluate HT-KIT in patients with advanced KIT-driven cancers, utilizing translational biomarkers of target engagement and early efficacy endpoints.
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