On September 2, 2025 Hoth Therapeutics, Inc. (NASDAQ: HOTH), a clinical-stage biopharmaceutical innovator, reported combined positive findings from multiple preclinical programs evaluating its precision antisense candidate HT-KIT, including compelling anti-tumor efficacy, a clean safety profile, and new GLP-validated bioanalytical results that exceeded internationally recognized regulatory thresholds (Press release, Hoth Therapeutics, SEP 2, 2025, View Source [SID1234655665]).
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
Preclinical Efficacy & Safety Highlights
Rapid Tumor Kill: HT-KIT triggered significant tumor cell death in preclinical models of gastrointestinal stromal tumors (GIST) and systemic mastocytosis as early as 24 hours post-treatment, with statistically significant tumor shrinkage observed by day 8.
Strong KIT Suppression: In vitro, HT-KIT achieved over 80% knockdown of KIT expression, the oncogenic driver in multiple aggressive cancers.
Clean Safety Profile: Multi-dose in vivo studies confirmed no off-target toxicity across critical organs including liver, kidneys, spleen, bone marrow, and thymus.
Dose-Dependent Biological Signal: A preclinical safety study demonstrated proportional liver engagement (increase from 1.11g to 1.32g at 3.0 mg/kg) with zero gross pathology at any organ site.
GLP-Validated Bioanalytical Results
The study, conducted by Altasciences Company, Inc. under OECD, FDA, and EMA GLP standards, demonstrated that HT-KIT meets or exceeds strict bioanalytical benchmarks:
Regulatory-Grade Validation: All calibration curve, quality control, and dilution integrity requirements passed with high reproducibility.
Superior Data Integrity: 90.5% of Incurred Sample Reanalysis (ISR) values fell within ±30%, well above the 66.7% regulatory minimum.
Extended Stability: HT-KIT remained stable in serum for 37 days at -80°C, surpassing the validated 28-day stability period, with further studies ongoing.
Flawless Compliance: No protocol or SOP deviations impacted study reliability.
"These results combine a rare and powerful story — tumor kill within 24 hours, clean safety across all systems, and GLP-validated reproducibility beyond regulatory standards," said Robb Knie, CEO of Hoth Therapeutics. "We believe HT-KIT has the potential to transform outcomes in KIT-driven cancers, and these milestones accelerate our path toward IND submission and first-in-human trials."
Next Steps
Hoth expects to integrate this bioanalytical data into its formal GLP toxicology package and is preparing for an IND.
About HT-KIT
HT-KIT is a precision antisense oligonucleotide designed to silence mutant KIT mRNA, an oncogenic driver in gastrointestinal stromal tumors (GIST), systemic mastocytosis, and certain leukemias. By targeting KIT expression at the genetic level, HT-KIT seeks to overcome resistance to tyrosine kinase inhibitors while minimizing systemic side effects.