EXCLUSIVE: Hoth Therapeutics' Antisense Therapy Demonstrates Efficacy Against KIT-Driven Cancers

Hoth Therapeutics Inc. HOTH on Tuesday announced combined findings from multiple preclinical programs evaluating its precision antisense candidate 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.

Preclinical Efficacy & Safety Highlights

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.

Also Read: Why Is Hoth Therapeutics Stock Flying Higher On Tuesday?

In vitro, HT-KIT achieved over 80% knockdown of KIT expression, the oncogenic driver in multiple aggressive cancers.

Multi-dose in vivo studies confirmed no off-target toxicity across critical organs, including liver, kidneys, spleen, bone marrow, and thymus.

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., demonstrated that HT-KIT meets or exceeds strict bioanalytical benchmarks:

All calibration curve, quality control, and dilution integrity requirements passed with high reproducibility.

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.

No protocol or SOP deviations impacted study reliability.

Next Steps

Hoth expects to integrate this bioanalytical data into its formal GLP toxicology package and is preparing for an Investigational New Drug (IND) application.

In June, Silo Pharma Inc. SILO announced a non-binding letter of intent to form a strategic 50:50 joint venture with Hoth Therapeutics to develop and commercialize a potential treatment for obesity and metabolic disease based on technology Hoth has exclusively licensed from the U.S. Department of Veterans Affairs (VA) that was co-developed by the VA and Emory University.

The novel therapeutic platform centers on glial cell line-derived neurotrophic factor (GDNF), a VA-invented biologic with demonstrated anti-obesity effects and metabolic regulation in preclinical models.

The platform is protected under U.S. Patent No. 10,052,362 and targets multiple high-burden conditions, including non-alcoholic fatty liver disease (NAFLD), type 2 diabetes, and central obesity.

Price Action: At the last check on Tuesday, HOTH stock was up 9.48% at $1.26 during the premarket session.

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