Hoth Therapeutics Unveils Promising Results for Antisense Therapy
Hoth Therapeutics Inc. announced significant findings on March 12, 2024, regarding its precision antisense therapy candidate, HT-KIT. This innovative treatment aims to target and silence mutant KIT mRNA, which plays a critical role in various cancers, including gastrointestinal stromal tumors (GIST) and systemic mastocytosis.
Preclinical trials demonstrated that HT-KIT induced substantial tumor cell death within 24 hours in models of GIST and systemic mastocytosis. By day eight, researchers observed statistically significant tumor shrinkage, indicating the therapy’s rapid efficacy. In vitro studies revealed that HT-KIT achieved over 80% knockdown of KIT expression, addressing a crucial oncogenic driver in aggressive cancer types.
Safety and Efficacy Highlights
The safety profile of HT-KIT shows promising results. Multi-dose in vivo studies confirmed no off-target toxicity across vital organs, including the liver, kidneys, spleen, bone marrow, and thymus. A preclinical safety assessment indicated proportional liver engagement, with an increase from 1.11 grams to 1.32 grams at a dosage of 3.0 mg/kg, without revealing any gross pathology at any organ site.
According to Altasciences Company Inc., the study met or exceeded stringent bioanalytical benchmarks. All calibration curve, quality control, and dilution integrity requirements were satisfied with high reproducibility. Notably, 90.5% of Incurred Sample Reanalysis (ISR) values fell within ±30%, exceeding the regulatory minimum of 66.7%. Furthermore, HT-KIT demonstrated extended stability in serum, remaining viable for 37 days at -80°C, surpassing the validated 28-day stability period.
Future Directions and Collaborations
Hoth Therapeutics plans to integrate this bioanalytical data into its formal Good Laboratory Practice (GLP) toxicology package as it prepares for an Investigational New Drug (IND) application. Earlier in June 2023, Silo Pharma Inc. expressed interest in a strategic collaboration with Hoth to develop a treatment for obesity and metabolic disease. This potential joint venture is based on a technology Hoth exclusively licensed from the U.S. Department of Veterans Affairs, which was co-developed with Emory University.
The therapeutic platform focuses on glial cell line-derived neurotrophic factor (GDNF), a biologic with proven anti-obesity effects and metabolic regulation in preclinical models. This platform is protected under U.S. Patent No. 10,052,362 and aims to address various high-burden conditions, including non-alcoholic fatty liver disease (NAFLD), type 2 diabetes, and central obesity.
As of the latest trading session, Hoth Therapeutics’ stock was up by 9.48%, reaching $1.26 during premarket hours. The ongoing developments in HT-KIT’s research and its strategic partnerships position Hoth Therapeutics as a key player in advancing treatments for challenging medical conditions.
