Hoth Therapeutics is turning to AI to help move its investigational targeted antisense oligonucleotide HT-KIT closer to the clinic, announcing the deployment of the OpenAI API to support development of the orphan-designated cancer therapy.
The patient-focused biopharmaceutical company has integrated the AI platform across its preclinical workflow, using it to assist with data analysis, modeling of cancer pathways, and preparation of regulatory documentation ahead of Investigational New Drug (IND) submission. This reflects a broader industry shift, as AI tools are increasingly applied beyond discovery and into translational and regulatory stages.
HT-KIT, which has received orphan drug designation, targets rare and aggressive cancers driven by KIT mutations, including systemic mastocytosis and gastrointestinal stromal tumors — areas of significant unmet medical need. After IND filing, the candidate is planned to move into phase I clinical evaluation.
Preclinical findings suggest activity in rare cancers. In both in vitro and in vivo models of systemic mastocytosis and gastrointestinal stromal tumors, HT-KIT achieved more than 80 percent reduction in KIT mRNA and KIT protein expression. In xenograft studies, the therapy also demonstrated statistically significant tumor-volume reductions within eight days, alongside apoptotic signaling consistent with suppression of the KIT pathway.
Safety data so far appear favorable, with no dose-limiting toxicities reported in preclinical studies. The company has also completed Good Laboratory Practice–validated bioanalytical methods to enable pharmacokinetic, biodistribution, and exposure-response analyses — supporting preparation for IND submission.
For Hoth, the use of AI is framed as an addition to its development efforts. CEO Robb Knie said the integration “supports execution of our IND-enabling strategy [...] as we advance toward phase I.”
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