Hoth Therapeutics announces ‘positive’ results for HT-KIT studies

Hoth Therapeutics announced that HT-KIT, a new molecular entity, for the treatment of advance systemic mastocytosis achieved positive preclinical results in a study sponsored by Hoth. HT-KIT is an antisense oligonucleotide that targets the proto-oncogene cKIT by inducing mRNA frame shifting and already has Orphan Drug Designation from the FDA. The sponsored preclinical research was conducted at NC State University over the past two years. The aim of the initial studies was to assess how long HT-KIT was functional in cancerous and non-cancerous mast cells, and to determine whether the cells could develop resistance to HT-KIT. Key findings from initial studies in the mouse model show: HT-KIT effectively kills human mast cells that rely on signaling through the KIT receptor to survive; The effects of a single dose of HT-KIT lasted for about 2 weeks; HT-KIT efficacy at reducing KIT expression lasted for 7 days. Overall, these data provide strong support for potential applications of HT-KIT outside of mastocytosis and mast cell leukemia where KIT may be expressed and could play a role in disease aggression. Further in vivo and mechanistic studies are underway at NCSU in these diseases.