Hoth Therapeutics, Inc. announced that HT-KIT, a new molecular entity, for the treatment of advance systemic mastocytosis (AdvSM) achieved positive preclinical results in a study sponsored by Hoth. HT-KIT is anense 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 ("NCSU") 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. Further in vivo and mechanical studies are underway at NCSU in these diseases. HT-KIT is a new molecular entity (NME) under development for treatment of mast cell derived cancers and anaphylaxis.

HT-KIT was developed Dr. Glenn Cruse, Assistant Professor at North Carolina State University and shares the same molecular class as Hoth's current HT-004 drug. Mutations in the KIT pathway have been associated with several human cancers, such as gastrointestinal stromal tumors and mast cell-derived cancers (mast cell leukemia and mast cell sarcoma). Based on the initial proof-of-concept success, Hoth intends to initially target mast cell neoplasms for development of HT-KIT, which is a rare, aggressive cancer with poor proggnosis.

HT-KIT has Orphan Drug Designation From the FDA. FDA Orphan Drug Designation is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States. Orphan drug status provides benefits to drug developers, including assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of post-approval marketing exclusivity.