Hoth Therapeutics, Inc. (NASDAQ: HOTH), a patient-focused biopharmaceutical company, today announced the U.S. Food and Drug Administration (FDA) designated HT-KIT, a new molecular entity, as an Orphan Drug for the treatment of mastocytosis.
HT-KIT is an antisense oligonucleotide that targets the proto-oncogene cKIT by inducing mRNA frame shifting. Preclinical studies have demonstrated that HT-KIT induces apoptosis of neoplastic mast cells and reduces metastasis associated with aggressive mastocytosis.
“This designation is an important milestone for HT-KIT, paving the drug development pathway to reach patients in need of new therapies for treatment of mastocytosis,” said Stefanie Johns, Ph.D., Chief Scientific Officer of Hoth. “It is an honor to develop a novel therapeutic like HT-KIT, which provides a new mechanism of action to tackle serious conditions such as aggressive systemic mastocytosis (ASM), mast cell leukemia (MCL), and systemic mastocytosis with associated hematological neoplasm (SM-AHN), acute myeloid leukemia, gastrointestinal stromal tumors, and other cKIT-driven diseases. Hoth will leverage provisions under the Orphan Drug Act to efficiently advance the HT-KIT therapeutic into the clinic.”
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.
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. The HT-KIT drug is designed to more specifically target the receptor tyrosine kinase KIT in mast cells, which is required for the proliferation, survival and differentiation of bone marrow-derived hematopoietic stem cells. 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 prognosis.