On September 11, 2023 Hoth Therapeutics, Inc. (NASDAQ: HOTH), a patient-focused biopharmaceutical company, reported that it has submitted a request for a Pre-Investigational New Drug (IND) meeting to the U.S. Food and Drug Administration (FDA) to discuss the proposed drug development program for HT-KIT, a new molecular entity, for the treatment of advance systemic mastocytosis (AdvSM) (Press release, Hoth Therapeutics, SEP 11, 2023, View Source [SID1234635075]). 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.
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Currently in the preclinical stage of development of HT-KIT, Hoth anticipates a meeting with the FDA to be scheduled during the fourth quarter of 2023. During the pre-IND meeting with the FDA, Hoth Therapeutics plans to discuss the overall proposed drug development program for HT-KIT including requirements for nonclinical, clinical pharmacology, clinical, chemistry, and manufacturing controls. Hoth also plans to present clinical trial designs for the IND-opening, phase 1 dose ranging and dose extension studies as well as a proposed follow-up phase 2 safety and efficacy study; both studies will be conducted in adult patients with advanced systemic mastocytosis (AdvSM). AdvSM includes patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SMAHN), and mast cell leukemia (MCL).
"Today’s FDA submission brings us one step closer to advancing HT-KIT for patients who are suffering with a rare aggressive form of cancer," stated Robb Knie, Chairman and CEO of Hoth Therapeutics. "We look forward to working closely with the FDA and advancing HT-KIT through the IND-enabling and clinical phases of development."
About HT-KIT
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. 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.