Hoth Therapeutics announced that the US Food and Drug Administration (FDA) has accepted their Preinvestigational New Drug submission for HT-KIT, which is designed for the treatment of advanced systemic mastocytosis (SM).

The submission, announced via news release, includes a request for a meeting with the FDA to discuss the development program for the new molecule.

“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.”

SM represents a group of rare diseases characterized by excessive proliferation and accumulation of mast cells in tissues and organs, including the bone marrow, liver, spleen, and gastrointestinal tract. The prognosis varies widely, but most patients die within 4 to 5 years following the diagnosis.

Read more about SM therapies

Most patients with SM have gain-of-function mutations in the KIT tyrosine kinase domain.

HT-KIT is an antisense oligonucleotide that targets mast cell neoplasms by inducing mRNA frame shifting in the proto-oncogene cKIT, thereby interfering with the proliferation, survival, and differentiation of bone marrow-derived hematopoietic stem cells. HT-KIT shares the same molecular class as HT-004, another Hoth Therapeutic drug.

HT-KIT already has US FDA Orphan Drug Designation, and at the upcoming meeting, Hoth will present clinical trial designs for Investigational New Drug opening, phase 1 dose ranging, and dose extension studies.

In addition, a phase 2 safety and efficacy study is planned for adult patients with advanced SM, which includes patients with aggressive systemic mastocytosis, systemic mastocytosis with an associated hematological neoplasm, and mast cell leukemia.


Hoth Therapeutics announces FDA accepts pre-investigational new drug (Pre-IND) submission for HT-KIT, treatment for cancer patients. News release. Hoth Therapeutics, Inc.; September 11, 2023.