Hoth Therapeutics, Inc. today announced a development updates for its novel cancer therapeutic, HT-KIT. Hoth’s innovative approach, which employs a chemically-stable antisense oligonucleotide to target the proto-oncogene KIT through by frameshifting KIT mRNA transcripts, has potential as a KIT-targeted therapeutic alone, or in combination with agents that target KIT signaling, in the treatment of KIT-associated malignancies.
Through a sponsored scientific research agreement with North Carolina State University, the team used the HT-KIT mRNA frame-shifting approach on mast cell leukemia cells in vitro and found that KIT protein expression, signaling and function were reduced. Treatment with HT-KIT prevented cancer cell growth and induced cell death over 72 hours. In a humanized mast cell leukemia mouse model, tumor growth and infiltration of other organs were reduced and tumor cell death increased when HT-KIT induced frameshifted c-KIT mRNA.
Hoth has filed several patent applications to protect this IP throughout the world.
“With our HT-KIT drug, we are flipping off a key cancer signal that’s involved in multiple aggressive cancers, such as systemic mastocytosis, mast cell leukemia, gastrointestinal stromal tumors and acute myeloid leukemia. Our approach avoids pitfalls related to KIT mutations by targeting mRNA. Our next round of preclinical studies are underway and we are excited to utilize the results for our planned Pre-IND meeting with FDA later this year,” stated Robb Knie, Chief Executive Officer of Hoth.
HT-KIT, a new molecular entity, was designated as an Orphan Drug for the treatment of mastocytosis earlier in 2022. HT-KIT Hoth has successfully completed manufacturing feasibility of the HT-KIT drug substance in collaboration with WuXi STA.
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.