Hoth Therapeutics (NASDAQ: HOTH) mRNA Frame-Shifting Therapeutic, HT-KIT, Proves Effective Against Aggressive Cancer Cells

Treatment with HT-KIT prevented cancer cell growth and induced death in neoplastic mast cells over 72 hours

HT-KIT inhibits tumor growth and systemic organ infiltration using both an allograft mastocytosis model and a humanized xenograft MC tumor model.

Orphan Drug status granted, successfully completed API manufacturing feasibility

NEW YORK, April 11, 2022 /PRNewswire/ — Hoth Therapeutics, Inc. (NASDAQ: HOTH), a patient-focused biopharmaceutical company, 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.

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.

About Hoth Therapeutics, Inc.

Hoth Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing new generation therapies for unmet medical needs. Hoth’s pipeline development is focused to improve the quality of life for patients suffering from skin toxicities associated with cancer therapy, mast-cell derived cancers and anaphylaxis, Alzheimer’s Disease, atopic dermatitis and other indications. To learn more, please visit https://ir.hoththerapeutics.com/.

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