HT-KIT / Hoth Therap, NC State University - LARVOL DELTA

Hoth Therapeutics Reports Positive Preclinical Safety Data for Cancer Fighting HT-KIT -- Dose-Dependent Liver Activity with No Observed Toxicity Supports IND Pathway (PRNewswire) - "Key Preclinical Findings (HT-KIT): Liver weight increased from 1.11g at 0 mg/kg to 1.32g at 3.0 mg/kg, consistent with pharmacological engagement; Kidney and spleen weights remained stable, indicating no off-target or systemic toxicity; Thymus and other critical organ weights were within normal range across all groups; No gross pathology or visible lesions observed in any treated animal....Hoth Therapeutics expects to initiate GLP toxicology studies, with plans to submit an Investigational New Drug (IND) application soon after."

IND • Preclinical • Hepatocellular Cancer

Hoth Therapeutics Secures Japanese Patent for Breakthrough HT-KIT Platform Targeting Mast Cell Diseases (PRNewswire) - "Hoth Therapeutics...announced that they have obtained Japan Country Patent No. 7677628 for the HT-KIT platform. The patent was officially granted by the Japan Patent Office on May 7, 2025, and exclusive protection is granted until August 27, 2039. The granted patent, titled 'Targeting KIT with Splice Switching Oligonucleotides to Induce Apoptosis of Mast Cells,' protects Hoth's novel approach of using splice-switching oligonucleotides (SSOs) to selectively disrupt KIT gene expression in mast cells-cells implicated in conditions ranging from chronic hives to rare cancers....Applicable to mast cell-driven inflammatory and oncologic conditions."

Patent • Inflammation • Oncology

Hoth Therapeutics Reports Positive Preclinical Results for HT-KIT, a Precision Antisense Therapy Targeting Rare and Aggressive KIT-Driven Cancers (PRNewswire) - "Hoth Therapeutics, Inc...announced compelling preclinical data for HT-KIT, its proprietary antisense oligonucleotide (ASO) therapeutic designed to target and silence aberrant KIT gene expression—implicated in a variety of rare, treatment-resistant cancers....Over 80% reduction in KIT expression in vitro using cancer cell lines harboring activating KIT mutations. Significant tumor growth in GIST and mast cell tumor animal models following systemic administration of HT-KIT. No observable off-target toxicity in liver, kidney, or bone marrow, suggesting a favorable safety profile....Hoth Therapeutics expects to file an Investigational New Drug (IND) application with the FDA in early 2026, with first-in-human Phase 1 trials planned shortly thereafter."

IND • Preclinical • Gastrointestinal Stromal Tumor

Hoth Therapeutics Advances Cancer Fighting HT-KIT Program with New Patent Filing and Preclinical Study Initiation (PRNewswire) - "Hoth Therapeutics, Inc...announced two major developments in the ongoing advancement of its proprietary antisense oligonucleotide (ASO) cancer fighting drug candidate, HT-KIT. The Company has filed amended claims with the U.S. Patent and Trademark Office for its lead ASO technology targeting MS4A6A and FcεRIβ—genes implicated in allergic inflammation and mast cell-related diseases. These refined claims enhance intellectual property protection for HT-KIT and further position it as a novel therapeutic platform with broad clinical potential, including use in treating conditions such as anaphylaxis, mastocytosis, and allergic asthma. In parallel, Hoth has initiated a GLP-compliant 4-week intravenous toxicity study with a 14-day recovery period in C57BL/6 mice. This preclinical study, conducted in partnership with OnTargetx R&D Inc. and ITR Laboratories, is designed to evaluate the safety profile of HT-KIT in support of upcoming regulatory filings."

Patent • Preclinical • Immunology • Inflammation

Hoth Therapeutics Announces Positive Preclinical Results for HT-KIT in Aggressive Cancer Gastrointestinal Stromal Tumors (GIST). HT-KIT Triggered Significant Tumor Cell Death as Early as 24 Hours Post-treatment (PRNewswire) - Key Findings from the Preclinical Study: Significant Reduction in KIT Expression - HT-KIT effectively reduced KIT receptor expression within 24 hours, with effects sustained for 72 hours. Induction of Tumor Cell Death - HT-KIT triggered significant tumor cell death as early as 24 hours post-treatment, while the lower dose led to delayed but substantial cell death at 72 hours. Decreased Tumor Cell Proliferation - HT-KIT treatment inhibited cell growth and proliferation in GIST-T1 cells, as confirmed by cell count reductions and decreased fluorescence intensity in proliferation assays....The company is currently conducting additional preclinical studies to further validate HT-KIT's efficacy and safety profile, with plans to initiate regulatory discussions for first-in-human trials."

Preclinical • Gastrointestinal Stromal Tumor

Hoth Therapeutics Partners with OnTargetx R&D to Advance Research for Cancer fighting HT-KIT Cancer Therapeutic (PRNewswire) - "Hoth Therapeutics...announced its collaboration with OnTargetx R&D Inc. to advance research for its cancer-fighting therapeutic, HT-KIT. This immunohistochemistry study, conducted in partnership with Charles River Laboratories Montreal ULC, represents a key step in the preclinical development of HT-KIT....The study focuses on: Development and optimization of staining methods for (c-Kit) markers, a critical target in HT-KIT therapeutic mechanism; Processing and qualitative evaluation of tissue samples by a board-certified pathologist to determine the presence of markers; Comprehensive reporting and insights to support the continued development of HT-KIT."

Commercial • Oncology

Hoth Therapeutics Announces Update on Cancer Therapeutic HT-KIT Results Suggesting the Treatment Effectively Inhibited Tumor Growth in Preclinical Treatment Study (PRNewswire) - "Hoth Therapeutics...is excited to announce highly promising early data from a preclinical study of its latest cancer treatment. The preliminary results demonstrate that the treatment successfully stabilizes tumor growth with remarkable consistency across subjects, showing potential as an effective therapeutic option in oncology....Tumor volume measurements in the treated group exhibited minimal changes, suggesting that the treatment effectively inhibited tumor growth. Additionally, all tumors in the treated animals displayed remarkable consistency in volume, with standard deviation error bars barely visible-a strong indication of the treatment's uniformity and reproducibility in suppressing tumor growth."

Preclinical • Oncology

Hoth Therapeutics and Aronnax Inc. Enter Master Services Agreement For HT-KIT Cancer Therapeutic (PRNewswire) - "Hoth Therapeutics, Inc...today announced that it has entered into a Master Services Agreement with Aronnax, Inc. for its HT-KIT cancer therapeutic....Aronnax will oversee the third-party provider, ITR Laboratories, conducting intravenous injection using increasing/decreasing doses for each subsequent group. A timeframe of forty-eight hours will be allowed between each dose group. This study will provide Hoth key metrics in both max dose and range finding elements which will help formulate its proposed clinical trial."

Licensing / partnership • Acute Myelogenous Leukemia • Gastrointestinal Stromal Tumor • Oncology

Hoth Therapeutics Engages Altasciences to Perform Key Study for HT-KIT Cancer Fighting Therapeutic (PRNewswire) - "Hoth Therapeutics, Inc...announced today that it has completed bioanalytical method development under its prior agreement with Altasciences and plans to further engage Altasciences to perform a key study of mouse blood and plasma analysis from samples collected in Hoth's sponsored scientific research conducted at NC State University. The blood and plasma samples collected are part of a dose response study in a humanized mouse model. The upcoming study will use the newly validated bioanalytical method developed at Altasciences to test the amount of HT-KIT from the dose response study and help determine how long HT-KIT remains in the blood stream post dosing."

Preclinical • Oncology

Hoth Therapeutics Announces it Has Successfully Completed Manufacturing of its Cancer fighting HT-KIT for IND-Enabling Studies (PRNewswire) - "Hoth Therapeutics, Inc...today announced it has completed the manufacturing of its drug substance HT-KIT using good laboratory practice (GLP). HT-KIT is an antisense oligonucleotide that targets the proto-oncogene cKIT by inducing mRNA frame shifting....Hoth has now successfully completed manufacturing of the HT-KIT drug substance in collaboration with WuXi STA Pharmaceutical. Hoth plans to use the GLP drug substance in its upcoming pre-clinical studies required for its investigational new drug (IND) submission."

Commercial • Oncology

Hoth Therapeutics Announces Successful Completion of Pre-IND Meeting with FDA Regarding Development of Hoth's Cancer Fighting HT-KIT (PRNewswire) - "Hoth Therapeutics, Inc...today announced today the successful completion of a pre-IND meeting with the U.S. Food and Drug Administration (FDA) regarding the Company's development plan for its HT-KIT therapeutic. The Pre-IND meeting was completed through written responses provided by the FDA Nonmalignant Hematology Division of Regulatory Operations for Cardiology, Hematology, Endocrinology, and Nephrology...The purpose of the requested meeting was to gain the Agency's agreement on the suitability the 505(b)(1) regulatory pathway for approval of HT-KIT; adequacy of referenced nonclinical information and the proposed drug development plan to support opening the IND; and to discuss any filing issues the Agency may have with the proposed product....Based on the FDA's feedback, Hoth intends to advance its IND-enabling activities for HT-KIT as planned."

FDA event • Oncology

Hoth Therapeutics Announces Positive Results Killing Cancer Cells in Both Gastrointestinal Stromal Tumors and Acute Myeloid Leukemia (PRNewswire) - "Hoth Therapeutics...announced that HT-KIT...achieved positive preclinical results in a study sponsored by Hoth....HT-KIT effectively kills human mast cells that rely on signaling through the KIT receptor to survive. The effects of a single dose of HT-KIT lasted for about 2 weeks. HT-KIT efficacy at reducing KIT expression lasted for 7 days....HT-KIT potently reduced KIT expression using GIST cells and that GIST cells rapidly died between 48 and 72 hours after exposure to HT-KIT....KIT expression was lower in the AML cells compared to either the mastocytosis or GIST cells, but was effectively and dose-dependently reduced with HT-KIT treatment over 72 hours."

Preclinical • Acute Myelogenous Leukemia • Gastrointestinal Stromal Tumor

FDA Grants Hoth Therapeutics Pre-IND Meeting for HT-KIT, Treatment for Cancer Patients (PRNewswire) - "Hoth Therapeutics, Inc...today announced the Pre-IND meeting requested on September 8th for HT-KIT with the U.S. Food and Drug Administration (FDA) has been granted to receive written responses only from the FDA that are targeted for delivery on November 7, 2023...The Pre-IND meeting topics include the acceptability of the overall proposed drug development program for HT-KIT including requirements for nonclinical, clinical pharmacology, clinical, chemistry, and manufacturing controls. The proposed clinical trial designs for the first clinical studies in the HT-KIT program are also included in the Pre-IND meeting topics to receive FDA feedback.....'Receiving feedback from the FDA in November positions us well to achieve our planned IND-enabling development activities on time with the eventual goal of initiating the first HT-KIT clinical trial in patients.'"

FDA event • Oncology

Hoth Therapeutics Announces FDA Accepts Pre-Investigational New Drug (Pre-IND) Submission for HT-KIT, Treatment for Cancer Patients (PRNewswire) - "Hoth Therapeutics, Inc...today announced 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)...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)."

FDA event • Aggressive Systemic Mastocytosis • Oncology

Hoth Therapeutics mRNA Frame-Shifting Therapeutic, HT-KIT, Proves Effective Against Aggressive Cancer Cells (PRNewswire) - "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."

Preclinical • Hematological Malignancies • Leukemia • Oncology

Hoth Therapeutics Announces it Has Successfully Completed Manufacturing Feasibility of the HT-KIT Drug Substance (PRNewswire) - "Hoth has successfully completed manufacturing feasibility of the HT-KIT drug substance in collaboration with WuXi STA. Hoth plans to pursue a Pre-IND meeting with FDA by the end of 2022."

Commercial • FDA event • Aggressive Systemic Mastocytosis • Oncology

Hoth Therapeutics Announces Orphan Drug Designation Granted to Cancer Therapeutic HT-KIT to Treat Mastocytosis (PRNewswire) - "Hoth Therapeutics...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."

Orphan drug • Aggressive Systemic Mastocytosis • Oncology

Hoth Therapeutics Announces Submission of Orphan Drug Designation Request for HT-KIT to Treat Mastocytosis (PRNewswire) - "Hoth Therapeutics, Inc...announced it submitted an Orphan Drug Designation Request to the US Food and Drug Administration (FDA) for HT-KIT for the treatment of mastocytosis....HT-KIT is an antisense oligonucleotide that targets the proto-oncogene cKIT by inducing mRNA frame shifting, resulting in apoptosis of neoplastic mast cells. The KIT signaling pathway is implicated in multiple diseases, including all types of mastocytosis (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 anaphylaxis."

Orphan drug • Acute Myelogenous Leukemia • Aggressive Systemic Mastocytosis • Hematological Malignancies • Leukemia • Mast Cell Leukemia • Oncology

Hoth Therapeutics Announces a Sponsored Research Agreement to Further Develop Novel mRNA Cancer Therapeutic HT-KIT (PRNewswire) - "Hoth Therapeutics, Inc...announced that it has signed a Sponsored Research Agreement with North Carolina State University ('NC State') to support the continued research and development of HT-KIT, a novel therapeutic for the treatment mast cell cancers....In addition, the research will expand therapeutic potential of HT-KIT for the treatment of other cancers where aberrant cKIT signaling contributes to the cancer progression, such as gastrointestinal stromal tumors (GIST) and acute myeloid leukemia (AML)."

Licensing / partnership • Acute Myelogenous Leukemia • Gastrointestinal Cancer • Gastrointestinal Stromal Tumor • Hematological Malignancies • Leukemia • Mast Cell Leukemia • Oncology

Hoth Therapeutics Inks API and Drug Product Contracts with WuXi STA to Advance Manufacturing of HT-KIT Cancer Therapeutic (PRNewswire) - "Hooth Therapeutics...announced today that it has entered into two contracts with STA Pharmaceutical, a subsidiary of WuXi AppTec (WuXi STA), for process development and manufacturing of the active pharmaceutical ingredient (API) and formulation of drug product for the continued development and commercialization of HT-KIT....The agreements between Hoth Therapeutics and WuXi STA are expected to accelerate the development of this new modality therapy with rapid and efficient PMO manufacturing scale-up from feasibility batches to GMP material....After completion of formulation development, Hoth intends to pursue a Pre-IND meeting with the US FDA."

Commercial • FDA event • Licensing / partnership • Aggressive Systemic Mastocytosis • Hematological Malignancies • Leukemia • Mast Cell Leukemia • Oncology

Hoth Therapeutics' mRNA 'Frameshifting' Therapy HT-KIT for Mast Cell Cancers Reduces Size, Spread - Research Published in Molecular Therapy (PRNewswire) - "Hoth Therapeutics...highlights recent findings from North Carolina State University, that its new treatment for mast cell cancers, known as HT-KIT, reduces the number of mast cells by "mutating" the messenger RNA (mRNA) before it can deliver instructions for manufacturing the gene responsible for cell proliferation...In a mouse model, tumor growth and infiltration of other organs were reduced, and tumor cell death increased when the frameshifted c-KIT mRNA was induced."

Preclinical • Oncology

Hoth Therapeutics' mRNA 'Frameshifting' Therapy HT-KIT for Mast Cell Cancers Reduces Size, Spread - Research Published in Molecular Therapy (PRNewswire) - "Hoth Therapeutics, Inc...highlights recent findings from North Carolina State University, that its new treatment for mast cell cancers, known as HT-KIT, reduces the number of mast cells by 'mutating' the messenger RNA (mRNA) before it can deliver instructions for manufacturing the gene responsible for cell proliferation....The cancer cells stopped proliferating and began dying within hours. In a mouse model, tumor growth and infiltration of other organs were reduced, and tumor cell death increased when the frameshifted c-KIT mRNA was induced."

Preclinical • Oncology

Hoth Therapeutics Shares Positive Preclinical Results of Novel HT-KIT Therapeutic (PRNewswire) - "The preclinical animal trials were conducted using humanized mast cell neoplasm models, representative in vitro and in vivo models for aggressive, mast cell-derived cancers, such as mast cell leukemia and mast cell sarcoma. Key findings of the mouse models found that HT-KIT: Reduces KIT expression and induces apoptosis of neoplastic human mast cells, Inhibits tumor growth in humanized xenograft mast cell neoplasia model, Reduces liver infiltration of neoplastic mast cells, Reduces tumor growth in isograft models of mast cell neoplasia."

Preclinical • Hematological Malignancies • Mast Cell Leukemia • Oncology

Hoth Therapeutics Plans Shareholder Presentation on HT-KIT Therapeutic’s Preclinical Results for Rare Cancers (PRNewswire) - “Hoth Therapeutics, Inc…announced today plans to host a virtual information session on its pipeline product, HT-KIT…on Thursday, June 3rd for interested shareholders…HT-KIT exhibited highly positive results in humanized mast cell neoplasm models, representative in vitro and in vivo models for aggressive, mast cell-derived cancers such as mast cell leukemia and mast cell sarcoma…Furthermore, systemic administration of the anti-cancer therapeutic in an in vivo humanized mast cell neoplasm model showed a significant reduction in tumors and neoplastic mast cell infiltration from the therapeutic group compared to the vehicle control group. There were no obvious signs of toxicity in the study animals, suggesting that the novel anti-cancer therapeutic was well-tolerated…Stefanie will also provide a brief overview of the studies that are slated to initiate in Q2 2021.”

Preclinical • Oncology

Hoth Therapeutics Announces Participation at the American Society of Clinical Oncology (ASCO) Annual Meeting (PRNewswire) - " Hoth Therapeutics, Inc...announced...that it will be an exhibitor at the American Society of Clinical Oncology (ASCO) Annual Meeting. Attendees will be able to visit the Hoth Therapeutics booth virtually starting May 24th through July 6, 2021. Hoth Therapeutics is dedicated to helping address unmet medical needs and improve the quality of life for patients living with rare cancers through the development of its assets, HT-001 and HT-KIT."

Clinical • Oncology