/C O R R E C T I O N -- Hoth Therapeutics, Inc./
Hoth Therapeutics (NASDAQ: HOTH) has announced positive preclinical results for HT-KIT, its antisense oligonucleotide therapy targeting KIT-driven cancers. The treatment demonstrated over 80% reduction in KIT expression in vitro and significant tumor growth inhibition in gastrointestinal stromal tumors (GIST) and mast cell tumor models.
Key achievements include successful targeting of mutant KIT mRNA transcripts with no observable off-target toxicity in liver, kidney, or bone marrow. The company plans to file an Investigational New Drug (IND) application with the FDA in early 2026, followed by Phase 1 trials. HT-KIT represents a potential alternative to current tyrosine kinase inhibitors (TKIs), addressing drug resistance concerns through its mRNA-level targeting approach.
Hoth Therapeutics (NASDAQ: HOTH) ha annunciato risultati preclinici positivi per HT-KIT, la sua terapia con oligonucleotidi antisenso mirata ai tumori guidati da KIT. Il trattamento ha mostrato una riduzione di oltre l'80% nell'espressione di KIT in vitro e una significativa inibizione della crescita tumorale nei modelli di tumori stromali gastrointestinali (GIST) e tumori delle mastcellule.
Tra i risultati principali vi è il successo nel colpire i trascritti mRNA mutanti di KIT senza tossicità off-target osservabile nel fegato, nei reni o nel midollo osseo. L'azienda prevede di presentare una domanda di Nuovo Farmaco Sperimentale (IND) alla FDA all'inizio del 2026, seguita da studi di Fase 1. HT-KIT rappresenta una possibile alternativa agli attuali inibitori della tirosina chinasi (TKI), affrontando il problema della resistenza ai farmaci grazie al suo approccio mirato a livello di mRNA.
Hoth Therapeutics (NASDAQ: HOTH) ha anunciado resultados preclínicos positivos para HT-KIT, su terapia con oligonucleótidos antisentido dirigida a cánceres impulsados por KIT. El tratamiento demostró una reducción de más del 80% en la expresión de KIT in vitro y una inhibición significativa del crecimiento tumoral en modelos de tumores del estroma gastrointestinal (GIST) y tumores de mastocitos.
Los logros clave incluyen el éxito en el direccionamiento de los transcritos mutantes de ARN mensajero de KIT sin toxicidad fuera del objetivo observable en hígado, riñón o médula ósea. La compañía planea presentar una solicitud de Nuevo Fármaco en Investigación (IND) ante la FDA a principios de 2026, seguida de ensayos de Fase 1. HT-KIT representa una alternativa potencial a los inhibidores actuales de tirosina quinasa (TKIs), abordando la resistencia a los medicamentos mediante su enfoque dirigido a nivel de ARN mensajero.
Hoth Therapeutics (NASDAQ: HOTH)는 KIT 주도 암을 표적으로 하는 안티센스 올리고뉴클레오티드 치료제 HT-KIT의 긍정적인 전임상 결과를 발표했습니다. 이 치료제는 시험관 내에서 KIT 발현을 80% 이상 감소시켰으며, 위장관 기질 종양(GIST) 및 비만 세포 종양 모델에서 종양 성장 억제를 크게 나타냈습니다.
주요 성과로는 간, 신장, 골수에서 관찰되는 비표적 독성 없이 돌연변이 KIT mRNA 전사체를 성공적으로 표적화한 점이 있습니다. 회사는 2026년 초에 FDA에 임상시험용 신약(IND) 신청서를 제출하고 1상 시험을 진행할 계획입니다. HT-KIT는 mRNA 수준에서 표적화하는 접근법을 통해 약물 내성 문제를 해결하며, 현재 티로신 키나제 억제제(TKI)에 대한 잠재적 대안으로 기대됩니다.
Hoth Therapeutics (NASDAQ : HOTH) a annoncé des résultats précliniques positifs pour HT-KIT, sa thérapie par oligonucléotides antisens ciblant les cancers dépendants de KIT. Le traitement a démontré une réduction de plus de 80 % de l'expression de KIT in vitro et une inhibition significative de la croissance tumorale dans des modèles de tumeurs stromales gastro-intestinales (GIST) et de tumeurs à mastocytes.
Les réalisations clés incluent le ciblage réussi des transcrits d'ARNm mutés de KIT sans toxicité hors cible observable au niveau du foie, des reins ou de la moelle osseuse. La société prévoit de déposer une demande d'Investigational New Drug (IND) auprès de la FDA au début de 2026, suivie d'essais de phase 1. HT-KIT représente une alternative potentielle aux inhibiteurs actuels de la tyrosine kinase (TKI), en répondant aux problèmes de résistance aux médicaments grâce à son approche ciblant l'ARNm.
Hoth Therapeutics (NASDAQ: HOTH) hat positive präklinische Ergebnisse für HT-KIT bekannt gegeben, seine Antisense-Oligonukleotid-Therapie, die auf KIT-getriebene Krebserkrankungen abzielt. Die Behandlung zeigte eine über 80%ige Reduktion der KIT-Expression in vitro und eine signifikante Hemmung des Tumorwachstums in Modellen gastrointestinaler stromaler Tumoren (GIST) und Mastzelltumoren.
Wichtige Erfolge umfassen die erfolgreiche Zielgerichtetheit auf mutierte KIT-mRNA-Transkripte ohne beobachtbare Off-Target-Toxizität in Leber, Niere oder Knochenmark. Das Unternehmen plant, Anfang 2026 einen Investigational New Drug (IND)-Antrag bei der FDA einzureichen, gefolgt von Phase-1-Studien. HT-KIT stellt eine potenzielle Alternative zu aktuellen Tyrosinkinase-Inhibitoren (TKIs) dar und adressiert Resistenzen durch seinen Ansatz, der auf mRNA-Ebene ansetzt.
- Over 80% reduction achieved in KIT expression during in vitro testing
- Significant tumor growth inhibition demonstrated in animal models
- No observable off-target toxicity in vital organs, suggesting favorable safety profile
- Novel approach targeting mRNA level could potentially avoid resistance mechanisms seen in current treatments
- IND filing not expected until early 2026, indicating a long pathway to potential commercialization
- Still in preclinical stage with no human trial data available
In the news release, Hoth Therapeutics Reports Positive Preclinical Results for HT-KIT, a Precision Antisense Therapy Targeting Rare and Aggressive KIT-Driven Cancers, issued 12-May-2025 by Hoth Therapeutics, Inc. over PR Newswire, we are advised by the company that the 3rd bullet section, 2nd bullet should read "Significant inhibition of tumor growth". The complete, corrected release follows:
Hoth Therapeutics Reports Positive Preclinical Results for HT-KIT, a Precision Antisense Therapy Targeting Rare and Aggressive KIT-Driven Cancers
- HT-KIT dramatically suppresses tumor growth in preclinical models of GIST and systemic mastocytosis.
- Treatment shows potent downregulation of oncogenic KIT mutations with no observed systemic toxicity.
- IND filing expected early 2026, opening pathway to first-in-human trials.
Preclinical Milestones:
- Over
80% reduction in KIT expression in vitro using cancer cell lines harboring activating KIT mutations. - Significant inhibition of 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.
HT-KIT is engineered to selectively bind to mutant KIT mRNA transcripts and block their translation, thereby preventing the production of the KIT protein, a critical driver of tumor growth in cancers such as gastrointestinal stromal tumors (GIST), systemic mastocytosis, and certain acute leukemias.
Preclinical Milestones:
- Over
80% reduction in KIT expression in vitro using cancer cell lines harboring activating KIT mutations. - Significant inhibition of 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.
"We believe HT-KIT represents a first-in-class approach to treating KIT-mutated cancers at the genetic level, offering hope for patients who have exhausted traditional therapies," said Robb Knie, CEO of Hoth Therapeutics. "The strength of our preclinical data positions HT-KIT as a powerful candidate for precision oncology. We are moving rapidly toward IND submission and are eager to begin human trials.
Current treatment options for KIT-driven cancers often rely on tyrosine kinase inhibitors (TKIs), which may lead to drug resistance or systemic side effects. HT-KIT offers a highly targeted alternative by attacking the disease at the mRNA level—upstream of protein expression—potentially avoiding the resistance mechanisms seen with small-molecule therapies.
Next Steps:
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. The company is actively engaging regulatory advisors and contract research partners to accelerate clinical development.
About HT-KIT
HT-KIT is a synthetic antisense oligonucleotide developed using proprietary gene-silencing technology licensed exclusively by Hoth. It is designed to inhibit KIT gene expression in tumors where KIT mutations are known oncogenic drivers. Preclinical studies suggest HT-KIT has the potential to overcome resistance seen in patients previously treated with TKIs.
About Hoth Therapeutics, Inc.
Hoth Therapeutics is a clinical-stage biopharmaceutical company dedicated to developing innovative, impactful, and ground-breaking treatments with a goal to improve patient quality of life. We are a catalyst in early-stage pharmaceutical research and development, elevating drugs from the bench to pre-clinical and clinical testing. Utilizing a patient-centric approach, we collaborate and partner with a team of scientists, clinicians, and key opinion leaders to seek out and investigate therapeutics that hold immense potential to create breakthroughs and diversify treatment options. To learn more, please visit https://ir.hoththerapeutics.com/ .
Forward-Looking Statement
This press release includes forward-looking statements based upon Hoth's current expectations, which may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws, and are subject to substantial risks, uncertainties, and assumptions. These statements concern Hoth's business strategies; the timing of regulatory submissions; the ability to obtain and maintain regulatory approval of existing product candidates and any other product candidates we may develop, and the labeling under any approval we may obtain; the timing and costs of clinical trials, and the timing and costs of other expenses; market acceptance of our products; the ultimate impact of any health epidemic, on our business, our clinical trials, our research programs, healthcare systems, or the global economy as a whole; our intellectual property; our reliance on third-party organizations; our competitive position; our industry environment; our anticipated financial and operating results, including anticipated sources of revenues; our assumptions regarding the size of the available market, benefits of our products, product pricing, and timing of product launches; management's expectation with respect to future acquisitions; statements regarding our goals, intentions, plans, and expectations, including the introduction of new products and markets; and our cash needs and financing plans. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. You should not place reliance on these forward-looking statements, which include words such as "could," "believe," "anticipate," "intend," "estimate," "expect," "may," "continue," "predict," "potential," "project" or similar terms, variations of such terms, or the negative of those terms. Although the company believes that the expectations reflected in the forward-looking statements are reasonable, the company cannot guarantee such outcomes. Hoth may not realize its expectations, and its beliefs may not prove correct. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, market conditions and the factors described in the section titled "Risk Factors" in Hoth's most recent Annual Report on Form 10-K and Hoth's other filings made with the U. S. Securities and Exchange Commission. All such statements speak only as of the date made. Consequently, forward-looking statements should be regarded solely as Hoth's current plans, estimates, and beliefs. Investors should not place undue reliance on forward-looking statements. Hoth cannot guarantee future results, events, levels of activity, performance, or achievements. Hoth does not undertake and specifically declines any obligation to update, republish, or revise any forward-looking statements to reflect new information, future events, or circumstances or to reflect the occurrences of unanticipated events, except as may be required by applicable law.
Investor Contact:
LR Advisors LLC Email: investorrelations@hoththerapeutics.com
www.hoththerapeutics.com
Phone: (678) 570-6791
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SOURCE Hoth Therapeutics, Inc.
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