Hoth Therapeutics Reports Positive Preclinical Results for HT-KIT, a Precision Antisense Therapy Targeting Rare and Aggressive KIT-Driven Cancers
- Over 80% reduction in KIT expression achieved in preclinical trials
- No observable toxicity in liver, kidney, or bone marrow
- Novel treatment approach potentially avoiding resistance mechanisms seen in current therapies
- Clear pathway to IND filing in early 2026
- Still in early preclinical stage with human trials not starting until 2026
- No revenue generation expected in the near term
- Success in preclinical trials doesn't guarantee clinical trial effectiveness
Insights
Hoth's HT-KIT antisense therapy shows promising preclinical results against KIT-driven cancers with 80% KIT reduction and minimal toxicity.
The preclinical data for Hoth Therapeutics' HT-KIT represents a significant advancement in the treatment landscape for KIT-driven cancers. The reported 80% reduction in KIT expression in vitro is particularly noteworthy, as KIT mutations are fundamental drivers in GIST and systemic mastocytosis pathogenesis. These cancers often develop resistance to traditional tyrosine kinase inhibitors (TKIs) like imatinib and sunitinib.
What makes this approach scientifically distinctive is that HT-KIT targets the disease upstream at the mRNA level rather than at the protein level like conventional TKIs. This mechanism potentially circumvents the common resistance pathways that develop with protein-targeting approaches. By preventing the translation of KIT mRNA transcripts, HT-KIT addresses the root cause of these cancers before protein synthesis occurs.
The absence of observable toxicity in critical organs including liver, kidney, and bone marrow suggests a favorable safety profile compared to current standard-of-care TKIs, which often cause significant adverse effects like hepatotoxicity, fatigue, and myelosuppression. The ability to selectively target mutant KIT while sparing normal KIT function would represent a crucial therapeutic advantage if confirmed in human trials.
The planned IND filing in early 2026 positions this agent on a reasonable development timeline. For context, patients with KIT-driven cancers that have exhausted available TKIs currently face limited options, with many relying on clinical trials or palliative care. A precision therapy that can overcome TKI resistance would address a critical unmet need in these orphan disease populations.
- 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 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.
FAQ
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