Hoth Therapeutics' Cancer Therapeutic HT-KIT Surpasses Preclinical Milestones With Potent Anti-Tumor Activity and GLP-Validated Bioanalytical Results, Exceeding Regulatory Standards

NEW YORK, Sept. 2, 2025 /PRNewswire/ -- Hoth Therapeutics, Inc. (NASDAQ: HOTH), a clinical-stage biopharmaceutical innovator, today announced combined positive findings from multiple preclinical programs evaluating its precision antisense candidate HT-KIT, including compelling anti-tumor efficacy, a clean safety profile, and new GLP-validated bioanalytical results that exceeded internationally recognized regulatory thresholds.

Preclinical Efficacy & Safety Highlights

• Rapid Tumor Kill: HT-KIT triggered significant tumor cell death in preclinical models of gastrointestinal stromal tumors (GIST) and systemic mastocytosis as early as 24 hours post-treatment, with statistically significant tumor shrinkage observed by day 8.
• Strong KIT Suppression: In vitro, HT-KIT achieved over 80% knockdown of KIT expression, the oncogenic driver in multiple aggressive cancers.
• Clean Safety Profile: Multi-dose in vivo studies confirmed no off-target toxicity across critical organs including liver, kidneys, spleen, bone marrow, and thymus.
• Dose-Dependent Biological Signal: A preclinical safety study demonstrated proportional liver engagement (increase from 1.11g to 1.32g at 3.0 mg/kg) with zero gross pathology at any organ site.

GLP-Validated Bioanalytical Results

The study, conducted by Altasciences Company, Inc. under OECD, FDA, and EMA GLP standards, demonstrated that HT-KIT meets or exceeds strict bioanalytical benchmarks:

• Regulatory-Grade Validation: All calibration curve, quality control, and dilution integrity requirements passed with high reproducibility.
• Superior Data Integrity: 90.5% of Incurred Sample Reanalysis (ISR) values fell within ±30%, well above the 66.7% regulatory minimum.
• Extended Stability: HT-KIT remained stable in serum for 37 days at -80°C, surpassing the validated 28-day stability period, with further studies ongoing.
• Flawless Compliance: No protocol or SOP deviations impacted study reliability.

"These results combine a rare and powerful story — tumor kill within 24 hours, clean safety across all systems, and GLP-validated reproducibility beyond regulatory standards," said Robb Knie, CEO of Hoth Therapeutics. "We believe HT-KIT has the potential to transform outcomes in KIT-driven cancers, and these milestones accelerate our path toward IND submission and first-in-human trials."

Next Steps

Hoth expects to integrate this bioanalytical data into its formal GLP toxicology package and is preparing for an IND.

About HT-KIT

HT-KIT is a precision antisense oligonucleotide designed to silence mutant KIT mRNA, an oncogenic driver in gastrointestinal stromal tumors (GIST), systemic mastocytosis, and certain leukemias. By targeting KIT expression at the genetic level, HT-KIT seeks to overcome resistance to tyrosine kinase inhibitors while minimizing systemic side effects.

About Hoth Therapeutics, Inc.

Hoth Therapeutics, Inc. (NASDAQ: HOTH) is a clinical-stage biopharmaceutical company developing targeted, first-in-class therapies for oncology, dermatology, and rare diseases.

For more information, visit http://www.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 the current coronavirus pandemic, or any other 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: [email protected]
www.hoththerapeutics.com
Phone: (678) 570-6791

SOURCE Hoth Therapeutics, Inc.