Hoth Therapeutics HT-001 Interim Results First-in-Class Topical Therapy Preserves Cancer Treatment While Resolving Dermatologic Side Effects

Rhea-AI Summary

Hoth Therapeutics (NASDAQ: HOTH) announced breakthrough interim results for its HT-001 Phase 2a clinical trial, achieving a 100% response rate in at least one primary endpoint for treating EGFR inhibitor-induced skin toxicities. The trial demonstrated that over 65% of patients reported reduced pain and itching, with no patients requiring dose reduction or discontinuation of their cancer treatment.

HT-001, a once-daily topical gel containing an FDA-approved NK1 receptor antagonist, targets inflammatory pathways triggered by EGFR inhibition. The therapy showed significant efficacy in preclinical studies and is being developed under the 505(b)(2) regulatory pathway, positioning it to potentially become the first FDA-approved treatment specifically for EGFRI-related skin toxicities, which affect up to 90% of cancer patients receiving EGFR inhibitors.

Positive

• 100% of patients achieved at least one primary endpoint of clinical dermatologic improvement
• Over 65% of patients experienced reduced pain and itching symptoms
• 0% of patients required dose reduction or discontinuation of cancer treatment
• No serious adverse events reported, demonstrating strong safety profile
• Potential to be first FDA-approved therapy for EGFRI-related skin toxicities

Negative

• None.

Insights

Oncology Clinical Researcher

Hoth's HT-001 shows promising 100% response rate in Phase 2a trial addressing critical EGFR inhibitor side effects that often disrupt cancer treatment.

The interim Phase 2a results for Hoth Therapeutics' HT-001 represent a potentially significant advancement in supportive oncology care. The reported 100% response rate in at least one primary endpoint for EGFR inhibitor-induced skin toxicities addresses a widespread clinical challenge - up to 90% of cancer patients on EGFR inhibitors experience these dermatologic side effects, which frequently necessitate dose reductions or treatment discontinuation.

What makes these results particularly compelling is that none of the patients required dose reduction or discontinuation of their cancer therapy during treatment with HT-001. This is crucial because maintaining full therapeutic dosing of EGFR inhibitors directly impacts cancer treatment efficacy and patient outcomes. The additional finding that over 65% of patients experienced reductions in pain and pruritus suggests meaningful quality-of-life improvements.

From a mechanistic perspective, HT-001's approach targeting the neuroinflammatory pathway via NK1 receptor antagonism represents a novel strategy. Rather than causing immunosuppression, it specifically addresses the Substance P-driven inflammatory cascade triggered by EGFR inhibition.

The development under the 505(b)(2) regulatory pathway is strategically advantageous, potentially accelerating approval by leveraging existing safety data for the active compound. If successful, HT-001 would become the first FDA-approved therapy specifically for EGFRI-related skin toxicities, filling a significant unmet need across multiple cancer types including non-small cell lung cancer, pancreatic, breast, colorectal, and head and neck cancers.

Hoth Therapeutics' HT-001 Achieves 100% Response Rate in at least one endpoint in Phase 2a Trial in PK Patients for EGFR Inhibitor-Related Skin Toxicities.

Hoth Therapeutics will host a Key Opinion Leader (KOL) event on, at 3:30PM EST to highlight recent clinical progress with HT-001, a novel topical therapeutic developed to address EGFR inhibitor-induced skin toxicities in cancer patients. This event will feature insights from derm-oncology and dermatology specialists Jonathan Hale Zippin M.D., Ph.D., and Adam Friedman M.D., F.A.A.D., who will present interim results from the ongoing Phase 2 trial and discuss how HT-001 could redefine supportive care standards for oncology patients.  Access/join the event through the following link: https://zoom.us/j/91353016981.

Phase 2a Trial Highlights (CLEER-001)

• 100% of enrolled patients in open-label cohort achieved at least one primary endpoint of clinical dermatologic improvement
• Over 65% reported reductions in pain and pruritus (itching)
• 0% required dose reduction or discontinuation of their EGFRI therapy.
• Topical therapy was well tolerated with no serious adverse events.

NEW YORK, June 24, 2025 /PRNewswire/ -- Hoth Therapeutics, Inc. (NASDAQ: HOTH), a clinical-stage biopharmaceutical company developing targeted therapies for rare and serious inflammatory conditions, today announced that its investigational candidate HT-001 met the primary efficacy endpoint in at least one metric in 100% of patients in its ongoing Phase 2a clinical study (CLEER-001) evaluating treatment for epidermal growth factor receptor inhibitor (EGFRI)-induced cutaneous toxicities.

EGFR inhibitors, used widely to treat non-small cell lung cancer (NSCLC), pancreatic, breast, colorectal, and head and neck cancers, are associated with dermatologic side effects in up to 90% of patients, often resulting in painful rashes, pruritus, dryness, nail changes, and alopecia. These adverse events frequently force dose reductions or treatment discontinuation, limiting therapeutic efficacy and patient outcomes.

"HT-001 is a breakthrough candidate with the potential to be the first FDA-approved therapy specifically targeting these EGFRI-related skin toxicities," said Robb Knie, CEO of Hoth Therapeutics. "The ability to preserve full-dose cancer treatment while improving patient quality of life addresses a critical unmet need across oncology."

Phase 2a Trial Highlights (CLEER-001)

• 100% of enrolled patients in open-label cohort achieved at least one primary endpoint of clinical dermatologic improvement
• Over 65% reported reductions in pain and pruritus (itching)
• 0% required dose reduction or discontinuation of their EGFRI therapy.
• Topical therapy was well tolerated with no serious adverse events.

HT-001 is a once-daily topical gel formulated with an FDA-approved neurokinin-1 receptor antagonist (NK1RA). This mechanism mitigates inflammatory pathways triggered by EGFR inhibition, particularly Substance P-driven responses that lead to skin breakdown. By targeting the neuroinflammatory axis, HT-001 reduces symptoms without immunosuppression or systemic toxicity.

Supporting Preclinical Data

In preclinical rat models co-treated with erlotinib (5.85 mg/kg/day), HT-001 significantly reduced:

• Dermatitis and alopecia severity
• Inflammatory markers including Substance P and neutrophil activity
• Disease progression even when HT-001 was introduced after symptom onset.

Additionally, in compassionate-use human cases, complete symptom resolution was observed within one week, with no recurrence for up to three weeks post-treatment discontinuation.

Regulatory and Development Pathway

HT-001 is being advanced under the 505(b)(2) regulatory pathway, enabling the use of existing safety data to accelerate development. Key milestones include:

• IND opened and chronic toxicology completed.
• Phase 2a trial (CLEER-001) currently underway in the U.S.
• Phase 2b/3 trial planning in progress

About Hoth Therapeutics
Hoth Therapeutics, Inc. (NASDAQ: HOTH) is a biopharmaceutical company developing innovative therapies for patients with unmet medical needs. The Company's pipeline includes treatments targeting rare diseases, inflammatory skin disorders, cancer, and neurological conditions. For more information, visit: 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: investorrelations@hoththerapeutics.com
www.hoththerapeutics.com
Phone: (678) 570-6791

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SOURCE Hoth Therapeutics, Inc.

FAQ

What are the key results from Hoth Therapeutics' (NASDAQ: HOTH) HT-001 Phase 2a trial?

The trial achieved a 100% response rate in at least one primary endpoint, with over 65% of patients reporting reduced pain and itching. No patients required dose reduction of their cancer treatment, and the therapy was well-tolerated with no serious adverse events.

How does Hoth Therapeutics' HT-001 work to treat EGFR inhibitor skin toxicities?

HT-001 is a once-daily topical gel containing an FDA-approved NK1 receptor antagonist that mitigates inflammatory pathways triggered by EGFR inhibition, particularly targeting Substance P-driven responses that lead to skin breakdown.

What percentage of cancer patients experience skin toxicities from EGFR inhibitors?

Up to 90% of patients receiving EGFR inhibitors experience dermatologic side effects, including painful rashes, itching, dryness, nail changes, and hair loss.

What is the regulatory pathway for Hoth Therapeutics' HT-001?

HT-001 is being developed under the 505(b)(2) regulatory pathway, which allows use of existing safety data to accelerate development. The Phase 2a trial is ongoing, with Phase 2b/3 trial planning in progress.

What types of cancers are treated with EGFR inhibitors that could benefit from HT-001?

EGFR inhibitors are widely used to treat non-small cell lung cancer (NSCLC), pancreatic, breast, colorectal, and head and neck cancers, making these patient populations potential beneficiaries of HT-001 therapy.