Medicus Pharma Receives FDA “Study May Proceed” Letter for NDA-Enabling Registrational SkinJect® Study in Patients with Gorlin Syndrome
Rhea-AI Summary
Medicus Pharma (NASDAQ: MDCX) received an FDA “Study May Proceed” letter for its NDA-enabling, registrational Phase 2b study SKNJCT-005, evaluating SkinJect® (D‑MNA) 200 mcg in Gorlin Syndrome patients with multiple basal cell carcinomas. The open-label, multicenter trial is expected to enroll up to 50 patients.
According to Medicus, FDA feedback led to protocol enhancements including mandatory baseline biopsy confirmation of nodular BCC, a composite primary endpoint requiring both clinical and histological clearance, in-person investigator assessments, aligned responder definitions, and extended follow-up from 24 weeks to at least two years to assess durability. Prior SKNJCT-003 data in 90 patients showed 64% clinical and 55% histological clearance at Day 57 after three 200‑mcg treatments.
The FDA also issued non–clinical-hold recommendations on pharmacokinetics, microneedle evaluation, genetic confirmation of Gorlin Syndrome, independent safety monitoring, statistical methods, and future randomized, blinded, comparator-controlled designs. Medicus plans to amend the SKNJCT-005 protocol and provide further updates as study initiation and enrollment planning advance.
Positive
- FDA “Study May Proceed” authorization for NDA-enabling Phase 2b SKNJCT-005
- Up to 50 patients in multicenter registrational trial targeting rare Gorlin Syndrome
- Prior SKNJCT-003 study showed 64% clinical and 55% histological clearance at Day 57
- Follow-up extended to at least two years, enhancing durability-of-response dataset
- Potential to be first FDA-approved lesion-directed therapy specifically for Gorlin Syndrome
Negative
- Follow-up extended from 24 weeks to ≥2 years, lengthening trial duration and data-readout timeline
- FDA highlights need for future randomized, blinded, comparator-controlled studies, implying more complex later-stage development
- SkinJect® remains in Phase 2b, with regulatory approval and commercialization still several stages away
Key Figures
Historical Context
| Date | Event | Sentiment | 24h Move | Catalyst |
|---|---|---|---|---|
| Jul 16 | FDA feedback study | Positive | +16.5% | FDA feedback and IRB approval supported a redesigned Teverelix Phase 2 study. |
| Jun 22 | Conference presentation | Neutral | -1.9% | Bio International presentation outlined SkinJect and Teverelix development programs for investors. |
| Jun 15 | FDA designation request | Positive | -5.2% | Company submitted an FDA Rare Pediatric Disease Designation request for SkinJect. |
| Jun 11 | Phase 2a study | Positive | +4.0% | Company initiated a Teverelix Phase 2a endometriosis study using genomic data. |
| Jun 08 | European study submission | Positive | -3.9% | Company submitted a European modification supporting planned Teverelix Phase 2b development. |
24h Move is the share-price change in the day after each event; other market factors may also have contributed.
Positive regulatory and clinical announcements showed mixed price alignment, with 2 aligned reactions and 3 divergences.
Key Terms
investigational new drug regulatory
histological clearance medical
pharmacokinetic assessments medical
data safety monitoring board medical
statistical analysis plan technical
basal cell carcinoma medical
AI-generated analysis. How Rhea-AI works. Not financial advice.
SKNJCT-005 registrational clinical study protocol strengthened through planned implementation of FDA recommendations, including combined clinical and histological clearance endpoints and extended patient follow-up
PHILADELPHIA, July 27, 2026 (GLOBE NEWSWIRE) -- Medicus Pharma Ltd. (NASDAQ: MDCX) (“Medicus” or the “Company”), a biotech/life sciences company focused on advancing the clinical development programs of novel and potentially disruptive therapeutic assets, today announced that the U.S. Food and Drug Administration (“FDA”) has completed its safety review of the Company's Investigational New Drug ("IND") application for SKNJCT-005 and issued a "Study May Proceed" letter authorizing the initiation of the Company's NDA-enabling registrational Phase 2b clinical study evaluating SkinJect® (D-MNA) 200 mcg in patients with Gorlin Syndrome presenting with multiple basal cell carcinomas ("BCCs").
The FDA authorization follows Medicus' submission of the SKNJCT-005 protocol and the Company's comprehensive response to the Agency's June 18, 2026, information requests.
“Receiving FDA clearance to initiate our registrational Gorlin Syndrome study represents a significant regulatory milestone for the SkinJect® program,” stated Dr. Raza Bokhari, Medicus Executive Chairman and CEO. "Importantly, the FDA's recommendations strengthen the registrational study by enhancing the scientific rigor of the efficacy assessments while further supporting the long-term evaluation of treatment durability. We believe these refinements position SkinJect® to generate an even more robust data package as we advance toward a potential NDA submission for patients with Gorlin Syndrome, a population with substantial unmet medical need, who frequently face repeated surgeries throughout their lifetimes. The ability to treat multiple lesions using a localized precision therapy represents an important advancement and what we believe could become a new standard of care for this patient population."
FDA Authorization Advances Registrational Development
SKNJCT-005 is designed as a Phase 2b, open-label, multicenter, NDA-enabling registrational study evaluating SkinJect® 200 mcg in patients with Gorlin Syndrome (Nevoid Basal Cell Carcinoma Syndrome) who develop multiple basal cell carcinomas requiring repeated surgical treatment. The study is intended to generate robust efficacy and safety data to support registration of SkinJect® in this rare disease population and is expected to enroll up to 50 patients.
SkinJect® is a proprietary dissolvable microneedle-array patch that delivers doxorubicin directly into basal cell carcinoma lesions, enabling localized treatment while minimizing systemic drug exposure. Previous Phase 1 and Phase 2 studies have demonstrated a favorable safety profile together with encouraging clinical and histological clearance rates in patients with basal cell carcinoma. The submitted protocol cites data from the completed 90-patient SKNJCT-003 study demonstrating
Protocol Strengthened Following FDA Review
As part of the FDA review process, the Agency provided constructive recommendations designed to enhance the scientific and regulatory utility of the study. Medicus agreed to incorporate these recommendations through a protocol amendment, including:
- requiring baseline biopsy confirmation of nodular basal cell carcinoma for all enrolled patients;
- revising the primary efficacy endpoint to require both clinical and histological clearance at a prespecified time point;
- requiring in-person investigator assessment of clinical clearance in conjunction with histological confirmation;
- aligning the responder definition with the revised composite endpoint; and
- extending patient follow-up from 24 weeks to at least two years to evaluate durability of response.
In its “Study May Proceed” letter of July 23, 2026, the FDA has also provided additional recommendations relating to pharmacokinetic assessments, evaluation of used microneedle arrays, genetic confirmation of Gorlin Syndrome, establishment of an independent Data Safety Monitoring Board, refinement of statistical methodology, development of a standalone Statistical Analysis Plan, and future incorporation of randomized, blinded, comparator-controlled study designs intended to support substantial evidence of effectiveness. These recommendations were specifically identified by the FDA as not constituting clinical hold issues. The company is evaluating these recommendations and intends to submit an amended SKNJCT-005 protocol incorporating the FDA's recommendations.
The Company expects to provide additional updates regarding study initiation and enrollment timing as development progresses.
Addressing a Significant Unmet Medical Need in Gorlin Syndrome:
Gorlin Syndrome is a rare autosomal dominant genetic disorder caused primarily by mutations affecting the Hedgehog signaling pathway, characterized by the lifelong development of multiple basal cell carcinomas beginning during childhood or early adulthood. Many patients develop dozens—or even hundreds—of tumors throughout their lifetime, often requiring repeated surgical excisions that can result in cumulative scarring, functional impairment and significant psychosocial burden. The current protocol notes that affected individuals may develop dozens to more than 1,000 basal cell carcinomas during their lifetime. The condition is estimated to affect approximately 1 in 30,000 to 60,000 individuals worldwide, corresponding to an estimated U.S. patient population of approximately 6,000 to 12,000 individuals and a substantially larger addressable population across major global markets.
SkinJect® is being developed as a lesion-directed, minimally invasive treatment designed to reduce reliance on repeated surgical procedures while preserving surgery as an option for lesions that do not respond or require definitive excision. If approved, SkinJect® has the potential to become the first FDA-approved lesion-directed therapy specifically developed for patients with Gorlin Syndrome and the first microneedle-based chemotherapy platform designed to achieve clinically meaningful visual and histological clearance of basal cell carcinoma lesions.
For further information contact:
Carolyn Bonner, President and Chief Financial Officer
(610) 636-0184
cbonner@medicuspharma.com
Anna Baran-Djokovic, SVP Investor Relations
(305) 615-9162
adjokovic@medicuspharma.com
About Medicus Pharma Ltd.
Medicus Pharma Ltd. (Nasdaq: MDCX) is a precision-guided biotech/life sciences company focused on accelerating the clinical development programs of novel and potentially disruptive therapeutic assets. The Company is actively engaged in multiple countries across three continents.
The Company’s current key therapeutic assets are:
SkinJect®, a novel localized immuno-oncology precision product focused on non-melanoma skin diseases, especially basal cell carcinoma (BCC) and Gorlin Syndrome, a rare autosomal dominant disease also called nevoid BCC syndrome.
Teverelix®, a next-generation GnRH antagonist, is a product under clinical development for cardiovascular high-risk advanced prostate cancer patients and patients with acute urinary retention relapse (AURr) episodes due to enlarged prostate, and endometriosis.
Medicus’ strategy is to advance select programs through Phase 2 proof-of-concept, key clinical and regulatory inflection points that substantially reduce development risk and increase attractiveness to potential pharmaceutical partners. By generating decision-grade clinical, regulatory and operational datasets, the Company seeks to create opportunities for strategic collaborations, regional licensing transactions and broader commercialization partnerships with established pharmaceutical companies. As data matures across its programs, Medicus intends to continue building differentiated development packages designed to maximize asset value while maintaining capital efficiency and development focus.
Cautionary Notice on Forward-Looking Statements
Certain information in this news release constitutes "forward-looking information" under applicable securities laws. "Forward-looking information" is defined as disclosure regarding possible events, conditions or financial performance that is based on assumptions about future economic conditions and courses of action and includes, without limitation, statements regarding the development of SkinJect® and the potential benefits thereof for those suffering with Gorlin Syndrome, the receipt of the FDA’s written feedback and “study may proceed” determination from the FDA for IND 178051 / SKNJCT-005 and the Company’s response to such feedback, the development, advancement and commercialization of SkinJect®, and the potential market opportunities related thereto, the Company’s expectations regarding reported efficacy findings of SkinJect®, the development of Teverelix® and expectations concerning, and future outcomes relating to, the development, advancement and commercialization of Teverelix® for AURr, cardiovascular high-risk advanced prostate cancer, women’s health indications like endometriosis, and the potential market opportunities related thereto; the Company’s expectations regarding the Phase 2 study, the timing and results thereof, including the number patients to be enrolled therein; the Company’s expectations regarding the generation of an early pharmacodynamic signal, decision-grade clinical evidence and a comprehensive Phase 2 dataset for Teverelix®, and the potential to accelerate clinical development, optimize capital allocation and support dose optimization and route selection; the potential for Teverelix® to establish a new treatment paradigm for recurrent AUR; and potential strategic partnering opportunities. Forward-looking statements are often but not always, identified by the use of such terms as "may", “on track”, “aim”, "might", "will", "will likely result", “could,” “designed,” "would", "should", "estimate", "plan", "project", "forecast", "intend", "expect", "anticipate", "believe", "seek", "continue", "target", “potential” or the negative and/or inverse of such terms or other similar expressions. These statements involve known and unknown risks, uncertainties and other factors, which may cause actual results, performance or achievements to differ materially from those expressed or implied by such statements, including those risk factors described in the Company's annual report on form 10-K for the year ended December 31, 2025, and in the Company's other public filings on EDGAR and SEDAR+, which may impact, among other things, the trading price and liquidity of the Company's common shares. Forward-looking statements contained in this news release are expressly qualified by this cautionary statement and reflect our expectations as of the date hereof and thus are subject to change thereafter. The Company disclaims any intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. Readers are further cautioned not to place undue reliance on forward-looking statements as there can be no assurance that the plans, intentions or expectations upon which they are placed will occur. Such information, although considered reasonable by management at the time of preparation, may prove to be incorrect and actual results may differ materially from those anticipated.
