Medicus Pharma CEO meets with lawmakers on Energy & Commerce Committee on Capitol Hill
Rhea-AI Summary
Medicus Pharma (NASDAQ: MDCX) said CEO Dr. Raza Bokhari and leadership met with senior members of the House Energy & Commerce Committee on April 27, 2026 to seek support for Orphan Drug designation, a registrational IND for SkinJect® in Gorlin Syndrome, and rare-disease pediatric voucher access.
Meetings included outreach with advocates and the Gorlin Syndrome Alliance and outline plans for regulatory and clinical development toward expedited access for high-burden patients.
Positive
- CEO and leadership held targeted Capitol Hill meetings to advance Orphan Drug designation for SkinJect
- Company is pursuing a registrational IND pathway focused on decision-grade clinical data
- Strategic collaboration with Gorlin Syndrome Alliance to pursue an Expanded Access IND for patients with multiple or inoperable BCCs
Negative
- MoU with Helix Nanotechnologies is explicitly non-binding and provides no assurance of a definitive collaboration
- Regulatory actions sought (Orphan designation, registrational IND, priority voucher) remain unapproved and subject to FDA discretion
News Market Reaction – MDCX
In the Apr 27 session, MDCX declined 5.68%, reflecting a notable negative market reaction. Argus tracked a trough of -3.6% from its starting point during tracking. Our momentum scanner triggered 4 alerts that day, indicating moderate trading interest and price volatility.
Data tracked by StockTitan Argus on the day of publication.
Key Figures
Historical Context
| Date | Event | Sentiment | 24h Move | Catalyst |
|---|---|---|---|---|
| Apr 17 | Orphan drug filing | Positive | +17.0% | Submission of FDA Orphan Drug Designation application for SkinJect in Gorlin Syndrome. |
| Apr 15 | Clinical data update | Positive | -25.0% | Phase 1 Teverelix data showing long-acting hormone suppression and acceptable safety. |
| Apr 06 | Trial protocol update | Positive | +9.8% | Submission of optimized Phase 2 protocol to FDA for Teverelix in acute urinary retention. |
| Apr 01 | Trial clarification | Positive | -8.5% | Clarification of positive Phase 2 SkinJect data, focusing on active treatment arms. |
| Mar 30 | KOL validation | Positive | -9.6% | Independent KOL validation of SkinJect Phase 2 data with strong response metrics. |
24h Move is the share-price change in the day after each event; other market factors may also have contributed.
Recent history shows mixed to negative price reactions to largely positive clinical and regulatory updates, with several declines following favorable SkinJect and Teverelix news.
Over the last month, Medicus issued multiple positive updates on SkinJect and Teverelix, including KOL validation of Phase 2 SkinJect data on Mar 30, clarification of Phase 2 results on Apr 1, an optimized Phase 2 Teverelix protocol on Apr 6, new Teverelix data on Apr 15, and an Orphan Drug Designation application for SkinJect on Apr 17. Price reactions have been inconsistent, with both strong gains and sharp selloffs, suggesting sentiment is fragile despite advancing programs.
Key Terms
orphan drug designation regulatory
investigational new drug (ind) regulatory
hedgehog signaling pathway medical
microneedle array medical
basal cell carcinoma medical
gnrh antagonist medical
acute urinary retention medical
expanded access ind regulatory
AI-generated analysis. How Rhea-AI works. Not financial advice.
Builds Support for Accelerated Access to SkinJect® for Gorlin Syndrome patients with a Rare Autosomal Dominant Disease
PHILADELPHIA, April 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 therapeutics assets, today announced that CEO Dr. Raza Bokhari, along with the Medicus leadership team, conducted a series of meetings on Capitol Hill with senior lawmakers across key healthcare committees to advance support for:
- Orphan Drug Designation for SkinJect® for Gorlin Syndrome
- Registrational Investigation New Drug (IND) approval for Gorlin Syndrome patients
- Rare disease pediatric FDA voucher program
These engagements included:
- Brett Guthrie — Chair of the House Energy & Commerce Committee, which has primary jurisdiction over U.S. healthcare policy
- John Joyce — Member, House Energy & Commerce Committee; Co-Chair, CongressionalSkin Cancer Caucus
- Diana Harshbarger — Vice Chair, House Energy & Commerce Subcommittee on Health
- Gus Bilirakis — Co-Chair, Congressional Rare Disease Caucus; Senior Member, Energy & Commerce Committee
These meetings reflect coordinated outreach to leadership directly involved in shaping U.S. healthcare legislation and rare disease policy.
In collaboration with the Gorlin Syndrome Alliance, discussions emphasized the urgent need for new treatment approaches, as patients with Gorlin syndrome often experience lifelong recurrence of basal cell carcinomas and are frequently described as “never cancer free,” highlighting the need for non-surgical therapeutic options such as SkinJect®.
“Our engagement with policymakers reflects a clear alignment around accelerating access to innovative therapies for patients with significant unmet need,” said Dr. Raza Bokhari, Executive Chairman & CEO of Medicus, “We believe SkinJect® has the potential to shift the treatment paradigm in Gorlin Syndrome by offering a localized, repeatable, non-surgical approach.”
The Company is concurrently advancing regulatory and clinical planning to support a registrational development pathway for SkinJect®, with a focus on generating decision-grade data to enable expedited development in high-burden patient populations.
Addressing a High-Burden Rare Disease
Gorlin Syndrome, also known as nevoid basal cell carcinoma syndrome, is a rare, inherited condition driven primarily by mutations in the PTCH1 gene, leading to dysregulation of the Hedgehog signaling pathway and early, recurrent tumor formation.
Patients with this condition may develop dozens to over 1,000 basal cell carcinomas over their lifetime, often beginning in childhood, requiring repeated surgical interventions that can result in significant physical and psychological burden.
Despite this high disease burden:
- There are no FDA-approved therapies specifically for BCC in Gorlin Syndrome
- Current treatments rely heavily on surgical excision or Mohs surgery
- Systemic therapies, such as Hedgehog inhibitors, are limited by tolerability and recurrence upon discontinuation
The Company believes SkinJect® has the potential to address this gap through a localized, repeatable, non-surgical treatment approach.
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 therapeutics assets. The Company is actively engaged in multiple countries across three continents.
Company’s key therapeutics 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, collectively representing a ~
Teverelix®, a next generation GnRH antagonist is a first-in-market product for cardiovascular high-risk advanced prostate cancer patients and patients with acute urinary retention relapse (AURr) episodes due to enlarged prostate, collectively representing a ~
The Company is actively engaged in following collaborations:
Skinject™ Platform Expansion
In August 2025, the Company announced its entry into a non-binding memorandum of understanding (MoU) with Helix Nanotechnologies, Inc. (HelixNano), a Boston-based biotech company focused on developing a proprietary advanced mRNA platform, in respect of their shared mutual interest in the development or commercial arrangement contemplated by the MoU. The MoU is non-binding and shall not be construed to obligate either party to proceed with a joint venture or any further development or commercial arrangement, unless and until definitive agreements are executed, and there can be no assurance that such definitive agreements will be executed.
The Company is exploring co-development of thermostable infectious disease vaccines combining HelixNano’s proprietary mRNA technology with the Medicus microneedle array delivery platform.
Patient Access and Advocacy
In October 2025, the Company announced a strategic collaboration with the Gorlin Syndrome Alliance (GSA) to advance compassionate access to SkinJect for patients suffering from Gorlin Syndrome, also known as nevoid basal cell carcinoma syndrome.
In collaboration with the Gorlin Syndrome Alliance, Medicus is pursuing an Expanded Access IND program to provide Gorlin Syndrome patients with multiple or inoperable BCCs access to SkinJect™, the Company’s investigational D-MNAs, under physician supervision.
AI Enabled Clinical Development
In December 2025, the Company signed a non-binding letter of intent to collaborate with Reliant AI Inc., a decision-intelligence company specializing in generative AI for the life sciences, to develop an AI-driven clinical data analytics platform to support capital-efficient and time-efficient clinical development through data-driven dynamic clinical-site selection, pharmacodynamic (PD) informed patient stratification, and enrollment forecasting. The initial phase of the collaboration is expected to support the upcoming Teverelix clinical study planned for 2026. There can be no assurance that a definitive agreement will be executed or that the proposed collaboration will proceed as contemplated.
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 Company’s ability to continue as a going concern, statements regarding the Company’s leadership and prospects, the collaboration with GSA including the potential benefits thereof for GSA, those suffering with Gorlin Syndrome and Medicus (including as it relates to the development of SkinJect™), ability to be approved for the Registrational IND Program to enable those suffering with Gorlin Syndrome to access SkinJect™ under physician-supervised treatment protocols, Orphan drug designation for Skinject the development of Teverelix and expectations concerning, and future outcomes relating to, the development, advancement and commercialization of Teverelix for AURr, high CV risk prostate cancer, women’s health indications like endometriosis, and the potential market opportunities related thereto, the MOU, including the potential signing of definitive agreements between Medicus and HelixNano and the development of thermostable infectious diseases vaccines by combining HelixNano’s proprietary mRNA vaccine platform with Medicus’s proprietary microneedle array (MNA) delivery platform, the Company’s aim to fast-track the clinical development program and convert the SKNJCT-003 exploratory clinical trial into a pivotal clinical trial, and approval from the FDA and the timing thereof, including with respect to the Company’s submission for approval in the FDA Commissioner's National Priority Voucher program, plans and expectations concerning, and future outcomes relating to, the development, advancement and commercialization of SkinJect through SKNJCT-003 and SKNJCT-004, and the potential market opportunities related thereto, the Company’s expectations regarding reported efficacy findings, the overall response rate and potential changes thereto, and whether there will be material changes to its reported SKNJCT-003 topline results and to secure an EOP2 meeting with the FDA in the first half of 2026, entry into definitive documents with Reliant and the expected terms thereof, engaging in proposed Medicus-sponsored studies currently contemplated in the Reliant non-binding letter of intent and the expected benefits thereof, the expansion of SKNJCT-003 into the United Kingdom and the potential benefits therefrom, the advancement of the SKNJCT-004 study and the potential results of and benefits of such study. 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.
