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 United Arab Emirates (UAE) Department of Health – Abu Dhabi ("DOH") has granted Investigational New Drug ("IND") authorization for the Company's PRECISION-E2 Phase 2a clinical study evaluating Teverelix® in women with moderate-to-severe symptomatic endometriosis, a chronic estrogen-dependent inflammatory disease that affects approximately one in ten women of reproductive age worldwide.

The authorization follows completion of the DOH's comprehensive scientific and regulatory review of the Company's chemistry, manufacturing and controls (CMC), non-clinical, clinical and safety documentation and approved the “Study to Proceed”, subject to completion of required site-specific Institutional Review Board ("IRB") and ethics approvals.

The PRECISION-E2 study represents a significant regulatory milestone in Medicus' women's health strategy and advances the development of Teverelix® into what the Company believes is one of the first prospective genomics-guided precision medicine development programs designed to identify women with moderate-to-severe endometriosis most likely to benefit from a long-acting GnRH antagonist.

"Today's IND authorization represents far more than approval to initiate another clinical study,” stated Dr. Raza Bokhari, Medicus Executive Chairman and CEO, "it validates a development strategy designed to combine precision endocrinology, pharmacology and population genomics within a prospective clinical trial. We believe PRECISION-E2 has the potential not only to optimize the future development of Teverelix® but also is an important step toward personalized therapy that could help redefine how women with endometriosis and other estrogen-driven diseases are stratified and may support future development across multiple women's health indications.”

PRECISION-E2 Study Design:

PRECISION-E2 (Precision Estradiol Suppression and Genomic Response Study in Endometriosis) is a prospective, randomized, placebo-controlled Phase 2a clinical study designed to evaluate the pharmacodynamics, safety, tolerability, clinical activity and exploratory genomic correlates of Teverelix® in approximately 84 women with moderate-to-severe symptomatic endometriosis across multiple investigational sites in the United Arab Emirates. 

Participants will be randomized equally among four treatment groups:

• Teverelix® 60 mg administered subcutaneously
• Teverelix® 90 mg administered subcutaneously
• Teverelix® 90 mg administered intramuscularly
• Placebo administered subcutaneously.

The study's primary pharmacodynamic endpoint is the proportion of participants achieving and maintaining serum estradiol within the established Barbieri therapeutic window (approximately 20–50 pg/mL) for at least 14 consecutive days following treatment.

Secondary and exploratory endpoints include pharmacokinetic parameters, hormonal biomarkers, bone turnover biomarkers, safety, immunogenicity, endometriosis-associated pain, quality-of-life assessments and exploratory genomic analyses. 

The Company intends to explore whether integration of these clinical, pharmacodynamic and genomic data can identify candidate treatment-response biomarkers associated with controlled estradiol suppression, clinically meaningful benefit and treatment tolerability. Any candidate biomarker or multi-gene treatment-response signature identified through PRECISION-E2 would require confirmation in subsequent clinical studies before being used to guide patient selection.

Why PRECISION-E2 Is Different

Unlike conventional endometriosis clinical studies that primarily evaluate safety and efficacy, PRECISION-E2 has been specifically designed to prospectively integrate genomic information with endocrine pharmacology, pharmacokinetics, biomarkers, safety assessments and patient-reported outcomes to better understand why individual women respond differently to hormonal therapy.

The Company believes PRECISION-E2 represents a new generation of endometriosis clinical research by prospectively integrating genomic and biological data into therapeutic development from the outset.

The study has been designed to integrate:

• Whole-genome information from participating women, subject to applicable approvals and governance requirements.
• Hormonal pharmacodynamics, including estradiol, luteinizing hormone and follicle-stimulating hormone.
• Pharmacokinetic exposure.
• Bone turnover biomarkers.
• Clinical symptom improvement.
• Patient-reported quality-of-life outcomes.
• Safety and tolerability; and
• Exploratory analyses of predefined genes and biological pathways associated with estrogen receptor signaling, estrogen synthesis and metabolism, gonadotropin signaling, endometriosis biology, inflammatory pathways and pain perception, including candidate genes such as ESR1, ESR2, CYP19A1, GNRHR, FSHR and LHCGR.

The Company believes this integrated approach has the potential to generate one of the most comprehensive precision medicine datasets assembled in endometriosis and to establish an important scientific foundation for future biomarker-informed development of Teverelix®.

Addressing a Significant Unmet Women's Health Need

Endometriosis is a chronic estrogen-dependent inflammatory disease affecting approximately one in ten women of reproductive age worldwide.

Beyond chronic pelvic pain, dysmenorrhea and infertility, the disease frequently affects education, employment, family planning, emotional wellbeing and long-term quality of life.

Despite multiple available therapies, many women continue to experience inadequate symptom control, treatment-limiting adverse effects, recurrence following treatment discontinuation and variability in therapeutic response.

PRECISION-E2 has been designed to evaluate whether Teverelix® can achieve rapid, controlled and sustained estradiol suppression while minimizing unwanted hypoestrogenic effects and simultaneously generating the pharmacodynamic, clinical and genomic data necessary to optimize dose selection, route of administration and future precision medicine strategies.