Egetis Therapeutics (EGTX) investor relations material

Strategic focus and product pipeline

• Specializes in orphan drug development, with late-stage focus and commercialization of Emcitate (tiratricol) for MCT8 deficiency, an ultra-rare genetic disorder.

• Emcitate is the first and only approved treatment for MCT8 deficiency in the EU, launched in Germany in May 2025, with US FDA PDUFA date set for September 28, 2026.

• Received multiple regulatory designations: Orphan Drug (EU/US), Breakthrough Therapy, Fast Track, and Rare Pediatric Disease, ensuring market exclusivity and potential for a Priority Review Voucher.

• Expansion potential into Resistance to Thyroid Hormone Type Beta (RTH-β), with orphan designation for this indication as well.

Clinical data and efficacy

• Robust clinical program includes Triac Trial I, EMC cohort, Triac Trial II, ReTRIACt, and survival studies, demonstrating significant and durable reduction in T3 levels and improved clinical outcomes.

• Real-world evidence shows a threefold reduction in mortality risk for MCT8 patients treated with tiratricol.

• Patient-centered outcomes include improved interaction, alertness, motor skills, and sleep, with high caregiver satisfaction.

• ETA guidelines recommend Emcitate as long-term therapy for all MCT8 deficiency patients.

Commercialization and market strategy

• EU launch executed in two waves, starting with Germany, France, Italy, and Spain, followed by broader European rollout.

• US launch planned for Q4 2026, with strong patient identification, early access programs, and payer engagement underway.

• Partnerships established for Japan (Fujimoto), Türkiye, Central/Eastern/Southeastern Europe (Er-Kim), and Gulf region (taiba rare).

• Lean, agile commercial team with deep rare disease expertise and targeted stakeholder engagement.