Egetis Therapeutics (EGTX) Corporate presentation summary

Strategic focus and company overview

• Integrated orphan drug company specializing in late-stage development and commercialization of therapies for rare diseases.

• Emcitate (tiratricol) is the lead product, targeting MCT8 deficiency, an ultra-rare genetic disorder with high unmet medical need.

• Emcitate supplied to over 230 patients in more than 25 countries through managed access programs.

• Listed on Nasdaq Stockholm with a strong leadership team experienced in rare disease drug development and commercialization.

Emcitate (tiratricol) clinical and regulatory milestones

• First and only approved treatment for MCT8 deficiency in the EU, launched in Germany in May 2025.

• FDA accepted NDA with Priority Review; PDUFA date set for September 28, 2026.

• Received Breakthrough Therapy, Orphan Drug, and Rare Pediatric Disease designations from FDA; market exclusivity of 10 years (EU) and 7 years (US).

• Robust clinical data from multiple studies, including Triac Trial I, EMC cohort, and ReTRIACt, demonstrating efficacy and safety.

• European Thyroid Association recommends Emcitate as long-term therapy for all MCT8 deficiency patients.

Clinical efficacy and patient outcomes

• Emcitate normalizes T3 levels, improves cardiovascular health, body weight, and reduces mortality risk by threefold in MCT8 deficiency.

• Long-term real-world data confirm sustained efficacy and benign safety profile over up to six years.

• Caregiver-reported outcomes show improvements in interaction, alertness, motor skills, and sleep.

• All parents in clinical studies preferred to continue tiratricol treatment.