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, with global supply to over 230 patients in more than 25 countries.

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

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

• Expansion plans include potential new indications such as RTH-beta and partnerships for global reach.

Clinical and regulatory milestones

• Emcitate received EU approval in February 2025 and was launched in Germany in May 2025.

• FDA granted Breakthrough Therapy, Orphan Drug, and Rare Pediatric Disease designations; NDA accepted March 2026.

• Positive Phase 3 ReTRIACt study results and robust clinical data from multiple trials support regulatory submissions.

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

• Patent protection in the US extends to 2045, strengthening the product's exclusivity.

Disease background and unmet need

• MCT8 deficiency is a severe, ultra-rare genetic disorder with a median life expectancy of 35 years and high childhood mortality.

• Patients experience severe intellectual disability, motor impairment, and require lifelong care.

• No prior approved therapies; Emcitate addresses a significant unmet medical and societal need.