Egetis Therapeutics (EGTX) investor relations material

Strategic focus and company overview

• Specializes in late-stage development and commercialization of orphan drugs, with a primary focus on Emcitate (tiratricol) for MCT8 deficiency.

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

• Holds multiple regulatory designations: Orphan Drug, Breakthrough Therapy, Fast Track, and Rare Pediatric Disease in the US and EU.

• Expanding market presence through in-house teams in the US and EU, and partnerships in Japan, Türkiye, and the Gulf region.

• Pipeline includes Aladote for paracetamol overdose, currently on hold until Emcitate submissions are completed.

MCT8 deficiency and unmet medical need

• MCT8 deficiency is a rare, X-linked disorder causing severe neurocognitive and motor impairment, with a median life expectancy of 35 years.

• Incidence estimated at 1 per 70,000 male births; high disease burden with 100% requiring lifelong care and ~30% dying in childhood.

• Main cause of mortality is sudden cardiac death; patients often remain undiagnosed due to lack of awareness.

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

Clinical efficacy and safety of Emcitate (tiratricol)

• Triac Trial I and real-world cohort studies show significant, durable reduction in serum T3, normalization of thyrotoxicosis, and improved bodyweight and cardiovascular status.

• Treatment associated with a threefold lower risk of mortality in MCT8 deficiency patients.

• Positive results from the ReTRIACt study support NDA submission, showing statistically significant differences in T3 levels between treatment and placebo.

• Safety profile is benign, with beneficial effects maintained up to six years.

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