Egetis Therapeutics (EGTX) Study Result summary

Study background and objectives

• Triac Trial II evaluated Emcitate (tiratricol) in children under 30 months with MCT8 deficiency, focusing on neurocognitive development and peripheral thyrotoxicosis over 96 weeks.

• The trial included 22 patients across multiple countries, dosed at more than double the amount per kilogram compared to previous studies.

• The primary endpoint combined gross motor function measures (GMFM-88) and infant development scales (BSID-III) versus historical or natural history controls.

• Secondary endpoints included achievement of motor milestones, normalization of thyroid hormone markers, and safety/tolerability.

• MCT8 deficiency is a severe, ultra-rare disease with no approved treatments, causing severe intellectual and motor disability, low body weight, cardiovascular issues, and significant unmet medical need.

Key results and findings

• Numerical improvements in neurocognitive development were observed, but primary endpoints did not reach statistical significance versus historical controls.

• Significant and durable reduction of T3 levels was confirmed in all patients, alleviating thyrotoxicosis features.

• Emcitate maintained a well-tolerated safety profile, even at higher doses in very young children.

• Data will be presented at upcoming medical meetings and submitted for peer-reviewed publication.

• The study was well executed, with robust data collection and no safety concerns.

Regulatory pathway and implications

• The marketing authorization application (MAA) was submitted to the EMA, including data from three studies and a natural history cohort; validated in October 2023.

• The FDA requires a placebo-controlled withdrawal study (ReTRIACt) for the NDA; Triac Trial II is complementary to both submissions.

• Triac Trial II results will be included in the EMA response to 120-day questions in August 2024, focusing on safety in young children.

• Regulatory timelines and probability of approval remain unchanged in both the EU and US; US NDA timing will be updated after ReTRIACt trial completion.

• Emcitate holds Orphan Drug and Rare Pediatric Disease Designations in the US and EU, and is positioned to become the first approved treatment for MCT8 deficiency.