Egetis Therapeutics (EGTX) Study Result summary

Study background and objectives

• Triac Trial II evaluated Emcitate (tiratricol) in children under 30 months with MCT8 deficiency, a severe ultra-rare disease with no approved treatments, focusing on neurocognitive development and T3 reduction over 96 weeks.

• The trial enrolled 22 patients across multiple international sites and used higher doses per kg than previous studies.

• Primary endpoints were changes in GMFM-88 and BSID-III scores versus historical controls from Triac Trial I.

• Secondary endpoints included reduction in serum T3 concentrations and assessment of safety and tolerability.

Key results and findings

• No statistically significant improvement was observed in neurocognitive development compared to historical controls.

• Significant and durable reductions in thyroid hormone T3 levels were confirmed in all patients, consistent with prior studies.

• The safety profile remained well-tolerated and favorable, even at higher dosing in younger children.

Regulatory and strategic implications

• Triac Trial II data will be included in the response to EMA's 120-day questions, focusing on safety in young children, with timelines for EMA and FDA submissions unchanged.

• The product's probability of approval is not considered affected by these results.

• US NDA will be based on T3 effects, chronic thyrotoxicosis data, and ongoing ReTRIACt trial results.