Egetis Therapeutics (EGTX) investor relations material

Introduction and purpose

• FDA granted a rolling NDA review for MCT8 deficiency treatment following a successful pre-NDA meeting, with the objective to seek FDA advice and agreement on the NDA submission content, focusing on the clinical data package.

Details of approval or decision

• Rolling NDA submission for MCT8 will begin in December 2025, with a complete NDA targeted for early 2026 and FDA review completion expected in Q3 2026, pending Priority Review.

• The NDA will be based on currently available clinical data from six sources, including multiple trials, studies, and the US Expanded Access Program.

• The retried/ReTRIACt study will be closed soon, considered complementary, and accrued data will be included in the NDA.

Impact on industry and stakeholders

• Emcitate is the first therapy targeting MCT8 deficiency, a rare disease with high unmet need, and approval could provide a treatment option for patients with severe neurological and metabolic symptoms.

• The drug holds Breakthrough Therapy and Rare Pediatric Disease Designations, potentially expediting review and access.

• The transition of oversight from the Rare Disease Division to the Endocrinology Division at the FDA has been positive, with strong agency appreciation for the unmet medical need and robust data.

• The collaborative approach with the FDA is seen as a substantial step toward approval and potential market access in the U.S.