Egetis Therapeutics (EGTX) Study Result summary

Study design and objectives

• ReTRIACt was a double-blind, placebo-controlled, randomized withdrawal study in MCT8 deficiency, including both treatment-naive and previously treated patients, with males aged 4+ on stable tiratricol therapy.

• Patients were titrated to stable serum T3 before randomization to continue tiratricol or switch to placebo for 30 days or until reaching a serum T3 rescue criterion.

• The study's primary endpoints were the rate of change in serum T3 and the need for T3 rescue, with type 1 error controlled via alpha recycling.

• The statistical analysis plan was revised after FDA feedback and submitted before database lock and unblinding.

• The study aimed to complement existing clinical data for a New Drug Application (NDA) submission.

Key results and outcomes

• Statistically significant difference in serum T3 rate of change between placebo and tiratricol groups (ratio 1.494, p=0.034); placebo group showed a higher rate of T3 increase (1.590) compared to tiratricol (1.064).

• Placebo group showed a 59% increase in T3 over 30 days, while tiratricol group showed essentially no change; all placebo patients had increased serum T3, while tiratricol patients had stable or decreased levels.

• All placebo patients had a larger T3 increase than any tiratricol patient, showing clear group separation.

• Four placebo patients met rescue criteria versus none in the tiratricol group (sensitivity analysis p=0.070); 50% of placebo patients required T3 rescue versus 14% in the tiratricol group (p=0.182).

• After the randomized period, placebo patients reinitiated tiratricol and T3 levels decreased as expected.

Regulatory and clinical development

• ReTRIACt is the final clinical study for Emcitate in MCT8 deficiency, complementing a robust dataset for NDA submission.

• NDA rolling submission planned for December 2025, with full submission in early 2026 and FDA review completion expected in Q3 2026 if Priority Review is granted.

• Emcitate holds Breakthrough Therapy, Orphan Drug, Fast Track, and Rare Pediatric Disease designations in the US.

• Approved in the EU in February 2025 and launched in Germany in May 2025.

• The ReTRIACt study is considered complementary evidence, with the NDA based on the totality of clinical data.