Catalyst overview
Research summary
This catalyst tracks a Phase 1/2 data readout for ECUR-506 in Ornithine transcarbmylase deficiency. The expected timing is based on the latest available company guidance and may change.
OTC-HOPE trial. Collab w/ iECURE. 3/6/24- MHRA approved CTA. 5/7/24 Fast Track Designation. 1/9/25- Reported efficacy & safety data. Finish enrollment in 2025 and provide complete data in H1 2026. 11/03/25:- Additional ECUR-506 data presented at several conferences; OTC-HOPE trial ongoing globally with full Phase 1/2 data expected in H1 2026. 01/07/26:- Received FDA RMAT and UK ILAP Innovation Passport; OTC-HOPE aligned on registrational endpoints; first infant complete response. 01/12/26:- FDA aligned OTC-HOPE endpoints and size for potential BLA; iECURE plans data release H1 2026. 03/12/26:- Reiterated OTC-HOPE Phase 1/2 plans for additional ECUR-506 data in first half of 2026. 03/18/26:- Selected for FDA CDRP pilot to support CMC readiness. Additional OTC-HOPE data expected in H1 2026. 04/28/26:- Preliminary OTC-HOPE data at ASGCT May 13, 2026. One-year infant data at SIMD May 18. 05/05/26:- Upcoming OTC-HOPE ASGCT oral shows first three dose cohorts; SIMD poster features one-year infant data. 05/13/26:- ASGCT preliminary OTC-HOPE data showed fewer HACs; SIMD 2026 efficacy update expected May 17-20. 05/18/26:- SIMD 2026 showed low-dose cohort efficacy; 18-month durability and significant HAE/HAC reductions. 08/06/26:- OTC-HOPE data showed 71% had no hyperammonemic crises; first infant had complete response. 08/20/26:- SSIEM oral update scheduled Aug 27, 2026 for OTC-HOPE safety and low-dose genome editing data.
Verification
Confirm timing and program details against the company announcement or filing.