Julian

Julian asked about alternative regulatory pathways for AMT-130 beyond traditional FDA channels, including leveraging existing or 4-year data for registration, and sought clarification on the submission plan and patient cohort for the 4-year data analysis.

Answer

CEO Matt Kapusta indicated that alternative avenues include pursuing opportunities outside the United States and leveraging patient and scientific community advocacy. CMO Walid Abi-Saab confirmed plans to amend the Statistical Analysis Plan for 4-year data submission to the FDA, noting it would include 12 patients at 4 years and all patients who have reached 3 years, though it's not expected to change the FDA's current stance on Phase I/II data alone.

Julian asked about uniQure's strategy to pursue regulatory paths beyond traditional FDA channels for AMT-130, including leveraging existing or 4-year data, and sought clarification on the specifics of the 4-year data submission to the agency.

Answer

Matt Kapusta, CEO, discussed exploring opportunities outside the U.S. and emphasizing patient and scientific community advocacy. Walid Abi-Saab, CMO, confirmed plans to amend the Statistical Analysis Plan for 4-year data submission to the FDA, which will include data from 12 patients at 4 years and all patients who reached 3 years, highlighting its importance for demonstrating durability and treatment effect.