Lydia

Lydia inquired about the split between non-transfusion and transfusion-dependent patients among the 44 ACTIVASE scripts and whether third-party services like IQVIA would accurately capture these scripts.

Answer

Tsveta Milanova, Chief Commercial Officer, indicated initial prescriptions are a mix of transfusion-dependent (larger proportion) and engaged symptomatic non-transfusion-dependent patients, expecting more transfusion-dependent initially, then scale-up from non-transfusion-dependent. She clarified that IQVIA would not be an accurate source due to a single specialty pharmacy, and Agios would provide direct prescription and revenue information.

Lydia asked for additional color on the split between non-transfusion and transfusion-dependent patients among the 44 ACTIVASE scripts and whether third-party services like IQVIA would accurately capture these scripts.

Answer

Chief Commercial Officer Tsveta Milanova explained that initial prescriptions are a combination of transfusion-dependent and engaged symptomatic non-transfusion-dependent patients, with transfusion-dependent patients initially forming a larger proportion, but non-transfusion-dependent patients driving later scale-up. She clarified that IQVIA would not be an accurate source for script tracking due to the single specialty pharmacy distribution model, and that Agios would provide prescription and revenue data.

Lydia asked about any statistical work or study design changes made for the osteogenesis imperfecta program following the second interim analysis.

Answer

Howard Horn, Chief Financial Officer, stated he was not aware of any changes. Eric Crombez, Chief Medical Officer, confirmed that the statistical plan remains in place, and internal modeling and rework reinforced confidence in the existing plan for the final data readout.