Study Design
DesignOpen-label, multicenter, three-arm
Enrollment165
Treatment Arms
Individualized prophylaxis 25-65 IU/kg every 3-5 days n=118
Weekly prophylaxis 65 IU/kg weekly n=24
On-demand 10-50 IU/kg per bleed n=23
[{"id":"along-ep1","name":"Annualized bleeding rate (ABR) on individualized prophylaxis","type":"PRIMARY","unit":"bleeds/year","results":[{"ci":"2.3-3.7","arm":"along-ind","label":"Mean ABR 2.9 (median Yr1: 1.6, Yr5: 0.5)","value":2.9},{"ci":"5.5-14.5","arm":"along-wk","label":"Mean ABR 8.9 (weekly)","value":8.9},{"arm":"along-od","label":"Mean ABR 37.3 (on-demand)","value":37.3}],"description":"Elocta is an extended half-life Factor VIII (Fc-fusion). The goal is reducing bleeds to near-zero while extending dosing intervals from every-other-day to every 3-5 days."}]
0 inhibitors in all 165 previously treated patients. No serious treatment-related AEs. In previously untreated patients (Kids A-LONG), ~33% inhibitor rate (consistent with class rate for any FVIII). Very clean safety profile.
Elocta is being cannibalized by Altuviiio (SOBI's own newer product) and competed by Hemlibra. Revenue declining in developed markets but still substantial in regions without Altuviiio/Hemlibra access. The SOBI Elocta-to-Altuviiio conversion is margin-accretive. Legacy product, not a driver.