Takeda Pharmaceuticals announced updated results from its phase 3/4 PROPEL clinical trial for ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated] at the 27th Annual International Society on Thrombosis and Haemostasis Congress (ISTH), in Melbourne, Australia.
The PROPEL study is a PROspective, randomized, multi-center study comparing the safety and efficacy of ADYNOVATE following PK-guided prophylaxis targeting two different Factor Eight (FVIII) trough activity Levels in subjects with severe hemophilia A. Eligible subjects had FVIII activity <1%, annualized bleed rate (ABR) ≥2, and transitioned from a previous SHP660 (ADYNOVATE) study or were 12–65 years old with ≥150 exposure days to plasma-derived or recombinant FVIII. After initial PK assessments, subjects were randomized to receive 12 months of PK-guided prophylaxis targeting FVIII trough levels of 1–3% (REF) or 8–12% (ELE) (1st 6 months: dose adjustment period). The primary outcome was the percent of subjects with a total ABR=0 (all bleeds) during the 2nd 6-month study period.
The updated results of the PROPEL study show that ADYNOVATE prophylaxis in severe hemophilia A patients may enhance a patient’s PK profile – by targeting FVIII trough levels of 8–12% (elevated prophylaxis arm, ELE) as compared with 1–3% (reference prophylaxis arm, REF). This represents a clinically meaningful trend towards more patients experiencing zero bleeds [62% ELE versus 42% REF, respectively; p=0.0545]. Patients randomized to the 8-12% target group also saw a reduced mean total annualized bleed rate (ABR); (1.6 ELE versus 3.6 REF, respectively) and a reduced mean spontaneous joint ABR (0.5 ELE versus 2.0 REF). The most common adverse reactions were headache, diarrhea, nausea and rash.
About Hemophilia A
Hemophilia is a chronic disease that causes longer-than-normal bleeding due to absent or deficient clotting factor in the blood. Hemophilia A is more common than hemophilia B, and affects about 158,225 people, whereas hemophilia B affects about 31,247 people worldwide. Treatment regimens typically include on-demand and/or regular prophylactic infusions of factor replacement therapy to control or prevent the risk of bleeding.
About ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated
ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated temporarily replaces the missing coagulation factor VIII that is needed for effective hemostasis.