Takeda New Data Back Benefit for Hemophilia A Personalized Prophylaxis

– Updated results from the Phase IIIb/IV PROPEL Study show that pharmacokinetic (PK)-driven dosing may be used to achieve FVIII target trough levels of 8–12%; and that selecting a patient-appropriate target FVIII level plus adjusting a dosing regimen to that patient’s PK characteristics, can improve the overall PK profile and may enhance outcomes, with no adverse event profile change – thus reinforcing the importance of PK-guided dosing and the potential benefit of personalized prophylaxis with ADYNOVATE¹

– Data presented alongside 47 other ISTH 2019 presentations showcasing the latest developments from Takeda’s hematology gene therapy pipeline and leading Factor portfolio

– Takeda’s robust presence at ISTH underscores its commitment to progressing scientific advancements for the bleeding disorders community

Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (“Takeda”), R&D-driven, global biopharmaceutical company with a leadership position in rare diseases, has today announced updated results from its phase IIIb/IV 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.

The latest results of the landmark 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].¹ 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).
• Reduced mean spontaneous joint ABR (0.5 ELE versus 2.0 REF)

The data supports the view that patients may benefit from PK-driven dosing that targets FVIII trough levels of 8–12%. The safety findings from this latest update were also comparable and consistent with previous ADYNOVATE trials.^1,2 Ongoing analyses will further characterize the relationship between PK-tailored dosing of ADYNOVATE FVIII levels and bleeding events.

Adapting the dosing regimen for an individual patient, guided by that patient’s individual PK characteristics, has great potential – for managing patients with hemophilia A, particularly those desiring greater bleed protection.

https://www.marketscreener.com/TAKEDA-PHARMACEUTICAL-CO-6491073/news/Takeda-Pharmaceutical-Unveil-New-Data-from-the-PROPEL-Study-at-ISTH-2019-Reinforcing-the-Potentia-28864713/