Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (“Takeda”), the global biotechnology leader in rare diseases, has announced results from its phase IIIb/IV clinical trial for ADYNOVATE at the 12th Annual Congress of the European Association of Haemophilia and Allied Disorders (EAHAD), taking place 6-9 February, in Prague, Czech Republic. 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 Levels in subjects with severe hemophilia A (see also Takeda Pharmaceutical Company Limited).
The study showed that ADYNOVATE prophylaxis targeting 8-12% (HIGH) vs 1-3 % (LOW) trough levels was associated with a trend toward a higher proportion of patients with a total annualized all bleed rate (ABR)=0 (66% HIGH vs 39% LOW; p=0.075). The HIGH group was also associated with a trend toward a lower total ABR, as well as a higher proportion of patients with all annualized joint bleed rate (AJBR)=0 (90% HIGH vs 68% LOW) and all spontaneous ABR=0 (84% HIGH vs 61% LOW). The data suggests that optimizing FVIII profiles through PK-driven dosing that targets trough levels 8-12% was consistently achievable and further treatment personalization for patients with hemophilia A should be considered. Safety profiles were comparable and consistent with previous ADYNOVATE trials. Ongoing analyses will characterize the relationship between PK-tailored dosing of ADYNOVATE FVIII levels and bleeding events.
“The study reiterates the importance of personalized prophylaxis for those living with hemophilia,” said Dr. med. Robert Klamroth, Head of the Department of Internal Medicine Angiology and Coagulation Disorders and Director of the Comprehensive Care Haemophilia Treatment Center and the Haemostasis and Thrombosis Unit at the Vivantes Klinikum in Berlin, Germany. “The findings suggest the need to measure actual FVIII levels and show a clear trend that if we’re able to keep FVIII levels in a higher range, there may be better patient outcomes, including enhanced bleed protection that could help more patients reach zero bleeds.”
“At Takeda, we are proud of the hematology heritage Shire and Baxalta built over 60 years and we plan on expanding on it through continued research and innovation, including continued focus on direct factor replacement,” said Dr. med. Wolfhard Erdlenbruch, Vice President Head of Global Medical Affairs Hematology, Takeda. “We are excited to be at EAHAD and share the results of PROPEL study. The zero all bleed rates and zero all spontaneous joint bleeds in this study have not been reported previously with people living with hemophilia A. This supports that factor levels matter and brings us a step closer to achieving our goal of optimized and personalized patient care, because every bleed matters.”
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