Takeda Data at ISTH 2021 Highlight the Benefits of Prophylaxis for Patients with Rare Bleeding Disorders

Takeda Data at ISTH 2021 Highlight the Benefits of Prophylaxis for Patients with Rare Bleeding Disorders

• Data Include Phase 3 Results Showing that the Primary Endpoint of Spontaneous, Treated Annualized Bleeding Rates During Prophylaxis with Recombinant von Willebrand Factor in Adults with von Willebrand Disease, was Met
• 12 Abstracts Presented Across Takeda’s Hematology Portfolio and Pipeline Support Takeda’s Commitment to Improving Patient Care Today as well as Transforming Future Care

OSAKA, Japan, July 20, 2021 – Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE:TAK) (“Takeda”) today announced the results of a phase 3 trial investigating the efficacy and safety of recombinant von Willebrand factor (rVWF) prophylaxis,¹ one of the 12 abstracts being presented at the International Society on Thrombosis and Haemostasis (ISTH) Virtual Congress 2021. Several of these abstracts explored the use of prophylaxis as part of Takeda’s commitment to improving outcomes for patients with rare bleeding disorders.

The prospective, phase 3, open-label, international multicenter study included 23 patients with severe von Willebrand disease (VWD). The study period was 12 months and included two arms; the prior on-demand (OD) group included patients previously taking OD VWF and the switch group included patients previously taking prophylactic plasma-derived von Willebrand factor (pdVWF) treatment [LPB0128]. Results showed that prophylaxis with rVWF effectively reduced spontaneous, treated annualized bleeding rates (sABRs) in patients previously treated OD, and the same level of hemostatic control was maintained in patients who switched from prophylaxis with pdVWF. The sABR was reduced by 91.5% on study (while receiving prophylactic rVWF) compared with historical sABR in prior OD patients, and sABR was maintained on-study in switch patients (sABR on-study: historical ratio (95% CI): 0.09 (0.02, 0.35) in prior OD arm; 0.55 (0.09, 3.52) in switch arm).  No new risks were identified, with no serious adverse events related to rVWF reported.¹ The pharmacokinetics (PK) of VWF:ristocetin cofactor (VWF:RCo) and FVIII pharmacodynamics (PD) [PB0917] were also studied and presented at the congress.²

“Stopping a bleed in VWD does not prevent subsequent bleeds, and the complications and unpredictable nature of these bleeds for many OD treated patients can impact their daily life,” commented Dr. Flora Peyvandi, Director of the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Ca’ Granda Ospedale Maggiore, Policlinico, University of Milan, Italy. “These exciting phase 3 results are important in improving understanding about the potential benefit of prophylaxis with rVWF and what this may offer patients with VWD in reducing spontaneous bleeds in the future.”

A literature review being presented at ISTH 2021 also studied the rates of bleeding-related complications, quality of life and healthcare resource utilization in VWD [PB0924].³ Data gathered from a further retrospective cohort study investigating the characteristics and management of patients with VWD in UK general practice discussed the occurrence of bleeding events in patients with a mild disease status [PB0927].⁴

Additional investigational data across Takeda’s hematology portfolio assessed the efficacy and safety of PK-guided prophylaxis in patients with hemophilia A. Results from the real-world German AHEAD study, which aims to examine long-term effectiveness and safety of antihemophilic factor (recombinant), rAHF (ADVATE),⁵ help to provide an understanding of the bleeding rates in patients with hemophilia A receiving PK-guided prophylaxis versus standard prophylaxis [PB0509].⁶ PK-guided prophylaxis was further studied in another real-world analysis using data from the CHESS II database to assess the impact on clinical and health resource utilization outcomes [PB0554].⁷ Data from a post hoc analysis of the PROPEL phase 3 study on the effects of PK-guided prophylaxis targeting specific FVIII trough levels with rurioctocog alfa pegol (ADYNOVATE) were also presented at the congress [PB0542].⁸

“At Takeda we are committed to helping make a difference to the lives of patients with rare hematological disorders, both today through our broad portfolio of treatments, and in the future by developing innovative solutions to address unmet needs,” commented Wolfhard Erdlenbruch, MD, Head, Global Medical Affairs Hematology. “The data presented at ISTH 2021 highlights the potential for rVWF to address unmet needs in VWD, as well as helping us to understand the clinical and resource utilization impact when individualizing treatment through PK-guided prophylaxis in hemophilia.”

Also featured at the congress was the phase 3 study design exploring TAK-755, an investigational recombinant ADAMTS13 replacement therapy being studied for use as prophylactic and OD treatment for patients with severe congenital thrombotic thrombocytopenic purpura (cTTP) [PB0837],⁹ and an abstract which reviewed the clinical burden of TTP [PB0843].¹⁰ Improving awareness and understanding around this rare and life-threatening blood disorder associated with ADAMTS13 deficiency,⁹ together with the development of TAK-755, forms part of Takeda’s commitment to invest in research that has the potential to transform the future care of even more patients affected by rare hematological disorders.