Takeda provides updates on its Pharmacokinetic-guided Prophylaxis Studies at ISTH 2020, reinforcing its commitment to advancing personalized care for people with bleeding disorders

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TAKEDA Pharmaceutical Company Limited has announced that 13 abstracts are being presented from the company’s Hematology portfolio and pipeline at the International Society on Thrombosis and Haemostasis 2020 Virtual Congress, ISTH 2020.

Among the data, Takeda spotlighted four abstracts to highlight its commitment to advancing personalized care through pharmacokinetic, PK-guided prophylaxis in people living with hemophilia or von Willebrand Disease, VWD, –including scientific updates in patients with hemophilia A from the Phase 3 PROPEL and Phase 3b CONTINUATION studies investigating potential benefits of personalized TAK-660 (rurioctocog alfa pegol) prophylaxis.

Two population studies into the PK/pharmacodynamics, PD, profiles of recombinant von Willebrand factor (rVWF), which provide data to assist in the optimization of rVWF personalized dosing strategies, were also presented.

Takeda addresses the unique needs of each patient with personalized care Takeda presented a total of 13 abstracts at ISTH 2020, available on the ISTH Congress Abstracts Site. Among them, Takeda spotlighted four abstracts which support its personalized care approach for patients with bleeding disorders, especially hemophilia and rVWD.

Prophylaxis for hemophilia A can help prevent spontaneous bleeds because even a single bleed may be life-threatening or contribute to permanent joint damage. I,ii Factor VIII (FVIII) prophylaxis can be tailored to the individual’s PK profile using multiple parameters of the PK curve, enabling adjusted dosing to achieve predictable FVIII levels to minimize bleeding risk.

Personalized treatment in hemophilia A aiming for higher trough levels by PK-guided dosing may help to optimize some outcomes by tailoring treatment for individual patient needs.

Supporting data presented at ISTH 2020 include:

Target joint resolution analysis from the PROPEL trial in hemophilia A iii [Abstract

PB0921]

Data from a post-hoc analysis of the Phase III PROPEL trial evaluating the efficacy and

target joint (TJ) resolution with PK-guided TAK-660 prophylaxis in patients with hemophilia A show that targeting 8–12% versus 1–3% FVIII trough levels were associated with reduced bleeding into TJs, reduced annualized bleed rate (ABR) irrespective of baseline TJ status, and TJ resolution within the first 6 months.

  • Personalizing prophylaxis with TAK-660 in Hemophilia A iv [Abstract PB0920]

An analysis exploring the potential benefits of personalized TAK-660 prophylaxis evaluated data on extended fixed dose or PK-tailored dosing from two respective studies – the Phase IIIb CONTINUATION and the Phase III PROPEL studies. Results from the two studies support the feasibility and efficacy of personalized TAK-660 prophylaxis by extending the fixed dose interval or by targeting elevated FVIII levels with PK-tailored dosing.

 

“Because every patient is unique, there is no ‘one-size-fits-all’ approach to hemophilia care. We are committed to improving outcomes and quality of life for each person living with a bleeding disorder,” commented Dr Carmen Escuriola-Ettingshausen, Co-director, Haemophilia Centre Rhein-Main – HZRM, Frankfurt-Mörfelden, Germany, one of the researchers leading the CONTINUATION and PROPEL studies. “The data presented at ISTH 2020 demonstrate the value that innovative, personalized approaches provide to the management of hemophilia.”

On the other hand, for patients with VWD, rVWF replacement therapy corrects VWF activity, as measured mostly by VWF:Ristocetin Cofactor (RCo), and leads consecutively to an endogenous increase of FVIII activity to hemostatically effective levels. The goals of Takeda’s PK studies in VWD are to understand the effects of rVWF dosing on VWF:RCo and FVIII activities and thus help patients living with VWD and their physicians achieve agreed treatment goals.

  • Population PK and PD models for rVWF in VWD v [Abstract PB1541]

Results from the modelling supported the feasibility of this PK/PD population model for describing VWF:RCo and FVIII activity in rVWF-treated patients with different VWD types, which could assist in the optimization of rVWF personalized dosing strategies.

The population PK/PD models aimed to characterize VWF:RCo and endogenous FVIII activity-time curves following administration of rVWF, based on data from Phase I and Phase III studies. · Exploring the relationship between multimeric pattern and VWF:RCo activity vi [Abstract PB1544]

Results from the modelling supported the feasibility of this PK population model for describing activity-time profiles of small, medium and large multimers following VFW dosing on VWF:RCo. The findings increase understanding of the effects of VFW dosing on VWF:RCo and FVIII activities which could potentially optimize treatment regimens for individual patient needs.

Takeda is committed to creating a world without bleeds

“Findings presented at ISTH 2020 reaffirm Takeda’s commitment to support personalized care as the optimal treatment approach for patients with bleeding disorders,” added Dr. med.Wolfhard Erdlenbruch, M.D., Vice President Head of Global Medical Affairs Hematology, Takeda. “Some of our data may be especially informative when considering the treatment of patients with hemophilia A requiring higher FVIII activity trough levels because of their active lifestyles and increased risk of injury-related bleeds. What we have presented at ISTH 2020 support the rationale for a personalized approach to prophylaxis for the management of both hemophilia and VWD, and have the potential to help the medical community change their patients’ lives for the better.”

In addition to data from the four aforementioned studies, Takeda presented real-world evidence (RWE) of extended half-life (EHL) prophylaxis with TAK-660 [Abstract PB0919], which showed that switching from standard half-life (SHL) FVIII to TAK-660 given at lower frequency and with weekly consumption resulted in reduced ABRs.

Results were also presented from the American Thrombosis and Hemostasis Network ATHN 2: Factor Switching Study [Abstract PB1049], which show that for the 52 participants in the study who switched from any FVIII concentrate to TAK-660, no new inhibitors were detected after 50 exposure days or 12 months. viii

A further seven studies presented included a retrospective chart review of gastrointestinal bleeding in VWD [Abstract PB1555], and pre-clinical and post-marketing updates on susoctocogalfa for the treatment of acquired hemophilia [Abstract PB0779].

– Jul. 13, 2020 @ 10:15 GMT |

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