Takeda a suspendu la sélection et le recrutement des études cliniques de ses thérapies géniques AAV8 pour le traitement de l’hémophilie A et B. Takeda a indiqué dans son courrier adressé à la FMH que la décision ne résultait pas de préoccupations liées à la sécurité concernant TAK-754 et TAK-748. « Nous évaluons actuellement les données provisoires de l’étude de phase I/II en cours portant sur TAK-754 dans l’hémophilie A, parallèlement au paysage actuel global de la thérapie génique dans l’hémophilie, » a expliqué Takeda. Les patients recrutés resteront sous observation clinique étroite conformément au protocole de l’étude et ceux qui devaient être inclus dans ces études feront l’objet d’une transition. Aucun patient n’a encore reçu de traitement dans l’étude de phase I/II de TAK-748 dans l’hémophilie B. Takeda tiendra la communauté informée dès lors qu’il aura terminé son évaluation.
Nous reprenons ici le courrier de Takeda (en anglais) :
19 August 2020
TAK-754 and TAK-748 Clinical Studies
As one of our partners we work closely with, we are writing today to share an important update with you on Takeda’s gene therapy in hematology. Takeda has recently made the decision to suspend screening and further enrollment in the TAK-754 and TAK-748 clinical studies, effective immediately.
As you may know, TAK-754 is an AAV8 gene therapy for the treatment of hemophilia A currently in Phase 1/2 clinical development, while TAK-748 is an AAV8 gene therapy for the treatment of hemophilia B in Phase 1/2 study start-up (no subject dosed as of today). In order to determine the most appropriate way forward for both TAK-754 and TAK-748, we are currently assessing the interim data from the ongoing Phase 1/2 study of TAK-754 in hemophilia A, together with the overall current gene therapy landscape in hemophilia. We will communicate more details on these results to the community in due time once our assessment has been finalized. It is important to note that this decision is not a result of any safety concerns with TAK-754 and TAK-748.
We have informed those investigators who are working directly on these studies. Our priority first and foremost, is to ensure the safety and wellbeing of patients. Those who are enrolled will continue to be under close clinical observation according to study protocol, and those who were planned to be enrolled into these studies will be informed and transitioned. We would like to emphasize that we remain committed to advancing gene therapies and very much believe in their potential. Our strategy is focused on delivering novel and transformative medicines to patients, and we will continue to work with our gene therapy platforms with the aim of delivering treatments in the future that can help make a meaningful difference in those living with hemophilia and other rare hematological diseases. We hope to continue the dialogue with your organization to help us inform that innovative focus.
We are proud to be a long-standing leader in bleeding disorders, and of our relation with the World Federation of Hemophilia of almost 30 years, which is grounded in our common goal to improve standards of care around the world. Takeda’s vision for a bleed-free world continues to fuel our passion every day to make a difference in the lives of those affected by bleeding disorders. Working together we hope to achieve it.