Press Release: Pivotal data demonstrate once-weekly efanesoctocog alfa provides superior bleed protection compared to prior factor prophylaxis

Pivotal data demonstrat e once-weekly efanesoctocog alfa provides superior bleed protection compared to prior factor prophylaxi s Investigational o nce - w eekly e fanesoctocog alfa prophylaxis met the primary efficacy endpoint providing clinically meaningful bleed protection for people with sever e hemophilia A R esults underscore th e ability of efanesoctocog alfa to sustain normal to near-normal factor levels and the potential to transform prophylactic treatment, providing people with hemophilia A with higher protection for longer Additional data showed e fanesoctocog alfa prophylaxis resulted in statistically significant and clinically meaningful improvements in physical health , pain intensity and joint health in patients on prior factor V III prophylaxis Paris and Stockholm – July 10 , 2022 – Sanofi and Swedish Orphan Biovitrum AB (publ) (Sobi® ) (STO:SOBI) presented for the first time today, in a late-breaking session at the 30th International Society on Thrombosis and Haemostasis (ISTH) Congress , positive results from the XTEND-1 pivotal Phase 3 study evaluating the safety, efficacy and pharmacokinetics of efanesoctocog alfa (BIVV001), an investigational factor VIII replacement therapy, in previously treated adults and adolescents ≥12 years with severe hemophilia A. The study met the primary efficacy endpoint, with once-weekly efanesoctocog alfa prophylaxis providing clinically meaningful bleed protection for people with severe hemophilia A. The median and mean annualized bleeding rates (ABR) were 0.00 (IQR: 0.00-1.04) and 0.71 (SD: 1.43) respectively. The study also met the key secondary endpoint, demonstrating superior bleed protection (p<0.0001) over="" prior="" factor="" viii="" prophylaxis="" with="" an="" estimated="" abr="" reduction="" of="" 77%="" and="" a="" mean="" 0.69="" compared="" to="" 2.96="" on="" prophylaxis,="" based="" intra-patient="" comparison="" (n="78)." in="" subset="" participants="" studied="" at="" baseline="" week="" 26,="" levels="" remained="" the="" normal="" near-normal="" range="" (="">40 IU/dL) for the majority of the week, and at 15 IU/dL at Day seven post-dose, providing increased factor activity level protection for patients with once-weekly prophylaxis. Annette von Drygalski, MD, PharmD Investigator, Professor and Director, Hemophilia and Thrombosis Treatment Center, UC San Diego " The phase 3 data demonstrate once - weekly efanesoctocog alfa 's potential to provide superior bleed protection , leading to substantial improvements in physical health, pain and joint health , by sustaining high factor levels for the majority of the week . These unprecedented results may offer people with hemophilia A the possibility to redefine their treatment expectations ." Data show adults and adolescents treated with once-weekly efanesoctocog alfa experienced statistically significant and clinically meaningful improvements in physical health (p=0.0001), pain intensity (p=0.0276), and joint health (p=0.0101) when comparing week 52 and baseline measurements.i Moreover, efanesoctocog alfa was effective at treating bleeds, including in target joints; 96.7% of bleeds were resolved with a single 50 IU/kg dose. Efanesoctocog alfa was well tolerated and inhibitor development to factor VIII was not detected. The most common treatment-emergent adverse events (>5% of participants overall) were headache, arthralgia, fall, and back pain. Dietmar Berger, MD, PhD Global Head of Development and Chief Medical Officer, Sanofi " We are committed to advancing innovati ve medicines that disrupt the status-quo and address the unmet needs that persist for people with rare conditions like hemophilia. T h ese robust data illustrate the promise of efanesoctocog alfa 's efficacy with once - weekly dosing and underscore it s potential as a therapy with best-in-disease efficacy ." Anders Ullman , MD, PhD Head of R&D and Chief Medical Officer, Sobi "We believe transforming the treatment paradigm for hemophilia A can only be achieved through elevating standards of care towards normal hemostasis . These data demonstrate the profile of efanesoctocog alfa in significant clinical terms, and further strengthen its potential to ultimately improve the lives of many ... Full story available on Benzinga.com