Completed XTEND-Kids Phase 3 study strengthens potential of
ALTUVIIIO™ to redefine expectations for treatment of children
<12 years of age with hemophilia A
Paris,
March 2,
2023. The XTEND-Kids phase 3 pivotal
study evaluating the safety, efficacy and pharmacokinetics of
ALTUVIIIO as once-weekly prophylaxis in previously treated patients
<12 years of age with severe hemophilia A met its primary
endpoint of safety, with no FVIII inhibitors detected in 74
children, with more than 50 children experiencing at least 50
exposure days, nearly a full year of treatment. The completion of
XTEND-Kids represents the final milestone needed for regulatory
submission in the EU.
Karin Knobe,
MD, PhDTherapeutic Area Head, Rare Diseases and
Rare Blood Disorders, Sanofi “At Sanofi, we never settle. We work
alongside patients, caregivers, and advocacy organizations to
understand the needs of the hemophilia community and pursue
first-in-class technologies to meet those needs. We strive for a
future where every child with hemophilia can play without fear,
travel free from a rigid treatment schedule, and pursue their
dreams unencumbered by worry.”
Hemophilia A is a rare, lifelong condition in which
the ability of a person’s blood to clot properly is impaired,
leading to excessive bleeds and spontaneous bleeds into joints that
can result in joint damage and chronic pain, and potentially impact
quality of life. The severity of hemophilia is determined by the
level of clotting factor activity in a person’s blood, and there is
a negative correlation between risk of bleeding and factor activity
levels.
ALUTVIIIO is a first-in-class, high-sustained FVIII
therapy approved by the US Food and Drug Administration (FDA) for
routine prophylaxis, on-demand treatment and control of bleeding
episodes, and perioperative management of bleeding in adults and
children in February 2023. Granted Breakthrough Therapy designation
by the FDA in May 2022 – the first FVIII therapy to receive this
designation—ALTUVIIIO also received Fast Track designation in
February 2021 and Orphan Drug designation in 2017. The European
Commission granted Orphan Drug designation in June 2019.
About XTEND-Kids The XTEND-Kids study (NCT04759131)
was an open-label, non-randomized interventional study of the
safety, efficacy, and pharmacokinetics of once-weekly ALTUVIIIO in
previously treated patients younger than 12 years of age with
severe hemophilia A. Patients received once-weekly ALTUVIIIO
prophylaxis (50 IU/kg) for 52 weeks which provided high-sustained
FVIII levels throughout the weekly dosing interval with a median
(IQR) annualized bleeding rate (ABR) of 0.00 (0.00, 1.02) and an
estimated mean (95% CI) ABR of 0.89 (0.56 ; 1.42). The primary
endpoint was the occurrence of inhibitor development (baseline to
52 weeks). No inhibitors were detected in this study.
About ALTUVIIIO™ALTUVIIIO [Antihemophilic Factor
(Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl] is a first-in-class
high-sustained factor VIII therapy that is designed to extend
protection from bleeds with once-weekly prophylactic dosing for
adults and children with hemophilia A. ALTUVIIIO has a 3 to 4 fold
longer half-life relative to standard and extended half-life factor
VIII products, providing high-sustained factor activity levels
throughout (≥40%) for most of the week and at 15% at the end of the
dosing interval. ALTUVIIIO is the first factor VIII therapy that
has been shown to break through the von Willebrand factor ceiling,
which imposes a half-life limitation on earlier generation factor
VIII therapies. ALTUVIIIO builds on the innovative Fc fusion
technology by adding a region of von Willebrand factor and
XTEN® polypeptides to extend its time in circulation. XTEN® is
a registered trademark of Amunix Pharmaceuticals, Inc.
About the XTEND Clinical ProgramsThe XTEND clinical
program is comprised of two Phase 3 trials in hemophilia A: XTEND-1
in people 12 years or older and XTEND-Kids in children younger than
12 years old. There is also an ongoing extension study
(XTEND-ed).
The Phase 3 XTEND-1 study
(NCT04161495) was an open-label, non-randomized
interventional study assessing the safety, efficacy, and
pharmacokinetics of once-weekly ALTUVIIIO in people 12
years of age or older (n=159) with severe hemophilia A who were
previously treated with factor VIII replacement therapy. The
study consisted of two parallel treatment arms — the prophylaxis
Arm A (n=133), in which patients who had received prior factor VIII
prophylaxis were treated with once-weekly
intravenous ALTUVIIIO prophylaxis (50 IU/kg) for 52
weeks, and the on-demand Arm B (n=26), in which patients who had
received prior on-demand factor VIII therapy began with
26 weeks of on-demand ALTUVIIIO (50 IU/kg), then switched
to once-weekly prophylaxis with ALTUVIIIO (50 IU/kg) for an
additional 26 weeks.
The primary efficacy endpoint of XTEND-1 was the
mean annualized bleeding rate (ABR) in Arm A, and the key
secondary endpoint was an intra-patient comparison of ABR during
the ALTUVIIIO weekly prophylaxis treatment period versus the
prior factor VIII prophylaxis ABR for a subset of
participants in Arm A who had participated in a
previous observational study (Study 242HA201/OBS16221).
The XTEND-Kids study (NCT04759131) was an
open-label, non-randomized interventional study of the safety,
efficacy, and pharmacokinetics of once-weekly ALTUVIIIO in
previously treated patients younger than 12 years of age with
severe hemophilia A. Patients received once-weekly ALTUVIIIO
prophylaxis (50 IU/kg) for 52 weeks. The primary endpoint was the
occurrence of inhibitor development.
About the Sanofi and Sobi collaborationSobi and
Sanofi collaborate on the development and commercialization of
Alprolix® and Elocta®/Eloctate®. The companies also collaborate on
the development and commercialization of efanesoctocog alfa, or
ALTUVIIIO in the US. Sobi has final development and
commercialization rights in the Sobi territory (essentially Europe,
North Africa, Russia and most Middle Eastern markets). Sanofi has
final development and commercialization rights in North America and
all other regions in the world excluding the Sobi territory.
About Sobi®Sobi is a specialised international
biopharmaceutical company transforming the lives of people with
rare and debilitating diseases. Providing reliable access to
innovative medicines in the areas of haematology, immunology and
specialty care, Sobi has approximately 1,600 employees across
Europe, North America, the Middle East, Asia and Australia. In
2022, revenue amounted to SEK 18.8 billion. Sobi’s share (STO:SOBI)
is listed on Nasdaq Stockholm. More about Sobi
at sobi.com, LinkedIn and YouTube.
About SanofiWe are an innovative global healthcare
company, driven by one purpose: we chase the miracles of science to
improve people’s lives. Our team, across some 100 countries, is
dedicated to transforming the practice of medicine by working to
turn the impossible into the possible. We provide potentially
life-changing treatment options and life-saving vaccine protection
to millions of people globally, while putting sustainability and
social responsibility at the center of our ambitions. Sanofi is
listed on EURONEXT: SAN and NASDAQ: SNY
Media RelationsSandrine
Guendoul | + 33 6 25 09 14 25
| sandrine.guendoul@sanofi.comSally
Bain | + 1 617 834 6026
| sally.bain@sanofi.comEvan Berland | +1 215
432 0234 | evan.berland@sanofi.comKate
Conway | + 1 508 364 4931
| kate.conway@sanofi.comVictor
Rouault | + 33 6 70 93 71 40
| victor.rouault@sanofi.com
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| eva.schaefer-jansen@sanofi.comArnaud
Delépine | + 33 6 73 69 36 93 |
arnaud.delepine@sanofi.comCorentine
Driancourt | + 33 6 40 56 92 21 |
corentine.driancourt@sanofi.comFelix
Lauscher | + 1 908 612 7239 |
felix.lauscher@sanofi.comTarik Elgoutni| + 1 617
710 3587 | tarik.elgoutni@sanofi.comNathalie
Pham | + 33 7 85 93 30 17 |
nathalie.pham@sanofi.com
Sobi Contacts:Media RelationsFor Sobi Media
contacts, click here.
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Sobi Investor Relations Team, click here.
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