Press Release: ISTH: Sanofi advances leadership in hemophilia with
new data for ALTUVIIIO and fitusiran
ISTH: Sanofi advances leadership in hemophilia
with new data for ALTUVIIIO and fitusiran
- Seven oral presentations across the
hemophilia portfolio reinforce Sanofi’s commitment to bring
potential first- and best-in-class treatments to the rare blood
disorders community
- Interim results from the long-term
XTEND-ed phase 3 study demonstrate once-weekly ALTUVIIIO continues
to provide highly effective bleed protection
- New ATLAS phase 3 study data
reinforce the potential of fitusiran to provide prophylaxis for
people with hemophilia A or B, with or without inhibitors
- New Drug Application for fitusiran
accepted for review by the US Food and Drug Administration, with a
PDUFA date of March 28, 2025
Paris, June 21, 2024. Sanofi
will present new data from its hemophilia portfolio at the 32nd
Congress of the International Society on Thrombosis and Haemostasis
(ISTH), taking place June 22-26, 2024, in Bangkok, Thailand.
Notable presentations on ALTUVIIIO [Antihemophilic Factor
(Recombinant), Fc-VWF-XTEN Fusion Protein] include long-term
interim phase 3 data on the efficacy and safety of the treatment in
adults and children with severe hemophilia A. Abstracts on
fitusiran include information on surgical experience as well as
long-term safety data from the ATLAS phase 3 clinical development
program in adults and adolescents with hemophilia A or B,
regardless of inhibitor status.
Dietmar BergerChief Medical
Officer, Global Head of Development “Our presence at this year’s
congress demonstrates our continued commitment to delivering
innovative first- and best-in-class solutions to the hemophilia
community. Hemophilia is a lifelong condition that significantly
impacts people living with the disease—from risk of bleeds and poor
joint health to increased risks during surgery. These data
reinforce why it’s critical to have treatment options, like
ALTUVIIIO and fitusiran, that deliver effective outcomes in
multiple scenarios and that can be used throughout a person’s life.
We look forward to working in partnership with regulatory agencies
to keep bringing novel options to those living with
hemophilia.”
ALTUVIIIOInterim analyses of XTEND-ed, a
long-term extension phase 3 study, showed that in adult and
pediatric populations, the use of ALTUVIIIO continued to provide
highly effective bleed prevention leading to improvement or
maintenance of joint health over a two-year period, and a safety
profile consistent with that reported in the initial studies. The
following abstracts will be presented at the meeting:
- “First Interim Analysis of
Clinical Outcomes in Adults and Adolescents With Severe Hemophilia
A Receiving Efanesoctocog Alfa Prophylaxis in XTEND-ed, a Phase 3
Long-term Extension Study”: In previously treated patients
(≥12 years old) who had the completed the XTEND-1 (Arm A/B) trial,
the mean annualized bleed rate (ABR) with ALTUVIIIO was 0.72
(standard deviation [SD])=1.26) for arm A and 0.42 (SD=0.89) for
arm B. No factor VIII inhibitors were detected (abstract
OC50.1).
- “Interim Analysis of Joint
Outcomes in Adult and Adolescent Patients with Severe Hemophilia A
Receiving Efanesoctocog Alfa During the Phase 3 XTEND-ed Long-Term
Extension Study”: In patients who continued to receive
once weekly ALTUVIIIO (50 IU/kg) in XTEND-ed, joint health had
improved or been maintained in adults and adolescents over a
two-year period, as measured by Hemophilia Joint Health Score total
score, total joint score, and subdomain scores (abstract
OC01.4).
- “Long-term Outcomes With
Efanesoctocog Alfa Prophylaxis for Previously Treated Children With
Severe Hemophilia A, an Interim Analysis of the Phase 3 XTEND-ed
Study": No factor VIII inhibitors were detected. The mean
ABR was 0.70 (SD=1.27), a rate similar to the mean ABR observed in
XTEND-Kids (abstract OC50.2).
Additional data being presented at ISTH show
that across clinical studies, ALTUVIIIO demonstrated effective
bleed protection when used for perioperative management in
participants with severe hemophilia A:
- “Perioperative Management
with Efanesoctocog Alfa in Adults, Adolescents, and Children with
Severe Hemophilia A in the Phase 3 XTEND Clinical
Program”: In 41 patients from the XTEND-1, XTEND-Kids, and
XTEND-ed studies who underwent 49 major surgeries, hemostasis was
maintained in all surgeries and hemostatic response with ALTUVIIIO
was rated as excellent in most surgeries (43/49) (abstract
OC14.1).
FitusiranAdditional analyses will be presented
at ISTH that support the potential of fitusiran as a first-in-class
treatment offering consistent bleed protection for patients with
hemophilia A or B, regardless of inhibitor status.
Novel results on the perioperative management of
hemophilia using fitusiran prophylaxis in the ATLAS clinical
development program demonstrated that major surgeries can be safely
performed in patients on fitusiran:
- “Surgical experience in
people with hemophilia A or B with and without inhibitors receiving
fitusiran”: 60 major surgeries, including 24 in
people with hemophilia with inhibitors, were conducted in the
fitusiran clinical development program at the time of this
analysis. Major surgeries were safely and effectively
conducted with fitusiran prophylaxis following bleed
management guidelines, irrespective of the patient’s inhibitor
status (abstract OC14.2).
Additional data presented at ISTH support the
favorable safety profile for fitusiran and demonstrate that
fitusiran prophylaxis under an antithrombin-based dosing regimen
(AT-DR) successfully mitigated the risk of thrombotic events (TEs)
and reduced the incidence of elevated liver enzymes and gallbladder
inflammation or gallstones.
The following abstracts will be presented at
ISTH:
- “Incidence of thrombotic
events in the fitusiran clinical development program”:
Fitusiran prophylaxis under an AT-DR led to a marked reduction in
TEs with substantially greater exposure on the AT-DR (abstract
OC40.2).
- “Hepatobiliary events in
the fitusiran clinical development program with the revised
AT-based dose regimen”: Fitusiran prophylaxis under
an AT-DR led to reductions in liver transaminase elevations and
cholecystitis/cholelithiasis events. Liver transaminase elevations
were infrequent and transient, and events of
cholecystitis/cholelithiasis resolved without clinical
complications with no fitusiran dose interruptions or
discontinuations (abstract OC40.3).
These presentations reinforce pivotal data that
were presented earlier this year from the phase 3 open-label
extension study (ATLAS-OLE) of fitusiran prophylaxis showing that
maintaining AT activity levels between 15-35% resulted in
clinically meaningful bleed control and a substantially improved
benefit risk profile in people with hemophilia A or B, with or
without inhibitors.
Regulatory submissions for fitusiran for the
treatment of hemophilia A or B in adults and adolescents with or
without inhibitors have been completed in China, Brazil and the US
with a US Food and Drug Administration (FDA) target action date of
March 28, 2025. The FDA also granted fitusiran Breakthrough Therapy
Designation for hemophilia B with inhibitors in December 2023.
About ALTUVIIIOALTUVIIIO [Antihemophilic Factor
(Recombinant), Fc-VWF-XTEN Fusion Protein] 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. In adults and adolescents,
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 within normal to near-normal
range, allowing for once-weekly administration. 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 other 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.
ALTUVIIIO is currently approved and marketed in
the US, Taiwan, and Japan. On June 17, 2024, it was approved by the
European Commission for the treatment and prevention of bleeds and
perioperative prophylaxis in hemophilia A under the name
Altuvoct.
ALTUVIIIO is the first factor VIII therapy to
receive Breakthrough Therapy Designation by the FDA in May
2022, Fast Track Designation in February 2021, and Orphan Drug
Designation in 2017. The European Commission granted Orphan
designation in June 2019.
About the XTEND-ed studyXTEND-ed (NCT04644575)
is a phase 3 multicenter, open-label three-arm study of the
long-term efficacy and safety of once-weekly ALTUVIIIO (50 IU/kg)
in previously treated patients with severe hemophilia A. The study
enrolled participants with severe hemophilia A from previous phase
3 studies, including adult and adolescent patients (≥12 years old)
who completed the XTEND-1 study (NCT04161495) and children (<12
years old) who completed the XTEND-Kids study (NCT04759131) (arm
A), newly initiated patients (arm B, China only), and newly
initiated patients with planned major surgery (arm C). Participants
received ALTUVIIIO prophylaxis for a total of 100 exposure days,
cumulative from the initial study (52 weeks) and this study.
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, Taiwan, and Japan. 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 SobiSobi is a specialized international
biopharmaceutical company transforming the lives of people with
rare and debilitating diseases. Providing reliable access to
innovative medicines in the areas of hematology, immunology, and
specialty care, Sobi has approximately 1,800 employees across
Europe, North America, the Middle East, Asia, and Australia. In
2023, revenue amounted to SEK 22.1 billion. Sobi's share (STO:SOBI)
is listed on Nasdaq Stockholm. More about Sobi at sobi.com and
LinkedIn.
About fitusiranFitusiran is currently under
clinical investigation, and its safety and efficacy have not been
evaluated by any regulatory authority. Fitusiran is a potential
first-in-class, antithrombin-lowering therapy for the prophylactic
treatment of people with hemophilia A or B, with or without
inhibitors. It is an investigational small volume, subcutaneously
administered small interference RNA (siRNA) therapeutic that aims
to prevent bleeds and rebalance hemostasis by lowering
antithrombin, a protein that inhibits blood clotting, to promote
thrombin generation. Fitusiran utilizes Alnylam Pharmaceutical
Inc.’s ESC-GalNAc conjugate technology, which enables subcutaneous
dosing with increased potency and durability. Fitusiran has the
potential to enable prophylaxis for people around the world living
with hemophilia A or B with or without inhibitors by virtue of its
low overall treatment burden, with as few as six small-volume
subcutaneous injections per year that do not require
refrigeration.
About the ATLAS clinical development programThe
efficacy and safety of fitusiran are being investigated in the
ATLAS clinical development program. The program includes the
completed phase 3 studies ATLAS-INH (NCT03417102), ATLAS-A/B
(NCT03417245), or ATLAS-PPX (NCT03549871) and the ongoing ATLAS-OLE
(NCT03754790) study, a single-arm, phase 3, open-label study
evaluating the safety and efficacy of fitusiran with a revised
AT-DR, which was designed to maintain an antithrombin target range
of 15%-35%. This study includes lower doses and less-frequent
dosing than earlier studies. ATLAS-NEO (NCT05662319) is an
additional phase 3 study currently recruiting participants to
assess the frequency of treated bleeding episodes with fitusiran
under the AT-DR in male adult and adolescent (≥12 years old)
participants with hemophilia A or B, with or without inhibitory
antibodies to factor VIII or IX, who have switched from their prior
standard-of-care treatment.
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 the world, 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
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