Press Release: Fitusiran prophylaxis reduced bleeds by 61% in
people with hemophilia A or B, with or without inhibitors, compared
to prior factor or bypassing agent prophylaxis
Fitusiran prophylaxis reduced bleeds by 61% in people with
hemophilia A or B, with or without inhibitors, compared to prior
factor or bypassing agent prophylaxis
- A median annualized
bleeding rate (ABR) of 0.0 was reported in the overall study
population during fitusiran prophylaxis (80 mg monthly)
- Fitusiran is a
novel, investigational subcutaneously administered small
interference RNA therapy, in development for the prophylactic
treatment of people with hemophilia A or B, with or without
inhibitor
Paris –
July 10, 2022 –
Positive data from the Phase 3 ATLAS-PPX study evaluating the
efficacy and safety of once-monthly fitusiran (80 mg) in adults and
adolescents with severe hemophilia A or B who were previously
treated with prior factor or bypassing agent (BPA) prophylaxis were
presented today in a late-breaking session at the International
Society on Thrombosis and Haemostasis (ISTH) 2022 Congress. The
study met the primary endpoint and demonstrated fitusiran
prophylaxis significantly reduced bleeding episodes compared to
prior factor or BPA prophylaxis.
Gili Kenet,
MDInvestigator, professor of Hematology, Director of the
Israeli National Hemophilia Center at Sheba Medical Center and head
of the Amalia Biron Thrombosis Research Institute of Tel Aviv
University, Tel Aviv, Israel
“There is a continued need for transformative
therapies that offer people with hemophilia consistent protection
while also reducing treatment burden. These phase 3 results are
encouraging and support fitusiran’s potential to provide people
with hemophilia A or B, regardless of inhibitor status, with a
meaningful reduction in bleeding episodes.”
Key findings in the Phase 3 ATLAS-PPX study
include the following:
- The overall
median annualized bleeding rate (ABR) was 0.0 for fitusiran
prophylaxis, compared to a median ABR of 4.4 with prior
prophylaxis.
- Fitusiran
prophylaxis resulted in a statistically significant reduction in
estimated ABR of 61.1% (p= 0.0008) versus factor or BPA
prophylaxis.
- 63.1% (n=41) of
adults and adolescents treated with fitusiran experienced zero
treated bleeds compared to 16.9% (n=11) with prior factor or BPA
prophylaxis.
- Median ABR for
treated bleeds was 0.0 with fitusiran prophylaxis for both
participants with and without inhibitors compared to 6.5 and 4.4
for participants with and without inhibitors, respectively, on
prior prophylaxis.
- Of the 67
participants exposed to a least one dose of fitusiran, the most
common adverse events (≥6 participants) were increased alanine
aminotransferase, nasopharyngitis, and upper respiratory tract
infection.
- Consistent with
the previously identified risk of fitusiran, suspected or confirmed
thromboembolic events were reported in 2 participants (3.0%).
Dietmar Berger MD, PhDGlobal
Head of Development and Chief Medical Officer
“These positive data support fitusiran’s
potential to transform prophylaxis treatment for people with
hemophilia A or B, with or without inhibitors, with a median annual
bleed rate of zero across all patient populations. Moreover, we are
excited to continue to explore fitusiran under an amended protocol
that focuses on dose optimization, including lower doses and less
frequent dosing regimens, with the potential for as few as six
injections per year.”
Additional data from the fitusiran clinical
program will be shared at the congress including:
- Consumption of
On-demand Factor Concentrates and Bypassing Agents for Management
of Breakthrough Bleeds with Fitusiran Prophylaxis in People with
Haemophilia A or B: An Analysis of Two Phase 3 Studies.
Oral presentation: OC40.3. July
11, 2:45 pm – 4:00 pm CET.
- Fitusiran, an
Investigational siRNA Therapeutic Targeting Antithrombin: Analysis
of Antithrombin Levels and Thrombin Generation from a Phase 3 Study
in People with Haemophilia A or B Without inhibitors. Oral
presentation: OC.50.2. July 12, 10:45 am
– 12:00 pm
- Fitusiran, an
Investigational siRNA Therapeutic Targeting Antithrombin: Analysis
of Antithrombin Levels and Thrombin Generation from a Phase 3 Study
in People with Haemophilia A or B With inhibitors. Poster
presentation: PB1152.
July 12, 6:30 pm -7:30 pm CET)
Collectively, these data add to a growing body
of evidence, including results from the ATLAS A/B and ATLAS-INH
Phase 3 studies, supporting fitusiran’s potential to transform
treatment for all people with hemophilia. Hemophilia A and B are
rare congenital bleeding disorders caused by a deficiency of factor
VIII and IX, respectively, resulting in insufficient thrombin
generation and ineffective clot formation further complicated in
patients who develop inhibitors to their factor treatment.
Sanofi is currently investigating the efficacy
and safety of fitusiran under an amended protocol which includes
lower doses and a less frequent dosing regimen maintaining an
antithrombin target range of 15-35% in all ongoing studies.
Fitusiran has the potential to provide prophylactic treatment for
all people with hemophilia A or B, with or without inhibitors, with
as few as six subcutaneous injections per year.
ATLAS-PPX Phase 3 study design (NCT03549871)
ATLAS-PPX is a multinational, open-label, Phase
3 study designed to evaluate the efficacy and safety of fitusiran
in adult and adolescents aged ≥12 years with severe hemophilia A or
B, with or without inhibitors, who have switched from prior factor
or bypassing agent prophylaxis. A total of 80 participants were
enrolled. In the study, participants continued their pre-study
prophylaxis regimen with factor or bypassing agents for a six-month
period followed by a switch to once-monthly fitusiran (80 mg)
administered subcutaneously for seven months.
The primary endpoint of the study was annualized
bleeding rate.
About fitusiran
Fitusiran is an investigational, subcutaneously
administered small interference RNA therapeutic in development for
the prophylactic treatment of people with hemophilia A or B, with
or without inhibitors. Fitusiran is designed to lower antithrombin,
a protein that inhibits blood clotting, with the goal of promoting
thrombin generation to rebalance hemostasis and prevent bleeds.
Fitusiran utilizes Alnylam Pharmaceutical Inc.’s ESC-GalNAc
conjugate technology, which enables subcutaneous dosing with
increased potency and durability. Fitusiran is currently under
clinical investigation and has not been evaluated by any regulatory
authority.
About Sanofi
We 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 RelationsSally Bain | + 1 617
834 6026 | sally.bain@sanofi.com
Investor RelationsEva
Schaefer-Jansen | + 33 7 86 80 56 39
| 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.comPriya
Nanduri | +1 617 764 6418 |
priya.nanduri@sanofi.com Nathalie
Pham | + 33 7 85 93 30 17 |
nathalie.pham@sanofi.com
Sanofi Forward-Looking Statements
This press release contains forward-looking
statements as defined in the Private Securities Litigation Reform
Act of 1995, as amended. Forward-looking statements are statements
that are not historical facts. These statements include projections
and estimates and their underlying assumptions, statements
regarding plans, objectives, intentions and expectations with
respect to future financial results, events, operations, services,
product development and potential, and statements regarding future
performance. Forward-looking statements are generally identified by
the words “expects”, “anticipates”, “believes”, “intends”,
“estimates”, “plans” and similar expressions. Although Sanofi’s
management believes that the expectations reflected in such
forward-looking statements are reasonable, investors are cautioned
that forward-looking information and statements are subject to
various risks and uncertainties, many of which are difficult to
predict and generally beyond the control of Sanofi, that could
cause actual results and developments to differ materially from
those expressed in, or implied or projected by, the forward-looking
information and statements. These risks and uncertainties include
among other things, the uncertainties inherent in research and
development, future clinical data and analysis, including post
marketing, decisions by regulatory authorities, such as the FDA or
the EMA, regarding whether and when to approve any drug, device or
biological application that may be filed for any such product
candidates as well as their decisions regarding labelling and other
matters that could affect the availability or commercial potential
of such product candidates, the fact that product candidates if
approved may not be commercially successful, the future approval
and commercial success of therapeutic alternatives, Sanofi’s
ability to benefit from external growth opportunities, to complete
related transactions and/or obtain regulatory clearances, risks
associated with intellectual property and any related pending or
future litigation and the ultimate outcome of such
litigation, trends in exchange rates and prevailing interest
rates, volatile economic and market conditions, cost containment
initiatives and subsequent changes thereto, and the impact that
COVID-19 will have on us, our customers, suppliers, vendors, and
other business partners, and the financial condition of any one of
them, as well as on our employees and on the global economy as a
whole. Any material effect of COVID-19 on any of the
foregoing could also adversely impact us. This situation is
changing rapidly and additional impacts may arise of which we are
not currently aware and may exacerbate other previously identified
risks. The risks and uncertainties also include the uncertainties
discussed or identified in the public filings with the SEC and the
AMF made by Sanofi, including those listed under “Risk Factors” and
“Cautionary Statement Regarding Forward-Looking Statements” in
Sanofi’s annual report on Form 20-F for the year ended December 31,
2021. Other than as required by applicable law, Sanofi does not
undertake any obligation to update or revise any forward-looking
information or statements.
Sanofi (BIT:1SAN)
Historical Stock Chart
Von Mär 2024 bis Apr 2024
Sanofi (BIT:1SAN)
Historical Stock Chart
Von Apr 2023 bis Apr 2024