Data from two Phase 3 studies demonstrating fitusiran significantly
reduced bleeds in people with hemophilia A or B, with or without
inhibitors, were featured at ASH’s plenary and late-breaking
sessions
Data from two Phase 3 studies
demonstrating fitusiran significantly reduced bleeds in
people with hemophilia A or B, with or without
inhibitors, were
featured at ASH’s plenary
and late-breaking
sessions
- Both Phase 3 studies achieved their primary and secondary
endpoints; fitusiran prophylaxis demonstrated significant and
clinically meaningful improvements in bleed protection across all
study populations
- A >89% reduction in annualized bleeding rate was
demonstrated with fitusiran prophylaxis in both studies when
compared to the control arms
- 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 inhibitors
Paris –
December 14,
2021 - Positive data from two Phase 3
studies evaluating the efficacy and safety of fitusiran, an
investigational small interference RNA (siRNA) therapy for the
prophylactic treatment of adults and adolescents with hemophilia A
or B, with or without inhibitors, are presented at the 63rd
American Society of Hematology (ASH) Annual Meeting. Results from
the ATLAS-A/B study, investigating fitusiran in people without
inhibitors, are being presented today in the Late-Breaking Abstract
Session and findings from the ATLAS-INH study, which evaluated
fitusiran in people with inhibitors to factor VIII or IX, were
shared in the Plenary Scientific Session on December 12,
2021.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. Fitusiran is a novel subcutaneous siRNA prophylactic
investigational therapy designed to lower antithrombin levels with
the goal of enhancing thrombin generation to rebalance hemostasis
in people with hemophilia, regardless of type or inhibitor
status.
The Phase 3 studies compared once monthly
fitusiran prophylaxis (80mg) with on-demand use of factor
concentrates in the ATLAS-A/B study, and on-demand use of bypassing
agents in the ATLAS-INH study. Across both clinical studies,
prophylactic treatment with fitusiran reduced annualized bleeding
rates by >89% compared to the control arms, showing a
statistically significant and clinically meaningful improvement in
bleeds when compared to on-demand treatments, and also showing
significant improvement in quality of life.“We are encouraged by
the data from these initial Phase 3 studies demonstrating
fitusiran’s potential as a new therapeutic option for people with
hemophilia A or B, regardless of inhibitor status,” says Dietmar
Berger, MD, PhD, Global Head of Development, Sanofi. “There
continues to be a significant need for transformative therapies
that offer people with hemophilia consistent protection from bleeds
while reducing treatment burden. These findings underscore
fitusiran’s potential to address these challenges and give hope to
patients, caregivers, and physicians.”The fitusiran Phase 3
clinical program is ongoing. Sanofi is currently investigating the
efficacy and safety of fitusiran under an amended protocol which
includes lower doses and an extended dosing regimen in all ongoing
adult and adolescent studies. Fitusiran has the potential to
provide prophylactic treatment for all people with hemophilia A or
B, with as few as six injections per year.
ATLAS-A/B Phase 3
Study
(NCT03417245)
ATLAS-A/B is a Phase 3 randomized, open-label
study investigating the efficacy and safety of fitusiran in males
≥12 years with severe hemophilia A or B without inhibitors who had
previously been treated with on-demand factor therapy. Study
participants (n=120) were randomized 2:1 to receive either
once-monthly 80mg subcutaneous fitusiran prophylaxis or on-demand
factor therapy for bleeding episodes. The primary endpoint is
annualized bleeding rate (ABR).
The key findings in this study include the
following:
- A statistically significant and
clinically meaningful reduction in treated annualized bleeding rate
of 89.9% in the fitusiran prophylaxis arm (95% CI 84.1%; 93.6%],
P <0.0001) compared to the factor on-demand arm.
- Median (interquartile range) annual
bleeding rate for treated bleeds is 0.0 (0.0; 3.4,) in the
fitusiran arm compared to 21.8 (8.4; 41.0) in the factor on-demand
arm.
- 50.6% (n=40) of study participants
in the fitusiran prophylaxis arm had zero treated bleeds compared
to 5.0% (n=2) of participants in the factor on-demand arm.
- The most common adverse events
reported in at least five (6.3%) participants in the fitusiran
prophylaxis arm were increased alanine aminotransferase (ALT),
upper respiratory tract infection, nasopharyngitis, abdominal pain,
increased aspartate aminotransferase (AST), cough, arthralgia,
asthma, gastritis, and headache.
- Treatment emergent adverse events
of special interest (TEAESIs) of any ALT or AST elevation >3 x
upper limit of normal were reported in the fitusiran prophylaxis
arm in 15 (19.0%) participants. There were no TEAESIs of suspected
or confirmed thromboembolism reported.
Phase 3 ATLAS-INH Study
(NCT03417102)
The ATLAS-INH study is a randomized, open-label
Phase 3 study designed to evaluate the safety and efficacy of
fitusiran in males ≥12 years with severe hemophilia A or B with
inhibitors to factor VIII or IX. Study participants (n=57)
receiving on-demand treatment with bypassing agents (BPA) were
randomized in a 2:1 ratio to receive once-monthly 80mg subcutaneous
fitusiran prophylaxis or continue with on-demand BPA. The primary
endpoint is annualized bleeding rate.
The key findings in this study include the
following:
- Fitusiran prophylaxis resulted in a
statistically significant reduction in treated annualized bleeding
rate of 90.8% (95% CI 80.8%; 95.6%), P<0.0001) in comparison to
treatment with BPAs.
- The median (interquartile range)
treated annualized bleeding rate is 0.0 (0.0; 1.7,) in the
fitusiran prophylaxis arm compared to 16.8 (6.7; 23.5) in the
on-demand BPA on-demand arm.
- 65.8% (n=25) participants in the
fitusiran prophylaxis arm had zero treated bleeds compared to 5.3%
(n=1)) in the BPA on-demand arm.
- The most common adverse events
reported in at least five (12.2%) participants in the fitusiran
prophylaxis arm were increased ALT, increased AST, upper abdominal
pain, increased gamma-glutamyl transferase, headache, upper
respiratory tract infection, arthralgia, increased blood alkaline
phosphatase, and increased transaminases.
- TEAESIs of ALT or AST elevation
>3 x upper limit of normal and suspected or confirmed
thromboembolism were reported in the fitusiran prophylaxis arm in
10 (24.4%) and 2 (4.9%) participants, respectively.
Fitusiran significantly reduced annualized
bleeding with a meaningful improvement in health-related quality of
life. Reported TEAEs in the fitusiran prophylaxis arm of ATLAS-A/B
and ATLAS-INH were generally consistent with previously identified
risks of fitusiran, or risks associated with the underlying disease
of severe hemophilia A or B.
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
sufficient 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 SanofiSanofi is dedicated to supporting
people through their health challenges. We are a global
biopharmaceutical company focused on human health. We prevent
illness with vaccines, provide innovative treatments to fight pain
and ease suffering. We stand by the few who suffer from rare
diseases and the millions with long-term chronic conditions.With
more than 100,000 people in 100 countries, Sanofi is transforming
scientific innovation into healthcare solutions around the
globe.
Media Relations
Contact Sally
Bain Tel: +1 (781)
264-1091 Sally.Bain@sanofi.com Investor
Relations Contacts ParisEva
Schaefer-JansenArnaud DelepineNathalie Pham
Investor Relations Contact North AmericaFelix
Lauscher
Tel.: +33 (0)1 53 77 45
45 investor.relations@sanofi.com
https://www.sanofi.com/en/investors/contact
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, 2020. 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