– New Data from an Ongoing Phase 2 Open-label
Extension Study with Fitusiran in Patients with Hemophilia A and B,
With or Without Inhibitors–
Alnylam Pharmaceuticals,
Inc. (Nasdaq:ALNY), the leading RNAi therapeutics
company and Sanofi Genzyme, the specialty care global business unit
of Sanofi, announced that new results from an ongoing Phase 2
open-label extension (OLE) study with the investigational
therapeutic, fitusiran, in patients with hemophilia A and B, with
or without inhibitors, will be presented in an oral presentation at
the International Society of Thrombosis and Haemostasis (ISTH)
Congress, being held from July 8 – 13, 2017 in Berlin, Germany. In
addition, preclinical data further elucidating fitusiran’s novel
mechanism of action will also be presented at the meeting.
“The data being presented at ISTH demonstrate the growing body
of clinical and nonclinical evidence that support fitusiran as a
potential treatment approach for patients with hemophilia A and B,
with and without inhibitors,” said Akin Akinc, Ph.D., Alnylam’s
Vice President and General Manager, Fitusiran.
“We are committed to furthering research to address the unmet
needs of people living with hemophilia,” said Baisong Mei, M.D.,
Ph.D., Sanofi’s Senior Global Project Head, Alnylam Portfolio. “We
look forward to having the data presented at ISTH regarding the
safety and clinical activity of fitusiran from this ongoing Phase 2
OLE study.”
Alnylam and Sanofi Genzyme presentations include:
- Fitusiran, an Investigational RNAi
Therapeutic Targeting Antithrombin for the Treatment of Hemophilia:
Interim Results from a Phase 2 Extension Study in Patients with
Hemophilia A or B with and without InhibitorsPresenter: K. John
Pasi, Royal London Haemophilia Centre, United KingdomSession:
Abstract Symposia Sessions, Clinical Research and Outcomes in
HemophiliaDate/Time: Monday, July 10, 8:45 – 10:15 a.m. ET (2:45 -
4:15 p.m. CEST)
- Development of a
Pharmacokinetic-Pharmacodynamic (PK-PD) Model of Fitusiran, an
Investigational RNAi Therapeutic Targeting Antithrombin for the
Treatment of Hemophilia in Patients with and without
InhibitorsPresenter: Husain Attarwala, Alnylam Pharmaceuticals,
United StatesSession: Poster Session, Exhibition Hall 4.2Date/Time:
Tuesday, July 11, 6:00 - 7:15 a.m. ET (12:00 - 2:15 p.m. CEST)
- Measurement of Thrombin Generation in
Conditions of Low AntithrombinPresenter: Peter Geisen,
Thrombinoscope BV, Maastricht, The NetherlandsSession: Poster
Session, Exhibit Hall 6.2Date/Time: Tuesday, July 11, 6:00 - 7:15
a.m. ET (12:00 - 2:15 p.m. CEST)
- The Combined Use of Bypassing Agents
with Antithrombin Reduction in Plasma of Hemophilia A and B
Patients with InhibitorsPresenter: Gili Kenet, National Hemophilia
Center, Sheba Medical Center, IsraelSession: Poster Session,
Exhibit Hall 4.2Date/Time: Tuesday, July 11, 6:00 - 7:15 a.m. ET
(12:00 - 2:15 p.m. CEST)
Alnylam will also be presenting nonclinical data on ALN-F12, a
development candidate targeting Factor XII (ALN-F12) for the
treatment of hereditary angioedema and the prevention of
thrombosis.
- Reduction of Hepatic Factor XII
Expression in Mice by ALN-F12 Inhibits Thrombosis without
Increasing Bleeding RiskPresenter: Jingxuan Liu, Alnylam
Pharmaceuticals, United StatesSession: Abstract Symposia Sessions,
Bleeding Management Under Oral AnticoagulationDate/Time: Monday,
July 10, 8:45 – 10:15 a.m. ET (2:45 - 4:15 p.m. CEST)
About HemophiliaHemophilia is a hereditary bleeding
disorder characterized by an underlying defect in the ability to
generate adequate levels of thrombin needed for effective clotting,
thereby resulting in recurrent bleeds into joints, muscles, and
major internal organs. There are approximately 200,000 persons
diagnosed worldwide with hemophilia A and hemophilia B.
Standard treatment for people with hemophilia currently involves
replacement of the deficient clotting factor either as prophylaxis
or “on-demand” therapy which can lead to a temporary restoration of
thrombin generation capacity. However, as many as one third of
people with severe hemophilia A will develop a neutralizing
antibody to their replacement factor – a very serious complication;
individuals with these ‘inhibitors’ become refractory to standard
replacement factor therapy.
About FitusiranFitusiran is an investigational,
once-monthly, subcutaneously administered RNAi therapeutic
targeting antithrombin (AT) for the treatment of hemophilia A and
B, with and without inhibitors. Fitusiran also has the potential to
be used for rare bleeding disorders. Fitusiran is designed to lower
levels of AT with the goal of promoting sufficient thrombin
generation to restore hemostasis and prevent bleeding. Fitusiran
utilizes Alnylam's ESC-GalNAc conjugate technology, which enables
subcutaneous dosing with increased potency and durability. The
clinical significance of this technology is under
investigation.
The safety and efficacy of fitusiran have not been evaluated by
the U.S. Food and Drug Administration or any other health
authority.
Alnylam - Sanofi Genzyme AllianceIn January 2014, Alnylam
and Sanofi Genzyme, the specialty care global business unit of
Sanofi, formed an alliance to accelerate the advancement of RNAi
therapeutics as a potential new class of innovative medicines for
patients around the world with rare genetic diseases. The alliance
enables Sanofi Genzyme to expand its rare disease pipeline with
Alnylam’s novel RNAi technology and provides access to Alnylam’s
R&D engine, while Alnylam benefits from Sanofi Genzyme’s proven
global capabilities to advance late-stage development and, upon
commercialization, accelerate market access for these promising
genetic medicine products.
In November 2016, Sanofi Genzyme elected to co-develop (through
Sanofi R&D) and co-commercialize fitusiran in the United
States, Canada and Western Europe, in addition to commercializing
fitusiran in its rest of world territories.
About RNAiRNAi (RNA interference) is a revolution in
biology, representing a breakthrough in understanding protein
synthesis in cells, and a completely new approach to drug discovery
and development. Its discovery has been heralded as "a major
scientific breakthrough that happens once every decade or so," and
represents one of the most promising and rapidly advancing
frontiers in biology and drug discovery today which was awarded the
2006 Nobel Prize for Physiology or Medicine. RNAi is a natural
process of gene silencing that occurs in organisms ranging from
plants to mammals. By harnessing the natural biological process of
RNAi occurring in our cells, the creation of a major new class of
medicines, known as RNAi therapeutics, is on the horizon. Small
interfering RNA (siRNA), the molecules that mediate RNAi and
comprise Alnylam's RNAi therapeutic platform, target the cause of
diseases by potently silencing specific mRNAs, with the goal of
preventing disease-causing proteins from being made.
About Alnylam PharmaceuticalsAlnylam (Nasdaq: ALNY) is
leading the translation of RNA interference (RNAi) into a whole new
class of innovative medicines with the potential to transform the
lives of patients who have limited or inadequate treatment options.
Based on Nobel Prize-winning science, RNAi therapeutics represent a
powerful, clinically validated approach for the treatment of a wide
range of debilitating diseases. Founded in 2002, Alnylam is
delivering on a bold vision to turn scientific possibility into
reality, with a robust discovery platform and deep pipeline of
investigational medicines, including three product candidates that
are in late-stage development or will be in 2017. Looking forward,
Alnylam will continue to execute on its "Alnylam 2020" strategy of
building a multi-product, commercial-stage biopharmaceutical
company with a sustainable pipeline of RNAi-based medicines. For
more information about our people, science and pipeline, please
visit www.alnylam.com and engage with us on Twitter at
@Alnylam.
About SanofiSanofi, a global healthcare leader,
discovers, develops and distributes therapeutic solutions focused
on patients' needs. Sanofi is organized into five global business
units: Diabetes and Cardiovascular, General Medicines and Emerging
Markets, Sanofi Genzyme, Sanofi Pasteur and Consumer
Healthcare.
Sanofi Genzyme focuses on developing specialty treatments for
debilitating diseases that are often difficult to diagnose and
treat, providing hope to patients and their families. Learn more at
www.sanofigenzyme.com.
Alnylam Forward Looking StatementsVarious statements in
this release concerning Alnylam's future expectations, plans and
prospects, including without limitation, Alnylam's views with
respect to the potential for RNAi therapeutics, including fitusiran
and ALN-F12, its plans regarding the reporting of clinical data
from its clinical trials of fitusiran and its preclinical studies
of ALN-F12, and its expectations regarding its STAr pipeline growth
strategy and its "Alnylam 2020" guidance for the advancement and
commercialization of RNAi therapeutics, constitute forward-looking
statements for the purposes of the safe harbor provisions under The
Private Securities Litigation Reform Act of 1995. Actual results
and future plans may differ materially from those indicated by
these forward-looking statements as a result of various important
risks, uncertainties and other factors, including, without
limitation, Alnylam's ability to discover and develop novel drug
candidates and delivery approaches, successfully demonstrate the
efficacy and safety of its product candidates, the pre-clinical and
clinical results for its product candidates, which may not be
replicated or continue to occur in other subjects or in additional
studies or otherwise support further development of product
candidates for a specified indication or at all, actions or advice
of regulatory agencies, which may affect the design, initiation,
timing, continuation and/or progress of clinical trials or result
in the need for additional pre-clinical and/or clinical testing,
delays, interruptions or failures in the manufacture and supply of
its product candidates, obtaining, maintaining and protecting
intellectual property, Alnylam's ability to enforce its
intellectual property rights against third parties and defend its
patent portfolio against challenges from third parties, obtaining
and maintaining regulatory approval, pricing and reimbursement for
products, progress in establishing a commercial and ex-United
States infrastructure, competition from others using
technology similar to Alnylam's and others developing products for
similar uses, Alnylam's ability to manage its growth and operating
expenses, obtain additional funding to support its business
activities, and establish and maintain strategic business alliances
and new business initiatives, Alnylam's dependence on third parties
for development, manufacture and distribution of products, the
outcome of litigation, the risk of government investigations, and
unexpected expenditures, as well as those risks more fully
discussed in the "Risk Factors" filed with Alnylam's most recent
Quarterly Report on Form 10-Q filed with the Securities and
Exchange Commission (SEC) and in other filings that Alnylam
makes with the SEC. In addition, any forward-looking
statements represent Alnylam's views only as of today and should
not be relied upon as representing its views as of any subsequent
date. Alnylam explicitly disclaims any obligation, except to the
extent required by law, to update any forward-looking
statements.
None of Alnylam's investigational therapeutics have been
approved by the U.S. Food and Drug
Administration, European Medicines Agency, or any other
regulatory authority and no conclusions can or should be drawn
regarding the safety or effectiveness of these therapeutics.
Sanofi Forward Looking StatementsThis 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 absence of guarantee that the
product candidates if approved will be commercially successful, the
future approval and commercial success of therapeutic alternatives,
Sanofi’s ability to benefit from external growth opportunities
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 conditions,
the impact of cost containment initiatives and subsequent changes
thereto, the average number of shares outstanding as well as those
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,
2016. Other than as required by applicable law, Sanofi does not
undertake any obligation to update or revise any forward-looking
information or statements.
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version on businesswire.com: http://www.businesswire.com/news/home/20170626006159/en/
Alnylam Pharmaceuticals, Inc.Investors and MediaChristine
Regan Lindenboom, 617-682-4340orInvestorsJosh Brodsky,
617-551-8276orSanofiMedia RelationsAshleigh Koss,
908-981-8745Mobile: 908-205-2572Ashleigh.koss@sanofi.comorSanofi
GenzymeCommunicationsLisa Clemence,
617-768-6699Lisa.clemence@sanofi.com
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