CAMBRIDGE, Mass. & PARIS--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, and Sanofi Genzyme, the specialty care global business unit of Sanofi, announced today that the U.S. Food and Drug Administration (FDA) has lifted the hold on clinical studies with fitusiran, including the Phase 2 open-label extension (OLE) study and the ATLAS Phase 3 program.
Alnylam and the FDA had previously reached alignment on new clinical risk mitigation measures, including protocol-specified guidelines and additional investigator and patient education concerning reduced doses of replacement factor or bypassing agent to treat any breakthrough bleeds in fitusiran studies. The FDA has now approved the protocol amendments and other updated clinical materials for fitusiran studies. Fitusiran is an investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of patients with hemophilia A and B. It is designed to lower levels of AT with the goal of promoting sufficient thrombin generation to restore hemostasis and prevent bleeding.
“We are pleased with the FDA’s decision to lift the clinical hold, as fitusiran holds the potential to help improve the lives of people living with hemophilia,” said Akin Akinc, Ph.D., Vice President and General Manager, Fitusiran at Alnylam. “With the additional risk mitigation measures in place, we look forward to the continued late-stage development of fitusiran and expect to resume dosing around year-end.”
About Fitusiran
Fitusiran is an investigational,
once-monthly, subcutaneously administered RNAi therapeutic targeting
antithrombin (AT) in development 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. Fitusiran has not been approved by
the FDA, EMA or any other regulatory authority for any indication and no
conclusions can or should be drawn regarding the safety or effectiveness
of this investigational therapeutic.
About Hemophilia
Hemophilia 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 400,000 people living with
hemophilia A and hemophilia B worldwide. 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, with
current factor replacement treatments people with hemophilia are at risk
of developing neutralizing antibodies or ‘inhibitors' to their
replacement factor, a very serious complication affecting as many as one
third of people with hemophilia A and a smaller fraction of people with
hemophilia B. People who develop inhibitors become refractory to
replacement factor therapy and are twice as likely to be hospitalized
for a bleeding episode.
Alnylam - Sanofi Genzyme Alliance
In January 2014, Alnylam
and Sanofi Genzyme 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 RNAi
RNAi (RNA interference) is a natural cellular
process of gene silencing that represents one of the most promising and
rapidly advancing frontiers in biology and drug development today. Its
discovery has been heralded as “a major scientific breakthrough that
happens once every decade or so,” and was recognized with the award of
the 2006 Nobel Prize for Physiology or Medicine. By harnessing the
natural biological process of RNAi occurring in our cells, 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, function upstream of today’s
medicines by potently silencing messenger RNA (mRNA) – the genetic
precursors – that encode for disease-causing proteins, thus preventing
them from being made. This is a revolutionary approach with the
potential to transform the care of patients with genetic and other
diseases.
About Alnylam Pharmaceuticals
Alnylam (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 people afflicted with rare genetic, cardio-metabolic, and hepatic
infectious diseases. Based on Nobel Prize-winning science, RNAi
therapeutics represent a powerful, clinically validated approach for the
treatment of a wide range of severe and 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 four product candidates
that are in late-stage development. 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 to address the needs of
patients who have limited or inadequate treatment options. Alnylam
employs over 600 people in the U.S. and Europe and is headquartered in
Cambridge, MA. For more information about our people, science and
pipeline, please visit www.alnylam.com
and engage with us on Twitter at @Alnylam
or on LinkedIn.
About Sanofi
Sanofi 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.
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.
Sanofi, Empowering Life.
Alnylam Forward Looking Statements
Various statements in
this release concerning Alnylam's future expectations, plans and
prospects, including without limitation, Alnylam's views with respect to
the potential for fitusiran for the treatment of people with hemophilia,
expectations regarding the timing for resumption of dosing in the
fitusiran Phase 2 OLE study and Phase 3 ATLAS program, and expectations
regarding 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.
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 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.
