| [November 09, 2017] |
 |
Alnylam Announces Successful Outcome Following FDA Type A Meeting to Discuss Fitusiran Program in Hemophilia
Alnylam
Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics
company, announced today a successful Type A meeting with the U.S. Food
and Drug Administration (FDA) where alignment was achieved on safety
measures and a risk mitigation strategy to enable resumption of dosing
in clinical studies with fitusiran, including the Phase 2 open-label
extension (OLE) study and the ATLAS Phase 3 program. With the completion
of the Type A meeting, the FDA will now consider removal of the clinical
hold upon final review of the amended protocols and other trial
materials. Fitusiran is an investigational RNAi therapeutic targeting
antithrombin (AT) for the treatment of patients with hemophilia A and B,
that 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 outcome of our meeting with the FDA regarding
next steps for the fitusiran clinical program and remain committed to
reinitiating the Phase 2 OLE and the Phase 3 ATLAS program around
year-end," said Akin Akinc, Ph.D., Vice President and General Manager,
Fitusiran. "Further, we look forward to continuing our partnership with
physicians and nurses to support education efforts on fitusiran safety,
which we believe are a critical step when introducing a new therapeutic
modality with the potential to address important unmet needs in
hemophilia."
The Company and the FDA 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.
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 (News - Alert)-GalNAc conjugate technology, which enables subcutaneous dosing with
increased potency and durability. The clinical significance of this
technology is under investigation. In September 2017, Alnylam
temporarily suspended dosing in all ongoing studies of fitusiran
following the observation of a fatal thrombotic serious adverse event
that occurred in a patient with hemophilia A without inhibitors who was
receiving fitusiran in the Phase 2 OLE study. All ongoing studies were
placed on clinical hold. Alnylam and fitusiran study investigators and
the FDA have now aligned on safety measures and a risk management
strategy for further advancement of fitusiran. Following regulatory and
institutional review and approval of amended study protocols and other
clinical materials implementing these measures, Alnylam intends to
resume fitusiran studies as soon as possible.
Fitusiran has not been approved by the FDA, EMA (News - Alert) 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 deficien 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, 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 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 (News - Alert) 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 (News - Alert) at @Alnylam
or on LinkedIn.
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 Phase
2 OLE study and Phase 3 ATLAS studies of fitusiran following regulatory
and institutional review and approval of amended study protocols and
other clinical materials implementing these measures, 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 (News - Alert)) 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.
View source version on businesswire.com: http://www.businesswire.com/news/home/20171109006479/en/
[ Back To TMCnet.com's Homepage ]
|
