Alnylam Pharmaceuticals Announces Sanofi Genzyme Opt-in Decision for Co-Development and Co-Commercialization of Fitusiran in Hemophilia and Rare Bleeding Disorders
Nov 14, 2016
- Sanofi Genzyme Elects not to Opt in for ALN-AS1 in Acute Hepatic
Porphyrias -
- Alnylam Intends to Commercialize ALN-AS1 Globally upon Product
Approval -
CAMBRIDGE, Mass.--(BUSINESS WIRE)--
Alnylam
Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading RNAi therapeutics
company today announced that, pursuant to the companies' global alliance
signed in January 2014, Sanofi Genzyme elected to opt in to co-develop
(through Sanofi R&D) and co-commercialize fitusiran, an investigational
RNAi therapeutic for the treatment of hemophilia and rare bleeding
disorders (RBD), in the United States, Canada and Western Europe. This
expanded right is in addition to their previously exercised opt-in
decision to develop and commercialize fitusiran in their rest of world
territories. The opt in decision was based on recent promising interim
clinical results from a Phase 1 study of fitusiran presented
at the World Federation of Hemophilia (WFH) in late July and additional
data that will be presented at the American Society of Hematology (ASH)
meeting in December. Alnylam is on track to initiate the fitusiran Phase
3 program in early 2017.
"This marks another milestone for this landmark collaboration and an
important step forward on the path to bringing RNAi therapeutics to
patients. This decision allows us to broaden the global reach and
accelerate the commercial development of fitusiran, a potentially
transformative approach to the treatment of hemophilia and rare bleeding
disorders, with a well-established partner in Sanofi Genzyme," said John
Maraganore, Ph.D., Chief Executive Officer of Alnylam. "We look forward
to continued collaboration with Sanofi Genzyme to advance fitusiran as
Alnylam transitions toward the commercial stage with our late-stage
pipeline."
"We are pleased to collaborate with Alnylam to develop this important
and innovative potential new treatment option for people living with
hemophilia across the world. We believe that significant unmet need
still exists, particularly for patients with inhibitors, and we are
excited by the promising early clinical data coming from the fitusiran
program," said David Meeker, M.D., Executive Vice President and Head of
Sanofi Genzyme. "This expanded collaboration with Alnylam supports our
deep and lasting commitment to patients with rare diseases. We look
forward to sharing our operational, regulatory and commercial experience
with Alnylam as we advance this investigational product."
Alnylam and Sanofi Genzyme will now co-develop and co-commercialize
fitusiran in the Co-Commercialization Territory (United States, Canada
and Western Europe), while Sanofi Genzyme will retain exclusive rights
to develop and commercialize the product in the Sanofi Genzyme Territory
(rest of world). Certain development and sales and marketing costs for
fitusiran will be shared 50/50 between Sanofi Genzyme and Alnylam. In
addition, Sanofi Genzyme will be required to make payments totaling up
to $75 million upon the achievement of development and regulatory
milestones for fitusiran. Upon the initiation of the first global Phase
3 clinical trial for fitusiran, Alnylam will earn a milestone payment of
$25 million. Sanofi Genzyme also will be required to pay tiered
double-digit royalties up to twenty percent on annual fitusiran net
sales in the Sanofi Genzyme Territory. The companies will share profits
equally in the Co-Commercialization Territory, where Alnylam expects to
book product sales.
Sanofi Genzyme has elected not to opt in for ALN-AS1, an investigational
RNAi therapeutic for acute hepatic porphyrias. The clinical dataset
informing the Sanofi Genzyme decision consisted of Part
A and Part B results from the ongoing ALN-AS1 Phase 1 study,
recently presented at the Society for the Study of Inborn Errors of
Metabolism (SSIEM) meeting in September. Based on this decision, Alnylam
intends to develop and commercialize ALN-AS1 globally upon product
approval. Alnylam will present initial results from Part C of the
ongoing Phase 1 study at the ASH meeting in December.
Alliance Background
In January 2014, Alnylam and Sanofi Genzyme, the specialty care global
business unit of Sanofi, formed an alliance to accelerate and expand the
development and commercialization of RNAi therapeutics across the world.
The alliance is structured as a multi-product geographic alliance in the
field of rare diseases. Alnylam retains product rights in the United
States, Canada and Western Europe, while Sanofi Genzyme obtained the
right to access certain programs in Alnylam's current and future Genetic
Medicines pipeline in the rest of the world through the end of 2019,
together with certain broader co-development/co-commercialization rights
and global rights for certain products.
About Fitusiran Phase 1 Study
The ongoing Phase 1 trial of fitusiran is being conducted in the United
States, Bulgaria, Russia, Switzerland, and the U.K. as a single- and
multi-dose, dose-escalation study comprised of four parts. Part A -
which is complete - was a randomized, single-blind, placebo-controlled,
single-dose, dose-escalation study (N=4 per cohort; 3:1 randomization of
fitusiran:placebo) in healthy volunteers. This part of the study was
completed after the first dose cohort received a single subcutaneous
dose of fitusiran at 30 mcg/kg. Part B of the study - which is also
complete - was an open-label, multi-dose, dose-escalation study that
enrolled 12 patients with severe hemophilia A or B. Patients in Part B
received three weekly subcutaneous injections of fitusiran at doses of
15, 45, or 75 mcg/kg. Part C of the study - which has completed dosing -
is an open-label, multi-dose, dose escalation study that enrolled 18
patients with moderate or severe hemophilia A or B without inhibitors.
Twelve patients in Part C received three monthly subcutaneous doses of
fitusiran at doses of 225, 450, 900, or 1800 mcg/kg. In addition, six
patients in Part C received three fixed monthly subcutaneous doses of
fitusiran at 80 mg. Part D of the study is designed to enroll up to 18
patients with inhibitors. Patients in Part D will receive three fixed
monthly subcutaneous doses of fitusiran at 50 mg or 80 mg. The primary
objective of Parts B, C, and D of the study is to evaluate the safety
and tolerability of multiple doses of subcutaneously administered
fitusiran in patients with hemophilia, with and without inhibitors.
Secondary objectives include assessment of clinical activity as
determined by lowering of circulating AT levels and increase in thrombin
generation at pharmacologic doses of fitusiran. In addition, exploratory
analyses of bleeding are being performed. In the U.K., enrollment has
been aided by the Southern Academic Coagulation Consortium (SACC).
About RNAi
RNAi (RNA interference) is a revolution in biology, representing a
breakthrough in understanding how genes are turned on and off 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, thereby preventing disease-causing
proteins from being made. RNAi therapeutics have the potential to treat
disease and help patients in a fundamentally new way.
About Alnylam Pharmaceuticals
Alnylam is a biopharmaceutical company developing novel therapeutics
based on RNA interference, or RNAi. The company is leading the
translation of RNAi as a new class of innovative medicines. Alnylam's
pipeline of investigational RNAi therapeutics is focused in 3 Strategic
Therapeutic Areas (STArs): Genetic Medicines, with a broad pipeline of
RNAi therapeutics for the treatment of rare diseases; Cardio-Metabolic
Disease, with a pipeline of RNAi therapeutics toward genetically
validated, liver-expressed disease targets for unmet needs in
cardiovascular and metabolic diseases; and Hepatic Infectious Disease,
with a pipeline of RNAi therapeutics that address the major global
health challenges of hepatic infectious diseases. In early 2015, Alnylam
launched its "Alnylam 2020" guidance for the advancement and
commercialization of RNAi therapeutics as a whole new class of
innovative medicines. Specifically, by the end of 2020, Alnylam expects
to achieve a company profile with 3 marketed products, 10 RNAi
therapeutic clinical programs - including 4 in late stages of
development - across its 3 STArs. The company's demonstrated commitment
to RNAi therapeutics has enabled it to form major alliances with leading
companies including Ionis, Novartis, Roche, Takeda, Merck, Monsanto, The
Medicines Company, and Sanofi Genzyme. In addition, Alnylam holds an
equity position in Regulus Therapeutics Inc., a company focused on
discovery, development, and commercialization of microRNA therapeutics.
Alnylam scientists and collaborators have published their research on
RNAi therapeutics in over 200 peer-reviewed papers, including many in
the world's top scientific journals such as Nature, Nature Medicine,
Nature Biotechnology, Cell, New England Journal of Medicine, and The
Lancet. Founded in 2002, Alnylam maintains headquarters in
Cambridge, Massachusetts. For more information about Alnylam's pipeline
of investigational RNAi therapeutics, please visit www.alnylam.com.
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 RNAi therapeutics,
including fitusiran and ALN-AS1, its expectations regarding the timing
of clinical studies and the presentation of clinical data, including for
its studies for fitusiran and ALN-AS1, its expectations regarding its
STAr pipeline growth strategy, its "Alnylam 2020" guidance for the
advancement and commercialization of RNAi therapeutics, and its plans
regarding the pursuit of pre-clinical programs 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 our
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.
The scientific information referenced in this news release relating to
fitusiran is preliminary and investigative. 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.
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Alnylam Pharmaceuticals, Inc.
Investors and Media:
Christine
Regan Lindenboom, 617-682-4340
or
Investors:
Josh
Brodsky, 617-551-8276
Source: Alnylam Pharmaceuticals, Inc.
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