- Sanofi Genzyme Elects not to Opt in for
ALN-AS1 in Acute Hepatic Porphyrias -
- Alnylam Intends to Commercialize ALN-AS1
Globally upon Product Approval -
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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version on businesswire.com: http://www.businesswire.com/news/home/20161114005456/en/
Alnylam Pharmaceuticals, Inc.Investors and
Media:Christine Regan Lindenboom, 617-682-4340orInvestors:Josh
Brodsky, 617-551-8276
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