Alnylam and Genzyme Form Transformational Alliance for RNAi Therapeutics as Genetic Medicines
Jan 13, 2014
- New Collaboration Expected to Accelerate and Expand Global Product
Value for RNAi Therapeutic Genetic Medicine Pipeline, Including "Alnylam
5x15" Programs -
- Alnylam Retains Broad Product Rights in North America and Western
Europe and Genzyme Obtains Rights to Access Alnylam's Genetic Medicines
Pipeline in the Rest of World and Receives Co-Development,
Co-Commercialization Rights or Global Rights to 3 Programs -
- Genzyme Becomes Major Alnylam Shareholder through Purchase of $700
Million of Equity at $80/Share, Bolstering Alnylam's Balance Sheet to
Increase Investment in RNAi Therapeutics and Enabling Alnylam's
Financial Independence through to Multiple Product Launches -
- Companies to Host Conference Call Monday, January 13 at 9:00 am ET,
6:00 am PT to Discuss Collaboration -
CAMBRIDGE, Mass.--(BUSINESS WIRE)--
Alnylam
Pharmaceuticals, Inc. (Nasdaq:ALNY), a leading RNAi therapeutics
company, and Genzyme, a Sanofi company (EURONEXT:SAN and NYSE:SNY),
announced today that they have formed a transformational alliance for
the development and commercialization of RNAi therapeutics as genetic
medicines. Genzyme and Alnylam have formed this new alliance to
accelerate and expand the development and commercialization of RNAi
therapeutics across the world. Alnylam will retain product rights in
North America and Western Europe, while Genzyme will obtain the right to
access Alnylam's current "5x15"1 and future genetic medicines
pipeline in the rest of the world (ROW), including global product rights
for certain programs. In addition, Genzyme becomes a major Alnylam
shareholder through an upfront purchase of $700 million of newly issued
stock at approximately $80/share, representing an approximately 12%
ownership position. This alliance significantly bolsters Alnylam's
balance sheet to over $1 billion in cash, enabling an increased
investment in the company's RNAi therapeutics pipeline and is expected
to secure Alnylam's financial independence through to multiple product
launches.
"Genzyme holds a longstanding commitment to improving the lives of
patients through the development and commercialization of treatments for
rare diseases, both through internal R&D and by working with valuable
external collaborators," said David Meeker, M.D., President and Chief
Executive Officer of Genzyme. "Our relationship with Alnylam has been
highly collaborative, and we believe that their world-class RNAi
technology holds the promise to provide a platform for sustained drug
development for rare genetic diseases for years to come. With this new
alliance, we are significantly broadening our relationship with Alnylam
and expanding Genzyme's pipeline of innovative medicines for the
treatment of genetic diseases."
"This new relationship with Genzyme is transformational for Alnylam. It
is a game changer for both the advancement of RNAi therapeutics as a new
class of genetic medicines to patients around the world, and for our
commitment to build a leading, independent biopharmaceutical company
that delivers value to our shareholders," said John Maraganore, Ph.D.,
Chief Executive Officer of Alnylam. "In this new alliance, Alnylam
benefits enormously from Genzyme's proven global capabilities, enabling
us to accelerate and expand market access for our ‘Alnylam 5x15'
products. At the same time, we retain our product rights in North
America and Western Europe, where we remain committed to develop and
commercialize our RNAi therapeutics pipeline. We also retain full global
product rights for all RNAi therapeutic products outside the genetic
medicine field. In addition, this new collaboration significantly
expands our balance sheet to over $1 billion in cash to increase our
investment in new RNAi therapeutic programs, while securing a cash
runway that we believe will allow us to develop and launch multiple
products as breakthrough medicines for patients in need."
The new alliance is structured as an exclusive relationship for the
worldwide development and commercialization of RNAi therapeutics in the
field of genetic medicines, which includes Alnylam's current "5x15"1
and future genetic medicine programs that reach human proof-of-concept
stages up until 2020. Alnylam retains product rights in North America
and Western Europe while Genzyme obtains rights to pipeline programs in
the ROW. Genzyme's rights are structured as an opt-in that is triggered
upon achievement of human proof-of-concept. Alnylam maintains
development control for all programs prior to Genzyme's opt-in and
maintains development and commercialization control for all programs in
its territory.
Upon the closing of the transaction, Genzyme will opt-in to patisiran
(ALN-TTR02) - an RNAi therapeutic currently in a Phase 3 trial for the
treatment of transthyretin (TTR)-mediated amyloidosis (ATTR) patients
with Familial Amyloidotic Polyneuropathy - for its ROW territories,
while Alnylam retains full product rights in North America and Western
Europe. Alnylam and Genzyme have also agreed to expand their current
collaboration on ALN-TTRsc - an RNAi therapeutic currently in a Phase 2
trial for the treatment of ATTR patients with TTR amyloid cardiomyopathy
- where the parties will co-develop and co-promote ALN-TTRsc in North
America and Western Europe; Alnylam will maintain development and
commercialization control with ALN-TTRsc and Genzyme will develop and
commercialize the product in its ROW territories. The companies believe
that this broadened collaboration on ALN-TTRsc will increase the
product's overall value, as significant market development and
commercial operational scale is warranted to maximize the opportunity
for both familial cardiac amyloidosis (FAC) and senile systemic
amyloidosis (SSA) manifestations of TTR cardiac amyloidosis.
In addition to its rights for Alnylam's current "5x15" programs and the
company's future genetic medicine programs in the Genzyme territory,
Genzyme has the right to either co-develop and co-promote ALN-AT3
for the treatment of hemophilia and other rare bleeding disorders (RBD)
in Alnylam's territory - with Alnylam maintaining development and
commercialization control - or to obtain a global license to
ALN-AS1 for the treatment of hepatic porphyrias. Genzyme will exercise
this selection right upon completion of human proof-of-concept for the
ALN-AT3 and ALN-AS1 programs. Finally, Genzyme has obtained the right
for a global license to a single, future genetic medicine program that
is not one of the currently defined Alnylam "5x15" programs1.
Alnylam retains global rights to any RNAi therapeutic genetic medicine
program that does not reach the human proof-of-concept stage by 2020,
subject to certain limited exceptions. Under the terms of the agreement,
Alnylam retains full rights to all current and future RNAi therapeutic
programs outside of the field of genetic medicines, including the right
to form new collaborations. Finally, the companies have agreed to enter
into exclusive discussion and negotiation for a research collaboration
to discover novel technologies for delivery of siRNAs in the central
nervous system (CNS), where both companies will share in any resulting
intellectual property.
The new Alnylam-Genzyme alliance is valued at well over $1 billion,
including equity, R&D funding, and potential milestone payments. Genzyme
will purchase $700 million of Alnylam stock, or approximately 8.8
million shares at a price of approximately $80/share, representing a 27%
premium to the 30-day trailing average of Alnylam stock calculated from
the last trading day prior to the agreement's execution. With this
issuance of the new shares, Genzyme becomes an approximately 12% owner
of Alnylam. As per the terms of the Share Purchase Agreement, Genzyme
has the right to purchase up to - but not more than - 30% of Alnylam
stock during the term of the active development and commercial
collaboration. A number of additional terms of the equity purchase are
described in Alnylam's Current Report on Form 8-K filing with the SEC in
association with execution of the agreement, and include the right for
Genzyme to nominate a board member if their stock ownership exceeds 20%,
plus other customary provisions related to voting rights, registration
rights, lock-up and selling restrictions, amongst other terms that are
customary in a share purchase agreement of this scope.
In addition to the upfront equity purchase, Alnylam will receive R&D
funding, starting on January 1, 2015, for programs where Genzyme has
elected to opt-in for development and commercialization. For "regional"
programs (e.g., patisiran) where Genzyme will develop and commercialize
in their ROW territory, Genzyme will pay 20% of global development
expenses. For "co-develop/co-promote" programs (i.e., ALN-TTRsc and
possibly ALN-AT3 if selected), Genzyme will pay 50% of global
development expenses. For "global" programs (i.e., Genzyme's future
"non-‘5x15'" global opt-in right and possibly ALN-AS1 if selected),
Genzyme will pay 100% of global expenses. In addition, Alnylam is
eligible to receive milestones totaling up to $75 million per product
for regional and co-develop/co-promote programs. In the case of global
Genzyme programs, Alnylam is eligible to receive up to $200 million in
milestones per product. Finally, Alnylam is also eligible to receive
tiered double-digit royalties up to 20% on net sales on all products
commercialized by Genzyme in its territories. In the case of Genzyme's
co-develop/co-promote products in the Alnylam territory, the parties
will share profits equally and Alnylam will book net sales revenues.
This transaction has been approved by the boards of both companies, and
is subject to customary closing conditions and clearances under the
Hart-Scott Rodino Antitrust Improvements Act.
Conference Call Information
Alnylam and Genzyme management will discuss this new alliance in a
conference call on January 13, 2014 at 9:00 am ET, 6:00 am PT. A slide
presentation will also be available on the News & Investors page of the
company's website, www.alnylam.com,
to accompany the conference call. To access the call, please dial
877-312-7507 (domestic) or 631-813-4828 (international) five minutes
prior to the start time and refer to conference ID 31887205. A replay of
the call will be available beginning at 12:00 pm ET, 9:00 am PT on
January 13, 2014. To access the replay, please dial 855-859-2056
(domestic) or 404-537-3406 (international), and refer to conference ID
31887205.
About RNA Interference (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 with a core
focus on RNAi therapeutics as genetic medicines, including programs as
part of the company's "Alnylam 5x15TM" product strategy.
Alnylam's genetic medicine programs are RNAi therapeutics directed
toward genetically defined targets for the treatment of serious,
life-threatening diseases with limited treatment options for patients
and their caregivers. These include: patisiran (ALN-TTR02), an
intravenously delivered RNAi therapeutic targeting transthyretin (TTR)
for the treatment of TTR-mediated amyloidosis (ATTR) in patients with
familial amyloidotic polyneuropathy (FAP); ALN-TTRsc, a subcutaneously
delivered RNAi therapeutic targeting TTR for the treatment of ATTR in
patients with familial amyloidotic cardiomyopathy (FAC); ALN-AT3, an
RNAi therapeutic targeting antithrombin (AT) for the treatment of
hemophilia and rare bleeding disorders (RBD); ALN-AS1, an RNAi
therapeutic targeting aminolevulinate synthase-1 (ALAS-1) for the
treatment of hepatic porphyrias including acute intermittent porphyria
(AIP); ALN-CC5, an RNAi therapeutic targeting complement component C5
for the treatment of complement-mediated diseases; ALN-PCS, an RNAi
therapeutic targeting PCSK9 for the treatment of hypercholesterolemia;
ALN-AAT, an RNAi therapeutic targeting alpha-1-antitrypsin (AAT) for the
treatment of AAT deficiency liver disease; ALN-TMP, an RNAi therapeutic
targeting TMPRSS6 for the treatment of beta-thalassemia and
iron-overload disorders; and ALN-ANG, an RNAi therapeutic for the
treatment of genetic forms of mixed hyperlipidemia and severe
hypertriglyceridemia, amongst other programs. As part of its "Alnylam
5x15" strategy, as updated in early 2014, the company expects to have
six to seven genetic medicine product candidates in clinical development
- including at least two programs in Phase 3 and five to six programs
with human proof of concept - by the end of 2015. The company's
demonstrated commitment to RNAi therapeutics has enabled it to form
major alliances with leading companies including Merck, Medtronic,
Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin, Cubist,
GlaxoSmithKline, Ascletis, Monsanto, The Medicines Company, and Genzyme,
a Sanofi company. In January 2014, Alnylam agreed to acquire Sirna
Therapeutics, a wholly owned subsidiary of Merck. 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, the New
England Journal of Medicine, and The Lancet. Founded in 2002,
Alnylam maintains headquarters in Cambridge, Massachusetts. For more
information, please visit www.alnylam.com.
About "Alnylam 5x15™" and Genetic Medicines
The "Alnylam 5x15" strategy, launched in January 2011, establishes a
path for development and commercialization of novel RNAi therapeutics as
genetic medicines. Alnylam's genetic medicine programs are RNAi
therapeutics directed toward genetically defined targets for the
treatment of diseases with high unmet medical need. These programs share
several key characteristics including: a genetically defined target and
disease expressed in the liver; the potential to have a major impact in
a high unmet need population; the ability to leverage the existing
Alnylam RNAi platform with clinically proven delivery to the liver; the
opportunity to monitor an early biomarker in Phase 1 clinical trials for
human proof of concept; and the existence of clinically relevant
endpoints for the filing of a new drug application (NDA) with a focused
patient database and possible accelerated paths for commercialization.
As updated in early 2014, the company expects to have six to seven
genetic medicine product candidates in clinical development - including
at least two programs in Phase 3 and five to six programs with human
proof of concept - by the end of 2015. The "Alnylam 5x15" programs
include: patisiran (ALN-TTR02), an intravenously delivered RNAi
therapeutic targeting transthyretin (TTR) in development for the
treatment of TTR-mediated amyloidosis (ATTR) in patients with familial
amyloidotic polyneuropathy (FAP); ALN-TTRsc, a subcutaneously delivered
RNAi therapeutic targeting TTR in development for the treatment of ATTR
in patients with familial amyloidotic cardiomyopathy (FAC); ALN-AT3, an
RNAi therapeutic targeting antithrombin (AT) in development for the
treatment of hemophilia and rare bleeding disorders (RBD); ALN-AS1, an
RNAi therapeutic targeting aminolevulinate synthase-1 (ALAS-1) in
development for the treatment of porphyria including acute intermittent
porphyria (AIP); ALN-CC5, an RNAi therapeutic targeting complement
component C5 in development for the treatment of complement-mediated
diseases; ALN-PCS, an RNAi therapeutic targeting PCSK9 in development
for the treatment of hypercholesterolemia; ALN-AAT, an RNAi therapeutic
targeting alpha-1-antitrypsin (AAT) for the treatment of AAT deficiency
liver disease; ALN-TMP, an RNAi therapeutic targeting TMPRSS6 in
development for the treatment of beta-thalassemia and iron-overload
disorders; and ALN-ANG, an RNAi therapeutic for the treatment of genetic
forms of mixed hyperlipidemia and severe hypertriglyceridemia, amongst
other programs. In 2014, Alnylam and Genzyme, a Sanofi company, formed a
multi-product geographic alliance on Alnylam's genetic medicine
programs. Specifically, Alnylam will lead development and
commercialization of programs in North America and Europe, while Genzyme
will develop and commercialize products in the rest of world. In
addition, Alnylam and Genzyme will co-develop and co-commercialize
ALN-TTRsc in North America and Europe.
Alnylam Forward-Looking Statements
Various statements in this press release concerning Alnylam's future
expectations, plans and prospects, including without limitation,
Alnylam's views with respect to the potential for RNAi therapeutics,
including the programs in its 5x15 pipeline, Genzyme's participation in
the development and commercialization of RNAi therapeutics, its
expectations regarding the receipt of potential R&D payments,
development, regulatory and sales milestones and royalties from Genzyme,
and its expectations regarding available cash for its operations through
multiple product launches, constitute forward-looking statements for the
purposes of the safe harbor provisions under The Private Securities
Litigation Reform Act of 1995. Actual results may differ materially from
those indicated by these forward-looking statements as a result of
various important 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 drug
candidates, the pre-clinical and clinical results for its product
candidates, which may not support further development of product
candidates, actions of regulatory agencies, which may affect the
initiation, timing and progress of clinical trials, Genzyme's ability to
successfully advance patisiran, ALN-TTRsc and other products in the
Genzyme territory, resulting in the potential payment of milestones and
royalties to Alnylam, as well as Alnylam's ability to develop and
commercialize such products in the rest of the world, the parties
ability to successfully co-develop and co-promote ALN-TTRsc and
potentially a second product in North America and Western Europe,
obtaining, maintaining and protecting intellectual property, Alnylam's
ability to enforce its patents against infringers and defend its patent
portfolio against challenges from third parties, obtaining regulatory
approval for products, competition from others using technology similar
to Alnylam's and others developing products for similar uses, Alnylam's
ability to 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, marketing, sales and distribution of products, the outcome
of litigation, 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
to update any forward-looking statements.
1For purposes of the Alnylam-Genzyme agreement, Alnylam's
current "5x15" programs include: patisiran, an RNAi therapeutic
targeting TTR for the treatment of ATTR patients with FAP; ALN-TTRsc, an
RNAi therapeutic targeting TTR for the treatment of ATTR patients with
TTR amyloid cardiomyopathy, including FAC and SSA; ALN-AT3, an RNAi
therapeutic targeting antithrombin (AT) for the treatment of hemophilia
and RBD; ALN-CC5, an RNAi therapeutic targeting complement component C5
for the treatment of complement-mediated diseases; ALN-AS1, an RNAi
therapeutic targeting aminolevulinate synthase-1 (ALAS-1) for the
treatment of hepatic porphyrias; and ALN-AAT, an RNAi therapeutic
targeting alpha-1-antitrypsin (AAT) for the treatment of liver disease
associated with AAT deficiency.
Alnylam Pharmaceuticals, Inc.
Cynthia Clayton, 617-551-8207
Vice
President, Investor Relations and Corporate Communications
or
Media:
Spectrum
Amanda
Sellers, 202-955-6222 x2597
Source: Alnylam Pharmaceuticals, Inc.
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