Alnylam Launches its RNAi 2010 Outlook for Key Scientific,
Clinical, and Business Initiatives; Announces Goals and Guidance for 2008
Advances RNAi Therapeutic for the Treatment of Huntington's Disease as Development
Program
Company Exceeds Financial Guidance; Ends 2007 with Greater than $450 Million
in Cash -
CAMBRIDGE, Mass., Jan 07, 2008 (BUSINESS WIRE) --
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics
company, today announced that it has launched its "RNAi 2010" outlook
for key scientific, clinical, and business initiatives as the company maintains
its commitment to translating the science of RNAi into innovative medicines
and building a leading biopharmaceutical company. The company also announced
its product and business goals for 2008, the initiation of a development program,
and updated its 2007 year-end cash position.
"We are more confident than ever regarding the significance of the RNAi
opportunity and Alnylam's leadership in realizing its fullest potential. Accordingly,
we have launched our 'RNAi 2010' plan as a new initiative focused on achieving
major scientific, clinical, and business milestones by the end of 2010,"
said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. "Execution
on this 'RNAi 2010' plan will continue to advance RNAi therapeutics as a transformative
approach for new medicines and our mission of building a leading biopharmaceutical
company founded on RNAi." RNAi 2010 Plan
Scientific Leadership.
By the end of 2010, Alnylam expects to broaden its leadership and significantly
expand the scope of delivery solutions for RNAi therapeutics. This will be
achieved by the continued scientific leadership of Alnylam scientists and
current academic and industry collaborators, but also a significant external
effort to form new delivery technology partnerships. Further, this effort
will include the significant expansion of the range of tissues and cell types
where the company aims to achieve efficient delivery of RNAi therapeutics
with both direct and systemic delivery approaches.
Clinical Pipeline.
By the end of 2010, Alnylam expects to have four or more RNAi therapeutic
programs in clinical development. These include direct and systemic RNAi programs,
Alnylam proprietary and 50/50 partnership programs, and siRNA (short interfering
RNA) and miRNA (microRNA) therapeutics.
New Business Collaborations.
Based on its scientific, clinical, and intellectual property leadership, the
company also expects to form four or more new major business collaborations
by the end of 2010. These are expected to include the completion of additional
broad platform alliances similar to the company's July 2007 partnership with
Roche. Completion of these business collaborations is expected to provide
the company significant resources and funding to advance Alnylam's proprietary
and 50/50 partnership pipeline programs.
2008 Goals and Guidance
Opportunity to Demonstrate Human Proof-of-Concept with RNAi Therapeutic.
Alnylam's lead program, ALN-RSV01 for the treatment of respiratory syncytial
virus (RSV) infection, is currently in a Phase II experimental infection study
in adult volunteers. Enrollment for this study was completed in December and
the study remains blinded. Top-line results from this study are expected early
in the first quarter of 2008 and offer the industry's first opportunity to
demonstrate human proof-of-concept with an RNAi therapeutic.
Advance Clinical Pipeline.
Assuming a positive outcome for its current Phase II experimental infection
study, Alnylam intends to initiate an additional Phase II study with ALN-RSV01
for the treatment of RSV in an adult patient population in the first half
of 2008. The company also expects to file a new investigational new drug (IND)
application from its development pipeline in 2008, with candidates including
ALN-PCS for the treatment of hypercholesterolemia and ALN-VSP for the treatment
of liver cancer.
Finally, the company expects that several clinical stage compounds under development
by third party Alnylam licensees will continue to
advance the expanding field of RNAi therapeutics.
Publish 10 or More Key Scientific Papers.
Alnylam expects to continue its leading scientific efforts with the publication
of key data related to in vivo efficacy and systemic delivery of RNAi therapeutics,
as well as advancements with miRNA therapeutics.
Form Two or More Major New Alliances.
In 2008, Alnylam intends to form two or more new alliances with leading companies
to continue to develop and fund its growing pipeline of RNAi therapeutics.
This includes potential platform license agreements such as the company's
July 2007 collaboration with Roche.
Continue Significant Intellectual Property (IP) Leadership.
In 2008, Alnylam expects to receive many additional patent issuances or grants
from patent offices around the world for fundamental, chemistry, delivery,
target, and miRNA therapeutic IP elements.
Maintain Solid Financial Performance.
Alnylam announced today that it now expects to achieve a 2007 year-end cash
position of greater than $450 million, as compared with its previous 2007
cash guidance of greater than $435 million. In 2008, the company aims to maintain
a solid financial position while executing on its product and business goals,
and expects to end the year with greater than $390 million in cash.
"Overall, 2007 was a very successful year in our efforts to deliver on
the promise of RNAi therapeutics. In 2008, consistent with our 'RNAi 2010'
plan, we intend to build on this substantial progress across the four key
dimensions of our business: scientific leadership, products, intellectual
property, and business execution," said Barry Greene, President and Chief
Operating Officer of Alnylam. "Importantly, we believe that advancement
of our science and product pipeline will continue to demonstrate the importance
of RNAi therapeutics as an innovative new class of drugs, and that these achievements
together with our intellectual property estate will continue to support our
ability to execute on major business opportunities."
Huntington's Disease Program
Alnylam also announced today that it has advanced an RNAi therapeutic development
program targeting the huntingtin gene for the treatment of Huntington's disease.
Huntington's disease is an autosomal dominant neurodegenerative genetic disease
that afflicts approximately 30,000 patients in the U.S., with an estimated
150,000 additional patients having a 50 percent risk of developing the disease.
The disease is caused by mutations in the huntingtin gene leading to expression
of a toxic mutated protein. This program, designated ALN-HTT, is in partnership
with Medtronic, Inc., which is structured as a 50/50 co-development/profit
share relationship in the U.S. market. In Europe, Medtronic is solely responsible
for development and commercialization. Alnylam scientists and collaborators
have published and presented in vivo data demonstrating efficacy for an siRNA
targeting the huntingtin gene in a mouse model of Huntington's disease (DiFiglia
et al., PNAS, 104, 17204 - 17209 (2007)).
"The scientific teams at Alnylam and Medtronic have made excellent progress
in our joint Huntington's program and we're excited to advance ALN-HTT as
a development program, extending our pipeline of RNAi therapeutics,"
said Akshay Vaishnaw, M.D., Ph.D., Vice President, Clinical Research of Alnylam.
"Pre-clinical data from our Huntington's disease program provide a strong
rationale to advance an RNAi therapeutic program for this important disease
where there are simply no effective therapies for patients today."
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. RNAi therapeutics target the cause
of diseases by potently silencing specific messenger RNAs (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 applying its therapeutic expertise
in RNAi to address significant medical needs, many of which cannot effectively
be addressed with small molecules or antibodies, the current major classes
of drugs. Alnylam is leading the translation of RNAi as a new class of innovative
medicines with peer-reviewed research efforts published in the world's top
scientific journals including Nature, Nature Medicine, and Cell. The company
is leveraging these capabilities to build a broad pipeline of RNAi therapeutics;
its most advanced program is in Phase II human clinical trials for the treatment
of respiratory syncytial virus (RSV) infection.
In addition, the company is developing RNAi therapeutics for the treatment
of a wide range of disease areas, including hypercholesterolemia, liver cancers,
and Huntington's disease. The company's leadership position in fundamental
patents, technology, and know-how relating to RNAi has enabled it to form
major alliances with leading companies including Medtronic, Novartis, Biogen
Idec, and Roche. To reflect its outlook for key scientific, clinical, and
business initiatives, Alnylam has established "RNAi 2010" which
includes the company's plan to significantly expand the scope of delivery
solutions for RNAi therapeutics, have four or more programs in clinical development,
and to form four or more new major business collaborations, all by the end
of 2010.
Alnylam is a joint owner of Regulus Therapeutics LLC, a joint venture focused
on the discovery, development, and commercialization of microRNA therapeutics.
Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts.
For more information, visit www.alnylam.com.
Alnylam Forward-Looking Statements
Various statements in this release concerning Alnylam's future expectations,
plans and prospects, including without limitation, the need for novel RNAi
therapeutics, Alnylam's views with respect to the potential for RNAi therapeutics,
including ALN-RSV01, and its expectations with respect to the timing and success
of its clinical and pre-clinical trials, the timing of regulatory filings,
its expectations regarding the development of efficient delivery of RNAi therapeutics,
the formation of new alliances, and its cash position at the end of 2007 and
2008, 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 risks related
to: Alnylam's approach to discover and develop novel drugs, which is unproven
and may never lead to marketable products; obtaining, maintaining and protecting
intellectual property; Alnylam's ability to enforce its patents against infringers
and to defend its patent portfolio against challenges from third parties;
Alnylam's ability to obtain additional funding to support its business activities;
Alnylam's dependence on third parties for development, manufacture, marketing,
sales and distribution of products; obtaining regulatory approval for products;
competition from others using technology similar to Alnylam's and others developing
products for similar uses; Alnylam's dependence on collaborators; and Alnylam's
short operating history; as well as those risks more fully discussed in the
"Risk Factors" section of its most recent quarterly report on Form
10-Q on file with the Securities and Exchange Commission. 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 does not assume any obligation to update any forward-looking statements.
SOURCE: Alnylam Pharmaceuticals, Inc.
Alnylam Pharmaceuticals, Inc.
Investors
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