Full Press Release Details
| Alnylam Pharmaceuticals, Inc. | ||||
| Cynthia Clayton Vice President, Investor Relations and Corporate Communications 617-551-8207 Liz Bryan (Media) Spectrum 202-955-6222 x2526 |
Alnylam Launches Alnylam 2020 Guidance for
Advancement and Commercialization of RNAi Therapeutics
By End of 2020, Company Expects to Achieve Profile with 3 Marketed Products, 10 RNAi Therapeutic Clinical Programs Including 4
in Late Stages Across 3 Strategic Therapeutic Areas (STArs)
Provides Goals and Guidance for 2015,
Exceeding its Alnylam 5x15 Guidance Established in 2011 and Further Updated in 2014
Guidance to End 2014 with Approximately $880 Million in Cash
Cambridge, Mass., January 8, 2015 Alnylam Pharmaceuticals,
Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today 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 3 Strategic Therapeutic Areas (STArs). As outlined at its R&D Day,
held on December 12, 2014, the company s 3 STArs include: Genetic Medicines, RNAi therapeutics for the treatment of rare diseases; Cardio-Metabolic Disease, RNAi therapeutics for the treatment of unmet needs in dyslipidemia, hypertension,
diabetes, and non-alcoholic steatohepatitis (NASH); and Hepatic Infectious Disease, RNAi therapeutics for the treatment of infectious diseases with liver involvement. In addition and as outlined further below, the company announced its pipeline
goals for 2015, putting the company on track to exceed its Alnylam 5x15 guidance established in January 2011 and further updated in January 2014.
Our Alnylam 5x15 product strategy put Alnylam on a trajectory to transition from a platform technology company to a platform-driven product
company. We successfully executed on this strategy, and are now on track to even exceed our expanded 2014 guidance. Alnylam 2020 is new guidance that marks our expected transition from a late-stage clinical development company to a
multi-product commercial-stage company with a sustainable development pipeline a profile that we believe has rarely been achieved in the biopharmaceutical industry. Specifically, Alnylam 2020 provides what we believe to be an
exciting path forward for Alnylam with multiple marketed products and a rich pipeline of programs in clinical development, including in
late stages, in our 3 Strategic Therapeutic Areas, or STArs , said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. Across our 3 STArs Genetic Medicines,
Cardio-Metabolic Disease, and Hepatic Infectious Disease we believe that we can address major unmet needs globally in a wide range of diseases with high-impact, differentiated medicines, and continue to build what we believe to be one of the
most robust product pipelines in biotech. By delivering on our near- and longer-term goals, we envision the potential to make a meaningful difference in the lives of patients and their caregivers, and, in so doing, maximize value for our
Alnylam also announced today its goals and guidance for 2015 across its 3 STArs, putting the company on track to exceed its
Alnylam 5x15 guidance introduced in January 2011 and further updated in January 2014 with 8 programs in clinical development, including 2 in Phase 3, with 5 having achieved human proof-of-concept results. The company s
2015 goals and guidance are as follows:
Genetic Medicine STAr, a broad pipeline of investigational RNAi therapeutics for rare diseases.
Cardio-Metabolic Disease STAr, a pipeline of investigational RNAi therapeutics
toward genetically validated, liver-expressed disease targets for unmet needs in cardiovascular and metabolic diseases.
Hepatic Infectious Disease STAr, a pipeline of investigational RNAi therapeutics that address the major global health challenges of
hepatic infectious diseases.
The company now expects to end 2014 with approximately $880 million in cash and intends to provide financial guidance for 2015 in
connection with its year-end 2014 financial results in February.
Alnylam management plans to present a company overview detailing these goals and
guidance at the 33rd Annual J.P. Morgan Healthcare Conference on Monday, January 12, 2015 at 10:00 a.m. PT (1:00 p.m. ET) at the Westin St. Francis Hotel in San Francisco, Calif. A live audio webcast of the presentation and the subsequent
Q&A session will be available on the Investors section of the company s website, www.alnylam.com. A replay of the presentation will be available on the Alnylam website within 48 hours after the event.
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
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 Merck, Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin, Cubist, GlaxoSmithKline, Ascletis, Monsanto, The Medicines Company, and Genzyme, a Sanofi company. 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.
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, its expectations regarding its STAr pipeline growth strategy, its Alnylam 2020 guidance for the advancement and commercialization of RNAi therapeutics,
its expectations for the filing of regulatory documents, the timing of the start of clinical studies and presentation of clinical data, and its expected cash position as of December 31, 2014, 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,
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 studies, 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 manage operating expenses, 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.