Full Press Release Details
Alnylam Outlines 2016 Goals for RNAi Therapeutics Pipeline
Company Plans to Advance Multiple Phase 3 Programs, Including Start of Two Fitusiran
Phase 3 Studies, and Expects Ten or More Major Clinical Data Readouts
and Three New IND Filings
Cambridge, Mass., January 11, 2016 Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today its
pipeline goals for 2016 across its Genetic Medicine, Cardio-Metabolic Disease, and Hepatic Infectious Disease Strategic Therapeutic Areas (STArs). Achieving these goals will advance the Company towards its Alnylam 2020 guidance, launched
in January 2015, where by the end of 2020 the Company expects to achieve a profile with three marketed products, 10 RNAi therapeutic clinical programs including four in late stages of development across its three STArs. As part of its
2016 guidance, the Company plans to advance multiple Phase 3 programs, including the start of two fitusiran Phase 3 trials in hemophilia, and expects to have at least ten major clinical data readouts and to file three new IND* applications.
By 2020, we expect to have multiple marketed products and a rich pipeline of
programs in clinical development, including multiple programs in late stages, across our three STArs. To enable this objective, our reproducible and modular platform provides what we believe to be a robust drug discovery and development
framework to advance a pipeline of potentially differentiated products, said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. We believe the pipeline goals we are announcing today bring us closer to achieving our Alnylam
2020 profile and becoming a fully integrated commercial-stage biopharmaceutical company. With multiple Phase 3 programs, ten or more clinical data readouts, and three new INDs expected in 2016, we believe that our pipeline is positioned to
make an increasingly meaningful difference in the lives of patients and to create significant value for our shareholders.
Genetic Medicine STAr,
a broad pipeline of investigational RNAi therapeutics for the treatment of rare diseases.
Cardio-Metabolic Disease STAr, a pipeline of investigational RNAi therapeutics toward
unmet needs in cardiovascular and metabolic diseases.
Hepatic Infectious Disease STAr, a pipeline of investigational RNAi therapeutics that address major global health challenges.
Alnylam is updating its 2015 cash guidance today to end with approximately $1.28B. The Company intends to provide financial guidance for
2016 in connection with its year-end 2015 financial results in February.
Alnylam management will present a company overview detailing these goals and
guidance at the 34th Annual J.P. Morgan Healthcare Conference today, Monday, January 11, 2016 at 10:00 a.m. PT (1:00 p.m. ET) at the Westin St. Francis Hotel in San Francisco, California. In addition, the Company plans to webcast its Q&A
breakout session immediately following the presentation at
10:30 a.m. PT (1:30 p.m. ET). A live audio webcast of the presentation and breakout session will be available on the Investors section of the Company s website, www.alnylam.com. A replay
will be available on the Alnylam website within 48 hours after the event.
Sanofi Genzyme Alliance
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 North America 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 (ROW) through the end of 2019, together with certain broader
co-development/co-commercialization rights and global rights for certain products. In the case of patisiran, Alnylam will advance the product in North America and Western Europe, while Sanofi Genzyme will advance the product in the
ROW. In the case of revusiran, Alnylam and Sanofi Genzyme will co-develop/co-commercialize the product in North America and Western Europe, while Sanofi Genzyme will advance the product in the ROW. In the case of fitusiran, Sanofi Genzyme has
elected to opt into the program for its ROW rights, while retaining its further opt-in right to co-develop and co-promote fitusiran with Alnylam in North America and Western Europe, subject to certain restrictions.
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 Merck, Medtronic, Novartis, Biogen, Roche, Takeda, Kyowa Hakko Kirin, Cubist,
GlaxoSmithKline, Ascletis, 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
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, 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, its expectations regarding the timing of the start of clinical studies and presentation of clinical data, the expected timing for completion of enrollment and the potential for over-enrollment in its APOLLO Phase 3 study, its
expected cash position as of December 31, 2015 and the expected timing for 2016 cash guidance, and its plans regarding the pursuit of pre-clinical programs, strategic partnership opportunities 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 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 be replicated or continue to occur in other subjects or in additional studies or otherwise support further development of product candidates, actions of regulatory agencies,
which may affect the initiation, timing and progress of clinical trials, 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.