Alnylam to Report New Clinical Results with Investigational RNAi Therapeutics for the Treatment of Transthyretin-Mediated Amyloidosis at the First European Congress on Hereditary ATTR Amyloidosis


- Company to Present New Data from Phase 2 Open-Label Extension (OLE) Studies with Patisiran and Revusiran -

- Company to Host Conference Call on Tuesday, November 3, at 7:00 a.m. ET -

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that Alnylam scientists and collaborators will present results from ongoing Phase 2 open-label extension (OLE) studies with its investigational RNAi therapeutics in development for the treatment of transthyretin (TTR)-mediated amyloidosis (ATTR amyloidosis) at the First European Congress on Hereditary ATTR Amyloidosis being held November 2-3, 2015 in Paris, France. The presentations will include 18-month patisiran data for the treatment of patients with Familial Amyloidotic Polyneuropathy (FAP) and 6-month revusiran data for the treatment of patients with TTR cardiac amyloidosis, including Familial Amyloidotic Cardiomyopathy (FAC) and Senile Systemic Amyloidosis (SSA), a non-hereditary form of TTR cardiac amyloidosis. The Company will host a conference call on Tuesday, November 3 at 7:00 a.m. ET to discuss these results.

"We are excited to share these data with world leading experts and clinicians in the ATTR community," said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. "We continue to collect safety and patient experience data in our OLE studies with the goal of providing much needed therapeutic options for patients with this difficult-to-treat condition."

Presentations by Alnylam scientists and collaborators at the meeting include:

  • DISCOVERY: A Study Examining the Prevalence of Transthyretin Mutations in Subjects Suspected of Having Cardiac Amyloidosis
Author:       Olakunle Akinboboye, Queens Heart Institute
Session: Epidemiology and Phenotypes in ATTR Amyloidosis

Monday, November 2, 9:30 - 10:30 a.m. CET

  • Analysis of Disease Progression in Patients with Transthyretin Cardiac Amyloidosis
Author:       Julian D. Gillmore, University College London Medical School
Session: New Tools for Diagnosis
Date/Time: Monday, November 2, 4:10 - 5:10 p.m. CET
  • Patients with Hereditary ATTR Amyloidosis Experience an Increasing Burden of Illness as the Disease Progresses
Author:       David Adams, French Reference Centre for FAP
Session: Poster session II
Date/Time: Tuesday, November 3, 7:45 - 8:30 a.m. CET
  • Phase 2 Open-Label Extension (OLE) Study of Patisiran, an Investigational siRNA Agent for Familial Amyloidotic Polyneuropathy (FAP)
Author:       David Adams, French Reference Centre for FAP
Session: Disease Modifying Therapy
Date/Time: Tuesday, November 3, 10:00 - 11:00 a.m. CET
  • Phase 2 Open-Label Extension (OLE) Study of Revusiran, an Investigational RNAi Therapeutic for the Treatment of Patients with Transthyretin Cardiac Amyloidosis
Author:       Julian D. Gillmore, University College London Medical School
Session: Disease Modifying Therapy
Date/Time: Tuesday, November 3, 10:00 - 11:00 a.m. CET

Conference Call Information
Alnylam management will discuss these data in a webcast conference call on Tuesday, November 3 at 7:00 a.m. ET. A slide presentation will also be available on the Investors page of the company's website,, 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 70778772. A replay of the call will be available beginning at 9:00 a.m. ET. To access the replay, please dial 855-859-2056 (domestic) or 404-537-3406 (international), and refer to conference ID 70778772.

Genzyme Alliance
In January 2014, Alnylam and Genzyme, a Sanofi company, 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 Genzyme obtained the right to access certain programs in Alnylam's current and future Genetic Medicines pipeline in the rest of the world (ROW,) including co-development/co-commercialization and/or global product rights for certain programs. In the case of patisiran, Alnylam will advance the product in North America and Western Europe, while Genzyme will advance the product in the ROW. In the case of revusiran, Alnylam and Genzyme will co-develop/co-commercialize the product in North America and Western Europe, while Genzyme will advance the product in the ROW.

About Transthyretin-Mediated Amyloidosis
Transthyretin (TTR)-mediated amyloidosis (ATTR amyloidosis) is an inherited, progressively debilitating, and often fatal disease caused by mutations in the TTR gene. TTR protein is produced primarily in the liver and is normally a carrier of vitamin A. Mutations in TTR cause abnormal amyloid proteins to accumulate and damage body organs and tissue, such as the peripheral nerves and heart, resulting in intractable peripheral sensory neuropathy, autonomic neuropathy, and/or cardiomyopathy. ATTR represents a major unmet medical need with significant morbidity and mortality; familial amyloidotic polyneuropathy (FAP) affects approximately 10,000 people worldwide and familial amyloidotic cardiomyopathy (FAC) is estimated to affect at least 40,000 people worldwide. FAP patients have a life expectancy of 5 to 15 years from symptom onset, and the only approved treatment options for early stage disease are liver transplantation, and tafamidis (approved in Europe). FAC is fatal within 2.5 to 5 years of diagnosis and treatment is currently limited to supportive care. Senile systemic amyloidosis (SSA) is a non-hereditary form of TTR cardiac amyloidosis caused by idiopathic deposition of wild-type TTR; its prevalence is generally unknown, but is associated with advanced age. There is a significant need for novel therapeutics to treat patients with TTR amyloid polyneuropathy and/or cardiomyopathy.

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 Merck, Medtronic, Novartis, Biogen, 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

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 patisiran and revusiran for the treatment of TTR-mediated amyloidosis, and its plans regarding commercialization of RNAi therapeutics, including patisiran and revusiran, 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.

Alnylam Pharmaceuticals, Inc.
Michael Mason, 617-551-8327
Vice President, Finance and Treasurer
Liz Bryan (Media), 202-955-6222 x2526

Source: Alnylam Pharmaceuticals, Inc.

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