CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today announced that The Committee for Orphan Medicinal Products (COMP) within the European Medicines Agency (EMA) adopted a positive opinion for ALN-TTR01 designation as an orphan medicinal product for the treatment of familial amyloidotic polyneuropathy (FAP), one of the predominant forms of transthyretin (TTR)-mediated amyloidosis (ATTR). ALN-TTR01 is a systemically delivered RNAi therapeutic being developed for the treatment of all forms of ATTR, which includes FAP and familial amyloidotic cardiomyopathy (FAC). A positive opinion by the COMP precedes official designation of ALN-TTR01 as an orphan drug by the European Commission.
“We are pleased to have received this positive opinion for Orphan Drug Designation from the EMA for our ALN-TTR program. Indeed, we believe that RNAi therapeutics targeting transthyretin could represent a major advance for the treatment of ATTR – a significant unmet medical need where breakthrough therapies are needed,” said Saraswathy Nochur, Ph.D., Vice President, Regulatory Affairs at Alnylam. “Our ongoing Phase I trial is actively enrolling patients and is focused on demonstrating safety and tolerability of ALN-TTR01; importantly, it is also designed to capture key data on clinical activity through measurements of serum TTR levels. We are on track to complete enrollment in this study by the end of the first half of this year and to report data in the third quarter.”
Orphan Drug Designation by the European Commission provides regulatory and financial incentives for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union (EU). In addition to a 10-year period of marketing exclusivity in the EU after product approval, Orphan Drug Designation provides companies with protocol assistance from the EMA during the product development phase, direct access to centralized marketing authorization, as well as reduced regulatory fees.
About TTR-Mediated Amyloidosis
Transthyretin (TTR)-mediated amyloidosis (ATTR) is a hereditary, systemic disease caused by mutations in the TTR gene. TTR protein is produced primarily in the liver and is normally a carrier for thyroid hormones and retinol binding proteins. Mutations in TTR cause abnormal amyloid proteins to accumulate in and damage body organs and tissue such as the peripheral nerves and heart, resulting in intractable peripheral sensory neuropathy, autonomic neuropathy, and/or cardiomyopathy. In its severest form, ATTR represents a major unmet medical need with significant morbidity and mortality as an orphan disease; FAP (familial amyloidotic polyneuropathy) affects approximately 5,000 to 10,000 people worldwide and FAC (familial amyloidotic cardiomyopathy) affects approximately 40,000 people worldwide. ATTR patients with FAP have a mean life expectancy of five to 15 years from symptom onset and the only treatment option is liver transplantation; as a result there is a significant need for novel therapeutics to treat patients who have inherited mutations in the TTR gene.
About ALN-TTR Program
ALN-TTR01 is a systemically delivered RNAi therapeutic being developed for the treatment of transthyretin (TTR)-mediated amyloidosis (ATTR), including familial amyloidotic polyneuropathy (FAP) and familial amyloidotic cardiomyopathy (FAC). Pre-clinical studies have shown that treatment with ALN-TTR01 results in both prevention and regression of pathogenic TTR deposits in peripheral tissues including dorsal root ganglia, sciatic nerve, stomach, and intestines in animal models. In addition, ALN-TTR01 administration in pre-clinical animal models was found to result in dose-dependent and durable, yet reversible silencing of the TTR gene and serum levels of TTR. ALN-TTR01 is formulated in a first generation lipid nanoparticle (LNP) developed by Tekmira Pharmaceuticals Corporation, and is currently in a randomized, placebo-controlled, dose escalation Phase I clinical trial. The primary objective of this study is to evaluate the safety and tolerability of a single dose of intravenous ALN-TTR01; secondary objectives include characterization of plasma and urine drug pharmacokinetics and assessment of pharmacodynamic activity based on measurements of circulating TTR serum levels. Alnylam expects to complete this Phase I study in the first half of 2011 and report data in the third quarter of 2011. In parallel, Alnylam is also advancing ALN-TTR02, which utilizes second-generation delivery technology. The ALN-TTR02 program is on track for an investigational new drug (IND) application filing in the second half of 2011. In 2012, following results from both ALN-TTR01 and ALN-TTR02 studies, Alnylam expects to advance this program into Phase II clinical studies.
About “Alnylam 5x15™”
The “Alnylam 5x15” strategy, launched in January 2011, establishes a path for development and commercialization of novel RNAi therapeutics to address genetically defined diseases with high unmet medical need. Products arising from this initiative share several key characteristics including: a genetically defined target and disease; the potential to have a major impact in a high unmet need population; the ability to leverage the existing Alnylam delivery platform; the opportunity to monitor an early biomarker in Phase I 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. This strategy leverages Alnylam’s clinical progress on siRNA delivery, including definitive human proof-of-concept data for systemic delivery. By 2015, the company expects to have five such RNAi therapeutic programs in advanced clinical development. These include ALN-TTR for the treatment of transthyretin-mediated amyloidosis (ATTR), ALN-PCS for the treatment of severe hypercholesterolemia, ALN-HPN for the treatment of refractory anemia, and two additional programs from the company’s ongoing discovery efforts that will be advanced into development later in 2011. Alnylam intends to commercialize the products arising under the “Alnylam 5x15” strategy itself in the United States and potentially certain other territories; the company will seek development and commercial partners in other global territories.
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 RNAs (siRNAs), 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 for the treatment of genetically defined diseases, including ALN-TTR for transthyretin-mediated amyloidosis (ATTR), ALN-PCS for severe hypercholesterolemia, and ALN-HPN for refractory anemia. As part of its “Alnylam 5x15TM” strategy, the company expects to have five RNAi therapeutic products for genetically defined diseases in advanced stages of clinical development by the end of 2015. Alnylam has additional partner-based programs in clinical or development stages, including ALN-RSV01 for respiratory syncytial virus (RSV) infection, ALN-VSP for liver cancers, and ALN-HTT for Huntington’s disease. The company’s leadership position on RNAi therapeutics and intellectual property have enabled it to form major alliances with leading companies including Merck, Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin, and Cubist. In addition, Alnylam and Isis co-founded Regulus Therapeutics, Inc., a company focused on discovery, development, and commercialization of microRNA therapeutics; Regulus has formed partnerships with GlaxoSmithkline and sanofi-aventis. Alnylam has also formed Alnylam Biotherapeutics, a division of the company focused on the development of RNAi technologies for application in biologics manufacturing, including recombinant proteins and monoclonal antibodies. Alnylam scientists and collaborators have published their research on RNAi therapeutics in over 100 peer-reviewed papers, including many in the world’s top scientific journals such as Nature, Nature Medicine, Nature Biotechnology, and Cell. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts. For more information, please visit www.alnylam.com.
Alnylam Forward-Looking Statement
Various statements in this release concerning Alnylam’s future expectations, plans and prospects, including without limitation, Alnylam’s expectations with respect to its “Alnylam 5x15” product strategy, Alnylam’s views with respect to the potential for RNAi therapeutics, including ALN-TTR01 and ALN-TTR02, ALN-PCS, ALN-HPN, ALN-VSP, ALN-RSV01 and ALN-HTT, its expectations with respect to the timing and success of its clinical and pre-clinical trials, including its plan to initiate clinical trials for ALN-TTR02 and ALN-PCS, the expected timing of regulatory filings, and the formation of new alliances and business ventures, 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; 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 timing and progress of clinical trials; 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 current and future 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.