EMA Recommends Orphan Designation for Alnylam’s RNAi Therapeutic for Transthyretin-Mediated Amyloidosis

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Alnylam Receives Positive Opinion for Orphan Drug Designation in the European Union for ALN-TTRsc, a Subcutaneously Delivered RNAi Therapeutic for the Treatment of Transthyretin-Mediated Amyloidosis (ATTR)

April 4, 2014

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion recommending ALN-TTRsc for designation as an orphan medicinal product for the treatment of transthyretin (TTR)-mediated amyloidosis (ATTR).

“We are very pleased to have received a positive opinion from the EMA COMP on our application for Orphan Drug Designation for ALN-TTRsc,” said Saraswathy (Sara) Nochur, Ph.D., Senior Vice President, Regulatory Affairs and Quality Assurance at Alnylam. “We believe RNAi therapeutics represent a promising new approach for the treatment of ATTR, with the potential to make a meaningful impact for patients with this progressive and debilitating disease. We look forward to sharing Phase 2 clinical data from our ALN-TTRsc program later in the year, and, assuming continued positive results, we plan to advance to a Phase 3 pivotal trial in ATTR patients with TTR cardiac amyloidosis by the end of the year.”

ALN-TTRsc is currently in a pilot Phase 2 clinical trial for the treatment of ATTR patients with TTR cardiac amyloidosis; this study is aimed at evaluating the tolerability of ALN-TTRsc in approximately 15 patients. In addition, the study will assess preliminary clinical activity as measured by knockdown of serum TTR levels and additional exploratory tests, such as cardiac imaging (including echocardiography and cardiac MRI), circulating cardiac biomarkers (NT-proBNP and troponins T and I), 6-minute walk test, New York Heart Association (NYHA) classification, and measures of heart failure symptoms and quality of life (Kansas City Cardiomyopathy Questionnaire and EQ-5D QOL). The company expects to present data from the Phase 2 trial in late 2014. Patients completing the Phase 2 trial will be eligible to participate in an open-label extension (OLE) study for further assessment of general tolerability and clinical activity with long-term dosing; the ALN-TTRsc Phase 2 OLE study is expected to be initiated in mid-2014. Assuming positive results, Alnylam expects to begin a Phase 3 trial in TTR cardiac amyloidosis patients by the end of 2014.

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), and where no satisfactory treatment is available. In addition to a 10-year period of marketing exclusivity in the EU after product approval, Orphan Drug Designation provides incentives for companies seeking protocol assistance from the EMA during the product development phase, and direct access to centralized marketing authorization.

In January 2014, Genzyme and Alnylam 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 obtains the right to access Alnylam’s current “5×15” 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 ALN-TTRsc, Alnylam and Genzyme are co-developing and co-commercializing 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) is an inherited, progressively debilitating, and fatal disease caused by mutations in the TTR gene. TTR protein is produced primarily in the liver and is normally a carrier for retinol binding protein. 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) affects at least 40,000 people worldwide. FAP patients have a life expectancy of five to 15 years from symptom onset, and the only approved treatment options for early stage disease are liver transplantation, and tafamidis (approved in Europe). The mean survival for FAC patients is approximately 2.5 years, and there are no approved therapies. 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.

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Cynthia Clayton, 617-551-8207
Vice President, Investor Relations and Corporate Communications
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Amanda Sellers, 202-955-6222 x2597

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