Alnylam Pharma (ALNY) Announces Completion of ENDEAVOUR Phase 3 Enrollment

August 8, 2016 8:03 AM EDT

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Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), announced that it has completed enrollment in its ENDEAVOUR Phase 3 study with revusiran, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of hereditary ATTR amyloidosis with cardiomyopathy (hATTR-CM), also known as familial amyloidotic cardiomyopathy (FAC). The ENDEAVOUR trial enrolled 206 patients with hATTR-CM, above the enrollment target of 200 patients. The Company expects to report data from the study in early 2018.

“ATTR amyloidosis is a progressive, life-threatening, orphan disease with limited treatment options. These patients often have a poor prognosis due to the rapid progression of their disease, and new therapeutic options are needed,” said Eric Green, Vice President, General Manager, TTR Program. “We’re excited to have now completed enrollment in our ENDEAVOUR Phase 3 study, ahead of expectations, and look forward to reporting data in early 2018."

The ENDEAVOUR Phase 3 trial is a randomized, double-blind, placebo-controlled, global study designed to evaluate the efficacy and safety of revusiran in patients with hATTR-CM. The co-primary endpoints of the study are the change compared to baseline in six-minute walk distance (6-MWD) and the percent reduction in TTR level between placebo- and revusiran-treated patients at 18 months. Secondary endpoints include a composite endpoint of cardiovascular (CV) mortality and cardiovascular hospitalization, New York Heart Association (NYHA) class, Kansas City Cardiomyopathy Questionnaire (KCCQ), and all-cause mortality. The trial was designed to enroll up to 200 hATTR-CM patients with a documented TTR mutation, including V122I or other mutations, in addition to amyloid deposits as identified by biopsy or technetium scan. Patients were randomized 2:1, revusiran to placebo, with revusiran administered subcutaneously at 500 mg daily for five days then weekly for 18 months.

All patients completing the ENDEAVOUR Phase 3 study may be eligible to enroll in a Phase 3 open-label extension (OLE) study (ENDEAVOUR-OLE).

Alnylam recently presented 12-month results from an ongoing Phase 2 OLE study of revusiran in patients with TTR cardiac amyloidosis. These data were presented at the XV International Symposium on Amyloidosis, held July 3 – 7, 2016, in Uppsala, Sweden.

About ATTR Amyloidosis

ATTR amyloidosis is a progressively debilitating and often fatal disease caused by deposition of transthyretin (TTR) in peripheral tissues. TTR protein is produced primarily in the liver and is normally a carrier of vitamin A. In hereditary ATTR amyloidosis (hATTR), 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. Hereditary ATTR amyloidosis represents a major unmet medical need with significant morbidity and mortality; hATTR amyloidosis with polyneuropathy (hATTR-PN) – also known as familial amyloidotic polyneuropathy (FAP) – affects approximately 10,000 people worldwide and hATTR amyloidosis with cardiomyopathy (hATTR-CM) – also known as familial amyloidotic cardiomyopathy (FAC) – is estimated to affect at least 40,000 people worldwide. hATTR-PN 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, certain countries in Latin America and Japan, where it is approved for all stages of disease). hATTR-CM is fatal within 2.5 to 5 years of diagnosis and treatment is currently limited to supportive care. Wild-type ATTR amyloidosis (wtATTR) – previously called 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 ATTR amyloidosis.

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 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.

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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