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Last $124.81 USD
Change Today -0.07 / -0.06%
Volume 378.2K
ALNY On Other Exchanges
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As of 8:10 PM 05/22/15 All times are local (Market data is delayed by at least 15 minutes).

alnylam pharmaceuticals inc (ALNY) Key Developments

Alnylam Pharmaceuticals, Inc. Receives Orphan Drug Designation from the United States Food & Drug Administration for Revusiran

Alnylam Pharmaceuticals, Inc. announced that the United States Food & Drug Administration (FDA) has granted Orphan Drug Designation to revusiran, an investigational RNAi therapeutic, for the treatment of transthyretin (TTR)-mediated amyloidosis (ATTR amyloidosis). Revusiran is currently in Phase 3 development for the treatment of Familial Amyloidotic Cardiomyopathy (FAC), one of the predominant clinical manifestations of ATTR amyloidosis. Revusiran is currently enrolling FAC patients in the ENDEAVOUR Phase 3 trial, a randomized, double-blind, placebo-controlled, global study designed to evaluate the efficacy and safety of revusiran in patients with FAC. The co-primary endpoints of the study are the change compared to baseline in 6-minute walk distance (6-MWD) and the percent reduction in serum TTR between placebo- and revusiran-treated patients at 18 months. Secondary endpoints include a composite endpoint of cardiovascular mortality and cardiovascular hospitalization, New York Heart Association (NYHA) class, Kansas City Cardiomyopathy Questionnaire (KCCQ), CV mortality, CV hospitalization and all-cause mortality. The trial is designed to enroll up to 200 FAC patients with a documented TTR mutation, including V122I or other mutations, in addition to amyloid deposits as identified by biopsy. Patients are being randomized 2:1, revusiran:placebo, with revusiran administered subcutaneously at 500 mg daily for five days then weekly for 18 months. The trial design was informed by natural history data which showed a mean decline of 140 meters in 6MWD over an 18-month period in FAC patients with mild-to-moderate heart failure. The ENDEAVOUR study was designed with 90% power to detect as little as a 39% difference in the 18-month change from baseline for 6-MWD between treatment groups, with a significance level of p < 0.05. An unblinded interim analysis for futility may be conducted when 50% of patients reach 18 months. All patients completing the ENDEAVOUR Phase 3 study will be eligible to enroll in a Phase 3 open-label extension (OLE) study. The FDA Office of Orphan Products Development (OOPD) mission is to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. The Orphan Drug Act provides incentives for sponsors to develop products for rare diseases. The Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S.

Alnylam Files Clinical Trial Application (CTA) for ALN-AAT, an Investigational RNAi Therapeutic for the Treatment of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease (Alpha-1 Liver Disease)

Alnylam Pharmaceuticals, Inc. has filed a Clinical Trial Application (CTA) with the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 1/2 clinical trial with ALN-AAT, a subcutaneously administered investigational RNAi therapeutic targeting alpha-1 antitrypsin (AAT) for the treatment of AAT deficiency-associated liver disease (alpha-1 liver disease). As per the CTA filing, the intended clinical study of ALN-AAT will be performed in normal healthy volunteers, and, then, in subjects with alpha-1 liver disease. ALN-AAT now becomes the company's sixth clinical stage program in its Genetic Medicine Strategic Therapeutic Area (STAr), the seventh clinical pipeline program overall, and the fifth clinical program employing the company's Enhanced Stabilization Chemistry (ESC)-GalNAc delivery technology. Consistent with previous guidance, the company expects that following approval of the CTA, it will initiate the Phase 1/2 study in late 2015, with initial data expected to be reported in early 2016. ALN-AAT is a subcutaneously administered investigational RNAi therapeutic that utilizes Alnylam's proprietary ESC-GalNAc-siRNA conjugate delivery technology. ESC-GalNAc-siRNA conjugates are designed to achieve targeted delivery of RNAi therapeutics to hepatocytes through uptake by the asialoglycoprotein receptor, and enable subcutaneous dosing with increased potency and durability and a wide therapeutic index. As per the filed CTA, the Phase 1/2 trial of ALN-AAT will be a randomized, single-blind, placebo-controlled study conducted in three parts. Parts A and B will be single-dose (Part A) and multi-dose (Part B), dose-escalation studies, designed to enroll up to a total of 48 healthy adult volunteers. Part C will be a multi-dose study in adults with the PiZZ mutation in their AAT gene and with mild-to-moderate liver fibrosis. The primary objective of the study is to evaluate safety and tolerability of single and multiple subcutaneous doses of ALN-AAT. Secondary objectives include evaluation of pharmacokinetics of ALN-AAT and clinical activity for ALN-AAT as measured by knockdown of serum AAT. In addition, biopsies will be obtained from subjects with alpha-1 liver disease to quantify the effects of treatment on levels of periodic acid-Schiff (PAS)-stained globules, a measure of AAT misfolding observed in the livers of alpha-1 liver disease patients.

Alnylam Pharmaceuticals, Inc. Reports Unaudited Consolidated Earnings Results for the First Quarter Ended March 31, 2015

Alnylam Pharmaceuticals, Inc. reported unaudited consolidated earnings results for the first quarter ended March 31, 2015. For the quarter, the company's net revenues from collaborators were $18,537,000 compared to $8,275,000 in the prior year period. Loss from operations was $52,222,000 against $269,064,000 a year ago. Loss before income taxes was $51,208,000 against $268,813,000 a year ago. Net loss was $50,777,000 or $0.62 per basic and diluted share against $250,943,000 or $3.70 per basic and diluted share a year ago. Interest income was $1,014,000 compared to $333,000 for the same period last year. Non-GAAP net loss was $50,777,000 or $0.62 basic and diluted per common share compared to $26,287,000 or $0.39 basic and diluted per common share for the same period last year. The increase in revenues in the first quarter of 2015 compared to the first quarter of the previous year was due primarily to revenue recognized in connection with the September 2014 amendment of remaining performance obligations under the Monsanto agreement, as well as services performed in connection with their performance obligations under agreements with The Medicines Company and Genzyme.

Alnylam Pharmaceuticals, Inc. to Report Q1, 2015 Results on May 07, 2015

Alnylam Pharmaceuticals, Inc. announced that they will report Q1, 2015 results at 4:30 PM, US Eastern Standard Time on May 07, 2015

Alnylam Pharmaceuticals, Inc., Q1 2015 Earnings Call, May 07, 2015

Alnylam Pharmaceuticals, Inc., Q1 2015 Earnings Call, May 07, 2015

 

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