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press release

May 5, 2017

Alnylam Reports Positive Results from Ongoing Phase 2 Open-Label Extension (OLE) Studies for Patisiran and Revusiran, in Development for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)

- Patisiran Demonstrates Continued Evidence for Potential Halting of Neuropathy Progression and First Ever Results Showing Statistically Significant Improvement in Nerve Fiber Density in Patients with Familial Amyloidotic Polyneuropathy (FAP) Patisiran APOLLO Trial on Track to Complete Enrollment in Next 3 4 Months; if Positive, Expect New Drug Application (NDA) Filing in 2017 Revusiran Demonstrates Robust and ...

press release

May 5, 2017

Alnylam Initiates Phase 1 Open Label Extension (OLE) Study with ALN-AT3, an Investigational RNAi Therapeutic for the Treatment of Hemophilia and Rare Bleeding Disorders

- Provides Opportunity for People with Hemophilia who Participated in Phase 1 Trial to Receive ALN AT3 on Ongoing Basis Pivotal Studies on Track to Begin in Mid 2016 CAMBRIDGE, Mass. (BUSINESS WIRE) Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it has initiated a Phase 1 open label extension ...

press release

May 5, 2017

Alnylam and Genzyme Announce that Genzyme Opts into ALN-AT3 Hemophilia Program for Development and Commercialization Outside of North America and Western Europe

-Marks First Product Opt In since Formation of Landmark 2014 Alliance for Global Advancement of RNAi Therapeutics as Genetic Medicines CAMBRIDGE, Mass. (BUSINESS WIRE) Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, and Genzyme, a Sanofi company (EURONEXT:SAN and NYSE:SNY), today announced that Genzyme has elected to opt into Alnylam's investigational ALN ...

press release

May 5, 2017

Alnylam Reports Positive Initial Clinical Results for ALN-AS1, an Investigational RNAi Therapeutic Targeting Aminolevulinic Acid Synthase 1 (ALAS1) for the Treatment of Acute Hepatic Porphyrias

- In Phase 1 Study of Asymptomatic High Excreter (ASHE) Patients with Acute Intermittent Porphyria (AIP), ALN AS1 Achieves up to 82% Lowering of Aminolevulinic Acid (ALA) and up to 93% Lowering of Porphobilinogen (PBG), the Toxic Heme Synthesis Intermediates that Mediate Porphyria Attacks Using Exosomal mRNA Preparations from Serum and Urine, ALN AS1 Showed Potent, ...

press release

May 5, 2017

Alnylam Initiates Phase 1/2 Clinical Trial for ALN-AAT, an Investigational RNAi Therapeutic for the Treatment of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease (Alpha-1 Liver Disease)

- Company Expects to Present Initial Clinical Results in Early 2016 CAMBRIDGE, Mass. (BUSINESS WIRE) Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it has initiated 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 ...

press release

May 5, 2017

Alnylam Pharmaceuticals Reports Second Quarter 2015 Financial Results and Highlights Recent Period Activities

- Advanced Pipeline of Seven Investigational Clinical Programs and Presented Positive Clinical Results for Three: Patisiran for Familial Amyloidotic Polyneuropathy (FAP), ALN AT3 for Hemophilia, and ALN CC5 for Complement Mediated Diseases In Addition to Upcoming Presentation of ALN PCSsc Clinical Results, Announces New Plan to Present Initial Clinical Data for ALN AS1 Porphyria Program at ...

press release

May 5, 2017

ALN-PCSsc Achieves Quarterly and Potentially Bi-Annual Subcutaneous Dose Regimen Profile for Effective LDL-C Lowering in Phase 1 Clinical Study

- Investigational First in Class PCSK9 Synthesis Inhibitor Achieves up to 83% Maximal and 64% Mean Maximum LDL C Lowering, Comparable to Published Results with Anti PCSK9 Monoclonal Antibodies, but with Clinically Significant Reductions in LDL C Clamped Down for More than 140 Days after Just a Single Dose ALN PCSsc Generally Well Tolerated with No Clinically ...

press release

May 5, 2017

Alnylam and Collaborators Report First-Ever Evidence that Patisiran Reduces Pathogenic, Misfolded Transthyretin (TTR) Monomers and Oligomers in TTR-Mediated Amyloidosis (ATTR Amyloidosis) Patients with Familial Amyloidotic Polyneuropathy (FAP)

- Patisiran, an Intravenously Administered Investigational RNAi Therapeutic Targeting TTR, Achieves an Approximately 90% Reduction in Misfolded Non Native Conformations of TTR (NNTTR) as Measured in Serum In Addition, Complete 12 Month Data (N=27) from Phase 2 Open Label Extension (OLE) Study Show a Mean 3.1 Point Decrease in Neuropathy Impairment Score (mNIS+7) at 12 Months, ...

press release

May 5, 2017

Alnylam Reports Positive Initial Clinical Results for ALN-CC5, an Investigational RNAi Therapeutic Targeting Complement Component C5 for the Treatment of Complement-Mediated Diseases

In Ongoing Phase 1/2 Study, Single Subcutaneous Doses of ALN CC5 Achieve Potent, Statistically Significant, and Highly Durable C5 Knockdown of up to 96% After Single Dose, ALN CC5 Also Achieves up to 92% Inhibition of Serum Complement Activity, Including up to 61% Inhibition of Serum Hemolytic Activity, and was Generally ...