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Biotech / Medical : Alnylam Pharmaceuticals, Inc. (ALNY)
ALNY 456.04+1.4%Oct 31 9:30 AM EDT
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From: A.J. Mullen9/15/2015 11:30:16 AM
   of 166
 
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, Dose-Dependent, and Durable Silencing of ALAS1 mRNA in Liver –

– ALN-AS1 also Generally Well Tolerated with No Clinically Significant Drug-Related Adverse Events to Date –

– Company has Now Transitioned to Part B of Phase 1 Study with Monthly Subcutaneous Dosing and Plans to Start Part C in AIP Patients Suffering from Recurrent Attacks in Early 2016 –

– Company to Host Conference Call Today, Tuesday, September 15, at 8:30 am ET to Discuss Results –

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