(By Balaseshan) Alnylam Pharmaceuticals Inc. (NASDAQ: ALNY) said it has presented new pre-clinical data on RNAi therapeutics for the treatment of hemophilia and other bleeding disorders at the 54th American Society of Hematology Annual Meeting.
Alnylam scientists presented data showing that ALN-AT3, a subcutaneously administered RNAi therapeutic targeting antithrombin (AT), yields potent, dose-dependent, and durable knockdown of AT in non-human primates (NHPs) with an up to four-fold increase in thrombin generation.
Alnylam's program in hemophilia comprises part of its "Alnylam 5x15" product strategy, by which the company aims to advance five programs in clinical development, including programs in advanced stages, by the end of 2015.
Alnylam scientists showed that subcutaneous administration of ALN-AT3 in NHPs results in potent, dose-dependent, and durable knockdown in serum AT, resulting in significant increases in thrombin generation. Alnylam expects to file an investigational new drug (IND) application for ALN-AT3 in mid-2013.
In addition, results from Alnylam's ALN-AT3 program will be discussed at an upcoming RNAi Roundtable on conjugate delivery that the company will host on December 14 at 11:00 am. The discussion will focus on progress with the company's proprietary GalNAc-conjugate platform to deliver RNAi therapeutics with subcutaneous dose administration.
The roundtable will also include a review of the company's two leading conjugate programs: ALN-TTRsc for the treatment of transthyretin-mediated amyloidosis (ATTR) and ALN-AT3 for the treatment of hemophilia and other bleeding disorders.
Hemophilias are hereditary disorders caused by genetic deficiencies of various blood clotting factors, resulting in recurrent bleeds into joints, muscles, and other major internal organs.
ALNY is trading up 4.68% at $17.43 on Monday. The stock has been trading between $6.88 and $21.38 for the past 52 weeks.