New User:

Forgot your password?

Stock Market & Financial Investment News

News Breaks
January 22, 2014
08:04 EDTALNYAlnylam initiates Phase 1 clinical trial with ALN-AT3
Alnylam Pharmaceuticals has initiated a Phase 1 study with ALN-AT3, a subcutaneously administered RNAi therapeutic targeting antithrombin for the treatment of hemophilia and rare bleeding disorders. ALN-AT3 has demonstrated efficacy in animal models of hemophilia, including in non-human primate models of induced hemophilia. Moreover, pre-clinical studies of ALN-AT3 support a wide therapeutic index in the hemophilia setting. ALN-AT3 is a key program in the company’s “Alnylam 5x15” product strategy, which is aimed at advancing multiple RNAi therapeutic genetic medicine programs into clinical development. Alnylam recently announced that it expects to exceed its original “Alnylam 5x15” guidance with six to seven genetic medicine programs in clinical development by the end of 2015, including at least two programs in Phase 3 and five to six programs that will have achieved human proof-of-concept results supporting further development. The Phase 1 study is being conducted in the U.K. as a single- and multi-dose, dose-escalation study consisting of two parts. Part A will be a randomized, single-blind, placebo-controlled, single-dose, dose-escalation study, enrolling up to 24 healthy volunteer subjects. The primary objective of this part of the study is to evaluate the safety and tolerability of a single low dose of ALN-AT3, with the potential secondarily to show changes in AT plasma levels at sub-pharmacologic doses. Part B of the study will be an open-label, multi-dose, dose-escalation study enrolling up to 18 people with moderate to severe hemophilia A or B. The primary objective of this part of the study is to evaluate the safety and tolerability of multiple doses of subcutaneously administered ALN-AT3 in hemophilia subjects. Secondary objectives include assessment of clinical activity as determined by knockdown of circulating AT levels and increase in thrombin generation at pharmacologic doses of ALN-AT3.
News For ALNY From The Last 14 Days
Sign up for a free trial to see the rest of the stories you've been missing.
November 11, 2015
10:46 EDTALNYAlnylam and The Medicines Co. reports trial results with ALN-PCSsc
Alnylam (ALNY) and The Medicines Company (MDCO) reported results from their ongoing Phase 1 clinical trial with ALN-PCSsc in an oral presentation at the American Heart Association Scientific Sessions 2015. ALN-PCSsc is an investigational RNAi therapeutic targeting PCSK9 - a genetically validated protein regulator of LDL receptor metabolism - being developed for the treatment of hypercholesterolemia. As reported previously, subcutaneous administration of ALN-PCSsc resulted in an up to 83% lowering of LDL-C, with an up to 64 +/- 5 percent mean maximum reduction, comparable to published results for anti-PCSK9 MAbs. In new results, the effects of ALN-PCSsc were found to be highly durable, with clinically significant and clamped reductions in LDL-C, supportive of a potential bi-annual subcutaneous dose regimen. Specifically, an up to 53 percent maximal and 47% least squares mean reduction in LDL-C was achieved at day 180 after just a single, low volume injection. In addition, ALN-PCSsc was shown to reduce a number of atherogenic lipids, including lipoprotein - or "Lp" - and total cholesterol, which are associated with increased risk of cardiovascular disease. ALN-PCSsc was generally well tolerated with no clinically significant drug-related adverse events. The development leadership of ALN-PCSsc has now transferred from Alnylam to The Medicines Company, who expects to initiate the ORION-1 Phase 2 study by the end of 2015.

Sign up for a free trial to see the rest of the stories you've been missing.
I agree to the disclaimer & terms of use