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Alnylam Pharmaceuticals Inc.

Reported Positive Preliminary Clinical Results in ALN-PCS Severe Hypercholesterolemia Program. Alnylam achieved positive preliminary results from its ongoing clinical trial of ALN-PCS, a systemically delivered RNAi therapeutic targeting proprotein convertase subtilisin/kexin type 9 (PCSK9) for the treatment of severe hypercholesterolemia. These results showed safety and tolerability as well as potent, robust, and statistically significant knockdown of PCSK9 plasma levels and reductions in low-density lipoprotein cholesterol (LDL-C) levels, a clinically validated endpoint. Results also documented for the first time major advances in delivery of RNAi therapeutics using second generation LNPs in human studies. The Phase I clinical study is ongoing with planned dose escalation and Alnylam expects to report additional data in the first half of 2012. Alnylam plans to partner its ALN-PCS program prior to initiating a Phase II clinical study.
Source: press release, 2/09/12. http://phx.corporate-ir.net/phoenix.zhtml?c=148005&p=irol-newsArticle&ID...

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Alnylam Initiates Phase I Clinical Study of ALN-PCS, an RNAi Therapeutic Targeting PCSK9 for the Treatment of Severe Hypercholesterolemia
- Study Provides Opportunity to Assess Human Proof of Concept with Second Generation Lipid Nanoparticles; Data Expected at Year's End -

CAMBRIDGE, Mass., Sep 26, 2011 (BUSINESS WIRE) --

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it has initiated dosing in its Phase I clinical trial with ALN-PCS, an RNAi therapeutic targeting proprotein convertase subtilisin/kexin type 9, or PCSK9, for the treatment of severe hypercholesterolemia. The study is aimed at evaluating the safety and tolerability of ALN-PCS in subjects with elevated low-density lipoprotein cholesterol (LDLc, or "bad" cholesterol). In addition, the study will evaluate clinical activity of ALN-PCS based on measurements of plasma PCSK9 protein and serum LDLc levels. Alnylam expects to present data from this study at year's end.
Source: press release, 9/26/11. http://phx.corporate-ir.net/phoenix.zhtml?c=148005&p=irol-newsArticle&ID...

Compound/DeviceSpecialtyIndicationCompound ClassTarget
ALN-PCSCardiovascularHypercholesterolemiaRNAi therapeuticPCSK9

Mechanism of action: ALN-PCS is an RNAi therapeutic designed to target proprotein convertase subtilisin/kexin type 9, or PCSK9, for the treatment of severe hypercholesterolemia. PCSK9 is a key regulator of LDL receptor levels.

Phase of Development: I

Event Type: Data: Phase I trial results

Dates: 2012-03-01 - 2012-06-30

Results:

Alnylam Reports Positive Clinical Results for ALN-PCS, an RNAi Therapeutic Targeting PCSK9 for the Treatment of Severe Hypercholesterolemia
- PCSK9 Synthesis Inhibitor Achieves up to 84% Knockdown of PCSK9 and 50% Lowering of LDL Cholesterol in Single Dose, Statin-Free Phase I Trial -
- New Results Highlight Continued Improved Efficacy and Safety for Alnylam Second-Generation Lipid Nanoparticles (LNP) -

CAMBRIDGE, Mass., Apr 20, 2012 (BUSINESS WIRE) --Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today positive results from its Phase I clinical trial of ALN-PCS, an RNAi therapeutic targeting PCSK9 for the treatment of severe hypercholesterolemia. ALN-PCS is a PCSK9 synthesis inhibitor that reduces intracellular and extracellular levels of PCSK9 resulting in lowered plasma levels of low-density lipoprotein cholesterol (LDL-C), or "bad" cholesterol. The new data were presented at the American Heart Association's Arteriosclerosis, Thrombosis and Vascular Biology 2012 Scientific Sessions held in Chicago. Results showed that administration of a single dose of ALN-PCS, in the absence of concomitant lipid-lowering agents such as statins, resulted in statistically significant and durable reductions of PCSK9 plasma levels of up to 84% and lowering of LDL-C of up to 50%. The new data also highlight continued improved efficacy and tolerability for Alnylam's second-generation lipid nanoparticle (LNP) delivery technology.
Source: press release, 4/20/12 http://phx.corporate-ir.net/phoenix.zhtml?c=148005&p=irol-newsArticle&ID...