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Alnylam

Capella

Alnylam welcomes you to Capella, the destination for updates on our progress in translating the science of RNAi toward innovative medicines.

Initial Evidence for Potential Correction of the Hemophilia Phenotype in Phase 1 Study of ALN-AT3

January 11, 2015

Initial Evidence for Potential Correction of the Hemophilia Phenotype in Phase 1 Study of ALN-AT3

We announced updated clinical results from the ongoing Phase 1 study of ALN-AT3, a subcutaneously administered, investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders (RBD).  New results were presented at the 2015 Goring Coagulation Conference in London.  Specifically, data were presented from the study’s second dose cohort in hemophilia subjects (n=3), where subcutaneous administration of ALN-AT3 resulted in an up to 70% knockdown of AT.  New results provide initial evidence for potential correction of the hemophilia phenotype associated with ALN-AT3 administration and AT knockdown.  Specifically, ALN-AT3 administration resulted in an increase in thrombin generation of up to 334% and a marked improvement in whole blood clotting.  In addition, the most advanced severe hemophilia A subject in the cohort has remained bleed free for 47 days without replacement factor prophylaxis as of the January 6, 2015 data cut-off date. In addition, ALN-AT3 administration remains generally well tolerated.



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Alnylam 2020

January 10, 2015

Alnylam 2020

In January 2015, we launched our “Alnylam 2020” guidance for the advancement and commercialization of RNAi therapeutics as a whole new class of innovative medicines.  Specifically, by the end of 2020, we expect to achieve a company profile with 3 marketed products, 10 RNAi therapeutic clinical programs – including 4 in late stages of development – across 3 Strategic Therapeutic Areas (STArs).  Our 3 STArs include: Genetic Medicines, RNAi therapeutics for the treatment of rare diseases; Cardio-Metabolic Disease, RNAi therapeutics for the treatment of unmet needs in dyslipidemia, hypertension, diabetes, and non-alcoholic steatohepatitis (NASH); and Hepatic Infectious Disease, RNAi therapeutics for the treatment of infectious diseases with liver involvement.  “Alnylam 2020” marks our expected transition from a late-stage clinical development company to a multi-product commercial-stage company with a robust and sustainable development pipeline – a profile that we believe has rarely been achieved in the biopharmaceutical industry.


Alnylam R&D Day 2014 Webcast and Presentations

December 12, 2014

Alnylam R&D Day 2014 Webcast and Presentations

On December 12, we hosted an R&D Day in New York City. Alnylam management and key opinion leaders discussed the latest progress as well as plans for the future development of our RNAi therapeutics pipeline. At this event, we announced our pipeline growth strategy for development and commercialization of RNAi therapeutics across three Strategic Therapeutic Areas (STArs): Genetic Medicines, Cardio-metabolic Disease, and Hepatic Infectious Disease.



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Positive Initial Phase 1 Clinical Results for ALN-AT3, in Development for the Treatment of Hemophilia and Rare Bleeding Disorders

December 8, 2014

Positive Initial Phase 1 Clinical Results for ALN-AT3, in Development for the Treatment of Hemophilia and Rare Bleeding Disorders

We presented positive initial Phase 1 data for ALN-AT3, an investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders. Preliminary results, presented at the 56th Annual Meeting of the American Society of Hematology (ASH), showed that subcutaneous administration of ALN-AT3 given once weekly for three weeks at low doses of 15 (N=3) or 45 (N=1) micrograms/kg (mcg/kg) resulted in an up to 57% knockdown of AT in hemophilia subjects.  The effects of ALN-AT3 lasted for about 60 days after a single dose. ALN-AT3 was found to be well tolerated in both healthy volunteers and hemophilia subjects enrolled in the study.  These initial results show preliminary evidence for potency and durability of RNAi therapeutics at mcg/kg subcutaneous doses in human studies, and are the first clinical data to be reported for Alnylam’s Enhanced Stabilization Chemistry (ESC)-GalNAc conjugate technology.




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Pre-Clinical Data with ALN-CC5 for Treatment of Complement Mediated Diseases Presented at ASH

December 7, 2014

Pre-Clinical Data with ALN-CC5 for Treatment of Complement Mediated Diseases Presented at ASH

We presented pre-clinical with ALN-CC5, an investigational RNAi therapeutic targeting complement component C5 for the treatment of complement-mediated diseases, at the American Society of Hematology (ASH) Meeting. Data showed an up to 99.2% knockdown of serum C5 and up to 96.2% inhibition of serum hemolytic activity in non-human primates (NHPs) with continued dosing for over seven months.



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New Paper Documenting Alnylam’s Pioneering Discovery of GalNAc-Conjugated siRNA

December 1, 2014

New Paper Documenting Alnylam’s Pioneering Discovery of GalNAc-Conjugated siRNA

We have published an article in the Journal of the American Chemical Society describing the discovery of GalNAc-conjugated siRNA as a novel strategy for delivery of RNAi therapeutics. This publication as a JACS “Communication” documents the landmark discovery by our scientists of GalNAc-conjugates as a potent and durable approach for subcutaneous administration of RNAi therapeutics with a wide therapeutic index.



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New Pre-Clinical Data on RNAi Therapeutic Programs for Cardio-Metabolic Diseases

November 17, 2014

New Pre-Clinical Data on RNAi Therapeutic Programs for Cardio-Metabolic Diseases

We presented pre-clinical data from our investigational RNAi therapeutic programs toward genetically validated targets in development for the treatment of cardiovascular metabolic diseases, including: ALN-PCSsc targeting PCSK9 for the treatment of hypercholesterolemia; ALN-AC3 targeting apolipoprotein C3 (apoC3) for the treatment of hypertriglyceridemia; and ALN-ANG targeting angiopoietin-like 3 (ANGPTL3) for the treatment of genetic forms of mixed hyperlipidemia and severe hypertriglyceridemia.  The data, presented at the American Heart Association (AHA) Scientific Sessions 2014, included new pre-clinical multi-dose data in non-human primates (NHPs) with over six months of dosing for ALN-PCSsc, showing robust and clamped knockdown of PCSK9 of up to 92% and reductions in LDL-C of up to 77% with a once-monthly subcutaneous dosing regimen.  These studies confirm the potential for a once-monthly, and possibly once-quarterly, low volume subcutaneous dose regimen, thus highlighting the emerging profile of our ESC-GalNAc conjugate delivery technology. We believe that ALN-PCSsc represents an innovative, differentiated, and well-validated approach for the treatment of hypercholesterolemia.



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Positive Initial Phase 2 Data with Revusiran (ALN-TTRsc)

November 14, 2014

Positive Initial Phase 2 Data with Revusiran (ALN-TTRsc)

We announced positive initial data from our Phase 2 clinical trial with revusiran (ALN-TTRsc), an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR cardiac amyloidosis. Initial results from the pilot Phase 2 study demonstrated that revusiran was generally well tolerated in patients with significant disease burden.  In addition, data showed an up to 98.2% knockdown of both mutant and wild-type forms of TTR – the disease-causing protein.  As would be expected with the short treatment duration of five weeks, there were no significant changes observed in exploratory clinical measurements.



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New Programs Added to Infectious Disease Pipeline: ALN-HDV and ALN-PDL

November 11, 2014

New Programs Added to Infectious Disease Pipeline: ALN-HDV and ALN-PDL

We have expanded our infectious disease pipeline with two new RNAi therapeutic programs. First, we are advancing ALN-HDV, an RNAi therapeutic targeting the hepatitis delta viral (HDV) genome in development for the treatment of HDV infection. We are also advancing ALN-PDL, an RNAi therapeutic targeting hepatocyte-expressed programmed death ligand 1 (PD-L1) in development for the treatment of chronic liver infections.



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Clinical and Pre-Clinical Data on RNAi Therapeutics at 10th Oligonucleotide Therapeutics Society Meeting

October 14, 2014

Clinical and Pre-Clinical Data on RNAi Therapeutics at 10th Oligonucleotide Therapeutics Society Meeting

We presented new data from multiple clinical and pre-clinical studies at the 10th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS), held October 12 – 15, 2014 in San Diego.  Among multiple presentations, we presented additional data from our Phase 1 trial with ALN-TTRsc showing rapid, dose-dependent, stable, and durable knockdown of serum TTR of up to 96.2%.  In addition, we presented pre-clinical data from a new program, ALN-GO1, an investigational RNAi therapeutic targeting glycolate oxidase (GO) in development for the treatment of primary hyperoxaluria type 1 (PH1), showing efficacy in rodent disease models.  Finally, we presented new pre-clinical research demonstrating that delivery of Enhanced Stabilization Chemistry (ESC)-GalNAc-siRNA conjugates to the lung achieves similar plasma exposure, efficacy, and duration of liver gene silencing as achieved by subcutaneous delivery.  This finding opens up the possibility for needle-less administration of RNAi therapeutics via inhalation for knockdown of liver disease genes.