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

Pre-Clinical Data on ALN-AAT at Digestive Disease Week

May 17, 2015

Pre-Clinical Data on ALN-AAT at Digestive Disease Week

We presented pre-clinical data on ALN-AAT, an investigational RNAi therapeutic targeting alpha-1 antitrypsin (AAT) for the treatment of AAT deficiency-associated liver disease. New data, presented at the Digestive Disease Week (DDW) meeting, held May 16 – 19, 2015, showed a robust knockdown of serum AAT of up to 93% in non-human primates (NHPs) with monthly subcutaneous dosing. This level of knockdown was highly durable, lasting for greater than 30 days following the final dose. Further, ALN-AAT was found to have a wide therapeutic index based on results from GLP toxicology studies. In addition, study results were reported from a transgenic mouse model of alpha-1 liver disease, where mice overexpress the human Z-AAT protein.



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12-Month Clinical Data from Patisiran Phase 2 Open-Label Extension (OLE) Study

April 21, 2015

12-Month Clinical Data from Patisiran Phase 2 Open-Label Extension (OLE) Study

We presented initial 12-month clinical data from our ongoing Phase 2 open-label extension (OLE) study with patisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin (TTR)-mediated amyloidosis in patients with familial amyloidotic polyneuropathy (FAP). Study results, presented at the 67th Annual Meeting of the American Academy of Neurology (AAN) being held April 18 – 25, showed a mean 2.5 point decrease in modified Neuropathy Impairment Score (mNIS+7) at 12 months in patients who had reached the 12-month endpoint (N=20) at the time of data cutoff. This decrease in neuropathy progression compares favorably to the 13 to 18 point increase in mNIS+7 at 12 months that can be estimated from the literature in untreated FAP patients with similar baseline characteristics.



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Pre-Clinical Results with ALN-AT3 Published in “Nature Medicine”

April 13, 2015

Pre-Clinical Results with ALN-AT3 Published in “Nature Medicine”

We have published pre-clinical results with ALN-AT3, an investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia, in Nature Medicine. The paper documents a broad set of pre-clinical data supporting the clinical advancement of ALN-AT3. Among the many findings reported, subcutaneous administration of ALN-AT3 led to potent, dose-dependent, and durable knockdown of AT in wild-type mice, hemophilia A mice, and non-human primates (NHPs). In addition, ALN-AT3 treatment improved hemostasis in hemophilia mice and normalized thrombin generation in a non-human primate “inhibitor” model of hemophilia A (HA). Furthermore, long-term ALN-AT3 administration – even at highly exaggerated doses – was shown to be well tolerated in hemophilia mice, supporting a wide therapeutic index in the disease setting.



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Results from Retrospective Natural History Study of Patients with Familial Amyloidotic Cardiomyopathy (FAC)

March 15, 2015

Results from Retrospective Natural History Study of Patients with Familial Amyloidotic Cardiomyopathy (FAC)

We presented results from a retrospective natural history study evaluating disease progression in transthyretin (TTR)-mediated amyloidosis (ATTR amyloidosis) patients with familial amyloidotic cardiomyopathy (FAC). These results were presented at a meeting with members of the Association of Black Cardiologists (ABC) at the American College of Cardiology (ACC) Annual Scientific Session, held March 14 – 16, 2015. Amongst other findings, study results showed a mean decline of 140 meters in 6-minute walk distance (6-MWD) over an 18-month period in FAC patients with mild-to-moderate heart failure. Also at the ACC meeting, we presented complete results from our Phase 2 clinical trial with revusiran, an investigational RNAi therapeutic for the treatment of FAC. Consistent with preliminary results presented last year, revusiran achieved an up to 98.2% knockdown of serum TTR – the disease-causing protein – and was found to be generally well tolerated.




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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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