Capella

Capella—the Online Voice of Progress in RNAi

Alnylam welcomes you to Capella, the destination for updates on our progress in translating the science of RNAi into innovative medicines. For Alnylam, Capella is our online voice for communicating the scientific progress we are making as we work to develop innovative medicines for patients.

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We reported new results from Part C of our Phase 1/2 clinical study of ALN-CC5, a subcutaneously administered investigational RNAi therapeutic targeting complement component C5 for the treatment of complement-mediated diseases, at the 58th Annual Meeting of the American Society of Hematology (ASH), held December 3 – 6, 2016 in San Diego, California.



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We reported positive interim results from our Phase 1 study of fitusiran in patients with hemophilia with inhibitors as well as from our Phase 2 open-label extension (OLE) study in hemophilia patients without inhibitors at the 58th Annual Meeting of the American Society of Hematology (ASH), held December 3 – 6, 2016 in San Diego, California.





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We reported positive initial results from Cohorts 1 and 2 in Part C of our Phase 1 study with givosiran (gi-VOH-si-ran), the International Nonproprietary Name for ALN-AS1, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyrias.  These results were presented at the 58th Annual Meeting of the American Society of Hematology (ASH), held December 3 – 6, 2016 in San Diego, California.



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We reported interim results from our ongoing EXPLORE study, a multinational, prospective, observational study aimed at characterizing the natural history and clinical management of patients with acute hepatic porphyrias (AHP) who experience recurrent attacks or receive prophylactic treatment to prevent attacks.  Results from 112 patients, of which 104 have acute intermittent porphyria (AIP), showed a mean of 9.5 acute attacks reported in the prior year with severe neuropathic pain as the cardinal feature in 100% of attacks, along with other symptoms including nausea or vomiting (>80%), fatigue (77%), and weakness (79%).  Approximately 64% of patients reported experiencing porphyria symptoms between attacks, with about 46% experiencing symptoms daily.  Patients also reported a diminished quality of life and significant healthcare utilization, with a mean of 4.6 overnight hospitalizations per year (range 0-70) and a mean hospital stay duration of 6.6 days (range 1-60).  Of the attacks reported on study, approximately 76% of them required treatment with heme or at a healthcare facility.


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We presented clinical and non-clinical data in a series of posters and oral presentations at the 12th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS), held September 25 – 28, 2016 in Montreal, Quebec.






 

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We presented initial clinical data from ALN-GO1, an investigational RNAi therapeutic targeting glycolate oxidase (GO) for the treatment of Primary Hyperoxaluria Type 1 (PH1), at the 17th Congress of the International Pediatric Nephrology Association (IPNA), held September 20 – 24, 2016 in Iguaçu, Brazil.



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We reported new pre-clinical results in animal models of myasthenia gravis (MG) from ALN-CC5, an investigational RNAi therapeutic in development for the treatment of complement-mediated diseases, at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting, held September 14-17 in New Orleans, Louisiana.


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We presented interim results from our ongoing Phase 1 clinical trial with ALN-AS1, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyrias (AHP), at the 2016 Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium, held from September 6 – 9, 2016 in Rome, Italy.



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We reported new data from Parts C and D of our Phase 1 study with fitusiran, an investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders, at the World Federation of Hemophilia (WFH) World Congress, held July 24-28, 2016 in Orlando, Florida.



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We reported new clinical data from our ongoing Phase 2 open-label extension (OLE) studies with patisiran and revusiran, investigational RNAi therapeutics targeting transthyretin (TTR) for the treatment of hereditary TTR-mediated amyloidosis (hATTR amyloidosis). These data were presented at the XV International Symposium on Amyloidosis held July 3 – 7, 2016 in Uppsala, Sweden.






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