Capella

Capella—the Online Voice of Progress in RNAi

Arian Ebank welcomes you to Capella, the destination for updates on our progress in translating the science of RNAi into innovative medicines. For Arian Ebank, 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 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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We reported data from 6 patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) enrolled in Part C of our ongoing Phase 1/2 clinical study with ALN-CC5, an investigational RNAi therapeutic targeting complement component 5 (C5) for the treatment of complement-mediated diseases. These data were presented at the 21st Congress of the European Hematology Association (EHA) Meeting in Copenhagen, Denmark, held June 9-12, 2016.  In this part of the study, ALN-CC5 was evaluated as a monotherapy or as an adjunct to eculizumab, an approved anti-C5 monoclonal antibody indicated for the treatment of PNH.



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We reported updated data from our ongoing Phase 1/2 clinical study with ALN-CC5, an investigational RNAi therapeutic targeting complement component C5 for the treatment of complement-mediated diseases. Data were presented at the 53rd Congress of the European Renal Association – European Dialysis and Transplant Association (ERA-EDTA) in Vienna, Austria, held May 21-24, 2016.  Results showed that administration of ALN-CC5 in healthy volunteers (N=44) achieved up to 99% knockdown of serum C5 and a mean maximum of 86% serum hemolysis inhibition in the highest dose group, with mean levels consistently greater than 80% inhibition through the 13 weeks of treatment. Results also showed a mean maximum CH50 inhibition of 99.6% and maximum inhibition up to 100% relative to baseline.

 

The effects of ALN-CC5 were found to be highly durable, with C5 knockdown clamped at over 90% for more than six months following a single dose. C5 knockdown and complement inhibition results support the potential for a once-quarterly dosing regimen when used in combination with the monoclonal antibody, eculizumab. Importantly, ALN-CC5 was shown to be generally well tolerated, with no serious adverse events and no drug-related discontinuations to date. All adverse events were mild or moderate in severity.


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We reported results from our ongoing Phase 2 open-label extension (OLE) study with patisiran at the 68th Annual Meeting of the American Academy of Neurology (AAN), held April 15 – 21, 2016 in Vancouver, British Columbia, Canada.  Complete 18-month data (N=27) from the study provided continued evidence that patisiran has the potential to halt neuropathy progression in patients with hATTR-PN (also known as familial amyloidotic polyneuropathy, or FAP), showing a mean 0.8-point decrease in mNIS+7, which compares favorably to an estimated increase in mNIS+7 of 22 to 26 points at 18 months based upon analysis of historical data sets in untreated hATTR-PN patients with similar baseline neurologic impairment. Patisiran administration was also associated with a statistically significant, approximately 77% median improvement in nerve fiber density as read histologically in a blinded manner from distal thigh sweat gland biopsy samples.
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We’re announcing the addition of a new program to our genetic medicines pipeline, ALN-F12, an investigational RNAi therapeutic targeting F12 for the treatment of Hereditary Angioedema (HAE). Pre-clinical data for ALN-F12 were presented at the American Academy of Allergy, Asthma & Immunology (AAAAI) Annual meeting, held March 4-7, 2016.

HAE is a genetic disorder characterized by episodes of severe swelling in various parts of the body, including the face, hands, feet, gastrointestinal tract, and airways. It is caused by a defect in the C1-inhibitor gene that results in poor control of contact pathway activation and excessive bradykinin generation. Elevated levels of bradykinin increase vascular permeability, ultimately causing the episodic swelling attacks that are characteristic of HAE. The F12 gene encodes Factor XII (FXII), which is at the top of the contact pathway cascade. Pre-clinical data showed that administration of ALN-F12 resulted in dose-dependent reduction of vascular permeability in two different mouse models of bradykinin-driven vascular leakage, demonstrating that suppression of F12 mRNA has the potential to mitigate excess bradykinin stimulation. Further, in non-human primates, a single subcutaneous dose of ALN-F12 at 3 mg/kg resulted in potent and durable knockdown of serum FXII of greater than 85 percent, with knockdown of over 50 percent sustained out to three months following administration.


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