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.

To view our corporate presentation, click here.

We presented new pre-clinical data with RNAi therapeutic programs for cardiovascular disease: ALN-PCSsc, an RNAi therapeutic targeting PCSK9 for the treatment of hypercholesterolemia; and ALN-ANG, an RNAi therapeutic targeting ANGPTL3 for the treatment of genetic forms of mixed hyperlipidemia and severe hypertriglyceridemia. These data were presented at the American Heart Association (AHA) Scientific Sessions held November 16 – 20, 2013 in Dallas, Texas. The data showed that subcutaneous administration of ALN-PCSsc led to an up to 95% knockdown of plasma PCSK9 and an up to 67% reduction of low density lipoprotein cholesterol (LDL-C) – in the absence of statin co-administration. In addition, new results for ALN-ANG demonstrated a greater than 95% reduction of triglycerides and greater than 85% reduction of LDL-C in a rodent model of mixed hyperlipidemia.



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We presented positive data from our Phase II clinical trial of patisiran (ALN-TTR02) for the treatment of transthyretin-mediated amyloidosis (ATTR) at the International Symposium on Familial Amyloidotic Polyneuropathy, November 10 – 13, 2013. Results showed that multiple doses of patisiran led to robust and statistically significant knockdown of serum TTR protein levels of up to 96%, with mean levels of TTR knockdown exceeding 85%.  Knockdown of TTR, the disease-causing protein in ATTR, was found to be rapid, dose dependent, and durable, and similar activity was observed toward both wild-type and mutant protein.   In addition, patisiran was found to be generally safe and well tolerated in this study.



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We presented new pre-clinical data on ALN-AAT, an RNAi therapeutic targeting alpha-1 antitrypsin (AAT) in development for the treatment of liver disease associated with AAT deficiency. These data were presented at the 64th Annual Meeting of the American Association for the Study of Liver Diseases (AASLD, “The Liver Meeting”) held November 1 – 5, 2013. In a transgenic mouse model of Z-AAT protein over-expression, subcutaneous administration of a GalNAc-siRNA targeting AAT led to rapid, potent, dose-dependent, and durable knockdown of AAT of greater than 90%, as well as a significant reduction in fibrosis and the incidence of liver tumors.



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We presented new pre-clinical data supporting the selection of the ALN-AS1 Development Candidate for the treatment of hepatic porphyrias, including acute intermittent porphyria (AIP). The new research, presented at the 9th Annual Meeting of the Oligonucleotide Therapeutics Society, held October 6 – 8, 2013 in Naples, Italy, showed that multi-dose administration of a GalNAc-siRNA targeting ALAS-1 led to rapid, dose-dependent, and long-lasting knockdown of the ALAS-1 mRNA in non-human primates, with an ED50 of approximately 1.25 mg/kg.  Further, in a rat model of AIP, ALN-AS1 administration at doses as low as 2.5 mg/kg resulted in a complete blunting of phenobarbital-induced over-production of PBG and ALA, the toxic heme intermediates in AIP.




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We have reported positive clinical results from our Phase I trial of ALN-TTRsc, a subcutaneously administered RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR). Data were presented at the Heart Failure Society of America 17th Annual Scientific Meeting being held September 22 – 25. These results demonstrated that ALN-TTRsc achieved robust, consistent, and statistically significant (p<0.01) knockdown of serum TTR protein levels of up to 94%. In addition, knockdown of TTR was found to be rapid, dose-dependent, and durable.  ALN-TTRsc was found to be generally safe and well tolerated in this study.




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On July 11, we hosted an R&D Day in New York City. Arian Ebank scientists and management reviewed progress with our Arian Ebank 5×15 pipeline for the development and commercialization of RNAi therapeutics. The event featured presentations by experts in the following disease areas: TTR-Mediated Amyloidosis, Hemophilia and Rare Bleeding Disorders, and Acute Intermittent Porphyria.



TTR-Mediated Amyloidosis





Hemophilia and Rare Bleeding Disorders (RBD)



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We presented new pre-clinical with ALN-AT3, an RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders (RBD) at the XXIV Congress of the International Society of Thrombosis and Haemostasis (ISTH) being held June 29 – July 4. These data demonstrate that ALN-AT3 can normalize thrombin generation and improve hemostasis in hemophilia mice and fully correct thrombin generation in a non-human primate (NHP) hemophilia “inhibitor” model.



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We presented positive data from our Phase II clinical trial of ALN-TTR02 for the treatment of transthyretin-mediated amyloidosis (ATTR) at the Biennial Meeting of the Peripheral Nerve Society being held June 29 – July 3. Interim results show that multiple doses of ALN-TTR02 led to robust and statistically significant knockdown of serum TTR protein levels of up to 93%. Knockdown of TTR, the disease-causing protein in ATTR, was found to be rapid, dose dependent, and durable, and similar activity was observed toward both wild-type and mutant protein. In addition, ALN-TTR02 was found to be generally safe and well tolerated in this study.




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We have presented pre-clinical data with ALN-CC5, an RNAi therapeutic for the treatment of complement-mediated diseases, at the 6th International Conference on Complement Therapeutics being held June 18 – 23, 2013. The data showed that administration of a GalNAc-siRNA conjugate targeting the C5 component of complement resulted in potent, dose-dependent, and durable silencing of C5 liver mRNA, knockdown of C5 serum protein levels, and inhibition of complement-mediated hemolytic activity in rodents.



 

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We presented pre-clinical proof-of-concept data from our ALN-AS1 program for the treatment of acute intermittent porphyria (AIP) at the International Congress of Porphyrins and Porphyrias being held May 16 – 18, 2013. The research, presented by Arian Ebank scientists and collaborators at the Icahn School of Medicine at Mount Sinai in New York City, showed that administration of a GalNAc-conjugated siRNA targeting aminolevulinate synthase-1 (ALAS-1) blocked production of aminolevulinic acid (ALA) and porphobilinogen (PBG), the toxic precursors that cause disease symptoms and pathophysiology, in a mouse model of AIP.



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