Presentations

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. [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/EXPLORE-AASLD-Slides_11Nov16.pdf" type="(540 KB PDF)"] View the EXPLORE natural history presentation [/spotlight-link]

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. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=991334" type=" "]Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/OTS_ALN-AAT_Phase-1_092816_Final2.pdf" type="(670 KB PDF)"]View the ALN-AAT Phase 1/2 presentation[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/GalNAc-siRNA_Nonclinical_Review_092816.pdf" type="(2 MB PDF)"]View the poster on review of non-clinical safety data for GalNAc-siRNAs[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/GalNAc-siRNA_vs_ASO_092816.pdf" type="(2 MB PDF)"]View the poster on GalNAc-siRNA vs. GalNAc-ASO[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/Cholesterol-siRNA_Muscle_Delivery_092816.pdf" type="(620 KB PDF)"]View the poster on cholesterol conjugates for muscle delivery[/spotlight-link]  

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. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=990764" type=" "]Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/IPNA_GO1_092416.pdf" type="(970 KB PDF)"]View the presentation[/spotlight-link]

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. [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/AANEM_ALN-CC5_MG_Presentation_09142016.pdf" type="(1 MB PDF)"]View the presentation[/spotlight-link]

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. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=988112" type=" "]Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/SSIEM-2016_AS1_Ph1_Capella_Deck_090716.pdf" type="(840 KB PDF)"]View the presentation[/spotlight-link]

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. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=980988" type=" "]Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/WFH-2016_Fitusiran_Ph-1_072516_2.pdf" type="(760 KB PDF)"]View the presentation[/spotlight-link]

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. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=977940" type=" "]Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ISA-2016_Patisiran-Ph-2-OLE_070116.pdf" type="(480 KB PDF)"]View the initial 24-month patisiran Phase 2 OLE data presentation[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ISA-2016_Correlation_070116.pdf" type="(360 KB PDF)"]View the TTR knockdown, mNIS +7 correlation data presentation[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ISA-2016_APOLLO_070116.pdf" type="(370 KB PDF)"]View the APOLLO baseline demographics presentation[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ISA-2016_Revusiran-Ph2-OLE_070116.pdf" type="(640 KB PDF)"]View the initial 12-month revusiran Phase 2 OLE data presentation[/spotlight-link]

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. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=975341" type=" "]Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/EHA_CC5_Ph1-2_061116.pdf" type="(330 KB PDF)"]View the presentation[/spotlight-link]

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. [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ERA-EDTA_CC5_Ph-1_052216.pdf" type="(880 KB PDF)"]View the presentation[/spotlight-link]

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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