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Alnylam Reports Complete 18-Month Data from Ongoing Phase 2 Open-Label Extension Study of Patisiran, an Investigational RNAi Therapeutic Targeting Transthyretin for the Treatment of Hereditary ATTR Amyloidosis with Polyneuropathy (hATTR-PN)

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Additional Relevant Details Alnylam the leading RNAi therapeutics company, announced today complete 18-month data from its ongoing Phase 2 open-label extension (OLE) study with patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of hereditary ATTR amyloidosis with polyneuropathy (hATTR-PN), also known as familial amyloidotic polyneuropathy (FAP). These new clinical data are being presented in an oral talk at the 68th Annual Meeting of the American Academy of Neurology (AAN) being held April 15 - 21, 2016 in Vancouver, British Columbia, Canada. Complete 18-month data (N=27) from the study provide continued evidence that patisiran has the potential to halt neuropathy progression in patients with hATTR-PN. In the first reported exploratory analysis of its kind, the degree of TTR knockdown observed in patients was shown to correlate with improvement in neuropathy impairment scores. Further, in this Phase 2 OLE trial, patisiran was found to be generally well tolerated with up to 25 months of treatment. Dosing continues in the patisiran Phase 2 OLE study, and the Company plans to report initial 24-month data from the trial in mid-2016.
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Apr 20, 2016
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Related Keywords Phase 2 Open-label Extention Study, Patisiran, Rnai Therapeutic Targeting Transthyretin, Attr Amyloidosis, Polyneuropathy, Hattr-pn