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Alnylam's patisiran shows promise of halting neuropathy progression in patients with genetic disorder

Apr. 21, 2015 8:47 AM ETAlnylam Pharmaceuticals, Inc. (ALNY) StockALNYBy: Douglas W. House, SA News Editor
  • At the 67th Annual Meeting of the American Academy of Neurology, Alnylam (NASDAQ:ALNY) presented 12-month clinical data from its ongoing Phase 2 open-label extension study of its RNAi therapeutic, patisiran, in development for the treatment of transthyretin-mediated amyloidosis in patients with familial amyloidotic polyneuropathy (FAP), a genetic disorder characterized by abnormal deposits of the protein amyloid in the body's tissues and organs. It is caused by mutations in the TTR gene, which provides instructions for producing transthyretin (TTR).
  • The results showed a mean 2.5-point decrease in modified Neuropathy Impairment Score at 12 months in patients who had reached the 12-month endpoint (n=20) at the time of data cutoff. This compares favorably with the 13 - 18-point increase cited in the literature in untreated FAP patients with similar baseline characteristics. Patients treated with patisiran achieved a sustained mean serum TTR knockdown at the 80% target level for ~16 months, with up to an 88% mean knockdown between doses. Researchers believe that TTR knockdown has the potential to halt neuropathy progression in FAP patients.
  • 18-month data should be available later this year. The company will host a conference call this morning at 9:00 am ET to discuss the results.
  • Shares are up 9% premarket on light volume.

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