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Alnylam shares surge 40% on drug results that may herald a new type of medicine

Patisiran, which is intended for the rare and progressive disease hereditary ATTR amyloidosis with polyneuropathy, is “the first ever positive Phase 3 results for an RNAi therapeutic, marking the potential arrival of an entirely new class of medicines,” said Alnylam ALNY, +52.04% Chief Executive John Maraganore.

The Wednesday news is a “big win for ALNY and RNAi,” especially after Alnylam halted a hemophilia drug’s development earlier this month and was forced to discontinue another Drug last fall, said Leerink Partners analyst Paul Matteis.

The results also appear to be a best-case scenario for the company, he said, noting that the safety data overall look “clean and sufficient to quell any concerns of revusiran,” which was discontinued last fall after patient deaths.

Individuals with ATTR amyloidosis have abnormal proteins in their body that can then damage bodily functions, affecting the heart and nervous and gastrointestinal systems.

Safety issues were also largely similar between the therapy and the placebo, except for mild-to-moderate peripheral edema, or swelling in the arms and legs, and infusion-related reactions, both of which were more commonly seen in patients on patisiran.

READ MORE (MarketWatch)

  • Cheer Alnylam's Drug, Pause on Its PlatformBloomberg
  • UPDATED: Alnylam achieves breakthrough RNAi success as PhIII patisiran study ...Endpoints News
  • After 20 Years, A Drug That Silences Genes SucceedsForbes
  • New Drug Shows Promise Against Rare Gene DisorderNewsmax
  • Alnylam Shares Skyrocket on Clinical Study Results - Biotech
  • Why Alnylam Pharmaceuticals Is Wednesday's Top Stock24/7 Wall St.
  • Alnylam jumps after late stage trial success for rare disease drugFinancial Times
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Alnylam shares surge 40% on drug results that may herald a new type of medicine


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