2017-09-26 Daily Pharma Scoop: Alnylam’s Big Win, Aradigm NDA and Rating, EXEL and AZN Japan expansions

  • Alnylam’s recent win with patisiran is discussed.
  • Aradigm files NDA, will see PDUFA in January.
  • AstraZeneca and Exelixis both have successful Japanese trials in different indications.

Analysis of top Seeking Alpha coverage: Alnylam

Today’s coverage is for Alnylam And Sanofi Achieve An RNAi First by Long Term Bio. Alnylam (ALNY) recently reported positive results (a p value of less than 0.00001) from its APOLLO trial of patisiran targeting familial amyloid polyneuropathy (‘FAP). FAP are a group of genetically transmitted rare nervous disorders where nerve lesions are induced due to deposition of amyloid fibrils. This is most commonly caused by mutated transthyretin (‘TTR), which is the target of patisiran. FAP is a rare disease, with only 1 in 100,000 people throughout most of the US and Europe having the disease, although the disease is more prevalent in some countries. However, the story here is twofold - first, that patisiran is the first RNAi therapy to pass a phase 3 trial, and two, patisiran will compete with Ionis Pharma’s (IONS) inotersen, which reported data in April and is one step ahead of ALNY as a result. RNAi and antisense and two RNA therapeutics approaches, and inotersen is antisense while patisiran is RNAi.

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