Alnylam Surprises Investors With Last-Minute HELIOS-B Revisions

 
• By Joseph Haas
  
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Alnylam pushed back the study’s top-line readout to June or July. But analysts said the changes increase the odds of demonstrating monotherapy benefit with Amvuttra in ATTR-CM.

Alnylam building
Alnylam's trial changes are intended to demonstrate monotherapy benefit with Amvuttra • Source: Shutterstock

Overshadowing the strong sales growth revealed in its fourth quarter 2023 earnings call, Alnylam Pharmaceuticals Inc. worried investors on 15 February with a late change to the endpoints and expected topline readout for the Phase III HELIOS-B study that it hopes will support expanding Amvuttra’s label into transthyretin amyloid cardiomyopathy (ATTR-CM), which would substantially increase the drug’s addressable population.

Key Takeaways

  • Alnylam is expanding the length of follow-up in its Phase III trial to add ATTR-CM to Amvuttra’s label, which it says would increase the drug’s target market ten-fold.

The second RNAi therapy from Alnylam approved for transthyretin-associated diseases, Amvuttra (vutrisiran) got US Food and Drug Administration approval in...

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