1. Scrip
  2. >>R&D
  3. >>Clinical Trials

Alnylam Surprises Investors With Last-Minute HELIOS-B Revisions

 
• By Joseph Haas

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 firm’s stock price fell on the news, but most analysts said the HELIOS-B protocol changes, while late, make sense and improve prospects for success

The second RNAi therapy from Alnylam approved for transthyretin-associated diseases, Amvuttra (vutrisiran) got US Food and Drug Administration approval in June 2022 to treat hereditary transthyretin-mediated amyloidosis (hATTR)-associated polyneuropathy. (Also see "Alnylam’s Amvuttra To Take Off From Onpattro’s Runway" - Scrip, 14 June, 2022.) It joined Onpattro (patisiran), which also is approved for hATTR but failed to get ATTR-CM added to its label based on findings from the Phase III APOLLO-B study

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Scrip for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required
Start Free Trial

Already a subscriber?

More from Clinical Trials

Leo Roars To A Profit In Q1 As Adbry Grows In US

 
• By Kevin Grogan

The Danish firm's atopic dermatitis drug is holding its own despite strong competition from Sanofi/Regeneron’s Dupixent.

Azafaros Banks Over €130m To Advance Nizubaglustat Into Phase III

 
• By Kevin Grogan

The therapy has been developed for GM1 and GM2 gangliosidoses and Niemann-Pick disease type C.

Lilly Builds Obesity Momentum With New SURMOUNT Data Besting Novo Nordisk

 
• By Alaric DeArment

The presentation and publication of the Phase IIIb study showing greater efficacy for Zepbound over Wegovy come at a time when competition has been heating up between the two obesity medications.

Insilico Fast-Tracks First AI-Designed TNIK Inhibitor For IPF

 
• By Dexter Jie Yan

InSilico will leapfrog Phase IIb to progress its AI-generated candidate for idiopathic pulmonary fibrosis directly into a Phase III trial in China, as it prepares for an IPO in Hong Kong.

More from R&D

Azafaros Banks Over €130m To Advance Nizubaglustat Into Phase III

 
• By Kevin Grogan

The therapy has been developed for GM1 and GM2 gangliosidoses and Niemann-Pick disease type C.

Lilly Builds Obesity Momentum With New SURMOUNT Data Besting Novo Nordisk

 
• By Alaric DeArment

The presentation and publication of the Phase IIIb study showing greater efficacy for Zepbound over Wegovy come at a time when competition has been heating up between the two obesity medications.

Which Firms Are Most Exposed To Prasad’s Possible Accelerated Approvals Clampdown?

 
• By Andrew McConaghie

Vaccine and accelerated approvals are expected to face greater scrutiny under new CBER leader Vinay Prasad, but he has promised “no light switch change” to policy, giving some reassurance to companies whose share prices have been battered.