On Deck For Alnylam: Three Phase III Starts In 2025

The RNAi specialist outlined plans to initiate three Phase III trials this year – two for nucresiran in ATTR amyloidosis and one for zilebesiran for hypertension – during an R&D day in New York.

(Shutterstock)
Key Takeaways

Alnylam showcased its RNAi pipeline during a four-hour R&D day in New York.

A big focus was the next-generation TTR silencer nucresiran and the hepatic angiotensinogen synthesis inhibitor zilebesiran for hypertension, both of which are moving into Phase III.

Further out, management highlighted Phase I assets in neuroscience, including mivelsiran for Alzheimer’s disease and ALN-HTT02 for Huntington’s disease.

The big milestone for Alnylam this year is the expected US Food and Drug Administration approval of Amvuttra (vutrisiran) for...

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

More from R&D

ASCO: Pushing The Frontiers Of ADCs

 

Antibody-drug conjugates are a major part of the oncology pipeline, and data presented at the American Society of Clinical Oncology meeting highlight the emergence of new technologies.

Skyhawk Looks To Soar With Huntington’s Hope

 
• By 

Oral RNA splicing modulator has gone into a Phase II/III trial

Scholar Rock Shows Muscle-Building PoC In Obesity

 

The company said it intends to maintain a focus on rare neuromuscular disease with apitegromab but could consider partnering in obesity.

Spyre Takes Aim At Roche And J&J In Inflammatory Diseases

 

The US firm was already looking to compete in ulcerative colitis, but now expands its ambitions by moving into rheumatoid arthritis as well.

More from Advanced Therapies

Sarepta Market Dynamics For Elevidys Imperiled By Second Patient Death

 
• By 

With two deaths in non-ambulatory DMD patients, Sarepta is attempting damage control and will ask the US FDA to advise, setting up a possible confrontation with CBER director Prasad.

BMS’s Breyanzi Stands To Further Expand Broad Lymphoma Coverage

 

The drugmaker presented data at a lymphoma meeting showing strong efficacy for the CAR-T in marginal zone lymphoma, the second most common indolent lymphoma.

Spain’s SpliceBio Secures Sanofi and Roche Support In $135m Financing

 
• By 

Series B cash will be used to advance its Stargardt disease gene therapy.