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

New Duchenne Results Put Avidity On Course For Filing

 
• By Andrew McConaghie

The potential first-in-class antibody oligonucleotide conjugate could treat a form of the muscle wasting disease, and could be the first of Avidity’s trio of rare disease drugs to gain approval.

(Shutterstock)

Avidity has further strengthened the case for its Duchenne muscular dystrophy candidate, delpacibart zotadirsen, with Phase I/II data showing it boosted levels of the missing dystrophin muscle protein.

Key Takeaways
  • Del zota restored total dystrophin levels to up to 58% of normal and cut by 80% creatine kinase, a key biomarker of muscle damage

The San Diego, CA-based company is developing delpacibart zotadirsen (del zota) as the lead candidate in its new class of RNA therapeutics called antibody oligonucleotide conjugates, and confirmed it was on course to file with the US Food and Drug Administration by

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 Business

In Brief: US FDA Delay Sends Biohaven Shares Down

 
• By Andrew McConaghie

The US regulator’s decision has taken Biohaven and investors by surprise, with the reason for a ‘major amendment’ status not clear.

In Brief: Sanofi Commits $20bn To US R&D And Manufacturing By 2030

 
• By Sushmita Panda

Move by French major aligns with broader industry efforts to localize production amid political pressure, as Trump pushes to onshore manufacturing and hints at easing tariff threats.

Eisai Forecasts 73% Leqembi Sales Rise In FY25 As Demand Expands

 
• By Mandy Jackson

Eisai spent most of its FY24 earnings call discussing plans for growing sales of Biogen-partnered Alzheimer’s drug Leqembi from JPY44.3bn ($304.1m) to JPY76.5bn ($525.1m) in FY25.