Wave Life Sciences soars on positive DMD trial results

25 September 2024

US RNA specialist Wave Life Sciences (Nasdaq: WVE) has announced positive interim data from the ongoing Phase II FORWARD-53 study of WVE-N531, which is an exon skipping oligonucleotide being investigated in boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping.

Wave Life noted that the interim analysis was conducted after 24 weeks of 10mg/kg dosing every two weeks (Q2W), and WVE-N531 demonstrated substantial dystrophin expression and that it was safe and well tolerated, with the news sending the firm’s shares up more than 53% to $8.19 by close of trading on Tuesday.

The FORWARD-53 data demonstrated "meaningful" improvements in serum biomarkers for muscle health, according to Wave, with WVE-N531 localizing in myogenic stem cells in the majority of participants and aiding in the regeneration of myofibers. The estimated half-life of WVE-N531 in muscle tissue was 61 days, something Wave says supports a possible monthly dosing regimen moving forward.

This article is accessible to registered users, to continue reading please register for free.  A free trial will give you access to exclusive features, interviews, round-ups and commentary from the sharpest minds in the pharmaceutical and biotechnology space for a week. If you are already a registered user please login. If your trial has come to an end, you can subscribe here.

Login to your account

Become a subscriber

 

£820

Or £77 per month

Subscribe Now
  • Unfettered access to industry-leading news, commentary and analysis in pharma and biotech.
  • Updates from clinical trials, conferences, M&A, licensing, financing, regulation, patents & legal, executive appointments, commercial strategy and financial results.
  • Daily roundup of key events in pharma and biotech.
  • Monthly in-depth briefings on Boardroom appointments and M&A news.
  • Choose from a cost-effective annual package or a flexible monthly subscription
The Pharma Letter is an extremely useful and valuable Life Sciences service that brings together a daily update on performance people and products. It’s part of the key information for keeping me informed

Chairman, Sanofi Aventis UK

Companies featured in this story

More ones to watch >


Today's issue

Company Spotlight





More Features in Biotechnology