Phase 1 Trial of WVE-210201 Positive for DMD Amenable to Exon 51 Skipping


WVE-210201 was granted both Orphan Drug and Rare Pediatric Disease designations from the FDA, as well as Orphan Drug status in the European Commission.

Dr Paul Bolno

Paul Bolno, MD, MBA, the president and chief executive officer of Wave Life Sciences

Paul Bolno, MD, MBA

Positive results from a phase 1 study of WVE-210201 (NCT03508947), an investigational agent for boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping, have been announced.1

Wave Life Sciences, the manufacturer of the therapy, revealed that the findings support the initiation of a phase 2/3 study. Additionally, based on the results of 4 ascending dose cohorts in the phase 1 trial as well as the final analysis (which is still pending), Wave Life has noted it has selected a dose for the aforementioned phase 2/3 trial, which is expected to begin in 2019. Final results of this phase 1 trial, as well as the details of that phase 2/3 trial, will be presented at future scientific meetings.

“These results mark an important milestone for the Duchenne community and toward our goal of inducing meaningful, natural dystrophin expression in boys with DMD who are amenable to exon 51 skipping,” Paul Bolno, MD, MBA, the president and chief executive officer of Wave Life Sciences, said in a statement. “This is an exciting time at Wave as we continue to partner with the patient and medical communities to advance our lead program in DMD. We thank all of the boys and their families who are participating in this Phase 1 trial and its open-label extension and are grateful for their trust and commitment.”

Preclinical experiments have shown that the WVE-210201 was associated with a 52% increase in the levels of dystrophin protein compared with normal skeletal muscle tissue.2 Previously, WVE-210201 was granted both Orphan Drug and Rare Pediatric Disease designations from the FDA, following its Orphan Drug status granted by the European Commission.

The phase 1 trial assessed 4 primary outcome measures of safety in 40 patients: the number of patients with adverse events (AEs), the severity of those AEs, the number of patients with serious AEs (SAEs), and the number of patients who withdrew due to AEs. Secondary outcomes consisted of the maximum observed concentration (Cmax) at day 1, day 2, and day 8; the time to Cmax (Tmax) at day 1, day 2, and day 8; and the area under the plasma concentration-time curve (AUC0-t) at day 1, day 2, and day 8.

The therapy is also being assessed in an ongoing, multi-dose, open-label extension trial, initiated in August 2018 to those completing the phase 1 trial, which Wave Life plans to use in its submission for accelerated approval with the FDA. According to Wave Life, “the company remains on track to deliver an interim analysis of dystrophin expression from muscle biopsies in boys receiving WVE-210201 in the OLE study in the second half of 2019.”

“PPMD continues to be optimistic about the progress the team at Wave Life Sciences is making with their stereopure exon 51 skipping program for Duchenne,” Pat Furlong, founding President and CEO of Parent Project Muscular Dystrophy (PPMD). “We are pleased to see WVE-210201 advance on the clinical development path and look forward to more updates from the Wave team.”


1. Wave Life Sciences Announces Positive Phase 1 Results for WVE-210201 in Duchenne Muscular Dystrophy (DMD) [press release]. Cambridge, MA: Wave Life Sciences; Published December 6, 2018. Accessed December 6, 2018.

2. M. Wood, J. Zhang, K. Bowman, et al. WVE-210201, an investigational stereopure oligonucleotide therapy for Duchenne muscular dystrophy, induces Exon 51 skipping and dystrophin protein restoration. Neuromuscular Disord. 2017;27(2):S217. doi: 10.1016/j.nmd.2017.06.442

Related Videos
Michael Levy, MD, PhD
Michael Kaplitt, MD, PhD
Michael Kaplitt, MD, PhD
video 4 - "Amyloid Cascade Hypothesis of Alzheimer’s Disease"
Video 3 - "Amyloid Precursor Protein and Amyloid Beta Species in Alzheimer’s Disease"
Svetlana Blitshteyn, MD, FAAN, director and founder of Dysautonomia Clinic
© 2024 MJH Life Sciences

All rights reserved.