Industry Partner News: Wave Life Sciences Announces Positive Phase 1 Results for WVE-210201 in Duchenne Muscular Dystrophy (DMD)

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December 6, 2018

Dear Duchenne Community,

We would like to share an exciting update about our lead Duchenne muscular dystrophy (DMD) clinical program. This morning, Wave Life Sciences announced positive safety and tolerability results from the WVE-210201 Phase 1 clinical trial in boys with Duchenne muscular dystrophy who are amenable to exon 51 skipping.

The data from this trial support our moving forward with a Phase 2/3 clinical trial of WVE-210201, which we intend to initiate in 2019, and propel us towards achieving our collective goal of inducing meaningful, natural dystrophin expression in boys with DMD.

For more information, we invite you to read our announcement here.

Importantly, thank you to all of the boys and their families who are participating in this Phase 1 trial and its open-label extension, as well as the advocacy organizations that have provided invaluable guidance and collaboration along the way. Your contributions and partnership are deeply appreciated.

At Wave Life Sciences, we are focused on delivering transformational therapies for patients with serious, genetically-defined diseases. Today’s announcement has taken us one step closer to achieving this objective. We look forward to continuing our engagement with you as we work together to make a difference for people impacted by Duchenne.

Sincerely,

Paul Bolno, MD, MBA
President and Chief Executive Officer

Wendy Erler, MBA
Vice President, Patient Advocacy and Market Insights
Wave Life Sciences