Revised Cash Guidance
As a result of the decisions announced above, Wave now expects that its existing cash and cash equivalents, together with expected and committed cash from existing collaborations, will enable Wave to fund its operating and capital expenditure requirements into the third quarter of 2021.
Investor Conference Call and Webcast
Wave management will host an investor conference call today at 8:00 a.m. ET. The conference call may be accessed by dialing +1 (866)220-8068 for participants based in the United States or +1 (470)495-9153 for participants based outside the United States, and entering conference ID 9967079. The live webcast may be accessed by visiting the “For Investors & Media” section of the Wave Life Sciences website at www.ir.wavelifesciences.com. Following the webcast, a replay will be available on the website.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a fatalX-linked genetic neuromuscular disorder caused predominantly byout-of-frame deletions in the dystrophin gene, resulting in absent or defective dystrophin protein. Dystrophin protein is needed for normal muscle maintenance and operation. Because of the genetic mutations in DMD, the body cannot produce functional dystrophin, which results in progressive and irreversible loss of muscle function, including the heart and lungs. Worldwide, DMD affects approximately one in 5,000 newborn boys.
About Suvodirsen
Suvodirsen is an investigational stereopure oligonucleotide previously in development as a treatment for patients with Duchenne muscular dystrophy (DMD) who have genetic mutations amenable to exon 51 skipping. Wave initiated clinical development of suvodirsen in November 2017 and completed a Phase 1 safety and tolerability study in early 2019. Based on an interim analysis from a Phase 1 Open Label Extension (OLE) study conducted in December 2019, Wave discontinued development of suvodirsen.
Approximately 13% of DMD patients have mutations amenable to treatment with an exon 51 skipping therapy. Exon-skipping technology is intended to induce cellular machinery to ‘skip over’ a targeted exon and restore the reading frame, resulting in the production of internally truncated, but functional dystrophin protein.
About Wave Life Sciences
Wave Life Sciences (Nasdaq: WVE) is a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases. Wave aspires to developbest-in-class medicines across multiple therapeutic modalities using PRISMTM, the company’s proprietary discovery and drug development platform that enables the precise design, optimization and production of stereopure oligonucleotides. Driven by a resolute sense of urgency, the Wave team is targeting a broad range of genetically defined diseases so that patients and families may realize a brighter future. To find out more, please visit www.wavelifesciences.com and follow Wave on Twitter @WaveLifeSci.
Forward-Looking Statements
This press release contains forward-looking statements concerning our goals, beliefs, expectations, strategies, objectives and plans, and other statements that are not necessarily based on historical facts, including statements regarding the following, among others: our commitment to advancing genetic medicines in diseases of the central nervous system, eye and liver; our intent to rapidly incorporate our learnings from the Phase 1 OLE study; our ability to deliver on the promise of our current and future pipeline; our plans to report topline clinical data from thePRECISION-HD2 trial; the future performance and results of our programs in clinical trials and in preclinical development; the potential benefits of PRISM and our stereopure oligonucleotides compared with stereorandom oligonucleotides; the benefit of nucleic acid therapeutics generally; and the anticipated duration of our cash runway. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including the following: our ability to finance our drug discovery and development efforts and to raise additional capital when needed; the ability of our preclinical programs to produce data sufficient to support our clinical trial applications and the timing thereof; our ability to continue to build and maintain the company infrastructure and personnel needed to achieve our goals; the