WAVE Life Sciences to advance next-generation nucleic acid therapies for Duchenne Muscular Dystrophy

WAVE’s DMD program is based on preclinical data demonstrating an approximate 25-fold improvement in exon-skipping efficiency compared to drisapersen and eteplirsen, suggesting the potential for improved potency and an enhanced ability to restore the production of functional dystrophin. In addition, WAVE’s proprietary muscle targeting technology has demonstrated substantial improvement in distribution to critical tissues in animal models, including skeletal muscle, diaphragm, and heart.

"We recognize the acute need of the Duchenne community for therapeutic options to address this devastating disease, and also appreciate regulators’ requirements for strong, well-validated, scientific evidence. Our goal is to fulfill both of these needs by bringing forward optimally designed drugs through robust clinical trials," said Paul Bolno, M.D., MBA, President and CEO of WAVE Life Sciences.

"Based on the strong preclinical data we’ve seen to date, we are highly encouraged that we are on track to develop exon-skipping medicines that maximize potency with a favorable safety profile. In addition, we plan to conduct rigorous, well-designed clinical trials that explore various predictive biomarkers and evaluate comprehensive endpoints."

WAVE’s planned clinical trials will include both ambulatory and non-ambulatory patients in order to evaluate preservation of walking ability, heart and lung function.

WAVE’s initial DMD candidate skips exon 51 in the dystrophin gene. The company will initiate IND-enabling studies in 2016 and intends to initiate its first clinical trial of the candidate in the second half of 2017.

WAVE will also leverage its novel stereopure chemistry platform to advance therapies targeting additional DMD-related exons to expand the potential impact of its promising approach to a broader group of patients.

WAVE will present data at the upcoming Parent Project Muscular Dystrophy’s (PPMD) Annual Connect Conference being held June 26 – 29, 2016 in Orlando, Florida.

"We are excited whenever a new company enters the Duchenne space," said Pat Furlong, Founding President and CEO of PPMD. "Individuals with Duchenne need and deserve options, and we are hoping this will become another tool in our arsenal."

As part of its commitment to developing better medicines for DMD, WAVE has extended its ongoing research collaboration with the University of Oxford to advance stereopure nucleic acid therapies for DMD across exons.

Through the collaboration, renowned researcher Matthew Wood, M.D., Ph.D., Professor of Neuroscience, Department of Physiology, Anatomy and Genetics, Medicine Sciences Division, University of Oxford, and his team will continue to work with WAVE to use the company’s proprietary platform to enhance oligonucleotide approaches, including exon-skipping, to address the rare genetic muscle disease.

"The data we have seen to date using WAVE’s novel approach to exon-skipping in DMD is very promising. I believe that academia and industry, working together, may be on the verge of a veritable medical revolution where we can potentially effectively and durably treat genetically based diseases such as DMD," said Professor Wood.

"Collaborations between academia and industry are critical now more than ever in order to collectively harness the latest scientific advancements to rapidly progress therapies for patients. We look forward to expanding our collaboration with the WAVE team and advancing the potential and benefits of stereopure oligonucleotide approaches for DMD."

About Duchenne Muscular Dystrophy (DMD)

Duchenne muscular dystrophy (DMD) is a fatal X-linked genetic neuromuscular disorder caused by mutations in the gene that encodes for the protein dystrophin. Dystrophin is needed for muscle maintenance and operation. Because of the genetic mutations, the body cannot produce functional dystrophin. Without dystrophin, muscles cannot work properly, and patients experience progressive loss of strength and serious medical complications involving the heart and lungs.

DMD affects approximately one in 3,500 newborn boys around the world; approximately 13% have mutations in Exon 51. There are currently no approved disease-modifying drugs available to treat DMD. Exon-skipping drugs are designed to "skip over" the mutated exon, enabling the gene to once again code for and produce functional dystrophin.