CureDuchenne, Prosensa to advance access to treatments for Duchenne Muscular Dystrophy patients

The new initiative will help restart dosing of medication for patients who participated in previous clinical studies of exon skipping drugs – a promising approach to the disease – and could help accelerate the development timelines of four exon skipping drug candidates.

CureDuchenne, and its partners, will provide Prosensa with $7 million in funding, which will help the company to:

• Commence dosing in a European Phase II clinical extension study of Prosensa’s Exon 44 skipping drug, PRO044;
• Initiate a placebo-controlled clinical trial for PRO044 in the U.S. in the first half of 2015 (this drug could help up to six percent of those with Duchenne);
• Re-initiate dosing for drisapersen clinical trial participants in North America and Europe and facilitate the drug’s NDA filing in the U.S. in 2014 (drisapersen could help up to 13 percent of the Duchenne population); and
• Support the development of other exon skipping compounds, PRO045 and/or PRO053 (which could help up to eight percent of the Duchenne population respectively).

Duchenne is a progressive muscle-wasting disease that has no approved treatment, although several potential solutions, including those from Prosensa, are in clinical development.

"We are so pleased to help Duchenne patients gain access to much needed experimental drugs — this is particularly important for the boys who participated in clinical trials," said Debra Miller, founder and CEO of CureDuchenne. "We were the first U.S. nonprofit organization to fund Prosensa and this new initiative will expand our relationship and support them in both getting experimental drugs to patients and accelerating PRO044, drisapersen, PRO045 and PRO053 drug development process. Providing access to potentially beneficial treatments to Duchenne patients is aligned with our mission to cure Duchenne."

"The ability for industry and nonprofit organizations like CureDuchenne to work collaboratively is crucial to developing much needed treatment options for rare diseases such as DMD," said Hans Schikan, CEO of Prosensa. "CureDuchenne has been a dedicated supporter of Prosensa since the company’s inception, and we are very appreciative of the additional funding for this important follow-on exon skipping program."

Duchenne affects approximately 1 in every 3,500 boys. Boys are usually diagnosed by the age of 5, and are in a wheelchair by 12. Most don’t survive their mid-20s. Duchenne patients are missing a key muscle protein called dystrophin. Without dystrophin, muscle cells easily become damaged and die resulting in muscle weakness, followed by heart and breathing failure. Exon skipping drugs trick the muscle cells to produce novel dystrophin by skipping missing, misaligned or faulty exons – sections of genes – on the patient’s own RNA.

CureDuchenne was the first U.S. nonprofit to fund Prosensa’s exon skipping research. Today’s announcement expands on CureDuchenne’s initial $1.3 million investment in Prosensa Holdings to fund exon skipping research in 2004. It is the latest example of how CureDuchenne’s venture philanthropy model helps speed drug development and treatment.

Through this model, CureDuchenne has supported promising research aimed at treating and curing Duchenne. Its investments have been leveraged into more than $100 million from biotech and pharmaceutical companies, venture capital funds, and other foundations toward research and development. As a result, seven research projects have advanced into human clinical trials thanks to CureDuchenne’s early-stage investments – including Prosensa’s drisapersen and PRO044.