CureDuchenne, a nonprofit focused on finding a cure for Duchenne muscular dystrophy (DMD), will be paying the rare disease-focused biotech firm Prosensa up to €5 million ($6.68 million) in convertible promissory notes with the aim of accelerating the development of and improve patient access to experimental DMD therapies.
In June, Prosensa received positive feedback from the FDA enabling it to pursue an NDA filing for drisapersen—a drug that, according to CureDuchenne, could help up to 13% of the DMD population—under an accelerated approval pathway. However, the FDA’s guidance also requested that the company perform a confirmatory study in a follow-on exon skipping drug with a similar mechanism of action prior to a potential approval for drisapersen.
As such, Prosensa says it plans to use CureDuchenne's funds to do the following:
Initiate a Phase II study in Europe by the end of 2014 and a placebo-controlled trial in the U.S. in the first half of 2015 for PRO044, an exon-skipping candidate for the treatment of DMD. Prosensa believes this clinical extension study could be used as one of two confirmatory studies to support accelerated approval for drisapersen, and CureDuchenne says PRO044 could help up to 6% of DMD sufferers.
Support re-dosing efforts for drisapersen trial participants in North America and Europe and facilitate the drug’s NDA filing in the U.S. in 2014.
Support development of PRO045 and PRO053, Prosensa’s other exon-skipping compounds. CureDuchenne says these candidates could help up to 8% of the DMD population respectively.
The funds come at an opportune time, as in January a research and collaboration agreement it had with GSK to advance drisapersen ended several months after the DMD candidate failed to meet its primary endpoint in a Phase III clinical study. Prosensa also just released its financial results for the second quarter of 2014. The firm's net loss for the quarter was €8.0 million ($10.69 million), compared to a net loss of €4.7 million ($6.28 million) for the second quarter of 2013. Plus, its revenue for the three months ending on June 30, 2014, was nil, in part due to a decrease in license revenue of €1.3 million ($1.74 million) and a decrease in collaboration revenue of €0.7 million (around $935,000) due to the termination of that GSK collaboration.
Prosensa says it's been collaborating with CureDuchenne since 2004, when CureDuchenne provided financial support to help advance Prosensa’s pipeline of DMD products. "CureDuchenne has been a dedicated supporter of Prosensa since the company’s inception, and we are very appreciative of the additional funding for our extensive DMD program," Prosensa CEO Hans Schikan said in a statement.