Rare Disease Report

CureDuchenne Gives Prosensa $7 Million To Restart Trials

AUGUST 12, 2014
James Radke

The not-for-profit advocacy group CureDuchenne announced they are giving the small biotech company Prosensa Holding N.V. $7 million to help Prosensa get some of their developmental drugs closer to market.  The four main drugs included in the agreement are:
  • Drisapersen (exon 51 skipping drug) to reinitiate phase 3 clinical trials and prepare for NDA filing
  • PRO044 (exon 44 skipping drug) to enter phase 2 clinical trial in EU and and phase 3 clinical trial in U.S.
  • PRO045 (exon 45 skipping drug)
  • PRO053 (exon 53 skipping drug)
In a press release, Debra Miller, founder and CEO of CureDuchenne said, “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 $7million investment expands CureDuchenne’s initial $1.3 million investment in Prosensa Holdings to fund exon skipping research in 2004.  It is also another example of how well the philanthropic model in the rare disease community can be used to accelerate drug development and treatment.

In a press release from Prosensa, Hans Schikan, CEO of Prosensa said, “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,” adding, “CureDuchenne has been a dedicated supporter of Prosensa since the company’s inception, and we are very appreciative of the additional funding for this
As we reported in June, Prosensa received guidance from FDA about their Duchenne program. Until then, their main drug, drisapersen had been put on hold after the company’s latest clinical trial failed to meet its primary endpoint.  However, subsequent analysis of the data from that trial has given the company hope that drisapersen is an effective treatment in the right patient. The FDA (and CureDuchenne) seem to agree.  In June, the FDA suggested the company conduct a historically controlled trial to compliment their drisapersen studies and to conduct a randomized, placebo-controlled trial of another exon-skipping drug with a similar mechanism of action (i.e., PRO044) to show that the trial design and endpoints being used are appropriate (i.e., dystrophin protein production). And in June, Prosensa announced they were pleased with FDA’s guidance and stated they would begin redosing patients in their drisapersen study that had been put on hold while looking into the logistics of starting the other trial with PRO044. 
Two months and $7million later, Prosensa now has a better understanding of how to proceed.
The money from CureDuchenne will be used to help Prosensa with the following:
  • Commence dosing in a European Phase 2 clinical extension study of PRO044
  • Initiate a placebo-controlled clinical trail for PRO044 in the United States in the first half of 2015
  • Re-initiate dosing for drisapersen clinical trial participants in North America and Europe adn facilitate the drugs NDA filing in the United States in 2014
  • Support the development of other exon skipping compones, PRO045 and/or PRO053

About Duchenne Muscular Dystrophy

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.
The dystrophen gene contains 79 exons and if one of them is damaged, it may lead to Duchenne muscular dystrophy.  One type of treatment called exon skipping, uses RNA based technologies to ‘skip’ over the damaged gene and allow the protein dystrophen to still be produced. This technology is being pursued by both Prosensa and Ssarepta to find a treatment for subgroups of patients with Duchenne with known gene mutations.

About CureDuchenne

CureDuchenne is a national nonprofit organization located in Newport Beach, Calif., dedicated to finding a cure for Duchenne, the most common and most lethal form of muscular dystrophy.

About Prosensa Holding N.V.

Prosensa (NASDAQ: RNA) is a Dutch biotechnology company engaged in the discovery and development of RNA-modulating therapeutics for the treatment of genetic disorders. Its primary focus is on Duchenne muscular dystrophy (DMD), myotonic dystrophy and Huntington's disease. Prosensa's current portfolio includes six compounds for the treatment of DMD, all of which have received orphan drug status in the United States and the European Union.

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