CureDuchenne Gives Prosensa $7 Million To Restart Trials


Philanthropic efforts could accelerate drug development and treatment.

Philanthropic efforts could accelerate drug development and treatment.

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 include involved 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 $7 million 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."

Click here to read the full article on Rare Disease Report.

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