Date: 2014-08-11
Type of information: Collaboration agreement
Compound: PRO044, PRO045, PRO053, drisapersen
Company: Prosensa (The Netherlands) CureDuchenne (USA - CA)
Therapeutic area: Genetic diseases - Neuromuscular diseases - Rare diseases
Type agreement: collaboration agreement
Action mechanism:
Disease: Duchenne Muscular Dystrophy
Details: * On August 11, 2014, CureDuchenne announced a new $7 million collaboration with Prosensa to help get experimental medicines to patients with Duchenne Muscular Dystrophy more quickly. The new initiative will help restart dosing of medication for patients who participated in previous clinical studies of exon skipping drugs 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). CureDuchenne was the first U.S. nonprofit to fund Prosensa’s exon skipping research. This new announcement expands on CureDuchenne’s initial $1.3 million investment in Prosensa Holdings to fund exon skipping research in 2004.
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