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DMD Race For First FDA Approved Drug: Prosensa Business Developments

June 10, 2013

This is the second in this week’s three part series on recent business developments for the three competitors and their orphan drugs in the Duchenne Muscular Dystrophy (DMD) space :

GSK is developing Drisapersen in collaboration with Prosensa. Prosensa is a Netherlands-based pharmaceutical company founded in 2002 that develops RNA-based therapeutics for genetic diseases. Drisapersen is a drug designed to skip over the defective exon 51 in DMD patients and to produce functional dystrophin. GSK has exclusive, worldwide rights to develop and commercialize Drisapersen from Prosensa under a 2009 deal.

Exon-skipping is a genetic intervention that alters dystrophin expression in the skeletal muscle. The DMD gene is the largest known human gene that consists of a coding sequence of 79 exons. Exon-skipping excludes particular exons from the dystrophin to bypass mutations. This type of treatment can be applied to approximately 72% of DMD patients (Per Review of Phase II and Phase III clinical trials for Duchenne Muscular Dystrophy. Scully M, Pandya S, Moxley R. Expert Opinion on Orphan Drugs. Jan 2013; 1(1): 33-46).

Drisapersen currently has several Phase II and III ongoing clinical trials :

  1. Phase II (US) – Clinical study to assess 2 doses of GSK2402968 in subjects with DMD
  2. Phase III (19 countries) – Clinical study to assess efficacy & safety of GSK2402968 in subjects with DMD
  3. Phase III (22 countries) – Open label study of GSK2402968 in subjects with DMD
  4. Treatment IND protocol (US) – Study of safety, tolerability, & efficacy of long-term administration of Drispersen in US subjects with DMD.

Prosensa, files on May 24, 2013, a F-1 filing with the US Securities and Exchange Commission (SEC) to raise up to $60 million in an IPO.  The company, who initially files confidentially on April 15, 2013, plans to list on the NASDAQ under the symbol ‘RNA’. J.P. Morgan and Citi are the joint bookrunners for the deal.

Prosensa currently has 6 exon-skipping compounds in development for treating DMD. The European Medicines Agency (EMA) assigns orphan drug status to Prosensa’s two preclinical compounds PRO052 and PRO055. The FDA grants ODD to Prosensa’s 4 preclinical compounds PrO045, PRO052, PRO053, and PRO055 in January 2013. Prosensa’s 2 other Exon-specific compounds, PRO044 and PRO051 (Drisapersen), receive ODD prior to 2013.

Please Note: “View from starting block of a competition swimming pool” by Rufino Uribe [CC-BY-SA-2.0] | Wikimedia Commons.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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