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Prosensa Files For Initial Public Offering (IPO)

May 27, 2013

A recent Blog Post discusses three companies that have recently filed for an Initial Public Offering (IPO) in the rare disease and orphan drug space:

•   PTC Therapeutics
•   Kamada
•   Bluebird bio.

Another orphan drug company, Prosensa, files this past Friday, 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 is a Netherlands-based pharmaceutical company founded in 2002 that develops RNA-based therapeutics for genetic diseases. Duchenne Muscular Dystrophy (DMD) has attracted a lot of interest from both investors and the media, due to impressive positive results from GlaxoSmithKline (GSK)/Prosensa’s orphan drug Drisapersen and their competitor, Sarepta Therapeutics’ orphan drug Eteplirsen. Both drugs are 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.

In a May 6, 2013 webinar, co-sponsored by the Muscular Dystrophy Association (MDA), Parent Project Muscular Dystrophy (PPMD), and CureDuchenne, GSK’s John Kraus discusses Phase 2b results that show that weekly injections of Drisapersen to DMD patients, result in a significant difference in walking distance compared to a placebo. Mr. Kraus says that the earliest the company would file an application for approval of Drisapersen in the US or Europe is the first quarter 2014. Mr. Kraus also goes on to say that :

•   “Data on dystrophin production related to drisapersen will be presented at the October 2013 meeting of the World Muscle Society
 •   The investigators saw some indications of adverse effects on kidney function related to drisapersen treatment, but that kidney biomarkers returned to normal during off-treatment periods of the phase 2b study
 •    They did not see other adverse events of concern in the phase 2b study, except for injection site reactions
•    They were encouraged that this study detected an effect of continuous drisapersen treatment compared to placebo treatment on walking distance despite the fact that the trial participants had an average age of 7, a time at which walking ability is fairly stable in DMD.”

Please reference the article in MDA’s online magazine Quest for further detail information about the Phase 2b clinical trial results.

Prosensa announces in January 2013, that it achieved Orphan Drug Designation (ODD) in the EU and the United States for all of its compounds for the treatment of DMD. 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: “Dollar Symbol Gold” by Rugby471 (Own work) [GFDL] | Wikimedia Commons.

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

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