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Prosensa, Orphan Drug Drisapersen, Duchenne Muscular Dystrophy, And The J.P. Morgan Healthcare Conference

January 9, 2013

       On Monday, January 7, 2013, at the 31st Annual J.P.Morgan Healthcare Conference in San Francisco, Prosensa gives a presentation on orphan drug Drisapersen (PRO051) – the main competitor to Sarepta Therapeutics’ orphan drug Eteplirsen for Duchenne Muscular Dystrophy (DMD).

Prosensa is partnering with GlaxoSmithKline for the development of Drisapersen, which is currently in late-stage Phase III clinical trials for DMD. The drug has orphan status in both the United States and in Europe. Prosensa also has RNA therapies for different variants of DMD.

       Prosensa did not webcast their presentation. Adam Feuerstein from, tweeted live from the Prosensa Presentation the following (Adam Feuerstein (@adamfeuerstein from – check out his #JPM13 liveblog) :

– Prosensa has deeper pipeline than Sarepta Therapeutics

– Phase 3 trial of Drisapersen for DMD is fully enrolled & 3 trials are completed; GSK holding placebo-controlled data until 06/13 – 07/13

– DEMAND II trial data to be released 3rd Q2013

– DEMAND III trial data to be released 4th Q2013

– PRO045, exon-45 skipping drug, has entered the clinic

– Adverse events are renal effects and injection site reactions, but don’t result in any dropouts.

As noted in a previous Orphan Druganaut Blog Post, Henri Termeer, former Genzyme CEO and Chairman who retired in 2011 after almost 30 years, joins Prosensa as a strategic adviser “providing advice for the biotech’s corporate strategy and growth plans’’. Mr. Termeer built Genzyme into a leader in rare diseases and orphan drugs.

On Wednesday, January 9, 2013, at the 31st Annual J.P. Morgan Healthcare Conference, NPS Pharmaceuticals will have a live webcast of their presentation at 9:00 AM Pacfic Standard Time/12:00 Noon EST (NY).

Correction to Blog Post made 01/16/13:  The sentence “Drisapersen study for 140 weeks had boys excluded due to rapid deterioration, similar to Sarepta Therapeutics’ Eteplirsen trial” has been deleted. No boys were excluded from the Prosensa 140-week study. Two boys were non-ambulatory from the start of the extension study, but all boys (including two that lost ambulation during the extension study) were included in Prosensa’s analysis of the 140 weeks data.


Orphan Druganaut Blog Post titled, “Review of Phase II & III Orphan Drug Clinical trials for Duchenne Muscular Dystrophy: Journal Article”.

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

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