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Prosensa Phase III

Horribly worded headline, but great news...

http://online.wsj.com/article/BT-CO-20110119-706326.html

Glaxo, Prosensa Starts Phase III Of Duchenne Medication Dystrophy

LONDON (Dow Jones)--GlaxoSmithKline PLC (GSK.LN) and Prosensa said Wednesday that the first patient has commenced treatment in the Phase III clinical study investigating GSK2402968 (968), in ambulant boys with Duchenne Muscular Dystrophy or DMD.

MAIN FACTS:

-Commencement of this study confirms previously announced plans to progress this asset into Phase III.

-This randomized, placebo controlled study will enroll 180 patients, from up to 18 countries, and is currently the most advanced ongoing study for this rare, severely debilitating, neuromuscular disease.

-The study is designed to assess the efficacy and safety of GSK968 6 mg/kg, once weekly, compared to placebo, for 48 weeks in ambulant boys over 5 years of age with DMD The primary efficacy endpoint is a measure of muscle function using the six minute walking distance test.

-Prosensa is a privately held biopharmaceutical company.

-GlaxoSmithKline shares at 1315 GMT up 8 pence, or 0.68%, at GBP1189.50 pence, valuing the company at GBP61.81 billion.

-By Razak Musah Baba, Dow Jones Newswires; 44-20-7842-9275; razak.baba@dowjones.com

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Permalink Reply by Keith Van Houten on January 24, 2011 at 6:11pm

I would imagine that if the Phase III trials outside the US start showing promise, GSK will bring their full resources to bear to get a US trial approved and moved through regulatory approval as soon as possible. I'm sure they need the US market to recoup their investment.

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Permalink Reply by Ofelia Marin on January 24, 2011 at 6:19pm

Completely agree.

Keith Van Houten said:

I would imagine that if the Phase III trials outside the US start showing promise, GSK will bring their full resources to bear to get a US trial approved and moved through regulatory approval as soon as possible. I'm sure they need the US market to recoup their investment.

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Permalink Reply by Rahul Grover on January 24, 2011 at 8:33pm

I know this has been asked before, but i don't recall seeing anyone knowing the answer, but do we know what the FDA's plan is for skipping of other exons? Would the FDA require trial phases for all other exon skipping? Has this discussion happened with the FDA? If not, is there one scheduled?

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Permalink Reply by Tulika on January 25, 2011 at 12:57am

Well I would personally still participate in the trial even if the toxicity issues are known. I know that managing the kidney and liver is still possible as compared to managing a body wide loss of muscle tissue.

A risk vs benefit case should alos be considered as compared to taking in isolation that the drug has toxicity issues.

Tapio said:

Does anyone happen to know what the toxicity concern is with this drug? I've heard bits and pieces but am unclear whether the toxicity they refer to has to do with the actual mechanics of exon skipping (the fact this would be toxic in a healthy body) or some side effect they are seeing with the drug? Wasn't it shown in the phase 2 study that the drug was well tolerated, or was some toxicity demonstrated in higher dosages in a separate test?

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Permalink Reply by Ofelia Marin on January 25, 2011 at 8:53am

I would do too...our regulatory agency however doesn't allow the start of trials...

Hoping this will change when some results from Phase 2 UK trial will surface.

Tulika said:

Well I would personally still participate in the trial even if the toxicity issues are known. I know that managing the kidney and liver is still possible as compared to managing a body wide loss of muscle tissue.

A risk vs benefit case should alos be considered as compared to taking in isolation that the drug has toxicity issues.

Tapio said:

Does anyone happen to know what the toxicity concern is with this drug? I've heard bits and pieces but am unclear whether the toxicity they refer to has to do with the actual mechanics of exon skipping (the fact this would be toxic in a healthy body) or some side effect they are seeing with the drug? Wasn't it shown in the phase 2 study that the drug was well tolerated, or was some toxicity demonstrated in higher dosages in a separate test?

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Permalink Reply by cheryl cliff on January 26, 2011 at 12:08pm

In the countries where clinical trials are moving forward, does anyone know if there is an agreement with the pharma (GSK in this case) to allow trial participants to continue on the exon skipping meds AFTER completion of the trial?