Please login to the form below

Not currently logged in
Email:
Password:

First efficacy data for GSK/Prosensa's muscular dystrophy candidate positive

Ups ante in race against Sarepta Therapeutics to market first Duchenne muscular dystrophy drug

First efficacy data for GSK/Prosensa's muscular dystrophy candidate positive

An antisense drug developed by Prosensa and GlaxoSmithKline (GSK) has improved walking ability for patients with Duchenne muscular dystrophy (DMS) in a phase IIb trial.

The positive data on the candidate, called drisapersen, ups the ante in a race between Prosensa/GSK and rival Sarepta Therapeutics to bring the first drug treatment for DMD to market.

The top-line trial results were revealed at the RNA & Oligonucleotide Therapeutics Conference in Cold Spring Harbor, US, yesterday, and revealed that drisapersen achieved a clinically meaningful improvement in the six-minute walking test in DMD patients compared to placebo after 24 weeks of treatment.

By the end of the study period, patients on continuous drisapersen were able to walk 35 metres further than those on placebo, with the difference maintained up to 48 weeks, although intermittent treatment with the drug conferred no significant benefit. There was also no difference in muscle strength compared to placebo for either drisapersen regimen.

Neither GSK nor privately-held Netherlands firm Prosensa put out a statement on the disclosure of the data at the time of writing, and analysts remarked the timing is a little odd given that they had previously said no results on the drug would be available until a phase III trial was completed later this year.

It has been suggested that the companies have brought forward their presentation in response to the news that Sarepta hopes to file for accelerated approval of its eteplirsen candidate in the US based on the results of its own phase IIb study, which was completed last July with positive 72-week data disclosed last week.

One immediate impact of the GSK/Prosensa announcement was a lift to Sarepta's shares, as eteplirsen has a very similar mechanism of action to drisapersen and the latest data validate its own programme.

Both drugs work via a process known as exon skipping, which partially corrects a defect at exon 51 in the gene coding for a protein called dystrophin that is the underlying cause of symptoms in DMD and other forms of muscular dystrophy. The result is the expression of a shortened form of dystrophin that restores some of the function of the healthy protein.

Analysts have suggested both drugs could achieve sales of up to $500m at peak, with some suggesting eteplirsen may have an edge in the marketplace in terms of safety, although clearly the clinical datasets for both agents remain small at the moment.

11th April 2013

From: Research

Share

Tags

Featured jobs

Subscribe to our email news alerts

PMHub

Add my company
Hanson Zandi

Hanson Zandi’s philosophy is to go that extra mile. Look deeper. Discuss further.Study longer. Work harder.This focussed work ethic combined...

Latest intelligence

Acute leukaemia patients get new online information centre that will help them get the best possible care
A unique collaboration between some of the world's leading leukaemia experts and the patient community in the UK and across Europe has resulted in the development of a one-of-a-kind educational...
How to conduct a pharma content audit
A pharma content audit is essential for identifying whether your site delivers comprehensive, valuable information for the purpose of meeting the needs of your content marketing efforts....
14_ipad_lowres.jpg
Do your customers feel that you’re listening to them?
When answering to data, excellent digital communication can create much stronger relationships between reps and customers....

Infographics