logo-loader

Summit provides renewed hope for sufferers of muscle wasting disease

Published: 11:45 26 Apr 2016 EDT

DMD
The disease affects boys and young men.
For sufferers of Duchenne Muscular Dystrophy, April 26, 2016 will go down as a black day as a drug created to treat a fatal muscle wasting disease was effectively nixed by an advisory committee to the all powerful US Food & Drug Administration.
 
Its developer, Sarepta Therapeutics Inc (NASDAQ:SRPT), hitherto a star of the biotech sector, saw the bottom fall out its world too as its shares slid by a third. 
 
They are down by three-quarters in the year to date – and at US$9.77 each are well off their high of US$53.48, achieved in late 2013.
 
At that point, the company was worth US$2.4bn reflecting the excitement surrounding eteplirsen, the DMD drug given short shrift by the committee advising the FDA.
 
While not binding, the US regulator normally takes advisory committee advice into consideration when reviewing New Drug (NDA) and Biologic License Applications (BLA). So the outlook is bleak for Sarepta. 
 
However there may be a ray of hope for sufferers in the form of a UK-based, Nasdaq-listed drug developer that is only just starting to register on investors’ radar screens.
 
Shares in Summit Therapeutics PLC (NASDAQ:SMMT, LON:SUMM) rose 4% after the FDA today allowed the company to broaden the scope of its phase II clinical trial of its drug ezutromid to trial sites in the US.
 
The green light provides a welcome boost to researchers working on the utrophin modulator, although it should be noted that ezutromid, formerly known as SMT C1100, is still in the formative stages of development.
 
“The level of interest in this hearing on eteplirsen is testament to the urgent need for therapeutic solutions for Duchenne muscular dystrophy,” said Glyn Edwards, Summit’s chief executive. 
 
“Summit Therapeutics has, in its utrophin modulator programme, an approach with a unique mechanism of action that has the potential to benefit the entire DMD population. 
 
“We look forward to bringing utrophin modulation to the many families who are not addressed by the approaches currently in development.”
 
In contrast to many current therapeutic approaches to DMD, the utrophin modulation used by Summit has the potential to treat all boys and young men with DMD, regardless of their underlying dystrophin gene mutation, Summit's statement said.
 
DMD is a progressive muscle wasting disease that affects around 50,000 boys and young men in the developed world, generally leading to death by the late twenties. 
The disease is caused by different genetic faults in the gene that encodes dystrophin, a protein that is essential for the healthy function of all muscles.
 

ARway.ai announces multiple new SaaS developer contracts in both the United...

ARway.ai (CSE:ARWY, OTCQB:ARWYF) Chief Executive Officer Evan Gappelberg joined Steve Darling from Proactive to announce multiple new SaaS developer sign-ups for its augmented reality experience platform, focusing on AR indoor navigation. These partnerships represent significant milestones in...

2 hours, 2 minutes ago