So what is the status of the trial, and what are we looking out for come data release?
Well, it’s a phase II that is extending into the US, out of the UK, and it’s set up as a proof of concept study. Preclinical and a phase I that looked at tolerability suggested some clinical evidence, but neither were wide enough to say for sure whether the drug works. This proof of concept trial is essentially trying to show that the drug is absorbed and taken up correctly, as well as attempting to establish a clinical benefit from the perspective of improved muscle function. The primary endpoint relates to a change from a pre-established baseline in magnetic resonance imaging parameters, looking at fat infiltration and inflammation of the leg muscles. Secondary (exploratory) endpoints include the six-minute walk distance, which is now a famous measuring stick based on the high profile of the Eteplirsen study, and what’s called the North Star Ambulatory Assessment as well as patient reported outcomes.
The trial is a 48-week trial, and the January 2017 data release will only address the first 24 weeks of treatment, so we are probably looking at topline some point during the second or third quarter of 2017.
So what’s the takeaway here? Well, Sarepta Therapeutics Inc (NASDAQ:SRPT) is grabbing all the headlines with Eteplirsen, but Summit Therapeutics PLC (ADR) (NASDAQ:SMMT) is hot on the company’s trail with its own DMD candidate, and has the potential to target a much wider patient population if it gets approval. There’s still plenty of time to go before any sort of NDA filing, but this doesn’t mean data can’t provide interim catalysts. The just kicked off phase II is one such opportunity for a number of catalysts, making the trial (and the company) one to watch throughout the latter half of 2016.
Note: This article is written by Mark Collins and originally published at Market Exclusive.