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Duchenne’s Muscular Dystrophy (DMD) is a hot topic right now. The furor surrounding Sarepta Therapeutics Inc (NASDAQ:SRPT)’s Eteplirsen, which just picked up a delay response from the FDA, and the announcement regarding BioMarin Pharmaceutical Inc. (NASDAQ:BMRN)’s now discontinued candidate are grabbing headlines, but these two companies are far from exclusive in the space.
Summit Therapeutics PLC (ADR) (NASDAQ:SMMT) just announced the first patient enrolled in a phase II for its lead candidate Ezutromid, and expects to offer up some interim (24 week) data as soon as January next year.
As the trials get going, and move towards reporting come January, we will likely see some interim releases alluding to efficacy and tolerability. Each of these updates has the potential to inject some upside momentum into the company’s market capitalization, making it an intriguing one to watch as we move forward. So, with this in mind, here’s some detail on the drug in question, and a look at whether Summit can compete with Sarepta’s offering if both get a green light.
First then, the drug. DMD is a condition that derives from a mutation in the gene that codes for dystrophin. Dystrophin is a protein that plays a vital role in the structural integrity of muscles, and when it is not produced (or is dysfunctional, depending on the mutation in question) it can cause the muscle wastage that is the hallmark of DMD.
As it turns out, we’ve got another protein called utrophin, which is structurally similar to dystrophin, and there is mounting evidence that it can mimic the latter functionally. Ezutromid is what’s called a utrophin modulator – it upregulates the production and release of utrophin. This utrophin (theoretically) then gets to work structurally reinforcing muscles and reversing the effect of long term dysfunctional dystrophin.
So what’s the difference between this and Eteplirsen, and what does this difference mean for Summit Therapeutics PLC (ADR) (NASDAQ:SMMT) going forward? DMD is caused by a host of different mutations, all of which have the same end result – dystrophin that doesn’t work. Sarepta Therapeutics Inc (NASDAQ:SRPT)’s Eteplirsen targets a number of these mutations, accounting for about 12-13% of the DMD patient population, and uses a gene editing technique to attempt to produce a sort of semi functional dystrophin alternative. In contrast, Ezutromid doesn’t have any effect on the dysfunctional dystrophin genes, so it can be used to treat the entire DMD population, if it gets an agency nod.
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