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Marinus Pharmaceuticals Inc (NASDAQ:MRNS) Might Be At The Start Of A Longer Term Revaluation

Marinus Pharmaceuticals Inc (NASDAQ:MRNS) Might Be At The Start Of A Longer Term Revaluation
Written by
Chris Sandburg
Published on
July 26, 2017
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Back in January, we published this piece outlining our thoughts on Marinus Pharmaceuticals Inc (NASDAQ:MRNS). At the time, the company had just put out data from a small and ongoing study of its lead asset in a rare condition and picked up a small bump in market capitalization on the back of the news. As we outlined in the piece, however, we felt that the relatively insignificant degree of appreciation was an underwhelming response to the news based on what it might mean for the company long-term.During the months subsequent to our initial coverage, Marinus has appreciated in value considerably, very much validating our bullish thesis on the stock. Just this month, we got an update associated with the drug and the trial in question and the company is now well into what could be a pivotal third quarter 2017 for shareholders.We like to make sure our readers get on top of any potential catalysts ahead of them impacting the market, so here's what to watch in Marinus near term.For those that missed our initial coverage and don't have the time to check back, we looked at a study of a drug called Ganaxolone, which Marinus is investigating as a potential therapy for a condition called CDKL5, which is a serious and rare genetic disorder that is caused by a mutation of the cyclin-dependent kinase-like 5 (CDKL5) gene. It primarily affects children, mostly girls, and results in severe disability including problems walking, talking and other issues associated with core functional capabilities.It's not nice and there's no really effective treatment on shelves right now.Marinus is trying to change that with Ganaxolone.The drug is what's called a CNS-selective GABAA modulator. GABAA is a receptor that plays a key role in the development and onset of seizures in these patients and, through the modulation of this receptor, Marinus is hoping that Ganaxolone can serve to reduce the seizures and also alleviate certain elements of the above-noted disabilities (many of which are exacerbated by the seizures in question).It's worth noting that the drug failed a phase 3 in epilepsy patients last year, but this was in adult patients and these sorts of drugs are reported to have a higher potential for efficacy in a pediatric population. The trial we are looking at here is targeting said pediatric population and the data we looked at back in January pointed towards a relatively strong degree of efficacy in the kids involved.Out of five patients studied, three patients demonstrated a marked reduction in seizure frequency and tolerability seems on par with current standard of care treatments.Sure, it is a small sample size and five patients isn't going to be enough to get this drug approved in this indication; the company will have to expand into a slightly larger population if it wants to achieve this goal.With that said, however, this is a rare pediatric disorder, meaning that the word 'slightly' is very pertinent here – chances are the FDA will be willing to accept a registration application based on only a relatively small multiple of the number of patients trialed in the ongoing study, purely because there isn't anything on the market for this indication right now and there aren’t that many patients suffering from the disease from which Marinus can pick to include in the study.So, with all that said, why are we looking at this one now?Well, the company just announced that the drug has picked up Orphan Designation in the US meaning, if it does get approved, it will have a strong foundation of advantageous factors on which Marinus can build a commercialization effort.That's news, but it's not a catalyst.What we are looking from a near-term catalyst perspective is the release of data from the above-discussed trial. The company stated that it should be released mid year and we are taking this to mean at the outside end of the third quarter.If the numbers that are released fall in line with those that we got a glance at back in January, Marinus is really going to run.As things stand, the company trades for an around 70% premium to the price at which we highlighted it. This, however, is only the start of a longer-term upside revaluation if we get the data we are looking for.Check out our previous coverage of this one here.We will be updating our subscribers as soon as we know more. For the latest updates on MRNS, sign up below!Image courtesy of Andy Leppard via FlickrDisclosure: We have no position in MRNS and have not been compensated for this article.

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