During 2017 so far, we have drawn our readers’ attention to Marinus Pharmaceuticals Inc (NASDAQ:MRNS) on two separate occasions.
The first was back in January, here, immediately subsequent to the company putting out some very early stage data rooted in one of its rare epilepsy programs. Markets basically ignored the news and we suggested that the data was strong, despite general market interpretation, and that this oversight was an opportunity to pick up some shares cheaply ahead of a rebalancing.
Fast-forward to June and we once again highlighted the company, and again, we did our decision to spotlight it in the same thesis. At that point, Marinus was trading at a 70% premium to its price in January, but the FDA had just given the drug in question Orphan Designation and we felt that, if the numbers from an upcoming data release were strong, that this would be just beginning for Marinus.
It’s now mid-September and the data we have been pointing to all year as being pivotal for Marinus just hit press. And just as we expected it might be, based on the early glimpse we got in January, the data is very strong. Marinus is up another 35% on the news and will open the session on Tuesday (the day after the release) at a 220% premium for the price at which we first highlighted this one as want to watch.
So what did we learn and what’s next?
By way of a brief introduction to this one, the drug in focus is called ganaxolone and it’s targeting a condition called CDKL5 disorder. CDKL5 disorder is rare genetic disorder caused by a mutation in what’s called the cyclin-dependent kinase-like 5 (CDKL5) gene and it primarily affects children, mostly girls, and results in severe disability. There aren’t any treatment options that are broadly effective in the syndication right now and it’s a strong (if not large) unmet need in pediatric healthcare.
The key metric against which efficacy would be measured in the trial was seizure frequency and severity, as seizures are a core and debilitating component of this disorder.
And to put it bluntly, the drug hit it out of the park.
As per the latest release, the median change in 28-day seizure frequency from baseline in the intent-to-treat (ITT) population (this was the primary endpoint of the trial) came in as a decrease of 43%. Similarly, the median change from baseline in seizure-free days in the ITT population (which was a key secondary endpoint in the study) came in as an increase of 78%.
In other words, not only are patients who took the drug having fewer seizures, they are also getting three more days without having any seizures at all. It’s tough to get across how much of a difference this sort of seizure free day can have for both patient and carer (generally parent in this instance) in this indication. That a drug like this can increase seizure free days by close to 80% is potentially a game changer.
So what’s next?
The company is about to take this drug into a phase 3 study that will, on initiation, be the first ever pivotal trial in this indication and in this pediatric population. Management is set to meet with the FDA near term in order to set a protocol for the study and – at that point – we will know what the company has to do in order to pick up approval. If the endpoints mimic those that were used for the phase 2 study, it will be a bonus the company and it will serve up a strong chance of success at trial completion.
Cash as of June 30, 2017, came in at just shy of $20 million, which should be enough to get the company through a pivotal in this indication.
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Image courtesy of Erik Drost via Flickr
Disclosure: We have no position in MRNS and have not been compensated for this article.