Marinus Pharmaceuticals, Inc. (MRNS) saw its shares climb on Monday after the company reported positive preliminary results from a mid-stage trial. Specifically the data came from Mirna’s Phase 2 study evaluating ganazolone as a treatment for orphan genetic disorders in CDKL5 patients. The enrollment for this study is still ongoing, with topline data expected in mid-2017.
For some background, CDKL5 is a severe, rare genetic disorder that results in early-onset, difficult-to-control seizures, and neuro-developmental impairment.
Four patients have been enrolled in this cohort of the study for an average treatment duration of five-months. Out of the four patients, three have experienced a notable reduction in seizure frequency compared to baseline ranging from 52% to 88%.
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All responders are continuing to receive treatment, two of which have completed six-months of treatment and have elected to participate in the study extension. Only one patient has discontinued the study after four-months of treatment due to lack of efficacy.
The safety data thus far are consistent with earlier studies where ganaxolone has shown to be generally safe and well-tolerated.
Dr. Jaakko Lappalainen, Vice President of Clinical Development of Marinus Pharma, commented:
We are encouraged by the results in these difficult-to-treat pediatric patients. Concurrent with completing this study, we will be evaluating the potential for breakthrough therapy and applying for orphan drug designation with the United States Food and Drug Administration. CDKL5 pediatric epilepsy may prove to be an attractive and efficient path for ganaxolone and we look forward to evaluating results from the final patients enrolled in this cohort of the study.
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Marinus underperformed the broad markets in 2016 with the stock down over 85%. However, excluding Monday’s move, the stock is up about 15% year to date in 2017.
Shares of Marinus were last trading up about 27% at $1.47, with a consensus analyst price target of $3.33 and a 52-week trading range of $0.82 to $6.76.