Marinus Pharmaceuticals Inc (NASDAQ:MRNS) recently announced that the four-person phase 2 study for ganaxolone drug has shown positive preliminary data. On account of this announcement, the company’s stock climbed almost 49.1% to $1.72 per share yesterday.
Will the Company’s pediatric epilepsy drug help control out-of-control seizures?
Ganaxolone drug of the company is the pediatric epilepsy drug, which is meant for the treatment of rare genetic disorder called CDKL5. The classic symptom of this rare genetic disorder CDKL5 is that it can lead to early onset of seizures, which eventually go out of hand and are too difficult to curtail.
Now the company is moving on to the mid-stage clinical trial of Ganaxolone drug. The company is now enrolling patients for this trial. In this mid-stage clinical trial, Marinus Pharmaceuticals Inc is hoping to enrol about 10 patients as of now. The company expects that the additional data on this trial may come out in the first half of this year, that is, by June 2017.
Progress of mid-stage clinical trial of Ganaxolone drug
As of now, it has been learnt that Marinus Pharmaceuticals has enrolled four patients for this mid-stage clinical trial. In the latest development, three among these patients have shown a positive sign. There has been considerable reduction in the frequency of seizures among these patients. The three patients have a reduced frequency of about 52% to 88%.
The company said that the fourth patient, however, shall end the participation in this trial due to the failure of seeing progress. The reason cited by company for ending the participation of fourth patient is “lack of efficacy.” This patient will no longer be a part of the study from May 2017.
Though the sample size is smaller, but according to Simos Simeonidis of the RBC Capital Markets, the range of frequency reduction of seizures and the response rate is of paramount importance. Simos Simeonidis calls the results “encouraging” because of the group of patients being “hard to treat.”