Marinus Pharmaceuticals Inc (NASDAQ:MRNS) is making some impressive gains this morning, currently up $1.28 and trading at $4.40 at the time of writing – a 41 percent bump. Before the market open today, the biopharmaceutical developer announced that its epilepsy drug, Ganaxolone, is ready to advance into a late-stage clinical trial after successfully passing through an open-label Phase 2 program for a very rare form of the condition called CDKL5 Disorder.
More than 14.3 million shares of Marinus have been traded so far today, up from the stock’s average volume of just 662,000 shares per day.
The company says that its CDKL5 Disorder research for Ganaxolone is now its lead pediatric orphan epilepsy program. The condition is a very rare and severe genetic epilepsy that leads to early-onset, treatment-refractory seizures along with delayed neurological development and “disabling” daily behavioral problems. At the moment, there are no FDA-approved products out there that effectively treat this disorder. Earlier in 2017, Ganaxolone was granted orphan drug status.
In the Phase 2 portion of the Ganaxolone clinical program, the oral formulation of the drug demonstrated a meaningful drop in seizure frequency in most of the patients being evaluated. On top of that, some patients reportedly experienced a boost in the amount of seizure-free days alongside behavioral benefits. Marinus says that it intends on meeting with regulatory bodies to settle on a clinical development path that would be necessary to score approval moving forward.
“We believe that the efficacy and safety profile of ganaxolone in this study provide compelling evidence that a definitive study in patients with CDKL5 disorder should be undertaken as soon as possible,” said the company’s Chief Medical Officer, Dr. Lorianne Masuoka. “The tolerability and complete lack of dose regimen-limiting safety concerns, as compared to the most widely used anti-epileptic drugs for this condition, could make ganaxolone an exceptional option for patients with CDKL5 disorder.”
In the Phase 2 program reported on today, the median change in 4-week seizure frequency was a drop of 43 percent, with a median boost in seizure-free days of 78 percent. Of the seven children who were enrolled in the program, five experienced a significant seizure reduction compared to baseline – with a median reduction of 65 percent.
On top of that, both investigators in the study and the children’s caregivers said that the patients had improvements in mood, behavior, sleep and attention. Ganaxolone was safe and well-tolerated overall, and no major adverse events were reported.
The author of this article holds no position in any of the companies mentioned above.