A little more than six months since its stock price was crushed by a failed late-stage program for its leading — and only — drug, tiny biotech microcap Marinus $MRNS saw its shares shoot up this morning after it offered a positive Phase II snapshot on a handful of patients who suffer from a rare genetic ailment.
Investigators flipped the first card of its Phase II hand for ganaxolone in treating CDKL5, which triggers severe seizures and neurological damage among the children afflicted by it. One of four patients had to drop out from a lack of response, but investigators say that the three others experienced a reduction in seizures ranging from 52% to 88%.
Presenting data at the annual meeting of the American Epilepsy Society, researchers also added seizure responses and EEG measurements for three patients — one with CDKL5 and two with PCDH19 pediatric epilepsy — underscoring a positive response in cutting the rate of seizures from baseline.
Investors liked what they saw, driving up shares about 40% in morning trading. It was a different story last June, when the company’s shares dived on the news that ganaxolone had failed a late-stage study for adult focal onset seizures. Its market cap at the end of day on Friday was a fragile $22 million.
It’s early days yet for this study, which is designed to enroll up to 10 patients with each of CDKL5 disorder, Lennox Gastaut Syndrome and PCDH19 pediatric epilepsy.
The drug development program here is similar to what Sage has been working on, with a focus on status epilepticus as well as postpartum depression.
Michael G. Chez, the director of Pediatric Neurology Research and Pediatric Epilepsy, Sutter Neuroscience Institute, said:
“I am impressed with the responder rate and magnitude of seizure control seen with ganaxolone in the initial CDKL5 patients. The CGIs (clinical global impression scales) are consistent with seizure control, with responders showing ‘much improved’ under this scale. I look forward to further evaluating these children and seeing the final results.”