Shares of the actively traded micro-cap biotech Marinus Pharmaceuticals Inc MRNS opened notably higher Thursday morning following a positive clinical readout from the company.
What Hapened
The shares, which suffered a setback in late July after a failed midstage trial of Ganaxolone in postpartum depression and have not recovered much since, could see a redemption of sorts following the readout.
Marinus said the Phase 2 study evaluating the intravenous formulation of Ganaxolone, a positive allosteric modulator of GABAA, in refractory status epilepticus, or RSE, met the primary endpoint, with no patients progressing to IV anesthetics within 24 hours of treatment initiation.
Status epilepticus is a rare epileptic seizure of prolonged duration, lasting more than 5 minutes for convulsive seizures or 30 minutes for non-convulsive.
The announcement followed the completion of top-line analysis of the open-label, dose-finding, proof-of-concept study that assessed efficacy, safety and pharmacokinetics.
The study enrolled 17 patients who received an IV infusion of ganaxolone as an adjunctive to second-line standard of care AED for up to 96 hours followed by a taper, with three doses — 500 mg/day, 650 mg/day and 713 mg/day — tested.
Marinus said Ganaxolone was found to have an acceptable safety profile for the RSE population in all dose groups.
Why It's Important
Status epilepticus affects about 75,000 patients in the U.S. each year, the company said in a statement. It can cause permanent damage to the brain and even death if not quickly brought under control.
RSE patients are those who fail second line treatment with IV AEDs. There are no currently approved therapies for RSE.
What Next
Marinus said the data have been accepted for an oral platform presentation at the Neurocritical Care Society 17th Annual Meeting in October.
The company said it is making preparations for an end-of-Phase 2 meeting with the FDA in the first quarter of 2020 to discuss next steps toward a potential Phase 3 pivotal study.
Marinus also said it is evaluating a new ready-to-use dose formulation that does not require compounding, mixing or diluting, likely ensuring immediate administration.
Data on the new formulation, which is to be tried in a few additional patients, will be included in the end-of-Phase 2 meeting, the company added.
Price Action
Marinus shares were rallying 20.45% to $1.65 Thursday morning.
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