Shares of small-cap biopharma Marinus Pharmaceuticals Inc MRNS were moving sharply higher to a new 52-week high Tuesday following the release of positive results for ganaxolone, the only investigational asset in its pipeline.
What Happened: Randor, Pennsylvania-based Marinus said its registrational Phase 3 trial dubbed Marigold produced positive results.
The trial is evaluating ganaxoline in children and young adults with CDKL5 deficiency disorder, or CDD, a rare genetic epilepsy with refractory seizures.
Patients who were given ganaxolone showed a significant 32.2% median reduction in 28-day major motor seizure frequency, compared to a 4% reduction for those receiving the placebo, achieving the primary endpoint, the company said.
The investigational drug was also generally well-tolerated, with a safety profile consistent with previous clinical studies and the most frequent adverse event being sleepiness.
Following the readout, SVB Leerink analyst Marc Goodman maintained an Outperform rating on Marinus shares and increased the price target from $6 to $8, as he raised the probability of success of the CDD program from 50% to 95%.
Why It's Important: Ganaxolone, a positive allosteric modulator of GABAA receptors, is being developed in intravenous and oral formulations in both acute and chronic care settings.
The company has had mixed results with the pipeline asset. A midstage study in postpartum depression failed in mid-2019.
Ganaxolone is being evaluated for multiple seizure disorders, including CDD, and treatment-resistant depression.
What's Next: Marinus said it plans to submit an NDA for ganaxolone in CDD to the FDA in mid-2021 and a marketing authorization application to the EMA by the end of the third quarter of 2021.
The company also said it plans to present the top-line results at an upcoming scientific meeting.
Issuing a pipeline update, Marinus said it received FDA feedback on the protocol for the planned Phase 3 study of intravenous ganaxolone in refractory status epilepticus.
The company said it is in discussions with the FDA to respond to the latter's feedback prior to enrolling patients into the trial. The company expects to release top-line results from the study in the first half of 2022.
Separately, Marinus announced a five-year cost-sharing contract with BARDA to support the Phase 3 study in RSE, including $21 million in non-dilutive financing and preclinical studies of ganaxolone in nerve agent exposure animal models.
Marinus stands to receive up to $30 million in additional optional funding contingent on favorable clinical and preclinical outcomes.
MRNS Price Action: Marinus shares were trading higher by 88.15% to $3.97 at last check Tuesday.
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