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Marinus Pharmaceuticals Provides Business Update

The following excerpt is from the company's SEC filing.

and reports third quarter 2015 financial results

Marinus Pharmaceuticals, Inc.

(Nasdaq:MRNS), a

biopharmaceutical company dedicated

to the development of innovative therapeutics to treat epilepsy and neuropsychiatric disorders

today

provided a business update on its clinical development activities and reported its financial results for the third quarter ended September 30, 2015.

Recent Highlights:

Successful End-of-Phase 2 meeting with the United States Food and Drug Administration (FDA) for ganaxo lone in focal onset seizures

Initiated clinical phase of ganaxolone intravenous (IV) clinical program in status epilepticus (SE)

Robust efficacy in benzodiazepine-resistant SE animal models seen with Ganaxolone IV

Preclinical data further supports clinical evidence that ganaxolone does not activate the human progesterone receptor

Announced interim observations from exploratory Phase 2 proof-of-concept trial in PCDH19 female pediatric epilepsy

“Marinus has made significant progress advancing our ganaxolone programs,” commented Christopher M. Cashman, Chief Executive Officer of Marinus Pharmaceuticals. “Of particular note is the addition of our ganaxolone intravenous formulation to our development portfolio. The GLP safety and tolerability studies with a clean safety profile coupled with robust efficacy observed in benzodiazepine-resistant SE animal models support the advancement of ganaxolone IV into the clinic. We are excited about the potential for ganaxolone IV in the acute care setting which, together with our convenient oral capsule and liquid formulations, provides a continuity of care for patients suffering from drug-resistant seizures.”

Business Update

Ganaxolone is a CNS-selective GABA

modulator being developed in three different dose forms (IV, capsule, and liquid) intended to maximize therapeutic reach to adult and pediatric patient populations in both acute and chronic care settings. Ganaxolone acts on a well-characterized target in the brain known to have both anti-seizure and anti-anxiety effects.

Ganaxolone IV – Status Epilepticus (Orphan Indication):

Marinus recently announced the

initiation of the clinical phase of its ganaxolone IV program and its initial target orphan indication in SE. SE is

a life-threatening condition, which, if not arrested immediately, can result in marked neuronal damage, cognitive impairment and death.

Data from preclinical studies yielded positive results testing ganaxolone IV in benzodiazepine-resistant SE. Two separate laboratories using different measurements tested ganaxolone IV in clinically translatable rodent models of SE and in both studies ganaxolone IV promoted survival and showed better or comparable reversal of seizures than the endogenous neurosteroid allopregnanolone.

Marinus plans to commence a Phase 1 clinical trial designed to evaluate the safety, tolerability and pharmacokinetics of ganaxolone IV in the first half 2016 followed by initiation of a clinical trial in SE patients later next year.

Ganaxolone IV for the hospital setting offers a new mechanism of action for the treatment of SE that is complemented by its oral dose forms, providing the potential for IV-to-oral continuation therapy as patients transition from acute care to outpatient settings.

Ganaxolone Capsule – Refractory Focal Onset Seizures:

Marinus has focused most of its recent clinical development efforts on advancing its outpatient chronic epilepsy indications where oral administration is required.

The most advanced clinical study with ganaxolone is the ongoing Phase 3 trial assessing the efficacy and safety of ganaxolone for adjunctive treatment of refractory focal onset seizures. Enrollment in this Phase 3 study is nearly complete with final enrollment in the registration component of the study expected to reach between 300-350 patients. Top-line data are expected to be announced mid-2016.

A successful End-of-Phase 2 meeting took place during the third quarter, where the FDA was in agreement with the planned path to support registration of ganaxolone for adjunctive treatment of focal onset seizures, which, among other anticipated preclinical and clinical studies, includes a single additional Phase 3 registration study. Marinus and the FDA were in general agreement on the design, population and primary endpoint for both the ongoing and planned second Phase 3 clinical study, pending submission of the protocol of the second...


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