In The Spotlight: Marinus Pharma

(RTTNews) - 2017 has been an exceptional year for Marinus Pharmaceuticals Inc. (MRNS) as the stock is up an impressive 823% year-to-date, way ahead of the iShares NASDAQ Biotechnology Index (IBB), which has returned just 18% during the same time frame.

Marinus is focused on developing therapeutics to treat epilepsy and neuropsychiatric disorders.

The Company's lead drug candidate is Ganaxolone, being developed in three different formulations namely IV, capsule and liquid. Ganaxolone is a synthetic analog of endogenous neurosteroid Allopregnanolone that targets GABAA receptor known for anti-seizure and anti-anxiety activity.

The Company's lead clinical program with Ganaxolone is in the indication of CDKL5 disorder, where the compound in oral formulation recently completed phase II testing successfully.

CDKL5 disorder is a serious and rare genetic disorder that is caused by a mutation of the cyclin-dependent kinase-like 5 (CDKL5) gene, located on the X chromosome. This disorder mostly affects girls, and is characterized by early-onset, difficult-to-control seizures and severe neuro-developmental impairment. Most children affected by CDKL5 cannot walk, talk, or care for themselves.

In the phase II study, the results of which were reported in September of this year, Oral Ganaxolone demonstrated substantial and durable anti-seizure efficacy in children with CDKL5 disorder.

Ganaxolone is designated an Orphan Drug by the FDA for the treatment of CDKL5 Disorder.

Based on the encouraging phase II data, Marinus intends to meet up with regulatory agencies to discuss the path forward with the goal of commencing a clinical study in 2018.

Ganaxolone in intravenous (IV) formulation is under a phase II trial in women diagnosed with severe Postpartum Depression, dubbed Magnolia Study.

Postpartum Depression, or PPD, is a type of depresssion that many women experience after having a baby. According to the Centers for Disease Control, 11 to 20% of women who give birth each year have postpartum depression symptoms.

Launched in June 2017, the Magnolia Study is recruiting women with severe PPD. Data from this study is expected in early 2018.

Another phase II study in Postpartum Depression with Ganaxolone has been initiated. This study, known as Amaryllis trial, is designed to evaluate Oral Ganaxolone in women with moderate PPD. Data from this study is expected in 2018.

The Company is also initiating a phase II feasibility study with Ganaxolone IV in patients with refractory status epilepticus (RSE).

Refractory status epilepticus (RSE) is a life-threatening condition in which seizures do not respond to first- and second-line anticonvulsant drug therapy (Source: NCBI).

Data from the phase II feasibility study is expected in 2018.

Financials & Key Numbers: (As of September 30, 2017)

Cash on hand: $62.9 million Accumulated deficit: $139.9 million *Shares Outstanding: 40.43 million

(*Data sourced from Yahoo Finance)

Shares of Marinus are currently well off their all-time high of $20.72, recorded in August 2015, say down by nearly 55%. Be that as it may, the stock has gone gangbusters this year!

MRNS has traded in a range of $0.82 to $9.87 over the last 52 weeks. The stock closed Thursday's trading at $9.33, up 15.47%.