Marinus Pharmaceuticals Provides Business Update and 2017 Financial Results
Near-term Clinical Value Catalysts
- Initiate Phase 3 pivotal study with oral ganaxolone in children with CDD (CDKL5 Deficiency Disorder) mid-2018
- Report top-line intravenous (IV) ganaxolone data from Phase 2 Magnolia study in women with severe postpartum depression (PPD) third quarter 2018.
- Report top-line oral ganaxolone data from Amaryllis study in women with moderate PPD fourth quarter 2018.
“2017 was a significant year for Marinus, and one that has positioned us for important data readouts and advancing ganaxolone into late stage development in 2018,” said
CDKL5 Deficiency Disorder (CDD)
- In November, the Company presented the successful results from its Phase 2 study evaluating the safety and efficacy of ganaxolone in children with CDD at the annual
CDKL5 Forumin Boston. Marinus was recognized as the leader in clinical research in CDD. The data presented showed that ganaxolone provided substantial and durable anti-seizure efficacy in children with CDD and was generally safe and well-tolerated with no serious adverse events.
- The last patient enrolled into the Phase 2 study, recently completed the six-month treatment period, and based on good seizure control, entered the one-year extension to the study. The majority of CDD patients from the Phase 2 study entered the one-year study extension and continue to receive ganaxolone.
- The Company is engaging in successful, collaborative discussions with regulatory agencies and expects to initiate a global, pivotal study in mid-2018.
Postpartum Depression (PPD)
- Marinus is enrolling patients into the Magnolia Study, a Phase 2 double-blind, placebo-controlled, dose-optimization clinical trial to evaluate the safety, efficacy and pharmacokinetics of ganaxolone in women diagnosed with severe PPD (Hamilton Depression Rating Scale (HAMD17) score ≥26). Patients randomized into the first part of the study will undergo an infusion of either ganaxolone or placebo and will be followed for 30 days. The goal of the first part of the study is to evaluate multiple regimens of intravenous (IV) ganaxolone, which will inform dosing for the second part of the study. Patients enrolled into the second part of the study could receive IV ganaxolone of various infusion lengths followed by administration of oral ganaxolone. The goal of the second part of the study is to identify an optimized dose or doses for further testing in phase 3. Based upon the effect size shown in a recent study for a compound with similar mechanism of action, the Company has increased targeted enrollment in this study. This increase in study scope and the corresponding forecast for patient recruitment have extended our expected timing for completion of the first part to the third quarter of 2018.
- The Company is enrolling patients into its
Amaryllis Study, a Phase 2 double-blind, placebo-controlled clinical trial to evaluate the safety, tolerability and efficacy of oral ganaxolone in women with moderate PPD (HAMD17 score > 20 and < 26). The study is designed to enroll approximately 50 women at 20 sites within the US. Data from this study are expected fourth quarter of 2018.
Status Epilepticus (SE)
- The Company has initiated its Phase 2 study with ganaxolone IV in patients with refractory status epilepticus (RSE). Data from this proof-of-concept study are expected fourth quarter of 2018.
Research and development expenses decreased to
General and administrative expenses increased
The Company reported net losses of
Readers are referred to, and encouraged to read in its entirety the Company’s Annual Report on Form 10-K for the year ended
|Marinus Pharmaceuticals, Inc.
Selected Financial Data (in thousands, except share and per share amounts)
|Cash and cash equivalents||$33,531||$26,178|
|LIABILITIES AND STOCKHOLDERS’ EQUITY|
|Total current liabilities||$2,544||$8,084|
|Notes payable, long-term portion||—||1,743|
|Other long term liabilities||120||141|
|Total stockholders’ equity||58,008||21,479|
|Total liabilities and stockholders’ equity||$60,672||$31,447|
|Year Ended December 31,|
|Research and development||$||12,376||$||22,005|
|General and administrative||6,667||6,237|
|Loss from operations||(19,043||)||(28,242||)|
|Per share information:|
|Net loss per share of common stock—basic and diluted||$||(0.80||)||$||(1.47||)|
|Basic and diluted weighted average shares outstanding||23,540,738||19,498,143|
To the extent that statements contained in this press release are not descriptions of historical facts regarding Marinus, they are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Words such as “may”, “will”, “expect”, “anticipate”, “estimate”, “intend”, “believe”, and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. Examples of forward-looking statements contained in this press release include, among others, statements regarding our interpretation of preclinical studies, development plans for our product candidate, including the development of dose forms, the clinical trial testing schedule and milestones, the ability to complete enrollment in our clinical trials, interpretation of scientific basis for ganaxolone use, timing for availability and release of data, the safety, potential efficacy and therapeutic potential of our product candidate and our expectation regarding the sufficiency of our working capital. Forward-looking statements in this release involve substantial risks and uncertainties that could cause our clinical development programs, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the conduct of future clinical trials, the timing of the clinical trials, enrollment in clinical trials, availability of data from ongoing clinical trials, expectations for regulatory approvals, the attainment of clinical trial results that will be supportive of regulatory approvals, and other matters, including the development of formulations of ganaxolone, and the availability or potential availability of alternative products or treatments for conditions targeted by the Company that could affect the availability or commercial potential of our drug candidates. Marinus undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of the Company in general, see filings Marinus has made with the
Executive Director, Investor & Strategic Relations
Source: Marinus Pharmaceuticals, Inc.