Marinus Pharmaceuticals Provides Business Update and Second Quarter 2018 Financial Results
02 August 2018
Near-term Clinical Value Catalysts
- Complete enrollment of patients in ganaxolone intravenous (IV) portion of the Phase 2 Magnolia study in women with severe postpartum depression (PPD) in third quarter 2018
- Report top-line data from ganaxolone IV portion of the Magnolia study in fourth quarter 2018
- Report top-line oral ganaxolone data from Amaryllis study in women with PPD in fourth quarter 2018
“We achieved an important milestone in the second quarter with the initiation of a pivotal Phase 3 clinical trial evaluating the use of oral ganaxolone in children and young adults with CDKL5 Deficiency Disorder (CDD),” commented
Mr. Cashman continued, “We are continuing to enroll patients with postpartum depression in both our Magnolia and Amaryllis studies and look forward to reporting top-line data from both studies in the coming months.”
CDKL5 Deficiency Disorder (CDD)
- The Company is enrolling patients in its pivotal Phase 3 clinical trial (Marigold Study) evaluating the use of oral ganaxolone in children and young adults with CDD. The Marigold Study is a global, double-blind, placebo-controlled, trial that will enroll approximately 70 patients between the ages of 2 and 21 with a confirmed disease-related CDKL5 gene variant. Patients will undergo a baseline period before being randomized to receive either ganaxolone (up to 1,800 mg/day) or placebo for 17 weeks, in addition to their existing anti-seizure treatment. Following the treatment period, all patients that meet certain eligibility requirements will have the opportunity to receive ganaxolone in the open label phase of the study. The study’s primary efficacy endpoint is percent reduction in seizures. Secondary outcome measures will include non-seizure-related endpoints to capture certain changes in behavioral and sleep disturbances that were reported in previous clinical studies with ganaxolone. There are currently no approved treatments for CDD.
Postpartum Depression (PPD)
- The Company is in the process of completing enrollment in the IV only portion of the Magnolia study, a Phase 2 double-blind, placebo-controlled, dose-optimization clinical trial to evaluate ganaxolone in women diagnosed with severe PPD (Hamilton Depression Rating Scale (HAMD17) score ≥26). The efficacy endpoint is change from baseline in the HAMD17 score. The Company expects to provide top-line data in the fourth quarter and discuss next steps in our PPD program, which include evaluating an IV followed by oral formulations of ganaxolone.
- Enrollment is on-going in the Company’s Amaryllis study, a Phase 2 clinical trial to evaluate the safety, tolerability and efficacy of oral ganaxolone in women with moderate PPD (HAMD17 score of ≥ 20, but < 26). Patients enrolled in the initial open label phase of the study receive one of multiple treatment regimens with ganaxolone capsules which include once-daily dosing. The efficacy endpoint is change from baseline in the HAMD17 score. Data from the open label phase are expected in the fourth quarter of 2018 and will inform later stage development of novel treatment paradigms that may include an IV followed by oral ganaxolone pivotal program. Upon successful completion of the open label phase, the study will continue as a double-blind placebo-controlled trial.
Status Epilepticus (SE)
- The Company is enrolling patients with refractory status epilepticus (RSE) in its Phase 2 study with ganaxolone IV. Initial data from this proof-of-concept study are expected in the fourth quarter of 2018.
Research and development expenses increased to
General and administrative expenses were
Readers are referred to, and encouraged to read in its entirety, the Company’s Quarterly Report on Form 10-Q for the quarter ended
Marinus Pharmaceuticals, Inc.
Selected Financial Data (in thousands, except share and per share amounts)
|Cash and cash equivalents||$21,467||$33,531|
|LIABILITIES AND STOCKHOLDERS’ EQUITY|
|Other long term liabilities||107||120|
|Total stockholders’ equity||45,020||58,008|
|Total liabilities and stockholders’ equity||$48,687||$60,672|
|Three Months Ended June 30,||Six Months Ended June 30,|
|Research and development||$||7,232||$||2,817||$||11,159||$||6,390|
|General and administrative||2,338||1,691||4,526||3,503|
|Loss from operations||(9,570||)||(4,508||)||(15,685||)||(9,893||)|
|Other income (expense)||1||(3||)||—||(12||)|
|Per share information:|
|Net loss per share of common stock—basic and diluted||$||(0.24||)||$||(0.21||)||$||(0.38||)||$||(0.47||)|
|Basic and diluted weighted average shares outstanding||40,395,650||21,985,213||40,384,429||21,288,545|
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