RADNOR, Pa.--(BUSINESS WIRE)--Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today provided an update on its clinical development activities and reported its financial results for the second quarter ended June 30, 2020.
“We are very pleased to report that the company’s clinical programs continue to progress in line with the current timelines most recently discussed at our June 30 R&D Day, with limited disruptions attributable to COVID-19,” said Scott Braunstein, M.D., Chief Executive Officer of Marinus. “We continue to expect topline data from our pivotal Phase 3 clinical trial in CDKL5 deficiency disorder in the third quarter, and in anticipation of this, we are planning for both our first NDA submission and the potential commercial launch of ganaxolone. Furthermore, we recently reported that the FDA granted Rare Pediatric Disease Designation for ganaxolone in CDD, an important milestone for both Marinus and patients suffering from this devastating condition.”
Dr. Braunstein added, “In addition to progressing our clinical trials, we are also strengthening the organization and preparing Marinus for the next phase of growth. The company continues to execute on the multiple initiatives and clinical trials we believe will drive long term value for shareholders, including efforts in developing next generation formulations. To bolster this strategy, we were pleased to appoint Chuck Austin to our Board of Directors, as well as adding new leadership in our research, clinical development, legal and commercialization teams to ensure we have strong and experienced talent around the table to guide the execution of Marinus’ mission.”
Pipeline Update:
Status Epilepticus (SE)
- Marinus is on track to start enrolling the Phase 3 study in Q3, with 50 of the targeted 100 U.S. sites already pre-selected.
- The Phase 3 study is a randomized, double-blind, placebo-controlled trial in SE patients who have failed benzodiazepines and 2 or more second line intravenous anti-epileptic drugs (AEDs). The trial is designed to enroll approximately 125 patients randomized to receive ganaxolone or placebo adjunctive to standard of care, and to provide greater than 90% power to detect a 30% efficacy difference between ganaxolone and placebo.
- Patients will receive a 36-hour infusion followed by a 12-hour taper for a total 48-hour treatment period, which, for patients randomized to receive ganaxolone, targets a plasma concentration of greater or equal to 500ng/mL for 12-hours (same target concentration, 50% longer duration as the target dose evaluated in Phase 2).
- The co-primary endpoints for the trial are (i) proportion of patients with SE cessation within 30 minutes of treatment initiation without medications for the acute treatment of SE and (ii) proportion of patients with no progression to IV anesthesia for 36 hours following treatment initiation.
- Topline data is expected in the first half of 2022.
CDKL5 Deficiency Disorder (CDD)
- Marinus remains on-track to report topline data from the pivotal Phase 3 Marigold Study in Q3, evaluating the use of oral ganaxolone in children and young adults with CDD. The global, double?blind, placebo?controlled, clinical trial enrolled 101 patients between the ages of 2 and 21 with a confirmed disease?related CDKL5 gene variant.
- On July 30, the company announced the FDA has granted Rare Pediatric Disease Designation for ganaxolone for the treatment of CDD.
- In advance of topline data, preparations have begun for an expanded access program (EAP) in CDD that would allow the company, on positive data, to offer ganaxolone to patients who were unable to participate in the Phase 3 trial.
- The company continues to enhance its plans for a potential NDA submission and the development of its commercial strategy.
Tuberous Sclerosis Complex (TSC)
- The first patient has been enrolled in the Phase 2 open?label trial to evaluate the safety and tolerability of adjunctive ganaxolone treatment in patients with TSC. The trial is expected to enroll approximately 30 patients ages 2 to 65.
- The protocol is designed for patients to undergo a four-week baseline period, followed by a 12-week treatment period where they will receive up to 600 mg of ganaxolone (oral liquid suspension) three times a day. Patients who meet eligibility criteria may continue ganaxolone treatment during a 24-week extension planned for the trial.
- The primary endpoint for the trial is percent change in 28-day primary seizure frequency for the treatment period relative to baseline. The company plans to analyze allopregnanolone sulfate levels as part of the trial efficacy analysis.
- The company expects to report topline data in the first half of 2021, subject to any delays that may be caused by COVID-19.
PCDH19 Related Epilepsy (PCDH19-RE)
- Marinus remains on-track to enroll approximately 25 patients in the ongoing Phase 2 Violet Study evaluating allopregnanolone sulfate as a biomarker and ganaxolone as a treatment in PCDH19-RE patients.
- The company believes the increased patient enrollment has the potential to strengthen the ability to detect a meaningful signal as proof-of-concept for the allopregnanolone sulfate biomarker hypothesis.
- On-track to report topline data from this proof-of-concept trial in the first half of 2021.
- Marinus will continue to evaluate if the allopregnanolone sulfate biomarker hypothesis could have broader utility in other targeted indications.
Corporate Update:
- Marinus raised $46 million in gross proceeds from an underwritten public offering of its common stock.
- Appointed Martha Manning, Esq., who has over 20 years of life sciences experience, as Vice President, General Counsel and Secretary.
- Appointed Charles Austin, a recognized leader in the pharmaceutical and medical device industries, to its Board of Directors.
- Hosted aclinical update and commercial overview on its pipeline programs in Status Epilepticus (SE), CDKL5 Deficiency Disorder (CDD) and Tuberous Sclerosis Complex (TSC), with four key opinion leaders participating in the event.
CDD Phase 3 Topline Clinical Data Conference Call:
- As mentioned previously, Marinus plans to host a conference call later in the third quarter coincident with the release of topline data from its Phase 3 CDD clinical trial.
Financial Update:
At June 30, 2020, the company had cash, cash equivalents and investments of $105.9 million, compared to $91.7 million at December 31, 2019. We believe that our cash and cash equivalents as of June 30, 2020 will enable us to fund our current scale of operating expenses and capital expenditures into 2022.
Research and development expenses increased to $11.8 million and $26.8 million for the three and six months ended June 30, 2020, respectively, as compared to $10.0 million and $18.9 million for the same periods in the prior year. The increases were due primarily to our ongoing enrollment in our CDD trial, which completed enrollment during the first quarter of 2020, and enhanced drug development activity, including preclinical studies and manufacturing activities in preparation for a potential NDA filing for CDD and a Phase 3 clinical trial in SE expected to commence in the third quarter of 2020.
General and administrative expenses were $4.1 million and $8.0 million for the three and six months ended June 30, 2020 as compared to $2.5 million and $6.2 million for the same periods in the prior year. The increases were due primarily to increased legal and consulting fees as we scale up our operations and prepare for potential commercialization, and noncash stock-based compensation.
The company reported net losses of $15.7 million and $34.3 million for the three and six months ended June 30, 2020, respectively, compared to $12.4 million and $24.9 million in the same period a year ago. Cash used in operating activities increased to $30.0 million for the six months ended June 30, 2020 compared to $20.4 million for the same period a year ago.
Readers are referred to, and encouraged to read in its entirety, the company’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2020, to be filed with the Securities and Exchange Commission, which includes further detail on the above-referenced transactions and the company’s business plans, operations, financial condition and results of operations.
| Marinus Pharmaceuticals, Inc. Selected Financial Data (in thousands, except share and per share amounts) (unaudited) | ||||||
| June 30, 2020 | December 31, 2019 | |||||
| ASSETS | ||||||
| Cash and cash equivalents | $ | 98,930 | $ | 90,943 | ||
| Investments | 6,966 | 739 | ||||
| Other assets | 7,547 | 7,160 | ||||
| Total assets | $ | 113,443 | $ | 98,842 | ||
| LIABILITIES AND STOCKHOLDERS’ EQUITY | ||||||
| Current liabilities | $ | 9,503 | $ | 8,031 | ||
| Other long term liabilities | 2,795 | 3,042 | ||||
| Total liabilities | 12,298 | 11,073 | ||||
| Total stockholders’ equity | 101,145 | 87,769 | ||||
| Total liabilities and stockholders’ equity | $ | 113,443 | $ | 98,842 | ||
| Three Months Ended June 30, | Six Months Ended June 30, | ||||||||||||
| 2020 | 2019 | 2020 | 2019 | ||||||||||
| Expenses: | |||||||||||||
| Research and development | $ | 11,752 | $ | 10,010 | $ | 26,756 | $ | 18,882 | |||||
| General and administrative | 4,130 | 2,502 | 7,980 | 6,169 | |||||||||
| Loss from operations | (15,882 | ) | (12,512 | ) | (34,736 | ) | (25,051 | ) | |||||
| Interest income | 212 | 90 | 398 | 186 | |||||||||
| Other expense, net | (5 | ) | (1 | ) | (9 | ) | (41 | ) | |||||
| Net loss | $ | (15,675 | ) | $ | (12,423 | ) | $ | (34,347 | ) | $ | (24,906 | ) | |
| Deemed dividends on convertible preferred stock | — | — | (8,880 | ) | — | ||||||||
| Net loss applicable to common shareholders | $ | (15,675 | ) | $ | (12,423 | ) | $ | (43,227 | ) | $ | (24,906 | ) | |
| Per share information: | |||||||||||||
| Net loss per share of common stock—basic and diluted | $ | (0.16 | ) | $ | (0.24 | ) | $ | (0.46 | ) | $ | (0.47 | ) | |
| Basic and diluted weighted average shares outstanding | 99,770,498 | 52,522,225 | 93,180,795 | 52,493,874 | |||||||||
About Marinus Pharmaceuticals
Marinus Pharmaceuticals, Inc. is a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders. Ganaxolone is a positive allosteric modulator of GABAA receptors that acts on a well-characterized target in the brain known to have anti-seizure, anti-depressant and anti-anxiety effects. Ganaxolone is being developed in IV and oral dose forms intended to maximize therapeutic reach to adult and pediatric patient populations in both acute and chronic care settings. Marinus has conducted the first ever Phase 3 pivotal trial in children with CDKL5 deficiency disorder and is conducting a Phase 2 trial in Tuberous Sclerosis Complex, as well as a Phase 2 biomarker-driven proof-of-concept trial in PCDH19-related epilepsy. The company intends to initiate a Phase 3 trial in status epilepticus. For more information visit www.marinuspharma.com.
