Marinus Pharmaceuticals Provides Business Update and Reports Fourth Quarter and Full Year


  • Received FDA approval of ZTALMY® (ganaxolone) for the treatment of seizures associated with CDKL5 deficiency disorder; first approved treatment for this rare form of genetic epilepsy

  • Awarded a Rare Pediatric Disease Priority Review Voucher by the FDA

  • Eligible for an additional $30 million of funding under the existing Oaktree Capital Management, L.P. credit agreement

  • Patient screening commenced for Phase 3 TrustTSC trial in tuberous sclerosis complex

  • Resupply of clinical trial materials has been moved up to May 2022 for Phase 3 RAISE trial in refractory status epilepticus

  • Marinus to host conference call today at 8:00 a.m. ET / 5 a.m. PT

RADNOR, Pa., March 21, 2022–(BUSINESS WIRE)–Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today reported business highlights and financial results for the fourth quarter and year ended December 31, 2021.

“This year has already proven to be pivotal for Marinus following the FDA’s approval of ZTALMY®, the first and only treatment for seizures associated with CDKL5 deficiency disorder,” said Scott Braunstein, M.D., Chief Executive Officer of Marinus Pharmaceuticals. “This achievement further validates our clinical development plans for ganaxolone as an innovative treatment option across a range of seizure disorders and sets the stage as we move into our next chapter of growth as a commercial stage company. We look forward to building on this momentum as the approval enables the potential for significant non-dilutive funding in the near-term to execute on our business strategy and invest in our future.”

Commercial Preparedness – CDKL5 Deficiency Disorder

ZTALMY® (ganaxolone) oral suspension received U.S. Food and Drug Administration (FDA) approval on March 18, 2022 for the treatment of seizures associated with cyclin-dependent kinase-like 5 deficiency disorder (CDD) in patients two years of age and older.

  • ZTALMY is expected to be commercially available in the U.S. through a specialty pharmacy beginning in July following scheduling by the U.S. Drug Enforcement Administration, which is expected within 90 days of FDA approval

  • To support the CDD community, Marinus plans to launch The ZTALMY One™ Program, a comprehensive patient services program to provide assistance with product access, ongoing support to patients, caregivers and their medical teams, and financial support to eligible patients

  • Commercial launch leadership is in place; 16 field representatives are expected to be onboarded in April and key discussions have been initiated with both government and commercial payers

Clinical Pipeline Update

CDKL5 Deficiency Disorder (CDD)

  • EMA’s Committee for Medicinal Products for Human Use opinion on the Marketing Authorization Application is expected by year end 2022

  • Committed to identifying opportunities throughout the world to improve the lives of more patients, including growing the CDD Expanded Access Program to Europe

Tuberous Sclerosis Complex (TSC)

  • Actively screening patients in the U.S. in the Phase 3 TrustTSC trial after commencing site initiations in the first quarter of 2022

    • Expect to include 60 sites, predominantly in the U.S., Western Europe, Canada and Israel

  • Topline data targeted first half of 2024

Status Epilepticus

  • Expect to resupply clinical trial material and resume recruitment for Phase 3 RAISE trial in refractory status epilepticus (RSE) by end of May

  • Continued U.S. site activations in RAISE trial with 52 sites now open

  • Phase 2 RESET trial in established status epilepticus is planned to begin U.S. enrollment in the second half of 2022

  • Phase 3 RAISE II trial in RSE (for European registration) now expected to begin enrollment in the first half of 2023

Next Generation Ganaxolone and Lennox-Gastaut Syndrome (LGS)

  • Second generation ganaxolone formulation Phase 1 topline data on track for mid-2022 readout

    • If Phase 1 data supports further clinical development, Phase 2 LGS trial expected to begin in the second half of 2022 utilizing second generation formulation

    • Expect to initiate modified release candidate development in 2022

  • The company has identified two prodrug candidates that are targeted to advance into initial Investigational New Drug (IND) enabling studies in the second half of 2022

Second generation ganaxolone formulation is being developed to improve pharmacokinetic (PK) characteristics and expand the therapeutic utility, including efficacy, dosing frequency and tolerability.

General Business and Financial Update

  • A Rare Pediatric Disease Priority Review Voucher (PRV) was awarded to Marinus by the U.S. Food and Drug Administration upon approval of ZTALMY®. The company intends to monetize the PRV to fund on-going operations, including continued clinical development and commercialization efforts for ganaxolone.

  • FDA approval of ZTALMY® makes available through December 31, 2022 an additional $30 million of funding under the existing Oaktree Capital Management, L.P. credit agreement, subject to the satisfaction of certain customary conditions described in the credit agreement.

  • In March 2022, Marinus entered an exclusive license agreement with Ovid Therapeutics to license patents and patent applications in the U.S., the EU and certain other countries in Europe related to the use of ganaxolone for the treatment of CDKL5 deficiency disorder.

Financial Results

  • At December 31, 2021, the company had cash, cash equivalents, and short-term investments of $122.9 million, compared to $140.0 million at December 31, 2020.

  • Recognized $1.5 million and $6.4 million in Biomedical Advanced Research and Development Authority (BARDA) federal contract revenue for the three and twelve months ended December 31, 2021, respectively, as compared to $1.5 million and $1.7 million for the three and twelve months ended December 31, 2020.

  • Research and development (R&D) expenses were $18 million and $73.5 million for the three and twelve months ended December 31, 2021, respectively, as compared to $13 million and $51.1 million, respectively, for the same periods in the prior year; the increase was due primarily to increased R&D headcount, increased clinical trial activity, including the RSE and TSC trials, and drug development activities for both the oral and IV formulations.

  • General and administrative (G&A) expenses were $10.6 million and $37.3 million for the three and twelve months ended December 31, 2021, respectively, as compared to $6 million and $18.5 million, respectively, for the same periods in the prior year; the primary drivers of the change were increased headcount to support scale up of the company’s operations and preparations for commercial launch.

  • Separately, and as a result of the European collaboration agreement with Orion, we booked full year 2021 collaboration revenue of $9 million and a one-time cost of collaboration expense of $1.5 million, both of which were fully recorded in the third quarter of 2021.

  • The company had net losses of $28.3 million and $98.8 million for the three and twelve months ended December 31, 2021, respectively; cash used in operating activities decreased to $55.5 million for the twelve months ended December 31, 2021, compared to $60.9 million for the same period a year ago.

  • Readers are referred to, and encouraged to read in its entirety, the company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2021, to be filed with the Securities and Exchange Commission, which includes further details on the company’s business plans, operations, financial condition, and results of operations.

Corporate Guidance

  • For the fiscal year 2022, the company expects BARDA revenues in the range of $7 to $10 million and total GAAP operating expenses, inclusive of G&A and R&D, to be in the range of $152 to $157 million, of which the company expects stock-based compensation to be approximately $17 million.

December 31,
2021

December 31,
2020

(unaudited)

ASSETS

Cash and cash equivalents

122,927

138,509

Investments

1,474

Other assets

13,913

10,479

Total assets

136,840

150,462

LIABILITIES AND STOCKHOLDERS’ EQUITY

Current liabilities

40,566

10,729

Long Term Debt, Net

40,809

Other long-term liabilities

1,979

2,534

Total liabilities

83,354

13,263

Total stockholders’ equity

53,486

137,199

Total liabilities and stockholders’ equity

136,840

150,462

Three Months Ended
December 31,

Twelve Months Ended
December 31,

2021

2020

2021

2020

(unaudited)

(unaudited)

Revenue:

Federal contract revenue

1,520

1,546

6,358

1,718

Collaboration revenue

8,987

Total revenue

1,520

1,546

15,345

1,718

Expenses:

Research and development

18,014

13,044

73,520

51,106

General and administrative

10,622

6,005

37,278

18,549

Cost of collaboration revenue

1,478

Loss from operations

-27,116

-17,503

-96,931

-67,937

Interest income

23

40

80

499

Interest expense

-1,553

-2,582

Other income (expense), net

341

-6

657

-37

Net loss and comprehensive loss

-28,305

-17,469

-98,776

-67,475

Deemed dividends on convertible preferred stock

-8,880

Net loss applicable to common shareholders

-28,305

-17,469

-98,776

-76,355

Per share information:

Net loss per share of common stock—basic and diluted

-0.77

-0.55

-2.69

-2.80

Basic and diluted weighted average shares outstanding

36,746,112

31,832,970

36,663,340

27,270,055

Conference Call Information

Monday, March 21, at 8:00 a.m. ET
Domestic: +1 (888) 550-5280
International: (646) 960-0813
Webcast Link: https://events.q4inc.com/attendee/991025183
Conference ID: 2696394

About Marinus Pharmaceuticals

Marinus is a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders. Ganaxolone is a neuroactive steroid GABAA receptor modulator that acts on a well-characterized target in the brain known to have anti-seizure effects. It is being developed in IV and oral dose formulations intended to maximize therapeutic reach to adult and pediatric patient populations in both acute and chronic care settings. For more information visit www.marinuspharma.com.

Forward-Looking Statements

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 expected clinical development plans, enrollment in our clinical trials, regulatory communications and submissions and product launches for ganaxolone, and the timing thereof; our commercialization plans; our expectations regarding scheduling by the U.S. Drug Enforcement Administration and the expected timing thereof; our plans to launch a…



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