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Marinus Pharmaceuticals Provides Business Update and Reports Fourth Quarter 2020 Financial Results

RADNOR, Pa.–(BUSINESS WIRE)–#CDDMarinus 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 and regulatory development activities and reported its financial results for the fourth quarter and fiscal year ended December 31, 2020.

“Although 2020 was extremely challenging for so many, we finished the year achieving several important milestones, which has provided momentum into 2021,” said Scott Braunstein, M.D., Chief Executive Officer of Marinus Pharmaceuticals. “We remain on track to submit a new drug application to the FDA for use of ganaxolone in CDKL5 deficiency disorder, continue active enrollment in our Phase 3 RSE trial, while advancing and expanding our oral programs. In the second half of 2021 our attention will continue to be focused on enrolling our Phase 3 trials in RSE and TSC, advancing our commercialization preparations for ganaxolone’s potential launch in CDKL5 deficiency disorder, and expanding our novel formulation development work. We are also looking to expand our oral and IV franchises into new target therapeutic indications with significant unmet medical need and a strong scientific rationale for the use of ganaxolone.”

“Based on the progress to date, as well as our analysis of all prior studies in a number of epilepsy indications, we believe that the oral formulation of ganaxolone has the potential to become an important drug in a variety of rare, pediatric seizure disorders, a tremendous complement to our expanding IV franchise,” Dr. Braunstein said.

Dr. Braunstein added, “In December, we raised $70 million, ending the year with $140 million of cash on hand, which provides financial support for our ongoing clinical trials, commercial planning and portfolio development. We continue to add talent throughout the organization, including in our commercial, clinical, legal, regulatory, and operations areas. Our focus remains on realizing ganaxolone’s potential to help those suffering from rare seizure disorders.”

Pipeline Update:

PCDH19 Related Epilepsy (PCDH19-RE)

“These findings are very encouraging for those living with PCDH19-related epilepsy, in which many patients continue to experience clusters of seizures that often require inpatient hospitalization. Reducing seizure frequency for this patient population is clinically meaningful,” said Joseph Sullivan, M.D., Professor of Neurology & Pediatrics, Director, UCSF Benioff Children’s Hospital Pediatric Epilepsy Center of Excellence.

Tuberous Sclerosis Complex (TSC)

CDKL5 Deficiency Disorder (CDD)

Refractory Status Epilepticus (RSE)

Corporate Update

CFO Transition

Edward F. Smith, Chief Financial Officer, will be leaving the company to pursue other career opportunities. Mr. Smith has been with the company since 2013, and during his tenure has driven the finance, accounting, information technology, and program management functions, as Marinus sharpened its focus onto its core rare epilepsy franchise. The company has appointed Steven Pfanstiel, who brings 16 years of J&J experience in various financial reporting and business support roles, and most recently at LifeScan Inc., a former J&J business unit focused on blood glucose monitoring, where he served as Vice President, Finance. Mr. Pfanstiel’s effective start date is April 12, 2021.

Financial Update

At December 31, 2020, the company had cash, cash equivalents and investments of $140.0 million, compared to $91.7 million at December 31, 2019. The company believes its cash and cash equivalents as of December 31, 2020 will enable it to fund the company’s current scale of operating expenses and capital expenditures into 2Q 2022. Marinus projects spend in the range of $18 to $20 million per quarter in 2021, offset by approximately $2 to $3 million per quarter from BARDA in 2021. This spend could increase in 3Q with the initiation of the TSC trial and ongoing expenses associated with the RAISE trial.

Marinus recognized $1.7 million in federal contract revenue in the year ended December 31, 2020 as a result of the BARDA contract the company entered into in September 2020. No federal contract revenue was recognized in the year ending December 31, 2019.

Research and development expenses increased to $13.0 million and $51.1 million for the three and twelve months ended December 31, 2020, respectively, as compared to $12.5 million and $43.0 million for the same periods in the prior year. The increase was due primarily to Marinus’ ongoing enrollment in the company’s Phase 3 Marigold Study, which top-line data were announced during the third quarter of 2020, and increased drug development activity, including preclinical studies and manufacturing activities in preparation for a potential NDA filing for ganaxolone in CDD and the recently initiated RAISE trial.

General and administrative expenses increased to $6 million and $18.6 million for the three and twelve months ended December 31, 2020, respectively, compared to $3.0 million and $11.5 million for the same periods in the prior year. The primary drivers of the increase were increased legal and consulting fees of $2.7 million and $1.5 million related to increased headcount as we scale up the company’s operations and prepare for potential commercialization, and noncash stock-based compensation of $1.6 million.

The company reported net losses of $17.5 million and $67.5 million for the three and twelve months ended December 31, 2020, respectively, compared to $15.4 million and $54.1 million in the same period a year ago. Cash used in operating activities increased to $60.9 million for the year ended December 31, 2020 compared to $48.6 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 year ended December 31, 2020, to be filed with the Securities and Exchange Commission, which includes further detail on the above-referenced matters 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

 

December 31,

 

December 31,

 

2020

 

2019

 

 

 

 

ASSETS

 

 

 

Cash and cash equivalents

$

138,509

 

$

90,943

Investments

 

1,474

 

 

739

Contract revenue receivable

 

1,646

 

 

Other assets

 

8,833

 

 

7,160

Total assets

$

150,462

 

$

98,842

LIABILITIES AND STOCKHOLDERS’

EQUITY

 

 

 

Current liabilities

$

10,729

 

$

8,031

Other long term liabilities

 

2,534

 

 

3,042

Total liabilities

 

13,263

 

 

11,073

Total stockholders’ equity

 

137,199

 

 

87,769

Total liabilities and stockholders’ equity

$

150,462

 

$

98,842

 

 

Three Months Ended December 31,

 

Twelve Months Ended December 31,

 

 

2020

 

2019

 

2020

 

2019

 

 

 

 

 

 

 

 

 

 

 

 

 

Federal contract revenue:

 

$

1,546

 

$

 

$

1,718

 

$

Expenses:

 

 

 

 

 

 

 

 

 

 

 

 

Research and development

 

$

13,044

 

$

12,512

 

$

51,106

 

$

42,966

General and administrative

 

 

6,005

 

 

2,960

 

 

18,549

 

 

11,456

Loss from operations

 

 

-17,503

 

 

(15,472)

 

 

(67,937)

 

 

(54,422)

Interest income

 

 

54

 

 

72

 

 

499

 

 

354

Other (expense) income

 

 

-20

 

 

(9)

 

 

(37)

 

 

(53)

Net loss

 

$

(17,469)

 

$

(15,409)

 

$

(67,475)

 

$

(54,121)

Per share information:

 

 

 

 

 

 

 

 

 

 

 

 

Net loss per share of common stock—basic and diluted

 

$

(0.55)

 

$

(1.02)

 

$

(2.80)

 

$

(3.97)

Basic and diluted weighted average shares outstanding

 

 

31,832,970

 

 

15,113,498

 

 

27,270,055

 

 

13,628,194

New Leadership Hires

Steven E. Pfanstiel, Chief Financial Officer (Effective April 12, 2021)

Mr. Steven Pfanstiel is an accomplished life sciences and pharmaceuticals industry professional with extensive experience developing and implementing financing strategies to support R&D, supply chain, and global commercialization organizations. He was most recently Vice President, Finance for LifeScan of Malvern, PA, a world leader in blood glucose monitoring known for OneTouch brand products for managing diabetes, where he was responsible for a finance team of over 40 and drove significant cost savings while optimizing investment in sales and marketing. Prior to LifeScan, Mr. Pfanstiel held financial leadership roles in a wide range of life sciences and pharmaceutical organizations, including Senior Director, Financial Planning and Analysis (FP&A) at Optinose; Senior Director, Global Strategic Marketing, at DePuy Synthes (Johnson & Johnson); Senior Director, North America Commercial, Worldwide Financial Reporting, and Portfolio for LifeScan & Animas (Johnson & Johnson). Prior to his roles with LifeScan and Optinose, Mr. Pfanstiel worked for 16 years in various divisions and operating companies of Johnson & Johnson. Mr. Pfanstiel holds a B.A. in Physics from Wabash College in Crawfordsville, IN; an M.S. in Environmental Systems Engineering from Clemson University in Clemson, SC; and an M.B.A. in Finance from the Kelley School of Business at Indiana University in Indianapolis, IN.

Ian Miller, MD, Vice President, Clinical Development

Dr. Ian Miller is a pediatric epileptologist with 14 years of experience at Nicklaus Children’s Hospital in Miami, where he most recently served as the Chair of the Neurology Department, and on the Board of Directors for First Choice Neurology (a private practice of over 80 neurologists). Within the hospital he was the founding Medical Director of the Ion Channel Epilepsy Program, co-Director of the Tuberous Sclerosis Program, and the Director of Neuroinformatics. Dr. Miller’s research experience includes participation in all five of the pivotal trials for cannabidiol, including one as a first author. He was also a primary investigator for fenfluramine, nasal diazepam, and neuropace, among others, and has provided input regarding trial design and endpoints for three early gene therapy candidates in SCN1A, and enrolled patients in one of them as PI. He is a member of the Medical Advisory Board for the Dravet Syndrome Foundation (DSF), and a member of the American Epilepsy Society. He is board certified in Neurology with a special qualification in Child Neurology, as well as Clinical Neurophysiology, Epilepsy, and Neuroimaging. Dr. Miller testified with the DSF at the FDA in 2018 regarding the need for an ICD10 code for Dravet syndrome, leading to the approval of the G40.83 and its subgroups in October of 2020. Dr. Miller received his M.D. degree from the University of Iowa, in Iowa City, where he grew up. He completed residency and fellowship training at the University of Utah and the University of Washington before completing Clinical Neurophysiology fellowship at Miami Children’s Hospital.

Ian Hunt, Vice President, Market Access & Channel Strategy

Ian Hunt has over 25 years of experience leading payer & channel strategy, field sales and marketing in commercial and pre-commercial programs for some of the industry’s foremost pharmaceutical and biotech companies. He has worked on the launch of over a dozen products and managed pricing and contracting for both Pharmacy and Medical Benefit products. He was most recently Vice President of Market Access; Payor and Site of Care Strategy & Marketing for Horizon Therapeutics where he was responsible for four Business Units, nine Brands, and investigational new products. Previously, Mr. Hunt served as Head, Value & Access – Strategy/Marketing at Bioverativ, Inc., Vice President, Head Global Market Access, Patient Services, and Channel Strategy for Sage Therapeutics, and Senior Director, Payer & Channel Strategy and Marketing for Genzyme. Mr. Hunt holds a Master of Science degree in Politics & Economics from the University of Bristol in the United Kingdom where he was a Rotary Scholar, and a B.A. from Troy University in Alabama.

Fred Halperin, Vice President, Marinus Technology Solutions (IT)

Fred Halperin brings more than 35 years of healthcare and technology experience to Marinus. Immediately prior to joining Marinus, Mr. Halperin was Senior Director, IT Strategy, Transformation and Enablement Services for Otsuka Pharmaceuticals where he led a global effort to define a 3-year IT Roadmap as well as establish several internal technology support functions and processes. Prior to Otsuka, Mr. Halperin worked in the Office of the CIO at Johnson & Johnson as the Chief of Staff to the Corporate Chief Information and Chief Technology officers. In this role, Mr. Halperin acted on their behalf as the primary technology liaison to the J&J Executive Committee and Board of Directors, led the creation and implementation of the enterprise IT strategy and managed several high-priority technology initiatives in partnership with the business lines and global technology infrastructure function. Prior to these recent corporate roles, Mr. Halperin was a senior life sciences technology practice leader with two leading global management consulting firms. Mr. Halperin earned his Bachelor of Science degree in both Computer Science and Industrial Psychology from Tulane University in New Orleans, LA.

About Ganaxolone

Ganaxolone, a positive allosteric modulator of GABAA receptors, is being developed in intravenous and oral formulations intended to maximize therapeutic reach to adult and pediatric patient populations in both acute and chronic care settings. Unlike benzodiazepines, ganaxolone exhibits anti-seizure and anti-anxiety activity via its effects on both synaptic and extrasynaptic GABAA receptors. Ganaxolone has been studied in more than 1,600 patients, both pediatric and adult, at therapeutically relevant dose levels and treatment regimens for up to four years.

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 potential. 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 recently completed the first ever Phase 3 pivotal trial in children with CDKL5 deficiency disorder and is conducting a Phase 3 trial in refractory status epilepticus, Phase 2 trial in tuberous sclerosis complex and a Phase 2 biomarker-driven proof-of-concept trial in PCDH19-related epilepsy. 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 clinical development plans for ganaxolone; our statement that we will complete enrollment in the CALM Study this week; our expectation to release top line TSC data from the CALM Study in Q3 2021; our plans to present the Violet Study at a scientific meeting later this year and to publish the trial in a scientific journal; our expectation to move to a Phase 3 trial in TSC in the third quarter of 2021; our plans to have a pre-NDA meeting with the FDA at the end of Q1 2021 for our planned mid-2021 NDA submission for CDD; our plans for the EMA pre-marketing authorization application meeting to support a MAA submission for CDD at the end of the third quarter of 2021; our plans for entering into an agreement with a European commercial partner, targeting completion in the summer of 2021; our expectation to have a vast majority of sites for the Phase 3 RAISE trial open by the end of Q2 2021 with topline data expected in 1H 2022; our plans for a separate RSE trial to be conducted in Europe to commence in the first half of 2022; our exploration of earlier intervention for treatment in SE in ESE, targeting convulsive patients in the emergency room who have been treated with and failed benzodiazepines; our plans for a potential mid-2021 NDA submission and a fiscal 2022 launch, if approved, of ganaxolone in CDD; our expectations to hold a meeting with the EMA in Q3 2021 with the first patient enrolled in a double blind, placebo controlled, global Phase 3 trial for TSC in Q4 2021; our expectations that our cash, cash equivalents and investments will be sufficient to fund our operating expenses and capital expenditures into the second quarter of 2022; the potential for our spend to increase in the third quarter of 2021 with the initiation of the TSC trial and ongoing expenses associated with the RAISE trial; the potential safety and efficacy of ganaxolone; expectations regarding our ability to receive and utilize a priority review voucher; the therapeutic potential of ganaxolone; our belief that ganaxolone has the potential to become an important drug in a variety of rare, pediatric seizure disorders; and our plans for an expanded access program for ganaxolone.

Contacts

Sasha Damouni Ellis

Vice President, Investor Relations & Corporate Communications

Marinus Pharmaceuticals, Inc.

484-253-6792

sdamouni@marinuspharma.com

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