Marinus Pharmaceuticals : Corporate Overview Slide Deck

Published: 2024-07-08 12:45:09 ET MRNS

Ryan (center)

Living with CDKL5 deficiency disorder

Corporate Presentation

July 2024

Nasdaq: MRNS

@MarinusPharma

Photo Credit: Kelly Crews Photography

Safe Harbor Statement

To the extent that statements contained in this presentation 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 presentation include, among others, statements regarding our ability to continue as a going concern; our expected revenue and expenses; our commercialization plans for ZTALMY® and clinical development plans for ganaxolone, and the expected timing thereof; the clinical development schedule and milestones; expected dosing in our clinical trials; our expected timing to begin and complete enrollment in our clinical trials; the expected trial design, target patient population and endpoints for our clinical trials; interpretation of scientific basis for ganaxolone use; timing for availability and release of data; the potential safety and efficacy and therapeutic potential of ganaxolone; timing and expectations regarding the potential benefits ZTALMY will provide for patients and physicians; timing and expectations regarding regulatory communications and submissions; expectations regarding our agreement with BARDA; expectations regarding our current and contemplated collaborations with ex-US partners, including the potential benefits and timing thereof; expectations regarding the potential market opportunities for our product candidates; expectations regarding patient populations; expectations regarding potential commercial alliances; expectations regarding our cash flow, cash projections and cash runway; expectations regarding the continued uptake of ZTALMY; expectations regarding the impact of on-going scientific and clinical research investments on our product candidates; expectations regarding operating margins; plans for commercial investments; plans to leverage existing our infrastructure and knowledge; our plans for the global access program and the expected benefits and timing thereof; and our expectations regarding future opportunities of oral and IV ganaxolone. Forward-looking statements in this presentation 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, uncertainties and delays relating to patient and physician acceptance of ZTALMY; our ability to obtain adequate market access for ZTALMY; our ability to comply with the U.S. Food and Drug Administration's ("FDA") requirement for additional post-market studies in the required timeframes; the timing of regulatory filings; the potential that regulatory authorities, including the FDA and the European Medicines Agency ("EMA"), may not grant or may delay approval for our product candidates; uncertainties and delays relating to the design, enrollment, completion, and results of clinical trials; unanticipated costs and expenses; early clinical trials may not be indicative of the results in later clinical trials; clinical trial results may not support regulatory approval or further development in a specified indication or at all; actions or advice of the FDA or EMA may affect the design, initiation, timing, continuation and/or progress of clinical trials or result in the need for additional clinical trials; our ability to obtain and maintain regulatory approval for our product candidates; our ability to obtain, maintain, protect and defend intellectual property for our product candidates; the potential negative impact of third party patents on our collaborators' or our ability to commercialize ganaxolone; delays, interruptions or failures in the manufacture and supply of our product candidates; the size and growth potential of the markets for our product candidates, and our ability to service those markets; our cash and cash equivalents may not be sufficient to support our operating plan for as long as anticipated; our expectations, projections and estimates regarding expenses, future revenue, capital requirements, and the availability of and the need for additional financing; our ability to obtain additional funding to support our commercial and clinical development programs; our dependence on ex-US partners to commercialize ZTALMY outside of the US; the potential for our ex-US partners to breach our collaboration agreements or terminate the agreements; and the availability or potential availability of alternative products or treatments for conditions targeted by us that could affect the availability or commercial potential of our product 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 our business in general, see filings we have made with the Securities and Exchange Commission. You may access these documentsfor free by visiting EDGAR on the SECweb site at www.sec.gov.

Ganaxolone Development Pipeline

Ganaxolone is a positive allosteric GABAA receptor modulator with a well-defined MOA designed to treat patients suffering from seizure disorders.

Ganaxolone is designed to modulate both synaptic and extrasynaptic GABAA receptors to calm over-excited neurons.

Oral Suspension

Intravenous

Phase 1

Phase 2

CDKL5 Deficiency Disorder

Tuberous Sclerosis Complex

TrustTSC Trial

Developmental Epileptic

Encephalopathies

Including Lennox-Gastaut Syndrome

Refractory Status Epilepticus

RAISE Trial

Ongoing trial

Planned future trial

Phase 3

Approved

FDA & EMA approved

Anticipated Milestones

Topline data first half of Q4 2024

Trial to begin 1H 2025

Topline data announced June 2024

ZTALMY® Clinical and Commercial Overview

Not for promotional use

ZTALMY® Has the Potential to Significantly Advance Epilepsy Treatment

FDA-approved in CDD

Well-characterized safety profile

Differentiated MOA addresses unmet need

CDD: CDKL5 deficiency disorder; MOA: mechanism of action

Scalable commercial infrastructure supports rapid expansion and adoption

Significant commercial opportunity

Phase 3 CDD Marigold Trial Data and Safety Summary

Phase 3 Marigold data publishedin The Lancet Neurology

First international CDKL5 guidelines publishedin Frontiers in Neurology

Patients taking ganaxolone experienced a significant reduction in seizure frequency

ReductionPercentMedian SeizuresMotorMajorofFrequency

30.7%

Ganaxolone reduced

the frequency of

monthly major motor

seizures by a median of

30.7% compared with

6.9% for placebo

(p=0.0036)*

Δ = 27.1% (47.9 - 9.6)**

6.9%

28-day

Ganaxolone Placebo

*Wilcoxon Rank-Sum Test **Hodges-Lehman Estimate of Median Difference

*Hodges-Lehmann estimate of median difference (95% confidence interval)

**Wilcoxon rank-sum test

Not for promotional use

Treatment Emergent Adverse Events (TEAE)

Preferred Term

Placebo (n=51)

Ganaxolone (n=50)

Any TEAE, n (%)

(88.2)

(86.0)

Somnolence

8 (15.7)

(36.0)

Pyrexia

(7.8)

9 (18.0)

Upper Respiratory

(5.9)

5 (10.0)

Tract Infection

Constipation

(5.9)

(6.0)

Salivary Hypersecretion

1 (2.0)

3 (6.0)

Sedation

(3.9)

(6.0)

Serious Treatment Emergent Adverse Events

Preferred Term

Placebo (n=51)

Ganaxolone (n=50)

Any Serious TEAE, n (%)

5 (9.8)

6 (12.0)

Bronchitis

0 (0.0)

1 (2.0)

Rhinovirus Infection

0 (0.0)

1 (2.0)

Urinary Tract Infection

0 (0.0)

1 (2.0)

Pneumonia Mycoplasmal

1 (2.0)

0 (0.0)

Pneumonia Viral

1 (2.0)

0 (0.0)

Respiratory Syncytial Virus

1 (2.0)

0 (0.0)

Bronchiolitis

Oxygen Saturation Decreased

0 (0.0)

1 (2.0)

Food Refusal

0 (0.0)

1 (2.0)

Pneumonia Aspiration

0 (0.0)

1 (2.0)

Hypoxia

1 (2.0)

0 (0.0)

Faecaloma

1 (2.0)

0 (0.0)

Hypotonia

1 (2.0)

0 (0.0)

Seizure

1 (2.0)

0 (0.0)

Unresponsive to Stimuli

1 (2.0)

0 (0.0)

Includes AEs that occurred >5% of subjects in

ganaxolone arm and ganaxolone > placebo

Phase 3 Open Label Extension Data in CDD

Reduction in monthly major motor seizure frequency through 2 years of the OLE*

Trial data published in Epilepsia

Patients achieved average seizure reductions of approximately 50% at two years of treatment

Patients who remained in the clinical trial at 2 years experienced sustained reduction in MMSF1

*Data as of June 30, 2022

extension analysis (Marigold Study). American Epilepsy Society. Dec 4-8, 2020. Virtual conference.

Not for promotional use

ZTALMY® Performance Metrics and Growth Drivers

U.S. net product revenue of $7.5M for the first quarter

>125% growth from Q1 2023

Continued strong new patient enrollments

Continued growth of new prescribers driving demand

Increased full year 2024 expected U.S. net product revenue to

$33M-$35M

Favorable reimbursement

dynamics across all payers, including both commercial and government programs

Achieved profitability on the ZTALMY

commercial investment in Q1 2024, ahead of original target

Growth Opportunities:

Not for promotional use

Significant Near-term Milestones Build on Commercial Success

Program

Indication

1H 2024

2H 2024

1H 2025

2H 2025

CDKL5 deficiency

Achieved

disorder

profitability*

Tuberous sclerosis

Completed

Phase 3 TrustTSC

Potential filing for FDA

Potential Launch

Phase 3

complex

readout

Approval

enrollment

Developmental and

Begin enrollment of

epileptic

Phase 2 trial

encephalopathies

Second-Gen

Lennox-Gastaut

IND Expected

Program

syndrome

Topline Data Readout Expected Before Year-End

*on ZTALMY commercial investment

Driving Global Access of ZTALMY® (ganaxolone)

Europe:Collaboration agreement with Orion Corporation for ganaxolone in CDD, TSC, RSE

China:Collaboration agreement with Tenacia Biotechnology for ganaxolone in CDD, TSC, SE

MENA:Distribution agreement with Biologix Fzco for ganaxolone

Marinus Access Program

Program initiated in Q4 2023 to expand global access to ZTALMY in non- partnered markets for appropriate patients with seizures associated with CDD

1Subject to achievement of certain clinical and commercial launch milestones related to CDD, TSC, and RSE and annualized sales thresholds for the oral and IV products

2Subject to achievement of regulatory approvals for CDD and TSC

Disclaimer

Marinus Pharmaceuticals Inc. published this content on 08 July 2024 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 08 July 2024 16:44:06 UTC.