Ryan (center)
Living with CDKL5 deficiency disorder
Corporate Presentation
March 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; our anticipated and potential financing plans; 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 ongoing scientific and clinical research investments on our product candidates; estimated net patient pricing of ZTALMY and related market access and payer coverage; expectations regarding long-term patient response and retention for ZTALMY; plans and expectations to optimize costs and expenses; 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 documents for free by visiting EDGAR on the SEC web site at www.sec.gov.
©2024 Marinus Pharmaceuticals. All Rights Reserved I | 2 |
Ganaxolone Development Pipeline
Ongoing trial | Planned future trial |
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
Oral
Suspension
Intravenous
Oral
Suspension
Second Generation Formulation
Phase 1 | Phase 2 |
CDKL5 Deficiency Disorder
Marigold Trial
Refractory Status Epilepticus
RAISE Trial
Tuberous Sclerosis Complex
TrustTSC Trial
Refractory Status Epilepticus
RAISE II Trial
Epileptic Encephalopathies
Lennox-Gastaut Syndrome
Phase 3 | Approved | Anticipated Milestones |
FDA & EMA approved
Interim analysis Q2 2024
Topline data Q4 2024
Enrollment completion Q4 2025
Trial to begin Q4 2024
Phase 2 LGS trial to begin 2025
©2024 Marinus Pharmaceuticals. All Rights Reserved I | 3 |
Ganaxolone U.S. Market Opportunity
Oral | Intravenous (IV) | Second Generation | |
Product | |||
Ganaxolone | Ganaxolone | ||
Development | |||
COMMERCIAL | PIPELINE | PIPELINE | PIPELINE |
Indication
Stage
Incidence / Prevalence
Seizure type(s)
CDKL5 deficiency | Tuberous sclerosis |
disorder | complex |
- Ph3 TrustTSC trial | |
underway | |
FDA and EMA approved | - Topline data anticipated |
Q4 2024 | |
- 1:40,000 live births¹ | - 1:6,000 live births2 |
- 90-100 U.S. newborns / year | - 9.6-11k highly refractory6 |
Generalized and | Focal seizures (primarily) |
focal seizures | |
Not for promotional use |
Refractory status epilepticus
- Ph3 RAISE trial underway | - Enrollment anticipated to |
- Interim analysis planned Q2 | |
conclude Q4 2025 | |
2024 | |
35k U.S. patients / year3,4
Continuous/persistent electrographic or clinical seizures
Lennox-Gastaut
syndrome
- Exploring application of extended-release technologies
- LGS trial anticipated to begin 2025
- 26:100,000 people5
- 48k U.S. patients
- Drop seizures
(atonic, tonic, or tonic- clonic)
- 90% intractable
¹Symonds et al. Incidence and phenotypes of childhood-onset genetic epilepsies: a | 4Rossetti and Lowenstein. Lancet Neurol. 2011 Oct; 10(10): 922-930 | |||
prospective population-based national cohort. Brain. 2019 Aug 1;142(8):2303-2318. | 5Trevathan et al. (1997). Epilepsia, 38(12), 1283-1288 | ©2024 Marinus Pharmaceuticals. All Rights Reserved | I | 4 |
²Curatolo P, Bombardieri R, Jozwiak S. Tuberous sclerosis. Lancet. 2008;372:657-68. | 6Highly refractory population estimated through ZS & Associates Opportunity Assessment market research | |||
³DeLorenzo RJ et al. J Clin Neurophysiol. 1995; 12: 316-325 | and internal assessment of ~40% of refractory TSC patients would represent a highly refractory population. |
Commercial Update
Not for promotional use
ZTALMY® Performance Metrics
U.S. net product revenue of $6.6M | U.S. net product revenue of $19.6M | U.S. net product revenue of $32M- |
for the fourth quarter of 2023 | for the full year 2023 | $34M expected for the fiscal |
year 2024 |
More than 165 patients active on | ~225 million covered lives, |
therapy at the end of 2023 | including both commercial and |
government programs |
Not for promotional use | ©2024 Marinus Pharmaceuticals. All Rights Reserved I | 6 |
A Comprehensive Commercial Strategy to Grow the ZTALMY® Brand
Patient identification | Activate the caregiver |
community |
Focused education to | Continuously enhance the |
HCPs to establish ZTALMY | patient experience |
as the standard of care for | |
CDD seizure management |
- Elevate by educating HCPs on the importance of determining the genetic etiology of patients with refractory epilepsy syndromes
- Increased investment in third party data expected to allow targeting of approximately 2x more CDD patients
-
Inspire through newly added
"Shining Moments " educational programing delivered directly to the caregiver community focused on ZTALMY and CDD the community
- Promotional education targeted to HCPs with a high propensity of having CDD patients and prescribing ZTALMY
- Data driven analytics and HCP segmentation strategies to deliver the right message, to the right HCP, at the right time
• Refine the ZTALMYOne patient support program to meet the evolving needs of the CDD community
Drive best in class practices, establish Marinus as a leader in refractory epilepsy, and build capabilities for future launches
Not for promotional use | ©2024 Marinus Pharmaceuticals. All Rights Reserved I | 7 |
Tuberous Sclerosis Complex
"Many individuals with TSC continue to experience uncontrolled seizures despite a cocktail of multiple antiepileptic drugs. Because new options are always needed, the TSC community welcomes clinical evaluation of new epilepsy treatments"
- Kari Luther Rosbeck, President & CEO of the Tuberous Sclerosis Alliance
Tuberous Sclerosis Complex (TSC)
CAUSE | • | Defect or mutation of TSC1 and/or TSC2 genes | |
INCIDENCE | • | ~1 in 6,000 live births1 | |
COMMON | • | Seizures, cognitive impairment, behavioral difficulties, skin/kidney/lung abnormalities, etc | |
SYMPTOMS | |||
EPILEPSY IN TSC | • Occurs in ~80-90% of those with TSC2 | ||
• | Two-thirds refractory to existing therapies2 | ||
• Seizures typically begin within first year of life (infantile spasms and/or focal seizures)3 | |||
TSC is one of the most common genetic epilepsies often exhibiting highly refractory seizures
despite existing therapies
- Hasbani DM & Crino PB 2018 Hand. Clin. Neurol.
- Henske EP et al.2016 Nat. Rev. Dis. Primers.
- Chu-ShoreCJ et al.2010 Epilepsia
©2024 Marinus Pharmaceuticals. All Rights Reserved I | 9 |
TSC Phase 2 Trial Results
Percent reduction in TSC-associated seizure frequency
100
80
60
40
20
0 -20-40-60-80
-100-200
Primary Endpoint Results: 16.6% median reduction in TSC-associatedseizures
16.6%
=median
Secondary and
Exploratory Analyses
focalinreductionPercent | (median)frequencyseizure | 30 | patientsofPercent | Proportion of patients with a ≥50% reduction | ||||
25.2 % | 50 | in TSC-associated seizure frequency | ||||||
45 | ||||||||
20 | 40 | 36.4% | ||||||
35 | 30.4 % | |||||||
30 | 25.0 % | |||||||
25 | ||||||||
10 | ||||||||
20 | ||||||||
15 | ||||||||
10 | ||||||||
0 | 5 | |||||||
0 | * | |||||||
Subjects with | ||||||||
Intent to Treat | +Cannabidiol +Everolimus | |||||||
Focal Seizure Types | ||||||||
(n=23) | (n=12) | (n=11) | ||||||
(n=19) | * Secondary endpoint | |||||||
Ganaxolone was generally well-tolerated with somnolence, sedation and fatigue reported as the most common adverse events; in addition, one treatment-related serious adverse event of seizure was reported in the trial
©2024 Marinus Pharmaceuticals. All Rights Reserved I 10
Attachments
- Original Link
- Original Document
- Permalink
Disclaimer
Marinus Pharmaceuticals Inc. published this content on 05 March 2024 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 05 March 2024 21:08:59 UTC.