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| Statement |
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| We're incredibly proud of the job that Christy's team has done within six or so months of launch, we had every state Medicaid program reimbursing ZTALMY |
| We are excited for the opportunity to bring ZTALMY to more CDD patients in need and believe our commercial strategy has us well positioned to realize the potential of this novel treatment |
| We have two key data readouts in RSE and TSC that is positive to drive significant growth for our ganaxolone franchise and we look forward to sharing these and other important updates in the months ahead |
| We finished 2023 with another strong quarter of enrollment and robust quarterly growth |
| And over the coming weeks after positive data, we will set up a meeting with the FDA, a pre-NDA CMC meeting very similarly what we did with ZTALMY and that was an incredibly successful strategy |
| As Christy mentioned, this increase from 2023 represents continued strong and steady execution on the launch |
| And we're confident in delivering the data set as we talked about and probably even more excited about sharing some of the secondaries in the fall as well, Andrew |
| First of all, I am proud of how we managed the business in 2023 |
| So the last few weeks -- the team has made great progress |
| This growing data set should drive a robust package for both the FDA filing and our health economic outcomes |
| We see the recent uptick in enrollment as a strong reflection of the potential market opportunity for the IV franchise |
| And we are looking forward, this has been a three year project, the labor of love I think our clinical team has done an amazing job at enrolling the right type of patients for the study |
| So what we do know is that the targeting that we've done has been very, very good from '22 to now |
| And so that continuous variable is extremely robust in terms of statistical power |
| The combination of our team's leadership, the commercial plans we have outlined and the success of ZTALMY gives us the confidence that ganaxolone has the potential to become a blockbuster franchise across CBD, TSC and RSE |
| That said, traditionally, you know the market better than us |
| 2024 will be a pivotal year for the company as we have built a solid foundation that has us well positioned to drive future growth |
| Together, the CDD, RSE and TSC markets represent a multibillion-dollar opportunity, where we believe we can take a firm leadership position for these disease states and other refractory epilepsies |
| We believe there is a strong business rationale and market opportunity for the expansion of our ZTALMY business into TSC, where we know there is a significant unmet need in refractory patients |
| This solid performance is the result of our strategy to establish ZTALMY as a critical treatment in the comprehensive management of seizures associated with CDD and to ensure that patients have seamless access to ZTALMY from prescription through fulfillment |
| We continue to see swift payer approval with time from enrollment to patients fill of approximately two weeks in the second half of 2023, representing a consistent improvement throughout the year and demonstrating payers' understanding of ZTALMY's impact on patients in need |
| Additionally, payer approvals of CDD prescriptions remain at nearly 100%, indicating strong payer recognition of the value of ZTALMY for these patients |
| And by the -- and when we finish the TSC study, we'll be happy to share some of the -- we haven't seen the blood levels in TSC, but we've seen them in other healthy volunteer studies or SAD, MAD studies -- and certainly, we're feeling good about the discontinuation rates in the real world |
| With corporate and system-level financial decision makers, we believe engaging with these key stakeholders can accelerate access and awareness leading to more favorable formulary placement and will ultimately provide patients with earlier access to treatment |
| And with the open-label extension data published late last year, we are able to emphasize ZTALMY's sustained efficacy and safety profile supporting the use of ZTALMY as a proven treatment for combating seizures associated with CDD |
| We have a very high reason to believe that the prodrug both ganaxolone and the cleavage of the prodrug structurally looks incredibly safe |
| So that's also encouraging on the efficacy side |
| We feel very good about it |
| We'll have improved cost of goods |
| Our experience with ZTALMY provides Marinus with a solid foundation for two potential commercial launches in 2025 |
| Statement |
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| And there are some other important business issues that we will talk about over the coming months on the prodrug program |
| Physicians are not perfect beasts, but we feel quite confident that the placebo rate will come in lower than our original assumption of 30% to 40% |
| In our Phase 2, we had one patient who was on 100 different drugs and just collecting all of that data to me -- it could create issues |
| today suffering from super-refractory status |
| We could not be more pleased with the baseline demographics of the patients enrolled, the high percentage of patients rolling over to the open-label portion of the study and the low overall discontinuation rates, which are substantially different than what we saw in Phase 2 |
| And the only thing I'll add to Christy's comments is that, the literature is quite clear that IV anesthesia leads to increased morbidity and mortality |
| We were also not afraid to make tough decisions, such as discontinuing the established status epilepticus trial and making other cost reductions to ensure adequate cash runway headed into two significant data readouts |
| I think when we started the study, we really could not get a very comfortable handle on where placebo rates would be in the study |
| And our clinical team has pressed every enrollment every physician on enrollment making it crystal clear that if a patient wants to be enrolled, the physician had to feel comfortable that IV anesthetic was an extra of choice |
| As Scott mentioned, one of the things that keeps him up at night is that these patients are quite sick |
| The company reported a net loss before income taxes of 41.8 million and 142.9 million for the 3 and 12 months ended December 31, 2023, as compared to a net loss before income taxes of 32.7 million and 16.4 million for the same period in the prior year |
| This analysis showed that even in the absence of IV anesthesia, refractory status that was treated with three or more IV anti-seizure medications had worse outcomes and longer lengths of stay |
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