Q3 2023 Marinus Pharmaceuticals Inc Earnings Call

Speaker 1: Greetings and welcome to the Marinus Pharmaceuticals third quarter 2023 financial results and business update call. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question and answer session. If you would like to ask a question during this time, simply press star 1 on your telephone keypad. If you wish to withdraw your question, you again press star 1.

Greetings and welcome to the <unk> Pharmaceuticals third quarter 2023 financial results and business update call. At this time all participants are in a listen only mode.

After the Speakers' presentation, there will be a question and answer session. If you'd like to ask a question. During this time simply press star one on your telephone keypad if.

If you wish to withdraw your question Hugh again press Star one.

For time, please restrict yourself to one question you may queue back up for a follow up if time permits and now it is my pleasure to introduce your host Sanya Weigel Senior Vice President Investor Relations Human resources and <unk>.

Speaker 1: For time, please restrict yourself to one question. You may queue back up for a follow-up if time permits. And now, it is my pleasure to introduce your host, Sonia Weigel, Senior Vice President, Investor Relations, Human Resources, and Corporate Affairs. You may begin, Miss Weigel.

Corporate Affairs, you may begin Ms Wigle.

Thank you and good morning.

Speaker 2: With me from there, and if for Dr Scott Brown team chairman and chief executive officer. Christy shaper, keep commercial officer. TJ lion business officer. Dr Joe hand medical officer. And see fan seal, keep financial officer and chief operating officer.

With me from Meredith for Doctor, Scott, Brian <unk>, Chairman and Chief Executive Officer Mr.

Mr Shaffer Chief commercial officer.

TJ Lyon Chief business Officer.

Dr. Joe Houlihan, Chief Medical Officer, and Bill <unk>, Chief Financial Officer, and Chief operating Officer.

Speaker 2: Before we begin, I would like to remind everyone that some of the statements we are making today are forward-looking statements under the security laws.

Before we begin I would like to remind everyone that some of the statements. We're making today forward looking statements under the securities laws.

Speaker 2: These forward-looking statements 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 such forward-looking statements.

These forward looking statements involve substantial risks and uncertainties that could cause our clinical development programs future.

Your result performance or.

Steven to differ significantly from those expressed or implied by such forward looking statements.

Speaker 2: These risks and uncertainties and risks associated with our business are described in the company's report filed with the Securities and Exchange Commission, including Form 10-K , 10-Q, and 8-K.

These risks and uncertainties.

And risks associated with our business are described in the company's report filed with the Securities and Exchange Commission.

<unk> Form 10-K, 10-Q and 8-K.

Speaker 2: I will now turn the call over to our CEO , Dr. Scott Bronstein.

I will now turn the call over to our CEO, Dr. Scott Braunstein.

Speaker 3: Thank you, Sonia, and welcome to our call. Before I turn to our progress in the third quarter, I want to express my appreciation to those of you who attended in person or via webcasts on a investor and analyst event in September . I also want to thank our key opinion leaders, Dr. Dar, Dr. Kamehgan, Dr. Khan, who reviewed their clinical experience treating patients with status and TSC.

Thank you Sonya and welcome to our call before I turn to our progress in the third quarter I want to express my appreciation to those of you who attended in person or via webcast, our investor and analyst event in September.

I also want to thank our key opinion leaders Dr. Dara Doctor team Hagan, Dr Khan, who reviewed their clinical experience treating patients with status and PSD.

Speaker 3: The third quarter of 2023 was marked by strong Zitomie sales growth, meaningful progress in expanding global access to Zitomie, continued advancement of our late-stage clinical programs, and ongoing development of our second-generation oral gonaxalone product.

The third quarter of 2023 was marked by strong holiday sales growth meaningful progress and expanding global active does it tell me continued advancement of our late stage clinical programs and ongoing development of our second generation oral <unk> product.

We are pleased to report that the only sale in the third quarter slightly exceeded our mid September expectation with revenues of $5 4 million.

Speaker 3: We are pleased to report that the Tommy sale in the 3rd quarter, slightly exceeded our mid September expectations. With revenue of 5.4M dollars. And we are once again, raising our guidance to the range of 18.5 to 19M dollars.

We are once again, raising our guidance to the range of 18 $5 million to $19 million.

Speaker 3: Just 4 full quarters into our launch, we are pleased to report our annual revenue run rate is well north of 20M dollars.

For full quarters into our launch we are pleased to report our annual revenue run rate is well north of $20 million.

These results validate our efforts to build a strong commercial organization and execute on a rare disease focused strategy.

Speaker 3: These results validate our efforts to build a strong commercial organization and execute on our rare disease focused strategy.

Speaker 3: Christy safer or chief commercial officer will review our achievements and the steps we are taking to prepare for 2 new potential launches. 2024 for concerns will be reached Verne riders on atell1

Christy Schaffer, our Chief commercial Officer will review, our achievements and the steps we are taking to prepare for two new potential launches in 2025.

Speaker 3: In addition to the early commercial success in the US, we are making meaningful progress in expanding access to zootomies for patients around the world.

In addition to the early commercial success in the U S. We are making meaningful progress in expanding access to <unk> for.

For patients around the world.

Speaker 3: Today, we announced the launch of our new Global Managed Access Program, designed to provide positions the opportunity to formally request the use of the polymy for their patients and geographies, where the product is not commercially available and as supported by local regulatory requirements.

Today, we announced the launch of our new Global managed access program designed to provide physicians the opportunity to formally request the use of <unk> for their patients in geographies, where the product is not commercially available and is supported by local regulatory requirements.

Speaker 3: We are pleased to be collaborating with German and experienced leader in this area who will facilitate the program.

We are pleased to be collaborating with Durbin and experienced leader in this area, who will facilitate the program.

Speaker 3: Comments lines are chief business officer will provide an overview following comments from Chris.

Tom Lyons, our Chief business Officer, who will provide an overview following comments from Christie.

Our commercial partners in the EU, China in Mena region have also made notable progress.

Speaker 3: Our commercial partners in the EU, China, and MENA regions have also made notable progress.

Speaker 3: The Center for Drug Evaluation of the China National Medical Product Administration granted priority review of the NDA submitted by PNASA Biotechnology for Zitolni in CDD.

Center for drug evaluation of the China National Medical product administration.

Granted priority review of the NDA submitted by beneath the biotechnology or is it only in CVD.

Speaker 3: Additionally, Orion Corporation continues to plan for the commercial launch of the tell me. And selected European countries in 2024. And we expect our partner in the MENA region biologics. That product available for distribution by the end of this year.

Additionally, Orion Corporation continues to plan for the commercial launch of this call me in selected European countries in 2024, and we expect our partner in the Mena region Biologics.

Product available for distribution by the end of this year.

We are proud that in such a short period of time Meredith has created a global network that will provide the opportunity for CBD patients and their families to access our novel anti <unk> therapy.

Speaker 3: We are proud that in such a short period of time, Marinus has created a global network that will provide the opportunity for seeded patients and their families to access our novel anti-convulpant therapy.

Let me take a few moments to review our clinical pipeline, starting with an update on the phase III <unk> trial of IV <unk> in refractory status epilepticus.

Speaker 3: Let me take a few moments to review our clinical pipeline. Starting with an update on the phase 3 raised trial of IV gonaxalone. In refractory status level up.

Speaker 3: In our last update provided at our investor and analyst event in September . We noted the race trial enrollment was on an upward trajectory since early August . Following the activations of all clinical sites.

In our last update provided at our Investor and analyst event in September we noted the raise trial enrollment was on an upward trajectory since early August following the activation of all clinical site.

Speaker 3: Although total enrollment has continued to grow and screening activity remains quite high. The rate of enrollment has continued to show more variability than we expect.

Although total enrollment has continued to grow and screening activity remains quite high.

<unk> enrollment has continued to show more variability than we expected.

Speaker 3: Based on current enrollment trends, we now project to enroll the number of patients required for the interim analysis by the end of the first quarter of 2024, rather than our previous guidance of January .

Based on current enrollment trends, we now project, we enrolled the number of patients required for the interim analysis by the end of the first quarter of 2024, rather than our previous guidance of January <unk>.

Speaker 3: As a result, we now anticipate top line data in the 2nd quarter of 2024. If the predefined stopping criteria for the interim analysis are met.

As a result, we now anticipate top line data in the second quarter of 2024, if the predefined stopping criteria for the interim analysis are met.

Speaker 3: The entire organization remains a deeply focused on advancing our day three clinical trials in refractory, satisfactory, status, epilepsitis, and TSC.

The entire organization remains acutely focused on advancing our phase III clinical trial in refractory status epilepticus and PSD.

We are confident in the benefit that <unk> could bring to critically ill patients and a significant commercial opportunity for the first novel therapy for acute status in well over a decade.

Speaker 3: We are confident in the benefits that IDGEN-AX balloon could bring to critically ill patients and a significant commercial opportunity for the first novel therapy for acute status and well over-adjected.

Speaker 3: We are committed to successfully completing both the raise and trust in 2024. And continue to make the investment to prepare for these commercial launch.

We are committed to successfully completing both the raised and cross PSC trial in 2024.

Need to make the investments to prepare for these commercial launches.

Speaker 3: Preparations are underway for an NDA piloting and we don't expect the additional delays to significantly impact the timing of our commercial loss.

Separations are underway for an NDA filing and we don't expect the additional delay and significantly impact the timing of our commercial launch.

Speaker 3: Finally, with the success of the Tommy, the recent extension of our cash runaway into Q4 2024 and tightening up our spend to focus on our most valuable market opportunity.

Finally, with the success of the Tommy the recent extension of our cash runway into Q4, 2024 and timing of our spend to focus on our most valuable market opportunities.

Speaker 3: We believe we have the appropriate resources required to complete 2 key data readouts and prepare for a bright future.

We believe we have the appropriate resources required to complete two key data readouts and prepare for a bright future.

Before I turn to our oral franchise I want to give a brief update on our reset trial and establish etsy.

Speaker 3: Before I turn to our oral franchise, I want to give a brief update on our reset trial in established SE. We have chosen to continue to the phase two reset trial in established status to focus additional resources on the expansion of our refractory clinical programs, including further investigation of a potential development opportunity in super refractory status.

We have chosen to discontinue the phase III reset trial and establish status and focus additional resources on the expansion of our refractory clinical programs, including further investigation of a potential development opportunity in super refractory status or.

Speaker 3: Our efforts will continue to focus on completing the race trial, accelerating the enrollment for race 2, as well as generating additional data that has the potential to support further studies in SRSE and RSE health economics outcomes.

Our efforts will continue to focus on completing the raised trial accelerating the enrollment breweries too as well as generating additional data that has the potential to support further studies and Srs.

And RSV health economic outcomes research.

Speaker 3: We will evaluate every investment the company is making and prioritize the opportunities that we believe will maximize shareholder value.

We will evaluate every investment the company is making and prioritize the opportunities that we believe will maximize shareholder value.

Moving to our oral franchise, we are actively enrolling patients in our global Phase III Trust PSC trial.

Speaker 3: Moving to our oral franchise, we are actively enrolling patients in our global phase 3 trust TSC child.

Speaker 3: We believe that the Tommy can address a significant unmet medical need for patient suffering, from refractory seizures associated with CSE. And we are currently the only product in phase three development for this indicator.

We believe that the Tony can address a significant unmet medical need for patients suffering from refractory seizures associated with DSD and we are currently the only product in phase III development for this indication.

Blinded discontinue rates in this trial remained low which gives us high confidence in the tolerability and potential efficacy of our new titration schedule.

Speaker 3: Blinded discontinue rates in this trial remain low, which gives us high confidence in the tolerability and potential efficacy of our new titration schedule.

We expect top line data mid 2024 and have every reason to believe that this study can replicate the success of the <unk>. The end of the Marigold trial, and our real world experience to date.

Speaker 3: We expect top line data mid 2024 and have every reason to believe that this study can replicate the success of the colony. Demon the marigold trial and our real world experience today.

With several key Readouts in 2024, we believe we have laid a strong foundation for near and long term growth and have prioritized key program in our cash runway accordingly.

Speaker 3: With several key readouts in 2024, we believe we have laid a strong foundation for near and long-term growth and have prioritized speed programs and our cash runaway accordingly.

Speaker 3: With that, I'd like to turn the call over to our Chief Commercial Officer, Kristi Schaper.

With that I'd like to turn the call over to our Chief commercial officer safer.

Thank you Scott and good morning.

Speaker 4: And please, to present the progress we have made in the third quarter and over the first year following our successful Zatami launch.

To prevent the progress we have made in the third quarter and over the first year following our actuals that Tommy launch.

We established the Tommy as a brand physicians and patients track, which validates our innovative approach and positions us for success.

Speaker 4: We establish the Tommy as a brand physicians and patients press, which validates our innovative approach and positions us for success with two near-term additional potential launches.

Near term additional potential launches.

Speaker 4: We continue to collaborate closely with our CDD Centers of Excellence, helping connect them with their community and local HCPs to maximize access to care for their patients.

We continue to collaborate closely with our CBD centers of excellence, helping connect them with our community and local HCP to maximize access to care for their patients.

As a result of the successful effort net product revenues have grown steadily quarter over quarter since the <unk> launch and.

Speaker 4: As a result of these successful efforts, net product revenues have grown steadily quarter over quarter since the Tommy's month.

Speaker 4: Importantly, our patient retention rate remains very strong at approximately 80%.

Importantly, our patient retention rate remained very strong at approximately 80%.

In the third quarter of 2023 net product revenues grew to $5 $4 million.

Speaker 4: In the third quarter of 2023, net product revenues grew to $5.4 million.

At the end of the third quarter, we had approximately 140 patients active on therapy and on average it takes approximately 13 days from prescription order kicked up down that.

Speaker 4: At the end of the third quarter, we had approximately 140 patients active on therapy and, on average, it takes approximately 13 days from prescription order to fulfillment.

Speaker 4: are 225 million covered lives with over 100% of on-label claims reimburse the date.

There are 225 million covered lives with over 100% of on label claims reimbursed to date.

Speaker 4: Hayers are continuing to recognize that this is a serious condition for truth. Reducing reimbursement time from four to two weeks.

Payers are continuing to recognize that this is a serious condition can treat reducing reimbursement time from four to two weeks.

Given our solid growth we are pleased to increase our annual revenue guidance for 2023 to a range of 18 $5 million to $19 million.

Speaker 4: Given our solid growth, we are pleased to increase our annual revenue guidance for 2023 to arrange $18.5 to $19 million.

Speaker 4: I want to thank the entire commercial team for the dedication and passion they demonstrate day in and day out to bring meaningful change to the lives of the patients we serve.

I wanted to thank the entire commercial team for the dedication and passion. They demonstrate day in day out to bring meaningful change to the lives of the patients we serve.

Looking ahead, our cross functional team of narrow nerf and play is working hard in preparation for the Aes annual meeting, where we will continue to establish narrowness as a committed member of the epilepsy community.

Speaker 4: Looking ahead, a cross-principital team of Marinus employees is working hard in preparation for the AES annual meeting, where we will continue to establish Marinus as a commended member of the epilepsy community.

We are excited to build on our present from last year's conference and our first year in attendance as a commercial organization.

Speaker 4: We are excited to build on our presence from last year's conference and our first year in attendance as a commercial organization.

Speaker 4: We plan to engage with over 300 US-based HCP attendees through various opportunities, including Arta Tomey Expebitbeur as a Tomey product presentation by Dr. Daniel Gossett from Sector's Trial Zorology and ongoing engagement with HCP's and Patient Advocacy Group.

We plan to engage with over 300 U S based HCP attendees for various opportunities including.

Tell me there.

Has that Tommy product presentation by Dr. Daniel got that from Texas child, Neurology and ongoing engagement with HCP and patient advocacy groups.

Speaker 4: Additionally, our scientific and medical teams have planned for several data presentations that Joe will discuss in more detail.

Additionally, our scientific and medical teams have planned for several data presentations that Jon will discuss in more detail.

The rare genetic epilepsy franchise continues to evolve the Tommy brand with new initiatives surrounding the importance of genetic testing early patient identification and caregiver engagement.

Speaker 4: The rare genetic epilepsy franchise continues to evolve the Sotami brand with new initiatives surrounding the importance of genetic testing, early patient identification, and caregiver engagement.

Speaker 4: All support our continued progress into 2024 and focus on the CDD community while allowing us to plan for an expanded indication in TSC.

All support our continued progress into 2024 and focus on the CDB community, while allowing us to plan for an expanded indication in TSA.

PSA planning and development is well underway with a clear focus on franchise efficient and the distinct needs of the TSA community.

Speaker 4: TSC planning and development is well underway with a clear focus on franchise efficiencies and the distinct needs of the TSC community.

Speaker 4: In terms of the next phase of our planning for the acute care franchise, we are making significant progress in preparation for the IV launch and are taking a strategic approach to plan for and build a hospital-focused acute care commercial organization.

In terms of the next phase of our planning for the acute care franchise, we are making significant progress in preparation for the IV launch and are taking a strategic approach to plan for and build the hospital acute care commercial organization.

Speaker 4: During our investor day event in September , we underscored the clinical gaps faced by refractory status patients who progressed through multiple ASMs or IV anesthesia, as well as the significant treatment costs tied to each approach.

During our Investor day event in September we underscore the clinical gap phase III refractory status patients progressed through multiple ASI or IV anesthesia.

As well as the significant treatment costs.

<unk> approach.

Speaker 4: Since then, we have fully outlined the substantial economic burden on the healthcare system while administering usual care to these patients.

Thanks, Dan we have fully outline the substantial economic burden on the health care system, while administering usual care to these patients.

These vital insight on bolstered our total value proposition and going into next year, we will be testing <unk>.

Speaker 4: These vital insights have both stirred our total value proposition and going into next year, we will be testing the key value messages across both clinical and financial stakeholders.

Messages across both clinical and financial stakeholders.

This feedback directly from our customer base will support and refine how we plan to address their distinct value drivers with the goal of maximizing the perceived value of IV <unk> and accelerating access.

Speaker 4: This feedback directly from our customer base will support and refine how we plan to address their distinct value drivers with the goal of maximizing the perceived value of Ivy can excellent and accelerating access.

Speaker 4: The Tell Me Success today provides a positive foundation for growth and a continued reason to believe that Gennaxebel and Revenue have the potential to reach an annual revenue of greater than $1 billion for the rare genetic epilepsy franchise and the acute care franchise combined.

Does it tell me success to date provides a positive foundation for growth and a continued reason to believe that <unk> revenues have the potential to reach an annual revenue of greater than $1 billion for the rare genetic epilepsy franchise and the acute care franchise combined.

Speaker 4: I'll now hand the call over to our Chief Business Officer, DJ Lyon, to discuss our managed access program and how we're expanding global access to the Tommie.

I'll now hand, the call over to our Chief business Officer, TJ Lion to discuss our managed access program and how we're expanding global access is it taught me.

Speaker 5: Thank you, Christie. I'd like to take a moment to provide more detail on our new Global Managed Access Program, announced this morning, concurrent with our third quarter finance.

Thank you Christy I'd like to take a moment to provide more detail on our new global managed access program announced this morning concurrent with our third quarter financials.

This initiative supports our mission to bring innovative medicines to patients with a CVD diagnosis.

Speaker 5: This initiative supports our mission to bring innovative medicines to patients with a CVD diagnosis.

Speaker 5: This continues to provide physicians access to the Tommy for qualifying CDD patients in geographies where the Tommy is not available commercially and as supported by local regulatory re

And to provide physicians access because of the Tommy for qualifying patients in geographies, where the economy is not available commercially and as supported by local regulatory requirements.

The Baroness exit program will be managed by Durbin the leader in the international management and distribution of specialized pharmaceuticals, including providing medical support in certain countries.

Speaker 5: Marit-Exas program will be managed by Durban, the leader in the International Management and Distribution of Specialized Pharmaceuticals, including providing medical support in certain countries.

Part of the unified groups product access division.

Speaker 5: Part of UNIFAR Group's product access division, there have been works in partnership with Global Pharmaceutical and Biotech Companies, to facilitate managed pharmaceutical product access programs throughout the world. The company has over 25 years of experience in designing and implementing X.

Urban works in partnership with global pharmaceutical and biotech companies facilitate managed pharmaceutical product access programs throughout the world.

The company has over 25 years of experience in designing and implementing actions.

Access request in geographies, where Marriott had the commercial arrangement in place will be managed by the local commercial organization in their respective territory and are not eligible for the Meredith access program.

Speaker 5: Access request in geographies where Marinus had the commercial arrangement in place will be managed by the local commercial organization in their respective territory and are not eligible for the Marinus exit.

Speaker 5: This little detail can be found in our press release issued this morning.

Additional details can be found in our press release.

Issued this morning.

Speaker 5: We believe this program has a potential to generate incremental revenue from Arranit and could be supported by funding from sources such as government agencies, local hospitals, charitable institutions, private insurance, and in some cases, private pay, depending upon.

We believe this program has the potential to generate incremental revenue for Meredith and could be supported by funding from sources, such as government agencies local hospitals charitable institutions.

Private insurance and in some cases private pay depending upon the country.

Speaker 5: Net of transaction fees, the revenue generated from this program will go to America. We're very pleased to be...

Net of transaction fees. The revenue generated from this program will go to America.

We're very pleased to be working with they have been on the launch of the Meredith access program. This program along with our collaboration agreements in place in Europe, China, and the Mena region.

Speaker 5: This program, along with our collaboration agreements in place in Europe , China, and the mean of region, will allow us to expand our global access strategy and help make it on the available for appropriate CDD patients and geographies where there are no approved treatment options and local regulation costs.

Allow us to expand our global access strategy and help makes it available for appropriate CBD patients in geographies, where there are no approved treatment options and local regulations allow.

Speaker 5: At this time, I would like to turn the call over to our Chief Medical Officer, Dr. Joe Lohan, for an update on our clinical programs in development.

At this time I would like to turn the call over to our Chief Medical Officer, Dr. Joe <unk> for an update on our clinical programs in development.

Speaker 3: Thank you, TJ. Hello, everyone. It's my pleasure to provide an overview of the pipeline progress we've made since our second quarter call. I'll start with our development programs.

Thank you T. J Hello, everyone. It's my pleasure to provide an overview of the pipeline progress we've made since our second quarter call.

I'll start with our development programs in status epilepticus.

Speaker 3: Scott mentioned the raised file of Ivy Genact alone and refractory status continues to enroll. With over 75% of the patients required for the interim analysis randomized to date.

Scott mentioned, the res trial of IV <unk> in refractory status continues to enroll with over 75% of the patients required for the interim analysis randomized to date.

Speaker 6: US Center to Council for the bulk of enrollment with new insights continuing to add patience.

U S centers account for the bulk of enrollment with numerous sites continuing to add patients.

Speaker 6: With that said, based on the current enrollment rates, we believe the number of patients required from the interim analysis will be enrolled by the end of the first quarter, with top-line data now expected in the second quarter of 2024.

With that said based on the current enrollment rates, we believe the number of patients required for the interim analysis will be enrolled by the end of the first quarter with topline data now expected in the second quarter of 2024.

As Scott mentioned.

Speaker 6: Got mentioned, although patient screening continues at a high rate, we're seeing more enrollment variability than anticipated.

Although patient screening continues at a high rate.

We're seeing more enrollment variability than anticipated.

Speaker 6: Our mountain consideration, limiting enrollment to the right patients to demonstrate the clinical benefit of the maximum in a highly refractory RSE population. I'm confident that the criteria for study.

Paramount consideration eliminate enrollment for the right patients to demonstrate the clinical benefit of <unk> and a highly refractory RSV population.

And I'm confident that the criteria for study qualification will be that.

Speaker 6: We continue to maintain strong emphasis on site engagement and education, including in-person site outreach from our medical science at a gofair scene, here-to-peer programs between site coordinators and principal investigators, virtual investigator meetings and space meetings.

We continue to maintain a strong emphasis on site engagement and education, including in person site outreach from our medical scientific affairs team spirit.

Peer to peer programs between site coordinators and the principal investigators.

Virtual investigator meetings and case reviews.

Now let me provide a brief overview of what to expect from the Illumina in Austin.

Speaker 6: Now, let me provide a brief overview of what to expect in the intermittent loss. Protocol provides for now.

Protocol provides for an analysis at 82 patients.

Speaker 6: which is powered at 94% in the effect of 40% difference between the maximum and the costee-bo.

Which is powered at 94% to detect a 40% difference between <unk> and Exelon and placebo.

As we've discussed previously the co primary endpoints herself Juan onset of activity as measured by the proportion of participants with Sce's location within 30 minutes without medications for the acute treatment status.

Speaker 6: As we discussed previously, the co-primary end point to step one onset of action is measured by the proportionate participants with a C cessation within 30 minutes without medications for the acute treatment status.

Speaker 6: and two, durability of effects. As reflected in the proportion of participants, you do not progress to IVNFB within 36 hours.

To durability of effect.

Reflected in the proportion of participants who do not progress to IV anesthesia within 36 hours.

The key secondary endpoints are first time two status location. Following study drug initiation, which is another way to assess onset of effect.

Speaker 6: The key secondary end points are first, I'm two statisticians following study drug initiation, which is another way to assess onset of effect.

Speaker 6: Second, lack of progressions IV in the city for 72 hours, following study, drug industry.

Second lack of progression to IV anesthesia for 72 hours following study drug industry AC.

Speaker 6: The lateral endpoint is important because of the stress and durability of the effect after the infusion of study medications completed.

The latter endpoints important because of assessing the durability of effect. After the infusion study medications is completed.

With topline data of the interim analysis will include results of the co primary and key secondary endpoints.

Speaker 6: The top line data of the error analysis will include results of the co-primary and C-secondary end point. We plan to announce these results because the study meets stopping criteria for ethics.

And we plan to announce these results to the study would meet the stopping criteria for efficacy.

Other secondary in health care utilization endpoints will be analyzed after all patients have completed the full study will be presented at upcoming scientific meetings.

Speaker 6: Other secondary and health care utilization endpoints will be analyzed after all patients have completed the full study. And we'll be presented with upcoming scientific meeting.

Speaker 6: Following a successful urinal, we plan to begin transitioning the majority of race sites to open-wabeled enrollment.

Following a successful interim.

We plan to begin transitioning the majority of <unk> sites to open label enrollment.

Speaker 6: and then shortly transition the subset of these sites to the raised food study.

And then shortly transmissions a subset of these sites to the race to study.

This will help support a smooth and rapid completion for raised two with.

Speaker 6: This will help support a smooth and rapid completion for race 2. With the goal of driving...

With the goal of driving improved timeline.

Speaker 6: We anticipate and roll in the first patient and raise two prior to the end of the end of the video.

We anticipate enrolling the first patients in res to prior to the end of the year.

Speaker 6: In addition to supporting EU regulatory filing, race-to-mell should provide an opportunity for expansion of the US wave.

In addition to supporting EU regulatory filing race through May also provide an opportunity for expansion of the U S label.

Speaker 6: We believe this study will serve a unique objective, which is to assess ibogaine exsalone in a broader RFE patient population.

We believe this study will serve a unique objective, which is to assess the hydrogen exelon and our broader RSV patient population.

Speaker 6: The importance of treating this earlier stage is supported by a recent study in Geometryology that emphasized the value of rapid stratification, showing the duration of independent predictors of subsequent neurologic disabilities.

The importance of treating earlier stage is supported by a recent study in Jama neurology and emphasize the value of rapid status location.

The duration of status as an independent predictor of subsequent neurologic disability.

Our research has shown that patients who had not progressed to IV anesthesia for a variety of reasons.

Speaker 6: Our research has shown that patients who would not progress to IV anesthesia for a variety of reasons

Instead receive ongoing treatment with second line IV Aedes also create a significant clinical and economic burden on health care system resources.

Speaker 6: Instead, receive ongoing treatment with second-line IBADs. Also create a significant clinical and economic burden on healthcare system resources. Create an important market opportunity.

An important market opportunity for IV.

Additionally, we continue to supply <unk> to physicians upon request under emergency <unk> for Super refractory status epilepticus.

Speaker 6: Additionally, we continue to supply IB-DN-X-Glombed to positions upon requests under emergency I&Ds for simple refractory status epilepticus or SRS-D.

Or src.

Speaker 6: Today, 21 patients have been treated for SRSE, including seven with a new dosing regimen developed specifically for this use. So far, results with a new reg...

To date 21 patients have been treated for FRC, including seven with a new dosing regimen developed specifically for this year.

So far results with the new regimen has been highly encouraging.

Because of its potential utility in treating srs or.

Speaker 6: Because of its potential utility and streaming SRS use, we're considering initiation of a proof of concept study with IDGN.

We're considering initiation of a proof of concept study with IV.

Investigators will present additional clinical data on patients treated with Blackstone for <unk> at the upcoming American Epilepsy Society meeting.

Speaker 6: Investigators will present additional clinical beta on patients treated with connectable for SRSC at the upcoming American epilepsy society meeting.

Speaker 6: I'd now like to discuss updates on our oral franchise, starting with CS.

I would now like to discuss updates on our oral franchise starting with PSP.

Seizures in TSV or Auckland treatment resistant despite the availability of newer disease specific anti seizure medications.

Speaker 6: Teachers and TSC are often treated resistant, despite the availability of newer disease, specific anti-seedrum medication.

Speaker 6: To address this unmet need, we're evaluating the maximum ESD patients with refractory disease.

To address this unmet need we're evaluating <unk> PSC patients with refractory seizures.

Speaker 6: This Phase 3 study, FUSTSC, is currently enrolling with top-line data anticipated in 2020.

This phase III study plus PSC is currently enrolling with top line data anticipated mid 2024.

As a reminder, we announced at our September Investor and analyst event Proto.

Speaker 6: our September anniversary. When the evening

Speaker 6: Go to call amendments of the trust CFC trial and final.

Protocol amendments and a trust PSP trial has been finalized.

The trials now anticipated to enroll 128 cases, which provides 90% power to detect a 25% reduction in TSA associated seizures.

Speaker 6: The trial now anticipated to enroll 128 citizens, which provides 90% power to detect a 25% reduction in PSA associated seizures.

Based on the blinded data the discontinuation rate in this trial remains low at approximately 10% only one treatment discontinuation to date attributed to the occurrence of tunnel.

Speaker 6: Based on the blinded data, the discontinuation rate in this trial remains low at approximately 10%, only one treatment discontinuation to date attributed to the occurrence of some.

This bolsters our confidence in the new titration schedule.

Speaker 6: This bolsters our confidence in the new titration schedule. And you can leave this new dosing paradigm to improve tolerability and therefore, potentially have a favorable spectrum efficacy.

We believe this new dosing paradigms and improved Tolerability, and therefore potentially have a favorable effect on efficacy as well.

A review of the baseline demographics to discuss PSP study provides some insight into the patients being enrolled.

Speaker 6: Our review of the baseline demographics in the CUSPST study provides some insight into the patients being enrolled.

Speaker 6: It's an extremely refractory population with a median baseline seizure rate of 52 per month. And with patients taking an average of 3.1 in common in anti-seizure medicine.

Extremely refractory population with a median baseline seizure rate of 52 per month.

With patients taking an average of three one concomitant anti seizure medications.

41% enrolled to date are taking afinitor and 25% are taking up a dialogue.

Speaker 6: 41% enrolled today are taking a finitour since 25% and taking up a dial.

Speaker 6: As a reminder, this will be the first study in TSC to evaluate a new anti-seizure treatment added to these medications.

As a reminder.

This will be the first study in PSC to evaluate a new anti seizure treatment added to these medications.

Speaker 6: Now, I'll turn to our 2nd generation product development. As we previously announced, we initiated enrollment in our multiple ascending dose or MAD study. With preliminary data from several cohorts expected by year end 2020.

Now I'll turn to our second generation product development as.

As we previously announced we initiated enrollment in a multiple ascending dose or Mad study.

With preliminary data from several cohorts expected by year end 2023.

Speaker 6: We're planning to finalize a clinical program for Lennon's guest O's syndrome in the first half of 2024 pending results for the Mad Drop.

We're planning to finalize our clinical program for loans guests both syndrome.

First half of 2024 pending results of the <unk> trial.

Speaker 6: If the data from the Mad Study are sufficiently informative, we could potentially move directly to a federal phase three step.

With the data from the Mad study are sufficiently informative, we could potentially move directly to a pivotal phase III study.

Speaker 6: You believe this new formulation would be the future of the Royal Fransize. And we'll continue to target its use in patient suffering from LGS and other refractory epilepsy.

We believe this new formulation can be the future of the oil franchise and will continue to target a juice in patients suffering from lgs and other refractory epilepsy.

Speaker 6: We saw a meaningful improvement in pharmaceutical networks in our single eighth-ending dose trial. And I hope that the Mad Study will...

We saw a meaningful improvement in pharmacokinetics and our single ascending dose trial.

Hopeful that the Mad study will reinforce our belief that the second generation formulation, we will have pharmacokinetic properties, allowing twice a day administration.

Speaker 6: second-generation formulation will have pharmacokinetic properties allowing twice a day of administration and will provide physician's the ability to individualize doses. This is a crucial aspect of seizure management and treatment reason for that.

We will provide physicians with the ability to individualized dosing.

This is a crucial aspect of seizure management and treatment resistant epilepsy.

Speaker 6: Also, we're pleased to share that all seven of our submitted abstracts were accepted as poster presentations at the upcoming American Epilepsy Society annual meeting. It will take place in Orlando from December 1st to 5th.

Also we're pleased to share that all seven of our submitted abstracts were accepted as poster presentations at the upcoming American Epilepsy Society annual meeting that will take place in Orlando from December one for Phil.

Speaker 6: Among these will be a post-depressantation from the open label extension of the Marigold study with seniors and patients follow-up, which will show data supporting the durability of effective minimum form, in treating refractory seizures associated with CDD.

Among these will be a poster presentation from the open label extension of the Marigold study with two years of patient follow up.

Which will show data supporting the durability of effect of the milestone in treating refractory seizures associated with CBD.

Speaker 6: We've recently begun to analyze data from patients with three years of open label follow up. And our preliminary review shows that geyser reductions are stable over this longer duration of treatment and are consistent with outcomes at three years.

We've recently begun to analyze data from patients with three years of open label follow up and our preliminary review shows that seizure reductions are sustainable over this longer duration of treatment.

Instant with outcomes at two years.

We'll be hosting a scientific exhibit at aes, highlighting the breadth of our <unk> Research program and we hope to see many of you there.

Speaker 6: We'll be hosting a scientific exhibit at AES highlighting the breadth of our Gnactual and research programs. And we hope to see many of you.

Speaker 6: To conclude, we have a number of key data announcements in the next year, and we remain resolute in our goal to successfully advance our ongoing clinical trials and develop new treatment options to help patients and families suffering from severe refractory seizures.

To conclude we have a number of key data announcements in the next year and we remain resolute in our goal to successfully advance our ongoing clinical trials and develop new treatment options to help patients and families.

For instance, severe refractory seizure disorders.

I'd now like to turn the call over to our CFO and CEO, Steve <unk>, who will provide you with a financial update thank.

Speaker 6: I'd now like to turn the call over to our CFO and CEO , Steve Pansteel, who will provide you with a financial up.

Speaker 7: Thanks, Joe. Thank you, morning everyone. I am pleased to be able to share our financial results of the third quarter of 2020.

Thanks, Joe and good morning, everyone I am pleased to be able to share our financial results for the third quarter of 2023.

Speaker 7: We ended the third quarter with cash, cash equivalence, and short term investments of 176.4 million, which is expected to provide cash runway into the fourth quarter of 2024.

We ended the third quarter with cash cash equivalents and short term investments of $176 4 million, which is expected to provide cash runway into the fourth quarter of 2020 for the.

Speaker 7: The catch balance includes net proceeds of 25.9 million from the sale of 3.7 million shares through our ATM facility in the quarter.

The cash balance includes net proceeds of $25 9 million from the sale of $3 7 million shares through our ATM facility in the quarter.

Speaker 7: Turning to our revenue and operating guidance. As Christy mentioned in her update, we experienced another strong quarter of the Tommy sales. Therefore, we are increasing our totaling annual guidance again with net revenues now projected to be in the range of 18.5 to 19 million, which represents an increase of 1.5 million on the lower end and 0.5 million on the higher end of the prior guidance.

Turning to our revenue and operating guidance as Christie mentioned in her update we experienced another strong quarter of the Tommy sales.

Therefore, we are increasing or its a Tommy annual guidance again with net revenue is now projected to be in the range of 18 $5 million to $19 million, which represents an increase of $1 5 million on the lower end and <unk> 5 million on the higher end of the prior guidance range.

For BARDA revenue, we continue to expect revenues to be in the range of $11 million to $12 million.

Speaker 7: For Barter Revenue, we continue to expect revenues to be in the range of 11 to 12 million.

Speaker 7: We project our gap operating expenses, inclusive of FSCNA and R&D expenses, being the range of 158 to 152 million, of which we expect approximately 15 million to be non-cash stock-based compensate.

We project, our GAAP operating expenses inclusive of SG&A and R&D expenses to be in the range of $158 million to $162 million of which we expect approximately $16 million to be noncash stock based compensation.

Speaker 7: This is a reduction from our prior guidance of 160 to 165 million and is driven by ongoing efforts to carefully manage cost and prudently invest in systemic commercialization and our ongoing phase three trial.

This is a reduction from our prior guidance of $160 million to $165 million and is driven by ongoing efforts to carefully manage costs and prudently investments atomic commercialization and our ongoing phase III trials.

I'll now take a few minutes to summarize our financial results.

For the third quarter of 2023, we recognized product revenues of $5 4 million and 13 million for the three and nine months ended September 32023, as compared to <unk> 6 million for each of the same period in the prior year.

Speaker 7: For the third quarter of 2023, we recognize product revenues of $5.4 million and $13 million for the three and nine months and its December 30, 2023 as compared to $0.6 million for each of the same periods in the prior year.

Speaker 7: These revenues consist of the Tommy product sales, which we launched in the third quarter of 2022.

These revenues consist of the Tommy product sales, which we launched in the third quarter of 2022.

Speaker 7: Separately, we recognize voter revenues of 1.9 million and 10.8 million for the three and nine months and it's September 30th, 2023 as compared to 1.8 million and 5.1 million for the same periods in the prior years. The increases driven primarily by activity associated with startup of our API on during

Separately, we recognize barter revenues of $1 9 million and $10 8 million for the three and nine months ended September 32023, as compared to $1 8 million and $5 1 million for the same periods in the prior year.

Increase was driven primarily by activity associated with startup of our API Onshoring initiative.

Research and development expenses were $23 7 million and 73 million for the three and nine months ended September 32023, as compared to $19 million and $58 5 million for the same periods in the prior year.

Speaker 7: We search and development expenses were 23.7 million and 73 million for the three and nine months ended September 30th, 2023. As compared to 19 million and 58.5 million for the same period in the prior year.

So year to date change was due to increased costs associated with our API ongoing effort increased DSD in RSV clinical trial activity and increased head count.

Speaker 7: The year-to-day change was due to increased cost associated with our API on-scoring efforts, increased DSV and RFC clinical trial activity, and increased set-down.

Speaker 7: As a reminder, the API-on-trowing effort is approximately 70% funded by Barta for the increased in R&D expenses, is partially offset by the increased part of revenues in 2020.

As a reminder, the API onshoring effort is approximately 70% funded by BARDA. So the increase in R&D expenses is partially offset by the increased part of revenues in 2023.

Speaker 7: Selling general and administrative expenses were 14.9 million and 45.8 million for the three of nine months ended September 30th 2023. I've compared to 13.4 million and 42.2 million for the same periods in the prior year.

Selling general and administrative expenses were $14 9 million and $45 8 million for the three and nine months ended September 32023, as compared to $13 4 million and $42 2 million for the same periods in the prior year.

Speaker 7: The primary drivers of the change in a year-to-day basis were increased pet count related to the US launch of the Tommy.

Primary drivers of the change on a year to date basis were increased head count related to the U S launch of the Tommy.

Speaker 7: Cancer Stincound was 1.9 million and 5.4 million to the three and nine months ended for September 30th, 2023, as compared to 0.5 million and 0.6 million for the same periods in the prior year.

Interest income was $1 9 million and $6 4 million for the three and nine months ended September 32023, as compared to zero point $5 million and <unk> 6 million for the same periods in the prior year.

Speaker 7: The increase in interest in town was driven by the overall increase in cash, cash equivalents, and short-term investments that increased yield on those bounds.

The increase in interest income was driven by the overall increase in cash cash equivalents and short term investments and increase the yield on those balances.

Interest expense was $4 2 million and $12 6 million for the three and nine months ended September 32023, as compared to $2 6 million and $7 million for the same periods in the prior year.

Speaker 7: Interest expense was 4.2 million and 12.6 million. So the three and nine months ended September 30th, 2023, that's compared to 2.6 million and 7 million for the same periods in the prior year.

Speaker 7: The increase is driven by drawdown of an additional 30 million of credit under the Oak Tree Agreement in March 2022 upon FDA approval for Satole and non-Cash interest expense related to our revenue interest financing with the Guard.

The increase was driven by drawdown of an additional $30 million of credit under the <unk> agreement in March 2022, upon FDA approval for the Tommy and noncash interest expense related to a revenue interest financing with regard.

Speaker 7: The company reported and that lost the foreign taxes of 33 million and 101.2 million for the three and nine months ended September 30th, 2023 has compared to net income before taxes of 75 million and 16.2 million for the same periods in the prior year. As a reminder, the prior year results included the one-time sale of our priority review voucher in the third quarter.

The company reported a net loss before income taxes of $33 million and $101 2 million for the three and nine months ended September 32023, as compared to net income before taxes of $75 million and $16 2 million for the same periods in the prior year.

As a reminder, the prior year results included the one time sale of our priority review voucher in the third quarter.

These totals include noncash stock based compensation expense of $4 million and $11 6 million for the three and nine months ended September 32023, as compared to $3 9 million and $11 1 million for the same periods in the prior year.

Cash used in operating activities was $91 million for the nine months ended September 30 of 2023 as compared to cash used in operating activities of $91 million for the same period in the prior year.

Speaker 7: Cash used in operating activities was $91 million for the nine months ended September 30, 2023, as compared to cash used in operating activities of $91 million for the same period in the prior year.

Before we move to Q&A I will make a few concluding remarks.

Speaker 7: Before we move to Q&A, I will make a few concluding remarks. We are making meaningful progress in a number of areas, including strong tatami sales growth, progress in expanding global access to tatami, continue the advancement of our oral and IV GANACS on clinical programs, and ongoing development of our second-generation product.

We are making meaningful progress in a number of areas, including strong <unk> sales growth progress in expanding global access because the Tommy.

Continued advancement of our oral and IV can axon clinical programs and ongoing development of our second generation product.

Speaker 7: As a result of these efforts, 2024 promises to be an exciting year for Marinus with 2D data readout to expect.

As a result of these efforts 2024 promises to be an exciting year for <unk> with two key data readouts expected.

Speaker 7: We look forward to seeing those of you attending the AES conference or one of the other investor or scientific conferences we will be attending in the coming months.

We look forward to seeing those of you attending the Aes conference or one of the other investor or scientific conferences, we will be attending in the coming months.

Speaker 7: Thanks again for your continued interest in Marinus. Operator, you may now open the call to question.

Thanks again for your continued interest in Mariner.

Operator, you May now open the call to questions.

Thank you if you ask a question. Please press star one on your telephone keypad, if you wish to remove yourself from the queue simply press star one again.

Speaker 1: Thank you. If you have a question, please press star one on your telephone keypad, and if you wish to remove yourself from Q simply press star one again. Again, we do ask that you refrain and keep yourself restricted to one question, you make you up for a follow-up.

When we do ask that you refrain and keep yourself restricted to one question you may queue up for a follow up.

One moment. Please for your first question.

Your first question comes from the line of Brian Abrahams of RBC capital markets. Your line is open.

Speaker 1: Your first question comes from the line of Brian Abraham's of RBC Capital Markets. Your line is over.

Speaker 8: Hey, guys. Good morning. Thanks for taking my question. Congrats on all the progress. On the RAISE study, can you talk about some of the ways you could potentially reaccelerate enrollment there without compromising the stringent enrollment criteria and the study conduct that you have? And curious your level of confidence and the potential to complete the interim cohort by the end of the first quarter and read out second quarter. And then, we'd love for you to maybe just quickly comment on what you're seeing in terms of the Talmid trends, the types of patients getting on, as that seems to be, that growth there seems to be accelerating even since September . Thanks.

Oh, Hey, guys. Good morning, Thanks for taking my question Congrats on all the progress.

<unk> study can you talk about some of the ways you could potentially reaccelerate enrollment there without compromising the stringent enrollment criteria in the study conduct that you have and curious your level of confidence in the potential to complete the interim cohort.

By the end of the first quarter and readout second quarter, and then would love. If you could maybe just quickly comment on what youre seeing in terms of the Tommy trends the types of patients getting on as that seems to be the growth there seems to be accelerating even since September. Thanks.

Thanks, Brian I will let me take <unk> question I'll pass it over to Joe If he has any other comments and then I'll pass over to Christie for her additional commentary on Tommy.

Speaker 3: Thanks, Brian . I will, let me take the raise question. I'll pass it over to Joe. He has any other comments and then I'll pass over to Christie for her additional commentary on the Talmie.

Speaker 3: I think we have seen with the raised trial as inflows of this study without question we have. And I've said this to lots of investors, we have weeks and months where we have very high enrollment and we have weeks and months that are still lower on a monthly basis for enrollment. We've got about three times the active number of sites.

Thank you.

We have seen with the raise trial ebbs and flows of this study without question, we have and I've said this to lots of investors, we have weeks and months, where we have very high enrollment and we have weeks or months that are still lower on a monthly basis for enrollment.

We've got about three times the active number of sites.

Speaker 3: recruiting patients looking for patients as we did earlier in the year. And I could not be prouder of the team the way they've really energized sight across the country within Canada and Australia. And we're modeling today really enrollment rates that are very much aligned with the first half of the year even though we have two to three more times of many sites up and running.

Recruiting patients looking for patients as we did earlier in the year.

Could not be prouder of the team and the way they really energize sites across the country within Canada, and Australia, and we're modeling today really enrollment rates that are very much aligned with the first half of the year, even though we have two to three more.

Times as many sites up and running in terms of actually accelerating from here and we are still confident that these sites are all playing.

Speaker 3: In terms of actually accelerating from here, we are still confident that these sites are all playing an active role in screening and those patients will...

An active role in screening those patients will will those sites will deliver patients and we still believe that the rate of enrollment can incrementally.

Speaker 3: Those sites will deliver patients and we still believe that the rate of enrollment can incrementally move ahead, but we wanted to be more conservative in our estimates to make sure that we did not have to move timeline. So I would say we feel very confident about more conservative timelines. We don't want to have to move them again. It's disappointing for us and I know it's disappointing for investors.

Move move ahead, but we wanted to be more conservative in our estimates to make sure that we did not have to move timeline. So I would say, we feel very confident about more conservative timelines, we don't want to have to move them again, it's disappointing for us and I know its disappointing for investors.

Speaker 3: But I will say I think the team has continued to be incredibly stringent in terms of the enrollment per se. And I think our last protocol amendment when we added language around only in rolling patients who are who require IVAN aesthetics, that did narrow the pool. I think we initially thought we'd have a placebo rate.

I will say I think the team has continued to be incredibly stringent in terms of the enrollment per se.

And I think with our last protocol amendment, when we added language or AUM only enrolling patients who are who require IV anesthetics that did narrow the pool.

We initially thought we'd have a placebo rate.

Speaker 3: of about 30% consistent with about a third of patients who would ultimately not go to IV anesthesia. And I think we feel today that <expletive> Burrade's gonna be much lower because of the more stringent criteria. And maybe to this discussion, Brian , we've really overpowered the study from where we started to where we are today. But that gives us extremely high confidence on the study stopping at the interim. Joe, any comments you wanna add there? And then we'll pass it to Chris here.

Of about 30% consistent with about a third of patients who would ultimately not good IV anesthesia and I think we feel today that placebo rate is going to be much lower because of the more stringent criteria and maybe to this discussion Brian we've really overpowered. The study from where we started to where we are today, but.

That gives us extremely high confidence on this study stopping at the interim Joe any comments you want to add there and then we'll pass it to Christy after that.

Speaker 6: Yeah, no, I'm also very confident about the enrollment. We're still, you know, as I mentioned, you know, we have high contact with sites. We're planning some new enrollment initiatives. We find that when patients, when the sites, you know, receive some motivational messages, we're gonna, you know, try to get messages out there to keep the study top of mind. As we finish up, you know, coming toward the end of enrollment, you typically see, you know, sites.

Yes, no I'm also very confident about the enrollment we're still.

As I mentioned, we have high contact with sites.

Planning, some new enrollment initiatives, we find that when patients when.

The sites.

Received some motivational messages we're going to.

Trying to get messages out there to keep the study top of mind as we finish up coming towards the end of enrollment you typically see sites.

Suddenly pick things up when they know that.

Speaker 6: Suddenly pick things up when they know that the time to enroll is limited. We hope to see that here, but we're maintaining our contact with the site. And the ebbs and flows, we expect to see things pick up again. Very confident about that. So I'm fully confident we're going to get to the enrollment target by the time we said.

The time to enroll is limited we hope to see that here, but we are maintaining our contact with the sites.

And the ebbs and flows we expect to see things pick up again.

Very confident about that so.

I am fully confident we're going to get to the enrollment target by the time, we said we would.

Speaker 3: And just the last thing I'll add Brian to Joe's comments is we're seeing substantially higher screening it.

Yeah, and just the last thing I'll add Brian to Joe's comments is we're seeing substantially higher screening activity and typically screening in the study has led to about a 50% enrollment rate and I would just say anecdotally in the month of October for example that screening rate was lower than 50.

Speaker 3: And typically screening in the study has led to about 50% enrollment rate. And I would just say anecdotally in the month of October , for example, that screening rate was lower than 50%. So I think that's just the ebbs and flows of the patients, but the activity level is extremely high, which is different than what we were seeing earlier.

So I think thats, just the ebbs and flows of the patients but the activity level is extremely high which is different than what we were seeing earlier in the year, so the sites or engage their screening.

Speaker 9: So the sites are engaged, they're screening, and just happen to be the last few weeks we've had...

And just happened to be the last few weeks, we've had more patients respond to standard therapy or not.

Speaker 9: more patients respond to standard therapy or not specifically getting enrolled. And I think that's important and gives us a lot of confidence in our...

Specifically, yet in the World and I think that's important and gives us a lot of confidence in our projections, sorry, sorry to cut you off there Joe Chris do you want to jump in on answer Tony.

Speaker 3: Sorry, sorry, cut your off, sir, Joe. Chris, you wanna jump in on, on the Tommy?

Speaker 4: Absolutely. Good morning, Brian . And thanks for the question. We as a team have been so encouraged with the meaningful progress that we continue to have. The types of patients that you mentioned continue to say the same. However, that's very, very broad. We see very, very young and we have seen some young adults also come into our enrollment progress. But what we have learned very significantly is that this brand is very promotionally responsive.

Absolutely good morning, Brian and thanks for the question.

We as a team has been so encouraged with the meaningful progress that we continue to have.

Types of patients that you mentioned continuing to stay the same however that is very very broad.

We see very very young and we have seen some young adults.

Also comments, you'll iron enrollment progress, but what we have learned very significantly that this brand is very promotional late responses.

Speaker 4: So what we've done, we spoke a little bit about last quarters. We have expanded our reach into the community. Our CIEWE's continued to be kind of central to our communication and...

So what we've done we spoke a little bit about last quarter as we have expanded our reach into the community. Our coes continued to be kind of central to our communication and.

Speaker 4: and our promotion, but then understanding that they work very closely with the community as well to get patients on therapy.

And our promotion, but then understanding that they work very closely with the community as well to get them.

Patients on therapy now, we also talked a little bit last quarter about additional ICD 10 leads that we have we have invested an additional set of data to get that reach even further and so that data continues to grow and then that just triangulate back to how we are moving our promotion and targeting physicians and caregivers even.

Speaker 4: Now, we also talked a little bit last quarter about additional ICB 10 leads that we have, we have invested in additional steps of data.

Speaker 4: to get that reach even farther. And so that data continues to grow. And then that just triangulates back to how we are moving our promotion and targeting positions and caregivers even farther. So we have a lot that we're doing and it's working. And again, we're just really, you know,

So there's a lot that we're doing and it's working.

And again, we're just really.

Zero down on the fact that this is a very very responsive market not only with physicians and caregivers and we continue.

Speaker 4: zeroed in on the fact that this is a very, very responsive market, not only with physicians and caregivers and we continue to to make sure that we are meeting these patients where they are and delivering what we need to.

To make sure that we are meeting these patients where they are and delivering what we need to.

Your next question comes from the line of Joseph till May of TD Cowen. Your line is open.

Speaker 1: Your next question comes from the line of Joseph Tome of Kidi Cowan. Your line is open.

Speaker 10: Hello there, good morning. Thank you for taking my question. Maybe just in terms of the enrollment, are you seeing certain sites are very strong and more important?

Hello. Good morning, Thank you for taking my question maybe.

Maybe just in terms of the enrollment or are you seeing certain sites are very strong in motors that you think you can rely on over the next quarter.

Speaker 10: and rely on it over the next quarter. Are there others that maybe have a high level of screen failure rates meaning that they don't really?

Are there others that maybe have a high level of screen failure rates, meaning that they don't really maybe get the criteria I guess is there a bias in the sites that you are enrolling.

Speaker 10: maybe get the criteria, I guess is there bias in the site that you're enrolling? I mean, you're some of these initiatives that you mentioned aimed at kind of, you know, increasing contact there. And maybe just a quick one on the basic database, how many patients above that 82 would you need for a filing? If any, thank you.

In the year. Some of these initiatives that you mentioned aimed at kind of.

Increasing contact there so maybe just a quick one on the.

Take your database, how many patients about that 82 would you need for profiling if any thank you.

Thanks, Joe Let me, let me say over the last question. Once we have the interim analysis, we will go to the FDA.

Speaker 3: Thanks Joe. Let me start with the last question. Once we have the interim analysis, we will go to the FDA and really have a discussion on the actual number of patients they would like to see.

Really haven't discussion on the actual number of patients they would like to see.

Speaker 9: on a drug. Certainly I'm a believer that this is always a function of risk reward.

On drugs, certainly I'm a believer that this is always a function of risk reward I'll, let Joe talk about what we've seen in a blinded fashion in terms of safety, but I think we feel very good about the safety profile.

Speaker 9: I'll let Joe talk about what we've seen in a blinded fashion in terms of safety, but I think we feel very good about the safety profile. We've talked openly about not only continuing the double-blind portion during the interim analysis, but...

We've talked openly about not only continuing the double blind portion during the interim analysis, but then flipping the study over to an open label study. We've got raised two sites up and running which is also using the same dosing protocol as rate. So I think we're going to have more than an ample number of pace.

Speaker 9: then flipping the study over to an open label study. We've got raised two sites up and running, which is also using the same dosing protocol as raised. So I think we're gonna have more than an ample number of patients who are getting a raised regimen.

<unk>, who are getting a raise regimen at the time of filing certainly given the the efficacy and safety data that we'll have in the phase III in terms of the sites I think the thing that.

Speaker 9: After time of filing, certainly given the efficacy and safety data that we'll have in the phase three.

Speaker 9: In terms of the sights, the thing that I've been most encouraged about and really since the summer.

I have been most encouraged about and.

Really since this summer.

Speaker 9: We added 10 or 15, I would say new sites since the summer and many of those have been producing quite nicely in the study. So I think we have a substantially broader number of sites that are participating. It's been a long time for some of the bigger sites that have been in the study, we're asking them to clean their databases now and really finalize the patient. So we are working our study coordinators hard.

We added 10 or 15, I would say new sites since the summer in many of those have been.

Producing quite nicely in this study so I think we have a substantially broader number of sites that are participating it's been a long time for some of the bigger sites that have been in the study were asking them to clean their databases now and really finalize other patients. So we are working our study.

Coordinators hard.

Speaker 9: But to your question, I think we have more sites that are screening and following patients. And I don't look at sites that are screening and have screen failures as something they're doing wrong. I think it's just a function. They're actively looking for patients. They're following the patients are getting consent.

To your question I think we have more sites that are screening.

And following patients and I don't I don't look at sites that are screening and have screen failures is something they're doing wrong. I think it's just a function there theyre actively looking for patients. They are following the patients are getting consent and either of the patients are responding to second line therapy and I'll remind folks are.

Speaker 9: and either the patients are responding to second-line therapy or mine folks.

Speaker 9: Our data would suggest it's probably happening sometime between 12 and 24 hours, which I would argue is pretty mediocre medical care overall, or the patient can't be consented, or there's a reason that ultimately the patient is contraindicated for the study. The bad patient with high renal or malinducency, liver failure, et cetera. But we generally take

Data would suggest it's probably happening sometime between 12, and 24 hours, which I would argue was pretty mediocre medical care overall or the patient can't be consented or there is a reason that ultimately the patient is contraindicated for this study patients with high renal.

Renal insufficiency liver failure et cetera, but we generally take.

Speaker 9: screening at the good sign and many of our best sites will have screen failures. That's expected. I think just we've had a little less luck with turning them into enrollment this month. Joe, anything you want to add about sites?

Screening is a good sign and <unk>.

Many of our best sites will have screen failures that expected I think just.

We've had a little less luck with turning them into enrollment this month, Joe anything new you want to add about site.

Speaker 6: Now I just reiterate, the recent enrollment has come from newer sites, sites that have gotten up and running more recently.

No.

I'd just reiterate the recent enrollment has come from.

<unk> sites sites that have gotten up and running more recently.

Speaker 6: So, as you know, again, it's got mentioned screening is high, enthusiasm is high, and some patients just end up getting screened out. I think if we, you know, I'm very encouraged by the continued screening efforts, and if we keep doing that, we'll enroll the study. You know, as an any study, there's variability in how sites perform, but we're seeing a broader representation of sites actively in...

So as you know again as Scott mentioned screening is high enthusiasm is high and some patients just ended up getting screened out I think if we you know I'm very encouraged by the continued screening efforts and if we keep doing that will.

We will enroll the study.

It's an early study there is variability in house sites perform but we are seeing.

Broader representation of sites.

<unk> enrolling in the study and screening.

Speaker 9: And Joe, the last Joe Tomay, the last time in all make, is that I think it's critical that our team, our clinicians and our psych coordinators continue to really make sure that we're enrolling the right patients and we continue the high.

And Joe the last Joe tell me the last comment I'll make is that I think it's critical that our team our clinicians and our.

Our site coordinators continue to really make sure that we're enrolling the right patients and we continue the high.

Speaker 9: the high standards that we've had to date, that's the only way the study is going to win and have a decisive victory. So it's always a catch-22 in that regard, but I have all the respect in the world for our clinical team, and our physicians, and our MSLs who are out there educating, but not bending the rules to enroll patients just so we hit the numbers faster. That's always a down...

The high standards that we've had to date, that's the only way. The study is going to win and have a decisive victory.

So it's always a catch 22 in that regard, but I have all the respect in the world for our clinical team and our physicians and our MSL or out there educating.

But not bending the rules to enroll patients just so we hit the numbers faster. So that's always the balance when you run a study like this.

Speaker 6: You mentioned safety. Yeah, and neglected to mention the safety looks good. Consistent with the phase two, we're not seeing any new safety signals. So that's looking favorable.

And then Scott you mentioned Joe Yes.

Yes, it was collected dimension to say the safety looks good.

Consistent with the phase two we're not seeing any new safety signals.

So that's looking favorable.

Thanks, Joe.

Your next question comes from the line of jewelry of true Securities. Your line is open.

Speaker 1: Your next question comes from line of Junley of truth securities. Your line is open.

Speaker 5: Hey, thanks for taking your questions. I just want to confirm that at 82 patients, the study is 94% power to detect a 4% effect size of the cold primary end point. But you haven't disclosed the stopping criteria. And you know, has the stopping criteria changed over time as you look at the enrollment, which has been a little slower than expected? You know, given that if you don't...

Hey, Thanks for taking our questions I just wanted to confirm that was at 82 patients. This study is 94% power to detect a 40% effect size on the co primary endpoints and but you haven't disclosed the stopping criteria.

And has the stopping criteria changed over time as you.

Look at the enrollment, which has been a little slower than expected.

Given that.

You don't stop at interim that he could actually split maybe a material type point of the whole study to read out.

Got you thanks for the question.

Speaker 6: Yeah, thanks for the question. Yeah, that's absolutely right. 94% power to detect a 40% treatment difference. Actually,

Yes, that's absolutely right, it's 94% power to detect a 40% treatment difference actually.

Speaker 6: We could see statistical significance at a delta lower than that. Down in the range of 25%, we would still see statistical significance.

We could see statistical significance.

I'd add a delta lower than that.

Down in the range of 25%, we would still see statistical significance and the stopping criteria are statistical significance. When you do an interim analysis there is always a.

Speaker 6: and the stopping criteria are statistical significance. When you do an interim analysis, there's always a spend in the alpha that you have to do. And that works out actually to have, if we went to the end, it actually would have a minimal impact on the statistical power at the end of the study. But even with the...

Spend in the Alpha that you have to do.

And that works out actually to have.

To the end of it actually would have a minimal impact on the statistical power at the end of the study.

But even even with.

It's zero to 93.

Speaker 6: 0.0293 uh... and but that's that is and with that we have ninety four percent power to detect that forty

But thats that is even with that we have 94% power to detect that 40% Delta.

Speaker 6: So again, well powered at the interim, very confident.

So again well powered at the interim very confident about it.

And Joe the only all at June to be clear, we have not moved the goalposts at all on that I think we had very conservative assumptions going into the trial as I mentioned earlier not having a good handle on exactly what the placebo rate would be we considerably you thought about a placebo rate, 30% or higher.

Speaker 9: And Joe, the only I'll add, June to be clear, we have not moved the goalpost at all on that. I think we had very conservative assumptions going into the trial, as I mentioned earlier, not having a good handle on exactly what the Poseba rate would be. We conservatively thought about a Poseba rate 30% or higher. And that's clearly at least what we believe to be the case today, we're seeing a much lower Poseba rate.

And that's clearly at least what we believe to be the case today are much we're seeing a much lower placebo rate, which.

Speaker 9: Just in our minds gives us a lot more flexibility in terms of hitting statistical significance. But we have not.

And our minds gives us a lot more flexibility in terms of hitting statistical significance.

But we have not moved the goalposts at all in that regard.

Operator next question.

Your next question comes from the line of Charles Duncan of Cantor Fitzgerald. Your line is open.

Speaker 1: Your next question comes from a line of Charles Duncan of Cantor Fitzgerald, your line is old.

Hey, good morning, Scott and team congrats on the progress commercially I appreciate the comments on placebo rate in stringency for race as well as just continuation rate for trust PSC all good but I didn't want to talk about that I'm on I talked about the match program site.

Speaker 11: a morning shot and team congrats on the progress commercially. Appreciate the comments on placebo rate and stringency for raise, as well as just continuation rate for trust TSE. All good, but I didn't want to talk about that. I want to talk about the maps program. So I had a question on maps. I guess...

I had a question on <unk> I guess.

Speaker 11: I'm kind of wondering what was the driver for the MATS program? Was it patient or prescriber request or was it your opportunity to identify? And can you give us a sense of the financial turns that there have been? How much, you know, call it dollar of sales or dollar revenue will you retain? Thanks.

Kind of wondering what was the driver for it Matt.

Match program was it patient or prescriber requests or was it your opportunity identification and can you give us a sense of the financial terms with their event how much.

Call. It dollar of sales or a dollar of revenue will you retain thanks.

Yeah.

Speaker 9: Thanks Charles, really appreciate the question. Since the launch is atalmy, we routinely, more than once or twice a month, receive requests from patients, families, physicians outside the US about access to the atalmy.

Thanks, Charles really.

I appreciate the question since the launches have told me we routinely.

More than once or twice a month to receive requests from patients families physicians outside the U S about access to the told me.

Speaker 9: And so much of what we do is spending time with not only the IFCR in the U.S., but the CDK alliances in Europe and in other countries and our efforts.

So much of what we do is spending time with not only the <unk> in the U S, but the CDK alliances in Europe and in other countries and our efforts with <unk>.

Speaker 9: The Chinese CDKL5 Alliance, for example, has not only helped us understand just how big that, how many patients are in China, over 600 that have been identified and growing.

The Chinese <unk> fiber lines. For example has not only helped us understand just how big that how many.

Patients are in China over over 600 that have been identified and growing and certainly has driven.

Speaker 9: and certainly has driven the orphan disease status and the priority review from the Chinese government. So we are in constant discussions with our alliance groups around the world, and I think it's critical for us to help provide the drug to those patients' families.

The orphan disease status and the priority review from the Chinese government. So we are in constant discussions with airlines groups around the world and I think it's critical for us.

To help provide the drug to those patients families.

Speaker 9: and advocacy groups. So first and foremost, that was our goal here. Secondly, I think we're in a position now with our supply that we can really think about a global expansion. I mean, that's a critical piece of this.

And advocacy groups, so first and foremost that was our goal here.

Secondly, I think we're in a position now with our supply that we can we can really think about a global expansion I mean, that's a critical piece of this.

Speaker 9: We're very comfortable with the two-year shelf life and all of those processes get a little bit more complicated as you go global. So I think we've made tremendous progress in our supply chain and our comfort level of supplying drugs.

Very comfortable with the two year shelf life and.

Those all of those processes get a little bit more complicated as you go global So I think we've made tremendous progress in our supply chain and our comfort level.

Supplying drug and I think we've had a lot of inbound interest in that regard from companies to partner with.

Speaker 9: I think we've had a lot of imbalance interest in that regard from companies to partner with. And so maybe TJ, I'll flip it over to you and Steve, if you have any comments to talk a little bit more about the economics and how we're thinking about price.

And so maybe TJ I'll flip it over to you and Steve If you have any comments to talk a little bit more about the economics and how we're thinking about pricing.

Thanks, Scott and thanks, Charles for the question.

Speaker 5: Thanks, Scott, and thanks, Charles for the question. We are, they're mentioning in the both the release and in the conversation today, has a lot of experience in the space, and EAP. And we're relying on their guidance on the pricing, where we anticipate both also where between EU pricing and project for the EU pricing and US pricing, thought on a country by country basis.

We are Jeremy as you mentioned and that's both the release and in the conversation today is a lot of experience in this space.

And we're relying on their guidance on the pricing.

Anticipate will fall somewhere in between.

EU pricing.

<unk> pricing U S function.

On a country by country basis so.

So.

That's our pricing.

Speaker 5: If the eye for your moment, you have to be finalized. And with respect to the proportion that we retain, it would be, it would be significant in majority of the top 100 net expenses.

Exactly at the moment, yet to be finalized and with respect to the proportion that we retain it would be.

It would make a significant majority of the top line.

Net transaction fees from Durbin.

Charles This is Steve.

Speaker 7: Your name is Dr. Shanty. I would just add that, you know, Derbys and Important Partner, but they're really administering the program on our behalf. You know, we aren't disclosing the fees in detail here, but they make up a, you know, a low percentage of the overall revenue.

I would just add that Durbin is an important partner, but theyre really administering the program on our behalf.

We aren't disclosing the fees.

In detail here, but they make up a low percentage of the overall revenue as expected.

Speaker 1: The next question comes from a line of Mark Goodman of Lereng Partners, your line is open.

Your next question comes from the line of Marc Goodman, <unk> Leerink partners. Your line is open.

Yeah with respect to so Tony I was wondering if you could just give us a flavor for the gross to net in the quarter and how to think about it into next year and now that you've been out there a while can you give us a sense of the number of patients that you think are truly they're the CTD patients what what's the what's the big number there that we can get.

Speaker 12: Yeah, with respect to the Tony I was wondering if you could just give us a flavor for the gross to net in the quarter and how to think about it into next year. And now that you've been out there a while, can you give us a sense of the number of patients that you think are truly there, the CDD patients, you know, what's the?

Speaker 12: What's the big number there that we can get to? It's reasonable. You know where the patients are and you feel pretty good that those are attainable. Are you seeing any off label in the 140? Thanks. Thanks, Mark Steve.

That's reasonable.

Where the patients are and you feel pretty good that those are attainable.

Are you seeing any off label in the 140.

Thanks, Mark Steve you want to start with gross to nets.

Yeah sure happy to happy to share a little bit of detail. There I think the gross to net deductions have been pretty consistent with our expectations. We've always said around 20%, it's actually been a little less than 20% over the course of the year on a year to date basis, we do see that the gross to net deductions coming down slightly.

Speaker 7: Yeah, sure. You know, happy to share a little bit of detail there. I think the grossennet deductions have been pretty consistent with our expectations. We've always said around 20%. It's actually been a little less than 20% over the course of the year on a year-to-date basis.

Speaker 7: We do see that the growth and ed deductions coming down slightly. We have about 50% of our patients on Medicaid as their primary, the other 50% commercial. But of that 50% of commercial, about 25% have secondary Medicaid access. As they get through their deductibles that Medicaid secondary drops off, and we see a little benefit from a growth to net. But they've been really consistent just below about 20%.

About 50% of our patients on Medicaid as their primary the other 50% commercial but of that 50% of commercial about 25% have secondary Medicaid access.

As they get through their deductibles that Medicaid secondary drops off and we see a little benefit from a gross to net but.

<unk> been really consistent just below about 20%.

Mark Lee, let me make one or two comments for you.

Speaker 9: More clearly, let me make one of your comments for a Passover to Christy to give you a little more color. I think we've always said we believe there are about a hundred newborns a year, about 2000 pediatric patients.

Pass over to Christie to give you a little more color I think we've always said we believe there are about 100 newborns a year about 2000 pediatric patients.

Speaker 9: The third party hospital, the third party ICT 10 codes, certainly are suggesting that there's well north of 800 patients today and that number is growing. And remember, this code's only been around for three or four years.

The third party hospital other third parties.

ICD 10 codes, certainly are suggesting that there is well north of 800 patients today and that number is growing and remember this code has only been around for three or four years I will tell you. We still know many of our centers of excellence still don't use the ICD 10 codes. So we certainly know there are more than that number.

Speaker 9: I will tell you we still know many of our centers of excellence still don't use the ICD-10 codes. So we certainly know there are more than that number of patients out there coming into the population. I would say the biggest thing we've seen.

Patients out there.

Moving into the population I would say the biggest thing we've seen is a nice that that's different from the time <unk> launches, we would have seen in adults being treated for the disease.

Speaker 9: is a knife that's different from the time of launch is we'd have seen adults being treated.

Speaker 9: for the disease, which has been a nice upside to how we thought about the market. So we certainly believe the market.

Which has been.

Nice.

Upside to how we thought about the market. So we certainly believe the market potential is larger given the fact that we're seeing adult neurologists treat patients and we did it originally Christy maybe I'll pass over to you for any additional thoughts.

Speaker 9: Potential is larger given the fact that we're seeing adult neurologist treat patients and we did it originally Chrissy, maybe I'll pass over to you for

Speaker 4: Yeah, thanks Scott and Mark thanks for the question Scott makes really great points there.

Yes, Thanks, Scott and Mark Thanks for the question Scott makes really great points there.

Speaker 4: So again, just to kind of zero in on it, we really believe that this 2000 addressable pediatric patients is very achievable.

So again just to kind of zero in on it we really believe that this 2000 addressable pediatric patients.

Very achievable and our continued progress really underscores that.

Speaker 4: and our continued progress really underscores that, with the fact that there are between 75 and 100 newborns a year, and we do not yet see that reflected in the ICT 10 codes. Now we know that we need to continue to drive that message forward with the usage of the code. I think old habits are hard to break for some physicians, and then we just continue with that method.

With the fact that there are between 75 and 100 newborn into year and we do not yet see that reflected in the ICD 10 codes now we know that we need to continue to drive that message forward looking.

With the usage of the code.

<unk>.

Old habits are hard to break for some physicians and then we just continue with that message.

Speaker 4: Another thing that continues to drive that number upward is not only our promotional efforts with genetic testing, but the AES also has a very, very large drive now to promote genetic testing not only in newborn patients, but in maybe those who have

Another thing that continues to drive that number upward and not only our promotional effort with genetics testing, but.

<unk> also has a very very large drive now to promote genetic testing not only in newborn patient, but and maybe that is who has a symptomatic diagnosis.

Speaker 4: a symptomatic diagnosis that don't have a diagnosis yet from a genetic cause of their epilepsy. So all of these things, not only what we're doing, but even in the epilepsy community are driving, you know, larger diagnoses of patients, which will help support not only just the 2000 addressable pediatric patients, but then again, maybe some older patients as well.

Have a diagnosis yet and from a genetic cause of their epilepsy. So all of these things not only what we're doing but even in the epilepsy community are driving larger diagnoses a patient search will help support not only just the 2000 addressable pediatric patients, but then again, maybe some older patients as well.

Your next question.

Speaker 1: Your next question comes from line of Andrew Tye of Jeffrey Jelinez.

Comes from the line of Andrew Tsai of Jefferies. Your line is open.

Speaker 13: Hey, thanks. Good morning. Appreciate all the updates. So maybe one more on the RSC.

Hey, thanks.

I appreciate all the update so maybe one more on the RSC.

Obviously the goal is to get to 82 patients by March of next year. So as we think about your.

Speaker 3: So as we think about your enrollment cadence, how should we think about the holiday period and November or December timeframe? Could enrollment flow down a little bit as people take time off or not necessarily? Or maybe set another way, where would you ideally like to be on enrollment by year and 2023? And then finally, could we expect you, when could we expect you to give you the next update on enrollment? Thank you. Yes, thank you. Let me start with the update, Andrew. I think we'll continue.

Enrollment cadence how should we think about the holiday period in November December timeframe could enrollment slowdown a little bit as people take time off are not necessarily or maybe said another way where would you ideally like to be on enrollment by year end 2023, and then finally can we expect U <unk>.

Can we expect you to give you the next update on enrollment. Thank you.

Yes.

Speaker 9: Let me start with the update Andrew. I think we'll continue the update. People certainly at JP Morgan would be the most logical time or the JP Morgan Healthcare Conference. The most logical time to update enrollment. Typically we've given a business update at that point in time. So we'll continue to share the enrollment numbers with you folks.

Let me just start with the update Andrew I think we'll continue to update people certainly at J P. Morgan would be the most logical time for the Jpmorgan Health care conference. The most logical time to update enrollment typically we'd given a business update.

At that point in time, so we'll continue to share the enrolment numbers with you folks.

Speaker 9: What we talked about for us to hit these enrollment numbers was fortify patients a month.

We had talked about or for us to hit these enrollment numbers was four to five patients a month.

Speaker 9: We felt very confident about that. With the current projections in our timing, we're really expecting three to four patients a month to get to the finish line for us to get. And so we try to be as conservative as we could in terms of that thinking.

Felt very confident about that with the current projections and our timing, we're really expecting 3% to four patients a month to get to the finish line.

For us to get and so we've tried to be as conservative as we could in terms of that thinking.

Speaker 9: At least my general view is that holidays weekend doesn't seem to have a big impact here. I would say last few weeks to months we've probably enrolled 40-50% of our patients on Saturday and Sunday. My wife used to call them Saturday night specials when I was in practice.

At least my general view is that holidays weekends doesn't seem to have a big impact here I would say the last few weeks to months, we've probably enrolled 40% 50% of our patients on Saturday and Sunday.

My wife used to call them Saturday night specials, when I was in practice.

Speaker 9: So I don't think we've seen a major difference in cadence around holidays. Certainly the weekends where you would think less staffing in the hospital would have an impact.

I don't think we've seen a major difference in cadence around holiday certainly the weekends, where you would think less staffing in the hospital would have an impact. So we're generally thinking about these these trends kind of having the good and bad weeks, but are going to smooth out to three to four patients per month, and that's going to get us to the finish line.

Speaker 9: So we're generally thinking about these trends kind of having their good and bad weeks, but are going to smooth out the three to four patients per month, and that's gonna get us to the finish.

Speaker 6: And as I said, we'll plan on that update. Jellie, do you want to add to that? No, I actually just remember New Year's Eve last year was a good time for enrollment. I don't think, yeah, this is different than a chronic outpatient.

As I said, we will plan on that update Joe anything you want to you want to add to that.

Actually just remember new year's Eve last year was a good time.

Im for enrollment.

I don't think this is different than a chronic outpatient study.

Speaker 6: And I don't think that we'd expect the holidays to have the same kind of impact that it has.

And I don't think that we'd expect the holidays to have the same kind of impact.

I'm a study like that.

Your next question comes from the line of Douglas Tsao of H C. Wainwright. Your line is open.

Speaker 1: Your next question comes from line of Douglas style of H.T. Rainwright. Your line is open.

Speaker 12: Hi, good morning. Thanks for taking the questions. I'm just curious in terms of the decision to discontinue the reset trial and you sort of obviously consider pursuing development.

Hi, good morning, Thanks for taking the questions I'm just curious in terms of the.

The decision to discontinue the reset trial.

And just sort of obviously consider pursuing development and <unk>.

Speaker 12: SRSC, is that driven by sort of the feasibility or just the sort of simplicity of how would fit in clinical practice? Maybe it's easier or just from a value proposition pricing standpoint. Thank you.

<unk>.

Is that driven by sort of stood at the feasibility or just the sort of simplicity of clinical how it would fit.

In clinical practice, maybe it's easier or just from a value proposition and pricing standpoint. Thank you.

Speaker 9: Yeah, thanks for the question Doug, really appreciate it. I think there's a lot of factors to play in here. I'll let Kristi comments from the commercial side, but I think overall the message that we're, you know, which I've always shared with investors, I already thought the ER opportunity would be a more complex one for us.

Yeah. Thanks for the question, Doug really appreciate it I think theres a lot of factors at play in here I'll, Let christi comments from the commercial side, but I think overall the message that we're in which Ive always shared with investors I always thought the opportunity would be more complex one for us.

Speaker 9: We need a lot of sales reps, they're higher pressures in the emergency room from a pricing standpoint. But I thought it was important for us to do this study to really start to understand dosing in a frontline setting. That being said, as we've done this study, the costs have just really gone up dramatically as we have to obtain community consent and there's a huge upfront cost.

We need a lot of sales reps there higher pressures in the emergency room from a pricing standpoint, but I thought it was important for us to do this study to really start to understand dosing in a frontline setting that being said as we've done this study.

The cost of just really gone up dramatically as we have to obtain community consent and there is a huge upfront cost.

Speaker 9: and then we can spend money for community consent and we may lose a site because they lose a coordinator or a physician lose territories and so I just looked at what we were spending we all did looked at what we were spending on the study and I was I was quite worried It was gonna just continue to grow

And then we can spend money for community concern and we may lose the site because they lose a coordinator or physician moves territories and so I just looked at what we were spending we all did looked at what we're spending on the study and I was quite worried it was going to just continue to grow.

Speaker 9: And we didn't have a great commercial vision for this aside of the business.

And we didn't have a great commercial vision for this side of the business. The flip side is that we continue to get lots of requests for a super refractory status, we've shared that data.

Speaker 9: The flip side is that we continue to get lots of requests for a super refractory status. We've shared that data a lot. We certainly think there is a regulatory path to expanding the label and getting to 63 grams of capitals. And we think it's a critically important and very meaningful business opportunity for us.

We certainly think there is a regulatory path to expanding the label and getting to 63 grams of Captisol and we think it's critically important and very meaningful business opportunity for us and I think as we sat down as a team and really strategize with our commercial team.

Speaker 9: And I think as we sat down as a team and really strategized with our commercial team and our senior MSL teams and we think about it, HEO or Health Economic Outcomes.

Our senior MSL teams as we think about it <unk> health economic outcomes.

Speaker 9: We recognize that we're going to have to do more in the space to really maintain our leadership and access to the drugs.

We recognize that we're going to have to do more in this space to really maintain our leadership and an access to the drug.

Speaker 9: So we really sat back as a team and said, we've got to spend our dollars wisely where we are today. It would be nice to continue the study, but given the cost of it, we think the dollars are going to be much more valuable.

We really sat back as a team and said we've got to spend our dollars wisely, where we are today it would be nice to continue the study, but given the cost of it. We think the dollars are going to be much more valuable, let's say in a call. It a phase 463 Gram captisol thousand milligram of good Exelon stuff.

Speaker 9: say in a call-it-a phase for 63 gram, capped as all, 1000 milligram, a good excellent study in SRC patients, one that physicians can understand, dosing, we can align with the FDA, and obviously we've seen a ton of interest given the number of patients that we've treated. So from a business standpoint, that's the way we're thinking about it. Chris, do you wanna add anything on the commercial side?

E S.

Or is he patients one that physicians can understand dosing, we can align with the FDA and obviously, we've seen a ton of interest given the number of patients that we've treated so.

From a business standpoint, that's the way we're thinking about it Chris you want to add anything on the commercial side.

Speaker 4: Yeah, thanks Doug. I think one thing that Scott mentioned was just the infrastructure that it would take for a promotional effort inside of the ER, which I think we could all say.

Yeah.

Thanks, Dan I think one thing that Scott mentioned was just the infrastructure that it would take for promotional effort inside of the AAR, which I think we can all say in pretty.

Speaker 4: pretty robust. And so, you know, who we are as Marinus is really focusing on an unmetacled lead and the total value proposition.

Pretty robust and so.

We are its narrowness Israeli focusing on an unmet medical need and the total value proposition and the Srs a population is quite large and what we mean by total value proposition.

Speaker 4: in the SRFD population, it's quite large. And what we mean by total value proposition is, you know, not only the economic value proposition, but the clinical value that we could potentially bring.

Not only the economic value proposition, but the clinical value that we could potentially bring is quite large and when we look at cost of care.

Speaker 4: It is quite large. When we look at costs.

Speaker 4: of care. Inside the hospital system and moving and progressing through status, it grows significantly as it gets to SRSD. And if we're focusing on it, nonmet medical needs, these patients are in dire need and these health care providers really need something that is effective for these patients. And we've been encouraged what Joe's team has shown us with some of the patients that have been treated and we know that these patients are.

Inside the hospital system, and moving and progressing through status. It grows significantly as it gets to us RSA and of course, focusing on non met medical need. These patients are in dire need in these health care providers really need something that is effective for these patients and we've been encouraged what Joe's team has shown us.

West none of the patients that had been traded in and we know that these patients are definitely responses.

Speaker 4: definitely responses and given circumstances. So again, looking at the economic and clinical value proper, these patients is quite large.

Given the circumstances and looking at the economic and clinical value prop for these patients is quite large.

Speaker 1: Thank you. That's all the time we have for questions today. I will now turn the call over to Scott for some closing remarks.

Thank you that's all the time, we have for questions today I will now turn the call over to Scott for some closing remarks.

Speaker 9: Well thanks everyone for dialing in, sorry that we couldn't get to all the Q&A today. I will openly admit that I'm a little bit under the weather and...

Well, thanks, everyone for dialing in sorry that we couldn't get to all the Q&A today I will openly admit that I'm a little bit under the weather.

Speaker 9: A little bit of a struggle for me to get through this call today, but appreciate you all dialing in and We are keeping our heads down and we'll be working to the finish line of both of these studies, which we're incredibly excited about

A little bit of a struggle for me to get through this call today, but I. Appreciate you all dialing in and we are keeping our heads down and we'll be working to the finish line on both of these studies, which we're incredibly excited about commercial team continues to do just a fantastic job and we didn't spend any time on it on the call today, but we will.

Speaker 9: Commercial team continues to do just a fantastic job and we didn't spend any time on it on the call today, but we will continue to expect to have data on our second generation program by year end. It's one that we're incredibly excited about as well. So thank you all for dialing in and we look forward to getting the finish line on these studies and we'll be in touch with you all shortly. Thanks so much. This concludes today's class. Thank you.

We continue to expect to have data on our second generation program by year end. It's one that we're incredibly excited about it as well. So thank you all for dialing in and we look forward to getting the finish line on these studies and we'll be in touch with you all shortly thanks so much.

This concludes today's conference call you may now disconnect.

Okay.

Speaker 9: Studies and will be in touch with you all shortly. Thanks so much.

These studies will be in touch with you all shortly thanks so much.

This concludes today's conference.