Q1 2020 Earnings Call

Greetings and welcome to the Meritas Pharmaceuticals first quarter 2020 earnings call. At this time all participants are listen only mode brief question and answer session will follow the formal presentation. If anyone should require operators system. During the conference. Please press star zero on your telephone keypad.

Operator: Greetings, and welcome to the Marinus Pharmaceuticals first quarter 2020 earnings call. At this time, all participants are in listen-only mode. A brief question and answer session will follow the formal presentation. If anyone should require operator assistance during the conference, please press star zero on your telephone keypad.

As a reminder, this conference is being recorded.

Operator: As a reminder, this conference is being recorded. It is now my pleasure to introduce your hosts, Sasha DeMoney Ellis, Vice President, Investor Relations. Thank you. You may begin, Ms. Ellis. Thank you.

And it's now my pleasure to introduce your host Sasa, the Monique Ellis Vice President Investor Relations. Thank you you may be getting the salad.

Thank you.

With me from Meritas are Dr., Scott Braunstein, Chief Executive Officer, Dr., Joe Houlihan, Chief Medical Officer, and Edward Smith, Chief Financial Officer.

Sasha Damouni Ellis: With me from Marinus are Dr. Scott Braunstein, Chief Executive Officer, Dr. Joe Hulihan, Chief Medical Officer, and Edward Smith, Chief Financial Officer. Before we begin, I would like to remind everyone that some of the statements made today could be termed as forward-looking under the securities law. These forward-looking statements, of course, are subject to certain risks and uncertainties that are associated with our business and covered in part in the companies' Form 10-K and 10-Q as filed with the Securities and Exchange Commission. I will now turn the call over to Scott.

Before we begin I would like to remind everyone that some of the statements made today could be termed as forward looking under the securities law.

These forward looking statements of course are subject to certain risks and uncertainties that are associated with our business uncovered in park and the company form 10-K and 10-Q.

And with the Securities and Exchange Commission.

I will now turn the call over to Scott.

Thank you saw show good morning, everyone and welcome to our first quarter Twentytwenty business and financial update.

Scott Braunstein: Thank you, Sasha. Good morning, everyone.

Scott Braunstein: And welcome to our first quarter 2020 business and financial update. As Sasha mentioned, today on the call, I have with me Dr. Joe Hulihan, Chief Medical Officer, who will provide updates on our clinical progress, and Ed Smith, our Chief Financial Officer, who will review our financial statements and the company's cash position. We will be available for questions at the end of the call.

As Sachin mentioned today on the call.

With me Dr., Joe Houlihan, Chief Medical Officer, who will provide updates on our clinical progress and Smith, our Chief Financial Officer, who will review our financial statement in the company's cash position.

We will be available for questions at the end of the call.

Scott Braunstein: Starting off today's call, I would like to acknowledge that this marks our first Earnings and Business Update Conference call. This is an important milestone that reflects the significant progress we have made and the evolution of Marinus to a company that is nearing pivotal trial data with the potential for both an NDA filing and a commercial launch. We have continued our momentum over the past few quarters with several milestones, including promising clinical data from our program in status epilepticus and important financing prior to year end. Additionally, we implemented a broader strategic clinical plan designed to unlock the potential of Gonadsalone across a range of rare sedor disorders and an encouraging interaction with the FDA that we will describe in more detail for you shortly. 2020 has already turned out to be an exciting time at Marinus, and we look forward to having our quarterly calls as an opportunity to keep our shareholders up-to-date and well-informed. I'd like to take a moment to acknowledge the ongoing COVID-19 pandemic.

Starting on today's call I would like to knowledge that these this marks our first earnings and business update conference call.

This is an important milestone that reflects the significant progress we have made and the evolution of Meredith to a company that is nearing pivotal trials either with the potential for both and engineering piloting and commercial launch.

We continued our momentum over the past few quarters with several milestones.

Including promising clinical data from our program that is once again.

An important financing prior to your and.

The implementation of the broader strategic clinical plan.

Turning to unlock the potential okay, that's all loan across a range of where she or disorders.

It is encouraging interaction with the idea that we will describe in more detail for you shortly.

Twentytwenty has already turned out to be an exciting time, it Meredith and we look forward to hobbies or quarterly calls as an opportunity to keep our shareholders, Judy and well informed.

I'd like to take a moment to acknowledge the I'm going Kobin 19, and then.

Scott Braunstein: As Marinus, we have been fortunate that as of today, we have avoided the major impacts and delays that have been so challenging for many of our colleagues across the life sciences. We are encouraged that our upcoming clinical milestones, which include top-line data from our phase three pivotal trial in CDKL5 deficiency disorder, or CDD, and the initiation of our phase two trial in tuberous sclerosis complex, or TSC, remain on track. I am grateful for the continued commitment and dedication of our team and our investigators. They have enabled us to successfully navigate this unprecedented and challenging time. This has been a difficult period for all of us, and our entire organization has risen to the occasion. Before summarizing our program highlights, I would like to take a moment to share with you our evolving vision for Marinus and Ganasalus. It has been slightly over a year since I stepped in as Executive Chairman and, as you know, roughly nine months since assuming the full-time CEO role.

Meritas you've been fortunate that as of today, we have avoided the major impacts Italy.

It's a challenging for many about colleagues across the life Sciences.

We don't courage that our upcoming clinical milestones, which include topline data from our phase three pivotal trial in T.K.L., five deficiency disorder, or CBD and the initiation of our phase two trial into gross square is just complex 40, yet.

Remain on track I.

I am grateful for the continued commitment and dedication of our team and our investigators have enabled us to.

To successfully navigate this unprecedented and challenging Todd.

She's been a difficult period for all of Us and I'll retire organization has risen to the cage.

Before summarizing our program quite like I would like to take a moment to share with you or Bobby Bidrin from Marriott and good absolute.

It's been slightly over a year since I stepped in as executive Chairman and as you know roughly nine months to be assuming the full time to your role.

Scott Braunstein: We have made a series of changes to the organization that should help drive our future success. These include key leadership hires and encouraging the focus of our core skill set. Continuing to improve our clinical trial design and execution, broadening our manufacturing capabilities, driving a renewed focus on treating patients with severe forms of epilepsy, and finally, demanding a clinical trial strategy that is based on strong science and meaningful unmet clinical needs throughout the epilepsy community. We see the opportunity to be a leader in the epilepsy space, both in and out of the hospital. Equally important, we will continue to apply strong financial discipline to the business while making the appropriate investments to help us achieve a successful development and commercial strategy.

Maybe series of changes to the organization that should help drive for future success.

These include the leadership hires.

Encouraging the focus of our core skill sets.

Continuing to improve our clinical trial design and execution.

Broadening.

That's great capabilities.

Driving a renewed focus on treating patients with severe forms the epilepsy and finally demanding a clinical trial strategy based on strong sorry.

Meaningful unmet clinical need throughout the epilepsy community.

We see the opportunity to be a leader in the former PC, both in and out of the hospital.

Equally important we will continue to apply strong financial discipline to the business, while making the appropriate investment to help us achieve its successful developmental and commercial strategy.

Later on the call Joe will provide more detail clinical update.

Scott Braunstein: Later in the call, Joe will provide more detailed clinical updates, but before that, I'd like to provide some brief program highlights. Let me start off with our Status Epilepticus Program, also referred to as STATUS or STP. Status is a rare condition where patients are in a prolonged state of continuous or near-continuous seizure activity that can lead to permanent damage to the brain and, in some cases, death.

Before that I'd like to provide some brief program highlights.

Let me start off with our status Epilepticus program.

Also referred to it started support.

That is the rare condition, where patients are in a prolonged the continuous or near continuous seizure activity that can lead to permanent damage to the brain and in some cases.

Last year, we reported encouraging data from ARQ <unk> two dose finding study of I think in absolute NSC with 100% of patients achieving the primary endpoint the pie that prevention of progression to our the anesthetic within 24 hours of treatment initiation.

Scott Braunstein: Last year, we reported encouraging data from our Phase 2 dose-finding study of IV Gonaxalone in SE, with 100% of patients achieving the primary endpoint, defined as prevention of progression to IV anesthetics within 24 hours of treatment initiation. In addition, we saw a rapid onset of action with a median time to SE cessation of five minutes across all doses evaluated. We also noted a numerical dose response trend with 100% of patients at the highest dose and at a targeted serum concentration of 500 nanograms per ml meeting the primary endpoint and having no recurrence of SE at the 72-hour time point. Additionally, in all patients available for follow-up, we saw no recurrence of SE up to the four-week visit.

In addition, we saw a rapid onset of action with a median time at Ccs teaching a five minutes across all doses about.

We also know did he numerical dose response trend with 100% of patients at the highest dose and.

Target the German concentration of 500 nanograms per ml meeting the primary endpoint and having no recurring put status at the 72 hour time point.

Additionally, in all patients available for follow up you know recurrence of SC up to the poor we did.

We are confident that connects the one has the potential to be a powerful new option capable of addressing the need for a safe and effective treatments for status.

Scott Braunstein: We are confident that Gonaxolone has the potential to be a powerful new option capable of addressing the need for a safe and effective treatment for status epilepticus. We believe that cannabis alone can provide a rapid onset of action and potentially play a role in preventing the devastating consequences of uncontrolled seizures. We have rapidly moved forward with our plans for a Phase III pivotal study and have made excellent progress, including a constructive end-to-Phase II meeting with the FDA. Joe will provide more details on our plans for the status program later in the call.

We believe that connects the loan can provide a rapid onset of action and potentially play a role in preventing the devastating consequences of uncontrolled seizures.

We have rapidly move forward with our plans for phase three pivotal study and have made excellent progress, including a constructive into phase two meeting with Yep Yep.

I will provide more detail and our plans for the status program later in the call.

Let's now move to the two our CBD program.

Scott Braunstein: Let's next move to our CDD program. CDD is a rare refractory form of pediatric epilepsy with limited effective treatment. Last quarter, we announced the completion of enrollment in the Marigold Study, our global double-blind, placebo-controlled, pivotal phase 3 trial evaluating oral gonaxalone in children and young adults with CDD. Importantly, we remain on track to report top-line data from the trials in Q3 of this year and have begun preparations for potential NDA files. We have also started to build a team to help lead our commercial strategy.

He is a rare refract reform pediatric epilepsy with limited epic teaches treatment.

Last quarter, we announced the completion of enrollment the Marigold study, our global double blind placebo controlled pivotal phase three trial.

Valuating World and that's the one in children and young adults with TBD.

Importantly, we remain on track to report topline beat up mid trials in Q3, this year and have begun preparations for the potential and we filed.

We've also started to build the team to help lead our commercial strategy.

Scott Braunstein: Our early commercial work has begun to fully evaluate the potential market opportunity for the use of Ganaxolone in patients suffering from CDD, as well as the possible expansion into the TSC population. We will be sharing those plans with you in greater detail should the Phase 3 trial meet its primary end. We are looking forward to sharing the dataset and our concurrent thinking about the program with you later this year.

Early commercial work has begun to fully evaluate potential market opportunity for the use of good that's alone in patients suffering from TBD.

Well as the possible expansion into the T. population.

We will be sharing those plans with you in greater detail should the phase three trial its primary endpoint.

We are looking forward to sharing that being said in our concurrent thinking about the program with you later this year.

As mentioned, we also have announced plans to expand the good that's one franchise into tier city.

Scott Braunstein: As mentioned, we also have announced our plans to expand the Ganaxalang franchise into TFC, which is a common cause of genetic epilepsy and a rare disorder that can affect many organs and lead to non-malignant tumors of the brain, skin, kidneys, heart, eyes, and lungs. The decision to expand into TSC was scientifically informed by our discovery of a new biomarker, alloprenaniline s We believe LOS may be an important biomarker to predict which rare genetic epilepsies are most likely to respond to treatment with Gonadsolone. Our analyses have indicated that TSE may be one of those rare genetic epilepsies that are impacted by the abnormal production of alloprenadolone, as well as other neurosteroids, thus increasing the likelihood that patients will benefit from gonaxalone treatment.

Which is a common cause of genetic epilepsy, and a rare disorder that can affect many oregon and reach non malignant tumor of the brain skin kidneys heart.

In London.

The decision to expand its yet see with signs typically informed by our discovery of a new biomarker Allopregnanolone soapy, where our west.

During our phase two study in PCB, each 19 related epilepsy.

We believe our west maybe an important biomarker to predict which rare genetic epilepsy.

Most likely to respond to treatment with good that's one.

Our analyses have indicated that T. S. C. Maybe one of those rare genetic epilepsy that are impacted by the abnormal production of allopregnanolone as well as other neuro steroids.

Increasing the likelihood the patient will benefit some good absolute treatment.

We believe this mechanistically relevant.

Scott Braunstein: We believe this mechanistically relevant, biology-driven approach is an important new chapter in our strategic advancement of Ganaxalone, which allows us to focus on indications with the greatest unmet medical need while also maximizing potential benefits for patients and our chance of clinical success. As part of the larger strategic clinical plan, we have made the careful decision to transition our ongoing Phase III violence study to a proof-of-concept trial evaluating LOS as a biomarker in patients with a confirmed PCVH19 mutation. As a reminder, PCVH19-related epilepsy is a serious and rare disease characterized by variable early-onset cluster seizures and comorbid cognitive and behavioral disturbances with or without accompanying intellectual disability.

Algae driven approach is an important new chapter in our strategic advancement of connects alone, which allows us to focus on indication with degrees unmet medical need while also maximizing potential benefit for patients and our chance of clinical success.

As part of the larger strategic clinical plan, we have made the careful decision to transition our ongoing phase three violent study.

You are proof of concept trial evaluating our Wes as they biomarker in patients with a confirmed PCB each 19 mutation.

As a reminder, PDH 19 related epilepsy, just curious to know and rare disease characterized by very bold early onset clusters teachers and co morbid cognitive behavioral disturbances with or without accompanying intellectual disability.

There were many factors that led us to limit trial enrollment, including the resources required for global study.

Scott Braunstein: There were many factors that led us to limit trial enrollment, including the resources required for a global study, the episodic nature of seizures in PCVH19 patients, the potential limited commercial opportunity associated with the indication, as well as the overall challenges associated with running a global pivotal study during the COVID-19 pandemic. Overall, we believe this transition will allow us to focus our capital resources on indications where there is a significant patient population that is currently underserved by available treatment. We strongly believe that this is the best use of our resources.

The thought of nature of seizures in PC, each 19 patients the potential limited commercial.

Opportunity associated with the indication as well as the overall challenges associated with running a global pivotal study during that Kobin 19 pandemic.

Overall, we believe this transition will allow us to focus our capital resources on indications where do they significant patient population that is currently underserved by available treatments.

We strongly believe that this is the best use of our resources.

Scott Braunstein: Joe will provide additional details on our updated clinical program for PCDH 19, but as part of our commitment to that community, we will continue to explore opportunities to provide Dinaxolone for patients that could substantially benefit from this therapy and will no longer be eligible for the violet study. Before I transition the call over to Joe for more detailed clinical updates, I'd like to spend a little time highlighting our corporate achievements for the quarter. We recently announced the formation of our Scientific Advisory Board and are fortunate to have six globally recognized seizure disorder experts. Dr. Hussain from Duke University School of Medicine, Dr. Rogalski from UC Davis Health, Dr. Hirsch from Yale Medicine, Dr. Vidkavich from Brigham and Women's Hospital, Dr. Trinkas from Paracels Medical University, and Dr. French from NYU Langone Health.

Joe will provide additional details on our updated clinical program for PC each 19.

But as part of our commitment to that community. We will continue to explore opportunities to provide thing that's one for patients that could substantially benefit from this therapy and will no longer be eligible for the pilot study.

Before I transition the call over to Joe for more detailed clinical update.

I'd like to spend a little time, highlighting our corporate achievements for the quarter.

We recently announced the formation of our scientific Advisory Board and are fortunate to have six globally recognized seizure disorder experts.

Dr Hussain from Duke University School of Medicine, Dr. Gargosky from you see needs help.

Dr Hirsch from Yale Medicine.

Dr been cabbage from Brigham and women's hospital.

After trinkets from that parents don't medical University, and Dr., French from and why you lend going help.

Scott Braunstein: While the accomplishments of the team are too long to list, I want to highlight that members of our newly formed SAVs have contributed to multiple anti-epileptic drug approvals, pioneered the field of neuroactive steroids, and are leading the establishment of international consensus on the diagnosis and treatment of status epileptica. We believe that the enthusiasm and participation of our scientific advisors is a testament to the potential of Ganaxalone to improve outcomes in patients with severe epilepsies and look forward to leveraging their guidance and expertise on clinical development and global registration strategies as we advance Ganaxalone through the clinic.

Well be accomplishments of the team or too long to list.

I want to highlight the that members of our newly corn seed be have contributed to multiple anti epileptic drug approvals pioneered the deal neuro after this year or and our meeting the establishment of international consensus on the diagnosis and treatment status epilepticus.

We believe that enthusiasm in participation of our scientific advisors is a testament to the potential we're gonna have swung to improve outcomes in patients with severe epilepsy and look forward to leveraging their guidance and expertise on clinical development and global reach global registration strategies.

As we advance connects will do the Clinton.

In addition, we were thrilled to announce the appointment of Sasha immediately as vice President of Investor Relations and corporate communications.

Scott Braunstein: In addition, we were thrilled to announce the appointment of Sasha DeMuniellis as Vice President of Investor Relations and Corporate Communications. Sasha brings extensive experience across diverse roles in health care and financial communications, as well as issues management and executive visibility, which will be invaluable to Marinus as we look to strengthen our relationship with key stakeholders, including investors, employees, advocacy groups, patient organizations, and the media. As you can see, we have put Sasha right to work. 2020 is going to be an exciting time at Marinus with pivotal trial data and the next phase of the development of Gynapsolone, which will include two additional late-stage clinical trials. We expect continued strong execution throughout the year and look forward to the implementation of our strategic plan to unlock the potential of Gonaxalone to transform the treatment paradigm for a number of severe and life-threatening epilepsy. With that, I would now like to turn the call over to our Chief Medical Officer, Joe Hulihan. Joe?

Sasha brings extensive experience across diverse roles in healthcare and financial communications as well as issues management and exactly visibility, which will be invaluable generic but as we look to strengthen our relationship with key stakeholders, including investors employees advocacy group.

Patient organization and then media.

As you can see you put itself you're right to work.

Twentytwenty is going to be an exciting time that Meredith.

Pivotal trial data and the next phase of the development of good NAPSLO, which will include two additional late stage clinical trials.

We expect continued strong execution throughout the year and look forward to the implementation of our strategic plan to unlock the potential of gonna have slowed to transform the treatment paradigm for a number of severe in life threatening epilepsy.

With that I would now like to turn the call over to our Chief Medical Officer, Joe Houlihan Joe.

Thank you Scott and good morning, everyone.

Joseph Hulihan: Thank you, Scott. Good morning, everyone.

As Scott mentioned these are exciting times at Mariners and I'd like to update you on our clinical plans with key milestones for 2020 and beyond.

Joseph Hulihan: As Scott mentioned, these are exciting times at Marinus, and I'd like to update you on our clinical plans and key milestones for 2020 and beyond. So, starting off with status epilepsy. Last year, we reported encouraging positive data from our Phase 2 dose-finding study, and based on these results, we've rapidly advanced our plans for a Phase 3 registration-enabling trial. We had a highly collaborative and productive end-of-phase 2 meeting with the FDA and can report that we've reached general alignment on key aspects of our Phase 3 trial, including the primary endpoints, the dosing paradigm, and the patient population. The study has two co-primary endpoints, which address the major criteria for efficacy in status epilepsy, stopping status epilepticus rapidly and maintaining seizure control.

So starting off with status epilepticus.

Last year, we reported encouraging positive data from our phase two dose finding study and based on these results Weve rapidly advanced our plans for a phase three registration enabling trial.

We had a highly collaborative unproductive into phase two meeting with the FDA.

And can report that Weve reached a new alignment on key aspects of our phase three trial design.

Including the primary endpoints the dosing paradigm in the patient population.

The study has to co primary endpoints, which address the major criteria for efficacy and status epilepticus.

Stopping status epilepticus rapidly and maintaining seizure control.

The selection of these endpoints was informed by the findings from phase two and give us reason to be optimistic about the outcome in phase three.

Joseph Hulihan: The selection of these endpoints was informed by the findings from Phase 2 and gives us reason to be optimistic about the outcome in Phase 2. In phase two, we saw that the median time to seizure cessation was five minutes, and it was less than 30 minutes for all but one patient. So, the first co-primary endpoint for phase three is the proportion of patients with seizure cessation within 30 minutes without additional treatment. This endpoint not only reflects the rapid onset of action.

In phase two we saw the median time to seizure cessation was five minutes.

It was less than 30 minutes for all but one patient.

So the first co primary endpoint for phase three is the proportion of patients with seizure cessation within 30 minutes without additional treatment.

This endpoint not only reflects rapid onset of action.

Joseph Hulihan: It also allows the physician to intervene quickly with alternative treatments if needed. The other co-primary endpoint is the percentage of patients without progression to IV anesthesia for at least 24 hours. In the Phase II study, none of the 17 patients required IV anesthesia within 24 hours of initiating treatment, which was the study's primary endpoint. This is meaningful as a proxy measure for control of status epilepticus and is also an important clinical and health economic endpoint in and of itself. Morbidity and mortality with IV anesthesia are considerable, and the need for ICU admission and prolonged hospital stays drive the cost of care. The Phase 2 study also informed our dose selection for Phase 3. And we have agreement from the FDA on this aspect of the study.

It also allows that position to intervene quickly with alternative treatments if needed.

The other co primary endpoint is the percentage of patients without progression to Ivy anesthesia for at least 24 hours.

The phase two study none of the 17 patients required Ivy anesthesia within 24 hours of initiating treatment, which was the studies primary endpoint.

This is meaningful as a proxy merger for control of status Epilepticus and is also an important clinical in health economic and point in of itself.

Morbidity and mortality with I'd be anesthesia or considerable and the need for I see you admission and prolong hospital stays drive the cost of care.

The phase two study also informed our dose selection for phase three and we have agreement from the FDA on this aspect of the study.

We found that maintenance of like an excellent blood level of at least 500 nanograms per ml for eight hours was a key aspect and completely controlling status.

Joseph Hulihan: for eight hours was a key aspect in completely controlling status. In Phase 3, based on formulation improvements, we will be able to maintain Ganaxalone at that target blood level for 12 hours rather than 8. The study will enroll approximately 125 patients whose status failed to respond to first-line treatment with a benzodiazepine and then two additional second-line anti-seizure medications, which was the average number of failed second-line treatments in the Phase 2 study. With that number of participants, the trial will have greater than 90% power to detect a 30% difference between the drug and placebo.

Phase three based on formulation improvements, we will be able to maintain an excellent that target blood level for 12 hours rather than.

The study will enroll approximately a 125 patients who status has failed to respond to first line treatment with a benzodiazepine.

And then two additional second line anti seizure medications, which was the average number of failed second line treatments in the phase two study.

With that number of participants the trial will have greater than 90% powered to detect a 30% difference between drug and placebo.

We look forward to continued dialogue with the FDA as we finalize the trial protocol and expect patient enrollment to begin in the third quarter of this year.

Joseph Hulihan: We look forward to continued dialogue with the FDA as we finalize the trial protocol and expect enrollment to begin in the third quarter of this year. I'm also pleased to share that we have already begun identifying and readying trial sites, which will ensure the rapid initiation of patient and We expect top-line data in the first half of 2022 and feel that the study will provide clinically meaningful results to guide care for a common and catastrophic neurologic emergency. Turning to CDD, as Scott mentioned, we have completed enrollment in the Marigold study, our pivotal phase three study evaluating oral gonaxalone in children and young adults with CDD. As a reminder, this global, double-blind, placebo-controlled trial has completed on-schedule enrollment of 101 patients between the ages of 2 and 21 with a confirmed CDKL5 gene mutation.

I'm also pleased to share that we have already begun identifying and readying trial sites, which will ensure the rapid initiation of patient enrollment.

We expect topline data in the first half of 2022.

It feels that this study will provide clinically meaningful results the guy care for our common catastrophic neurologic emergency.

Turning to CDB.

Scott review, we have completed enrollment in the Marigold study our pivotal phase three study evaluating Oregon acts alone in children and young adults with CBD.

As a reminder, this global double blind placebo controlled trial has completed on schedule and moments of 101 patients between the ages, two and 21 with a confirmed CDK all five June patients.

Joseph Hulihan: Today, I can provide an update that the discontinuation rate continues to be less than 10% and that we are encouraged by the continued high rate of enrollment in the open-label extension arm of the trial. We are fast approaching our last patient visit and remain on track to report top-line data from the trial in the third quarter of this year. We've been able to keep the required trial assessments on track despite disruptions from COVID-19, and the use of electronic seizure diaries is allowing us to access and analyze patient data remotely. We have actively begun preparations for the NB filing that would follow a successful Phase 3 outcome. Moving next to Tuberous Sclerosis Complex, or TSC, our newest clinical program, which marks the next step in our biomarker-driven strategy to target indications where we believe Ganaxalone is most likely to benefit patients. As Scott said, TSC is a complex disease with diverse manifestations and is a leading cause of genetically determined epilepsy. Seizures of PSC can be one of the most difficult aspects of the illness, with multiple seizure types that can change across a patient's life and that are often treatment resistant.

Today I can provide an update the discontinuation rate continues to be less than 10%.

And that we are encouraged by the continued high rate of enrollment in the open label extension arm of the trial.

We are fast approaching our last patient visit and remain on track report topline data from the trial in the third quarter of this year.

We've been able to keep the required trial assessments on track despite disruptions from cobot 19.

The use of electronic seizure diaries is allowing us to access and analyze patient data remotely.

We have active wouldn't be gun preparations for the N.B. filing that would follow a successful phase three outcome.

Moving next to Toubro sclerosis complex or T. S C.

Our newest clinical programs, which marks the next step in our biomarker driven strategy to target indications, where we believed in Acs loan is most likely to benefit patients.

As Scott said PSC is a complex disease with diverse manifestations.

And as a leading cause of genetically determined epilepsy.

The seizures of P.S.. So you can be one of the most difficult aspects illness.

Multiple seizure types that can change across a patients lifespan and that are often treatment resistance.

And our discovery of Aloe us as a potential response biomarker was especially relevant to individuals with TSC.

Joseph Hulihan: And our discovery of ALOES as a potential response biomarker was especially relevant to individuals with TSC, since an imbalance in endogenous neurosteroid levels has been identified as a potential factor in seizure causation. Ganaxialone would directly target this imbalance. Our discovery of LOS as a potential biomarker for epilepsy has also allowed us to investigate a range of other genetic epilepsies to determine where we would expect an axolotl to have the highest likelihood of being affected. If confirmed, this finding would provide a goal for individualized treatment, one that focuses the use of medication in those patients most likely to benefit, while also preventing the challenging, often years-long journey epile Our Phase II open-label trial in TSC will evaluate the safety and tolerability of adjunctive connexalone in approximately 30 patients from ages 2 to 65 with highly refractory seizures.

Since an imbalance in endogenous steroid levels has been identified as a potential factor in seizure causation.

Your next alone would directly target this imbalance.

Our discovery of al or less as a potential biomarker for epilepsy has also allowed us to investigate a range of other genetic epilepsies can determine where are we would expect an x. alone had the highest likelihood of being affected.

If confirmed this finding realize a goal for individualized freezes.

One that focuses the use of medication in those patients most likely to benefit.

Also preventing the challenging often years long journey epilepsy patients can face while searching for an effective treatment.

Or face to open label trial, and T.S.C., we'll evaluate the safety and tolerability of adjunct to connect Sloane.

Approximately 30 patients for majors to the 65 with highly refractory seizures.

Study participants will receive up to 600 milligrams of the oral liquid suspension of can axles three times a day for 12 weeks.

Joseph Hulihan: Study participants will receive up to 600 milligrams of oral liquid suspension of Conexalone three times a day for 12 weeks. Patients who meet eligibility criteria may continue Gonaxalone treatment for a 24-week extension. The primary endpoint is percent reduction in monthly seizure frequency relative to baseline. In line with our biomarker strategy, we plan to analyze whether there is an association between ALOES levels and seizures.

Patients, who meet eligibility criteria and they continue can axle and treatment in the 24 week extension.

The primary endpoint as percent reduction monthly seizure frequency relative to baseline.

One with our biomarker strategy, we plan to analyze whether there's an association between our west levels and seizure improvements.

We've already made considerable progress and preparing for the initiation of this trial.

Joseph Hulihan: We've already made considerable progress in preparing for the initiation of this trial, with one of the six planned trial sites already open. We anticipate beginning to screen and enroll patients during this quarter and do not anticipate any delays in trial progress related to COVID-19. We plan to report top-line data from the study in the third quarter of 2020. Finally, I'd like to touch on the VIOLET study, our trial in PCDH19-related epilepsy. Earlier in the call, Scott detailed that we've decided to transition the ongoing phase three program to a proof of concept.

With one of the six plan trial sites already open.

We anticipate beginning to screeners enroll patients during this quarter and do not anticipate any delays and trial progress related to cope with Nike.

We plan to report topline data from the study in the third quarter of 2021.

Finally, I'd like to touch on the violence study or trial P. C. D H 19 related epilepsy.

Earlier in the call Scott detailed that we've decided to transition ongoing phase three program to a proof of concept study.

Joseph Hulihan: This study will stratify patients into one of two biomarker groups based on LOS levels, and they will be randomized to ganaxalone or placebo within each stratum. The trial will consist of a prospective baseline period to collect seizure data, followed by a 17-week double-blind treatment phase. Patients randomized to Ganaxalone will titrate over four weeks to a dose of up to 600 milligrams of oral liquid suspension three times a day and maintain that dose for 13 weeks.

This study will stratify patients into one or two biomarker groups based on our west levels and they will be randomized to get an x. alone or placebo within each strategy.

The trial will consist of a prospective baseline period to collect seizure data.

Followed by a 17 week double blind treatment phase.

Patients randomized to get an actual loan will tie trade over four weeks to a dose of up to 600 milligrams of oral liquid suspension three times, a day and maintain that goes for 13 weeks.

Joseph Hulihan: We expect to complete the double-blind portion of the trial with approximately 15 to 20 patients and anticipate top-line data in the first half of 2021. With these studies, we have developed a clinical program focused on rare epilepsies with high-end medications where we believe Ganaxalone has real potential to improve patient outcomes. In the next few months, we'll see many program milestones with new data from our pivotal study and the initiation of late stage trials, and we look forward to sharing our progress with you. Before I turn the call over to Ed to review our financials, I'd like to sincerely thank our investigators and study site personnel. Their dedication to their patients and to advancing studies that could yield new treatments has ensured the continuity of our programs despite the rapidly evolving challenges to the health care system posed by COVID-19.

We expect to complete the double blind portion of the trial with approximately 15 to 20 patients and anticipate topline data in the first half of 2021.

With these studies, we have developed the clinical program focused on rare epilepsies with high unmet need now, where we believed and axle own has real potential to improve patient outcomes.

The next few months, we'll see many program milestones with new data from our pivotal study and the initiation of late stage trials, and we look forward to sharing or progress with you.

Before I turn the call over to add to review our financials I'd like to sincerely. Thank our investigators and study site personnel.

Whose dedication to their patients into advancing studies that could yield new treatments has ensured the continuity of our programs. Despite the rapidly evolving challenges to the health care system posed by covalently team.

Joseph Hulihan: And most importantly, on behalf of Marinus, I would like to thank the patients and their families who participated in these trials. We are increasingly confident that our efforts will lead to meaningful improvements for patients and families who must deal with the challenges of such severe neurologic disorders. With that, I'll turn the call over to Ed. Thanks, Joe. And good morning, everyone.

Most importantly on behalf of Meritas I'd like to thank the patients and their families who participated in these trials.

We are increasingly confident that our efforts will be the meaningful improvements for patients and families who must deal with the challenges of such severe neurologic disorders.

With that I'll turn the call over that.

Thanks, Joe and good morning, everyone. Our financial results for the first quarter or 20, Twond and released this morning reflect the continued expenses associated with our ongoing clinical development programs work in Acs alone.

Ed Smith: Our financial results for the first quarter of 2020, released this morning, reflect the continued expenses associated with our ongoing clinical development programs for Ganaxialone. For the first quarter of 2020, R&D expenses were $15 million, compared with $8.9 million for the same period in 2019. This increase in research and development expense is mostly attributable to the NDA enabling clinical and manufacturing activities in support of our most advanced clinical program in CDD. In addition, we have begun to incur costs associated with our preparations to commence a Phase III pivotal trial in status epilepticus, which are partially offset by reduced non-seizure disorder costs driven by the completion of our Phase II PPD studies last year. General and administrative costs are consistent quarter to quarter, which were $3.9 million versus $3.7 million for the first quarter of this year and last, respectively.

For the first quarter up 2020, R&D expenses were 15 million compared with 8.9 million for the same period in 2019.

This increase in research and development expenses, mostly attributable to the India, enabling clinical and manufacturing activities in support of our most advanced clinical program in C D.

In addition, we have begun to incur costs associated with our preparations to commence a phase three pivotal trial in status epilepticus, which are partially offset by reduced non seizure disorder costs driven by the completion of our phase two P.T.D. studies last year.

General and administrative costs are consistent quarter to quarter, which were 3.9 million versus 3.7 billion for the first quarter of this year at last respectively.

Our net loss for the first quarter. A 2020 was 18.7 million for 32 cents per basic and diluted share compared with a net loss of 12 and a half million for 24 cents per basic and diluted share for the same period in 2019.

Ed Smith: The net loss for the first quarter of 2020 was $18.7 million, or $0.32 per basic and diluted share, compared with a net loss of $12.5 million, or $0.24 per basic and diluted share, for the same period in 2019.

As of March 31st 2020, we had approximately $77.8 million in cash cash equivalents and investments compared with approximately 91.7 million at the end of the here.

Ed Smith: 2019

Ed Smith: As of March 31, 2020, we had approximately $77.8 million in cash equivalents and investments.

Our current cash and investments without taking into consideration any pick any potential cash inflows to the company provide runway into the third quarter 2021, assuming our current scale of operations as described earlier by Scott Joe.

Ed Smith: Compared with approximately 91.7 million at the end of the year.

Ed Smith: Our current cash and investments, without taking into consideration any potential cash inflows to the company, provide runway into the third quarter of 2021, assuming our current scale of operations as described earlier by Scott. Before opening the call for questions, I'd like to thank our investors for their continued support and confidence in our program.

Before opening the call for questions I'd like to thank or investors for their continued support and confidence in our programs. I also want to of course, thank the entire management team and all Meritas employees for their hard work dedication to our mission. We look forward to continued momentum and progress through 2000.

Ed Smith: Program. I also want to, of course...

Morning.

Thank you and with that we will now open the call for questions.

Ed Smith: I want, of course, to thank the entire management team and all Marinus employees for their hard work and dedication to our mission. We look forward to continued momentum and progress through 2020.

Thank you.

Well now be conducting a question and answer session. If he would like to ask the question.

Please press star one on your telephone keypad confirmation tola indicate that your line is in the question Q.

A press star to if you'd like to remove your question from the Q4 participants using speaker equipment. It may be necessary to pick up your headsets before pressing the star keys. One moment. Please why we poll for questions.

Operator: Thank you, and with that, we will now open the call for questions. Thank you. We'll now be conducting a question and answer session. If you would like to ask a question, please press star 1 on your telephone keypad. A confirmation tone will indicate that your line is in the question queue. You may press star 2 if you'd like to remove your question from the queue. For participants using speaker equipment, it may be necessary to pick up your handset before pressing the star keys. One moment, please, while we poll for questions. The first question is from Marc Goodman, SVB Lear Inc. Please go ahead, sir. Yes, morning guys.

The first question, there's some Marc Goodman SVB Leerink. Please go ahead Sir.

This morning, guys. So first question is should we assume from.

The conversation about Pcs, stating that this is basically a switch.

From that to TSC as far as a program that you're going to be pursuing you know over the goal line to get approval for that indication.

And second of all can you talk about how you have avoided these co that clinical trial issues. It just seems like everybody seems to be having trouble and it's great to hear that that you're not and you don't plan to but can you talk about that and why it is and there was something specifically in the press release about CDD phase ones.

Marc Harold Goodman: So my first question is, should we assume from the conversation about PCH-19 that this is basically a switch? From that to TSC as far as a program that you're going to be pursuing, you know, over the goal line to get approval for that indication, and second of all, can you talk about how you've avoided COVID clinical trial issues? It just seems like everybody seems to be having trouble, and it's great to hear that you're not, and you don't plan to, but can you talk about why it is, and there was something specifically in the press release about CDD phase one supportive trials, no delays, or there could be delays. A little more color on what you meant there. Thanks.

Sort of trials no delays or are there could be delays.

Just give some more color and what you meant there. Thanks.

Sure Good morning, Mark I hope, you're doing well and.

As we kicked it off this morning, and as everyone can appreciate Joe myself as south charge in different locations and we're trying our best to meet our phones before we answer so theres a delay we'll we'll make sure we remind everyone. So let me let me tackle the first question Mark about our strategic plan.

T CDH 19 relative to T. S C.

Yes, you should assume that's yet see in our mind in is a program that we'd like to see move rapidly through phase two and with a positive outcome on T.D.D.

Scott Braunstein: Good morning Marc, hope you're doing well, and as we kick it off this morning, as everyone can appreciate, Joe, myself, Ed, and Sasha are in different locations, and we're trying our best to mute our phones before we answer so if there's a delay, we'll make sure we remind everyone. So let me tackle the first question, Marc, about our strategic plan, TCD-H19, relative to TSC Yes, I think you should assume that TSC, in our minds, is a program that we'd like to see move rapidly through phase two, and with a positive outcome for TDD and a strong proof-of-concept study for TSC, we would be very interested in initiating phase three in the first half of next year for TSC, again, if our programs all point in that direction. We're thinking about that for several reasons.

And our strong proof of concept study in T.S.C., we wouldn't be very interested in initiating the phase three in the first half of next year for T.S.C. again, if if if our programs all point in that direction.

We're thinking about that for several reasons, we think the biomarker signal and TSC is equally strong at least from a blood sample and do the standpoint to support the potential efficacy connects alone and T.S.C., we think the market is substantially larger.

Oh feedback with the investigator community that there's a huge amount of interest to study additional jobs in the TSC population and we are very interested in it in and seeing how a proof of concept data planes out on the on the flipside PCB.

Scott Braunstein: We think the biomarker signal in TSC is equally strong, at least from a blood sample and data standpoint to support the potential efficacy of Gonaxalone in TSC. However, we think the market is substantially larger. Our feedback with the investigator community is that there is a huge amount of interest in studying additional drugs in the TSC population, and we are very interested in seeing how our proof-of-concept data plays out. On the flip side, for PCVH19... As you know, Marc, this was a study that was already underway when I took over as Executive Chairman. I think in the early days of our market research work, I had some concerns about the market potential here. We saw in the clinical trial that the number of patients that were out there, we certainly could find enough to do a phase three, but we were concerned that, ultimately, this was a much smaller market opportunity. I think I've spoken with all of you that, very different than CDD, and I would say even in the TSC population, many children with PCVH19 are well controlled on their current anti-seizure regimens, and it's really only a handful of patients, somewhere between 30, 40, and 50% that wind up needing supplemental medications. And again, it's very episodic.

Each 19.

As you know Mark. This was the study that is already underway when I took over as executive Chairman I think coming early days of our market research work.

I had some concerns about the market potential here, we saw in the clinical trial, but the number of patients that were out there, but we certainly could find enough to do a phase three but we were concerned that ultimately this was a much smaller market opportunity I think I've spoken with all abuse.

That's very different than TV, and I would say, even the TSC population many children with P.T.H. 19.

Well controlled on their current anti seizure regimens and it's really only a handful of patients.

Somewhere between 30, 40, and 50% that wind up meeting supplemental medications and again, it's it's a very episodic Sasha so.

Our decision to move away from the things three trial or the filing strategy with PCH 19 is really one of the resource allocation now that being said Mark I think we can really seen some true proof of concept data from what I would now consider phase two study in PCB 18, 19 were going to continue.

Scott Braunstein: So our decision to move away from a phase three trial and a filing strategy with PCVH-19 is really one of resource allocation. Now, that being said, Marc, I think we can really see some true proof of concept data from what I would now consider a phase two study in PCVH-19. We're going to continue to run that as a blinded study, so we'll be able to look at our results in at least a good number of patients for both patients that produce very low aloe levels and abnormally high aloe levels. And our hope would be, if all programs continue to show positive data, or at least our phase three programs show positive data, we would strongly consider a strategy of a biomarker study in the future that included multiple genetic orphan epilepsies, PCVH19 being a part of that, to really think about a broader allo biomarker strategy and phase four program. So that's the logic around the switch, really, from PC

You do run that as a blinded study will be able to look at least it in a good number of patients at <unk> at our results in both patients that produce very low salary levels and that and abnormally high at low levels.

And our hope would be if all programs continue to show positive data or at least our phase three programs show positive data, we would strongly consider a strategy of that other biomarker study in the future.

That included multiple genetic orphan epilepsies, PCB, aged 18 being part of that to really think about a broader allo biomarker.

Strategy at <unk> and paid for program, so that the logic around the the switch really from PCB age thing to tier C.

I think we can really condensed timelines if things all work in our favor and as as we said the market opportunity is is several times larger and I think the unmet significantly larger as well.

Scott Braunstein: I think we can really condense timelines if things all work in our favor, and as we said, the market opportunity is several times larger, and I think the unmet need is significantly larger as well. And I'll ask Joe to make a few comments in a minute as well, but just to move to the COVID clinical trial issue, we have been fortunate. A lot of it is serendipity, a lot of it is timing, a lot of it is our trial design, but from a CDD standpoint, the fact that our efficacy data is an electronic diary system that patients are doing at home relieves a lot of stress from a visit standpoint, patients needing to go to the hospital. That being said, our team was very early in moving to telephonic visits for the investigators.

And Oh, I'll ask Joe to make a few comments it up in a minute as well, but just to move to the Tobin a clinical trial issue we had been fortunate.

A lot of it is serendipity a lot of it it's timing a lot of it is our trial designs, but.

From a tsunami standpoint, the fact that are.

That's good to see data is an electronic diary system that patients who are doing at home.

That that will move a lot of stress for about a visit standpoint patients means come to the hospital that being said our team was very early in moving to tell upon it visits for the investigators are.

Scott Braunstein: We talked about that in February, and much of that activity started to move really before businesses were closed here in the U.S. We looked at our supply chain very early in February. We had some investors in the middle of February, and that really kind of got our minds thinking about all the things we could do. And so we feel very fortunate from a standpoint of the CDD trial that we're really in very good shape with the tail end of that trial. The efficacy data, and patient visits can all be done telephonically or at home, so we feel very confident about the timing of that trial. Status is a little bit of a wait and see, right? I mean, we are fortunate that, again, at the end of phase two meeting with the agency, telephonically, we heard from the agency very quickly in terms of the final meeting minutes.

We talked about that in February and Weve EM and much of that activity started to move really before businesses were close here in the U.S.. We looked at we looked at our supply chain very early in February.

We had some is that investors that the office middle of February of that really kind of got our minds thinking about all the things we could do and so we feel very fortunate from that standpoint into C.D. trial that to really had very good shape with a tailored to that trial. The efficacy data the patient visits can can all be done telephone.

The or at home until we feel very confident about the timing around that trial status is a little bit of a wait and see right. I mean, we are fortunate again and the piece to meeting with the agency Telephonically, we heard from the agency very quickly in terms of the final.

Meeting minutes.

And our hope and expectation.

Scott Braunstein: And our hope and expectation is that as the world starts to open up... Our ability to go into phase 3 in the second half of the summer should have minimal effect, of course, if COVID-19 re-emerges and hospitals are extremely busy. That will be a real challenge, but at least where we stand today, we're feeling reasonably confident in our ability for that trial to open as we had planned. Our clinical operations team is doing an incredible job of working with sites now who want to be a part of the trial. Those numbers, in terms of recruiting our sites, are right on time.

As the world starts to open up.

Our ability to go into phase three in the second half of the summer should should have minimal effect course of cobot 19 re emergence and hospitals or or are extremely busy that will be a real challenge.

But at least where we stand today, we're feeling reasonably confident our ability for that trial to open as we had planned art political ops team is doing an incredible job of working with sites now want to be a part of the trial those numbers in terms of recruitment in the far sites right on time.

So that's been that's right now we feel very confident that as long as the world doesn't get worse than the U.S. in particular doesn't get worse, we feel as though our phase three clinical should be on track.

Scott Braunstein: So that's been, that's, right now, we feel very confident that as long as the world doesn't get worse, and the US, in particular, doesn't get worse, we feel as though our Phase 3 plan should be on track. In terms of the Phase I trials, as many of you know, there are several supporting trials that are required for FDA approval. We, like other companies, have been affected by the closure of Phase I centers. But my head of plan operations, Matt Hall, and I talked about this last week, and we're seeing the Phase I centers starting to reopen. We've actually heard about one of the CROs that we use will actually put all our Phase I patients into a hotel. If it all goes well, they'll be screened for COVID.

In terms of the phase one trials there as many of you know there are several supporting trials that are required for FDA approval.

We like other companies that have been affected by things weren't centers closing, but my head of Clin ops that hall, and I talked about this last week and we're seeing the things one center starting to reopen we've actually heard about one of one of the CR routes that we use.

Actually put or fees when patients into a hotel if it all goes well they'll be screen for Tobin there'll be placed in a hotel and then brought into a phase one center as as a potential solution and so our expectation is that are things one work, which is not rate limiting for our and be a four or five.

Scott Braunstein: They'll be placed in a hotel and then brought into a Phase I center as a potential solution. And so our expectation is that our Phase I work, which is not rate-limiting for our NDA or our filing, those trials will have a one to three month delay, but we expect them all to be back on track in the summer or the fall. Joe, any comments on either your general thoughts about TSC, PCVH19, or clinical trial work that I didn't mention that you want to add?

Doing well those trials will have a a one to three month delay, but but we expect them all through the back on track in the summer or fall, Joe any comments on either.

Your general thoughts about T has seen PCH 19, or what clinical trial work that did I didn't mention that that you weren't ahead.

Scott Braunstein: Yeah, no, thanks, Scott. Well, I think for PCDH-19 and TSC, you know, we're going to continue it as a proof of concept. I think that one would have been more challenging to enroll, and perhaps that's a marker of the lesser degree perhaps of unmet need in terms of rapidity of enrollment, and I think TSC is particularly interesting in terms of biomarkers, I believe. For the enrollment issues with CDD, again Scott mentioned serendipity, the team enrolled that trial, the enrollment was on track, and it was completely enrolled around the time the COVID-19 problem, We didn't need to screen anyone.

Oh, Thanks, Scott well I concur.

The age 19, and TSC, we're going to continue it as are the PC gauge 19 studies proof of concept.

I think got one would have been more challenger to enroll and.

And perhaps that's a.

Cool about marker of the.

Lesser degree, perhaps an unmet need a in terms of repetitive enrollment.

And I think Trc is particularly interesting in terms of a biomarker hypothesis.

For the enrollment issues with CDD again, Scott mentioned serendipity the team enrolled that trial.

At the enrollment was on track and that was completely enrolled around the time covert 19 problems.

Hit and so we were able to do the remote patient visits we didn't need to screen anymore patients.

Joseph Hulihan: For more information, visit www.fema.gov.

ER and deal PC dates on team was earlier phase.

unknown: PCDH19 was in an earlier phase; patients needed to be screened, and that had to be done in person, but the CDD visits could be done remotely, patients could get remote labs, and so again, the team did everything they could to get things done on time and do as much as they could remotely. And also, Scott mentioned we have the electronic seizure diaries, so we can access that efficacy data.

Patients needed to be screens and that had to be done in person, but the CD do business could be done remotely patients can get remote labs, and so again the team did.

Everything they could.

To to get things done on time and do as much. The can remotely and also you know Scott mentioned, we have the electronic seizure diary. So we can.

Access that the efficacy data remotely.

Marketing follow ups or should I don't want to thank you. Thanks.

Joseph Hulihan: Marc, any follow-up questions, or should we go to the next question? Thank you.

Marc Harold Goodman: Thanks.

Operator: But

Next question is some Jay Olson Oppenheimer. Please go ahead Sir.

Jay Olson: The next question is from Jay Olson, Oppenheimer. Please go ahead, sir.

Oh, Hey, congrats on the progress and thanks for taking the questions. There's an F. T. A decision coming up later this year for Epidiolex her TSC and I was wondering how you expect that to impact the treatment landscape, assuming that drugs approved for T.S.C. and how do you expect it to improve.

Scott Braunstein: Oh hey, congratulations on the progress, and thanks for taking the questions. There's an FDA decision coming up later this year for Epidiolex for TSC, and I was wondering how you expect that to impact the treatment landscape, assuming that the drug is approved for TSC, and how you expect it to impact enrollment in your TSC study and eventually differentiate Ganaxone, and perhaps with the Alleles biomarker data you're collecting? Thanks for the question, Jane. Thanks for the nice comments, and I appreciate you dialing in today.

In fact enrollment of your T.S.P. study and eventually differentiate can axon, perhaps with the Algawise biomarker data you're collecting.

[noise] They said they answer the question Joan Thanks, with a nice comments and appreciate you dialing in today, we <unk>, we did a lot of work around the TSC program at the yes last December.

Scott Braunstein: We did a lot of work around the TSC program at ATS last December, and the feedback that we were getting from our investigators, and some of you are very comfortable with today, that there are quite a few TSC patients already on Epidiolex. The product is either being used via prescription off-label today, or quite a few patients through expanded access. In sites that are using Epidiolex or Expanded Access, they are excluded from our trial because that's part of the clinical trial design for the GW program. That's very logical and seen all the time that you can't add on to a new therapy.

And the feedback that we were getting from our index investigators and something we're very comfortable with today that there are quite a few TLC TLC patients already on Epidiolex.

The product either being used.

Do you prescription off label today, or a quite a bit of UBS of patients through expanded access and sites that are you using epidiolex are exposed to that so they are excluded from our trial because that's part of that the clinical trials.

For the the GW program, that's very logical and then seen all the time that you can't add up to a new therapy. So all of our sites have been chosen where things were 100% of our physicians. Our investigators are not using at that I like the expanded access form.

Scott Braunstein: So all of our sites have been chosen where 100% of our physicians and our investigators are not using Epidiolex in an expanded access format. We would expect quite a few patients in our phase two study to be on Epidiolex as background therapy. And certainly, we think that'll be very valuable in terms of the read, and we don't see the approval of Epidiolex later this year in TSC at all being a rate-allowing factor. And in fact, some of the centers that we've already talked to and will begin screening shortly are very excited about the opportunity and have told us they have a significant number of patients that they are interested in enrolling in the study. So we think that that approval will not at all impact our enrollment. Joe, any other comments that you'd like to add about Epidiolex?

Matt.

We wouldn't expect quite a few patients in our phase two study to beyond Epidiolex his background therapy.

And certainly we think that will be very valuable in terms of the read and we don't see the approval of Epidiolex later this year Ntsc at all being a rate limiting factor and in fact, some of the centers that we've already talked to and has now.

I will begin screening shortly.

Our are very excited about the opportunity and they told us they have a significant number of patients that they are interested in are rolling in the study. So we think that that in that approval will not at all impact our involvement Joe any other comments that you'd like to add around epidiolex.

Oh yeah.

Joseph Hulihan: Yeah, from a clinical standpoint, unfortunately, there's still a lot of unmet need. Every patient responds differently to medications. Our investigators tell us, as Scott mentioned, that they have patients that will qualify for the study. Despite the availability of Epidiolex or any other seizure medication, seizures in PSE can be quite...

From a clinical standpoint, Unfortunately, there's still a lot of unmet need every patient response differently to medications our investigators.

Tell us as Scott mentioned that they have patience that more qualified for the study.

Despite.

The available to them dialects or any other you know seizure medications seizures and PSC can be quite.

Refractory so I think we'll be on track for enrollment of the study.

Joseph Hulihan: Factory. So I think we will be on track for enrollment. Great, that's very helpful. Thank you. And if I could maybe squeeze in a house...

Great. That's very helpful. Thank you and if I could maybe squeezing a housekeeping question about the S. Three that you filed last week, there seems to be some confusion about the common and preferred share components and the conversion. So maybe if you could please describe your plans there that would be great.

Ed Smith: I have a housekeeping question about the S3 that you filed last week. There seems to be some confusion about the common and preferred share components and the conversion, so maybe if you could please describe your plans there, that would be great. Thanks Jay. Ed, do you want to take that? Yeah, I got it.

Thanks, Jay I'll turn it over to Ed and everyone take that.

Yeah I got it thank you and good morning Ah, Yes, yes, three that we filed last week relates to the preferred stock that we sold as part of the financing that we did in December so essentially it's a registration statement that covers the common stock.

Ed Smith: I got it. Thank you, and good morning. Yeah, so the S3 that we filed last week relates to the preferred stock that we sold as part of the financing that we did in December. So essentially, it's a registration statement that covers the common stock that underlies the preferred, and the preferred that we sold in December reflects both the increase in our authorized share count and, secondly, the effectiveness of that registration statement that we just filed. The preferred will, in large part, mandatorily convert into common stock, so we would expect that to occur either later this month or next month.

That underlies the the preferred and the preferred that we sold in December upon Oh.

The increase in the authorized share count and secondly, the effectiveness of that registration statement that we just filed a the preferred was in large part mandatorily convertible into common stock. So we would expect that to occur either later this month or next month.

Jay Olson: Great, that's very helpful. Congratulations again on the progress and thanks again for taking the questions. Thanks Jay, stay well.

Great. That's very helpful. Congrats again on the progress and thanks again for taking the questions.

Thanks, Jay stay well.

We have a question from afraid Lithia Young Cantor Fitzgerald. Please go ahead.

Alethia Young: We have a question from Alethia Young, Cantor, and Fitzgerald. Please go ahead. Hey guys, thanks for taking my questions, and uh, see what we got from the progress. Um, I guess, a couple. One, can you talk a little bit about the conversations you guys had with the FDA about the 30% effect size, and had you guys kind of expected it to be in that range that you would need, or, kind of, if you could provide anything incremental there? I thought it was, I thought, um, I guess I Is it kind of a matter of just that, I think people have contested that before, just that maybe with the COVID situation, it's not the best place to put your chips, especially in the biomarker, you know, kind of probably still needs to be proven somewhat to some degree and have a follow-up likely. [inaudible]

Yeah. So my question.

I got from the fact that.

A couple why don't I can you talk a little bit about the conversations about the I see I am I right. This fact side.

Have you guys kind of stuck with it being not raising it neither competing write anything incremental there I thought it was right or not we got something below Oh, My my favorite like.

And then that they think the last night there.

Is it kinda that not only just that I think people cut that start before I guess.

Maybe that went because I think lakes and it's not that that places that your check it's actually saying maybe in the biomarker you know kind of them probably still needs to be prevented somewhat to some degree.

Follow up cycle.

[laughter] thankfully, yeah, I hope, you're saying well and great to your puppy getting bigger everyday to I will let me handle the P.H. 19 question and then I'll turn it over to Joe for a little bit more commentary on the FDA interaction.

Scott Braunstein: Thanks Alicia. I hope you're staying well and it's great to see your puppy getting bigger every day too.

Scott Braunstein: I will...

Scott Braunstein: And then I'll turn it over to Joe for a little bit more commentary on the FDA interaction. And I'll just say on the FDA interaction: we're just really happy that we got to a point with the agency where we are very much aligned about the best way to move forward with this study. And it's a complex study, so it wasn't an easy interaction, and as I said, I think I'll turn it over to Joe for a little bit more commentary on PCDH19. I would like to say a few things.

And I'll just say on the gain traction. We're just really happy that we are that we got to a place with the agency that when you're very much aligned about the best way to move forward with the study and it's a complex studies. So it if it was a wasn't easy interaction or.

And as I said, I think I'll turn it over to Joe for a little bit more commentary there on P. C. D H 19.

I would say a few things again this was the study that has.

Scott Braunstein: Again, this was a study that was challenging from the day that we started it. I think we realized it was going to take a lot of resources on a global scale, and certainly that type of study in a COVID-19 environment is particularly tricky. We have a lot of great things going on at the company, and one of the decisions I had to make from an organizational standpoint was, was this a good allocation of our individual resources?

He has been challenging from the day that that we started that I think we realized it was going to take a lot of resources on a global scale and certainly that type of study. It in a kobin 19 environment is particularly tricky we have a lot of great.

Things going on at the company and one of the decisions I had to make from an organizational standpoint is there was this a good our allocation of our individual resource dollars the side, but having you know our people get on planes and and and really happened take on some real challenges.

Scott Braunstein: Aside from dollars, but having our people get on planes and really have to take on some real challenges to then ultimately have a study that has, as I said, limited market potential. Now, that being said, we think the biomarker signal is still very important and very real. I think what we saw in PCVH19 has unequivocally driven us to TSC, and I think what we're seeing in all three of our programs, and you'll see it in our new slide deck that will come out later this morning, you can see that all these disease states really clump, so to speak, in terms of the underproduction of alloprenanolone in this young population. And so we believe there is a No question PCVH19 drove us to TSC, and no question we still think it can be a part of the story.

You then ultimately have studied that had what I would you know as I said has limited market potential now that being said, we think the biomarker signal is still very important and very real I think what we saw in P. CDH 19 has unequivocally driven us.

T S C and I think what we're seeing in all three of our programs.

And you will see then our new slide that that'll come out later. This morning, you can see that all these disease states really clump so to speak in terms of the underproduction of Allopregnanolone in this in this young population and so we believe there is a real signal there no question.

<unk> PCBK snaking drove us to T. S C.

And no question, we still think it can be a part of the story, but today, where we are from a people standpoint from a resource standpoint, we think that trend that study to a proof of concept study is the right decision for the company [laughter] and as I mentioned earlier in the marks question.

Scott Braunstein: But today, where we are from a people standpoint, from a resource standpoint, we think that turning that study into a proof-of-concept study is the right decision for the company. And as I mentioned earlier in Mark's question, I think we can quickly accelerate the TSC program into phase three on the heels, and should we see a positive phase three in CVD, certainly we'll have proof-of-concept data in-house around year end, and be able to share with you on TSC early next year. But we'll be looking very aggressively to move that program forward, and should we see positive signals from our other trials earlier. Let me turn over the FDA question to Joe. Joe, do you want to take that? Sure.

I think we can it quickly accelerate the pet food program into a phase three on these yields and should we see a positive piece Threem TVB and certainly we'll have proof of concept data in house around your end and be able to share with you on T.S.C. early next year, but we.

We'll be looking very aggressively to move that program forward.

Sure you shouldn't see positive signals from or other trials earlier I'll, Let me turn over the FDA question to Joe Joe you want to take that.

Sure Yeah.

No we had up.

Joseph Hulihan: We had a collegial discussion with the FDA about the trial design, and we gained, I think, agreement on the major outlines of the study. I think we're working out details at this point. It's a complex trial; it's not a well-worn path. But it was, you know, a collaborative meeting with suggestions from the FDA about, did you consider this and that? And so I was very pleased with the outcome of the meeting. We're continuing to talk with the FDA, and hopefully, I think we'll have the final protocol. The Vodna Protocol will be finalized soon, and we look forward to sharing all the details once we do.

We Joel I called Collegial discussion with the FDA about the trial design and we gained I think agreement on the major outlines of the study I think we're working out.

Details at this point.

No there it's a it's a complex trial, it's not a well worn path.

But it was you know collaborative meeting with suggestions from the FDA about did you consider this not and so while I was very pleased with the outcome of the meeting.

We're continuing to talk with.

With the FDA and.

Hopefully I think we'll have the final protocol.

While the critical finalized soon.

And I look forward to sharing all the details once we do.

And then that the follow up or is there an animal on the and the trial the email [laughter] [laughter].

Joseph Hulihan: And then can I ask a follow-up question? Is there an animal in the trawl that you mentioned? I'm sorry, I'm going to pause now. Is there an animal in the mouth?

[laughter].

Per status no.

Alethia Young: Um, for status, no. And you asked about the sample size. Yeah, I think we're still having the same Sample Size Calculation. You know that that that's holding true and that will be able to enroll again, hopefully, as Scott mentioned there when COVID-19 comes, you know, there's a recurrence. We'll have to address that, but I think we're in good shape in that regard. Thanks, Alethea. The next question is from Douglas Tsao, HC Wayne Wright.

You asked about the sample size, yeah I think.

We I.

I think we're still having the same.

Sample size calculation I think we'll.

You know that that.

Thats holding true.

And.

That will be able to enroll again hopefully here. So you know Scott mentioned or.

Uh huh.

You know there's <unk>.

Cobot 19 comes you know there's a recurrence we'll have to address that I think we're in good shape in that regard.

Great.

Thanks only here.

The next question is from Douglas Sal.

H.C. Wainwright. Please go ahead Sir.

Douglas Dylan Tsao: Please go ahead, sir.

Operator: Hi, good morning. Thanks for taking the questions. Just in terms of the status trial, I know

Hi, good morning, Thanks for taking your questions I'm just in terms of the status trial I know.

You know coming out of phase two just given the the the the results there was sort of a thought was trying to incorporate shipped into some patients a little bit earlier it sounds like in terms of the initial thoughts on the phase three it's gonna be very similar in terms of patients new name.

Douglas Dylan Tsao: The results, there was sort of a thought of trying to incorporate treatment for some patients a little bit earlier. But it sounds like, in terms of the initial thoughts on phase 3, it's going to be very similar in terms of patients needing to have failed a couple other anti-epileptics, so as well as benzodiazepines.

You have held a couple of other anti epileptic, so as well as benzodiazepine.

Douglas Dylan Tsao: So, I think that it's a good idea. I think that's a good idea. I think that's a good idea. I think that's a good idea. I think that's a good idea. I think that's a good idea. I think that's a good idea.

It is that just you know sort of trying to go with that replicated study that had you know incredibly strong results or were there. Some other thoughts in terms of the ability to execute a trial just given you know cobot 19 that might be a little bit more complex. Thank you.

Scott Braunstein: Is that just, you know, sort of trying to replicate a study that had, you know, incredibly strong results? Or were there some other thoughts in terms of the ability to execute a trial just given COVID-19 that might be a little bit more complex? Thank you.

Thanks, Doug I'll kick it off and then I'll turn it over to Joe as well I think first and foremost we were surprised in the phase two dogs that <unk>.

Scott Braunstein: Thanks, Doug. I'll kick it off, and then I'll turn it over to Joe as well. I think, first and foremost, we were surprised in Phase II, Doug, that our Phase II design allowed intervention after one failed therapy, one failed anti-epileptic, and we were somewhat surprised that 14 out of 17 of those patients had failed at least two prior anti-epileptics. Part of our thinking for this phase three is we want to really show the value proposition, the health economic impact. And I think we feel very confident, particularly from the data in the public domain and all our experience now with the medical community, that if these patients have failed a benzodiazepine and two prior anti-epileptics, their probability of responding to a fourth agent is extremely low, and that's really driving our thinking around the placebo rate. So as we think about a placebo or a delta of 30%, I think we can very well absorb a much higher placebo rate than we would expect to see in the real world setting. And that was, I think, a critical piece of the thinking process. I think the other piece, Doug, is that there is a real world out there.

Our phase to design, a loud intervention after one fail therapy, one failed anti epileptic and we were somewhat surprised that 14 out of 17 of those patients failed at least two prior anti epileptics and in most cases benzodiazepine, where the the mean was about 2.9 prior treat.

Mitch part of our thinking for this stage three and we want to really show the the value proposition that health economic impact.

And I didn't we feel very confident particularly given the data in the public domain and all our experience now with the the medical community that these patients had failed the benzodiazepine and two prior anti epileptic their probability or responding to it.

Of course agent is extremely low and that that's really driving our thinking around the placebo rate. So as we think about a placebo or a delta of 30% I think we can very well absorb a much higher placebo rate that we.

I would expect to see in the real World said, it and that was that I think a critical piece of the thinking I think the other piece studies, that's the real world out there when you talk to any one of these EPL apologists or narrow intensive is what you see is.

Scott Braunstein: When you talk to any one of these epileptologists or neurointensivists, what you see is... After a benzodiazepine, you'll get a single agent and then typically a second agent of a different class. For example, you may start with a sodium channel blocker and then move to a non-sodium channel blocker, as an example. And so I think that's the real world that we are living in, and we want to actually run the trial in that very real world. Before I turn it over to Joe for his comments, I think you bring up a really important point Doug that where we're starting is not where we want to end up. So, give or take, we know that there are somewhere between 30 and 40,000 refractory patients in the U.S. But the status market is significantly larger than that, north of 100,000.

After a benzodiazepine you'll get a single agent and then typically a second agent of a different class.

May start with the sodium channel blocker, and then move to a non sodium channel blocker as as an example, and so I think that's the real world that we that we are living in and we want to actually run the trial, that's very real world before I turn over to Joe for his comments I think you bring up a really important point.

That's where we're starting is not where we want it and up so give or take we know that they're somewhere between 30 and 40000 refractory patients in the U.S., but the status market is significantly larger than that north of 100000, it's the second largest.

Scott Braunstein: It's the second largest neurologic emergency in the United States behind stroke. And when we look at the ESET data as an example, three drugs that are commonly used today, none of those drugs have more than a 50% response rate, slightly below that. And so we believe we have a drug that has a substantially higher rate of success in the sickest patients. And ultimately, this drug, in our view, should move up into frontline and second-line therapy. But we think the right way to do that is to run the registration trial in a refractory population and then build on that with a Phase IV program in first- and second-line patients. And, by the way, that's a different study. That's really an emergency room study, while our Phase III will be a neuro-ICU study. So, I'll stop there. Joe, any other comments that you'd like to add?

Neurologic emergency in United States behind stroke, and when we look at the east that data as an example, three drugs that are commonly used today, none of those drugs have more than a 50% response rate slightly below that and so we believe we have a drug that has a some stuff.

And actually higher rate of success in the sickest patients and ultimately this drug into our view should move up into a front line in second line therapy, but we didn't the right way to do that is to run the registration trial.

As you know refractory population and then build on that with thing for program in first and second line patients and by the way. That's a different study that's really an emergency room study, while our phase three will be in neuro I see you study.

So I'll stop there Joe any any other comments it that you'd like to add.

Joseph Hulihan: No, not a whole lot, Scott. At the point at which we're intervening in the trial, people will be on the brink of having to go to IV anesthesia, and so if the study treatment works, we avert progression to IV anesthesia. Patients who don't respond, patients on placebo, they can be rapidly escalated to the next level of care, so and The data suggest that, as Scott mentioned, the response at that point in the progression of care is low. The study population is very similar to that in Phase II. The etiologies, we've, you know, modified that slightly. But essentially, the study population is very close to phase two.

Not no not a whole lot Scott you know the point at which were intervening in the trial.

People will be on a on the.

Brink of having to go to I'd be anesthesia and so if the study treatment works, we avert progression to Ivy anesthesia.

Patients, who don't respond the patients on placebo they can be rapidly escalate into the next level of care. So.

And.

The data suggest that as Scott mentioned the response at that point in the progression of care is low.

Study population is very similar to face too.

The etiologies, Oh, we you know.

Modified that slightly but essentially.

Study population is a very close to phase two.

And just there's a follow up.

Douglas Dylan Tsao: And just as a follow-up to that, Joe, maybe there are any, you know, sort of changes versus the Phase II sort of based on learning that you think are worth highlighting?

To that Joe maybe are there any you sort of changes versus the phase two sort of based on learnings that you think are worth highlighting just in terms of enhancing the you know the probability of success in just given the results that you saw in the in that Phase. Two study you know that's not a lot of room improvement, but just curious what types of things that you might have might.

Douglas Dylan Tsao: [inaudible]

Douglas Dylan Tsao: I'm just curious what types of things that you might have changed.

Changed.

Joseph Hulihan: Well, the drug dosing is probably the most relevant thing to your question. So, in phase two... We were lucky to have data that showed us that the key thing in terms of efficacy, more so than the total daily dose, was maintaining a target blood level. And patients who maintained a target blood level, 500 nanograms per mL.

Well the ER the drug or the thing is probably the most relevant they are to your question. So in phase two.

A good data you know we were lucky to have data that showed us that the key thing in terms of efficacy or more so than the total daily dose who is maintaining a target blood levels.

In patients who maintain the target from blood level to 500 nanograms per ml or above.

Joseph Hulihan: There were three groups, and one group maintained that level for four hours. Another group maintained that blood level for eight hours, and the third group didn't achieve that blood level at all during the infusion. The group that maintained the blood level for eight hours did better. I mean, status was controlled very well in all groups, but we had continuous EEG, and looking at the EEG, that eight-hour infusion did better in controlling EEG activity. So in phase three, because we have a new formulation, we can actually extend that to 12 hours. And that'll, we hope, give us a better chance of, quote, breaking the seizures or breaking the status. Anywhere, early in care or late in care, the idea is to interrupt the abnormal circuitry and allow the brain to reset, whether it's a benzodiazepine that's given and clears very quickly, but hopefully, it will break the status on the other end, you know, on the anesthesia.

For.

There were three groups one group maintained that levels were four hours. Another group maintained at for eight hours. The third group Didnt achieve that blood level at all during infusion.

The group that maintain the blood level for eight hours.

Did better I mean status was controlled a very well in all groups, but we have continuous even GE at looking at the G.

Eight hour infusion.

Ah did better in controlling the GE activity so on phase three.

Because we have a new formulation, we can actually extend that.

12 hours and now we hope give us a better chance of quote breaking the seizures are breaking the status any of the.

Early in care latent care.

The idea is to interrupt the abnormal circuitry and allow the brain to reset.

Whether it's a benzodiazepine.

Thats given that clears very quickly but.

Hopefully it will break the status on the other and lobby anesthesia.

It's kept onboard for 24 hours or longer.

Obviously don't continue it forever you keep the patients under anesthesia for certain period of time of enlighten that hopefully you've broken the status. So we're hoping that that 12 hours rather than eight hours would give us an even better chance to them.

Joseph Hulihan: So we're hoping that 12 hours, rather than 8 hours, would give us an even better chance to win, to intervene and stop the status. And I think that's the major difference, yeah.

To intervene and stop the status.

And I think that's okay. The major difference here.

Okay, Great and then just one maybe one follow up on the MD epilepsy program. It does it sound like after CDD, you're going to really focus much more on the sort of bought the allo biomarker sort of pathway and it how do you envision that from a red pursuing that from a regulatory.

Douglas Dylan Tsao: Okay, great. And then just one, maybe one follow-up on the...

Douglas Dylan Tsao: Does it sound like after CDD you're going to really focus much more on the sort of aloe fire marker?

Joseph Hulihan: Normal.dotm

Joseph Hulihan: How do you envision pursuing that from a regulatory standpoint? I think that, um... You know, it fits within the personalized medicine bucket, as Scott mentioned, depending on the signal from CDD. We may look more broadly.

Endpoint.

I think that.

No it fits within the personalized medicine buckets as Scott mentioned.

Depending on the signal from C D D.

We may look more broadly.

And at a bar the biomarker across developmental epilepsies, the genetic epilepsies and to further study the LLS hypothesis and so between CBD in P.S.C.

Joseph Hulihan: at the biomarker across the developmental epilepsies, the genetic epilepsies, and to further study the ALOS hypothesis. And so between CDD and PSC, I think that'll inform us well about what we do next in terms of where we go with pediatric developmental epilepsy.

Think that'll inform us well about what we do next in terms of where we go with pediatric developmental epilepsy.

Great. Thank you so Mike.

Douglas Dylan Tsao: Great, thank you so much. Thanks, Doug. I noticed it's 9.30, and I want to be cognizant of the time this morning.

Thanks, Doug I noticed it's 930 and I want to be cognizant under time. This morning. So operator, why don't we take one more call. One more question come from a color and my apologies that we didn't get to a few more calls.

Scott Braunstein: So, operator, why don't we take one more call, one more question from a caller? And my apologies that we didn't get to a few more calls, but we'll be available all day and afternoon to take questions later today. So, operator, one more question, please.

But will be available all after all day in the afternoon to take questions. Later today, so operator or more question. Please.

Operator: Thank you. We have a question from Michael Higgins, Landenburg, and Taubman. Please go ahead, sir. Morning guys. Thanks for speaking one more in here. Congratulations on the continued progress despite the

Thank you.

A question from Michael Higgins Landenburg Thalmann. Please go ahead Sir.

Morning, guys. Thanks for sneaking one more in here congrats on a continued progress despite the challenging conditions. A couple quick questions on the pipeline development I'm Marigold, you mentioned the enrollment into the open label extension is high can you give us some more detail as to how high that is what might that might be.

Michael Higgins: I'm Merigold. You mentioned that enrollment into the Open Label Extension is high. Can you give us some more detail as to how high that is, what rate that might be?

Scott Braunstein: Thanks, Michael. I think starting off, I'd say we would expect in any study where there are not a lot of opportunities or treatments out there for children with a bad seizure disorder, the open-label numbers to be high. We're not giving those numbers specifically, but certainly, there's nothing that we've seen on the open label side that gives us reason for concern, and I think we have to take those results cautiously. I think on the discontinuation side, that's an important one that we're also looking at, and we know the side effects and the tolerability of the drug extremely well, and again, we're very happy that we're not seeing anything that's surprising there. And I think one of the things we had in our trial design was a lot of flexibility around dosing, and so

[noise]. They thanks, Michael I think starting off I'd say, we would expect and any study where there's not a lot of opportunities or or treatments out there for four children with with a bad seizure disorder. We would expect the open label on numbers to be high.

We're not giving those numbers specifically, but certainly there is nothing that we've seen on the open label side that makes us.

Gives us reason for concern and I think we have to take those.

Those results you know cautiously I think on the discontinuation side and.

That's an important one that we're also looking at and we know this side effects and the tolerability of the drug extremely well and again, we're we're very happy that we're not seeing anything that's surprising there and I think one of the things we had in our trial design was a lot of flexibility around around dosing and so it's something I think.

The investigator community very comfortable with as well. So so we're going to say right now Michael but but thanks for the question and Ah.

Dan that upgrade I'm going to just go to closing remarks.

Michael Higgins: [inaudible] Okay. Bye.

Okay.

So I just want to thank you all for joining us today for our first quarterly conference call.

Scott Braunstein: So I just want to thank you all for joining us today for our first quarterly conference call. 2020 will be an exciting, milestone-rich year for Marinus, and we thank you all for your continued support and confidence. We look forward to providing updates in the coming months as we advance Gnatsalone to a potentially transformative treatment for rare and seizure disorders. Sasha has an upcoming event that she wanted to discuss with you, Sasha?

2020 will be an exciting milestone, which year for Marin us when we think Youre all for your continued support and confidence we look forward to providing updates in the coming months since we advanced connects loan to potentially transformative treatment for rare in seizure disorders.

Russia has an upcoming events that she wanted to discuss your view Sasha.

Sasha Damouni Ellis: Thanks Scott. So, on June 30th, we will be showcasing our clinical efforts with Ganaxalone to improve the current treatment landscape, and we'll highlight commercial updates and potential pipeline expansion opportunities during our virtual R&D day. The event will include presentations from Marinus Management, Key Opinion Leaders, and the Advocacy Group. Thank you everyone for joining the call today. This concludes today's teleconference. You may disconnect your lines at this time. Thank you for your participation, and be safe out there.

Thanks Scott.

So on June Thirtyth, we will be showcasing our clinical efforts that can act alone to improve the current treatment landscape and we'll highlight commercial uptake on potential pipeline expansion opportunities during our first of all our indeed day.

Yeah that will include presentations from Mariner management key opinion leaders and ethic secret.

Thank you everyone for joining the call today.

This concludes todays teleconference. You may disconnect. Your lines at this time, thank you for your participation and be safe out there.

[noise].