Q3 2021 Marinus Pharmaceuticals Inc Earnings Call
This momentum throughout the end of the year and into what we believe will be a pivotal 2022, as we await FDA action and plan for our evolution to a commercial stage company.
None of this would be possible without the hard work of our dedicated Mariners employees and the support of our advocacy partners. We look forward to the exciting developments over the coming months and we want to thank our shareholders for their support and encouragement operator can you now open the call for questions.
Absolutely if he would like to ask a question. Please press star one on your telephone keypad.
Again, Thats star one to ask an audio question. Please hold while we compile the Q&A roster.
And your first question comes from the line of Jin Li with <unk> Securities. Please state your question.
Hi, Thanks for the update and for taking our questions.
So.
So the first question how has the NDA process and so far specifically do you need a preapproval inspection and if so has that been done yet and has there been any changes to the need for the AD com.
Thanks Joon this is Scott.
Good morning.
We're having we lost our Internet service throughout the greater Philadelphia area. So many of US are just using our phones. This morning.
So let me let me walk you through what.
And where we stand with the FDA.
We filed the NDA.
In the summer in July and since that time, we've had very constructive dialogue and responses from the FDA. We held the mid cycle review meeting with the FDA. This week.
FDA has been asking as expected several questions of the filing which we're taking is very positive they are engaging and interacting.
They have.
As for several clinical site reviews. The first has been completed.
With really no no issues raised not surprisingly they are looking at our largest clinical sites as of today. They have not asked or in manufacturing inspection site, but we are preparing for that.
They have confirmed in our mid cycle review meeting that at this time there are not requesting a panel. So we feel as though we are on track as expected for an action date.
In the March timeframe of 2022.
And we've been incredibly pleased with the interactions in the agency <unk> been a great partner I think David asked very fair questions and.
Our team is working to answer all those questions.
A normal process in terms of the review Joe is there anything you wanted to add that I didn't comment on no.
I think you mentioned Scott I mean, we've got the.
Intensive inspection readiness.
Activities going on.
And the interactions with the agency.
Very productive and cordial so we're optimistic.
Great. Thanks for all the color and just wanted to add one more question regarding your natural history study that Youre doing in collaboration with Mineral Foundation, you mentioned other biotech organizations are they by the company and its.
So do they all have also congressionally trust activities.
I understand the dynamics there. Thank you.
Yes.
I think all but one have.
<unk> stated commercial interest.
And CBD, if I recall, Scott is that correct.
When we looked at that yesterday.
And one way or another or more broadly in epileptic encephalopathy.
And I would include different approaches some are pure approaches to the seizure activity that that these children.
<unk>.
Have others are looking at genetic approaches to really correct the disease in its entirety. So I think it's in.
It's a broad based industry a collaboration with the Lulu Foundation and.
And in June it's interesting because.
We now take for quote unquote granted that we have.
The opportunity to receiver.
A rare pediatric disease voucher for this disease state, we have a lot of that to Alex Betty our head of scientific affairs and the work that he did because there's really very limited public data on the natural history of this disease. Our phase three study is actually though one of them.
The largest natural history databases for patients who were treated with placebo.
A large database in Australia, but really limited information and so that was a critical piece for us putting all those pieces together to get Alex did an awesome job and are in a critical piece of the fda's understanding and granting us the priority disease.
<unk>.
Scott.
No.
Alright.
Okay.
Yes.
Yes, yes, I reminded there is one point.
Yes.
This is really aims candid study is really aimed at the non seizure.
Aspects of the disease and developing disease modifying therapies. So I'll stop now I'll, let you go.
I was just curious if the opportunity was actually much bigger than we had modeled or if you are hoping to find.
Pockets of patients that are yet to be discovered through this collaboration but.
Just let me take that so many companies interested in this.
At <unk>.
Supposedly ultra rare disease. So thank you so much.
Sure.
Your next question comes from the line of Marc Goodman with SBB Leerink. Please proceed with your question.
Hi, Thanks for taking my question, just really on a line for Mark.
Can you provide more color on the site activation for the raise trials.
And do you have any overlap in the U S side the crowd. The three studies that raise raised III under resale trials. Thank you.
Sure.
At the beginning of getting the study started we were really confronted with widespread.
Covid and distraction from.
Our clinical trials and and problems like status resources in hospitals being diverted to COVID-19.
So we have started to take there.
Very aggressive approach.
Toward.
Communicating with sites and doing.
Hey.
Listening tour as it were virtually some some will be in person.
Each hospital is different and requires different solutions.
So we're actually talking about.
Staffing up to address that having our physicians address it as well.
Part of it.
Relationships as well getting in front of the site and speaking with them. So we are starting to show some initial results.
We've got over 60 sites activated and.
Optimistic that we're on track to complete enrollment on time by the end of next year.
Yes, let me let me add a few comments just to clarify the 60 sites. That's our year end target. We're at 44 today, but we've got another set of sites that are really ready to go and our clinical ops team is moving in that direction.
I think they are a big part of the question too is how are we looking at sites across the three studies as a reminder, the reset trial is going to be specifically ER based trial those patients who are coming in with convulsive status.
We are using just a small number of sites that were very active in the <unk> trial.
And.
And that group of patients to a very large degree don't overlap with the phase three raise population <unk>.
Two trial, we're going to kick that trial off in Europe.
We're going we will add U S sites, but we don't plan on adding U S sites until the rave trial is very close or at completion. So from a company perspective, our first goal. Our first priority is raised and that Registrational trial.
The <unk> II trial is critically important for both European approval and market expansion, but we will start with key European sites and as a reminder, standard of care is slightly different in Europe and reset in 2022 will really be just a handful of sites that are in the emergency room, and we don't see.
A meaningful overlap in fact, when we talk to physicians in the emergency room they.
They tell us they really get one shot at these convulsive patients.
Which is why we'll be combining <unk> with standard of care.
Very different than our our raise population that largely nonconvulsive population that will fail one or several.
Therapies before moving to general anesthesia, Joe I Hope I Didnt cut you off were there other comments that you will have to make.
No just to add a little bit of detail on the reset study that's going to involve substantial and so if you really look at the.
Patient pathway when they come to the emergency room.
Those patients as Scott mentioned for the reset trial are really a different population we don't expect.
Either the reset trial to compromise enrollment in the res trial at shared sites.
Patients different treating physicians.
Ultimately if we achieve both indications I think there'll be.
Pretty much separate populations.
Thanks, that's very helpful. Just a real quick follow up question. So we recorded collaboration revenue of $9 million a quarter should we expect similar revenue for that line in the coming quarters.
Yes, Hi, this is Steve I can I can weigh in on that so the $9 million that we brought in and recognized as revenue was really primarily related to the upfront associated with the Orion deal. So that's an allocation of kind of the value.
That we that we take out the upfront of the deal so.
So that's I view that as more of a one time as we get through some of the MTO metabolite work, which is related to the claw back and other actions, we will have periods, where we are.
Allocate more revenue from that deal over but thats not an ongoing number where we would expect to see the ongoing revenues coming in is once we have sales will have royalties there'll be other milestones going with that but thats more of an upfront deal accounting activity than anything else.
Thanks, Thanks for the color.
Your next question comes from the line of Joseph <unk> with.
With Cowen <unk> Company. Please state your question.
Hi, there good morning, and thank you for taking my questions. The first one just on the next generation formulations, I know youre going to events to enter the clinic next year is the plan to kind of assess those and take one forward or is it possible that they would have profiles that may be amenable to sort of.
Specific epilepsy disorders, you would take them both forward.
And then second when we think about the launch and in CBD is there a patient population that would be sort of the earlier adopter Argus patients built up or.
Would it really just be on the physician and patient preference once launched.
Joe why don't I kick it off and then I'll turn it over to you.
Let me start with the latter question, Joe and good morning.
CVD.
As a reminder, our phase III population the average age of that of that patient group was six years old.
These children typically present very early in life.
Genetic testing more often than not by the age of one to two will by the by the published literature will have a very high failure rate about 85% of the patients will fail anti epileptic therapies at the end of the year and so our expectation is that this population.
We'll be presenting after several failures relatively early and it's interesting Joe because we're putting a lot of thought into that process now and continuing to look at the data.
We'll start to have ICD 10 data by we've had it for about a year, but we're really going to look at that data very closely at year end going into the launch, but our general expectations. Early days of this launch is going to be a younger population. They are in many of them are seen or taken care of.
In centers of excellence.
However, we really feel as though we can we can hit.
We can interact with a significant number of physicians, who are really treating the bulk of these patients.
On the second generation.
Compounds.
I'll turn most of this over to Joe but.
What I would say is right now I think we're looking at second generation programs that would target both.
Very friendly pediatric dosing schedule and in the adult dosing schedules. So right now we're thinking about this being a sprinkle in the pediatric population and of course, a solid dosage formulation in adults and that more than likely for us to get to a modified release formulation.
<unk> will probably take the form of two different products at the end of the day.
Right now.
We very likely will be able to test three three.
Three different formulations in 2022.
And the key for us is going to be replicating a PK curve that we think is differentiated compared to the first generation Kodak's Lone star.
Start to think about the modified release profile start to think about.
The rate titration schedule with the new formulation and of course moving into the correct patient population Jojo.
Joe who and maybe I'll turn it over to you any other thoughts on.
What do you think.
As an epilepsy, just about the CVD population or any other comments you'd like to make on the on the next generation program.
Yes, Thanks Scott.
One of the principles for managing these refractory epilepsy that you'd be able to have dose individualization for patients tolerating the drug to go up on the dose.
And.
I think that.
Having a new formulation, which got consistent delivery and.
Linear.
Dose exposure relationship.
It will really be a big advantage and then.
Alternatively.
And modified release profile that allows at least twice a day dosing.
In terms of CBD, one of the principles of epileptic encephalopathy says that the seizures, where seizures caused the other neuro developmental impairment to be worse I think that supports from a clinical standpoint that we will see early patients are younger patients treated early to.
To try and aggressively control the seizures and potentially <unk>.
<unk> developmental or slow developmental deterioration.
Perfect that is very helpful. Thank you very much again.
Your next question comes from the line of Andrew Tsai with Jefferies. Please proceed with your question.
Hi, This is <unk> on for Andrew. Thank you for the update today you have a question about the CBD program. So youll be providing the metabolite data to the FDA, maybe two or three months before them to do.
Do you think that Tony it floats and what drives your confidence that this won't be an issue.
Paducah extension would be unlikely and we at because you've seen quite a few can do pension this year.
Yeah. Thanks for the question.
And just to clarify we have now begun the MQ metabolite work.
You've talked about having it available publicly early in Q1 and that is 100% on track.
The <unk> metabolite issue has been one that has kept me very busy as well as the CMC team in our in our preclinical team from the time I joined the organization because at the time that I joined two years ago.
We had not done a thorough metabolite work of <unk> loan.
We did the first detailed work in 2019, where we saw the same two metabolite and from that period on we've had very constructive dialogue with the agency about it we put a ton of resources behind it.
About a six step process to synthetically recreate the <unk> metabolite.
So that in of itself is a major undertaking.
We are now we spend a meaningful amount of resources to actually now synthesize it in the lab. So that we can run the appropriate tests to UBS agency comfort now I'd also say, we've treated 1900 patients with <unk> and we feel as though it's got an impeccable safety profile, but because we.
Haven't metabolite, which is greater than 10% in the blood there's an FDA.
Regulation about the work that needs to be done. So we talk with the agency we aligned on our plan over a year ago, a big piece of that plan with showing that <unk>.
<unk> metabolite does not have activity to geologic activity and we've demonstrated that.
And we've also walk the agency through the plan that we would have in terms of better better.
Aligning with prior guideline.
They have been very supportive of our plan.
This will be a relatively.
Short single Page report.
Throwing that it has any.
Toxic.
Tox risk.
And.
And we will actually.
In all probability request a type C meeting to really review the long term plan for our metabolite worked with them before the <unk> date. So they are expecting this work we don't see it as a major amendment.
Right now we've provided them quite a bit of data and certainly they have not yet asked us about it but we are.
Very prepared for them to ask questions about this and have these results in hand to share with them over the coming weeks. So it's one they've been a great partner I think it has helped us quite a bit to be very.
Open with the agency.
We continue to have very good dialogue. So we don't see this.
As an issue that that will that will cause a delay in the approval one never knows.
But certainly given the interactions to date, we don't see it as a major risk.
Okay, and then you want to add.
Is there anyone out there.
Scott already covered it.
Thanks.
And thanks for that and just a follow up on the commercial opportunity here. So how comfortable are you with 2022 consensus sales numbers.
Consensus is maybe at $3 million for third quarter and $5 million for fourth quarter, and you've mentioned how the conversion of patients from your <unk> program to take six to nine months. So I just want to manage expectations here.
Okay.
Should we be expecting some inventory build in third quarter.
Okay.
Steve do you want to talk about the inventory.
Yeah, I can touch on both of those so we're certainly still in the planning stages finalizing pricing. So I think it's premature to put out any kind of revenue guidance at this point, so I think more to come down the road.
But obviously with the <unk> date in March and DEA scheduling, taking three months, we're looking at kind of a mid year launch timing for the U S market. So there will be some some period than when we're working with payers and other things. So it will take some time to ramp up our patients and really have kind of significant revenue streams.
In terms of inventory, we're certainly going to want to make sure we have.
Adequate inventory in place for a strong launch we're looking at our EAP and open label patients and helping to transition those as quickly as we can if approved so.
We will make sure that we have adequate plans in place I think that's that's probably as much as I can say on that one.
Yeah.
Yeah, the only thing I'll add to Steve's comments is that we've been pretty transparent that from the time of DEA scheduling.
Which we would expect to be a little before July one window. Purdue date is march 20th in and so.
We look for DEA schedule and the end of June we have been very clear that it will take three to six months for payers to really start reimbursing an excellent. So I do think that's an important consideration, but we have quite a few patients on an open label extension, we're getting increasing interest in the expanded access program.
<unk> every month, which is great to see we've been able to really make the expanded access program incrementally easier so for us in 'twenty two it's really about patient identification, making sure. There's good access for patients and we're quite confident that there is a meaningful unmet need these patients are well identified.
And the commercial team will be in <unk>.
<unk> position going into the second half of 'twenty two.
Alright wonderful thank you.
Your next question comes from the line of <unk> Yang with Cantor Fitzgerald. Please state your question.
Hi, Thanks for taking my question. This has been out in front of it Dan.
Maybe another one on the new formulations.
Any chance that there will be more than Q formulations that enter the clinic next year and overall what do you think is the cadence over the next year it's fair.
<unk> formulations.
Well thanks for the question.
We really run the process of our new formulations in parallel we've looked at several as Joe mentioned, we've looked at several drug delivery technology platforms. We do believe that two of those specific platforms will go into the clinic in 2022, we're also running in parallel.
Drug work and we do believe we will have a candidate selected in 2022, although I'll be honest I think it's.
It really depends on what the final pro drug looks like and how much preclinical work, we would have to do and it's much harder to predict whether we would absolutely have a third in the clinic in 2002 and that would be really around the pro drug work respectively.
Again, how much preclinical data that we would require how different the molecule looks like good excellent and what the FDA regulations are so it is a little bit early and so I apologize if I jumped the gun a little bit there, but certainly we feel very confident about having three different platforms.
Well define it in 'twenty two on the clinical team has been really terrific Joe.
Our new head of clinical operations.
Mark.
Has done a great job thinking about our clinical program here, we've had contributions across the organization as I mentioned earlier and we think we can do really relatively straightforward phase one work to understand what.
What the PK is looked like of this new compound and that and given we know so much about the PK of can exelon in so much that we've learned over the last few years, it's really going to help us we believe accelerate the programs.
Joe do you want to you want to add any comments around the clinical development programs.
I mean I think.
There are number of different pathways that we've mapped out for potential clinical development.
Okay.
The timing it depends to some extent on which one we take whether we do a full blown.
Phase two study or which even if we do a full phase III with some dose exploration and I think that we would do that as efficiently as possible potentially with.
Novel trial designs or something else too.
Speed the time to complete it or we could do additional phase one work.
And then go straight to phase III. So there are a number of different brands.
A lot of it depends on the profile, we see in the first in human studies.
Okay. Thank you.
Yeah.
Your next question comes from the line of Jay Olson with Oppenheimer. Please state your question.
Hey, guys. This is Matt on for Jay Thanks for taking our question.
So we were wondering if you could just please speak about any physician feedback that you received at the neuro critical care Society meeting in October about the basically RSC data.
And then also what kind of data or analyses, we could expect to see at Aes in December that would be great. Thank you.
Yes.
We had very productive meetings with the neuro critical care Society.
We really concentrated on speaking to.
Current investigators and potential investigators about the phase III study.
Our scientific Affairs group, Alex <unk> Ryback from our scientific Affairs group led a focus group about the phase III study. The feedback was very positive we are getting positive feedback from sites across the board about the study design and about the.
Ability to execute the study.
We did identify.
Some potential additional sites.
And we're looking at the types of sites.
Weather.
We need to go to Quaternary kind of referral centers, but maybe large academic hospitals and.
Community hospitals.
The rural or urban settings, who see a range of patients and take care of them themselves rather than transferring them out.
B the places to go and we identified two sites and have a profile.
Different type of site, we're looking at several of those so we got great feedback across the board and great ideas in terms of.
Initiatives at sites.
To help them with enrollment so it's a very productive meeting.
Joe and the highlights on Aes.
Sure.
Yeah on the embargo that you wanted to talk about.
Yes, no we will be presenting data across the board from.
Our programs.
In terms of TLC and the.
Our CBD program.
As well as the IV program, So really we had as we said.
Nine abstracts submitted and accepted for presentation.
Okay got it that makes sense I appreciate you taking the questions.
Yeah.
Your next question comes from the line of Brian's Corny with Baird. Please proceed with your question.
Hey, good morning, everyone and thank you for taking my question just wanted to get a little more color on the new formulations like moving into the clinic and maybe you could just help us understand sort of the draw.
Drivers as you see in terms of some of the safety issues on the anti seizure effects. I know you said, you're kind of targeting minimization of peak to trough exposure.
I guess is there a.
Specific plasma exposure level at peak that you kind of consider too high and what plasma exposure level, you really kind of want to be out of it.
12 hours post dosing or are any interim period to kind of be within your target profile in humans.
Yes.
Scott you want I can start off a little bit go ahead Joe.
I'll kick it off so we're finding one of the things about the current formulation patients.
Patients respond some patients respond to relatively low exposures.
On the flip side, some patients have high exposures and tolerate the drug very well and so I think the responses are.
In terms of especially in terms of tolerability differ by patient population.
Perhaps a GSE.
<unk> has more of a sensitivity to side effects like somnolence, whereas lgs maybe more.
Maybe closer to the CBD population.
So in general the midpoint of the range, we see for efficacy is around 140 150 nanograms per ml.
We'll get a better idea of Tolerability.
<unk>, we want to go for I think above.
Patients tolerate three hungry, but on a population basis may be closer to 200 is what we want to look at but then again as I said with the ability to go up higher for patients, who tolerated and who need the extra control and so ultimately the peak to trough variability would be read.
Reduced we think by a modified release formulation and so again.
Immediate release targeting a profile that lends itself to that perhaps a higher <unk> than we use in immediate release, but that may lend itself to increasing.
The area under the curve modified release.
Formulation.
So Scott I don't know if you have anything to yes. Thanks Joe.
Add a few things so I think you raise a great question Brian.
We have to do this and progressive step so step one is showing reproducible bioavailability.
And.
And so we wanted to make sure that all patients are achieving similar blood concentrations no patient has left behind which has really been the problem with the current formulation roughly 20% to 25% of patients are just not going to achieve adequate blood levels and almost every study we looked at where the drug has quote unquote.
Failed, we've seen serum plasma concentrations below 50 nanograms per ml.
And I think to a large degree the titration schedule has helped that quite a bit in terms of tolerability and more patients getting to serum concentrations that can have therapeutic effects.
As one piece, but.
Back to the specifics we want to see that.
That reproducibility across all patients we are going to look for higher C. Max is in this phase one study, but really that's a function of <unk>.
Seeing better bioavailability.
From the get go is something that we feel we can reproduce and then to Joe's point once we achieve those higher C. Max as we can then use.
More gradual titration schedule too tough to replicate that in all patients and our CMC team has is already starting to do the work on how we would then modified the release and in our internal team and they've done fantastic work really believe as we have a high probability of success.
Modified release.
With different technologies out there, but we've got one or two that we've already identified so.
Omar without better bioavailability and I think once we see that in the phase one studies, we will really be able to move aggressively to an MLR strategy and I think what's equally important is that.
We understand that if we wanted to be competitive in lgs.
Twice a day schedule is really important to me if you go back and look at the history of this molecule and the sale of the phase III and focal seizures that was a commercial decision that the company needed a <unk> formulation.
Adam was they didn't have the right formulation for VIP. So I think you've got to have the right formulation going into a <unk> schedule.
We're confident we can do that in conjunction with the ongoing development program. We think we can get real proof of concept data with what would be an immediate release formulation.
And begin that work in 2022, but ultimately our goal would be a phase III study with a modified release formulation.
Again, minimizing certainly peaks and minimizing troughs, and then being able to target a 100 or 150 or 200 nanograms per ml.
In that type of.
In a phase III design, and I think that will yield unquestionably the strongest efficacy results doing it with the appropriate titration schedule will certainly maximize tolerability and I think if you do both of those things we've got to we've got a really competitive and exciting oral franchise.
Alright, Thanks, Scott that's really helpful. Thank you.
Your next question comes from the line of Jason Butler with JMP Securities. Please proceed with your question.
Hi, Thanks for taking the question just one on.
Partnership priorities for China, and Japan, just any thoughts you have at this point about how much additional clinical data you would need to support regulatory approvals in those countries. Thanks.
It depends Jason its great question.
In China, there is a SaaS path for orphan disease indications.
Indications, where the Chinese government has specifically.
Already anointed those diseases.
And Trc is on that list as of today CBD is not on that list, but something that very important for us to discuss with the advocacy groups to try to move.
To try to add CBD to that list, where where U S data can be used for the registration process.
On the on the Japanese side of the equation, we've already done in house work.
Feel very comfortable that our preclinical programs are all.
Well within.
Except for an ultimate Japanese approval, our plan is to really start phase one.
Work in Japanese patients in 2022 and have an interaction with the regulatory bodies in Japan in 2022, but it's a relatively straightforward path. We've already had an outside agency confirm that pathway for us our clinical team and strategy team I think is.
Now very well versed with with that path and as a reminder, as of today cannabinoids are not CBD or not on the market in Japan, I know that the GW folks who are working very hard and have created a pathway there but as of today. The product is not on the market. So again.
The unmet need in PSC is quite high as of today, It certainly CBD as well and so we think this is something that.
As will be relatively straightforward.
We're ready to commit to the early work in.
And we will continue to have very vibrant discussions moving forward.
Corporate helps yes, just a quick follow up unless I guess theres important IP question as well, but as you think about those.
Those potential partnerships is the focus primarily on <unk> or could it also include additional formulations.
Oh, Thanks, It's a great question, it's interesting win win.
Yes.
Heavily involved in strategic partnerships at the Sierra with Chinese partners. There were a lot of folks who are really interested in being a manufacturing partner.
And I don't see that as a critical piece of the puzzle today I'm much more focused and I've asked our strategy team to really focus on partners in China, who can really help expedite our clinical.
It is in our clinical programs.
I think we've seen that work really nicely in the car T space.
And in other oncology spaces.
And we've already talked to several strategics, who do you believe they could help us on the clinical.
The clinical front one example.
We are hungry to start and infantile spasm program and we certainly think that.
Chinese partner with good clinical.
Our strong clinical team great Kols connections clearly help us expedite that program in 2022, so our focuses.
As of today is really finding a strategic partner, who has the clinical skill sets in and potentially can really help accelerate.
Our clinical pipeline.
Great. Thanks, Scott.
Yeah.
Ladies and gentlemen, please limit your question to one and one follow up. Your next question comes from the line of Douglas Tsao with H C. Wainwright. Please state your question.
Hi, good morning, Thanks for taking the questions just I'm just curious in terms of the Super Super refractory status epilepticus patients here Youre supplying drug to are you collecting the data from those and have you seen any results and I'm. Just curious is there some consistency in terms of where you are making recommendation and term.
How that should be dosed.
Yes.
In terms of the dosing, we're working with the sites.
It looks like the.
We're allowing a higher dose in the RSV study.
The cap on.
Captisol is 50 grams.
We're going up and that gives you a exposure of the maximum daily exposure around 830 milligrams you can actually we're actually going up to a little bit over a 1000 milligrams you can ask loan at the maximum dose and allowing some rebalancing for the patients who need it so.
We're still.
Trying to find.
The maximum.
Effective regimen.
Rather than waiting until the patients on anesthetics.
Looking at initiating the drug as they taper down looking at different dosing regimens bolus versus an infusion.
We haven't seen a consistent pattern.
In terms of results.
It's worked in many patients.
And some patients that has the work, but the pattern isn't consistent we've seen kids respond.
That's been one thing, but but older patients with very refractory epilepsies have also responded.
So it's been a small number of patients not enough yet to really.
Draw conclusions, but again as we said we will continue to supply it.
<unk> requested.
And it looks.
Potentially promising although as we've said we don't have any plans to pursue an indication our goal is to try it.
If we can prevent a lot of patients who are getting to that stage.
That that would be the ultimate goal.
And Kevin do you want to talk a little bit I'm sorry, Doug.
Hello, Joe respond to your last piece.
Joe what what's your do you want to talk a little bit about your thinking about a publication strategy or whether we as Meredith undertake.
Some some other path.
So have you chat about that yes.
Yes no.
Absolutely publish and present the results of the.
An entire series at the appropriate time.
We've had a case studies.
Presented.
The patients some of the patients who've done well, but we will publish the results.
And.
And put them out there.
It could be obligated to do that Scott I don't know if you have questions.
Comments beyond that.
The only thing I would add Doug is and I think it's important first of all I really want to thank our physicians on the team.
Henry <unk> module guests your who have really worked closely with investigators in these really refractory patients. Because this is a whole new paradigm right, Doug and not only are we dosing in the slightly over 1000 milligrams, a day, but often for four or five days in patients.
And.
And so the regimen will be different in one of the things I'm, particularly interested in our <unk>.
Current pro drug approach is that that project would be very applicable to an IV formulation. There may be some theoretical advantages why perjured might actually get to the brain, even incrementally faster than the next loan today, but I think our current formulation can get to the brain quite quickly.
And stays in the brain, but what I really like about our approach. Our approach is it really eliminates the need for captisol and that creates the opportunity either to really flex our muscle on dosing paradigms and so we're equally focused on moving the IV drug program forward aggressively.
To really allow us to do much more than we're able to do today with captisol. So the folks at ligand they've been great partners for us and they continue to be but.
Yes.
It's more difficult developing two drugs in one rather than than a single drug and really right. Now Captisol has created more limitations in our program then can axle itself sorry did I cut you off yet another question.
Yes.
Obviously I understand why you want to prioritize just wanted to ask about the reset and raise trials and I understand why you want to prioritize.
Getting enrollment.
And not potentially sort of impacting that with reset, but just as those trials fill up would you anticipate expanding universe of both the reset universe and.
Or are they sort of I know, you're sort of not youre targeting centers that arent overlapping is there a different profile.
Between the two trials that youre thinking about.
Well, let me just say the reset trial is a big undertaking for US Big piece of this is is gaining community consent, it's quite a bit of work I'm really appreciative of Sasha and her team and how much. She has helped with our clinical operations team. We've got to go into the community we've got a really.
Educate the community on the study we've got to get there by and we've got to run a certain number of of educational programs.
Again as these patients at the emergency room. There is no time for them to be consensus. So it's a big undertaking and our proof of concept study.
Well I think you have some great data points and we're also going to use some form of the gene that study, which has never been done before when you looked at you said they did not use the EG.
Critical outcome measure in that study. So it's one that we're really excited about I think let's be honest, Doug we're doing a lot. We have about 15 clinical trials ongoing we see this is a critically important piece of the IV story I think commercially what we want to understand is what's the right dosing paradigm.
<unk> for the frontline study when we launch into the RSC marketplace and ultimately.
We'll run the appropriate registrational trial in early status, but.
We still believe the bigger market opportunity is in the refractory setting and I think what we're going to actually sit down with our F&B and talk about is what are the other phase four strategies that we should be thinking about to really maximize adoption I think <unk>. Two is a fabulous studies to really expand the <unk>.
Use of this product in the refractory setting with the right outcome measures.
But.
These are still relatively small studies and the scope of a very common disease and I think that's going to leave us a lot of opportunity to do more post approval. The frontline setting is really meaningful to us as well but.
We are eye on the prize is certainly refractory first and foremost and in frontline and an important piece, but we've got some we've got some time to figure that one out.
Joe anything you want to add.
No Scott nothing I think that covers it.
There are no questions at this time I would now like to turn the floor back to Scott Braunstein for any additional or closing remarks.
Well, thank you operator, and thanks, everyone for the questions Ive seen that we've done well over our hours. So I'm glad for no. Other questions really appreciate all your support for the analyst community, our investors and our investors and and just wanted to say again. Thank you to the mayor and his team they've been working really hard this is an incredibly.
<unk> environment in the hospital space today.
And.
I'm really proud of how well the team is is meeting this very difficult challenge.
And we look forward to speaking to you next quarter. Thanks again.
Thank you for participating in today's conference call. You May now disconnect your lines at this time.
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Sure.
Yes.