Q3 2022 Marinus Pharmaceuticals Inc Earnings Call

Okay.

Ladies and gentlemen, greetings and welcome to the Meredith Pharmaceuticals third quarter, 2022 financial results and business update call.

At this time all participants are in a listen only mode.

After the speaker's presentation, there will be a question and answer session if.

If you would like to ask a question. During this time simply press the star key followed by the number one on your telephone keypad. If you would like to withdraw your question Press Star one once again.

And now it's my pleasure to introduce your host Sasha <unk> Ellis Vice President of Corporate Affairs, and Investor Relations you may begin to Minneapolis.

Thank you and good morning with me from Baroness are Dr. Scott Braunstein, Chief Executive Officer, Christy Schaefer, Chief Commercial Officer, Dr. Joe Houlihan, Chief Medical Officer, and Steve said, it's still Chief Financial Officer.

Before we begin I would like to remind everyone that some of the statements. We're making today are forward looking statements under the securities laws. These forward looking statements involve substantial risks and uncertainties that could cause our clinical development programs future results performance or achievements to differ.

Lee from those expressed or implied by such forward looking statements.

These risks and uncertainties and risks associated with our business are described in the company's reports filed with the Securities and Exchange Commission, including forms 10-K, 10-Q and 8-K.

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

Thank you Sasha and welcome to our call I'm extremely proud of the progress. The team has made as a commercial company. Following the FDA approval of the told me we initiated the launch at the end of July and are excited to report our early launch metrics for the first time.

In a few moments I will turn the call over to Kristy Shaffer to discuss our commercial progress, including strong early demand in payer coverage determinations. We are confident in the investments we have made in our commercial infrastructure and believe we are executing on a successful launch.

On the clinical side of the business, we remain focused on advancing our pipeline and prioritizing our two phase III programs in refractory status epilepticus and tuberous sclerosis complex.

Starting with TFC, we've been actively engaging with the community, including our participation in the recent World T. S. He conference.

After our teams interactions with both clinicians and families. We continue to believe that there is a significant unmet need for individuals suffering with refractory seizures associated with TFC and that the Tommy has the potential to be an important option for these patients and families.

Moving to our IV program as previously communicated we continue to implement important amendment to the raise protocol, including expanding the inclusion criteria with the specific goal of broadening the patient population that would be eligible for treatment with <unk> alone and accelerating enrollment in the <unk>.

Yeah.

The amended protocol has been adopted across a growing number of clinical sites and we expect full adoption by year end.

<unk> continued to express enthusiasm to enroll a larger number of patients under the amended protocol and I will leave a further discussion for Joe to review in his prepared remarks.

We continue to expect data from the raise trial in the second half of next year and data from the phase three trusts T. S. C trial in the first quarter of 2024. Similarly, the early stage programs for both the IV and oral franchises progress as previously discussed.

Finally, some comments on how we plan on bringing zoo, telling me to patients and families globally.

In Europe , we are on track to submit complete responses to the Ema's day 120 list of questions for the <unk> marketing authorization application by the end of November which would result in a cgmp opinion by the end of the first quarter of 2023.

I want to acknowledge the entire Meredith team and our partners at Orion Corporation for their tireless efforts to ensure that our responses to the ema's questions are complete and robust together with Orion. Our teams continued to prepare for a potential European commercial launch next year.

In addition, we believe that there is a broader global opportunity for <unk> alone and are exploring further ex U S. Commercial alliances we are targeting the strategic agreement for China by year end and another important global collaboration in 2023.

With the sale of our priority review voucher, the exercise of the contract option by BARDA and completion of the revenue interest financing agreement with <unk>, we have executed on our plans to extend our projected cash runway with non dilutive financing into early 'twenty four.

Creating the opportunity to secure a U S based source of API to improve our future business fundamentals.

Furthermore, with the equity offering we announced last night, which is expected to close on November 10th We now project, our cash balance to carry us into the second half of 2024.

Additionally, we are delighted to expand our shareholder base and at the same time.

Our existing shareholders for their commitment to the organization.

Upon closing the equity offering our new cash balance will allow us to continue to invest in the ongoing <unk> told me launch our development pipeline and prepare for two additional potential commercial launches.

Now I would like to turn the call over to our Chief commercial officer, Christy Schaefer for updates on the commercial launch of this told me Christy.

Thanks, Scott and good morning, I'm pleased to share our early progress on the commercial launch of the economy in the U S where we have seen continued high enthusiasm from both physicians and caregivers, reflecting the need in this community and the potential that the Tommy could provide and senior management.

We lifestyle told me on July 28, and by the end of the third quarter generated $560000 and net product revenue.

During the first nine weeks of launch we received over 80 day prescription enrollment forms more than 30 of which were for new commercial patient not previously treated with the Tommy with the remainder encompassing the transition of all U S. D. D D O L Lee and EAP patients to commercial.

Alex.

Importantly, these prescriptions are coming from a diverse prescriber base and that was 40 unique accounts, including interests from outside of our 265 target accounts. We believe this speaks to the unmet medical need and the CBD community as well as the strength of our digital marketing tools publication strategy and Rob.

Bust education and awareness efforts ahead of launch.

In addition to a strong level of new patient enrollment and prescriber activity. We're also encouraged by the early trends, we're seeing from payers with approximately 40% of <unk> patients with completed prescription enrollment forms on reimbursed therapy by the end of the third quarter.

As a reminder, we expect approximately 60% of the CBD patient population well access coverage for both fee for service and managed Medicaid with the remaining 40% being managed commercially.

As of November 1st the Tommy received positive coverage criteria from over 15 payers, representing approximately 17 million lives and we expect the majority of the remaining coverage statements in the fourth quarter.

In addition, 57% of all U S. Commercial plans have already extended label coverage to the Tommy.

We continue to believe payers appreciate the impact of the disease on patients and their families as well as the unmet medical need within the CBD treatment landscape and the Tommy profile as the first and only product indicated for seizures associated with D. D D.

The field interactions are being supported by our educational speaker programs as well as to product theaters, one which took place in October at the child Neurology Society annual meeting in the second planned in December at the American Epilepsy Society annual meeting.

Our foremost the Tommy Speaker Bureau commences in December with a meaningful number of programs planned for the end of next year.

We will continue to educate and raise awareness within the CBD patient community starting with the CDB community Webinar early next year.

We have also been actively engaging with leading patient advocacy organizations, which have proven to be an important source of two way communication.

These groups help share information within the patient community and provide a channel for feedback to ensure we're meeting the evolving needs of the CVD communities.

We're grateful for the insights and support that the IFC are PDK all by the alliance and the Liberal Foundation provides the families living with a D D.

We are very pleased with our early launch progress and are grateful for the impact. We believe we are making on CBD patients and families. We are excited to build on our strong commercial foundation and look forward to continuing to work with patients caregivers physicians and payers over the coming months.

As a final update to further our commitment and continue our progress towards building an acute care hospital franchise. We have brought on an additional commercial later to Mike Kane Christopher the cell who has a strong track record of success and hospital launch experience to support the development of our U S commercial strategy for status.

That ticket.

I'll now hand, the call over to our Chief Medical Officer, Joe Houlihan to discuss our ongoing development programs, including the data we continue to generate and presented at scientific conferences, which remain critical to supporting our launch and driving awareness of the Tommy.

Thank you Christine and Hello, everyone.

First I'd like to provide an update on our IV programs in status epilepticus and dive a bit deeper into where we stand with the rays Protocol Amendment, we announced in June this year.

As we've previously shared we broaden the inclusion criteria to permit randomization of patients who've been treated with high doses of IV anesthetics, such as protocol for up to 18 hours rather than excluding any patient who had been treated with high dose IV anesthetics, regardless of how the administration of these agents.

We believe this is an important change because it will allow for the enrollment of patients transferred to the ICU other hospitals or the emergency room two of the most common pathways for identification of refractory status epilepticus.

The original protocol that excluded many of these patients represented a significant proportion of the phase III trial population.

I think it's also important to mention that the patients enrolled prior to implementation of the amendment would still meet eligibility criteria under the revised protocol.

Scott mentioned the majority of Reis sites have implemented a protocol amendment with the remaining sites expected to transition to the revised protocol by year end.

Investigators are expressed considerable enthusiasm about the potential of the changes in the protocol to allow recruitment of a greater number of eligible patients.

We continue to expand the number of participating study sites in the U S and plan to expand the centers in Canada, and Australia, all of which we expect will be activated under the amended protocol.

In August and November we held investigator meetings in the U S to educate our sites on the updated protocol and we continue to work closely with them to support timely study enrollment.

We recently reviewed the baseline characteristics of the phase III raise trial patients we're pleased to share that as of September .

Baseline characteristics of the phase III patients closely resemble those of the patients enrolled in our phase two RSV study.

Participants in both studies are roughly the same age.

Similar proportion have a history of epilepsy and they have similar mean baseline seizure burdens and status up electric is severity.

These demographics are outlined on slide 29 of our November corporate debt.

In addition in September we presented data from the Phase II <unk> study and an overview of the ongoing raise trial at the London Innsbruck Colloquium on status uplift tickets.

The presentation received a positive response from those in attendance, who are leading researchers and clinicians in the field.

Press the raise trial represents an advance in clinical research on refractory status.

A condition for which there was no data from rigorously conducted placebo controlled studies.

We also had several Marinus representatives attended the neuro critical care Society annual meeting in October .

We presented two posters with data from the IV can excellent program, one of which was designated as a distinguished poster presentation.

There, we had the opportunity to meet with many investigators and other team members can raise study sites. Additionally.

Additionally, neuro critical care physicians from other leading centers expressed interest in participating in the trial.

Looking to the future of the rate of studies, we will continue to actively monitor what's working well with a site level and where we can provide additional support for trial recruitment and execution.

There's also the potential for expansion of the age range of eligible patients.

Currently the trial can enroll patients ages 12 years and over we.

We will shortly be submitting a request to the FDA with supporting data to potentially allows us to include younger patients.

The higher incidence of status epilepticus and children under 12, this change could expand the pool of eligible patients and further support timely study enrollment.

As previously disclosed we have.

The option for an independent data monitoring committee to conduct an interim analysis when two thirds of participants have completed the study approximately 82 patients.

At that time, if the interim is conducted the committee would recommend either stopping the study for efficacy or continuing to full enrollment.

While conducting the interim analysis may affect the sensitivity of the study to detect the treatment effect should it continue to fall enrollment.

We have designed the interim analysis for the raise study to have a minimal effect on the efficacy outcome fully enrolled trial.

We're pleased with the changes we have made to support enrollment of the raise trial and continue to expect top line data in the second half of 2023.

Planning continues for a separate phase III <unk> trial, the res III study to support a marketing authorization application in Europe .

The protocol is finalized and enrollment is expected to begin in the second half of 2023.

This trial will not only serve as a critical piece of the European approval strategy. We believe it also has the potential to further broaden the indications for IV <unk> in the U S moving.

Moving on to the phase III reset study and establish status epilepticus, we have activated the first site and expect to begin enrollment this year and we continue to advance the exception from informed consent process for additional sites.

Now I'd like to turn to our Oregon excellent programs.

As Steve stated in her remarks, we continue to work to increase awareness of Tommy across the scientific and medical community.

October 13th we presented two posters at the child Neurology Society meeting from our pivotal phase III <unk> study <unk>.

<unk> open label extension data that showed continued seizure reduction over a two year period.

Although 24 month data was available for only <unk> of the 54 patients remaining in the open label extension.

Reduction in major motor seizure frequency for this subset continued to improve with a median reduction of 53% at the 24 month Mark versus a 37% reduction at the conclusion of the double blind phase.

The discontinuation rate was about 30% during the first year of the open label phase, but declined to about 10% during the second.

These data in total suggest that patients who remain on treatment long term may demonstrate continued reductions in seizure frequency.

We also presented data from a post hoc analysis from the anxiety depression and mood scale. There was administered during the Marigold study.

It showed that <unk> was associated with improvements in certain domains of behavior, specifically reductions and the compulsive behavior and many hyperactive components of this scale.

While these findings would require confirmation through further research suggests that there may be potential benefits of treatment beyond seizure reduction.

Moving to our Phase III Trust TFC trial in tuberous sclerosis complex. We are pleased to share that we are actively screening patients.

Getting 85 clinical sites predominantly in the U S and Europe with Activations in Canada, and Israel expected by year end and Australia in the first half of 2023.

Carriage with the high level of enthusiasm in the medical community for this trial and continue to anticipate top line data in the first quarter of 2024.

Finally, I'd like to provide you with an update on our second generation formulations.

As shared last quarter, we saw promising initial results from our phase one single ascending dose study of the first of our re formulation candidates, which we believe support advancing it further in clinical development.

<unk> in the Phase one study received reformulated, Oregon actually at doses ranging from 100 900 milligrams with the current oral suspension given as a reference control.

The PK profile of the new formulation showed increases in overall exposure relative to the maximum concentration or <unk>. Therefore, this could potentially allow for upward titration beyond the dose of the current oral suspension to further optimize efficacy without producing a concomitant increase in <unk> related adverse effects.

Such as some loans.

The next steps in the evolution of this program include enrollment of an additional phase one study cohort to assess clinical doses above 900 milligrams to determine whether we can achieve additional increases in overall exposure relative to peak concentration.

We expect the data to be available by the end of the first quarter of 2023.

We will also be performing PK modeling to determine the next steps in development of this formulation, including the need for a study with multiple <unk> doses, which we would be in a position to initiate in the first half of 2023.

We anticipate selecting one of the second generation can excellent formulations for a phase II study in Lennox <unk> syndrome to begin in 2023.

Lennox <unk> syndrome is highly treatment refractory and we believe the new oral formulation has the potential to provide more consistent and predictable exposure to <unk>, which.

Which we expect will allow physicians to individualized dosing to achieve an optimal response for each patient.

Additionally, we have developed several pro drug candidates that could offer further improvements in efficacy tolerability and dose individualization as well as operational and manufacturing efficiencies.

Lead oral and IV pro drug candidates have been selected for additional IND, enabling studies with phase one data targeted for 2024.

We look forward to continuing to raise awareness within the medical and scientific community over the coming months and we will have a robust presence at the American epilepsy Society annual meeting in December <unk>.

Including presentations of data from our oral and IV can excellent programs during the general sessions as well as during Mariner scientific exhibit.

As a clinical team we remain focused on our phase III clinical programs in status epilepticus and tuberous sclerosis complex.

Advancing our re formulation pro drug candidates as well as our continued support for <unk> and CTG.

Now I'll turn the call over to our CFO , Steve <unk>, who will provide you with a financial update.

Thanks, Joe and good morning to everyone before discussing our financial results for the third quarter 2022, I would like to provide a few key financial highlights since our prior call.

As previously touched on by Scott in his opening remarks, we have been incredibly active on the deal making front over the past several months, adding over $150 million in funding to our cash position in the near term.

Specifically in August we closed on the sale of a rare pediatric disease priority review voucher receiving growth funding of $110 million.

That was followed in September with an additional $12 million of increased funding from BARDA as they exercise the first contract option to support our API Onshoring initiative.

Just recently in October we completed a royalty monetization agreement with regard to health care, which provided an upfront payment of $32 5 million for future royalty payments related to the U S revenues associated with <unk>, including the Tommy.

We ended the quarter with cash and cash equivalents of $168 2 million, which does not include the funds received from the cigar revenue interest financing agreement.

Adding the net funds from that recent deal results in a cash balance of approximately $200 million, which is projected to be sufficient to fund our operations into the first quarter of 2024 inclusive of maintaining the minimum cash balance of $15 million required under our credit agreement.

As Scott highlighted upon the closing of the equity offering announced last night, we project our cash balance to carry us into the second half of 2024.

I'll now move into our financial results for the third quarter of 2022, and I'm, particularly excited to share the results of our first quarter was the Tommy product sales in the U S.

For the third quarter of 2022, we recorded as the Tommy net product revenues of 560000, which represents just over two months of actual commercial launch this.

This revenue consists of the Tommy product sales to our single specialty pharma partner Orsini and includes an estimate of gross to net deductions inclusive of expected Medicaid discounts.

Focusing on BARDA revenues, only we recognized revenues of $1 8 million and $5 1 million for the three and nine months ended September 32022, respectively, as compared to $1 1 million and $4 8 million in each of the same periods in the prior year.

Research and development expenses increased to $19 million and $58 5 million for three and nine months ended September 32022, respectively, as compared to $18 4 million and $55 5 million for the same periods in the prior year.

<unk> was due primarily to costs associated with increased R&D head count and startup of the Phase III Trust PSC trial.

Selling general and administrative expenses increased to $13 4 million and $42 2 million for the three and nine months ended September 32022, respectively, as compared to $9 5 million and $26 7 million for the same periods in the prior year.

Primary drivers of the change were preparation for and launch of the Tommy and additional support for scale up of the company's operations.

As previously mentioned the third quarter of 2022 included a net gain of $107 4 million related to the PIV sale.

As a result, the company reported net income of $73 3 million and $14 5 million for the three and nine months ended September 32022, respectively as compared to net losses of $19 5 million and $70 5 million for the same periods in the prior year.

These totals include noncash stock based compensation expense of $3 9 million and $11 1 million for the three and nine months ended September 32022, respectively, as compared to $2 8 million and $10 9 million for the same periods in the prior year.

Cash used in operating activities was $91 million for the nine months ended September 32022, as compared to cash used in operating activities of $33 7 million for the same period in the prior year as.

As a reminder, our 2021 operating cash flow total includes $30 million of funding from the signing of our European collaboration agreement with Orion.

Now I will turn the call back to Scott, who will provide concluding remarks.

Thanks, Steve This has been a busy and exciting time for the Mariners team and we are pleased with the early launch results. The progress we've made towards a potential approval in Europe next year and the organizations research efforts. We look forward to building on this commercial foundation with the Tommy and advancing our two phase three program.

<unk> over the next 12 months.

To conclude I would like to mention that <unk> will be holding an investor breakfast Monday morning December 5th at a yes, we will be meeting with a variety of physicians and advocacy organizations over the course of the weekend and hope to see they're live Monday morning.

This meeting will give you the opportunity to meet with our commercial and scientific leadership, who have been integral to this exciting launch.

<unk> will provide more details on the event over the coming weeks.

Operator can you now open the call to questions.

Yes. Thank you.

As a reminder, if you would like to ask a question Press Star then the number one on your telephone keypad.

And we will pause for just a moment to compile the Q&A roster.

Okay.

And we will take our first question from Marc Goodman with S. V. B Securities. Your line is open.

Okay.

Good morning, Mark you may be muted.

Yeah.

Sure.

Operator, we don't hear anything.

I don't hear Mr. Goodman, either Mr. Goodman, if you can hear US. Please re queue. We will move to our next question from Joseph <unk> with Cowen and company. Your line is open.

Hi, there good morning, and thank you for taking our questions.

First one on the call me itself.

Sales are growing and it looks like consensus for the year.

Hitting around $2 5 million I guess based on what you know of a launch so far is there some number that youre comfortable with and can you comment on any.

Talking patterns that we should be.

And then on RFP.

The upper end of the enrollment for both the phase during the phase III.

We discussed potentially going down at 212 years or below is there any difference.

Difference in response prior to your to your.

Normal.

Based on.

Broad acreage that'd be helpful. Thank you.

Steve why don't I turn it over to you for does it told me question and then we'll let we'll let Joe take the RSC questions.

Yes, good morning, Joe Thanks for the question with regard to the Tommy revenues, we arent providing guidance for 'twenty, two where 23, so I won't speak to that specifically, but as we said we're really focused on the number of patients on therapy. We are focused on driving access across government and commercial payers were really extreme with I assume.

We are happy with our progress on both those fronts. We felt like we really have a really strong trajectory there and justice first couple months of launch and Additionally, as we mentioned we'll be launching speaker programs. Later this year, we're going to have a strong presence at aes. So.

We're really bullish on our launch trajectory here, but we're going to hold off on guidance for now in terms of inventory impact.

We're working with a single specialty pharma partner, we have a really tight distribution.

<unk>.

Think of our warehouse and our specialty pharma partner being within just a few hours of each other.

Given that we measure the inventory impact on the order of weeks not months.

That's going to be the case for the foreseeable future. So.

Very limited inventory stocking impact and that will be the case moving forward.

Hey, Steve before we turn it over to Joe maybe you want to remind Joe told me in the audience about our what we've talked about breakeven and how we're feeling about that relative to the first few months of launch.

Yeah Joe.

To add a little more color in terms of the number of scripts in that trajectory.

We've said that we think this $25 million investment in the CBD commercialization, it's really a two year to breakeven type of investments so by the middle of 'twenty four.

We should be annualizing at a sales run rate of around $30 million.

That with a 90% gross profit margin on the product should cover that $25 million commercial investment, we think it'll be a pretty steady build.

Over that time timeframe, and we think that that breakeven at $30 million happens probably at around 240 to 240 to 250 million page or 240 to 250 patients on therapy.

Great.

Joe before I turn over to you I just wanted to comment a little bit for Joe to them in the folks in the audience that.

The raise trial itself, primarily we're using adult sites, but we have about 10 sites who are in the pediatric age range, typically 12 and above and so the extension of the trial to younger patients would primarily affect those sites that right now have a limited 12 and above.

<unk>, but in terms of our expectation and the success of the drug in younger populations I'll turn it over to Joe and Joe You May also want to comment on some of our experience and Src with with the Maxwell.

Yeah, no absolutely Scott.

We don't I think what you were asking about Joe there was a little bit of background noise.

About.

There.

Response in children compared to adults.

We don't expect our children.

To do certainly any worse.

At least as well as adults and we have had some we've treated now about 14 patients under emergency IND.

For Super refractory status, and we've had some great responses and the kids.

And so we expect the kids who enrolled in the trial to do well and actually this amendment should give us.

Good boost in terms of the eligible population because in terms of kids status is.

More common in kids under 12, then in adolescence.

So we're hoping to go down to age two.

And we're sending requests the FDA very shortly about that.

Perfect. Thank you very much it's very helpful.

And we will take our next question from Andrew Tsai with Jefferies. Your line is open.

Alright, Thanks, and good morning, Congrats on all the progress maybe one on CBD another one on RSC.

On the CD launch.

50 patients seems pretty strong as of September 30th.

Previously you said not to expect a bolus is that still the case for you guys and just curious, whereas the total patient count today relative to the 50 as of September 30, and then secondly for RFC can you just remind us the latest and greatest on what you expect the plus.

Zeebo response rates could be for the two co primary endpoints and why that should be the case so percent of patients having a cessation within 30 minutes and then percent of patients not moving to IV anesthesia that'd be very helpful. Thank you.

Thanks, Andrew Joe you want to take the first question on RSC and then we can turn it over to Stephen Christiane TDD.

Yeah.

So.

We were very conservative in terms of sample size estimation for the trial.

We got.

You have the placebo response from our investigators and our advisory Board.

And for the first co primary endpoint response within 30 minutes.

I think the placebo response, frankly, I hate to be overconfident, but it will be negligible.

The IDE.

The thought that status would stop on its own.

Within a half hour.

I'm confident about that.

So.

Conservatively, 10% placebo response potentially.

The second co primary endpoint escalation to IV anesthesia again.

We surveyed.

Our sites and.

They said, we gave them the profile of the patient coming into the study.

That's where we got the estimate the sample size calculations based on up 45% placebo response in the 75%.

I mean, 75% placebo response studies progression to IV anesthesia.

45% progression in the can ask alone arm.

I think that's extremely.

Conservative.

So the 30% Delta and actually that gives us 90% over 90% power and we talked about the interim analysis. We also that's also well powered.

That's the point, we get to 82 patients.

I think.

That's that's still gives us a very high amount of power to detect a treatment difference.

Yeah.

Steve you want to kick off on the numbers and then we'll turn it over to Christine for any other comments.

Yeah sure sure Andrew I think in terms of the bolus, maybe I'll start there we had just over 20 patients in our EAP open label program that would come over over time so.

It's not a huge bolus that we'd expect from that program and I'm not sure. If that's what you were specifically referencing but I think the other piece that's really important to see is when we had talked about this previously we had said hey, it could take three months or more on average for patients to work through the reimbursement process. We know payers will put LTC blocks or other things in place, which just can take some.

Time to work through.

I think our payer team has done a really phenomenal job of really being out there getting good patient access I think our pricing strategy has paid off extremely well.

Extremely well and in fact, we're seeing about 50% of our patients being reimbursed and approved within 30 days.

Some are going to take longer but we're really proud of what we've been able to do so these patients are you just going to come over.

I'm in over time as they work through that payer process, but.

To have that many patients on after nine weeks I think shows the work we've done on the payer side Christy I don't know if you wanted to add anything there.

Yeah. Thanks for the question, Andrew I think that.

Dave hit it on the on the head there however, just to reiterate that as number 20 patients and our AA Pan allele programs.

With just over 30 patients that were naive to <unk> of.

Of those 30 patients.

We saw a nice balance of when we saw those enrollment forms. So there certainly was no bolus has done in the first couple of weeks and we are continuing to see interest that is equal to that that we saw early in the launch day. So only a few weeks and but we're encouraged by the effort as well.

Thanks for the color. Thank you.

Thanks, Andrew.

Okay.

As a reminder, the star one if you would like to ask a question and we will take our next question from June .

With true with Securities. Your line is open.

Hi.

This is awesome on for Joon, thanks for taking my questions.

Just wondering if you've seen an uptick in enrollment per site that have implemented the new protocol for ways given that it should be easier to recruit patients.

And then maybe on the previous question. If you could just talk a little bit more about three months or more to work through the reimbursement process.

I'm, just a little bit of color on that would be interesting. Thank you.

Yes.

I'll take the first one on the rave trial.

Couldn't feel more confident about where we are today relative to even six or nine months ago.

I think there are a lot of factors.

Playing an important role here first.

As in the hospital system was really overrun a year ago, we've had several hundred personnel changes in hospitals, where the studies.

Been taking place and we really feel like that.

It is now stabilized to a meaningful degree and we've talked about that on the call. So we really kind of appeal.

And overall.

Hospital standpoint, the worst is behind the hospital systems I'd say secondly, we have done everything in our power to really help with those hospital systems. We've added several teams too to the core clinical team including.

Three great Pharm DS, who are interacting with pharmacists and personnel say thirdly.

Joe and the medical team has been out on the road meeting with key sites until we feel as though all of those things together, we really now have a significant number of sites that can be important contributors to the study we're seeing an increased number of sites enrolling patients in the study. This is always a tricky one because it is an issue.

Incidents study until we have we don't want to get too ahead of ourselves in terms of our timing.

We talk about publicly but we certainly are enthusiastic about where we are today I feel like the teams made great progress Joe has really taken a lot of it is on its own and I'm working closely with sites and making sure were getting them to the mechanics of what we knew would be a complex study. So I feel as though we're in a better place than we've been in.

The very long time, probably the early days of Covid that we have more engagement than we've had we continue to see encouraging signs on enrollment and we're confident about ending. This study later. This later this year Joe any other comments you want to make that.

That I missed.

No I mean, I think the investigators are very positive about this amendment.

And.

Universally.

This is going to allow.

Especially the patients who come in from other hospitals are emergency departments that were excluded before.

Come into the study so we've gotten great feedback on it and we're really optimistic about the impact it's going to happen.

Great and Chris do you want to talk a little bit about the reimbursement process.

Absolutely and just taking a quick step back here, Steve mentioned and that we can take up to three months or more to get through the enrollment process and we make we mapped that out just based on a couple of typical payer hurdles early and launches.

Such as NBC blocks, and really lack of published payer policy and coverage.

What I would like to say is that we're super proud that we've been able to accelerate this payer access and drive an efficient payer adjudication process that we're already seeing approximately half of our scripts covered within 30 days or less.

So with an ultra rare disease population, sometimes there are medical necessity request and all of this takes a little bit of time, but we're proud of the access that we have and the speed at which we've been able to get to them.

Christy I'm just going to add on.

It's really been a great relationship with not only our payer team our medical science liaison team, but the payors and our advocacy groups I think the payers really understand that in in these ultra orphan conditions.

That have genetic diagnoses or other.

There are not a lot of options for patients.

We've done a lot on the education side, our advocacy groups have other advocacy groups like the TSA alliance as well and so we really think this is a business that has sustainability given the unmet need. So I think the early days of taught US a lot of different things not only that TBD, but the viability.

<unk> for us is a really differentiated.

Orphan treatment for for these genetic disorders.

More refractory.

<unk> next question operator.

Thank you we will take our next question from Brian <unk> with Baird. Your line is open.

Okay.

Hi, This is Luke on for Brian Thanks for taking the question.

So I think you said 40 unique accounts for the 50 something completed forms to your knowledge do the accounts you've reached thus far still have patients to work through or did either existing accounts have.

Arms filled for the majority of their patients.

And then just one more.

So you noted about like 40% of those completed enrollment firms run therapy by the end of Q. You gave favorable you give color on favorable coverage determinations to the beginning of November .

Especially since you are saying about half are getting reimbursed within a month at this point if I heard correctly could you provide a general idea of what additional proportion beyond that 40% have moved under reimbursed therapy.

Through early November thanks.

Yeah look it's a good question and I'm going to turn it over to Stephen Christy, but you make a good point that people should really remember as we go through these numbers. We're reporting numbers due the end of September which means if a patient got a new script the third week in September .

We're in that 40% number right. So so we're incredibly excited that early days early prescriptions.

The carriers both on the commercial side in EBIT on the Medicaid side have made coverage determination and we continue to see that on a very regular basis and quite honestly. The vast majority of scripts are not being delayed not being denied.

And so we could not be more enthusiastic so from that standpoint, Chris Steve maybe I'll turn it over to you for any other comments and see if you want to add.

Yeah happy to so.

Scott really.

Ken pointed the fact that these prescriptions are coming and week by week and so because we've already gotten to half of them adjudicating within 25 to 30 days, we're super proud of that however, what you're not saying is that as that came in late in September and even those that have come in now.

To hit on about those 40 accounts that we're seeing right now that have.

Engaged in a process where thing equal number of unique health care providers that are prescribed as well so not only do we have the H D. D. D centers of excellence that are across the United States. We also have a target base of about 265 accounts that we're targeting today. What this tells US is that I think we got our.

Targeting right early on here and the launch days and Ah witness representation I think that that breadth of interest not only from accounts that positions us well really dictate success.

I think we yet to see a physician that says I don't have any more patients.

Because we have incident of about 1% and 40000 will continue to see patients that not only a gen two and above.

Indication, but there's going to be new.

Patients in the database as well as weak as we go on for three years.

And maybe I'll just make one other comment I think that's important if you think about the two big buckets here commercial payers that Medicaid, we're clearly making tremendous progress on the commercial side.

And good steady progress on Medicaid and as we've always guided to and this launch we're confident that by early 'twenty three and again Medicaid has a a routine and a timeline, which is pretty ingrained both from a mandatory or optional side by that first quarter 'twenty. Three we will have the vast majority of it.

Medicaid patients with with policies in place. So we are right, where we wanted to be I would say, where we're pleasantly ahead of schedule on the reimbursement side and again I think what we're really focusing on is the cadence of new patients going forward.

And then as we've kind of made it clear on this call. We're really happy where we are both from the diversity of those patients.

And theyre not really coming solely from our centers of excellence, Steve anything we missed that you wanted to add.

No I think we've had all the key topics there.

Great.

Operator can we take the next question.

Thank you. Our next question comes from Douglas Tsao with H C. Wainwright Your line is open.

Hi, Thanks for taking my question. So just maybe following up on the commercial launch.

It sounds like you've seen prescriptions coming from 40 accounts I'm just curious does that represent 40.

Unique prescribers or do some accounts have multiple prescribers and I'm just curious in terms of the total universe of.

265 hospitals.

How many have you had an engagement with so far and.

Identified patients that they have and potentially could come on therapy at some point over the next 12 months. Thank you.

Yeah.

Sure Chris do you want to kick it off here.

Absolutely. Thanks for the question. So just to go back to it 265 target that we have a balanced approach on you know we've got targets that we're mapping out between a through D. So a b C. D. Here on every level not only.

Influence across the country, but then number of patients that we believe that they could be potentially trading so of those 40 accounts and I guess to mention that we havent equal number of breadth of physician some.

The account to do have more than one physician that are treating patients.

We tend to have multi disciplinary approach for city D patients so you'll have.

Several physicians there. We also had accounts that were very distinctly with not only their nurse practitioners.

What other ancillary staff that focuses on these patients. So we're getting a large breadth of not only accounts that physicians and health care providers as well that again is encouraging and tells us that our targeting strategy has worked.

Chris You had a specific question from Doug and I think I can say this but you should clarify when we say accounts. We are seeing unique physician practices not unique positions that correct does that with doug's question.

That is correct, but not only are there unique accounts, but they are unique physicians as well.

Okay.

Yes.

No. It did and I guess also I'm just curious to the extent that you know.

Do you have a sense of.

Is there a spectrum and how many patients each account is treating.

Or is it fairly evenly distributed.

Okay.

A lot of that.

Sorry, Scott.

Out of that information were gathering now.

Our MSL team did a significant amount of work early in the days to be able to map where patients are we're confirming a lot of that data on outbound countries.

All patients across the United States, but right now we do not have any specific amount of patients at each individual account and that's information that we will continue to inform.

Okay, great. Thanks, Doug Yeah.

Maybe I'll just add again, we could have expected early days of this launch to only come from the Ato that have roughly 20% to 50 patients right.

Alright that would've been very logical and certainly one or two coes that were big and rollers in the phase three had been very big commercial supporters, but that's the minority. The majority then we have our next layer of accounts, which Christy walked you through to those roughly 250 key accounts, but what's been interesting is we've seen scripts.

Not only it's not only at those 250 accounts, but some other accounts where physicians have read the phase III publication downloaded the prior authorization form from our website and submitted that effectively spontaneously without meeting. The representatives. So I think we need to give a little kudos to our marketing team who has really created.

<unk> a great website, a great informational portal for physicians and we know in our discussions with the IFC are in other alliance groups at many of these patients are treated on a more local basis and they're only going to their centers of excellence once or twice a year. So it's clear to us.

Asset positions are not necessarily waiting for those visits some are actively prescribing today.

<unk> had several thousand hits from both caregivers and physicians on our website.

And so we really feel we're getting some some great traction early days and again I'll. Thank our public our publication team in an.

Alex and that he's team on driving that phase III publication of great editorial in the lancet inclusion in the global <unk> five.

Sidelines as a recommended therapy all of those things have I think really broadened the reach of the drug in early days again early and we know that the families and patients have a lot of medical problems and see a lot of physicians until we know theres going to be some rate limiting.

Great limiting steps for for families to get to physicians, but early days again, we could not be happier with the depth and breadth of the launch.

That's helpful.

Nobody is Scott and just as a follow up but I think your point is it's an important one about not just getting volume from the theories I'm. Just curious do you have a sense I mean, I think you mentioned the website and and knowledge of the phase III do you have a sense of like is there a pattern for for folks who often I know, it's difficult because obviously youre not necessarily interacting with them but for.

These scripts that are just kind of for lack of a better phrase just kind of a PRN and the system do you have a sense of where that awareness is coming from.

I'm going to turn it over to Christi, who.

A reminder has spent many years.

Her career in the rare orphan disease space, So Chris maybe I'll turn it over to you for maybe general comments about the ultra orphan space and what you think.

Being early days here.

Yeah, absolutely so again.

We had enormous kind of warm entry into the CLO, but a good amount of our time was spent out in the community understanding how these patients are moving between the Coa and the community.

We have a minimal amount of prescriptions that are I would say just showing up rather without any involvement from either our medical affairs team or a direct promotional effort from our sales team.

So right now again to Scott's point of understanding how these patients are being treated when they're showing up in the C. O M versus their community setting we're mapping that out right now, but again. This is a direct you now.

<unk> by which you know good promotional effort from not only our marketing.

Medical affairs and promotional effort from our sales team.

Okay, great. Thank you.

Thanks, Doug.

And we will take our next question from Jay Olson with Oppenheimer. Your line is open.

Yeah.

Congrats on all the progress and thanks for taking the questions now.

Now that you sold the <unk> can you talk about how you plan to invest a $110 million proceeds and how are you prioritizing asset allocation and then could you share any thoughts on business development opportunities to diversify your portfolio, especially since there are a number of assets available at <unk>.

Relatively low valuations. Thank you.

Yeah.

Steve you want to talk a little bit of balance sheet, and then I'll talk a little bit about the assets.

Absolutely, yes, good morning, Jay Thanks for the call in terms of the use of the proceeds then and obviously in addition to the PIV, we brought in $30 million plus from the cigar deal and obviously the offering we announced.

All of those.

We will go into continuing investment in our commercialization of the Tommy our continued investment in our clinical development, which includes the two phase III trials, we have ongoing the RSV trial and the trustee of C trial, plus will start a second European based raise trial next year that'll be a phase III as well and then general expense.

In terms of working capital.

Capital expenditures as we build up our IV infrastructure as well so those funds what kind of support all of those those activities in terms of prioritization I think we've been we've been clear the commercialization of the Tommy is incredibly important.

Obviously.

We're extremely focused on the phase threes, we have going with the RSC readout expected second half of next year in Q1 for TSA.

Just obvious critical priorities from an investment perspective, Scott I'll turn it back to you.

Thanks, Steve.

I think what you're hearing from US is that we're having we're just really enjoying working in the ultra orphan disease space I think working with the advocacy groups and helping these patients get therapies for very severe conditions.

It gets a lot of us through.

Through the day with with a lot of excitement there are challenges in the ultra orphan.

Disease business, and I think the epilepsy space, even more complicated because of these patients multiple medical condition, but to my earlier point I mean, we're really seeing very strong reimbursement from the community from our Payors and and the opportunity to really share this information.

With physicians broadly so we love this model and we would love to expand our presence in this space. We think there are some really great targets out there, we're spending more and more time looking at other mechanisms of action. We're certainly still very invested in our second generation program, which.

Anything can drive increased efficacy with a great side effect profile, which is so critical in these patients who are really suffering from so many other medical conditions.

But we're certainly happy that we can we can take some real resources and and look at other assets in the space.

Thank Chris Theater teams done a great job on the commercial side and it would be great to think about adding another product to the portfolio over time I think we're it's incredibly focused on this launch.

We are very hopeful will be an important PSC launch in 'twenty five and of course, the RSC launch around the same time.

So I think Chris <unk> got a lot on her plate, we're thrilled to have a new commercial lead on the RV side of the business is going to complement our sales leadership on the oral side tremendously well. So most of our time today is looking at relatively early stage assets nothing late stage.

And in the epilepsy side I think we've become pretty good at developing these drugs and I feel incredibly fortunate that we have Joe Dr. Ian Miller, who is fantastic so really a strong scientific team.

Another of Lepton widgets on the team who is who has been critically important on our payer side. So we've got some really strong intellectual firepower.

We're doing our best to look at all the compounds that are out there and obviously the markets have been.

Pretty tough on all all the all the companies in the biotech space, we're certainly seeing the ultra orphan neuro diseases for lack of a better being left behind we have a lot of inbound calls from companies, who have broad portfolios and can't find the resources to work in the ultra.

Orphan neuro space. So we do think there'll be some opportunities for us over time, we've got to stay focused on our execution on our late stage programs, but but I think we'd love to do more in this space.

We all really enjoy it.

Yes.

Thanks for the question J operator, we'll go to the next question.

We have no further questions at this time, so I would turn the call back to Mr. Scott Braunstein for closing remarks.

Well, thank you and thanks, everyone for dialing in today. Thank you for all the questions.

It really could not be more excited about where we stand today early days that told me launch in and the progress we're making on the clinical side as well as the expanded pipeline in the now the extended cash runway. So we really appreciate all your support.

And your time today and look forward to seeing the lives.

Have a good day everyone.

Ladies and gentlemen, this concludes today's call and we thank you for your participation you may now disconnect.

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Yes.

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