Marinus Pharmaceuticals, Inc. (NASDAQ: MRNS) Third Quarter 2022 Results Conference Call November 8, 2022 08:30 AMm. ET
Participating companies
Sasha Damouni Ellis – Vice President – Corporate Affairs and Investor Relations
Scott Braunstein – Executive Director
Christy Shafer – Sales Director
Joe Hulihan – Medical Director
Steve Pfanstiel, Chief Financial Officer
Conference Call Participants
Joseph Thome – Cowen and Company
Andrew Tsai – Jefferies
Douglas Tsao – H. C. Wainwright
Jay Olson-Oppenheimer
Operator
Ladies and gentlemen, greetings and welcome to Marinus Pharmaceuticals’ third quarter 2022 cash effects and business updates. [Operator Instructions]
And now I’m pleased to introduce you to your host, Sasha Damouni Ellis, Vice President of Corporate Affairs and Investor Relations. Possibly I could start, Mrs Damouni Ellis.
Sasha Damouni Ellis
Thank you and tomorrow. I am joined through Marinus through Dr. Scott Braunstein, Executive Director; Christy Shafer, Chief Commercial Officer; Dr. Joe Hulihan, Chief Medical Officer; and Steve Pfanstiel, Chief Financial Officer.
Before we begin, I would like to remind everyone that some of the statements we make are forward-looking statements within the meaning of securities laws. execute results, functionality or achievements to differ slightly from those expressed or implied by such forward-looking statements. These dangers, uncertainties and dangers related to our business are described in the Company’s reports filed with the Securities and Exchange Commission, adding Forms 10-K, 10-Q and 8-K.
Now I’ll pass it on to our CEO, Scott Braunstein.
Scott Braunstein
Thank you, Sasha. Et welcome to our call. I am incredibly proud of the progress the team has made as an advertising company following the FDA approval of ZTALMY. We introduced the launch at the end of July and are excited about our first launch metrics for the first time.
In a few moments, I will pass the call to Christy Shafer to talk about the progress of our business, adding the early determinations of the payer policy and the highest call. We are confident in the investments we have made in our commercial infrastructure and are executing a successful launch.
On the clinical business side, we remain focused on advancing our portfolio and prioritizing our two Phase 3 systems in refractory epileptic and tuberous sclerosis complex.
Starting with TSC, we have actively engaged with the community, adding our participation in the recent TSC Global Conference. After our team’s interactions with physicians and families, we continue to say that there is a significant unfulfilled desire for others with TSC-related refractory attacks, and that ZTALMY has the potential to be a vital option for those patients and their families.
By moving to our IV program, as announced in the past, we continue to implement vital improvements to the RAISE protocol, adding the expansion of inclusion criteria for the explicit purpose of expanding the patient population who would be eligible for treatment with IV ganaxolone and accelerating enrollment in the study. The modified protocol has been followed at an increasing number of clinical sites and we expect full adoption by the end of the year. another discussion for Joe to review in his ready comments.
We are still expecting data from the RAISE test in the current part of next year and data from the TrustTSC Phase 3 trial in the first quarter of 2024. Similarly, entry-level systems for IV and oral franchises are progressing as previously reported.
Finally, some comments on how we plan to bring ZTALMY to patients and families around the world. In Europe, we are on track to submit full responses to the EMA Day 120 list of questions for CDD marketing authorization application by the end of November. which would result in a CHMP opinion until the end of the first quarter of 2023. I must thank the entire Marinus team and our partners at Orion Corporation for their tireless efforts to ensure that our answers to EMA’s questions are complete and robust. Together with Orion, our groups continue to prepare for the launch of a European announcement imaginable next year.
In addition, there is a broader global opportunity for ganaxolone and we are exploring other opportunities outside the United States. commercial alliances. We aim for a strategic deal for China by the end of the year and some other vital global collaboration in 2023. With the sale of our precedence review guarantee, the termination of a contract option through BARDA, and the termination of interest on the revenue financing agreement with Sagard, we have executed our plans to expand our projected money track with undiluted financing through early 2024 while creating the opportunity to secure a US-based API source. We are in the U. S. to decorate our long-term business fundamentals.
In addition, with the percentage offer announced last night, which is expected to close on November 10, we now expect our cash balance to take us to the current part of 2024. In addition, we are excited to expand our shareholder base. and at the same time thank our existing shareholders for their commitment to the organization. At the end of the percentage provided, our new money balance will allow us to continue investing in the continued launch of ZTALMY, our progression pipeline and prepare for two future advertising releases.
Now, I would like to pass the call to our Chief Commercial Officer, Christy Shafer, for updates on the launch of ZTALMY. Christy?
Christy Shafer
Thank you Scott. Et smart morning. I am pleased to share our initial progress in launching the ZTALMY announcement in the United States, where we have noticed a constant enthusiasm on the part of physicians and caregivers reflecting the wishes of this network and ZTALMY’s potential offering in crisis management. We introduced ZTALMY on July 28 and at the end of the third quarter, we generated $560,000 in net product revenue.
In those first nine weeks of launch, we won more than 50 CDD prescription enrollment forms, of which more than 30 were for newly announced, previously untreated ZTALMY-treated patients, with the rest covering the transition from across the U. S. to the U. S. U. S. , CDD, OLE and EAP Patients to advertise products.
It’s vital to note that those recipes come from a diverse prescriber base of more than 40 exclusive accounts, adding interest from the outside to our 265 target accounts. Virtual marketing tools, publishing strategy and pre-launch education and outreach efforts.
In addition to a highlight of new patient enrollment and prescriber activity, we are also encouraged by the early endings we are seeing among payers, as approximately 40% of CDD patients have finished the prescription enrollment bureaucracy with the remedy reimbursed by the end of the third trimester.
As a reminder, we anticipate that approximately 60% of the CDD patient population will have a Medicaid-paid and controlled policy, while the remaining 40% will be commercially controlled. As of November 1, ZTALMY obtained positive coverage criteria from more than 15 payers. representing approximately 70 million lives, and we expect most of the remaining policy briefs in the fourth quarter.
In addition, 57% of all U. S. industry plans are in the U. S. The U. S. government has already extended the labeling policy to ZTALMY. We continue to believe that payers appreciate the effect of the disease on patients and their families, as well as the unmet medical desires in the CDD treatment landscape and the ZTALMY profile is the first and only product indicated for CDD-associated seizures.
Field interactions are supported by our educational speaker systems as well as two product theaters, one taking place in October at the Society of Child Neurology Annual Meeting and scheduled for December at the American Epilepsy Society Annual Meeting. Our official ZTALMY speaker office starts in December with a significant number of systems planned until the end of next year.
We will continue to teach and raise awareness in the CDD patient network beginning with the CDD network webinar early next year. We have also actively engaged with leading patient advocacy organizations, which have proven to be a source of two-way communication.
These teams provide percentage information within the patient network and provide a feedback channel to ensure we meet the conversion desires of CDD communities. We are grateful for the concepts and that IFCR, CDKL5 Alliance and LouLou Foundation provide to families living on contracts
We are very pleased with the progress of our early launch and are grateful for the impact we believe we will have on CDD patients and their families. patients, caregivers, doctors and payers in the coming months.
As a final update to continue our commitment and progress towards creating an acute care hospital franchise, we have hired another Commercial Manager for my team, Kristin Rudisill, who has a strong track record of good luck and hospital discharge experience for the progression of our business strategy in the U. S. For prestige epilepsy.
I now give the floor to our Chief Medical Officer, Joe Hulihan, to speak about our ongoing progression programs, adding the knowledge we continue to generate and provide at the clinical meetings that continue to be critical to our launch and spread the word about ZTALMY.
Joe Hulihan
Thank you, Christy, and good morning everyone. First of all, I would like to provide an update on our IV systems in epileptic prestige and deepen our position a little with the modification of the RAISE protocol that we announced in June of this year. As we have already shared, we are expanding the Inclusion Criteria to allow randomization of patients who have been treated with maximum doses of intravenous anesthetics, such as propofol, for up to 18 hours rather than excluding any patient who has been treated with maximum doses of intravenous anesthetics, regardless of the brevity of management of those agents. .
We believe this is a significant replacement, as it will allow enrollment of patients transferred to intensive care from other hospitals or the emergency room. Two of the most common pathways for epilepsy refractory to identity or prestige. The original protocol excluded many who represented a significant proportion of the population from the Phase 2 trial.
I think it’s also to mention that patients enrolled prior to implementation of the improvement would still meet the eligibility criteria under the revised protocol. As Scott mentioned, most RAISE sites have implemented protocol enhancement, and the remaining sites are expected to transfer to the revised protocol by the end of the year.
The researchers expressed great excitement about the possibility of making adjustments to the protocol to allow for the recruitment of more eligible patients. We continue to increase the number of attractive RAISE exam sites in the U. S. We will be activated under the modified protocol.
In August and November, we held meetings of researchers in the US. We are in the U. S. to inform our sites about the updated protocol and we continue to work intensively with them for timely enrollment in studies. We recently reviewed the initial characteristics of patients in Phase 3 RAISE. We are pleased to report that, in September, the baseline characteristics of Phase 3 patients closely resemble those of patients enrolled in our Phase 2 CSR study.
Participants in either study are about the same age. One proportion have a history of epilepsy and have initial loads of seizures and epilepsy of prestige severity. These demographics are described on slide 29 of our November corporate presentation.
In addition, in September, we presented knowledge of phase 2 ESR and a review of the ongoing RAISE trial at the London-Innsbruck Colloquium on status epilepticus. The presentation garnered a positive reaction from participants who are leading researchers and physicians in the field.
They explain that the RAISE trial represents a breakthrough in clinical studies on refractory status, a condition for which there was no knowledge of rigorously conducted placebo-controlled studies. Several Marinus representatives also attended the annual meeting of the Neurocritical Care Society in October, where we presented two posters containing knowledge of the ganaxolone IV program, one of which was designated as a featured poster presentation. There, we had the opportunity to meet many scholars and other members of the RAISE exam sites team. In addition, neurocritical care physicians at other primary centers have an explicit interest in participating in the trial.
Looking ahead to the long-term RAISE study, we will continue to actively monitor what is working well at the site point and where we can provide more for trial recruitment and execution. There is also the possibility of expanding the age diversity of eligible patients. Currently, the trial can enroll patients over the age of 12. We will soon submit an application to the FDA with knowledge that has allowed us to prospectively include more young patients. Given the higher incidence of prestige epilepticus in children under 12 years of age. , this update could expand the pool of eligible patients and further inspire timely enrollment in the study.
As stated above, we have the option of having an independent data monitoring committee act as an interim investigation when two-thirds of the participants have completed the examination, or approximately 82 patients. At that point, if the interim assessment is carried out, the committee will go ahead, either avoiding the examination for reasons of potency or proceeding with the full registration. Although conducting an interim study might cause the sensitivity of a study to encounter a treatment effect, it should be continued until registration is complete. We designed the interim study of the RAISE examines to have minimal effect on the final efficacy results of a fully enrolled trial.
We are pleased with the changes we have made to aid RAISE trial enrollment and continue to expect first-line data in the current part of 2023. Planning continues for a separate Phase 3 CSR trial, the RAISE 2 study to assist a marketing authorization application in Europe. The protocol is finalized and recruitment is expected to begin in the current part of 2023. This trial will not only serve as an essential component of the European approval strategy. It also has the possibility to expand further. the indication of ganaxolone IV in the United States.
Let’s move on to the Phase 2 RESET exam in established prestige epilepticus. We activated the first one and plan to begin enrollment in the U. S. This year and continue to make progress on the informed consent exception for more.
Now, I would like to move on to our oral ganaxolone programs. As Christy said in her remarks, we continue to work to raise awareness about ZTALMY in the clinical and medical community. On October 13, we presented two posters at the Neurology Society Child Assembly of our pivotal Marigold Phase 3 study, adding open-label extension knowledge that showed continued relief in seizures over a two-year period.
Although 24-month knowledge was only available for 16 of the 54 patients who remained on the open-label extension, relief in primary motor seizure frequency for this subgroup continued with mean relief of 53% at 24 months compared with 30. 7. % relief at the end of the double-blind phase.
The dropout rate was around 30% in the first year of the open phase, but decreased to around 10% in the second. frequency of seizures. We also present knowledge from a post hoc investigation of the anxiety, depression and temperament scale. The Marigold study was administered, which showed that ZTALMY was linked to innovations in certain habit spaces, namely discounts on compulsive habit and hyperactive manic. parts of the scale. While those findings require confirmation through further research, they recommend that there are possibly potential benefits of the remedy beyond reducing seizures.
Moving on to our Phase 3 TrustTSC trial in the tuberous sclerosis complex, we are pleased to learn that we are actively evaluating patients. We are 85 clinical sites primarily in the U. S. The U. S. and European developments in Canada and Israel are planned until the end of the year and in Australia in the first part of 2023. We are encouraged by the medical network’s maximum enthusiasm for this trial and continue to anticipate sales for the first quarter of 2024.
Finally, I would like to give you an update on our moment generation formulations. As shared last quarter, we saw promising first-line effects from a Phase 1 ascending single-dose study of the first of our reformulation candidates, which we helped in clinical development. Phase 1 examining participants gained reformulated oral ganaxolone at doses ranging from 100 to 900 milligrams with existing oral suspension provided as a baseline control.
The pharmacokinetic profile of the new formulations showed increases in overall exposure from peak concentration or Cmax. Therefore, this can potentially allow an increase in dose beyond the dose of the existing oral suspension to further optimize efficacy without generating a concomitant increase. in Cmax-related adverse events such as Sonance.
The next steps in the evolution of this program come with recruiting an additional Phase 1 examination cohort to compare single doses greater than 900 milligrams to, if we can, achieve greater increases in overall exposure from peak concentration. We expect the knowledge to be to be had until the end of the first quarter of 2023. We will also carry out PK modeling for the next steps in the development of this formula, adding the need for a study with several increasing doses, which we could start in the first part of 2023.
We plan to choose one of the second-generation ganaxolone formulas for a phase 2 study in Lennox-Gastaut syndrome that will begin in 2023. Lennox-Gastaut syndrome is highly treated refractory and new oral formulations have the potential to offer a more consistent and predictable supply. exposure to ganaxolone, in which we would allow physicians to individualize the dose to achieve an optimal reaction for each patient.
In addition, we have developed several pro-drug applicants that can deliver additional innovations in efficacy, tolerability and dose individualization, as well as operational and production efficiency. IND studies with specific Phase 1 knowledge for 2024. We look to the future to proceed to raise awareness in the medical and clinical network in the coming months, and we will have a strong presence at the Annual Meeting of the American Epilepsy Society in December, adding knowledge presentations of our oral and ganaxolone systems in the general sessions, as well as at the Marinus Science Expo. As a clinical team, we remain focused on our Phase 3 clinical systems and prestige epileptic complex and tuberous sclerosis, advancing our reformulation and prodrug applicants, as well as our continuation for ZTALMY and CDD.
I will now pass the word to our CFO, Steve Pfanstiel, who will provide you with a financial update.
Steve Pfanstiel
Thank you Joe and good morning everyone.
Before discussing our monetary effects for the third quarter of 2022, I would like to share with you some key monetary aspects from our call beyond. months, adding more than $150 million in investment to our short-term money position. Specifically, in August, we closed the sale of our Pediatric Rare Disease Priority Review Voucher, receiving $110 million in gross investment. This was followed in September by another $12 million in BARDA investment by exercising the first contract option for our API offshoring initiative.
Most recently, in October, we entered into a royalty monetization agreement with Sagard Healthcare, which includes a $32. 5 million upfront payment for long-term royalty bills similar to U. S. revenue. Relevant U. S. with ganaxolone, adding ZTALMY. cash equivalents of $168. 2 million, which does not come with the budget obtained from Sagard’s interest financing agreement. The addition of the net budget from this recent transaction results in a money balance of approximately $200 million, which will be sufficient to finance. Our operations through the first quarter of 2024; adding to maintain the minimum money balance of $15 million required under our credit agreement. As Scott noted at the end of the percentage offer announced last night, we expect our money balance to take us to the current part of 2024.
I will now turn to our monetary effects for the third quarter of 2022, and I am pleased to share our first quarter effects with sales of ZTALMY products in the United States. For the third quarter of 2022, we recorded a ZTALMY net profit of $560,000, representing just over two months of effective advertising launch. This gain is comprised of sales of ZTALMY products to our exclusive specialty pharmaceutical spouse, Orsini, and includes estimated net expansion deductions in addition to expected Medicaid reimbursements.
Focusing on BARDA revenue alone, we see revenue of $1. 8 million and $5. 1 million for the 3 and nine months ended September 30, 2022, respectively, compared to $1. 1 million and $4. 8 million for each of the same periods last year. Research and progress Construction expenses increased $19 million and $58. 5 million for the three and nine months ended September 30, 2022, respectively, compared to $18. 4 million and $55. 5 million for the same periods of the previous fiscal year. The replacement is basically due to construction-related prices on R staff.
Selling, general and administrative expenses increased to $13. 4 million and $42. 2 million for the 3- and nine-month periods ended September 30, 2022, respectively, compared to $9. 5 million and $26. 7 million for the same periods of the prior fiscal year. were preparing and launching ZTALMY and beyond to expand the company’s operations. As mentioned above, the third quarter of 2022 included a net profit of $107. 4 million similar to the sale of GRP.
As a result, the Company reported a net revenue source of $73. 3 million and $14. 5 million for the three and nine months ended September 30, 2022, respectively, compared to net losses of $19. 5 million and $70. 5 million for the same periods last year. The totals come with a non-cash stock-based redemption expense of $3. 9 million and $11. 1 million for the 3 and nine months ended Sept. 30, 2022, respectively, compared to $2. 8 million and $10. 9 million for the same periods last year.
Cash used in operating activities was $91 million for the nine months ended September 30, 2022, compared to money spent on operating activities of $33. 7 million during the same era last year. As a reminder, our overall operating money for 2021 includes $30 million in funding from the signing of our European partnership agreement with Orion.
I will now turn the floor over to Scott, who will make his closing remarks.
Scott Braunstein
Thank you, Steve.
It has been a busy and exciting time for the Marinus team and we are pleased with the results of the initial launch, the progress made towards potential approval in Europe next year and the organisation’s research efforts. We look forward to building on this advertising base with ZTALMY and advancing our two Phase 3 systems over the next 12 months.
In closing, I would like to mention that Marinus will host an investor breakfast on Monday morning, December 5 at AE. We will be meeting with doctors and powerhouse organizations over the weekend and hope to see them live there on Monday morning. This assembly will give you the opportunity to meet our commercial and clinical leaders who were an integral component of this exciting launch. Sasha will provide more main points about the occasion in the coming weeks.
Operator, can you open the question form now?
Q&A session
Operator
Yes, thanks. [Operator Instructions] We will answer the first one by Marc Goodman of SVB Securities. Your line is open.
Scott Braunstein
Hello Marc. Possibly you would be silenced. Operator, we don’t hear anything.
Operator
Let Mr. Goodman, if he listens to us, queue again. We’ll move on to the next one by Joseph Thome of Cowen and Company. Your line is open.
Joseph Thomé
Hello everyone. Hello and thank you for answering our questions. Perhaps a novelty in ZTALMY. Es smart to see sales start rolling. It turns out that the consensus for the year is around $2. 5 million. garage patterns that we are aware of?
And then, at the time of CSR, I taught that the upper enrollment limit for Phase 2 and Phase 3 is 8 years, and then there is talk of the option of going down to 12 years or less. Does it make a difference in reaction to treatment or addition?To the popular general care based on this kind of broad [indistinguishable] that would be useful?Thank you.
Scott Braunstein
Steve, why don’t you turn to you for ZTALMY’s question and then let’s let Joe answer the CSR question?Yes
Steve Pfanstiel
Yes. Hello, Jo. Thanks for the question. In terms of ZTALMY’s revenue, we provide forecasts for 222 or 2023, so I wouldn’t possibly communicate about that specifically, but as we said, we’re concentrating on the number of patients in therapy. We focus on selling access within government and ad payers. We are too satisfied with our progress on both fronts. We feel like we’re having a very strong track record there in the first two months of launch, and also, as we mentioned, we’re going to launch speaker systems later this year. We’re going to have a strong presence at EA. So we’re positive about our launch trajectory here, however, let’s wait for now.
In terms of inventory impact, we work with a single specialty pharmacist spouse. We have a very strict distribution process. Imagine that our warehouse and our specialty pharmacist spouse are separated by only a few hours. Since we measure the effect of inventories on the order of weeks, not months, this will be the case for the foreseeable future. Therefore, a very limited effect on inventory storage, and this will be the case in the future.
Joseph Thomé
Hey, Steve, before you speak, Joe, you’d like to remind ZTALMY and our audience. . . What do we talk about break-even and how do we feel about it compared to the first few months of launch?
Steve Pfanstiel
Joe, just to bring up a little more color in terms of the number of dashes in this trajectory. We’ve said it: We believe the $25 million investment in CDD commercialization is a two-year investment to break even. By mid-2024, we believe we deserve to annualize at a sales rate of around $30 million. That, along with a 90% gross profit margin on earnings, is expected to cover that $25 million business investment. We think it’s going to be a pretty solid build during that era and we think the $30 million break-even point will likely come down between 240 and 250 million patients: 240, 250 patients in therapy.
Scott Braunstein
Super. Et Joe, before I give you the floor, just wanted to make a small comment to ZTALMY and the audience about the fact that the RAISE trial itself primarily uses adult sites, but we have about 10 sites that are in the pediatric age range, usually 12 years or older. Therefore, extending the trial to younger patients would primarily be sites that lately have a limited population of 12 years or older. But in terms of our expectations and the good fortune of the drug in younger populations, I’m going to give the floor to Joe. And Joe, you can also comment on some of our reports on SRSE with ganaxolone.
Joe Hulihan
Sí. No, absolutely, Scott. We don’t know, I think what you’re asking about Joe, there was a bit of background noise, but it was about his reaction in young people rather than adults. Don’t expect young people to get worse at least as well as adults. And we’ve had some: we’ve now treated about 14 patients in super-refractory prestige emergency INDs, and we’ve had the right reactions in young people. So we will be waiting for young people to sign up to pass the test, and indeed this amendment gives us a good seasoning in terms of eligible population, because in terms of young people, prestige is no rarer in young people under 12 than in teenagers. So we hope to move on to the age of two and will submit an application to the FDA very soon about it.
Joseph Thomé
Perfect. Thank you so much. That’s very helpful.
Operator
And we’re going to take our next Andrew Tsai consultation with Jefferies. Your line is open.
Andrew Tsaï
Hello. Thank you and good morning. Congratulations on all the progress. So, maybe one in CDD, another in CSR. Thus, at the launch of the fixed-term contract, 50 patients are strong on September 30. You already said that this was not to be expected, is this still your case?And out of curiosity, where is the total number of patients today compared to September 50-30?
And then secondly for CSR; Can you remind us what you expect from placebo reaction rates for the two co-primary endpoints and why?So, the percentage of patients who quit smoking within 30 minutes, and then the percentage of patients who don’t. Transfer to intravenous anesthesia, would that be very helpful? Thank you.
Scott Braunstein
Thank you, André. Joe, first you need to answer about CSR, so we can pass it on to Steve and Christy about CDD?
Joe Hulihan
So we were very conservative in terms of estimating the duration of the pattern for the trial. Our researchers and advisory board gave us insight into the placebo reaction. And for the first reaction to the co-primary endpoint within 30 minutes, I think the placebo reaction, frankly, I hate being too sure of myself, but it will be insignificant. The idea that prestige would avoid it on its own in part of an hour. I’m sure of that. So, conservatively, 10% reaction to placebo potentially.
At the time of escalation from the co-primary endpoint to IV anesthesia, again, we surveyed our sites and they said we were entering the profile, the patient entering the study. That’s where they gave us the estimate. The calculation of the pattern length is based on a 45% placebo reaction and a 75% placebo reaction; that is, 75% is a progression to IV anesthesia and 45% progression in the ganaxolone arm. I think it’s incredibly conservative. So, the delta of 30%, and in fact it gives us more than 90% strength. And we talked about interim research, which is also very difficult to the point where we reached 82 patients, I think, which still gives us much greater strength than stumbling over the difference in treatment.
Scott Braunstein
Steve, you have to start with the numbers, and then we’re going to turn it over to Christy to comment further?
Steve Pfanstiel
Yes, of course, Andrew, I think in terms of bowling, I’ll possibly start there. We had just over 20 patients in our open EAP program that would come in time. Therefore, it is not a massive bolus that we [indistinguish] from this program. And I don’t know if that’s what you were referring to specifically. But I think the other thing that’s vital is that when we talked about it before, we said, hey, it can also take 3 months or more on average for patients to go through the reimbursement process. We know that payers will set up blocks of NDCs or other things, which would possibly take some time. But I think our payer team has done a phenomenal job of being there, smart access for patients. I think our pricing strategy has paid off enormously.
And in fact, about 50% of our patients are reimbursed and approved within 30 days. Some will take longer, but we are proud of what we have been able to do. Therefore, those patients will come in longer than they deserve. Be part of this payer process. But having so many patients after nine weeks, I think, shows the paintings we’ve done on the paymaster side.
Christy, I don’t know if you were looking to upload something there?
Christy Shafer
Yes, thanks for the question, Andrew. I think Steve hit him on the head there. However, just to reiterate those numbers, 20 patients in our [indistinguishable] PAD systems with just over 30 patients who had not received ganaxolone. Of those 30 patients, we saw a smart balance between when we saw those enrollment forms. So, there wasn’t a bolus from them in the first two weeks, and we continue to see interest equivalent to what we saw at the start of launch day. So, only a few weeks, but we are also encouraged by the effort.
Andrew Tsaï
Thank you. That’s all I had. Thank you.
Scott Braunstein
Thank you André.
Operator
[Operator Instructions] And we’re going to take our next Joon Lee consultation with Truist Securities, their line is open.
unidentified analyst
Hi, I am [indistinguishable] for Joon. Thank you for answering the questions. I wonder if you’ve noticed an increase in the number of registry sites that implemented the new protocol for RAISE, given that it’s less difficult to recruit eligible patients.
And then, in the previous question, if you can communicate a little more about the 3 months or more for the paintings in the refund process. It would be interesting a little color about that. Thank you.
Scott Braunstein
Yes, I will take the first one in the test RAISE. Es we may not be any more sure of where we are today than we were six or nine months ago. I think there are many points that play a vital role here. First of all, I think the hospital formula was actually surpassed. a year ago. We had several staffing adjustments of another hundred people in the hospitals where the studies were conducted, and we actually feel that that has now stabilized to a significant degree. And we talked about that on the call. So we actually have the impression, from the general point of view of the hospital, that the worst thing is the hospital formulas.
So I would say that we have done everything we can to help those hospital systems. We have added several groups to the core clinical team, adding 3 perfect pharmacists who interact with pharmacists and staff.
Third, Joe and the medical team have been on the road to meeting with key sites, so we feel like all of those things together, we actually now have a significant number of sites that can be significant contributors to the study. We’re seeing an accumulation in the number of sites enrolling patients in the study. It’s a complicated consultation because it’s an incident study, so we have, we don’t need to get too far ahead in terms of time and what we’re talking about. Publicly, however, we are excited about where we are today.
I feel that the groups have made wonderful progress. Joe takes a lot of that on his own and works a lot with the sites and makes sure we get them through the mechanisms of what we knew was a complex exam. So I feel like we’re in a more wonderful position than we’ve been in a long time, probably the early days of COVID and we have more engagement on site than we had, we continue to see encouraging symptoms on the record, and we’re confident that this exam will be over later this year. Joe, do you have any other comments I missed?
Joe Hulihan
No, I mean, I think the researchers are very positive about this amendment. And universally, this will especially allow patients coming from other hospitals or emergency departments who in the past were excluded from participating in the study. So we’ve had some wonderful feedback on this and we’re really sure of the effect it will have.
unidentified analyst
Super. Et Chris, would you like to let us know a little bit about the refund process?
Christy Shafer
Just going back a step here, Steve commented that it may take up to 3 months or more to complete the registration procedure, and we have mapped it based on some typical payer hurdles at the beginning of releases like NDC. Locks and unpublished policies and payer coverage. What I would like to say is that we are very proud to have been able to expedite this payer access and conduct an effective payer arbitration procedure that we already see over some of our scripts covered within 30 days or less.
So with an ultra-rare disease population, there are requests for medical necessity, and all of this takes a little bit of time, but we’re proud of the existing access we have and the speed with which we’ve been able to reach them. .
Scott Braunstein
Chris, I’ll just add, I think it’s actually a wonderful appointment not only with our paying team, our medical science league liaison team, but also with the payers and our defense teams. I think payers really perceive that in those ultra-orphan situations that have genetic diagnoses or other AEDs, there are a lot of characteristics for patients. And I think we’ve done a lot on the education side, our defense teams have done it, other defense teams like TSC Alliance as well. It is a corporate that has sustainability given the unmet need.
So I think the early days taught us a lot of other things, not only about CDD, but also about viability for us as a de facto otheriado orphan remedy for those genetic disorders and more refractory encephalopathies. Next question, operator.
Operator
We’ll be taking our next Brian Skorney with Baird. Your line is open.
unidentified analyst
Hi, I’m Luke for Brian. Thank you for answering the question. I think you said 40 exclusive accounts for the 50 or so bureaucracies that were finished. Based on your knowledge, do the math you’ve come to so you still have patients to treat or do. Have those existing accounts broken bureaucracy for most of your patients?
And then just one more, keep in mind that about 40% of the finalized enrollment bureaucracy was in treatment at the end of the third quarter, you tested positive, you colored favorable policy determinations in early November, especially since you’re talking About a portion is reimbursed within a month at this point. If I listened correctly, can you give me a general idea of the additional proportion beyond that 40% who won the reimbursed treatment until the beginning of November?Thank you.
Scott Braunstein
Luke, that’s a smart question, and I’ll pass it on to Steve and Christy, but you’re pointing out a smart point that other people deserve not to forget when we go through unit numbers. We report the numbers until the end of September, which means that if a patient receives a new script in the third week of September. They’re at that 40% number, right?
So, we’re incredibly excited that in the early days, early prescriptions, companies on the advertising side, and even on the Medicaid side made policy decisions. And we continue to see that very regularly and honestly, the vast majority of non-delayed scripts are not rejected. So, we couldn’t be more excited about that. Christy, maybe I’ll turn to you for more feedback and see if you need to add.
Christy Shafer
Yes, satisfied with. So Scott really put his finger on the fact that those prescriptions come in week after week. And since we’ve already received that part from them within 25 to 30 days, we’re very proud of it. However, what you don’t see are the ones that arrived at the end of September and even the ones that arrived now, I would love to communicate to you about the 40 accounts that we are seeing right now that have been involved in the procedure that we are seeing an equivalent number of exclusive accounts physical care providers who have also prescribed.
So, not only do we have 8 CDD Centers of Excellence spread across the U. S. We also have a target base of approximately 265 accounts that we are targeting today. What that tells us is that I think they gave us our guidance very early here on premiere days. And with this representation, I believe that this interest not only of the accounts, but also of the doctors, dictates success.
I think we haven’t noticed a doctor yet who says, I don’t have patients anymore because we have an incident of about one in 40,000. We will continue to see patients who not only age at age two or older for our indication, but there will be new patients in the database over the years.
Scott Braunstein
And maybe I’ll just make another comment that I think is important. If you think about the two big cubes here, the ad payers and Medicaid, we’re obviously making massive progress on the industry front and stable smart progress on Medicaid. We’ve guided you in this launch, we’re sure that until early 2023 and again, Medicaid has a regimen and a timeline, which is pretty much ingrained in one mandatory and optional aspect.
But through this first quarter of 2023, we will have the vast majority of Medicaid patients with policies in place. Therefore, we are where we need to be. I would say we are pleasantly ahead of schedule on the payment side. And again, I think what we’re focusing on is the speed of new patients in the future.
And because we made it transparent on this call, we’re pleased to be a part of the diversity of those patients and not just come from our centers of excellence. Steve, is there anything we missed that you wanted to add?
Steve Pfanstiel
No, I know, we touched on all the key issues there.
Scott Braunstein
Super. Operator, why don’t we take the following question?
Operator
The next one comes from Douglas Tsao with H. C. Wainright. Your line is open.
Douglas Tsao
Hi, thank you for answering the questions. So, perhaps to track the launch of the ad, it seems that he saw a recipe of 40 accounts. I’m curious, does this constitute 40 exclusive prescribers or do some accounts have prescribers?And I’m curious in terms of the total universe of 265 hospitals, how much have you had a commitment to so far and met the patients who have and in all likelihood may go through treatment sometime in the next 12 months?Thank you.
Christy Shafer
I’m sorry.
Scott Braunstein
Christy you to free him? Yes.
Christy Shafer
Thank you for the question. So, to get back to that, 265 targets for which we have a balanced technique, we have targets that we map between A and D, then A, B, C, D here at their level, just an influence across the country, however also the number of patients that we think they can potentially treat.
So, of those 40 accounts, I just commented that we have an equivalent number of doctors. Some accounts have more than one doctor treating patients. EOCs tend to have a multidisciplinary technique for fixed-term patients. You will find several doctors.
We also have accounts that work very clearly not only with their nurse practitioners, but also with other assistants who focus on those patients. Therefore, we have a wide diversity of not only accounts, but also doctors and healthcare providers. This is again encouraging and tells us that our targeting strategy has worked.
Scott Braunstein
Christy, you had Doug’s express question, and I think I can say that, but you explain that when we communicate about accounts, we are communicating about exclusive medical practices, not exclusive medical practices. Was that Doug’s question?
Christy Shafer
Exactly. But there are only exclusive accounts, but there are also exclusive doctors.
Douglas Tsao
It is ok. Thank you.
Scott Braunstein
Doug, have you noticed your question?
Douglas Tsao
Yes, no, no. Yes, no, he did. And I also guess I’m curious about how much a concept has: is there a spectrum and how many patients does each account treat?Or is it slightly distributed?
Christy Shafer
A lot of this information, sorry, Scott. A lot of this information we collect now, our MSL team did a lot of work early on to map where patients are. We verify much of this information and cover more patients across the United States. United, even at this time we do not have an express number of patients in each individual account and this is data that we will continue to report.
Douglas Tsao
It is ok. Super. Thank you.
Scott Braunstein
And Doug, yes, I’ll just add, again, I think we might have expected the first few days of this launch to come just from the 8 EOCs that have 20 to 50 patients each, right?And in fact, one or two EOCs who were great recruiters in Phase 3 were big supporters of advertising, but that’s the minority, not the majority.
Then we have our next layer of accounts, which Christy has guided you through those 250 key accounts. where doctors read the Phase 3 publication, downloaded the prior authorization form from our website, and spontaneously submitted it without meeting with the representative. So I think we should give some credit to our marketing team for creating a wonderful website, a wonderful inshapeation portal for doctors.
And we know from our discussions with IFCR and other alliance teams that many of those patients are treated more locally and only stop at their centers of excellence once or twice a year. It is transparent to us that doctors do not necessarily wait for those scale ats. Some are actively prescribing today. We have gained several thousand scales of caregivers and doctors on our website.
And then we feel like we’re getting wonderful traction early on. And back, I will thank our publishing team and Alex Aimetti’s team for leading this phase 3 publication, a fair editorial in The Lancet, inclusion in the global CDKL5 rules as a recommended therapy. All those things that I think extended the success of the drug in its early days. Again, early and we know that those families and those patients have a lot of medical problems, they see a lot of doctors. And we know that there will be rate limitation steps for families to move to doctors. But at first, back again, we couldn’t be happier with the intensity and breadth of the launch. Is it useful?
Douglas Tsao
No fees, Scott. Just as a follow-up, because I think his point is: it’s vital not only to see the volumes of the EOCs. I’m just curious, does it have a concept, I mean, I think, you discussed the online page and the wisdom of Phase 3. Do you have a concept, like, is there a style for other people that, and I know it’s complicated because obviously you don’t necessarily interact with them?But for scripts that are just, for lack of a greater expression, simply appearing in the system. Do you have any concept of where this consciousness comes from?
Scott Braunstein
I pass the word to Christy, who, as a reminder, has spent many years of her career in the area of rare orphan diseases. So, Christy, I’ll pass it on to you for some general comments about the area of ultra-orphans. and what you think we’re seeing here in the early days.
Christy Shafer
Yes, absolutely. Again, we had warm access to the WCC, but we spent much of our time on the network to understand how those patients move between the WCC and the network. So, we have a minimal amount of prescriptions that I would say come in without any involvement. from our medical affairs team or direct promotional efforts from our sales team.
Right now, again, to Scott’s point about how those patients are treated, when they come to the WCC in relation to their network setup. We are mapping this right now, again, this is a straightforward procedure where a clever promotional effort is not just our medical marketing matters and the promotional effort of our sales team.
Douglas Tsao
It is ok. Super. Thank you.
Scott Braunstein
Thanks Doug.
Operator
And we’re going to take our next consultation from Jay Olson with Oppenheimer. Your line is open.
Jay Olson
Congratulations on all the progress and thank you for answering the questions. Now that you’ve sold the GRP, can you tell us how you plan to invest the $110 million in profits and how you prioritize asset allocation?Do you care about trading progression opportunities to diversify your portfolio, especially since there are a number of assets available at low valuations?Thank you.
Scott Braunstein
Steve, get a little bit out about the balance sheet, and then I’ll let you know a little bit about assets.
Steve Pfanstiel
Yes. Hi, Jay. Thanks for the call. In terms of product usage and in addition to GRP, we raised over $30 million from the Sagard deal and from the offering we announced. All of this will lead to continued investment in our commercialization of ZTALMY, our continued investment in our clinical development, which includes the two Phase 3 trials we have underway, the CSR trial and the TrustTSC trial, and we will launch a momentary RAISE trial in Europe next year. It will also be a phase 3.
And then overhead in terms of operating capital, capital expenditures as we build our infrastructure IV. So, that budget will include all those activities. In terms of prioritization, I think we’ve been clear, the commercialization of ZTALMY. It’s incredibly important. Obviously, we are incredibly focused on the 3 stages we have with the expected CSR reading in the current part of next year in the first quarter for TSC. These are just key priorities that are evident from an investment perspective. Scott, I’ll give you the ground again.
Scott Braunstein
Thank you, Steve. Jay, I think what you’re hearing from us is that we have a. . . We actually enjoy running in the box of ultra-orphan diseases. I think we’re passionate about working with advocacy teams and helping those patients. Get treatments for very serious situations on the day. There are demanding situations in the ultra-orphan disease sector and I think the area of epilepsy is even more confusing because those patients have multiple medical situations. But for the above points, I mean, we actually see a very significant reimbursement from our payer network and the opportunity to percentage that data with doctors in general. So we like this style and would like to expand our presence in the area. We think there are some very smart goals.
We spend more and more time reading other mechanisms of action. In fact, we are still very committed to our second-generation program, in which we can increase efficacy with a fair-looking effect profile, which is so critical in those patients who actually suffer from so many other medical conditions. But we are really satisfied to be able to take genuine resources and take a look at other assets in the space. And I think Christy and her team did a wonderful job commercially. And it would be wonderful to think about adding some other product to the portfolio over time. I think we’re incredibly focused on this launch and in fact expect there to be a significant TSC launch in 25. And, of course, the launch of CSR almost at the same time. So I think Christy turns out to have a lot to do. We are excited to have a new sales manager in the IV aspect of the company, who will complement our sales leadership incredibly well in the oral aspect.
So most of our time today is spent on relatively early-level assets, not anything at a complex level on the epilepsy side. I think we’ve been successful in creating those drugs, and I feel incredibly fortunate to have Joe, Dr. Ian Miller, who is fantastic, a strong clinical team. We have another epileptologist on the team, who has been critically vital on our payer side. So we have very strong intellectual firepower. composed out there. And obviously, markets have been quite difficult for all corporations in the biotech sector. In fact, we see ultra-orphan neurodiseases due to lack of being left behind.
We’ve won many incoming calls from corporations that have giant portfolios and can’t locate the resources to work in the ultra-orphan neurological space. Therefore, we believe that there will be opportunities for us over time. We want to remain focused on our execution of our late-stage programs, but I think we’d like to do more in the space. We all appreciate it. Thank you for the question. Jay. The operator will move on to the next question.
Operator
We have no additional questions at this time. I give the ground back to Mr Scott Braunstein for the last word.
Scott Braunstein
Well, thank you and thank you all for calling today. Thank you for any questions. In fact, we couldn’t be more excited about where we are now, the early days of this ZTALMY launch and the clinical progress we are making, as well as the pipeline expansion and now the extended cash flow. So we really appreciate all your time today and look forward to seeing you live. Have a great day for everyone.
Operator
Ladies and gentlemen, this concludes today’s call. Thank you for your participation. You can now log out.