Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS) Q3 2022 Earnings Call Transcript

Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS) Q3 2022 Earnings Call Transcript November 8, 2022

Marinus Pharmaceuticals, Inc. beats earnings expectations. Reported EPS is $1.89, expectations were $0.47.

Operator: Ladies and gentlemen, greetings and welcome to the Marinus Pharmaceuticals’ Third Quarter 2022 Financial Results and Business Update Call. And now it’s my pleasure to introduce your host, Sasha Damouni Ellis, Vice President of Corporate Affairs and Investor Relations. You may begin Ms. Damouni Ellis.

Sasha Damouni Ellis: Thank you and good morning. With me from Marinus are Dr. Scott Braunstein, Chief Executive Officer; 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 are making today are forward-looking statements under the securities laws. These forward-looking statements and both substantial risks and uncertainties that could cause our clinical development programs, future results, performance or achievements to differ significantly from those expressed or implied by such forward-looking statements. These risks and uncertainties and risks associated with our business are described in the company’s reports filed with the Securities and Exchange Commission, including Form 10-K, 10-Q and 8-K. I will now turn the call over to our CEO, Scott Braunstein.

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 ZTALMY. 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 Christy Shafer to discuss our commercial progress, including strong early demand and 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 3 programs in refractory status epilepticus and tuberous sclerosis complex.

Starting with TSC, we’ve been actively engaging with the community, including our participation in the recent World TSC Conference. After our team’s 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 TSC, and that ZTALMY 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 amendments 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 IV ganaxolone and accelerating enrollment in the study. The amended protocol has been adopted across a growing number of clinical sites and we expect full adoption by year end.

Sites continue 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 3 TrustTSC 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 ZTALMY 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 CDD Marketing Authorization Application by the end of November, which would result in a CHMP opinion by the end of the first quarter of 2023.

I want to acknowledge the entire Marinus team and our partners at Orion Corporation for their tireless efforts to ensure that our responses to the EMA questions are complete and robust. Together with Orion, our teams continue to prepare for potential European commercial launch next year. In addition, we believe that there is a broader global opportunity for ganaxolone and are exploring further ex-U.S. commercial alliances. We are targeting a 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 a contract option by BARDA and completion of the revenue interest financing agreement with Sagard, we have executed on our plans to extend our projected cash runway with non-diluted financing into early 2024 while creating the opportunity to secure a U.S.-based source of API to improve our future business fundamentals.

Furthermore, with the equity offering announced last night, which is expected to close on November 10, 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 thank our existing shareholders for the commitment to the organization. Upon closing the equity offering, our new cash balance will allow us to continue to invest in the ongoing ZTALMY 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 Shafer, for updates on the commercial launch of the ZTALMY. Christy?

Christy Shafer: Thank you, Scott. And good morning. I’m pleased to share our early progress on the commercial launch of the ZTALMY 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 ZTALMY could provide in seizure management. We launched ZTALMY on July 28 and by the end of the third quarter generated $560,000 in net product revenue. During those first nine weeks of launch, we received over 50 CDD prescription enrollment forms, more than 30 of which were for new commercial patients, not previously treated with the ZTALMY, with the remainder encompassing the transition of all U.S., CDD, OLE, and EAP patients to commercial products.

Importantly, these prescriptions are coming from a diverse prescriber base of over 40 unique accounts, including interest from outside of our 265 target accounts. We believe this speaks to the unmet medical need in the CDD community as well as the strength of our digital marketing tools, publication strategy and robust 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 CDD 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 CDD patient population will access coverage through both fee-for-service and managed Medicaid with the remaining 40% being managed commercially.

As of November 1, ZTALMY received positive coverage criteria from over 15 payers representing approximately 70 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 ZTALMY. 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 CDD treatment landscape and ZTALMY profile is the first and only product indicated for seizures associated with CDD. The field interactions are being supported by our educational speaker programs as well as two product theater, one which took place in October at the Child Neurology Society Annual Meeting and the second planned in December at the American Epilepsy Society Annual Meeting.

Our formal ZTALMY speaker bureau commences in December with a meaningful number of programs planned through the end of next year. We will continue to educate and raise awareness within the CDD patient community starting with the CDD 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 CDD communities. We’re grateful for the insights and support that the IFCR, CDKL5 alliance and the LouLou Foundation provide the families living with CDD. We are very pleased with our early launch progress and are grateful for the impact we believe we are making on CDD 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 leader to my team, Kristin Rudisill who has a strong track record of success and hospital launch experience to support the development of our U.S. commercial strategy for status epilepticus. I’ll now hand the call over to our Chief Medical Officer, Joe Hulihan to discuss our ongoing development programs, including the data we continue to generate and present at scientific conferences which remain critical to supporting our launch and driving awareness of ZTALMY.

Joe Hulihan: Thank you, Christy, 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 RAISE 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 propofol for up to 18 hours rather than excluding any patient who had been treated with high dose IV anesthetics regardless of how brief the administration of these agents may have been. We believe this is an important change because it will allow for the enrollment of patients transferred to the ICU from other hospitals or the emergency room.

Two of the most common pathways for identification or refractory status epilepticus. The original protocol excluded many of these patients who represented the significant proportion of the Phase 2 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. As Scott mentioned, the majority of RAISE sites have implemented the protocol amendment with the remaining sites expected to transition to the revised protocol by year end. Investigators have 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 RAISE 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 3 RAISE trial patients. We’re pleased to share that as of September, the baseline characteristics of the Phase 3 patients closely resemble those of the patients enrolled in our Phase 2 RSE study. Participants in both studies are roughly the same age. A similar proportion have a history of epilepsy and they have similar lean baseline seizure burdens and status epilepticus severity. These demographics are outlined on Slide 29 of our November corporate deck. In addition, in September, we presented data from the Phase 2 RSE study and an overview of the ongoing RAISE trial at the London-Innsbruck Colloquium on status epilepticus.

The presentation received a positive response from those attendance who are leading researchers and clinicians in the field. They express that 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 attend the Neurocritical Care Society Annual Meeting in October where we presented two posters with data from the IV ganaxolone 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 from RAISE study sites. Additionally, neurocritical care physicians from other leading centers expressed interest in participating in the trial.

Looking to the future of the RAISE study, we will continue to actively monitor what’s working well at 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 will shortly be submitting a request to the FDA with supporting data that potentially allowed us to include younger patients. Given the higher incidence of status epilepticus in 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 conduct of an interim analysis may affect the sensitivity of a study to detect a treatment effect should be continue to full enrollment. We have designed the interim analysis for the RAISE study to have a minimal effect on the efficacy outcome of a 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 3 RSC trial, the RAISE 2 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 indication for IV ganaxolone in the U.S. Moving on to the Phase 2 RESET study in established status epilepticus. We have activated the first site and expect to begin U.S. 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 oral ganaxolone programs. As Christy stated in her remarks, we continue to work to increase awareness of ZTALMY across the scientific and medical community. On October 13, we presented two posters at the Child Neurology Society meeting from our pivotal Phase 3 Marigold study, including open-label extension data that showed continued seizure reduction over a two-year period.

Although 24 month data was available for only 16 of the 54 patients remaining in the open-label extension, the 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 30.7% 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 the 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. It was administered during the Marigold study, which showed ZTALMY was associated with improvements in certain domains of behavior, specifically reductions in the compulsive behavior and manic hyperactive components of the scale.

While these findings would require confirmation through further research, they suggest that there may be potential benefits of treatment beyond seizure reduction. Moving to our Phase 3 TrustTSC trial in tuberous sclerosis complex, we are pleased to share that we are actively screening patients. We’re targeting 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. We’re encouraged 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 a Phase 1 single ascending dose study of the first of our reformulation candidates, which we believe support advancing it further in clinical development.

Participants in the Phase 1 study received reformulated oral ganaxolone that doses ranging from 100 to 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 Cmax. 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 Cmax related adverse effects such as Sonance. The next steps in the evolution of this program include enrollment of an additional Phase 1 study cohort to assess single 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 ascending doses, which we would be in a position to initiate in the first half of 2023. We anticipate selecting one of the second generation ganaxolone formulations for a Phase 2 study in Lennox-Gastaut Syndrome to begin in 2023. Lennox-Gastaut Syndrome is highly treated refractory and we believe the new oral formulation has the potential to provide more consistent and predictable exposure to ganaxolone, which we expect would allow physicians to individualize dosing to achieve an optimal response for each patient. Additionally, we’ve 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 1 data targeted for 2024. We look forward to continuing to raise awareness within the medical and scientific community over the coming months, and we’ll have a robust presence of the American Epilepsy Society annual meeting in December, including presentations of data from our oral and ganaxolone programs during the general sessions, as well as during Marinus Scientific Exhibit. As a clinical team, we remain focused on our Phase 3 clinical programs and Status Epilepticus and Tuberous Sclerosis Complex, advancing our reformulation and pro-drug candidates, as well as our continued support for ZTALMY and CDD. Now, I’ll turn the call over to our CFO, Steve Pfanstiel, who will provide you with a financial update.

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Steve Pfanstiel: 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 our rare pediatric disease priority review voucher receiving gross funding of $110 million. That was followed in September with an additional $12 million of increased funding from BARDA as they exercised the first contract option to support our API on-shoring initiative.

Just recently in October, we completed a royalty monetization agreement with Sagard Healthcare, which provided an upfront payment of $32.5 million for future royalty payments related to U.S. revenues associated with ganaxolone including ZTALMY. We ended the quarter with cash and cash equivalence of $168.2 million, which does not include the funds received from the Sagard 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 with ZTALMY product sales in the U.S. For the third quarter of 2022 we recorded the ZTALMY net product revenues of $560,000, which represents just over two months of actual commercial launch. This revenue consists of the ZTALMY product sales to our single-specialty pharma partner Orsini and includes the estimate of growth net deductions inclusive of expected Medicaid discounts. Focusing on BARDA revenues only we recognize revenues of $1.8 million and $5.1 million for the three and nine ended September 30, 2022 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 in $19 million and $58.5 million for the three and nine months ended September 30, 2022 respectively as compared to $18.4 million and $55.5 million for the same periods in the prior year. The change was due primarily to cost associated with increased R&D headcount and startup of the Phase 3 TrustTSC Trial. Selling, general and administrative expenses increased to $13.4 million and $42.2 million for the three and nine months ended September 30, 2022 respectively as compared to $9.5 million and $26.7 million for the same periods in the prior year. The primary drivers of the change were preparation for and launch of ZTALMY 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 PRV 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 30, 2022 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 non-cash stock based compensation expense of $3.9 million and $11.1 million for the three and nine months ended September 30, 2022 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 30, 2022 as compared to cash used in operating activities of $33.7 million for the same period in the prior year. 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’ll turn the call back to Scott, who will provide concluding remarks.

Scott Braunstein: Thanks, Steve. This has been a busy and exciting time for the Marinus team and we are pleased with the early launch results, the progress we’ve made toward a potential approval in Europe next year and the organization’s research efforts. We look forward to building on this commercial foundation with ZTALMY and advancing our two Phase 3 programs over the next 12 months. To conclude, I would like to mention that Marinus will be holding an investor breakfast Monday morning, December 5 at AEs. We will be meeting with a variety of physicians and efficacy organizations over the course of the weekend and hope to see you there 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. Sasha will provide more details on the event over the coming weeks. Operator, can you now open the call to questions?

Q&A Session

Operator: Yes. Thank you. We’ll take our first question from Marc Goodman with SVB Securities. Your line is open.

Scott Braunstein: Good morning, Marc. You may be muted. Operator, we don’t hear anything.

Operator: I don’t hear Mr. Goodman either. Either Mr. Goodman, if you can hear us please re-queue. We will move to our next question from Joseph Thome with Cowen and Company. Your line is open.

Joseph Thome: Hi there. Good morning, and thank you for taking our questions. Maybe a first one on ZTALMY itself. Good to see the sales start rolling in. It looks like consensus for the year is hovering around $2.5 million. I guess based on what you know of the launch so far, is this a number that you’re comfortable with and can you comment on any stocking patterns that we should be aware off? And then second on RSC, I taught the upper end of the enrollment for both the Phase 2 and the Phase 3 is eight years and then you discuss potentially going down to kind of 12 years or below. Is there any difference in response either to therapy or addition to normal standard of care based on this sort of broad that would be helpful? Thank you.

Scott Braunstein: Steve, why don’t I turn over to you for the ZTALMY question, and then we’ll let Joe to the RSC question. Yeah,

Steve Pfanstiel: Yes. Good morning, Joe. Thanks for the question. With regard to ZTALMY revenues we are providing guidance for 222 or 2023 so I won’t speak to that specifically, but as we said, we’re really focused on the number of patients on therapy. We’re focused on driving access across government and commercial payers. We’re really extreme with €“ extremely happy with our progress on both those fronts. We feel like we real have a really strong trajectory there in just this 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 are working with a single specialty pharma partner. We have a really tight distribution process. 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 a very limited inventory stocking impact, and that will be the case moving forward.

Joseph Thome: Hey, Steve, before we turn over, Joe, maybe you want to remind the ZTALMY and the audience about our €“ what we talked about break even and how we’re feeling about that relative to the first few months of launch?

Steve Pfanstiel: Yes. Joe, just to add a little more color in terms of the number of scripts in that trajectory. We’ve said that €“ we think the $25 million investment in the CDD commercialization is really a two-year to breakeven type of investment. So by the middle of 2024 we think 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 million to 250 million patients €“ 240, 250 patients on therapy.