Mirum Pharmaceuticals, Inc.

Hello, everyone, and welcome to the Mirim Pharmaceuticals Third Quarter 2024 Financial Results and Business Update. My name is Seb, and I'll be the operator for your call today. If you would like to ask a question during the Q&A session, please press star 1 on your telephone keypad. If you would like to withdraw your question, please press star 2. I will now hand you over to Andrew McKibben, Vice President of Investor Relations and Finance. Please go ahead.

Thanks, Seb, and good morning, everyone. I'd like to welcome you to Merum Pharmaceuticals' third quarter 2024 conference call. I'm joined today by our CEO, Chris Peets, our President and Chief Operating Officer, Peter Radovich, our Chief Medical Officer, Joanne Kwan, and Eric Bierkolt, our Chief Financial Officer. Earlier today, Merum issued a news release announcing the company's results for the third quarter 2024. Copies of this news release and SEC filings can be found in the Investors section of our website. Before we start, I'd like to remind you that during the course of this conference call, we will be making certain forward-looking statements based on management's current expectations, including statements regarding Merrim's programs and market opportunities for its approved medicines and product candidates. These statements represent our judgment as of today and inherently involve risks and uncertainties that may cause actual results to differ materially from the results discussed. We are under no duty to update these statements. Please refer to the risk factors in our latest Form 10-Q and subsequent SEC filings for more information. With that said, I'd like to turn the call over to Chris. Chris?

Thanks, Andrew, and good morning, everyone. It has been another outstanding quarter for Merum. With strong commercial execution and advancing pipeline, we have made excellent progress across our 2024 strategic objectives, accelerating our growth as a leader in rare disease. As a reminder, our goals this year have been to drive continued growth across our three commercial medicines, expand the labels for both Livmarley and Pfeffik and Kenodial and CTX, leverage the strength of IVAT inhibition in the adult settings of PSC and PVC, and continue expanding our portfolio of medicines for rare diseases. We have delivered on all of these this year. Specifically in the third quarter, all three commercial medicines saw continued growth, with net product sales of $90.3 million, an 89% increase from the third quarter of 2023. This very strong quarter was driven by the continued underlying trends across the brands, with increased PFIC and international uptake for Lipmarlin. With this strong execution from our commercial team, we are increasing our 2024 guidance to $330 to $335 million in full-year net product revenue. We have also made excellent progress with our pipeline. The list of that has been granted breakthrough designation for cholestatic pruritus and PVC based on the Vantage study interim results. As a reminder to qualify for this designation, the FDA requires preliminary clinical evidence that indicates that the drug may demonstrate substantial improvement over existing therapy on at least one clinically significant endpoint. This designation emphasizes the continued unmet need in PBC and the strong early treatment effects seen in the interim results, which have now been accepted as a late-breaking abstract at the upcoming American Association for the Study of Liver Diseases meetings. Our clinical studies in PBC and PSC remain on track with our enrollment guidance, and the Phase III EXPAND studies evaluating lipmarly in additional settings of cholestatic gyritis is now underway. For kinadiol, I'm pleased to announce we received priority review for our NDA and CTX with the PDUFA date of December 28th. And finally, I'm also happy to share that we've expanded our early development pipeline with the addition of MRM3379, a candidate for the treatment of patients with Fragile X syndrome. Fragile X syndrome is a rare genetic disorder that is the leading cause of intellectual disability that affects approximately 50,000 male patients across the U.S. and Europe with no approved therapies. Strategically, this program aligns well with the capabilities we are building in rare genetic neurology to support kinadiol on CTX. We will continue to look for opportunities to leverage our industry-leading rare disease expertise in patient settings where there's a clear need for high-impact medicines. I'm proud of all of our progress to date in 2024 and the potential ahead from here. And I'll now turn the call over to Peter to go over the commercial business.

Peter? Thanks, Chris. I'm pleased to say that we had another strong quarter with total net product sales of 90.3 million. For Liv Marley, total global net product sales grew to 59.1 million in the third quarter. U.S. sales were 43.5 million, while international sales were 15.6 million. In the U.S., we continue to see solid growth in allogeal syndrome and are seeing added contribution from PFIC as reimbursement has come through earlier than we expected. Internationally, we continue to see strong underlying demand growth in allogeal syndrome from both our core markets and our distributor partners, despite pricing headwinds. With Livmarly now approved in PFIC in Europe, pricing and reimbursement negotiations are underway. We also saw continued steady growth from Holbaum and Kinadol, which had 31.2 million of net product sales in the third quarter. And the commercial team is looking forward to our PDUCA date for CTX in December. Overall, it's been a tremendous year for the commercial business. We continue to grow our impact for Algea syndrome patients. We expanded the label for Livmarli, allowing us to extend our reach to the PFIC community. We effectively navigated price negotiations in Europe And we successfully integrated the bioasset portfolio, leading to record sales. With the increased full-year guidance of $330 to $335 million, we look forward to ending the year on a high note and continuing our strong execution into 2025. With that, I'll turn it over to Joanne. Joanne?

Thanks, Peter. It was a productive quarter for our pipeline, and I'm excited to share our progress. First, I want to highlight the progress we're making with Felixivet. We're thrilled with the breakthrough therapy designation for Velixibat as a potential treatment for cholestatic pruritus in patients with PBC. This designation highlights the significant burden patients face and the need for an effective treatment. The decision was based on the positive interim analysis of the Phase 2B Vantage study, which showed statistically significant improvement versus placebo in pruritus for PBC patients treated with Velixibat. I'm happy to share that we will be presenting this data as a late breaker at AASLD's deliver meeting next week. For both the PSC and PBC programs, we've had great engagement with investigators and advocacy groups over the last few months. We're happy with how both studies are progressing and remain on track to full enrollment by the second half of 2025 for PSC and in 2026 for PBC. Moving on to expand. As a reminder, the study represents an exciting potential label expansion opportunity for Live Marley in additional settings We have started opening sites and are excited to start screening patients. Looking toward the end of the year, we're on track for our December 28th PDUFA date for Kenodial and CTX. This is an exciting step forward in extending our presence in rare genetic neurology. Next, I'm thrilled by the opportunity in Fragile X Syndrome with MRM3379. Fragile X Syndrome, or FXS, is the most common monogenic cause of intellectual disability. It is also characterized by anxiety and language delays. It is an X-linked disorder, and males tend to be more severely affected. MRM3379 is a selective phosphodiesterase, or PDE, 4D inhibitor, and this mechanism has shown proof of concept in Fragile X in the clinic. We believe MRM3379's brain penetrant profile is differentiating, and we plan to evaluate its potential in males with FXS in a Phase II study. We plan to start this study next year. I'm pleased with the progress we made this quarter across our different assets and look forward to providing updates in the future. I'll now turn it over to Eric. Eric? Thanks, Joanne.

Earlier today, we issued a press release that included financial results for the third quarter, which I'll briefly summarize. Overall, we continued our trend of strengthening quarter-over-quarter financial performance. I'm excited to announce that in Q3, we achieved positive operating cash flow for the first time. Net product revenue in the third quarter 2024 was $90.3 million compared to net product revenues of $47.7 million in the third quarter last year. Total operating expenses for the quarter and the September 30 were $103.9 million, which includes R&D expense of $31.7 million, SG&A expense of $50.5 million, and cost of sales of $20.8 million. The total operating expense for the quarter included approximately $18 million of non-cash, stock-based compensation, and depreciation and amortization expense, of which approximately $6 million was included in cost of sale. For the quarter ended September 30, net loss was $15 million, or $0.32 a share. Our cash, cash equivalents, and investments was $293.8 million as of September 30, a reduction of $1.6 million from the previous quarter. The third quarter cash use included the payment of a $10 million milestone to Takeda upon European approval of the Liv Marley PFIC indication. The upfront fee for MRM 3379 was $7.5 million, which we will pay in the fourth quarter with cash on hand. Under the deal terms, we are subject to a $7.5 million phase three start milestone and certain regulatory and sales-based milestones. In summary, we continue to grow the commercial business and advance and enhance our drug development pipeline, all while remaining financially independent and fiscally disciplined. Now, I'll turn the call back over to Chris for final comments. Thanks, Eric.

It has been a great quarter for Miriam. Our commercial programs are performing well with strong growth resulting from increased full-year guidance. Our pipeline is delivering with FLEXVAT's recent breakthrough designation and the initiation of the EXPAND study for Livmarlie and added MRM 3379 with the plan phase two start next year. And the Miriam team has driven all of this with financial discipline, setting us up for a strong finish to the year. With that, operator, please open the call for questions.

Thank you. As a reminder, to ask a question, please press star 1 on your telephone keypad. If you would like to withdraw your question, please press star 2. First question is from Gavin Clark Gartner from Evercore ISI. Please go ahead.

Hey, guys. Congrats on all the progress, and thanks for taking the questions. So I just wanted to focus in on Fragile X. So first question here. Maybe just for those who are less familiar, what's the current registrational endpoint in Fragile X? Is it behavioral or cognitive clinical endpoints? And is there any opportunity to pursue an accelerated path?

Kevin, thanks for the question. I'll actually turn it over to Joanne to talk about our strategy and thinking on endpoints.

Yeah. Hi, Kevin. Thanks for the question. You know, our thinking is that we can use the NIH toolbox as a registration endpoint. Obviously, we've just acquired the program, so we haven't had further discussions with the FTA. But, you know, we do think this is a very viable path forward. We've recognized it in the past. Different endpoints have been used, and I think that's also been problematic for the field. And if you look at the history, there really have been no positive, real positive studies other than for the Ptomelanus, which is also a PD4 inhibitor. However, with our molecule, we think that there's the selectivity and really the high brain penetration actually provides some advantages in developing it forward. So we're really, you know, excited in terms of moving this forward and do think that there's a viable path forward with the FDA.

And just to add one comment on the NIH toolbox endpoint. This is a, you know, the patient conducted a test and, you know, some of all of the programs that we have here at Merrim leaning on the patient-reported outcomes and our experience with that. clinical plan.

Got it. That's helpful. And are you planning to have any engagement with regulators prior to initiating the Phase II, or will next engagement likely be end of Phase II meetings?

Yeah, thanks, Gavin, for the follow-up. We will be engaging with the FDA, you know, early next year on this program. Got it. Thanks. Thanks for the questions.

Our next question is from Jessica Phi at JPMorgan. Please go ahead.

Hey, guys. Good morning. Thanks for taking my questions. I had a couple. First, on Velexabat, can you share a little more detail on the data we should expect from the late-breaking presentation at AASLD from the Vantage interim? How much of an update will this be? What would you focus the street on? And what subgroups, if any, will you present? That's one. Second, Liv Marley looks like growth was really solid quarter over quarter, year over year. I think last year, 3Q, you talked about seasonal headwinds. Did you see any of that this year? And then lastly, on business development and the context of this latest in-licensing, how do you see the company evolving with this asset kind of going beyond the kind of rare liver disease kind of legacy focus? And do you expect to kind of remain active on the BizDev front on the back of this in licensing? Thank you.

Thanks, Jess, for the question. I'll touch on the quick comments on Velixibet and BD strategy and let Peter speak to the commercial trends. Now, for the Velixibet late breaker, you know, we're just a handful of days away from these being released. So, just kind of point you towards that. And the update's coming soon with data there. Real focus, the top line is already out there, right, that we've seen just really strong response on pruritus. That is the registrational endpoint we're pursuing. So still very excited about this data and excited to share the update. On BD strategy, I think just to take a step back and put the 3379 addition as an early stage program, we do see this as one of several steps that we've been pursuing now for years here at Merham with a team that's been focused on rare disease, in particular rare genetic disease, and programs that are overlooked that can add a lot of value. So the 3379 program is a great example of that, where it fits well with our capabilities in terms of how genetic diseases get diagnosed and treated. on the development side, a thoughtful approach to endpoints and patient-reported outcomes. And really, the last comment I'll make, I see this as kind of a bookend of an example of things that we look at between the transaction last year, bringing on commercial products. You'll see this as kind of the earlier end of the spectrum in terms of where we focus our time for BD strategy. With that, maybe I'll pass it over to Peter on the commercial side.

Yeah, with regards to the Liv-Marley trends, Jesse, as you mentioned, in 2023, you know, we did comment on a bit of a slowdown in some of the summer months with Liv-Marley. You know, it really has not been something we observed at all in 2024. This quarter was really solid continued growth in algeo syndrome in line with kind of what we've been seeing with algeo syndrome and a strong kind of long performance from PFIC in the U.S. as well as strong growth in international.

Thank you.

Thanks for the question. Our next question is from Manny Faruha from LeeRink Partners. Please go ahead.

Hey, guys. Good morning. You have Brian on for Manny. Thanks for taking our questions and congrats on the quarter. I guess a few on 3379. Can you just provide any specifics around the deal terms such as royalties or future milestones that we should expect for this program? And then more specifically on the OpEx line, you know, do you expect this program to impact OpEx moving forward or is it pretty negligible? And then just one on the commercial front, you know, can you share any color on where you're seeing this acceleration in PfeC is coming from? So, you know, whether that be new patient starts or switches from a competitor and what that means for growth moving forward? Thanks.

Brad, thanks for the question. I'll pass it off to Eric and Peter to touch on both questions.

Yeah, thanks. So I did provide some comments on the financial terms in my commentary that we just went through. So the upfront was $7.5 million, and really the only sort of pre-regulatory approval milestone is the $7.5 million phase three stock milestone. Our royalties we're not going to comment on, but they're very typical for a deal of this stage, so pretty modest. In terms of the increase in spend, it's too early to be too specific because we're early in terms of developing our plans for this asset, both on the clinical side as well as the manufacturing side. But to give you just a rough sense in terms of percentage of our R&D spend, you could think about it being sort of in the mid-teens, percentage-wise.

Yeah, and just to kind of put that in perspective of how we looked at bringing this program into MIRM, see it as very much an efficient, early-stage program that is quite targeted investment to get to proof of concept. So the phase two design and investment into that from the deal terms to the clinical spend, really not a big step in terms of the amount of dollars spent.

And yeah, Brian, to your question on what's driving the PFIT performance, I think an overarching comment is really reimbursement. Our market access and reimbursement team did a great job securing coverage for PFIT kind of sooner than we would anticipate. based on even our LGO approval as well as other rare disease approvals when you usually expect a few more quarters of new-to-market policies where reimbursement might be slower to come online. So that came online faster than we thought. In terms of sources of demand, we've talked in the past about our expanded access clinical trial rollover patients being in about the low 20s in the U.S. that were ready to be rolled over after launch. And then We are seeing de novo demand. Probably the majority of those are IVAT naive, and we are seeing some switches as well.

Awesome.

Thank you.

Thanks for the questions.

Our next question is from , from Stiefel. Please go ahead.

Hey, Greg. Good morning. Thanks for taking our questions, and congrats on the quarter looking pretty bright ahead. I guess a couple of questions from me as well. Just thinking about the OPEX growth, or I guess not OPEX, but the revenue guidance increase, just curious if it is primarily driven by allogel growth ahead in Q4, or is it really going to be about PFIC as we think about modeling purposes? When we think about expand, Joanne, can you expand a little bit more? You said sites are opening. But in terms of outlook on cadence of patient enrollment and any kind of timeline that you envision in terms of enrollment completion, that would be great. And then lastly, on the MRM3379, just curious about your overall strategy on the neurology side. I mean, Fragile X is the starting point here, but are you thinking about expanding on the PDE4 inhibition for other indications? Is there anything that's lower hanging fruit after Fragile X? Thanks so much.

Thanks for the question. Joanne, do you want to start with the EXPAND question and move from there?

Yeah, thanks for the question. In terms of EXPAND, we're right on track in terms of where we are in terms of the study starts. So we have sites up and running and we expect to start screening shortly. You know, our guidance in terms of enrollment timeline has not changed from before. So we expect to, you know, hopefully in a few months or so. So that's really right on track. And then on the sales trends.

Yeah, yeah. So, yeah, we obviously raised our guidance to 330, 335 in terms of what drives that. You know, we continue to see strong algeo growth in the U.S. kind of in line with historical trends. And, yeah, the uptick that we saw in Q3 and this kind of coming forward in our guidance in Q4 is really, really driven by the PFIC launch in the U.S.

That's great. And just to kind of touch on the, you know, the the broader opportunity for PDD4D, there may be potential in other indications, but we're approaching this first in a very focused way to get to proof of concept in Fragile X in the Phase II program before we consider putting broader dollars against it. So, you know, really looking forward to generating that first proof of concept data and what that can mean for Fragile X patients, and we'll go from there.

Makes sense. Thanks for taking our questions, guys.

Thanks, David. The next question is from Brian Scorny at Baird. Please go ahead.

Hey, good morning, guys. Thank you for taking my question. On the PDE4D, I know shinogis run a phase three with a zephyr molast in Fragile X. And so I'm just kind of wondering, is the phase two data with that sort of the basis of the licensing deal here? And then can you maybe compare and contrast your molecule with the shinogi one? And would you anticipate initiating phase two before the readout of that study next year, or would you maybe wait on those results to decide on potential endpoints and power? Thanks.

Yeah, thanks for the question, Brian. That did weigh into how we looked at the program. The phase two proof of concept data from that program, I think, are quite interesting. I'll let Joanne speak to a little bit of the differentiation we see. But just first in terms of study start timing, I see these as somewhat independent, though we are obviously monitoring that . But given the differentiated profile, I think that we could see stronger results than what comes out of that program. I'll let Joanne speak to some of what we liked about this to bring it into the pipeline.

Yeah. Hi, Brian. Thanks for the question. You know, the molecule we have is highly selective, and I think one of the key differentiation points from the other molecule is just the high brain-to-plasma ratio, simply because, you know, what we're trying to do is alter the cyclic AMP levels within the brain. And so, we think that our molecule has some potential advantages here. So, I think that's the key differentiation point, and obviously, we'll keep that in mind as we look at results for the Tomlinson Program. We're excited by the potential for MRM-3379 and other potential applications.

Great. Thank you. Thanks for the question. The next question is from David Lebowitz from Citi. Please go ahead.

Thank you very much for taking my question. Can you compare the decisions to license the trivia assets with 3379 and what you were thinking in general about your strategy relative to business development going forward?

Thanks, David. I can give a little bit of color, and I'll turn it over to Peter to talk about some of the things that we're active looking, you know, the spaces we're active looking at and kind of how the search came to both of these products. But the way I've put some perspective on these two transactions, and frankly, actually the founding transaction of Merum and acquiring Lithmarly and Velixadat, is really looking for overlooked value that the Merum team can grow and expand on. And the spectrum of types of transactions we've looked at from commercial to phase one slash phase two ready is where I think our team has really deep expertise in rare disease, in particular in rare disease settings that are genetically driven, where you can use patient reported outcomes, have a lot of experience with that. And so on the commercial side, gives us the ability to drive financial performance on the clinical side gives us the ability to build future growth into the company so see those uh quite different transactions working together uh really well to build out the profile of mirim i'll turn over to peter to talk about kind of what um what we look at yeah yeah i mean i think you can it's a it's a great question you can look at these two transactions uh you know the transaction with revere for the bile acid products came by way of us being in the pediatric liver space knowing knowing what

you know, may be underappreciated and create significant value for healthcare professionals, patients, caregivers, and that, you know, led to our insight there, and we're happy with the performance that we've had in over a year of having these products in our hands, continuing to grow these to, you know, record highs. I think, you know, one point about Kenadol, interestingly, is most of the prescribers are neurologists, and so really for over the last year, we've kind of been in this Neurofield, even though it's a biolastic replacement product, the manifestations are neurologic, and that's kind of gotten us into this field and, you know, the medical conferences, et cetera, where we kind of, you know, sort of gained insight and, you know, what may be another underappreciated program in 3379. So I think, you know, one theme is, you know, high impact for patients and underappreciated rare disease programs.

Thank you. Thanks for the questions.

The next question comes from Steve Seedhouse at Raymond James Financial. Please go ahead.

Hi, this is Timur Vanagopal for Steve Seedhouse. Congrats on the quarter. We have a couple of questions about Fragile X. So first one is, do you have plans to enroll patients younger than 18 years old? And at what point could you enroll those patients? And then in terms of the molecule specifics, do you know how much more CM activity you can achieve in the brain That is, how much is the Tomilast leaving on the table versus normal? Thank you.

I'll start and let Joanne finish on some of the details, but I appreciate the question here. We are planning to interact with FDA early next year to finalize some of the Phase II elements, but do have some initial thinking on what we're going to propose. Caveat that with, you know, we'll update it with regulatory input.

Yeah, thanks for the question. In terms of the age range, you know, it's typical in these kinds of programs to start with adult patients and then try to step down in age. And that's the basic approach that we will have as well. Currently, you know, we have information from the prior phase one program, which included a fairly large number of adults, healthy volunteers, and elderly patients. And so we'll use that strategy and then step down in age. And that we think going down in age will be particularly helpful for Fragile X because most patients with Fragile X are diagnosed around age three. So having something for children will be particularly impactful for this disease. In terms of how much more central nervous kind of system activity, that's a pretty difficult thing to gauge. We do know that, you know, our brain to plasma ratio is actually quite a bit higher. So we think that that will give us some big advantages here. Obviously, the proof will be in the pudding in terms of looking at results, but we're pretty confident that this is an important differentiating feature for 3379 compared to any of the other compounds here in the clinic.

Thank you. Thanks for the question. The next question is from Mike Ols at Morgan Stanley. Please go ahead.

uh good morning thanks for taking the question uh maybe just one on kinadol and ctx now that you sort of have a padufa date late december maybe just remind us what the potential market opportunity is there and what impact getting that in the label might have on on sales thanks yeah thanks for the question mike um yeah so we're you know really really excited about the potential approval and the opportunity to you know be out there with active promotion just a reminder well

Keenadol is approved under a different indication and available under emergency use here for CTX. This will be a chance to have it on label and with active promotion. We think that the best literature estimates suggest there's about 1,000 to 2,000 prevalent patients in the US with maybe only about 10% of those diagnosed. So a lot of our effort on disease state awareness and then ultimately upon approval promotion we'll try to increase that diagnosis rate. And to the extent we can increase it even a little bit, I think it could have a meaningful impact on the current sales trajectory you see reflected in our numbers today.

Great. Thank you. Thanks for the question.

The next question comes from John Wallaban from JMP. Please go ahead.

Hi. This is Catherine on for John. I have a quick question about kind of what you're seeing at CUS and this global expansion for Marley, if you could comment on that. And then any updates to your views on the PFIC launch, given how strong the quarter's been?

Thanks for the questions, Catherine. Yeah, the one comment I'd make on PFIC overall, you know, this is early days and seeing, you know, good initial demand. But some of this was really just pulled forward of reimbursement so that's uh we do see this step up as really being a stronger quarter than um than others because of that earlier reimbursement but i'll let peter speak to some of the international uh aspects yeah yeah absolutely i mean we uh we've now uh you know have pricing and reimbursement approval analogy oh and uh the four major eu markets plus several mid-sized markets so really

Really proud of the execution our team has delivered there and, you know, distributor partners around the world continue to find more allogeal syndrome patients that we potentially benefit with Marley. So, you know, the allogeal launch in Europe and international markets continues to go well. And yeah, the PFIC approval came recently. So that is, you know, obviously not reflected in international sales now. Maybe as you move into the back half of next year, mid to back half of next year with pricing and reimbursement coming in.

Thanks so much.

Great. Thanks for the question. The next question comes from Ed Arthur from HC Wainwright. Please go ahead.

Hi. Good morning, everyone. This is Thomas Yip asking a couple questions for Ed. Thank you for putting out our questions. So first question, perhaps following on to this question, can you give us more details on peer discussions for PSEC and the U.S. and EU? I suppose specifically, what percentage of target subscribers have you reached so far?

Yeah, I mean, the beauty of PFIC really in every market where I've been is the prescribers are the same as algeol syndrome and maybe even a subset of those, quite frankly. So I think we've, at this point, you know, in the U.S. launch of PFIC reached substantially all of them and have seen, you know, a nice interest in the profile and great demand in sales as you're seeing in the Q3 numbers. You know, early days in Europe, I think as we Um, about reaching Germany and places where you can launch more quickly, but I think we'll be reaching the vast majority of our providers as we move into next year. And. As the individual countries pricing and reimbursement comes closer to where you've got conversations are.

Got it. And is that perhaps another question for Likmali? Just wondering if you can discuss enrollment progress for the expense study or perhaps any initial investigative feedback that you have so far.

Thanks, Thomas, for the question. I mean, I can just touch on that briefly in that, you know, sites are just now being opened. So we have sites open and starting to screen patients. So too early to comment on the initial, you know, But I will remind you that the whole idea for the study and the study design really came from prescriber interest and investigator interest and looking to get compassionate use access for patients that are, that kind of fit within the protocol of the study. So this is an indication that's driven by physician and patient demand to add it to the Litmarly label. So still see a lot of that same dynamic that led us to put the study together in the first place.

Understood. Perhaps one more question from us. So, with the licensing of the 579, does that mean the recurrent disorder is now a disease target for Miriam or just rare genetic diseases in general?

Yeah, thanks for the question there. I mean, overall, this doesn't change what we're looking for. And we see rare disease itself as somewhat of a therapeutic area. Because the commonality and how patients are diagnosed in these genetic conditions, the thoughtful approach that you need on endpoints, that all plays across all of these different settings. And on the commercial team side, have commercialized and are active with a number of different therapeutic areas and prescribers. So while we see there's opportunities to build in some of these subspecialties, we do see opportunity outside of them as well, because we've shown that we can add value across a number of different settings, both clinically and commercially.

Understood. Thank you again for the questions. Looking forward to hearing more about 3DCM in the coming months.

Thanks for the questions. This concludes the Q&A session. I'll now hand the floor back to Chris Peets, CEO, to conclude the call.

Great. Thanks, everyone, for joining us today, and have a great day. Goodbye.

Thank you all for joining today's Miriam Pharmaceuticals Third Quarter 2024 Results Call. You may now disconnect.