This conference call transcript was computer generated and almost certianly contains errors. This transcript is provided for information purposes only.EarningsCall, LLC makes no representation about the accuracy of the aforementioned transcript, and you are cautioned not to place undue reliance on the information provided by the transcript.
spk09: Hello and welcome to the Mirum Pharmaceuticals first quarter of 2024 financial results and business update. My name is Terry and I'll be the conference operator today. All lines have been placed on mute to prevent any background noise. There will be an opportunity to ask questions today and you can do this by pressing star followed by one on your telephone keypads. I will now hand the call over to Andrew McKibben, Vice President of Investor Relations to begin. Please go ahead.
spk13: Thanks, Terry, and good afternoon, everyone. I'd like to welcome you to Merum Pharmaceuticals' first 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 Bierkult, our Chief Financial Officer. Earlier today, Merum issued a news release announcing the company's results for the first quarter 2024. Copies of this news release and SEC filings can be found in the investor section of our website. Before we begin, I'd like to remind you that during the course of this conference call, we will be making certain forward-looking statements about Merrim and our programs based on management's current expectations, including statements regarding Merrim's current and future business plans, development programs and regulatory expectations, strategies, prospects, market opportunities, and financial expectations. Merrim is under no duty to update these statements, and they are subject to numerous risks and uncertainties, and actual results could differ materially from the results anticipated by these statements. Investors should read the risk factors set forth in Miriam's 10Q for the quarter ended March 31st, 2024, and any subsequent reports filed with the SEC. With that said, I'd like to turn the call over to Chris. Chris?
spk03: Thanks, Andrew, and good afternoon to everyone. 2024 is tracking to be another year of significant growth for us, and I'm very pleased to highlight our progress across key strategic objectives to grow the commercial business, expand the indications of our commercial medicines, and advance Velixivet to market. We continue to build value while delivering on our commitment to create and commercialize life-changing medicines for rare disease. First, driving growth across our commercial medicines. Total net product sales this past quarter were $68.9 million, representing a 137% increase from the first quarter of 2023. Livmarli continues its strong performance. Newly diagnosed and prevalent patients continue to come to treatment in both the U.S. and internationally. And we are well positioned to meet our full year revenue guidance of $310 to $320 million, driven by continued demand increases across all medicines, international launches, and contribution from the PFIC approval. Second, we are also making significant progress towards expanding the impact of our potentially life-changing medicines through new approvals and label expansions. In March, we announced the U.S. approval of Livmarli and PFIC, an important milestone for Merim and the PFIC patient community. This approval represents the culmination of years of dedication from patients, researchers, and our team. We are excited to be able to bring Livmarli to this community, particularly those patients with rare genetic types of PFIC. The pivotal data was also just published this week in the Lancet, highlighting the improvements in itch, bile acids, bilirubin, and growth seen with Livmarli treatment. And we're off to a great start with positive reception post-approval. We've also made great progress preparing our upcoming NDA submission for Keen et al. for the treatment of CTX. And third, we are advancing Velixibet and PSC and PBC, which are more common adult cholestatic settings, where we can apply the scientific and regulatory insights from the Livmarli program to address bile acid accumulation in patients suffering from these diseases. This quarter, we will be taking an important step in advancing the program with our interim analyses of the in the VISTA's PSC and Vantage PBC studies, which are scheduled in June. We see both indications of significant opportunities as there are no approved therapies for PSC and no approved therapies to treat cholestatic pruritus in PBC. In summary, we continue to make excellent progress across our core strategic objectives, supported by a strong underlying financial position that will allow us to further execute on upcoming opportunities. And with that, I'll turn the call over to Peter to discuss our commercial business.
spk12: Peter? Thanks, Chris. Our commercial teams delivered another strong quarter with continued demand growth for Lib Marley across all geographies. Underlying growth dynamics remain strong across our medicines and geographies, and we are tracking well towards achieving our full year revenue guidance of 310 to 320 million. For Lidmarli, total global net product sales grew to 42.8 million in the first quarter, up from 41.4 million in the fourth quarter of 2023 and 29.1 million a year ago. U.S. Lidmarli sales were 30.8 million and international Lidmarli sales were 12.1 million. Our U.S. Algeo business continues to benefit from durable demand expansion and total Lidmarli prescriptions, with a mix of older and newly diagnosed patients starting treatment. Internationally, we are also seeing sustained demand growth from our core markets, and we continue to launch in new countries, most recently in Italy. Our U.S. business was impacted by the Change Healthcare cybersecurity incident affecting our specialty pharmacy. This resulted in a temporary disruption to insurance claims processing during the quarter, which we estimate had approximately $3 million impact on Q1 sales across our products. Turning to the recent approval of Livmarly for colsat, puritis, and PFIC patients, this is an important step forward for the business And I'm happy to say that our approval has been well received by the physician and patient community. And discussions with payers are progressing well. We continue to anticipate reimbursement and paid dispenses to materialize over the next few quarters. Turning to Kolbam and Keenadal, we recognized net product sales of 26.1 million in the first quarter of this year. Overall, I'm pleased with the strong demand we have seen year to date and how this positions us towards achieving our full-year guidance of $310 to $320 million. I look forward to continued growth in the coming quarters and years to come. And with that, I'll turn it over to Joanne. Joanne?
spk00: Thanks, Peter. We have a lot to look forward to this quarter as we continue to advance our pipeline. First, I would like to take a few minutes to talk about our upcoming interim analyses in June for our Vista's PSC study and Vantage PBC study. Starting with the VISTA's PSC study, a blinded interim analysis will be conducted to support dose selection. We have pre-specified an efficacy threshold for continuation, which is based on prior experience with IVET inhibitors in cholestatic pruritus. Using these criteria, the independent data monitoring committee will review the data and recommend whether to continue the study with a selected dose or to unblind if the thresholds for safety or efficacy are not met. The starting point for the study design assumed a treatment difference of 1.75 points in pruritus and a standard deviation of 3. As a reminder, pruritus is assessed on a 10-point numerical rating scale. This approach allows us to accomplish three key objectives. First, we want to confirm a meaningful treatment effect. Second, we want to select the best dose. And third, by keeping ourselves blinded to the interim results, patients from the interim will be included in the potentially pivotal data set. This gets us to pivotal data faster, and at the same time, we have reassurance of a meaningful treatment effect. We will also be sharing top-line data from the interim analysis of the VANTAGE study in PBC. As a reminder, this study allows patients with both normal and elevated ALK-FOS who are on ursodiol. The interim data set included 32 patients across three arms with two active doses and placebo. The objective for the interim is to select the appropriate dose to take forward to the pivotal portion of the study. Given the historical data with iBET inhibitors in PVC, we believe this is adequately sized to select a dose and show a trend on efficacy. The interim is not designed to show statistical significance. At the interim, we expect to share top-line data on pruritus improvement, safety, and other biomarkers such as serum bile acids. Both of these are seamless, adaptive study designs, and we continue to enroll with the goal of supporting registration. Enrollment in both studies is progressing well. These studies represent an important step towards addressing the accumulation of bile acids in broader patient groups with adult cholestasis, where a significant portion of patients lack adequate treatment options for cholestasis and a severe symptomatic burden. We'll provide an update on projections for completion of enrollment for both studies when we announce the interims in June. 2024 is off to a great start, and I look forward to sharing our progress with you this year. With that, I'll now turn the call over to Eric to discuss our financial results. Eric?
spk08: Thanks, Joanne. Earlier today, we issued a press release that included financial results for the first quarter, which I'll briefly summarize. Total revenue in the first quarter of 2024 was $69.2 million compared to total revenue of $31.6 million in the first quarter last year. Total operating expenses for the quarter ended March 31st, where $95.7 million which includes R&D expenses of $32.2 million, SG&A expenses of $45.6 million, and cost of sales of $17.8 million. The total operating expense for the quarter included approximately $17.1 million of non-cash charges. For the quarter ended March 31st, net loss was $25.3 million or $0.53 per share. Our cash, cash equivalents, and investments increased to $302.8 million as of March 31, 2024, up from $286.3 million at the end of last year, primarily due to a reduction in working capital. We expect that our working capital balances will vary from quarter to quarter depending on timing of payments and inventory investments. So in summary, our business continues to be well funded and we are in an excellent position to support the advancement of our pipeline and expansion of our global commercial business. Now I'll turn the call back over to Chris for final comments.
spk03: Thanks, Eric. It's been a great start to the year and our business continues to grow. We remain on track for a full year revenue guidance. We're executing across our label expansion opportunity launches. and are very much looking forward to the velixibat interims ahead. And with that, operator, please open the call for questions.
spk09: Thank you. If you would like to ask a question, please press star followed by one on your telephone keypads now. If you would like to remove yourself from the queue, it's star followed by two. And when preparing to speak, please ensure your line is unmuted locally. Your first question on the line comes from Jessica Fye of JP Morgan. Please go ahead. Your line is open.
spk10: Great. Good afternoon. Thanks for taking my question. For the two Valixabad studies, can you remind us what background medications patients are allowed to be on that may also address pruritus and how that kind of factors into your expectations for the results, if at all?
spk03: Thanks, Jess, for the question. I'll ask Joanne to jump in and talk a little bit about the background setting.
spk00: Yeah, thanks, Chris, and thanks, Jessica, for the question. You know, we think actually that the way that we design the studies actually make it really broadly applicable for both of these populations. So, for instance, in PBC, we're allowing patients who are either on or not on ursodiol, and we're allowing patients with any level of outfall. So, a little different than some of the other trials that have been for the other agents kind of in this area. So, we really think this translates to use in first-line ultimately for PPC. For PSC, as you know, there's no other therapies. And we know that a majority of patients with both these diseases, PSC and PPC, do have pruritus. So we think this, the way we've designed the studies with a very broad inclusion criteria do allow us to translate kind of a very real world issue into our studies and therefore give us useful information.
spk10: Great. And maybe just one more if I could. Can you remind us what to expect from a tolerability standpoint for Velixibat?
spk00: Yeah. Well, thanks for the follow-up, Jessica. So, you know, we know IVAT inhibitors quite well. And I will say so far, you know, Velixibat kind of tracks in what we know. And we know exactly how to dose these. And we know exactly what to look for. So, we don't expect really any surprises in this way.
spk10: Great. Thank you.
spk09: Thanks, Jess. Thank you. Your next question comes from Manu Foruha from Lee Rink Partners. Your line is now open.
spk06: Thanks so much for taking the question, guys. A quick one. You mentioned about a $3 million impact this quarter, it sounds like, on change healthcare issue. To what extent is that sort of a one-time lost revenue as opposed to revenue that might pop up in sort of a recognition function next quarter. And separately, when you think about that $3 million, as we try and back that out and think about underlying demand metrics, how is that separated between your products this quarter and sort of the split between Live Morally versus the acquired TBTX assets?
spk03: Thanks for the question, Marty. I'll let Peter kind of dive into the detail. Short answer though, it's a one-time effect, but Peter can give a little color on the background here.
spk12: Yeah, one-time effects that, you know, it's concluded by the end of the quarter, so don't expect to see any lingering effects from this in future quarters. And, you know, the you know, overall demand for all of our products grew, total prescriptions grew over the quarter. So, you know, not really, you know, that's why we're very confident in the 310 to 320. And I think, and you also asked about the 3 million, you know, I think assigning 3 million by individual product is kind of probably false precision here. I mean, we kind of think about the 3 million as an impact across the U.S. business.
spk06: Okay, but is it reasonable to assume the great majority of it was driven by Liv Marley, given the geographics of those products?
spk12: I think it's probably balanced. You can think about it generally in proportion to the size of the products.
spk06: Okay, that's helpful. And as we think about between now and the end of the year, staying on commercial questions, obviously you maintain your guidance. Should we think about the tempo between now and reaching somewhere in 310 to 320 as fairly consistent? Is more of the growth back end weighted into like 3Q into 4Q? How should we think about that from a modeling perspective now that we're sort of deeper into the year?
spk12: Yeah, I think, I mean, the way we think about it is generally consistent. I mean, the cadence of demand is, you know, strong and we see it growing quarter to quarter. You will have PFIC coming on, although, you know, as we've commented, most of 2024, we expect many of the PFIC dispenses to be pre-drug and, you know, they're probably contributing more in 2025. That might be the one dynamic that comes into play more later in the year. But generally, I think it's a pretty consistent build towards the 310 to 320. Okay.
spk06: That's helpful. Thanks, guys. Next question.
spk09: The next question on the line comes from Gavin Clark-Gartner of Evercore ISI. Please go ahead. Your line is now open.
spk11: Hey, congrats on the progress, and thanks for taking my questions. First, on PSC, I believe you noted there was a 1.75 expected pruritus benefit and a three standard deviation that was informing your powering assumptions. But was that 1.75 absolute or placebo-adjusted? And maybe just remind us your expectations for the placebo arm for this trial.
spk00: Yeah, so, yeah, thanks for the question, Gavin. So, by 1.75, we mean the treatment difference, so active compared to placebo. And we took some fairly conservative assumptions, you know, by putting that together. And as you know, you know, those are always kind of a starting point for where you kind of put the study. But we did want to share at least our starting point for looking at the study design.
spk11: Yeah, that's helpful. And are you able to share the baseline pruritus scores for either trial for Velixibab?
spk00: You know, not at this point. We'll be happy to share information when we share the interims in June with you. So I think that would be – we'll look forward to that along with you folks.
spk11: Sounds good. Just the last one. Any updates on the potential for orphan drug status for PFIC in the EU?
spk03: Thanks for the follow-up there. This is one that we are continuing in dialogue with the European regulators and hope to have an update soon, but still come back to really strong conviction in our our data for the Live Marley program and PFIC providing some real advantages for patients. So, hoping to have an update on that one soon. Sounds good. Thanks, guys. Yeah, thanks for the questions.
spk09: The next question on the line comes from Dagon Ha from CIFL. Please go ahead. Your line is open.
spk02: Hey, good afternoon, guys. Thanks for taking my questions. Maybe two-part question. On the PFIX side of the story, I was wondering if you can comment on your dialogue with the physicians, given the label disparity between this and Bilvay for the time being. And when you think about the reimbursement dialogue, will there need to be subsequent dialogues to be had once you get the label expansion done? And switching over to Velixabad, bearing in mind the interim update in June for both Vista's and Vantage, How are you guys thinking about sort of the Glaxo drug towards the back end of this year, and how might that impact your PBC strategy if both come out positive? Thanks so much.
spk03: Yeah, thanks for the questions. Maybe I'll just make a quick comment on the Valixabad competition briefly and then pass over to Peter to talk about PFIC. And, you know, what we're seeing and kind of how we've approached the dosing for Velixibat I think provides the potential for a real advantage in terms of activity level. It's something we've learned across all of the IBAT programs, in particular all the work we've done with Liv Marley and Velixibat on understanding where we're at on the dose response curve. I think it provides the potential to have really strong activity here. Of course, this is something we'll see play out with the actual datasets as they come forward. but excited about the dosing regimens that we're evaluating in the Vantage study and what that can mean for patients. Maybe Peter can speak to the PFIC questions. Sure.
spk12: Yeah, thanks for the questions. Feedback on the Marley profile and PFAC has been very positive. I think we've, you know, a lot of favorable, you know, feedback on the FC profile that was observed in the MARCH study as reflected in the label, as well as the broader Genetic types of that are included in the labeling, which can sometimes make a difference in market access, depending on the on the payers policies. So, really, really favorable feedback from clinicians and patients happy to have live Marley available for those patients. And, yeah, pair conversations so far going well, it's early days still, but have had really happy with the policies that have emerged. And then in terms of updated policies after, as you mentioned, a potential label update for younger than five years of age, I'd expect that those, I mean, there's a lot of payers in the U.S., so it's heterogeneous, but generally those would occur pretty quickly. We saw that with AllerGO when the initial label was one year of age and older and then lowered the age. Those subsequent follow-up conversations generally occur pretty quickly to update policies.
spk02: Great. Thanks for taking our questions.
spk14: Yep, thanks for the questions.
spk09: The next question on the line comes from Steven Seedhouse of Raymond James. Please go ahead.
spk05: Hey, good afternoon. Thanks for taking the questions. Two separate questions. I'll just ask them both now because they're pretty straightforward. First, on the PBC readout, you mentioned pritis improvement, safety, and serum bile acids would be the focus of that data release. And I'm just curious if you'll also be sharing liver function tests, ALKFOS, ALT, AST, bilirubin, just to get a sense of, even from a safety standpoint, what's happening there, if there's any impact of, you know, de novo bile acid synthesis on any of those parameters. And then the second one is just on business development. We'd be curious your comments or thoughts on just the overall view of that or priorities for Merum you know, over the next call at 12 to 18 months, are you thinking about expanding the pipeline or focusing on Biloxibat primarily? Thank you.
spk03: Yeah, thanks, Steve, for the question. I'll speak to and comment on kind of our business development efforts and let Joanne come back on the PPC interims. And for, you know, our strategy and approach to business development, you know, remains consistent with what we've done over the course of Miriam. It's very much in our DNA. It's how the company came to be and started. and its approach of being disciplined about making sure anything that we look to bring on is something that we can add value to that's at terms and something that helps grow the company and really across a number of different rare disease settings is where we're looking. I think we're in a position where we're quite lucky in that there's a lot of growth in the commercial business label expansion opportunities, the velixibat developments, that there's no urgency to do something so we can remain disciplined in looking at ways to grow the company. Maybe Joanne can speak to the PBC question.
spk00: Yeah, thanks, Chris, and thanks for the question. You know, as I mentioned, this is an interim analysis, so it's pretty limited in terms of scope. We're mainly looking at it to ensure safety and to select a dose moving forward. So, you know, with that, we'll look at pruritus, we'll look at, you know, serum bile acids and safety in particular. You know, the data sets can be pretty limited, so we think it'll be quite limited in terms of making any conclusions, certainly, about any other parameters. I think we'd look to the final data set for that.
spk05: Thanks so much.
spk15: Thanks for the questions.
spk09: The next question on the line comes from Brian Scorny of Baird. Please go ahead. Your line is open.
spk07: Hi, this is Luke on for Brian. As we set expectations for Vantage, in particular thinking about a comp to the Cella-Delpar response study, do you think the subgroup in that study with baseline NRS greater than four is a reasonable comp for pruritus benefits? And then are you aware of the 11-point NRS scale they used in that study is the same as the itch-reported outcome scale that you're using?
spk03: Thanks, Luke, for the questions. Yeah, I mean, the scale used, it is similar. I mean, there's some very minor differences, but for adult pruritus measurements and registrational studies, this is all in line with FDA guidance. They use a 0 to 10 scale. That's what we're using in our study. So, there is some, definitely some similarity there and the treatment effect in that subset that they looked at, you know, it's not too far off with how we looked at our kind of. Change from placebo assumptions and empowering right where we looked at the 1.75 difference from placebo. So, I'm looking at potentially a little bit more effect from. from an IVAT, but, you know, in general, it's really not too far off if you're thinking about study design assumptions. Of course, we're quite excited about getting this data and seeing what that looks like, particular change from baseline, which is really what the patient experiences and what you're doing to address the burden of disease.
spk07: Great. Thank you.
spk03: Thanks for the question.
spk09: The next question on the line comes from David Lebowitz of Citi. Please go ahead. Your line is open.
spk01: Thank you very much for taking my question. On the 1.75 point difference on pruritus, could you just elaborate as to whether you're talking about through the blinded portion or through the actual pivotal portion at a subsequent time point? And perhaps give us some view of of what that point and how you will use it to consider upsizing if that is needed, what type of thresholds we could expect.
spk00: Yeah, well, thanks for the question. You know, we're not going to get into the details of the study design, but I just provided some of the, you know, numbers so that you could get a sense of what kind of treatment effect we're looking at. The 1.75 treatment difference and the three in terms of standard deviation, it's just a general number that we're looking for the overall design of the study. So we won't be sharing any specifics in terms of, you know, kind of where we are with the interim. Obviously, we'll share the results of the interim since we'll be blinded. We certainly hope that we'd be continuing the study. So that's what we hope to see in June.
spk03: And one add to that, David, is that the The measurement is actually looking at over time, right? It's the months three, four, five, or sorry, four, five, and six actually of the treatment effect for the final analysis. That does a lot to add power and try to deal with any potential for placebo response because you're looking at multiple time points and how they roll into the analysis. That's another kind of factor to the study design, applying what we used from the PFIC study and the adult settings.
spk01: Got it. Thank you very much for taking my question.
spk03: Yeah, thanks for the question.
spk09: The next question on the line comes from John Walden of Citizens JMP. Please go ahead. Your line is open.
spk04: Hey, thanks for taking the question. Just a logistic one from me on PBC and PSE. When you select a dose, do the patients on the prior dose get to roll back in at either placebo or the new dose, or do you have to re-enroll patients? Can you comment on how long enrollment could take to complete in both those studies? Thanks.
spk03: Thanks, John, for the question. We'll give a more formal update on what we expect to see for the, to get to the full data set in June when we have the interim. But I'll let Joanne speak to a little bit of the mechanics of how patients flow through the study.
spk00: Yeah. So we're going to be continuing, you know, we are continuing enrollment in the study at this point. And then, you know, we'll be continuing with one active dose and placebo. So we expect to, you know, include all the patients ultimately in the analysis. when ultimately unblind the whole data set.
spk01: Thanks for the question, John.
spk09: Next question on the line comes from Morgan Stanley. Please go ahead. Your line is open.
spk14: Hi, this is Rohit on for Mike. Thanks for taking our questions. Can you just provide any color on the ongoing launch prep for PFIC? And when do you expect patients on the expanded access program to get on paid drugs? Thanks.
spk12: Yeah, thanks for the question, Rohit. In the U.S., obviously, launch is underway. You know, seeing, you know, prescriptions come in for live mortality for PFIC patients now. You know, we have talked about it. We have, you know, about 25 patients in the U.S. who are on clinical drug. Most of those are eligible to roll over at this time, and we'd expect them to come over to commercial drug in the coming quarters throughout this year. So that's how we see the cadence playing forward. Thank you.
spk09: The next question on the line comes from Ed Arce from H.C. Wainwright. Please go ahead. Your line is open.
spk15: Hi, good afternoon, everyone. This is Thomas Yip asking a couple of questions for Ed. Thank you for the kind of questions. So first, following up on the U.S. performance for the Somali PFEG, just wonder of the 42.8 million net sales in first quarter. How much was it from PFIC approximately? And also, what are some early launch metrics that investors can look to?
spk03: Thanks, Thomas, for the question. In Q1, there really, there's no PFIC contribution in there yet. And we're just, the approval came in March and just now kind of rolling over those clinical patients. So expect that revenue contribution to be pretty minimal from PFIC over the next quarter or two as we get into the back half of the year where we expect more full reimbursement.
spk15: Got it. And then switching gears to the European front, have you had any interaction with either EMA or CHMP recently given your expectation on a recommendation in the first half of this year, and if positive, any ongoing commercial preparations for European market and for PFIC?
spk03: Thanks for the follow-up there. On the EMA discussion, we have been active in discussing with EMA. As mentioned, feel confident in our arguments and hope to have an update on that soon, so no formal determination yet. And maybe Peter can speak a little bit to the launch prep in Europe for PHIC.
spk12: Yeah, certainly upon a potential approval, we would be ready to launch Live Marley and PHIC in Europe. Prescribers for, you know, PHIC are essentially identical to the prescribers of Live Marley for Alishiel syndrome. So, you know, we'll be ready to go with dossier submissions to health technology agencies, et cetera, towards pricing and reimbursement at the level as well.
spk15: Okay, and one last question from us. Can you discuss some of the main drivers for your product sales slightly by 2 million quarter to quarter? Can you highlight some major factors?
spk12: Yeah, I mean, you know, we mentioned the, you know, the change healthcare cyber attack was kind of in play for our entire portfolio. I think if you look back over time, you know, in the bile acid product sales, there's quarter-to-quarter volatility given the ultra-rare nature of the disease in a small number of patients over time. But do see, you know, an opportunity to continue to build on those products. This year, you know, mid-single-digit year-on-year growth consistent with what historically is our expectation. Going forward, really excited about potential approval by FDA next year for Kinadol and CTX and the chance to go out and find more patients there.
spk15: Understood. Thank you again for the kind of questions. We look forward to the C-HMP recommendations soon and also your June presentation for Lexibet. Sounds good. Thanks, Thomas.
spk09: We have no further questions. Therefore, I will hand back the call to Chris Peet, CEO, for final remarks.
spk03: Great. Thank you all for joining us today. Really appreciate the interest in Miriam and our programs. Have a good evening. Bye-bye.
spk09: This concludes today's conference call. Thank you all for joining. You may now disconnect your lines.
Disclaimer