NASDAQ:MIRM Mirum Pharmaceuticals Q1 2024 Earnings Report $43.69 +0.27 (+0.62%) Closing price 05/2/2025 04:00 PM EasternExtended Trading$43.68 -0.02 (-0.03%) As of 05/2/2025 04:05 PM Eastern Extended trading is trading that happens on electronic markets outside of regular trading hours. This is a fair market value extended hours price provided by Polygon.io. Learn more. Earnings HistoryForecast Mirum Pharmaceuticals EPS ResultsActual EPS-$0.54Consensus EPS -$0.40Beat/MissMissed by -$0.14One Year Ago EPSN/AMirum Pharmaceuticals Revenue ResultsActual Revenue$69.22 millionExpected Revenue$69.71 millionBeat/MissMissed by -$490.00 thousandYoY Revenue GrowthN/AMirum Pharmaceuticals Announcement DetailsQuarterQ1 2024Date5/8/2024TimeN/AConference Call DateWednesday, May 8, 2024Conference Call Time4:30PM ETUpcoming EarningsMirum Pharmaceuticals' Q1 2025 earnings is scheduled for Tuesday, May 6, 2025, with a conference call scheduled on Wednesday, May 7, 2025 at 4:30 PM ET. Check back for transcripts, audio, and key financial metrics as they become available.Conference Call ResourcesConference Call AudioConference Call TranscriptPress Release (8-K)Quarterly Report (10-Q)Earnings HistoryCompany ProfilePowered by Mirum Pharmaceuticals Q1 2024 Earnings Call TranscriptProvided by QuartrMay 8, 2024 ShareLink copied to clipboard.There are 13 speakers on the call. Operator00:00:01Hello, and welcome to the Mirum Pharmaceuticals First Quarter 20 24 Financial Results and Business Update. My name is Terry, and I'll be the conference operator today. I will now hand the call over to Andrew McKibben, Vice President of Investor Relations to begin. Please go ahead. Speaker 100:00:30Thanks, Terry, and good afternoon, everyone. I'd like to welcome you to Mirum Pharmaceuticals' Q1 2024 Conference Call. I'm joined today by our CEO, Chris Peetz our President and Chief Operating Officer, Peter Radovich our Chief Medical Officer, Joanne Kwan and Eric Bjerkholt, our Chief Financial Officer. Earlier today, Merrim 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 Investors section of our website. Speaker 100:00:59Before 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 Miriam and our programs based on management's current expectations, including statements regarding Miriam's current and future business plans, development programs and regulatory expectations, strategies, prospects, market opportunities and financial expectations. Merum 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 10 Q for the quarter ended March 31, 2024, and any subsequent reports filed with the SEC. With that said, I'd like to turn the call over to Chris. Chris? Speaker 200:01:41Thanks, 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 valexibat 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 $68,900,000 representing a 137% increase from the Q1 of 2023. Speaker 200:02:22LIV Marley 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 $310,000,000 to $320,000,000 driven by continued demand increases across all medicines, international launches and contribution from the PFIC approval. 2nd, we are also making significant progress towards expanding the impact of our potentially life changing medicines through new approvals and label expansions. Speaker 200:02:56In March, we announced the U. S. Approval of LIVMARELY and PFIC, an important milestone for Miram 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 LID Marley to this community, particularly those patients with rare genetic types of PFIC. Speaker 200:03:17The pivotal data was also just published this week in The Lancet highlighting the improvements in itch, bile acids, bilirubin and growth seen with the LIV Marley 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 KEYNOTE for the treatment of CTX. And third, we are advancing volixibat in PSC and PBC, which are more common adult cholestatic setting, where we can apply the scientific and regulatory insights from the Lymph Marley 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 voxibat in the VISTA's PSC and VANTAGE PBC studies, which are scheduled in June. Speaker 200:04:05We see both indications as significant opportunities as there are no approved therapies PSC and no approved therapies to treat cholestatic pruritus and 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. Peter? Thanks, Chris. Speaker 300:04:34Our commercial teams delivered another strong quarter with continued demand growth for LibMarley 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 $310,000,000 to $320,000,000 For Lid Marley, total global net product sales grew to $42,800,000 in the Q1, up from $41,400,000 in the Q4 of 2023 $29,100,000 a year ago. U. S. Lidmarley sales were $30,800,000 and international Lidmarley sales were 12,100,000 dollars Our U. Speaker 300:05:16S. Alagille business continues to benefit from durable demand expansion in total Lid Marley 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. Speaker 300:05:40Business 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 $3,000,000 impact on Q1 sales across our products. Turning to the recent approval of Lidmarly for CholeSat pruritus in 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 pay dispenses to materialize over the next few quarters. Speaker 300:06:22Turning to coal bomb and Kenadol, we recognized net product sales of $26,100,000 in the Q1 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 $310,000,000 to 320,000,000 dollars 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? Speaker 400:06:48Thanks, 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 vaxxavat, 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 surpassed site and efficacy threshold for continuation, which is based on prior experience with Ibad inhibitors in cholestatic arthritis. Speaker 400:07:18Using 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 3 key objectives. First, we want to confirm a meaningful treatment effect. Speaker 400:07:502nd, 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 outclass who are on Ursodiol. Speaker 400:08:19The interim data set includes 32 patients across 3 arms with 2 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 Ibad inhibitors and TBC, we believe this is adequately sized to select the 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. Speaker 400:08:53Both 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. Speaker 400:09:27With that, I'll now turn the call over to Eric to discuss our financial results. Eric? Speaker 500:09:33Thanks, Joanne. Earlier today, we issued a press release that included financial results for the Q1, which I'll briefly summarize. Total revenue in the Q1 2024 was $69,200,000 compared to total revenue of $31,600,000 in the Q1 last year. Total operating expenses for the quarter ended March 31 were $95,700,000 which includes R and D expenses of $32,200,000 SG and A expenses of 45,600,000 dollars and cost of sales of 17,800,000 approximately $17,100,000 of non cash charges. For the quarter ended March 31, net loss was $25,300,000 or $0.53 per share. Speaker 500:10:28Our cash, cash equivalents and investments increased to $302,800,000 as of March 31, 2024, up from $286,300,000 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. Speaker 200:11:12Thanks, 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 are executing across our label expansion opportunity, launches and are very much looking forward to the filixibat interims ahead. And with that, operator, please open the call for questions. Operator00:11:33Thank Your first question on the line comes from Jessica Fye of JPMorgan. Please go ahead. Your line is open. Great. Good afternoon. Operator00:11:56Thanks for taking my question. For the 2 valexovad 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? Speaker 200:12:18Thanks, Jess, for the question. I'll ask Joanne to jump in and talk a little bit about the background setting. Speaker 400:12:23Yes. Thanks, Chris, and thanks, Jess, for the question. 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 TBC, we're allowing patients who are either on or not on Ursodiol and we're allowing patients with varying with any level of VAP FOS. So, a little different than some of the other trials that have been for the other agents kind of in this area. Speaker 400:12:48So, we really think this translates to use in first line ultimately for PBC. For PSC, as you know, there's no other therapies. And we know that a majority of patients with both these diseases, PSC and PBC, 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. Great. Operator00:13:19And maybe just one more if I could. Can you remind us what to expect from a tolerability standpoint for voxelabat? Speaker 400:13:30Yes. Well, thanks for the follow-up, Jessica. So we know IVAD inhibitors quite well. And I will say so far, you know, belixibat kind of tracks in what we know. And we know exactly how to dose these and we know exactly what to look for. Speaker 400:13:45So, we don't expect really any surprises in this way. Operator00:13:52Great. Thank you. Speaker 600:13:55Thanks, Jess. Operator00:13:56Thank you. Your next question comes from Manu Foroohar from Leerink Partners. Your line is now open. Speaker 700:14:05Thanks so much for taking the question guys. A quick one. You mentioned about a $3,000,000 impact this quarter, it sounds like, some changed health care issue. To what extent is that sort of a one time loss revenue as opposed to revenue by pop up in sort of a recognition function next quarter? And separately, when you think about that $3,000,000 dollars as we try and back that out and think about kind of underlying demand metrics, how is that separated between your products this quarter and sort of the split between the Marley versus the acquired TPx assets? Speaker 200:14:41Thanks for the question, Mani. I'll let Peter kind of dive into the details. I mean, short answer to those, it's a one time effect, but Peter can give a little color on the background here. Speaker 300:14:51Yes. One time effects that has concluded by the end of the quarter. So don't expect to see any lingering effects from this in future quarters. And the overall demand for all of our products grew, total prescriptions grew over the quarter. So not really that's why we're very confident in the $310,000,000 to $320,000,000 And I think and you also asked about the $3,000,000 I think assigning $3,000,000 by individual product kind of probably false precision here. Speaker 300:15:26I mean, we kind of think about $3,000,000 as an impact across the U. S. Business. Speaker 700:15:35Okay. But is it reasonable to assume that the great majority of it was driven by Liv Marley given the geographics of those products? Speaker 300:15:45I think it's probably balanced. You could think about it generally in proportion to the size of the products. Speaker 700:15:55Okay, that's helpful. And as we think about between now and the end of the year, staying on commercial questions, obviously, you maintained your guidance. Should we think about the tempo between now and reaching somewhere in 3.20 to 3.20 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? Speaker 300:16:23Yes, I think I mean, the way we think about it is generally consistent. I mean, the cadence of demand is strong and we see it growing quarter to quarter. You will have PFIC coming on, although as we've commented, most of 2024, we expect many of the PFIC dispensers to be pre drug and that 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. Speaker 700:16:52Okay. That's helpful. Thanks guys. Speaker 600:16:56Thanks for the question. Operator00:16:59The next question on the line comes from Gavin Clark Gartner of Evercore ISI. Please go ahead. Your line is now open. Speaker 800:17:08Hey, 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 3 standard deviation that was informing your powering assumptions. But was that 1.75 absolute or placebo adjusted? Maybe just remind us your expectations for the placebo arm for this trial? Speaker 400:17:33Yes. So, yes, 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 by putting that together. And as you know, those are always kind of a starting point for where you kind of put the study. Speaker 400:17:54But we did want to share at least our starting point for looking at the study design. Speaker 800:18:02Yes. That's helpful. And are you able to share the baseline pruritus scores for either trial for valexibat? Speaker 400:18:11Not at this point. When we 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. Speaker 800:18:25That sounds good. Just a last one. Any updates on the potential for orphan drug status for PFIC in the EU? Speaker 200:18:35Thanks 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 data for the Litt Marley program in PFIC, providing some real advantages for patients. So hoping to have an update on that one soon. Speaker 300:19:05Sounds good. Thanks guys. Speaker 600:19:08Yes. Thanks for the questions. Operator00:19:12The next question on the line comes from Day Gunhav from Stifel. Please go ahead. Your line is open. Speaker 900:19:19Hey, good afternoon guys. Thanks for taking my questions. Maybe 2 part question. On the P6 side of the story, I was wondering if you can comment on your dialogue with the physicians given the label disparity between this and Bilvey 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? Speaker 900:19:41And switching over to valexibat, bearing in mind the interim update in June for both Vestas and Vantage, how are you guys thinking about sort of the Galaxo drug towards the back end of this year? And how might that impact your PBC strategy if both come out positive? Thanks so much. Speaker 200:20:01Yes. Thanks for the questions. Maybe I'll just make a quick comment on valexovad competition briefly and then pass over to Peter to talk about PFIC. And what we're seeing and kind of how we've approached the dosing for valexovat, I think provides potential for a real advantage in terms of activity level. And so that we've learned across all of the IBAT programs, in particular, all the work we've done with Lyth Marley and voxelabat on understanding where we're at on the dose response curve, I think provides the potential to have really strong activity here. Speaker 200:20:35Of course, this is something we'll see play out with the actual data sets 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. Speaker 300:20:52Sure. Yes, thanks for the questions. Feedback on the Marley profile and PFAC has been very positive. I think we've a lot of favorable feedback on the efficacy profile that was observed in the March study is reflected in the label as well as the broader genetic types of PFIC that are included in the labeling, which can sometimes make a difference in market access depending on the payers' policies. So really, really favorable feedback from clinicians and patients, happy to have Lyub Marley available for those patients. Speaker 300:21:24And yes, payer conversations so far are going well. It's kind of 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 5 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 Alagille when the initial label was 1 year of age and older and then lower the age. Speaker 300:21:51Those subsequent follow-up conversations generally occur pretty quickly to update policies. Speaker 900:21:57Great. Thanks for taking our questions. Speaker 600:22:01Yes. Thanks for the questions. Operator00:22:05The next question on the line comes from Steven Seedhouse of Raymond James. Please go ahead. Speaker 1000:22:12Hey, good afternoon. Thanks for taking the questions. 2 separate questions. I'll just ask them both now because they're pretty straightforward. First on the PBC readout, you mentioned price improvement safety and serum bile acids will be the focus of that data release. Speaker 1000:22:28And I'm just curious if you'll also be sharing liver function tests, ALT plus ALT, KST, bilirubin, just to Speaker 1100:22:36get a sense of even from Speaker 1000:22:38a safety standpoint, what's happening there, if there's any impact of de novo bile acid synthesis on any of those parameters? And then the second one is just on business development. Would be curious your comments or thoughts on just the overall view of that or priorities for Miriam over the next call it 12 to 18 months? Are you thinking about expanding the pipeline or focusing on blixibat primarily? Thank you. Speaker 200:23:09Thanks, Steve, for the question. I'll speak to and comment on kind of our business development efforts and let Joanne come back on the PBC interims. And for our strategy and approach to business development remains consistent with what we've done over the course of MiRIM. It's very much in our DNA. It's how the company came to be and started. Speaker 200:23:30And 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 valexovat developments that there's no urgency to do something. So we can remain disciplined in looking at ways to grow the company. And then maybe Joanne can speak to the PBC question. Speaker 400:24:14Yes. Thanks, Chris, and thanks for the question. 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 the dose moving forward. Speaker 400:24:26So with that, we'll look at pruritus, we'll look at serum bile acids and safety in particular. 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. Speaker 700:24:45Thanks a lot. Speaker 600:24:48Thanks for the questions. Operator00:24:52The next question on the line comes from Brian Skorney of Baird. Please go ahead. Your line is open. Speaker 300:24:59Hi. This is Luke on for Brian. As we set expectations for Vantage, in particular thinking about a comp to the seladelpar response study, Do you think the subgroup in that study with baseline NRS greater than 4 is a reasonable comp for pruritus benefit? And then, are you aware of the 11 point NRS scale that you used in that study is the same as the itch reported outcome scale that you're using? Speaker 200:25:27Thanks Luke for the questions. Yes, I mean the scale used, it is similar. I mean there's some very minor differences, but for adults, pruritus measurements and registrational studies that's kind of this is all in line with FDA guidance. That uses a 10 scale. That's what we're using in our study. Speaker 200:25:46So there is some definitely some similarity there. And the treatment effect in that subset that they looked at, it's not too far off with how we looked at our kind of change from placebo assumptions and powering, right, where we looked at the 1.75 difference from placebo. So I'm looking at potentially a little bit more effect from an IVAT. But in general, it's really not too far off if you're thinking about steady 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. Speaker 1100:26:33Great. Thank you. Speaker 600:26:36Thanks for the question. Operator00:26:39The next question on the line comes from David Lebowitz of Citi. Please go ahead. Your line is open. Speaker 1200:26:46Thank you very much for taking my question. On the 1.75 point difference on psoriasis, 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 what that point and how you will use it to consider upsizing if that is needed, what type of thresholds we could expect? Speaker 400:27:20Yes. Well, thanks for the question. We're not going to get into the details of the study design, but I just provided some of the 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 3 in terms of standard deviation is 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 kind of where we are with the interim. Speaker 400:27:49Obviously, we'll share the results of the interim and since we'll be blinded, we certainly hope that we'd be continuing the study. So that's what we hope to be done with you in June. Speaker 200:27:57And one add to that, David, is that the measurement is actually looking at over time, right? It's the months 3, 4, 5 sorry, 4, 5, and 6, 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. So that's another kind of factor to the study design applying what we used from the PFIC study in the adult settings. Speaker 1200:28:29Got it. Thank you very much for taking my question. Speaker 600:28:33Yes. Thanks for the question. Operator00:28:37The next question on the line comes from John Walden of Citizens JMP. Please go ahead. Your line is open. Speaker 300:28:45Hey, thanks for taking the question. Just logistic one from me on PBC and PSC. When you select a dose, 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. Speaker 200:29:11Thanks, John, for the question. Just on timelines, 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. Speaker 400:29:28Yes. So we're going to be continuing we are continuing enrollment in the study at this point. And then we'll be continuing with one active dose and placebo. So we expect to include all the patients ultimately in the analysis when we can ultimately unblind the whole data set. Speaker 600:29:56Thanks for the question, John. Operator00:29:59Next question on the line comes from Mike Bowles of Morgan Stanley. Please go ahead. Your line is open. Speaker 200:30:07Hi. 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 drug? Speaker 200:30:18Thanks. Speaker 300:30:21Yes. Thanks for the question, Rohit. In U. S, obviously, launch is underway, seeing prescriptions come in for livmarly and copepit patients now. We have talked about we have about 25 patients in the U. Speaker 300:30:37S. 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 going forward. Speaker 700:30:55Thank you. Operator00:30:59The next question on the line comes from Ed Arce from H. C. Wainwright. Please go ahead. Your line is open. Speaker 1100:31:08Hi, good afternoon, everyone. This is Thomas Yip asking a couple of questions for Ed. Thank you for taking my questions. So first, following up on U. S. Speaker 1100:31:17Performance for LUXALLE PFAG. Just wonder of the $42,800,000 net sales in the Q1, how much was it from PFIC approximately? And also what are some early launch metrics that investors can update? Speaker 200:31:38Thanks Thomas for the question. In Q1, there really there is no PFIC 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 2 as we get into the back half of the year where we expect more full reimbursement. Speaker 1100:32:03Got it. And then switching gears to the European front, have you any interaction with either EMA or CHMP recently given your expectation on recommendation in the first half this year? And if positive, any ongoing commercial preparations for European market for PFIC? Speaker 200:32:31Thanks for the follow-up there. On the EMA discussion, over the 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 PFIC. Speaker 300:32:52Yes. Certainly, upon a potential approval, we would be ready to launch Lid Marley and P. Baker in Europe. Prescribers for P. Baker essentially identical to the prescribers of Lid Marley for Algill syndrome. Speaker 300:33:07So we'll be ready to go with dossier submissions to health technology agencies etcetera to work with pricing and reimbursement at that time and attribute level as well. Speaker 1100:33:20Okay. And that's one last question from us. Can you discuss some of the main drivers for the biologics of product sales that's slightly $5,000,000 quarter to quarter? Can you provide some major factors? Speaker 300:33:41Yes. I mean, we mentioned the change healthcare cyber attack was kind of in play for our entire portfolio. I think if you look back over time, in the bile acid product sales, there is quarter to quarter volatility, given the ultra rare nature of the disease in small number of patients over time. But do see an opportunity to continue to build on those products this year, mid single digit year on year growth consistent with what historically is our expectation. And going forward, really excited about potential approval by FDA next year for Kenadol and CTX and the chance to go out and find more patients there. Speaker 1100:34:28Understood. Thank you again for the kind of questions. We look forward to the CXMP recommendation soon and also your June presentation for elixir. Sounds good. Thanks, Thomas. Operator00:34:43We have no further questions. Therefore, I will hand back the call to Chris Peete, CEO, for final remarks. Speaker 200:34:51Great. Thank you all for joining us today. Really appreciate the interest in Miriam and our programs. Have a good evening. Bye bye. Operator00:35:02This concludes today's conference call. Thank you all for joining. You may now disconnect your lines.Read morePowered by Conference Call Audio Live Call not available Earnings Conference CallMirum Pharmaceuticals Q1 202400:00 / 00:00Speed:1x1.25x1.5x2x Earnings DocumentsPress Release(8-K)Quarterly report(10-Q) Mirum Pharmaceuticals Earnings HeadlinesMirum Pharmaceuticals, Inc. (NASDAQ:MIRM) Receives Consensus Rating of "Buy" from BrokeragesApril 24, 2025 | americanbankingnews.comMirum Pharmaceuticals receives FDA approval for Livmarli tablet formulationApril 15, 2025 | markets.businessinsider.comShocking AI play that’s beats Nvidia by a country mileYou’ve seen the headlines about Nvidia. Now Tim Sykes is sounding the alarm — because what CEO Jensen Huang is about to announce could change the AI market once again. Experts already predict the total addressable market could climb past $20 trillion. But Sykes believes most investors have missed what’s coming next. He’s tracking a new shift — and says the biggest gains are still ahead.May 4, 2025 | Timothy Sykes (Ad)FDA Approves Mirum Pharmaceuticals’ Drug For Rare Liver Disease Patients In Tablet Form: Retail Stays BullishApril 14, 2025 | msn.comMirum’s LIVMARLI Now FDA Approved in Tablet FormulationApril 14, 2025 | finance.yahoo.comMirum Pharmaceuticals Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)April 10, 2025 | businesswire.comSee More Mirum Pharmaceuticals Headlines Get Earnings Announcements in your inboxWant to stay updated on the latest earnings announcements and upcoming reports for companies like Mirum Pharmaceuticals? Sign up for Earnings360's daily newsletter to receive timely earnings updates on Mirum Pharmaceuticals and other key companies, straight to your email. Email Address About Mirum PharmaceuticalsMirum Pharmaceuticals (NASDAQ:MIRM), a biopharmaceutical company, focuses on the development and commercialization of novel therapies for debilitating rare and orphan diseases. Its lead product candidate is LIVMARLI (maralixibat), an orally administered and minimally absorbed ileal bile acid transporter (IBAT) inhibitor that is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the United States and internationally. The company is also involved in the commercialization of Cholbam, a cholic acid capsule, which is approved as treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects and for adjunctive treatment of patients with peroxisomal disorders, including peroxisome biogenesis disorder-Zellweger spectrum disorder and Smith-Lemli-Opitz syndrome; and Chenodal, a tablet, which is approved for the treatment of radiolucent stones in the gallbladder, and under Phase 3 development for the treatment cerebrotendinous xanthomatosis. In addition, it develops Volixibat, an oral and minimally absorbed agent designed to inhibit IBAT, currently under Phase 2b clinical trial for the treatment of adult patients with cholestatic liver diseases. The company was incorporated in 2018 and is headquartered in Foster City, California.View Mirum Pharmaceuticals ProfileRead more More Earnings Resources from MarketBeat Earnings Tools Today's Earnings Tomorrow's Earnings Next Week's Earnings Upcoming Earnings Calls Earnings Newsletter Earnings Call Transcripts Earnings Beats & Misses Corporate Guidance Earnings Screener Earnings By Country U.S. Earnings Reports Canadian Earnings Reports U.K. Earnings Reports Latest Articles Amazon Earnings: 2 Reasons to Love It, 1 Reason to Be CautiousMeta Takes A Bow With Q1 Earnings - Watch For Tariff Impact in Q2Palantir Earnings: 1 Bullish Signal and 1 Area of ConcernVisa Q2 Earnings Top Forecasts, Adds $30B Buyback PlanMicrosoft Crushes Earnings, What’s Next for MSFT Stock?Qualcomm's Earnings: 2 Reasons to Buy, 1 to Stay AwayAMD Stock Signals Strong Buy Ahead of Earnings Upcoming Earnings Palantir Technologies (5/5/2025)Vertex Pharmaceuticals (5/5/2025)Realty Income (5/5/2025)Williams Companies (5/5/2025)CRH (5/5/2025)Advanced Micro Devices (5/6/2025)American Electric Power (5/6/2025)Constellation Energy (5/6/2025)Marriott International (5/6/2025)Energy Transfer (5/6/2025) Get 30 Days of MarketBeat All Access for Free Sign up for MarketBeat All Access to gain access to MarketBeat's full suite of research tools. 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There are 13 speakers on the call. Operator00:00:01Hello, and welcome to the Mirum Pharmaceuticals First Quarter 20 24 Financial Results and Business Update. My name is Terry, and I'll be the conference operator today. I will now hand the call over to Andrew McKibben, Vice President of Investor Relations to begin. Please go ahead. Speaker 100:00:30Thanks, Terry, and good afternoon, everyone. I'd like to welcome you to Mirum Pharmaceuticals' Q1 2024 Conference Call. I'm joined today by our CEO, Chris Peetz our President and Chief Operating Officer, Peter Radovich our Chief Medical Officer, Joanne Kwan and Eric Bjerkholt, our Chief Financial Officer. Earlier today, Merrim 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 Investors section of our website. Speaker 100:00:59Before 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 Miriam and our programs based on management's current expectations, including statements regarding Miriam's current and future business plans, development programs and regulatory expectations, strategies, prospects, market opportunities and financial expectations. Merum 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 10 Q for the quarter ended March 31, 2024, and any subsequent reports filed with the SEC. With that said, I'd like to turn the call over to Chris. Chris? Speaker 200:01:41Thanks, 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 valexibat 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 $68,900,000 representing a 137% increase from the Q1 of 2023. Speaker 200:02:22LIV Marley 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 $310,000,000 to $320,000,000 driven by continued demand increases across all medicines, international launches and contribution from the PFIC approval. 2nd, we are also making significant progress towards expanding the impact of our potentially life changing medicines through new approvals and label expansions. Speaker 200:02:56In March, we announced the U. S. Approval of LIVMARELY and PFIC, an important milestone for Miram 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 LID Marley to this community, particularly those patients with rare genetic types of PFIC. Speaker 200:03:17The pivotal data was also just published this week in The Lancet highlighting the improvements in itch, bile acids, bilirubin and growth seen with the LIV Marley 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 KEYNOTE for the treatment of CTX. And third, we are advancing volixibat in PSC and PBC, which are more common adult cholestatic setting, where we can apply the scientific and regulatory insights from the Lymph Marley 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 voxibat in the VISTA's PSC and VANTAGE PBC studies, which are scheduled in June. Speaker 200:04:05We see both indications as significant opportunities as there are no approved therapies PSC and no approved therapies to treat cholestatic pruritus and 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. Peter? Thanks, Chris. Speaker 300:04:34Our commercial teams delivered another strong quarter with continued demand growth for LibMarley 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 $310,000,000 to $320,000,000 For Lid Marley, total global net product sales grew to $42,800,000 in the Q1, up from $41,400,000 in the Q4 of 2023 $29,100,000 a year ago. U. S. Lidmarley sales were $30,800,000 and international Lidmarley sales were 12,100,000 dollars Our U. Speaker 300:05:16S. Alagille business continues to benefit from durable demand expansion in total Lid Marley 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. Speaker 300:05:40Business 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 $3,000,000 impact on Q1 sales across our products. Turning to the recent approval of Lidmarly for CholeSat pruritus in 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 pay dispenses to materialize over the next few quarters. Speaker 300:06:22Turning to coal bomb and Kenadol, we recognized net product sales of $26,100,000 in the Q1 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 $310,000,000 to 320,000,000 dollars 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? Speaker 400:06:48Thanks, 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 vaxxavat, 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 surpassed site and efficacy threshold for continuation, which is based on prior experience with Ibad inhibitors in cholestatic arthritis. Speaker 400:07:18Using 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 3 key objectives. First, we want to confirm a meaningful treatment effect. Speaker 400:07:502nd, 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 outclass who are on Ursodiol. Speaker 400:08:19The interim data set includes 32 patients across 3 arms with 2 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 Ibad inhibitors and TBC, we believe this is adequately sized to select the 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. Speaker 400:08:53Both 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. Speaker 400:09:27With that, I'll now turn the call over to Eric to discuss our financial results. Eric? Speaker 500:09:33Thanks, Joanne. Earlier today, we issued a press release that included financial results for the Q1, which I'll briefly summarize. Total revenue in the Q1 2024 was $69,200,000 compared to total revenue of $31,600,000 in the Q1 last year. Total operating expenses for the quarter ended March 31 were $95,700,000 which includes R and D expenses of $32,200,000 SG and A expenses of 45,600,000 dollars and cost of sales of 17,800,000 approximately $17,100,000 of non cash charges. For the quarter ended March 31, net loss was $25,300,000 or $0.53 per share. Speaker 500:10:28Our cash, cash equivalents and investments increased to $302,800,000 as of March 31, 2024, up from $286,300,000 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. Speaker 200:11:12Thanks, 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 are executing across our label expansion opportunity, launches and are very much looking forward to the filixibat interims ahead. And with that, operator, please open the call for questions. Operator00:11:33Thank Your first question on the line comes from Jessica Fye of JPMorgan. Please go ahead. Your line is open. Great. Good afternoon. Operator00:11:56Thanks for taking my question. For the 2 valexovad 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? Speaker 200:12:18Thanks, Jess, for the question. I'll ask Joanne to jump in and talk a little bit about the background setting. Speaker 400:12:23Yes. Thanks, Chris, and thanks, Jess, for the question. 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 TBC, we're allowing patients who are either on or not on Ursodiol and we're allowing patients with varying with any level of VAP FOS. So, a little different than some of the other trials that have been for the other agents kind of in this area. Speaker 400:12:48So, we really think this translates to use in first line ultimately for PBC. For PSC, as you know, there's no other therapies. And we know that a majority of patients with both these diseases, PSC and PBC, 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. Great. Operator00:13:19And maybe just one more if I could. Can you remind us what to expect from a tolerability standpoint for voxelabat? Speaker 400:13:30Yes. Well, thanks for the follow-up, Jessica. So we know IVAD inhibitors quite well. And I will say so far, you know, belixibat kind of tracks in what we know. And we know exactly how to dose these and we know exactly what to look for. Speaker 400:13:45So, we don't expect really any surprises in this way. Operator00:13:52Great. Thank you. Speaker 600:13:55Thanks, Jess. Operator00:13:56Thank you. Your next question comes from Manu Foroohar from Leerink Partners. Your line is now open. Speaker 700:14:05Thanks so much for taking the question guys. A quick one. You mentioned about a $3,000,000 impact this quarter, it sounds like, some changed health care issue. To what extent is that sort of a one time loss revenue as opposed to revenue by pop up in sort of a recognition function next quarter? And separately, when you think about that $3,000,000 dollars as we try and back that out and think about kind of underlying demand metrics, how is that separated between your products this quarter and sort of the split between the Marley versus the acquired TPx assets? Speaker 200:14:41Thanks for the question, Mani. I'll let Peter kind of dive into the details. I mean, short answer to those, it's a one time effect, but Peter can give a little color on the background here. Speaker 300:14:51Yes. One time effects that has concluded by the end of the quarter. So don't expect to see any lingering effects from this in future quarters. And the overall demand for all of our products grew, total prescriptions grew over the quarter. So not really that's why we're very confident in the $310,000,000 to $320,000,000 And I think and you also asked about the $3,000,000 I think assigning $3,000,000 by individual product kind of probably false precision here. Speaker 300:15:26I mean, we kind of think about $3,000,000 as an impact across the U. S. Business. Speaker 700:15:35Okay. But is it reasonable to assume that the great majority of it was driven by Liv Marley given the geographics of those products? Speaker 300:15:45I think it's probably balanced. You could think about it generally in proportion to the size of the products. Speaker 700:15:55Okay, that's helpful. And as we think about between now and the end of the year, staying on commercial questions, obviously, you maintained your guidance. Should we think about the tempo between now and reaching somewhere in 3.20 to 3.20 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? Speaker 300:16:23Yes, I think I mean, the way we think about it is generally consistent. I mean, the cadence of demand is strong and we see it growing quarter to quarter. You will have PFIC coming on, although as we've commented, most of 2024, we expect many of the PFIC dispensers to be pre drug and that 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. Speaker 700:16:52Okay. That's helpful. Thanks guys. Speaker 600:16:56Thanks for the question. Operator00:16:59The next question on the line comes from Gavin Clark Gartner of Evercore ISI. Please go ahead. Your line is now open. Speaker 800:17:08Hey, 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 3 standard deviation that was informing your powering assumptions. But was that 1.75 absolute or placebo adjusted? Maybe just remind us your expectations for the placebo arm for this trial? Speaker 400:17:33Yes. So, yes, 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 by putting that together. And as you know, those are always kind of a starting point for where you kind of put the study. Speaker 400:17:54But we did want to share at least our starting point for looking at the study design. Speaker 800:18:02Yes. That's helpful. And are you able to share the baseline pruritus scores for either trial for valexibat? Speaker 400:18:11Not at this point. When we 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. Speaker 800:18:25That sounds good. Just a last one. Any updates on the potential for orphan drug status for PFIC in the EU? Speaker 200:18:35Thanks 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 data for the Litt Marley program in PFIC, providing some real advantages for patients. So hoping to have an update on that one soon. Speaker 300:19:05Sounds good. Thanks guys. Speaker 600:19:08Yes. Thanks for the questions. Operator00:19:12The next question on the line comes from Day Gunhav from Stifel. Please go ahead. Your line is open. Speaker 900:19:19Hey, good afternoon guys. Thanks for taking my questions. Maybe 2 part question. On the P6 side of the story, I was wondering if you can comment on your dialogue with the physicians given the label disparity between this and Bilvey 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? Speaker 900:19:41And switching over to valexibat, bearing in mind the interim update in June for both Vestas and Vantage, how are you guys thinking about sort of the Galaxo drug towards the back end of this year? And how might that impact your PBC strategy if both come out positive? Thanks so much. Speaker 200:20:01Yes. Thanks for the questions. Maybe I'll just make a quick comment on valexovad competition briefly and then pass over to Peter to talk about PFIC. And what we're seeing and kind of how we've approached the dosing for valexovat, I think provides potential for a real advantage in terms of activity level. And so that we've learned across all of the IBAT programs, in particular, all the work we've done with Lyth Marley and voxelabat on understanding where we're at on the dose response curve, I think provides the potential to have really strong activity here. Speaker 200:20:35Of course, this is something we'll see play out with the actual data sets 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. Speaker 300:20:52Sure. Yes, thanks for the questions. Feedback on the Marley profile and PFAC has been very positive. I think we've a lot of favorable feedback on the efficacy profile that was observed in the March study is reflected in the label as well as the broader genetic types of PFIC that are included in the labeling, which can sometimes make a difference in market access depending on the payers' policies. So really, really favorable feedback from clinicians and patients, happy to have Lyub Marley available for those patients. Speaker 300:21:24And yes, payer conversations so far are going well. It's kind of 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 5 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 Alagille when the initial label was 1 year of age and older and then lower the age. Speaker 300:21:51Those subsequent follow-up conversations generally occur pretty quickly to update policies. Speaker 900:21:57Great. Thanks for taking our questions. Speaker 600:22:01Yes. Thanks for the questions. Operator00:22:05The next question on the line comes from Steven Seedhouse of Raymond James. Please go ahead. Speaker 1000:22:12Hey, good afternoon. Thanks for taking the questions. 2 separate questions. I'll just ask them both now because they're pretty straightforward. First on the PBC readout, you mentioned price improvement safety and serum bile acids will be the focus of that data release. Speaker 1000:22:28And I'm just curious if you'll also be sharing liver function tests, ALT plus ALT, KST, bilirubin, just to Speaker 1100:22:36get a sense of even from Speaker 1000:22:38a safety standpoint, what's happening there, if there's any impact of de novo bile acid synthesis on any of those parameters? And then the second one is just on business development. Would be curious your comments or thoughts on just the overall view of that or priorities for Miriam over the next call it 12 to 18 months? Are you thinking about expanding the pipeline or focusing on blixibat primarily? Thank you. Speaker 200:23:09Thanks, Steve, for the question. I'll speak to and comment on kind of our business development efforts and let Joanne come back on the PBC interims. And for our strategy and approach to business development remains consistent with what we've done over the course of MiRIM. It's very much in our DNA. It's how the company came to be and started. Speaker 200:23:30And 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 valexovat developments that there's no urgency to do something. So we can remain disciplined in looking at ways to grow the company. And then maybe Joanne can speak to the PBC question. Speaker 400:24:14Yes. Thanks, Chris, and thanks for the question. 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 the dose moving forward. Speaker 400:24:26So with that, we'll look at pruritus, we'll look at serum bile acids and safety in particular. 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. Speaker 700:24:45Thanks a lot. Speaker 600:24:48Thanks for the questions. Operator00:24:52The next question on the line comes from Brian Skorney of Baird. Please go ahead. Your line is open. Speaker 300:24:59Hi. This is Luke on for Brian. As we set expectations for Vantage, in particular thinking about a comp to the seladelpar response study, Do you think the subgroup in that study with baseline NRS greater than 4 is a reasonable comp for pruritus benefit? And then, are you aware of the 11 point NRS scale that you used in that study is the same as the itch reported outcome scale that you're using? Speaker 200:25:27Thanks Luke for the questions. Yes, I mean the scale used, it is similar. I mean there's some very minor differences, but for adults, pruritus measurements and registrational studies that's kind of this is all in line with FDA guidance. That uses a 10 scale. That's what we're using in our study. Speaker 200:25:46So there is some definitely some similarity there. And the treatment effect in that subset that they looked at, it's not too far off with how we looked at our kind of change from placebo assumptions and powering, right, where we looked at the 1.75 difference from placebo. So I'm looking at potentially a little bit more effect from an IVAT. But in general, it's really not too far off if you're thinking about steady 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. Speaker 1100:26:33Great. Thank you. Speaker 600:26:36Thanks for the question. Operator00:26:39The next question on the line comes from David Lebowitz of Citi. Please go ahead. Your line is open. Speaker 1200:26:46Thank you very much for taking my question. On the 1.75 point difference on psoriasis, 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 what that point and how you will use it to consider upsizing if that is needed, what type of thresholds we could expect? Speaker 400:27:20Yes. Well, thanks for the question. We're not going to get into the details of the study design, but I just provided some of the 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 3 in terms of standard deviation is 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 kind of where we are with the interim. Speaker 400:27:49Obviously, we'll share the results of the interim and since we'll be blinded, we certainly hope that we'd be continuing the study. So that's what we hope to be done with you in June. Speaker 200:27:57And one add to that, David, is that the measurement is actually looking at over time, right? It's the months 3, 4, 5 sorry, 4, 5, and 6, 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. So that's another kind of factor to the study design applying what we used from the PFIC study in the adult settings. Speaker 1200:28:29Got it. Thank you very much for taking my question. Speaker 600:28:33Yes. Thanks for the question. Operator00:28:37The next question on the line comes from John Walden of Citizens JMP. Please go ahead. Your line is open. Speaker 300:28:45Hey, thanks for taking the question. Just logistic one from me on PBC and PSC. When you select a dose, 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. Speaker 200:29:11Thanks, John, for the question. Just on timelines, 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. Speaker 400:29:28Yes. So we're going to be continuing we are continuing enrollment in the study at this point. And then we'll be continuing with one active dose and placebo. So we expect to include all the patients ultimately in the analysis when we can ultimately unblind the whole data set. Speaker 600:29:56Thanks for the question, John. Operator00:29:59Next question on the line comes from Mike Bowles of Morgan Stanley. Please go ahead. Your line is open. Speaker 200:30:07Hi. 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 drug? Speaker 200:30:18Thanks. Speaker 300:30:21Yes. Thanks for the question, Rohit. In U. S, obviously, launch is underway, seeing prescriptions come in for livmarly and copepit patients now. We have talked about we have about 25 patients in the U. Speaker 300:30:37S. 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 going forward. Speaker 700:30:55Thank you. Operator00:30:59The next question on the line comes from Ed Arce from H. C. Wainwright. Please go ahead. Your line is open. Speaker 1100:31:08Hi, good afternoon, everyone. This is Thomas Yip asking a couple of questions for Ed. Thank you for taking my questions. So first, following up on U. S. Speaker 1100:31:17Performance for LUXALLE PFAG. Just wonder of the $42,800,000 net sales in the Q1, how much was it from PFIC approximately? And also what are some early launch metrics that investors can update? Speaker 200:31:38Thanks Thomas for the question. In Q1, there really there is no PFIC 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 2 as we get into the back half of the year where we expect more full reimbursement. Speaker 1100:32:03Got it. And then switching gears to the European front, have you any interaction with either EMA or CHMP recently given your expectation on recommendation in the first half this year? And if positive, any ongoing commercial preparations for European market for PFIC? Speaker 200:32:31Thanks for the follow-up there. On the EMA discussion, over the 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 PFIC. Speaker 300:32:52Yes. Certainly, upon a potential approval, we would be ready to launch Lid Marley and P. Baker in Europe. Prescribers for P. Baker essentially identical to the prescribers of Lid Marley for Algill syndrome. Speaker 300:33:07So we'll be ready to go with dossier submissions to health technology agencies etcetera to work with pricing and reimbursement at that time and attribute level as well. Speaker 1100:33:20Okay. And that's one last question from us. Can you discuss some of the main drivers for the biologics of product sales that's slightly $5,000,000 quarter to quarter? Can you provide some major factors? Speaker 300:33:41Yes. I mean, we mentioned the change healthcare cyber attack was kind of in play for our entire portfolio. I think if you look back over time, in the bile acid product sales, there is quarter to quarter volatility, given the ultra rare nature of the disease in small number of patients over time. But do see an opportunity to continue to build on those products this year, mid single digit year on year growth consistent with what historically is our expectation. And going forward, really excited about potential approval by FDA next year for Kenadol and CTX and the chance to go out and find more patients there. Speaker 1100:34:28Understood. Thank you again for the kind of questions. We look forward to the CXMP recommendation soon and also your June presentation for elixir. Sounds good. Thanks, Thomas. Operator00:34:43We have no further questions. Therefore, I will hand back the call to Chris Peete, CEO, for final remarks. Speaker 200:34:51Great. Thank you all for joining us today. Really appreciate the interest in Miriam and our programs. Have a good evening. Bye bye. Operator00:35:02This concludes today's conference call. Thank you all for joining. You may now disconnect your lines.Read morePowered by