NASDAQ:MIRM Mirum Pharmaceuticals Q4 2023 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-$0.66Consensus EPS -$0.34Beat/MissMissed by -$0.32One Year Ago EPSN/AMirum Pharmaceuticals Revenue ResultsActual Revenue$69.55 millionExpected Revenue$66.73 millionBeat/MissBeat by +$2.82 millionYoY Revenue GrowthN/AMirum Pharmaceuticals Announcement DetailsQuarterQ4 2023Date2/28/2024TimeN/AConference Call DateWednesday, February 28, 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)Annual Report (10-K)Earnings HistoryCompany ProfilePowered by Mirum Pharmaceuticals Q4 2023 Earnings Call TranscriptProvided by QuartrFebruary 28, 2024 ShareLink copied to clipboard.There are 13 speakers on the call. Operator00:00:00Good afternoon, ladies and gentlemen. Thank you for joining today's Merrim Pharmaceuticals Reports Q4 and Year End 2023 Financial Results and provides business update. My name is Tia, and I will be your moderator for today's call. All lines will be muted during the presentation portion of the call with an opportunity for questions and answers at the end. It is my pleasure to pass the call over to Andrew McKibbin, Vice President of Investor Relations and Finance. Operator00:00:30Please proceed. Speaker 100:00:33Thanks, Kia, and good afternoon, everyone. I'd like to welcome you to Mirum Pharmaceuticals' Q4 2023 conference call. I'm joined today by our CEO, Chris Peetz our President and Chief Operating Officer, Peter Radovich our Chief Scientific Officer and Head of Research, Tam Big our Chief Medical Officer, Joanne Kwan and Eric Biercol, our Chief Financial Officer. Earlier today, Merrim issued a news release announcing the company's results for the Q4 and full year 2023. Copies of this news release and SEC filings can be found in the Investors section of our website. Speaker 100:01:04Before we begin, I'd like to remind you that during the course of this call, we will be making certain forward looking statements about Mirim and our programs based on management's current expectations, including statements regarding current and future business plans, development programs and regulatory expectations, strategies, prospects, market opportunities and financial expectations. Emera is under no duty to update these statements, and they are subject to numerous risks and uncertainties, and actual results could differ materially from those results anticipated by these statements. Investors should read the risk factors set forth in Miriam's 10 Q for the quarter ended September 30, 2023 and the subsequent reports filed with the SEC. With that said, I'd like to turn the call over to Chris. Chris? Speaker 200:01:43Thanks, Andrew, and good afternoon, everyone. I'm excited to kick off an update on the many achievements for Miriam in 2023, another year of significant growth for us and cover highlights for what lies ahead for the company. Our continued progress in 2023 and opportunity ahead reflects the dedication of the Miram team to making a difference in the lives of patients and their families around the world. Over the year, we transformed our business with a broadened reach to patients in the U. S. Speaker 200:02:11And internationally, achieving $179,000,000 in net product sales and 142% year over year growth in total revenue. This was driven by continued strong growth for expansion of our commercial portfolio with the acquisition and integration of Kenadol and Colbond. We have now built a growing self sustaining leading rare disease business that is positioned for a great 2024 and beyond. The last year has shown the value creation potential of our strategy to drive growth in our commercial medicines, unlock the potential of upcoming label expansion opportunities, leverage our expertise in cholestasis in adult settings and continue to grow the pipeline. Looking at the year ahead for execution on our strategy, overall from the current commercial business, we expect to achieve $310,000,000 to $320,000,000 of net product revenue in 2024. Speaker 200:03:09This is expected to be driven by growth across all 3 commercially available medicines. We also have multiple regulatory and clinical catalysts this year to advance our pipeline. For Le Marley and PFIC, we are on track for our PDUFA date on March 13. We're excited for the opportunity to bring the strong results of the MARGE PFIC Phase 3 study to patients with a potential label expansion. We're also preparing for the submission of the positive RESTORE Phase III results of Kenadol and CTX in the first half of the year, an important label enabling opportunity. Speaker 200:03:43And we've also made great progress with the VISTAs and VANTAGE studies for valexibat in PSC and PDC. Patients for the interim analyses have been enrolled and we expect to conduct a dose selection interims of these adaptive studies in the first half of the year. We see the PSC program as an opportunity to bring the first ever therapy to market for this progressive burdensome disease. I'm proud of all of our progress in 2023 and the potential ahead for Mira. We look forward to continuing to grow the impact of our programs, providing life changing medicines to patients with rare disease. Speaker 200:04:16Now before I turn the call over to Peter to discuss commercial business, I wanted to welcome our new Chief Medical Officer, Doctor. Joanne Kwan, who joined Miram in January. We are thrilled to have Joanne and her extensive leadership and scientific expertise helping to drive the value creation we see ahead for Miram. We'll hear from her later on this call. And for now, I'll turn the call over to Peter to discuss our commercial business. Speaker 200:04:39Peter? Speaker 300:04:40Thanks, Chris. Speaker 100:04:41We are excited by the tremendous progress we have made across our U. S. And international businesses, coming off of 2023 where we saw 138% growth in total net product sales compared to 2022. We are already seeing the benefits of our newly expanded pediatric hepatology franchise and enhanced offering amongst the patients and prescriber community. I'm proud of the Miram team's focus on patients, while simultaneously driving the seamless integration of Polvol and Kenadol. Speaker 100:05:12And this is evident in the $69,600,000 total net product sales we achieved in the Q4 of 2023, which reflected growth across all three commercial medicines. For LIVARLE, total global net product sales grew to $41,400,000 in the 4th quarter. Our U. S. Business saw $31,400,000 $111,000,000 for the quarter year respectively, representing approximately 63% year over year growth. Speaker 100:05:44And internationally, we are seeing consistent strong uptake as well, but we continue to anticipate quarter over quarter variability in international revenue. Moving on to KEYDOL and Pohlbaum, the Q4 was the 1st full quarter with these two medicines under Miriam's leadership. And I'm very happy with how quickly we have been able to transition these medicines into our business. In the Q4, we recognized net product sales of $28,100,000 the highest quarter to date for the bile acid products. And looking ahead, we expect steady growth for these 2 medicines in line with historical trends. Speaker 100:06:24Taking all the strong dynamics in the business together, we expect net product sales to be $310,000,000 to $320,000,000 for 2024. This represents over 70% growth from 2023 with increased contribution from all commercially available medicines. In summary, 2023 was a fantastic year for RT as we expanded our portfolio to 3 commercial products and grew our presence both in the U. S. And worldwide. Speaker 100:06:53We continue to see the tremendous impact of all three products across their patient communities and we look forward to further expansion in 2024 as we stay committed to our mission of delivering life changing medicines to patients worldwide. And with that, I'll turn it over to Joanne. Joanne? Thanks, Peter. Speaker 300:07:13Before I begin, I'd like to say that I'm thrilled to join Europe. This experienced team has accomplished so much in such a short period of time, and I'm excited to continue the momentum and progress in the treatment of rare diseases. In 2024, we are looking forward to multiple regulatory and development milestones. From a regulatory perspective, we're tracking well towards our PFIC PDUFA date of March 13 and expect a decision from EMA in the first half of the year. Regarding Kenadol and CTX, we're making good progress here as well. Speaker 300:07:45We've had positive interactions with the FDA and no additional clinical studies are required to support our NDA submission, which is planned in the first half of twenty twenty four. Turning to valexibat, we're also looking forward to our upcoming interim analyses in our VISTAs PSC study and our VANTAGE study in PBC patients. We are on track for the interim analysis to read out in the first half of this year. Note that the studies are now continuing to enroll patients with the goal of supporting registration. As a reminder, the Visto's PSC study will have a blinded interim analysis to support dose selection. Speaker 300:08:22Based on the data, this will allow patients from the selected dose and placebo arms to be included in the pivotal data set. For the larger indication of PBC, the study is designed so that we unblind and show the top line results from an interim analysis, which will include data on pruritus improvement, change in serum bile acids and safety. Both of these studies represent an important step towards addressing the accumulation of bile acids in broader patient groups with adult full of phases where a significant portion of patients are without adequate treatment options for their cholestasis and a severe symptomatic burden. I'm excited to be part of this team and look forward to sharing our progress with VIVITR. With that, I'll now turn the call over to Eric to discuss our financial results. Speaker 300:09:08Eric? Speaker 400:09:10Thanks, John. Earlier today, we issued a press release that included financial results for the quarter full year, which I'll briefly summarize. Note our 10 ks filing is planned for next week as our auditors finalize the first 404 and other audit procedures for Miran. Total revenue in the Q4 2023 was $69,600,000 and for the year ended December 31, total revenue was $186,400,000 including total net product sales of $178,900,000 This is compared to total revenues of $27,900,000 $77,100,000 for the Q4 and full year 2022, respectively. For operating expenses for the year ended December 31 were $293,000,000 which includes research and development expenses of 102,600,000 SG and A expenses of $145,900,000 and cost of sales of 44,500,000 dollars For the year ended December 31, 2023, net loss was $160,900,000 or $3.94 per share. Speaker 400:10:28Net loss for the year included non cash stock based compensation expense of 35,000,000 and intangible amortization of $10,400,000 This intangible amortization is largely reflected in our cost of goods sold. In the Q4, COGS also reflected a reserve in excess of $5,000,000 for inventory, primarily related to the bile acid acquisition. For the year ended December 31, 2023, our cash used in operating activities was $70,900,000 down from $120,100,000 the year before. We had cash, cash equivalents and investments of $286,300,000 as of December 31, 2023. We remain well funded with a strong and self sustaining business. Speaker 400:11:23Our increasing revenue base places us in an exceptional position to grow the business through clinical development and continued expansion of our global commercial presence over the coming years. Now I'll turn the call back over to Chris for final comments. Speaker 500:11:40Thanks, Eric. Speaker 200:11:41It has been a strong year for Miram, finishing with the biggest ever revenue quarter for Lymph Marley and the bile acid portfolio. We are poised for another great year in 2024 with 3 growing commercial products and with several clinical and regulatory catalysts as we strive to make continued advances for rare disease patients around the world. We look forward to keeping you updated on our exciting progress throughout the year. And with that, operator, please open the call for questions. Operator00:12:10We will now begin the Q Speaker 600:12:45This is Nick on for Jess. Congrats on the quarter and thanks for taking our questions. Maybe looking at the 2024 net product sales guidance of $310,000,000 to $320,000,000 Can you maybe discuss a bit more about how much of the Marley growth is baked in there versus contributions from Kitadong, Colmum? And also comment a little bit on Lebmarly and Elagil and maybe provide an update on what you're seeing on the competitive dynamics for BELLETE, if any? Speaker 200:13:13Yes. Thanks for the question. First, just from a starting point, we're not giving product level guidance at this point, but really great dynamics across the products. And I'll let Peter kind of give a little color on what we're seeing across the business. Speaker 700:13:25Yes. And I think as you think about the components and Speaker 100:13:28the color commentary there, the biologics product portfolio, we expect it to grow in line with historical trends, which have been kind of on the order of Speaker 700:13:36mid single digit year on year growth rates. And then looking into the Marley growth case in the U. S, which has been strong over the last over all the quarters of its marketing, expected to see it continue to move forward with that cadence. And then on the international side, we do expect quarter to quarter variability there, probably international sales. And then your other question about competitive dynamics now as you go, I think as you can see from the numbers here, it's really you haven't seen a measurable impact from those at all really the La Marlin growth trajectory hasn't been impacted by that. Speaker 600:14:13Great. And maybe just one more, thinking about the VANTAGE study and how it allows for patients both the first line setting, second line setting post EDCA. Beyond the rate of pruritus observed across these two settings, can you maybe talk a little bit more about the differences in how it presents and or maybe the severity of itch that is observed in these two patient populations? Speaker 200:14:35Joanne and I can speak to the difference between the lines therapy. Speaker 300:14:39Yes. So as you know, pruritus is a big issue in both of these diseases. We have a broad patient population that we're accessing in terms of the vocab studies. And actually there's this is broader than, for instance, some of the other entrants in this space who are looking at biochemical abnormalities. I'll remind you that we have agreement from FDA that pruritus is a registrational endpoint, and both of these studies are actually positioned to be registrational after the interim analysis. Speaker 300:15:07So we feel pretty confident in terms of both the study design, the drug itself, or dose selection and moving forward at this point. Operator00:15:24The next question comes from the line of Gavin Clark Gardner with Evercore ISI. Please proceed. Speaker 800:15:31Hi, this is Yasha on for Gavin. Just two questions for us. For the first one, for Lid Marley, especially with the start of the year, just wondering if you have seen patients being reweighted and put on higher doses as they grow? Speaker 700:15:47Happy to do that. Speaker 400:15:47That's the question we have and Speaker 700:15:49we published some of that information last year. Not surprisingly, it's a rate based product. Speaker 100:15:55And then in the growth data Speaker 700:15:57that we've seen published with Marley, we have made you see weight based dose adjustments. Speaker 800:16:05Awesome. And then one more for the EU business. Do you have any commentary on potential implications of Ipsen refiling odevixibat odevixibat in the EU under a different brand name, not with orphan status? And then also want to confirm that there'd be no reimbursement or access impact on Wood Marley in the EU? Speaker 200:16:26Thanks for the question. At this point, we're focused on getting access broadly across all international markets for Lymphomarli and Alagille and having great progress as we see demand continuing to grow across Europe and other international markets. Can't really speak too much to the BELVE procedure, other than that we're making great progress getting LYMARLE. It really rolled out a standard of care in Alagille across all markets. Speaker 800:17:03Great. Thank you. Speaker 200:17:06Thanks for the question. Operator00:17:07Thank you. The next question comes from the line of Mani Faruah with Leerink Partners. Please proceed. Speaker 500:17:17Hi, good afternoon. This is Lillian Songo on for Mani. I'm sorry, I couldn't hear, and apologies if it was asked earlier. I heard the question, but we couldn't hear the answer. This was regarding the growth expected for LIFO in terms of weight based increases as the patient get older. Speaker 500:17:35And to add to that, if you could comment in terms of how should we look how should we think about the way and then the dosage for new patients versus existing ones? Speaker 200:17:50Yes. So the question and just to make sure that I recap it here and ask for any color from Peter. Basically, the original question was around, do we see adjustments to dose as patients are on therapy over time, per the label. And we do see that over time. I think, Lutz mentioned that there's been a recent poster presentation on it. Speaker 200:18:16So that is a dynamic that we see out with the treating physicians. And so were you able to the microphone pick us up this time? Speaker 500:18:34Yes, we could hear you really clearly. Thank you. And as a follow-up regarding the Chinnabales Shonam assets, could you maybe comment in terms of what the international growth opportunities are for those assets? Speaker 200:18:50Yes. So question about Kenadol Coal Bomb Growth Opportunities. And I'll let Peter speak a little bit to the dynamic of what we have today and then also a little bit about the Kenadol label expectations expansion opportunity for CTX. Speaker 100:19:06Yes. With both products, we continue to see growth in line with historical trends, certainly with KEYNOTE Speaker 700:19:14and recently announced Phase 3 data for CTS and potential approval next year for CTS. I think there's an opportunity key adults never been promoted for CTS before. So I think there's an opportunity to find more patients through disease state awareness and hopefully increasing the diagnosis rate. One thing we know about CTS is maybe only about 10% of the patients are diagnosed and under management. So the opportunity there is Speaker 100:19:42to try to identify more patients and Speaker 700:19:45to them on therapy. Operator00:19:52Thank you. Thank you. Speaker 200:19:55Thanks for the questions. Operator00:19:58The next question comes from the line of Mike Coles with Morgan Stanley. Please proceed. Hi. Speaker 900:20:06This is Rohit on for Mike. Thanks for taking our questions. Can you just talk about your expectations for the upcoming LIV MARLY, PDUFA in PFIC and any launch prep that's associated? And do you think that any patients are currently using it off label? Thank you. Speaker 200:20:23Thanks for the question, Rohit. On the regulatory front, we're kind of where we expect to be, so excited about PDUFA date coming up. And I'll let Peter speak a little bit to how we're preparing for that and some of the first opportunities we see there. Speaker 100:20:38Yes. We're really excited about the potential approval for LiMARLE and PFIC. Speaker 700:20:43I think the Mars Speaker 100:20:44data reinforce the strong value proposition that LiMARLE Speaker 700:20:46offers to our various stakeholder groups. Physician prescribing universe is essentially identical to the physician prescribing universe is essentially identical to the AlloSure audience. So there's really no need for meaningful operating expense increased access. Speaker 100:21:06And we do have Speaker 700:21:07a number of patients in the mid-20s who are receiving clinical rollover or extended access, the barley, who would be eligible to roll over the commercial upon approval. Speaker 900:21:26Thank you. Speaker 200:21:28Thanks for the question. Operator00:21:31The next question comes from the line of Steve Seedhouse with Raymond James. Please proceed. Speaker 1000:21:38Yes. Hi. This is Timur Ivannikov on for Steve Seedhouse. So our first question is related to the gross margin. I think you mentioned there was an inventory charge related to the acquisition in 4Q. Speaker 1000:21:51Just what are your expectations for gross margin in 2024 and perhaps also the OpEx trajectory in 2024? Speaker 400:22:04For us, gross margin, we do expect that the intangible amortization will continue. I mean, it's largely related to the bile acid acquisition intangibles, which we're amortizing over 50 quarters. As far as sort of traditional cost of goods, it consists of the actual cost of products and royalties. So that will continue at approximately the same sort of percentage of sales. And then we did have a larger than we expect going forward amortization or reserve for the inventory that came with the acquisition. Speaker 400:22:45So we might have some reserve in some quarters, but not to the extent we had in the Q4. In terms of overall operating expenses, R and D and SG and A, 4th quarter is probably pretty representative of what we expect the next few quarters. Speaker 1000:23:09Okay. Thank you very much. And then just a question on your overall enrollment dynamics in VISTUS Advantage. I think in VISTUS, your interim is only on 45 patients. So can you comment about the overall enrollment target dates? Speaker 1000:23:26And do you see any differences between the studies in terms of the enrollment dynamics? Speaker 200:23:34Thanks for the question. I mean, the simple answer here is we have patients in for the interim. We're continuing to enroll for Part 2 now. And we'll be able to give better guidance for the full study enrollment at that interim when it comes up. Speaker 1000:23:54Okay. Appreciate it. Thank you very much. Speaker 200:23:57Yes. Thank you. Operator00:24:00The next question comes from the line of David Lebowitz with Citi. Please proceed. Speaker 1100:24:06Hi, this is Divanjana on for David. Thanks for taking our call. We wanted to ask about the blinded PSC interim analysis. So beside the dose selection, would you share any other data points at a blinded basis? Speaker 300:24:24So we will be blinded in this interim. And this is designed so that if it passes the threshold, it is designed to be predictive of a clinic meaningful and statistically significant positive pivotal analysis in the end. So that's all I can really share with you at this point. And it's really the threshold was developed based on what we know about the drug, this class of drugs, PSC in general and pruritus. So we feel pretty confident in terms of the design being robust and this being kind of well set up to produce a registration study for us. Speaker 200:25:03And in terms of communication at the point of interim for the VISTA study, assuming a positive interim, we would communicate that the interim has occurred and study continues as planned, which would be the extent of the information that we receive here on the team as well. Speaker 1100:25:19Okay. So as a follow-up to that, could you tell us a bit more about I mean, while unlikely, is there any other scenario like that would play out if somehow like if you see that the efficacy is not crossing the threshold on a blind blinded basis that was pre specified? Speaker 200:25:40So the study design in the event where that threshold is not met basically converts to an open interim analysis. We'd be able to look at the data, share top line findings of it and what the next steps are for the study in that scenario. Speaker 1100:26:00Okay. Thank you for the insight. Speaker 200:26:04Yes. Thanks for the questions. Operator00:26:06Thank you. The next question comes from the line of Brian Skorney with Baird. Please proceed. Speaker 900:26:16Hi, Ann. On PBC beyond pruritus, how are you thinking about the impact on outcomes and other markers in Vantage? And how can this help you make the case to physicians and patients? Speaker 200:26:31Yes. Thanks for the question. I'll let Joanne speak a little bit to kind of the registrational plan for PBC. Speaker 300:26:38Yes. So in terms of the interim, we're just going to be looking at top line. So, pruritus, serum bile acid safety, as top line. And as you know, age is a big issue in this particular patient population for which there's really no particular therapies at this point. So we're pretty confident that this study, the way it's designed actually addresses an important unmet medical need at this point. Speaker 200:27:03Yes. And just to recap, one of the points made earlier, the eligibility criteria for the VANTAGE study does not have an alkaline phosphatase criteria. So it includes patients that are traditionally thought of as first line that have really only UDCA as on label and still experience the same rates and severity of pruritus as later lines of treatment. So a lot of unmet need across the PBC patient population. Speaker 700:27:38Great. Thanks. Speaker 200:27:41Thanks for the question. Operator00:27:42Thank you. The next question comes from the line of Ed Arce with H. C. Wainwright. Please proceed. Speaker 1200:27:51Hi, everyone. Thanks for taking my questions and congrats on another strong quarter. I have 3, if I may. Firstly, on the VANTAGE study in PBC, as you said, it's a blinded data readout. Obviously, this is, as you mentioned, basically pruritus versus placebo and whether it's stat sig. Speaker 1200:28:15But I'm also wondering if there are specific thresholds of activity on pruritus that you're looking at, not just so much to proceed, but also as you think about the competitive landscape and where you'd like to see that come in? Secondly, if you could speak a bit about the PFIC opportunity in terms of 2024, sort of the sales, the speed of the sales ramp, kind of what you're expecting the trajectory to be through the remaining quarters of this year? And then lastly, I'm sorry, I didn't hear when Peter was reviewing, I think it was Peter, the numbers for LIV Marley, both U. S. And international as well as bile acid products for the Q4. Speaker 1200:29:07Thanks so much. Speaker 200:29:11Great. Thanks for the question. Maybe I'll just hit on recap one of the points on Vantage and then pass it over Peter to talk about PFIC and recap some of those sales numbers. And the way we think about Vantage and valexovat and PVC, it really comes down to this the highlight here on lines of therapy and where various agents are labeled and expected to be labeled. The majority of patients are first line patients and the majority of them have pruritus. Speaker 200:29:43So that's really the primary unmet need that we're going after. So showing a significant impact on that pruritus, we think will be very clinically meaningful. And that's the lens that we're going to be taking towards that interim data when we look at it and make a decision on next steps and continuing to roll into the pivotal portion of the study. With that, I'll pass over to Peter on the next two points. Speaker 700:30:08Yes. Sorry, I'll try to speak up a little bit here. Speaker 100:30:10So on the PFIC 2024 revenue expectation piece, I think we'll be upon a potential approval here in March, we'll Speaker 200:30:20be spending a fair bit Speaker 100:30:22of time working closely with payers get LiDARLE positioned in their new to market policies. So I think if you think about 2024, we'd expect a lot of free drug or PAP with PFIC really coming in more in 2025, somewhat similar to what we saw in the Alagille initial launch where the early quarters were high PAP drug. And then in terms Speaker 400:30:48of the Speaker 100:30:48numbers from Q4, LIDMARLE totaled $41,400,000 worldwide, dollars 31,400,000 US10 International and then bile acid products were $28,100,000 for Q4. Speaker 1200:31:06Great. And then maybe just further clarification on PFIC. Appreciate the comments. Just wondering given that you mentioned earlier there's about 20 ish patients right now on expanded access. How soon would you expect those to be able to roll over onto commercial drug once again? Speaker 100:31:30Yes. What we saw in the Alagille launch is that occurred in a quarter or 2 generally is what we saw. And so, yes, I think that's probably consistent with our expectation for those 25 or so. Speaker 1200:31:45Thanks so Speaker 200:31:47much. Thanks for the questions. Operator00:31:49Thank you. The next question comes from the line of Jonathan Willoughby with Citizens JMP. Please proceed. Speaker 1100:31:58Hi, this Speaker 500:31:59is Catherine on for John. I just had 2 kind of quick questions. One time to kind of follow-up on valexibat and just kind of the how you guys see it fitting into the PBC paradigm in the setting of other approved agents that might potentially be effective also on pruritus, whether it's an excuse to see as kind of the combo therapy, sort of a monotherapy for earlier patients? And then also, just as far as preparing for the PFIP launch, I know you said that there's not going to be too much of an investment as far as SGA goes. But like what else is kind of being done to sort of get the word out there about PFIC or do the most of these physicians already know that it's coming? Speaker 200:32:40Thanks for the questions. And just to kind of recap the PBC positioning one more time here. The VANTAGE study includes first line patients that really are when you look at the eligibility criteria for the PPAR programs, for example, and how basafibrates gets used outside the U. S, we are upstream of that setting where when a patient is biochemically controlled, they can still have substantial pruritus, right? That's the area where IVAC can play a very unique role in being in front of the other second line therapies, the PPARs and OCA, for example. Speaker 200:33:24So pretty unique positioning that we see for PBC. I'd add on, in PSC, really exciting opportunity here where valexovat is positioned to be the first and only therapy for PSC by using pruritus as an endpoint really gives an outcome to use for bringing a new therapy to these patients. So quite excited about the positioning there. And pass over to Peter for part 2 here. Speaker 100:33:55Yes. In regards to BPIC, obviously, there's been there's no promotion until an approval. But I mean, I just kind of emphasize that it's a really small number of physicians that take care of these patients in the U. S. I mean, you're probably talking about on the order of 100 physicians in the entire country that take care of PFIC patients. Speaker 100:34:15There have been presentations at the major medical conferences on the data, Phase Speaker 700:34:203 data as well as Speaker 100:34:21the Phase 2 data going back for years. So I think there's a kind of a relatively high awareness of the data. And certainly, from the Alagille approval and commercialization, a lot of comfort with Marley's clinical profile, kind of familiarity with using it, prescribing it, working with our patient support hub, the Miramax's Plus program, co pay support, etcetera. So I think that's probably a fair summary of where things stand today. Speaker 500:34:52Thanks so much. Speaker 200:34:55Thanks for the questions. Operator00:34:58Thank you. There are no additional questions at this time. I will pass it back to Chris Peetz for any closing remarks. Speaker 200:35:07Great. Thanks again for everyone for joining us for today. During the call, I did want to put a plug in for Rare Disease Day tomorrow. Today, to build awareness for the more than 7,000 rare diseases impacting patients around the world and celebrate some of the meaningful advances in research that we've seen in recent years. Tomorrow, as part of Rare Disease Day, we're excited to be able to share a manuscript published in hepatology featuring long term data and patients with Alagille syndrome treated with Leimarley. Speaker 200:35:36So please join us in supporting the many advocacy groups that passionately lead efforts to support patients in research in rare disease tomorrow. And have a great evening. Goodbye. Operator00:35:49That concludes today's conference call. Thank you. You may now disconnect your line.Read morePowered by Conference Call Audio Live Call not available Earnings Conference CallMirum Pharmaceuticals Q4 202300:00 / 00:00Speed:1x1.25x1.5x2x Earnings DocumentsPress Release(8-K)Annual report(10-K) 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.comElon just did WHAT!?As you may recall, Biden and the Fed were working on a central bank digital currency, or CBDC. Had they gotten away with it, the Fed and U.S. banks could have seized control of our financial lives forever. But Trump stopped them cold on January 23rd, 2025, when he outlawed CBDCs… Paving the way for Elon Musk's secret master plan.May 4, 2025 | Brownstone Research (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. Start Your 30-Day Trial MarketBeat All Access Features Best-in-Class Portfolio Monitoring Get personalized stock ideas. Compare portfolio to indices. Check stock news, ratings, SEC filings, and more. Stock Ideas and Recommendations See daily stock ideas from top analysts. Receive short-term trading ideas from MarketBeat. Identify trending stocks on social media. Advanced Stock Screeners and Research Tools Use our seven stock screeners to find suitable stocks. Stay informed with MarketBeat's real-time news. Export data to Excel for personal analysis. Sign in to your free account to enjoy these benefits In-depth profiles and analysis for 20,000 public companies. Real-time analyst ratings, insider transactions, earnings data, and more. Our daily ratings and market update email newsletter. Sign in to your free account to enjoy all that MarketBeat has to offer. Sign In Create Account Your Email Address: Email Address Required Your Password: Password Required Log In or Sign in with Facebook Sign in with Google Forgot your password? Your Email Address: Please enter your email address. Please enter a valid email address Choose a Password: Please enter your password. Your password must be at least 8 characters long and contain at least 1 number, 1 letter, and 1 special character. Create My Account (Free) or Sign in with Facebook Sign in with Google By creating a free account, you agree to our terms of service. This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.
There are 13 speakers on the call. Operator00:00:00Good afternoon, ladies and gentlemen. Thank you for joining today's Merrim Pharmaceuticals Reports Q4 and Year End 2023 Financial Results and provides business update. My name is Tia, and I will be your moderator for today's call. All lines will be muted during the presentation portion of the call with an opportunity for questions and answers at the end. It is my pleasure to pass the call over to Andrew McKibbin, Vice President of Investor Relations and Finance. Operator00:00:30Please proceed. Speaker 100:00:33Thanks, Kia, and good afternoon, everyone. I'd like to welcome you to Mirum Pharmaceuticals' Q4 2023 conference call. I'm joined today by our CEO, Chris Peetz our President and Chief Operating Officer, Peter Radovich our Chief Scientific Officer and Head of Research, Tam Big our Chief Medical Officer, Joanne Kwan and Eric Biercol, our Chief Financial Officer. Earlier today, Merrim issued a news release announcing the company's results for the Q4 and full year 2023. Copies of this news release and SEC filings can be found in the Investors section of our website. Speaker 100:01:04Before we begin, I'd like to remind you that during the course of this call, we will be making certain forward looking statements about Mirim and our programs based on management's current expectations, including statements regarding current and future business plans, development programs and regulatory expectations, strategies, prospects, market opportunities and financial expectations. Emera is under no duty to update these statements, and they are subject to numerous risks and uncertainties, and actual results could differ materially from those results anticipated by these statements. Investors should read the risk factors set forth in Miriam's 10 Q for the quarter ended September 30, 2023 and the subsequent reports filed with the SEC. With that said, I'd like to turn the call over to Chris. Chris? Speaker 200:01:43Thanks, Andrew, and good afternoon, everyone. I'm excited to kick off an update on the many achievements for Miriam in 2023, another year of significant growth for us and cover highlights for what lies ahead for the company. Our continued progress in 2023 and opportunity ahead reflects the dedication of the Miram team to making a difference in the lives of patients and their families around the world. Over the year, we transformed our business with a broadened reach to patients in the U. S. Speaker 200:02:11And internationally, achieving $179,000,000 in net product sales and 142% year over year growth in total revenue. This was driven by continued strong growth for expansion of our commercial portfolio with the acquisition and integration of Kenadol and Colbond. We have now built a growing self sustaining leading rare disease business that is positioned for a great 2024 and beyond. The last year has shown the value creation potential of our strategy to drive growth in our commercial medicines, unlock the potential of upcoming label expansion opportunities, leverage our expertise in cholestasis in adult settings and continue to grow the pipeline. Looking at the year ahead for execution on our strategy, overall from the current commercial business, we expect to achieve $310,000,000 to $320,000,000 of net product revenue in 2024. Speaker 200:03:09This is expected to be driven by growth across all 3 commercially available medicines. We also have multiple regulatory and clinical catalysts this year to advance our pipeline. For Le Marley and PFIC, we are on track for our PDUFA date on March 13. We're excited for the opportunity to bring the strong results of the MARGE PFIC Phase 3 study to patients with a potential label expansion. We're also preparing for the submission of the positive RESTORE Phase III results of Kenadol and CTX in the first half of the year, an important label enabling opportunity. Speaker 200:03:43And we've also made great progress with the VISTAs and VANTAGE studies for valexibat in PSC and PDC. Patients for the interim analyses have been enrolled and we expect to conduct a dose selection interims of these adaptive studies in the first half of the year. We see the PSC program as an opportunity to bring the first ever therapy to market for this progressive burdensome disease. I'm proud of all of our progress in 2023 and the potential ahead for Mira. We look forward to continuing to grow the impact of our programs, providing life changing medicines to patients with rare disease. Speaker 200:04:16Now before I turn the call over to Peter to discuss commercial business, I wanted to welcome our new Chief Medical Officer, Doctor. Joanne Kwan, who joined Miram in January. We are thrilled to have Joanne and her extensive leadership and scientific expertise helping to drive the value creation we see ahead for Miram. We'll hear from her later on this call. And for now, I'll turn the call over to Peter to discuss our commercial business. Speaker 200:04:39Peter? Speaker 300:04:40Thanks, Chris. Speaker 100:04:41We are excited by the tremendous progress we have made across our U. S. And international businesses, coming off of 2023 where we saw 138% growth in total net product sales compared to 2022. We are already seeing the benefits of our newly expanded pediatric hepatology franchise and enhanced offering amongst the patients and prescriber community. I'm proud of the Miram team's focus on patients, while simultaneously driving the seamless integration of Polvol and Kenadol. Speaker 100:05:12And this is evident in the $69,600,000 total net product sales we achieved in the Q4 of 2023, which reflected growth across all three commercial medicines. For LIVARLE, total global net product sales grew to $41,400,000 in the 4th quarter. Our U. S. Business saw $31,400,000 $111,000,000 for the quarter year respectively, representing approximately 63% year over year growth. Speaker 100:05:44And internationally, we are seeing consistent strong uptake as well, but we continue to anticipate quarter over quarter variability in international revenue. Moving on to KEYDOL and Pohlbaum, the Q4 was the 1st full quarter with these two medicines under Miriam's leadership. And I'm very happy with how quickly we have been able to transition these medicines into our business. In the Q4, we recognized net product sales of $28,100,000 the highest quarter to date for the bile acid products. And looking ahead, we expect steady growth for these 2 medicines in line with historical trends. Speaker 100:06:24Taking all the strong dynamics in the business together, we expect net product sales to be $310,000,000 to $320,000,000 for 2024. This represents over 70% growth from 2023 with increased contribution from all commercially available medicines. In summary, 2023 was a fantastic year for RT as we expanded our portfolio to 3 commercial products and grew our presence both in the U. S. And worldwide. Speaker 100:06:53We continue to see the tremendous impact of all three products across their patient communities and we look forward to further expansion in 2024 as we stay committed to our mission of delivering life changing medicines to patients worldwide. And with that, I'll turn it over to Joanne. Joanne? Thanks, Peter. Speaker 300:07:13Before I begin, I'd like to say that I'm thrilled to join Europe. This experienced team has accomplished so much in such a short period of time, and I'm excited to continue the momentum and progress in the treatment of rare diseases. In 2024, we are looking forward to multiple regulatory and development milestones. From a regulatory perspective, we're tracking well towards our PFIC PDUFA date of March 13 and expect a decision from EMA in the first half of the year. Regarding Kenadol and CTX, we're making good progress here as well. Speaker 300:07:45We've had positive interactions with the FDA and no additional clinical studies are required to support our NDA submission, which is planned in the first half of twenty twenty four. Turning to valexibat, we're also looking forward to our upcoming interim analyses in our VISTAs PSC study and our VANTAGE study in PBC patients. We are on track for the interim analysis to read out in the first half of this year. Note that the studies are now continuing to enroll patients with the goal of supporting registration. As a reminder, the Visto's PSC study will have a blinded interim analysis to support dose selection. Speaker 300:08:22Based on the data, this will allow patients from the selected dose and placebo arms to be included in the pivotal data set. For the larger indication of PBC, the study is designed so that we unblind and show the top line results from an interim analysis, which will include data on pruritus improvement, change in serum bile acids and safety. Both of these studies represent an important step towards addressing the accumulation of bile acids in broader patient groups with adult full of phases where a significant portion of patients are without adequate treatment options for their cholestasis and a severe symptomatic burden. I'm excited to be part of this team and look forward to sharing our progress with VIVITR. With that, I'll now turn the call over to Eric to discuss our financial results. Speaker 300:09:08Eric? Speaker 400:09:10Thanks, John. Earlier today, we issued a press release that included financial results for the quarter full year, which I'll briefly summarize. Note our 10 ks filing is planned for next week as our auditors finalize the first 404 and other audit procedures for Miran. Total revenue in the Q4 2023 was $69,600,000 and for the year ended December 31, total revenue was $186,400,000 including total net product sales of $178,900,000 This is compared to total revenues of $27,900,000 $77,100,000 for the Q4 and full year 2022, respectively. For operating expenses for the year ended December 31 were $293,000,000 which includes research and development expenses of 102,600,000 SG and A expenses of $145,900,000 and cost of sales of 44,500,000 dollars For the year ended December 31, 2023, net loss was $160,900,000 or $3.94 per share. Speaker 400:10:28Net loss for the year included non cash stock based compensation expense of 35,000,000 and intangible amortization of $10,400,000 This intangible amortization is largely reflected in our cost of goods sold. In the Q4, COGS also reflected a reserve in excess of $5,000,000 for inventory, primarily related to the bile acid acquisition. For the year ended December 31, 2023, our cash used in operating activities was $70,900,000 down from $120,100,000 the year before. We had cash, cash equivalents and investments of $286,300,000 as of December 31, 2023. We remain well funded with a strong and self sustaining business. Speaker 400:11:23Our increasing revenue base places us in an exceptional position to grow the business through clinical development and continued expansion of our global commercial presence over the coming years. Now I'll turn the call back over to Chris for final comments. Speaker 500:11:40Thanks, Eric. Speaker 200:11:41It has been a strong year for Miram, finishing with the biggest ever revenue quarter for Lymph Marley and the bile acid portfolio. We are poised for another great year in 2024 with 3 growing commercial products and with several clinical and regulatory catalysts as we strive to make continued advances for rare disease patients around the world. We look forward to keeping you updated on our exciting progress throughout the year. And with that, operator, please open the call for questions. Operator00:12:10We will now begin the Q Speaker 600:12:45This is Nick on for Jess. Congrats on the quarter and thanks for taking our questions. Maybe looking at the 2024 net product sales guidance of $310,000,000 to $320,000,000 Can you maybe discuss a bit more about how much of the Marley growth is baked in there versus contributions from Kitadong, Colmum? And also comment a little bit on Lebmarly and Elagil and maybe provide an update on what you're seeing on the competitive dynamics for BELLETE, if any? Speaker 200:13:13Yes. Thanks for the question. First, just from a starting point, we're not giving product level guidance at this point, but really great dynamics across the products. And I'll let Peter kind of give a little color on what we're seeing across the business. Speaker 700:13:25Yes. And I think as you think about the components and Speaker 100:13:28the color commentary there, the biologics product portfolio, we expect it to grow in line with historical trends, which have been kind of on the order of Speaker 700:13:36mid single digit year on year growth rates. And then looking into the Marley growth case in the U. S, which has been strong over the last over all the quarters of its marketing, expected to see it continue to move forward with that cadence. And then on the international side, we do expect quarter to quarter variability there, probably international sales. And then your other question about competitive dynamics now as you go, I think as you can see from the numbers here, it's really you haven't seen a measurable impact from those at all really the La Marlin growth trajectory hasn't been impacted by that. Speaker 600:14:13Great. And maybe just one more, thinking about the VANTAGE study and how it allows for patients both the first line setting, second line setting post EDCA. Beyond the rate of pruritus observed across these two settings, can you maybe talk a little bit more about the differences in how it presents and or maybe the severity of itch that is observed in these two patient populations? Speaker 200:14:35Joanne and I can speak to the difference between the lines therapy. Speaker 300:14:39Yes. So as you know, pruritus is a big issue in both of these diseases. We have a broad patient population that we're accessing in terms of the vocab studies. And actually there's this is broader than, for instance, some of the other entrants in this space who are looking at biochemical abnormalities. I'll remind you that we have agreement from FDA that pruritus is a registrational endpoint, and both of these studies are actually positioned to be registrational after the interim analysis. Speaker 300:15:07So we feel pretty confident in terms of both the study design, the drug itself, or dose selection and moving forward at this point. Operator00:15:24The next question comes from the line of Gavin Clark Gardner with Evercore ISI. Please proceed. Speaker 800:15:31Hi, this is Yasha on for Gavin. Just two questions for us. For the first one, for Lid Marley, especially with the start of the year, just wondering if you have seen patients being reweighted and put on higher doses as they grow? Speaker 700:15:47Happy to do that. Speaker 400:15:47That's the question we have and Speaker 700:15:49we published some of that information last year. Not surprisingly, it's a rate based product. Speaker 100:15:55And then in the growth data Speaker 700:15:57that we've seen published with Marley, we have made you see weight based dose adjustments. Speaker 800:16:05Awesome. And then one more for the EU business. Do you have any commentary on potential implications of Ipsen refiling odevixibat odevixibat in the EU under a different brand name, not with orphan status? And then also want to confirm that there'd be no reimbursement or access impact on Wood Marley in the EU? Speaker 200:16:26Thanks for the question. At this point, we're focused on getting access broadly across all international markets for Lymphomarli and Alagille and having great progress as we see demand continuing to grow across Europe and other international markets. Can't really speak too much to the BELVE procedure, other than that we're making great progress getting LYMARLE. It really rolled out a standard of care in Alagille across all markets. Speaker 800:17:03Great. Thank you. Speaker 200:17:06Thanks for the question. Operator00:17:07Thank you. The next question comes from the line of Mani Faruah with Leerink Partners. Please proceed. Speaker 500:17:17Hi, good afternoon. This is Lillian Songo on for Mani. I'm sorry, I couldn't hear, and apologies if it was asked earlier. I heard the question, but we couldn't hear the answer. This was regarding the growth expected for LIFO in terms of weight based increases as the patient get older. Speaker 500:17:35And to add to that, if you could comment in terms of how should we look how should we think about the way and then the dosage for new patients versus existing ones? Speaker 200:17:50Yes. So the question and just to make sure that I recap it here and ask for any color from Peter. Basically, the original question was around, do we see adjustments to dose as patients are on therapy over time, per the label. And we do see that over time. I think, Lutz mentioned that there's been a recent poster presentation on it. Speaker 200:18:16So that is a dynamic that we see out with the treating physicians. And so were you able to the microphone pick us up this time? Speaker 500:18:34Yes, we could hear you really clearly. Thank you. And as a follow-up regarding the Chinnabales Shonam assets, could you maybe comment in terms of what the international growth opportunities are for those assets? Speaker 200:18:50Yes. So question about Kenadol Coal Bomb Growth Opportunities. And I'll let Peter speak a little bit to the dynamic of what we have today and then also a little bit about the Kenadol label expectations expansion opportunity for CTX. Speaker 100:19:06Yes. With both products, we continue to see growth in line with historical trends, certainly with KEYNOTE Speaker 700:19:14and recently announced Phase 3 data for CTS and potential approval next year for CTS. I think there's an opportunity key adults never been promoted for CTS before. So I think there's an opportunity to find more patients through disease state awareness and hopefully increasing the diagnosis rate. One thing we know about CTS is maybe only about 10% of the patients are diagnosed and under management. So the opportunity there is Speaker 100:19:42to try to identify more patients and Speaker 700:19:45to them on therapy. Operator00:19:52Thank you. Thank you. Speaker 200:19:55Thanks for the questions. Operator00:19:58The next question comes from the line of Mike Coles with Morgan Stanley. Please proceed. Hi. Speaker 900:20:06This is Rohit on for Mike. Thanks for taking our questions. Can you just talk about your expectations for the upcoming LIV MARLY, PDUFA in PFIC and any launch prep that's associated? And do you think that any patients are currently using it off label? Thank you. Speaker 200:20:23Thanks for the question, Rohit. On the regulatory front, we're kind of where we expect to be, so excited about PDUFA date coming up. And I'll let Peter speak a little bit to how we're preparing for that and some of the first opportunities we see there. Speaker 100:20:38Yes. We're really excited about the potential approval for LiMARLE and PFIC. Speaker 700:20:43I think the Mars Speaker 100:20:44data reinforce the strong value proposition that LiMARLE Speaker 700:20:46offers to our various stakeholder groups. Physician prescribing universe is essentially identical to the physician prescribing universe is essentially identical to the AlloSure audience. So there's really no need for meaningful operating expense increased access. Speaker 100:21:06And we do have Speaker 700:21:07a number of patients in the mid-20s who are receiving clinical rollover or extended access, the barley, who would be eligible to roll over the commercial upon approval. Speaker 900:21:26Thank you. Speaker 200:21:28Thanks for the question. Operator00:21:31The next question comes from the line of Steve Seedhouse with Raymond James. Please proceed. Speaker 1000:21:38Yes. Hi. This is Timur Ivannikov on for Steve Seedhouse. So our first question is related to the gross margin. I think you mentioned there was an inventory charge related to the acquisition in 4Q. Speaker 1000:21:51Just what are your expectations for gross margin in 2024 and perhaps also the OpEx trajectory in 2024? Speaker 400:22:04For us, gross margin, we do expect that the intangible amortization will continue. I mean, it's largely related to the bile acid acquisition intangibles, which we're amortizing over 50 quarters. As far as sort of traditional cost of goods, it consists of the actual cost of products and royalties. So that will continue at approximately the same sort of percentage of sales. And then we did have a larger than we expect going forward amortization or reserve for the inventory that came with the acquisition. Speaker 400:22:45So we might have some reserve in some quarters, but not to the extent we had in the Q4. In terms of overall operating expenses, R and D and SG and A, 4th quarter is probably pretty representative of what we expect the next few quarters. Speaker 1000:23:09Okay. Thank you very much. And then just a question on your overall enrollment dynamics in VISTUS Advantage. I think in VISTUS, your interim is only on 45 patients. So can you comment about the overall enrollment target dates? Speaker 1000:23:26And do you see any differences between the studies in terms of the enrollment dynamics? Speaker 200:23:34Thanks for the question. I mean, the simple answer here is we have patients in for the interim. We're continuing to enroll for Part 2 now. And we'll be able to give better guidance for the full study enrollment at that interim when it comes up. Speaker 1000:23:54Okay. Appreciate it. Thank you very much. Speaker 200:23:57Yes. Thank you. Operator00:24:00The next question comes from the line of David Lebowitz with Citi. Please proceed. Speaker 1100:24:06Hi, this is Divanjana on for David. Thanks for taking our call. We wanted to ask about the blinded PSC interim analysis. So beside the dose selection, would you share any other data points at a blinded basis? Speaker 300:24:24So we will be blinded in this interim. And this is designed so that if it passes the threshold, it is designed to be predictive of a clinic meaningful and statistically significant positive pivotal analysis in the end. So that's all I can really share with you at this point. And it's really the threshold was developed based on what we know about the drug, this class of drugs, PSC in general and pruritus. So we feel pretty confident in terms of the design being robust and this being kind of well set up to produce a registration study for us. Speaker 200:25:03And in terms of communication at the point of interim for the VISTA study, assuming a positive interim, we would communicate that the interim has occurred and study continues as planned, which would be the extent of the information that we receive here on the team as well. Speaker 1100:25:19Okay. So as a follow-up to that, could you tell us a bit more about I mean, while unlikely, is there any other scenario like that would play out if somehow like if you see that the efficacy is not crossing the threshold on a blind blinded basis that was pre specified? Speaker 200:25:40So the study design in the event where that threshold is not met basically converts to an open interim analysis. We'd be able to look at the data, share top line findings of it and what the next steps are for the study in that scenario. Speaker 1100:26:00Okay. Thank you for the insight. Speaker 200:26:04Yes. Thanks for the questions. Operator00:26:06Thank you. The next question comes from the line of Brian Skorney with Baird. Please proceed. Speaker 900:26:16Hi, Ann. On PBC beyond pruritus, how are you thinking about the impact on outcomes and other markers in Vantage? And how can this help you make the case to physicians and patients? Speaker 200:26:31Yes. Thanks for the question. I'll let Joanne speak a little bit to kind of the registrational plan for PBC. Speaker 300:26:38Yes. So in terms of the interim, we're just going to be looking at top line. So, pruritus, serum bile acid safety, as top line. And as you know, age is a big issue in this particular patient population for which there's really no particular therapies at this point. So we're pretty confident that this study, the way it's designed actually addresses an important unmet medical need at this point. Speaker 200:27:03Yes. And just to recap, one of the points made earlier, the eligibility criteria for the VANTAGE study does not have an alkaline phosphatase criteria. So it includes patients that are traditionally thought of as first line that have really only UDCA as on label and still experience the same rates and severity of pruritus as later lines of treatment. So a lot of unmet need across the PBC patient population. Speaker 700:27:38Great. Thanks. Speaker 200:27:41Thanks for the question. Operator00:27:42Thank you. The next question comes from the line of Ed Arce with H. C. Wainwright. Please proceed. Speaker 1200:27:51Hi, everyone. Thanks for taking my questions and congrats on another strong quarter. I have 3, if I may. Firstly, on the VANTAGE study in PBC, as you said, it's a blinded data readout. Obviously, this is, as you mentioned, basically pruritus versus placebo and whether it's stat sig. Speaker 1200:28:15But I'm also wondering if there are specific thresholds of activity on pruritus that you're looking at, not just so much to proceed, but also as you think about the competitive landscape and where you'd like to see that come in? Secondly, if you could speak a bit about the PFIC opportunity in terms of 2024, sort of the sales, the speed of the sales ramp, kind of what you're expecting the trajectory to be through the remaining quarters of this year? And then lastly, I'm sorry, I didn't hear when Peter was reviewing, I think it was Peter, the numbers for LIV Marley, both U. S. And international as well as bile acid products for the Q4. Speaker 1200:29:07Thanks so much. Speaker 200:29:11Great. Thanks for the question. Maybe I'll just hit on recap one of the points on Vantage and then pass it over Peter to talk about PFIC and recap some of those sales numbers. And the way we think about Vantage and valexovat and PVC, it really comes down to this the highlight here on lines of therapy and where various agents are labeled and expected to be labeled. The majority of patients are first line patients and the majority of them have pruritus. Speaker 200:29:43So that's really the primary unmet need that we're going after. So showing a significant impact on that pruritus, we think will be very clinically meaningful. And that's the lens that we're going to be taking towards that interim data when we look at it and make a decision on next steps and continuing to roll into the pivotal portion of the study. With that, I'll pass over to Peter on the next two points. Speaker 700:30:08Yes. Sorry, I'll try to speak up a little bit here. Speaker 100:30:10So on the PFIC 2024 revenue expectation piece, I think we'll be upon a potential approval here in March, we'll Speaker 200:30:20be spending a fair bit Speaker 100:30:22of time working closely with payers get LiDARLE positioned in their new to market policies. So I think if you think about 2024, we'd expect a lot of free drug or PAP with PFIC really coming in more in 2025, somewhat similar to what we saw in the Alagille initial launch where the early quarters were high PAP drug. And then in terms Speaker 400:30:48of the Speaker 100:30:48numbers from Q4, LIDMARLE totaled $41,400,000 worldwide, dollars 31,400,000 US10 International and then bile acid products were $28,100,000 for Q4. Speaker 1200:31:06Great. And then maybe just further clarification on PFIC. Appreciate the comments. Just wondering given that you mentioned earlier there's about 20 ish patients right now on expanded access. How soon would you expect those to be able to roll over onto commercial drug once again? Speaker 100:31:30Yes. What we saw in the Alagille launch is that occurred in a quarter or 2 generally is what we saw. And so, yes, I think that's probably consistent with our expectation for those 25 or so. Speaker 1200:31:45Thanks so Speaker 200:31:47much. Thanks for the questions. Operator00:31:49Thank you. The next question comes from the line of Jonathan Willoughby with Citizens JMP. Please proceed. Speaker 1100:31:58Hi, this Speaker 500:31:59is Catherine on for John. I just had 2 kind of quick questions. One time to kind of follow-up on valexibat and just kind of the how you guys see it fitting into the PBC paradigm in the setting of other approved agents that might potentially be effective also on pruritus, whether it's an excuse to see as kind of the combo therapy, sort of a monotherapy for earlier patients? And then also, just as far as preparing for the PFIP launch, I know you said that there's not going to be too much of an investment as far as SGA goes. But like what else is kind of being done to sort of get the word out there about PFIC or do the most of these physicians already know that it's coming? Speaker 200:32:40Thanks for the questions. And just to kind of recap the PBC positioning one more time here. The VANTAGE study includes first line patients that really are when you look at the eligibility criteria for the PPAR programs, for example, and how basafibrates gets used outside the U. S, we are upstream of that setting where when a patient is biochemically controlled, they can still have substantial pruritus, right? That's the area where IVAC can play a very unique role in being in front of the other second line therapies, the PPARs and OCA, for example. Speaker 200:33:24So pretty unique positioning that we see for PBC. I'd add on, in PSC, really exciting opportunity here where valexovat is positioned to be the first and only therapy for PSC by using pruritus as an endpoint really gives an outcome to use for bringing a new therapy to these patients. So quite excited about the positioning there. And pass over to Peter for part 2 here. Speaker 100:33:55Yes. In regards to BPIC, obviously, there's been there's no promotion until an approval. But I mean, I just kind of emphasize that it's a really small number of physicians that take care of these patients in the U. S. I mean, you're probably talking about on the order of 100 physicians in the entire country that take care of PFIC patients. Speaker 100:34:15There have been presentations at the major medical conferences on the data, Phase Speaker 700:34:203 data as well as Speaker 100:34:21the Phase 2 data going back for years. So I think there's a kind of a relatively high awareness of the data. And certainly, from the Alagille approval and commercialization, a lot of comfort with Marley's clinical profile, kind of familiarity with using it, prescribing it, working with our patient support hub, the Miramax's Plus program, co pay support, etcetera. So I think that's probably a fair summary of where things stand today. Speaker 500:34:52Thanks so much. Speaker 200:34:55Thanks for the questions. Operator00:34:58Thank you. There are no additional questions at this time. I will pass it back to Chris Peetz for any closing remarks. Speaker 200:35:07Great. Thanks again for everyone for joining us for today. During the call, I did want to put a plug in for Rare Disease Day tomorrow. Today, to build awareness for the more than 7,000 rare diseases impacting patients around the world and celebrate some of the meaningful advances in research that we've seen in recent years. Tomorrow, as part of Rare Disease Day, we're excited to be able to share a manuscript published in hepatology featuring long term data and patients with Alagille syndrome treated with Leimarley. Speaker 200:35:36So please join us in supporting the many advocacy groups that passionately lead efforts to support patients in research in rare disease tomorrow. And have a great evening. Goodbye. Operator00:35:49That concludes today's conference call. Thank you. You may now disconnect your line.Read morePowered by