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spk13: Good afternoon, ladies and gentlemen. Thank you for joining today's Merrim Pharmaceuticals Reports Q4 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. If you would like to ask a question, please press star one on your telephone keypad. It is my pleasure to pass the call over to Andrew McKibbin, Vice President of Investor Relations and Finance. Please proceed.
spk07: Thanks, Tia, and good afternoon, everyone. I'd like to welcome you to Miriam Pharmaceutical's fourth quarter 2023 conference call. I'm joined today by our CEO, Chris Peets, our President and Chief Operating Officer, Peter Radovich, our Chief Scientific Officer and Head of Research, Pam Bigg, our Chief Medical Officer, Joanne Kwan, and Eric Bierkult, our Chief Financial Officer. Earlier today, Miriam issued a news release announcing the company's results for the fourth quarter and full year 2023. Copies of this news release and SEC filings can be found in the Investors section of our website. Before we begin, I'd like to remind you that during the course of this call, we will be making certain forward-looking statements about Miriam 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. Miriam 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 10Q 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.
spk06: Chris? Thanks, 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 Mirim 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. and internationally, achieving $179 million in net product sales and 142% year-over-year growth in total revenue. This was driven by continued strong growth for Live Marley and the expansion of our commercial portfolio with the acquisition and integration of Keenadal and Colbon. 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 to $320 million of net product revenue in 2024. This is expected to be driven by growth across all three commercially available medicines. We also have multiple regulatory and clinical catalysts this year to advance our pipeline. for Livmarley and PFIC. We are on track for our PDUFA date on March 13th. We're excited for the opportunity to bring the strong results of the March PFIC phase three study to patients with a potential label expansion. We're also preparing for the submission of the positive restore phase three results of Kinadol and CTX in the first half of the year, an important label enabling opportunity. And we've also made great progress with the VISTAs and Vantage studies for Velixibat and PSC and PDC. Patients for the interim analyses have been enrolled, and we expect to conduct the dose selection interims of these adaptive studies in the first half of the year. We see the PFC 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. Now, before I turn the call over to Peter to discuss our commercial business, I wanted to welcome our new chief medical officer, Dr. Joanne Kwan, who joined Miriam 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 Miriam. 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. Peter?
spk09: Thanks, Chris.
spk07: We are excited by the tremendous progress that 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 patient and prescriber community. I'm proud of the MIRIN team's focus on patients, while simultaneously driving the seamless integration of Colbon and Penadol. And this is evident in the 69.6 million of total net product sales we achieved in the fourth quarter of 2023, which reflected growth across all three commercial medicines. For Livmarlie, total global net product sales grew to 41.4 million in the fourth quarter. Our U.S. business saw 31.4 million and 111 million for the quarter and year respectively. representing approximately 63% year-over-year growth. And internationally, we are seeing consistent strong uptake as well, but we continue to anticipate quarter-to-quarter variability in international revenue. Moving on to Kingdall and Holbaum, the fourth quarter was the first 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 fourth quarter, we recognized net product sales of 28.1 million, the highest quarter to date for the bile acid products. And looking ahead, we expect steady growth for these two medicines in line with historical trends. Taking all the strong dynamics in the business together, we expect net product sales to be 310 to 320 million 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 our team as we expanded our portfolio to three commercial products and grew our presence both in the U.S. and worldwide. We 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?
spk03: Thanks, Peter. Before 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 are tracking well towards our PFIC-DUFA date of March 13th and expect a decision from the EMA in the first half of the year. Regarding Ketadol and CTX, we're making good progress here as well. We'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 2024. Turning to the looks of that, we're also looking forward to our upcoming interim analyses in our VISTA's 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 VISTA's PSC study will have a blinded interim analysis to support dose selection. Based upon 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 the 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 cholestasis, 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 their progress with you this year. With that, I'll now turn the call over to Eric to discuss our financial results. Eric?
spk14: Thanks, John. Earlier today, we issued a press release that included financial results for the quarter and full year, which I'll briefly summarize. Note, our 10-K filing is planned for next week as our auditors finalize the first 404B and other audit procedures from ERIN. Total revenue in the fourth quarter 2023 was $69.6 million, and for the year ended December 31st, total revenue was $186.4 million, including total net product sales of $178.9 million. This is compared to total revenues of $27.9 million and $77.1 million for the fourth quarter and full year 2022 respectively. For operating expenses for the year ended December 31st were 293 million, which includes research and development expenses of 102.6 million, SG&A expenses of 145.9 million, and cost of sales of 44.5 million. For the year ended December 31st, 2023 net loss was $160.9 million or $3.94 per share. Net loss for the year included non-cash stock-based compensation expense of $35 million and intangible amortization of $10.4 million. This intangible amortization is largely reflected in our cost of goods sold. In the fourth quarter, COGS also reflected a reserve in excess of $5 million for inventory, primarily related to the bile acid acquisition. So the year ended December 31, 23, our cash used in operating activities was $70.9 million, down from $120.1 million the year before. We have cash, cash equivalents, and investments of $286.3 million as of December 31st, 2023. We remain well-funded with a strong and self-sustaining business. Our 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.
spk06: Thanks, Eric. It has been a strong year for Miriam, finishing with the biggest ever revenue quarter for Liv Marley and the BioLoss portfolio. We are poised for another great year in 2024 with three 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.
spk13: We will now begin the QA session. If you would like to ask a question, please press star followed by 1 on your touch-tone keypad. If for any reason you would like to remove that question, please press star followed by 2. Again, to ask a question, press star 1. As a reminder, if you are using a speakerphone, please remember to pick up your handset before asking your question. We will pause here briefly to allow questions to generate in queue. The first question comes from the line of Jessica Pfei with Jeff P. Morgan. Please proceed.
spk08: Hey, this 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 to 320 million, can you maybe discuss a bit more about how much of Marley growth is baked in there versus contributions from Kenadon Colbaum? And also, comment a little bit on the Marlene and maybe provide an update on what you're seeing on the competitive dynamics with Build With May, if any.
spk06: Yeah, thanks for the question. And 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.
spk07: Yeah, and I think if you think about the components, some of the color commentary there, you know, the bile acid product portfolio, we expect it to grow in line with historical trends, which have been kind of on the order of bits per 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, you know, over all the quarters of its marketing, We do expect quarter-to-quarter variability there for the LeVar Lake International sales. And then your other question about competitive dynamics and knowledge yield, and I think as you can see from the numbers here, it's really, we haven't seen a measurable impact from those at all, really. The LeVar Lake Road trajectory hasn't been impacted by that.
spk08: Great. 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?
spk06: Yeah, Joanne and I can speak to the differences between the lines of therapy.
spk03: Yeah, so, you know, as you know, pruritus is a big issue in both of these diseases. You know, we have a broad patient population that we're accessing in terms of the BOLIXBAP studies. And actually, 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 agreed from FDA that pruritus is a registration endpoint, and both of these studies are actually positioned to be registration after the interim analysis. So we feel pretty confident in terms of both the study design, the drug itself, our dose selection, and moving forward at this point.
spk08: Great. Thanks.
spk13: Thank you. The next question comes from the line of Gavin Clark-Gardner with Infracore ISI. Please proceed.
spk04: Hi, this is Yesha. I'm here, Gavin. Just two questions for us. For the first one, for Liv 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?
spk07: I'm happy to answer the question. We have published some of that information last year. You know, not surprisingly, it's a weight-based product. And the growth data that we've seen published with Marley, we're happy to see weight-based adjustments.
spk04: Awesome. And then one more. For the EU business, do you have any commentary on potential implications of Ipsen refiling Odovixiba in the EU under a different brand name? not with orphan status, and then also want to confirm that there would be no reimbursement or access impact on Livmarli in the EU?
spk06: Thanks for the question. At this point, we're focused on getting access broadly across all international markets for Livmarli and Algiel, and having great progress as we see demands continuing to grow across Europe and other international markets. Can't really speak too much to the bill of a procedure, other than that we're making great progress getting with Marley. It really rolled out a standard of care analogy across all markets.
spk13: Great, thank you.
spk01: Thanks for the question.
spk13: Thank you. The next question comes from the line of Mani Barura with Lurink Partners. Please proceed.
spk05: Hi, good afternoon. This is Lillian Songo on Harmony. 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 lymphoma in terms of weight-based increase as the patient gets older. And to add to that, if you could comment in terms of how should we look or should we think about the weight and then the dosage for a new patient versus existing ones?
spk06: Yeah, 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 it was mentioned that there's been a recent poster presentation on it So that is a dynamic that we see out with the treating physicians. And so we're able to, did the microphone pick us up this time?
spk05: Yeah, we could hear you really clearly, thank you. And as a follow-up regarding the Kinabalu showdown assets, Would you maybe comment in terms of what the international growth opportunities are for those assets?
spk06: Yeah, so a question about Keen et al. 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 Keen et al. label expectations of expansion opportunity for CTX.
spk07: Yeah, I think with both products, we continue to see growth in line with historical trends, certainly with Ketadol and the recently announced Phase 3 data for CTFs and potential approval next year for CTFs. I think there's an opportunity. Ketadol has never been promoted for CTFs before, so I think there's an opportunity to find more patients through disease-safe 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 to try to identify more patients and get them on therapy.
spk05: Thank you.
spk13: Thank you.
spk05: Thanks for the questions.
spk13: The next question comes from the line of Mike Colt with Morgan Stanley. Please proceed.
spk09: Hi, this is Rohit on for Mike. Thanks for taking our questions. Can you just talk about your expectations for the upcoming Live Marley PDUFA and PFIC and any launch prep that's associated? And do you think that any patients are currently using it off-label? Thank you.
spk06: Thanks for the question, Rohit. On the regulatory front, we're where we expect to be, so excited about the 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.
spk07: Yeah, we're really excited about the potential approval for the Marley and PPIC. I think the Mars data reinforced the strong value proposition that Marley offers. various stakeholder groups, and I think that will certainly help us. The physician prescribing universe is essentially identical to the allergy audience, so there's really no need for meaningful operating expense increase to access it. And we do have a number of patients in the mid-20s who are receiving clinical rollover or extended access to Marley who would be eligible to roll
spk09: Thank you.
spk01: Next question.
spk13: The next question comes from the line of Steve Seathouse with Raymond James. Please proceed.
spk02: Yes. Hi. This is Timur Ivanikov on for Steve Seathouse. 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. just what are your expectations for gross margin in 2024 and perhaps also the OPEX trajectory in 2024?
spk14: For gross margin, we do expect that the intangible amortization will continue. I mean, it's largely related to the bile acid acquisition intangibles, which were amortizing over 50 quarters. As far as sort of traditional cost of goods, it consists of the actual cost of product and royalty. So that will continue 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. We might have some reserve in some quarters, but not to the extent we had in the fourth quarter. In terms of overall operating expenses, R&D and SG&A, fourth quarter is probably pretty representative of what we expect the next few quarters.
spk02: Okay, thank you very much. And then just a question on your overall enrollment dynamics in this disadvantage. I think in VISTAs, your interim is only on 45 patients. So can you comment about the overall enrollment target dates? And do you see any differences between the studies in terms of the enrollment dynamics?
spk06: Thanks for the question. I mean, the simple answer here is we have patients in for the interim. We're continuing to enroll for part two now. and we'll be able to give better guidance for the full study enrollment at that interim when it comes up.
spk02: Okay, I appreciate it. Thank you very much. Yep, thank you.
spk13: The next question comes from the line of David Libeliz with Citi. Please proceed.
spk12: Hi, this is Devajana on for David. Thanks for taking our call. We wanted to ask about the blinded PSE interim analysis. So besides the dose selection, would you share any other data points on the blinded basis?
spk03: So we will be blinded in this interim. And, you know, it's designed so that if it passes the threshold, It is designed to be predictive of a clinically 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, you know, this 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.
spk06: In terms of communication at the point of the 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.
spk12: Okay. 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 the Somehow, like, you know, if you see that, like, you know, the efficacy is not crossing the threshold on a blinded basis, that would be specified?
spk06: So 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, you know, what the next steps are for the study in that scenario.
spk12: Okay, thank you for the insight.
spk06: Yep, thanks for the questions.
spk13: Thank you. The next question comes from the line of Brian Scorney with Baird. Please proceed.
spk15: Brian, on PDC 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?
spk06: Yeah, and thanks for the question. I'll let Joanne speak a little bit to kind of the registrational plan for PBC.
spk03: Yeah, so in terms of the interim, we're just going to be looking at top line. So, pruritus, serum valence, and safety as top line. And as you know, you know, it's just a big issue in this particular patient population for which there's really no particular good therapies at this point. So we're pretty confident that this study, the way it's designed, actually addresses an important medical need at this point.
spk06: Yeah, 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.
spk15: Great. Thanks.
spk03: Thanks for the question.
spk13: Thank you. The next question comes from the line of Ed Arth with AC Ringright. Please proceed.
spk10: Hi, everyone. Thanks for taking my questions, and congrats on another strong quarter. I have three, 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 puritis versus placebo and whether it's stat-sig. But I'm also wondering if there are specific thresholds of activity on Puritas 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 Live Marley, both US and international, as well as the bile acid products for the fourth quarter. Thanks so much.
spk06: Great. Thanks for the question. Maybe I'll just hit on, recap one of the points on Vantage and then pass it over to Peter to talk about PFIC and recap some of those sales numbers. And, you know, the way we think about Vantage and Velixibat and PBC, it really comes down to this, you know, 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. So 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 it over to Peter on the next two points.
spk07: Sorry, I'll try to speak up a little bit here. So on the PFIC 2024 revenue expectation piece, I think we'll be, upon a potential approval here in March, we'll be spending a fair bit of time working closely with payers to get Liv Marley positioned in their new-to-market policies. So I think if you think about 2024, we'd expect a lot of free drugs or PAPs with PFIC really coming in more in 2025, somewhat similar to what we saw in the algeol initial launch where the early quarters were high PAP drug. And then in terms of the numbers from Q4, with Marley total 41.4 million worldwide, 31.4 US, 10 international, and then bile acid products were 28.1 million for Q4.
spk10: Great. 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 drugs once again?
spk07: Yeah, what we saw in the AllerGeo launch is that occurred in a quarter or two, generally, is what we saw. And so, yeah, I think that's probably consistent with our expectation for those 25 or so. Thanks so much.
spk10: Thanks for the questions.
spk13: Thank you. The next question comes from the line of Jonathan Willoughby with Citizens JMP. Please proceed.
spk11: Hi, this is Catherine on for John. I have two kind of quick questions. One kind of follow-up on Velexibat and just kind of 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 you see it as kind of the combo therapy, sort of a monotherapy for earlier patients. And then also, just as far as preparing for the PFIC launch, I know you said that there's not going to be too much of an investment as far as SGA goes, but what else is kind of being done to sort of get the word out there about the PFIC, or do most of these physicians already know that it's coming?
spk06: Thanks for the questions. And just to kind of recap the PPC positioning one more time here, the Vantage study is includes first-line patients that really are, when you look at the eligibility criteria for the PPAR programs, for example, and how basal fibrates get used outside the U.S., we are upstream of that setting where when a patient is biochemically controlled, they can still have substantial pruritus. That's the area where IVAC can play a very unique role in being in front of the other second-line therapies, PPARs and OCA, for example. So pretty unique positioning that we see for PBC. I'd add on, you know, in PSC, really exciting opportunity here where Velixibed is positioned to be, you know, the first and only therapy for PSC. by using puritis 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 it over to Peter for part two here.
spk07: Yeah, in regards to BPIC, obviously there's been, you know, 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. There have been presentations at the major medical conferences on the data, phase three data, as well as the phase two data going back for years. So I think there's a kind of a relatively high awareness of the data. And certainly from the AllerGeo 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 Plus program, you know, co-pay support, et cetera. So I think that's probably a fair summary of where things stand today.
spk11: Thanks so much. Thanks for the questions.
spk13: Thank you. There are no additional questions at this time. I will pass it back to Chris Peets for any closing remarks.
spk06: Great. Thanks again for everyone for joining us for today. Before we end the call, I do want to put a plug in for Rare Disease Day tomorrow. It's a day 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 Allergy Syndrome treated with Livmarly. So 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.
spk13: That concludes today's conference call. Thank you. You may now disconnect your line.
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