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Operator
Good afternoon. I would like to welcome you all to the Miriam Pharmacuticals conference call. My name is Brica and I will be the event specialist running today's call with you. All lines are on mute for the presentation portion of the call today with an opportunity for questions and answers at the end. If you would like to ask a question, please press star then one on your telephone keypad. I would now like to pass the conference over to your host, Andrew McKibbin. Vice President of Investor Relations, to begin. So, Andrew, you may begin.
Brica
Thanks, Priyanka, and good afternoon, everyone. I'd like to welcome you to Miriam Pharmaceuticals' third quarter 2023 conference call. I'm joined today by our President and CEO, Chris Pease, our Chief Operating Officer, Peter Radovich, our Head of Research and Development, Pam Begg, and our newest team member, Eric Bjerkel, our Chief Financial Officer. Earlier today, Miriam issued a news release announcing the company's results for the third quarter 2023. Copies of this news release and SEC filings can be found in the investor section of our website. Full details and updates from the quarter can be found in our news release and 10Q issued today. Before we begin, I'd like to remind you that during the course of this conference call, we will be making certain forward-looking statements about Miriam and our programs based on management's current expectations, including statements regarding Merrim's current and future business plans, development programs, and regulatory expectations, strategies, prospects, market opportunities, and financial expectations. Merrim is under no duty yet to update these statements, and they are subject to numerous risks and uncertainties, and actual results could differ materially from the results anticipated by these statements. Investors should read the risk factors set forth in Merrim's 10K for the year ended December 31st, 2022, and any subsequent reports filed with the SEC. With that said, I'd like to turn the call over to Chris. Chris?
Priyanka
Thanks, Andrew, and good afternoon to everyone. This was another strong quarter of execution at Merrim. The team has continued to grow our business by focusing on delivering life-changing medicines for patients and their families living with rare diseases. We've achieved multiple goals this quarter, including realizing total revenue of $47.7 million for the quarter. We saw continued strong global adoption of Lipmarli. We also completed the acquisition of two high-impact and synergistic commercial medicines, Kinadol and Colbon. We continued our commitment to delivering on our pipeline with the recent positive Restore Phase 3 study readout and upcoming clinical and regulatory milestones. And all of this has been accomplished while improving our financial position in terms of performance and balance sheets. These achievements come alongside another special milestone for Mirim coming up this weekend, our five-year anniversary of launching the company. I'd like to thank the Mirim team's dedication, perseverance, and unmatched spirit for making all of our success possible in a short time, with much more to come. Now, moving to Live Marley, we see steady demand growth in the U.S. and internationally. This reflects the positive impact of Live Marley for patients and families in the Allershield Syndrome community, and also the focused execution of the Mirim team to make this important medicine accessible to patients around the world. I am proud of what we've been able to accomplish with Livmarley and Al-Jil syndrome and what that means for our execution on our expanded portfolio. This quarter, we grew our business with the acquisition of two commercial medicines, Kinadol and Colbomb, and have assumed sponsorship of a broadly used cholestasis genetic testing panel, vital for diagnosing several rare genetic disorders that is free to patients and providers. This has created a leading pediatric hepatology franchise and also brings significant and growing revenue contribution with approximately $100 million in annual net product sales from the bile acid portfolio. Regarding our pipeline, we have made good progress here as well. In October, we announced positive data from the Phase III Restore Study, evaluating Kinadol in patients with cerebral tendinous xanthomatosis, or CTX. The data showed Kenadol has a significant impact across key measures of the disease and this phase three data set and the potential approval of Kenadol for CTX is a critical step forward for CTX patients. We plan to submit a new drug application for CTX to the FDA in the first half of 2024. We also look forward to significant upcoming milestones and opportunities for Lyfmarly with the March 13 PDUFA date for PFIC and our upcoming phase two data and biliary atresia. And for Elixabed, we expect to conduct our interim analyses in PSC and PBC in the first half of next year. Now, before I turn the call over to Peter to discuss our commercial business, I want to welcome our new CFO, Eric Bjerkel, who joined Mirum in September. We are thrilled to have Eric and his extensive leadership experience helping to drive the growth and value creation we see ahead for Mirum. We'll hear from him later in the call. And with that, I'll turn the call over to Peter. Peter?
Andrew
Thanks, Chris. With total revenue reaching 47.7 million in Q3, we are excited by the tremendous progress that we have made across our U.S. and international businesses. Our team continues to bring Live Marley to more patients worldwide, and we have taken significant steps in establishing a leading pediatric hepatology franchise through the ongoing execution of our commercial strategy with Live Marley and the addition of pinadol and colbon. Starting with Live Marley, In the third quarter, our revenues grew to $39 million in total net product sales, which represents over 100% growth before the addition of the bile acid portfolio. This reflects the life-changing impact that Marley is having in algeo patients with pruritus and the excellent execution of our commercial team. The U.S. business, which saw $28.6 million this quarter, continues to grow. Internationally, we see similar strength in patient uptake in Germany and France, as well as distributor markets. Going forward, we continue to anticipate quarter-to-quarter variability in international revenue. Overall, we are looking forward to our continued expansion in both the U.S. and globally, and are excited for the opportunity to broaden access to Livmarly to patients with Allergy L-Syndrome and future indications. Moving to Keendal and Holbaum, The addition of these medicines presents a significant opportunity to both augment live Marley's growth and significantly enhance our relationships with the patient communities and prescribers and pediatric. There are striking similarities between the commercial models for these medicines. So, in the hands of one team, we expect top and bottom line synergies in terms of our commercial strategy. Our pediatric hepatology team has been extremely effective, so we don't plan to change a model that is working well. But we do see some opportunity to augment our efforts with the expertise that we have brought in from Trevere. Pediatric hepatologists are the main call point for Livmarley and Colbaum, and we will continue to deepen our relationships with these prescribers through our expanded offering of high-impact medicines and our branded cholestasis genetic testing panels. which is an important diagnostic tool that positions Miriam as a partner from the start of the patient journey. While Polbaum and Keen et al have grown steadily over the last several years, we see opportunity to improve diagnosis and increase treatment across these indications. In CTX, for example, the consequences of the disease first manifest in organs other than the liver, such as juvenile bilateral cataracts, and may not be linked to CTX until much later in adulthood, usually after irreversible neurological damage has occurred. To address this gap in diagnosis and treatment, we've increased investment in disease awareness and diagnosis among specialties where this disease often first presents, such as ophthalmology, neurology, and medical genetics. This expanded team will allow us to reach more patients earlier in their journey and is a key step as we prepare for a potential approval and launch in CTX. In summary, we are thrilled by the addition of the new products and team members and look to build on the tremendous momentum we've achieved delivering important medicines to even more patients with rare liver diseases. On that note, I'll turn the call over to Pam. Pam?
Chris
Thanks, Peter. Last month, our team announced positive data from the Phase III Restore Study evaluating Kenadol in CTX. The study objective was to evaluate the safety and efficacy of Kinadol by measurement of urine bile alcohols and other biomarker measures, including cholesterol. The study showed a marked and statistically significant improvement across the primary and all key secondary endpoints, demonstrating a broad and consistent effect with Kinadol in patients with CTX. As a reminder, disease progression in CTX is due to the accumulation of cholesterol in the tissue and brain. And we were thrilled to see the depth of improvement across all measures. In particular, the cholesterol results are a critical finding. Prior to this, it was not certain that treatment with Kinadol could impact cholesterol levels this quickly. And the results of the RESTORE trial highlight the importance to diagnose and treat CTX patients as early as possible. Additionally, a greater proportion of patients receiving placebo required rescue therapy, demonstrating the immediate impact of Kinadol in CTX. And while Kinadol is currently standard of care in CTX, and recognized as a medical necessity by the FDA, we're very pleased that the results confirm the life-changing impact Kinadol can have for these patients. We're grateful to the work that your team has put into the development and execution of this study, including selection of the endpoints, which were developed in close collaboration with the FDA. And with these strong results, we believe we are well positioned for our NDA filing, which we expect in the first half of next year. Moving on to clinical milestones, we're looking forward to reporting top-line data later this year from our Phase IIb Embark study of Lipmarli for pediatric patients with biliary atresia, which will be the first placebo-controlled data with an IBAHT inhibitor in this setting. For Velixibat in PFC and PBC, we expect to report on the interim analyses in the first half of next year. We continue to push on screening activities and are looking forward to sharing the interim data. Lastly, I'm proud of the growing body of important clinical data and real-world evidence validating the role of Lube-Marley for patients with Allergy Health Syndrome and PFIC. The body of research we presented at the NASPGAN meeting recently, including data from our March PFIC Phase III study and real-world experience in Allergy Health Syndrome speaks to the tremendous effects across the clinical and real-world settings. In summary, Mirren's leadership position in pediatric hepatology continues to build. And we remain committed to growing our presence across these indications. We also look forward to the multiple upcoming data readouts across settings where we believe IVAT inhibition can play a key role in changing the treatment paradigm for patients living with biliary atresia, PSC, and PPC. And with that, I'll now turn the call over to Eric to discuss our financial results. Eric?
Peter
Thanks, Pam. Before I begin, I'd like to mention how pleased I am to have joined the MIRAM team at such an exciting time for the company, and I look forward to helping advance new medicines for patients with rare diseases. Now for the third quarter financial results. Earlier today, we issued a press release that included financial results for the quarter, which I'll briefly summarize. Full details can be found in the Form 10-Q also filed today. As highlighted by Chris and Peter, we recognize $47.7 million of net product sales in the third quarter, a 47% increase over net sales the previous quarter. This reflects product sales of $38.7 million for the Marley, as well as $9 million for Kinadol and Colban. I'd like to point out that for the bile acid products, we only recognize product sales since the date of the transaction closed on August 31st, but we'll see the full quarterly contribution of these medicines in the fourth quarter of this year. Our total operating expenses for the quarter were $72.9 million, which includes R&D expenses of $26.1 million, SG&A expenses of $36.5 million, and cost of sales of $10.2 million. For the quarter ended September 30, net loss was $23.6 million, or $0.57 a share. Net loss for the quarter included non-cash stock-based compensation expense of $8.4 million and intangible amortization of $2.6 million. At the close of the third quarter and at September 30, 2023, we had cash, cash equivalents, and investments of $306 million. In summary, we're well-funded with a growing revenue base, which places us in an exceptional position to execute on our business plan, including execution of our clinical trials 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.
Priyanka
Thanks, Eric. As you can tell, Merum has continued to execute on all fronts. We've built the leading franchise in pediatric hepatology, with three standard of care medicines and the leading genetic testing tool. We are seeing strong sales growth across all of our medicines. We've generated important phase three data to support the filing for approval of Keen et al and CTX. We have several clinical and regulatory catalysts ahead as we strive to make continued advances for rare disease patients around the world. And we've accomplished all of this while putting Merum in a strong financial position. We look forward to keeping you updated on our exciting progress as we head into 2024. And with that, operator, please open the call for questions.
Operator
Thank you. If you would like to ask a question, please press star then 1 on your telephone keypad. We have the first question from Josh Schimmer of Kantor. Your line is now open.
Josh Schimmer
Thanks very much for taking the question, and congrats on another exceptional quarter. I just wanted to ask, because Ipsen on their call indicated that they had seen some switching of patients from Liv Merle to Bilvay for allogeal syndrome. Curious as to what you're actually seeing from your lens and whether you expect their launch to impede the strong growth curve you've been on for Liv Merle. Thank you.
Priyanka
Thanks, Josh, for the question. I'll hand it over to Peter to talk about the competitive dynamic.
Andrew
Thanks, Josh. Indeed, you know, LaMarley has been approved and marketed in the U.S. for over two years now, and Q3 is the first quarter where there was another product available and algeol syndrome. And I think that what we really point you to is the headline tape. The net revenue that we saw in Q3 was a really strong step up over Q2. In fact, even a little stronger than what we saw from Q1 to Q2 in terms of Liv Marley U.S. sales. So we think the story is there that Liv Marley's growth trajectory is very strong and it's not been impacted.
Josh Schimmer
Can you comment on the split between U.S. and rest of world as well? Can you comment on whether you have seen any attrition of patients to Bilvay? And if so, how many and why do you think they might have switched?
Priyanka
Well, I think on the, you know, Peter commented on the U.S. dynamic. Internationally, the regulatory situation is a bit different where we've not seen in Europe, for example, an approval of Bilvay and Algeel syndrome. And so we are continuing to grow and add patients there. It's a really strong situation for international markets, really anchored around Europe at this point.
Peter
Great. Thank you. Thanks for the questions.
Operator
Thank you. Your next question comes from Gavin Clark Gartner of Evercore ISR.
Gavin Clark Gartner
Hey, congrats on the quarter and thanks for taking my questions. So for the EMBARQ readout, when is the baseline bilirubin value taken in relation to the Casai procedure?
Chris
Yeah, thanks for the question. So the baseline bilirubin that we see in the EMBARQ study is very similar to what's observed in the natural history. Pre-Casai, it's about close to about 10, which has been shown in a few literature papers about 9.5 to 10. And post-CASI, we see it around seven. And so that really leaves a lot of room in the six months for improvement of treatment. So looking forward to sharing that data later this year.
Gavin Clark Gartner
Just to clarify, is the baseline value for the trial after the CASI when it's around the seven value?
Chris
It is. It's after the CASI. Okay. Got it.
Gavin Clark Gartner
Thanks for taking the question. Thanks, Kevin.
Operator
We now have Manish Ruha of Learing Partners. The line's open.
Peter
Hey, congrats on a strong quarter and thanks for sending the question in. I guess a broader question around sort of looking past approval of the NDA now that you have the general face to pot of data in hand. How should we think about avenues that you have available to you to accelerate growth, whether through pricing, promotional opportunities, et cetera, that perhaps weren't available when it was prior to having that data and FDA approval? And how do we think about avenues to accelerate growth in that asset and potentially expand margins and the consequence?
Andrew
Yeah, thanks for the question, Manny. Yeah, I think, you know, we anticipate FDA approval of Genadol and CTX, potential approval in 2025. And, you know, thinking from that point, you know, really the opportunity here is to increase the diagnosis rate. Once you have a diagnosed patient in a clinic, we see substantially all patients are offered Keendel already. It's really seen as a necessity. But we do know from KOLs and literature, diagnosis rates are quite low. We think only 10% of patients approximately are diagnosed. We've spent a lot of time thinking about what can we do to increase that 10%. Obviously, you don't have to do a lot to make a difference in terms of the growth of the product. So we have a team and efforts around promoting disease state awareness, genetic testing, and neurology, ophthalmology, medical genetics, to try to find more patients, essentially, and bring more patients into a diagnosis and under care.
Peter
Great. And then I guess secondarily on the pipeline, obviously a lot of focus on biliary atresia given the unmet need there. Presuming that you see a positive result on bilirubin, some perhaps directional benefit on event-rate transplant, obviously there's a power to that. Can you walk us through what the time horizon would be and what the steps are? for you to engage with regulators and then be able to perhaps provide us and investors in a public setting an update on what the filability or path to approval would be, that indication?
Priyanka
Thanks for that question, Mani. The failure attrition timelines, kind of laying them out in total, expect the top line data from the EMBARQ study by the end of the year. And we'll then take that data the way you described, you know, having a positive bilirubin analysis and supportive trends on outcomes would be a really strong outcome here that we'd be excited to take forward to FDA. We'd love to have that meeting in the first half of the year and, you know, sometime towards mid-year be able to update publicly on what our plans are on taking it forward based on FDA input.
Peter
Great. That's helpful. Is it reasonable to assume that that might be something we'd hear from you guys, like, in the first half of next year? Or is it just too early to kind of make those assumptions around, like, or how narrow that timeline might be?
Priyanka
We, you know, these types of interactions are dependent on scheduling with FDA. So, as we get closer through getting to a meeting with FDA, we'd be able to provide some clarity. For now, the best guidance we have is, you know, having an update by mid-year next year.
Peter
Great. That's helpful. I know you have other analysts waiting. I'll hop off the line. Excellent. Thanks for the question.
Operator
Thank you. Your next question comes from the line of Steve Seedhouse of Raymond James.
Steve
Thanks. Good afternoon, everyone. I wanted to ask about Embark first. Do you have a sense, based on the demographic you've enrolled, of the number of clinical events you'd be able to analyzed by the time of the top line data and also the registry work you guys are doing in parallel. Will that be available and part of the top line analysis before year end? Second, I just wanted to ask about the PBC and PSC data. It's, I guess, delayed. Maybe you'd refute that, but the and widened in terms of just the expectation on when the timing would be of those in terms. Can you just talk about some of the inputs there as well. Thanks so much.
Chris
Yeah, thanks for the question. I can answer these. So, you know, with regard to your first question on the events for bilirubin, we are seeing events coming in, and as you can expect, these patients with a highly progressive disease, but this is blinded data, and we'll be able to share top line more information with you towards the end of the year. With regard to the registry, we're working very closely with the Natural History Registry, as you alluded to, And what we're really looking at is aligning our patient population and pulling patients from that registry to really understand in this particular embarked population what bilirubin levels are really prognostic for transluntory survival. And that will hopefully, you know, will help to bolster supportive information as part of our discussion with the FDA. With regard to PSC and PBC, yeah, so we've seen, you know, some screen failures come in. Since our last earnings call, a lot of these are coming from the e-diary compliance. So patients have to complete a pruritus score daily. So we're working closely with the sites to minimize that. There's been a few patients that have screened sales for liver labs, screening liver labs, and then a couple for qualifying pruritus scores. But we remain actively screening and really excited to share data next year.
Steve
All right. Thanks so much. Thanks for the question.
Operator
We now have David Leibowitz of Citi.
David Leibowitz
Thank you very much for taking my question. Considering the EMA recently reaffirmed its decision to not give orphan disease status to Build A for ALGS, how do you believe that leads through or does not lead through to Thanks for the question, David.
Priyanka
Yeah, it's a really interesting situation watching this unfold. And I think just for a little bit of background on this, you know, the key factor is to maintain orphan designation is to have some significant benefit over available therapies. That's our understanding of what the recent outcome was with EMA. on their recent allogel syndrome decision. And we're waiting to hear back for final feedback from EMA. We're optimistic about the data set that the March PFIX study provides in that it's a broader genetic profile of patients. It starts at a younger age. It's a stronger response rate. Those are all things that we included in our submissions and some of the briefing materials on this point. And we should have an update on feedback from the agency by the end of the year.
David Leibowitz
Thanks for taking my question. Yeah, thanks for the question.
Operator
We now have Brian Smolny of EDD.
Brian Smolny
Hey, good afternoon, guys. Thanks for taking the question. My question is also on MPAR. Can you just give us a little bit of an idea of how you sort of characterize the primary, the change in bilirubin, what that means versus the secondary endpoint of getting patients below two megs per deciliter. And just in time, in terms of expectations for the placebo arm on that secondary endpoint, I think the Children Liver Disease Research Network, about 50% of infants post-CASI at three months had bilirubin less than two. Is that a reasonable assumption for the placebo rate or for that endpoint in embarking? Is there anything to consider in the design that might make that more or less? Thanks.
Chris
Yeah, thanks for the question. So maybe I'll start with your last question first. So in the natural history, you know, that shows that somewhere between 7.5 to 10 from the literature post-CASAI, and less than half of those patients will clear jaundice. So the majority of those patients will remain elevated to varying degrees. And as you alluded to, those patients that do really well and have good established bile flow, less than two milligrams per deciliter, they'll do pretty well. But the majority of them are really between two and greater than six. And so we'll also be looking at if we shift patients from high risk, meaning greater than six, to moderate risk between two and six, and maybe those from moderate risk to low risk. So we'll be looking at bilirubin in all different ways. And our primary endpoint to answer your first question is looking at the percent change difference between active and placebo at month six using an MMRM analysis. And I think that was it. Was there another question?
Operator
Thank you. We now have. Ed Ace of HC Wainwright.
Ed Ace
Hi, good afternoon, everyone. This is Thomas Yip asking a couple of questions for Ed. Thank you for taking our questions. So first, congratulations on the positive restore data set earlier this month, earlier last month, actually. So can you talk a little bit about kindergarten and day filing and CTX in the first half of next year? In addition to restore data, what do you anticipate will be called the data package? Would that be historical data or post-marketing usage data?
Priyanka
Thanks for the question. Yeah, the NDA approach we see as relatively straightforward here for Keen et al. There's been extensive back and forth with FDA on what they're expecting for the NDA and the discussion of the restore phase three study design. So that was something that we did work on and got comfortable that this was, there was good alignment with FDA that this would support an NDA for the CTX indication. And the NDA will include kind of the typical battery of additional data analyses that you'd see for a small molecule application. So nothing particularly unique about the NDA overall.
Ed Ace
Okay. Got it. And then perhaps just one more question from us. For the Maui and Algae syndrome in the U.S. market as we get close to the end of the year, what's your anticipated growth level for Algae syndrome for 2024?
Peter
We have not provided any guidance on 24 at this point, and we're still discussing internally whether we will and if so, what and when. So stay tuned on that.
Ed Ace
Okay, understood. Okay, thank you again for taking all the questions, and looking forward to an embarked data readout. Thanks, Thomas.
Operator
Thank you. We now have our final question on the line from John Wollaben of JMP Security.
John Wollaben
Hey, thanks for taking the question. Just on Kinadol and Colbalm, wondering if you could talk a little bit about the early experience with those products and then also the genetic testing. You know, what kind of leverage do you think that will give you for Marley down the road? And then how should we think about, you know, a CTX approval and its potential impact on sales?
Andrew
Thanks for the question, John. You know, it's been, you know, really exciting to bring these products and the genetic testing into Merim. You know, I kind of commented on some of the dynamics in the script. Our liver team now is, you know, focused on promoting both Liv Marley and Colbom to pediatric hepatologists, as well as, you know, kind of having a leadership role with the col-stasis genetic testing, really kind of enhances the value proposition that the Miriam team can bring to our customers. The prescribers and other healthcare professionals certainly improves access and ability to, you know, kind of having reasons to be in front of their customers. So really excited about what this is doing for Miriam's profile and, you know, supporting Liv Marley's growth. So I think that's, you know, one point. And then, yeah, as we move forward into 2024, 2025, Continue to expect the bile acid products to deliver load amidst single digit growth. Expect, you know, we think we can, you know, we're excited about the investments we can make in CTS and trying to increase the diagnosis rate there, find more patients, find them earlier when we could probably have a larger impact.
Peter
Thank you. Thanks, John.
Operator
Thank you. As a reminder, it's start one to ask any further questions. I can confirm we have no further questions. So I'd like to turn the call back over to Chris for any final remarks.
Priyanka
Great. Thank you for joining today's call. Just as a note to close out here, it has been a strong quarter for Miriam. The biggest ever quarter of revenue. Additional medicines in the portfolio, multiple clinical and regulatory catalysts ahead, and strong balance sheet. Have a great evening. Goodbye.
Operator
Thank you all for joining. I can confirm that does conclude today's call. Please have a lovely rest of your day and you may now disconnect your lines.
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