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spk02: And thank you all for joining. I would like to welcome you all to the Miriam Pharmacute Calls Report, Second Quarter 2023 Financial Results and Provides Business Update. My name is Brica and I'll be your moderator for today's call. 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 turn the call over to Andrew McGibbon, VP of Investor Relations and Finance. So, Andrew, please go ahead.
spk09: Thanks, Brika, and good afternoon, everyone. I'd like to welcome you to Miriam Pharmaceutical's second quarter 2023 conference call. I'm joined today by our President and CEO, Chris Peets, our Chief Operating Officer, Peter Radovich, and our Head of Research and Development, Pam Vick. Earlier today, Merim issued a news release announcing the company's results for the second quarter of 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. 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 Merim. Our pending acquisition of Travere's bile acid product portfolio and our program is based on management's current expectations. including statements regarding Miriam's current and future business and commercial plans, development programs, regulatory expectations, strategies, prospects, market opportunities, and financial forecasts and guidance. 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 the results anticipated by these statements. Investors should read the risk factors set forth in Miriam's filings with the SEC. With that said, I'll turn the call over to Chris. Chris?
spk08: Thank you, Andrew. And good afternoon to everyone joining us on the call today. We are thrilled to discuss yet another quarter of strong growth at Neuron. We are on track to become a leading multi-product franchise in pediatric hepatology through the impact of Live Marley for patients globally, continued progress of the Lixabat in the clinic, and the pending acquisition of Kinadol and Colbom. The last quarter saw substantial progress on our five-part strategy, which is to grow Live Marley in the U.S., launch Livmarly in international markets, establish new indications for Livmarly, advance and commercialize Elixabat and adult cholestatic indications, and acquire additional high-impact medicines for rare disease. We've made great strides on all of these areas of strategic focus so far this year. First, on growing Livmarly's commercial business, in the second quarter, We performed well against our guidance of 50% year-over-year net product sales growth in the U.S. in 2023, as well as growth from international markets, achieving $32.5 million of Live Marley revenue and $37.5 million total revenue. We are also excited about the recent progress in international markets with our approval in Canada and a growing list of countries selling Live Marley in distributor markets. Livmarly indication expansion efforts are also making great progress with our PFIC application under review with the December 13th, 2023 PDUFA date. We're looking forward to the potential label expansion based on the compelling March phase three data and bringing Livmarly to PFIC patients. In addition, the Embark biliary atresia study is fully enrolled, heading towards top line data later this year. Elixabat is also progressing nicely towards interim analyses in PSC and PBC later this year. positioning those studies to expand into their potentially registrational portions. And finally, on the fifth strategic pillar of product acquisition, we are advancing towards closing the acquisition of Kinadol and Colbomb, two commercial products with over an aggregate of $100 million of annual net sales in 2022. As we've shared recently, we expect this acquisition to deepen our leadership in pediatric hepatology and accelerate our growth across our product line. We expect the transaction to close in the third quarter and are excited about the fit of these bile acid replacement medicines with Marley and the upcoming phase three readout for Keenadol and CTX. Financially, we are well positioned to execute on our strategy. We ended the second quarter with a $330 million cash balance and Q2 was another quarter of financial growth with relatively steady operating expense levels and a growing top line compared to the first quarter. The pending addition of Kinadol and Kolbam offers an opportunity to further accelerate our revenue with a highly synergistic business. Concurrent to this pending acquisition, we entered a private placement agreement for $210 million supported by several existing and new investors, which we'll use to fund the upfront payment in connection with the acquisition. This financing allows us to maintain the strength of our balance sheet and positions us for continued growth. With that, I'll pass the call over to Peter to discuss our commercial business in more detail before Pam gives an R&D update.
spk04: Peter? Thanks, Chris. We continue to see strong demand for Live Marley, with $32.5 million in Live Marley net product sales in the second quarter of 2023, which represents 86% growth over the second quarter of 2022. Our U.S. business continues to perform well with net product sales of 26.2 million. This growth was driven by a strong underlying new patient demand. Notably, in Q2, we saw an increase in uptake by patients less than 12 months of age, consistent with the Lib Marley label expansion to three months of age and older in March. Early treatment in AllerGeal syndrome is important to limit the impact of the disease. and we're pleased to see Livmarly addressing this need in the real world setting. The US business performance continues to track very well against our guidance of 50% growth in 2023 net product sales. Turning to international markets, we continue to see strong de novo demand in Germany and France launches, in line with what we saw in the US launch of Livmarly. Pricing and reimbursement discussions are ongoing in major European markets, which we expect will continue throughout 2023 with full launches in most European countries starting in 2024. We also anticipate use of name patient programs in European and distributor markets to continue to grow throughout 2023. Overall, we are making great progress and are looking forward to our potential label expansion for PFIC in December in the United States and the upcoming readout of the EMBARQ study in biliary atresia. We see the total opportunity for Live Marley across Allergy Syndrome, PFIC, and BA as greater than $1 billion, and that this opportunity is strengthened by our recently announced agreement to acquire Keenadol and Colbomb, which will deepen our presence in pediatric hepatology and establish a focused, multi-product franchise. There is a substantial amount of top and bottom line synergy to unlock by putting the bile acid portfolio and live Marley together in the hands of one commercial team. As the marketing efforts are similar in scope and the approach to market access product distribution and patient services are quite complimentary. We expect the bile acid products to continue to grow a low single digit rates annually. with the potential indication expansion to CTX for Keenadal providing an upside opportunity to increase patient diagnosis and treatment upon approval. Financially, the strong second quarter net sales of $32.5 million for Livmarlie and $27.5 million for Keenadal and Kolbam reported today by Travere totals approximately 60 million for the future combined franchise, with growth expected across all three products. In summary, we are building on the tremendous momentum and potential of our expanded portfolio to deliver these important medicines to patients with rare liver diseases. On that note, I'll turn the call over to Pam. Pam?
spk01: Thanks, Peter. We are thrilled to be heading into a data-rich second half of the year. First, as Chris mentioned, we are eagerly awaiting the December 13th BDUFA date for PfeC. Secondly, we announced in May the completed enrollment for the EMBARQ Phase II study in biliary atresia. This study has a six-month primary endpoint assessing bilirubin, and we're on track for the top-line data readout later this year, which will be the first placebo-controlled data with an IVAT inhibitor in this setting. I'm also pleased to say that the Phase IIb VISTA study of elixibat in PFC currently has the total number of patients randomized or in screening required for the interim analysis. This interim will be triggered when 45 randomized patients have been on study for a minimum of three months and is expected around year end. In addition, the interim analysis for the Phase IIb Vantage study in PBC is also expected around the same time. As a reminder, after these interim analyses, both studies will move into the potentially pivotal portion of the trial. And finally, turning toward the pending acquisition, we have high confidence in the success of the Restore Phase 3 trial, evaluating Kinadol in patients with CTX, and look forward to top-line data in the fourth quarter. If positive, this study could support a label expansion for CTX with the potential for orphan exclusivity. Now, regarding our ongoing efforts to present the growing body of data on Live Marley, this summer we presented 12 abstracts at both the EASL and European Pediatric Hepatology Congresses. These data demonstrated Livmarly's significant ability to reduce bilirubin, symptoms associated with allogel syndrome and PFIC, as well as real-world data in which patients with allogel syndrome came off of the liver transplant waiting list after Livmarly treatment. In addition, we published a seminal analysis in hepatology demonstrating that pruritus, serum bile acids, and bilirubin are significantly predictive for transplant and event-free survival in allogeal syndrome, showing that 93% of Live Marley-treated patients with pruritus reductions remain transplant-free after six years. So in summary, the upcoming data readout across indications, along with the ongoing robust clinical evidence of Live Marley in both allogeal and PFIC, cements our leadership position in pediatric hepatology, and we remain committed to growing our presence in rare disease. And with that, I'll turn the call back over to Chris. Chris?
spk08: Thanks, Pam. Mirab is proud to be the leader in pediatric hepatology. The team continues to deliver high growth with Livmarly and make important advances in treatment options for cholestatic disease across our clinical programs. At the close of our acquisition of Kinadol and Colbomb, expected in the third quarter this year, we will have three approved rare disease products, an upcoming PDUFA date for label expansion for Livmarly, and four additional indications in development in high-need orphan settings with upcoming clinical readouts. We are exceptionally positioned to expand our leadership and impact in rare disease and look forward to bringing these important medicines to patients around the world. With that, operator, please open the call for questions.
spk02: Thank you. If you would like to ask a question, please press star then 1 on your telephone key. We have our first question from Steve Seedhouse of Raymond James. Your line is open.
spk03: Hey, good afternoon, guys. I wanted to ask a couple questions about Embar. The first one is just, are you, like, are patients screened and consented and ultimately enrolled in the study before they've had their CASAI procedure or in the days or weeks after? And I'm curious if there's a lead-in sort of before treatment where you track bilirubin kinetics and see sort of what the response is to the surgery before initiating treatment. I have a couple of follow-ups as well.
spk01: All right. Thanks for the question. Appreciate it. So for the EMBARQ study, the patients immediately post-CASI. So the discussion happens sometimes prior to the CASI procedure when they learn of the diagnosis after the baby is born. And so the consent process can happen either before the concise surgery or immediately after the concise surgery, but the goal is to get these patients on to the was to get we're now fully enrolled was to get these patients into the study as quickly as possible. There are no criteria for bilirubin in the study so we're not looking at any bilirubin effects post the site we're enrolling all patients and we're really excited to have this study read out towards the end of the year.
spk03: Okay, great thanks for that. And then just on the 6 month analysis. I'm curious if there is a formal assessment of, like, transplant or other liver events already in that 6 month time point. Or, let's say, just through last follow up of all the patients that are. Enrolled by the time you announced the 6 month bilirubin analysis.
spk01: Yeah, thanks. So for the six-month primary endpoint, as you note, is bilirubin, but we are assessing in other key secondary outcomes transplant at that six-month time point, as well as other thresholds of bilirubin that are predictive of transplant. And then, you know, these patients at the end of the double-blind treatment period roll over into open-label extension, so we'll continue to follow those patients long-term.
spk03: Okay, and then just the last question I have was just on that open label extension. So I think correct me if I'm wrong. I think it would go through 24 months total. So, 18 months post the 6 month analysis. And the question is. Do you think you'll have the ability to leverage natural history control cohort data? You know, in similar ways that just based on some quality databases and analogy on for instance. Here for biliary atresia, or if not, is that work that. you would hope to do at Mirum in the coming year? Thanks so much.
spk01: Yeah, that's a great question. We're actually doing that right now. We're working with natural history registries, looking at kind of aligning those patients with the patients that are enrolled in our study and understanding a little bit more about thresholds and prognostic markers for transplant-free survival. And we'll continue to work with those registries throughout. We're looking at six-month time points, and then we'll obviously continue to look at long-term treatment versus natural history. So, all of that will be kind of as much as we can in the timeframe. The package will contain our data set plus work that we're doing supported by the natural history, and we'll have a discussion with the FDA at that time.
spk03: Great. Thanks so much.
spk02: Your next question comes from Manu Kuruha of SCB Learing.
spk06: Just there now, I guess. Thanks for taking the question, guys. A quick commercial question. I know we had a little bit of discussion last year around 2Q, 3Q seasonality. Can you give us a sense of where those trends are now? How should we think about modeling? And sort of what are the sort of key metrics we should be following or thinking about as we think about going through each season this year?
spk04: Thanks for the question, Manny. Yeah, you know, we feel that what we've seen in Q2 is really strong demand that feel really good about how the business is pointed heading into Q3. But, you know, we're worth mentioning we're just now at the beginning of August, so we'll have to see how things unfold over the next couple months. But overall, feel really good about how the business is pointed going forward.
spk06: Great, that's helpful. And can you, separately, looking forward post-presumptive deal close of the acquired assets, how should we think about margin evolving over time? Obviously, you're going to have a little bit of expense that you bring on, but that's going to be coming with a substantial amount of revenue. Is there room for further margin expansion going forward? On what time horizon might we see that as we transition from this Q4 first quarter with with revenues from all the assets put together through next year.
spk08: Thanks, Mike, for the question. The overall picture of bringing Kenadol and Coalbomb into Merum, we do see a tremendous amount of synergy. So they will contribute substantially to margin at Merum because of the overlap in our business. A lot of that behind some of the rationale of why we're so excited about bringing these products into our portfolio. We're not going to be guiding towards margin, but what I can say is that this puts us on a very sound financial footing, and we do expect all three products to continue growing for the foreseeable future. So that will continue to improve financial performance as we go.
spk06: Great, that's really helpful. Thanks guys. Congrats on the quarter. Yeah, thanks.
spk02: We now have David. The city bank, please go ahead when you're ready.
spk00: Hi, thanks for taking on question. This is on for David. So we wanted to ask if you could update us on the timing of the closure of the Travier transaction. And also, could we have some insight as to how the operating spend might shift post-closure? Thank you.
spk08: Yeah, thanks for the question. We're tracking towards closing this quarter, so we do expect a Q3 close. And then as we move into a post-close time period, we're excited about jumping right in to bring these products over to Mirim. Overall, they'll have a high impact in terms of margin and cash contribution right at the start in Q4. So see that synergy will play through in having these products in our hands. So expect that impact to show up with improved performance in Q4 from having these products at Mirim.
spk00: Okay, so maybe just a quick follow up on that. So, what's your strategy on incorporating this product into your current pipeline? Yeah, thanks for that question.
spk08: Absolutely, yeah. We can, I'll have Peter speak through some of the planning, but these fit very well with Live Marley and the current Live Marley business. I want Peter to get into some of the strategies we have planned.
spk04: Thanks, Chris. Yeah, we're really excited about bringing these products into Merum and excited about bringing several really talented folks from Travere into Merum as well. You know, we have a team right now that's been out, you know, promoting with Marley to pediatric hepatologists and anticipate, you know, that team continues. And also we'll have a team that focuses on physicians of specialties that are often involved in identifying patients with bile acid synthesis disorders, Zellweger spectrum disorders, et cetera, that focus on neurology, ophthalmology, medical genetics. So have a liver-focused team and what we're calling a metabolic-focused team and modest expansion to our field footprint. So pretty excited about how that plays out going forward.
spk00: Okay. That's very helpful. Thank you.
spk06: Thanks for the question.
spk02: Thank you. We now have Ed of HCWainwright.
spk05: Hi. Good afternoon, everyone. This is Thomas Yip asking a couple of questions for Ed. Thank you for taking our questions. Perhaps first question regarding the Embark data, regarding data positive and given that Molly's orphaned drug destination for Valerie and Tricia, Do you anticipate and park top line data along with national history data that you mentioned earlier? Do you think collectively that's enough to support with Molly's approval?
spk08: Thanks, Thomas, for the question. Overall, that's our strategy to take that package to FDA. It's too early to say what the determination will be at this point. bilirubin has not yet been fully established as an endpoint for approval, and that's the work that we're doing with the EMBARQ data and the natural history work. So, looking forward to putting together what we think will be a really compelling dataset.
spk05: Okay, got it. And then perhaps, can you go over some major differences that you know of so far between the FDA and the EMA what they're looking for for people with ample and for very rich users.
spk08: Thanks for the question there. We see a pretty common point of view in what we've had for our conversations. And I think it's worth noting that there really is not a lot of clinical data or research that's been done in this space. So the EMBARQ study really is a landmark. It's going to be the largest and first study of IVAT in the setting, and one of the few studies that actually has an intervention that early after the CASAI, so really important data set to bring forward to advance care for these patients.
spk05: Got it. And then perhaps one final question for Bolexibat. You mentioned the interim analysis for both. and advantage studies both expected later this year. Can you tell us what type of data we can expect to see?
spk01: Yeah, thanks for the question. So for the PSC VISTA study, this is a blinded interim, so we won't be sharing any data. But it is for dose selection, and then the study will upsize if need to or not, and then carry on a potentially registrational Emily Early- Part two of the study so wouldn't expect any top line data out there, but would expect to be able to. Emily Early- communicate that the study is is carrying on and continuing on for the PBC vantage study, we will be sharing top line data, this is an open interim analysis and so we'll be able to have a look at the data and we'll be we'll be sharing that with you later.
spk05: Okay, understood. Thank you again for the kind of questions and looking forward to data from the Vantage study. Thanks, Thomas.
spk02: Thank you. If you'd like to ask any further questions, please press star then 1 on your telephone keypad. Our next question comes from Brian Scorney with Baird.
spk07: Hi, this is Charlie on for Brian. I wanted to ask about the SG&A. We've seen it climbing somewhat steadily over the past several quarters. Just wanted to know, particularly as you start to integrate the bile acid team from Travere, if we should expect that to keep rising, what kind of trajectory that's going to take. And along a similar line, thinking about the looks of that, as that hopefully gets closer to getting commercialized, what kind of regulatory strategy are you taking going forward? And considering that these are adult liver diseases, will this require a different commercial sales force? Or do you think that you can also leverage the field force that you have currently? Thank you.
spk08: Thanks for the questions. I'll comment a bit on SG&A and regulatory strategy and task force. Peter talked about the field team. And for SG&A, we have Yeah, what we see is kind of this modest increases over prior quarters, really driven by initially the launch in the U.S. and now some of the international markets that we've brought on. So just for the base business, you know, some incremental SG&A increase over time is what we'd expect. And then with adding in Kinadol and Colbaum, that will bring incremental SG&A expense, but really modest compared to the top line opportunity. As Peter was mentioning earlier, there's a lot of synergy in how we approach those markets and the fit with Marley. So we are bringing some people over who have great experience with those products from Travere, excited about bringing those folks into the Merrim family. But overall, the total contribution will be quite attractive with these products in our hands. Now, as we move forward to Velixibat, which will be an opportunity to expand into adult hepatology, first, the endpoint in registration to comment on, these studies incorporate pruritus as the primary endpoint. Similar to Allergy Syndrome and the ongoing review for PFIC, the pruritus is the outcome for approval, and that's what we're using for the registration approach for the adult indications. I'll let Peter comment on the commercialization strategy. Yeah, interesting.
spk04: So, yeah, I think we would be able to leverage our commercialization team and infrastructure to, you know, launch Felicibat into adults. Our current team is primarily pediatric-focused. Maybe it goes to the higher decile, larger volume adult liver doctors, but we would look to expand to a broader group.
spk07: Great. Thanks a ton for the color, and congrats on the quarter. Okay. Thanks for the questions.
spk02: Thank you. I'd like to hand the call back over to Chris for any final remarks.
spk08: Great. Thanks, operator. And thanks again to everyone for joining today's call. Have a great day. Goodbye.
spk02: Thank you all for joining. I can confirm this does conclude today's call. Please have a lovely rest of your day, and you may now disconnect.
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