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spk02: Ladies and gentlemen, welcome to the Miro Pharmaceuticals Report's first quarter 2023 financial results and profile business update. My name is Glenn and I'll be the operator for today's call. If you would like to ask a question during the presentation, you may do so by pressing star 1 on the telephone keypad. I'll now hand you over to your host, Andrew McKibbin, to begin. Andrew, please go ahead.
spk06: Thanks, Glenn, and good afternoon, everyone. I'd like to welcome you to Miriam Pharmaceuticals' first 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 Bigg. Earlier today, Miriam issued a news release announcing the company's results for the first quarter 2023. Copies of this news release and SEC filings can be found in the Investors 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 Merrim 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 forecasts and guidance. Merrim 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. Andrew Barber, Investors agreed the risk factors set forth the miriams 10 K, for the year ended December 31 2022 and any subsequent reports filed with the SEC with That said, I like to turn the call over to Chris Chris.
spk07: Chris Van Orden, Thank you, Andrew and good afternoon to everyone joining us on the call today. Chris Van Orden, it's been another exceptional quarter for miriam as we continue to advance our leadership position as a high growth rare disease company focused on life changing medicine. Our clinical, regulatory, and business achievements in the first quarter, with total revenue of $31.6 million, are driven by our team's passion and expertise in addressing urgent unmet needs for patients with rare disease. With our five-part strategy to become a global leader in rare disease in place, we're pleased with the progress we've already made in 2023. First, we've continued to build on the successful launch of Live Marley and Allergy Syndrome in the U.S. tracking well on our guidance of 50% year-over-year net product sales growth, driven by new patient starts. Second, our international business is also building well, with a recent launch in Germany and early access programs in France and other international partner markets. We look forward to additional launches around the globe later this year and into 2024. Third, we've accomplished an important step in our strategy to expand the label for Lidmarli, In the first quarter, we announced label expansion to include patients three months and older. In addition, we submitted a supplemental NDA and European equivalent with our positive Livmarli PFIC phase three data. Our team is preparing for the December 13th PDUFA date and is excited about bringing Livmarli as a new treatment option advancing care for PFIC patients. We also anticipate providing top line data from the EMBARQ study in biliary atresia in the second half of this year a third potential indication for Livmarly. And fourth, in advancing into adult indications, we are conducting potentially pivotal studies for Velixibet in primary sclerosing cholangitis and primary biliary cholangitis, and look forward to providing interim updates from these studies in the second half of this year. In the fifth part of our strategy, we continue to evaluate opportunities across rare diseases to leverage Mirim's industry-leading capabilities. We also recently took a significant step towards enabling our strategy with convertible note financing of approximately $316 million aggregate principal amount. This financing enabled the repurchase of the nearly 10% royalty committed to our prior royalty investor and added additional growth capital to the balance sheet. We were excited to see the high level of interest in supporting our strategy in this well oversubscribed transaction. We continue to project that our balance sheet provides three plus years of runway beyond cashflow breakeven. We have started 2023 off with great momentum, and I'm excited about what the Mirim team is poised to achieve this year and beyond as we provide patients with life-changing medicines. And 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.
spk03: Peter? Thanks, Chris. We are pleased with the $29.1 million in Livmarli net product sales in the first quarter of 2023, which included $24.7 million from the U.S. and $4.4 million from international markets. The U.S. growth was driven by the addition of new patients, as well as a strong refill cadence. And we're pleased to report that substantially all of the key pediatric liver accounts in the U.S. have prescribed Livmarli. Based on their positive clinical experience and ease of access with Mirim Access Plus, healthcare professionals report that they intend to increase the use of Livmarli across their AllerGel syndrome patients. Taken together, the US business performance is tracking well against our guidance for 50% growth in 2023 net product sales. Turning to international markets, in Q1, we launched Livmarli in Germany, and it's off to a great start. We've already converted early access program patients to commercial drug, and we have seen impressive de novo demand in line with what we saw in the US launch. Pricing and reimbursement discussions are ongoing in major European markets, which we expect to unfold throughout 2023, with full launches in European countries beyond Germany starting in 2024. Also, we expect main patient sales in European and distributor markets to continue throughout 2023, although we anticipate quarter-to-quarter variability in order patterns. In summary, Live Marley is in a strong position after a year and a half in market as the first and only treatment for Algeo syndrome that is providing rapid symptomatic relief and long-term improvement in outcomes. Dr. Matthew Sholl, M.D.: : With marley is becoming standard of care and treating cold status period is with over 600 allergy l syndrome patients treated globally to date. Dr. Matthew Sholl, M.D.: : Clinicians have gained familiarity with and confidence in the marley's robust clinical profile, as well as its exceptional access and patient support. Dr. Matthew Sholl, M.D.: : And we believe this provides a strong competitive position propelling a continued growth story and allergy l syndrome. with exciting pediatric cholestasis indication expansion opportunities just on the horizon. And on that note, I'll turn the call over to Pam. Pam?
spk01: Thanks, Peter. In the first quarter, we achieved important milestones in our pipeline that support the growth of our programs and ability to impact more patients. As a reminder, the central problem in all of these diseases studied in our pipeline is accumulation of bile acids. And across these cholestatic settings, IVAT inhibition has been shown to directly reduce toxic bile acid accumulation, the associated debilitating symptomatic burden, and analogy of Liv Marley showed improved long-term outcomes versus standard of care. The benefits of profound IVAT inhibition was shown by the impressive data from our March PFIC Phase III study announced at the end of last year. And we're happy to share that our supplemental NDA submission was accepted with the DUFA date of December 13th of this year. And in April, we also submitted for approval of Livmarly in PFIC to the EMA for patients two months of age and older. Now, the data from the March PFIC study proved our hypothesis where greater IVAT inhibition using higher dosing regimens of Livmarly drove a highly statistically significant reduction in both pruritus and serum bile acids across the broadest range of genetic PFIC types studied to date. In addition, early and statistically significant improvements in growth, as well as in bilirubin, We're seeing in patients treated with Livmarly where 40% of patients with abnormal bilirubin levels at baseline normalized their bilirubin versus zero in the placebo group. Now, these data suggest an overall clinical improvement beyond pruritus for these patients, and we have heard strong positive feedback from the physician community, and if approved, look forward to the opportunity to expand Livmarly to a broad pediatric population. We're also happy to announce that we remain on track for top-line data readout for our biliary atresia program for the second half of this year. This study will provide the first placebo-controlled data for an IVAT inhibitor in this setting, and we're really excited to share more when the study is completed. As a reminder, the primary endpoint in the biliary atresia study assesses bilirubin at 26 weeks, as bilirubin is the most predictive marker of disease progression and transplant in this setting. And the dosing regimen in the BA study is the same as the March PFIC study, and the bilirubin improvements we observed in March are extremely encouraging as we look forward to our biliary atresia readout. Now, turning to our pipeline programs and adult cholestatic indication. We expect interim analyses from our studies of Elizabeth in both PSC and PBC in the second half of this year. We've previously shown that marked reductions in pruritus and serum bile acids can be achieved with iBAT inhibition in PSC, underscoring the potential for velixibat in this setting. And currently, there are no approved therapies for PSC, and roughly 60% of patients are being actively managed for pruritus with largely ineffective off-label therapies, none of which lower bile acids. Building off of our learnings from our pediatric programs, we are dosing at a higher level and BID dosing regimen, which adds to our confidence in these upcoming intramuralities. And finally, I'm proud of our team's academic and collaborative efforts as we continue to advance the benefit for patients from clinical studies to the real world. We're committed to leveraging our deep experience across all of these cholestatic disease settings as we continue our scientific leadership. And with that, I'll turn the call back over to Chris. Chris?
spk07: Thanks, Pam. Merrim is poised for continued growth throughout the years ahead. We anticipate that 2023 will be another transformative year with strong commercial growth potential PFIC label expansion, top-line data of the first randomized dataset of an IDAT inhibitor in biliary atresia, and interim analyses from our potentially pivotal felixivite studies. We remain excited about the growth prospects and potential of Mirim as we work to provide new medicines to patients with rare disease. And with that, operator, please open the call for questions.
spk02: Thank you. Ladies and gentlemen, if you'd like to ask a question, please press star followed by 1 on the telephone keypad now. When preparing to ask your question, please ensure your phone is unmuted locally. We have our first question. It comes from Jessica Phi from J.P. Morgan. Jessica, your line is now open.
spk10: Hey, this is Nick on for Jess. Thanks for taking our questions. Two quick ones for me. One, could you just talk a little bit about the factors that resulted in that timing shift for the Phase 2b VISTA trial mid-2023, the second half of 2023. Is that more of a narrowing or a shift?
spk01: Yeah, hi. I'm happy to answer that question. Thanks for the question. So the reason for the delay in the PSD study, it's really a slight delay and mostly based on screen fails. And there's a number of reasons why patients are screening out. One is a mix of lab values. Some pruritus score compliance during screening where they have to put in their pruritus scoring. And if they're not compliant, they also get kicked out because we want to make sure that they can remain in the study. And then also some for itch score that is not meeting the minimum threshold to enter the study. What we're doing with some of this is we've got plans on advancing tools and education on how we standardize training and interpretation of those itch scores and just continuing to drive forward. And we're starting to see more movement and good activity.
spk10: Great. And as a quick follow-up, I know there have been some PBC trials in the past that have run into issues with high placebo responses on itch. Can you kind of just discuss how the Vantage trial is designed to help kind of mitigate those risks?
spk01: Yeah, thanks for the question. So in our PBC clarity study, as you know, we, there was a, we all ourselves had a placebo response rate. So we took those learnings, applied them into this study design. We baked some of that into the assumptions for our analysis, and we also adjusted our study design to accommodate for placebo failures and account for that. I don't think I can give specifics about the way that we're doing that in the trial, but we have thought very deeply about that and taken that into account.
spk10: Great. Thanks so much.
spk02: Thanks for the questions. Thank you. Our next question comes from Manny Furuha from SVP Securities. Manny, your line is now open.
spk08: A quick question on biliary atresia. How should we think about the scale of the opportunity, both in terms of the number of patients, which I think is fairly well defined, but also at what point would patients be How long would they be treated? Is the expectation here that you would pursue a lifelong study? Would you expect from your clinical feedback that physicians, in the case of successful study commercialization, would, after one year, two years, some period of time, look to withdraw patients rather than leave them on drugs for life?
spk07: Thanks, Monty, for the question. My one just kind of first comment, you know, filariatresia is the most common indication for liver transplant in pediatrics. So really, in terms of scale of unmet need and impact you can have, this is a really important indication. I actually asked Peter to maybe speak to a little bit of how to think about how this evolves over time and duration of treatment, because there are some nuances on how we think this indication can build over time.
spk03: Yeah, I think as you think about that piece of it, Manny, the biliary atresia is, you know, more common than AllerGeo and PFIT by a fair bit when you think about it from an incidence perspective, probably on, you know, threefold more common on newborn incidence in AllerGeo. But because the dynamics that Chris is describing, there's much less of a prevalent pool there because it's generally pretty rapidly progressive and heads towards liver transplant. So we'd envision in the kind of early years after approval, you know, the, you know, eligible patient population being, you know, kind of on the order of kids who were born, you know, within the last 12 to maybe 36 months. But as you, you know, if the drug does what we hope it does and get patients on therapy and, again, hopefully keep them from liver transplant, that would build over time and I think could be better than the other two indications.
spk08: That's really helpful. And when we talk about what the drug hopes one can do. Obviously, there's a little less certainty around the regulatory dynamic here because you can't really ask the child's age. Give us a sense of how you think about target effect size, target product profile, and is there more certainty in your view on what you need to show for clinical uptake, or is that also a little bit up in the air as our regulatory questions?
spk07: Thanks for the question there. I mean, there's a couple of aspects to consider in thinking about the regulatory conversations that we'll have after this data set is unblinded. Regulators are data driven. They do have patient interest in mind. And so I think if we show a really convincing data set paired with advancing some of the understanding of the disease and the importance of bilirubin, which is some work we're also doing in parallel. That'll be the basis for a really good case and strong conversation on why the EMBARQ study could potentially support approval in biliary atresia. We do need to generate that data, and that's all kind of baked into the study design. I think the one thing that I'd point to in addition to The bilirubin data that Pam talked about from the PFIC study, just looking at mean change in bilirubin. There's also categories that are really well established on risk profile for transplant in terms of level of bilirubin at three months. So we have a close eye on that being able to down categorize patients on the bilirubin score, you know, above six, two to six and below two. for Billy Rubin, each step is a meaningful difference in risk of outcome. So we'll look at those in a kind of responder or shift table format. All right.
spk08: That's really helpful. Thanks, guys. Thanks for the question.
spk02: Thank you. With our next question comes from Steve Seathouse from Raymond James. Steve, your line is now open.
spk05: Hi, two questions for me. This is Ryan Deschner on for Steve Seedhouse. Now that we're more than a year into the Marley launch, have you seen an appreciable step up in sales due to the increases in average patient weight? And how often are treating physicians adjusting dose for growth and other reasons?
spk03: Thanks for the question. You know, what we see there is I think about once a year, you know, patients are weighed and they're considered for dose increases. We do see dose increases in the real-world setting, consistent with the prescribing information for Lib Marley. Really, the majority, though, of the growth we're seeing is just de novo demand and new patient starts.
spk05: Excellent. And then also, in what areas do you anticipate potentially being able to differentiate from competitor, Modavixibet and bilir atresia. Thank you.
spk07: Oh, and in terms of, thanks for the question there, in terms of bilir atresia, I think that time to having a randomized data set was a big part of our thinking and our strategy here. The EMBARQ study on track to have data second half of this year will give us a really sizable lead in terms of having data on Livmarly, a really robust data set on Livmarly in this setting. We also have a feature of the study that is not in the Bilvey study, where at six months, patients roll on to open-label therapy. So the effect of starting treatment later is really important to look at from the prevalent patients that are out there. So that's a really key part that we're looking at here. And then lastly, similar to what we've seen in the other settings, our higher dose with Marley, I think we expect to have a bigger clinical impact. So I feel like we've really advanced our understanding of how to dose IVAT inhibitors and really think that that's going to show up in our top line data.
spk05: Thank you very much.
spk02: Thanks for the question. Thank you. We have our next question comes from from CT. Your line is now open.
spk00: Hi, and thanks for taking our call. I'm on for from CT. So we were wondering if you could comment on the ex-U.S. inventory build and what should we expect in terms of the sales trajectory ex-U.S. going forward?
spk03: In terms of the $4.4 million in sales we saw in Q1, a lot of that was from Germany and Western Europe where there's really very little, if You know, effectively zero inventory, true demand sales there as we as we get started in those countries. We do have, you know, some other the balance of the sales and international are named patient programs in distributor markets of markets that we're not directly selling in. But our partners are central, eastern Europe, Middle East. And generally what happens in those dynamics is there's a country-specific mechanism and an individual patient identified that the physician and our distributor partner work through a local reimbursement access mechanism. Oftentimes those patients get three, six, and even some cases we've seen 12 months of supply ordered for one patient. So that's kind of where, and so obviously you're not gonna see a consistent refill cadence every 30 days like you see in the U.S. with those kind of orders. So that's kind of the dynamic we see. And in terms of visibility and the guidance we have for 50% growth is based on the U.S. sales. In the international setting, we're really excited about the feedback we're getting, and we're excited that Germany's gone really well. And I think from sort of the patient position perspective, it looks a lot like the U.S. But it's just too early to provide firm guidance on international, given that we've just kind of getting started there.
spk00: That's very helpful. Thank you.
spk02: Thanks for the question. Thank you. With our next question, it comes from Ed Oggs from XC Wing Rights. Ed, your line is now open.
spk04: Hi, everyone. This is Thomas Yip asking a couple of questions for Ed. First, congratulations on a very impressive report. So just trying to tease out this of the 600 patients with ARD syndrome best been treated with Molly globally. Can you tell us roughly what percentage stay on chronic treatment and if you can provide some major factors for patients to stay on with Marley or, by far, stay off with Marley as well. We appreciate it.
spk07: Thanks for the question. The 600 number, really, we see as a really strong statement on familiarity with and interest in prescribing with Marley. That number comes from all sources over the history of the program for patients with Allergial Syndrome who have been prescribed or been part of a clinical study protocol. So it's really kind of all sources that feed into that. Not really a number that makes sense to parse out to the, you know, by country or by source. But what I can say is that across all of the different ways that patients have found their way to Live Marley Clinical Study, expanded access program, commercial products, compassionate use products in different formats, you do see the same profile of really high compliance, high persistence. Some of these patients is six plus years staying with the program because of the profound impact that it can have.
spk04: Thanks. Thanks for the clarification. Perhaps sticking with algea syndrome, with the U.S. label expansion down to three months of age and above, can you just tell us what your expectations are both for top line and also from a strategic standpoint as well?
spk07: Yeah, on the label expansion for algea down to three months of age, really important step because that's where most patients are diagnosed. So it allows Livmarly as a treatment option early on. Overall, top line impacts, keep in mind that these are younger, smaller patients. Really important for the long-term view, but staying consistent with our guidance of 50% year-over-year growth in the U.S. and international as we get further in, we'll look at how we fold that into guidance as well.
spk04: Got it. And that perhaps switching gears to PSAC with the application submitted to the EMA in Europe, when should we expect the next milestone, including a CHMD opinion perhaps?
spk07: Yeah, so the PDUFA date we shared in December, excited about that. Potential timing for an opinion from CHMP would be late Q3 or in Q4.
spk04: Got it. Perhaps one final question. This one is financial. With the convertible offering closing in April, can you tell us what your estimated cash went all the way out to?
spk07: Yeah, so it's similar. TAB, Mark McIntyre:" described in the prepared remarks really are fully funded with the current business to cash flow break even and positive so three plus years really meant to just be an indicator of the strength that we have there and see runway. TAB, Mark McIntyre:" To operate the business for the long term here continue to grow the products.
spk04: Got it. Understood. Thank you so much. Thank you again for taking our questions. Looking forward to the data.
spk07: Yeah, thanks for the questions.
spk02: Thank you. So our next question comes from from . Brian, your line is now open.
spk09: Hi, this is Luke. I'm for Brian. Just a couple on Embark. Do you have an update on the scope of detail you might expect to include with the top line readout beyond just bilirubin in particular, or maybe any outcomes data? And then are you able to generally characterize the geographic dispersion of the enrolled patients in terms of US versus other territories? Thanks.
spk07: So thanks for the question. And overall on top line, you know, other than the primary, you can't really speak to specifically what else would be in there other than getting material updates disclosed. The study's not powered for outcomes at that time point, so unclear if we would have anything to share on that point. And then geographic presence of sites really around the globe, and we're seeing activity across all geographies. Nothing really to point to there.
spk09: Great, thanks. And then just one on the PFIC launch. Do you think this will be a lean add-on or do you see any need to invest further into the commercial team?
spk07: I'll ask Peter to maybe describe some of the launch plans.
spk03: Yeah, we don't see a need for incremental OPEX for PFIC. It's In fact, the PFIC patients are probably taken care of by a subset of the prescribers who have prescribed Lew-Marley today for Allishield syndrome.
spk09: Good. I'll hop back in queue. Thank you.
spk04: Thanks for the questions.
spk02: Thank you. As a reminder, ladies and gentlemen, if you'd like to ask any further questions, Please press star, followed by 1 on the headphone keypad now. We have no further questions on the line. Great.
spk07: Thank you, operator. And a big thank you to everyone for joining today's call. I hope you have a great day. Goodbye.
spk02: Thank you. Ladies and gentlemen, this concludes today's call. Thank you for joining. You may now disconnect your line.
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