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spk08: Good afternoon. My name is Chris, and I'll be your conference operator today. At this time, I'd like to welcome everyone to the Miriam Q2 2022 business update. All lines have been placed on mute to prevent any background noise. After the speaker's remarks, there will be a question and answer session. If you would like to ask a question during this time, simply press star, then the number one on your telephone keypad. To withdraw your question, please press star one again. Thank you. Ian Clements, Chief Financial Officer. You may begin.
spk06: Thanks, Chris. Good afternoon, everyone. I'd like to welcome you to Miriam Pharmaceuticals' second quarter 2022 conference call. I'm joined today by our President and CEO, Chris Peets, our Chief Operating Officer, Peter Radovich, and our Head of R&D, Pam Vig. Earlier this afternoon, Miriam issued a news release announcing the company's results for the second quarter of 2022. Copies of this news release and SEC filings can be found in the Investors section of our website. Before we begin, we'd like to remind you that during the course of this conference call, we will be making certain forward-looking statements about MIRM and our programs based on management's current expectations, including statements regarding MIRM's business plans, development programs, strategies, prospects, market opportunities, and financial forecasts and guidance. MIRM 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 10-K for the year ended December 31st, 2021, and any subsequent reports filed with the SEC. With that said, I'd like to turn the call over to Chris. Chris?
spk10: Thank you, Ian, and good afternoon to everyone.
spk09: It's been another great quarter for Miriam. In the second quarter, we achieved $17.5 million in Marley net sales, which reflects our team's commitment and expertise. and the life-changing impact of Livmarly for allogeal patients with cholestatic pruritus. This quarter's progress furthers our goal in becoming a leader in rare disease. We are proud of our launch success and look forward to continued growth in the United States as Livmarly becomes standard of care in this setting. The success and impact for patients with allogeal syndrome in the U.S. are why we are so excited about what's to come across all of our programs. First, the U.S. launch shows what's possible for the upcoming potential European and broader international approvals of Livmarley and Algeo syndrome. This disease has severe burden around the globe, and we look forward to realizing the promise of Livmarley for patients more broadly outside the U.S., starting with the anticipated fourth quarter European approval. Second, the clinical impact we've seen in Algeo syndrome in our pivotal data, as well as recent presentations showing improvement in long-term outcomes, provides evidence of the potential for Livmarly and Velixibet to improve lives across a number of cholestatic settings. We are progressing our five late-stage Livmarly and Velixibet clinical programs, which will generate multiple data readouts over the next 18 months, the first of which will be data from our landmark PFIC Phase III study, an important label expansion opportunity for Livmarly later this year. We have a lot to look forward to as we head into a busy end to 2022. The progress of Livmarly commercialization and in our promising pipeline is made possible by both the unwavering dedication of our team and our collective mission to bring important new treatments to patients around the world. With that, I'll turn it over to Peter, who will provide a commercial update. Peter?
spk10: Thanks, Chris.
spk12: We are delighted with Miriam's ongoing launch success as we get Liv Marley, the first and only FDA-approved medication for this disease, into the hands of more patients. Today, I'll share further color around Liv Marley's 17.5 million net product sales and what we're seeing in the commercial business. As a reminder, there is no inventory in our reported product sales, which are therefore a very accurate representation of patient demand. Now, taking a closer look, second quarter revenue was driven by a steady addition of new Allergy Syndrome patients as well as consistent retail cadence with limited treatment discontinuations, which continue to mirror what we saw in the clinical trial site. On the payer side, we've achieved our goal of over 90% of Live Marley dispenses being reimbursed, which has been a real strong point of the launch. We are thrilled about the rapid acceptance of Live Marley by physicians, payers, and patients in the first three quarters on the market. But it's clear we're still in the early days of launch with significant room to grow in the United States. In fact, our market research suggests that physicians intend to substantially increase utilization of Live Marley beyond the group that has received a prescription since launch. So looking forward, we expect continued quarter-over-quarter growth in the United States. Now turning to our plans for Live Marley's international launches, We are excited about the potential European approval of Lidmarli in the fourth quarter. The Mirim team in Western Europe has been active this year in early engagement with physicians, payers, and other key stakeholders. As is typical of launching a rare disease medicine in Europe, we expect reimbursement to progress country by country over the year or two following approval. We expect to launch first in Germany shortly after EMA approval followed by other major markets in Western Europe. Beyond Western Europe, our eight commercialization partners are also making great progress towards introducing Livmarly to new AllerGeal patient populations worldwide, with potential approvals starting as early as this year in these geographies. In total, outside the United States, we currently have over 100 AllerGeal syndrome patients on the Livmarley Clinical Program that we expect to roll over to commercial therapy upon approvals and local commercial availability. And on top of this, in the PPET Clinical Program, we currently have nearly 100 patients ongoing worldwide who also will be eligible to roll over to commercial therapy upon regulatory approvals and local commercial availability. And now, I'll hand the call over to Pam to provide an update on our pipeline.
spk04: Thanks, Peter. In the second quarter of 2022, our team has continued to build a foundation for our important upcoming milestones for 2022 and 2023, which positions us well to deliver life-changing medicines to rare disease patients. First up, we expect top-line data from our March P6 Phase III clinical trial in the fourth quarter of this year. This landmark study builds on the findings of the Indigo Phase II study of Liv Marley and P6. in which dramatic clinical response led to breakthrough designation for PFIC2. Patients with sustained serum bile acid response in indigo also demonstrated five-year transplant-free survival with Livmarli, a remarkable finding in this disease. Also in these responders, we saw significant improvements in pruritus, growth, and liver function. And all of our learnings from the long-term indigo experience have been applied to our March PFIC Phase III study. Now, as a reminder, this is the largest randomized PFIC clinical study conducted to date with more than 90 patients randomized. This study includes all PFIC subtypes at higher doses than was previously tested in our Phase II clinical study. And we look forward to sharing top-line results next quarter. We also have four additional late-stage studies that we expect to read out in 2023. First, the Phase II B VISTA study of Elixabat in primary sclerosing cholangitis. physicians believe that to be a major advancement in the treatment of this progressive disease with no approved therapies. We now expect to conduct the interim analysis mid-year 2023. This revised timeline is largely driven by the current environment for running clinical studies with impacts on site openings outside the U.S. and the related supply chain. We're optimistic about the direction of this study, and we have recently had several international site openings and patient screens. Steady conduct has otherwise gone well with some patients now in the open-label extension portion of the study, and we continue to get positive feedback from sites. Regarding the OHANA study in patients with intrapatic cold cases of pregnancy, we recently launched a streamlined protocol amendment. We're enrolling patients for the open-label phase of the study and are planning for an interim readout in the first half of next year. Finally, in the second half of next year, we remain on track for data from both the Phase 2B Vantage study in adults with primary biliary cholangitis, as well as primary data from our Liz Marley Phase 2B Embark study in biliary atresia. And across our pipeline and publications, we remain dedicated to driving academic and collaborative research in rare disease. And we are proud of the significant data presentations we delivered this quarter at both Espigan and Eazl. And all of these noteworthy presentations on long-term outcomes and response with Liz Marley in Allergy Syndrome can be found in the Publications and Presentations section of our corporate website. And on that note, I will turn the call over to Ian. Ian?
spk06: Thanks, Pam. Earlier today, we issued a press release that included financial results for the quarter, which I'll briefly summarize. Additionally, full details can be found in the Form 10-Q, also filed today. As highlighted by Chris and Peter, we recognized $17.5 million of net product sales in the second quarter, a 61% sequential increase over the first quarter of 2022. We expect continued growth of Litmari revenues over the coming quarters. Our total operating expenses for the quarter were $48.9 million, which includes research and development expenses of $25.4 million, SG&A expenses of $21 million, and cost of sales of $2.5 million. I should also note that these numbers include expense related to non-cash stock-based compensation of $6.6 million. For the quarter ended June 30, 2022, net loss was $26.9 million, or 84 cents per share. Merriman remains well-funded, and at the close of the second quarter, ended June 30, 2022, we had cash, cash equivalents, and investments of $225 million. To further strengthen our position, we recently reduced our royalty and milestone obligations to Live Marley and Belexabat with the acquisition of Sotergen, which was previously a licensing partner and is now a wholly owned subsidiary of Miri. In summary, with our growing top line contribution and efficient business model, we're in a strong position to achieve critical milestones and expand our global commercial presence over the coming years. Now I'll turn the call back over to Chris for any final comments. Chris?
spk09: Thanks, Ian. And thanks, everyone, for joining today. To close, Miriam continues to execute on all fronts in 2022 as we deliver on our commitment to make meaningful change for rare disease with Live Marley and our development pipeline. With $17.5 million in net product sales, we achieved this quarter. It brings us to $28.4 million year-to-date. And we intend to build on this trajectory in the months and years ahead through continued growth of Live Marley in the U.S., potential additional approvals and launches in Europe and beyond, and through pipeline opportunities as we look to announce top line data from our March PFIC phase three study later this year, the first of several key data sets over the coming 18 months. And with that, operator, please open the line for questions.
spk08: Thank you. As a reminder, if you'd like to ask a question, please press star then one on your telephone keypad. Our first question is from Jessica with JPMorgan. Your line is open.
spk05: Hey, guys. Thanks for taking my question. Nice results this quarter. Where do you expect patient ads on Live Marley to come from going forward? Is it more from broadening to new physicians or from existing prescribers going deeper within their patients, maybe into more moderate patients if they initially started Live Marley in the most severe? And then thinking about the Europe launch, how should we think about the average price you might achieve in Europe relative to the U.S.? And lastly, when the Phase 3 March PPIC data reads out, what in particular are you going to be looking for in that data set in comparison to Albarrio's BILVE data in PFIC?
spk10: Thanks for the question, Jess.
spk09: I'll turn it over to Peter to touch on those two commercial topics, and then we'll circle back on the PFIC data. Thanks for the question, Jess.
spk12: In terms of where we expect to see growth in the United States with regards to new patient ads, I think primarily it's the latter phenomenon you described. A lot of our target positions have prescribed initially, as you note, often to maybe the more severely affected patients, have gained comfort with Marley, and we see them kind of growing their prescriptions over time into their patient populations. There may be, to a lesser extent, some newer accounts that come online as well, but really predominantly the former phenomenon you note. In terms of European pricing, it's probably a little bit early to get into specifics as we look towards approval in Q4. but fundamentally feel really confident in the Marley's value proposition and the data that underpins the submission and that Marley can achieve a strong price in the European countries, kind of on par with other rare disease medicines.
spk09: And circling back to the PFIC question, I touched the opening comment in the past at the PAM to talk through some of the analysis points. But overall, we're looking to match or beat the really tremendous effects that we saw in the Indigo Phase 2 study that led to breakthrough designation already with FDA for PfeC type 2. And a lot of that's built into the analysis plan.
spk04: Yeah, and I just would add to that. I think we're just really excited about this study because, as mentioned, it's the largest PFIC population that's ever been studied with over 90 patients enrolled, and I think that's a pretty significant number when you look at the rarity of the disease. In this study, you know, the primary endpoint is in the primary cohort of PFIC2, and so what we're looking at as a primary endpoint is on pruritus. Key secondary endpoints include serum bile acid, and then there's a step-down approach using hierarchical methodology in which we'll then look at the all of the patients, which includes PPIC2 as well as the other subtypes. So the totality of the PPIC population, then again looking at the primary endpoint and then the key secondary endpoints. So a lot of data to come and really looking forward to sharing that with you next quarter.
spk03: Our next question is from Manny Brouhar with SVB.
spk08: Your line is open.
spk00: Hi, good afternoon. This is Lily Nisango on behalf of Manny. I just had a follow-up question on the PSEC data. First, would you be able to provide a little more granularity in timing of presentation? Should we expect the data to be presented at an upcoming medical meeting? And secondly, in terms of the data that we may see, where would you anticipate differentiation from competitive assets?
spk09: Thanks for the question, Lily. On timing, we have nothing more specific than Q4. We will plan to share top-line findings when we conduct the unblinding and look to get the details presented at a conference, the next one that would make sense. And I think too early to say which one that would be. In terms of differentiation, I think the key points that we think about as we design the study was from the findings on dose ranging across all the prior clinical work with Marley, suggests that at these higher doses that we're using in the March-PFIC Phase III study, you can expect more bile acid clearing. And so that should then translate into better clinical response. So we'll be looking for response rates on pruritus and serum bile acids to see how those compare competitively.
spk03: The next question is from Yasmeen Rahimi with Piper Sandler.
spk08: Your line is open.
spk01: Hi, guys. Thank you so much for taking my question. This is Lauren on for Yaz. Just two questions. One, could you shed some light onto why timelines for Volixibab moved from 4Q22 to 2023? And then the second question about the PFIC study as well. What do you expect will be an outstanding data set? And then how soon could you get that data packaged into the label? And then have you started to warehouse these patients ready to put on Live Marley? Thank you so much.
spk09: Great. Thanks for the question. I'll start with the PFIC follow-up and then let Pam speak to some of the other pipeline questions. And in terms of the data timing, when we unblind in Q4, We'll look to turn around and submit that for label expansion relatively quickly. So we'd expect to get it in early next year, in the first half of next year, and work through review to have subsequent approvals for PFIC based on that data. And in terms of patients available, as Peter mentioned, we already have nearly 100 PFIC patients currently on drug active around the globe. with an expanded access program also open for geographies around the globe as well that will eventually be eligible for commercial rollover once we get to those approvals and reimbursements. So, yeah, we do have a very active clinical program in PFIC that will be eligible for rollover down the road. I'll let Pam speak to the voicemail timelines.
spk04: Yeah, so thanks for the question. So for the VISTA study, which is a PFP study, You know, we're enrolling and, you know, what we're seeing is that we have patients on the long-term extension open-label phase of the study, and so we remain really positive that that's a great sign patients are remaining on drugs. With regard to the timelines, you know, I think that there's, you know, pretty typical of what's been seen across the industry, frankly, with some slower startups, some staffing shortages, do a hangover from COVID and still existing as well as supply chain issues. So all of that with lab kits and things like that. So all of that is, I think, been mitigated and lots of these issues and sites are now up and running. We've got new centers that are now being opening outside of the US and patients are in screening. So we're feeling pretty good about where we are today and looking forward to that data readout next year.
spk01: Thank you so much. Thank you.
spk08: The next question is from Ed Arce with HC Wainwright. Your line is open.
spk11: Great. Thanks for taking my questions. And congrats on another strong quarter. Two questions for me. Firstly, on the $17.5 million in Live Marley sales, just wanted to dig in a little deeper, if you could, and describe what really are some of the key drivers to that rapid growth. What, in your view, is really working so far during the launch? And then secondly, how many patients are on commercial drug as opposed to other pre-commercial programs? And then of that, what is the split between those sort of naive and those that have come on to commercial drug as a rollover from trials. Thank you so much.
spk09: Great. Thanks, Ed, for the question. I'll take a little bit on kind of that last part of the question. I'll let Peter speak a little bit of what we're seeing in the field, what's being effective and dynamic on uptake. I think the first point to make on actual current patient numbers on commercial drug given that there is no inventory in our model, it is pretty clear. So that 17.5, if you divide it by what the approximate net price is per patient per quarter, you can see that roughly translates to about 200 patients on drug this last quarter. And we give you that direction just so it's pretty simple to track from the net sales, which is really what we're using to gauge our performance. In terms of the U.S. rollover, it really is very much a minority of the patients on drug. The vast majority of patients in the U.S. were prescribed after the approval. And one thing that kind of gives us a lot of optimism about ex-U.S. growth is that the 100 patients that we mentioned for the Allergy Syndrome Expanded Access and Clinical Program is much larger than what we had in the U.S. comparatively. But without that, I'll let Peter talk a little bit about what we're seeing out there in the field.
spk12: Thanks, Chris. It's really been a steady addition of new patients over time since approval. Really high compliance persistence rates, which is consistent with what we expected from our clinical trial and a product like Live Marley, which has a rapid symptomatic benefit, the patients can feel the product very quickly after they start taking it. And likewise, if you forget to take it for a few days, you'll notice that as well. So we see very high compliance persistence and very low discontinuation rates. So those phenomena together with steady new patient ads is really what's driving the growth. Maybe the final point I'd add is we mentioned that over 90% of our dispenses were reimbursed this last quarter. So really happy with that. I've seen the payers come online and now the vast majority of our expenses are paid.
spk03: Great. Thanks. That's helpful. Thanks for the question.
spk08: The next question is from Ryan Scorny with Baird. Your line is open.
spk07: Hi, guys. Charlie on for Brian today. Thanks for taking the question. Congrats on the quarter. I just wanted to get a little more color on how you're thinking about the magnitude of commercial potential in PFIC compared to Allergial Syndrome, especially given kind of what we've seen in space. It's been lagging behind in terms of sales compared to how you've been doing in ALGS. So I'd just be curious how you're thinking about those, especially given We've heard from a KOL that they should be similar prevalence and incidence rates, so it would just be great to get your thoughts on that.
spk09: Thanks, Charlie, for the question. That's actually a good one to dig into because there's a couple dynamics there. Overall, from what we see, this is both literature and what we're seeing in conversations with some of the prescribers and pediatric GIs and hepatologists that see these patients, is that allogel syndrome is much more common. And we kind of see it as likely in the three to four times the size of PFIC overall for that 2,000 to 2,500 kids in the U.S. that we talked about for allogel syndrome. One of the kind of interesting dynamics that makes some of the literature and epidemiology a little bit harder to be precise for PFIC is that it is a recessive disorder. So it does vary by geography much more than allogeal syndrome does. So kind of the dynamic you mentioned there where you may have one KOL who sees a similar number of PCIC patients as allogeal patients, that often is very much city or state dependent. And there are several centers where the opposite is what you find when you go have these conversations where allogeal syndrome is very common and they may not see a PCIC patient hardly at all. So it does vary by geography. It makes the PSIC numbers a little bit harder to nail down.
spk08: Great, thanks. Thanks for the question. Again, that's Star 1 if you'd like to ask a question. The next question is from Steve Seedhouse with Raymond James. Your line is open.
spk10: Hi, this is Ryan Deschner out for Steve Seedhouse.
spk09: I was just wondering if you think it's possible to demonstrate a reduction in preterm birth, given not only the clinical outcome importance, but also the pharmaeconomic importance in the ICP program. Thanks, Brian, for the question. I'll let Pam jump in on this one.
spk04: Yeah, so, you know, it's a great question. Thanks for the question. So, I think, you know, when you look at the literature on elevated serum bile acids, I think what you're alluding to is that, you know, any increase in serum bile acids is an increased risk to the fetus. And so obviously what we are also looking for in one of our secondary endpoints in the potentially registrational portion of the OHANA ICP study is that we are looking at those outcomes as well. And so therefore, any reduction that you would see in serum bile acids hopefully translate to those untoward fetal outcomes. So paratus looking is obviously the primary. That's the most near-term outcome. FDA likes that endpoint. Looking at serum bile acids and then also looking at other events such as untoward fetal outcomes.
spk03: But that will be in the Part 2 portion of the study. Got it. Thank you very much.
spk08: We have no further questions at this time. I'll turn it over to Chris Peets for any closing remarks.
spk09: Great. Thanks, Chris. And thank you, everyone, for joining today. Have a great evening. Bye.
spk08: Ladies and gentlemen, this concludes today's conference call. Thank you for participating. You may now disconnect.
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