Mirum Pharmaceuticals Q3 2023 Earnings Call Transcript

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November 2, 2023

There are 12 speakers on the call.

Operator

Good afternoon. I would like to welcome you all to the Miriam Pharmaceuticals Conference Call. My name is Brika, 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. I would now like to pass the conference over to your host, Andrew McGibbon, Vice President of Investor Relations to begin.

Operator

So Andrew, you may begin.

Speaker 1

Thanks, Bhriga, and good afternoon, everyone. I'd like to welcome you to Mira Pharmaceuticals' Q3 2023 conference call. I'm joined today by our President and CEO, Chris Peetz our Chief Operating Officer, Peter Radovich our Head of Research and Development, Pam Bigg and our newest team member, Eric Bjerkhold, our Chief Financial Officer. Earlier today, Miriam issued a news release announcing the company's results for the Q3 2023. Copies of this news release and SEC filings can be found in the Investors section of our website.

Speaker 1

Full details and updates from the quarter can be found in our news release and 10 Q 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 Mirim and our programs based on management's current expectations, including statements regarding Merum's current and future business plans, development programs and regulatory expectations, Strategies, Prospects, Market Opportunities and Financial Expectations. Members are 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

Speaker 2

the risk factors set forth

Speaker 1

in Miriam's 10 ks for the year ended December 31, 2022, and any subsequent reports filed with the SEC. With that said, I'd like to turn the call over to Chris. Chris?

Speaker 2

Thanks, Andrew, and good afternoon to everyone. This was another strong quarter of execution at Miram. 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 have achieved multiple goals this quarter, including realizing total revenue of $47,700,000 for the quarter. We saw continued strong global adoption of Lip Marley.

Speaker 2

We also completed the acquisition of 2 high impact and synergistic commercial medicines, Kenadol and Colvom. 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 sheet. These achievements come alongside another special milestone for Miram coming up this weekend, our 5 year anniversary of launching the company. I'd like to thank the Miram team's dedication, perseverance and unmatched spirit for making all of our success possible in a short time with much more to come.

Speaker 2

Now moving to LIVARELY, we see steady demand growth in the U. S. And internationally. This reflects the positive impact of LIVARELY for patients and families in the Alachua Syndrome community and also the focused execution of the Miram team to make this important medicine accessible to patients around the world.

Speaker 1

I am proud of what

Speaker 2

we've been able to accomplish with Lymphmarly and Abengill syndrome and what that means for our execution on our expanded portfolio. This quarter, we grew our business with the acquisition of 2 commercial medicines, kenadol and kohlbaum, 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 $100,000,000 in 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 3 RESTORE study evaluating Kenadol in patients with cerebral tendinous xanthomatosis or CTX.

Speaker 2

The data showed KEYNOTELL has a significant impact across key measures of the disease and this Phase 3 data set and the potential approval of KEYNOTELL for CTX As 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 twenty twenty four. We also look forward to significant upcoming milestones and opportunities for Lymphmarly with the March 13 PDUFA date for PFIC in our upcoming Phase 2b data in biliary atresia. And for elixibat, 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 Durekle, who joined Miriam 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 Miram.

Speaker 2

We'll hear from him later in the call. And with that, I'll turn the call over to Peter. Peter?

Speaker 3

Thanks, Chris. With total revenue reaching $47,700,000 in Q3, We are decided by the tremendous progress that we have made across our U. S. And international businesses. Our team continues to bring LYBARLE to more patients worldwide and we have taken significant steps in establishing a leading pediatric hepatology franchise.

Speaker 3

Through the ongoing execution of our commercial strategy with Lubmarly and the addition of Kenadol and Colbond. Starting with Le Marley, in the Q3, our revenues grew to $39,000,000 in total net product sales, which represents over 100% growth before the addition of the bile acid portfolio. This reflects the life changing impact Marley is having an Alagille patients with pruritus and the excellent execution of our commercial team. The U. S.

Speaker 3

Business, We saw $28,600,000 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.

Speaker 3

And globally and are excited for the opportunity to broaden access to LUB MARLI to patients with Alagille syndrome and future indications.

Speaker 4

Moving to KEYNOTE and Pohlbaum.

Speaker 3

The addition of these medicines presents a significant opportunity to both augment LYMIRLI's growth and significantly enhance our relationships with the patient communities and prescribers in pediatric hepatology. There are striking similarities between the commercial models for these medicines. So at the hand 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. Though we do see some opportunity to augment our efforts with the expertise that we have brought in from Trevir.

Speaker 3

Pediatric hepatologists are the main call point for the Marley and Covalon 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 panel, Which is an important diagnostic tool that positions Miriam as a partner from the start of the patient journey. While Polbom and Kenadol 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 manifests 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 have 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 it's a key step as we prepare for a potential approval and launch in CTX.

Speaker 3

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 On that note, I'll turn the call over to Pam. Pam?

Speaker 5

Thanks, Peter. Last month, our team announced by measurement of urine bile alcohols and other biomarker measures, including cholesterol. The study showed a market and statistically significant improvement across the primary and all key secondary endpoints, demonstrating a broad and consistent effect of kenadol 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.

Speaker 5

Prior to this, it was not certain that treatment with Kenadol 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 Kenadol and CTX. And while quinadol is currently standard of care in CTX and recognized as a medical necessity by the FDA, we are very pleased that the results conducting a life changing impact KienaDol can have for these patients. We're grateful to the work that Travir 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.

Speaker 5

And with these strong results, we believe we are well positioned for our NDA filing, which we expect in the first half of Sure. Moving on to clinical milestones, we're looking forward to reporting top line data later this year from our Phase 2b EMBLAQ study of LYF Marley for pediatric patients with biliary atresia, which will be the 1st placebo controlled data with an IBAT inhibitor in this setting. For valexibat in PSC 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 interim data. Lastly, I'm proud of the growing volume of important clinical data and real world evidence validating the role of Lismarley for patients with Alagille syndrome and PFIC.

Speaker 5

The body of research we presented at the NAST began meeting recently, Including data from our March PFIC Phase 3 study and real world experience in Alagille syndrome speaks to the tremendous effects across the clinical and real world settings. In summary, Miriam'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 iBA inhibition can play a key role in changing the treatment paradigm for patients living with biliary atresia, PSC and PBC. Here. And with that, I'll now turn the call over to Eric to discuss our financial results.

Speaker 5

Eric?

Speaker 4

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 Q3 financial results. Earlier today, we issued a press release that included financial results As highlighted by Chris and Peter, we recognized $47,700,000 of net product sales in the 3rd quarter, A 47% increase over net sales the previous quarter. This reflects product sales of $38,700,000 for LiMARLE As well as $9,000,000 for Kinodol and Kolbahn.

Speaker 4

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 31, but we'll see the full quarterly contribution of these medicines in the Q4 of this year. Our total operating expenses for the quarter were $72,900,000 Which includes R and D expenses of $26,100,000 SG and A expenses of $36,500,000 And cost of sales of $10,200,000 For the quarter ended September 30, net loss was 23,600,000 or $0.57 a share. Net loss for the quarter included noncash stock based compensation expense of 8,400,000 An intangible amortization of $2,600,000 At the close of the Q3 ended September 30, 2023, We have cash, cash equivalents and investments of $306,000,000 In summary, we're well funded with a growing revenue base, Now I'll turn the call back over to Chris for final comments.

Speaker 2

Thanks, Eric. As you can tell, Miriam has continued to execute on all fronts. We've built the leading franchise in pediatric hepatology with 3 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 3 data to support the filing for approval of KEYNOTELE and CTX.

Speaker 2

We have several 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. We have the first question from Josh Schimmer of Cantor. Your line is now open.

Speaker 6

Thanks very much for taking the question and congrats on another exceptional quarter. Just wanted to ask because Ipsen on their call that they had seen some switching of patients from Livmerli to Bilvay for Alagille 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 Lymphomarley. Thank you.

Speaker 2

Thanks, Josh, for the question. I'll hand over to Peter to talk about

Speaker 3

the competitive dynamic. Yes. Thanks, Josh. Indeed, LaMarley has been approved and marketed in the U. S.

Speaker 3

For over 2 years now and Q3 is the Q1 where there was another product available in Algell 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 So we think the story is there that Le Marley's growth trajectory is very strong and it's not been impacted.

Speaker 6

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 BILVE? And if so, how many and why do you think they might have switched?

Speaker 2

Well, I think on the 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 Bilbao and Alagille syndrome. And so we Are continuing to grow and add patients there.

Speaker 2

It's a really strong situation for international markets really anchored around Europe at this point.

Operator

Your next question comes from Gavin Clark Gartner of Evercore ISI.

Speaker 7

Hey,

Speaker 2

So for the EMBARK readout, when is the baseline bilirubin value taken in relation to the Kasai procedure?

Speaker 5

Yes. Thanks for the question. So the baseline bilirubin that we see in the IMbark study is very similar to what's observed in the natural history. Pre Kasai, it's about close to about 10, which has been shown in a few literature papers about 9.5 to 10. And post Kasai, we see it around 7.

Speaker 5

And so that really leaves a lot of room in the 6 months for improvement of treatment. Looking forward to sharing that data later this year.

Speaker 2

Just to clarify, is the baseline value for the trial after the KAKAI when It's around the $7,000,000,000

Speaker 5

It is. It's after the site.

Speaker 2

Okay, got it.

Speaker 8

Congrats on a strong quarter and thanks for setting the question in.

Speaker 7

I guess a broader question around

Speaker 8

sort of looking past approval

Speaker 7

of the NDA and now that you have the general face How should we think about avenues that you have available to you to accelerate growth, Whether through pricing, promotional opportunities, etcetera, that perhaps weren't available when it's prior to having that data on FDA approval. And how should we think about avenues to accelerate growth in that asset or potentially expand margins as a consequence?

Speaker 3

Yes. Thanks for the question, Manny. Yes, I think we anticipate FDA approval of conducting a CTS potential approval in 2025. And thinking from that point, 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 chemo already.

Speaker 3

It's really seen as a necessity. So but we do know from KOLs and literature, diagnosis rates are quite low. We think only 10% of Patients approximately are diagnosed. So 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.

Speaker 3

So we're we have a team and efforts Promote 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.

Speaker 7

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 results on bilirubin, some perhaps Directional benefit on event rate transplant, obviously, there's no 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 on in a public setting An update on what the filability or path to approvability would be that indication?

Speaker 2

Thanks for that question, Mani. The biliary atresia timelines kind of laying them out in total. Expect The top line data from the IMbark study by the end of the year and we'll then take that data The way you described 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 look to have that meeting in the first half of the year and Sometime towards mid year be able to update publicly on what our plans are on taking it forward based on FDA input.

Speaker 7

Great. That's helpful. Is it reasonable to assume 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?

Speaker 2

We these case of interactions are dependent on scheduling with FDA. So as we get closer through getting to

Speaker 7

a meeting with FDA. We'd be

Speaker 2

able to provide some clarity. For now, the best guidance we have is having an update by mid year next year.

Speaker 7

Great. That's helpful. I know you have other analysts waiting. I'll hop off the line.

Speaker 2

Excellent. Thanks for the question.

Operator

Thank you. Your next question comes from the line of Steve Seedhouse of Raymond James.

Speaker 9

Thanks. Good afternoon, everyone. I wanted to ask about Embark first. Just 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.

Speaker 9

2nd, I just wanted to ask about the PBC and PSC Seed Data, it's I guess delayed, maybe you'd refute that, but the and widened in terms of just the on when the timing would be of those interns. Can you just talk about some of the inputs there as well? Thanks so much.

Speaker 5

Yes. Thanks for the question. I can answer these. So 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.

Speaker 5

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 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 pulling patients from that registry to really understand in this particular Embark population what bilegulin levels are really prognostic for Translum free survival and that will hopefully will help to bolster supportive information as part of our discussion with the FDA. With regard to PSC and PBC, yes, so we've seen some screen failures come in since our last earnings call. A lot of these are coming from the e diary compliance.

Speaker 5

So patients have to complete pruritus score daily. So we're working closely with 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.

Speaker 9

All right. Thanks so much.

Speaker 2

Thanks for the question.

Operator

We now have David Leibovitz of Citi.

Speaker 10

Thank you very much for taking my question. Considering the EMA recently We affirmed this decision to not give orphan disease status to Bill A for ALGS. How do you believe that leads through or does not lead through to Limbari relative to PFIC.

Speaker 2

Thanks for the question, David. Yes, it's a really interesting situation watching this unfold. And I Just for a little bit of background on this, 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 the recent Algell syndrome decision. And we're waiting to hear back for final feedback from EMA.

Speaker 2

We're optimistic about the data set that the March PFIC study provides and that it's a broader genetic Profile of patients, it starts at a younger age, and 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.

Speaker 10

Thanks for taking my question.

Speaker 2

Yes. Thanks for the question.

Operator

We now have Brian Swamy of Baird.

Speaker 11

Hey, good afternoon guys. Thanks for taking the question. My question is also on Empar. Can you just give us a little bit Have an idea of how you sort of characterize the primary, the change in bilirubin and what that means versus the secondary endpoint of getting patients below to make And just in time, in terms of expectations for the placebo arm on that secondary endpoint, I think the Children's Liver Disease Research Network About 50% of infants post Kasai at 3 months had biliary been less than 2. So is that a reasonable assumption for the placebo rate or for that endpoint embarking, is there anything to consider in the design that might make that more or less?

Speaker 11

Thanks.

Speaker 5

Yes. Thanks for the question. So maybe I'll start with Your last question first. So in the natural history, that shows that somewhere between 7.5% to 10% from the literature, post Kasai and less than half of those patients will here, jaundice. So the majority of those patients will remain elevated to varying degrees.

Speaker 5

And as you alluded to those patients That are do really well and have good established bio flow, less than 2 milligrams per deciliter. They'll do pretty well, but the majority of them are really between 2 and greater than 6. And so we'll also be looking at, if we shift patients from high risk, I mean, greater than 6 to moderate risk between 26 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 proportion the percent change difference between active in placebo at month 6 using an MMR analysis.

Speaker 5

And today, I think that was it. Was there another question?

Operator

Thank you. We now have Ed Ayers of H. C. Wainwright.

Speaker 8

Hi, good afternoon, everyone. This is Thomas Yip asking a couple of questions Brad, thank you for taking our questions. So first, congratulations on the positive resource data set early this month, Early last month actually. So can you talk about a little bit about KINZOL NDA filing and CTX In addition to restore data, what do

Speaker 2

you anticipate will be part of

Speaker 8

the data package? Will that be historical data or post marketing usage data?

Speaker 2

Thomas, thanks for the question. Yes, the NDA approach we see as relatively straightforward here for Keane et al. There's been extensive back and forth with FDA on what they're expecting for the NDA in the discussion of the RESTORE Phase 3 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 are that you'd see for A small molecule application.

Speaker 2

So nothing particularly unique about the NDA overall.

Speaker 8

Okay, got it. And then perhaps just one more question from us. For Limbaly and Algiers syndrome in U. S. Market as we get close to the end of the year, What's your anticipated growth level of growth for in Allergan's income for 2024?

Speaker 4

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.

Speaker 8

Okay, understood. Okay, thank you again for taking the questions and looking forward to And Bark, there are no.

Speaker 2

Thanks, Thomas.

Operator

Thank you. We now have our final question on the line from John Wallerben of JMP Securities.

Speaker 9

Hey, thanks for taking the question. Just on KINODA and cobalt, wondering if you could talk a little bit about the early experience of those products And then also the genetic testing, what kind of leverage do you think that will give you for Lip Marley down the road? And then how should we think about CTX approval and its potential impact on sales? Thanks.

Speaker 3

Thanks for the question, John. It's been really exciting to bring these products and the genetic testing into Merum. Yes, I commented on some of the dynamics in the script. Our liver team now is focused on promoting both Lou Marley and Cole Baum to pediatric hepatologists as well as kind of having a leadership role with the cold states of genetic testing, which 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 kind of having reasons to be in front of their customers. So really excited about what this is doing for Merrim's profile and supporting the Marley's growth.

Speaker 3

So I think That's one point. And then yes, as we move forward into 2024, 2025, continue to expect the We think we can we're excited about the investments we can make in CTS and trying Increase the diagnosis rate there, find more patients, find them earlier when we could probably have a larger impact.

Speaker 9

Helpful color. Thank you.

Speaker 2

Thanks, John.

Operator

Thank you. I can confirm we have no further questions. So I'd like to turn the call back over to Chris for any final remarks.

Speaker 2

Great. Thank you for joining today's call. Just as a note to close out here, it has been a strong quarter for Mirum, 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 line.

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Mirum Pharmaceuticals Q3 2023
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