Mirum Pharmaceuticals Q4 2023 Earnings Call Transcript

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February 28, 2024

There are 13 speakers on the call.

Operator

Good afternoon, ladies and gentlemen. Thank you for joining today's Merrim Pharmaceuticals Reports Q4 and Year End 2023 Financial Results and provides business update. My name is Tia, and I will be your moderator for today's call. All lines will be muted during the presentation portion of the call with an opportunity for questions and answers at the end. It is my pleasure to pass the call over to Andrew McKibbin, Vice President of Investor Relations and Finance.

Operator

Please proceed.

Speaker 1

Thanks, Kia, and good afternoon, everyone. I'd like to welcome you to Mirum Pharmaceuticals' Q4 2023 conference call. I'm joined today by our CEO, Chris Peetz our President and Chief Operating Officer, Peter Radovich our Chief Scientific Officer and Head of Research, Tam Big our Chief Medical Officer, Joanne Kwan and Eric Biercol, our Chief Financial Officer. Earlier today, Merrim issued a news release announcing the company's results for the Q4 and full year 2023. Copies of this news release and SEC filings can be found in the Investors section of our website.

Speaker 1

Before we begin, I'd like to remind you that during the course of this call, we will be making certain forward looking statements about Mirim and our programs based on management's current expectations, including statements regarding current and future business plans, development programs and regulatory expectations, strategies, prospects, market opportunities and financial expectations. Emera is under no duty to update these statements, and they are subject to numerous risks and uncertainties, and actual results could differ materially from those results anticipated by these statements. Investors should read the risk factors set forth in Miriam's 10 Q for the quarter ended September 30, 2023 and the 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, everyone. I'm excited to kick off an update on the many achievements for Miriam in 2023, another year of significant growth for us and cover highlights for what lies ahead for the company. Our continued progress in 2023 and opportunity ahead reflects the dedication of the Miram team to making a difference in the lives of patients and their families around the world. Over the year, we transformed our business with a broadened reach to patients in the U. S.

Speaker 2

And internationally, achieving $179,000,000 in net product sales and 142% year over year growth in total revenue. This was driven by continued strong growth for expansion of our commercial portfolio with the acquisition and integration of Kenadol and Colbond. We have now built a growing self sustaining leading rare disease business that is positioned for a great 2024 and beyond. The last year has shown the value creation potential of our strategy to drive growth in our commercial medicines, unlock the potential of upcoming label expansion opportunities, leverage our expertise in cholestasis in adult settings and continue to grow the pipeline. Looking at the year ahead for execution on our strategy, overall from the current commercial business, we expect to achieve $310,000,000 to $320,000,000 of net product revenue in 2024.

Speaker 2

This is expected to be driven by growth across all 3 commercially available medicines. We also have multiple regulatory and clinical catalysts this year to advance our pipeline. For Le Marley and PFIC, we are on track for our PDUFA date on March 13. We're excited for the opportunity to bring the strong results of the MARGE PFIC Phase 3 study to patients with a potential label expansion. We're also preparing for the submission of the positive RESTORE Phase III results of Kenadol and CTX in the first half of the year, an important label enabling opportunity.

Speaker 2

And we've also made great progress with the VISTAs and VANTAGE studies for valexibat in PSC and PDC. Patients for the interim analyses have been enrolled and we expect to conduct a dose selection interims of these adaptive studies in the first half of the year. We see the PSC program as an opportunity to bring the first ever therapy to market for this progressive burdensome disease. I'm proud of all of our progress in 2023 and the potential ahead for Mira. We look forward to continuing to grow the impact of our programs, providing life changing medicines to patients with rare disease.

Speaker 2

Now before I turn the call over to Peter to discuss commercial business, I wanted to welcome our new Chief Medical Officer, Doctor. Joanne Kwan, who joined Miram in January. We are thrilled to have Joanne and her extensive leadership and scientific expertise helping to drive the value creation we see ahead for Miram. We'll hear from her later on this call. And for now, I'll turn the call over to Peter to discuss our commercial business.

Speaker 2

Peter?

Speaker 3

Thanks, Chris.

Speaker 1

We are excited by the tremendous progress we have made across our U. S. And international businesses, coming off of 2023 where we saw 138% growth in total net product sales compared to 2022. We are already seeing the benefits of our newly expanded pediatric hepatology franchise and enhanced offering amongst the patients and prescriber community. I'm proud of the Miram team's focus on patients, while simultaneously driving the seamless integration of Polvol and Kenadol.

Speaker 1

And this is evident in the $69,600,000 total net product sales we achieved in the Q4 of 2023, which reflected growth across all three commercial medicines. For LIVARLE, total global net product sales grew to $41,400,000 in the 4th quarter. Our U. S. Business saw $31,400,000 $111,000,000 for the quarter year respectively, representing approximately 63% year over year growth.

Speaker 1

And internationally, we are seeing consistent strong uptake as well, but we continue to anticipate quarter over quarter variability in international revenue. Moving on to KEYDOL and Pohlbaum, the Q4 was the 1st full quarter with these two medicines under Miriam's leadership. And I'm very happy with how quickly we have been able to transition these medicines into our business. In the Q4, we recognized net product sales of $28,100,000 the highest quarter to date for the bile acid products. And looking ahead, we expect steady growth for these 2 medicines in line with historical trends.

Speaker 1

Taking all the strong dynamics in the business together, we expect net product sales to be $310,000,000 to $320,000,000 for 2024. This represents over 70% growth from 2023 with increased contribution from all commercially available medicines. In summary, 2023 was a fantastic year for RT as we expanded our portfolio to 3 commercial products and grew our presence both in the U. S. And worldwide.

Speaker 1

We continue to see the tremendous impact of all three products across their patient communities and we look forward to further expansion in 2024 as we stay committed to our mission of delivering life changing medicines to patients worldwide. And with that, I'll turn it over to Joanne. Joanne? Thanks, Peter.

Speaker 3

Before I begin, I'd like to say that I'm thrilled to join Europe. This experienced team has accomplished so much in such a short period of time, and I'm excited to continue the momentum and progress in the treatment of rare diseases. In 2024, we are looking forward to multiple regulatory and development milestones. From a regulatory perspective, we're tracking well towards our PFIC PDUFA date of March 13 and expect a decision from EMA in the first half of the year. Regarding Kenadol and CTX, we're making good progress here as well.

Speaker 3

We've had positive interactions with the FDA and no additional clinical studies are required to support our NDA submission, which is planned in the first half of twenty twenty four. Turning to valexibat, we're also looking forward to our upcoming interim analyses in our VISTAs PSC study and our VANTAGE study in PBC patients. We are on track for the interim analysis to read out in the first half of this year. Note that the studies are now continuing to enroll patients with the goal of supporting registration. As a reminder, the Visto's PSC study will have a blinded interim analysis to support dose selection.

Speaker 3

Based on the data, this will allow patients from the selected dose and placebo arms to be included in the pivotal data set. For the larger indication of PBC, the study is designed so that we unblind and show the top line results from an interim analysis, which will include data on pruritus improvement, change in serum bile acids and safety. Both of these studies represent an important step towards addressing the accumulation of bile acids in broader patient groups with adult full of phases where a significant portion of patients are without adequate treatment options for their cholestasis and a severe symptomatic burden. I'm excited to be part of this team and look forward to sharing our progress with VIVITR. With that, I'll now turn the call over to Eric to discuss our financial results.

Speaker 3

Eric?

Speaker 4

Thanks, John. Earlier today, we issued a press release that included financial results for the quarter full year, which I'll briefly summarize. Note our 10 ks filing is planned for next week as our auditors finalize the first 404 and other audit procedures for Miran. Total revenue in the Q4 2023 was $69,600,000 and for the year ended December 31, total revenue was $186,400,000 including total net product sales of $178,900,000 This is compared to total revenues of $27,900,000 $77,100,000 for the Q4 and full year 2022, respectively. For operating expenses for the year ended December 31 were $293,000,000 which includes research and development expenses of 102,600,000 SG and A expenses of $145,900,000 and cost of sales of 44,500,000 dollars For the year ended December 31, 2023, net loss was $160,900,000 or $3.94 per share.

Speaker 4

Net loss for the year included non cash stock based compensation expense of 35,000,000 and intangible amortization of $10,400,000 This intangible amortization is largely reflected in our cost of goods sold. In the Q4, COGS also reflected a reserve in excess of $5,000,000 for inventory, primarily related to the bile acid acquisition. For the year ended December 31, 2023, our cash used in operating activities was $70,900,000 down from $120,100,000 the year before. We had cash, cash equivalents and investments of $286,300,000 as of December 31, 2023. We remain well funded with a strong and self sustaining business.

Speaker 4

Our increasing revenue base places us in an exceptional position to grow the business through clinical development and continued expansion of our global commercial presence over the coming years. Now I'll turn the call back over to Chris for final comments.

Speaker 5

Thanks, Eric.

Speaker 2

It has been a strong year for Miram, finishing with the biggest ever revenue quarter for Lymph Marley and the bile acid portfolio. We are poised for another great year in 2024 with 3 growing commercial products and with several clinical and regulatory catalysts as we strive to make continued advances for rare disease patients around the world. We look forward to keeping you updated on our exciting progress throughout the year. And with that, operator, please open the call for questions.

Operator

We will now begin the Q

Speaker 6

This is Nick on for Jess. Congrats on the quarter and thanks for taking our questions. Maybe looking at the 2024 net product sales guidance of $310,000,000 to $320,000,000 Can you maybe discuss a bit more about how much of the Marley growth is baked in there versus contributions from Kitadong, Colmum? And also comment a little bit on Lebmarly and Elagil and maybe provide an update on what you're seeing on the competitive dynamics for BELLETE, if any?

Speaker 2

Yes. Thanks for the question. First, just from a starting point, we're not giving product level guidance at this point, but really great dynamics across the products. And I'll let Peter kind of give a little color on what we're seeing across the business.

Speaker 7

Yes. And I think as you think about the components and

Speaker 1

the color commentary there, the biologics product portfolio, we expect it to grow in line with historical trends, which have been kind of on the order of

Speaker 7

mid single digit year on year growth rates. And then looking into the Marley growth case in the U. S, which has been strong over the last over all the quarters of its marketing, expected to see it continue to move forward with that cadence. And then on the international side, we do expect quarter to quarter variability there, probably international sales. And then your other question about competitive dynamics now as you go, I think as you can see from the numbers here, it's really you haven't seen a measurable impact from those at all really the La Marlin growth trajectory hasn't been impacted by that.

Speaker 6

Great. And maybe just one more, thinking about the VANTAGE study and how it allows for patients both the first line setting, second line setting post EDCA. Beyond the rate of pruritus observed across these two settings, can you maybe talk a little bit more about the differences in how it presents and or maybe the severity of itch that is observed in these two patient populations?

Speaker 2

Joanne and I can speak to the difference between the lines therapy.

Speaker 3

Yes. So as you know, pruritus is a big issue in both of these diseases. We have a broad patient population that we're accessing in terms of the vocab studies. And actually there's this is broader than, for instance, some of the other entrants in this space who are looking at biochemical abnormalities. I'll remind you that we have agreement from FDA that pruritus is a registrational endpoint, and both of these studies are actually positioned to be registrational after the interim analysis.

Speaker 3

So we feel pretty confident in terms of both the study design, the drug itself, or dose selection and moving forward at this point.

Operator

The next question comes from the line of Gavin Clark Gardner with Evercore ISI. Please proceed.

Speaker 8

Hi, this is Yasha on for Gavin. Just two questions for us. For the first one, for Lid Marley, especially with the start of the year, just wondering if you have seen patients being reweighted and put on higher doses as they grow?

Speaker 7

Happy to do that.

Speaker 4

That's the question we have and

Speaker 7

we published some of that information last year. Not surprisingly, it's a rate based product.

Speaker 1

And then in the growth data

Speaker 7

that we've seen published with Marley, we have made you see weight based dose adjustments.

Speaker 8

Awesome. And then one more for the EU business. Do you have any commentary on potential implications of Ipsen refiling odevixibat odevixibat in the EU under a different brand name, not with orphan status? And then also want to confirm that there'd be no reimbursement or access impact on Wood Marley in the EU?

Speaker 2

Thanks for the question. At this point, we're focused on getting access broadly across all international markets for Lymphomarli and Alagille and having great progress as we see demand continuing to grow across Europe and other international markets. Can't really speak too much to the BELVE procedure, other than that we're making great progress getting LYMARLE. It really rolled out a standard of care in Alagille across all markets.

Speaker 8

Great. Thank you.

Speaker 2

Thanks for the question.

Operator

Thank you. The next question comes from the line of Mani Faruah with Leerink Partners. Please proceed.

Speaker 5

Hi, good afternoon. This is Lillian Songo on for Mani. I'm sorry, I couldn't hear, and apologies if it was asked earlier. I heard the question, but we couldn't hear the answer. This was regarding the growth expected for LIFO in terms of weight based increases as the patient get older.

Speaker 5

And to add to that, if you could comment in terms of how should we look how should we think about the way and then the dosage for new patients versus existing ones?

Speaker 2

Yes. So the question and just to make sure that I recap it here and ask for any color from Peter. Basically, the original question was around, do we see adjustments to dose as patients are on therapy over time, per the label. And we do see that over time. I think, Lutz mentioned that there's been a recent poster presentation on it.

Speaker 2

So that is a dynamic that we see out with the treating physicians. And so were you able to the microphone pick us up this time?

Speaker 5

Yes, we could hear you really clearly. Thank you. And as a follow-up regarding the Chinnabales Shonam assets, could you maybe comment in terms of what the international growth opportunities are for those assets?

Speaker 2

Yes. So question about Kenadol Coal Bomb Growth Opportunities. And I'll let Peter speak a little bit to the dynamic of what we have today and then also a little bit about the Kenadol label expectations expansion opportunity for CTX.

Speaker 1

Yes. With both products, we continue to see growth in line with historical trends, certainly with KEYNOTE

Speaker 7

and recently announced Phase 3 data for CTS and potential approval next year for CTS. I think there's an opportunity key adults never been promoted for CTS before. So I think there's an opportunity to find more patients through disease state awareness and hopefully increasing the diagnosis rate. One thing we know about CTS is maybe only about 10% of the patients are diagnosed and under management. So the opportunity there is

Speaker 1

to try to identify more patients and

Speaker 7

to them on therapy.

Operator

Thank you. Thank you.

Speaker 2

Thanks for the questions.

Operator

The next question comes from the line of Mike Coles with Morgan Stanley. Please proceed. Hi.

Speaker 9

This is Rohit on for Mike. Thanks for taking our questions. Can you just talk about your expectations for the upcoming LIV MARLY, PDUFA in PFIC and any launch prep that's associated? And do you think that any patients are currently using it off label? Thank you.

Speaker 2

Thanks for the question, Rohit. On the regulatory front, we're kind of where we expect to be, so excited about PDUFA date coming up. And I'll let Peter speak a little bit to how we're preparing for that and some of the first opportunities we see there.

Speaker 1

Yes. We're really excited about the potential approval for LiMARLE and PFIC.

Speaker 7

I think the Mars

Speaker 1

data reinforce the strong value proposition that LiMARLE

Speaker 7

offers to our various stakeholder groups. Physician prescribing universe is essentially identical to the physician prescribing universe is essentially identical to the AlloSure audience. So there's really no need for meaningful operating expense increased access.

Speaker 1

And we do have

Speaker 7

a number of patients in the mid-20s who are receiving clinical rollover or extended access, the barley, who would be eligible to roll over the commercial upon approval.

Speaker 9

Thank you.

Speaker 2

Thanks for the question.

Operator

The next question comes from the line of Steve Seedhouse with Raymond James. Please proceed.

Speaker 10

Yes. Hi. This is Timur Ivannikov on for Steve Seedhouse. So our first question is related to the gross margin. I think you mentioned there was an inventory charge related to the acquisition in 4Q.

Speaker 10

Just what are your expectations for gross margin in 2024 and perhaps also the OpEx trajectory in 2024?

Speaker 4

For us, gross margin, we do expect that the intangible amortization will continue. I mean, it's largely related to the bile acid acquisition intangibles, which we're amortizing over 50 quarters. As far as sort of traditional cost of goods, it consists of the actual cost of products and royalties. So that will continue at approximately the same sort of percentage of sales. And then we did have a larger than we expect going forward amortization or reserve for the inventory that came with the acquisition.

Speaker 4

So we might have some reserve in some quarters, but not to the extent we had in the Q4. In terms of overall operating expenses, R and D and SG and A, 4th quarter is probably pretty representative of what we expect the next few quarters.

Speaker 10

Okay. Thank you very much. And then just a question on your overall enrollment dynamics in VISTUS Advantage. I think in VISTUS, your interim is only on 45 patients. So can you comment about the overall enrollment target dates?

Speaker 10

And do you see any differences between the studies in terms of the enrollment dynamics?

Speaker 2

Thanks for the question. I mean, the simple answer here is we have patients in for the interim. We're continuing to enroll for Part 2 now. And we'll be able to give better guidance for the full study enrollment at that interim when it comes up.

Speaker 10

Okay. Appreciate it. Thank you very much.

Speaker 2

Yes. Thank you.

Operator

The next question comes from the line of David Lebowitz with Citi. Please proceed.

Speaker 11

Hi, this is Divanjana on for David. Thanks for taking our call. We wanted to ask about the blinded PSC interim analysis. So beside the dose selection, would you share any other data points at a blinded basis?

Speaker 3

So we will be blinded in this interim. And this is designed so that if it passes the threshold, it is designed to be predictive of a clinic meaningful and statistically significant positive pivotal analysis in the end. So that's all I can really share with you at this point. And it's really the threshold was developed based on what we know about the drug, this class of drugs, PSC in general and pruritus. So we feel pretty confident in terms of the design being robust and this being kind of well set up to produce a registration study for us.

Speaker 2

And in terms of communication at the point of interim for the VISTA study, assuming a positive interim, we would communicate that the interim has occurred and study continues as planned, which would be the extent of the information that we receive here on the team as well.

Speaker 11

Okay. So as a follow-up to that, could you tell us a bit more about I mean, while unlikely, is there any other scenario like that would play out if somehow like if you see that the efficacy is not crossing the threshold on a blind blinded basis that was pre specified?

Speaker 2

So the study design in the event where that threshold is not met basically converts to an open interim analysis. We'd be able to look at the data, share top line findings of it and what the next steps are for the study in that scenario.

Speaker 11

Okay. Thank you for the insight.

Speaker 2

Yes. Thanks for the questions.

Operator

Thank you. The next question comes from the line of Brian Skorney with Baird. Please proceed.

Speaker 9

Hi, Ann. On PBC beyond pruritus, how are you thinking about the impact on outcomes and other markers in Vantage? And how can this help you make the case to physicians and patients?

Speaker 2

Yes. Thanks for the question. I'll let Joanne speak a little bit to kind of the registrational plan for PBC.

Speaker 3

Yes. So in terms of the interim, we're just going to be looking at top line. So, pruritus, serum bile acid safety, as top line. And as you know, age is a big issue in this particular patient population for which there's really no particular therapies at this point. So we're pretty confident that this study, the way it's designed actually addresses an important unmet medical need at this point.

Speaker 2

Yes. And just to recap, one of the points made earlier, the eligibility criteria for the VANTAGE study does not have an alkaline phosphatase criteria. So it includes patients that are traditionally thought of as first line that have really only UDCA as on label and still experience the same rates and severity of pruritus as later lines of treatment. So a lot of unmet need across the PBC patient population.

Speaker 7

Great. Thanks.

Speaker 2

Thanks for the question.

Operator

Thank you. The next question comes from the line of Ed Arce with H. C. Wainwright. Please proceed.

Speaker 12

Hi, everyone. Thanks for taking my questions and congrats on another strong quarter. I have 3, if I may. Firstly, on the VANTAGE study in PBC, as you said, it's a blinded data readout. Obviously, this is, as you mentioned, basically pruritus versus placebo and whether it's stat sig.

Speaker 12

But I'm also wondering if there are specific thresholds of activity on pruritus that you're looking at, not just so much to proceed, but also as you think about the competitive landscape and where you'd like to see that come in? Secondly, if you could speak a bit about the PFIC opportunity in terms of 2024, sort of the sales, the speed of the sales ramp, kind of what you're expecting the trajectory to be through the remaining quarters of this year? And then lastly, I'm sorry, I didn't hear when Peter was reviewing, I think it was Peter, the numbers for LIV Marley, both U. S. And international as well as bile acid products for the Q4.

Speaker 12

Thanks so much.

Speaker 2

Great. Thanks for the question. Maybe I'll just hit on recap one of the points on Vantage and then pass it over Peter to talk about PFIC and recap some of those sales numbers. And the way we think about Vantage and valexovat and PVC, it really comes down to this the highlight here on lines of therapy and where various agents are labeled and expected to be labeled. The majority of patients are first line patients and the majority of them have pruritus.

Speaker 2

So that's really the primary unmet need that we're going after. So showing a significant impact on that pruritus, we think will be very clinically meaningful. And that's the lens that we're going to be taking towards that interim data when we look at it and make a decision on next steps and continuing to roll into the pivotal portion of the study. With that, I'll pass over to Peter on the next two points.

Speaker 7

Yes. Sorry, I'll try to speak up a little bit here.

Speaker 1

So on the PFIC 2024 revenue expectation piece, I think we'll be upon a potential approval here in March, we'll

Speaker 2

be spending a fair bit

Speaker 1

of time working closely with payers get LiDARLE positioned in their new to market policies. So I think if you think about 2024, we'd expect a lot of free drug or PAP with PFIC really coming in more in 2025, somewhat similar to what we saw in the Alagille initial launch where the early quarters were high PAP drug. And then in terms

Speaker 4

of the

Speaker 1

numbers from Q4, LIDMARLE totaled $41,400,000 worldwide, dollars 31,400,000 US10 International and then bile acid products were $28,100,000 for Q4.

Speaker 12

Great. And then maybe just further clarification on PFIC. Appreciate the comments. Just wondering given that you mentioned earlier there's about 20 ish patients right now on expanded access. How soon would you expect those to be able to roll over onto commercial drug once again?

Speaker 1

Yes. What we saw in the Alagille launch is that occurred in a quarter or 2 generally is what we saw. And so, yes, I think that's probably consistent with our expectation for those 25 or so.

Speaker 12

Thanks so

Speaker 2

much. Thanks for the questions.

Operator

Thank you. The next question comes from the line of Jonathan Willoughby with Citizens JMP. Please proceed.

Speaker 11

Hi, this

Speaker 5

is Catherine on for John. I just had 2 kind of quick questions. One time to kind of follow-up on valexibat and just kind of the how you guys see it fitting into the PBC paradigm in the setting of other approved agents that might potentially be effective also on pruritus, whether it's an excuse to see as kind of the combo therapy, sort of a monotherapy for earlier patients? And then also, just as far as preparing for the PFIP launch, I know you said that there's not going to be too much of an investment as far as SGA goes. But like what else is kind of being done to sort of get the word out there about PFIC or do the most of these physicians already know that it's coming?

Speaker 2

Thanks for the questions. And just to kind of recap the PBC positioning one more time here. The VANTAGE study includes first line patients that really are when you look at the eligibility criteria for the PPAR programs, for example, and how basafibrates gets used outside the U. S, we are upstream of that setting where when a patient is biochemically controlled, they can still have substantial pruritus, right? That's the area where IVAC can play a very unique role in being in front of the other second line therapies, the PPARs and OCA, for example.

Speaker 2

So pretty unique positioning that we see for PBC. I'd add on, in PSC, really exciting opportunity here where valexovat is positioned to be the first and only therapy for PSC by using pruritus as an endpoint really gives an outcome to use for bringing a new therapy to these patients. So quite excited about the positioning there. And pass over to Peter for part 2 here.

Speaker 1

Yes. In regards to BPIC, obviously, there's been there's no promotion until an approval. But I mean, I just kind of emphasize that it's a really small number of physicians that take care of these patients in the U. S. I mean, you're probably talking about on the order of 100 physicians in the entire country that take care of PFIC patients.

Speaker 1

There have been presentations at the major medical conferences on the data, Phase

Speaker 7

3 data as well as

Speaker 1

the Phase 2 data going back for years. So I think there's a kind of a relatively high awareness of the data. And certainly, from the Alagille approval and commercialization, a lot of comfort with Marley's clinical profile, kind of familiarity with using it, prescribing it, working with our patient support hub, the Miramax's Plus program, co pay support, etcetera. So I think that's probably a fair summary of where things stand today.

Speaker 5

Thanks so much.

Speaker 2

Thanks for the questions.

Operator

Thank you. There are no additional questions at this time. I will pass it back to Chris Peetz for any closing remarks.

Speaker 2

Great. Thanks again for everyone for joining us for today. During the call, I did want to put a plug in for Rare Disease Day tomorrow. Today, to build awareness for the more than 7,000 rare diseases impacting patients around the world and celebrate some of the meaningful advances in research that we've seen in recent years. Tomorrow, as part of Rare Disease Day, we're excited to be able to share a manuscript published in hepatology featuring long term data and patients with Alagille syndrome treated with Leimarley.

Speaker 2

So please join us in supporting the many advocacy groups that passionately lead efforts to support patients in research in rare disease tomorrow. And have a great evening. Goodbye.

Operator

That concludes today's conference call. Thank you. You may now disconnect your line.

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Key Takeaways

  • Mirum reported $179 million in net product sales for 2023, a 142% year-over-year increase, and expects $310–$320 million in 2024 net product revenue driven by its three commercial medicines.
  • The company achieved $69.6 million in Q4 net product sales—LIVMARLI at $41.4 million (63% US growth) and the newly integrated bile acid products (KEYDOL and POLBOND) at $28.1 million—demonstrating successful acquisition integration.
  • Key 2024 catalysts include a March 13 PDUFA decision for LIVMARLI in PFIC, first-half NDA submission for Kenadol in CTX, and interim analyses from valoxibat trials in PSC and PBC to support dose selection and registration.
  • Despite a 2023 net loss of $160.9 million ($3.94/share), Mirum improved cash usage—operating cash burn fell to $70.9 million—and ended the year with $286.3 million in cash, cash equivalents and investments.
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Earnings Conference Call
Mirum Pharmaceuticals Q4 2023
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