Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) Q4 2023 Earnings Call Transcript

Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) Q4 2023 Earnings Call Transcript February 28, 2024

Mirum Pharmaceuticals, Inc. misses on earnings expectations. Reported EPS is $-0.66 EPS, expectations were $-0.33. MIRM isn’t one of the 30 most popular stocks among hedge funds at the end of the third quarter (see the details here).

Operator: Good afternoon, ladies and gentlemen. Thank you for joining today’s Mirum 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 question-and-answers at the end. [Operator Instructions] It is my pleasure to pass the call over to Andrew McKibben, Vice President of Investor Relations and Finance. Please proceed.

Andrew McKibben: Thanks, Tia and good afternoon, everyone. I’d like to welcome you to Mirum Pharmaceuticals fourth quarter 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, Pam Vig; our Chief Medical Officer, Joanne Quan; and Eric Bjerkholt, our Chief Financial Officer. Earlier today, Mirum issued a news release announcing the company’s results for the fourth quarter and full year 2023. Copies of this news release and SEC filings can be found in the Investors section of our website. 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 Mirum, 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.

Mirum 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 Mirum’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?

Chris Peetz: Thanks, Andrew and good afternoon, everyone. I’m excited to kick off an update on the many achievements for Mirum in 2023, another year of significant growth for us and cover highlights for what lies ahead for the company. Our continued progress in 2023 an opportunity ahead, reflects the dedication of the Mirum 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 US and internationally achieving $179 million in net product sales and 142% year-over-year growth in total revenue. This was driven by continued strong growth for LIVMARLI and the expansion of our commercial portfolio with the acquisition and integration of CHENODAL and CHOLBAM.

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 million to $320 million of net product revenue in 2024. This is expected to be driven by growth across all three commercially available medicines. We also have multiple regulatory and clinical catalysts this year to advance our pipeline.

For LIVMARLI 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 March PFIC Phase III study to patients with a potential label expansion. We’re also preparing for the submission of the positive RESTORE Phase III results of CHENODAL and CTX in the first half of the year an important label-enabling opportunity. And we also made great progress with the VISTAS and VANTAGE studies for volixibat in PSC and PBC. Patients for the interim analysis have been enrolled and we expect to conduct the 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 Mirum. We look forward to continuing to grow the impact of our programs providing life-changing medicines to patients with rare disease. Now before I turn the call over to Peter to discuss our commercial business, I wanted to welcome our new Chief Medical Officer, Dr. Joanne Quan who joined Mirum in January. We are thrilled to have Joanne and her extensive leadership and scientific expertise helping to drive the value creation we see ahead from Mirum. 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. Peter?

Peter Radovich: Thanks Chris. We are excited by the tremendous progress that we have made across our US 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 Mirum team’s focus on patients while simultaneously driving the seamless integration of CHOLBAM and CHENODAL and this is evident in the $69.6 million of total net product sales we achieved in the fourth quarter of 2023, which reflected growth across all three commercial medicines. For LIVMARLI total global net product sales grew to $41.4 million in the fourth quarter.

Our US business saw $31.4 million and $111 million for the quarter and year respectively, representing approximately 63% year-over-year growth. And internationally we are seeing consistent strong uptake as well, but we continue to anticipate quarter-to-quarter variability in international revenue. Moving on to CHENODAL and CHOLBAM. The fourth quarter was the first full quarter with these two medicines under Mirum’s leadership, and I’m very happy with how quickly we have been able to transition these medicines into our business. In the fourth quarter, we recognized net product sales of $28.1 million, the highest quarter to-date for the bile acid products. And looking ahead we expect steady growth for these two medicines in line with historical trends.

Taking all the strong dynamics in the business together, we expect net product sales to be $310 million to $320 million 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 our team as we expanded our portfolio to three commercial products and grew our presence both in the US and worldwide. 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?

Joanne Quan: Thanks Peter. Before I begin, I’d like to say that I’m thrilled to join Mirum. 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 are tracking well towards our PFIC PDUFA date of March 13th and expect a decision for the EMA in the first half of the year. Regarding CHENODAL and CTX we’re making good progress here as well. 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 2024.

Turning to Volixibat. We’re also looking forward to our upcoming interim analysis in our VISTAS PSC study and our VANTAGE study in PBC patients. We are on track for the interim analysis to readout 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 VISTAS PSC study will have a Blinded Interim Analysis to support Dose Selection. Based upon 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 the 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 Cholestasis where a significant portion of patients are without adequate treatment options for their Cholestasis and its Severe Symptomatic Burden. I’m excited to be part of this team. And look forward to sharing our progress to you this year. With that, I’ll now turn the call over to Eric, to discuss our financial results. Eric?

Eric Bjerkholt: Thanks, Joanne. Earlier today we issued a press release that included financial results for the quarter and full year, which I’ll briefly summarize. Note, our 10-K filing is planned for next week as our auditors finalize the first 404(b) and other audit procedures for Mirum. Total revenue in the fourth quarter 2023 was $69.6 million. And for the year ended December 31st, total revenue was $186.4 million including total net product sales of $178.9 million. This is compared to total revenues of $27.9 million and $77.1 million for the fourth quarter and full year 2022, respectively. For operating expenses for the year ended December 31st were $293 million which includes research and development expenses of $102.6 million, SG&A expenses of $145.9 million and cost of sales of $44.5 million.

For the year ended December 31st 2023, net loss was $160.9 million or $3.94 per share. Net loss for the year included non-cash, stock-based compensation expense of $35 million and intangible amortization of $10.4 million. This intangible amortization is largely reflected in our cost of goods sold. In the fourth quarter COGS also reflected a reserve in excess of $5 million for inventory, primarily related to the Bile Acid acquisition. For the year ended December 31st 2023, our cash used in operating activities was $70.9 million, down from $120.1 million the year before. We had cash, cash equivalents and investments of $286.3 million as of December 31st 2023. We remain well funded with a strong and self-sustaining business. 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.

Chris Peetz: Thanks Eric. It has been a strong year for Mirum, finishing with the biggest ever revenue quarter for LIVMARLI and the Bile Acid portfolio. We are placed for another great year in 2024 with three 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.

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Q&A Session

Operator: We would now being the Q&A session [Operator Instructions] The first question comes from the line of Jessica Fye with JPMorgan. Please proceed.

Unidentified Analyst: Hey. This is Nick on for Jess. Congrats on the quarter. And thanks for taking our question. Maybe looking at the 2024 net product sales guidance of $310 million to $320 million can you maybe discuss a bit more about how much LIVMARLI growth is baked in there versus contributions from CHENODAL and CHOLBAM? And also, comment a little bit on LIVMARLI and Alagille maybe provide an update on what you’re seeing on the competitive dynamics for Bylvay if any?

Chris Peetz: Yeah. Thanks for the question. And 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.

Peter Radovich: Yeah. And I think as you think about the component in some of the color commentary there, the bile acid product portfolio we expect it to grow on historical trends, which have been kind of on the order of mid-single-digit year-on-year growth rates. And then looking into LIVMARLI growth case in the US has just been strong over the last overall quarters of its marketing, expected to see it continue to move forward with that team. And then on the international side, we do expect quarter-to-quarter variability there for the LIVMARLI international sales. And then your other question about competitive dynamics in Alagille. I think as you can see from the numbers here it’s really — we haven’t seen a measurable impact from those at all the LIVMARLI growth trajectory hasn’t been impacted by that.

Unidentified Analyst: Great. And maybe just one more. Thinking about the VANTAGE study and how do Alagille patients go to first-line setting second-line setting post CDCA 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?

Chris Peetz: Joanne and I can speak to the difference between the line therapy.

Joanne Quan: Yeah. 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 volixibat 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 provide us with the registrational endpoint and both of these studies are actually positioned for registration after the interim analysis. So we feel pretty confident in terms of both the study design the drug itself our dose selection and moving forward at this point.

Unidentified Analyst: Great. Thanks.

Chris Peetz: Thanks for the questions.

Operator: The next question comes from the line of Gavin Clark-Gartner with Evercore ISI. Please proceed.

Unidentified Analyst: Hi. This is Yesha on for Gavin. Just two questions for us. For the first one for LIVMARLI especially with the start of the year just wondering if you have seen patients being reweighted for higher doses as they grow?

Peter Radovich: Happy to take the question. We have published some of that information last year. Not surprisingly it is a weight-based product. And the growth that we’ve seen published with LIVMARLI I think we have — we did see weight-based dose adjustments.

Unidentified Analyst: All right. And then one more for the EU business. Do you have any commentary on potential implications of ITCH and refiling odevixibat in EU under a different brand name not with orphan status? And then I also want to confirm that there would be no reimbursement or access impact on LIVMARLI in the EU?

Chris Peetz: Yes. Good question. At this point, we’re focused on getting access broadly across all international markets for LIVMARLI 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 Bylvay procedure. Other than that, we’re making great progress getting LIVMARLI really rolled out as standard of care in Alagille across all markets.

Unidentified Analyst: Great. Thank you.

Chris Peetz: Thanks for the question.

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

Lili Nsongo: Hi. Good afternoon. This is Lili Nsongo on for Mani. I’m sorry I couldn’t hear — and I apologize, if it was asked earlier, I heard a question, but we couldn’t hear the answer. This was regarding the growth expected for LIVMARLI in terms of weight-based increases as the patients get older. And to add to that if you could comment in terms of how should we look — or should we think about the weight and then the dosage for new patients versus existing ones?

Chris Peetz: Yeah. So the question — and just to make sure that I recap it here and ask for any color from Peter. Basically, the question there — the original question was around, do we see adjustments to dose as patients that are on therapy over time through the label. And we do see that over time. I think we mentioned that there’s been a recent poster presentation on it. So that is a dynamic that we see out with the treating physicians. And so we’re able to — did the microphone pick us up this time?

Lili Nsongo: Yeah. Yeah, we could hear you very clearly. Thank you. And as a follow-up regarding the volixibat asset, could you maybe comment in terms of what the international growth opportunities are for those assets?

Chris Peetz: Yeah. So a question about CHENODAL CHOLBAM 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 CHENODAL label expectations expansion opportunity for CTX.