Mirum Pharma (MIRM) Transcription de l'appel sur les résultats

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DATE
Tuesday, Nov. 4, 2025 at 8 a.m. ET
CALL PARTICIPANTS
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Chief Executive Officer — Christopher Peetz
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President and Chief Operating Officer — Peter Radovich
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Chief Medical Officer — Joanne M. Quan
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Chief Financial Officer — Eric Bjerkholt
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Full Conference Call Transcript
Christopher Peetz: Thanks, Andrew, and good afternoon, everyone. 2025 continues to be an outstanding year for Mirum. We've created a leading rare disease company, purpose-built to create and deliver life-changing medicines to patients. Our success comes from that foundation. The team deeply connected to patients and families, turning their insights into meaningful therapies and measurable performance. In the third quarter, we delivered strong commercial results, advanced our clinical pipeline and strengthened our financial foundation. I'm proud of the way our team continues to execute with focus and consistency. First, on commercial performance. We reported third quarter revenue of $133 million, representing a nearly 50% year-over-year increase over the same period last year.
This performance reflects the strength and breadth of our commercial portfolio, including continued momentum from the U.S. PFIC launch and expanding demand from our international markets. Turning to R&D. We remain on track for three potentially pivotal readouts over the next 18 months. First up is the VISTAS Phase IIb study in PSC. With enrollment complete, we expect to announce top line data in the second quarter of 2026. With the successful interim analysis last year and a consistent body of supporting data with IBAT inhibitors across multiple cholestatic diseases, we're optimistic about volixibat's potential to become the first approved treatment in this setting.
We are also progressing well with our VANTAGE study of volixibat in PBC, the EXPAND study of LIVMARLI in ultra-rare cholestatic conditions, and our newly initiated Phase II study of MRM-3379 in Fragile X syndrome. We've also taken meaningful steps to further strengthen our financial performance. This quarter, our cash balance grew significantly, and we recognized positive net income for the first time. This is an important milestone that highlights the operating leverage in our commercial model. In sum, it's been another solid quarter of execution for Mirum. I want to thank the entire Mirum team for their continued dedication to patients.
We built a high-growth cash flow-positive rare disease company with a broad pipeline and global footprint, and we're just getting started. And with that, I'll hand the call over to Peter. Peter?
Peter Radovich: Thanks, Chris. Q3 was another excellent quarter for Mirum with total net product sales of $133 million. This was driven by continued robust performance of LIVMARLI in both the U.S. and international markets as well as steady contribution from our bile acid portfolio. LIVMARLI net product sales totaled $92 million for the quarter. In the U.S., LIVMARLI demand remains healthy in both Alagille syndrome and PFIC with $64 million in net product sales. Alagille syndrome growth remains durable, and PFIC continues to contribute meaningfully, reflecting the real-world benefit of expanded diagnosis and increased genetic screening.
As we begin reaching into broader segments of the medical community, particularly adult-focused providers, we're finding that genetic testing is still less embedded in practice and often requires more education and dialogue. So we view this as an area where sustained engagement can continue to drive incremental gains. Internationally, LIVMARLI demand continues to grow with $28 million in net product sales this quarter. Demand across our direct and partner markets remains robust, supported by expanding reimbursement and launches in new geographies. Q3 was the first full quarter of commercialization for our partner, Takeda in Japan, with in-market adoption dynamics generally consistent with LIVMARLI's U.S. launch.
Our Bile Acid Medicines, CHOLBAM and CTEXLI generated $41 million in net product sales this quarter, supported by increased CTX patient finding following CTEXLI's FDA approval earlier this year. And I'm happy to say that we now expect to land in the upper end of our prior full year 2025 guidance range with $500 million to $510 million in revenues. This reflects the continued strength of our U.S. business in both Alagille syndrome and PFIC, steady contributions from our bile acid portfolio, along with the typical quarter-to-quarter variability in international partner and distributor ordering patterns. Looking ahead, we continue to see substantial growth potential across our portfolio with peak revenue potential for LIVMARLI, volixibat and MRM-3379 each exceeded $1 billion.
And then with that, I'll turn it over to Joanne for an update on the pipeline. Joanne?
Joanne M. Quan: Thanks, Peter. I'm pleased to provide an update on the continued progress across our clinical pipeline, where we're seeing continued collaboration and momentum with physicians and patients across all of our ongoing studies. Starting with volixibat, we completed enrollment in the Phase IIb VISTAS study in primary sclerosing cholangitis, or PSC, and expect to announce top line data in the second quarter of 2026. PSC represents a significant area of unmet need with no approved therapies and limited treatment options. We're deeply grateful to the investigators and the PSC patient community for their partnership in advancing this important study. As a reminder, the outcome of the interim analysis of the VISTAS study last year was what we'd hoped for.
The recommendation was to keep the current sample size, which we believe reflects a strong signal for the final analysis. It's worth noting that the study was powered using conservative assumptions, a placebo-adjusted treatment effect of 1.75 points and standard deviation of 3. A case series is being presented at AASLD of 8 PSC patients treated with maralixibat under our compassionate use program, a continuation of a case series presented earlier this year at DDW. All of these patients had meaningful reductions in pruritus and 4 of the 8 had complete resolution. This data supports the role for IBAT inhibition as a treatment for PSC. Turning to PBC.
The VANTAGE study continues to progress well, and we expect to complete enrollment next year. Interim data presented last year demonstrated statistically significant improvement in pruritus, meaningful reductions in serum bile acids and encouraging improvements in fatigue. We're excited to advance this study through the confirmatory stage. Additional analyses from the VANTAGE interim will be presented at AASLD, which highlights the decreases in fatigue and improvement in sleep in volixibat patients as well as showing a decrease in IL-31 in treated patients. Our EXPAND study evaluating LIVMARLI in additional settings of cholestatic pruritus is also enrolling well. This study is designed to broaden access to patients across multiple rare cholestatic diseases who currently have few or no treatment options.
It represents a meaningful label expansion opportunity, and we're targeting enrollment completion in 2026. Finally, I'm excited to share that we've initiated our Phase II study of MRM-3379, our brain-penetrant PDE4D inhibitor for Fragile X syndrome. The preclinical data we recently presented from a mouse FMR1 knockout model of Fragile X, showed that MRM-3379 reversed the disease phenotype across multiple behavioral assessments and increases our confidence in the importance of this pathway in Fragile X. Overall, we're very encouraged by the progress across our development programs and look forward to upcoming milestones in 2026. With that, I'll turn the call over to Eric to discuss our financial results. Eric?
Eric Bjerkholt: Thanks, Joanne, and good afternoon, everyone. We delivered another solid quarter of financial performance, highlighted by total net product revenue of $133 million, representing a 47% increase over the prior year and reflecting growth across all our commercial medicines. This quarter included approximately $5 million in sales to our partner, Takeda in Japan. We do not expect additional sales to Takeda in Q4 of this year. Total operating expense for the quarter ended September 30 was $130 million, which includes R&D expense of $43 million, SG&A expense of $62 million and cost of sales of $26 million. Expenses for the quarter included noncash stock-based compensation expense of $18 million and intangible amortization and other noncash items of $6 million.
The intangible amortization and other noncash items expense are largely reflected in our cost of sales. Our cash operating margins continued to improve, and we delivered GAAP profitability in the third quarter, generating approximately $3 million in net income. While this reflects the strength and scalability of our business model, we view quarterly GAAP profitability as a milestone, not yet a consistent expectation as we continue to invest in growth. Cash, cash equivalents and investments were $378 million at September 30, an $85 million increase from the beginning of the year. We continue to be well funded and financially independent, providing us the resources required to expand our patient impact and grow our business.
With that, I'll turn the call back to Chris.
Christopher Peetz: Thanks, Eric. Before we open the call for questions, I want to close by reflecting on what's been an incredibly productive quarter. Across every dimension of our business, commercial, clinical and operational, we continue to execute with purpose and discipline, anchored by the same patient-centric approach that's driven our success from the start. That's what's enabled us to become a high-growth cash flow positive, leading rare disease company. Thanks again to the Mirum team and to the patients and families who inspire our work every day. With that, operator, please open the call for questions.
Operator: [Operator Instructions] Our first question will be from the line of Jessica Fye with JPMorgan.
Abdulqudus Tahlil: This is Abdul on for Jess. We just have two questions. What are going to be the key drivers of LIVMARLI's performance as we look ahead to 2026? And can you talk about why the midpoint of the new guidance range now implies 4Q reps flat sequentially from 3Q? I don't think we saw that dynamic last year.
Christopher Peetz: Abdul, thanks for the question. On key drivers into 2026, I mean, we see a lot of basically what we have today rolling forward into next year. We expect that we'll probably give guidance early in the year next year on what that year looks like. But we are in early innings of the PFIC launch, both in the U.S. and internationally. So expect that to continue to build in over time. And I think for the guidance this year for Q4, maybe I ask Peter to speak to what we see from kind of the quarter-to-quarter dynamics...
Peter Radovich: Thanks, Chris. Yes, and I appreciate the question, Abdul. The main dynamic is tried to highlight in our prepared remarks. We see growth for LIVMARLI U.S. We see the bile acid portfolio continuing to do what it does. It's really the LIVMARLI international line where we expect variability as we move quarter-to-quarter. As we've talked about before, that business has periodic large orders from distributors, and we saw those come in, in Q3. We also mentioned that we had Takeda revenue in Q3, which we also had Q1 and Q2 that we don't expect in Q4. So there's a fair bit of an inventory build there. So that's really the dynamic that's in the LIVMARLI international line.
Operator: The next question will be from the line of Josh Schimmer with Cantor.
Joshua Schimmer: Maybe I have two quick ones. First, what trends are you seeing in terms of adoption of the solid tablet formulation of LIVMARLI? And what percent of sales are for that versus the liquid? And then for volixibat, what are you thinking in terms of the appropriate price analogs, especially after we've seen a significant increase in rare orphan disease prices perhaps over the last year, particularly for conditions that perhaps are less prevalent than PBC and more aligned with PSC.
Peter Radovich: Thanks for the questions, Josh. So in terms of the solid tablet, just launched in the U.S. in mid-June. So this is really our first full quarter with it. And what we've seen a very encouraging kind of uptake and really switches from the liquids. So if you look at the prescribing information, patients are eligible to switch if they're at least 25 kilos. I think what I could say is that a substantial proportion of those who are eligible based on their weight, are switching. So certainly excited about what that can mean long term in terms of persistence and adherence and an easier single tablet per dose format that will be preferred by these adolescents and adults.
So excited about that dynamic. And then yes, volixibat pricing. Obviously, I haven't made a final decision there, monitored those dynamics that you're talking about. We've kind of base case thinking, you can look at the other -- PPARs and the other products that are kind of approved in PBC at 130 to 150, but we're still analyzing. I think it's kind of too early to say what the right pricing strategy is for volixibat.
Operator: The next question will be from the line of Gavin Clark-Gartner with Stifel.
Gavin Clark-Gartner: Just had one. What's your expectation for Paragraph IV filers? Maybe just helpful to lay out your confidence in your whole IP portfolio, especially around the method patents and including volixibat.
Christopher Peetz: Gavin, thanks for the question. Overall, I mean, we're in the window where we could potentially see that and kind of all routine for this point in the life cycle for LIVMARLI, and really quite confident in our overall IP position. In particular, you mentioned the method patents that are specific to dosing of LIVMARLI in these indications. So we've seen this has been really the key fundamental observation that's made all of Mirum possible and the IP behind it, we see is quite strong and in a great position and prepared to defend it. So more to come if and when we do see any filers, but nothing to date.