Q3 2025 Mirum Pharmaceuticals Inc Earnings Call & Buiness Update
Speaker #1: Hello and welcome to the Mirum Pharmaceuticals, Inc. third Quarter 2020 financial results and business update . My name is Harry and I'll be your operator today .
Speaker #1: All lines are currently in listen only mode and there will be an opportunity for Q&A after management's prepared remarks . To enter the queue questions , please press star followed by one on your telephone keypad .
Speaker #1: I would now like to hand the conference over to Andrew McKibben , SVP of Strategic Finance and Investor Relations . Please go ahead .
Speaker #2: Thanks , Harry , and good afternoon , everyone . I'd like to welcome you to Mirum Pharmaceuticals, Inc. Third Quarter 2025 conference call .
Speaker #2: I'm joined today by our CEO , Chris Peetz , our President and Chief Operating Officer , Peter Radovich . Our Chief Medical Officer , Joanne Quan .
Speaker #2: And Eric Bjerkholt, our Chief Financial Officer. Earlier today, Mirum issued a news release announcing the company's results for the third quarter of 2025.
Speaker #2: Copies of this news release and SEC filings can be found in the investors section of our website . Before we start , I'd like to remind you that during the course of this conference call , we will be making certain forward looking statements based on management's current expectations , including statements regarding Miriam's program and market opportunities for its approved medicines and product candidates .
Speaker #2: These statements represent our judgment and knowledge of events as of today and inherently involve risks and uncertainties that may cause actual results to differ materially from the results discussed .
Speaker #2: We are under no duty to update these statements . Please refer to the risk factors in our latest form 10-q and subsequent SEC filings .
Speaker #2: For more information . With that said , I'd like to turn the call over to Chris . Chris . Thanks , Andrew , and good afternoon , everyone .
Speaker #2: 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 .
Speaker #2: Our success comes from that foundation , a team deeply connected to patients and families , turning their insights into meaningful therapies and measurable performance .
Speaker #2: In the third quarter , we delivered strong commercial results , advance our clinical pipeline , and strengthened our financial foundation . I'm proud of the way our team continues to execute with focus and consistency .
Speaker #2: 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 .
Speaker #2: This performance reflects the strength and breadth of our commercial portfolio , including continued momentum from the US launch and expanding demand from our international markets .
Speaker #2: Turning to R&D , we remain on track for three potentially pivotal readouts over the next 18 months . First up is the Vistas Phase two study in PSC with enrollment complete , we expect to announce top line data in the second quarter of 2026 successful interim analysis last year and a consistent body of supporting data with Ibat inhibitors across multiple cholestatic diseases .
Speaker #2: We're optimistic about the potential to become the first approved treatment in this setting. We're also progressing well with our Vantage study of Elizabet and PDC.
Speaker #2: The expand study of Livmarli and Ultrarare Cholestatic conditions , and our newly initiated phase two study of MRM 3379 . In fragile X syndrome .
Speaker #2: 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 .
Speaker #2: This is an important milestone that highlights the operating leverage in our with a commercial model . Some it's been another solid quarter of execution for Mirum .
Speaker #2: I want to thank the entire Mirum team for their continued dedication to patients . We've built a high growth cash flow positive rare disease company with a broad pipeline and global footprint , and we're just getting started .
Speaker #2: And with that , I'll hand the call over to Peter . Peter . Thanks , Chris .
Speaker #3: Q3 was another excellent quarter for mirror with total net product sales of 133 million . This was driven by continued robust performance of Livmarli in both the US and international markets , as well as steady contribution from our bile acid portfolio .
Speaker #3: Livmarli net product sales totaled 92 million for the quarter in the US . Livmarli demand remains healthy in both Alagille syndrome and with 64 million in net product sales .
Speaker #3: Alagille syndrome growth remains durable and 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 , were finding that genetic testing is still less embedded in practice and often requires more education and dialogue .
Speaker #3: 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 .
Speaker #3: 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 the Marlies , US launch .
Speaker #3: Our bile acid medicines , Cholbam and CTC generated 41 million in net product sales . This quarter , supported by increased CHT patient finding following FDA approval earlier this year .
Speaker #3: 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 to $510 million in revenues .
Speaker #3: 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.
Speaker #3: Looking ahead , we continue to see substantial growth potential across our portfolio , with peak revenue potential for Livmarli , Volixibat and MRM 3379 each exceeding 1 billion .
Speaker #3: And with that , I'll turn it over to Joanne for an update on the pipeline . Joanne .
Speaker #2: Thanks , Peter .
Speaker #4: 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 .
Speaker #4: Starting with Volixibat . We completed enrollment in the phase two B Vista 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 .
Speaker #4: 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 Vista study last year was what we'd hoped for .
Speaker #4: 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 .
Speaker #4: A placebo-adjusted treatment effect of 1.75 points and a standard deviation of 3. A case series is being presented at AASLD of eight PSC patients treated with Maralixibat under our Compassionate Use program.
Speaker #4: A continuation of a case series presented earlier this year at D.W. All of these patients had meaningful reductions in pruritus, and four of the eight had complete resolution.
Speaker #4: This data supports a role for IBD inhibition as treatment for PSC . Turning to PBC , the vantage study continues to progress well , and we expect to complete enrollment next year .
Speaker #4: 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.
Speaker #4: Additional analyses from the vantage interim will be presented at aasld , which highlights the decreases in fatigue and improvement in sleep in patients , as well as showing a decrease in IL 31 .
Speaker #4: In treated patients . For expense study evaluating Livmarli and additional settings . Of cholestatic pruritus is also enrolling well . This study is designed to broaden access to patients across multiple rare diseases who currently have few or no treatment options .
Speaker #4: 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 two study of MRM 3379 .
Speaker #4: Our brain-penetrant PDE 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.
Speaker #4: 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.
Speaker #4: Eric .
Speaker #5: Thanks , John , 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 .
Speaker #5: And reflecting growth across all our commercial medicines . This quarter included approximately 5 million in sales through our partner Takeda in Japan . We do not expect additional sales to techera in Q4 of this year .
Speaker #5: Total operating expense for the quarter ended September 30th was 130 million , which includes R&D of 43 million . G&A expense of 62 million , and cost of sales of 26 million .
Speaker #5: Expenses for the quarter included non-cash stock based compensation expense of 18 million , and intangible amortization and other non-cash items of 6 million .
Speaker #5: The intangible amortization and other non-cash items expense are largely reflected expense in our cost of sales . Our cash operating margin continued to improve , and we delivered GAAP profitability in the third quarter , generating approximately 3 million in net income .
Speaker #5: While this reflects the strength and scalability of our business model with you . Quarterly GAAP profitability as a milestone not yet a consistent expectation as we continue to invest in growth .
Speaker #5: Cash , cash equivalents and investments were 378 million at September 30th and 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 .
Speaker #5: With that , I'll turn the call back to Chris . Thanks .
Speaker #2: 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 .
Speaker #2: That's what's enabled us to become a high growth cash flow positive , leading rare disease company . Thanks again to the team and to the patients and families who inspire our work every day .
Speaker #2: With that , operator , please open the call for questions .
Speaker #1: Thank you . Now , opening the call , if you would like to ask a question , please press star One on your telephone keypad .
Speaker #1: If you change your mind and would like to exit the queue , please press star followed by two . And finally , when preparing to ask your question , please ensure that your phone is unmuted locally .
Speaker #1: Our first question will be from the line of Jessica FYE with J.P. Morgan . Please go ahead . Your line is open .
Speaker #6: Hey , this Abdul on for Jess . We just had two questions . What are going to be the key drivers of Marley's performance ?
Speaker #6: As we look ahead to 2026 ? And can you talk about why the midpoint of the new guidance range now implies for Q flat sequentially from three Q ?
Speaker #6: I don't think we saw that dynamic last year. Thanks.
Speaker #2: Abdul , thanks for the thanks for the question . On key drivers in the 2026 . I mean , we see a lot of basically what we have today rolling forward into next year .
Speaker #2: 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 launch , both in the US and internationally .
Speaker #2: So expect that to continue to build in over time . And I think for the guidance this year and for Q4 , maybe , Peter , to speak to what we see from the quarter to quarter dynamics .
Speaker #2: Yeah . And then . guidance .
Speaker #3: Thanks , Chris . Yeah . Appreciate the question . After all , the main dynamic has tried to highlight in our prepared remarks , we see growth for Marley us .
Speaker #3: We see the bile acid portfolio continuing to do what it does . It's really the live Marley International line where we expect variability as we move quarter to quarter .
Speaker #3: You know , as we've talked about before , that business has periodic large orders from from distributors . And we saw we saw those come in in Q3 .
Speaker #3: We also mentioned that we had to cater revenue in Q3 , which we also had Q1 and Q2 , that we don't expect in Q4 .
Speaker #3: So it's a fair bit of inventory build there . So that's really the dynamic is in the Marley International line .
Speaker #6: Thank you .
Speaker #2: Answer the question .
Speaker #1: The next question will be from the line of Josh Schimmer with Cantor. Please go ahead; your line is open.
Speaker #7: Thanks for taking the questions . Maybe I had two quick ones . First . What trends are you seeing in terms of adoption of the solid tablet formulation of Livmarli ?
Speaker #7: 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 .
Speaker #7: Thank you .
Speaker #3: Thanks for the questions , Josh . Yeah . So in terms of the solid tablet , just launched in the US and mid-June .
Speaker #3: So this is really our first full quarter with it . And what we've we've seen a very encouraging kind of uptake . And really switches from the liquid .
Speaker #3: So if you look at the prescribing information patients are eligible to switch if they're at least 25 kilos . I think what I can say is that substantial proportion of those who are eligible , based on their weight , are switching .
Speaker #3: So certainly excited about what that can mean long term in terms of persistence and adherence . And an easier single , you know , single tablet per dose format .
Speaker #3: That'll be preferred by these adolescents and adults . So excited about that dynamic . And then , yeah , it looks about pricing .
Speaker #3: Obviously I haven't made a final decision there of monitored those dynamics that you were talking about . We've kind of , you know , based case thinking .
Speaker #3: You can look at the other pars and the other products that are that are kind of approved in PBC at the 130 to 150 .
Speaker #3: But we're still analyzing. I think it's kind of too early to say what the right pricing strategy is for that.
Speaker #7: Thank you .
Speaker #2: Thanks for the questions .
Speaker #1: The next question will be from the line of Gavin Clark , Gartner with Evercore . Please go ahead . Your line is open .
Speaker #8: Hey guys . Thanks for taking the question . Just had one . What's your expectation for paragraph for filers ? Maybe just helpful to lay out your confidence in your whole IP portfolio , especially around the method patents and including volixibat .
Speaker #8: Thanks .
Speaker #2: Hey Gavin , thanks for the question . Overall , I mean , the we're we're in the window where we could potentially see that and kind of all all routine for this point in the lifecycle for Livmarli .
Speaker #2: And really quite confident in our overall IP position . In particular , you mentioned the the method patents that are specific to dosing of livmarli in these indications .
Speaker #2: We've seen this is 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 .
Speaker #2: And and a great position and prepared to defend it . So more to come if and when we we do see any filers but nothing to date .
Speaker #8: Great . Thanks .
Speaker #2: Thanks for the question .
Speaker #1: The next question will be from the line of James Kandula with Stiefel . Please go ahead . Your line is open .
Speaker #9: Hey , thanks for taking the question . This is Mark on for James . So , you know , recently on earnings Shionogi seemed to suggest it's still an open question around sort of what exactly the best endpoint is for their fragile X study .
Speaker #9: You wanted to see if you guys had any perspectives on that and sort of the implications for for your program that you initiated this year .
Speaker #9: And then we had a second question on PSC . And these patients typically kind of have inflammatory disease , sort of like comorbidities .
Speaker #9: And we know that Ibat inhibitors by nature sort of have some of these GI side effects . So curious your thoughts on on the safety risks there .
Speaker #9: And if you can see sort of anything in the blinded data on GI side effects and whether those looked any materially different than , say , PBC or Aligos .
Speaker #9: Thank you .
Speaker #2: Yeah , thanks for the question mark . I can't really speculate too much on Shionogi's update and what's going on underneath that , but turned it to Joanne to talk a little bit about our endpoint strategy and what we're our approach on our programs .
Speaker #4: Yeah , thanks for the question . You know , we feel that we're in a good spot at this point . You know , the preclinical data in terms of this pathway and the importance of this pathway and fragile X is quite strong .
Speaker #4: We recently presented some preclinical data with our compound in a mouse model mouse knockout model , which supports efficacy . And us moving forward .
Speaker #4: And then we've also , you know , had very good engagement with the community , with patients and with physicians . We also had a very successful and engaging pre-ind meeting with the FDA earlier this year .
Speaker #4: And they're entirely aware of the range of endpoints that we're looking at . And we're , you know , well aware of the types of validation that are needed for these types of outcomes .
Speaker #4: So I think we're actually in a in a pretty good spot . You know , a lot of interest from the community . And we're looking forward to conducting this study and seeing what we see .
Speaker #2: And then on the PSC safety sample that actually looked at Joanne for that .
Speaker #4: Yeah . And so with regard to that , you know , for the PSC study , you know , we've had a data monitoring committee following with us .
Speaker #4: And so no issues have been raised , no suggested modifications to the protocol . So we feel pretty comfortable . There's no big safety issues here .
Speaker #4: Feel pretty comfortable with , you moving forward with the way the protocol was initially designed . So it's not no issues have emerged .
Speaker #4: Their .
Speaker #2: Profile overall is consistent with what we know about Ibat at this point .
Speaker #9: Thanks .
Speaker #2: Thanks for the questions .
Speaker #1: The next question will be from the line of Joseph Thome with TD Cowan . Please go ahead . Your line is open .
Speaker #2: Hi there. Good afternoon. Thank you for taking my questions, and congrats on the progress.
Speaker #10: Maybe on the PSC study now that that one is fully enrolled , are you able to talk a little bit about the the baseline criteria of the patients that were enrolled , especially as it relates to the population that was studied in the interim ?
Speaker #10: Analysis population ? And maybe second , can you also discuss a little bit the importance of hitting on quality of life measures or bile acid , in addition to itch and will that those secondary endpoints be provided in the top line release in the second quarter ?
Speaker #10: Thank you .
Speaker #2: Thanks , Joseph , for the question . I think overall , we've not you know , plan to present or analyze some of the baseline criteria at this point .
Speaker #2: What we know from what we can say more generally from the enrollment criteria and what was in the interim , is the patients are selecting for itch .
Speaker #2: So we do have . Quite elevated baseline Pruritis scores . And it's from what we're seeing , it's quite representative of the PSC population in terms of background , disease , background , medications , things like that .
Speaker #2: So overall , kind of in line with what we expected for the population . And shifting to the question about endpoints , you know , the focus from a regulatory standpoint is 100% on that Pruritis endpoint being the outcome that we've discussed with FDA .
Speaker #2: We do expect to are excited about and expect to see based on other settings . Expect to see movement on things like fatigue and the bile acids .
Speaker #2: Bile acids obviously , being a key mechanistic marker , fatigue being a really important measure for patients . But again , those are secondary for a reason .
Speaker #2: The regulatory path is entirely through that pruritis endpoint .
Speaker #10: Thank you .
Speaker #2: Thanks for the question .
Speaker #1: The next question will be from the line of Ryan Deshna with Raymond James . Please go ahead . Your line is open . Thanks .
Speaker #3: And congrats on the quarter . Can you remind . us what .
Speaker #2: Went into the .
Speaker #1: Decision to opt for .
Speaker #10: Bid dosing .
Speaker #2: For the . expand study ? And how would you expect the dosing instructions .
Speaker #1: To look .
Speaker #2: On an expanded .
Speaker #1: Label .
Speaker #11: In cholestatic pruritus patients? And I have a follow-up. Thanks.
Speaker #2: Thanks , Ryan , for the question . Let me . The simple answer is empirical . Right . So this is based on observations we've had in compassionate use settings at dose levels that have explored across a range in this kind of all in the bracket of these elevated dose levels from the label up to the label .
Speaker #2: And empirically , this is where we've seen really great response stories from compassionate use examples . And Ryan , you said you had .
Speaker #11: A follow Rick . Yeah . Real quick . How big of an impact have the government shutdown been so far for things like genetic screening programs and other programs related to allergy and thanks today .
Speaker #2: No impact that we've seen across all of our interactions with customers and really across the across the business .
Speaker #11: Thank you .
Speaker #2: Questions .
Speaker #1: From the line of Manny with Leerink Partners , please go ahead . Your line is open .
Speaker #6: Hey guys . We have Ryan on for Manny . Thanks for taking our question and congrats on the quarter . Can you talk a little bit about the pace of New ads that you guys saw in the third quarter compared to the second quarter ?
Speaker #6: I know you talked a lot about genetic testing and new patient diagnoses . And then maybe more broadly , as you guys start to see , you know , consistent positive cash flow and you have several launches on the horizon , maybe just talk through your BD strategy about adding more products to the pipeline .
Speaker #6: Thanks .
Speaker #2: Yeah , thanks for the question . I turn it over to Peter to jump into those .
Speaker #3: Yeah . You know , in terms of the pace of ads , it continues to be healthy . It continues to come from a broad .
Speaker #3: You know , patient population . Everything from infants to adults that we've kind of commented on that is a dynamic where the paradigm is really being changed with adult providers to think about genetic cholestasis as kind of a clinical entity , to be suspicious about .
Speaker #3: So that's kind of a educational effort . And there , know , some of the major academic medical centers are on board with that .
Speaker #3: And they're looking into genetic causes of cholestatic liver diseases . And the patients , they can't explain with with other diseases . But most , most aren't .
Speaker #3: Right . So that's just kind of a gradual effort . And but it's continuing to bear fruit in Q3 . Then the second .
Speaker #3: Oh yeah . Do you want to .
Speaker #2: Sure . I mean , the thing we'd say on BD is since the beginning of the company , that's really been at our , at our core is looking for underappreciated programs .
Speaker #2: So we continue to do that . And , you know , expect to always be active doing that . But we're in just a .
Speaker #2: Fantastic position where there there's no urgency and no need . So we have a very
Speaker #2: bar you . And as you can see from . The programs we've brought in since the start of the company . Look for good value creation opportunity .
Speaker #2: So that will continue to be the standard . We take going forward . And plenty in the company to to grow and build .
Speaker #2: And , you know , optimistic about adding more down the road .
Speaker #6: Appreciate it . Thanks again .
Speaker #2: Thanks for your questions .
Speaker #1: Next question will be from the line of Mike olds with Morgan Stanley . Please go ahead . Your line is open .
Speaker #12: Hi , this is Rohit on for Mike . Thanks for taking our questions . Just with the recent linerixibat for announced for GSK , how do you see the competitive dynamics playing out in PBC ?
Speaker #12: Thanks .
Speaker #2: Thanks for the question . I think two two overarching things to think about for the competitive landscape in PBC . One is just kind of a reminder on lines of therapy and where the the volixibat program plays and in the vantage study there , there is no baseline alkaline phosphatase criteria .
Speaker #2: So our our program and incorporates both first and second line PBC settings . So those that have stable alkaline phosphatase on Udca that likely wouldn't be a treatment candidate for for some of the parts that are recently launched but still have itch , that is a that's a candidate for for volixibat study .
Speaker #2: And we expect ultimately that marketed treatment . And then with respect to Linerixibat as a competitor , we're very excited about the interim data that we saw from the vantage study and what it means for the dose level .
Speaker #2: That was selected . In the placebo adjusted difference that we saw on itch in that data set is striking , led to breakthrough designation , and it's really everything that we'd hoped to see from all that we've learned about dosing of these , of this mechanism , in these settings .
Speaker #2: So quite excited about the competitive profile of Bat , given that highly active dose level .
Speaker #12: Thank you .
Speaker #2: Thanks for your questions .
Speaker #1: The next question will be from the line of John Wohlleben with citizens . Please go ahead . Your line is open .
Speaker #3: Hey , thanks for taking the question .
Speaker #11: Wondering if .
Speaker #3: You guys are anticipating seeing similar disease modifying .
Speaker #11: Effects over time with Volixibat, as you saw with Livmarli and PSC and PBC, and if so, what would be the time frame?
Speaker #11: And do you think that would be an important consideration for adoption and use over time ?
Speaker #2: Hey Joe , thanks for the question . I mean , the overarching first thought there is the first readouts here we think are probably too soon to be looking at that and focused on the itch endpoint and really see that as the that's that's the launch profile .
Speaker #2: But I'll turn it over to Joanne to talk through some of what we'll be looking at and what we'll be able to see over time from the program.
Speaker #4: Yeah , thanks for the question . You know , as Chris alluded to , the whole discussion , especially with the regulators , has been around how do we get in PSC approved ?
Speaker #4: And clearly that's with pruritus or pruritis endpoint at this point in in the field of PSC , that's really the only something approval endpoint .
Speaker #4: Obviously , we'll look at other things . Look , you know , longer term we do expect those types of endpoints may take quite a long time to evolve .
Speaker #4: We'll continue to follow these patients . And obviously we'll continue to engage with the agency in terms of appropriate endpoints do think a concrete path forward is with pruritus .
Speaker #4: And we're pretty confident in terms of , you know , the ability of Elizabeth to affect that in a positive way for patients .
Speaker #11: Thanks , guys .
Speaker #1: Thank you . And with no further questions on the line at this time , I would like to hand the call back to Chris for some closing remarks .
Speaker #11: Great .
Speaker #2: Thanks again , everyone , for joining us today and for your continued support . We look forward to updating you next quarter . Good afternoon .
Speaker #1: This will conclude the Mirum Pharmaceuticals, Inc. third quarter 2020 financial results and business update . Thank you to everyone who is able to join us today .
Speaker #1: You may now disconnect your lines .