MIRM

Image source: The Motley Fool. Wednesday, Aug. 5, 2026 at 4:30 p.m. ET SVP of Strategic Finance and Investor Relations - Andrew McKibben Chief Executive Officer - Christopher Peetz President and Chief Operating Officer - Peter Radovich Chief Financial Officer - Eric H. Bjerkholt Chief Development Officer - Lara Longpre Operator: Good afternoon, and welcome to Mirum Pharmaceuticals Second Quarter 26 Earnings Conference Call. My name is Alexandra, and I will be your operator today. All lines are currently in a listen-only mode. And there will be an opportunity for Q&A after management's prepared remarks. I would now like to hand the conference over to Andrew McKibben, SVP of Strategic Finance and Investor Relations. Please go ahead. Andrew McKibben: Thank you, Alexandra, and good afternoon, everyone. I would like to welcome you to Mirum Pharmaceuticals second quarter 26 conference call. I am joined today by our Chief Executive Officer, Christopher Peetz our President and Chief Operating Officer, Peter Radovich and Eric H. Bjerkholt, our Chief Financial Officer. Lara Longpre, our Chief Development Officer, will be joining us for the Q&A portion of the call. Joanne Quan, our Chief Medical Officer, could not be with us today due to a family matter. Earlier today, Mirum issued a press release announcing the company's results for the second quarter of 26. Copies of the press release and our SEC filings are available on the Investors section of our website. Before we start, I would 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 Mirum's programs and market opportunities for its approved medicines and product candidates and financial guidance. 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. We are under no duty to update these statements. Please refer to the risk factors in our latest Form 10 Q and subsequent filings for more information about these risks and uncertainties. With that said, I would like to turn the call over to Christopher. Christopher? Christopher Peetz: Thanks, Andrew, and good afternoon, everyone. At Mirum, we are growing a rare disease leader focused on delivering high impact medicines for often overlooked diseases. This quarter demonstrates continued progress with strong commercial execution on our approved medicines as we head into the potential launch of our fourth commercial medicine later this year. We have a busy pipeline with multiple pivotal readouts in the quarters ahead, all delivered with the strength in capital structure and overall financial performance, giving us greater capacity to invest throughout the business. In the second quarter, our commercial business generated $176 million in net product sales, reflecting strong demand across the portfolio and excellent execution by our team. Based on this performance, we are increasing our full year 2026 net product sales guidance to $680 million to $700 million Fueled by the strong commercial performance, we are driving towards the next phase of Mirum's growth with multiple milestones over the coming months. Our next commercial milestone will be the potential launch of Zilurgisertib for FOP with the PDUFA date next month. This is fast progress for a program added to our rare genetic business, only in the second quarter. Peter will cover more of the launch profile in his remarks. Moving to the pipeline for our rare liver business, important to spend some time today on volixibat and PSC. Which just had some key U. S. Regulatory interactions. First, as a reminder of the background of the study of volixibat in cholestatic pruritus in PSC, we designed this adaptive study with input from the FDA as a pivotal trial for this difficult clinical setting. Including alignment on study duration, endpoints, and analysis plan. As we have announced previously and presented at EASL this year, VISTAS met its primary endpoint showing highly significant improvements in pruritus in the primary cohort. With consistent significant results also observed in a second cohort of patients with milder baseline pruritus. We are excited to share that the FDA has now granted breakthrough therapy designation for volixibat in cholestatic pruritus due to PSC based on these strong results. We see this as recognition of the potential for volixibat to address a serious unmet need in PSC As planned, we recently held a pre NDA discussion with the agency about the submission of an NDA based on the VISTA study. In the meeting, the FDA recommended conducting a phase 3 study. We believe the VISTA study provides a robust and clear dataset to characterize the use of volixibat in patients with pruritus due to PSC, and is a clinically and statistically highly persuasive study. VISTAS is the largest randomized clinical study conducted in patients with pruritus due to PSC with an extensive overall data package that includes more than 180 PSC patients, randomized 1-year safety exposure data for over 100 PSC patients and growing, results from an independent committee evaluating liver safety, all totaling over 600 subjects across the clinical program to date. So while we are not currently aligned on the NDA submission package, we will be engaging in discussions with the FDA on how to further supplement our planned submission based on the VISTA study. This engagement will delay the planned timing of our NDA submission, which we are now targeting for the first half of next year. We are positioned to move quickly once we have further clarity from the agency. We will provide updates as we work towards our goal of bringing a much needed therapy to this unaddressed clinical setting. In parallel, the VANTAGE study in PBC is progressing well, and has completed enrollment reaching over 330 patients randomized. In PBC, our earlier breakthrough therapy designation has enabled more dialogue with the agency during the conduct of the study. We have recent feedback from FDA for Vantage to serve as a pivotal study of volixibat in pruritus due to PBC if the study is successful at its Q1 top line readout next year. Our next clinical readout for the rare liver business is expected to be volovitug's AZURE-1 top line results later this quarter. This is the readout of the Phase III portion following strong results in phase IIb portion earlier this year, and we also continue to expect the AZURE-4 data in the Q4, which keeps us on track for a potential BLA submission for this breakthrough therapy designated program in the first half of next year. And rounding out the rare liver pipeline highlights, the Phase III EXPAND study of Livmarli and additional rare cholestatic conditions remain on track for top-line data in the Q4. So putting this all together, we are advancing these clinical programs from a position of financial strength. Our commercial business continues to generate meaningful cash, providing the capacity to invest in potential launches, clinical development, opportunistic business development, where we see a compelling strategic fit and the potential to create value. I am proud of the team's progress and the promise of our current medicines and pipeline I am excited about what lies ahead for Mirum. And with that, I will turn the call over to Peter to discuss our commercial performance and launch readiness in more detail. Peter? Peter Radovich: Thanks, Christopher. The second quarter was another strong quarter for Mirum's commercial business. With total net product sales of $176 million for Livmarli and the bile acid medicines both continue to perform well And based on the demand we see, we are increasing our full year 2026 net product sales guidance to $680 million to $700 million. Second quarter net product sales for Livmarli were $129 million, with the U. S. Contributing 92 million Alagille growth remains durable, supported by continued new patient starts, sustained persistence on therapy, and weight based dose increases. PFIC continues to be an important driver of growth, fueled by new diagnosis. We are particularly encouraged by the growing contribution from adult PFIC patients. We are seeing an increase in prescriptions from adult liver providers as awareness of later onset PFIC grows. And genetic testing becomes more routine. Based on claims data as well as insights from 2 years in market, we now estimate an addressable adult PFIC population of at least 2,000 patients in The United States with likely a similar number in Europe. And because genetic testing remains less established in adult practice, then the 2,000 addressable patients do not yet have a PFIC diagnosis. And we see a meaningful opportunity to continue expanding diagnosis through education. Internationally, Livmarli continues to grow across our direct and partner markets. Contributing $37 million for the quarter. We are seeing contributions from established markets, and expanding reimbursement in additional geographies. On the rare genetic disease side of the business, our bile acid medicines continue to provide steady contribution generating $48 million in net product sales for the quarter. Like our rare liver business, our rare genetics-- our rare genetics business is also poised for growth with the recent addition of Zilurgisertib for FOP. Data from the pivotal Phase II progress study of Zilurgisertib presented at ENDO showed a compelling clinical profile in FOP patients ages 12 and older. Based on the strong efficacy observed and the convenience of oral dosing, we believe Zilurgisertib has the potential to offer an attractive profile to patients with this severely debilitating disease. If approved by the FDA, the initial launch opportunity is expected to focus on patients ages 12 and older, with potential future expansion into younger patients supported by additional cohorts in the PROGRESS study. And following a recent late cycle meeting with the FDA, we believe the NDA is proceeding as expected towards the September PDUFA date and we are preparing for potential U. S. Launch in the Q4. The U. S. Launch will heavily leverage the rare genetics team we have in place now that currently markets our bile acid medicine. As the physicians who manage FOP are largely concentrated in the same specialized centers where CTEXLI and CHOLBAM are prescribed. Building on the efficiency of our rare genetics business. Also, a marketing application for Zilurgisertib has been submitted in Europe. And we will provide updates as this filing progresses. Overall, we are pleased with the continued execution across the commercial organization. We are seeing strong demand throughout the existing portfolio, while making the investments necessary to support the next wave of potential launches. We believe that we are well positioned for the remainder of the year and beyond. With that, I will turn it over to Eric to discuss the financial results. Eric? Eric H. Bjerkholt: Thanks, Peter, and good afternoon, everyone. Today, I will walk through the financials of another excellent quarter from Mirum. Net product sales for the second quarter were $176 million compared to net product sales of $128 million in the second quarter of last year. Cash equivalents and investments as of June 30 were $561 million compared with $391 million at the beginning of the year. In the second quarter and first half of 26, the cash contribution margin from our commercial business was in the high-50s percent approximately a 5 percentage point improvement over the year before. Total operating expense for the quarter ended June 30 was $290 million, $19 million of which includes 16 million of in-process R&D expense associated with the upfront payment for licensing Zilurgisertib. R&D expense of $76 million including $29 million related to the development of volovitug, SG&A expense of $66 million, and cost of sales of $23 million, all excluding stock based compensation expense and intangible amortization. Stock based compensation, intangible amortization, and other noncash expenses totaled $37 million for the quarter. Operating cash flow was positive in the second quarter despite the expected increase in R&D expense. During the quarter, we significantly improved our capital structure through the issuance of $690 million aggregate principal amount of 0% coupon convertible notes due in June 2032. Net proceeds from the offering were $672 million. In conjunction with this financing, we settled 75% of the outstanding 2029 notes added $197 million of cash to the balance sheet and significantly reduced our interest expense. These transactions further strengthen our balance sheet and extend our financial flexibility to execute our strategy. Our commercial business continues to scale and our pipeline includes multiple near term value drivers. With that, I will turn the call back to Christopher for closing remarks. Christopher Peetz: Thanks, Eric. Taking stock of where we are we are continuing to drive our strategy to bring important medicines to underserved rare disease patients. Commercial business is thriving, now tracking towards 680 to $700 million in net product sales for the year. Livmarli is well on its way to realizing its over $1 billion peak revenue potential. And our rare genetics team is thrilled about the potential launch of Zilurgisertib for FOP patients in the coming months. On the pipeline, we are focused on a collaborative discussion with FDA on the VISTA study in PSC. Clinical results are clear, and breakthrough therapy designation gives us further opportunity to engage with FDA. All said, we have a plan to advance volixibat for PSC patients. The balance of the pipeline is firing on all cylinders. Recapping our upcoming clinical milestones, we expect clinical readouts from the Phase III AZURE-1 and AZURE-4 studies of volovitug over the balance of the year which will enable our planned BLA submission in the first half of next year. Next quarter, we also expect to announce top line results from the EXPAND study of Livmarli and additional rare cholestatic diseases setting up the potential for an sNDA next year as well. And into 2027, we expect top line results from the VANTAGE study of volixibat in PBC in Q1. And finally, we are on track with MRN-338, with proof of concept data in fragile X syndrome next year. Importantly, we are building an organization capable of reaching many more people living with overlooked rare diseases while delivering strong financial performance. I want to thank the Mirum team for their continued focus and execution, and to the patients, families, and physicians who continue to partner with us in this work. With that, operator, please open the call for questions. Operator: We will now begin the question and answer session. Please limit yourself to 1 question and 1 follow-up. If you would like to ask a question, please press *1 to raise your hand. To withdraw your question, press *1 again. We ask that you pick up your handset when asking a question to allow for optimum sound quality. If you are muted locally, please remember to unmute your device. Please standby while we compile the Q and A roster. Your first question comes from the line of Ryan Deshner with Raymond James. Your line is now open. Please go ahead. Ryan Deschner: Hi. Good afternoon. Congrats on the strong results. Curious what your overall take is on the potential read through from the BOLD study evaluating odevixibat in patients with biliary atresia? And I have a follow-up question. Christopher Peetz: Thanks, Ryan, for the question. The BOLD study and the study of note in our pipeline that includes biliary atresia patients, the EXPAND study I mean, to put it simply, are asking very different questions. I would equate the BOLT study more to what we ran previously with EMBARK, looking at patients in the very acute setting trying to reduce bilirubin or extend transplant free survival. The question we are asking in EXPAND ties much more to where we have seen IBAT perform quite well in other settings. We are looking at older patients than what was in BOLD and in EMBARK and evaluating pruritus changes it is something that we have seen an impact from IBAT therapy. Over and over again now in different clinical settings. So we feel there is not really a read through or connection between BOLD, and what we are looking at in the upcoming EXPAND readout. Ryan Deschner: Appreciate it. And then, just on the PSC regulatory discussions, what key topics do you anticipate sort of addressing with these future discussions? Thank you. Christopher Peetz: Yeah, thanks for the follow-up on that. I mean, just to kind of reiterate some of the background here. We did extensively discuss the VISTA study design with FDA Back in the pre IND setting, the FDA acknowledged that pivotal intent. Described the design as reasonable, gave direct feedback on duration, analysis plan, all these things that designed the study that we read out so successfully earlier this year. And frankly, the situation now that we have seen is in the meeting, there was a new team from FDA. And so we think there is a lot of work to kind of get them up to speed on the backdrop here. So the history designing and conducting VISTA and what the study means in a PSC setting. So we think it is going to be an iterative approach to kind of get them up to speed, only with the current data package, but with the history of the program. Very helpful. Thanks, Christopher. Thanks for the questions. Operator: Your next question comes from the line of Gavin Clark-Gartner with Evercore ISI. Your line is now open. Please go ahead. Gavin Clark-Gartner: Hey, guys. Thanks for taking the questions. First, what was this new FDA team's rationale to conduct an additional phase 3? Like, was it more of a safety database question, or was it more around the efficacy side? Christopher Peetz: Follow-up, Gavin. You know, the comments about a phase 3 recommendation from them. Really were not specific. So we do not have great clarity on what specifically they are looking for. There were comments across the board on more general on efficacy which we think VISTAs very clearly addresses, and they actually commented on that in the meeting. On the safety side, in the discussion, the couple of things that were brought up were IDD safety and the backdrop with IVAT GI effects. And liver safety, You know, those were designed into VISTAs as key things to evaluate. So we think it is all there in the VISTA study, and it is more this is more about familiarity with study design and some of the questions that were asked. Another thing I would point out is that the breakthrough designation actually was issued after the meeting. So I think that is gives us a great opportunity to go back in and build up familiarity with this dataset and work toward an NDA. Gavin Clark-Gartner: Okay. That makes sense. And just a quick follow-up. Is this a team that you have engaged with before on any of your other programs? Do you have any experience working with them? And I kind of on a similar point, what other regulatory precedents would support approval based on the similar type of phase 2 b setting? Thank you. Christopher Peetz: Thanks for the follow-up. In terms of the team, a lot of times the correspondences written, so you do not have perfect clarity on who is behind some of the written correspondence. So it is hard to answer that question to be honest. In terms of precedent, I mean, really have looked across all the IBAD settings where you are seeing an Alagille where the first approval was based on 4-week randomized withdrawal data. All the way to the more recent LIVMARLI approval in PBC pruritus with a 6-month placebo controlled study that frankly looks a lot like VISTAS. it is a little bit larger because PBC is a more prevalent indication. So there is pretty clear precedent for IBAT in cholestatic pruritus. Settings. That line up with VISTAs. It goes back to the whole way that we designed the study in the prior conversations with the agency. I am sorry. I may have misheard something. Did you say that this was only happening via written correspondence, or was this pre NDA in person with the FDA or virtually? The pre the pre NDA was in person. So when you were asking about prior and other interactions, some of those have been written. So we do not know who is behind some of the other written correspondence. This meeting was in person. Okay. Got it. Thanks so much. Yeah. Thanks for the question. Operator: Your next question comes from the line of Mike Ulz with Morgan Stanley. Your line is now open. Please go ahead. Rohit Bhasin: Hi. This is Rohit Bhasin on for Mike. Thanks for taking our questions. Just based on your conversation with the FDA, do you see any read through to the PBC Vantage study Is it possible they will request a phase 3 trial for that 1? And also, in terms of commercial prep for FOP, can you talk about where you currently stand in what is outstanding? Thanks. Christopher Peetz: Thanks, Rohit. I will speak to PBC and then pass it over to Peter to talk about FOP. On the VANTAGE study, as you may recall, we were granted breakthrough designation after the interim analysis in 2024. So that actually gave us a great opportunity to have conversations similar to what we expect to go through here with VISTAs. So in the correspondence after the breakthrough designation on Vantage, actually received a pretty clear feedback from FDA on what we should do to consider Vantage a pivotal study. And we were able to address those. So primarily, the comments related to the overall size, that is part of why we have ended up with over 3 hundred and 30 patients in Vantage. And also the analysis plan and some of the specifics on how the pruritus endpoint is analyzed. Peter Radovich: And, Rohit, yeah, with regards the commercial prep for Zilurgisertib potential approval and launch in Q4. that is going really well. As I mentioned, we did were able to drop that in the bats of our rare genetics team and had a couple territories there. A lot of, you know, typical prelaunch activity and profiling accounts. And understanding where the treaters are. These patients are well identified. there is about 300 or so diagnosed and managed in The US and highly specialized centers Had a had a good presence at the end of meeting earlier this summer as well. So excited about the progress of the end of the review and Working towards launch readiness in Q4. Thank you. Thanks for the question. Operator: Your next question comes from the line of Brian Skorney with Baird. Your line is now open. Please go ahead. Brian Skorney: Hey. Good afternoon, team. I am gonna be annoying and also ask about FDA's issues with VISTAs, just given that it seems like a pretty straightforward data set. Can you just refresh our memories? Is the review team here is this within the Division of Hepatology? Is Kati Donahue, the Office Director? Was she involved in the meeting? Is Frank Anania still the Division Director and was he in the meeting? And you know, I mean, I hear they were not very clear about why they wanted phase 3, but, I mean, did they sort of mention, like, a need for replication it just seems like the key value here is so robust that there is not really another question that could be answered with a phase 3 other than maybe longer term follow-up? So, yeah, any sort of guidance there is helpful. Christopher Peetz: Thanks for the question, Brian. On some of the specifics of people kind of in the room, I would say is, you know, the office leadership, we think maybe has changed, and leadership was not in the room in our review meeting. And kind of getting into some of the I mean, specifically on efficacy, you know, the I think it is like you are pointing out, I think it is very easily addressed by the VISTA data. And getting breakthrough afterwards, I think, is a nod in that direction. So I have given some of the discussion in the room, I think that is something that we can readily address through iterative conversation with FDA. I do not know if that helps answer your question. Yeah. Brian Skorney: So maybe if I could just ask 1 thing on the PDUFA with Zilurgisertib. I think you would have had probably the late cycle review meeting in the last month or so. Just any commentary on how that went? Your level of confidence going into labeling discussions? Peter Radovich: Yeah. Thanks for the question, Brian. That yeah. We did have the late cycle meeting, Meeting went very well. So we are you know, we feel the application is progressing, you know, quite well towards the PDUFA date. So Full systems go to prepare for potential launch in Q4. Great. Thank you. Thanks for the questions. Operator: Your next question comes from the line of Kalpit Patel with Wolfe Research. Your line is now open. Please go ahead. Yesha Patel: Yeah. Hey. Good afternoon. Thanks for taking the question. 1 more on the regulatory interaction here. In your communication with the agency, has there been any dialogue so far on running a post approval confirmatory study instead? Of running a phase 3. Or should investors assume that, you know, a Phase III is what is in the works right now? Christopher Peetz: Thanks for the question. I think the direct answer is that conversation with FDA did not get to that level of specifics for post approval requirements. I can comment a little bit on the pathway here that using pruritus is the basis for full approval. So we do not expect to have a post approval study in the sense of kind of confirmatory accelerated approval type format. What we would expect and has been added for other IBAT approvals is some level of post approval registry or kind of long term monitoring. So and so we do think that would be appropriate for this setting as well. Okay. Thank you. Thanks for the questions. Operator: Your next question comes from the line of James Condulis with Stifel. Your line is now open. Please go ahead. James Condulis: Hey. Thanks for taking my question. and congrats on a great Live Marley quarter. Just maybe to be annoying again, 1 more on PSC. I guess, like, to clarify, is a phase 3 on the table, or, you know, are you confident that this can be resolved you know, through, like you said, iterations on sort of the data Just wondering sort of, like, what your base case is and, you know, sort of what informs your confidence of guiding to a first half 27 resubmission? Thanks. Christopher Peetz: Yeah. Thanks for the question, James. And as we looked at this proposal for a phase 3, and given the VISTA results, I think the key question is what would you learn from another study in this setting? And VISTA is the largest ever conducted in PSC pruritus with clear, definitive results. it is a big enough safety database for a setting like PSC. So do not see what would be gained by going down that road. And find that just the weight of evidence here is really convincing. So I feel good about where we stand now with breakthrough designation. To work through this. Thanks. Thanks for your question. Operator: Your next question comes from the line of Lisa Walter with RBC. Your line is now open. Please go ahead. Lisa Walter: Good afternoon. Thanks for taking our questions. Kind of 1 more on the NDA filing delay here for velexibat. Could this mean that we see more BD in the near term as potentially PSC revenues may now be pushed out? And I just wanna ask as well, could you add any clarity on whether the FDA is still accepting pruritus as an approvable endpoint? Thanks so much. Christopher Peetz: Thanks for the questions, Lisa. You know, first thing on I will answer your second part first, which is absolute clarity that pruritus is an approvable endpoint. that is what this discussion is all focused on. So there is there is no question there. On the BD front, you know, we always approached BD on being always active and always opportunistic. So do not see this really relating to overall level of activity we have or our criteria and what we are looking to bring in. Thanks for the question. Operator: Your next question comes from the line of Joe Schwartz with Leerink Partners. Your line is now open. Please go ahead. Analyst: Hi. Thanks. Hypothetically, if the FDA does not budge, does VantagePVC have any ability to strengthen the volixibat regulatory package for PSC. For instance, if, a filing in PBC is an NDA, and a filing in PSC as an sNDA, could that order of operations be successful without having to do another Phase III? Christopher Peetz: Joe, thanks for the question. What I would say is potentially, and we are getting into some hypothetical down the road. The base plan is to get the PSC NDA submitted first. But as we think about the VANTAGE data, another large randomized study playing into a very related pruritus condition, I do see some weight to that. And how we approach that in terms of parallel or sequenced NDAs is something that we will get to if we end up in that scenario. Okay. Thanks. And then question on volovitug. Did any baseline characteristics for patients enrolled in the interim analysis cohort of Azure 1 appear to correlate with better response in the phase 2 b portion of the trial. And how do the baseline characteristics for patients enrolled in the full Azure 1 population and Azure 4 compare? I mean, the quick answer to that is no. Nothing obvious that really comes out. We see response for volovitug across really all patients. So, it is a very broad based response. And that cuts across baseline viral RNA levels, ALT levels, geography, kind of every way we have looked at it, consistently see patients responding to volovitug. Thank you. Thanks for the question. Operator: Your next question comes from the line of Jessica Fye with JP Morgan. Your line is now open. Please go ahead. Jessica Fye: Hey, guys. Good afternoon. Thanks for taking my question. So more on volixibat. When you, I think in your prepared remarks referred to some options to supplement the VISTAs trial, Curious what you could supplement it with. And second, just based on where we stand right now, what gives you the confidence to outline first half of 27 as the new PSC filing timeline. Thank you. Christopher Peetz: Yeah. Thanks for the questions, Jessica. Yeah. In terms of supplementing it, the real simple 1 is actually something I mentioned in the prepared remarks as well, is kind of the data cutoff timing. Where we are able to pretty easily allow more safety data to accrue, like to get to 100 patients at the 12 month mark in the open label follow-up. So that was 1 of the kinda quick offerings in there. I think some of the other things could be towards things that were mentioned in earlier questions and what could be offered up for post marketing registry work and things like that. In terms of the timing, we feel first half is very achievable, and it is really based on having enough room to have an iteration or 2 with FDA. So it is less about time we need to prepare the submission, frankly, because we are-- things are ready to go quite quickly after we kinda have the input we need. it is more about that iteration with FDA. Really, that is that is kind of an estimate based on experience. You know, we have we have done the thing the applications like this before back to the original Alagille application where it took a couple of meetings along the way to kind of build up to that NDA filing for Livmarli in Alagille, And so it is it is a type of situation that we have worked through before. Operator: Your next question comes from the line of Joseph Thome with TD Cowen. Your line is now open. Please go ahead. Joseph Thome: Hi there. Good afternoon, and thank you for taking my questions. Maybe just in setting expectations in terms of when we should hear next steps on sort of the filing progress? I guess, what are you anticipating in terms of relaying kind of your FDA interactions to the street? And then I think in your prepared remarks, you indicated that this necessarily will not flip over to PBC, and that you have some feedback that Vantage will be a registrational study. I guess, to your knowledge, is that this new group that conveyed that information 1 point of clarification just on the updated guidance. Is there anything for FOP in your updated product guidance, or would that be above and beyond? What you have outlined today? Thank you. Christopher Peetz: Thanks for the questions. I will take the first couple and pass it over for the FOP question. You know, in terms of providing an update, our view is that this is likely an iterative process, and so we would plan to give an update when we have kind of a material update. So you do not wanna be sharing the blow by blow on what might be e email correspondence even with FDA. And shifting on to the PBC question, those interactions were written correspondence, so do not know exactly if it is the same exact people behind it, but what gives a lot of comfort there is recency. Right? So this has happened over the past year. That we have had those PDC written correspondence. Eric H. Bjerkholt: And on guidance, the 680 to 700 million does not include FOP. So anything we see in Q4 would be above and beyond, although we really expect the revenue to start more in 2020 Thank you. Operator: Your next question comes from the line of Jonathan Wolleben with Citizens. Your line is now open. Please go ahead. Jonathan Wolleben: Hey, Christopher. I am wondering if you could just you know, talk us through what this iterative dialogue looks like in terms of you know, using breakthrough therapy designation or formal meeting status and, you know, scheduling of these, just for a little bit more expectations on that flow of information between you and the agency. Christopher Peetz: Yeah. Thanks for the follow-up, John. 1 of the benefits of breakthrough designation is it does allow for more frequent advice from FDA, more frequent interactions And so quite simply, that just means we will be able to reach out more in informally and also have more advice meetings. How those sequence out really depends on how the dialogue with FDA progresses. So we cannot really give too much more specific at this point. But we will keep you guys updated as we make progress through it. Operator: Your next question comes from the line of Ramakanth Swayampakula. With h c Wainwright Your line is now open. Please go ahead. Swayampakula Ramakanth: Thank you. This is Swayampakula Ramakanth from H.C. Wainwright. Couple of really quick questions. You know, based on the interactions that you have had so far, for the PSC, PSC indication you know, are you planning to make any changes at all in your approach for the for the PBC indication, you know, once the VANTAGE data comes out, And, also, if you do go ahead and submit in the first half 27, you know, notwithstanding the recommendation. You know, did you run into a risk of refuse to file kind of a situation? And let's say everything goes fine and you still continue to apply, would you expect an AdCom at the end of this? Christopher Peetz: Thanks, RK, for the question. In terms of the PBC approach and given the recent interactions, I think we have direct input for that indication for vantage. So feel like that is on a good course. So it would not make changes at this point what we are doing in PBC. We have kind of already made recent adjustments to accommodate what FDA asked for having Vantage being confirmed as a pivotal study. And then on some of these questions about submission risks, I think is how I would describe the question, that is the reason for this iterative interaction with FDA is try to work through things that might be an RTF risk and try and get those off the table. And frankly, you know, an AdCom might be something that could be helpful given the huge impact that volixibat has shown in a really terrible setting for patients. You know, the relief, dryness relief, improvement in sleep and fatigue that patients experience with volixibat treatment. Is really life changing. So I think that could be 1 way that this gets highlighted. Thank you. Thanks for answering my questions. Thanks for the question. Operator: There are no further questions at this time. Christopher Peetz: We will now turn the call back to Christopher Peetz for closing remarks. Great. Well, thank you all for joining us today, and hope you have a great afternoon. Operator: This concludes today's call. Thank you for attending. You may now disconnect. Before you buy stock in Mirum Pharmaceuticals, consider this: The Motley Fool Stock Advisor analyst team just identified what they believe are the 10 best stocks for investors to buy now… and Mirum Pharmaceuticals wasn’t one of them. The 10 stocks that made the cut could produce monster returns in the coming years. 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Our analysts just identified a stock with the potential to be the next Nvidia. Tell us how you invest and we'll show you why it's our #1 pick. Tap here. Increased full-year 2026 net product sales guidance to $680 million to $700 million, driven by durable growth in Alagille syndrome and expanding adult PFIC diagnosis. Identified a significant growth opportunity in adult PFIC, estimating at least 2,000 addressable patients in the U.S. and a similar number in Europe as genetic testing becomes more routine. Achieved a high-50s percent cash contribution margin from the commercial business, representing a 5 percentage point year-over-year improvement in operational efficiency. Strengthened the capital structure through a $690 million convertible note offering, extending financial flexibility and reducing interest expense while settling 75% of 2029 notes. Attributed the delay in the volixibat PSC NDA submission to a lack of alignment with a new FDA review team regarding the sufficiency of the VISTA study as a pivotal package. Maintained that the VISTA study remains the largest randomized clinical study in PSC pruritus, providing a robust safety and efficacy dataset that management believes is clinically persuasive. Leveraged the existing rare genetics commercial infrastructure to prepare for the potential Q4 launch of Zilurgisertib, targeting approximately 300 identified FOP patients in the U.S. Targeting a first-half 2027 NDA submission for volixibat in PSC, allowing for iterative dialogue with the FDA under the newly granted Breakthrough Therapy Designation. Anticipating top-line results from the Phase III VANTAGE study in PBC in Q1 2027, which the FDA has indicated could serve as a pivotal study if successful. Expecting multiple near-term clinical readouts, including Phase III AZURE-1 and AZURE-4 results for volovitug, supporting a planned BLA submission in the first half of 2027. Preparing for the potential U.S. launch of Zilurgisertib in Q4 2026, following the September PDUFA date, with initial focus on patients aged 12 and older. Projecting top-line data from the Phase III EXPAND study of Livmarli in additional rare cholestatic conditions in Q4 2026, setting up a potential sNDA next year. The FDA recommended conducting an additional Phase III study for volixibat in PSC, though management is seeking to supplement the existing VISTA data instead. Breakthrough Therapy Designation for volixibat in PSC was granted after the pre-NDA meeting, which management views as a positive signal for future engagement. Recorded $16 million in in-process R&D expense during the quarter associated with the upfront payment for licensing Zilurgisertib. Management noted that while the FDA review team for PSC appeared new, recent written correspondence for the PBC program has provided clearer registrational requirements. One stock. Nvidia-level potential. 30M+ investors trust Moby to find it first. Get the pick. Tap here. Management noted the FDA's recommendation was not highly specific but included general comments on efficacy and safety, specifically regarding GI effects and liver safety. Management believes the Breakthrough Therapy Designation granted after the meeting provides a platform to build the agency's familiarity with the existing VISTA dataset. Management clarified that the EXPAND study evaluates older patients and focuses on pruritus, whereas the BOLD study targeted acute bilirubin reduction and transplant-free survival. Expressed confidence that there is no direct read-through between the two studies due to different patient populations and clinical endpoints. Management acknowledged that data from the large randomized VANTAGE study in PBC could potentially add weight to the overall volixibat pruritus evidence. Indicated that the order of operations for filings (parallel vs. sequenced) would be determined as the dialogue with the agency progresses. Management confirmed with absolute clarity that the FDA still accepts pruritus as an approvable endpoint, and the current discussions are focused on the sufficiency of the data package. Noted that for full approval based on pruritus, they expect post-marketing registries rather than confirmatory efficacy studies.

Interested in Mirum Pharmaceuticals, Inc.? Here are five stocks we like better. Mirum raised its 2026 product-sales outlook to $680 million–$700 million after second-quarter sales increased to $176 million from $128 million a year earlier. LIVMARLI led growth with $129 million in sales, supported by new patient starts, treatment persistence and expansion in PFIC. The planned U.S. filing for volixibat in PSC pruritus was delayed to the first half of 2027 after the FDA recommended another Phase III study, although Mirum believes existing VISTAS data can support approval. The company expects additional pipeline readouts, including PBC data in early 2027 and potential FOP approval in September. Mirum strengthened its financial position, ending June with $561 million in cash, cash equivalents and investments. A $690 million convertible-notes offering generated $672 million in net proceeds and helped the company settle 75% of its 2029 notes. Mirum Pharma: A Rare Disease Growth Story to Watch Mirum Pharmaceuticals (NASDAQ:MIRM) reported second-quarter net product sales of $176 million and raised its full-year 2026 product sales outlook to $680 million to $700 million, while outlining several upcoming clinical and regulatory milestones across its rare liver and rare genetic disease portfolios. Second-quarter product sales rose from $128 million in the prior-year period. Chief Executive Officer Chris Peetz said the company’s commercial performance and strengthened capital structure provide capacity to invest in prospective launches, clinical development and business-development opportunities. → SpaceX’s First Earnings Report Could Decide Whether Shorts or Bulls Have Control LIVMARLI generated $129 million in second-quarter net product sales, including $92 million in the United States and $37 million internationally, according to President and Chief Operating Officer Peter Radovich. The company cited new patient starts, therapy persistence and weight-based dose increases as contributors to continued growth in Alagille syndrome. Mirum also said progressive familial intrahepatic cholestasis, or PFIC, remains a driver of LIVMARLI growth. Radovich pointed to increased awareness of later-onset PFIC among adult liver providers and more routine genetic testing. Based on claims data and two years of commercial experience, Mirum estimates an addressable adult PFIC population of at least 2,000 patients in the U.S., with a likely similar population in Europe. → 3 Drone Stocks That Should Soar After the Summer Slump The company’s bile acid medicines generated $48 million in quarterly net product sales. Mirum said its rare genetics business could expand with a potential fourth-quarter U.S. launch of zilurgisertib for fibrodysplasia ossificans progressiva, or FOP, if the product receives FDA approval. Radovich said the FDA review of zilurgisertib was proceeding as expected following a late-cycle meeting, with a September PDUFA date. The company expects an initial launch, if approved, to focus on FOP patients aged 12 and older. Mirum plans to use its existing rare genetics commercial team, which markets Ctexli and CHOLBAM and already serves specialized treatment centers that manage FOP patients. → The Bitcoin Comeback May Already Be Underway—2 ETFs for Exposure Chief Financial Officer Eric Bjerkholt said the updated $680 million to $700 million revenue guidance does not include potential FOP revenue. He said the company expects revenue from the potential product launch to begin more meaningfully in 2027. Mirum said the FDA granted breakthrough therapy designation to volixibat for cholestatic pruritus associated with primary sclerosing cholangitis, or PSC, following results from the VISTAS study. The study met its primary endpoint and showed statistically significant improvements in pruritus in the primary cohort, according to the company. However, Peetz said a recent pre-NDA meeting resulted in an FDA recommendation to conduct a Phase III study. Mirum is not currently aligned with the agency on the proposed NDA package and has delayed its planned filing for volixibat in PSC to the first half of 2027. Management maintained that the VISTAS data set is sufficient to support an application. Peetz said the trial was designed with FDA input and represents the largest randomized clinical study in PSC pruritus, with more than 180 randomized PSC patients and more than 100 patients with one-year safety exposure in the ongoing follow-up. During the question-and-answer session, Peetz said the agency’s comments supporting another Phase III study were not specific and covered general efficacy and safety considerations. He said the company believes VISTAS addressed efficacy, gastrointestinal safety and liver-safety considerations. Mirum intends to use the breakthrough designation to support more frequent interactions with the FDA and expects an iterative dialogue before submission. Peetz said the company may supplement the package with additional accumulated safety data, including longer-term follow-up, and could discuss post-marketing registry or monitoring requirements. He also stated that pruritus remains an approvable endpoint and that Mirum does not expect an accelerated-approval-style confirmatory study after approval. Mirum said enrollment has been completed in the Phase III VANTAGE study of volixibat for pruritus associated with primary biliary cholangitis, or PBC, with more than 330 patients randomized. The company expects top-line results in the first quarter of 2027. Management said it has received recent FDA feedback that VANTAGE can serve as a pivotal study if successful. Peetz said prior discussions related primarily to study size and the analysis plan for the pruritus endpoint, which the company addressed during the trial. Elsewhere in the rare liver pipeline, Mirum expects top-line results later in the quarter from the Phase III portion of the AZURE-1 study of brelovitug. The company also expects four-year data in the fourth quarter and said those results could support a planned biologics license application submission in the first half of 2027. Peetz said the company expects a further Phase III readout, AZURE-4, over the remainder of the year. Mirum also expects fourth-quarter top-line data from the Phase III EXPAND study of LIVMARLI in additional rare cholestatic conditions. Peetz said positive results could support a supplemental NDA next year. The company remains on track to report proof-of-concept data for MRM-3379 in Fragile X syndrome in 2027. Cash, cash equivalents and investments totaled $561 million as of June 30, compared with $391 million at the start of the year. Bjerkholt said commercial cash contribution margin was in the high 50% range during the second quarter and first half, an improvement of about five percentage points from the previous year. Operating expenses totaled $219 million during the quarter, including a $16 million in-process research and development charge tied to the upfront licensing payment for zilurgisertib. Operating cash flow was positive despite increased research and development spending, Bjerkholt said. During the quarter, Mirum issued $690 million in aggregate principal amount of zero-coupon convertible notes due in June 2032, producing $672 million in net proceeds. The company used the transaction in part to settle 75% of its outstanding 2029 notes, adding $197 million of cash to its balance sheet and reducing interest expense, according to Bjerkholt. Mirum Pharmaceuticals, Inc is a late-stage biopharmaceutical company dedicated to the development and commercialization of innovative therapies for rare cholestatic liver diseases. The company's primary focus lies in addressing the unmet medical needs of patients suffering from genetic and progressive forms of pediatric liver disorders, where limited treatment options currently exist. Mirum's lead product candidate, maralixibat (Livmarli), is an ileal bile acid transporter inhibitor designed to reduce systemic bile acid accumulation and alleviate associated pruritus and liver damage. This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to [email protected]. The article "Mirum Pharmaceuticals Q2 Earnings Call Highlights" was originally published by MarketBeat. 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Mirum Pharmaceuticals MIRM reported a loss of 80 cents per share (excluding certain one-time expenses) for the second quarter of 2026, wider than the Zacks Consensus Estimate of a loss of 77 cents. The company had reported a loss of 12 cents per share in the year-ago quarter. Revenues in the second quarter totaled $176.2 million, up 37.9% year over year. The figure also beat the Zacks Consensus Estimate of $165 million. The top line was driven by strong growth of its marketed products, Livmarli (maralixibat) and bile acid medicines, Cholbam and Ctexli (chenodiol). Livmarli is approved for treating cholestatic pruritus in patients with Alagille syndrome worldwide. The drug is also approved for treating certain patients with progressive familial intrahepatic cholestasis in the United States and Europe. The FDA has also approved a new tablet formulation of Livmarli for the treatment of cholestatic pruritus in patients with Alagille syndrome and progressive familial intrahepatic cholestasis. The oral tablet was launched in the United States in June 2025, which is likely to offer convenience for older patients. Mirum acquired Travere Therapeutics’ bile acid products in August 2023, which added the latter’s Cholbam capsules and Ctexli tablets to its portfolio of commercialized drugs. Shares of Mirum have rallied 34% so far this year compared with the industry’s rise of 3.4%. Image Source: Zacks Investment Research Livmarli’s net product sales were $128.7 million in the second quarter, reflecting an increase of 46% year over year. Livmarli sales in the United States were $92 million, reflecting strong demand across all indications. In ex-U.S. markets, Livmarli sales were $37 million. Net product sales of bile acid products, comprising Cholbam and Ctexli tablets, were $47.5 million in the quarter, reflecting an increase of 20% year over year. The company did not record any license and other revenues in the reported quarter. Research and development expenses increased almost 96.3% year over year to $90.5 million. Selling, general and administrative expenses totaled $81.5 million, up almost 28.8% from the year-ago quarter’s level. As of June 30, 2026, Mirum had cash, cash equivalents and investments worth $561.3 million compared with $420.6 million as of March 31, 2026. In view of the strong performance of its marketed products, Mirum raised its revenue guidance for 2026. The company now expects worldwide net product sales of $680-$700 million in 2026 compared with the previous expectation of $660-$680 million. In March 2026, Mirum completed enrollment in the phase III EXPAND study evaluating Livmarli for treating additional rare cholestatic conditions. Top-line data from the same is expected in the fourth quarter of 2026. Mirum’s lead pipeline candidate, volixibat, is currently being evaluated in two phase IIb studies for treating patients with primary biliary cholangitis or PBC (the VANTAGE study) and primary sclerosing cholangitis or PSC (the VISTAS study). The company recently had a pre-new drug application (NDA) meeting with the FDA for volixibat in cholestatic pruritus due to PSC. Mirum is now planning additional discussions with the FDA before a potential NDA submission for volixibat in PSC in the first half of 2027. The FDA has granted Breakthrough Therapy designation and Orphan Drug designation to volixibat for the treatment of cholestatic pruritus due to PSC. Meanwhile, the company expects to report top-line data from the VANTAGE study in the first quarter of 2027. Mirum recently completed the acquisition of privately held biotech Bluejay Therapeutics. Through this deal, it added brelovitug — a fully human monoclonal antibody being developed to treat chronic hepatitis delta virus (“HDV”) — to its pipeline. Top-line data from the phase III AZURE-1 and AZURE-4 studies evaluating brelovitug in HDV are expected in the third and fourth quarters of 2026, respectively. Mirum recently in-licensed exclusive worldwide rights to zilurgisertib, a once-daily oral ALK2 inhibitor from Incyte INCY. The candidate is being developed for treating fibrodysplasia ossificans progressiva (FOP). In June 2026, Mirum and Incyte announced positive phase II results from Cohort 1 of the PROGRESS study evaluating zilurgisertib in adolescents and adults aged 12 years and older with FOP. Cohort 1 results showed meaningful reductions in total heterotopic ossification (HO) lesion volume, new HO lesions and flare activity in adolescents and adults with FOP. These results were shared in a late-breaking presentation at ENDO 2026, the Endocrine Society’s annual meeting. The FDA has accepted the new drug application seeking approval for zilurgisertib in FOP under Priority Review with a target action date of Sept. 26, 2026. Upon potential approval, Mirum expects to launch zilurgisertib in the fourth quarter of 2026. Also, a marketing application for zilurgisertib in FOP has been submitted in Europe. Mirum Pharmaceuticals, Inc. price-consensus-eps-surprise-chart | Mirum Pharmaceuticals, Inc. Quote Mirum currently carries a Zacks Rank #2 (Buy). Some other top-ranked stocks in the biotech sector are Repligen RGEN and Liquidia Corporation LQDA, each currently sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here. Over the past 60 days, estimates for Repligen’s 2026 earnings per share have risen from $1.99 to $2.06, while estimates for 2027 have increased from $2.57 to $2.62 during the same time. RGEN shares have declined 3.4% year to date. Repligen’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 16.80%. Over the past 60 days, estimates for Liquidia’s 2026 earnings per share have risen from $2.97 to $3.02, while estimates for 2027 have increased from $4.81 to $5.31 during the same time. LQDA shares have surged 158.4% year to date. Liquidia’s earnings beat estimates in three of the trailing four quarters, while missing the same on the remaining occasion, with the average surprise being 54.40%. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Mirum Pharmaceuticals, Inc. (MIRM) : Free Stock Analysis Report Incyte Corporation (INCY) : Free Stock Analysis Report Repligen Corporation (RGEN) : Free Stock Analysis Report Liquidia Corporation (LQDA) : Free Stock Analysis Report This article originally published on Zacks Investment Research (zacks.com). Zacks Investment Research

- Q2 2026 net product sales of $176 million - 2026 net product sales guidance increased to $680 million to $700 million - Volixibat granted Breakthrough Therapy and Orphan Drug Designations for cholestatic pruritus due to PSC - Pre-NDA meeting held for volixibat in cholestatic pruritus due to PSC; additional discussions planned before potential NDA submission, now targeted in H1 2027 - Enrollment completed in VANTAGE study of volixibat in cholestatic pruritus due to PBC; data expected Q1 2027 - Conference call to provide business updates today, August 5 at 1:30 p.m. PT / 4:30 p.m. ET FOSTER CITY, Calif., August 05, 2026--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease company, today reported financial results for the second quarter 2026 and provided a business update. "Mirum delivered another strong quarter, with continued commercial momentum supporting an increase to our full-year net product sales guidance and launch readiness for the upcoming potential approval of zilurgisertib," said Chris Peetz, Chief Executive Officer of Mirum. "The FDA’s decision to grant volixibat Breakthrough Therapy and Orphan Drug Designations underscores the strength of the VISTAS efficacy data and the serious unmet need in PSC. VISTAS was designed with FDA input as a pivotal study and met its primary endpoint with highly significant results. However, at our recent pre-NDA meeting, the agency recommended conducting a Phase 3 study. We intend to hold further discussions with the FDA before a potential NDA submission in the first half of 2027 based on VISTAS." Q2 and Recent Highlights Commercial: Raising Full Year Net Product Sales Guidance to $680 Million to $700 Million Second quarter 2026 global net product sales of $176.2 million. Second quarter 2026 LIVMARLI® net product sales were $128.7 million, representing 46% growth over second quarter 2025 net product sales. Second quarter 2026 Bile Acid Medicines net product sales were $47.5 million, representing 20% growth over second quarter 2025 net product sales. Regulatory and Pipeline: Advancing Toward Multiple Milestones U.S. FDA granted volixibat Breakthrough Therapy Designation for the treatment of cholestatic pruritus due to primary sclerosing cholangitis (PSC) and Orphan Drug Designation for PSC. Participated in pre-NDA meeting with U.S. FDA for volixibat in cholestatic pruritus due to PSC; planning additional discussions with the FDA before potential NDA submission, now targeted in H1 2027. Brelovitug AZURE-1 and AZURE-4 Phase 3 studies in chronic hepatitis delta virus (HDV) topline results expected in Q3 and Q4 2026, respectively. LIVMARLI EXPAND Phase 3 study in cholestatic pruritus due to additional rare cholestatic conditions topline results expected in Q4 2026. Completed enrollment in the volixibat VANTAGE Phase 2b study in cholestatic pruritus due to primary biliary cholangitis (PBC); topline results expected in Q1 2027. Presented positive pivotal Phase 2 results from the PROGRESS study of zilurgisertib in fibrodysplasia ossificans progressiva (FOP) at ENDO 2026; Prescription Drug User Fee Act (PDUFA) target action date for the zilurgisertib NDA is September 26, 2026. Corporate & Financial: Strong Balance Sheet and Financial Independence Total revenue for the quarter ended June 30, 2026 was $176.2 million compared to $127.8 million for the quarter ended June 30, 2025. Total operating expenses were $218.8 million for the quarter ended June 30, 2026 compared to $132.8 million for the quarter ended June 30, 2025. Total operating expenses for the quarter ended June 30, 2026 included: Issued $690.0 million aggregate principal amount of 0.00% convertible senior notes due 2032. Settled $237.2 million aggregate principal amount of 4.00% convertible senior notes due 2029, which represented approximately 75% of the then-outstanding notes. As of June 30, 2026, Mirum had unrestricted cash, cash equivalents, and investments of $561.3 million compared to $391.4 million as of December 31, 2025. Business Update Conference Call Mirum will host a conference call today, August 5 at 1:30 p.m. PT / 4:30 p.m. ET, to provide business updates. Join the call using the following details: Conference Call Details: US/Toll-Free: + 1 833 461 5787International: +1 585 542 9983Access Code: 789239699 You may also access the call via webcast by visiting the Investors section of Mirum’s corporate website. The archived webcast will be available for replay. About LIVMARLI® (maralixibat) oral solution and LIVMARLI® (maralixibat) tablets LIVMARLI® (maralixibat) is an orally administered, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for two pediatric cholestatic liver diseases. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. three months of age and older and in Europe for patients two months of age and older. It is also approved in the U.S. for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) 12 months of age and older and in Europe for the treatment of PFIC in patients three months of age and older. For more information for U.S. residents, please visit LIVMARLI.com. LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for the treatment of ALGS and PFIC. LIVMARLI is currently being evaluated in the Phase 3 EXPAND study in additional settings of cholestatic pruritus. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum’s clinical trials section on the company’s website. IMPORTANT SAFETY INFORMATION Limitation of Use: LIVMARLI is not for use in PFIC type 2 patients who have a severe defect in the bile salt export pump (BSEP) protein. LIVMARLI can cause side effects, including: Liver injury. Changes in certain liver tests are common in patients with ALGS and PFIC but can worsen during treatment. These changes may be a sign of liver injury. In PFIC, this can be serious or may lead to liver transplant or death. Your healthcare provider should do blood tests and physical exams before starting and during treatment to check your liver function. Tell your healthcare provider right away if you get any signs or symptoms of liver problems, including nausea or vomiting, skin or the white part of the eye turns yellow, dark or brown urine, pain on the right side of the stomach (abdomen), bloating in your stomach area, loss of appetite or bleeding or bruising more easily than normal. Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea and stomach pain. Your healthcare provider may advise you to monitor for new or worsening stomach problems including stomach pain, diarrhea, blood in your stool or vomiting. Tell your healthcare provider right away if you have any of these symptoms more often or more severely than normal for you. A condition called Fat Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body fat is common in patients with ALGS and PFIC but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment and may monitor for bone fractures and bleeding which have been reported as common side effects. US Prescribing Information EU SmPC Canadian Product Monograph About CHOLBAM® (cholic acid) capsules The FDA approved CHOLBAM® (cholic acid) capsules in March 2015, the first FDA-approved treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects, and for adjunctive treatment of patients with peroxisome biogenesis disorder-Zellweger spectrum disorder. The effectiveness of CHOLBAM has been demonstrated in clinical trials for bile acid synthesis disorders and the adjunctive treatment of peroxisomal disorders. An estimated 200 to 300 patients are current candidates for therapy. CHOLBAM (cholic acid) Indication CHOLBAM is a bile acid indicated for Treatment of bile acid synthesis disorders due to single enzyme defects. Adjunctive treatment of peroxisomal disorders, including Zellweger spectrum disorders, in patients who exhibit manifestations of liver disease, steatorrhea, or complications from decreased fat-soluble vitamin absorption. LIMITATIONS OF USE The safety and effectiveness of CHOLBAM on extrahepatic manifestations of bile acid synthesis disorders due to single enzyme defects or peroxisomal disorders, including Zellweger spectrum disorders, have not been established. IMPORTANT SAFETY INFORMATION WARNINGS AND PRECAUTIONS – Exacerbation of liver impairment Monitor liver function and discontinue CHOLBAM in patients who develop worsening of liver function while on treatment. Concurrent elevations of serum gamma glutamyltransferase (GGT) and alanine aminotransferase (ALT) may indicate CHOLBAM overdose. Discontinue treatment with CHOLBAM at any time if there are clinical or laboratory indicators of worsening liver function or cholestasis. ADVERSE REACTIONS The most common adverse reactions (≥1%) are diarrhea, reflux esophagitis, malaise, jaundice, skin lesion, nausea, abdominal pain, intestinal polyp, urinary tract infection, and peripheral neuropathy. Please see full Prescribing Information for additional Important Safety Information. About CTEXLI® (chenodiol) tablets CTEXLI® (chenodiol) tablets is FDA-approved for the treatment of adults with cerebrotendinous xanthomatosis (CTX). Chenodiol is another name for chenodeoxycholic acid (CDCA). CDCA is a naturally occurring bile acid that was originally approved for the treatment of people with radiolucent stones in the gallbladder. CTEXLI was evaluated as part of the Phase 3 RESTORE study, the first and only clinical trial for CTX. CTX is a rare progressive disease that can affect the brain, spinal cord, tendons, eyes and arteries. IMPORTANT SAFETY INFORMATION CTEXLI can cause side effects, including: Liver Injury: You will need to undergo laboratory testing before starting and while taking CTEXLI to check your liver function. Changes in certain liver tests may occur during treatment and may be a sign of liver injury. This can be serious. Stop taking CTEXLI immediately and tell your healthcare provider right away if you get any signs or symptoms of liver problems, including, stomach (abdomen) pain, bruising, dark-colored urine, feeling tired (fatigue), bleeding, yellowing of the skin and eyes, nausea, and itching. Most Common Side Effects: Diarrhea, headache, stomach pain, constipation, high blood pressure, muscular weakness, and upper respiratory tract infection. Tell your healthcare provider about all the medications that you take, as CTEXLI may interact with other medicines. US Prescribing Information About Volixibat Volixibat is an investigational oral, minimally absorbed agent designed to selectively inhibit the ileal bile acid transporter (IBAT). Volixibat may offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids through inhibition of IBAT, thereby reducing bile acids systemically and in the liver. Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (PSC) (VISTAS study), and primary biliary cholangitis (PBC) (VANTAGE study). In 2026, Mirum shared that the Phase 2b VISTAS study of volixibat in PSC met its primary endpoint, with statistically significant and clinically meaningful reductions in pruritus observed in patients treated with volixibat. Volixibat’s safety profile in the study was generally consistent with the known effects of IBAT inhibition. Volixibat has been granted FDA Breakthrough Therapy designation for the treatment of cholestatic pruritus due to PSC. In 2024, Mirum announced positive interim results from the Phase 2b VANTAGE study of volixibat in PBC. No new safety signals were observed in the study. Volixibat has been granted FDA Breakthrough Therapy designation for the treatment of cholestatic pruritus due to PBC. About Brelovitug Brelovitug is an investigational, highly potent, pan-genotypic, fully human immunoglobulin G1 (IgG1) monoclonal antibody (mAb) that targets the surface antigen (anti-HBsAg) on both the hepatitis delta virus (HDV) and the hepatitis B virus (HBV). Brelovitug is designed to neutralize and remove hepatitis B and hepatitis D virions and deplete HBsAg-containing subviral particles. Brelovitug has FDA Breakthrough Therapy designation for the treatment of chronic HDV infection and PRIME and Orphan designations from the European Medicines Agency. In 2026, Mirum announced that in the Phase 2b portion of the AZURE-1 study in HDV, treatment with brelovitug demonstrated strong antiviral activity in HDV and achieved the primary composite endpoint of virologic response and alanine aminotransferase (ALT) normalization at Week 24 in both brelovitug dose arms as compared to the delayed treatment arm. Favorable safety and tolerability profiles were observed. Brelovitug is currently being evaluated in the global Phase 3 AZURE clinical program. Mirum owns worldwide rights to brelovitug. About Zilurgisertib Zilurgisertib is an investigational, oral, small molecule, activin receptor-like kinase 2 (ALK2) inhibitor in development for the treatment of Fibrodysplasia Ossificans Progressiva (FOP). Zilurgisertib is designed to inhibit the ALK2 receptor, which is abnormally active in most patients with FOP and leads to bone formation in soft tissues, a process known as heterotopic ossification (HO). FOP is an ultra-rare genetic disease that affects approximately 300 patients in the U.S. and 900 worldwide, with diagnosis typically occurring in early childhood. Zilurgisertib was evaluated in the PROGRESS pivotal Phase 2 study, which formed the basis of a new drug application (NDA). The FDA has accepted the NDA for zilurgisertib in FOP under Priority Review with a Prescription Drug User Fee Act (PDUFA) date of September 26, 2026. Mirum Pharmaceuticals, Inc. licensed zilurgisertib from Incyte for worldwide development and commercialization. About MRM-3379 MRM-3379 is an in-licensed investigational oral therapy being evaluated for the treatment of Fragile X syndrome (FXS). It is a selective phosphodiesterase-4D (PDE4D) inhibitor designed to enhance cAMP signaling. MRM-3379 may offer a novel approach to improving cognition, language, and daily function in individuals with FXS. MRM-3379 has been granted FDA Fast Track designation for the treatment of FXS. The BLOOM Phase 2 clinical study of MRM-3379 is currently underway in FXS. Males ages 16 to 45 will be randomly assigned to receive one of three dose levels of MRM-3379 or placebo for 12 weeks. An open-label cohort of boys ages 13 to 16 will receive the lowest dose, in order to explore effects of treatment in younger boys, closer to the age of diagnosis. The study’s primary endpoint is safety and tolerability, the key secondary endpoint is the NIH Toolbox Crystallized Cognition Composite (CCC), and several exploratory endpoints will assess potential effects on mood, behavior, and other symptoms that are relevant to this population. Mirum owns worldwide rights to MRM-3379. About Mirum Pharmaceuticals Mirum Pharmaceuticals (NASDAQ: MIRM) is a leading rare disease company with a global footprint of approved products and a broad pipeline of investigational medicines. Purpose-built to bring forward breakthrough medicines for people with overlooked conditions, Mirum focuses on rare liver and rare genetic diseases, where it has built deep expertise and strong connections to patient communities. The company’s commercial portfolio includes LIVMARLI® (maralixibat) for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC), CHOLBAM® (cholic acid) for bile-acid synthesis disorders, and CTEXLI® (chenodiol) for cerebrotendinous xanthomatosis (CTX). Mirum’s clinical-stage pipeline includes volixibat, an IBAT inhibitor in late-stage development for primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), brelovitug, a fully human monoclonal antibody in late-stage development for chronic hepatitis delta virus (HDV), zilurgisertib, an ALK2 inhibitor under regulatory review with the FDA for fibrodysplasia ossificans progressiva (FOP), and MRM-3379, a PDE4D inhibitor being evaluated for Fragile X syndrome (FXS). Mirum’s success is driven by a team dedicated to advancing high impact medicines through strategic development, disciplined execution and purposeful collaboration across the rare disease ecosystem. Learn more at www.mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and X. Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, commercial results for our approved products, including continued growth in year-over-year net product sales, achievement of our 2026 financial guidance, our anticipated successes in 2026, including continued commercial momentum, the results, enrollment, conduct and progress of our ongoing and planned studies for our product candidates, including the timing and results of interim and topline analyses of our ongoing studies, the occurrence, timing and results of our discussions with the FDA regarding volixibat, potential submission and approval of NDA filings, the timing of any submissions and approvals of NDA filings and the potential commercial launch of our product candidates. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as "anticipate," "expected," "will," "could," "would," "guidance," "target," "intend," "plan," "potential" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirum’s business in general, risks and uncertainties associated with pharmaceutical development and commercialization in general, the impact of geopolitical and macroeconomic events, and the other risks described in Mirum’s Annual Report on Form 10-K for the year ended December 31, 2025 and subsequent filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. Mirum and the Mirum logo are trademarks of Mirum Pharmaceuticals, Inc. View source version on businesswire.com: https://www.businesswire.com/news/home/20260805277342/en/ Contacts Investor Contact:Andrew [email protected] Media Contact:Meredith [email protected]

- Cohort 1 results presented at ENDO 2026 demonstrate meaningful reductions in total heterotopic ossification (HO) lesion volume, new HO lesions and flare activity in adolescents and adults with FOP - U.S. Food and Drug Administration (FDA) accepted the New Drug Application (NDA) for zilurgisertib in FOP under Priority Review FOSTER CITY, Calif. & WILMINGTON, Del., June 14, 2026--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq:MIRM) and Incyte (Nasdaq:INCY) today announced pivotal Phase 2 results from Cohort 1 of the PROGRESS study evaluating zilurgisertib, an investigational oral activin receptor-like kinase 2 (ALK2) inhibitor, in adolescents and adults (≥12 years of age) with fibrodysplasia ossificans progressiva (FOP). Results were shared in a late-breaking rapid-fire presentation at ENDO 2026, the Endocrine Society’s annual meeting. Results from Cohort 1 of the PROGRESS study demonstrated a consistent treatment effect across measures of disease activity and durability through Week 48. During the open-label extension, no new HO lesions were observed among patients who continued to receive zilurgisertib or among placebo-treated patients who crossed over to active treatment at Week 24. "The findings presented at ENDO represent an important milestone for the zilurgisertib program and further strengthen the growing body of clinical evidence supporting its potential as a treatment for FOP," said Steven Stein, M.D., Executive Vice President, Chief Medical Officer and Head of Late-Stage Development at Incyte. "People living with FOP and their families urgently need additional treatment options," said Joanne Quan, M.D., Chief Medical Officer at Mirum Pharmaceuticals. "These results reinforce our confidence in the potential of zilurgisertib and our commitment to working with Incyte to bring this important program forward as we prepare for potential commercialization and support the FOP community." Cohort 1 of the PROGRESS study evaluated zilurgisertib 100 mg once-daily in 63 adolescents and adults (≥12 years of age) with FOP. Patients were randomized 1:1 to receive zilurgisertib (n=32) or placebo (n=31) during a 24-week, placebo-controlled, double-blind period, followed by an open-label extension period. Baseline demographics and disease characteristics were generally balanced between treatment groups, with a mean age of approximately 21 years and evidence of recent disease activity prior to enrollment. A total of 61 patients had 48-week whole-body CT scan data available at the time of the open-label extension analysis. Key efficacy findings included: Fewer patients receiving zilurgisertib developed new HO lesions at Week 24, with an 81% reduction versus placebo (p=0.0986). 99.9% reduction in total volume of new HO lesions in patients receiving zilurgisertib versus placebo at Week 24 (nominal p-value<0.0001). Reduction in total existing HO lesion volume compared with an increase observed in placebo-treated patients at Week 24 (nominal p-value=0.004). Among patients receiving zilurgisertib, no new HO lesions were observed and total HO lesion volume continued to decrease from Week 24 to Week 48. Among patients who crossed over from placebo to zilurgisertib, no new HO lesions were observed and total HO lesion volume decreased from Week 24 to Week 48. Key Efficacy Findings (Week 24 Placebo-Controlled Period and Week 48 Crossover) Zilurgisertib was generally well-tolerated during the 24-week placebo-controlled period of the study. Data showed: Most adverse events were mild or moderate in severity. No adverse events led to treatment discontinuation or dose reduction. Serious adverse events and Grade ≥3 adverse events occurred at low rates in both treatment groups. The most commonly reported adverse events among patients receiving zilurgisertib were FOP flare-up or aching/pain due to FOP (25%), headache (21.9%), upper respiratory tract infection (21.9%), arthralgia (18.8%), epistaxis (12.5%), and nausea (12.5%). The full abstract is available on the Endocrine Society’s ENDO 2026 website. Detailed analyses are also posted on the Publications & Presentations section of Mirum’s website. The U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for zilurgisertib for the treatment of FOP in patients 12 years of age and older and granted Priority Review. The Prescription Drug User Fee Act (PDUFA) target action date for zilurgisertib is September 26, 2026. About Zilurgisertib Zilurgisertib is an investigational, oral, small molecule, activin receptor-like kinase 2 (ALK2) inhibitor in development for the treatment of Fibrodysplasia Ossificans Progressiva (FOP). Zilurgisertib is designed to inhibit the ALK2 receptor, which is abnormally active in most patients with FOP and leads to bone formation in soft tissues, a process known as heterotopic ossification (HO). FOP is an ultra-rare genetic disease that affects approximately 300 patients in the U.S. and 900 worldwide, with diagnosis typically occurring in early childhood. Zilurgisertib was evaluated in the PROGRESS pivotal Phase 2 study, which formed the basis of a new drug application (NDA). The FDA has accepted the NDA for zilurgisertib in FOP under Priority Review with a Prescription Drug User Fee Act (PDUFA) date of September 26, 2026. Mirum Pharmaceuticals, Inc. licensed zilurgisertib from Incyte for worldwide development and commercialization. About the PROGRESS Study PROGRESS is a global, randomized, double-blind, placebo-controlled Phase 2 study evaluating the efficacy and safety of zilurgisertib in patients with fibrodysplasia ossificans progressiva (FOP). PROGRESS Cohort 1 enrolled patients 12 years of age and older who were randomized 1:1 to receive zilurgisertib 100 mg once daily or placebo during a 24-week double-blind treatment period, followed by an open-label extension. Additional PROGRESS cohorts will evaluate the efficacy and safety of zilurgisertib in patients ages 6 to <12 years of age (Cohort 2) and in patients ages 2 to <12 years of age (Cohort 3). The primary endpoint of the study is the proportion of Cohort 1 patients with new heterotopic ossification (HO) lesions at Week 24 as assessed by whole-body CT scan data. Key secondary endpoints include the number and total volume of new HO lesions, changes in total HO lesion volume and flare activity through Week 24. About Mirum Pharmaceuticals Mirum Pharmaceuticals (NASDAQ: MIRM) is a leading rare disease company with a global footprint of approved products and a broad pipeline of investigational medicines. Purpose-built to bring forward breakthrough medicines for people with overlooked conditions, Mirum focuses on rare liver and rare genetic diseases, where it has built deep expertise and strong connections to patient communities. The company’s commercial portfolio includes LIVMARLI® (maralixibat) for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC), CHOLBAM® (cholic acid) for bile-acid synthesis disorders, and CTEXLI® (chenodiol) for cerebrotendinous xanthomatosis (CTX). Mirum’s clinical-stage pipeline includes volixibat, an IBAT inhibitor in late-stage development for primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), brelovitug, a fully human monoclonal antibody in late-stage development for chronic hepatitis delta virus (HDV), zilurgisertib, an ALK2 inhibitor under regulatory review with the FDA for fibrodysplasia ossificans progressiva (FOP), and MRM-3379, a PDE4D inhibitor being evaluated for Fragile X syndrome (FXS). Mirum’s success is driven by a team dedicated to advancing high impact medicines through strategic development, disciplined execution and purposeful collaboration across the rare disease ecosystem. Learn more at www.mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and X. About Incyte® Incyte is redefining what’s possible in biopharmaceutical innovation. Through deep scientific expertise and a relentless focus on patients, we have built an established portfolio of first-in-class medicines and an extensive portfolio of next-generation medicines across our key franchises: Hematology, Oncology and Inflammation & Autoimmunity. To learn more, visit Incyte.com and Investor.Incyte.com. Follow us on social media: LinkedIn, X and Instagram. Mirum Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the Company’s planned participation at a scientific congress, Mirum’s continued advancement of zilurgisertib with Incyte, the likelihood of a FDA approval pathway for zilurgisertib and the potential benefit of zilurgisertib in real world settings versus scientific presentations of data. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as "expected," "will," "could," "would," "guidance," "potential," "continue" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirum’s business in general, the impact of geopolitical and macroeconomic events, and the other risks described in Mirum’s Annual Report for the year ended December 31, 2025, filed with the Securities and Exchange Commission on February 25, 2026, and subsequent filings with the Securities and Exchange Commission, which are available at www.sec.gov. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. Incyte Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding the presentation of data from the PROGRESS study; the potential for zilurgisertib to become a treatment option for people living with FOP; expectations regarding ongoing and future clinical trials for zilurgisertib, including the timing of such trials; and Incyte’s aspirations and goals as set forth under the heading "About Incyte." Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including the sufficiency of clinical trial data to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials and the ability to enroll subjects in accordance with planned schedules; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; the efficacy or safety of Incyte’s and its partners’ products; the ability of Incyte and its partners to achieve commercial success for their marketed products and product candidates, if approved; Incyte’s and its partners’ ability to obtain and maintain protection of intellectual property for their products and technology; Incyte’s reliance on third parties and partners; the acceptance of Incyte’s and its partners’ products in the marketplace; market competition, sales, marketing, manufacturing and distribution requirements; greater than expected expenses, including expenses relating to litigation or strategic activities; and those risks and uncertainties discussed in greater detail in Incyte’s reports filed with the U.S. Securities and Exchange Commission, including its annual report on Form 10-K for the year ended December 31, 2025, and its quarterly report on Form 10-Q for the quarter ended March 31, 2026. Incyte disclaims any intent or obligation to update these forward-looking statements. Mirum and the Mirum logo are trademarks of Mirum Pharmaceuticals, Inc. View source version on businesswire.com: https://www.businesswire.com/news/home/20260614539468/en/ Contacts Mirum Investor Contact: Andrew [email protected] Mirum Media Contact: Meredith [email protected] Incyte Investor Contact: [email protected] Incyte Media Contact: [email protected]

It has been about a month since the last earnings report for Mirum Pharmaceuticals, Inc. (MIRM). Shares have lost about 10.1% in that time frame, underperforming the S&P 500. But investors have to be wondering, will the recent negative trend continue leading up to its next earnings release, or is Mirum Pharmaceuticals due for a breakout? Before we dive into how investors and analysts have reacted as of late, let's take a quick look at its most recent earnings report in order to get a better handle on the important catalysts. Mirum incurred a loss of 39 cents per share (excluding certain one-time expenses) in the first quarter of 2026, narrower than the Zacks Consensus Estimate of a loss of 40 cents. The company reported a loss of 30 cents per share in the year-ago quarter. Revenues in the first quarter totaled $159.9 million, up 43.3% year over year. The figure also beat the Zacks Consensus Estimate of $148 million. The top line was driven by the strong growth of Livmarli and bile acid medicines, Cholbam and Ctexli. Livmarli’s net product sales were $113.8 million in the first quarter, reflecting an increase of 55% year over year. Livmarli sales in the United States were $84 million, reflecting strong demand across all indications. In ex-U.S. markets, Livmarli sales were $30 million. Net product sales of bile acid products, comprising Cholbam and Ctexli tablets, were $46.1 million in the first quarter, reflecting an increase of 20% year over year. The company did not record any license and other revenues in the reported quarter. Research and development expenses increased almost 138.8% year over year to $97.9 million. Selling, general and administrative expenses totaled $96.3 million, up almost 66.9% from the year-ago quarter’s level. As of March 31, 2026, Mirum had cash, cash equivalents and investments worth $420.6 million compared with $391.4 million as of Dec. 31, 2025. Reflecting the strong performance of its marketed products, Mirum raised the full-year revenue guidance for 2026. The company now expects worldwide net product sales of approximately $660-$680 million in 2026, compared with the previous expectation of $630-$650 million. It turns out, estimates review have trended downward during the past month. The consensus estimate has shifted -41.47% due to these changes. At this time, Mirum Pharmaceuticals has a poor Growth Score of F, however its Momentum Score is doing a bit better with a D. Following the exact same course, the stock was allocated a grade of D on the value side, putting it in the bottom 40% for this investment strategy. Overall, the stock has an aggregate VGM Score of F. If you aren't focused on one strategy, this score is the one you should be interested in. Estimates have been broadly trending downward for the stock, and the magnitude of these revisions indicates a downward shift. Notably, Mirum Pharmaceuticals has a Zacks Rank #3 (Hold). We expect an in-line return from the stock in the next few months. Mirum Pharmaceuticals belongs to the Zacks Medical - Biomedical and Genetics industry. Another stock from the same industry, Axsome Therapeutics (AXSM), has gained 5.1% over the past month. More than a month has passed since the company reported results for the quarter ended March 2026. Axsome reported revenues of $191.2 million in the last reported quarter, representing a year-over-year change of +57.4%. EPS of -$1.26 for the same period compares with -$0.80 a year ago. Axsome is expected to post a loss of $0.83 per share for the current quarter, representing a year-over-year change of +9.8%. Over the last 30 days, the Zacks Consensus Estimate remained unchanged. Axsome has a Zacks Rank #3 (Hold) based on the overall direction and magnitude of estimate revisions. Additionally, the stock has a VGM Score of D. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Mirum Pharmaceuticals, Inc. (MIRM) : Free Stock Analysis Report Axsome Therapeutics, Inc. (AXSM) : Free Stock Analysis Report This article originally published on Zacks Investment Research (zacks.com). Zacks Investment Research

- Late-breaking rapid-fire presentation to include results from Cohort 1 of the placebo-controlled PROGRESS study evaluating zilurgisertib in fibrodysplasia ossificans progressiva (FOP) FOSTER CITY, Calif. & WILMINGTON, Del., June 04, 2026--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) and Incyte (Nasdaq: INCY) today announced that pivotal Phase 2 results from the PROGRESS study evaluating zilurgisertib, an investigational ALK2 inhibitor, in patients with fibrodysplasia ossificans progressiva ("FOP") will be presented at ENDO 2026, the Endocrine Society’s annual meeting, taking place June 13-16, 2026, in Chicago, Illinois. The late-breaking rapid-fire presentation will include results from Cohort 1 of the placebo-controlled PROGRESS study, which enrolled patients 12 years of age and older and formed the basis of the New Drug Application (NDA) for zilurgisertib to the U.S. Food and Drug Administration (FDA). In April 2026, Mirum entered into an exclusive license agreement with Incyte for worldwide rights to zilurgisertib. "ENDO 2026 is an important milestone for the zilurgisertib FOP program as we share pivotal results from the PROGRESS study in patients living with this debilitating disease," said Steven Stein, M.D., Executive Vice President, Chief Medical Officer and Head of Late-stage Development at Incyte. "These data add to the growing clinical understanding of zilurgisertib’s potential in FOP as Incyte and Mirum continue to advance toward the FDA’s Priority Review PDUFA date of September 26, 2026." "FOP is a devastating, progressive disease that profoundly impacts patients and families," said Joanne Quan, M.D., Chief Medical Officer at Mirum Pharmaceuticals. "At Mirum, in partnership with Incyte, we are committed to advancing zilurgisertib with urgency as we work toward potentially bringing a needed new treatment option to people living with FOP." Congress Presentation Additional details regarding the presentation are as follows: Abstracts accepted for presentation at ENDO 2026 will be available once published through the Endocrine Society’s ENDO 2026 website. About Zilurgisertib Zilurgisertib is an investigational, oral, small molecule, activin receptor-like kinase 2 (ALK2) inhibitor in development for the treatment of Fibrodysplasia Ossificans Progressiva (FOP). Zilurgisertib is designed to inhibit the ALK2 receptor, which is abnormally active in most patients with FOP and leads to bone formation in soft tissues, a process known as heterotopic ossification (HO). FOP is an ultra-rare genetic disease that affects approximately 300 patients in the U.S. and 900 worldwide, with diagnosis typically occurring in early childhood. Zilurgisertib was evaluated in the PROGRESS pivotal Phase 2 study, which formed the basis of a new drug application (NDA). The FDA has accepted the NDA for zilurgisertib in FOP under Priority Review with a Prescription Drug User Fee Act (PDUFA) date of September 26, 2026. Mirum licensed zilurgisertib from Incyte for development and commercialization globally. About the PROGRESS Study PROGRESS is a global, randomized, double-blind, placebo-controlled Phase 2 study evaluating the efficacy and safety of zilurgisertib in patients with fibrodysplasia ossificans progressiva (FOP). PROGRESS Cohort 1 enrolled patients 12 years of age and older who were randomized 1:1 to receive zilurgisertib 100 mg once daily or placebo during a 24-week double-blind treatment period, followed by an open-label extension. Additional PROGRESS cohorts will evaluate the efficacy and safety of zilurgisertib in patients ages 6 to <12 years of age (Cohort 2) and in patients ages 2 to <12 years of age (Cohort 3). The primary endpoint of the study is the proportion of Cohort 1 patients with new heterotopic ossification (HO) lesions at Week 24 as assessed by whole-body CT scan data. Key secondary endpoints include the number and total volume of new HO lesions, changes in total HO lesion volume and flare activity through Week 24. About Mirum Pharmaceuticals Mirum Pharmaceuticals (NASDAQ: MIRM) is a leading rare disease company with a global footprint of approved products and a broad pipeline of investigational medicines. Purpose-built to bring forward breakthrough medicines for people with overlooked conditions, Mirum focuses on rare liver and rare genetic diseases, where it has built deep expertise and strong connections to patient communities. The company’s commercial portfolio includes LIVMARLI® (maralixibat) for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC), CHOLBAM® (cholic acid) for bile-acid synthesis disorders, and CTEXLI® (chenodiol) for cerebrotendinous xanthomatosis (CTX). Mirum’s clinical-stage pipeline includes volixibat, an IBAT inhibitor in late-stage development for primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), brelovitug, a fully human monoclonal antibody in late-stage development for chronic hepatitis delta virus (HDV), zilurgisertib, an ALK2 inhibitor under regulatory review with the FDA for fibrodysplasia ossificans progressiva (FOP), and MRM-3379, a PDE4D inhibitor being evaluated for Fragile X syndrome (FXS). Mirum’s success is driven by a team dedicated to advancing high impact medicines through strategic development, disciplined execution and purposeful collaboration across the rare disease ecosystem. Learn more at www.mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and X. About Incyte® Incyte is redefining what’s possible in biopharmaceutical innovation. Through deep scientific expertise and a relentless focus on patients, we have built an established portfolio of first-in-class medicines and an extensive portfolio of next-generation medicines across our key franchises: Hematology, Oncology and Inflammation & Autoimmunity. To learn more, visit Incyte.com and Investor.Incyte.com. Follow us on social media: LinkedIn, X and Instagram. Mirum Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the Company’s planned participation at a scientific congress, Mirum’s continued advancement of zilurgisertib with Incyte, the FDA approval pathway for zilurgisertib and the potential benefit of zilurgisertib in real world settings versus scientific presentations of data. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as "expected," "will," "could," "would," "guidance," "potential," "continue" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirum’s business in general, the impact of geopolitical and macroeconomic events, and the other risks described in Mirum’s Annual Report for the year ended December 31, 2025, filed with the Securities and Exchange Commission on February 25, 2026, and subsequent filings with the Securities and Exchange Commission, which are available at www.sec.gov. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. Incyte Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding the potential and promise suggested by the Phase 2 PROGRESS results, the potential for zilurgisertib to become a treatment option for people living with FOP, Incyte’s plans and expectations for the PROGRESS study and Incyte’s aspirations and goals as set forth under the heading "About Incyte". Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including the sufficiency of clinical trial data to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; the ability of Incyte’s collaborators to achieve commercial success for their marketed products and product candidates, if approved; Incyte’s and Incyte’s collaborators’ ability to obtain and maintain protection of intellectual property for their products and technology; Incyte’s reliance on third parties and partners; the acceptance of Incyte’s collaborators’ products in the marketplace; market competition, sales, marketing, manufacturing and distribution requirements; and those risks and uncertainties discussed in greater detail in Incyte’s reports filed with the U.S. Securities and Exchange Commission, including its annual report on Form 10-K and its quarterly report on Form 10-Q for the quarter ended March 31, 2026. Incyte disclaims any intent or obligation to update these forward-looking statements. Mirum and the Mirum logo are trademarks of Mirum Pharmaceuticals, Inc. View source version on businesswire.com: https://www.businesswire.com/news/home/20260604863826/en/ Contacts Mirum Investor Contact: Andrew [email protected] Mirum Media Contact: Meredith [email protected] Incyte Investor Contact [email protected] Incyte Media Contact [email protected]