Q2 2026 Mirum Pharmaceuticals Inc Earnings Call

August 5, 2026

Operator 2: Good afternoon, welcome to Mirum Pharmaceuticals' Q2 2026 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.

Operator: Good afternoon, welcome to Mirum Pharmaceuticals' Q2 2026 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.

Speaker #2: Afternoon, and welcome to Mirum Pharmaceuticals' second quarter 2026 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.

Speaker #2: I would now like to hand the conference over to Andrew McGibben, SVP of Strategic Finance and Investor Relations, please go ahead.

Speaker #3: Thank you, Alexandra, and good afternoon, everyone. I'd like to welcome you to Mirum Pharmaceuticals' second quarter 2026 conference call. I'm joined today by our Chief Executive Officer, Chris Peetz, our President and Chief Operating Officer, Peter Radovich, and Eric Bjerkholt, our Chief Financial Officer.

Andrew McKibben: Thank you, Alexandra, good afternoon, everyone. I'd like to welcome you to Mirum Pharmaceuticals' Q2 2026 conference call. I'm joined today by our Chief Executive Officer, Chris Peetz, our President and Chief Operating Officer, Peter Radovich, and Eric Bjerkholt, our Chief Financial Officer. Laura 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 Q2 2026. Copies of the press release and our SEC filings are available on the investors section of our website.

Speaker #3: Lara Longprey, 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.

Speaker #3: Earlier today, Mirum issued a press release 2026. Copies of the press release and our SEC filings are available on the investors section of our website.

Speaker #3: Before we start, I'd like to remind you that during the course of this conference call, we will be making certain forward-looking statements based on management's current expectations, including statements regarding Mirum's programs and market opportunities for its approved medicines and product candidates, and financial guidance.

Andrew McKibben: Before we start, I'd like to remind you that during the course of this conference call, we will be making certain forward-looking statements based on management's current expectations, including statements regarding 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 SEC filings for more information about these risks and uncertainties. With that said, I'd like to turn the call over to Chris. Chris?

Speaker #3: These statements represent our judgment and knowledge of events as of today and inherently involve risks and uncertainties that may cause actual results to announcing the company's results for the second quarter of differ materially from the results discussed.

Speaker #3: We are under no duty to update these statements. Please refer to the risk factors in our latest Form 10-Q and subsequent SEC filings for more information about these risks and uncertainties.

Speaker #3: With that said, I'd like to turn the call over to Chris. Chris?

Speaker #4: Thanks, Andrew. And good afternoon, everyone. At Mirum, we're 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.

Chris Peetz: Thanks, Andrew, good afternoon, everyone. At Mirum, we're 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 a strengthened capital structure and overall financial performance, giving us greater capacity to invest throughout the business. In Q2, 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 to 700 million.

Speaker #4: We have a busy pipeline with multiple pivotal readouts in the quarters ahead, all delivered with the strengthened capital structure and overall financial performance, giving us greater capacity to invest throughout the business.

Speaker #4: In the second quarter, our commercial business generated 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 to $700 million.

Speaker #4: Fueled by this strong commercial performance, we're 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 Zolygcerted for SOP, with a PDUFA date next month.

Chris Peetz: Fueled by the strong commercial performance, we're 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 a PDUFA date next month. This is fast progress for a program added to our rare genetic business only in Q2. Peter will cover more of the launch profile in his remarks. Moving to the pipeline for our rare liver business, it's important to spend some time today on volixibat and PSC, which just had some key US regulatory interactions. As a reminder of the background of the VISTA study of volixibat and cholestatic pruritus and 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.

Speaker #4: 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.

Speaker #4: Moving to the pipeline for our rare liver business, it's important to spend some time today on Valixavat and PFC, which just had some key U.S. developments.

Speaker #4: regulatory interactions. First, as a reminder of the background of the Vista study of valixavat and cholestatic pruritus and PFC, 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.

Speaker #4: As we've announced previously and presented at ESEL this year, Vista's 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.

Chris Peetz: We've announced previously and presented at EASL this year, VISTA 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. The FDA recommended conducting a phase III study.

Speaker #4: We are excited to share that the FDA has now granted breakthrough therapy designation for valixavat and cholestatic pruritus due to PFC, based on these strong results.

Speaker #4: We see this as recognition of the potential for valixavat to address a serious unmet need in PFC. As planned, we recently held a pre-NDA discussion with the agency about the submission of an NDA based on the Vista study.

Speaker #4: In the meeting, the FDA recommended conducting a phase three study. We believe the Vista study provides a robust and clear dataset to characterize the use of valixavat in patients with pruritus due to PFC and is a clinically and statistically highly persuasive study.

Chris Peetz: We believe the VISTA study provides a robust and clear data set to characterize the use of volixibat in patients with colitis due to PSC and is a clinically and statistically highly persuasive study. VISTA is the largest randomized clinical study conducted in patients with colitis 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. 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 H1 of next year.

Chris Peetz: We believe the VISTA study provides a robust and clear data set to characterize the use of volixibat in patients with colitis due to PSC and is a clinically and statistically highly persuasive study. VISTA is the largest randomized clinical study conducted in patients with colitis due to PSC, with an extensive overall data package that includes more than 180 PSC patients randomized, one-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. 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 H1 of next year.

Speaker #4: VISTAS is the largest randomized clinical study conducted in patients with pruritus due to PFC, with an extensive overall data package that includes more than 180 PFC patients randomized, one-year safety exposure data for over 100 PFC patients, and growing results from an independent committee evaluating liver safety—all totaling over 600 subjects across the clinical program today.

Speaker #4: 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.

Speaker #4: This engagement will delay the planned timing of our NDA submission, which we are now targeting for the first half of next year. We're positioned to move quickly once we have further clarity from the agency, and we'll provide updates as we work towards our goal of bringing a much-needed therapy to this unaddressed clinical setting.

Chris Peetz: 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 colitis 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 brelovitug's AZUR-1 top-line results later this quarter. This is the readout of the phase III portion, following strong results of the phase IIb portion earlier this year.

Speaker #4: In parallel, the Vantage study and PDC is progressing well, and has completed enrollment reaching over 330 patients randomized. In PDC, our earlier breakthrough therapy designation has enabled more dialogue with the agency during the conduct of the study, and we have recent feedback from FDA for Vantage to serve as a pivotal study of valixavat and pruritus due to PDC if the study is successful at its first quarter top-line readout next readout for the rare liver business is expected to be below a tug's Azure One top-line results later this quarter.

Speaker #4: This is the readout of the phase three portion following strong results of the phase two B portion earlier this year, and we also continue to expect Azure Four data in the fourth quarter, which keeps us on track for a potential BLA submission for this breakthrough therapy designated program in the first half of next year.

Chris Peetz: We also continue to expect AZUR-4 data in Q4, which keeps us on track for a potential BLA submission for this breakthrough therapy-designated program in H1 of next year. Rounding out the rare liver pipeline highlights, the phase III EXPAND study of LIVMARLI and additional rare cholestatic conditions remains on track for top-line data in Q4. 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, and 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, and I am excited about what lies ahead for Mirum.

Speaker #4: And rounding out the rare liver pipeline highlights, the phase three expand study of Livmarley and additional rare cholestatic conditions remains on track for top-line data in the fourth quarter.

Speaker #4: 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, and opportunistic business development where we see a compelling strategic fit and the potential to create value.

Speaker #4: I'm proud of the team's progress and the promise of our current medicines and pipeline, and I'm excited about what lies ahead for Mirum. And with that, I'll turn the call over to Peter to discuss our commercial performance and launch readiness in more detail.

Chris Peetz: With that, I will turn the call over to Peter to discuss our commercial performance and launch readiness in more detail. Peter?

Speaker #4: Peter.

Speaker #5: Thanks, Chris. The second quarter was another strong quarter for Mirum's commercial business, with total net product sales of $176 million. Livmarley 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 to $700 million.

Peter Radovich: Thanks, Chris. The Q2 was another strong quarter for Mirum's commercial business, with total net product sales of $176 million. LIVMARLI and the bile acid medicines both continue to perform well. Based on the demand we see, we are increasing our full year 2026 net product sales guidance to $680 to $700 million. Q2 net product sales for LIVMARLI were $129 million, with the US 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 diagnoses. We're particularly encouraged by the growing contribution from adult PFIC patients. We're seeing an increase in prescriptions from adult liver providers as awareness of later-onset PFIC grows and genetic testing becomes more routine.

Speaker #5: The second quarter net product sales for Livmarley were $129 million, with the U.S. contributing $92 million. Alogel growth remains durable, supported by continued new patient starts, sustained persistence on therapy, and weight-based dose increases.

Speaker #5: PFAT continues to be an important driver of growth, fueled by new diagnoses. We're particularly encouraged by the growing contribution from adult PFAT patients. We're seeing an increase in prescriptions from adult liver providers as awareness of later-onset PFAT grows and genetic testing becomes more routine.

Speaker #5: Based on claims data as well as insights from two years in-market, we now estimate an addressable adult PFAT population of at least 2,000 patients in the United States, with likely a similar number in Europe.

Peter Radovich: 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. Because genetic testing remains less established in adult practices than in pediatric, we believe the vast majority of the estimated 2,000 addressable patients don't yet have a PFIC diagnosis. 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.

Speaker #5: And because genetic testing remains less established in adult practices than in pediatric, we believe the vast majority of the estimated 2,000 addressable patients don't yet have a PFAT diagnosis, and we see a meaningful opportunity to continue expanding diagnosis through education.

Speaker #5: 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.

Speaker #5: On the rare genetic disease side of the business, our bile acid medicines continue to provide a steady contribution generating $48 million in net product sales for the quarter.

Speaker #5: Like our rare liver business, our rare genetic our rare genetics business is also poised for growth with the recent addition of Zalurga Certive for FOP.

Peter Radovich: Like our rare liver business, 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. Following a recent late-cycle meeting with the FDA, we believe the NDA is proceeding as expected towards the September PDUFA date. We are preparing for potential US launch in the Q4.

Speaker #5: Data from the pivotal phase two progress study of Zalurga Certive presented at ENDO showed a compelling clinical profile and FOP patients ages 12 and older.

Speaker #5: Based on the strong efficacy observed and the convenience of oral dosing, we believe Zalurga Certive has the potential to offer an attractive profile to patients with the severely debilitating disease.

Speaker #5: 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.

Speaker #5: And following a recent late cycle meeting with the FDA, we believe the NDA is proceeding as expected towards the September PDUPA date and we are preparing for potential U.S.

Speaker #5: Launch in the fourth quarter. The U.S. launch will heavily leverage the rare genetics team we have in place now that currently markets our bile acid medicines, as the physicians who manage FOP are largely concentrated in the same specialized centers where Sutexley and Colbalm are prescribed, building on the efficiency of our rare genetics business.

Peter Radovich: The US launch will heavily leverage the rare genetics team we have in place now that currently markets our bile acid medicines, as the physicians who manage FOP are largely concentrated in the same specialized centers where Staxyn and CHOLBAM are prescribed, building on the efficiency of our rare genetics business. Also, a marketing application for zilurgisertib has been submitted in Europe. We will provide updates as this filing progresses. Overall, we are pleased with the continued execution across the commercial organization. We're 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'll turn it over to Eric to discuss the financial results. Eric?

Speaker #5: Also, a marketing application for Zalurga Certive 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.

Speaker #5: We're seeing strong demand throughout the existing portfolio while making the investments necessary to support the next wave of potential launches. And we believe that we are well positioned for the remainder of the year and beyond.

Speaker #5: With that, I'll turn it over to Eric to discuss the financial results. Eric?

Speaker #3: Thanks, Peter. And good afternoon, everyone. Today, I'll walk through the financials of another excellent quarter for 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.

Eric Bjerkholt: Thanks, Peter, and good afternoon, everyone. Today, I'll walk through the financials of another excellent quarter for Mirum. Net product sales for Q2 were $176 million compared to net product sales of $128 million in Q2 of last year. Cash, cash equivalents, and investments as of 30 June were $561 million compared with $391 million at the beginning of the year. In Q2 and H1 of 2026, the cash contribution margin from our commercial business was in the high 50s%, approximately a 5 percentage point improvement over the year before. Total operating expense for the quarter ended 30 June was $19 million, which includes $16 million of in-process R&D expense associated with the upfront payment of licensing zilurgisertib.

Speaker #3: Cash, cash equivalents, and investments as of June 30th were $561 million compared with $391 million at the beginning of the year. In the second quarter and first half of 2026, the cash contribution margin from our commercial business was in the high 50th percent, approximately a 5 percentage point improvement over the year before.

Speaker #3: Total operating expense for the quarter ended June 30th was $290 million, which includes $16 million of in-process R&D expense associated with the upfront payment of licensing, Zalurga Certive.

Speaker #3: R&D expense of $76 million including $29 million related to the development of Brelovitag, SG&A expense of $66 million, and cost of sales of $23 million all excluding stock-based compensation expense and intangible amortization.

Eric Bjerkholt: R&D expense of $76 million, including $29 million related to the development of brelovitug, 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 non-cash expenses totaled $37 million for the quarter. Operating cash flow was positive in Q2 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.

Speaker #3: Stock-based compensation, intangible amortization, and other non-cash expenses totaled $37 million for the quarter. Operating cash flow was positive in the second quarter despite the expected increase in R&D expense.

Speaker #3: During the quarter, we significantly improved our capital structure through the issuance of $690 million aggregate principal amount of 0 percent coupon convertible notes due in June 2032.

Speaker #3: Net proceeds from the offering were $672 million. In conjunction with this financing, we settled 75 percent of the outstanding 2029 notes, added $197 million of cash to the balance sheet, and significantly reduced our interest expense.

Speaker #3: 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.

Eric Bjerkholt: Our commercial business continues to scale, and our pipeline includes multiple near-term value drivers. With that, I'll turn the call back to Chris for closing remarks.

Speaker #3: With that, I'll turn a call back to Chris for closing remarks.

Speaker #5: 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, with Marley as well on its way to realizing its over $1 billion peak revenue potential.

Chris 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 million 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. 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 to PSC patients. The balance of the pipeline is firing on all cylinders.

Speaker #5: And our rare genetics team is thrilled about the potential launch of Zalurga Certive for FOP patients in the coming months. On the pipeline, we are focused on a collaborative discussion with FDA on the VISTA study and PSC.

Speaker #5: The clinical results are clear and breakthrough therapy designation gives us further opportunity to engage with FDA. All said, we have a plan to advance fully to batch the PSC patients.

Speaker #5: The balance of the pipeline is firing on all cylinders. Recapping our upcoming clinical milestones, we expect clinical readouts from the phase three of 01 and 04 studies of Brelovitag over the balance of the year, which will enable our planned BLA submission in the first half of next year.

Peter Radovich: Recapping our upcoming clinical milestones, we expect clinical readouts from the phase III AZUR1 and AZUR4 studies of brelovitug over the balance of the year, which will enable our planned BLA submission in H1 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.

Speaker #5: Next quarter, we also expect to announce top-line results from the EXPAN study of both Maralixibat and additional rare cholestatic diseases, setting up the potential for an sNDA next year as well.

Speaker #5: And into 2027, we expect top-line results from the Vantage study of fully to batch PBC first quarter and finally, we're on track with MRM 3379 for proof of concept data and fragile X syndrome next year.

Chris Peetz: Into 2027, we expect top-line results from the VANTAGE Study of volixibat at PBC Q1. Finally, we're on track with MRM-3379 for proof of concept data in Fragile X syndrome next year. Importantly, we're 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, the patients, families, and physicians who continue to partner with us in this work. With that, operator, please open the call for questions.

Speaker #5: Importantly, we're 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 the patients, families, and physicians who continue to partner with us in this work.

Speaker #5: With that, operator, please open the call for questions.

Speaker #4: We will now begin the question and answer session. Please limit yourself to one question and one follow-up. If you would like to ask a question, please press star one to raise your hand.

Operator 2: We will now begin the question-and-answer session. Please limit yourself to one question and one follow-up. If you would like to ask a question, please press star one to raise your hand. To withdraw your question, press star one 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 stand by while we compile the Q&A roster. Your first question comes from the line of Ryan Deschner with Raymond James. Your line is now open. Please go ahead.

Operator: We will now begin the question-and-answer session. Please limit yourself to one question and one follow-up. If you would like to ask a question, please press star one to raise your hand. To withdraw your question, press star one 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 stand by while we compile the Q&A roster. Your first question comes from the line of Ryan Deschner with Raymond James. Your line is now open. Please go ahead.

Speaker #4: To withdraw your question, press star one again. We ask that you pick up your handset when asking a question to allow for optimum sound quality.

Speaker #4: If you are muted locally, please remember to unmute your device. Please stand by while we compile the Q&A roster. Your first question comes from the line of Ryan Deschner, with Raymond James.

Speaker #4: Your line is now open. Please go ahead.

Speaker #5: Hi, good afternoon. Congrats on the strong results. I'm curious what your overall take is on the potential read-through from the BOLD study evaluating Otavixa batch in patients with biliary atresia?

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. I have a follow-up question.

Speaker #5: And then I have a follow-up question.

Speaker #6: And thanks, Ryan, for the question. Yeah, the BOLD study and the study of note in our pipeline, that includes biliary atresia patients, the Expan study, I mean, to put it simply, are asking very different questions.

Chris 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, to put it simply, are asking very different questions. I would equate the BOLD 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, where we are looking at older patients than what was in BOLD and 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. We feel there is not really a read-through or connection between BOLD and what we are looking at in the upcoming EXPAND readout.

Speaker #6: I would equate the BOLD 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.

Speaker #6: The question we're asking in EXPAN ties much more to where we've seen IBAT perform quite well in other settings, where we're looking at older patients than what was in BOLD and EMBARK, and evaluating pruritus—something that we've seen an impact from IBAT therapy over and over again now in different clinical settings.

Speaker #6: So we feel there's not really a read-through or connection between BOLD and what we're looking at in the upcoming EXPAN readout.

Speaker #5: 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.

Ryan Deschner: Appreciate it. Just on the PSC regulatory discussions, what key topics do you anticipate sort of addressing with these future discussions? Thank you.

Speaker #6: Yeah, I think for the follow-up on that, I mean, just kind of to reiterate some of the background here, we did extensively discuss the VISTA study design with FDA.

Chris Peetz: Thanks for the follow-up on that. Just to 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 the 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. Frankly, the situation now that we have seen is in the meeting, there was a new team from FDA. We think there is a lot of work to get them up to speed on the backdrop here. The history, designing and conducting VISTA and what the study means in a PSC setting. We think it is going to be an iterative approach to get them up to speed, not only with the current data package, but with the history of the program.

Speaker #6: Back in the pre-'90 setting, the FDA acknowledged the pivotal intent, described the design as reasonable, and gave direct feedback on duration, analysis plan—all these things that designed the study that we read out so successfully earlier this year.

Speaker #6: Now, frankly, the situation now that we've seen is in the meeting, there was a new team from FDA, and so we think there's a lot of work to kind of get them up to speed on the backdrop here so that the history designing and conducting VISTAs and what this study means in a PSC setting.

Speaker #6: So we think it's going to be an iterative approach to kind of get them up to speed, not only with the current data package, but with the history of the program.

Speaker #5: Very helpful. Thanks, Chris.

Ryan Deschner: Very helpful. Thanks, Chris.

Speaker #6: Thanks for the questions.

Chris Peetz: Thanks for the questions.

Speaker #4: Your next question comes from the line of Gavin Clark-Gartner, with Evercore ISI. Your line is now open; please go ahead.

Operator 2: Your next question comes from the line of Gavin Clark-Gartner with Evercore ISI. Your line is now open. Please go ahead.

Operator: Your next question comes from the line of Gavin Clark-Gartner with Evercore ISI. Your line is now open. Please go ahead.

Speaker #7: Hey, guys. Thanks for taking the questions. First, what was this new FDA's team's rationale to conduct an additional phase three? Like, was it more of a safety database question, or was it more around the efficacy side?

Gavin Clark-Gartner: Hey, guys. Thanks for taking the questions. First, what was this new FDA's team's rationale to conduct an additional phase III? Was it more of a safety database question or was it more around the efficacy side?

Speaker #6: For the follow-up, Gavin, the comments about a phase three recommendation, from them, really were not specific. So we don't have great clarity on what specifically they're looking for.

Chris Peetz: For the follow-up, Gavin, the comments about a phase III recommendation from them really were not specific. We don't have great clarity on what specifically they're looking for. There were comments across the board on more general on efficacy, which we think VISTA very clearly addresses, and they actually commented on that in the meeting. On the safety side, the discussion, the couple of things that were brought up were DILI safety and the backdrop with IBAT GI effects and liver safety. Those were designed into VISTA as key things to evaluate. We think it's all there in the VISTA study, and this is more about familiarity with study design and some of the questions that were asked. Another thing I'd point out is that the breakthrough designation actually was issued after the meeting.

Speaker #6: There were comments across the board, more general on efficacy, which we think VISTASEAL very clearly addresses, and they actually commented on that in the meeting.

Speaker #6: On the safety side, the discussion the couple of things that were brought up were IBD safety in the backdrop with IBAT GI effects, and liver safety.

Speaker #6: Those were designed into VISTAs as key things to evaluate. So, we think it's all there in the VISTA study, and this is more about familiarity with study design and some of the questions that were asked.

Speaker #6: Another thing I'd point out is that the breakthrough designation actually was issued after the meeting, so I think that gives us a great opportunity to go back in and build up familiarity with this dataset and work us towards an NDA.

Chris Peetz: I think that gives us a great opportunity to go back in and build up familiarity with this data set and work us towards an NDA.

Speaker #7: 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?

Gavin Clark-Gartner: Okay. That makes sense. 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? Kind of on a similar point, what other regulatory precedents would support approval based on the similar type of phase IIb setting? Thank you.

Speaker #7: 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 two V setting?

Speaker #7: Thank you.

Speaker #6: I think for the follow-up, in terms of the team, a lot of times the correspondence is written, and so you don't have perfect clarity on who's behind some of the written correspondence.

Chris Peetz: Thanks for the follow-up. In terms of the team, a lot of times the correspondence is written, and so you don't have perfect clarity on who's behind some of the written correspondence. It's difficult to answer that question, to be honest. In terms of precedent, recently I've looked across all the IBAT settings where you're seeing an Alagille where the first approval was based on four-week randomized withdrawal data, all the way to the more recent LIVMARLI approval in PBC pruritus with a six-month placebo-controlled study that frankly looks a lot like VISTAS. It's a little bit larger because PBC is a more prevalent indication. There's 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 and the prior conversations with the agency.

Speaker #6: So it's difficult to answer that question to be honest. And in terms of precedent, I mean, we really had looked across all the IBAT settings where you've seen analogy where the first approval was based on four-week randomized withdrawal data.

Speaker #6: All the way to the more recent Livmarli approval and PBC pruritus with a six-month placebo-controlled study that, frankly, looks a lot like VISTAs. It's a little bit larger because PBC is a more prevalent indication.

Speaker #6: So there's 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.

Speaker #7: I'm 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?

Gavin Clark-Gartner: I'm 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?

Chris Peetz: The pre-NDA was in person, so when you were asking about prior and other interactions, some of those have been written, so we don't know who is behind some of the other written correspondence. This meeting was in person.

Speaker #6: The pre-NDA was in person. So when you were asking about prior and other interactions, some of those have been written. So we don't know who is behind some of the other written correspondence.

Speaker #6: This meeting was in person.

Speaker #7: Okay. Got it. Thanks so much.

Gavin Clark-Gartner: Okay, got it. Thanks so much.

Speaker #6: Yep. Thanks for the question.

Chris Peetz: Yeah. Thanks for the question.

Speaker #4: Your next question comes from the line of Mike Oates with Morgan Stanley. Your line is now open; please go ahead.

Operator 2: Your next question comes from the line of Mike Oltz with Morgan Stanley. Your line is now open. Please go ahead.

Operator: Your next question comes from the line of Mike Oltz with Morgan Stanley. Your line is now open. Please go ahead.

Speaker #8: Hi. This is Rohit on for Mike. Thanks for taking our questions. Just based on your conversation with the FDA, do you see any read-through to the PVC vantage study?

[Analyst] (Morgan Stanley): Hi, this is Rohit 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'll request a phase III trial for that one? Also, in terms of commercial prep for FOP, can you talk about where you currently stand and what's outstanding? Thanks.

Speaker #8: Is it possible they'll request a Phase 3 trial for that one? And also, in terms of commercial prep for FOP, can you talk about where you currently stand and what's outstanding?

Speaker #8: Thanks.

Speaker #6: Thanks, Rohit. I'll stick to PVC 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.

Chris Peetz: Thanks, Rohit. I'll 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. That actually gave us a great opportunity to have conversations similar to what we expect to go through here with VISTA. In the correspondence after the breakthrough designation on VANTAGE, we actually received pretty clear feedback from FDA on what we should do to consider VANTAGE a pivotal study. We were able to address those. Primarily the comments related to the overall size. That's part of why we've ended up with over 330 patients in VANTAGE and also the analysis plan and some of the specifics on how the pruritus endpoint is analyzed.

Speaker #6: So that actually gave us a great opportunity to have conversations similar to what we expect to go through here with VISTAs. And so in the correspondence after the breakthrough designation on vantage, we actually received a pretty clear feedback from FDA on what we should do to consider vantage a pivotal study.

Speaker #6: And we were able to address those. So primarily, the comments related to the overall size, that's part of why we've ended up with over 330 patients in vantage, and also the analysis plan and some of the specifics on how the pruritus endpoint is analyzed.

Speaker #8: And Rohit, yeah, with regards to the commercial prep for those liver assertive potential approval and launch in Q4, that's going really well. As I mentioned, you get a we're able to drop that in the bags of our rare genetics team and add a couple of territories there.

Peter Radovich: Rohit, yeah, with regards to the commercial prep for the zilurgisertib potential approval and launch in Q4, that's going really well. As I mentioned, we were able to drop that in the bags of our rare genetics team and add a couple of territories there. A lot of typical pre-launch activity and profiling accounts and understanding where the treaters are. These patients are well identified. There's about 300 or so diagnosed and managed in the US in highly specialized centers. Had a good presence at the ENDO meeting earlier this summer as well. Excited about the progress of the NDA review and working towards launch readiness in Q4.

Speaker #8: A lot of typical pre-launch activity and profiling accounts and understanding where the treaters are. These patients are well identified. There's about 300 or so diagnosed and managed in the US and highly specialized centers.

Speaker #8: Had a good presence at the endo meeting earlier this summer as well. So I'm excited about the progress of the NDA review and working towards launch readiness in Q4.

Speaker #8: Thank you.

[Analyst] (Morgan Stanley): Thank you.

Speaker #6: Thanks for the question.

Chris Peetz: Thanks for the question.

Speaker #4: Your next question comes from the line of Brian Scorney with Baird. Your line is now open; please go ahead.

Operator 2: Your next question comes from the line of Brian Skorney with Baird. Your line is now open. Please go ahead.

Operator: Your next question comes from the line of Brian Skorney with Baird. Your line is now open. Please go ahead.

Speaker #9: Hey, good afternoon, team. I'm going to be annoying and also ask a little bit on FDA's issues with VISTAs. Just given that it seems like a pretty straightforward dataset, can you just refresh our memories?

Brian Skorney: Hey, good afternoon, team. I'm going to be annoying and also ask a little bit on FDA's issues with VISTAS, just given that it seems like a pretty straightforward data set. Can you just refresh our memories as the review team here is within the division of hepatology. Is Kathleen Donohue still the office director? Was she involved in the meeting? Is Frank Anania still the division director, and was he in the meeting? I hear they weren't very specific about why they wanted phase III, did they sort of mention a need for replication? It just seems like the P value here is so robust that there's not really another question that could be answered with a phase III other than maybe longer-term follow-up. Yeah, any sort of guidance there is helpful.

Speaker #9: Is the review team here is within the Division of Hepatology. It's Katie Donahue still the office director? Was she involved in the meeting? Is Frank Ananya still the vision director?

Speaker #9: And was he in the meeting? And I mean, I hear that they weren't very specific about why they wanted phase three, but I mean, did they sort of mention a need for replication?

Speaker #9: It just seems like the p-value here is so robust that there's not really another question that could be answered with a phase three, other than maybe longer-term follow-up.

Speaker #9: So, yeah, any sort of guidance there is helpful.

Speaker #6: Yeah, thanks, Brian, for the question. On some of the specifics of people kind of in the room, what I would say is the office leadership, we think, maybe has changed, and leadership was not in the room in our review meeting.

Chris Peetz: Thanks, Brian, for the question. On some of the specifics of people in the room, what I would say, the office leadership, we think maybe has changed, and leadership was not in the room in our review meeting. Getting into specifically on efficacy, like you're pointing out, I think it's very easily addressed by the VISTAS data. Getting Breakthrough afterwards I think is a nod in that direction. Given some of the discussion in the room, I think that's something that we can readily address through iterative conversation with FDA. I don't know if that answers your question.

Speaker #6: And kind of getting into some of the I mean, specifically on efficacy, the I think it's like you're pointing out, I think it's very easily addressed by the VISTAs data.

Speaker #6: Getting breakthrough afterwards, I think, is a nod in that direction. So given some of the discussion in the room, I think that's something that we can readily address through iterative conversation with FDA.

Speaker #6: I don't know if that answers your question.

Speaker #9: Brian, thanks. Yeah, it does. Maybe if I could just ask one thing on the PDUFA with Ziller? Yesterday, I think we would have had probably the late-cycle review meeting in the last month or so. Just any commentary on how that went and your level of confidence going into labeling discussions?

Brian Skorney: Yeah, it does. Maybe if I could just ask one thing on the PDUFA with zilurgisertib. I think you would've had probably the late-cycle review meeting in the last month or so. Just any commentary on how that went and your level of confidence going into labeling discussions?

Speaker #6: Yeah. Thanks for the question, Brian. Yeah, we did have the late cycle meeting. Meeting went very well. So we feel the application is progressing quite well towards the PDUFA date.

Peter Radovich: Yeah. Thanks for the question, Brian. Yeah, we did have the late-cycle meeting. Meeting went very well. We feel the application is progressing quite well towards the PDUFA date. The whole system's go to prepare for potential launch in Q4.

Speaker #6: So, full systems go to prepare for potential launch in Q4.

Speaker #9: Great. Thank you.

Brian Skorney: Great. Thank you.

Speaker #6: Thanks for the questions.

Chris Peetz: Thanks for the questions.

Speaker #4: Your next question comes from the line of Kalpit Patel with Wolf Research. Your line is now open; please go ahead.

Operator 2: Your next question comes from the line of Kalpit Patel with Wolfe Research. Your line is now open. Please go ahead.

Operator: Your next question comes from the line of Kalpit Patel with Wolfe Research. Your line is now open. Please go ahead.

Speaker #2: Yeah. Hey, good afternoon, and thanks for taking the question. One more on the regulatory interaction here. In your communication with the agency, has there been any dialogue so far on potentially running a post-approval confirmatory study instead of running a phase three?

Kalpit Patel: Yeah. Hey, good afternoon, and thanks for taking the question. One more on the regulatory interaction here. In your communication with the agency, has there been any dialogue so far on potentially running a post-approval confirmatory study instead of running a phase III? Should investors assume that a phase III is what's in the works right now?

Speaker #2: Or should investors assume that a phase three is what's in the works right now?

Speaker #6: Thanks for the question. I think one the direct answer is that the conversation with FDA didn't get to that level of specifics for post-approval requirements.

Chris Peetz: Thanks for the question. I think the direct answer is that the conversation with FDA didn't get to that level of specifics for post-approval requirements. I can comment a little bit on the pathway here that using pruritus is a basis for full approval. We don't expect to have a post-approval study in the sense 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 long-term monitoring. We do think that would be appropriate for this setting as well.

Speaker #6: I can comment a little bit on kind of the pathway here that using pruritus is the basis for full approval. So we don't expect to have a post-approval study in the sense of kind of confirmatory accelerated approval-type format.

Speaker #6: What we would expect and has been added for other IBAD approvals is some level of post-approval registry or kind of long-term monitoring. And so we do think that would be appropriate for this setting as well.

Speaker #2: Okay. Thank you.

Kalpit Patel: Okay. Thank you.

Speaker #6: Thanks for the questions.

Chris Peetz: Thanks for the question.

Speaker #4: Your next question comes from the line of James Condolles with Stifel. Your line is now open; please go ahead.

Operator 2: Your next question comes from the line of James Condulis with Stifel. Your line is now open. Please go ahead.

Operator: Your next question comes from the line of James Condulis with Stifel. Your line is now open. Please go ahead.

Speaker #9: Hey, thanks for taking my question. And congrats on a great Liv Marley quarter. Just maybe to be annoying again, one more on PSC. I guess just to clarify, is a phase three on the table, or are you confident that this can be resolved through, like you said, iterations on sort of the data?

James Condulis: Hey, thanks for taking my question. Congrats on a great LIVMARLI quarter. Just maybe to be annoying again, one more on PSC. I guess just to clarify, is a phase III on the table or are you confident that this can be resolved through, like you said, iterations on the data? Just wondering what your base case is and what informs your confidence of guiding to a H1 2027 resubmission. Thanks.

Speaker #9: Just wondering, sort of, what your base case is and what informs your confidence in guiding to a first half '27 resubmission? Thanks.

Speaker #6: Yeah. Thanks for the question, James. And as we looked at this proposal for a phase three, and given the VISTAs results, I think the key question is what would you learn from another study in this setting?

Chris Peetz: Yeah, thanks for the question, James. As we looked at this proposal for a phase III, and given the VISTA results, I think the key question is: What would you learn from another study in this setting? VISTA is the largest ever conducted in PSC pruritus. Clear definitive results. It's a big enough safety database for a setting like PSC. We don't see what would be gained by going down that road, and find that just the weight of evidence here is really convincing. Feel good about where we stand now with breakthrough designation to work through this.

Speaker #6: I mean, VISTAs is the largest ever conducted in PSC pruritus. Clear, definitive results. It's a big enough safety database for a setting like PSC.

Speaker #6: So we don't see what would be gained by going down that road. And fine that just the weight of evidence here is really convincing, so feel good about where we stand now with breakthrough designation.

Speaker #6: To work through this.

Speaker #9: Thanks.

James Condulis: Thanks.

Speaker #6: Thanks for the question.

Chris Peetz: Thanks for the question.

Speaker #4: Your next question comes from the line of Lisa Walter with RBC. Your line is now open; please go ahead.

Operator 2: Your next question comes from the line of Lisa Walter with RBC. Your line is now open. Please go ahead.

Operator: Your next question comes from the line of Lisa Walter with RBC. Your line is now open. Please go ahead.

Speaker #10: Oh, good afternoon. Thanks for taking our questions. Just one more on the NDA filing delay here for Volixabet. Could this mean that we might see more BD in the near term, as potentially PSC revenues may be pushed out?

Lisa Walter: Oh, good afternoon. Thanks for taking our questions. One more on the NDA filing delay here for volixibat. Could this mean that we see more BD in the near term as potentially PSC revenues may now be pushed out? I just want to ask as well, could you add any clarity on whether the FDA is still accepting pruritus as an approvable endpoint? Thanks so much.

Speaker #10: And I just want to ask as well, could you add any clarity on whether the FDA is still accepting a pruritus as an approvable endpoint?

Speaker #10: Thanks so much.

Speaker #6: Great. Thanks for the questions, Lisa. The first thing on the answer to your second part first, which is absolute clarity that pruritus is an approvable endpoint.

Chris Peetz: Great. Thanks for the questions, Lisa. I'll answer your second part first, which is the absolute clarity that pruritus is an approval endpoint. That's what this discussion is all focused on. There's no question there. On the BD front, we've always approached BD on being always active and always opportunistic. Don't see this really relating to the overall level of activity we have or our criteria and what we're looking to bring in. Thanks for the question.

Speaker #6: That's what this discussion is all focused on. So there's no question there. On the BD front, we've always approached BD on being always active and always opportunistic.

Speaker #6: So don't see this really relating to the overall level of activity we have or our criteria and what we're looking to bring in. Thanks for the question.

Speaker #4: Your next question comes from the line of Joe Schwartz with Leerink Partners. Your line is now open; please go ahead.

Operator 2: Your next question comes from the line of Joseph Schwartz with Leerink Partners. Your line is now open. Please go ahead.

Operator: Your next question comes from the line of Joseph Schwartz with Leerink Partners. Your line is now open. Please go ahead.

Speaker #11: Hi. Thanks. Hypothetically, if the FDA doesn't budge, does Vantage PBC have any ability to strengthen the Volixabet regulatory package for PSC, for instance, if a filing in PBC is an NDA and a filing in PSC is an SNDA?

Joseph Schwartz: Hi. Thanks. Hypothetically, if the FDA doesn't budge, does VANTAGE PBC 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 is an sNDA, could that order of operations be successful without having to do another phase III?

Joe Schwartz: Hi. Thanks. Hypothetically, if the FDA doesn't budge, does VANTAGE PBC 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 is an sNDA, could that order of operations be successful without having to do another phase III?

Speaker #11: Could that order of operations be successful without having to do another phase three?

Speaker #6: Andrew, thanks for the question. All right. What I'd say is potentially, I mean, we're getting into some hypotheticals down the road. The base plan is to get the PSCs into NDA submitted first.

Chris Peetz: Hey, Joe. Thanks for the question. What I'd say is potentially, we're getting into some hypotheticals down the road. The base plan is to get the PSC's NDA submitted first. 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'll get to if we end up in that scenario.

Speaker #6: 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.

Speaker #6: And how we approach that in terms of parallel or sequenced NDAs is something that we'll get to if we end up in that scenario.

Speaker #9: Okay. Thanks. And then a question on Berlovita. Did any baseline characteristics for patients enrolled in the interim analysis cohort of Azure One appear to correlate with better response in the phase two B portion of the trial?

Joseph Schwartz: Okay, thanks. A question on brelovitug. Did any baseline characteristics for patients enrolled in the interim analysis cohort of AZUR-1 appear to correlate with better response in the phase II-B portion of the trial? How do the baseline characteristics for patients enrolled in the full AZUR-1 population and AZUR-4 compare?

Joe Schwartz: Okay, thanks. A question on brelovitug. Did any baseline characteristics for patients enrolled in the interim analysis cohort of AZUR-1 appear to correlate with better response in the phase II-B portion of the trial? How do the baseline characteristics for patients enrolled in the full AZUR-1 population and AZUR-4 compare?

Speaker #9: And how do the baseline characteristics for patients enrolled in the full Azure One population and Azure Four compare?

Speaker #6: I mean, the quick answer to that is no, nothing obvious that really comes out. And we see response for berloVita across really all patients.

Chris Peetz: I think the quick answer to that is no, nothing obvious that really comes out, and we see response for brelovitug across really all patients. It's a very broad-based response, and that cuts across baseline viral RNA levels, ALT levels, geography. Every way we've looked at it, you consistently see patients responding to brelovitug.

Speaker #6: So, it's a very broad-based response, and that cuts across baseline viral RNA levels, ALT levels, geography—kind of every way we've looked at it.

Speaker #6: You consistently see patients responding to Berlovita.

Speaker #9: Thank you.

Joseph Schwartz: Thank you.

Joe Schwartz: Thank you.

Speaker #6: Thanks for the question.

Chris Peetz: Thanks for the question.

Speaker #4: Your next question comes from the line of Jessica Phi with JP Morgan. Your line is now open; please go ahead.

Operator 2: Your next question comes from the line of Jessica Fye with J.P. Morgan. Your line is now open. Please go ahead.

Operator: Your next question comes from the line of Jessica Fye with J.P. Morgan. Your line is now open. Please go ahead.

Speaker #12: Hey, guys. Good afternoon. Thanks for taking my question. So more on Volixabet. When you, I think, can prepare remarks referred to some options to supplement the VISTAs trial, curious what you could supplement it with.

Jessica Fye: Hey, guys. Good afternoon. Thanks for taking my question. More on volixibat. When you, I think in prepared remarks, referred to some options to supplement the VISTA trial, curious what you could supplement it with?

Speaker #12: 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?

Jessica Fye: Second, just based on where we stand right now, what gives you the confidence to outline H1 2027 as the new PSC filing timeline? Thank you.

Speaker #12: Thank you.

Speaker #6: Yeah. Thanks for the questions, Jessica. Yeah. In terms of supplementing it, I mean, the real simple one is actually something I mentioned in the prepared remarks as well, is kind of the data cutoff timing.

Chris Peetz: Thanks for the questions, Jessica. In terms of supplementing it, I think the real simple one is actually something I mentioned in the prepared remarks as well, is kind of the data cutoff timing, where we're 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. That was one of the kind of 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 H1 is very achievable, and it's really based on having enough room to have an iteration or two with FDA.

Speaker #6: We're able to pretty easily allow more safety data to accrue to get to 100 patients at the 12-month mark in the open label follow-up.

Speaker #6: So that was one of the kind of 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.

Speaker #6: In terms of the timing, we feel first half is very achievable. And it's really based on having enough room to have an iteration or two with FDA.

Speaker #6: So it's less about time we need to prepare the submission, frankly, because we're things are ready to go quite quickly after we kind of have the input we need.

Chris Peetz: It's less about time we need to prepare the submission, frankly, because things are ready to go quite quickly after we have the input we need. It's more about that iteration with FDA. Really that's kind of an estimate based on experience. We've done 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 and Alagille. It's a type of situation that we've worked through before.

Speaker #6: It's more about that iteration with FDA. And really, that's kind of an estimate based on experience. We've done applications like this before back to the original allogeneal al application where it took a couple of meetings along the way to kind of build up to that NDA filing for with Marley and allogeneal.

Speaker #6: And so it's a type of situation that we've worked through before.

Speaker #4: Your next question comes from the line of Joseph Tomi with TD Cohen. Your line is now open; please go ahead.

Operator 2: Your next question comes from the line of Joseph Thome with TD Cowen. Your line is now open. Please go ahead.

Operator: Your next question comes from the line of Joseph Thome with TD Cowen. Your line is now open. Please go ahead.

Speaker #13: 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.

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, where are you anticipating in terms of relaying kind of your FDA interactions to the street? I think in your prepared remarks, you indicated that this necessarily won't 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? One 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've outlined today? Thank you.

Speaker #13: I guess, where are you in terms of anticipating relaying your FDA interactions to the Street? And then, I think in your prepared remarks, you indicated that this necessarily won't flip over to PBC, and that you have some feedback that Vantage will be a registrational study.

Speaker #13: I guess, to your knowledge, is it this new group that conveyed that information? And then, one point of clarification, just on the updated guidance.

Speaker #13: Is there anything for FOP in your updated product guidance or would that be above and beyond what you've outlined today? Thank you.

Speaker #6: Yeah. Thanks for the questions. I'll take the first couple and pass it over for the FOP question. In terms of providing an update, our thinking is that this is likely an iterative process.

Chris Peetz: Yeah. Thanks for the questions. I'll take the first couple and pass it over for the FOP question. In terms of providing an update, our thinking is that this is likely an iterative process. We would plan to give an update when we have kind of a material update. You don't want to be sharing the blow-by-blow on what might be email correspondence even with FDA. Shifting onto the PBC question, those interactions were written correspondence. Don't know exactly if it's the same exact people behind it, but what gives a lot of comfort there is recency, right? This has happened over the past year that we've had those PBC written correspondence.

Speaker #6: And so we would plan to give an update when we have kind of a material update. So I don't want to be sharing the blow-by-blow on what might be email correspondence even with FDA.

Speaker #6: And shifting onto the PDC question, those interactions were written correspondence. So, we don't know exactly if it's the same exact people behind it, but it gives a lot of comfort there is recency, right?

Speaker #6: So this happened over the past year, that we've had those PDC written correspondence.

Speaker #9: Then 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 2027.

Eric Bjerkholt: On guidance, the $680 to 700 million does not include FOP. Anything we see in Q4 would be above and beyond, although we really expect the revenue to start more in 2027.

Speaker #13: Thank you.

Joseph Thome: Thank you.

Speaker #4: Your next question comes from the line of John Voliban with Citizen. Your line is now open; please go ahead.

Chris Peetz: Next question.

Operator 2: Your next question comes from the line of John Wolleben with Citizen. Your line is now open. Please go ahead.

Operator: Your next question comes from the line of John Wolleben with Citizen. Your line is now open. Please go ahead.

Speaker #14: Hey, Chris. I'm wondering if you could just talk us through what this iterative dialogue looks like in terms of using breakthrough therapy or formal meeting status and scheduling of these just for a little bit more expectations on that flow of information between you and the agency.

Jonathan Wolleben: Hey, Chris. I'm wondering if you could just talk us through what this iterative dialogue looks like in terms of using breakthrough therapy or formal meeting status and scheduling of these, just for a little bit more expectations on that flow of information between you and the agency.

Jon Wolleben: Hey, Chris. I'm wondering if you could just talk us through what this iterative dialogue looks like in terms of using breakthrough therapy or formal meeting status and scheduling of these, just for a little bit more expectations on that flow of information between you and the agency.

Speaker #6: Yeah. Thanks for the follow-up, John. One of the benefits of breakthrough designation is it does allow for more frequent advice from FDA, more frequent interactions.

Chris Peetz: Thanks for the follow-up, John. One of the benefits of breakthrough designation is it does allow for more frequent advice from FDA, more frequent interactions. Quite simply, that just means we'll be able to reach out more informally, and also have more advice meetings. How those sequence out really depends on basically how the dialogue with FDA progresses. We can't really give too much more specific at this point. We will keep you guys updated as we make progress through it.

Speaker #6: And so quite simply, that just means we'll be able to reach out more informally and also have more advice meetings. How those sequence out really depends on basically how the dialogue with FDA progresses.

Speaker #6: So I can't really give too much more specific at this point. But we will keep you guys updated as we make progress through it.

Speaker #4: Your next question comes from the line of Ramakanth Swayampukula with HC Wainwright. Your line is now open. Please go ahead.

Operator 2: Your next question comes from the line of Ramakant Swayampukula with H.C. Wainwright. Your line is now open. Please go ahead.

Operator: Your next question comes from the line of Ramakant Swayampukula with H.C. Wainwright. Your line is now open. Please go ahead.

Speaker #15: Thank you. This is Okay from HC Wainwright. A couple of really quick questions. Based on the interactions that you have had so far, for the PSC, PSC indication, are you planning to make any changes at all in your approach for the PBC indication based once the Vantage data comes out?

Ramakant Swayampukula: Thank you. This is RK from H.C. Wainwright. Couple of really quick questions. Based on the interactions that you have had so far for the PSC indication, are you planning to make any changes at all in your approach for the PBC indication once the VANTAGE data comes out? If you do go ahead and submit in H1 2027, notwithstanding the recommendation, do you run into a risk of refuse to file kind of a situation? Let's say everything goes fine and you still continue to apply, would you expect an AdCom at the end of this?

Ramakanth Swayampakula: Thank you. This is RK from H.C. Wainwright. Couple of really quick questions. Based on the interactions that you have had so far for the PSC indication, are you planning to make any changes at all in your approach for the PBC indication once the VANTAGE data comes out? If you do go ahead and submit in H1 2027, notwithstanding the recommendation, do you run into a risk of refuse to file kind of a situation? Let's say everything goes fine and you still continue to apply, would you expect an AdCom at the end of this?

Speaker #15: And also, if you do go ahead and submit in the first half of '27, notwithstanding the recommendation, do you run into a risk of refuse to file kind of a situation?

Speaker #15: And let's say everything goes fine and you still continue to apply. Would you expect an ADCOM at the end of this?

Speaker #6: Thanks, Okay, 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.

Chris Peetz: Thanks, RK, for the question. In terms of the PBC approach, given the recent interactions, I think we have direct input for that indication for VANTAGE. Feel like that is on a good course. Wouldn't make changes at this point to what we're doing in PBC. We've kind of already made recent adjustments to accommodate what FDA asked for having VANTAGE being confirmed as a pivotal study. On some of these questions about submission risks, I think is how I would describe the question. That's the reason for this iterative interaction with FDA, is to try to work through things that might be an RTF risk and try and get those off the table. Frankly, in AdCom, it might be something that could be helpful given the huge impact that volixibat has shown in a really terrible setting for patients.

Speaker #6: So I feel like that is on a good course. So I wouldn't make changes at this point to what we're doing in PBC. We've kind of already made recent adjustments to accommodate what FDA asked for for having Vantage being confirmed as a pivotal study.

Speaker #6: And then on some of these questions about submission risks, I question. That's the reason for this interactive interaction with FDA is to try to work through things that might be an RTF risk and try and get those off the table.

Speaker #6: And frankly, an AdCom might be something that could be helpful, given the huge impact that politics has had in really terrible settings for patients.

Chris Peetz: The pruritus relief, improvement in sleep and fatigue that patients experience with volixibat treatment is really life-changing. I think that could be one way that this gets highlighted.

Speaker #6: The relief, pruritus relief, improvement in sleep and fatigue that patients experience with Felix about treatment is really life-changing. So I think that could be one way that this gets highlighted.

Speaker #15: Thank you. Thanks for answering my questions.

Ramakant Swayampukula: Thank you. Thanks for answering my questions.

Ramakanth Swayampakula: Thank you. Thanks for answering my questions.

Speaker #6: Thanks for the question.

Chris Peetz: Thanks for the question.

Speaker #4: There are no further questions at this time. I will now turn the call back to Chris Peetz for closing remarks.

Operator 2: There are no further questions at this time. I will now turn the call back to Chris Peetz for closing remarks.

Operator: There are no further questions at this time. I will now turn the call back to Chris Peetz for closing remarks.

Speaker #6: Great. Well, thank you all for joining us today. And I hope you have a great afternoon.

Chris Peetz: Well, thank you all for joining us today, and hope you have a great afternoon.

Speaker #4: This concludes today's call. Thank you for attending. You may now disconnect.

Operator 2: This concludes today's call. Thank you for attending. You may now disconnect. This event has now concluded. Thank you for joining Mirum Pharmaceuticals' Report Q2 2026 Financial Results and Provides Business Update. The line will disconnect automatically.

Operator: This concludes today's call. Thank you for attending. You may now disconnect. This event has now concluded. Thank you for joining Mirum Pharmaceuticals' Report Q2 2026 Financial Results and Provides Business Update. The line will disconnect automatically.

Speaker #1: This event has now concluded. Thank you for joining Mirum Pharmaceuticals' report second quarter 2026 financial results and provides business update. The line will disconnect automatically.

Q2 2026 Mirum Pharmaceuticals Inc Earnings Call

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