Q2 2024 Mirum Pharmaceuticals Inc Earnings Call

? ? ? ? ?

Carla: Good afternoon everyone, and welcome to the Mirum Pharmaceutics report second quarter 2024 financial results and business update. My name is Carla, and I will be coordinating your call today. During the presentation, you will have the opportunity to ask questions by pressing star followed by one on your telephone keypad, and if you change your mind, please press star followed by two. I will now hand you over to Andrew McKibben, Vice President of Investor Relations and Finance, to begin. Andrew, please go ahead.

Unknown Executive: Good afternoon, everyone, and welcome to the Mirum Promissivity Report, the Concordor 2024 financial results, and provides business update.

Karla: Good afternoon everyone and welcome to the Mirum Pharmaceutics report second quarter 2024 financial results and provides business update. My name is Carla and I will be coordinating your call today. During the presentation, you will have the opportunity to ask questions by pressing star followed by one on your telephone keypad.

Carla: My name is Carla, and I will be coordinating in court today.

Carla: During the presentation, you will have the opportunity to ask questions while pressing star followed by one on your telephone keypad. And if you change your mind, press star followed by two.

Andrew Mckibben: I will now hand you over to Andrew McKibben, Vice President of Investor Relations and Finance, to begin.

Andrew Mckibben: And if you change your mind, blink for a star followed by two. I will now hand you over to Andrew McKibben, Vice President of Investor Relations and Finance to begin. Andrew, please go ahead.

Andrew Mckibben: Andrew, please go ahead. Thanks, Carla, and good afternoon, everyone. I'd like to welcome you to Mirum Pharmaceuticals' second quarter of 2024 conference call. I'm joined today by our CEO, Chris Peetz, our President and Chief Operating Officer, Peter Radovich, our Chief Medical Officer, Joanne Quan, and Eric Bjerkholt, our Chief Financial Officer. Earlier today, Mirum issued a news release announcing the company's results to the second quarter of 2024. Copies of this news release and STC filings can be found in the investor's section of our website.

Andrew Mckibben: Thanks, Carla, and good afternoon, everyone. I'd like to welcome you to Mirum Pharmaceuticals' second quarter 2024 conference call. I'm joined today by our CEO, Chris Peetz, our President and Chief Operating Officer, Peter Radovich, our Chief Medical Officer, Joanne Quan, and Eric Bjerkholt, our Chief Financial Officer. Earlier today, Mirum issued a news release announcing the company's results for the second quarter of 2024. Copies of this news release and SEC filings can be found in the Investors section of our website.

Andrew Mckibben: Thanks, Carla, and good afternoon, everyone. I'd like to welcome you to Mirum Pharmaceutical's second quarter 2024 conference call. I'm joined today by our CEO , Chris Peetz, our President and Chief Operating Officer, Peter Radovich, our Chief Medical Officer, Joanne Quan, and Eric Bjerkholt, our Chief Financial Officer.

Speaker Change: Earlier today, Mirum issued a news release announcing the company's results for the second quarter of 2024. Copies of this news release and SEC filings can be found in the Investors section of our website.

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. These statements represent our judgment 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 FAC filings for more information.

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. These statements represent our judgment 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. With that said, I'd like to turn the call over to Chris. Okay?

Speaker Change: 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.

Speaker Change: These statements represent our judgment as of today and inherently involve risks and uncertainties that may cause actual results to differ materially from the results discussed.

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

Andrew Mckibben: With that said, I'd like to turn the caller over to Chris.

Chris Peetz: Chris? Thanks, Andrew, and good afternoon, everyone. I'm excited to share with you the outstanding progress we've made in this quarter with our commercial medicines and pipeline. It's been a strong quarter across the board with continued growth, important regulatory achievements, and positive the looks of that interim results. On the commercial side, adoption of our medicines continues to grow total net product sales of $77.8 million across with Marley, Candidol, and Colbaugh, representing a 139 percent increase from the second quarter of last year. Building on the strength, we've also achieved important regulatory milestones for our commercial medicines. We submitted our NVA for Candidol and CTX, which it approved, will allow us to take additional steps to reach this underdiagnosed population.

Unknown Executive: Thanks, Andrew, and good afternoon to everyone. And finally, we took an important step towards advancing the prospects of that towards potentially pivotal data with the positive interim readouts of the VISTAs PSC and Vantage PBC studies.

Chris Peetz: Thanks, Andrew, and good afternoon to everyone. I'm excited to share with you the outstanding progress we've made this quarter with our commercial medicines and pipelines. It's been a strong quarter across the board with continued growth, important regulatory achievements, and positive ELIXIVET interim results. On the commercial side, adoption of our medicines continues to grow, with total net product sales of $77.8 million for Marley, Ketanol, and Colvon, representing a 139% increase from the second quarter of last year.

Chris Peetz: Building on these strengths, we've also achieved important regulatory milestones for our commercial medicine. We submitted our NDA for Kenodiol and CTX, which, if approved, will allow us to take additional steps to reach this underdiagnosed population and provide an opportunity for urban exclusivity. For Luke Marley, I'm very happy to say that we are now approved for cholestatic paritis and PFIC in the U.S. and for the treatment of PFIC in Europe, and we recently announced a U.S. label update to reduce that age to 12 months and older.

Chris: Thanks Andrew and good afternoon to everyone. I'm excited to share with you the outstanding progress we've made this quarter with our commercial medicines and pipeline. It's been a strong quarter across the board with continued growth, important regulatory achievements, and positive Elixabat interim results.

Chris: On the commercial side, adoption of our medicines continues to grow, with total net product sales of $77.8 million across McMarley, Kienanol, and Colvon, representing a 139% increase from the second quarter of last year.

Chris: Building on the strength, we've also achieved important regulatory milestones for our commercial medicines. We submitted our NDA for kinediol and CTX, which, if approved, will allow us to take additional steps to reach this underdiagnosed population and provide an opportunity for orphan exclusivity.

Chris Peetz: And provide an opportunity for urban exclusivity. For the Marley, I'm very happy to say that we are now approved for colostatic arthritis and PFIC in the US and for the treatment of PFIC in Europe. And we recently announced the US label update to reduce that age to 12 months and older. We're looking forward to bringing the Marley to PFIC patients, given the impressive clinical impact that we've seen in this population. I'm also excited to announce the initiation of another potentially label-enabling study for live Marley and colostatic paritis. I'll let you and speak to some of the details, but in short, colostatic paritis is not limited to eligible syndrome, PFIC, PFC, and PBC.

Chris: For Legmarly, I'm very happy to say that we are now approved for cholestatic paritis and PFIC in the U.S. and for the treatment of PFIC in Europe. And we recently announced a U.S. label update to reduce that age to 12 months and older.

Chris Peetz: We're looking forward to bringing Marley to PFIC patients given the impressive clinical impact that we've seen in this population. I'm also excited to announce the initiation of another potentially label-enabling study for live Marley and cholestatic paritis. I'll let Joanne speak to some of the details, but in short, cholestatic pruritus is not limited to allele syndrome, PFIC, TFC, and PBC. We see multiple additional rare disease settings where patients develop cholestasis and experience significant pruritus.

Chris: We're looking forward to bringing Marley to PFIC patients given the impressive clinical impact that we've seen in this population.

Chris: I'm also excited to announce the initiation of another potentially label-enabling study for live marley and cholestatic pruritus. I'll let Joanne speak to some of the details, but in short, cholestatic pruritus is not limited to allele syndrome, PFIC, TSC, and PBC.

Chris Peetz: We see multiple additional rare disease settings where patients develop colostasis and experience significant pruritus. Supported by high interest from physicians and compelling response case studies, we are launching the Expand study to bring Liz Marley to patient communities that would otherwise be challenging to study individually. Collectively across these settings, we estimate there are at least 500 patients in the US alone that would be eligible for this indication. And finally, we took an important step towards advancing the licks of that towards potentially pivotal data with the positive interim readouts of the VISTA's PSC and Vantage PBC studies.

Joanne: We see multiple additional rare disease settings where patients develop cholestasis and experience significant pruritus.

Chris Peetz: Supported by high interest from physicians and compelling response case studies, we are launching the EXPAND study to bring Liz Marley to patient communities that would otherwise be challenging to study individually. Collectively across these settings, we estimate there are at least 500 patients in the U.S. alone that would be eligible for this indication. And finally, we took an important step towards advancing the look of that towards potentially pivotal data with the positive interim readouts of the VISTAs PSC and Vantage PBC studies.

Joanne: Supported by high interest from physicians and compelling response case studies, we are launching the EXPAND study to bring Liz Marley to patient communities that would otherwise be challenging to study individually.

Joanne: Collectively across these settings, we estimate there are at least 500 patients in the U.S. alone that would be eligible for this indication.

Unknown Executive: Thanks, Thomas, for the question.

Speaker Change: And finally, we took an important step towards advancing the looks of that towards potentially pivotal data with the positive interim readouts of the VISTA's PSC and Vantage PBC studies.

Chris Peetz: Our VISTA's PSC study exceeded the pre-specified efficacy threshold and is continuing enrollment to the full study readout. With no approved therapies and PSC, we are positioned to bring the first medicine to this patient community. The interim results of the Vantage study and PBC were also very encouraging, with a statistically significant improvement in paritis and a patient population that spans first and second-line PBC.

Chris Peetz: Our VISTAs PSC study exceeded the pre-specified efficacy threshold and is continuing enrollment to the full study readout. With no approved therapies and PFC, we are positioned to bring the first medicine to this patient community. The interim results of the Vantage Study and PBC were also very encouraging, with a statistically significant improvement in pruritus in a patient population that spans first and second line PVC. I'm happy to say that enrollment is progressing well for both programs. It was a packed second quarter for Mirum. So to dive into the details, I'll turn the call over to Peter to start with our commercial. Peter.

Speaker Change: Our VISTA's PSC study exceeded the pre-specified efficacy threshold and is continuing enrollment to the full study readout. With no approved therapies in PSC, we are positioned to bring the first medicine to this patient community.

Speaker Change: The interim results of the Vantage Study in PBC were also very encouraging, with a statistically significant improvement in pruritus and a patient population that spans first and second line PBC.

Chris Peetz: I'm happy to say that enrollment is progressing well for both programs. It was a packed second quarter for Mirum, so to dive into the details, we'll turn the call over to Peter to start with our commercial business.

Speaker Change: I'm happy to say that enrollment is progressing well for both programs.

Peter: It was a packed second quarter for Mirum. So to dive into the details we'll turn the call over to Peter to start with our commercial business. Peter?

Unknown Executive: Thanks, Chris. I'm pleased to report another strong quarter across all three commercial products, and we continue to track well towards our full-year revenue guidance of $310 to $320. We're also beginning to see prescriptions for PEPID. Internationally, Marley demand growth was strong. I'm pleased to say that we achieved a favorable outcome in our price negotiations with Germany. We expect this to run its course in the next quarter or two.

Peter Radovich: Thanks, Chris. I'm pleased to report another strong quarter across all three commercial products, and we continue to track well towards our full year revenue guidance of $310 to $320. Starting with Libmarly, total global net product sales grew to $47.2 million this quarter, which represents a 45% increase compared to the same quarter last year. In the U.S., sales were $35.5 million, while international sales were $11.7 million. Growth continues to be driven by new Algae-L syndrome patient additions, comprised of both prevalent patients and newly diagnosed.

Peter Radovich: Thanks, Chris. I'm pleased to report another strong quarter across all three commercial products, and we continue to track well towards our full year revenue guidance of 310 to 320 million. Starting with Lib Marley, total global net product sales grew to 47.2 million this quarter, which represents a 45% increase compared to the same quarter last year. In the U.S., sales were 35.5 million, while international sales were 11.7 million. Growth continues to be driven by new Alligial Syndrome patient additions, comprised of both prevalent patients and newly diagnosed. The dynamic we expect to persist going forward. We're also beginning to see prescriptions for PPIT patients, and our recent label expansion to include patients 12 months in order, provides incremental opportunity given that PPIT is generally diagnosed when children are young.

Peter: Thanks, Chris. I'm pleased to report another strong quarter across all three commercial products, and we continue to track well towards our full year revenue guidance of $310 to $320 million.

Peter: Starting with Libmarly, total global net product sales grew to 47.2 million this quarter, which represents a 45% increase compared to the same quarter last year.

Peter: In the U.S., sales were $35.5 million, while international sales were $11.7 million.

Peter: Growth continues to be driven by new Algeal Syndrome patient additions comprised of both prevalent patients and newly diagnosed, a dynamic we expect to persist going forward.

Peter Radovich: This dynamic we expect to persist going forward. We're also beginning to see prescriptions for PEPID, and our recent label expansion to include patients 12 months and older provides incremental opportunity given that PFIC is generally diagnosed when children are young.

Peter: We're also beginning to see prescriptions for PFIC patients, and our recent label expansion to include patients 12 months and older provides incremental opportunity, given that PFIC is generally diagnosed when children are young.

Peter Radovich: Internationally, Marley demand growth was strong. I'm pleased to say that we achieved a favorable outcome in our price negotiations with Germany. Stemming from this, we saw some price reference impact on international sales in Q2. We expect this to run its course in the next quarter or two. We were also very happy with the European Commission's recent endorsement of Liv Marley for PFIC, three months and older, which highlights the significant benefit of Liv Marley for these patients. Lastly, we saw nice demand growth from Colvom and Keynadol in the second quarter, where we recognized net product sales of 30.5%.

Peter Radovich: Internationally, Lib Marley demand growth was strong, and I'm pleased to say that we achieved a favorable outcome in our price negotiations with Germany. Stemming from this, we saw some price reference impact on international sales in Q2. We expect this to run its course in the next quarter or two.

Speaker Change: Internationally, the Marley demand growth was strong. I'm pleased to say that we achieved a favorable outcome in our price negotiations with Germany.

Peter: Stemming from this, we saw some price reference impact on international sales in Q2. We expect this to run its course in the next quarter or two.

Peter Radovich: We were also very happy with the European Commission's recent endorsement of Lib Marley for PPIT, three months and older, which highlights the significant benefit of Lib Marley for these patients.

Speaker Change: We were also very happy with the European Commission's recent endorsement of Livmarli for PFIT three months and older, which highlights the significant benefit of Livmarli for these patients.

Peter Radovich: Lastly, we saw nice demand growth from Colbom and Keene at all in the second quarter, where we recognized net product sales of 30.5 million. Overall, I'm thrilled with the continued strong commercial performance in the first half of the year, and proud of the mayor and team's continued execution. We are on a solid path to achieve our full year guidance of 310 to 320 million and continued growth.

Peter: Lastly, we saw nice demand growth from Colbaum and Cunadal in the second quarter, where we recognized net product sales of $30.5 million.

Peter Radovich: Overall, I'm thrilled with the continued strong commercial performance in the first half of the year and proud of the Mirum team's continued execution. We are on a solid path to achieve our full year guidance of $310 to $320 million and continued growth. And with that, I'll turn it over to Joanne.

Speaker Change: Overall, I'm thrilled with the continued strong commercial performance in the first half of the year and proud of the Miriam team's continued execution.

Speaker Change: We are on a solid path to achieve our full year guidance of $310 to $320 million and continued growth.

Joanne Quan: And with that, I'll turn it over to Joanne.

Joanne Quan: Dr. Anne. Thanks, Peter. We had an exceptional quarter, highlighted by the impressive internal analyses for the VISTA study in PSC and the Vantage study in PVC. I'll give a quick recap of the results. Starting with the VISTA study in PSC, we set a pre-specified threshold for continuation based on both efficacy and safety. The blinded interim analysis met this threshold in our independent data review committee, recommended proceeding with the 20 milligram BOD dose, and that the study continued without any changes. The blinded analysis confirms a meaningful treatment effect and also allows us to include these patients in the total patient number for the final analysis.

Joanne Quan: Thanks Peter. We had an exceptional quarter, highlighted by the impressive interim analyses of the VISTA study in PSC and the Vantage study in PVC. I'll give a quick recap of the results. Starting with a VISTA study in TSE, we set a pre-specified threshold for continuation based on both efficacy and safety. The blinded interim analysis met this threshold, and our independent data review committee recommended proceeding with the 20 mg BID dose and that the study continue without any changes.

Joanne: And with that, I'll turn it over to Joanne. Joanne? Thanks, Peter. We had an exceptional quarter, highlighted by the impressive interim analyses for the VISTA study in PSC and the Vantage study in PVC. I'll give a quick recap of the results.

Unknown Executive: Starting with a VISTA study in TSE, we set a pre-specified threshold for continuation based on both efficacy and safety. The blinded interim analysis met this threshold, and our independent data review committee recommended proceeding with the 20mg BID dose and that the study continue without any changes. The blind analysis confirms a meaningful treatment effect and also allows us to include these patients in the total patient number for the final analysis. We're happy with how the study is enrolling and anticipate completing enrollment in the second half of 2020. Shifting gears a bit, I would like to talk about the new Phase 3 Expansion.

Joanne: Starting with the VISTA study in PSE, we set a pre-specified threshold for continuation based on both efficacy and safety. The blinded interim analysis met this threshold and our independent data review committee recommended proceeding with a 20 mg BOD dose and that the study continue without any changes.

Joanne Quan: The blind analysis confirms a meaningful treatment effect and also allows us to include these patients in the total patient number for the final analysis. We're happy with how the study is enrolling and anticipate completing enrollment in the second half of 2020. Moving on to the Vantage Study and TBC, we're thrilled with the interim results. Both doses of elixivate showed a substantial and statistically significant reduction in itch, an approximately 2.3 point improvement over placebo.

Speaker Change: The blind analysis confirms a meaningful treatment effect and also allows us to include these patients in the total patient number for the final analysis.

Joanne Quan: of Health Sciences. We're happy with how the study is enrolling and anticipate completing enrollment in the second half of 2025.

Speaker Change: We are happy with how the study is enrolling and anticipate completing enrollment in the second half of 2025.

Joanne Quan: Moving on to the Vantage Study in TBC, we're thrilled with the interim results. Both doses of elixivate showed us substantial and statistically significant reduction in inch and approximately 2.3 point improvement over placebo. Based on this, the Vantage Study will also continue with the 20 milligram viaductose, consistent with the business study. The results support elixivates potential and an important advance for patients suffering from the postatic prevents. We also observe reductions in serum bile acids and improvements across multiple dimensions on the PBC 40, most notably fatigue. We look to complete enrollment in 2026 for this larger study.

Speaker Change: Moving on to the Vantage study in PBC, we're thrilled with the interim results. Both doses of Elixabat showed a substantial and statistically significant reduction in itch and approximately 2.3 point improvement over placebo.

Joanne Quan: Based on this, the Vantage study will also continue with the 20 milligram VI-D dose consistent with the risk. The results support Elixabet's potential as an important advance for patients suffering from close contact. We also observed reductions in serum bile acids and improvements across multiple dimensions on the PBC40, most notably fatigue. We look to complete enrollment in 2026 for this larger study. Overall, these results are significant for PBC patients, suggesting that melixivab has the potential to set a new standard in addressing the burden of post-traumatic stress disorder. We've already ramped up enrollment efforts and are targeting up to 100 total. Shifting gears a bit, I would like to talk about the new Phase 3 Expansion.

Speaker Change: Based on this, the VANTAGE study will also continue with the 20 milligram V.I.D. dose consistent with the VISTA study.

Joanne: The results support Elixabet's potential as an important advance for patients suffering from cholestatic pruritus.

Joanne: We also observed reductions in serum bilasins and improvements across multiple dimensions on the PBC40, most notably fatigue.

Joanne: We look to complete enrollment in 2026 for this larger study.

Joanne Quan: Overall, these results are significant for PBC patients, suggesting that the elixivate has the potential to set a new standard in addressing the burden of postasis. We've already ramped up enrollment efforts and are targeting up to 100 total sites.

Joanne: Overall, these results are significant for PBC patients, suggesting that melixivab has the potential to set a new standard in addressing the burden of cholestasis. We've already ramped up enrollment efforts and are targeting up to 100 total sites.

Joanne Quan: Shifting gears a bit, I would like to talk about the new Phase 3 Expand study. Mirum has received a number of requests for compassionate use for the Marley in patients with colostatic paritis across a variety of ultra rare indication patients. We believe these conditions share a common patchy neck and colostasis leading to elevated serum bile acids, which results in persistent paritis. Based on the good responses we've seen in some individuals receiving compassionate use, we're optimistic that that live Marley can play a significant role in the treatment of paritis for these patients. Expand is a randomized double-blind placebo-controlled study evaluating the Marley for true unit paritis for 20 weeks.

Joanne: Shifting gears a bit, I would like to talk about the new Phase 3 EXPAND study.

Joanne Quan: Mirum has received a number of requests for compassionate use with Marley in patients with cholestatic varitis across a variety of ultra-rare indications. We believe these conditions share a common pathogenic mechanism, cholestasis, leading to elevated serum bile acids, which results in persistent parietal. Based on the good responses we've seen in some individuals receiving compassionate use, we are optimistic that Livmarly can play a significant role in the treatment of paritis for these patients.

Speaker Change: Mirum has received a number of requests for compassionate use with Marley in patients with cholestatic varitis across a variety of ultra-rare indications.

Speaker Change: We believe these conditions share a common pathogenic mechanism, cholestasis, leading to elevated serum bile acids, which results in persistent pariahs.

Joanne: Based on the good responses we've seen in some individuals receiving compassionate use, we are optimistic that Litmarly can play a significant role in the treatment of paritis for these patients.

Joanne Quan: EXPAND is a randomized, double-blind, placebo-controlled study evaluating the MARL-E for treatment of pruritus for 20 years. Phan's study will enroll patients with cholestatic paritis associated with a range of conditions such as biliary atresia, secondary sclerosine cholangitis, and other less common conditions. Our target is to enroll approximately 45 patients, and we expect to complete enrollment in 2020. I look forward to providing further updates on VISTAs, Vantage, and EXPAND in the coming quarters. I'll now turn it over to Eric to discuss our finances. Eric?

Joanne: EXPAND is a randomized, double-blind, placebo-controlled study evaluating the MARLE for treatment of pruritus over 20 weeks.

Joanne Quan: Fand study will enroll patients with colostatic paritis associated with a range of conditions, such as barrier atreja, secondary sclerosine colonitis, and other less common conditions. Our target is to enroll approximately 45 patients, and we expect a complete enrollment in 2026. I look forward to providing further updates on visits, vantage, and expand in the coming quarters.

Speaker Change: Pham's study will enroll patients with cholestatic ferritis associated with a range of conditions such as biliary atresia, secondary sclerosing cholangitis, and other less common conditions.

Joanne: Our target is to enroll approximately 45 patients, and we expect to complete enrollment in 2026.

Eric: I look forward to providing further updates on VISTAs, Vantage, and EXPAND in the coming quarters. I'll now turn it over to Eric to discuss our financial results. Eric?

Eric Bjerkholt: I'll now turn it over to Eric to discuss our financial results.

Eric Bjerkholt: Eric? Thanks, Joanne. Earlier today, we issued a press release that included financial results for the second quarter, which I'll briefly summarize. Mets product revenue in the second quarter, 2024, was 77.8 million compared to net product revenues of 32.5 million in the second quarter last year. Total operating expense for the quarter and the June 30, were 102 million, which includes ID expense of 32.7 million, SGNA expense of 49.2 million, and cost of sale of 20.2 million. A total operating expense for the quarter included approximately 17.7 million, a non-cash charge of which 5.7 million was included in cost of sale.

Eric Bjerkholt: Thanks, Joanne. Earlier today, we issued a press release that included financial results for the second quarter, which I'll briefly summarize. Net product revenue in the second quarter 2024 was $77.8 million compared to net product revenues of $32.5 million in the second quarter last year. Total operating expense for the quarter ended June 30 was $102 million, which included R&D expense of $32.7 million, SG&A expense of $49.2 million, and cost of sales of $20.2 million.

Eric: Thanks Joanne. Earlier today we issued a press release that included financial results for the second quarter which I'll briefly summarize.

Eric: Net product revenue in the second quarter of 2024 was $77.8 million compared to net product revenue of $32.5 million in the second quarter last year.

Unknown Executive: Total operating expenses for the quarter ended June 30 were $102 million, which included R&D expense of $32.7 million, SG&A expense of $49.2 million, and cost of sale of $20.2 million. Cash used in the second quarter included the payment of a $10 million milestone to Takeda upon FDA approval of the Lidmarli-PFICC indication. With our robust commercial performance and continued financial discipline, we are in an excellent position to support the development of our pipeline and growth of our commercial business.

Eric: Total operating expense for the quarter ended June 30 were $102 million, which includes R&D expense of $32.7 million, SG&A expense of $49.2 million, and cost of sale of $20.2 million.

Eric Bjerkholt: The total operating expense for the quarter included approximately $17.7 million of non-cash charges, of which $5.7 million was included in cost of sale. For the quarter ended June 30, 2024, the net loss was $24.6 million, or $0.52 per share. Our cash, cash equivalents, and investment was $295.4 million as of June 30, 2024, a reduction of $7.4 million from the end of the prior quarter. Cash used in the second quarter included the payment of a $10 million milestone to Takeda upon FDA approval of the Lidmarli PFIC indication.

Eric: A total operating expense for the quarter included approximately $17.7 million of non-cash charges, of which $5.7 million was included in cost of sale.

Eric Bjerkholt: For the quarter and the June 30, 24, net loss was 24.6 million, or 52 cents per share. A cash, cash equivalent, and investment was 295.4 million as of June 30, 24, a reduction of 7.4 million from the end of the private quarter. Cash used in the second quarter included the payment of a 10 million milestone to Titillate upon FDA approval of the with mildly p-fick indication. With our robust commercial performance and continued financial discipline, we are in an excellent position to support the development of our pipeline and growth of our commercial business.

Eric: For the quarter ended June 30, 2024, net loss was $24.6 million or $0.52 per share.

Eric: Our cash, cash equivalents, and investment was $295.4 million as of June 30, 2024, a reduction of $7.4 million from the end of the prior quarter.

Speaker Change: Cash used in the second quarter included the payment of a $10 million milestone to Takeda upon FDA approval of the Lee Marley PFIC indication.

Eric Bjerkholt: With our robust commercial performance and continued financial discipline, we are in an excellent position to support the development of our pipeline and growth of our commercial business. Now, I'll turn the call back over to Chris for his final comment. Thanks.

Speaker Change: With our robust commercial performance and continued financial discipline, we are in an excellent position to support the development of our pipeline and growth of our commercial business.

Chris Peetz: Now, I'll turn a call back over to Chris for final comment. Thanks, Eric. It's been a great first half of the year.

Eric: Now I'll turn the call back over to Chris for a final comment.

Chris Peetz: Thanks, Eric. It's been a great first half of the year. I'm proud of the Mirum team and our strong execution. We are well positioned to continue to advance our four strategic priorities, to grow our commercial business, to expand the indications of our approved medicines, Advanced, Elixabeth, and Adult Cold Stasis, and to continue to look for opportunities to grow the pipeline. With that, Operator, please open the call for questions

Chris: Thanks, Eric.

Chris Peetz: I'm proud of the Mirum team and our strong execution. We are well positioned to continue to advance our four strategic priorities. To grow our commercial business, expand the indications of our approved medicines, advance the licks about in adult color stasis, and continue to look for opportunities to grow the pipeline.

Chris: It's been a great first half of the year.

Chris: I'm proud of the Mirum team and our strong execution. We are well positioned to continue to advance our four strategic priorities.

Unknown Executive: to grow our commercial business and continue to look for opportunities to grow the pipeline.

Speaker Change: to grow our commercial business, expand the indications of our improved medicines, advance the Lixabet and adult cholestasis, and continue to look for opportunities to grow the pipeline. With that, operator, please open the call for questions.

Unknown Executive: With that, operator, please open the call for questions. We will now begin the question and answer session. If you'd like to ask a question, please press power followed by one on your telephone K-POP. If you change your mind, please press power followed by two. When preparing to ask your question, please ensure your devices are muted locally.

Operator: We will now begin the question and answer session. If you would like to ask a question, please press star followed by 1 on your telephone keypad. If you change your mind, please press star followed by 2. When preparing to ask your question, please ensure your device is unmuted locally. And our first question comes from Daegon Ha from STIFO.

Chris: we will now begin to question and answercession

Speaker Change: If you would like to ask a question, please press star followed by 1 on your telephone keypad. If you change your mind, please press star followed by 2. When preparing to ask your question, please ensure your device is unmuted locally.

Agam: And our first question comes from They Gone Half from TFO. Good afternoon, guys. Thanks for taking our questions, and congrats on the progress. Two from us, one commercial and one clinical, I guess, starting with commercial. As we look at the approval label across Alligial and PFIC in the U.S., it's kind of a palindrome between Liv Marley and Bill Vey.

Chris: And our first question comes from Dagon Ha from TIFO.

Dae Ha: Hey, good afternoon, guys. Thanks for taking our questions and congrats on the progress. Two from us, one commercial and one clinical, I guess. Starting with commercial, as we look at the approval labels across AllerGeo and PFIC in the US, it's kind of a palindrome between Live Marley and Bilvay. So I was hoping you could maybe comment a little bit on what you've learned from perhaps Bilvay's experience in AllerGeo that you can implement to broaden Live Marley's reach within the PFIC segment before further label expansion can come down the pike. And then, on the clinical side, Joanne, enrollment progressing favorably sounds great. Is there any initiative for you to perhaps accelerate enrollment into VISTAs and Vantage Trials? Thanks so much.

Unknown Executive: Hey, good afternoon, guys. Thanks for taking our questions and congrats on the progress. Two from us, one commercial and one clinical, I guess. Starting with commercial, as we look at the approval labels across AllerGeo and PFIC in the US, it's kind of a palindrome between Live Marley and Bilvay. So I was hoping you could maybe comment a little bit on what you've learned from perhaps Bilvay's experience in AllerGeo that you can implement to broaden Live Marley's reach within the PFIC segment before further label expansion can come down the pike. And then, on the clinical side, Joanne, enrollment progressing favorably sounds great. Is there any initiative for you to perhaps accelerate enrollment into VISTAs and Vantage Trials? Thanks so much. Anything to take on further questions?

Dagon Ha: Hey, good afternoon, guys. Thanks for taking our questions and congrats on the progress. Two from us, one commercial, one clinical, I guess.

Dagon Ha: Starting with commercial, as we look at the approval label across Alageel and PFIC in the U.S., it's kind of a palindrome between Liv Marley and Bilvay. So I was hoping if you could maybe comment a little bit on what you've learned from perhaps Bilvay's experience in Alageel that you can implement to

Agam: I was hoping, if you can maybe comment a little bit on what you've learned from, perhaps, Bill Vey's experience in Alligial that you can implement to broad and live Marley's reach within the PFIC segment before further label expansion can come down the pike. And then, in terms of clinical side, Joanne, enrollment progressing favorably sounds great. Is there any initiative for you to perhaps accelerate enrollment into VISTA's Advantage trial? Thanks so much.

Chris: Broaden Liv Marley's reach within the PFIC segment before further label expansion can come down the pike.

Speaker Change: And then in terms of clinical side, Joanne, enrollment progressing favorably sounds great. Is there any initiative for you to perhaps accelerate enrollment into VISTAs and Vantage Trials? Thanks so much.

Chris Peetz: Thank you, Agam, for the question.

Chris Peetz: for the question. On the first kind of a comment on the labeling and label expansion we've seen for LibMarly, and we can circle back for any follow-up questions, but what I think you're asking is just how the sequencing has gone for Marley. And it's played out really well. I think we're in a position of very strong leadership in Europe, where we're the only product approved for both indications. In the U.S., we're now on a very equal footing, where we're now approved for both indications.

Chris Peetz: On the first kind of a comment on the labeling and label expansion we've seen for Liv Marley, and can circle back for the follow-up question. But what I think you're asking is just how the sequencing has gone for Liv Marley. It's played out really well. I agree in a position of a very strong leadership in Europe where we're the only products approved for both indications. In the U.S., we're now, we see about very equal footing where we're now approved for both indications. Initial reception has been quite strong, and the data for Liv Marley and both indications, we think, tells a really compelling story for prescribers, and that's what we're seeing play out in the real world.

Speaker Change: take on for the question you on the first pend of a comments on to the labeling and lal expansion we've seen for ly

Speaker Change: And can circle back for any follow-up questions, but what I think you're asking is just how the sequencing has gone for the Marley and it's it's played out

Speaker Change: really well. I think we're in a position of very strong leadership in Europe where

Speaker Change: We're the only products approved for both indications. In the U.S., we're now, we see a very equal footing where we're now approved for both indications. Initial reception has been quite strong, and the data for Lipmarly in both indications

Chris Peetz: The initial reception has been quite strong, and the data for lip Marley in both indications, we think tells a really compelling story for prescribers, and that's what we're seeing play out in the real world. So in terms of that kind of label sequencing, I think it's largely played out and in the favor of lip Marley. And I'll let Joanne maybe comment on the enrollment strategies. Yeah, thank you.

Chris: We think tells a really compelling story for prescribers, and that's what we're seeing play out in the real world. So, in terms of that kind of label sequencing, I think it's largely played out and to the favor of Marlene.

Joanne Quan: So, in terms of that label sequencing, I think it's largely played out and to the favor of Liv Marley.

Joanne Quan: And I'll let Joanne know the comments on the enrollment strategies. Yeah, thanks for the question. Like I said, we're happy with how the enrollment is going. As you've noticed, we've been very excited by the interim analysis results and the PBC and the PSC, and we should share that with our investigators, and they share the excitement on that and really are seeing the potential impact that this has as a medicine for both patient populations. We're continuing to work with our existing sites and continuing the expansion as we previously talked about. So we're happy with where our enrollment is going.

Speaker Change: And I'll let Joanne make the comments on the enrollment strategies.

Joanne Quan: Yeah, thanks for the question. You know, like I said, we're happy with how the enrollment is going. You know, as you've noticed, we've been very excited by the interim analysis results for the PBC and the PSC, and we share that with our investigators, and they share the excitement about that and really are seeing the potential impact that this has as a medicine for both patient populations. You know, we're continuing to work with our existing sites and continuing the expansion as we previously talked about. So, you know, we're happy with where our enrollment is going.

Joanne: Yeah, thanks for the question. You know, like I said, we're happy with how the enrollment is going. You know, as you've noticed, we've been very excited by the interim analysis results on the PBC and the PSC, and we share that with our investigators, and they share the excitement on that and really are seeing the potential impact that this has as a medicine for both patient populations.

Unknown Executive: Great, thank you very much.

Speaker Change: We're continuing to work with our existing sites and continuing the expansion as we previously talked about. So we're happy with where our enrollment is going.

Agam: Great, thank you very much.

Speaker Change: great thank you very much

Ani Foroohar: The next question comes from Ani Foroohar from Lurink Farmers. Thanks for taking the question. A couple of quick ones.

Mani Foroohar: The next question comes from Mani Foroohar from Lyrinc Partners.

Speaker Change: Thanks for the questions.

Speaker Change: The next question comes from Mani Foroohar from Lyrinc Partners.

Mani Foroohar: Hey, guys, thanks for taking the questions. A couple of quick ones. First, when we think about the novel expansion population in cholestatic pruritus, can you give us a sense of how you think about the duration of that study and the time horizon within which we might see results, recognizing it's a little bit of a heterogeneous population?

Ani Ferroja: Hey guys, thanks for taking the question. A couple of quick ones. First, when we think about the novel expansion population in cholestatic pruritus, can you give us a sense of...

Ani Foroohar: First, I want to think about the novel expansion population called Static Curitis. Can you give us a sense of how you think about the duration of that study and the time horizon with whom I see results, recognizing it's a little bit of a heterogeneous population, and then we have a commercial quick follow-up.

Ani Ferroja: How you think about the duration of that study and the time horizon, which we might see results, recognizing it's a little bit of a heterogeneous population, and then we have a commercial quick follow-up.

Joanne Quan: And then we have a quick commercial follow-up. Sure, thanks for the questions. Maybe Joanne and Peter can take the follow-up. Okay. So...

Joanne Quan: Sure, thanks for the questions. Maybe Joanne and Peter take follow-up? Sure.

Unknown Executive: Sure, thanks for the questions. Maybe Joanne and Peter can take the follow-up. OK. So...

Speaker Change: Sure, thanks for the questions. Maybe Joanne and I'll let Peter take the follow-up. Sure. So, you know, we are just launching the city now, and as we said, we plan to complete enrollment in 2026.

Joanne Quan: Sure. So, you know, we are just launching the study now. And as we said, we plan to complete enrollment in 2026. And it is a bit of a heterogeneous group of patients. But we expect that, you know, some patients will have biliary atresia as a cause, some secondary sclerosis, cholangitis, and then a variety of other causes. This is really based on our experience and compassion. So, you know, we're actually pretty confident in terms of our understanding of the role of IVAD inhibitors in the treatment of polycytic paritis and think that this extends its potential use to a wide variety of patients who, you know, each of these would be difficult to study. So we're excited to study them all in this expanded study and get some results and hopefully support some wider use.

Joanne Quan: So, you know, we are just launching the city now, and as we said, we plan to complete enrollment in 2026. It is a bit of a heterogeneous group of patients, but we expect that, you know, some patients will have biliratrizia as a cause, some secondary school or single angitis, and then a variety of other causes. And this is really based on our experience and compassion to use. So, you know, we're actually pretty confident in terms of our understanding the role of IVAT inhibitors and treatment and polysetic paritis, and think that this extended this potential used to a wide variety of patients who, you know, each of these would be difficult to study.

Speaker Change: It is a bit of a heterogeneous group of patients, but we expect that, you know, some patients will have early atresia as a cause, some secondary sclerosing, cholangitis, and then a variety of other causes. And this is really based on our experience in compassionate youths.

Speaker Change: So, you know, we're actually pretty confident in terms of our understanding the role of IVAD inhibitors in treatment of polycystic arthritis and think that this extends its...

Speaker Change: This potentially used to a wide variety of patients who, you know, each of these, it would be difficult to study. So we're excited to study them all in this EXPAND study and get some results and hopefully support some wider use.

Peter Radovich: So, we're excited to study them all in this expand study and get some results in the hopefully support some wider use. That's helpful.

Unknown Executive: That's helpful. And then when you think about sort of a more commercial side, obviously, there's

Unknown Executive: That's helpful. And then

Peter Radovich: And then, when you think about sort of a more on the commercial side, obviously, there's a little bit of a, there's some element of seasonality in this market, and sort of depends on swings one way early in the year, swing intensive swing the other, certainly around 3K, or as we saw a couple of years ago. How do we think about the tempo of new patient ads and any sort of operational details we should think about in terms of seasonality across the next few quarters looking forward? Yeah, so thanks for the question. I mean, one thing to just kind of remind on reflecting back to Q1, you know, we did see in the U.S.

Peter Radovich: And when you think about sort of more on the commercial side, obviously, there's a little bit of a... There's some element of seasonality in this market, and sort of the pendulum swings one way early in the year, tends to swing the other, certainly around 3Q, as we saw a couple years ago. How should we think about the tempo of new patient ads and any sort of operational details we should think about in terms of seasonality across the next few quarters looking ahead?

Speaker Change: That's helpful.

Speaker Change: And then when you think about sort of...

Speaker Change: I'm more on the commercial side.

Speaker Change: Obviously, there's some element of seasonality in this market, and the pendulum swings one way early in the year, tends to swing the other, certainly around 3Q as we saw a couple years ago. How do we think about the tempo?

Speaker Change: of new patient ads and any sort of operational details we should think about in terms of seasonality across the next few quarters looking forward.

Unknown Executive: Yeah, thanks for the question. One, you know, one thing to just kind of remind you of, reflecting back on Q1, we did see in the U.S. with Liv Marley and the bioacid products impact on our Q1 numbers from the changed healthcare cyber attacks. That's one thing to keep in mind as you think about quarter to quarter trends here. And, you know, as far as seasonality goes across three products, I can't say that we've identified, you know, you know, any real seasonality in these products.

Peter Radovich: Yeah, thanks for the question. One, you know, one thing to just kind of remind you of, reflecting back on Q1, we did see in the U.S. with Liv Marley and the bioacid products impact on our Q1 numbers from the changed healthcare cyber attacks. That's one thing to keep in mind as you think about quarter to quarter trends here. And, you know, as far as seasonality goes across three products, I can't say that we've identified, you know, you know, any real seasonality in these products.

Speaker Change: Yeah, thanks for the question. I mean, I want, you know, one thing to just kind of remind on, reflecting back to Q1, you know, we did see in the U.S. with Liv Marley and the bio-acid products impact on our Q1 number from the

Peter Radovich: with Liv Marley and the Biolassant products' impact on our Q1 number from the Change Health Care cyber attacks. That's one thing to keep in mind if you think about quarter-to-quarter trends here. And, you know, as far as seasonality goes across green products, I can't say that we've identified, you know, any real seasonality in these products. I mean, they're, you know, kind of ultra rare to rare products with relatively low underlying volume, which can lead to kind of quarter-to-quarter variability, quite frankly. But whether that occurs in one quarter versus another, I can't say that there's a really strong effect there.

Speaker Change: chang all care ceverber attacks that' one thing to keep in mind as you think about quarter-quarter trends here and as part asthe seasonality goes cost threeam products and i can't say that we've identified

Speaker Change: you know any real seasonality and these products mean they're you know kind of ultr rare to rare products with relatively low underlying volume which can't lead to kind of quarter a orter variability quite frankly but whether that occurs and

Unknown Executive: I mean, they're, you know, kind of ultra-rare to rare products with, you know, relatively low underlying volume, which can lead to kind of quarter to quarter variability, quite frankly. But whether that occurs in one quarter versus another, I can't say that there's a really strong effect.

Peter Radovich: I mean, they're, you know, kind of ultra-rare to rare products with, you know, relatively low underlying volume, which can lead to kind of quarter to quarter variability, quite frankly. But whether that occurs in one quarter versus another, I can't say that there's a really strong effect there.

Speaker Change: One quarter versus another, I can't say that there's a really strong effect there.

Peter Radovich: Okay, and so we, and on what time horizon should we expect sort of the sort of U.S. typing reference to dynamics to play out? That's probably what we should think about, sort of recurring and sort of eroding itself, playing out over the course of a couple quarters. That's primarily a 2-2-3-2 event. Like, how do we think about breaking that into sort of how we model? Yeah, exactly. We saw the effect in Q2; we expected in Q3, and you know, our expectation is by the time you're in Q4, that that effect is gone, and all of the kind of demand volume growth that we're seeing flows through to the top line.

Unknown Executive: Okay, and should we end on one?

Peter Radovich: Time Horizon. So we expect sort of the sort of OUS pricing reference to come back.

Speaker Change: Okay, and should we, and on what time horizon should we expect sort of the sort of OUS pricing reference dynamics to play out? That's something we should think about sort of recurring and sort of eroding itself playing out over the course of the next couple quarters. Is that primarily 2Q, 3Q events? How do we think about?

Peter Radovich: Reference dynamics to play out. That's something we should think about recurring and sort of eroding itself playing out over the course of a couple quarters. That primarily is 2Q3Q events, like how do we think about baking that into sort of how we model? Yeah, exactly.

Peter Radovich: Yeah, exactly. We saw the effect in Q2. We expect it in Q3. And, you know, our expectation is that by the time you're into Q4, that effect is gone, and all of the kind of demand volume growth that we're seeing flows through to the top line.

Speaker Change: baking that into sort of how we model.

Speaker Change: Yeah, exactly. We saw the effect in Q2, we expect it in Q3, and, you know, our expectation is by the time you're into Q4 that that effect is gone, and all of the kind of demand volume growth that we're seeing flows through to the top line.

Unknown Executive: Perfect. Thanks, guys. I know you've got a bunch of people in the queue. I'll hop off.

Ani Foroohar: Perfect, thanks, guys. I know you got a bunch of people with you. I'll hop off.

Speaker Change: Perfect. Thanks, guys. I know you got a bunch of people in the queue. I'll hop off.

Gavin Clark: Thanks for the questions. Our next question comes from Gavin, Clark Gottener from Evercourt, ISA.

Speaker Change: Thanks for the questions.

Gavin Clark: Our next question comes from Gavin Clark Gartner from Evercore ISA.

Speaker Change: Our next question comes from Gavin Clark-Gottner from Evercore ISA.

Ed Arce: Hi guys, this is Ed Arce. I'm for Gavin. Thanks for taking our question. And would have two, the first one, could you just touch on the level confidence in that 45 patient sample size for expand, and maybe how you powered the study and then one follow up after that. Yeah, yeah, thanks for the question. I would think I'll let Joanne kind of comment a bit, but I think the one thing to kind of say to give some context here is that now, at this point, I bet in these full static settings, I think we've seen a pattern here that you can draw a really dramatic response if you're at the right dose on biolasses of charitis.

Yashan: Hi guys, this is Yashan for Gavin. Thanks for taking our questions. And we just have two.

Yashan: Hi guys, this is Yashan for Gavin. Thanks for taking our question. We just have two. The first one, could you just touch on the level of confidence in that 45 patient sample size for Xpand and maybe how you powered the study and then one follow-up after that?

Yashan: The first one, could you just touch on the level of confidence in that 45 patient sample size for EXPAND and maybe how you powered the study and then one follow-up after that?

Unknown Executive: Thanks for the question. I think I'll, I'll let Joanne kind of comment a bit. But I think the one thing to kind of say to get some context here.

Chris Peetz: Thanks for the question. I think I'll, I'll let Joanne kind of comment a bit. But I think the one thing to kind of say to get some context here is that now, at this point, I've asked you in these full static settings.

Speaker Change: Yesha, thanks for the question. I think I'll let Joanne kind of comment a bit, but I think the one thing to kind of say to get some context here

Speaker Change: is that now, at this point, using iBATs in these full static settings...

Chris Peetz: I think we've seen a pattern here that you can drive a really dramatic response if you're at the right dose for bile acids of cirrhitis. So the thinking behind this study design is based on having actually seen that same profile play out in a number of compassionate use patients. So really strong evidence from the individual case studies that, in aggregate, basically make up this population. So I feel good about launching the study. That's why we designed it, and that's what compelled us to put it together. Maybe Joanne can speak a little bit about sample size. Yeah, thanks for the question.

Speaker Change: I think we've seen a pattern here that you can drive really dramatic response if you're at the right dose on bilasses of cirrhitis.

Joanne Quan: So the thinking behind the study design is based on having actually seen that same profile play out in the number of compassionate use patients. So it's really strong evidence from the individual case studies that, in aggregate, basically make this population. So feel good about launching the study; that's why we designed it, and that's what helped us to put it together. Are you drawing a little bit of sample size?

Joanne: So the thinking behind the study design is based on having actually seen that same profile play out in a number of compassionate use patients.

Speaker Change: So really strong evidence from the individual case studies that, in aggregate, basically make this population. So I feel good about launching the study. That's why we designed it. That's what compelled us to put it together. Maybe Joanne can speak a little bit about sample size.

Joanne Quan: Yeah, thanks for the question. So the sample size was based on powering based on our primary endpoint, which is an observatory digital. So this is a scale that we understand well based on our previous experience, prior studies of modeling. So we feel pretty confident that this is an appropriate sample size for us to see a solid treatment effect. Awesome, thank you.

Joanne Quan: Yeah, thanks for the question. So, you know, the sample size was based on powering based on the primary endpoint, which is an observer-rated issue. So this is a scale that we understand well, based on our previous experience in prior studies with Marley. So we feel pretty confident that this is an appropriate sample size for us to see a solid treatment effect.

Speaker Change: Yeah, thanks for the question. So, you know, the sample size was, you know, based on powering based on our primary endpoint, which is an observer rated ISRO.

Speaker Change: So this is a scale that we understand well, based on our previous experience, you know, prior studies with Marley. So we feel pretty confident that this is an appropriate sample size for us to see a solid treatment effect.

Joanne Quan: And then the follow-up question was, was there any data from EMBARQ that makes you confident enrolling VA patients in this trial?

Unknown Executive: And then one follow-up question was, was there any data from EMBARQ that makes you confident enrolling VA patients in this?

Joanne Quan: One follow was there any data from Embark that makes you confident in rolling being patient in this trial? Yeah, well, thanks for the question.

Speaker Change: Awesome, thank you. And then one follow-up was, was there any data from MBARC that makes you confident in enrolling VA patients in this trial?

Unknown Executive: I think it's important to note that the patient population that we're enrolling at EXPAND is actually quite different from the one that we did enroll in EMBARQ. With EXPAND, we're really taking patients really at any point in their journey. And for bileoesthesia, many of these patients will have had a CASI a number of years ago, but over the course of time, their condition deteriorates, and they may develop with that. So this is quite a different population than in BARCC, where we took incident patients around the time that they had their CASI. So, this is quite a different patient population, and on top of that, we're including other causes of cholestatic varitis in the clinic. Yeah.

Speaker Change: I think it's important to note that the patient population that we're enrolling at EXPAND is actually quite different than the one that we did enroll in EMBARQ.

Joanne Quan: You know, I think it's important to note that the patient population that we're rolling expand is actually quite different than the ones that were that we did enroll in Embark. We'll expand on really, you know, taking patients really at any point of their journey. And for Billy, a huge amount of these patients will have had a cassette number of years ago, but over the course of time their condition deteriorates, and they may develop the same. So this is quite a different population than EMBARK where we took really incident patients around the time that they had a cassette.

Speaker Change: With Xpand, we're really, you know, taking patients really at any point of their journey. And for Billy Oshiza, many of these patients will have had a KASI a number of years ago, but over the course of time, their condition deteriorates and they may develop a PSY.

Speaker Change: So this is quite a different population than in BARCC, where we took really incident patients around the time that they had a good time. So this is quite a different patient population, and on top of that, we're including other causes of cholestatic arthritis with chronic disease.

Joanne Quan: So this is quite a different patient population, and on top of that, we're including other causes of colostatic brightest with chronic disease.

Chris Peetz: Yes, I'd also add that in those compassionate use case studies, there have been biliary atresia patients in there quite a bit older than the embark age where they're enrolled as infants, and you do see that kind of hallmark response to IVAD treatment. So I think we've now figured out how to dose these medicines and what settings to advance them in.

Joanne Quan: Yes, I'd also add on that in those compassionate use case studies, there have been billionaire atries to patients in there quite a bit older than the embark age where they're enrolled as infants. And you do see that kind of hallmarked response to IVAT treatment. So I think we've now figured out how to dose these medicines and what settings to advance them in. Super helpful. Thank you so much.

Speaker Change: Yes, I'd also add on that in those compassionate use case studies, there have been biliary atresia patients in there.

Speaker Change: Quite a bit older than the Embark age where they're enrolled as infants. And you do see that kind of hallmark response to IVAD treatment. So I think we've now figured out how to dose these medicines and what settings to advance them in.

Unknown Executive: Super helpful. Thank you so much.

Speaker Change: Super helpful. Thank you so much.

Speaker Change: Yep, thanks for the questions.

Jessica Fye: The next question comes from Jessica Fye, from JPMorgan.

Jessica Fye: The next question comes from Jessica Fye from J.P. Morgan.

Speaker Change: The next question comes from Jessica Fye from J.P. Morgan.

Jessica Fye: Hey guys, good afternoon. Thanks for taking my questions out of view. First, where should we look for presentation of the interim PBC data for Valyxba, and what kind of additional details should we expect when you present those results? For example, are there subgroups where we should expect to learn more? Could we see it results broken down by severity of disease is defined by ALP levels or additional data on liver biomarkers or more details on the improvements observed in the fatigue dimension of the CDC 40.

Jessica Fye: Hey, guys, good afternoon. Thanks for taking my questions. I have a few.

Jessica Fye: Hey guys, good afternoon. Thanks for taking my questions. I had a few.

Chris Peetz: First, where should we look for presentation of the interim PBC data for valixibat? And what kind of additional details should we expect when you present those results? For example, are there subgroups where we should expect to learn more?

Jessica Fye: First, where should we look for presentation of the interim PBC data for felixibat and what kind of additional details should we expect when you present those results?

Chris Peetz: Could we see its results broken down by severity of diseases defined by ALP levels or additional data on liver biomarkers or more details on the improvements observed in the fatigue dimension of the PBC40? And then, separately, on the business development front, can you provide a bit of color about just what type of assets and which disease areas are most interesting to you? And lastly, can you just comment on your IP estate across LiveMarly and valixibat in terms of what IP you currently have and any pending applications? Thank you.

Speaker Change: For example, are there subgroups where we should expect to learn more? Could we see itch results broken down by severity of diseases defined by ALP levels?

Speaker Change: or additional data on liver biomarkers or more details on the improvements observed in the fatigue dimension of the PPC-40.

Jessica Fye: And then, separately, on the business development front, can you provide a bit of color about just what type of assets in which to these areas are most interesting to you?

Speaker Change: And then, separately, on the business development front, can you provide a bit of color about

Speaker Change: Just what type of assets and which disease areas are most interesting to you? And lastly, can you just comment on your IP estate across Live Marley and Belixibat in terms of what IP you currently have and any pending applications? Thank you.

Jessica Fye: And lastly, can you just comment on your IPF state across live Marley and Valyxba in terms of what IPU currently have and any pending applications? Thank you.

Chris Peetz: Thanks, Jessica, for the questions.

Peter Radovich: Thanks, Jess, for the questions. I can kind of, maybe I'll take a shot at the first and the last and then pass it over to Peter to talk about our DD strategy. First comment on the further data from the PBC study, we're preparing an abstract, and we'll work on getting it submitted for an upcoming Congress. You can't really predict when and where that lands and what's in it, so just say that we're looking at a number of the elements that you talked about there to consider for that abstract.

Chris Peetz: I can kind of maybe I'll take a shot at the first and the last and pass it over to Peter to talk about our DD strategy. First comment on the further data from the PBC study. We're preparing an abstract. We'll work on getting it submitted for an upcoming congress. You can't really predict when and where that lands and what's in it. So, you know, just say that we're looking at a number of the elements that you talked about there to consider for that abstract.

Speaker Change: Thanks, Jess, for the questions. I can kind of, maybe I'll take a shot at the first and the last and pass it over to Peter to talk about our DD strategy.

Speaker Change: First comment on the further data from the PBC study. We're preparing an abstract. We'll work on getting it submitted for an upcoming Congress. You can't really predict when and where that lands and what's in it.

Peter: Just say that we're looking at a number of the elements that you talked about there to consider for that abstract.

Peter Radovich: And then on intellectual property, actually, I'll direct you to our corporate deck on the backup slides. We have a summary of that that's recently been added and highlight the 2040 family of granted and pending patents that really tie back to the quite unique dosing profile for IVAT in general. With Marley and Elixabat in particular, that's led to all of these great advances we've seen across the programs that we're talking about here. And there's more detail in the backup of our public materials there that you can reference. Maybe Peter can talk about the BD strategy. Sure. Yeah. Thanks for the question, Jeff. We, you know, we're focused and rare.

Eric Bjerkholt: And then on intellectual property, actually directly to our corporate deck and the backup slides, we have a summary of that that's been recently added and highlight the 2040 family of granted and pending patents that really tie back to the quite unique dosing profile for I bat in general and live Marley and Valyxba in particular. That's led to all of these great advances we've seen across the programs that we're talking about here. And there's more detail in this in the backup of our public materials there that you can reference.

Speaker Change: And then on intellectual property, actually I'll direct you to our corporate deck and the backup slides. We have a summary of that that's been recently added.

Speaker Change: and highlight the 2040 family of granted and pending patents.

Speaker Change: That really tied back to the quite unique dosing profile for IBAT in general, and Livmarly and Elixabat in particular, that's led to all of these great advances we've seen across the programs that we're talking about here.

Peter: And there's more detail in the backup of our public materials there that you can reference.

Peter Radovich: Maybe Peter can talk about DD strategy. Sure. Yeah, thanks for the question, Jess. We were focused in rare disease. We really like rare pediatric opportunities. You know, essentially asking ourselves the question: programs where we could add a lot of value underappreciated programs. We have a really strong development, regulatory, commercial group in rare disease. So, looking in that kind of a corridor. And we have a high bar. We're very disciplined. We have a really strong base business with a great runway of catalyst in front of it. So, we take a lot of scrutiny at these opportunities.

Speaker Change: Maybe Peter can talk about BD strategy. Sure. Yeah, thanks for the question, Jeff. We, you know, we're focused in rare disease. We really like rare pediatric opportunities.

Peter Radovich: Sure. Thanks for the question, Jess. We're focused on rare disease. We really like rare pediatric opportunities. Essentially, we asked ourselves the question, programs where we could add a lot of value, underappreciated programs. We have a really strong development, regulatory, and commercial group in rare diseases, so we are looking in that kind of a corridor. And we have a high bar. We're very disciplined. We have a really strong base business with a great runway of catalysts in front of it, so we take a lot of scrutiny to these opportunities. Thank you. The next question comes next.

Speaker Change: You know essentially asking ourselves the question, programs where we could add a lot of value under appreciated programs.

Speaker Change: We have a really strong development, regulatory, commercial group in rare disease. So looking in that kind of a corridor. And we have a high bar. We're very disciplined. We have a really strong base business with a great runway of catalysts.

Speaker Change: We take a lot of scrutiny to these opportunities.

Speaker Change: Thank you.

Mike O's: The next question comes from Mike O's from Morgan Stanley.

Speaker Change: Any questions?

Unknown Executive: The next question comes from Mike Ulz from Morgan Stanley.

Operator: The next question comes from Mike Ulz from Morgan Stanley. Hi, this is Rohan on behalf of Mike. Thanks for taking

mike elath: The next question comes from Mike Oth from Morgan Stanley .

Robert: Hi, this is Robert from Mike. Thanks for taking our questions. Just in peak effect, you expect to pursue a label expansion to patients below 12 months.

Ramanan: Hi, this is Ramanan for Mike. Thanks for taking our questions. Just in PFIC, do you expect to pursue a label expansion to patients below 12 months? And can you just talk about how many patients are typically diagnosed that are below 12 months and what the opportunity there is? Thanks.

Robert: And I can just talk about how many patients are typically diagnosed that are below 12 months and with the opportunity there to take a look at the next question. Thanks.

Chris Peetz: Thanks for having a question. Overall, we feel that our label now is this PFIC expansion down to 12 months. It's in a really strong place.

Unknown Executive: Thanks for the question. Overall, we feel that our label, now with this PFIC expansion down to 12 months, is in a really strong place. We are, similar to what we do with eligible patients, evaluating the potential to submit yet another SMDA based on the infant data that's now mature. But frankly, we haven't come to a decision on that yet. So it's something that we could pursue. I feel that we are in a position now where we can capture most of the live Marley targeted patients for both allele syndrome and. The next question.

Chris Peetz: Thanks. We have a question. You know, overall, we feel that our label now, with this PFIC expansion down to 12 months, is in a really strong place. We are, you know, similar to what we do with eligible. We're evaluating the potential to submit yet another SMDA based on the infant data that's now mature, but frankly, we haven't come to a decision on that yet. So it's something that we could pursue. So I feel that we're in a position now where we capture most of the live Marley targeted patients for both allergy ill syndrome and the next question.

Speaker Change: Thanks for the question. Overall, we feel that our label now with this PFIC expansion down to 12 months, it's in a really strong place. We are, similar to what we do with eligible, we're evaluating the potential to submit yet another SMDA based on the infant data that's now mature.

Chris Peetz: We are similar to what we do with ology over evaluating the potential to submit yet another S-N-D-A based on the infant data that's now mature. But, frankly, I haven't come to a decision on that yet. So, it's something that we could pursue. So, feel that we're in a position now where we capture most of the live-marly targeted patients for both Allogial syndrome and PFIC.

Speaker Change: but frankly haven't come to a decision on that yet so it's something that we could pursue. I feel that we're in a position now where we capture most of the Liv Marley targeted patients for both Alajil syndrome and TFIC.

Chris Peetz: Thank you. Thanks for the question.

mike elath: Thank you.

David Libowitz: The next question comes from David Libowitz from CT. Thank you very much for taking my question. With respect to the EXPAND trial, given the heterogeneous population, how do you think the FDA would view it from a label expansion perspective?

Speaker Change: Thanks for the question.

Operator: The next question comes from David Lebowitz from Citi.

david liber: The next question comes from David Lebowitz from Citi.

David Lebowitz: Thank you very much for taking my question. With respect to the EXPAND trial, given the heterogeneous population, how do you think the FDA would view it from a label expansion perspective?

Joanne Quan: Thanks for the question, David.

David Lebowitz: Thanks for the question, David. Last, Joanne, maybe to talk a little bit about some of the thinking that went into that and the discussion with the FDA. Yeah.

Joanne Quan: Last Joanne, maybe to talk a little bit about some of the thinking that went into that and discussion with FDA. Thanks, David, for the question. You know, interesting that you asked the question that way because, in fact, the study came about because the FDA actually had suggested it. You know, we were receiving a compassionate news request, and so the FDA at one point said, you know, why don't you put these into a study. So, the expense that he has been designed, you know, keeping that input in mind. And so, you know, we do think the commonality is really coincidental, right?

Joanne: Thanks for the question, David. Last, Joanne, maybe to talk a little bit about some of the thinking that went into that and discussion with FDA. Yeah, thanks, David, for the question. You know, interesting that you asked the question that way because, in fact, the study came about because the FDA actually had suggested it.

Joanne Quan: Thanks, David, for the question. It's interesting that you asked the question that way because, in fact, the study came about because the FDA actually had suggested it. You know, we were receiving a compassionate use request, and so the FDA at one point said, you know, why don't you put this into a study? So this expanded study has been designed, you know, keeping that input in mind. And so, you know, we do think the commonality is really cholestatic arthritis.

Speaker Change: We were receiving a compassionate user request, and so the FDA at one point said, you know, why don't you put these into a study?

Speaker Change: So this, the EXPAND study has been designed, you know, keeping that input in mind.

Speaker Change: And so, you know, we do think the commonality is really cholecystic paritis.

Joanne Quan: I just, you know, call a stasis elevated syndrome bile acids and we know the effect that it has that I have on the horizon that's hitting. So, we're pretty confident in terms of, you know, our ability to fix the study and really in terms of the results that we once think is asking. Thanks, David.

Joanne Quan: Colostasis, Elevated Serum Bile Acids, and we know the effect that it has on pruritus. So we're pretty confident in terms of our ability to execute the study and really in terms of the results that we see. Once, once, I think it's over.

Unknown Executive: Colostasis, Elevated Serum Bile Acids, and we know the effect that it has on pruritus.

Speaker Change: you know, cholestasis, elevated serum bile acids, and we know the effect that it has, that I have on pruritus in that setting.

Speaker Change: So we're pretty confident in terms of our ability to execute this study and really in terms of the results that we'll see once I think it's executed.

Speaker Change: Got it. Thanks for taking my question.

David Libowitz: Thanks for the question.

Steven Seedhouse: The next question is from Steven Seedhouse from Raymond James, Financial Ink. Yeah, good afternoon. Thanks for the questions. Just don't expand. And how are you going to be measuring brightest because it seems like this trial would include pediatric end adult patients, depending on the conditions. So I'm just curious how you're going to standardize measuring the endpoint across disease types. Yeah, thanks for the question. We have an observer-rated intro, which has been validated and which we do have experience with in prior studies. And that's really designed for pediatric population. So, you know, it's right to be recognized that in a pediatric population can have different end points.

Steven Seedhouse: The next question is from Steven Seedhouse of Raymond James Financial Inc.

Speaker Change: Thanks for the question.

Speaker Change: The next question is from Steven Seedhouse from Raymond James Financial, Inc.

Steven Seedhouse: Yeah, good afternoon. Thanks for the questions. I just thought I'd expand.

Steven Seedhouse: Yeah, good afternoon. Thanks for the questions. Just thought I'd expand.

Steven Seedhouse: Um, how are you going to measure pruritus? Because it seems like this trial would include pediatric and adult patients, depending on the condition. So I'm just curious how you're going to standardize measuring the endpoint across disease types.

Steven Seedhouse: How are you going to be measuring pruritus, because it seems like this trial would include pediatric and adult patients, depending on the condition. So I'm just curious how you're going to standardize measuring the endpoint across disease types.

Joanne Quan: Yeah, thanks for the question. We have an observer-rated itch row that has been validated and which we have experience with in prior studies. And that's really designed for pediatric patients. So, you know, it's right of you to recognize that in a pediatric versus adult population, we can have different endpoints. But, you know, we feel pretty confident in terms of the design of the study and the selection of the endpoint and the fact that we have experience with this endpoint and know how it behaves.

Speaker Change: yes i think for the question we kind an observer rate hitr which has been validated and which we do have experienced within prior studies and that's really designed for a pedatric population

Unknown Executive: So, you know, it's right of you to recognize that in a pediatric versus adult population, we can have different endpoints. But, you know, we feel pretty confident in terms of the design of the study and the selection of the endpoint and the fact that we have experience with this endpoint and know how to handle it.

Speaker Change: So, you know, it's right of you to recognize that in a pediatric versus adult population we can have different endpoints, but, you know, we feel pretty confident in terms of the design of the study and the selection of the endpoint, and the fact that we have experience with this endpoint and know how to handle it.

Steven Seedhouse: But, you know, we feel pretty confident in terms of the design of the study and the selection at the end point. And the fact that we have experience with this endpoint is no problem. Yeah, and just to kind of follow up on that pediatric data set, that's the primary cohort: 45 patients. And that's where the primary analysis is. So the adults' scores will be part of the supplemental cohort. And think of the analysis plan designed in that way. Primary is based on the pediatric score. Oh, okay, and so the adults would be self-reported. It sure would there be a second observer?

Chris Peetz: Yeah, and just to kind of follow up on that, that pediatric data set, that's the primary cohort, 45 patients, that's where the primary analysis is. So the adult scores will be part of the supplemental cohort, and think of the analysis plan designed in that way. It's based on that.

Unknown Executive: Yeah, and just to kind of...

Speaker Change: Yeah, and just to kind of...

Speaker Change: Follow up on that, that pediatric data set, that's the primary cohort, 45 patients, that's where the primary analysis is, so the adult scores will be part of the supplemental cohort, and think of the analysis plan designed in that way, primary is based on the pediatric score.

Unknown Executive: Okay, and so the adults would have self-reported itch, or would there be a second observer? Yeah, so how does the end point look?

Speaker Change: Okay, and so the adults would be self-reported itch or would there be a second observer?

Unknown Executive: Yeah, so how does the employer look? Okay, and then the formulation here is it's just going to be the same liquid formulation that's commercially available? And then just, last question, and thanks for the multi-part question.

Steven Seedhouse: Yeah, so, yeah. How does the employee care about it? Right. So, for the adults, it's a self-reported endpoint. And that's one reason for us having them in a separate cohort. Based on our discussions with clinicians, you know, and just our experience with compassion and use, we do believe the majority of the patients will be pediatric. But we do think there will be some adult patients, and so we're studying them in a supplementary form, and we'll analyze those two. Okay, and then the formulation here is it's just going to be the same liquid formulation that's commercially available.

Speaker Change: yes how does the emple your book

Unknown Executive: Right, so yeah, for the adults, it's a self-reported endpoint. And that's one reason for us having them in a separate cohort. Based on our discussions with clinicians, you know, and just our experience with compassionate use, we do believe the majority of patients will be pediatric patients. But we do think there will be some adult patients. And so we're studying them in a supplemental analysis. Okay, and then the formulation here is it's just going to be the same liquid formulation that's commercially available? That's right, yep. And then just one last question, and thanks for the multi-part question.

Speaker Change: Right. So, for the adults, it's a self-reported endpoint, and that's one reason for us having them in a separate cohort. Based on our discussions with clinicians, you know, and just our experience of compassionate use, we do believe the majority of the patients will be pediatric.

Speaker Change: But we do think there will be some adult patients, and so we're studying them in a supplemental form.

Speaker Change: and we'll analyze those too.

Speaker Change: okay and then the formulation here is it's just going to be the same liquid formulation that's commercially available

Steven Seedhouse: That's right. Yeah. Okay.

Speaker Change: That's right, yep.

Steven Seedhouse: And then just last question, and thanks for the multi-part question. Are B8, are billiard regiapations that enrolled and expand that were maybe predict either on enrollment and that's our, excuse me, that enrolled and embark eligible to enroll in expand or is anyone who's been treated compassionately eligible to enroll in this study or are you excluding anyone with prior exposure to live Marley? Well, first, just on the, you know, to reiterate on the differences in the setting, the embark population was so young; they were too young, even really to recognize for the most part. You don't see that show up until basically what would have been the very end of even after the time period that Embark was looking at.

Speaker Change: Okay.

Speaker Change: And then just last question, and thanks for the multi-part question, are biliary atresia patients that enrolled in EXPAND that were maybe PRYDEC either on enrollment and that's, or excuse me, that enrolled in EMBARQ?

Unknown Executive: Are biliary atresia patients that enrolled and expanded who were maybe paretic either on enrollment and that's, or excuse me, that enrolled and embarked? Eligible to enroll in EXPAND, or anyone who's been treated?

Unknown Executive: Are biliary atresia patients that enrolled and expanded who were maybe parodic either on enrollment and that's, or excuse me, that enrolled and embarked? Eligible to enroll in EXPAND, or is anyone who's been treated? Compassionately. Eligible to enroll in this study, or are you excluding anyone with prior exposure to

Speaker Change: Is anyone eligible to enroll in EXPAND, or has anyone who's been treated compassionately

Speaker Change: Eligible to enroll in this study or are you excluding anyone with prior exposure to Liv Marley?

Chris Peetz: Well, first, just on the reiterate on the differences in the setting, the embark population was so young; they were too young, even really, to recognize pruritus, for the most part, you don't see that show up until basically, what would have been the very end, or even after the time period that embark was looking at. So it's a very distinct setting to go after the Yeah, you know, we're going to be taking this, getting towards grade school-age children in a lot of instances.

Speaker Change: Well, first, just to reiterate on the differences in the setting, the EMBARQ population was so young, they were too young even really to recognize pruritus for the most part. You don't see that show up until...

Speaker Change: Basically, what would have been the very end or even after the time period that Embark was looking at. So it's a very distinct setting to go after these, you know, what are going to be taking us getting towards grade school age children for a lot of instances.

Chris Peetz: So it's a very distinct setting to go after these, you know, what are going to be getting towards grade school age children for a lot of instances. So just to follow up on that, Chris, if there are patients that responded by biolasses or billiard rubin or some metric in embark, are they eligible to enroll and expand if there are billiard regiapations? Thank you. Yeah, the EMBARK study results recall that you know, we really in that setting, you're not seeing a response signal, right. So those patients either had successful cussi procedures. You wouldn't expect them to progress again for several years, or they had a transplant already.

Chris Peetz: So, just to follow up on that, Chris, if there are patients that responded by bilasses or bilirubin or some other metric and embark, are they eligible to enroll and expand if they're a biliary atresia patient?

Speaker Change: So, just to follow up on that, Chris, if there are patients that responded by bilases or bilirubin or some metric and embark, are they eligible to enroll and expand if they're a biliary atresia patient?

Chris Peetz: The comment I'd make there, yeah, the EMBARQ study results recall that in that setting, you're not really seeing a response signal, right, so those patients either had successful CASI procedures, you wouldn't expect them to progress again for several years, or they had a transplant already, so it's kind of, it's, we didn't even see it as a really relevant question. We can circle back on, I don't know the exclusion criteria off the top of my head right now, but it was, it's just such a different patient population; you wouldn't necessarily see those necessarily connect. Okay, that makes sense.

Speaker Change: and many more. Thank you. Thank you.

Speaker Change: I mean, the comment I'd make there, yeah, the EMBARQ study results recall there that

Speaker Change: We really, in that setting, you're not seeing a response signal, right? So those patients are either had successful CASI procedures.

Speaker Change: You wouldn't expect them to progress again for several years, or they had a transplant already. So it's kind of, it's, it's, we didn't even see it as a really relevant question.

Chris Peetz: So it's kind of, it's, we didn't even see it as a really relevant question. We can circle back on; I don't know the exclusion criteria. I'll talk my head right now, but yeah, it was just such a different patient population. You wouldn't see those necessarily connect. Okay, makes sense.

Speaker Change: We can circle back on, I don't know the exclusion criteria off the top of my head right now, but it was, it's just such a different patient population, you wouldn't see those necessarily connect.

Operator: Okay, that makes sense. Thanks so much.

Chris Peetz: Thanks a lot.

Speaker Change: Okay, makes sense. Thanks so much.

Brian's Granny: The next question comes from Brian's granny from Bayes. Hi, this is Charlie on for Brian. Just a couple questions on the biolasset portfolio. Previously, it was sitting around, you know, low to mid 20 per quarter.

Speaker Change: Thank you.

Brian Skorney: The next question comes from Brian Skorney from Baird.

Speaker Change: The next question comes from Brian Skorney from Baird.

Charlie: Hi, this is Charlie on behalf of Brian. Just a couple questions on the bile acid portfolio. Previously, it was sitting around, you know, low to mid-20 per quarter. So just wondering, you said it was demand growth, but what you're seeing, if you could give a little more detail there, as well as if you're planning for any other opportunities to expand the value you're getting out of coal bomb. Thank you.

Unknown Executive: Hi, this is Charlie on behalf of Brian. Just a couple questions on the bile acid portfolio. Previously, it was sitting around, you know, low to mid-20 per quarter. So just wondering, you said it was demand growth, but what you're seeing, if you could give a little more detail there, as well as if you're planning for any other opportunities to expand the value you're getting out of Coal Bomb. Thank you

Speaker Change: Hi, this is Charlie on for Brian . Just a couple questions on the bile acid portfolio. Previously, it was sitting around, you know,

Peter Radovich: So just wondering, you know, you said it's demand growth, but what you're seeing if you could give a little more detail there as well as if you're planning for any other opportunities to expand the value you're getting out of coal bomb. Thank you.

Charlie: load to mid twenty per quter so just wondering you know you said it's demand growth but what you're seeing if you could give a little more detail there as well as if you're planning for any other opportunities to expand the value you're getting out of coalbom thank you

Peter Radovich: Yeah, thanks for the question.

Unknown Executive: Yeah, thanks for the question. I'll ask Peter to Yeah, you know, and I think I'd kind of go back to

Peter Radovich: I'll ask Peter to give some color. Yeah, you know, and I think I can go back to with the violence of products that, you know, if you're looking at Q1 versus Q2, just a reminder that Q1 was artificially low because of the cyber attack that occurred that you know, pharmacy claim processing in the U.S. So that's one, one dynamic keep in mind. We do expect kind of steady demand growth, kind of in line with historical averages and what you've seen over time, with, you know, reminding that there is quarter-to-quarter variability with these products.

Speaker Change: Yeah, thanks for the question. I'll ask Peter to...

Peter Radovich: Yeah, you know, and I think I'd kind of go back to the bioasset products that, you know, if you're looking at Q1 versus Q2, just a reminder that Q1 was artificially low because of the cyber attack that occurred that impacted pharmacy claim processing in the U.S. So that's just one dynamic to keep in mind. You know, we do expect kind of steady demand growth kind of in line with historical averages and what you've seen over time, reminding you that there is quarter to quarter variability with these products.

Peter: Yeah, you know, and I think I'd kind of go back to with the bile acid products that, you know, if you're looking at Q1 versus Q2, just a reminder that Q1 was artificially low because of the cyber attack that occurred that impacted, you know,

Speaker Change: Pharmacy Claim Processing in the U.S.

Speaker Change: One dynamic to keep in mind, we do expect kind of steady demand growth kind of in line with historical averages and what you've seen over time.

Peter Radovich: And in terms of Colbaum expansion, yeah, we think the Colbaum label is in a great spot. It, you know, facilitates the use and reimbursement of that product across the various settings where it's been established. So, you know, no major plans right now to focus on expansion there.

Speaker Change: You know, reminding you that there is quarter-to-quarter variability with these products.

Peter Radovich: And in terms of cobalm expansion, yeah, that we think the cobalm label is a great spot. It, you know, facilitates the use and reimbursement of that product across the various settings where it's been established. So, you know, no major plans right now to focus on expansion there.

Speaker Change: And in terms of Colebaum expansion, yeah, we think the Colebaum label is in a great spot. It, you know, facilitates the use and reimbursement of that product across the various settings where it's been established. So, you know, no major plans right now to focus on expansion.

Speaker Change: expansion there

Unknown Executive: Thanks for the question, Trent.

Unknown Executive: Thanks for the question, Trevor.

Speaker Change: Thanks for the question, Trent.

John Lillivan: The next question comes from John Lillivan from Citizen's GMP. Hey, thanks for taking the questions. A couple on P6 for me. Wondering, you know, without talking about sales here, you know, any, you know, metrics on the P6 launch in the U.S. These are going well. I think that you guys can improve upon throughout the rest of the year.

John Wolleben: The next question comes from John Wolleben from Citizens GMP.

Speaker Change: The next question comes from John Wolleben from Citizens GMP.

Unknown Executive: Hey, thanks for taking the questions. A couple on PFIG for me. Wondering, you know, without talking about sales here, any metrics on the PFIG launch in the U.S., things are going well, things that you guys can improve upon throughout the rest of the year. And then, with the label expansion recently, can you discuss the added risk of propylene glycol toxicity? Any observation of that in the clinical trials, or is this just a risk that the FDA wanted to include?

Unknown Executive: Hey, thanks for taking the questions. A couple on PFIG for me. Wondering, you know, without talking about sales here, any metrics on the PFIG launch in the U.S., things are going well, things that you guys can improve upon throughout the rest of the year. And then, with the label expansion recently, can you discuss the added risk of propylene glycol toxicity? Any observation of that in the clinical trials, or is this just a risk that the FDA wanted to include?

John Wolleben: Hey, thanks for taking the questions. A couple on PFIG for me. Wondering, you know, without talking about sales here, you know, any, you know, metrics on the PFIG launch in the U.S.,

Speaker Change: Things are going well, things that you guys can improve upon throughout the rest of the year. And then with the label expansion recently, can you discuss the added risk of the propylene glycol toxicity? Any observation of that in the clinical trials, or is this just a risk that FDA wanted to include?

John Lillivan: And then with the label expansion recently, can you discuss the added risk of the coupling glycol toxicity? Any observation that in the clinical trials or just the risk that they want to include?

Peter Radovich: Yeah, kind of break that up between Peter and Joanne to talk about the commercial and safety aspects. Yeah, thanks for the question. Certainly real happy with how we've come out of the gate with P6 have seen. We had, you know, a number of patients, you know, transition from our clinical trial, expanded access programs to commercial drugs that have proceeded very well, as well as in the novel prescriptions for P6 patients to come in. You know, we've talked about it before that we expect, you know, revenue contribution from P6 in 2024 to be, you know, pretty modest given reimbursement.

Peter Radovich: Yeah, I'll kind of break that up between Peter and Joanne to talk about the commercial and safety aspects.

Speaker Change: Yeah, I'll kind of break that up between Peter and Joanne to talk about the commercial and safety aspects.

Joanne Quan: Yeah, thanks for the question. Certainly, we're really happy with how we've come out of the gate with PFIC, and we have seen a number of patients transition from our clinical trial expanded access programs to commercial drugs, that's proceeded very well, as well as de novo prescriptions for PFIC patients coming in. We've talked about it before that we expect revenue contribution from PFIC in 2024 to be pretty modest given reimbursement.

Peter: Yeah, thanks for the question. Certainly real happy with how we've come out of the gate with PFIC. I've seen, we had, you know, a number of patients, you know, transition from our clinical trial, expanded access programs to commercial drug. That's proceeded very well, as well as, you know, de novo prescriptions for PFIC patients come in.

Speaker Change: We've talked about it before that we expect revenue contribution from PFIC.

Speaker Change: And I think we expect 2024 to be pretty modest given reimbursement. We expect a fair bit of free drug shipment this year for PFIC. It is contemplated a little bit in our guidance, but really expect 2025 to be where PFIC starts contributing more.

Peter Radovich: We expect a fair bit of free drug shipment this year for P6. It is contemplated, you know, a little bit in our guidance, but, you know, really expect 2025 to be where P6 starts contributing more. So, you know, good, good start.

Joanne Quan: We expect a fair bit of free drug shipment this year for PFIC; it is contemplated, you know, a little bit in our guidance, but, you know, really expect 2025 to be where PFIC starts contributing more. So, you know, a good, good start. And then, with President Propylene Gladfollow and the clinical development program, I'll let Joanne speak to that. Yeah, thanks for the question. Just to be clear, we have not seen PG toxicity yet.

Joanne Quan: And then, with the presence of the propylene glycol in the clinical development program, I'll let Joanne speak to that.

Speaker Change: so good good start and then what president the propleing on the clinical development program all show ans speak to that yeah that thinks for the question just be clear we have not seen pg texasity in our clinical studies and in the context of the defect level expansion

Unknown Executive: Thanks for the question. Just to be clear, we have not seen PG toxicity in our clinical studies, and in the context of the PFIC label expansion, we and the FDA looked pretty carefully and kind of combed through all of that patient experience data. You know, this concern from the FDA really arose in light of the younger patients being considered for the expansion. And, you know, we have seen warnings about PG toxicity with other labels, for instance, so this is not new to us.

Joanne Quan: Yeah, thanks for the question. Just to be clear, we have not seen PG toxicity in our clinical studies. And in the context of the difficult level expansion, we and the FDA looked pretty carefully and kind of came through all of that patient experience data.

Joanne Quan: Thanks for the question. Just to be clear, we have not seen PG toxicity in our clinical studies, and in the context of the PFIC label expansion, we and the FDA looked pretty carefully and kind of combed through all of that patient experience data. You know, this concern from the FDA really arose in light of the younger patients being considered for the expansion. And, you know, we have seen warnings about PG toxicity with other labels, for instance, so this is not new to us.

Speaker Change: We and the FDA looked pretty carefully and kind of combed through all of that patient experience data.

Joanne Quan: You know, this concerns from the FDA really arose in light of the younger patients being considered for the expansion. And, you know, we have seen warns about PG toxicity with all the, for instance, so this is not, not you to us. You know, we do feel comfortable that the physicians who are describing this know their patients; they know what to look for, they are properly monitoring. So we think from a practical perspective that this is well handled.

Speaker Change: This concern from the FDA really arose in light of the younger patients being considered for the expansion. And we have seen warnings about PG toxicity with other labels, for instance, so this is not new to us.

Joanne Quan: You know, we do feel comfortable that the physicians who are prescribing this know their patients, they know what to look for, and they're appropriately monitoring. So we think from a practical perspective that this is well handled in terms of the routine patient experience.

Unknown Executive: You know, we do feel comfortable that the physicians who are prescribing this know their patients, they know what to look for, and they're appropriately monitoring. So we think from a practical perspective that this is well handled in terms of routine patient care.

Speaker Change: You know, we do feel comfortable that the physicians who are prescribing this know their patients, they know what to look for, they're appropriately monitoring, so we think from a practical perspective that this is well handled in terms of the routine patient care.

Joanne Quan: and Trump, so that's the routine patient care. Got it.

Ed Arce: Thanks. Thanks for the question.

Speaker Change: Got it. Thanks.

Speaker Change: Thanks for the question.

Thomas: And the next question is from Ed Arce from H.C. Hi, everyone.

Ed Arce: And the next question is from Ed Arce from HC Wainwright.

Speaker Change: And the next question is from Ed Ars from HC Wainwright.

Thomas: Hi, everyone. This is Thomas here asking a couple of questions for Ed. Thank you so much for taking our questions. Perhaps first, just wondering, for Kimmy Dahl, how would you characterize the additional commercial opportunity in CCX as a non-legal indication, I suppose in terms of quantity and also relative to our immunodeficient cells?

Thomas: This is Thomas. I've been a couple questions for Ed. Thank you so much for the kind of questions. Perhaps first, just wondering for Kenidaw, how you characterize the additional commercial opportunity and C.C.X. as a non-legal indication supporting terms of quantity and also how relative to our accumulative health.

Speaker Change: Hi everyone, this is Thomas here asking a group of questions for Ed. Thank you so much for taking our questions.

Thomas: Perhaps first, I'm just wondering for King and Dahl,

Unknown Executive: How would you characterize the additional commercial opportunity in CCX as a non-legal indication, I suppose in terms of quantity and also relative to our immunodeficient cells?

Thomas: How do you characterize the additional commercial opportunity in CCX as a non-legal indication? I suppose in terms of quantity and also...

Speaker Change: Relative to our existing cells.

Peter Radovich: Thanks, Thomas, for the question.

Thomas: Thanks, Thomas, for the question. I'll ask Peter to comment on that. Meanwhile, I've got some color on the undiagnosed population for CTX.

Unknown Executive: Thanks, Thomas, for the question. I'll ask Peter to comment on that and get some color on the undiagnosed population for CTX.

Peter Radovich: I'll ask Peter to comment on that. Get some color on the undiagnosed population for C.C.X. Yeah, and we look at the C.C.X. Opportunity, the best estimates from literature, market research, K.O.L., probably somewhere between 1,000 to 2,000 prevalent patients with C.C.X. In the United States, but only 10% of those are diagnosed right now is the best estimate of what we see. The biggest opportunity is that we can increase that 10% to something higher. So we are investing in disease state awareness, initiatives reaching out to physicians who may see these patients as they're presenting with various symptoms on their journey to a diagnosis and trying to both increase the rate of diagnosis as well as speed up the time to get to the diagnosis.

Peter: Thanks, Thomas, for the question. I'll ask Peter to comment on that and get some color on the undiagnosed population for CTX.

Unknown Executive: Again, you know, we, as we look at the CTX opportunity, the best estimates from literature, market research, and KOLs are probably somewhere between 1,000 to 2,000 prevalent patients with CTX in the United States, but only 10% of those are diagnosed right now. That's the best estimate of what we see. So, you know, probably the biggest opportunity is if we can increase that 10% to something higher.

Peter Radovich: Again, you know, we, as we look at the CTX opportunity, the best estimates from literature, market research, and KOLs are probably somewhere between 1,000 to 2,000 prevalent patients with CTX in the United States, but only 10% of those are diagnosed right now. That's the best estimate of what we see. So, you know, probably the biggest opportunity is if we can increase that 10% to something higher.

Peter: Again, you know, we, as we look at the CTX opportunity, you know, the best estimates from literature, market research, KOLs,

Peter: Probably somewhere between 1,000 to 2,000 prevalent patients with CTX in the United States.

Unknown Executive: but only ten percent of those our diagnosed right now it is the best best estimate of what we see

Unknown Executive: so there's probably the biggest opportunity if we can increase that ten percent to something higher so we are investing intodisease state awareness

Unknown Executive: So we are, you know, investing in disease state awareness initiatives, reaching out to physicians who may see these patients as they're presenting with various symptoms on their journey to a diagnosis and trying to both increase the rate of diagnosis as well as speed up the time to get to the diagnosis. And we have seen a lot of interest from prescribers, neurologists, and other physicians who kind of take an interest in that. And then, you know, if Ketanediol is approved by FDA and we're able to promote it, certainly the product has never been promoted before. So I think there'll be an opportunity out there and even for the diagnosis patients in clinic, you know, raise awareness of the benefits of and potential benefits of Ketanediol, support reimbursement, things like that.

Peter Radovich: So we are, you know, investing in disease state awareness initiatives, reaching out to physicians who may see these patients as they're presenting with various symptoms on their journey to a diagnosis and trying to both increase the rate of diagnosis as well as speed up the time to get to the diagnosis. And we have seen a lot of interest from prescribers, neurologists, and other physicians who kind of take an interest in that. And then, you know, if Kenodiol is approved by FDA and we're able to promote it, certainly the product has never been promoted before. So I think there'll be an opportunity out there and even for the diagnosis patients in clinic, you know, raise awareness of the benefits of and potential benefits of Kenodiol, support reimbursement, things like that.

Unknown Executive: and maybe one more from us. This one for the Lexibak.

Peter: you know, initiatives, reaching out to physicians who may see these patients as they're presenting with various symptoms on their on their journey to a diagnosis and trying to

Peter: both increase the rate of diagnosis as well as speed up the time to get to the diagnosis

Thomas: And have seen a lot of interest from, you know, prescribers, you know, neurologists and other physicians kind of take interest in that. And then, you know, if Kenidaw is approved by FDA and we're able to promote, you know, certainly the product has never been promoted before. So they'll be out to you out there. And even for the diagnosis, patients in clinic in raise awareness of the benefits of 10% of Kenidaw support, reimbursement, things like that.

Unknown Executive: and have seen a lot of interest from, you know, prescribers, you know, neurologists and...

Unknown Executive: and other positions kind of take interest in that. And then, you know, if Kenodiol is approved by FDA and we're able to promote...

Unknown Executive: Certainly the product has never been promoted before, so I think there will be an opportunity out there, and even for the diagnosis patients in clinic, you know, raise awareness of the benefits of, potential benefits of Kenodiol, support reimbursement, things like that.

Thomas: And this is thanks.

Unknown Executive: Regarding Vantage and this case, has the FDA or the NRA reviewed the interim data that you have so far? Has there been any feedback from any regulatory agency?

Unknown Executive: And maybe one more from us. This one for the Lexibak.

Thomas: And maybe one more from us. There's this one for the let's see back. We're going vintage, vintage, and this is. Has the FDA or the M.A.

Unknown Executive: And that's it. Thanks. And maybe one more from us, just this one for the Lexibag, regarding Vantage in this case.

Thomas: Review the interim data that you have so far, any feedback from any regulatory agency. Thanks so much for the question. You have the in the cadence here of regulatory review. We have a head of pre-IMD discussion designing the studies, and the next opportunity would be after we have final results is what our plans are. So no kind of interim discussions on plans. Okay. Thank you.

Speaker Change: Has the FDA or the NRA reviewed the interim data that you have so far and any feedback from any regulatory agency?

Unknown Executive: Regarding Vantage and this case, has the FDA or the NRA reviewed the interim data that you have so far? Has there been any feedback from any regulatory agencies?

Unknown Executive: Thanks, Thomas, for the question. Cadence here of regulatory review, we have had a pre-IND discussion designing the studies, and the next opportunity would be after we have final results to discuss what our plans are. So, no interim discussions on plans.

Unknown Executive: Thanks, Thomas, for the question.

Speaker Change: Cadence here of regulatory review. We have had a pre-IND discussion designing the studies and the next opportunity would be after we have final results is what our plans are. So no interim discussions on plans.

Unknown Executive: Okay, thank you. Thank you so much for the kind questions.

Unknown Executive: Thank you so much for the panel questions. Yeah, thanks for the questions.

Unknown Executive: Okay, thank you. Thank you so much for the kind of questions.

Speaker Change: Yeah, thanks for the questions.

Unknown Executive: And that was our next question.

Operator: And that was our last question. So I will hand it back over to Craig Peetz, the CEO, for any final remarks.

Unknown Executive: So I will have back over to cream state to see you for any final remarks. Thank you, operator, and thank you all for joining us today. We appreciate the support for our mirror in our programs and have a good evening. Bye.

Unknown Executive: And that was our last question, so I will hand back over to Corinne Peetz, the CEO , for any final remarks.

Chris Peetz: Thank you, operator, and thank you all for joining us today. We appreciate the support for Mirum and our programs. Have a good evening.

Operator: And this concludes today's call. Thank you for joining us. You may now disconnect from the call.

Speaker Change: thank you operator and thank you allfor joining us today we appreciate the support for mramin our programs have a good evening by

Unknown Executive: And it's going to be today's call. Thank you for joining. We went out to connect from a call.

Speaker Change: And this concludes today's call. Thank you for joining. You may now disconnect from the call.