Financial Data and Key Metrics Changes - Total revenues for Q1 2025 reached $111.6 million, representing a 61% increase compared to Q1 2024 [5] - The company updated its full-year revenue guidance to $435 million to $450 million, driven by strong growth across all commercial medicines [5][11] - Net product revenue for Q1 2025 was $111.6 million, up from $69.2 million in Q1 2024 [15] - Cash, cash equivalents, and investments at March 31, 2025, were $298.6 million, compared to $292.8 million at the beginning of the year [15] - Operating expenses for Q1 2025 totaled $126.8 million, including R&D expenses of $46 million and SG&A expenses of $57.7 million [15] Business Line Data and Key Metrics Changes - Total net product sales for LIVMARLI reached $73.2 million in Q1 2025, a growth of over 70% compared to Q1 2024 [11] - U.S. LIVMARLI sales were $49.5 million, driven by robust new patient demand [11] - International LIVMARLI sales were $23.7 million, with strong demand growth in direct European markets [12] - Bile acid products generated $38.4 million in net product sales, representing a 47% growth year-over-year [13] Market Data and Key Metrics Changes - The approval of LIVMARLI in Japan for PFIC and Alagille syndrome is expected to enhance market presence [6] - Citexly received FDA approval for the treatment of CTX, with promotional efforts underway to reach the underdiagnosed community [6][13] - The company is focusing on engaging healthcare professionals to identify patients with CTX following the recent approval [13] Company Strategy and Development Direction - The company aims to continue advancing its commercial medicines and high-impact pipeline, with a focus on rare diseases [5][19] - The VISTA study of velixibat is nearing completion of enrollment, with top-line data expected in Q2 2026 [8] - The company plans to initiate a Phase II study for MRM-3379 in fragile X syndrome later this year [10][19] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the strong start to the year and the growth of commercial medicines exceeding initial expectations [18] - The company is well-positioned for continued leadership in rare diseases, with a strong financial position and cash flow positivity expected for the full year [16][19] - Management highlighted the importance of recent regulatory approvals in supporting long-term growth potential [18] Other Important Information - The cash contribution margin from the commercial business improved from approximately 47% in Q1 2024 to approximately 53% in Q1 2025 [16] - The company is focused on raising disease awareness to improve patient identification for CTX [67] Q&A Session Summary Question: Insights on velexibat PBC data and discontinuation rates - Management noted that the data matured positively in the interim analysis, with strong response curves and minimal discontinuations due to diarrhea [21][22] Question: Additional IP around LIVMARLI tablet formulation and adoption - The tablet formulation has resulted in novel IP, with an allowed patent expected to extend coverage [24] - Management anticipates strong adoption among older children and adolescents due to the convenience of a single tablet [25] Question: Comparison of interim Phase II PBC data with competitors - Management highlighted the strengths of the pelixibat program, particularly in placebo-adjusted outcomes [31] Question: Update on LIVMARLI access versus competitors - Access to LIVMARLI in the U.S. is strong, with no significant barriers noted [39] Question: Expectations for the VISTA study and competitive positioning - Management expressed optimism for velexibat's activity level based on robust data from the VANTAGE study [43][46] Question: Update on inventory levels and operating expenses - Management clarified that the recent inventory increase is related to new international partners and is expected to normalize [52] Question: PFIC launch performance and sustainability of profitability - The PFIC launch has exceeded expectations, and while cash flow positivity is expected, profitability in a GAAP sense is not anticipated soon [73]

Financial Data and Key Metrics Changes - Total revenues for Q1 2025 reached $111.6 million, representing a 61% growth compared to Q1 2024 [5] - The company updated its full-year revenue guidance to $435 million to $450 million, driven by strong growth across all commercial medicines [5][10] - Net product revenue for Q1 2025 was $111.6 million, up from $69.2 million in Q1 2024 [13] - Cash, cash equivalents, and investments at March 31, 2025, were $298.6 million, compared to $292.8 million at the beginning of the year [13] - Operating expenses for Q1 2025 totaled $126.8 million, including R&D expenses of $46 million and SG&A expenses of $57.7 million [13][15] Business Line Data and Key Metrics Changes - Total net product sales for LIVMARLI reached $73.2 million in Q1 2025, a 70% increase compared to Q1 2024 [10] - U.S. LIVMARLI sales were $49.5 million, driven by robust new patient demand [10] - International LIVMARLI sales were $23.7 million, with strong demand growth in direct European markets [10] - Bile acid products generated $38.4 million in net product sales, representing a 47% growth year-over-year [12] Market Data and Key Metrics Changes - The approval of LIVMARLI's tablet formulation is expected to enhance patient convenience and drive adoption among older children and adolescents [10][24] - The company has seen an increase in new CTX patients since the FDA approval of Citexly in February 2025 [12][66] - The market for PSC is characterized as unsatisfied, with no FDA-approved therapies currently available, positioning velixibat favorably [46] Company Strategy and Development Direction - The company is focused on advancing its commercial medicines and high-impact pipeline, with three important regulatory approvals received since the start of the year [6][17] - The VISTA study of velixibat is nearing completion of enrollment, with top-line data expected in Q2 2026 [7][18] - The company plans to initiate a Phase II study for MRM-3379 in fragile X syndrome later this year [9][18] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the strong start to the year and the growth of commercial medicines exceeding initial expectations [17] - The company is well-positioned for continued leadership in rare diseases, with a strong financial position and cash flow positive outlook for the full year [15][72] Other Important Information - The cash contribution margin from the commercial business improved from approximately 47% in Q1 2024 to approximately 53% in Q1 2025 [15] - The company is focused on raising disease awareness to improve patient identification for underdiagnosed conditions [66] Q&A Session Summary Question: Insights on the additional velexibat PBC data - Management noted that the data matured positively in the interim analysis, with strong response curves [20][21] Question: Additional IP around the Libmarli tablet formulation - The tablet formulation has resulted in novel IP, with an allowed patent expected to extend coverage [22][23] Question: Comparison of interim Phase II PBC data with competitors - Management highlighted strengths in the pelixibat program, particularly in placebo-adjusted differences [30] Question: Update on Marley access versus competitors - Access to LIVMARLI in the U.S. is strong, with no barriers anticipated for either indication [38] Question: Expectations for the VISTA study - Management is optimistic about velexibat's activity level, using VANTAGE as a benchmark [42] Question: Update on patient identification for Citexly - There has been a nice uptick in patient identification since the FDA approval, attributed to promotional efforts [66][64] Question: Sustainability of profitability into 2026 - The company expects to be cash flow positive for the full year but does not anticipate profitability in a GAAP sense soon [72]

Financial Data and Key Metrics Changes - Total revenues for Q1 2025 reached $111.6 million, representing a 61% growth compared to Q1 2024 [4] - The company updated its full-year revenue guidance to $435 million to $450 million, driven by robust growth from all three commercial medicines [4][10] - Net product revenue for Q1 2025 was $111.6 million, compared to $69.2 million in Q1 2024 [13] - Cash, cash equivalents, and investments at March 31, 2025, were $298.6 million, up from $292.8 million at the beginning of the year [13] - Operating expenses for Q1 2025 totaled $126.8 million, including R&D expenses of $46 million and SG&A expenses of $57.7 million [13][14] Business Line Data and Key Metrics Changes - Total net product sales for LIVMARLI reached $73.2 million in Q1 2025, an increase of over 70% compared to Q1 2024 [10] - U.S. LIVMARLI sales were $49.5 million, driven by strong new patient demand across indications [10] - International LIVMARLI sales were $23.7 million, with strong demand growth in direct European markets [10] - Bile acid products generated $38.4 million in net product sales, representing a 47% growth over the same quarter last year [12] Market Data and Key Metrics Changes - The approval of LIVMARLI in Japan for PFIC and Alagille syndrome is expected to enhance market presence [5] - Citexly received FDA approval for the treatment of CTX, with promotional efforts underway to reach the underdiagnosed community [5] - The company is focused on engaging healthcare professionals to identify patients with CTX following the recent FDA approval [12] Company Strategy and Development Direction - The company aims to advance its commercial medicines and high-impact pipeline, with a focus on rare diseases [4][16] - The VISTA study of velixibat is nearing completion of enrollment, with top-line data expected in Q2 2026 [6][17] - The company plans to initiate a Phase II study for MRM-3379 in fragile X syndrome later this year [9][17] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the strong start to the year and the growth of commercial medicines exceeding initial expectations [16] - The company is well-positioned for continued leadership in rare diseases, with a strong financial position and cash flow positivity expected for the full year [15][17] - Management highlighted the importance of recent regulatory approvals in supporting long-term growth potential [16] Other Important Information - The cash contribution margin from the commercial business improved from approximately 47% in Q1 2024 to approximately 53% in Q1 2025 [14] - The company is focused on maintaining financial independence and executing its business plan effectively [15] Q&A Session Summary Question: Insights on velexibat PBC data and discontinuations - Management noted excitement over the data maturation in the interim analysis, with strong response curves and minimal discontinuations due to diarrhea [19][20] Question: Additional IP around LIVMARLI tablet formulation - The tablet formulation has resulted in novel IP, with an allowed patent expected to grant soon, extending coverage to February 2043 [22] Question: Comparison of interim Phase II PBC data with competitors - Management highlighted strengths in the pelixibat program, emphasizing strong outcomes in both change from baseline and placebo-adjusted differences [30] Question: Update on LIVMARLI access versus competitors - Access to LIVMARLI in the U.S. is strong, with no barriers noted for either indication, supported by a strong clinical value proposition [37] Question: Expectations for the VISTA study and competitive positioning - Management expressed optimism for velexibat's activity level in the VISTA study, with a strong market position anticipated due to the lack of approved therapies for PSC [45][46] Question: Update on patient identification for Citexly - Management reported a nice uptick in patient identification since the FDA approval, driven by promotional efforts and disease awareness initiatives [64] Question: PFIC launch performance and sustainability of profitability - The PFIC launch has exceeded expectations, and while cash flow positivity is anticipated, profitability in a GAAP sense is not expected soon due to non-cash charges [70][71]