LIVMARLI

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Mirum(MIRM) - 2025 Q2 - Earnings Call Transcript
2025-08-06 21:32
Financial Data and Key Metrics Changes - Total revenues for the second quarter reached $128 million, representing a 64% increase compared to the same quarter last year [6][19] - The company raised its full-year revenue guidance for 2025 to a range of $490 million to $510 million, indicating nearly 50% top-line growth [7][13] Business Line Data and Key Metrics Changes - Net product sales for Lidmarley were approximately $128 million, driven by strong demand in both the U.S. and international markets [10][19] - In the U.S., Lidmarley generated about $57 million in net product sales, with increased patient awareness contributing to higher volumes [10][11] - Internationally, Lidmarley sales reached $31 million, supported by expanding reimbursement and strong demand [12][13] Market Data and Key Metrics Changes - The U.S. market for Lidmarley is seeing increased patient penetration, with approximately 70-75% persistence rates after one year for Alagille syndrome patients [25][59] - The company is experiencing growth in the PFIC market due to increased awareness and genetic testing, leading to more diagnoses [11][59] Company Strategy and Development Direction - The company's strategy focuses on commercial execution, scientific innovation, and financial discipline, with a commitment to delivering life-changing medicines for rare diseases [5][20] - The pipeline includes three late-stage studies expected to yield pivotal data over the next 24 months, particularly in primary sclerosing cholangitis [8][16] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's position and the opportunities ahead, highlighting the strong performance of its medicines and pipeline [20] - The company is optimistic about Lidmarley's potential to exceed $1 billion in revenue, driven by ongoing growth in Alagille syndrome and PFIC indications [14][62] Other Important Information - The company reported operating cash flow positivity for the quarter, with cash and investments totaling $322 million, reflecting a $29 million increase from the end of the previous year [19] Q&A Session Summary Question: What are the overall therapy persistence rates for Lidmarley? - Management indicated that persistence rates for Alagille syndrome patients are around 70-75% after one year, with lower attrition in subsequent years [25] Question: Can you provide details on the expected revenue distribution between Lidmarley and the bile acid business? - Management did not break down guidance by specific products but noted that trends observed year-to-date are expected to continue [33] Question: What are the main drivers of growth in Lidmarley sales? - Key drivers include increased awareness of PFIC and the introduction of a single tablet formulation, which has received positive feedback [39][41] Question: Are there any inventory impacts in the second quarter? - Inventory impacts are primarily relevant to Japan and Takeda, with no significant inventory issues in the U.S. or Europe [44] Question: What is the status of the Fragile X study? - The company has received clearance from the FDA and is on track to initiate the Phase II study by the end of the year [52] Question: How well penetrated is the company in the Alagille and PFIC markets? - The company estimates approximately 50% penetration in the Alagille syndrome market, with ongoing efforts to increase awareness and patient identification [59][60]
Mirum Pharmaceuticals (MIRM) Earnings Call Presentation
2025-07-03 08:58
Business Overview - Mirum Pharmaceuticals projects net product sales of $310-320 million for 2024[9, 17] - The company has three approved rare disease products and three additional indications in late-stage development[8, 13] - Mirum's cash balance is $303 million, positioning it to execute its strategy[14] LIVMARLI (maralixibat) - In Alagille Syndrome (ALGS), 93% of participants experienced at least a 1-point reduction in ItchRO[Obs] in cholestatic pruritus[30, 95] - In ALGS, 83% of patients with a >1-point reduction in ItchRO[Obs] remained transplant-free 6 years after starting LIVMARLI[30, 100, 102] - In Progressive Familial Intrahepatic Cholestasis (PFIC), 62% of patients achieved minimal to no itch after 26 weeks of treatment[34, 35, 104] CHENODAL - CHENODAL addresses Cerebrotendinous Xanthomatosis (CTX), with a US prevalence of approximately 1,000-2,000 patients[13, 49] - RESTORE Phase 3 study showed a 20-fold increase in urine 23S-Pentol (bile alcohol) after placebo withdrawal (p<0.0001)[122] - RESTORE Phase 3 study showed a 50-fold increase in plasma 7αC4 after placebo withdrawal (p<0.0001)[122] Volixibat - Primary Sclerosing Cholangitis (PSC) affects approximately 54,000 patients across the US & EU, with ~65% experiencing active pruritus[13, 59] - In a CAMEO study, patients with PSC experienced a 70% mean reduction in pruritus and a 40% mean reduction in bile acids after 14 weeks of treatment with an IBAT inhibitor[61]
Mirum(MIRM) - 2025 Q1 - Earnings Call Transcript
2025-05-07 21:32
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]
Mirum(MIRM) - 2025 Q1 - Earnings Call Transcript
2025-05-07 21:32
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]
Mirum(MIRM) - 2025 Q1 - Earnings Call Transcript
2025-05-07 21:30
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]
Mirum(MIRM) - 2024 Q4 - Earnings Call Transcript
2025-02-27 12:10
Financial Data and Key Metrics Changes - Total net product sales for 2024 were $336.4 million, a 88% increase from $178.9 million in 2023 [24][27] - Fourth quarter 2024 net product sales were $99.4 million, representing a 43% year-over-year increase from $69.5 million [24][25] - The company ended 2024 with cash, cash equivalents, and investments of $293 million, an increase of approximately $7 million from the start of the year [27] Business Line Data and Key Metrics Changes - LIVMARLI's total net product sales for 2024 were $213.3 million, a 50% increase compared to 2023 [25] - Bile acid medicines achieved total net product sales of $123.1 million for 2024, with fourth quarter sales of $35 million, representing approximately 25% growth over the fourth quarter of 2023 [25][26] Market Data and Key Metrics Changes - The company expects to add close to $100 million to its top-line in 2025, with anticipated net product sales between $420 million and $435 million [12][27] - The market for MRM-3379 in Fragile X syndrome is estimated at $1 billion, primarily focusing on the 50,000 male patients in the US [39][40] Company Strategy and Development Direction - The strategic priorities for 2025 include global growth of commercial medicines, advancing the pipeline, selectively pursuing product acquisition opportunities, and maintaining scientific and financial discipline [8][9] - The company aims to expand LIVMARLI's label and drive Volixibat towards pivotal data in adult cholestasis [8][14] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong demand for LIVMARLI in Alagille syndrome and PFIC, anticipating continued growth [10][12] - The company is positioned for sustained growth in the rare disease market, leveraging its financial position for potential acquisitions [33][34] Other Important Information - The FDA approved CTEXLI for cerebrotendinous xanthomatosis (CTX) in adults, which is expected to enhance promotional efforts and patient reach [12][51] - The company reported being cash flow positive in 2024 and expects to maintain this status in 2025 [27] Q&A Session Summary Question: Strategic implications of convertible notes and capital allocation - Management highlighted the unique position of the company in the rare disease market, allowing for potential acquisitions and value creation opportunities [33][34] Question: Market penetration and competition for MRM-3379 - The market potential for MRM-3379 is based on a 50,000-patient market in the US, with substantial upside beyond the initial estimate [39][40][42] Question: Alagille market penetration and growth drivers - Current penetration for eligible treatment population in the US is around 40%, with growth driven by new diagnoses and increased patient starts [46][48] Question: Impact of CTX approval on sales trajectory - Management expects a gradual increase in patient diagnoses and treatment conversions to CTEXLI, impacting sales positively over time [51][52] Question: Patient mix for LIVMARLI in Q4 2024 - Most LIVMARLI patients are currently from Alagille syndrome, but there has been a notable increase in PFIC patient starts [56][58] Question: Commercial team expansion for LIVMARLI - No significant expansion of the commercial team is anticipated, as the same prescribing universe will be leveraged for the potential label expansion [62] Question: Timeline for topline data from Volixibat study - Topline data is expected approximately six months after enrollment completion, which is targeted for the second half of 2025 [84][85] Question: Patient experience in the EXPAND study - Positive treatment outcomes have been observed in biliary atresia patients treated through compassionate use, supporting the need for the EXPAND study [90][91]