Summary of Mirum Pharmaceuticals Inc (MIRM) Conference Call Company Overview - **Company**: Mirum Pharmaceuticals Inc (MIRM) - **Date of Conference**: May 21, 2025 - **Speakers**: Eric Viarholt (CFO), Andrew McKibbin (SVP of Finance and IR) Key Points Commercial Performance - **Record Revenue**: In Q1, LIVMARLI generated approximately $73 million in revenue, with about two-thirds from the U.S. and one-third from international markets [3][4] - **Market Penetration**: The company is nearing 50% penetration in the U.S. for Alagille syndrome, with an addressable population of 1,000 to 1,200 patients [5][6] - **Growth Expectations**: Significant growth opportunities are anticipated for both Alagille syndrome and PFIC (Progressive Familial Intrahepatic Cholestasis) throughout the patent life, which extends to February 2040 [5][6] International Expansion - **Geographic Reach**: LIVMARLI is now available in over 25 countries, with plans for continued expansion, particularly in major Western European countries [6][7] - **Partnerships**: Takeda has recently received approval to launch LIVMARLI in Japan, with expectations for a successful market entry [7] Clinical Studies and New Formulations - **EXPAND Study**: A basket study is underway for additional indications, including cholestatic pruritus, with an estimated 500 addressable patients [9][10] - **Tablet Formulation**: A new tablet formulation has been approved, which is expected to enhance market uptake by providing a more convenient option for older patients transitioning from liquid formulations [11][12] Bile Acid Portfolio - **Revenue Growth**: The bile acid portfolio, including Cetexly and Colban, generated $37 million in Q1, marking a 50% increase compared to the previous quarter [18][19] - **Patient Diagnosis**: Currently, only about 10% of potential patients for CTX (Cholestanol Storage Disease) are diagnosed, indicating a significant opportunity for patient finding efforts [19] Pruritus in PSC and PBC - **Market Opportunity**: There are approximately 30,000 patients in the U.S. with PSC (Primary Sclerosing Cholangitis), with pruritus being a major issue for these patients [21][22] - **Treatment Landscape**: Current treatments for pruritus are largely ineffective, creating a substantial opportunity for IBAT inhibitors like LIVMARLI [23][24] - **Clinical Data**: Over half of the patients in previous studies using LIVMARLI for PSC reported resolution of itching, indicating strong efficacy [25] Regulatory Pathway - **Approval Strategy**: The company is optimistic about the regulatory pathway for pruritus in PSC, as it is considered an outcome measure that can lead to full approval [33][34] Intellectual Property - **Patent Protection**: LIVMARLI has IP protection until 2040, with the new tablet formulation extending IP to 2043 [48][49] Competitive Landscape - **Dosing Optimization**: The company emphasizes the importance of dosing in achieving effective treatment outcomes, particularly in comparison to competitors [40][41] Additional Insights - **Durability of Effects**: Long-term data indicates consistent and durable responses in patients treated with LIVMARLI, with improvements in both pruritus and fatigue [36][37] - **Payer Landscape**: The payer environment is described as receptive, particularly for treatments that provide tangible benefits to patients [42][43] This summary encapsulates the key insights and developments discussed during the conference call, highlighting Mirum Pharmaceuticals' strategic direction, market opportunities, and clinical advancements.

Core Insights - Mirum Pharmaceuticals reported a narrower loss of 30 cents per share in Q1 2025, compared to a loss of 54 cents per share in the same quarter last year, and better than the Zacks Consensus Estimate of a loss of 39 cents [1][2] - The company's revenues for Q1 2025 reached $111.6 million, reflecting a year-over-year increase of nearly 62%, surpassing the Zacks Consensus Estimate of $98 million [2][3] Financial Performance - Livmarli's net product sales were $73.2 million in Q1 2025, marking a 71% increase year over year, with U.S. sales at $49.5 million and ex-U.S. sales at $23.7 million [6] - Net product sales from bile acid products, including Cholbam and Ctexli, totaled $38.4 million in Q1 2025, up 47% year over year [7] - Research and development expenses rose by 42.8% year over year to $46 million, while selling, general, and administrative expenses increased by 26.5% to $57.7 million [7][8] Guidance and Future Outlook - Mirum raised its full-year revenue guidance for 2025 to a range of $435-$450 million, up from the previous estimate of $420-$435 million, due to strong demand for its commercial products [9] - The company is evaluating Livmarli in a phase III study for treating pruritus in rare cholestatic conditions, with enrollment expected to complete in 2026 [10] Recent Developments - The FDA approved a new tablet formulation of Livmarli for treating cholestatic pruritus in patients with Alagille syndrome and progressive familial intrahepatic cholestasis [10] - Ctexli tablets received FDA approval for treating adults with cerebrotendinous xanthomatosis, becoming the first approved treatment for this condition [11] - Mirum's lead pipeline candidate, volixibat, is in two phase IIb studies for primary biliary cholangitis and primary sclerosing cholangitis, with enrollment expected to complete in 2026 [11][12]

分组1 - Mirum Pharmaceuticals reported a quarterly loss of $0.30 per share, better than the Zacks Consensus Estimate of a loss of $0.39, and improved from a loss of $0.54 per share a year ago, representing an earnings surprise of 23.08% [1] - The company posted revenues of $111.59 million for the quarter ended March 2025, surpassing the Zacks Consensus Estimate by 13.32%, and compared to year-ago revenues of $69.22 million [2] - Mirum Pharmaceuticals has surpassed consensus EPS estimates two times over the last four quarters and topped consensus revenue estimates four times during the same period [2] 分组2 - The stock has lost about 0.9% since the beginning of the year, while the S&P 500 has declined by 4.7% [3] - The current consensus EPS estimate for the coming quarter is -$0.35 on revenues of $102.78 million, and for the current fiscal year, it is -$1.23 on revenues of $427.82 million [7] - The Medical - Biomedical and Genetics industry is currently in the top 35% of Zacks industries, indicating a favorable outlook for stocks within this sector [8]

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]

Financial Performance - For the three months ended March 31, 2025, the company reported a net loss of $14.7 million, compared to a net loss of $25.3 million for the same period in 2024, indicating a 42% improvement in net loss year-over-year[113]. - Total revenue for the three months ended March 31, 2025, was $111.6 million, an increase of 61.2% compared to $69.2 million for the same period in 2024[135]. - Product sales, net increased to $111.6 million in Q1 2025 from $68.9 million in Q1 2024, driven by the commercialization of Livmarli and Bile Acid Medicines[136]. - The net loss for the three months ended March 31, 2025, was $14.7 million, a decrease of 42% compared to a net loss of $25.3 million in the same period in 2024[135]. - Interest income decreased to $3.0 million in Q1 2025 from $3.6 million in Q1 2024, primarily due to lower yields on investments[141]. Cash and Deficit - As of March 31, 2025, the company had an accumulated deficit of $658.9 million, up from $644.2 million as of December 31, 2024[113]. - The company had unrestricted cash, cash equivalents, and investments of $298.6 million as of March 31, 2025, compared to $292.8 million as of December 31, 2024[113]. - The accumulated deficit increased to $658.9 million as of March 31, 2025, compared to $644.2 million as of December 31, 2024[143]. - Net cash used in operating activities was $2.0 million for the three months ended March 31, 2025, reflecting a net loss of $14.7 million offset by adjustments of $24.0 million[162]. - Net cash provided by operating activities was $15.2 million for the three months ended March 31, 2024, reflecting a net loss of $25.3 million partially offset by adjustments of $16.5 million[163]. - Cash used in operating activities reflected changes in net operating assets of $11.3 million, primarily related to an increase in accounts receivable[162]. - Cash provided by operating activities reflected changes in net operating assets of $23.9 million, consisting primarily of a decrease in accounts receivables[163]. - The company maintains significant amounts of cash and cash equivalents at one financial institution that is in excess of federally insured limits[166]. Expenses - Research and development expenses rose to $46.0 million in Q1 2025, up 42.9% from $32.2 million in Q1 2024, primarily due to increased costs associated with product development and personnel[139]. - Selling, general and administrative expenses increased to $57.7 million in Q1 2025, a rise of 26.5% from $45.6 million in Q1 2024, mainly due to higher personnel costs and marketing expenses[140]. - The company anticipates an increase in research and development expenses as it continues to develop its product candidates, including volixibat and MRM-3379[123]. - Selling, general, and administrative expenses are expected to rise to support commercialization efforts and increased operational costs as a global biopharmaceutical public company[126]. Future Outlook - The company expects total product sales of its approved medicines to continue to increase annually, although revenue may experience fluctuations due to large periodic orders from partners[114][117]. - The company anticipates continued net losses as it invests in research, development, and commercialization activities for its product candidates[148]. - The company expects to satisfy future cash needs through existing capital, revenue from approved medicines, and potential equity offerings or collaborations[115]. - The company has entered into a Sales Agreement allowing for the potential sale of up to $200.0 million of common stock through an "at-the-market" offering[146]. Regulatory and Development Updates - The FDA granted orphan exclusivity for chenodiol for the treatment of cerebrotendinous xanthomatosis (CTX), with FDA approval received in February 2025 for commercialization under the brand name Ctexli[119]. - The company completed the acquisition of assets from Travere Therapeutics, primarily related to the development and commercialization of bile acid medicines, on August 31, 2023[108]. - The company initiated the Phase 3 EXPAND study for Livmarli in the fourth quarter of 2024, targeting rare cholestatic conditions[111]. Market Risk - The primary exposure to market risk is interest income sensitivity, which is affected by changes in the general level of U.S. interest rates[166]. - A near-term 10% fluctuation of the USD exchange rate could result in a potential change in the fair value of net assets and liabilities denominated in foreign currency by approximately $1.2 million[169]. - The company has outstanding $316.2 million aggregate principal of the Notes as of March 31, 2025, with a fair value of approximately $517.7 million[167].

Financial Performance - Total revenue for Q1 2025 was $111.6 million, compared to $69.2 million in Q1 2024, representing a 61% increase[5] - Total revenue for Q1 2025 was $111.585 million, a significant increase of 60.8% compared to $69.222 million in Q1 2024[27] - Product sales, net, reached $111.585 million in Q1 2025, while license and other revenue was negligible at $0 million compared to $305 thousand in Q1 2024[27] - The net loss for Q1 2025 was $14.677 million, a reduction of 42.5% from a net loss of $25.279 million in Q1 2024[27] - Basic and diluted net loss per share improved to $(0.30) in Q1 2025 from $(0.54) in Q1 2024[27] Revenue Sources - LIVMARLI net product sales for Q1 2025 were $73.2 million, reflecting a 71% growth over Q1 2024[5] - Bile Acid Medicines net product sales reached $38.4 million, indicating a 47% increase compared to Q1 2024[5] Guidance and Projections - The company raised its full-year revenue guidance to between $435 million and $450 million[3] - The VISTAS study in primary sclerosing cholangitis (PSC) is expected to complete enrollment in Q3 2025, with topline data anticipated in Q2 2026[1] Product Developments - LIVMARLI oral tablet formulation has received FDA approval, providing a convenient single tablet dose option[2] - LIVMARLI has been approved in Japan for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC)[5] - The company plans to initiate a Phase 2 study for MRM-3379 in Fragile X Syndrome in 2025[5] Expenses and Financial Position - Total operating expenses for Q1 2025 were $126.8 million, compared to $95.7 million in Q1 2024[5] - Total operating expenses increased to $126.768 million in Q1 2025, up from $95.690 million in Q1 2024, reflecting a rise of 32.4%[27] - Research and development expenses increased to $46.044 million in Q1 2025, compared to $32.222 million in Q1 2024, marking a rise of 43.0%[27] - Total stock-based compensation expenses were $15.803 million in Q1 2025, compared to $11.450 million in Q1 2024, indicating a 38.2% increase[27] Cash and Assets - As of March 31, 2025, unrestricted cash, cash equivalents, and investments totaled $298.6 million, up from $292.8 million at the end of 2024[5] - Cash and cash equivalents decreased to $211.822 million as of March 31, 2025, down from $222.503 million at the end of 2024[29] - Total assets increased to $690.245 million as of March 31, 2025, compared to $670.754 million at the end of 2024, reflecting a growth of 2.3%[29] - Total stockholders' equity rose to $233.256 million as of March 31, 2025, up from $225.640 million at the end of 2024[29]

Group 1 - The article discusses the performance of Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM), noting that despite a previous "Sell" rating given in October, the stock price has increased significantly over the past six months [2] - The investing group Haggerston BioHealth provides insights for both novice and experienced biotech investors, including catalysts to watch, buy and sell ratings, and forecasts for major pharmaceutical companies [2] - The article emphasizes the importance of detailed financial models and market analysis in making informed investment decisions within the biotech sector [2] Group 2 - The author, Edmund Ingham, has over five years of experience in covering biotech, healthcare, and pharma, having compiled detailed reports on more than 1,000 companies [2] - The article does not contain any personal investment positions or plans to initiate such positions in the companies mentioned, indicating an objective analysis [3] - Seeking Alpha clarifies that past performance is not indicative of future results and that the views expressed may not represent the platform as a whole [4]

Core Insights - Mirum Pharmaceuticals' lead product, Livmarli, is approved for treating cholestatic pruritus in patients with Alagille syndrome and certain patients with progressive familial intrahepatic cholestasis [1][2] - Livmarli's sales reached $213.3 million in 2024, marking a 50.4% year-over-year increase, although the company's heavy reliance on this drug raises concerns [2] - Mirum's stock has surged 83.2% over the past year, contrasting with an 8.5% decline in the industry [2] Product and Pipeline Developments - Livmarli is currently being evaluated in the phase III EXPAND study for treating pruritus in rare cholestatic conditions, with enrollment expected to complete in 2026 [3] - Mirum acquired Travere Therapeutics' bile acid products in August 2023, adding Cholbam and chenodiol tablets to its portfolio [3][4] - Ctexli (chenodiol) tablets were approved by the FDA in February 2025 for treating cerebrotendinous xanthomatosis, becoming the first approved treatment for this condition [4] Financial Performance and Concerns - Despite the growth from bile acid products, the company remains heavily dependent on Livmarli for revenue, which poses risks if sales do not meet expectations [4][9] - Disappointing results from the phase IIb EMBARK study for biliary atresia led to the discontinuation of the clinical study, highlighting potential pipeline setbacks [10] Market Position - Mirum currently holds a Zacks Rank 4 (Sell), indicating a less favorable market position compared to other biotech stocks like Gilead Sciences, Dynavax Technologies, and Arvinas, which have better rankings [11]