Mirum Pharmaceuticals Inc (MIRM) Q1 2025 Earnings Call May 07, 2025 04:30 PM ET Company Participants Andrew McKibben - Vice President of Investor Relations and FinanceChris Peetz - President and Chief Executive OfficerPeter Radovich - COO & PresidentEric Bjerkholt - Chief Financial OfficerSelena Zhang - Global capital marketsRyan Mcelroy - Equity Research AssociateRyan Deschner - Vice President - Equity Research Conference Call Participants Gavin Clark-Gartner - AnalystJessica Fye - Managing Director & Equi ...

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]

Financial Data and Key Metrics Changes - Total net product sales for 2024 reached $336.4 million, a significant increase from $178.9 million in 2023, representing an 88% year-over-year growth [24][27] - In Q4 2024, total net product sales were $99.4 million, compared to $69.5 million in Q4 2023, marking a 43% increase [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 generated total net product sales of $213 million in 2024, a 50% increase compared to 2023 [10][25] - The bile acid medicine portfolio achieved total net product sales of $123 million for 2024, with Q4 sales of $35 million, reflecting approximately 25% growth over Q4 2023 [11][25] Market Data and Key Metrics Changes - The company anticipates net product sales between $420 million and $435 million for 2025, indicating continued growth [12][27] - The market for MRM-3379 in Fragile X syndrome is estimated to be a $1 billion opportunity, primarily focusing on the 50,000 male patients in the US [39][40] Company Strategy and Development Direction - The strategic priorities for 2025 include expanding the global growth of commercial medicines, advancing the pipeline, selectively pursuing product acquisitions, and maintaining scientific and financial discipline [8][9] - The company aims to leverage its financial position to acquire rare disease products that present significant value-creation opportunities [33] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong demand for LIVMARLI in Alagille syndrome and PFIC, expecting continued growth [10][12] - The approval of CTEXLI for CTX is seen as a significant opportunity to reach more patients and enhance sales [12][51] Other Important Information - The company achieved positive interim results for Volixibat in both VISTAS PSC and VANTAGE PBC studies, leading to breakthrough designation in PBC [14][21] - The Phase 3 EXPAND study for LIVMARLI is on track for completion in 2026, with expectations for significant growth opportunities [21][22] Q&A Session Summary Question: Strategic question regarding capital allocation and business development - Management indicated that the strong earnings performance and stock trading above conversion price provide flexibility for business development opportunities [32][33] Question: Market penetration and competition for MRM-3379 - Management discussed the $1 billion revenue potential based on a 50,000 patient market in the US, with potential for other indications [38][39][40] Question: Alagille market penetration - Management estimated that approximately 40% of eligible Alagille syndrome patients are currently treated, indicating significant growth potential [46][47] Question: Impact of CTX approval on sales trajectory - Management expects a gradual increase in sales as diagnosed patients transition to CTEXLI [51][52] Question: Patient mix for LIVMARLI - Management confirmed that most LIVMARLI patients are Alagille syndrome patients, with increasing contributions from PFIC patient starts [56][58] Question: Commercial team expansion for LIVMARLI - Management stated that no significant expansion of the commercial team is anticipated, as the same prescribing universe will be leveraged [62] Question: Enrollment timeline for VANTAGE study - Management indicated that topline data from the VANTAGE study is expected approximately six months after enrollment completion [84][85] Question: Patient experience in EXPAND study - Management shared positive outcomes from compassionate use cases for biliary atresia, reinforcing confidence in the EXPAND study [90][91]

Core Insights - Mirum Pharmaceuticals reported a wider loss of 49 cents per share in Q4 2024, compared to the Zacks Consensus Estimate of a loss of 27 cents, and a loss of 66 cents per share in the same quarter last year [1] - The company's revenues for Q4 totaled $99.4 million, reflecting a year-over-year increase of 42.8%, surpassing the Zacks Consensus Estimate of $97 million [2] - Mirum's stock has increased by 70.3% over the past year, while the industry has declined by 9.5% [3] Financial Performance - Research and development expenses rose by 42.4% year-over-year to $44 million, while selling, general, and administrative expenses increased by 22.9% to $56.8 million [4] - As of December 31, 2024, the company had cash, cash equivalents, and marketable securities totaling $292.8 million, slightly down from $293.8 million as of September 30, 2024 [5] - For the full year 2024, Mirum generated revenues of $336.9 million, up from $186.4 million in the previous year, and reported a loss of $1.85 per share, an improvement from a loss of $4.00 per share in the prior year [8] Future Outlook - Mirum anticipates worldwide net product sales of approximately $420-$435 million and expects to achieve positive cash flow in 2025 [9] - The company is conducting a phase III study for Livmarli and has received FDA approval for Ctexli, the first treatment for cerebrotendinous xanthomatosis [10] - Mirum's lead pipeline candidate, volixibat, is under evaluation in two phase IIb studies, with enrollment expected to be completed in 2026 [11] - A phase II study for the PDE4D inhibitor, MRM-3379, is planned for Fragile X syndrome later in 2025 [12]

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]

分组1 - Mirum Pharmaceuticals reported a quarterly loss of $0.49 per share, which was worse than the Zacks Consensus Estimate of a loss of $0.27, representing an earnings surprise of -81.48% [1] - The company posted revenues of $99.41 million for the quarter ended December 2024, exceeding the Zacks Consensus Estimate by 2.40%, and showing a year-over-year increase from $69.55 million [2] - Over the last four quarters, Mirum Pharmaceuticals has surpassed consensus revenue estimates three times [2] 分组2 - The stock has gained approximately 20.8% since the beginning of the year, outperforming the S&P 500's gain of 1.3% [3] - The current consensus EPS estimate for the upcoming quarter is -$0.21 on revenues of $98.79 million, and for the current fiscal year, it is -$0.70 on revenues of $436.29 million [7] - The Medical - Biomedical and Genetics industry, to which Mirum Pharmaceuticals belongs, is currently ranked in the top 24% of over 250 Zacks industries, indicating a favorable outlook for the sector [8]