Core Insights - Mirum Pharmaceuticals has initiated the BLOOM Phase 2 clinical study for MRM-3379 targeting Fragile X syndrome (FXS) [1] - FXS is the most common inherited form of intellectual disability and autism spectrum disorder, affecting approximately 50,000 males in the U.S. and E.U. [1] - Currently, there are no approved therapies for Fragile X syndrome [1]

Group 1 - Biotech stocks are prominent on the IBD 50 list, with Stoke Therapeutics (STOK) and Mirum Pharmaceuticals (MIRM) either in or forming bases [1] - A specific biotech stock has shown a sell signal after a breakout that failed to progress [1] - Biotech companies have significantly rebounded from April lows, indicating a positive trend in the sector [1] Group 2 - Novo Nordisk's stock increased due to a lighter-than-expected discount for Ozempic and Wegovy under the Inflation Reduction Act, with a price cut of 71% for Ozempic [2]

Mirum Pharmaceuticals Conference Call Summary Company Overview - **Company**: Mirum Pharmaceuticals (NasdaqGM:MIRM) - **Focus**: Commercial rare disease company with three approved products targeting ultra-rare genetic conditions - **Recent Performance**: Reported $133 million in top-line revenue for Q3 2025, achieving positive net income for the first time [3][5] Key Highlights Financial Performance - Achieved $133 million in revenue for Q3 2025, marking a significant milestone in financial performance [3] - Positive net income reported for the first time, indicating an efficient business model in rare disease settings [5] Product Pipeline and Upcoming Milestones - **Velexibat**: Expected top-line data from the PSC program in Q2 2026, with additional studies in PBC and label expansion for LIVMARLI [4] - **LIVMARLI**: Strong performance in Alagille syndrome with approximately 50% market penetration in the U.S. [8] - **PFIC Launch**: Exceeded expectations with strong initial uptake driven by product profile and patient identification through genetic testing [20][22] Market Dynamics - **Alagille Syndrome**: LIVMARLI shows high response rates in pruritus and serum bile acid reduction, leading to long-term patient retention [8][9] - **PFIC Market**: Early signs of growth attributed to improved patient identification and awareness of genetic testing, expanding the potential patient pool [22][25] - **Competitive Landscape**: Mirum holds approximately 80% market share in Alagille syndrome and about 50% in PFIC, with a focus on expanding the total market [10][27] Product Formulation and Compliance - Introduction of a tablet formulation for LIVMARLI has improved patient compliance, particularly among older patients [12][13] Pricing Dynamics - Gradual growth in pricing expected as more patients are treated, with dose adjustments contributing to revenue over time [15][19] Future Growth Potential - Long-term view for LIVMARLI projected at over $1 billion, with Alagille syndrome contributing approximately 40% and PFIC around 30% [28] Additional Insights - **Genetic Testing**: Increasing adoption of genetic testing is crucial for diagnosing hard-to-identify patients, particularly in adult hepatology settings [7][22] - **Market Development**: Efforts are underway to enhance physician awareness of pruritus in PSC patients, which is often under-discussed [47] - **EXPAND Study**: Aiming to address ultra-rare cholestatic pruritus, with a significant number of patients fitting the profile identified [31][33] Conclusion Mirum Pharmaceuticals is positioned for continued growth with a strong product pipeline, effective market strategies, and a focus on expanding patient access through genetic testing and improved formulations. The company is optimistic about its future prospects, particularly with LIVMARLI and velexibat, as it navigates the competitive landscape in rare diseases.

Core Insights - Mirum Pharma demonstrates leadership in liver disease innovation by presenting data from its LIVMARLI® and Volixibat clinical programs at AASLD's The Liver Meeting® [1] Group 1 - Mirum Pharma showcases its advancements in liver disease treatment through clinical data [1] - The presentation at AASLD's The Liver Meeting® highlights the company's commitment to addressing liver diseases [1]

Core Insights - Mirum Pharmaceuticals reported earnings of 5 cents per share for Q3 2025, surpassing the Zacks Consensus Estimate of a loss of 10 cents, and improved from a loss of 30 cents per share in the same quarter last year [1][8] - Revenues for Q3 reached $133 million, a year-over-year increase of 47.1%, exceeding the Zacks Consensus Estimate of $132 million, driven by strong sales of Livmarli and bile acid products [2][8] Financial Performance - Livmarli's net product sales were $92 million in Q3, reflecting a 56% increase year over year, with U.S. sales at $64 million and international sales at $28 million [6][8] - Net product sales of bile acid products, including Cholbam and Ctexli, totaled $40.8 million in Q3, marking a 31% year-over-year increase [9] - Research and development expenses rose by 35.6% year over year to $43 million, while selling, general, and administrative expenses increased by 22.6% to $61.9 million [9] Guidance and Outlook - The company tightened its 2025 revenue guidance to a range of $500-$510 million, up from the previous range of $490-$510 million, with the Zacks Consensus Estimate currently at $508.9 million [11] Product Developments - Livmarli is approved for treating cholestatic pruritus in patients with Alagille syndrome and certain patients with progressive familial intrahepatic cholestasis [3] - A new tablet formulation of Livmarli was launched in the U.S. in June 2025, aimed at providing convenience for older patients [4] - Mirum acquired Travere Therapeutics' bile acid products in August 2023, enhancing its portfolio with Cholbam and Ctexli [4] Pipeline Updates - Mirum is conducting a phase III EXPAND study for Livmarli in rare cholestatic conditions, with enrollment expected to complete in 2026 [12] - The lead pipeline candidate, volixibat, is under evaluation in two phase IIb studies for primary biliary cholangitis and primary sclerosing cholangitis [12][13] - A phase II study on the newly in-licensed PDE4D inhibitor, MRM-3379, for Fragile X syndrome has been initiated [13] Market Performance - Mirum's shares have increased by 71.7% year to date, significantly outperforming the industry average rise of 10.9% [5]

Financial Performance - Mirum Pharmaceuticals reported quarterly earnings of $0.05 per share, exceeding the Zacks Consensus Estimate of a loss of $0.10 per share, and showing improvement from a loss of $0.30 per share a year ago, resulting in an earnings surprise of +150.00% [1] - The company achieved revenues of $133.01 million for the quarter ended September 2025, surpassing the Zacks Consensus Estimate by 0.85% and increasing from $90.38 million in the same quarter last year [2] Stock Performance - Mirum Pharmaceuticals shares have increased approximately 71.3% since the beginning of the year, significantly outperforming the S&P 500's gain of 16.5% [3] - The stock currently holds a Zacks Rank 2 (Buy), indicating expectations for it to outperform the market in the near future [6] Future Outlook - The current consensus EPS estimate for the upcoming quarter is -$0.15 on revenues of $137.68 million, and for the current fiscal year, it is -$0.67 on revenues of $508.94 million [7] - The Medical - Biomedical and Genetics industry, to which Mirum Pharmaceuticals belongs, is currently ranked in the top 39% of over 250 Zacks industries, suggesting a favorable outlook for the sector [8]

Financial Data and Key Metrics Changes - The company reported third quarter revenue of $133 million, representing a nearly 50% year-over-year increase [5][16] - Total net product revenue increased by 47% compared to the prior year, reflecting growth across all commercial medicines [16] - The company achieved positive net income for the first time, generating approximately $3 million in net income [7][17] Business Line Data and Key Metrics Changes - LIVMARLI net product sales totaled $92 million for the quarter, with $64 million in the U.S. and $28 million internationally [9][10] - The bioacquired portfolio, including CHOLBAM and CHENODAL, generated $41 million in net product sales this quarter [10] - The company expects to land in the upper end of its prior full year 2025 guidance range, with projected revenues of $500-$510 million [10] Market Data and Key Metrics Changes - Demand for LIVMARLI in the U.S. remains healthy, particularly in Alagille syndrome and PFIC, with continued growth expected [9] - Internationally, LIVMARLI demand continues to grow, supported by expanding reimbursement and launches in new geographies [10] - The first full quarter of commercialization for Takeda in Japan showed in-market adoption dynamics consistent with LIVMARLI's U.S. launch [10] Company Strategy and Development Direction - The company aims to create and deliver life-changing medicines to patients, focusing on rare diseases [5] - There is a strong emphasis on expanding the commercial portfolio and advancing the clinical pipeline, with three potentially pivotal readouts expected over the next 18 months [6][7] - The company is optimistic about the peak revenue potential for LIVMARLI, Volixibat, and MRM-3379, each exceeding $1 billion [11] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's ability to continue executing with focus and consistency, highlighting the importance of patient-centric approaches [5][18] - The company remains well-funded and financially independent, providing resources for expansion and growth [17] - Management noted that while quarterly GAAP profitability is a milestone, it is not yet a consistent expectation as the company continues to invest in growth [17] Other Important Information - The company completed enrollment in the phase 2b VISTAS study in primary sclerosing cholangitis (PSC) and expects to announce top-line data in the second quarter of 2026 [12] - The company is actively monitoring the competitive landscape, particularly regarding pricing strategies for Volixibat [28][68] Q&A Session Summary Question: Key drivers of LIVMARLI's performance in 2026 and guidance for Q4 - Management expects continued growth from the PFIC launch and other existing dynamics, with guidance for Q4 reflecting variability in international sales [20][22] Question: Trends in adoption of LIVMARLI's solid tablet formulation - The solid tablet formulation has seen encouraging uptake, with a substantial proportion of eligible patients switching from the liquid form [26][27] Question: Expectations for Paragraph IV filers and IP portfolio confidence - Management expressed confidence in the overall IP position, particularly regarding method patents for LIVMARLI [32][34] Question: Insights on Fragile X study endpoints and safety risks in PSC - Management is optimistic about the endpoint strategy for Fragile X and noted no significant safety issues in the PSC study [40][42] Question: Impact of government shutdown on genetic screening programs - No impact has been observed on genetic screening programs or business interactions due to the government shutdown [56] Question: Pace of new PFIC adds and business development strategy - The pace of new PFIC adds remains healthy, with ongoing educational efforts to increase awareness among providers [60][63]

Financial Data and Key Metrics Changes - The company reported third quarter revenue of $133 million, representing a nearly 50% year-over-year increase [4][16] - Total net product revenue increased by 47% compared to the prior year, reflecting growth across all commercial medicines [16] - The company achieved positive net income for the first time, generating approximately $3 million in net income [5][17] Business Line Data and Key Metrics Changes - LIVMARLI net product sales totaled $92 million for the quarter, with $64 million from the US and $28 million from international markets [7][9] - The bioacquired medicines, CHOLBAM and CHENODAL, generated $41 million in net product sales this quarter [9] - The company expects to land in the upper end of its prior full year 2025 guidance range, with projected revenues of $500-$510 million [9] Market Data and Key Metrics Changes - Demand for LIVMARLI in the US remains healthy, particularly in Alagille syndrome and PFIC, supported by expanded diagnosis and increased genetic screening [8] - Internationally, LIVMARLI demand continues to grow, with significant contributions from new geographies and expanding reimbursement [9] - The company noted variability in international partner and distributor ordering patterns affecting revenue projections [9] Company Strategy and Development Direction - The company aims to advance its clinical pipeline with three potentially pivotal readouts expected over the next 18 months [5] - The focus remains on building a high-growth, cash flow positive rare disease company with a broad pipeline and global footprint [6] - The company is actively looking for underappreciated programs to add to its pipeline, maintaining a high bar for new product acquisitions [50] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the continued growth potential across the portfolio, with peak revenue potential for LIVMARLI, Volixibat, and MRM-3379 each exceeding $1 billion [10] - The company highlighted the importance of sustained engagement with the medical community to drive incremental gains in genetic testing and diagnosis [8] - Management emphasized the strength and scalability of the business model, viewing quarterly GAAP profitability as a milestone rather than a consistent expectation [17] Other Important Information - The company completed enrollment in the phase 2b VISTAS study in primary sclerosing cholangitis (PSC) and expects to announce top-line data in the second quarter of 2026 [11] - The VANTAGE study continues to progress well, with interim data showing statistically significant improvements in pruritus and other key measures [12] - The company initiated a phase 2 study of MRM-3379 for Fragile X syndrome, with promising preclinical data supporting its efficacy [13] Q&A Session Summary Question: Key drivers of LIVMARLI's performance in 2026 and guidance for Q4 revenue - Management expects continued growth from the PFIC launch and noted that international revenue may show variability due to large distributor orders [20][22] Question: Trends in adoption of LIVMARLI's solid tablet formulation and pricing for Volixibat - The solid tablet formulation has seen encouraging uptake, with a substantial proportion of eligible patients switching from the liquid form [25][26] - Pricing for Volixibat is still under analysis, with comparisons to other PPARs in the market [26] Question: Expectations for Paragraph IV filers and confidence in IP portfolio - Management expressed confidence in the overall IP position, particularly regarding method patents for LIVMARLI [30] Question: Insights on Fragile X study endpoints and safety risks in PSC - Management is optimistic about the endpoint strategy for Fragile X and noted no significant safety issues in the PSC study [33][37] Question: Impact of government shutdown on genetic screening programs - No impact has been observed on genetic screening programs or business interactions due to the government shutdown [46] Question: Pace of new PFIC adds and business development strategy - The pace of new PFIC adds remains healthy, with ongoing educational efforts to increase awareness among adult providers [48] - The company continues to seek underappreciated programs for its pipeline, maintaining a high standard for new acquisitions [50] Question: Competitive dynamics in PBC with recent market entrants - Management highlighted the unique positioning of Volixibat in the PBC treatment landscape, emphasizing its potential in both first and second-line settings [52]

Financial Performance - Net income for the three months ended September 30, 2025, was $2.9 million, compared to a net loss of $14.2 million for the same period in 2024[112]. - The company reported a net income of $2.9 million for the three months ended September 30, 2025, compared to a net loss of $14.2 million in 2024, indicating a significant turnaround[135]. - Total revenue for the three months ended September 30, 2025, was $133.0 million, compared to $90.4 million in 2024, marking a 47.2% increase[135]. - For the nine months ended September 30, 2025, net product sales were $372.4 million, a 57.0% increase from $237.0 million in the same period of 2024[146][147]. - Livmarli sales for the nine months ended September 30, 2025, were $253.6 million, up from $149.2 million in 2024, representing a 69.9% increase[148]. Cash and Investments - Unrestricted cash, cash equivalents, and investments increased to $378.0 million as of September 30, 2025, from $292.8 million as of December 31, 2024[112]. - As of September 30, 2025, the company had $378.0 million in unrestricted cash, cash equivalents, and investments, up from $292.8 million as of December 31, 2024[155]. - Net cash provided by operating activities was $49.8 million for the nine months ended September 30, 2025, compared to $15.4 million for the same period in 2024[171][174]. - Net cash used in investing activities was $25.0 million for the nine months ended September 30, 2025, significantly lower than $90.8 million for the same period in 2024[175][176]. - Net cash provided by financing activities was $31.1 million for the nine months ended September 30, 2025, compared to $12.4 million for the same period in 2024[177]. Expenses - Research and development expenses for the three months ended September 30, 2025, were $43.0 million, an increase of 35.4% from $31.7 million in 2024[140]. - Selling, general and administrative expenses rose to $61.9 million for the three months ended September 30, 2025, up 22.5% from $50.5 million in 2024[141]. - Selling, general and administrative expenses increased by $37.5 million to $182.9 million for the nine months ended September 30, 2025, primarily due to a $24.5 million rise in personnel and compensation-related expenses[151]. - Total operating expenses for the nine months ended September 30, 2025, were $389.9 million, compared to $300.9 million in 2024, reflecting a 29.0% increase[146]. Accumulated Deficit - The accumulated deficit as of September 30, 2025, was $661.8 million, up from $644.2 million as of December 31, 2024[112]. - The accumulated deficit increased to $661.8 million as of September 30, 2025, compared to $644.2 million as of December 31, 2024[155]. Product Development and Sales - The company expects total product sales of its approved medicines to continue increasing annually, despite potential fluctuations due to large periodic orders[113]. - The increase in net product sales was primarily driven by the continued commercialization of Livmarli, which generated $92.2 million in sales, up from $59.1 million in 2024, reflecting a 57.0% increase[136][137]. - The NDA for chenodiol for the treatment of cerebrotendinous xanthomatosis (CTX) was submitted in 2024, with FDA approval received in February 2025[108]. - The FDA granted Breakthrough Therapy Designation for volixibat as a potential treatment for cholestatic pruritus in patients with primary biliary cholangitis (PBC) in October 2024[110]. - The company completed the acquisition of assets from Travere Therapeutics, primarily related to the development and commercialization of chenodiol and Cholbam, on August 31, 2023[107]. - The company has entered into a license agreement for MRM-3379, an allosteric inhibitor of PDE4D, aimed at treating Fragile-X Syndrome[106]. Future Outlook - Research and development expenses are expected to increase as the company continues to develop volixibat and execute the EXPAND study for Livmarli[123]. - Selling, general and administrative expenses are anticipated to rise to support commercialization efforts and increased operational costs as a public company[125]. - The company anticipates continued net losses in the foreseeable future due to ongoing research and development efforts and commercialization activities[159]. - The company has $316.2 million in aggregate principal of Notes outstanding as of September 30, 2025, with a fixed interest rate[179]. - A near-term 10% fluctuation in the USD exchange rate could result in a potential change in the fair value of foreign currency-denominated net assets and liabilities by approximately $2.6 million[181].

Revenue Performance - Third quarter 2025 total revenue was $133 million, compared to $90.4 million for the same quarter in 2024, representing a 47% year-over-year increase[4] - LIVMARLI net product sales for the third quarter 2025 were $92.2 million, reflecting a 56% growth over the third quarter 2024[4] - Bile Acid Medicines net product sales for the third quarter 2025 were $40.8 million, indicating a 31% increase compared to the same period in 2024[4] - Total revenue for the three months ended September 30, 2025, was $133.01 million, a 47.2% increase from $90.38 million in the same period of 2024[26] - Product sales, net for the nine months ended September 30, 2025, reached $372.38 million, compared to $236.98 million in 2024, reflecting a 57.1% growth[26] Financial Guidance and Expenses - The updated revenue guidance for 2025 is between $500 million and $510 million[1] - Total operating expenses for the third quarter 2025 were $130.4 million, up from $103.1 million in the same quarter of 2024[4] - Research and development expenses for the three months ended September 30, 2025, were $42.96 million, up 35.4% from $31.71 million in 2024[26] - The company incurred total stock-based compensation expenses of $18.13 million for the three months ended September 30, 2025, compared to $11.91 million in the same period of 2024, a 52.5% increase[26] Income and Assets - Net income for the three months ended September 30, 2025, was $2.91 million, compared to a net loss of $14.24 million in the same period of 2024[26] - Total current assets increased to $530.58 million as of September 30, 2025, from $392.79 million at the end of 2024, representing a 35.1% increase[28] - Cash and cash equivalents rose to $282.02 million as of September 30, 2025, compared to $222.50 million at the end of 2024, a 26.8% increase[28] - Total stockholders' equity increased to $292.05 million as of September 30, 2025, from $225.64 million at the end of 2024, reflecting a 29.3% growth[28] - Total liabilities as of September 30, 2025, were $493.07 million, up from $445.11 million at the end of 2024, indicating an increase of 10.8%[28] - The company reported a basic net income per share of $0.06 for the three months ended September 30, 2025, compared to a loss of $0.30 per share in the same period of 2024[26] Clinical Studies and Trials - Enrollment in the Volixibat VISTAS study for primary sclerosing cholangitis (PSC) is complete, with topline data expected in the second quarter of 2026[4] - The Phase 2 study for MRM-3379 in Fragile X Syndrome has been initiated[4] - The Volixibat VANTAGE study in primary biliary cholangitis (PBC) is expected to complete enrollment in 2026, with topline data anticipated in the first half of 2027[4] - LIVMARLI EXPAND Phase 3 study for pruritus in rare cholestatic conditions is expected to complete enrollment in 2026, with topline data expected in the first half of 2027[4]