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]

Financial Performance and Guidance - Mirum Pharmaceuticals expects net product sales of $500-510 million in 2025[7, 100] - The company had a strong financial position with $378 million in cash balance[8, 101] - Mirum anticipates achieving positive cash flow in 2025[8, 100] Approved Products and Pipeline - Mirum has 3 approved ultra-rare medicines[7, 9] - LIVMARLI is approved for Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC)[9, 14] - CHOLBAM and CTEXLI are bile acid replacement therapies for rare genetic diseases[9, 26] - Volixibat is in development for Primary Sclerosing Cholangitis (PSC) and Primary Biliary Cholangitis (PBC) with positive interim analysis[9, 44] - MRM-3379 is a PDE4D inhibitor in Phase 2 development for Fragile X Syndrome (FXS)[9, 78] Clinical Trials and Milestones - EXPAND Phase 3 study is enrolling patients with ultra-rare cholestatic pruritus, excluding PSC, PBC, ICP, ALGS and PFIC, with enrollment completion expected in 2026[9, 39, 42] - VISTAS study (PSC) expects confirmatory topline data in Q2 2026[9, 63] - VANTAGE study (PBC) expects enrollment completion in 2026[9, 72]

Core Insights - Mirum Pharmaceuticals' lead product, Livmarli, is the primary revenue driver for the company, significantly contributing to its top-line growth [1][3][13] Product Overview - Livmarli is an orally administered ileal bile acid transporter (IBAT) inhibitor approved for treating cholestatic pruritus in patients with Alagille syndrome (ALGS) and certain patients with progressive familial intrahepatic cholestasis (PFIC) [2] - A new tablet formulation of Livmarli was approved by the FDA earlier this year, enhancing convenience for older patients [3] Financial Performance - In the first half of 2025, Livmarli's net product sales reached $161.4 million, reflecting a 79.1% year-over-year increase [3] - Mirum has raised its revenue guidance for 2025 to a range of $490-$510 million, up from the previous estimate of $435-$450 million, due to strong sales performance [6][13] Market Dynamics - Mirum's acquisition of Travere Therapeutics' bile acid products in 2023 has diversified its revenue stream, adding Cholbam capsules and Ctexli tablets to its portfolio [5] - Competition is intensifying in the market, particularly from Albireo AB's Bylvay, which directly competes with Livmarli [8] Pipeline Developments - Mirum's lead pipeline candidate, volixibat, is currently undergoing evaluation in two phase IIb studies for primary biliary cholangitis and primary sclerosing cholangitis [9] Valuation and Stock Performance - Year-to-date, Mirum's shares have increased by 74.6%, outperforming the industry average of 7.4% [14] - The company's shares are trading at a premium, with a price/book ratio of 14.21 compared to the industry average of 3.35 [15] - The Zacks Consensus Estimate for 2025 loss per share has improved from 84 cents to 69 cents, with 2026 estimates shifting from a loss of 13 cents to earnings of 3 cents [16]

Core Insights - CorMedix (CRMD) and Mirum Pharmaceuticals (MIRM) are focusing on treatments for underserved medical conditions, creating niches in areas often overlooked by larger pharmaceutical companies [1] CorMedix (CRMD) - CorMedix's lead product, DefenCath, is the first FDA-approved antimicrobial catheter lock solution in the U.S., aimed at reducing catheter-related bloodstream infections in adult patients undergoing chronic hemodialysis [2] - DefenCath generated $78.8 million in net revenues in the first half of 2025, prompting CorMedix to raise its full-year revenue guidance for 2025 [6] - The company plans to expand DefenCath's label into total parenteral nutrition to increase its customer base [7] - CorMedix acquired Melinta Therapeutics for $300 million, adding seven marketed therapies and diversifying its revenue streams [8] - The acquisition of Melinta is a strategic move to mitigate competition in the CRBSI market from established players like Pfizer and Amphastar [10] - The Zacks Consensus Estimate for CorMedix's 2025 sales and EPS indicates a year-over-year increase of approximately 411% and 607%, respectively [19] Mirum Pharmaceuticals (MIRM) - Mirum's lead product, Livmarli, is approved for treating cholestatic pruritus in patients with Alagille syndrome and has generated $161.4 million in net product sales in the first half of 2025, reflecting a 79.1% year-over-year increase [3][14] - Livmarli's sales momentum is expected to continue, supported by a new tablet formulation launched in June 2025 [13] - MIRM's revenue is heavily reliant on Livmarli, making it vulnerable to regulatory setbacks and competitive pressures, particularly from Albireo's Bylvay [18][29] - Mirum's strategic acquisition of Travere Therapeutics' bile acid products has diversified its revenue stream, generating $78 million in the first half of 2025 [17] - The Zacks Consensus Estimate for Mirum's 2025 sales implies a year-over-year increase of around 51%, with a projected 61% narrowing of loss per share [19] Comparative Analysis - Year-to-date, CRMD shares have gained 39.1%, while MIRM shares have increased by 78.7%, compared to a 3.5% return for the industry [24] - MIRM's shares trade at a higher price/book (P/B) ratio of 14.35 compared to CRMD's 3.47, indicating a more expensive valuation [25] - CorMedix is viewed as a stronger investment option due to its expanding product portfolio and growth trajectory, while Mirum's reliance on Livmarli poses risks [27][31]

Core Insights - New options for Mirum Pharmaceuticals Inc (MIRM) began trading this week, with a focus on contracts expiring on November 21st [1] Options Analysis - A put contract at the $70.00 strike price has a current bid of $0.40, allowing investors to purchase shares at an effective cost basis of $69.60, which is a 6% discount to the current trading price of $74.69 [2][3] - The likelihood of the put contract expiring worthless is estimated at 70%, potentially yielding a 0.57% return on cash commitment or 3.31% annualized [3] - A call contract at the $80.00 strike price has a current bid of $1.00, offering an 8.45% total return if the stock is called away at expiration [6][8] - The $80.00 strike represents a 7% premium to the current stock price, with a 60% chance of the covered call expiring worthless, allowing the investor to retain both shares and premium [8] Volatility Metrics - Implied volatility for both the put and call contracts is approximately 41%, while the actual trailing twelve-month volatility is calculated at 40% [9]

Summary of Mirum Pharmaceuticals FY Conference Call Company Overview - **Company**: Mirum Pharmaceuticals (NasdaqGM:MIRM) - **Industry**: Biopharmaceuticals focused on rare diseases - **Products**: Three commercialized small molecule drugs: LIVMARLI, CHOLBAM, and CHENODAL [1][2] Financial Performance - **Revenue Guidance**: Management raised revenue guidance for 2025 to a range of $490 million to $510 million, reflecting robust revenue gains in the first half of 2025 compared to 2024 [1][2] - **Cash Flow**: The company is operating cash flow positive and has a strong balance sheet [2] Product Details LIVMARLI - **Indications**: Approved for Alagille syndrome and PFIC (Progressive Familial Intrahepatic Cholestasis) [3] - **Market Growth**: The PFIC indication has shown unexpected growth, particularly in adult populations, leading to increased patient identification [4][5] - **Clinical Profile**: LIVMARLI has a differentiated clinical profile, showing benefits in pruritus, growth, and bilirubin improvements [4] CHENODAL - **Indication**: Approved for CTX (Cerebrotendinous Xanthomatosis) with seven years of orphan exclusivity [3] CHOLBAM - **Indication**: Approved for bile acid synthesis disorders [3] International Expansion - **Partnerships**: Strong performance from international business, particularly in Japan with partner Takeda, who received approvals for Alagille and PFIC [6] Clinical Pipeline EXPAND Trial - **Objective**: Expanding the label of LIVMARLI to include additional settings of cholestatic pruritus, targeting at least 500 pediatric patients in the U.S. [7][8] VISTA Study - **Indication**: Evaluating volixibat for PSC (Primary Sclerosing Cholangitis) with an expected top-line readout in Q2 of the following year [9][10] - **Market Size**: Approximately 30,000 patients in the U.S., with a significant unmet need for effective treatments [9][10] AVANTAGE Study - **Indication**: Evaluating volixibat for PBC (Primary Biliary Cholangitis) with positive interim data showing significant improvements in pruritus and fatigue [17][18] MRM-3379 - **Indication**: Targeting Fragile X syndrome, a genetic mutation leading to autism spectrum disorder, with a market opportunity of around 25,000 patients in the U.S. [22][23] - **Phase 2 Study**: Expected to start in Q4 of the current year, focusing on different age groups [25] Competitive Landscape - **Volixibat Positioning**: Positioned as a first-line treatment option for pruritus in PBC, differentiating from existing therapies that primarily target second-line settings [20][21] Future Outlook - **Clinical and Commercial Catalysts**: Continued growth of all three medicines, initiation of Phase 2 in Fragile X, and upcoming data readouts for PSC and PBC expected in 2026 [27] - **Portfolio Expansion**: The company is actively looking for opportunities in the rare disease space, focusing on adding value and ensuring alignment with current capabilities [28][29]

Company Overview - Mirum Pharmaceuticals is focused on rare diseases and has three approved medicines projected to generate revenue between $490 million and $510 million this year, indicating a strong growth trajectory in its commercial business [4]. Pipeline Developments - The company recently announced the completion of enrollment for the VISTAS Phase IIb study of Volixibat for Primary Sclerosing Cholangitis (PSC), which is one of three potentially pivotal studies expected over the next 18 months, with top-line data anticipated in the second quarter of next year [4]. - Additionally, there is a follow-up indication for Volixibat in primary biliary cholangitis and a label expansion opportunity for LIVMARLI with the EXPAND study [4].