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].

Summary of Mirum Pharmaceuticals FY Conference Call Company Overview - **Company**: Mirum Pharmaceuticals (NasdaqGM:MIRM) - **Focus**: Rare disease-focused biopharmaceutical company - **Revenue Projection**: Expected revenue between $490 million to $510 million for the year [2][3] Key Products and Pipeline - **Approved Medicines**: Three approved medicines with a strong commercial business - **LIVMARLI**: - Recent label expansion from PFIC to a second indication, leading to significant growth - Projected peak sales of over $1 billion [3][15] - Strong performance driven by new patient diagnoses and flexible formulations [3][6] - **Volixibat**: - Recently completed enrollment for the VISTAS Phase 2b study in PSC, with top-line data expected in Q2 next year [2][30] - Targeting symptoms like pruritus as a primary endpoint for registration [30][33] - **MRM-3379**: - New program for Fragile X, starting Phase 2 study with a focus on cognitive endpoints [52][54] Market Dynamics - **LIVMARLI Growth**: - Strong growth in both U.S. and international markets, particularly in Alagille syndrome and PFIC [5][11] - Underestimated patient population for PFIC, leading to proactive diagnosis support [9][10] - **International Expansion**: - Direct markets include Canada and Western Europe, with over 30 countries now reimbursing the product [11][12] - **Bile Acid Portfolio**: - CHOLBAM and CHENODAL showing growth, with recent quarterly revenue of $40 million [28] Clinical Development Insights - **VISTAS Study**: - Focused on pruritus in PSC patients, with a significant patient population of approximately 30,000 in the U.S. [30][31] - Interim analysis showed promising results, with a clinically meaningful effect size expected [35][39] - **VANTAGE Study**: - Targeting PBC with a larger patient population, expected to complete enrollment next year [47] - Competitive landscape includes another IBAT therapy from GSK, with Mirum positioned favorably based on interim data [50] Regulatory and Strategic Considerations - **Regulatory Interactions**: - Constructive interactions with the FDA, with no significant issues reported [58] - **AI Utilization**: - Leveraging AI for efficiency in document drafting and regulatory processes [57] - **Market Positioning**: - Focus on underappreciated programs and repositioning mechanisms as part of business development strategy [55] Conclusion - Mirum Pharmaceuticals is positioned for significant growth with a strong pipeline and expanding market presence, particularly with LIVMARLI and volixibat. The company is actively addressing unmet needs in rare diseases while navigating regulatory landscapes effectively.

Mirum Pharmaceuticals (MIRM) 2025 Conference Summary Company Overview - **Company**: Mirum Pharmaceuticals - **Focus**: Rare disease therapeutics with three approved medicines - **Current Revenue Guidance**: $490 to $510 million, cash flow positive, financially independent [2][76] Key Products and Market Insights LIVMARLI - **Current Market Penetration**: Approximately 50% in the U.S. and mature European markets for Alagille syndrome [14] - **Patient Population**: Estimated 2,000 to 2,500 prevalent cases in the U.S., with 75% experiencing pruritus [18] - **Incidence Rate**: About 1 in 50,000 births [19] - **Newborn Screening**: Not necessary as most patients present with neonatal jaundice [16] SATEXLY - **Patient Identification Strategy**: Focused on identifying patients with CTX through a dedicated sales team and screening efforts [4] - **Diagnosis Rate**: Literature suggests only about 10% of patients are diagnosed [4] - **Newborn Screening Efforts**: Ongoing pilot projects to include CTX in newborn screening, with promising results from New York [9][10] PFIC (Progressive Familial Intrahepatic Cholestasis) - **Target Demographic**: Older children, adolescents, and adults [27] - **Patient Identification**: Focus on gastroenterologists and hepatologists to identify patients with idiopathic cholestasis [30] Financial Performance and Projections - **Revenue Recognition Model**: Revenue recognized upon family signing for LIVMARLI, reflecting demand sales [17] - **Takeda Partnership**: Significant revenue generation from Japan, with a scaling down of royalties expected over time [35][36] - **Future Profitability**: Potential for Mirum to become a very profitable company in the coming years, assuming successful product launches [76] Research and Development - **Pipeline**: Four clinical trials from three different product candidates, with three believed to be registration trials [2] - **Expanded Basket Study**: Aimed at treating patients with cholestatic pruritus beyond Alagille and PFIC, with an estimated 500 or more patients targeted [49][61] Strategic Planning and Risk Management - **Long-term Strategy**: Discussions on cash flow deployment and potential acquisitions are anticipated as profitability increases [78] - **Risk Appetite**: Expected to evolve with increased profitability, with a focus on validated mechanisms and lower biology risk [80][82] Core Competencies - **Strengths**: Late-stage clinical development, regulatory expertise, and strong commercial capabilities in rare diseases [85][88] Additional Insights - **Market Dynamics**: The company is optimistic about the potential for growth in the rare disease market, particularly with ongoing education and awareness efforts among healthcare providers [70][71] - **Patient Engagement**: Emphasis on building relationships with physicians and patient advocates to enhance product visibility and adoption [85][88]

Group 1 - The biotech forum has seen significant discussions around covered call opportunities in recent quarters [1] - Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) was previously identified as a solid investment opportunity, with recommendations to accumulate shares [2] - The company has reported two quarters of results since the last analysis, indicating ongoing performance tracking [2] Group 2 - The investing group, The Biotech Forum, offers a model portfolio featuring 12-20 high upside biotech stocks, along with live chat for trade discussions and weekly research updates [2]

Core Insights - Mirum Pharmaceuticals reported a narrower loss of $0.12 per share for Q2 2025, compared to a loss of $0.52 per share in the same quarter last year, and better than the Zacks Consensus Estimate of a loss of $0.31 per share [1][8] - Revenues for Q2 2025 reached $127.8 million, reflecting a year-over-year increase of 64.1%, surpassing the Zacks Consensus Estimate of $108 million, driven by strong sales of Livmarli and newly acquired bile acid products [2][8] - The company raised its revenue guidance for 2025 to a range of $490 million to $510 million, up from the previous estimate of $435 million to $450 million, due to the strong performance of Livmarli [10] Financial Performance - Livmarli's net product sales were $88.2 million in Q2 2025, marking an 87% increase year-over-year, with U.S. sales at $57 million and ex-U.S. sales at $31.2 million [7][8] - Net product sales from bile acid products, including Cholbam and Ctexli, totaled $39.6 million in Q2 2025, reflecting a 30% year-over-year increase [7] - Research and development expenses rose by nearly 41% year-over-year to $46.1 million, while selling, general, and administrative expenses increased by approximately 28.7% to $63.3 million [9] Recent Developments - Mirum is conducting a phase III EXPAND study for Livmarli to treat pruritus in rare cholestatic conditions, with enrollment expected to complete in 2026 [11] - The FDA approved a new tablet formulation of Livmarli, launched in the U.S. in June 2025, aimed at improving convenience for older patients [11] - The lead pipeline candidate, volixibat, is under evaluation in two phase IIb studies for primary biliary cholangitis and primary sclerosing cholangitis, with top-line data expected in Q2 2026 [12] Stock Performance - Mirum's shares increased by 8.6% in after-hours trading on August 6, 2025, following the better-than-expected results and raised revenue guidance [4] - Year-to-date, Mirum's stock has risen by 29.1%, significantly outperforming the industry average increase of 0.2% [6]

Financial Data and Key Metrics Changes - Total revenues for the second quarter reached $128 million, representing a 64% increase compared to the same quarter last year [6][19] - The company raised its full-year revenue guidance for 2025 to a range of $490 million to $510 million, indicating nearly 50% top-line growth [7][13] Business Line Data and Key Metrics Changes - Net product sales for Lidmarley were approximately $128 million, driven by strong demand in both the U.S. and international markets [10][19] - In the U.S., Lidmarley generated about $57 million in net product sales, with increased patient awareness contributing to higher volumes [10][11] - Internationally, Lidmarley sales reached $31 million, supported by expanding reimbursement and strong demand [12][13] Market Data and Key Metrics Changes - The U.S. market for Lidmarley is seeing increased patient penetration, with approximately 70-75% persistence rates after one year for Alagille syndrome patients [25][59] - The company is experiencing growth in the PFIC market due to increased awareness and genetic testing, leading to more diagnoses [11][59] Company Strategy and Development Direction - The company's strategy focuses on commercial execution, scientific innovation, and financial discipline, with a commitment to delivering life-changing medicines for rare diseases [5][20] - The pipeline includes three late-stage studies expected to yield pivotal data over the next 24 months, particularly in primary sclerosing cholangitis [8][16] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's position and the opportunities ahead, highlighting the strong performance of its medicines and pipeline [20] - The company is optimistic about Lidmarley's potential to exceed $1 billion in revenue, driven by ongoing growth in Alagille syndrome and PFIC indications [14][62] Other Important Information - The company reported operating cash flow positivity for the quarter, with cash and investments totaling $322 million, reflecting a $29 million increase from the end of the previous year [19] Q&A Session Summary Question: What are the overall therapy persistence rates for Lidmarley? - Management indicated that persistence rates for Alagille syndrome patients are around 70-75% after one year, with lower attrition in subsequent years [25] Question: Can you provide details on the expected revenue distribution between Lidmarley and the bile acid business? - Management did not break down guidance by specific products but noted that trends observed year-to-date are expected to continue [33] Question: What are the main drivers of growth in Lidmarley sales? - Key drivers include increased awareness of PFIC and the introduction of a single tablet formulation, which has received positive feedback [39][41] Question: Are there any inventory impacts in the second quarter? - Inventory impacts are primarily relevant to Japan and Takeda, with no significant inventory issues in the U.S. or Europe [44] Question: What is the status of the Fragile X study? - The company has received clearance from the FDA and is on track to initiate the Phase II study by the end of the year [52] Question: How well penetrated is the company in the Alagille and PFIC markets? - The company estimates approximately 50% penetration in the Alagille syndrome market, with ongoing efforts to increase awareness and patient identification [59][60]

Financial Data and Key Metrics Changes - Total revenues for the second quarter reached $128 million, representing a 64% increase compared to the same quarter last year [6][19] - The company raised its full-year revenue guidance for 2025 to a range of $490 million to $510 million, indicating nearly 50% top-line growth [6][13] Business Line Data and Key Metrics Changes - Net product sales for Lidmarley were approximately $128 million, driven by strong demand in both the U.S. and international markets [10] - In the U.S., Lidmarley generated about $57 million in net product sales, with increased patient awareness contributing to higher volumes [11][12] - Internationally, Lidmarley sales reached $31 million, supported by expanding reimbursement and strong demand [12][13] - The bile acid portfolio contributed approximately $40 million in revenue, benefiting from steady demand [13] Market Data and Key Metrics Changes - The U.S. market for Lidmarley is seeing increased recognition of PFIC diagnoses, with more patients presenting later in childhood or adulthood [11] - The company is experiencing durable growth in international markets, particularly with the recent launch of Lidmarley in Japan [12] Company Strategy and Development Direction - The company’s strategy focuses on commercial execution, scientific innovation, and financial discipline, with ongoing progress in all areas [6] - The pipeline includes three late-stage milestones, with significant studies expected to report data in the next 24 months [7][15] - Lidmarley is positioned to potentially become a billion-dollar revenue brand, driven by growth in Alagille syndrome and PFIC indications [14][56] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing momentum of commercial medicines and the pipeline, highlighting the potential for significant patient impact [5][21] - The company is optimistic about the upcoming clinical milestones and the overall growth trajectory [7][19] Other Important Information - The company reported operating cash flow positivity for the quarter, with cash and investments totaling $322 million [20] - The cash contribution margin from the commercial business exceeded 50% in the second quarter [20] Q&A Session Summary Question: What are the overall therapy persistence rates for Lidmarley? - Management indicated that persistence rates for Alagille syndrome are stable, with approximately 70-75% of patients remaining on therapy after one year [25][26] Question: Can you provide details on expected revenue distribution between Lidmarley and the bile acid business? - Management did not break down guidance by specific products but noted that trends observed year-to-date are expected to continue [33] Question: What are the main drivers of growth in Lidmarley sales? - Key drivers include increased awareness of PFIC and the introduction of a single tablet formulation, which has received positive feedback [37][38] Question: Any insights on inventory impacts in Q2 and Takeda order cadence? - Inventory impacts are primarily relevant in Japan, with expectations of periodic orders from Takeda leading to quarter-to-quarter variability [42][43] Question: What is the status of the Fragile X study? - The company has received clearance from the FDA and is on track to initiate the Phase II study by the end of the year [48] Question: How well penetrated is the company in the Alagille and PFIC markets? - Management estimates approximately 50% penetration in the Alagille market, with ongoing efforts to increase awareness and patient identification in PFIC [55][56] Question: Are any patients in the EXPAND study already on Lidmarley through compassionate use? - The EXPAND study is designed for patients who have not previously been treated, with strong interest from patient populations [61]

Summary of Risks Associated with Our Business [Summary of Risks](index=3&type=section&id=Summary%20of%20Risks) Key business risks include the commercial success of medicines, reliance on third parties, clinical development uncertainty, and competition - Commercial success hinges on marketing approved medicines and is subject to market acceptance and reliance on third-party manufacturing[8](index=8&type=chunk) - The business depends on the success of product candidates in **lengthy and expensive clinical testing** with uncertain outcomes[8](index=8&type=chunk) - The company faces **significant competition** and may require substantial additional financing for commercialization and development[8](index=8&type=chunk) - **Reliance on licensed intellectual property** and the ability to maintain IP protection are critical to the business[8](index=8&type=chunk) PART I. FINANCIAL INFORMATION [Item 1. Financial Statements (Unaudited)](index=4&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) Unaudited financial statements show significant revenue growth and a reduced net loss, driven by strong product sales [Condensed Consolidated Balance Sheets](index=4&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Total assets grew to $725.8 million, supported by increases in cash and receivables, alongside a rise in liabilities and stockholders' equity Condensed Consolidated Balance Sheet Highlights (in thousands) | Account | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | **Total Current Assets** | $453,398 | $392,788 | | Cash and cash equivalents | $228,122 | $222,503 | | Accounts receivable | $106,836 | $78,286 | | **Total Assets** | **$725,825** | **$670,754** | | **Total Current Liabilities** | $144,796 | $126,551 | | **Total Liabilities** | **$470,667** | **$445,114** | | **Total Stockholders' Equity** | **$255,158** | **$225,640** | [Condensed Consolidated Statements of Operations](index=5&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) Total revenue increased 64% year-over-year to $127.8 million, significantly narrowing the quarterly net loss to $(5.9) million Statement of Operations Summary (in thousands, except per share data) | Metric | Q2 2025 | Q2 2024 | 6 Months 2025 | 6 Months 2024 | | :--- | :--- | :--- | :--- | :--- | | **Total Revenue** | **$127,785** | **$77,875** | **$239,370** | **$147,097** | | Product sales, net | $127,785 | $77,760 | $239,370 | $146,677 | | **Total Operating Expenses** | **$132,774** | **$102,107** | **$259,542** | **$197,797** | | Cost of sales | $23,421 | $20,227 | $46,439 | $38,057 | | Research and development | $46,067 | $32,672 | $92,111 | $64,894 | | Selling, general and administrative | $63,286 | $49,208 | $120,992 | $94,846 | | **Loss from Operations** | **$(4,989)** | **$(24,232)** | **$(20,172)** | **$(50,700)** | | **Net Loss** | **$(5,861)** | **$(24,638)** | **$(20,538)** | **$(49,917)** | | **Net Loss Per Share** | **$(0.12)** | **$(0.52)** | **$(0.42)** | **$(1.06)** | [Condensed Consolidated Statements of Cash Flows](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Net cash from operations was $10.1 million, and the company's cash position increased by $5.7 million to $228.6 million Cash Flow Summary for Six Months Ended June 30 (in thousands) | Category | 2025 | 2024 | | :--- | :--- | :--- | | Net cash provided by operating activities | $10,079 | $11,409 | | Net cash used in investing activities | $(22,733) | $(72,730) | | Net cash provided by financing activities | $15,047 | $8,496 | | **Net increase (decrease) in cash** | **$5,674** | **$(52,831)** | | **Cash at end of period** | **$228,602** | **$233,495** | [Notes to Unaudited Condensed Consolidated Financial Statements](index=9&type=section&id=Notes%20to%20Unaudited%20Condensed%20Consolidated%20Financial%20Statements) Notes detail strong product sales growth, intangible asset composition, and the convertibility status of senior notes - The company has three approved medicines: **LIVMARLI® (maralixibat), Cholbam® (cholic acid), and Chenodal®/Ctexli® (chenodiol)**[23](index=23&type=chunk) Product Sales, Net by Medicine (in thousands) | Product | Q2 2025 | Q2 2024 | 6 Months 2025 | 6 Months 2024 | | :--- | :--- | :--- | :--- | :--- | | Livmarli | $88,160 | $47,231 | $161,384 | $90,076 | | Bile Acid Medicines | $39,625 | $30,529 | $77,986 | $56,601 | | **Total** | **$127,785** | **$77,760** | **$239,370** | **$146,677** | Product Sales, Net by Geography (in thousands) | Region | Q2 2025 | Q2 2024 | 6 Months 2025 | 6 Months 2024 | | :--- | :--- | :--- | :--- | :--- | | United States | $95,532 | $65,080 | $182,454 | $121,191 | | Rest of the world | $32,253 | $12,680 | $56,916 | $25,486 | | **Total** | **$127,785** | **$77,760** | **$239,370** | **$146,677** | - The **4.00% Convertible Senior Notes** due 2029 became convertible at the option of holders as the stock price condition was met[81](index=81&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=25&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management attributes revenue growth to Livmarli and Bile Acid Medicines sales, while rising operating expenses reflect increased commercial and R&D activities [Results of Operations](index=29&type=section&id=Results%20of%20Operations) Q2 2025 product sales grew by $50.0 million, driven by Livmarli, alongside increased R&D and SG&A expenses - The increase in product sales for Q2 2025 was driven by continued commercialization of Livmarli and sales of Bile Acid Medicines, including an **$11.0 million positive change in estimate**[135](index=135&type=chunk) - R&D expenses increased primarily due to higher spending on **volixibat and MRM-3379 programs** and a $10.2 million increase in personnel costs[139](index=139&type=chunk)[143](index=143&type=chunk) - SG&A expenses rose mainly due to an **$8.9 million increase in personnel-related expenses** to support commercial activities[140](index=140&type=chunk) [Liquidity and Capital Resources](index=32&type=section&id=Liquidity%20and%20Capital%20Resources) The company holds $321.7 million in unrestricted cash and investments, deemed sufficient to fund operations for at least 12 months - The company had **$321.7 million of unrestricted cash, cash equivalents and investments** as of June 30, 2025, sufficient to fund operations for at least the next 12 months[152](index=152&type=chunk)[156](index=156&type=chunk) - The company has an effective automatic shelf registration and a **$200.0 million ATM offering program**, neither of which had been used as of June 30, 2025[154](index=154&type=chunk)[155](index=155&type=chunk) - Holders of the convertible notes have the option to convert their notes during Q3 2025, which the company may settle in cash, stock, or a combination[163](index=163&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=35&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company's primary market risks are interest rate fluctuations on its investments and foreign currency exposure, mainly to the Euro and Swiss Franc - The company's primary market risk exposures are **interest rate sensitivity** on its cash and investments and **foreign currency exchange rate risk**[174](index=174&type=chunk)[176](index=176&type=chunk) - A hypothetical **10% fluctuation of the USD exchange rate** could result in a potential change of approximately **$1.6 million** in the fair value of net assets and liabilities denominated in foreign currency[177](index=177&type=chunk) [Controls and Procedures](index=35&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective as of June 30, 2025, with no material changes to internal controls - Management concluded that as of June 30, 2025, the company's **disclosure controls and procedures were effective**[178](index=178&type=chunk) - **No material changes** to the company's internal control over financial reporting occurred during the three months ended June 30, 2025[180](index=180&type=chunk) PART II. OTHER INFORMATION [Legal Proceedings](index=37&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently involved in any legal proceedings expected to have a material adverse effect on its financial condition - Management believes there are currently **no pending claims or actions** that could have a material adverse effect on the company's results of operations, financial condition, or cash flows[182](index=182&type=chunk) [Risk Factors](index=37&type=section&id=Item%201A.%20Risk%20Factors) Key risks include commercialization challenges, dependence on third parties, competition, intellectual property vulnerabilities, and future financing needs - Risks to commercialization include the ability to profitably market approved medicines, achieve market acceptance, and **secure adequate reimbursement from payors**[184](index=184&type=chunk)[186](index=186&type=chunk)[191](index=191&type=chunk) - The business is **highly dependent on third parties** for manufacturing, distribution, and conducting clinical trials, creating risks of disruption and delays[320](index=320&type=chunk)[324](index=324&type=chunk) - The company faces **significant competition** from other pharmaceutical companies, including those with greater resources or developing similar treatments[267](index=267&type=chunk)[268](index=268&type=chunk) - Intellectual property risks include the **lack of composition-of-matter patents for Livmarli** and reliance on method-of-use patents[363](index=363&type=chunk)[379](index=379&type=chunk) - Financial risks include the need for **substantial additional financing** and potential dilution to stockholders from equity offerings or note conversions[341](index=341&type=chunk)[346](index=346&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=92&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company reports no unregistered sales of equity securities during the covered period - There were **no sales of unregistered equity securities** during the quarter[472](index=472&type=chunk) [Exhibits](index=93&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed with the Form 10-Q, including required CEO and CFO certifications and XBRL data files - Exhibits filed include **CEO and CFO certifications** pursuant to Sarbanes-Oxley Sections 302 and 906, and Inline XBRL documents[476](index=476&type=chunk)

[Q2 2025 Highlights and Business Update](index=1&type=section&id=Q2%202025%20Highlights%20and%20Business%20Update) Mirum Pharmaceuticals reported strong Q2 2025 results with $128 million total revenue, raising full-year guidance to $490-$510 million, and advancing its late-stage pipeline Q2 2025 Key Metrics | Metric | Value | | :--- | :--- | | Total Revenue | $128 million | | 2025 Revenue Guidance | Increased to $490 - $510 million | - Commercial strength was noted in the **International business** and the **U.S. Progressive Familial Intrahepatic Cholestasis (PFIC) launch**[2](index=2&type=chunk) - The company anticipates **three significant late-stage clinical milestones in 2026**[2](index=2&type=chunk) - The **VISTAS study** of volixibat in Primary Sclerosing Cholangitis (PSC) is expected to complete enrollment this quarter, with topline data planned for **Q2 2026**[2](index=2&type=chunk)[4](index=4&type=chunk) [Commercial Performance](index=1&type=section&id=Commercial%20Performance) Global net product sales reached $127.8 million in Q2 2025, driven by strong LIVMARLI growth and the U.S. launch of a single oral tablet dose Q2 2025 Net Product Sales (YoY Growth) | Product Line | Q2 2025 Sales | YoY Growth | | :--- | :--- | :--- | | LIVMARLI | $88.2 million | 87% | | Bile Acid Medicines | $39.6 million | 30% | | **Total Global Net Product Sales** | **$127.8 million** | **N/A** | - A **single oral tablet dose** of LIVMARLI was launched in the U.S. in **June 2025**[4](index=4&type=chunk) [Pipeline Development](index=1&type=section&id=Pipeline%20Development) Mirum's late-stage pipeline is advancing with key clinical milestones expected in 2026, including topline data for volixibat in PSC and a new Phase 2 study for MRM-3379 - Key upcoming clinical milestones include **Volixibat (VISTAS study in PSC)** with topline data expected in **Q2 2026**, **Volixibat (VANTAGE study in PBC)** with enrollment completion expected in **2026**, and **LIVMARLI (EXPAND Phase 3 study)** with enrollment completion expected in **2026**[4](index=4&type=chunk) - A **Phase 2 study** for **MRM-3379**, a PDE4D inhibitor for Fragile X Syndrome (FXS), is planned to initiate in **Q4 2025**[4](index=4&type=chunk)[22](index=22&type=chunk) [Financial Results](index=1&type=section&id=Financial%20Results) Mirum reported Q2 2025 total revenue of $127.8 million, significantly narrowing its net loss to $5.9 million, and maintaining a strong cash position of $321.7 million - As of June 30, 2025, Mirum had **$321.7 million** in unrestricted cash, cash equivalents, and investments, an increase from **$292.8 million** as of December 31, 2024[4](index=4&type=chunk) [Condensed Consolidated Statement of Operations](index=5&type=section&id=Condensed%20Consolidated%20Statement%20of%20Operations) Q2 2025 total revenue grew 64% to $127.8 million, significantly reducing the net loss to $5.9 million despite increased operating expenses Q2 2025 Statement of Operations (in thousands, except per share data) | Metric | Q2 2025 | Q2 2024 | | :--- | :--- | :--- | | Total revenue | $127,785 | $77,875 | | Total operating expenses | $132,774 | $102,107 | | Loss from operations | $(4,989) | $(24,232) | | Net loss | $(5,861) | $(24,638) | | Net loss per share | $(0.12) | $(0.52) | - Total operating expenses for Q2 2025 included **$24.5 million** of non-cash expenses, compared to **$17.7 million** in Q2 2024[4](index=4&type=chunk) [Condensed Consolidated Balance Sheet](index=6&type=section&id=Condensed%20Consolidated%20Balance%20Sheet) As of June 30, 2025, total assets increased to $725.8 million, with total liabilities at $470.7 million and stockholders' equity at $255.2 million Balance Sheet Highlights (in thousands) | Account | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | Cash, cash equivalents & investments | $321,664 | $292,841 | | Total assets | $725,825 | $670,754 | | Total liabilities | $470,667 | $445,114 | | Total stockholders' equity | $255,158 | $225,640 | [Product Portfolio Overview](index=2&type=section&id=Product%20Portfolio%20Overview) Mirum's commercial portfolio includes LIVMARLI®, CHOLBAM®, and CTEXLI™, with ongoing label expansion for LIVMARLI and pipeline advancement for volixibat in adult cholestatic diseases - **LIVMARLI® (maralixibat)** is approved for cholestatic pruritus in patients with **Alagille syndrome (ALGS)** and **progressive familial intrahepatic cholestasis (PFIC)**[6](index=6&type=chunk) - **Volixibat** is an investigational IBAT inhibitor being evaluated for **primary sclerosing cholangitis (PSC)** and **primary biliary cholangitis (PBC)**, and has received **breakthrough therapy designation** for PBC[12](index=12&type=chunk) - **CHOLBAM® (cholic acid)** is approved for **bile acid synthesis disorders** and adjunctive treatment of **peroxisomal disorders**[13](index=13&type=chunk) - **CTEXLI™ (chenodiol)** is approved for the treatment of adults with **cerebrotendinous xanthomatosis (CTX)**[17](index=17&type=chunk)

Core Viewpoint - The market anticipates Mirum Pharmaceuticals, Inc. (MIRM) will report a year-over-year increase in earnings driven by higher revenues for the quarter ended June 2025, with actual results being crucial for stock price movement [1][2]. Financial Expectations - The consensus estimate indicates a quarterly loss of $0.31 per share, reflecting a year-over-year improvement of +40.4%. Revenues are projected to reach $108.4 million, representing a 39.2% increase from the same quarter last year [3]. - The consensus EPS estimate has been revised 6.92% higher in the last 30 days, indicating a positive reassessment by analysts [4]. Earnings Surprise Potential - The Most Accurate Estimate for Mirum Pharmaceuticals is higher than the Zacks Consensus Estimate, resulting in an Earnings ESP of +8.65%, suggesting a strong likelihood of beating the consensus EPS estimate [12]. - Historical performance shows that Mirum Pharmaceuticals has beaten consensus EPS estimates in two out of the last four quarters, with a notable surprise of +23.08% in the last reported quarter [13][14]. Industry Context - In the Zacks Medical - Biomedical and Genetics industry, Tango Therapeutics, Inc. (TNGX) is expected to report a loss of $0.35 per share for the same quarter, indicating a year-over-year decline of -45.8%. Revenue expectations for Tango are $6.19 million, down 68.9% from the previous year [18][19]. - Tango Therapeutics has a Zacks Rank of 2 (Buy) and an Earnings ESP of +0.57%, suggesting a likelihood of surpassing the consensus EPS estimate [20].