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].

Business Overview - Mirum Pharmaceuticals projects net product sales of $310-320 million for 2024[9, 17] - The company has three approved rare disease products and three additional indications in late-stage development[8, 13] - Mirum's cash balance is $303 million, positioning it to execute its strategy[14] LIVMARLI (maralixibat) - In Alagille Syndrome (ALGS), 93% of participants experienced at least a 1-point reduction in ItchRO[Obs] in cholestatic pruritus[30, 95] - In ALGS, 83% of patients with a >1-point reduction in ItchRO[Obs] remained transplant-free 6 years after starting LIVMARLI[30, 100, 102] - In Progressive Familial Intrahepatic Cholestasis (PFIC), 62% of patients achieved minimal to no itch after 26 weeks of treatment[34, 35, 104] CHENODAL - CHENODAL addresses Cerebrotendinous Xanthomatosis (CTX), with a US prevalence of approximately 1,000-2,000 patients[13, 49] - RESTORE Phase 3 study showed a 20-fold increase in urine 23S-Pentol (bile alcohol) after placebo withdrawal (p<0.0001)[122] - RESTORE Phase 3 study showed a 50-fold increase in plasma 7αC4 after placebo withdrawal (p<0.0001)[122] Volixibat - Primary Sclerosing Cholangitis (PSC) affects approximately 54,000 patients across the US & EU, with ~65% experiencing active pruritus[13, 59] - In a CAMEO study, patients with PSC experienced a 70% mean reduction in pruritus and a 40% mean reduction in bile acids after 14 weeks of treatment with an IBAT inhibitor[61]

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

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

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

Financial Data and Key Metrics Changes - Total revenues for Q1 2025 reached $111.6 million, representing a 61% increase compared to Q1 2024 [5] - The company updated its full-year revenue guidance to $435 million to $450 million, driven by strong growth across all commercial medicines [5][11] - Net product revenue for Q1 2025 was $111.6 million, up from $69.2 million in Q1 2024 [15] - Cash, cash equivalents, and investments at March 31, 2025, were $298.6 million, compared to $292.8 million at the beginning of the year [15] - Operating expenses for Q1 2025 totaled $126.8 million, including R&D expenses of $46 million and SG&A expenses of $57.7 million [15] Business Line Data and Key Metrics Changes - Total net product sales for LIVMARLI reached $73.2 million in Q1 2025, a growth of over 70% compared to Q1 2024 [11] - U.S. LIVMARLI sales were $49.5 million, driven by robust new patient demand [11] - International LIVMARLI sales were $23.7 million, with strong demand growth in direct European markets [12] - Bile acid products generated $38.4 million in net product sales, representing a 47% growth year-over-year [13] Market Data and Key Metrics Changes - The approval of LIVMARLI in Japan for PFIC and Alagille syndrome is expected to enhance market presence [6] - Citexly received FDA approval for the treatment of CTX, with promotional efforts underway to reach the underdiagnosed community [6][13] - The company is focusing on engaging healthcare professionals to identify patients with CTX following the recent approval [13] Company Strategy and Development Direction - The company aims to continue advancing its commercial medicines and high-impact pipeline, with a focus on rare diseases [5][19] - The VISTA study of velixibat is nearing completion of enrollment, with top-line data expected in Q2 2026 [8] - The company plans to initiate a Phase II study for MRM-3379 in fragile X syndrome later this year [10][19] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the strong start to the year and the growth of commercial medicines exceeding initial expectations [18] - The company is well-positioned for continued leadership in rare diseases, with a strong financial position and cash flow positivity expected for the full year [16][19] - Management highlighted the importance of recent regulatory approvals in supporting long-term growth potential [18] Other Important Information - The cash contribution margin from the commercial business improved from approximately 47% in Q1 2024 to approximately 53% in Q1 2025 [16] - The company is focused on raising disease awareness to improve patient identification for CTX [67] Q&A Session Summary Question: Insights on velexibat PBC data and discontinuation rates - Management noted that the data matured positively in the interim analysis, with strong response curves and minimal discontinuations due to diarrhea [21][22] Question: Additional IP around LIVMARLI tablet formulation and adoption - The tablet formulation has resulted in novel IP, with an allowed patent expected to extend coverage [24] - Management anticipates strong adoption among older children and adolescents due to the convenience of a single tablet [25] Question: Comparison of interim Phase II PBC data with competitors - Management highlighted the strengths of the pelixibat program, particularly in placebo-adjusted outcomes [31] Question: Update on LIVMARLI access versus competitors - Access to LIVMARLI in the U.S. is strong, with no significant barriers noted [39] Question: Expectations for the VISTA study and competitive positioning - Management expressed optimism for velexibat's activity level based on robust data from the VANTAGE study [43][46] Question: Update on inventory levels and operating expenses - Management clarified that the recent inventory increase is related to new international partners and is expected to normalize [52] Question: PFIC launch performance and sustainability of profitability - The PFIC launch has exceeded expectations, and while cash flow positivity is expected, profitability in a GAAP sense is not anticipated soon [73]

Financial Data and Key Metrics Changes - Total revenues for Q1 2025 reached $111.6 million, representing a 61% growth compared to Q1 2024 [5] - The company updated its full-year revenue guidance to $435 million to $450 million, driven by strong growth across all commercial medicines [5][10] - Net product revenue for Q1 2025 was $111.6 million, up from $69.2 million in Q1 2024 [13] - Cash, cash equivalents, and investments at March 31, 2025, were $298.6 million, compared to $292.8 million at the beginning of the year [13] - Operating expenses for Q1 2025 totaled $126.8 million, including R&D expenses of $46 million and SG&A expenses of $57.7 million [13][15] Business Line Data and Key Metrics Changes - Total net product sales for LIVMARLI reached $73.2 million in Q1 2025, a 70% increase compared to Q1 2024 [10] - U.S. LIVMARLI sales were $49.5 million, driven by robust new patient demand [10] - International LIVMARLI sales were $23.7 million, with strong demand growth in direct European markets [10] - Bile acid products generated $38.4 million in net product sales, representing a 47% growth year-over-year [12] Market Data and Key Metrics Changes - The approval of LIVMARLI's tablet formulation is expected to enhance patient convenience and drive adoption among older children and adolescents [10][24] - The company has seen an increase in new CTX patients since the FDA approval of Citexly in February 2025 [12][66] - The market for PSC is characterized as unsatisfied, with no FDA-approved therapies currently available, positioning velixibat favorably [46] Company Strategy and Development Direction - The company is focused on advancing its commercial medicines and high-impact pipeline, with three important regulatory approvals received since the start of the year [6][17] - The VISTA study of velixibat is nearing completion of enrollment, with top-line data expected in Q2 2026 [7][18] - The company plans to initiate a Phase II study for MRM-3379 in fragile X syndrome later this year [9][18] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the strong start to the year and the growth of commercial medicines exceeding initial expectations [17] - The company is well-positioned for continued leadership in rare diseases, with a strong financial position and cash flow positive outlook for the full year [15][72] Other Important Information - The cash contribution margin from the commercial business improved from approximately 47% in Q1 2024 to approximately 53% in Q1 2025 [15] - The company is focused on raising disease awareness to improve patient identification for underdiagnosed conditions [66] Q&A Session Summary Question: Insights on the additional velexibat PBC data - Management noted that the data matured positively in the interim analysis, with strong response curves [20][21] Question: Additional IP around the Libmarli tablet formulation - The tablet formulation has resulted in novel IP, with an allowed patent expected to extend coverage [22][23] Question: Comparison of interim Phase II PBC data with competitors - Management highlighted strengths in the pelixibat program, particularly in placebo-adjusted differences [30] Question: Update on Marley access versus competitors - Access to LIVMARLI in the U.S. is strong, with no barriers anticipated for either indication [38] Question: Expectations for the VISTA study - Management is optimistic about velexibat's activity level, using VANTAGE as a benchmark [42] Question: Update on patient identification for Citexly - There has been a nice uptick in patient identification since the FDA approval, attributed to promotional efforts [66][64] Question: Sustainability of profitability into 2026 - The company expects to be cash flow positive for the full year but does not anticipate profitability in a GAAP sense soon [72]

Financial Data and Key Metrics Changes - Total revenues for Q1 2025 reached $111.6 million, representing a 61% growth compared to Q1 2024 [4] - The company updated its full-year revenue guidance to $435 million to $450 million, driven by robust growth from all three commercial medicines [4][10] - Net product revenue for Q1 2025 was $111.6 million, compared to $69.2 million in Q1 2024 [13] - Cash, cash equivalents, and investments at March 31, 2025, were $298.6 million, up from $292.8 million at the beginning of the year [13] - Operating expenses for Q1 2025 totaled $126.8 million, including R&D expenses of $46 million and SG&A expenses of $57.7 million [13][14] Business Line Data and Key Metrics Changes - Total net product sales for LIVMARLI reached $73.2 million in Q1 2025, an increase of over 70% compared to Q1 2024 [10] - U.S. LIVMARLI sales were $49.5 million, driven by strong new patient demand across indications [10] - International LIVMARLI sales were $23.7 million, with strong demand growth in direct European markets [10] - Bile acid products generated $38.4 million in net product sales, representing a 47% growth over the same quarter last year [12] Market Data and Key Metrics Changes - The approval of LIVMARLI in Japan for PFIC and Alagille syndrome is expected to enhance market presence [5] - Citexly received FDA approval for the treatment of CTX, with promotional efforts underway to reach the underdiagnosed community [5] - The company is focused on engaging healthcare professionals to identify patients with CTX following the recent FDA approval [12] Company Strategy and Development Direction - The company aims to advance its commercial medicines and high-impact pipeline, with a focus on rare diseases [4][16] - The VISTA study of velixibat is nearing completion of enrollment, with top-line data expected in Q2 2026 [6][17] - The company plans to initiate a Phase II study for MRM-3379 in fragile X syndrome later this year [9][17] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the strong start to the year and the growth of commercial medicines exceeding initial expectations [16] - The company is well-positioned for continued leadership in rare diseases, with a strong financial position and cash flow positivity expected for the full year [15][17] - Management highlighted the importance of recent regulatory approvals in supporting long-term growth potential [16] Other Important Information - The cash contribution margin from the commercial business improved from approximately 47% in Q1 2024 to approximately 53% in Q1 2025 [14] - The company is focused on maintaining financial independence and executing its business plan effectively [15] Q&A Session Summary Question: Insights on velexibat PBC data and discontinuations - Management noted excitement over the data maturation in the interim analysis, with strong response curves and minimal discontinuations due to diarrhea [19][20] Question: Additional IP around LIVMARLI tablet formulation - The tablet formulation has resulted in novel IP, with an allowed patent expected to grant soon, extending coverage to February 2043 [22] Question: Comparison of interim Phase II PBC data with competitors - Management highlighted strengths in the pelixibat program, emphasizing strong outcomes in both change from baseline and placebo-adjusted differences [30] Question: Update on LIVMARLI access versus competitors - Access to LIVMARLI in the U.S. is strong, with no barriers noted for either indication, supported by a strong clinical value proposition [37] Question: Expectations for the VISTA study and competitive positioning - Management expressed optimism for velexibat's activity level in the VISTA study, with a strong market position anticipated due to the lack of approved therapies for PSC [45][46] Question: Update on patient identification for Citexly - Management reported a nice uptick in patient identification since the FDA approval, driven by promotional efforts and disease awareness initiatives [64] Question: PFIC launch performance and sustainability of profitability - The PFIC launch has exceeded expectations, and while cash flow positivity is anticipated, profitability in a GAAP sense is not expected soon due to non-cash charges [70][71]