Financial Performance - The net loss for the three months ended September 30, 2022, was $35.7 million, compared to $47.1 million for the same period in 2021, indicating a 24% improvement year-over-year[137] - The net loss for the three months ended September 30, 2022, was $35.7 million, a decrease of $11.4 million from a net loss of $47.1 million in the same period of 2021[174] - The net loss for the nine months ended September 30, 2022, was $99.2 million, a reduction of $42.3 million compared to a net loss of $141.5 million in the same period in 2021[185] Revenue and Sales - Total revenue for the three months ended September 30, 2022, was $18.8 million, an increase of $13.8 million from $5.0 million in the same period of 2021[174] - Livmarli product sales reached $18.8 million for the three months ended September 30, 2022, compared to zero in the same period of 2021[175] - Product sales, net were $47.2 million for the nine months ended September 30, 2022, compared to zero for the same period in 2021 due to the commercial launch of Livmarli[186] Expenses - Research and development expenses decreased to $26.2 million for the three months ended September 30, 2022, down $4.3 million from $30.5 million in the same period of 2021[178] - Selling, general and administrative expenses increased to $22.5 million for the three months ended September 30, 2022, up $5.2 million from $17.4 million in the same period of 2021[174] - Research and development expenses decreased to $75.7 million for the nine months ended September 30, 2022, down $27.9 million from $103.7 million in the same period of 2021[190] Cash and Investments - Cash, cash equivalents, restricted cash equivalents, and investments totaled $285.3 million as of September 30, 2022, compared to $261.5 million as of December 31, 2021[137] - As of September 30, 2022, the company reported cash, cash equivalents, and investments sufficient to fund operations for at least the next 12 months[201] - The company maintains significant cash reserves at a financial institution exceeding federally insured limits, exposing it to interest rate risk[222] Future Outlook - The company anticipates continued losses as it conducts ongoing clinical trials and commercial activities for Livmarli, with product sales expected to increase in the future[138] - The company expects to see increased product sales as it engages further with healthcare providers in the U.S. and globally[151] - The company anticipates continued net losses as it invests in research, product development, and market expansion for Livmarli[202] Clinical Trials and Product Development - The company reported positive topline data from the MARCH Phase 3 clinical trial for PFIC in October 2022, with further data expected from the EMBARK Phase 2b clinical trial in biliary atresia in the second half of 2023[130] - The company is developing volixibat for cholestatic liver diseases, with clinical trials showing significant activity on bile acid markers[131] Financing Activities - An underwritten public offering in August 2022 resulted in net proceeds of $86.1 million after deducting underwriting discounts and expenses[136] - The company issued and sold 1,160,915 shares of common stock during the nine months ended September 30, 2022, generating gross proceeds of $22.1 million[197] - Net cash provided by financing activities for the nine months ended September 30, 2022, was $109.4 million, primarily from a public offering and stock sales[217] Contractual Obligations and Risks - The company has contractual obligations related to license agreements that may result in future payments based on development and sales milestones[208] - The company has entered into the RIPA, with interest rate risk exposure linked to actual and forecasted net product sales, which may impact interest expense recognized each reporting period[223] - The company does not expect inflation to have a significant impact on its results of operations, despite ongoing geopolitical and macroeconomic events[225]

Corporate Presentation September 2022 Forward-Looking Statements This presentation contains "forward-looking" statements that are based on our management's beliefs and assumptions and on information currently available to management. Forward-looking statements include all statements other than statements of historical fact contained in this presentation, including information concerning our business strategy, objectives and opportunities. Forward- looking statements are subject to known and unknown risks, u ...

Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) Q2 2022 Earnings Conference Call August 4, 2022 4:30 PM ET Company Participants Ian Clements - CFO Chris Peetz - President and CEO Peter Radovich - COO Pam Vig - Head, R&D Conference Call Participants Jessica Fye - JPMorgan Lili Nsongo - SVB Ed Arce - H.C. Wainright Ryan Deschner - Raymond James Operator Good afternoon. My name is Chris, and I'll be your conference operator today. At this time, I'd like to welcome everyone to the Mirum Q2 2022 Business Update. [Oper ...

WASHINGTON, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2022 UNITED STATES SECURITIES AND EXCHANGE COMMISSION OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ________to ________ Commission File Number: 001-38981 Mirum Pharmaceuticals, Inc. (Exact name of registrant as specified in its charter) Delaware 83-1281555 ( ...

Financial Data and Key Metrics Changes - Total revenue for Q1 2022 was $12.9 million, including $10.9 million from net product revenue of LIVMARLI and $2 million in licensing revenue from a partner in China [22] - Operating expenses for the quarter totaled $45.6 million, with R&D expenses at $24.1 million and SG&A expenses at $19.1 million [23] - The company had cash, cash equivalents, and investments of $239.9 million at the end of Q1 2022 [23] Business Line Data and Key Metrics Changes - LIVMARLI generated net product revenue of $10.9 million in Q1 2022, reflecting strong demand dynamics and refill cadence [7][12] - Approximately 75% of expenses were reimbursed in Q1, with expectations for this rate to increase to 90% or higher in Q2 and beyond [12] Market Data and Key Metrics Changes - The payer mix for LIVMARLI currently stands at approximately 50% Medicaid and 50% commercial [12] - The company is preparing for international launches of LIVMARLI, with anticipated approval in Europe later in 2022 and early access programs being initiated [15] Company Strategy and Development Direction - Mirum Pharmaceuticals aims to capitalize on a greater-than-$500 million U.S. market opportunity in Alagille syndrome while progressing with five other late-stage clinical programs [9] - The company is focused on innovative research for rare diseases to expedite the development of new medicines [8] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in continued growth throughout the year, driven primarily by the U.S. market [28][40] - The company is optimistic about the potential for LIVMARLI to address unmet medical needs in Alagille syndrome and other cholestatic conditions [24] Other Important Information - The MARCH-PFIC Phase III clinical trial is fully enrolled, with top-line data expected in Q4 2022 [16] - The INDIGO Phase II data has been published, showing significant improvements in patient outcomes [19] Q&A Session Summary Question: What drove the strong revenue of $10.9 million? - Management noted strong demand dynamics and refill cadence, with expectations for continued growth above the previously stated floor of $50 million for the year [27][30] Question: What is the current status of drug reimbursement? - Management expects to achieve a reimbursement rate of 90% in Q2 and beyond [31] Question: How is the LIVMARLI launch progressing in the U.S.? - The conversion of expanded access patients to commercial product was rapid, and the company is seeing strong adoption among targeted key accounts [33] Question: What are the timelines for the European launch? - The first launch will be in Germany following EMA approval, with additional Western European countries targeted for 2023 [35][36] Question: How does the company plan to maintain brand awareness for LIVMARLI? - The focus will be on rolling out access for as many patients as possible and publishing data from the clinical program [45] Question: What is the expected R&D spend trend? - R&D spending is anticipated to remain flat throughout the year, with some studies winding down while others ramp up [42] Question: Is the company seeking collaborators for additional PFIC genetic subtypes? - The company is actively looking for opportunities to grow and develop underappreciated programs in pediatric rare diseases [73]

LIVMARLI for Alagille Syndrome (ALGS) - LIVMARLI is FDA-approved for cholestatic pruritus in ALGS patients [7], with a potential market opportunity exceeding $500 million in the US [7, 9] - A 6-year analysis demonstrated a 70% reduction in clinical outcomes (liver transplantation, biliary diversion surgery, hepatic decompensation, or death) with LIVMARLI compared to an untreated cohort [21] - Q1 2022 total product net revenue for LIVMARLI was $10.9 million, with approximately 75% of dispenses reimbursed [9] Pipeline and Development - The company has 5 late-stage indications under development [7, 12], including Progressive Familial Intrahepatic Cholestasis (PFIC), Biliary Atresia, Primary Sclerosing Cholangitis (PSC), Intrahepatic Cholestasis of Pregnancy (ICP), and Primary Biliary Cholangitis (PBC) [12] - MARCH Phase 3 data for PFIC is expected in Q4 2022 [12, 47, 80] - EMBARK study data for Biliary Atresia is expected in 2023 [12, 55, 80] - Interim data for VISTAS (PSC) and OHANA (ICP) are expected in Q4 2022, with VANTAGE (PBC) interim data expected in 2023 [12, 59, 80] Volixibat for Adult Cholestasis - Volixibat is being targeted for adult cholestasis, including ICP, PSC, and PBC [58, 59] - There are approximately 40,000 ICP cases per year in the US [59] - There are approximately 130,000 PBC cases per year in the US [59] Financial Status - The company reported $12.9 million net revenue in Q1 2022, with $10.9 million in net product revenue [78] - The company has a strong balance sheet with $239.9 million as of March 31, 2022 [78]

PART I. FINANCIAL INFORMATION [Item 1. Financial Statements (Unaudited)](index=4&type=section&id=Item%201.%20Financial%20Statements%20%28Unaudited%29) The company reported first Livmarli sales of **$10.9 million**, total revenue of **$12.9 million**, and a net loss of **$36.6 million** [Condensed Consolidated Balance Sheets](index=4&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Total assets decreased to **$278.1 million** from **$294.7 million**, with a corresponding reduction in liabilities Condensed Consolidated Balance Sheet Highlights (in thousands) | Balance Sheet Item | March 31, 2022 (Unaudited) | December 31, 2021 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $46,272 | $31,340 | | Short-term investments | $93,636 | $125,201 | | Total current assets | $155,268 | $166,592 | | Total assets | $278,148 | $294,651 | | **Liabilities & Equity** | | | | Total current liabilities | $34,553 | $42,596 | | Revenue interest liability, net | $132,940 | $129,923 | | Total liabilities | $169,216 | $174,439 | | Total stockholders' equity | $108,932 | $120,212 | [Condensed Consolidated Statements of Operations](index=5&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) The company reported first product sales of **$10.9 million**, total revenue of **$12.9 million**, and a net loss of **$36.6 million** Statement of Operations Summary (in thousands, except per share data) | Line Item | Three Months Ended Mar 31, 2022 | Three Months Ended Mar 31, 2021 | | :--- | :--- | :--- | | Product sales, net | $10,892 | $— | | License revenue | $2,000 | $— | | **Total revenue** | **$12,892** | **$—** | | Cost of sales | $2,424 | $— | | Research and development | $24,088 | $38,134 | | Selling, general and administrative | $19,116 | $9,479 | | **Total operating expenses** | **$45,628** | **$47,613** | | Loss from operations | ($32,736) | ($47,613) | | **Net loss** | **($36,606)** | **($50,532)** | | Net loss per share, basic and diluted | ($1.17) | ($1.68) | [Condensed Consolidated Statements of Cash Flows](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Operating cash outflow was **$39.7 million**, with net cash increasing by **$14.9 million** from investing and financing Cash Flow Summary (in thousands) | Cash Flow Activity | Three Months Ended Mar 31, 2022 | Three Months Ended Mar 31, 2021 | | :--- | :--- | :--- | | Net cash used in operating activities | ($39,669) | ($25,210) | | Net cash provided by (used in) investing activities | $36,500 | ($32,784) | | Net cash provided by financing activities | $18,104 | $6,594 | | **Net increase (decrease) in cash** | **$14,932** | **($51,409)** | [Notes to Unaudited Condensed Consolidated Financial Statements](index=9&type=section&id=Notes%20to%20Unaudited%20Condensed%20Consolidated%20Financial%20Statements) The notes detail business, accounting policies, FDA approval of Livmarli, liquidity, and financing arrangements - The company received **FDA approval for LIVMARLI® (maralixibat) on September 29, 2021**, for treating **cholestatic pruritus in Alagille syndrome (ALGS) patients** aged one year and older[29](index=29&type=chunk) - Management believes that its unrestricted cash, cash equivalents, and investments of **$139.9 million** as of March 31, 2022, are **sufficient to fund operations for at least the next twelve months**[31](index=31&type=chunk) - The company has a Revenue Interest Purchase Agreement (RIPA) with an affiliate of Oberland Capital, under which it has received **$115.0 million** in financing. In return, the Purchasers receive tiered revenue interest payments based on annual net sales of Livmarli, starting at **9.75%**[63](index=63&type=chunk)[65](index=65&type=chunk)[66](index=66&type=chunk) - In January 2022, a regulatory milestone was achieved by partner CANbridge, triggering a **$2.0 million milestone payment** to the company, which was recorded as license revenue[81](index=81&type=chunk) - During Q1 2022, the company sold **995,897 shares of common stock** through its "at the market" (ATM) sales agreement, raising net proceeds of **approximately $17.4 million**[89](index=89&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=23&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses Livmarli's commercial launch, a reduced net loss of **$36.6 million**, and sufficient liquidity - The company's lead product, Livmarli, is approved in the U.S. for **cholestatic pruritus in Alagille syndrome (ALGS) patients**. The company is also developing Livmarli for **Progressive Familial Intrahepatic Cholestasis (PFIC)** and **Biliary Atresia (BA)**, and its second product candidate, **volixibat, for adult cholestatic liver diseases**[111](index=111&type=chunk)[112](index=112&type=chunk) Results of Operations Comparison (in thousands) | | Three Months Ended Mar 31, 2022 | Three Months Ended Mar 31, 2021 | Change | | :--- | :--- | :--- | :--- | | **Total revenue** | **$12,892** | **$—** | **$12,892** | | Cost of sales | $2,424 | $— | $2,424 | | Research and development | $24,088 | $38,134 | ($14,046) | | Selling, general and administrative | $19,116 | $9,479 | $9,637 | | **Net Loss** | **($36,606)** | **($50,532)** | **$13,926** | - The **$14.0 million decrease in R&D expenses** was primarily due to a **$17.0 million decrease in license fees** (milestone payments) compared to the prior year. This was partially offset by increased clinical trial expenses for volixibat programs and higher personnel costs[149](index=149&type=chunk)[151](index=151&type=chunk) - The **$9.6 million increase in SG&A expenses** was driven by a **$4.9 million increase in personnel costs** (including **$1.4 million in stock-based compensation**), a **$2.9 million increase in professional services** for commercial launch, and a **$1.8 million increase in other administrative expenses**[150](index=150&type=chunk) - The company believes its cash, unrestricted cash equivalents, and investments will be **sufficient to fund current operations for at least the next 12 months** from the filing date of the report[155](index=155&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=32&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) Primary market risks include interest rate sensitivity, variable RIPA interest rates, and minor foreign currency exposure - The company's primary market risk is interest rate sensitivity on its cash and investment portfolio, but due to the short-term nature of its instruments, a **100-basis-point change in interest rates is not expected to have a material impact**[174](index=174&type=chunk) - The effective interest rate on the **Revenue Interest Purchase Agreement (RIPA) liability may vary based on forecasts of net product sales**, which could materially impact interest expense recognized each period[175](index=175&type=chunk) - **Foreign currency risk from operations in Switzerland and contracts denominated in Swiss Francs and Euros is not currently considered significant**[176](index=176&type=chunk) [Controls and Procedures](index=32&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded disclosure controls and procedures were effective with no material changes in internal controls - The company's management, including the CEO and CFO, concluded that as of March 31, 2022, the company's **disclosure controls and procedures were effective**[178](index=178&type=chunk) - **No changes in internal control over financial reporting occurred during the first quarter of 2022** that have materially affected, or are reasonably likely to materially affect, the company's internal controls[180](index=180&type=chunk) PART II. OTHER INFORMATION [Legal Proceedings](index=33&type=section&id=Item%201.%20Legal%20Proceedings) The company is not involved in any legal proceedings expected to materially affect its financial condition - **Management believes there are currently no pending claims or actions against the company that would have a material adverse effect** on its results of operations, financial condition, or cash flows[183](index=183&type=chunk) [Risk Factors](index=33&type=section&id=Item%201A.%20Risk%20Factors) Key risks include Livmarli commercialization, manufacturing reliance, financing, and intellectual property protection - The success of the business depends on the **continued successful commercialization of Livmarli, its only FDA-approved product**[185](index=185&type=chunk) - The company has **limited marketing and sales experience** and **relies completely on third parties for manufacturing, including sole-source suppliers**[187](index=187&type=chunk)[208](index=208&type=chunk) - The company has a **limited operating history, has incurred significant losses since inception, and expects to continue incurring losses for the foreseeable future**[217](index=217&type=chunk) - **Substantial additional financing will be needed** to continue commercialization, develop product candidates, and implement operating plans. Failure to obtain financing could force delays or elimination of programs[343](index=343&type=chunk) - The company is **dependent on intellectual property licensed from third parties**, and **termination of these licenses could result in the loss of significant rights**[331](index=331&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=84&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) No unregistered sales of equity securities occurred during the period - **None**[459](index=459&type=chunk) [Defaults Upon Senior Securities](index=84&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) This item is not applicable - **Not applicable**[460](index=460&type=chunk) [Mine Safety Disclosures](index=84&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is not applicable - **Not applicable**[461](index=461&type=chunk) [Other Information](index=84&type=section&id=Item%205.%20Other%20Information) No other information to report for the period - **None**[462](index=462&type=chunk) [Exhibits](index=85&type=section&id=Item%206.%20Exhibits) This section lists exhibits including corporate documents, CEO/CFO certifications, and XBRL data files - The exhibits filed include **CEO and CFO certifications pursuant to Sarbanes-Oxley Act Sections 302 and 906, and Inline XBRL documents**[464](index=464&type=chunk)

Corporate Presentation March 2022 Forward-Looking Statements This presentation contains "forward-looking" statements that are based on our management's beliefs and assumptions and on information currently available to management. Forward-looking statements include all statements other than statements of historical fact contained in this presentation, including information concerning our business strategy, objectives and opportunities. Forward- looking statements are subject to known and unknown risks, uncer ...

Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) Q4 2021 Earnings Conference Call March 9, 2022 4:30 PM ET Corporate Participants Ian Clements - Chief Financial Officer Chris Peetz - President, Chief Executive Officer Peter Radovich - Chief Operating Officer Pamela Vig - Chief Scientific Officer Ian Clements - Chief Financial Officer Conference Call Participants Jessica Fye - JPMorgan Mani Foroohar - SVB Leerink Ryan Deschner - Raymond James Ed Arce - H.C. Wainwright Josh Schimmer - Evercore Yasmeen Rahimi - Piper ...

(Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2021 OR UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K Delaware 83-1281555 (State or other jurisdiction of incorporation or organization) 950 Tower Lane, Suite 1050, Foster City, California 94404 (Address of principal executive offices) (Zip Code) (I.R.S. Employer Identification No.) Registrant's telephone number, including area code: (65 ...