Mirum Pharmaceuticals, Inc.

Previous wording

On October 22, 2024, we completed a license agreement with Enthorin Therapeutics, LLC and Dart Neuroscience LLC granting us the worldwide right to develop and commercialize MRM-3379, an allosteric inhibitor of Phosphodiasterase 4d (“PDE4D”). We intend to develop MRM-3379 for the treatment of Fragile-X Syndrome (“FXS”) and expect to initiate a Phase 2 clinical trial in 2025.

Current wording

On October 22, 2024, we completed a license agreement with Enthorin Therapeutics, LLC and Dart Neuroscience LLC granting us the worldwide right to develop and commercialize MRM-3379, an allosteric inhibitor of Phosphodiesterase 4D (“PDE4D”). We intend to develop MRM-3379 for the treatment of Fragile-X Syndrome (“FXS”) and expect to initiate a Phase 2 clinical trial in the fourth quarter of 2025.

Previous wording

The FDA approved Cholbam in March 2015, as the first FDA-approved treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects, and for adjunctive treatment of patients with peroxisomal disorders, including peroxisome biogenesis disorder-Zellweger spectrum disorder (“PBD-ZSD”). Chenodiol is standard of care for the treatment of cerebrotendinous xanthomatosis (“CTX”) in the United States with a medical necessity recognition by the FDA and was commercialized under the brand name Chenodal. We submitted a new drug application (“NDA”) for chenodiol for the treatment of CTX in 2024 and received FDA approval for the treatment of adults with CTX in February 2025, which will be commercialized under the brand name Ctexli. We currently commercialize Chenodal and Cholbam, and plan to commercialize Ctexli, in the U.S. through our specialized and focused commercial team. We have also assumed license and distribution agreements with several rare disease companies for the commercialization of Cholbam and chenodiol in additional countries.

Current wording

The FDA approved Cholbam in March 2015, as the first FDA-approved treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects, and for adjunctive treatment of patients with peroxisomal disorders, including peroxisome biogenesis disorder-Zellweger spectrum disorder (“PBD-ZSD”). Chenodiol is standard of care for the treatment of cerebrotendinous xanthomatosis (“CTX”) in the United States with a medical necessity recognition by the FDA and was commercialized under the brand name Chenodal. We submitted a new drug application (“NDA”) for chenodiol for the treatment of CTX in 2024 and received FDA approval for the treatment of adults with CTX in February 2025, which is commercialized under the brand name Ctexli. We currently commercialize Cholbam and Ctexli in the U.S. through our specialized and focused commercial team. We have also assumed license and distribution agreements with several rare disease companies for the commercialization of Cholbam and chenodiol in additional countries.

Previous wording

Our net loss was $14.7 million and $25.3 million for the three months ended March 31, 2025 and 2024, respectively. As of March 31, 2025, we had an accumulated deficit of $658.9 million, compared to $644.2 million as of December 31, 2024. As of March 31, 2025, we had unrestricted cash, cash equivalents and investments of $298.6 million, compared to unrestricted cash, cash equivalents and investments of $292.8 million as of December 31, 2024.

Current wording

Our net loss was $5.9 million and $24.6 million for the three months ended June 30, 2025 and 2024, respectively, and $20.5 million and $49.9 million for the six months ended June 30, 2025 and 2024, respectively. As of June 30, 2025, we had an accumulated deficit of $664.7 million, compared to $644.2 million as of December 31, 2024. As of June 30, 2025, we had unrestricted cash, cash equivalents and investments of $321.7 million, compared to unrestricted cash, cash equivalents and investments of $292.8 million as of December 31, 2024.

Previous wording

Our U.S. revenue from product sales, net further depends on our prescription mix of commercial payors, Medicaid and amounts of free medicines provided under our patient assistance program. We expect our prescription mix and resulting gross to net adjustment in the U.S. to remain consistent. Our revenue from product sales is recognized when the control of the product is transferred. Under our license agreement with Takeda as well as agreements with distributors, we may receive large periodic orders for our products. The timing of these orders can be inconsistent and can create significant quarter-to-quarter variation in product sales.

Current wording

Our U.S. revenue from product sales, net further depends on our prescription mix of commercial payors, Medicaid and amounts of free medicines provided under our patient assistance program. We expect our prescription mix and resulting gross to net adjustment in the U.S. to remain materially consistent. Our revenue from product sales is recognized when the control of the product is transferred. Under our license agreement with Takeda as well as agreements with distributors, we may receive large periodic orders for our products. The timing of these orders can be inconsistent and can create significant quarter-to-quarter variation in product sales. In addition, we recognize our best estimate of the consideration that we expect to receive when control of the inventory is transferred to our licensed partners and distributors. Such estimates may be complex and include estimates as to if and when our distributors and licensed partner’s sales in the market will occur. Estimates are reviewed and updated quarterly as additional information, including in-market pricing and sales information of our authorized distributors and licensed partners, becomes known which may cause variability of quarterly revenue particularly during periods of product launch.

Previous wording

Although we expect product revenues to increase as we continue commercial activities for our approved medicines, we may not achieve commercial success. Certain of our approved medicines, including the Bile Acid Medicines, are subject to immediate competition from compounded and generic entrants, as the abbreviated new drug application (“ANDA”) and NDA for these drug products have no remaining or current patent exclusivity. Chenodiol is standard of care for the treatment of CTX in the U.S. with a medical necessity recognition by the FDA. We submitted an

Current wording

Although we expect product revenues to increase as we continue commercial activities for our approved medicines, we may not achieve commercial success. Certain of our approved medicines, including the Bile Acid Medicines, are subject to immediate competition from compounded and generic entrants, as the abbreviated new drug application (“ANDA”) and NDA for these drug products have no remaining or current patent exclusivity. Chenodiol is standard of care for the treatment of CTX in the U.S. and was commercialized with a medical necessity recognition by the FDA until February 2025. We submitted an NDA for chenodiol for the treatment of CTX in 2024 and received FDA approval for the treatment of adults with CTX in February 2025, which is now commercialized under the brand name Ctexli. The FDA has granted orphan exclusivity for chenodiol for the treatment of CTX.

Previous wording

There have been no significant changes during the three months ended March 31, 2025, except as described below, in our critical accounting policies and estimates as compared to the critical accounting policies and estimates disclosed in the section titled “Management’s Discussion and Analysis of Financial Condition and Results of Operations” included in our Annual Report.

Current wording

There have been no significant changes during the three and six months ended June 30, 2025, except as described below, in our critical accounting policies and estimates as compared to the critical accounting policies and estimates disclosed in the section titled “Management’s Discussion and Analysis of Financial Condition and Results of Operations” included in our Annual Report.

Previous wording

Revenues from direct product sales are recorded net of reserves established for applicable discounts and allowances that are offered within contracts with our customers, including amounts from payors and other third parties on behalf of our customers. For revenues from distributors and our licensed partner, Takeda, we record net product sales at the time control of the product is transferred, based on the estimated variable consideration to be received. The transaction price, which may include fixed or variable consideration may be subject to constraint and is included in the product sales price only to the extent that it is probable that a significant reversal of the amount of the cumulative revenue recognized will not occur in a future period. We recognize our best estimate of the consideration that we expect to receive when control of the inventory is transferred to our customer and revenue is recognized. Such estimates may be complex and include estimates as to if and when our distributors and licensed partner’s sales in the market will occur. Estimates are reviewed and updated quarterly as additional information, including in-market sales information of our authorized distributors and licensed partners, becomes known. Actual amounts may ultimately differ from our estimates. If actual results vary, we adjust these estimates, which could have an effect on earnings in the period of adjustment.

Current wording

Revenues from direct product sales are recorded net of reserves established for applicable discounts and allowances that are offered within contracts with our customers, including amounts from payors and other third parties on behalf of our customers. For revenues from distributors and our licensed partner, Takeda, we record net product sales at the time control of the product is transferred, based on the estimated variable consideration to be received. The transaction price, which may include fixed or variable consideration may be subject to constraint and is included in the product sales price only to the extent that it is probable that a significant reversal of the amount of the cumulative revenue recognized will not occur in a future period. We recognize our best estimate of the consideration that we expect to receive when control of the inventory is transferred to our customer and revenue is recognized. For our distributor and license partner sales, such estimates may be more complex and include estimates as to if and when our distributors and licensed partner’s sales in the market will occur. These estimates are reviewed and updated quarterly as additional information, including in-market sales information of our authorized distributors and licensed partners, becomes known. Actual amounts may ultimately differ from our estimates. If actual results vary, we adjust these estimates, which could have an effect on earnings in the period of adjustment.

Previous wording

•for volixibat programs, an increase of $5.1 million, primarily due to increased expenses associated with conduct of the PSC and PBC trials;

Current wording

•for volixibat programs, an increase of $4.7 million, primarily due to increased expenses associated with conduct of the PSC and PBC trials as well as manufacturing development expenses;

Previous wording

Selling, general and administrative expenses were $57.7 million for the three months ended March 31, 2025, an increase of $12.1 million compared to the three months ended March 31, 2024. The increase was primarily due to increases of $7.7 million in personnel and other compensation-related expenses, including an increase of $2.7 million in stock-based compensation, reflecting an increase in the number of our selling, marketing and administrative employees to support commercial activities for our approved medicines, $2.6 million in advertising, promotion and medical affairs expenses associated with commercial activities primarily driven by the Bile Acid Medicines and PFIC, and $1.6 million in other general administrative expenses.

Current wording

Selling, general and administrative expenses were $63.3 million for the three months ended June 30, 2025, an increase of $14.1 million compared to the three months ended June 30, 2024. The increase was primarily due to increases of $8.9 million in personnel and other compensation-related expenses, including an increase of $2.6 million in stock-based compensation, reflecting an increase in the number of our selling, marketing and administrative employees to support commercial activities for our approved medicines, $2.6 million in general administrative expenses, $1.7 million in advertising, promotion and medical affairs expenses associated with commercial activities, and $0.9 million of expenses associated with post marketing approval studies.

Previous wording

Interest income was $3.0 million for the three months ended March 31, 2025, a decrease of $0.6 million compared to the three months ended March 31, 2024 largely due to lower yields on investments.

Current wording

Interest income was $3.0 million for the three months ended June 30, 2025, a decrease of $0.5 million compared to the three months ended June 30, 2024 largely due to lower yields on investments.

Previous wording

Product sales, net was $111.6 million for the three months ended March 31, 2025, compared to $68.9 million for the three months ended March 31, 2024. The increase in product sales, net was a result of our continued commercialization of Livmarli in the U.S. for the treatment of ALGS and PFIC, and in certain international markets directly or through distributor and partner orders and from our sales of the Bile Acid Medicines.

Current wording

Product sales, net was $239.4 million for the six months ended June 30, 2025, compared to $146.7 million for the six months ended June 30, 2024. The increase in product sales, net was a result of our continued commercialization of Livmarli in the U.S. for the treatment of ALGS and PFIC, and in certain international markets directly or through distributor and partner orders and from our sales of the Bile Acid Medicines.

Previous wording

For the three months ended March 31, 2025, cost of sales was $23.0 million, compared to $17.8 million for the three months ended March 31, 2024. The increase in cost of sales was primarily a result of increases in royalty expense of $3.5 million on net sales of Livmarli and the Bile Acid Medicines under licensing agreements and a $2.0 million increase primarily associated with excess purchase commitments for APIs associated with Ctexli. These increases were offset by lower product cost of sales of $1.6 million primarily related to the Bile Acid Medicines as we substantially completed the sale of acquired inventory in prior periods which had been recorded at fair value.

Current wording

For the six months ended June 30, 2025, cost of sales was $46.4 million, compared to $38.1 million for the six months ended June 30, 2024. The increase in cost of sales was primarily a result of increases in royalty expense of $7.3 million on net sales of Livmarli and the Bile Acid Medicines under licensing agreements, a $1.3 million increase primarily associated with increased PDUFA fees associated with the approval of our solid dose formulation in Livmarli and higher commercial supply chain costs of $2.1 million. These increases were partially offset by lower product cost of sales of $4.0 million primarily related to the Bile Acid Medicines, as we substantially completed the sale of acquired inventory in prior periods which had been recorded at fair value.

Previous wording

•for personnel related expenses, an increase of $2.2 million related primarily to increased employee headcount to support our development pipeline; and

Current wording

•for personnel related and stock-based compensation expenses, an increase of $13.8 million related primarily to increased employee headcount and related equity award grants to support our development pipeline; and

Previous wording

•for license fees, an increase of $5.0 million due to a development milestone payment associated with our EXPAND study.

Current wording

•for license fees, an increase of $5.0 million due to a development milestone payment associated with our EXPAND study, partially offset by

Previous wording

Since inception, we have funded our operations primarily through debt, equity, revenue interest financings and, to a lesser extent, cash from our product sales and license and collaboration revenue. We had $298.6 million of unrestricted cash, cash equivalents and investments as of March 31, 2025, compared to unrestricted cash, cash equivalents and investments of $292.8 million as of December 31, 2024. Since inception, we have incurred operating losses. As of March 31, 2025, we had an accumulated deficit of $658.9 million, compared to $644.2 million as of December 31, 2024.

Current wording

Since inception, we have funded our operations primarily through debt, equity, revenue interest financings and, to a lesser extent, cash from our product sales and license and collaboration revenue. We had $321.7 million of unrestricted cash, cash equivalents and investments as of June 30, 2025, compared to unrestricted cash, cash equivalents and investments of $292.8 million as of December 31, 2024. Since inception, we have incurred operating losses. As of June 30, 2025, we had an accumulated deficit of $664.7 million, compared to $644.2 million as of December 31, 2024.

Previous wording

Our principal source of liquidity is product revenue from sales of approved medicines. Until such time, if ever, as we can generate sufficient product revenue from sales of our approved medicines, our current product candidates or any future product candidates, if approved, to fund operations, we expect to finance our cash needs through a combination of equity offerings, debt financings and potential collaboration, license or development agreements. Our primary cash needs are for day-to-day operations and to fund our working capital requirements. To the extent that we raise additional capital through the sale of equity or convertible debt securities, ownership interest will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect rights as a stockholder. Debt financing and preferred equity financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends.

Current wording

Our principal source of liquidity is product revenue from sales of our approved medicines. In recent quarters, liquidity from product revenues has been sufficient to fund current operations. There can be no assurances that future revenues will continue to be sufficient to fund operations. Should product revenues from our currently approved medicines, our current product candidates or any future product candidates, if approved, be insufficient to fund operations, we would expect to finance our cash needs through a combination of equity offerings, debt financings and potential collaboration, license or development agreements. Our primary cash needs are for day-to-day operations and to fund our working capital requirements. To the extent that we raise additional capital through the sale of equity or convertible debt securities, ownership interest will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect rights as a stockholder. Debt financing and preferred equity financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends.

Previous wording

During the first quarter of 2025, the last reported sale price of our common stock exceeded 130% of the conversion price of the Notes for more than 20 trading days during the 30 consecutive trading days ended March 31, 2025. As a result, the Notes are convertible at the option of the holders of the Notes during the second quarter of 2025, the quarter immediately following the quarter when the conditions were met, as stated in the terms of the Notes. If holders of the Notes elect to convert their Notes, we may elect to settle such conversions by paying or delivering, as applicable, cash, shares of our common stock or a combination of cash and shares of our common stock.

Current wording

During the second quarter of 2025, the last reported sale price of our common stock exceeded 130% of the conversion price of the Notes for more than 20 trading days during the 30 consecutive trading days ended June 30, 2025. As a result, the Notes are convertible at the option of the holders of the Notes during the third quarter of 2025, the quarter immediately following the quarter when the conditions were met, as stated in the terms of the Notes. If holders of the Notes elect to convert their Notes, we may elect to settle such conversions by paying or delivering, as applicable, cash, shares of our common stock or a combination of cash and shares of our common stock.

Previous wording

Net cash used in operating activities was $2.0 million for the three months ended March 31, 2025, reflecting our net loss of $14.7 million offset by adjustments of $24.0 million. Adjustments consisted primarily of stock-based compensation, depreciation and amortization of our intangible assets and fixed assets, and charges associated with excess and obsolete inventory and firm commitment losses. Additionally, cash used in operating activities reflected changes in net operating assets of $11.3 million, primarily related to an increase in accounts receivable and payments made for accrued compensation and related benefits during the three months ended March 31, 2025. The net use of cash was offset by an increase in accrued sales deductions and royalties due to the growth from our product sales in the three months ended March 31, 2025, an increase in accrued interest in relation to the Notes and an increase in accrued milestone payments for the achievement of a maralixibat development milestone during the period.

Current wording

Net cash provided by operating activities was $10.1 million for the six months ended June 30, 2025, reflecting our net loss of $20.5 million offset by adjustments of $48.5 million. Adjustments consisted primarily of stock-based compensation, depreciation and amortization of our intangible assets and fixed assets, and charges associated with excess and obsolete inventory and firm commitment losses. Additionally, cash provided by operating activities reflected a cash outflow for changes in net operating assets of $17.9 million, primarily related to an increase in accounts receivable and payments made for inventory, prepaid assets and accrued compensation and related benefits partially offset by an increase in accrued sales deductions and royalties due to the growth from our product sales in the six months ended June 30, 2025 and an increase in accrued clinical trial and contract manufacturing expenses during the period.

Previous wording

Net cash provided by operating activities was $15.2 million for the three months ended March 31, 2024, reflecting our net loss of $25.3 million partially offset by adjustments of $16.5 million. Adjustments consisted primarily of stock-based compensation, and depreciation and amortization of our intangible assets and fixed assets. Additionally, cash provided by operating activities reflected changes in net operating assets of $23.9 million, consisting primarily of a decrease in accounts receivables due to timing of payments received related to sales of our approved medicines, an increase in accounts payable, accrued expenses and other liabilities primarily related to the increase in accrued sales deductions and accrued royalties payable due to our increased net product sales, and an increase in accrued interest expense related to the Notes issued in April 2023.

Current wording

Net cash provided by operating activities was $11.4 million for the six months ended June 30, 2024, reflecting our net loss of $49.9 million partially offset by adjustments of $35.8 million. Adjustments consisted primarily of stock-based compensation, and depreciation and amortization of our intangible assets and fixed assets. Additionally, cash provided by operating activities reflected changes in net operating assets of $25.5 million, consisting primarily of a decrease in accounts receivables due to timing of payments received related to sales of our approved medicines, and an increase in accounts payable, accrued expenses and other liabilities primarily related to the increase in accrued sales deductions due to our product sales in the six months ended June 30, 2024.

Previous wording

Net cash used in investing activities was $16.1 million for the three months ended March 31, 2025, primarily due to purchases of investments offset by proceeds from maturities of investments.

Current wording

Net cash used in investing activities was $22.7 million for the six months ended June 30, 2025, primarily due to purchases of investments offset by proceeds from maturities of investments.

Previous wording

Net cash provided by financing activities was $6.4 million for the three months ended March 31, 2025, due to proceeds from employee equity award exercises.

Current wording

Net cash provided by financing activities was $15.0 million for the six months ended June 30, 2025, due to proceeds from employee equity award exercises.

Previous wording

Net cash provided by financing activities was $1.2 million for the three months ended March 31, 2024, due to proceeds from employee equity award exercises.

Current wording

Net cash provided by financing activities was $8.5 million for the six months ended June 30, 2024, due to proceeds from employee equity award exercises.