Mirum Pharmaceuticals Inc

Previous wording

We are a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. We have three approved medicines: LIVMARLI® (maralixibat) oral solution (“Livmarli”), Cholbam® (cholic acid) capsules (“Cholbam”), and Chenodal® (chenodiol) tablets (“Chenodal”).

Current wording

We are a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. We have three approved medicines: LIVMARLI® (maralixibat) oral solution (“Livmarli”), Cholbam® (cholic acid) capsules (“Cholbam”), and Chenodal® (chenodiol) tablets (“Chenodal” or “chenodiol”).

Previous wording

Livmarli is a novel, orally administered, minimally-absorbed ileal bile acid transporter (“IBAT”) inhibitor (“IBATi”) that is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (“ALGS”) in the U.S. and various other countries around the world and for cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (“PFIC”) in the United States (“U.S.”). We market and commercialize Livmarli in the U.S. and certain countries in Europe through our specialized and focused commercial team. We have also entered into license and distribution agreements with several rare disease companies for the commercialization of Livmarli in additional countries. Additionally, we are developing Livmarli for PFIC outside of the U.S. We submitted a marketing authorization variation to the European Marketing Agency (“EMA”) in April 2023, for which we expect an approval recommendation from the Committee for Medicinal Products for Human Use (“CHMP”) in the first half of 2024, based on positive data from our MARCH Phase 3 clinical trial of Livmarli in patients with PFIC.

Current wording

Livmarli is a novel, orally administered, minimally-absorbed ileal bile acid transporter (“IBAT”) inhibitor (“IBATi”) that is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (“ALGS”) in the United States (“U.S.”) and various other countries around the world and for cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (“PFIC”) in the U.S. and and for the treatment of PFIC in the European Union (“EU”). We market and commercialize Livmarli in the U.S. and certain countries in Europe through our specialized and focused commercial team. We have also entered into license and distribution agreements with several rare disease companies for the commercialization of Livmarli in additional countries.

Previous wording

On August 31, 2023, we completed the acquisition of assets of Travere Therapeutics, Inc. (“Travere”) that are primarily related to the development, manufacture (including synthesis, formulation, finishing or packaging) and commercialization of Chenodal and Cholbam (also known as Kolbam, and together with Chenodal, the “Bile Acid Medicines”) pursuant to an asset purchase agreement dated July 16, 2023 (such acquisition, the “Bile Acid Portfolio Acquisition”). 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 peroxisome biogenesis disorder-Zellweger spectrum disorder (“PBD-ZSD”) and Smith-Lemli-Opitz syndrome (“SLOS”). Chenodal is approved for the treatment of radiolucent stones in the gallbladder and has received medical necessity recognition by the FDA for the treatment of cerebrotendinous xanthomatosis (“CTX”). We commercialize Chenodal and Cholbam in the U.S. through our specialized and focused commercial team. We have also assumed several license and distribution agreements with several rare disease companies for the commercialization of Chenodal and Cholbam in additional countries. In October 2023, we reported positive topline data from our RESTORE clinical trial evaluating Chenodal in patients with CTX. We plan to submit a new drug application for Chenodal for the treatment of CTX to the FDA in the first half of 2024.

Current wording

On August 31, 2023, we completed the acquisition of assets of Travere Therapeutics, Inc. (“Travere”) that are primarily related to the development, manufacture (including synthesis, formulation, finishing or packaging) and commercialization of Chenodal and Cholbam (also known as Kolbam, and together with Chenodal, the “Bile Acid Medicines”) pursuant to an asset purchase agreement dated July 16, 2023 (such acquisition, the “Bile Acid Portfolio Acquisition”). 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 peroxisome biogenesis disorder-Zellweger spectrum disorder (“PBD-ZSD”) and Smith-Lemli-Opitz syndrome (“SLOS”). Chenodal is approved for the treatment of radiolucent stones in the gallbladder and has received medical necessity recognition by the FDA for the treatment of cerebrotendinous xanthomatosis (“CTX”). We commercialize Chenodal and Cholbam in the U.S. through our specialized and focused commercial team. We have also assumed several license and distribution agreements with several rare disease companies for the commercialization of Chenodal and Cholbam in additional countries. In October 2023, we reported positive topline data from our RESTORE clinical trial evaluating Chenodal in patients with CTX and we submitted a new drug application for chenodiol for the treatment of CTX to the FDA in June 2024.

Previous wording

We are also advancing our product candidate, volixibat, a novel, oral, minimally-absorbed agent designed to inhibit IBAT, for the treatment of adult patients with cholestatic liver diseases. We are developing volixibat in the setting of primary sclerosing cholangitis (“PSC”) and primary biliary cholangitis (“PBC”). Volixibat has been studied in over 400 adults for up to 48 weeks. Clinical trials of volixibat have shown significant activity on IBAT and bile acid markers such as 7αC4, fecal bile acids and cholesterol, demonstrating potent biological activity. We expect to conduct an interim analysis of our VISTAS Phase 2b clinical trial in PSC and report interim data from our VANTAGE Phase 2b clinical trial in PBC, both in the first half of 2024.

Current wording

We are also advancing our product candidate, volixibat, a novel, oral, minimally-absorbed agent designed to inhibit IBAT, for the treatment of adult patients with cholestatic liver diseases. We are developing volixibat in the setting of primary sclerosing cholangitis (“PSC”) and primary biliary cholangitis (“PBC”). Volixibat has been studied in over 400 adults for up to 48 weeks. Clinical trials of volixibat have shown significant activity on IBAT and bile acid markers such as 7αC4, fecal bile acids and cholesterol, demonstrating potent biological activity. We conducted an interim analysis of our

Previous wording

Our net loss was $25.3 million and $30.1 million for the three months ended March 31, 2024 and 2023, respectively. As of March 31, 2024, we had an accumulated deficit of $581.5 million, compared to $556.2 million as of December 31, 2023. As of March 31, 2024, we had cash and cash equivalents of $302.8 million, compared to cash and cash equivalents of $286.3 million as of December 31, 2023.

Current wording

Our net loss was $24.6 million and $74.0 million for the three months ended June 30, 2024 and 2023, respectively, and $49.9 million and $104.2 million for the six months ended June 30, 2024 and 2023, respectively. As of June 30, 2024, we had an accumulated deficit of $606.2 million, compared to $556.2 million as of December 31, 2023. As of June 30, 2024, we had unrestricted cash, cash equivalents and investments of $295.4 million, compared to cash and cash equivalents of $286.3 million as of December 31, 2023.

Previous wording

We anticipate we will continue to generate losses for the foreseeable future as we continue commercial activities for our approved medicines, conduct our ongoing and planned clinical trials, seek regulatory approvals for our product candidates and make potential milestone payments to the licensors and other third parties from whom we have in-licensed or acquired our product candidates. We expect total product sales of our approved medicines will continue to increase on an annual basis, though we expect quarterly fluctuations. We have entered into collaboration arrangements with other companies whereby we are entitled to receive upfront and license fees, development and sales-based milestones, and tiered royalties based on sales of commercialized products. In select countries, we have entered into distribution agreements for the sale of Livmarli and such distributors may have fluctuating purchase levels of Livmarli. As a result, our net losses may fluctuate significantly from quarter-to-quarter and year-to-year.

Current wording

We anticipate we will continue to generate net losses for the foreseeable future as we continue commercial activities for our approved medicines, conduct our ongoing and planned clinical trials, seek regulatory approvals for our product candidates and make potential milestone payments to the licensors and other third parties from whom we have in-licensed or acquired our product candidates. We expect total product sales of our approved medicines will continue to increase on an annual basis. We have entered into license and collaboration arrangements with other companies whereby we are entitled to receive upfront and license fees, development and sales-based milestones, and tiered royalties based on sales of commercialized products. In select countries, we have entered into distribution agreements for the sale of our approved medicines and such distributors may have fluctuating purchase levels. As a result, our net losses may fluctuate significantly from quarter-to-quarter and year-to-year.

Previous wording

We concurrently entered into a transitional services agreement with Travere, pursuant to which Travere is obligated to perform certain services for a period of time, not anticipated to last beyond 12 months post-close, with respect to our use and operation of the Bile Acid Medicines.

Current wording

We concurrently entered into a transitional services agreement with Travere, pursuant to which Travere is obligated to perform certain services for a period of time with respect to our use and operation of the Bile Acid Medicines. This agreement has since expired according to its terms.

Previous wording

Under the Shire License Agreement and Assigned License Agreements, to date, we have accrued or paid aggregate development, regulatory and sales milestones of $81.0 million related to our Livmarli and volixibat programs.

Current wording

Under the Shire License Agreement and Assigned License Agreements, to date, we have accrued or paid aggregate development, regulatory and sales milestones of $91.0 million related to our Livmarli and volixibat programs.

Previous wording

We have three approved medicines: Livmarli, Cholbam and Chenodal. We expect total product sales of our approved medicines will continue to increase on an annual basis, though we expect quarterly fluctuations.

Current wording

We have three approved medicines: Livmarli, Cholbam and Chenodal. We expect total product sales of our approved medicines will continue to increase on an annual basis.

Previous wording

Our U.S. revenue from product sales, net further depends on our prescription mix of commercial payors, Medicaid and free medicines under our patient assistance program. We expect our prescription mix and resulting gross to net adjustment in the U.S. to remain consistent; however, we cannot reliably estimate it given our limited experience in the market. Our rest of world revenue from product sales, net primarily depends on our contractual obligations with our distributors and results of pricing negotiations with governmental authorities in certain European countries where we launch Livmarli and expect to launch our other product candidates, if approved. In addition, in certain countries, governments place large periodic orders. The timing of these orders can be inconsistent and can create quarter-to-quarter variation in revenue.

Current wording

Our U.S. revenue from product sales, net further depends on our prescription mix of commercial payors, Medicaid and free medicines under our patient assistance program. We expect our prescription mix and resulting gross to net adjustment in the U.S. to remain consistent. Our rest of world revenue from product sales, net primarily depends on our contractual obligations with our distributors and results of pricing negotiations with governmental authorities in certain European countries where we launch Livmarli and expect to launch our other product candidates, if approved. In addition, in certain countries, governments place large periodic orders. The timing of these orders can be inconsistent and can create quarter-to-quarter variation in revenue.

Previous wording

As of the date of our acquisition of the Bile Acid Medicines from Travere, inventory acquired was valued at its fair value. As a result, our cost of sales exceeds the historical cost to manufacture the inventory and has a negative impact on our gross margin. We expect to sell the acquired inventory within the next two years from the balance sheet date. In addition, in connection with the acquisition, we have firm commitments for the purchase of minimum order quantities for active pharmaceutical ingredients. We periodically evaluate these firm commitments to determine if these commitments are in excess of our needs. If any net loss is determined, we record a charge to cost of sales in the period identified.

Current wording

As of the date of our acquisition of the Bile Acid Medicines from Travere, inventory acquired was valued at its fair value. As a result, our cost of sales exceeds the historical cost to manufacture the inventory and has a negative impact on our gross margin. We expect to sell the majority of acquired inventory that is recorded at fair value within the next year from the balance sheet date. In addition, in connection with the acquisition, we have firm commitments for the purchase of minimum order quantities for active pharmaceutical ingredients. We periodically evaluate these firm commitments to determine if these commitments are in excess of our needs. If any net loss is determined, we record a charge to cost of sales in the period identified.

Previous wording

We incur interest expense on our convertible notes and on the RIPA. Interest on our convertible notes consists of a 4% per annum fixed rate of interest and amortization of debt discount and amortization costs. Interest on the RIPA consisted primarily of costs associated with our liability and non-cash interest costs associated with the amortization of the related debt discount and deferred issuance costs. Prior to extinguishment of the RIPA in the second quarter of 2023, we imputed interest expense associated with this liability using the effective interest rate method which is calculated based on the rate that would enable the debt to be repaid in full over the anticipated life of the arrangement.

Current wording

We incur interest expense on our convertible notes and incurred interest expense on the RIPA. Interest on our convertible notes consists of a 4% per annum fixed rate of interest and amortization of debt discount and amortization costs. Interest on the RIPA consisted primarily of costs associated with our liability and non-cash interest costs associated with the amortization of the related debt discount and deferred issuance costs. Prior to extinguishment of the RIPA in the second quarter of 2023, we imputed interest expense associated with this liability using the effective interest rate method which is calculated based on the rate that would enable the debt to be repaid in full over the anticipated life of the arrangement.

Previous wording

The preparation of financial statements and related disclosures in conformity with U.S. generally accepted accounting principles (“GAAP”) and our discussion and analysis of our financial condition and operating results require our management to make judgments, assumptions and estimates that affect the amounts reported, including the amount of assets, liabilities, expenses and the disclosure of contingent assets and liabilities. Management bases its estimates on historical experience, known trends and events, and on various other assumptions it believes to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities. Actual results may differ materially from these estimates under different assumptions or conditions.

Current wording

The preparation of financial statements and related disclosures in conformity with U.S. generally accepted accounting principles (“GAAP”) and our discussion and analysis of our financial condition and operating results require our management to make judgments, assumptions and estimates that affect the amounts reported. Management bases its estimates on historical experience, known trends and events, and on various other assumptions it believes to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities. Actual results may differ materially from these estimates under different assumptions or conditions.

Previous wording

There have been no significant changes during the three months ended March 31, 2024 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, 2024 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

Product sales, net was $68.9 million for the three months ended March 31, 2024, compared to $29.1 million for the three months ended March 31, 2023. The increase in product sales, net was a result of sales of the Bile Acid Medicines following the completion of the Bile Acid Portfolio Acquisition in August 2023, our continued commercialization of Livmarli in certain international markets directly or through partner market supply orders and in the U.S.

Current wording

Product sales, net was $77.8 million for the three months ended June 30, 2024, compared to $32.5 million for the three months ended June 30, 2023. The increase in product sales, net was a result of sales of the Bile Acid Medicines following the completion of the Bile Acid Portfolio Acquisition in August 2023 and our continued commercialization of Livmarli in the U.S. and in certain international markets directly or through partner market supply orders.

Previous wording

License and other revenue was $0.3 million for the three months ended March 31, 2024, compared to $2.5 million for the three months ended March 31, 2023. The decrease in license and other revenue was primarily due to the achievement of a regulatory milestone by GC Biopharma associated with our license agreement in 2023.

Current wording

License and other revenue was $0.1 million for the three months ended June 30, 2024, compared to $5.0 million for the three months ended June 30, 2023. The decrease in license and other revenue was primarily due to the achievement of a regulatory milestone by CANbridge Pharmaceuticals, Inc. (“CANbridge”) associated with our license agreement in 2023.

Previous wording

For the three months ended March 31, 2024, cost of sales was $17.8 million, compared to $5.0 million for the three months ended March 31, 2023. The increase in cost of sales was primarily a result of $4.1 million of amortization of acquired intangibles primarily associated with our acquisition of the Bile Acid Medicines in August 2023, increased royalties expense of $4.0 million on net sales of Livmarli and the Bile Acid Medicines under licensing agreements, increased product cost of sales of $2.9 million related to the Bile Acid Medicines and to a lesser extent for Livmarli, and other period costs of $1.4 million associated with increased commercial supply chain expenses and other general expenses.

Current wording

For the three months ended June 30, 2024, cost of sales was $20.2 million, compared to $6.8 million for the three months ended June 30, 2023. The increase in cost of sales was primarily a result of increased royalty expense of $4.6 million on net sales of Livmarli and the Bile Acid Medicines under licensing agreements, $4.3 million of amortization of acquired intangibles primarily associated with our acquisition of the Bile Acid Medicines in August 2023, increased product cost of sales of $3.1 million related to the Bile Acid Medicines and to a lesser extent for Livmarli, increased excess inventory reserves of $0.7 million and other period costs of $0.7 million associated with increased commercial supply chain expenses and other general expenses.

Previous wording

•for stock-based compensation, an increase of $1.1 million associated with incremental employee equity awards;

Current wording

•for stock-based compensation, an increase of $0.9 million associated with incremental employee equity awards;

Previous wording

•for personnel related expenses, an increase of $2.8 million related to increased employee headcount to support our development pipeline; and

Current wording

•for personnel related expenses, an increase of $1.6 million related to increased employee headcount to support our development pipeline; and

Previous wording

•for other expense, an increase of $3.3 million, primarily due to other general research and development activities.

Current wording

•for other expense, an increase of $3.1 million, primarily due to other general research and development activities.

Previous wording

Selling, general and administrative expenses were $45.6 million for the three months ended March 31, 2024, an increase of $15.4 million compared to the three months ended March 31, 2023. The increase was primarily due to an increase of $5.9 million in personnel and other compensation related expenses, including an increase of $1.7 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, an increase of $4.1 million in advertising, promotion and medical affairs expenses associated with commercial activities for our approved medicines, an increase of $3.7 million in expenses primarily related to accounting, legal, compliance, public relations and international expansion activities, and an increase of $1.4 million in other general administrative expenses.

Current wording

Selling, general and administrative expenses were $49.2 million for the three months ended June 30, 2024, an increase of $16.3 million compared to the three months ended June 30, 2023. The increase was primarily due to an increase of $7.1 million in personnel and other compensation-related expenses, including an increase of $2.3 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, an increase of $5.5 million in advertising, promotion and medical affairs expenses associated with commercial activities for our approved medicines, an increase of $3.2 million in expenses primarily related to accounting, legal, compliance, public relations and international expansion activities., and an increase of $0.5 million in other general administrative expenses.

Previous wording

Interest expense was $3.6 million for the three months ended March 31, 2024, a decrease of $0.7 million compared to the three months ended March 31, 2023. For the three months ended March 31, 2024, interest expense consisted of $3.6 million related to our convertible notes. For the three months ended March 31, 2023, interest expense of $4.2 million was related to the RIPA.

Current wording

Interest expense was $3.6 million for the three months ended June 30, 2024, a decrease of $0.2 million compared to the three months ended June 30, 2023. For the three months ended June 30, 2024, interest expense consisted of $3.6 million related to our convertible notes. For the three months ended June 30, 2023, interest expense consisted of $2.9 million related to our convertible notes issued during the period and $0.8 million related to the RIPA prior to extinguishment.

Previous wording

•for volixibat programs, an increase of $2.2 million, primarily due to increased expenses associated with conduct of the PSC and PBC trials of $3.3 million, partially offset by lower expenses of $0.8 million related to discontinuation of the intrahepatic cholestasis of pregnancy clinical trial;

Current wording

•for volixibat programs, an increase of $5.4 million, primarily due to increased expenses associated with conduct of the PSC and PBC trials of $6.2 million, partially offset by lower expenses of $0.6 million related to discontinuation of the intrahepatic cholestasis of pregnancy clinical trial;

Previous wording

Interest income was $3.6 million for the three months ended March 31, 2024, an increase of $1.4 million compared to the three months ended March 31, 2023. The increase was primarily due to higher interest earned on our cash equivalents and investment balances largely due to higher money market yields.

Current wording

Interest income was $7.1 million for the six months ended June 30, 2024, an increase of $1.2 million compared to the six months ended June 30, 2023. The increase was primarily due to higher interest earned on our cash equivalents and investment balances largely due to higher money market yields and higher invested balances.

Previous wording

Other income (expense), net was income of $1.8 million for the three months ended March 31, 2024, compared to expense of $0.8 million for the three months ended March 31, 2023. The income for the three months ended March 31, 2024 was primarily attributable to unrealized currency gains on net assets and liabilities denominated in foreign currency. The expense for the three months ended March 31, 2023 primarily related to fair value remeasurement of liabilities associated with our acquisition of Satiogen.

Current wording

Other income (expense), net was income of $2.1 million for the six months ended June 30, 2024, compared to expense of $1.1 million for the six months ended June 30, 2023. The income for the six months ended June 30, 2024 was primarily attributable to unrealized currency gains on net assets and liabilities denominated in foreign currency. The expense for the six months ended June 30, 2023 primarily related to fair value remeasurement of liabilities associated with our acquisition of Satiogen.

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 $302.8 million of cash and cash equivalents as of March 31, 2024, compared to cash and cash equivalents of $286.3 million as of December 31, 2023. Since inception, we have incurred operating losses and negative cash flows from operations. As of March 31, 2024, we had an accumulated deficit of $581.5 million, compared to $556.2 million as of December 31, 2023.

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 $295.4 million of unrestricted cash, cash equivalents and investments as of June 30, 2024, compared to cash and cash equivalents of $286.3 million as of December 31, 2023. Since inception, we have incurred operating losses and negative cash flows from operations. As of June 30, 2024, we had an accumulated deficit of $606.2 million, compared to $556.2 million as of December 31, 2023.

Previous wording

In September 2022, we filed an automatic statement on Form S-3 with the SEC (the “2022 Shelf Registration”), which became effective upon filing, pursuant to which we registered for sale from time to time in one or more offerings an unlimited amount of any combination of our common stock, preferred stock, debt securities and warrants, so long as we continue to satisfy the requirements of a “well-known seasoned issuer” under SEC rules. This automatic shelf registration statement will remain in effect for up to three years from the date it became effective. As of March 31, 2024, we have not issued any securities pursuant to the 2022 Shelf Registration. We may lose well-known seasoned issuer status in the future upon the occurrence of certain events prescribed by the Securities Act or the rules and regulations of the SEC occurring after we file this Quarterly Report if, at the time of those certain events, the worldwide market value of our voting and non-voting common equity held by our non-affiliates does not equal $700.0 million or more, calculated as of a date within 60 days prior to such events. If that were to occur and we were no longer considered a well-known seasoned issuer, we anticipate needing to amend our automatic shelf registration statement on Form S-3 prior to the occurrence of such events in order to sell securities under that Form S-3 on an ongoing basis.

Current wording

In September 2022, we filed an automatic shelf registration statement on Form S-3 with the SEC (the “2022 Shelf Registration”), which became effective upon filing, pursuant to which we registered for sale from time to time in one or more offerings an unlimited amount of any combination of our common stock, preferred stock, debt securities and warrants, so long as we continue to satisfy the requirements of a “well-known seasoned issuer” under SEC rules. This automatic shelf registration statement will remain in effect for up to three years from the date it became effective. As of June 30, 2024, we have not issued any securities pursuant to the 2022 Shelf Registration.

Previous wording

On November 2, 2023, we entered into a Sales Agreement (the “2023 Sales Agreement”) with Leerink and Cantor Fitzgerald & Co. (the “Sales Agents”), pursuant to which we may, from time to time, sell up to an aggregate amount of $200.0 million of our common stock through the Sales Agents in an “at-the-market” offering (the “ATM Offering”) pursuant to the 2022 Shelf Registration. We are not required to sell shares under the 2023 Sales Agreement. Sales of our common stock, if any, under the 2023 Sales Agreement may be made in any transactions that are deemed to be “at the market offerings” as defined in Rule 415 under the Securities Act. We will pay a given designated Sales Agent a commission of up to 3.0% of the aggregate gross proceeds of any shares of common stock sold through it pursuant to the 2023 Sales Agreement. No sales may be made under the 2023 Sales Agreement until a prospectus supplement relating to the ATM Offering is filed with the Securities and Exchange Commission.

Current wording

On November 2, 2023, we entered into a Sales Agreement (the “2023 Sales Agreement”) with Leerink and Cantor Fitzgerald & Co. (the “Sales Agents”), pursuant to which we may, from time to time, sell up to an aggregate amount of $200.0 million of our common stock through the Sales Agents in an “at-the-market” offering (the “ATM Offering”) pursuant to the 2022 Shelf Registration. We are not required to sell shares under the 2023 Sales Agreement. Sales of our common stock, if any, under the 2023 Sales Agreement may be made in any transactions that are deemed to be “at the market offerings” as defined in Rule 415 under the Securities Act. We will pay a given designated Sales Agent a commission of up to 3.0% of the aggregate gross proceeds of any shares of common stock sold through it pursuant to the 2023 Sales Agreement. As of June 30, 2024, we have not issued any securities pursuant to the 2023 Sales Agreement.

Previous wording

In August 2020, the SEC declared effective the 2020 Shelf Registration covering the sale of up to $300.0 million of our securities. Also, in August 2020, we entered into the 2020 Sales Agreement with Leerink pursuant to which we could issue and sell, in an at the market offering, from time to time, shares of common stock having an aggregate offering price of up to $75.0 million under the 2020 Shelf Registration through Leerink acting as the sales agent and/or principal. During the year ended December 31, 2023, we issued and sold 658,206 shares of common stock pursuant to the

Current wording

In August 2020, the SEC declared effective the 2020 Shelf Registration covering the sale of up to $300.0 million of our securities. Also, in August 2020, we entered into the 2020 Sales Agreement with Leerink pursuant to which we could issue and sell, in an at the market offering, from time to time, shares of common stock having an aggregate offering price of up to $75.0 million under the 2020 Shelf Registration through Leerink acting as the sales agent and/or principal. The 2020 Shelf Registration expired in August 2023, and no further sales may be made under the 2020 Sales

Previous wording

2020 Sales Agreement resulting in aggregate gross proceeds to us of $15.0 million. For the year ended December 31, 2023, the net proceeds to us after deducting sales commissions to Leerink and other issuance expenses were approximately $14.5 million. As of December 31, 2023, we have issued and sold an aggregate of 2,125,090 shares of common stock pursuant to the 2020 Sales Agreement, resulting in aggregate gross proceeds to us of $43.7 million. The 2020 Shelf Registration expired in August 2023, and no further sales may be made under the 2020 Sales Agreement.

Current wording

Agreement. Prior to the expiration of the 2020 Shelf Registration, we issued and sold an aggregate of 2,125,090 shares of common stock pursuant to the 2020 Sales Agreement, resulting in aggregate gross proceeds to us of $43.7 million.

Previous wording

Based on our current and anticipated level of operations and cash generated from sales of our approved medicines, we believe our existing cash and cash equivalents will be sufficient to fund current operations through at least the next 12 months from the filing of this Quarterly Report on Form 10-Q and beyond.

Current wording

Based on our current and anticipated level of operations and cash generated from sales of our approved medicines, we believe our existing unrestricted cash, cash equivalents and investments will be sufficient to fund current operations through at least the next 12 months from the filing of this Quarterly Report on Form 10-Q and beyond.

Previous wording

Although Livmarli has been approved for the treatment of cholestatic pruritus in patients with ALGS in patients three months of age and older and cholestatic pruritus in patients with PFIC five years of age and older in the U.S. and for the treatment of cholestatic pruritus in ALGS patients two months of age and older in Europe and we expect product revenues to increase as we continue commercial activities, Livmarli 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 ANDA and NDA for these drug products have no remaining or current patent or non-patent exclusivity. In addition, Chenodal is approved for radiolucent stones in the gallbladder yet is used primarily as standard of care for CTX. The FDA has provided a letter acknowledging Chenodal as a medical necessity for CTX, but prescriptions for this indication are not on label. As such, any change in this view from the FDA could adversely affect our ability to sell Chenodal in this indication. Unless and until Chenodal is approved for CTX, we cannot market or promote Chenodal for CTX which limits its growth potential.

Current wording

Although Livmarli has been approved for the treatment of cholestatic pruritus in patients with ALGS in the U.S. and various other countries around the world and for cholestatic pruritus in patients with PFIC in the U.S. and PFIC in the EU and we expect product revenues to increase as we continue commercial activities, Livmarli 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 ANDA and NDA for these drug products have no remaining or current patent or non-patent exclusivity. In addition, Chenodal is approved for radiolucent stones in the gallbladder yet is used primarily as standard of care for CTX. The FDA has provided a letter acknowledging Chenodal as a medical necessity for CTX, but prescriptions for this indication are not on label. As such, any change in this view from the FDA could adversely affect our ability to sell Chenodal in this indication. Unless and until Chenodal is approved for CTX, we cannot market or promote Chenodal for CTX which limits its growth potential.

Previous wording

Our principal source of liquidity is product revenue from sales of approved medicines, and, to a lesser extent, from our collaboration agreements. 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 approved medicines, and, to a lesser extent, from our license and collaboration agreements. 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.

Previous wording

If we raise additional funds through collaborations, strategic alliances or marketing, distribution or licensing arrangements with third parties, we may be required to relinquish valuable rights to our technologies, future revenue streams, research programs or product candidates or to grant licenses on terms that may not be favorable to us. As a result of adverse geopolitical and macroeconomic developments, such as the ongoing military conflicts between Ukraine and Russia and in Israel and the surrounding areas, and related sanctions, actual and anticipated changes in interest rates, economic inflation and the responses by central banking authorities to control such inflation, bank failures, the global credit and financial markets have experienced volatility and disruptions, including severely diminished liquidity and credit availability, declines in consumer confidence, declines in economic growth, increases in unemployment rates and uncertainty about economic stability. If the equity and credit markets deteriorate, it may make any necessary debt or equity financing more difficult, more costly and more dilutive. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our drug development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.

Current wording

If we raise additional funds through collaborations, strategic alliances or marketing, distribution or licensing arrangements with third parties, we may be required to relinquish valuable rights to our technologies, future revenue streams, research programs or product candidates or to grant licenses on terms that may not be favorable to us. Additionally, if the equity and credit markets deteriorate from adverse geopolitical and macroeconomic developments or otherwise, it may make any necessary debt or equity financing more difficult, more costly and more dilutive. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our drug development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.

Previous wording

In April 2023, we completed an offering of $316.3 million aggregate principal of the Notes, which includes the exercise of the initial purchasers’ option in full. The offering resulted in net proceeds of $305.3 million after deducting the initial purchasers’ discounts and commissions and offering expenses. The Notes are our senior, unsecured obligations and accrue interest at a rate of 4.00% per annum, payable semi-annually in arrears on May 1 and November 1 of each year. Our first interest payment was due on November 1, 2023. The Notes will mature on May 1, 2029, unless earlier converted, redeemed or repurchased by us. The terms of these Notes are further described in Note 10 to our unaudited condensed consolidated financial statements.

Current wording

In April 2023, we completed an offering of $316.3 million aggregate principal of the Notes, which includes the exercise of the initial purchasers’ option in full. The offering resulted in net proceeds of $305.3 million after deducting the initial purchasers’ discounts and commissions and offering expenses. The Notes are our senior, unsecured obligations and accrue interest at a rate of 4.00% per annum, payable semi-annually in arrears on May 1 and November 1 of each year. The Notes will mature on May 1, 2029, unless earlier converted, redeemed or repurchased by us. The terms of these Notes are further described in Note 10 to our unaudited condensed consolidated financial statements.

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 non-cash adjustments of $18.8 million. Non-cash adjustments consisted primarily of $11.5 million of stock-based compensation, $5.7 million of depreciation and amortization of our intangible assets and fixed assets, $2.2 million related to the cost of sale of inventory acquired in the Bile Acid Portfolio Acquisition and a $0.2 million charge associated with excess and obsolete inventory and firm commitment losses, offset by $1.7 million of unrealized foreign exchange gains. Additionally, cash provided by operating activities reflected changes in net operating assets of $21.7 million, consisting primarily of a $13.0 million decrease in accounts receivable due to timing of payments received related to sales of our approved medicines, a $11.3 million increase in accounts payable, accrued expenses and other liabilities resulting primarily from a $6.4 million increase in accrued sales deductions due to our product sales in the current quarter, and a $3.2 million increase in accrued interest expense related to the Notes issued in April 2023, a $0.9 million decrease in prepaid and other current assets and payments made for the purchase of inventory amounting to $3.2 million.

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 non-cash adjustments of $40.4 million. Non-cash adjustments consisted primarily of $23.3 million of stock-based compensation, $11.5 million of depreciation and amortization of our intangible assets and fixed assets, $4.6 million related to the cost of sale of inventory acquired in the Bile Acid Portfolio Acquisition and a $1.7 million charge associated with excess and obsolete inventory and firm commitment losses, offset by $2.1 million of unrealized foreign exchange gains. Additionally, cash provided by operating activities reflected changes in net operating assets of $20.9 million, consisting primarily of a $7.5 million decrease in accounts receivable due to timing of payments received related to sales of our approved medicines, a $18.2 million increase in accounts payable, accrued expenses and other liabilities resulting primarily from a $13.6 million increase in accrued sales deductions due to our product sales in the six months ended June 30, 2024, a $2.3 million decrease in prepaid and other current assets and payments made for the purchase of inventory amounting to $6.6 million.

Previous wording

Net cash used in operating activities was $18.8 million for the three months ended March 31, 2023, reflecting our net loss of $30.1 million partially offset by non-cash items of $14.0 million. Non-cash items consisted primarily of $8.6 million of stock-based compensation, $4.2 million of effective interest expense in connection with the RIPA, $1.3 million of depreciation and amortization of our intangible assets and fixed assets, $0.8 million net accretion of discounts on our investments and $0.5 million expense related to the change in fair value of liabilities arising from our acquisition of Satiogen having to do with certain purchase price adjustments and indemnification obligations and common stock to be issued as contingent consideration upon achievement of a certain milestone. Additionally, cash used in operating activities reflected changes in net operating assets of $2.6 million, consisting primarily of a $1.2 million decrease in accounts payable, accrued expenses and other liabilities, a $0.7 million increase in accounts receivable related to sales of Livmarli, a $0.8 million increase in inventory, a $0.3 million decrease in other assets and a $0.2 million decrease in operating lease liability.

Current wording

Net cash used in operating activities was $36.1 million for the six months ended June 30, 2023, reflecting our net loss of $104.2 million partially offset by non-cash items of $74.2 million. Non-cash items consisted primarily of $16.9 million of stock-based compensation, $5.1 million of effective interest expense in connection with the RIPA, $2.7 million of depreciation and amortization of our intangible assets and fixed assets, $0.8 million net accretion of discounts on our investments and $0.5 million expense related to the change in fair value of liabilities arising from our acquisition of Satiogen having to do with certain purchase price adjustments and indemnification obligations and common stock to be issued as contingent consideration upon achievement of a certain milestone. Additionally, cash used in operating activities reflected changes in net operating assets of $6.1 million, consisting primarily of a $8.0 million decrease in accounts payable, accrued expenses and other liabilities, a $11.3 million increase in accounts receivable related to sales of Livmarli, a $1.8 million increase in inventory, a $0.2 million decrease in other assets and a $0.4 million decrease in operating lease liability.

Previous wording

Net cash provided by investing activities was $27.0 million for the three months ended March 31, 2023, primarily due to proceeds of $64.5 million from maturities of investments, partially offset by $22.4 million used in purchases of investments and $15.0 million for a milestone payment associated with approval of Livmarli by the European Commission for the treatment of cholestatic pruritus in patients with ALGS two months of age and older.

Current wording

Net cash provided by investing activities was $77.6 million for the six months ended June 30, 2023, primarily due to proceeds of $120.0 million from maturities of investments, partially offset by $27.3 million used in purchases of investments and $15.0 million for a milestone payment associated with approval of Livmarli by the European Commission for the treatment of cholestatic pruritus in patients with ALGS two months of age and older.

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.

Previous wording

Net cash provided by financing activities was $13.0 million for the three months ended March 31, 2023, primarily due to net proceeds of $14.5 million from the issuance and sale of common stock under the Sales Agreement with Leerink, pursuant to which we issued and sold an aggregate of 658,206 shares of common stock at a weighted-average price of $22.79 per share, and proceeds of $1.4 million from employee equity award exercises, partially offset by $2.9 million of revenue interest payments made under the RIPA.

Current wording

Net cash provided by financing activities was $127.7 million for the six months ended June 30, 2023, primarily due to net proceeds of $305.3 million from the issuance of convertible notes, net proceeds of $14.5 million from the issuance and sale of common stock under the Sales Agreement with Leerink, pursuant to which we issued and sold an aggregate of 658,206 shares of common stock at a weighted-average price of $22.79 per share, and proceeds of $3.5 million from employee equity award exercises, partially offset by $195.6 million of revenue interest payments made under the RIPA.