Ultragenyx Pharmaceutical Inc

Previous wording

Ultragenyx Pharmaceutical Inc., we or the Company, is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultrarare genetic diseases. We have built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

Current wording

Ultragenyx Pharmaceutical Inc., we or the Company, is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. We have built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

Previous wording

Approved Products and Clinical Product Candidates

Current wording

Approved Therapies and Clinical Product Candidates

Previous wording

Evkeeza is a fully human monoclonal antibody administered by IV, that binds to and blocks the function of angiopoietin-like 3, or ANGPTL3, a protein that plays a key role in lipid metabolism, developed for the treatment of HoFH, a rare inherited condition. HoFH occurs when two copies of the genes causing familial hypercholesterolemia are inherited, one from each parent, resulting in dangerously high levels (>400 mg/dL) of low-density lipoprotein-cholesterol, or LDL-C, which is bad cholesterol. Patients with HoFH are at risk for premature atherosclerotic disease and cardiac events as early as their teenage years. Evkeeza is approved in the U.S., where it is marketed by our partner Regeneron Pharmaceuticals, or Regeneron. It is also approved in the European Economic Area, or EEA, Brazil and Japan as a first-in-class therapy for use together with diet and other LDL-C lowering therapies. In these regions, it is generally approved to treat adults and adolescents aged five years and older with clinical HoFH. There are approximately 3,000 to 5,000 patients with HoFH in the developed world outside of the U.S.

Current wording

Evkeeza is a fully human monoclonal antibody administered by IV, that binds to and blocks the function of angiopoietin-like 3, or ANGPTL3, a protein that plays a key role in lipid metabolism, developed for the treatment of HoFH, a rare inherited condition. HoFH occurs when two copies of the genes causing familial hypercholesterolemia are inherited, one from each parent, resulting in dangerously high levels (>400 mg/dL) of low-density lipoprotein-cholesterol, or LDL-C, which is bad cholesterol. Patients with HoFH are at risk for premature atherosclerotic disease and cardiac events as early as their teenage years. Evkeeza is approved in the U.S., where it is marketed by our partner Regeneron Pharmaceuticals, or Regeneron. It is also approved in the European Economic Area, or EEA, Brazil, Mexico, and Japan as a first-in-class therapy for use together with diet and other LDL-C lowering therapies. In these regions, Evkeeza is generally approved to treat adults and adolescents aged five years and older with clinical HoFH. There are approximately 3,000 to 5,000 patients with HoFH in the developed world outside of the U.S.

Previous wording

UX143 (setrusumab) is a fully human monoclonal antibody administered by IV that inhibits sclerostin, a protein that acts on a key bone-signaling pathway by inhibiting the activity of bone-forming cells and promoting bone resorption. Setrusumab is being developed for the treatment of OI, or brittle bone disease, which is caused by variants in the COL1A1 or COL1A2 genes, leading to either reduced or abnormal collagen and changes in bone metabolism. There are an estimated 60,000 patients in the developed world affected by OI. UX143 has received orphan drug designation from the U.S. Food and Drug Administration, or FDA, and European Medicines Agency, or EMA, Rare Pediatric Disease designation and Breakthrough Designation from the FDA, and was accepted into the EMA’s Priority Medicines program, or PRIME, program. Setrusumab is subject to our collaboration agreement with Mereo and is the lead clinical asset in our bone endocrinology franchise.

Current wording

UX143 (setrusumab) is a fully human monoclonal antibody administered by IV that inhibits sclerostin, a protein that acts on a key bone-signaling pathway by inhibiting the activity of bone-forming cells and promoting bone resorption. Setrusumab is being developed for the treatment of OI, or brittle bone disease, which is caused by variants in the COL1A1 or COL1A2 genes, leading to either reduced or abnormal collagen and changes in bone metabolism. There are an estimated 60,000 patients in the developed world affected by OI. UX143 has received orphan drug designation from the U.S. Food and Drug Administration, or FDA, and European Medicines Agency, or EMA, Rare Pediatric Disease designation and Breakthrough Designation from the FDA, and was accepted into the EMA’s Priority Medicines, or PRIME, program. Setrusumab is subject to our collaboration agreement with Mereo and is the lead clinical asset in our bone endocrinology franchise.

Previous wording

GTX-102 is an antisense oligonucleotide, or ASO, administered by intrathecal injection (IT) that inhibits expression of the paternal UBE3A antisense. GTX-102 is being developed for the treatment of Angelman syndrome, a debilitating and rare neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene. There are an estimated 60,000 patients in the developed world affected by Angelman syndrome. GTX-102 has received Fast Track Designation, Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA and has been accepted into the EMA’s PRIME program.

Current wording

GTX-102 is an antisense oligonucleotide, or ASO, administered by intrathecal injection that inhibits expression of the paternal UBE3A antisense. GTX-102 is being developed for the treatment of Angelman syndrome, a debilitating and rare neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene. There are an estimated 60,000 patients in the developed world affected by Angelman syndrome. GTX-102 has received Fast Track Designation, Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA and has been accepted into the EMA’s PRIME program.

Previous wording

UX701 is an AAV type 9 gene therapy, administered by a one-time IV infusion that is designed to deliver a truncated form of the ATP7B gene. UX701 is being developed for the treatment of patients with Wilson disease, which affects more than 50,000 patients in the developed world. UX701 has received Orphan Drug Designation in the U.S. and in the EU. UX701 has received a Fast Track Designation from the FDA.

Current wording

UX701 is an AAV type 9 gene therapy, administered by a one-time IV infusion that is designed to deliver a truncated form of the ATP7B gene. UX701 is being developed for the treatment of patients with Wilson disease, which affects approximately 50,000 patients in the developed world. UX701 has received Orphan Drug Designation in the U.S. and in the EU. UX701 has received a Fast Track Designation from the FDA.

Previous wording

As previously announced, in July 2024, we successfully completed an End of Phase 2 meeting with the FDA supporting our Phase 3 study plans for GTX-102. The pivotal Aspire Phase 3 study will be a global, randomized, double-blind, sham-controlled trial and will include a 48-week primary efficacy analysis period enrolling approximately 120 patients with Angelman syndrome with a genetically confirmed diagnosis of full maternal UBE3A gene deletion. The primary endpoint will be improvement in cognition assessed by Bayley-4 cognitive raw score. It will also include the key secondary endpoint of the Multi-domain Responder Index, or MDRI, across all five domains of cognition, receptive communication, behavior, gross motor function, and sleep. Individual secondary endpoints were also discussed and aligned on with the FDA for the domains of communication, behavior, motor function and sleep. We also discussed with the FDA plans to initiate the Aurora study, an additional, open-label clinical study to evaluate the safety and efficacy of GTX-102 for the treatment of patients with other Angelman syndrome genotypes and in other age groups.

Current wording

We are currently randomizing and dosing patients in Aspire, the 48-week Phase 3 study of GTX-102. Approximately 120 patients, between four and 17 years of age, with a genetically confirmed diagnosis of full maternal UBE3A gene deletion will be enrolled and randomized 1:1 to the GTX-102 or sham comparator group. The primary endpoint will be improvement in cognition assessed by Bayley-4 cognitive raw score, and the key secondary endpoint will be the Multi-domain Responder Index (MDRI) across the five domains of cognition, receptive communication, behavior, gross motor function, and sleep. We expect to complete enrollment for the Phase 3 study in the second half of 2025.

Previous wording

In May 2024, we announced positive topline results from our Phase 3 GlucoGene study for the treatment of patients aged eight years and older. The study achieved its primary endpoint, demonstrating that treatment with DTX401 resulted in a statistically significant and clinically meaningful reduction in daily cornstarch intake compared with placebo at Week 48.

Current wording

In May 2024, we previously disclosed that the Phase 3 GlucoGene study for the treatment of patients aged eight years and older with GSDIa achieved its primary endpoint, demonstrating that treatment with DTX401 resulted in a statistically significant and clinically meaningful reduction in daily cornstarch intake compared with placebo at Week 48. At Week 48, patients entered a 48-week Crossover Period where patients previously treated with placebo were treated with DTX401. In May 2025 we announced data from this Crossover Period, that demonstrated greater reductions in total daily cornstarch were observed in both the ongoing DTX401 group (-60%) with a mean follow-up of 120 weeks and the Crossover Placebo to DTX401 group (-64%) with a mean duration on therapy with DTX401 of 69 weeks. As of the data cut-off, glycemic control was maintained in participants treated with DTX401 despite significant reductions in daily cornstarch intake. DTX401 has demonstrated a consistent and acceptable safety profile with no new safety signals identified as of the data cut-off.

Previous wording

We have incurred net losses in each year since inception. Our net loss was $133.5 million and $435.8 million for the three and nine months ended September 30, 2024, respectively, and $159.6 million and $483.4 million for the three and nine months ended September 30, 2023, respectively. Substantially all of our net losses have resulted from costs incurred in connection with our research and development programs and from selling, general and administrative costs associated with our operations.

Current wording

We have incurred net losses in each year since inception. Our net losses were $151.1 million and $170.7million for the three months ended March 31, 2025 and 2024, respectively. Substantially all of our net losses have resulted from costs incurred in connection with our research and development programs and from selling, general and administrative costs associated with our operations.

Previous wording

Our total revenues were $139.5 million and $395.4 million for the three and nine months ended September 30, 2024, respectively, and $98.1 million and $306.9 million for the three and nine months ended September 30, 2023, respectively. The increase in revenue was largely driven by increases in demand for our approved products.

Current wording

Our total revenues were $139.3 million and $108.8 million for the three months ended March 31, 2025 and 2024, respectively. The increase in revenue was largely driven by an increase in demand for our approved products.

Previous wording

As of September 30, 2024, we had $824.7 million in available cash, cash equivalents, and marketable debt securities.

Current wording

As of March 31, 2025, we had $563.0 million in available cash, cash equivalents, and marketable debt securities.

Previous wording

Our management’s discussion and analysis of our financial condition and results of operations is based on our Condensed Consolidated Financial Statements, which have been prepared in accordance with U.S. generally accepted accounting principles. The preparation of these Condensed Consolidated Financial Statements requires us to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the financial statements, as well as the reported expenses incurred during the reporting periods. Our estimates are based on our historical experience and on various other factors that we believe are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying value of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions. There have been no material changes in our critical accounting policies during the three and nine months ended September 30, 2024, as compared to those disclosed in “Management’s Discussion and Analysis of Financial Condition and Results of Operations — Critical Accounting Policies and Significant Judgments and Estimates” in our Annual Report.

Current wording

Our management’s discussion and analysis of our financial condition and results of operations is based on our Condensed Consolidated Financial Statements, which have been prepared in accordance with U.S. generally accepted accounting principles. The preparation of these Condensed Consolidated Financial Statements requires us to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the financial statements, as well as the reported expenses incurred during the reporting periods. Our estimates are based on our historical experience and on various other factors that we believe are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying value of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions. There have been no material changes in our critical accounting policies during the three months ended March 31, 2025, as compared to those disclosed in “Management’s Discussion and Analysis of Financial Condition and Results of Operations — Critical Accounting Policies and Significant Judgments and Estimates” in our Annual Report.

Previous wording

Comparison of the three and nine months ended September 30, 2024 to the three and nine months ended September 30, 2023:

Current wording

Comparison of the three months ended March 31, 2025 to the three months ended March 31, 2024:

Previous wording

Our product sales increased by $34.9 million and $84.8 million for the three and nine months ended September 30, 2024, respectively, compared to the same periods in 2023. The increases are primarily due to an increase in demand for Crysvita in Latin America resulting from an increase in the number of patients on therapy, launch of Evkeeza in Japan and several markets in Europe, Middle East and Africa territories, or EMEA, and continued increase in demand for our other approved products.

Current wording

Our product sales increased by $29.0 million for the three months ended March 31, 2025 compared to the same period in 2024. The increase is primarily due to an increase in demand for Crysvita in Latin America resulting from an increase in the number of patients on therapy, the launch of Evkeeza in Japan and several markets in Europe, Middle East and Africa territories, or EMEA, and the continued increase in demand for our other approved products.

Previous wording

Cost of sales increased by $10.0 million and $26.7 million for the three and nine months ended September 30, 2024, respectively, compared to the same periods in 2023. The increases in cost of sales were due to an increase in demand for our approved products, primarily Crysvita in Latin America and Evkeeza in Japan and several markets in EMEA.

Current wording

Cost of sales increased by $11.1 million for the three months ended March 31, 2025, compared to the same period in 2024. The increase in cost of sales was due to an increase in demand for our approved products, primarily Crysvita in Latin America and Evkeeza in EMEA and Japan.

Previous wording

Total research and development expenses increased by $12.9 million and $22.2 million for the three and nine months ended September 30, 2024, respectively, compared to the same periods in 2023. The increase in research and development expenses was primarily due to:

Current wording

Total research and development expenses decreased by $12.7 million for the three months ended March 31, 2025 compared to the same period in 2024. The decrease in research and development expenses was primarily due to:

Previous wording

for biologic and nucleic acid programs, increases of $11.4 million and $26.6 million for the three and nine months ended September 30, 2024, respectively, primarily related to the continued clinical progress of the UX143 and GTX102 programs and associated clinical development and manufacturing expenses, partially offset by a reduction in development expense on UX053 for the treatment of Glycogen Storage Disease Type III due to a greater focus on our other late-stage and larger indication clinical programs;

Current wording

for biologic and nucleic acid programs, an increase of $0.4 million, primarily related to the continued clinical progress of the GTX102 program and associated clinical development and manufacturing expenses, partially offset by a reduction in development expense on UX143;

Previous wording

for other research and development expenses, decreases of $2.8 million and $4.6 million for the three and nine months ended September 30, 2024, respectively, primarily related to decreased staffing and cost efficiencies to support internal manufacturing, and administrative and general support.

Current wording

for other research and development expenses, an increase of $1.8 million, primarily related to increased staffing to support internal manufacturing, and administrative and general support.

Previous wording

We expect our annual research and development expenses to continue to moderate in the future as we advance our product candidates through clinical development. The timing and amount of expenses incurred will depend largely upon the outcomes of current or future clinical studies for our product candidates as well as the related regulatory requirements, manufacturing costs, and any costs associated with the advancement of our preclinical programs.

Current wording

We expect our annual research and development expenses to moderate in the future as we advance our product candidates through clinical development. The timing and amount of expenses incurred will depend largely upon the outcomes of current or future clinical studies for our product candidates as well as the related regulatory requirements, manufacturing costs, and any costs associated with the advancement of our preclinical programs.

Previous wording

We expect annual selling, general and administrative expenses to continue to moderate in the near-term as we continue to support our approved products and multiple clinical-stage product candidates, and thereafter we expect these expenses to increase as we plan to launch additional products.

Current wording

We expect annual selling, general and administrative expenses to increase in the future as we continue to support our existing approved products, multiple clinical-stage product candidates, and planned launches of additional products.

Previous wording

Interest income increased by $4.9 million and $8.8 million for the three and nine months ended September 30, 2024, respectively, compared to the same periods in 2023, primarily due to higher marketable debt securities balances.

Current wording

Interest income decreased by $2.0 million for the three months ended March 31, 2025 compared to the same period in 2024, primarily due to lower marketable debt securities balances.

Previous wording

For the three and nine months ended September 30, 2024, respectively, we recorded net increases in the fair value of our equity investments of $0.7 million and $0.4 million due to unrealized gains on our investment in Solid Biosciences Inc., or Solid, common stock.

Current wording

For the three months ended March 31, 2025 and 2024, we recorded a net decrease of $0.2 million and a net increase of $3.7 million, respectively, in the fair value of our equity investments due to unrealized loss and gain, respectively, on our investment in Solid Biosciences Inc., or Solid, common stock.

Previous wording

The non-cash interest expense on liabilities for sales of future royalties decreased by $2.0 million and $1.2 million for the three and nine months ended September 30, 2024, respectively, compared to the same periods in 2023. These decreases were primarily the result of a decrease in overall liability balances as payments have exceeded interest expense incurred. To the extent the royalty payments are greater or less than our initial estimates or the timing of such payments is materially different than our original estimates, we prospectively adjust the effective interest rate.

Current wording

The non-cash interest expense on liabilities for sales of future royalties decreased by $1.5 million for the three months ended March 31, 2025, compared to the same period in 2024, primarily due to a reduction in total royalty obligation balances as a result of increased royalties generated from our collaboration partner, KKC. To the extent the royalty payments are greater or less than our initial estimates or the timing of such payments is materially different than our original estimates, we prospectively adjust the effective interest rate.

Previous wording

Other income increased by $3.8 million and other expense decreased by $2.0 million for the three and nine months ended September 30, 2024, respectively, compared to the same periods in 2023. These changes were primarily due to fluctuations to our exposures in foreign exchange rates.

Current wording

Other income increased by $2.4 million for the three months ended March 31, 2025, compared to the same period in 2024. These changes were primarily due to fluctuations to our exposures in foreign exchange rates.

Previous wording

The provision for incomes taxes decreased by a nominal amount for the three and nine months ended September 30, 2024, respectively, compared to the same periods in 2023.

Current wording

The provision for incomes taxes increased by $0.9 million for the three months ended March 31, 2025, compared to the same period in 2024.

Previous wording

To date, we have funded our operations primarily from the sale of our equity securities, revenues from our commercial products, the sale of certain future royalties, and strategic collaboration arrangements.

Current wording

To date, we have funded our operations primarily from the sale of our equity securities, revenue from our commercial products, the sale of certain future royalties, and strategic collaboration arrangements.

Previous wording

As of September 30, 2024, we had $824.7 million in available cash, cash equivalents, and marketable debt securities. We believe that our existing capital resources will be sufficient to fund our projected operating requirements for at least the next twelve months. Our cash, cash equivalents, and marketable debt securities are held in a variety of deposit accounts, interest-bearing accounts, corporate bond securities, commercial paper, U.S. government securities, asset-backed securities, and money market funds. Cash in excess of immediate requirements is invested with a view toward liquidity and capital preservation, and we seek to minimize the potential effects of concentration and credit risk.

Current wording

As of March 31, 2025, we had $563.0 million in available cash, cash equivalents, and marketable debt securities. We believe that our existing capital resources will be sufficient to fund our projected operating requirements for at least the next 12 months. Our cash, cash equivalents, and marketable debt securities are held in a variety of deposit accounts, interest-bearing accounts, corporate bond securities, commercial paper, U.S. government securities, asset-backed securities, and money market funds. Cash in excess of immediate requirements is invested with a view toward liquidity and capital preservation, and we seek to minimize the potential effects of concentration and credit risk.

Previous wording

In June 2024, we completed an underwritten public offering in which 8,782,051 shares of common stock were sold, including the exercise in full by the underwriters of their option to purchase an additional 1,346,153 shares, at a public offering price of $39.00 per share. In connection with the offering, we sold to certain investors pre-funded warrants, in lieu of common stock, to purchase 1,538,501 shares of common stock at a purchase price of $38.999 per pre-funded warrant, which equals the public offering price per share of common stock less the $0.001 exercise price per share of each pre-funded warrant. The total proceeds that we received from the offering were $381.0 million, net of underwriting discounts and commissions.

Current wording

In June 2024, we completed an underwritten public offering for the sale of shares of common stock and pre-funded warrants. The total proceeds that we received from the offering were $381.0 million, net of underwriting discounts and commissions.

Previous wording

In February 2024, we entered into a Sales Agreement with Cowen and Company, LLC, or Cowen, pursuant to which the Company may offer and sell shares of the Company’s common stock having an aggregate offering proceeds up to $350.0 million, from time to time, in ATM offerings through Cowen. No shares were sold under this agreement during the nine months ended September 30, 2024.

Current wording

In February 2024, we entered into a Sales Agreement with Cowen and Company, LLC, or Cowen, pursuant to which the Company may offer and sell shares of the Company’s common stock having an aggregate offering proceeds up to $350.0 million, from time to time, in ATM offerings through Cowen. As of March 31, 2025 no shares have been sold under this agreement.

Previous wording

Cash used in operating activities for the nine months ended September 30, 2023 was $390.9 million and primarily reflected a net loss of $483.4 million, partially offset by non-cash items of $121.0 million, net, which consisted primarily of non-cash collaboration royalty revenues, interest expense related to the sale of future royalties, net of amounts capitalized, stock-based compensation, amortization of discounts on marketable debt securities, and depreciation and amortization. The change in operating assets and liabilities also reflected a net use of cash of $28.4 million, primarily due to an increase in accounts receivable primarily related to timing of invoices received, increased revenues, and order timing, a decrease in accounts payable, accrued liabilities, and other liabilities, primarily due to timing of payments and receipt of invoices, as well as a decrease in manufacturing accruals and the payout of the 2022 annual bonuses, partially offset by a net decrease in other assets primarily due to a decrease in prepaid manufacturing.

Current wording

Cash used in operating activities for the three months ended March 31, 2025 was $166.5 million and primarily reflected a net loss of $151.1 million, partially offset by non-cash items of $41.2 million, net, which consisted primarily of stock-based compensation, amortization of discounts on marketable debt securities, depreciation and amortization, non-cash royalty revenue, and non-cash interest expense related to the sale of future royalties. The change in operating assets and liabilities also reflected a net use of cash of $56.6 million, primarily due to a net decrease in accounts payable, accrued and other liabilities primarily due to the payout of the 2024 annual bonuses and decreases in accrued collaboration for payment of a milestone to a collaboration partner of $30.0 million as well as an increase in prepaid manufacturing expense, partially offset by a decrease in accounts receivable related to timing of orders and collections.

Previous wording

Cash used in operating activities for the nine months ended September 30, 2024 was $334.7 million and primarily reflected a net loss of $435.8 million, partially offset by non-cash items of $109.9 million, net, which consisted primarily of stock-based compensation, amortization of discounts on marketable debt securities, depreciation and amortization, non-cash royalty revenue, and non-cash interest expense related to the sale of future royalties. The change in operating assets and liabilities also reflected a net use of cash of $8.8 million, primarily due to an increase in accounts receivable primarily related to an increase in sales of our approved products and timing of when orders were received, partially offset by a net decrease in other assets primarily due to a decrease in prepaid manufacturing.

Current wording

Cash used in operating activities for the three months ended March 31, 2024 was $190.7 million and primarily reflected a net loss of $170.7 million, partially offset by non-cash items of $36.0 million, net, which consisted primarily of stock-based compensation, amortization of discounts on marketable debt securities, the change in fair value of equity investments, depreciation and amortization, non-cash royalty revenue, and non-cash interest expense related to the sale of future royalties. The change in operating assets and liabilities also reflected a net use of cash of $56.0 million, primarily due to an increase in accounts receivable primarily related to an increase in sales of our approved products and timing of when orders were received, partially offset by a net decrease in accounts payable, accrued and other liabilities primarily due to the payout of the 2023 annual bonuses and decreases in accrued collaboration for the payment of a regulatory milestone to a collaboration partner.

Previous wording

Cash used in investing activities for the nine months ended September 30, 2024 was $120.5 million and was primarily related to $98.9 million from net activities in marketable debt securities, combined with payments to Regeneron of $12.5 million for the achievement of milestones under the collaboration agreement recorded as an intangible asset.

Current wording

Cash provided by investing activities for the three months ended March 31, 2025 was $119.9 million and was primarily related to $137.0 million from net activities in marketable debt securities, partially offset by the payment to a collaboration partner of $15.0 million for the achievement of a milestone under the collaboration agreement recorded as an intangible asset.

Previous wording

Cash provided by investing activities for the nine months ended September 30, 2023 was $275.0 million and was primarily related to $325.0 million from net activities in marketable debt securities, offset by purchases of property, plant, and equipment of $42.7 million, primarily related to the fit-out of our gene therapy manufacturing facility.

Current wording

Cash provided by investing activities for the three months ended March 31, 2024 was $94.4 million and was primarily related to $109.7 million from net activities in marketable debt securities, partially offset by the payment to a collaboration partner of $10.0 million for the achievement of a milestone under the collaboration agreement recorded as an intangible asset.

Previous wording

We anticipate that, excluding non-recurring items, we will continue to generate annual losses for the next several years as we continue the development of, and seek regulatory approvals for, our product candidates, and continue with commercialization of approved products. We may require additional capital to fund our operations, to complete our ongoing and planned clinical studies, to commercialize our products, to continue investing in early-stage research capabilities to promote our pipeline growth, to continue to acquire or invest in businesses or products that complement or expand our business, including future milestone payments thereunder, and to further develop our general infrastructure and such funding may not be available to us on acceptable terms or at all.

Current wording

We anticipate that, excluding non-recurring items, we will continue to generate annual losses in the near term as we continue the development of, and seek regulatory approvals for, our product candidates, and continue with commercialization of approved products. We may require additional capital to fund our operations, to complete our ongoing and planned clinical studies, to commercialize our products, to continue investing in early-stage research capabilities to promote our pipeline growth, to continue to acquire or invest in businesses or products that complement or expand our business, including future milestone payments thereunder, and to further develop our general infrastructure and such funding may not be available to us on acceptable terms or at all.

Previous wording

Future minimum lease payments under non-cancellable leases as of September 30, 2024, were approximately $42.5 million, of which $13.4 million is due within one year.

Current wording

Future minimum lease payments under non-cancellable leases as of March 31, 2025, were approximately $46.6 million, of which $13.9 million is due within one year.

Previous wording

Manufacturing and service contract obligations primarily relate to manufacturing of inventory for our approved products. As of September 30, 2024, we had obligations of approximately $35.6 million, of which $23.2 million is due within one year.

Current wording

Manufacturing and service contract obligations primarily relate to manufacturing of inventory for our approved products. As of March 31, 2025, we had obligations of approximately $97.2 million, of which $84.9 million is due within one year.