Ultragenyx Pharmaceutical Inc.

Previous wording

UX143 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. UX143 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 Therapy Designation from the FDA, and was accepted into the EMA’s Priority Medicines, or PRIME, program. UX143 is subject to our collaboration agreement with Mereo and is the lead clinical asset in our bone endocrinology franchise.

Current wording

UX143 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. UX143 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 FDA and EMA Rare Pediatric Disease designation and Breakthrough Therapy Designation from the FDA, and was accepted into the EMA’s Priority Medicines, or PRIME, program. UX143 is subject to our collaboration agreement with Mereo.

Previous wording

In July 2025, we announced that all patients in the 48-week Phase 3 Aspire study have been enrolled. In total, 129 patients, between four and 17 years of age, with a genetically confirmed diagnosis of full maternal UBE3A gene deletion were enrolled and randomized 1:1 to the GTX-102 or the sham comparator group. Data from this study are expected in the second half of 2026.

Current wording

In July 2025, we announced that all patients have been enrolled in the 48-week Phase 3 Aspire study, our pivotal study evaluating patients with Angelman syndrome. In total, 129 patients, between four and 17 years of age, with a full maternal UBE3A gene deletion were enrolled and randomized 1:1 to the GTX-102 or the sham comparator group. Data from this study are expected in the second half of 2026.

Previous wording

Enrollment has begun in the Phase 2/3 Aurora study, which will evaluate GTX-102 in other Angelman syndrome genotypes and ages.

Current wording

In October 2025, we announced enrollment had begun in the Phase 2/3 Aurora study, which evaluates GTX-102 in other Angelman syndrome genotypes and ages. Enrollment in the study continued to progress during the first quarter of 2026 and is expected to be completed in the second half of 2026.

Previous wording

In September 2025, we completed enrollment of five patients in Cohort 4 in the ongoing, dose-finding, stage of the pivotal Cyprus2+study of UX701 for the treatment of Wilson disease. During Stage 1, the safety and efficacy of UX701 is being evaluated across four, sequential dosing cohorts (Cohort 1; 5.0 x 10^12 GC/kg; Cohort 2: 1.0 x 10^13 GC/kg: Cohort 3; 2.0 x 10^13 GC/kg and Cohort 4; 4.0 x 10^13 GC/kg). Data from Stage 1 of this study are expected in the first half of 2026.

Current wording

In September 2025, we completed enrollment of five patients in Cohort 4 in the ongoing, dose-finding, stage of the pivotal Cyprus2+study of UX701 for the treatment of Wilson disease. During Stage 1, the safety and efficacy of UX701 is being evaluated across four, sequential dosing cohorts (Cohort 1; 5.0 x 10^12 GC/kg; Cohort 2: 1.0 x 10^13 GC/kg: Cohort 3; 2.0 x 10^13 GC/kg and Cohort 4; 4.0 x 10^13 GC/kg). Data from Stage 1 of this study are expected in 2026.

Previous wording

We have incurred net losses in each year since inception. Our net losses were $180.4 million and $446.4 million for the three and nine months ended September 30, 2025, respectively, and $133.5 million and $435.8 million for the three and nine months ended September 30, 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.

Current wording

We have incurred net losses in each year since inception. Our net losses were $185 million and $151 million for the three months ended March 31, 2026 and 2025, 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 $159.9 million and $465.7 million for the three and nine months ended September 30, 2025, and $139.5 million and $395.4 million for the three and nine months ended September 30, 2024, respectively. The increase in revenue was largely driven by an increase in demand for our approved products.

Current wording

Our total revenues were $136 million and $139 million for the three months ended March 31, 2026 and 2025, respectively. The decrease in revenue was largely driven by timing of orders for Crysvita in Latin America.

Previous wording

As of September 30, 2025, we had $447.3 million in available cash, cash equivalents, and marketable debt securities.

Current wording

As of March 31, 2026, we had $534 million in available cash, cash equivalents, and marketable 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 nine months ended September 30, 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.

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, 2026, 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, 2025 to the three and nine months ended September 30, 2024:

Current wording

Comparison of the three months ended March 31, 2026 to the three months ended March 31, 2025:

Previous wording

Cost of Sales (dollars in thousands)

Current wording

Cost of Sales (dollars in millions)

Previous wording

Research and Development Expenses (dollars in thousands)

Current wording

Research and Development Expenses (dollars in millions)

Previous wording

The following tables provide a breakout of our research and development expenses by individual product candidate under each major clinical program type and other research and development categories:

Current wording

The following table provides a breakout of our research and development expenses by individual product candidate under each major clinical program type and other research and development categories:

Previous wording

Total research and development expenses increased by $46.1 million and $36.6 million for the three and nine months ended September 30, 2025, respectively, compared to the same periods in 2024. The changes in research and development expenses were primarily due to:

Current wording

Total research and development expenses increased for the three months ended March 31, 2026 compared to the same period in 2025. The increase in research and development expenses was primarily due to:

Previous wording

for gene therapy programs, increases of $19.9 million and $13.3 million for the three and nine months ended September 30, 2025, respectively, primarily due to an increase in UX111 manufacturing costs in preparation for commercial launch, partially offset by the timing of the UX401 manufacturing runs for which costs were incurred during the three and nine months ended September 30, 2024, but which did not recur for the three and nine months ended September 30, 2025;

Current wording

for gene therapy programs, an increase primarily due to UX111 and DTX401 manufacturing costs in preparation for commercial launch;

Previous wording

for biologic and nucleic acid programs, increases of $25.8 million and $38.4 million for the three and nine months ended September 30, 2025, respectively, primarily due to an increase in UX143 manufacturing costs in preparation for commercial launch as well as the continued clinical progress of GTX102 and associated clinical development and manufacturing costs;

Current wording

for biologic and nucleic acid programs, an increase primarily related to UX143 manufacturing settlements as well as the continued clinical progress of the GTX102 program and associated clinical development and manufacturing expenses;

Previous wording

Selling, General and Administrative Expenses (dollars in thousands)

Current wording

Selling, General and Administrative Expenses (dollars in millions)

Previous 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.

Current wording

We expect annual selling, general and administrative expenses to increase in the near term as we expand commercial activities in preparation for launches of additional products, while continuing to support our existing approved products and multiple clinical-stage product candidates.

Previous wording

Interest income decreased by $4.9 million and $8.5 million for the three and nine months ended September 30, 2025, respectively, compared to the same periods in 2024, primarily due to lower marketable debt securities balances.

Current wording

Interest income decreased for the three months ended March 31, 2026 compared to the same period in 2025, primarily due to lower marketable securities balances.

Previous wording

Non-cash Interest Expense on Liabilities for Sales of Future Royalties (dollars in thousands)

Current wording

Non-cash Interest Expense on Liabilities for Sales of Future Royalties (dollars in millions)

Previous wording

The non-cash interest expense on liabilities for sales of future royalties decreased by $1.6 million and $5.0 million for the three and nine months ended September 30, 2025, respectively, compared to the same periods 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.

Current wording

The non-cash interest expense on liabilities for sales of future royalties increased for the three months ended March 31, 2026, compared to the same period in 2025, primarily due to additional interest expense from the sale of future royalties to OMERS in November 2025. 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

As of September 30, 2025, we had $447.3 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.

Current wording

As of March 31, 2026, we had $534 million in available cash, cash equivalents, and marketable 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 securities are held in a variety of deposit accounts, interest-bearing accounts, corporate bond securities, commercial paper, U.S. government 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 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 September 30, 2025, 2,217,138 shares have been sold under this agreement for net proceeds of $79.7 million.

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 million, from time to time, in ATM offerings through Cowen. To date, we have sold 2.2 million shares under the ATM for net proceeds of $80 million. No shares were sold under the ATM during the three months ended March 31, 2026.

Previous wording

The following table summarizes our cash flows for the periods indicated (in thousands):

Current wording

The following table summarizes our cash flows for the periods indicated (in millions):

Previous wording

Cash used in operating activities for the nine months ended September 30, 2025 was $366.2 million and primarily reflected a net loss of $446.4 million, partially offset by non-cash items of $103.9 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 $23.6 million, primarily due to a net decrease in accounts payable, accrued, and other liabilities, primarily due to a decrease in accrued collaboration for payments of milestones to collaboration partners.

Current wording

Cash used in operating activities for the three months ended March 31, 2026 was $197 million and primarily reflected a net loss of $185 million, partially offset by non-cash items of $39 million, net, which consisted primarily of stock-based compensation, amortization of discounts on marketable 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 $51 million, primarily due to a net decrease in accounts payable, accrued and other liabilities primarily due to the payout of 2025 annual bonuses and decreases in accrued manufacturing due to payment or settlement of accrued costs related to stoppage of manufacturing for UX143, 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, 2025 was $166 million and primarily reflected a net loss of $151 million, partially offset by non-cash items of $41 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 million, primarily due to a net decrease in accounts payable, accrued and other liabilities primarily due to the payout of 2024 annual bonuses and decreases in accrued collaboration for payment of a milestone to a collaboration partner of $30 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 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 a collaboration partner of $12.5 million for the achievement of milestones under the collaboration agreement recorded as an intangible asset.

Current wording

Cash used in investing activities for the three months ended March 31, 2026 was $49 million and was primarily related to $43 million from net activities in marketable securities, combined with the payment to a collaboration partner of $5 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, 2025 was $308.2 million and was primarily related to $329.6 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.

Current wording

Cash provided by investing activities for the three months ended March 31, 2025 was $120 million and was primarily related to $137 million from net activities in marketable securities, partially offset by the payment to a collaboration partner of $15 million for the achievement of a milestone under the collaboration agreement recorded as an intangible asset.

Previous wording

We expect to satisfy future cash needs through existing capital balances, revenue from our commercial products, and a combination of public or private equity offerings, debt financings, collaborations, strategic alliances, licensing arrangements, and other marketing and distribution arrangements. Please see “Risk Factors—Risks Related to Our Financial Condition and Capital Requirements.”

Current wording

We expect to satisfy future cash needs through existing capital balances, revenue from our commercial products, and a combination of public or private equity offerings, debt financings, collaborations, strategic alliances, licensing arrangements, sales of future royalties and other marketing and distribution arrangements. Please see “Risk Factors—Risks Related to Our Financial Condition and Capital Requirements.”

Previous wording

Future minimum lease payments under non-cancellable leases as of September 30, 2025, were approximately $40.3 million, of which $15.1 million is due within one year.

Current wording

Future minimum lease payments under non-cancellable leases as of March 31, 2026, were approximately $40 million, of which $14 million is due within one year.

Previous wording

Manufacturing and service contract obligations primarily relate to manufacturing of product for our clinical stage pipeline. As of September 30, 2025, we had obligations of approximately $125.4 million, of which $92.9 million is due within one year.

Current wording

Manufacturing and service contract obligations primarily relate to manufacturing of product for our clinical stage pipeline, the majority of which are due in the next 12 months.