Ultragenyx Pharmaceutical Inc.
For the period ended Jun 30, 2026. Filed Aug 5, 2026.
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A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Jun 30, 2026 · Filed Aug 5, 2026
RARE$15.43-0.9%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2026-03-31; filed 2026-05-06.
0001193125-26-206960
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2026-06-30; filed 2026-08-05.
0001193125-26-333233
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 115 previous paragraphs and 118 current paragraphs.
80 unchanged; 28 reworded; 10 added; 6 removed; 1 previous and 0 current paragraphs rearranged.
Tables are not compared: 7 previous and 13 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (10)
Current report
Milestones and Upcoming Catalysts
Current report
Dojolvi for the treatment of LC-FAOD
Current report
In May 2026, we announced that Dojolvi was listed on the National Health Insurance (NHI) drug price list and was officially launched in Japan following the receipt of manufacturing and marketing approval under the Conditional Approval System for Pharmaceuticals on March 23, 2026.
Current report
The Phase 3 Enh3ance study continues with patients in both treatment and placebo-crossover groups progressing through 64 weeks of follow-up. Data from the second primary endpoint, which evaluates reduction in treatment burden, including use of ammonia scavengers and dietary management, across both the treatment and placebo-crossover groups following treatment with DTX301, are expected in the first half of 2027.
Current report
Our product sales increased for the three and six months ended June 30, 2026, as compared to the same periods in 2025. The increase was largely due to increased product sales for Crysvita, primarily in LATAM territories due to an increase in the number of patients as well as timing of shipments, and the continued expansion of Evkeeza in territories outside of the United States.
Current report
Our Crysvita royalty revenue increased for the three and six months ended June 30, 2026, as compared to the same periods in 2025. The increases were primarily due to an increase in the number of patients on therapy and timing of orders.
Current report
Cost of sales increased for the three and six months ended June 30, 2026, as compared to the same periods in 2025 primarily due to increased Crysvita product sales in LATAM and Evkeeza product sales in territories outside of the United States.
Current report
Cash Provided by Financing Activities
Current report
Cash provided by financing activities for the six months ended June 30, 2026 was $3 million and was primarily related to net proceeds from the issuance of common stock under equity plans.
Current report
Cash provided by financing activities for the six months ended June 30, 2025 was $84 million and was primarily related to net proceeds from our ATM offering.
Removed paragraphs (6)
Previous report
Recent Program Updates
Previous report
In May 2026, we provided an update on the Phase 1/2 data as of a March 2026 cut-off date. At that time, a total of 74 patients had been treated with GTX-102, with 66 patients continuing follow-up in the long-term extension (LTE) study. Phase 1/2 patients have been on continuous therapy for an average of more than three years and some patients are in their fifth year of treatment , with patients generally receiving the 14 mg quarterly, maintenance dose. As of the data cut-off date, GTX-102 maintained a consistent safety profile, even after multiple years of chronic treatment, demonstrating no new cases of transient lower extremity weakness nor any other recurring drug-related serious adverse events. We plan to present the updated Phase 1/2 long-term efficacy and safety data at a future scientific meeting.
Previous report
In March 2026, we announced positive results from the Phase 3 Enh3ance study of DTX301. At Week 36 in the randomized, double-blind placebo-controlled period of the trial, DTX301 patients (n=18) demonstrated a statistically significant and clinically meaningful 18% (p=0.018) reduction in 24-hour plasma ammonia (AUC0-24) into the normal range compared to placebo (n=19). Eight of nine patients with abnormal ammonia AUC0-24 at baseline also reached normal ammonia levels rapidly, which were generally maintained during this treatment period. At Week 24, patient global impression scale (PGIC) for overall OTC symptoms (n=15) showed 71% of DTX301 patients were much improved (equivalent to +3), compared to 0% of placebo patients. DTX301 was well tolerated with an acceptable safety profile.
Previous report
Our product sales decreased for the three months ended March 31, 2026 as compared to the same period in 2025. The decrease was largely due to a decrease in Crysvita product sales primarily due to timing of orders in Brazil, partially offset by an increase in Evkeeza product sales due to the continued expansion in patients and geographies in several markets in EMEA, in Japan and in Canada.
Previous report
Cost of sales increased by a nominal amount for the three months ended March 31, 2026 as compared to the same period in 2025.
Previous report
for other research and development expenses, a decrease primarily related lower internal manufacturing costs.
Reworded paragraphs (28)
Previous wording
Dojolvi is a highly purified, synthetic, 7-carbon fatty acid triglyceride administered orally, designed to provide medium-chain, odd-carbon fatty acids as an energy source and metabolite replacement, developed for people with LC-FAOD. LC-FAOD represents a set of rare metabolic diseases that prevents the conversion of fat into energy and can cause low blood sugar, muscle rupture, and heart and liver disease. Dojolvi is approved in the U.S. and certain other regions as a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed LC-FAOD. There are approximately 8,000 to 14,000 patients in the developed world with LC-FAOD.
Current wording
Dojolvi is a highly purified, synthetic, 7-carbon fatty acid triglyceride administered orally, designed to provide medium-chain, odd-carbon fatty acids as an energy source and metabolite replacement, developed for people with LC-FAOD. LC-FAOD represents a set of rare metabolic diseases that prevents the conversion of fat into energy and can cause low blood sugar, muscle rupture, and heart and liver disease. Dojolvi is approved in the U.S., Japan, and certain other regions as a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed LC-FAOD. There are approximately 8,000 to 14,000 patients in the developed world with LC-FAOD.
Previous wording
In February 2026, we announced that the FDA has accepted for review the BLA seeking approval of DTX401 for the treatment of Glycogen Storage Disease Type Ia (GSDIa). The FDA granted the BLA Priority Review and assigned a Prescription Drug User Fee Act
Current wording
In February 2026, we announced that the FDA has accepted for review the Biologics License Application, or BLA, seeking approval of DTX401 for the treatment of Glycogen Storage Disease Type Ia (GSDIa). The FDA granted the BLA Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) action date of August 23, 2026. The FDA previously informed the Company, in April 2026, that an Advisory Committee meeting was not anticipated.
Previous wording
In April 2026, we announced that the FDA had accepted our resubmitted BLA seeking accelerated approval for UX111 as a treatment for patients with Sanfilippo syndrome Type A. The submission included substantial longer-term data that was presented at the WORLDSymposium™ 2026, which demonstrated up to eight years of follow-up and showed further clinical improvement relative to the decline observed in natural history, and a durable treatment effect across clinical evaluations and multiple biomarkers, while maintaining an acceptable safety profile. In February 2025, the FDA granted the BLA Priority Review and, in April 2026, assigned a PDUFA action date of September 19, 2026.
Current wording
In April 2026, we announced that the FDA had accepted our resubmitted BLA seeking accelerated approval for UX111 as a treatment for patients with Sanfilippo syndrome Type A. The FDA granted the BLA Priority Review and assigned a PDUFA action date of September 19, 2026.
Previous 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.
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 September or October 2026 timeframe.
Previous 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.
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 half 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 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 the fourth quarter of 2026.
Previous wording
In March 2026, we announced that the FDA cleared our Investigational New Drug (IND) application for UX016, an investigational small molecule prodrug of sialic acid (SA), that is being evaluated as a substrate replacement therapy for GNE myopathy. The UX016 program is funded through clinical proof-of-concept by an external venture philanthropy agreement with a patient group, which includes the Phase 1/2 study that is expected to begin enrolling in the second half of 2026.
Current wording
In March 2026, we announced that the FDA cleared our Investigational New Drug, or IND, application for UX016, an investigational small molecule prodrug of sialic acid, or SA, that is being evaluated as a substrate replacement therapy for GNE myopathy. The UX016 program is funded through clinical proof-of-concept by an external venture philanthropy agreement with a patient group, which includes the Phase 1/2 study that is expected to begin enrolling in the second half of 2026.
Previous wording
In January 2026, topline safety and efficacy data from both studies were presented and included data on bone mineral density, vertebral fractures, and patient reported outcomes on pain and physical function. Based on our analysis of the data across the two global Phase 3 studies, we believe there is a basis to engage regulatory agencies to determine if there is a path forward for UX143.
Current wording
In January 2026, topline safety and efficacy data from both studies were presented and included data on bone mineral density, vertebral fractures, and patient reported outcomes on pain and physical function. We believe the data across two global Phase 3 studies suggest that setrusumab has a meaningful effect on bone disease in OI. We will continue to engage regulatory agencies to determine the necessary data to support a regulatory filing and if there is a potential path forward for UX143.
Previous 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.
Current wording
We have incurred net losses in each year since inception. Our net losses were $92 million and $277 million for the three and six months ended June 30, 2026, respectively and $115 million and $266 million for the three and six months ended June 30, 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 $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.
Current wording
Our total revenues were $214 million and $350 million for the three and six months ended June 30, 2026, respectively, and $167 million and $306 million for the six months ended June 30, 2025, respectively. The increase in revenue was largely driven by an increase in demand for our approved products, as well as timing of shipments.
Previous wording
In February 2026, we implemented a strategic restructuring plan designed to reduce operating expenses and focus resources on our highest value drivers, including a reduction in workforce of approximately 10% and the curtailment of certain operating activities, including UX143 manufacturing activities. During the three months ended March 31, 2026, we recognized total restructuring charges of $28 million in research and development expense and $2 million in selling, general and administrative expenses in our Condensed Consolidated Statements of Operations. As of March 31, 2026, $10 million of restructuring charges were included in accounts payable and accrued liabilities in our Condensed Consolidated Balance Sheet. We expect substantially all remaining cash payments related to the restructuring to be made during the first half of 2026. See “Note 13. Restructuring Expense” to the financial statements for additional information.
Current wording
In February 2026, we implemented a strategic restructuring plan designed to reduce operating expenses and focus resources on our highest value drivers, including a reduction in workforce of approximately 10% and the curtailment of certain operating activities, including UX143 manufacturing activities. During the three and six months ended June 30, 2026, we recognized total restructuring charges of nil and $28 million, respectively, in research and development expense and nil and $2 million, respectively in selling, general and administrative expenses in our Condensed Consolidated Statements of Operations. All expected cash payments related to the restructuring were completed during the six months ended June 30, 2026. See “Note 13. Restructuring Expense” to the financial statements for additional information.
Previous wording
As of March 31, 2026, we had $534 million in available cash, cash equivalents, and marketable securities.
Current wording
As of June 30, 2026, we had $436 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 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.
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 and six months ended June 30, 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 months ended March 31, 2026 to the three months ended March 31, 2025:
Current wording
Comparison of the three and six months ended June 30, 2026 to the three and six months ended June 30, 2025:
Previous 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:
Current wording
Total research and development expenses increased for the three and six months ended June 30, 2026, compared to the same periods in 2025. The increase in research and development expenses was primarily due to:
Previous wording
for gene therapy programs, an increase primarily due to UX111 and DTX401 manufacturing costs in preparation for commercial launch;
Current wording
for gene therapy programs, an increase for the three and six months primarily due to DTX401 and UX111 manufacturing costs in preparation for commercial launch, partially offset by timing of UX701 manufacturing runs;
Previous 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;
Current wording
for biologic and nucleic acid programs, a decrease for the three months and an increase for the six months, respectively, primarily related to the closeout of existing manufacturing commitments for UX143 as well as the continued clinical progress of the GTX-102 program and associated clinical development and manufacturing expenses; and
Previous wording
for stock-based compensation, a decrease due to the timing of grants and lower expense for modified awards under the restructuring plan; and
Current wording
for stock-based compensation, a decrease for the three and six months, due to the timing of grants and lower expense from lower headcount associated with the restructuring plan.
Previous wording
Selling, general and administrative expenses had a nominal increase for the three months ended March 31, 2026 compared to the same period in 2025.
Current wording
Selling, general and administrative expenses had a nominal increase for each of the three and six months ended June 30, 2026, compared to the same periods in 2025.
Previous 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.
Current wording
Interest income decreased for the three and six months ended June 30, 2026, compared to the same periods in 2025, primarily due to lower marketable securities balances.
Previous 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.
Current wording
The non-cash interest expense on liabilities for sales of future royalties increased for the three and six months ended June 30, 2026, compared to the same periods 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 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.
Current wording
As of June 30, 2026, we had $436 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 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.
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 and six months ended June 30, 2026.
Previous 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.
Current wording
Cash used in operating activities for the six months ended June 30, 2026 was $294 million and primarily reflected a net loss of $277 million, partially offset by non-cash items of $63 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 $80 million, primarily due to a net increase of accounts receivable related to timing of orders and collections, combined with a decrease in accounts payable, accrued and other liabilities primarily due to decreases in accrued manufacturing due to payment or settlement of accrued costs related to stoppage of manufacturing for UX143 and the payout of 2025 annual bonuses.
Previous 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.
Current wording
Cash used in operating activities for the six months ended June 30, 2025 was $275 million and primarily reflected a net loss of $266 million, partially offset by non-cash items of $70 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 $79 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 million, an increase in prepaid manufacturing expense.
Previous 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.
Current wording
Cash used in investing activities for the six months ended June 30, 2026 was $5 million and was primarily related to the payment to a collaboration partner for the achievement of a milestone under the collaboration agreement recorded as an intangible asset.
Previous 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.
Current wording
Cash provided by investing activities for the six months ended June 30, 2025 was $191 million and was primarily related to $210 million from net activities in marketable debt 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
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.
Current wording
Future minimum lease payments under non-cancellable leases as of June 30, 2026, were approximately $36 million, of which $13 million is due within one year.
Rearranged previous paragraphs (1)
Previous report — wording carried elsewhere
(PDUFA) action date of August 23, 2026. The FDA informed the Company that an Advisory Committee meeting is not anticipated at this time.