Ultragenyx Pharmaceutical Inc.
For the period ended Sep 30, 2026. Filed Nov 4, 2026.
View on SEC.govAbout this filing
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.
Sep 30, 2026 · Filed Nov 4, 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 2026-06-30; filed 2026-08-05.
0001193125-26-333233
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2026-09-30; filed 2026-11-04.
0001193125-26-445718
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 118 previous paragraphs and 128 current paragraphs.
61 unchanged; 38 reworded; 29 added; 19 removed; 0 previous and 0 current paragraphs rearranged.
Tables are not compared: 13 previous and 15 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 (29)
Current report
GENGLYCOS, formerly known as DTX401, is an adeno-associated virus serotype 8, or AAV8, gene therapy administered by IV that delivers a functional copy of the G6PC gene to restore glucose-6-phosphatase activity developed for the treatment of GSDIa. GSDIa is an ultra-rare genetic metabolic disorder caused by a deficiency of the enzyme needed to release glucose from the liver to the bloodstream. The deficiency reduces the liver’s ability to control glucose levels and is associated with potentially life-threatening hypoglycemia episodes and other serious complications, requiring rigorous nutritional management that involves a burdensome, around-the-clock regimen of raw cornstarch intake as an oral glucose replacement therapy. Glucose control with cornstarch is crude with large swings in glucose, and patients instead end up spending a large fraction of their day significantly hyperglycemic to avoid hypoglycemic episodes. GSDIa affects 1,500 to 2,500 patients in the U.S. and 6,000 to 8,000 worldwide within commercially accessible geographies.
Current report
FAYUVI, formerly known as UX111, is an adeno-associated virus 9, or AAV9, gene therapy, administered by a one-time IV infusion that provides thecross-correcting enzyme that enables the breakdown of Heparan sulfate developed for the treatment of MPS IIIA. MPS IIIA is an ultra-rare, fatal lysosomal storage disease that primarily affects the brain and is marked by rapid, progressive neurodegeneration beginning in early childhood. Children with MPS IIIA typically experience progressive global developmental delay, followed by the loss of cognitive, language, and motor function, ultimately leading to early death. MPS IIIA is estimated to affect approximately 3,000 to 5,000 patients in commercially accessible geographies, with a median life expectancy of 15 years.
Current report
In August 2026, we announced the FDA granted accelerated approval for GENGLYCOS, in adult and pediatric patients eight years and older with GSDIa. We also received a Rare Pediatric Disease Priority Review Voucher (PRV) upon this approval.
Current report
In October 2026, we entered into a definitive agreement to sell the PRV we received upon GENGLYCOS approval for a sales price of $210 million. The sale was completed on October 30, 2026.
Current report
In September 2026, we announced that the FDA granted standard full approval of FAYUVI for the treatment of pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome Type A). We also received a PRV upon this approval.
Current report
In October 2026, we entered into a definitive agreement to sell the PRV we received upon FAYUVI approval for a sales price of $203 million.
Current report
In November 2026, we announced that the Phase 1/2 study has begun and is evaluating safety and dose of UX016 and its impact on muscle strength in adults with GNEM. The UX016 program is fully funded through clinical proof-of-concept by an external venture philanthropy agreement with a patient group, which supports all costs for the Phase 1/2 study, which will begin dosing soon.
Current report
In November 2026, we announced that additional data in the dose-finding, stage of the Cyprus2+study of UX701 for the treatment of Wilson disease, including data from the fourth dosing cohort, demonstrated clinical activity in some patients, but did not meet our criteria we had set to advance the program to the pivotal, Phase 3, stage of development. UX701 demonstrated a favorable safety profile across all cohorts dosed.
Current report
We will terminate further development of this program and are working with clinical sites and patient advocacy groups to support the patient community through this transition.
Current report
In September 2026, we announced results from the Phase 3 Aspire study for apazunersen in Angelman syndrome. The study did not achieve the primary endpoint of change from Baseline in Bayley-4 cognitive raw score nor the key secondary endpoint of net response in Multidomain Responder Index (MDRI). The safety profile observed in the Aspire study was consistent with Phase 1/2.
Current report
In Aspire, the randomized groups were comparable at baseline and consistent with the patients studied in Phase 2. There were no differences between the treated and control groups that could support efficacy in the Bayley Cognition raw scores nor in the MDRI when looking at net response or mean changes of the individual five endpoints included in the MDRI.
Current report
We are winding down this program and are working with clinical sites and patient advocacy groups to support the patient community through this transition.
Current report
Inventory
Current report
We expense costs associated with the manufacture of our products prior to regulatory approval. Capitalization of such costs begins upon regulatory approval of the product for batches commenced after that date. Prior to FDA approval of GENGLYCOS in August 2026 and FAYUVI in September 2026, manufacturing and related costs for those products were expensed as research and development; accordingly, those costs were not capitalized and are not reflected in cost of sales after the respective approval dates. We expect inventory balances to increase as we produce products and capitalize the full costs of manufacturing, and we expect cost of sales to increase in relation to product revenues as we deplete the previously expensed inventory.
Current report
Inventory manufactured after regulatory approval is valued at the lower of cost and net realizable value, and cost is determined using the average cost method. Fixed production overhead is allocated to inventory based on the normal capacity of our manufacturing facilities. Overhead in excess of the amount allocated on that basis is recognized in cost of sales in the period incurred and is not capitalized.
Current report
We periodically review our inventories for excess amounts or obsolescence and write down obsolete or otherwise unmarketable inventory to estimated net realizable value.
Current report
Manufacturing costs incurred prior to approval for GENGLYCOS and FAYUVI were recorded as research and development expense. We estimate the cost of the inventory manufactured prior to approval, and expensed as research and development as of September 30, 2026, at approximately $88 million for FAYUVI and $113 million for GENGLYCOS.
Current report
Fixed manufacturing overhead was not allocated to individual production runs before approval, because no inventory was capitalized in those periods. We have therefore estimated the cost of this inventory by applying manufacturing overhead from a representative period of normalized production to the batches comprising the inventory on hand at approval.
Current report
There were no sales of GENGLYCOS or FAYUVI during the three and nine months ended September 30, 2026, and as a result there was no impact on cost of sales from the inventory manufactured and expensed prior to approval.
Current report
GENGLYCOS and FAYUVI are each administered as a single infusion at a dose determined by the patient's body weight. The number of vials required to treat a patient, and accordingly the cost of goods attributable to that patient, varies materially with patient weight. As a result, cost of sales as a percentage of the related product revenue will vary from period to period with the weight profile of the patients treated in that period, and will continue to do so after the previously expensed inventory has been fully consumed.
Current report
As the number of patients who can be treated from this inventory depends on their weights, the period in which inventory manufactured prior to approval is fully consumed may differ significantly from our estimates. We currently estimate that the inventory manufactured prior to approval will be fully consumed around the end of 2027 for FAYUVI and around the end of 2028 for GENGLYCOS. The actual consumption period will also depend on demand, the number of patients treated, the weight of the patients, the use of this inventory to supply our patient access and compassionate use programs, which draw on the same supply as commercial demand, and expiration dating.
Current report
We expect our gross margin percentage to decrease as we sell products that reflect the full cost of manufacturing and as we deplete the inventory expensed prior to approval.
Current report
Total research and development expenses decreased for the three and nine months ended September 30, 2026, compared to the same periods in 2025. The decreases in research and development expenses were primarily due to:
Current report
for gene therapy programs, a decrease for the three months and an increase for the nine months, respectively. The decrease for the three months was primarily due to timing of manufacturing costs for UX111. The increase for the nine months primarily reflects timing of manufacturing runs for DTX401, partially offset by decreases from the timing of manufacturing runs and development activities for UX701 and DTX301;
Current report
We expect annual selling, general and administrative expenses to remain relatively stable in the near term, as increased commercial investments to support the launch of two newly approved products are expected to be offset by general and administrative cost savings from our restructuring plans.
Current report
Benefit From (Provision For) Income Taxes (dollars in millions)
Current report
The increase in benefit from income taxes for the three and nine months ended September 30, 2026, compared to the same periods in 2025, was primarily due to the release of federal and state valuation allowances as a result of the reclassification of the GENGLYCOS in-process research and development intangibles from indefinite to finite-lived intangibles. There was no similar income tax benefit for the three and nine months ended September 30, 2025.
Current report
Subsequent to September 30, 2026, we announced the sale of the PRVs received upon the approvals of GENGLYCOS and FAYUVI. The GENGLYCOS PRV sale closed on October 30, 2026. The sale of the FAYUVI PRV remains subject to satisfaction of customary closing conditions and is expected to occur prior to the end of 2026. We expect to receive combined net proceeds of $349 million from the sale of both PRVs.
Current report
Cash Provided by Investing Activities
Removed paragraphs (19)
Previous report
We were founded in April 2010 by our President and Chief Executive Officer, Emil Kakkis, M.D., Ph.D., and are led by a management team experienced in the development and commercialization of rare disease therapeutics. Our strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.
Previous report
DTX401 is an adeno-associated virus 8, or AAV8, gene therapy clinical candidate, administered by a one-time IV infusion that is designed to deliver stable expression and activity of G6Pase-α, an essential enzyme in glycogen and glucose metabolism. DTX401 is being developed for the treatment of patients with GSDIa, and is the most common genetically inherited glycogen storage disease, with an estimated 6,000 patients in the developed world. A Pediatric Investigation Plan, or PIP, was accepted by the EMA. The DTX401 program has received Rare Pediatric Disease, RMAT, Fast Track, and Orphan Drug designations in the U.S., and PRIME and Orphan Medicinal Product Designations in the EU.
Previous report
UX111 (formerly ABO-102) is an adeno-associated virus 9, or AAV9, gene therapy product candidate, administered by a one-time IV infusion that provides the cross-correcting enzyme that enables the breakdown of Heparan sulfate, or HS. UX111 is being developed for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease with no approved treatment, which primarily affects the central nervous system. There are an estimated 3,000 to 5,000 patients in the developed world affected by Sanfilippo syndrome type A. The program was acquired through an exclusive license agreement with Abeona Therapeutics, or Abeona, that was announced in May 2022. The UX111 program has received Regenerative Medicine Advanced Therapy, or RMAT, Fast Track, Rare Pediatric Disease, and Orphan Drug Designations in the U.S., and PRIME and Orphan Medicinal Product designations in the EU.
Previous report
GTX-102 (apazunersen) for the treatment of Angelman Syndrome
Previous report
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 Breakthrough Therapy Designation, 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 report
UX701 (rivunatpagene miziparvovec) for the treatment of Wilson Disease
Previous report
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 report
Dojolvi for the treatment of LC-FAOD
Previous 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.
Previous report
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 report
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 report
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 report
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 report
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 report
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 report
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 report
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 report
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 report
Cash (Used in) Provided by Investing Activities
Reworded paragraphs (38)
Previous wording
Our current approved therapies and clinical-stage pipeline consist of four product categories: biologics, small molecules, AAV gene therapy, and nucleic acid product candidates. The following table summarizes our approved products and pipeline of clinical product candidates:
Current wording
Our current approved therapies and clinical-stage pipeline consist of three product categories: biologics, small molecules and AAV gene therapy product candidates. The following table summarizes our approved products and pipeline of 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, 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.
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 pediatric patients 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
DTX401 (pariglasgene brecaparvovec) for the treatment of Glycogen Storage Disease Type Ia, or GSDIa
Current wording
GENGLYCOS for the treatment of Glycogen Storage Disease Type Ia, or GSDIa
Previous wording
UX111 (rebisufligene etisparvovec) for the treatment of Sanfilippo syndrome type A, or MPS IIIA
Current wording
FAYUVI for the treatment of Sanfilippo syndrome type A, or MPS IIIA
Previous wording
UX016 for the treatment of GNE Myopathy
Current wording
UX016 for the treatment of GNE myopathy, or GNEM
Previous wording
UX016 is a small-molecule prodrug composed of sialic acid (SA; also known as N‑acetylneuraminic acid [NANA]) and a C16 fatty acid tail designed to improve biodistribution to target tissues, like muscle, more effectively and efficiently than free SA. UX016 is being developed for the treatment of GNE myopathy, also known as hereditary inclusion body myopathy (HIBM) and Nonaka Myopathy, which is caused by the body's inability to produce adequate sialic acid leads to progressive muscle wasting and severe disability. There are an estimated 10,000 patients in the developed world affected by GNE myopathy. UX016 is funded through clinical proof-of-concept through our venture philanthropy agreement with a patient group.
Current wording
UX016 is a small-molecule prodrug composed of sialic acid (SA; also known as N‑acetylneuraminic acid NANA) and a C16 fatty acid tail designed to improve biodistribution to target tissues, like muscle, more effectively and efficiently than free SA. UX016 is being developed for the treatment of GNE myopathy, also known as hereditary inclusion body myopathy (HIBM) and Nonaka Myopathy, which is caused by the body's inability to produce adequate sialic acid leads to progressive muscle wasting and severe disability. There are an estimated 10,000 patients in the developed world affected by GNE myopathy. UX016 is fully funded through clinical proof-of-concept through a venture philanthropy agreement with a patient group.
Previous wording
DTX401 for the treatment of GSDIa
Current wording
GENGLYCOS for the treatment of GSDIa
Previous wording
UX111 for the treatment of MPS IIIA
Current wording
FAYUVI for the treatment of MPS IIIA
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. 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.
Current wording
In October 2026, data with pre- and post-hoc analyses from Orbit and Cosmic were presented at the American Society for Bone and Mineral Research 2026 Annual Meeting showing a meaningful benefit on bone mineral density, fractures and other endpoints in the two Phase 3 studies. The Company 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
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 wording
The Phase 3 Enh3ance placebo-controlled study showed substantial improvement in ammonia control and reduction need for scavenger medicines along with profound improvement in global impression scale. The study continues with patients in both treatment and placebo-crossover groups progressing through 64 weeks of follow-up to evaluate the second primary endpoint of the reduction in the use of ammonia scavengers and dietary management. Data are expected in the first half of 2027.
Previous wording
UX016 for the treatment of GNE myopathy
Current wording
UX016 for the treatment of GNEM
Previous wording
We are a biopharmaceutical company with a limited operating history. To date, we have invested substantially all of our efforts and financial resources in identifying, acquiring, and developing our products and product candidates, including conducting clinical studies and providing selling, general and administrative support for these operations. 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
We are a biopharmaceutical company with a limited operating history. To date, we have invested substantially all of our efforts and financial resources in identifying, acquiring, and developing our products and product candidates, including conducting clinical studies and providing selling, general and administrative support for these operations. 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, strategic collaboration arrangements, and the sale of PRVs.
Previous 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.
Current wording
We have incurred net losses in each year since inception. Our net losses were $118 million and $395 million for the three and nine months ended September 30, 2026, respectively and $180 million and $446 million for the three and nine months ended September 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 $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.
Current wording
Our total revenues were $173 million and $523 million for the three and nine months ended September 30, 2026, respectively, and $160 million and $466 million for the three and nine months ended September 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 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.
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 nine months ended September 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 cash payments related to the restructuring were completed during the first half of 2026. See “Note 13. Strategic Restructuring Plan” to the financial statements for additional information.
Previous wording
As of June 30, 2026, we had $436 million in available cash, cash equivalents, and marketable securities.
Current wording
As of September 30, 2026, we had $401 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 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.
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. Other than for the changes for inventory below, there have been no material changes in our critical accounting policies during the three and nine months ended September 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 and six months ended June 30, 2026 to the three and six months ended June 30, 2025:
Current wording
Comparison of the three and nine months ended September 30, 2026 to the three and nine months ended September 30, 2025:
Previous wording
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 wording
Our product sales increased for the three and nine months ended September 30, 2026, as compared to the same periods in 2025. The increase was largely due to increased product sales for EVKEEZA, primarily in Europe, Middle East and Africa territories due to timing of shipments as well as an increase in the number of patients, and the continued expansion of DOJOLVI in all territories. CRYSVITA product sales also contributed to the increase for the nine months ended September 30, 2026 as compared to the same period in 2025, primarily due to improved patient access in Türkiye. For the three months ended September 30, 2026, CRYSVITA product sales decreased as compared to the same period in 2025 due to the timing of orders.
Previous wording
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 wording
Our CRYSVITA royalty revenue increased for the three and nine months ended September 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.
Previous wording
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 wording
Cost of sales increased for the three and nine months ended September 30, 2026, as compared to the same periods in 2025 primarily due to increased EVKEEZA product sales in territories outside of the United States.
Previous 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
Current wording
for biologic and nucleic acid programs, a decrease for the three and nine months, primarily related to the closeout of existing manufacturing commitments for UX143, partially offset by increases from the timing of clinical development and manufacturing activities for the GTX-102 program; and
Previous 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.
Current wording
for stock-based compensation, a decrease for the three and nine months, due to the timing of grants and lower expense from lower headcount associated with the restructuring plan.
Previous wording
We expect a decrease in research and development expenses in the near term. This expected decline is primarily driven by the expected completion of several Phase 3 clinical programs and a strategic restructuring of our workforce and expenditures to better match our current pipeline requirements.
Current wording
We expect a decrease in research and development expenses in the near term. This expected decline is primarily driven by the completion of several Phase 3 clinical programs, including the approval of two gene therapy products which will result in manufacturing costs to be capitalized to inventory after the approval date rather than expensed to research and development, as well as the restructuring activities of our workforce and expenditures to better match our current pipeline requirements.
Previous 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.
Current wording
Selling, general and administrative expenses increased for each of the three and nine months ended September 30, 2026, compared to the same periods in 2025, primarily due to launch preparation.
Previous 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.
Current wording
Interest income decreased for the three and nine months ended September 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 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.
Current wording
The non-cash interest expense on liabilities for sales of future royalties increased for the three and nine months ended September 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
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.
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, strategic collaboration arrangements, and the sale of PRVs.
Previous 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.
Current wording
As of September 30, 2026, we had $401 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 and six months ended June 30, 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 nine months ended September 30, 2026.
Previous 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.
Current wording
Cash used in operating activities for the nine months ended September 30, 2026 was $337 million and primarily reflected a net loss of $395 million, partially offset by non-cash items of $92 million, net, which consisted primarily of stock-based compensation, amortization of discounts on marketable securities, depreciation and amortization, non-cash royalty revenue, non-cash interest expense related to the sale of future royalties, and a decrease in deferred tax liabilities as a result of the release of federal and state valuation allowances as a result of the reclassification of the GENGLYCOS in-process research and development intangibles from indefinite to finite-lived intangibles. The change in operating assets and liabilities also reflected a net use of cash of $34 million, primarily due to a net increase of accounts receivable related to timing of orders and collections from our collaboration partners.
Previous 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.
Current wording
Cash used in operating activities for the nine months ended September 30, 2025 was $366 million and primarily reflected a net loss of $446 million, partially offset by non-cash items of $103 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 $23 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.
Previous 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.
Current wording
Cash provided by investing activities for the nine months ended September 30, 2026 was $53 million and was primarily related to $64 million from net activities in marketable securities, partially offset by 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 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.
Current wording
Cash provided by investing activities for the nine months ended September 30, 2025 was $308 million and was primarily related to $330 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
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 wording
Cash provided by financing activities for the nine months ended September 30, 2026 was $13 million and was primarily related to net proceeds from the issuance by Amlogenyx of SAFEs along with issuance of common stock under our equity plans.
Previous wording
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.
Current wording
Cash provided by financing activities for the nine months ended September 30, 2025 was $84 million and was primarily related to net proceeds from our ATM offering.
Previous 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.
Current wording
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 June 30, 2026, were approximately $36 million, of which $13 million is due within one year.
Current wording
Future minimum lease payments under non-cancellable leases as of September 30, 2026, were approximately $32 million, of which $12 million is due within one year.