Quarterly Report for Quarter Ending September 30, 2026 (Form 10-Q)

Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations

The following discussion and analysis of our financial condition and results of operations should be read in conjunction with the accompanying unaudited Condensed Consolidated Financial Statements and related notes in Item 1 and with the audited Consolidated Financial Statements and the related notes included in our Annual Report on Form 10-K for the year ended December 31, 2025, or Annual Report.

Overview

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

Approved Therapies and Clinical Product Candidates

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:

Approved Products

CRYSVITA for the treatment of X-Linked Hypophosphatemia, or XLH, and Tumor-Induced Osteomalacia, or TIO

CRYSVITA is a fully human monoclonal antibody administered via subcutaneous injection, that targets fibroblast growth factor 23, or FGF23, developed for the treatment of XLH. XLH is a rare, hereditary, progressive, and lifelong musculoskeletal disorder characterized by renal phosphate wasting caused by excess FGF23 production. There are approximately 48,000 patients with XLH in the developed world, including approximately 36,000 adults and 12,000 children. CRYSVITA is the only approved treatment that addresses the underlying cause of XLH. CRYSVITA is approved in the U.S., the EU and certain other regions for the treatment of XLH in adult and pediatric patients one year of age and older.

CRYSVITA is also approved in the U.S. and certain other regions for the treatment of FGF23-related hypophosphatemia in TIO, associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized in adults and pediatric patients 2 years of age and older. There are approximately 2,000 to 4,000 patients with TIO in the developed world. TIO can lead to severe hypophosphatemia, osteomalacia, fractures, fatigue, bone and muscle pain, and muscle weakness.

We are collaborating with Kyowa Kirin Co., Ltd., or KKC, and Kyowa Kirin, a wholly owned subsidiary of KKC, on the development and commercialization of CRYSVITA globally.

MEPSEVII for the treatment of Mucopolysaccharidosis VII, or MPS VII

MEPSEVII is an enzyme replacement therapy administered intravenously, or IV, that replaces the missing enzyme (beta-glucuronidase), developed for the treatment of MPS VII or Sly syndrome. MPS VII is a rare lysosomal storage disease that often leads to multi-organ dysfunction, pervasive skeletal disease, and death. MPS VII is one of the rarest MPS disorders, affecting an estimated 200 patients in the developed world. MEPSEVII is approved in the U.S., the EU and certain other regions for the treatment of children and adults with MPS VII.

DOJOLVI for the treatment of Long-chain Fatty Acid Oxidation Disorders, or LC-FAOD

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.

EVKEEZA for the treatment of Homozygous Familial Hypercholesterolemia, or HoFH

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.

GENGLYCOS for the treatment of Glycogen Storage Disease Type Ia, or GSDIa

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.

FAYUVI for the treatment of Sanfilippo syndrome type A, or MPS IIIA

FAYUVI, formerly known as UX111, is an adeno-associated virus 9, or AAV9, gene therapy, administered by a one-time IV infusion that provides the cross-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.

Clinical Product Candidates

DTX301 (avalotcagene ontaparvovec) for the treatment of Ornithine Transcarbamylase, or OTC, deficiency

DTX301 is an AAV8 gene therapy product candidate, administered by a one-time IV infusion that is designed to deliver stable expression and activity of the OTC gene. DTX301 is being developed for the treatment of patients with OTC deficiency, which is the most common urea cycle disorder, and there are approximately 10,000 patients in the developed world with OTC deficiency, of which we estimate approximately 80% are classified as late-onset, our target population. DTX301 has received Orphan Drug Designation in both the U.S. and in the EU and Fast Track Designation in the U.S.

UX143 (setrusumab) for the treatment of Osteogenesis Imperfecta, or OI

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.

UX016 for the treatment of GNE myopathy, or GNEM

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.

Milestones and Upcoming Catalysts

GENGLYCOS for the treatment of GSDIa

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.

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.

FAYUVI for the treatment of MPS IIIA

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.

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.

UX143 for the treatment of OI

In December 2025, we announced that the Phase 3 Orbit and Cosmic studies did not achieve their primary endpoint of reduction in annualized clinical fracture rate compared to placebo (Orbit) or bisphosphonates (Cosmic).

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.

DTX301 for the treatment of OTC deficiency

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.

UX016 for the treatment of GNEM

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.

UX701 for the treatment of Wilson disease

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.

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.

GTX-102 for the treatment of Angelman syndrome

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.

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.

We are winding down this program and are working with clinical sites and patient advocacy groups to support the patient community through this transition.

Financial Operations Overview

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.

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.

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.

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.

As of September 30, 2026, we had $401 million in available cash, cash equivalents, and marketable securities.

Critical Accounting Estimates

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.

Inventory

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.

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.

We periodically review our inventories for excess amounts or obsolescence and write down obsolete or otherwise unmarketable inventory to estimated net realizable value.

Results of Operations

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

Revenue (dollars in millions)

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.

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.

Cost of Sales (dollars in millions)

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.

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.

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.

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.

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.

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.

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.

Research and Development Expenses (dollars in millions)

Research and development expenses include internal and external costs incurred for research and development of our programs and program candidates and expenses related to certain technology that we acquire or license through business development transactions. These expenses consist primarily of clinical studies performed by contract research organizations, manufacturing of drug substance and drug product performed by contract manufacturing organizations and at our gene therapy manufacturing facility, materials and supplies, fees from collaborative and other arrangements including milestones, licenses and other fees, personnel costs including salaries, benefits and stock-based compensation, and overhead allocations consisting of various support and infrastructure costs.

Clinical programs include study conduct and manufacturing costs related to clinical program candidates. Translational research includes costs for preclinical study work and costs related to preclinical programs prior to IND filing. Upfront license, acquisition, and milestone fees include any significant expenses related to strategic licensing agreements. Approved products include costs for disease monitoring programs for post-marketing clinical studies, medical affairs activities to support scientific discovery efforts on existing programs, and regulatory costs for unapproved regions. Infrastructure costs include direct costs related to laboratory, IT, and equipment depreciation costs, and overhead allocations for human resources, IT, and other allocable costs.

We manage our research and development expenses by identifying the research and development activities we expect to be performed during a given period and then prioritizing efforts based on anticipated probability of successful technical development and regulatory approval, market potential, available human and capital resources, scientific data and other considerations. We regularly review our research and development activities based on unmet medical need and, as necessary, reallocate resources among our research and development portfolio that we believe will best support the long-term growth of our business. We allocate and analyze certain operational expenses by individual product candidates, specifically costs to conduct clinical studies, including expenses incurred with clinical research organizations, direct manufacturing costs, and salaries and benefits. Other operational expenses are not allocated and analyzed by individual product candidates. For instance, costs associated with Chemistry, Manufacturing and Controls, or CMC costs, are primarily purchases of materials for our internal gene therapy manufacturing activities that qualify as research and development expenses at the time of purchase but for which the allocation and consumption of such costs by a specific product candidate is not determined; accordingly, CMC costs for gene therapy programs are generally spread across multiple product candidates. Although we do track and allocate certain operational R&D costs at the individual product candidate level, as described above and as reflected in the table below, we do not fully track and allocate research and development expenses at the individual product candidate level.

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:

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:

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.

Selling, General and Administrative Expenses (dollars in millions)

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.

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.

Interest Income (dollars in millions)

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.

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

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.

Benefit From (Provision For) Income Taxes (dollars in millions)

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.

Liquidity and Capital Resources

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.

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.

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.

In November 2025, we received net proceeds of $392 million from OMERS for the sale of a percentage of our future royalties on CRYSVITA in the U.S. and Canada.

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.

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

Cash Used in Operating Activities

Our primary use of cash is to fund operating expenses, which consist primarily of research and development and commercial expenditures. Due to our significant research and development expenditures, we have generated significant operating losses since our inception. Cash used to fund operating expenses is affected by the timing of when we pay these expenses, as reflected in the change in our outstanding accounts payable and accrued expenses.

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.

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.

Cash Provided by Investing Activities

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.

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.

Cash Provided by Financing Activities

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.

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.

Funding Requirements

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.

If we are unable to raise additional capital in sufficient amounts or on terms acceptable to us, we may be required to delay, limit, reduce the scope of, or terminate one or more of our clinical studies, research and development programs, future commercialization efforts, or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.

Our future funding requirements will depend on many factors, including the following:

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."

Contractual Obligations and Commitments

Material contractual obligations arising in the normal course of business primarily consist of operating and finance leases and manufacturing and service contract obligations.

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.

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.

We generally expect to satisfy these commitments with cash on hand and cash provided by operating activities. The terms of certain of our licenses, royalties, development and collaboration agreements, as well as other research and development activities, require us to pay potential future milestone payments based on product development success. The amount and timing of such obligations are unknown or uncertain.