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Ultragenyx Pharmaceutical Inc.

For the period ended Jun 30, 2025. Filed Aug 6, 2025.

View on SEC.gov

About 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.

Jun 30, 2025 · Filed Aug 6, 2025

RARE$15.43-0.9%Stored close · as of Nov 6, 2026

What changed in Management’s Discussion and Analysis?

Read the current filing

Previous report: 10-Q

Period ended 2025-03-31; filed 2025-05-07.

0000950170-25-064726

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

Current report: 10-Q

Period ended 2025-06-30; filed 2025-08-06.

0000950170-25-103290

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

The 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 120 previous paragraphs and 122 current paragraphs.

77 unchanged; 37 reworded; 7 added; 5 removed; 1 previous and 1 current paragraphs rearranged.

Tables are not compared: 10 previous and 19 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 (7)

Current report

GTX-102 (apazunersen) for the treatment of Angelman Syndrome

Current report

The Phase 2/3 Aurora study, which will evaluate GTX-102 in other Angelman syndrome genotypes and ages, is expected to initiate in the second half of 2025.

Current report

In July 2025, we announced receiving a Complete Response Letter, or CRL, from the FDA for UX111. The CRL cited specific chemistry, manufacturing and controls, or CMC, related observations that we expect to be readily addressable, related to facilities and processes, and are not directly related to the quality of the product. We will be working with the FDA over the next few months to resolve the observations. Once resolution is achieved with the FDA, we expect to resubmit the BLA and anticipate up to a six-month review period to follow the resubmission.

Current report

Clinical review had been ongoing and the FDA acknowledged that the neurodevelopmental outcome data provided are robust and the biomarker data provide additional supportive evidence. The CRL did not note any review issues related to the clinical data package nor clinical inspections, and asked that updated clinical data from current patients be included in the resubmission.

Current report

Cash provided by financing activities for the six months ended June 30, 2025 was $83.8 million and was primarily related to net proceeds from our ATM offering.

Current report

Cash provided by financing activities for the six months ended June 30, 2024 was $384.9 million and was primarily related to net proceeds from the issuance of common stock and pre-funded warrants in connection with the underwritten public offering.
Removed paragraphs (5)

Previous report

In February 2025, the FDA granted Priority Review to the previously submitted BLA for UX111 with a Prescription Drug User Fee Act, or PDUFA, action date of August 18, 2025.

Previous report

The pivotal, Stage 2 protocol was amended to a 52-week, randomized, open-label, active-controlled design, evaluating the safety and efficacy of UX701 following dose selection in Stage 1. The Stage 2 primary endpoints include comparisons between the UX701 and active control groups of change in 24-hour urinary copper from Baseline at Week 52 and percent reduction in standard of care medication by Week 52.

Previous report

Cash provided by financing activities for the three months ended March 31, 2025 was $0.2 million.

Previous report

Cash used in financing activities for the three months ended March 31, 2024 was $0.1 million.
Reworded paragraphs (37)

Previous wording

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

Current wording

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

Previous wording

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

Current wording

GTX-102 is an antisense oligonucleotide, or ASO, administered by intrathecal injection that inhibits expression of the paternal UBE3A antisense. GTX-102 is being developed for the treatment of Angelman syndrome, a debilitating and rare neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene. There are an estimated 60,000 patients in the developed world affected by Angelman syndrome. GTX-102 has received 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 wording

In January 2025, we announced that the Phase 3 Orbit study is progressing to the second interim analysis expected in mid-2025. Patients in the Cosmic study also continue to be treated with either setrusumab or IV-BP therapy and will be evaluated in parallel with the second Orbit interim analysis in mid-2025. If Orbit progresses to full study completion in the fourth quarter of 2025, Cosmic will also continue to a data readout, to align with the Orbit readout without spending alpha at a mid-year interim assessment.

Current wording

In July 2025, we announced that the Phase 3 Orbit study is progressing to the final analysis expected around the end of 2025. In their review of the data at the second interim analysis for Orbit, the Data Monitoring Committee, or the DMC, informed us that UX143 demonstrated an acceptable safety profile and that we should continue the study to the final analysis. Patients in the Cosmic study also continue to be treated with either setrusumab or IV-BP therapy and will be evaluated in parallel with the final Orbit analysis around the end of the year.

Previous wording

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

Current wording

In July 2025, we announced that all patients in the 48-week Phase 3 Aspire study have been enrolled. In total, 129 patients, between four and 17 years of age, with a genetically confirmed diagnosis of full maternal UBE3A gene deletion were enrolled and randomized 1:1 to the GTX-102 or the sham comparator group. The primary endpoint will be improvement in cognition assessed by

Previous wording

These results will be included as part of a BLA submission expected in mid-2025.

Current wording

These results will be included as part of a BLA submission which is now expected in the fourth quarter of 2025.

Previous wording

In February 2025, we announced enrollment had been completed in the Phase 3 Enh3ance study of DTX301 for the treatment of OTC deficiency with a total of 37 patients randomized 1:1 to DTX301 or placebo. The co-primary endpoints are the percentage of patients who achieve a response as measured by the change in 24-hour plasma ammonia levels and discontinuation or reduction ammonia-scavenger medications and protein-restricted diet. Based on an amended protocol, the change in 24-hour ammonia levels will be measured through Week 36, after which the study would unblind and patients will be followed for a total of up to 64 weeks to determine the complete responders able to move safely to both ammonia-scavenger medications and protein-restricted diet control.

Current wording

In February 2025, we announced enrollment had been completed in the Phase 3 Enh3ance study of DTX301 for the treatment of OTC deficiency with a total of 37 patients randomized 1:1 to DTX301 or placebo. The co-primary endpoints are (i) the percentage of patients who achieve a response as measured by the change in 24-hour plasma ammonia levels and (ii) discontinuation or reduction ammonia-scavenger medications and protein-restricted diet. Based on an amended protocol, the change in 24-hour ammonia levels will be measured through Week 36, after which the study will unblind and patients will be followed for a total of up to 64 weeks to determine the complete responders able to move safely to both ammonia-scavenger medications and protein-restricted diet control.

Previous wording

In May 2025, we announced enrollment had begun in Cohort 4 of 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). We expect to enroll five patients in Cohort 4 who will receive immunomodulation therapy with rituximab and tacrolimus, in addition to the prophylactic oral corticosteroid regimen patients in Cohorts 1 through 3 received, prior to being dosed with UX701. Enrollment in Cohort 4 is expected to complete in the second half of 2025.

Current wording

Enrollment is on track in Cohort 4 of 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). Enrollment in Cohort 4 is expected to be completed in the second half of 2025.

Previous wording

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

Current wording

We have incurred net losses in each year since inception. Our net losses were $115.0 million and $266.0 million for the three and six months ended June 30, 2025, respectively, and $131.6 million and $302.3 million for the three and six months ended June 30, 2024, respectively. Substantially all of our net losses have resulted from costs incurred in connection with our research and development programs and from selling, general and administrative costs associated with our operations.

Previous wording

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

Current wording

Our total revenues were $166.5 million and $305.8 million for the three and six months ended June 30, 2025, and $147.0 million and $255.9 million for the three and six months ended June 30, 2024, respectively. The increase in revenue was largely driven by an increase in demand for our approved products.

Previous wording

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

Current wording

As of June 30, 2025, we had $539.0 million in available cash, cash equivalents, and marketable debt securities.

Previous wording

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

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 six months ended June 30, 2025, as compared to those disclosed in “Management’s Discussion and Analysis of Financial Condition and Results of Operations — Critical Accounting Policies and Significant Judgments and Estimates” in our Annual Report.

Previous wording

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

Current wording

Comparison of the three and six months ended June 30, 2025 to the three and six months ended June 30, 2024:

Previous wording

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

Current wording

Our product sales increased by $7.0 million and $36.0 million for the three and six months ended June 30, 2025, respectively, compared to the same periods in 2024. The increase for the three months ended June 30, 2025 was primarily due to continued progress of the launch of Evkeeza in Japan and in several markets in Europe, Middle East and Africa territories, or EMEA, and the continued increase in demand for our other approved products, partially offset by a decrease in Crysvita product revenue related to timing of orders in Latin America. The increase for the six months ended June 30, 2025 is primarily due to continued progress of the launch of Evkeeza in Japan and several markets in EMEA and an increase in demand for Crysvita in Latin America resulting from an increase in the number of patients on therapy, and the continued increase in demand for our other approved products.

Previous wording

Our Crysvita royalty revenue increased by $1.4 million for the three months ended March 31, 2025 as compared to the same period in 2024.

Current wording

Our Crysvita royalty revenue increased by $12.5 million and $13.9 million for the three and six months ended June 30, 2025, respectively, as compared to the same periods in 2024. The increases were primarily due to an increase in the number of reimbursed patients on therapy.

Previous wording

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

Current wording

Cost of sales increased by $1.7 million and $12.9 million for the three and six months ended June 30, 2025, respectively, compared to the same periods in 2024. The increase for the three months ended June 30, 2025 was primarily due to an increase in demand for our approved products, partially offset by a decrease related to timing of Crysvita orders in Latin America. The increase in cost of sales for the six months ended June 30, 2025 was primarily due to an increase in demand for Crysvita in Latin America and Evkeeza in EMEA and Japan, and the continued increase in demand for our other approved products.

Previous wording

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

Current wording

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

Previous wording

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

Current wording

Total research and development expenses increased by $3.2 million and decreased by $9.5 million for the three and six months ended June 30, 2025, respectively, compared to the same periods in 2024. The changes in research and development expenses were primarily due to:

Previous wording

for gene therapy programs, a decrease of $10.3 million, primarily due to the timing of the UX701 manufacturing runs and the recognition of contract manufacturing costs for DTX301 for the three months ended March 31, 2024 which did not recur for the three months ended March 31, 2025, partially offset by an increase in UX111 manufacturing costs in preparation for commercial launch;

Current wording

for gene therapy programs, an increase of $3.7 million and a decrease of $6.6 million for the three and six months ended June 30, 2025, respectively. The increase for the three months ended June 30, 2025, was primarily due to an increase in UX111 manufacturing costs in preparation for commercial launch, partially offset by the timing of the UX401 manufacturing runs for the three months ended June 30, 2024, which did not recur for the three months ended June 30, 2025. The decrease for the six months was primarily due to the timing of the UX701 and DTX301 manufacturing runs and the recognition of contract manufacturing costs for DTX301 for the six months ended June 30, 2024 which did not recur for the six months ended June 30, 2025, partially offset by an increase in UX111 manufacturing costs in preparation for commercial launch;

Previous wording

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

Current wording

for biologic and nucleic acid programs, increases of $11.7 million and $12.6 million for the three and six months ended June 30, 2025, respectively, primarily related to the continued clinical progress of the GTX102 and UX143 programs and associated clinical development and manufacturing costs;

Previous wording

for translational research, a decrease of $5.5 million, primarily related to a decrease in manufacturing expense for IND-stage projects;

Current wording

for translational research, decreases of $3.5 million and $9.1 million for the three and six months ended June 30, 2025, respectively, primarily related to a decrease in manufacturing costs for IND-stage projects;

Previous wording

for approved products, a decrease of $1.5 million for the three months ended March 31, 2025, primarily due to reduced compassionate use costs for Dojolvi;

Current wording

for approved products, increases of $3.0 million and $1.6 million for the three and six months ended June 30, 2025, respectively, primarily due to increased compassionate use costs for Dojolvi;

Previous wording

for infrastructure, an increase of $2.1 million, primarily related to the allocation of certain staffing costs; and

Current wording

for infrastructure, decreases of $2.8 million and $0.8 million for the three and six months ended June 30, 2025, respectively, primarily related to the allocation of certain staffing costs; and

Previous wording

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

Current wording

for other research and development expenses, decreases of $8.5 million and $7.2 million for the three and six months ended June 30, 2025, respectively, primarily related to decreased staffing and cost efficiencies to support internal manufacturing, and administrative and general support.

Previous wording

Selling, general and administrative expenses increased by $9.6 million for the three months ended March 31, 2025 compared to the same period in 2024. The increase was primarily due to higher employee compensation costs as well as the recognition of reimbursement for a cost share arrangement that impacted selling, general and administrative expenses for the three months ended March 31, 2024, which did not recur during the three months ended March 31, 2025.

Current wording

Selling, general and administrative expenses increased by $6.0 million and $15.7 million for the three and six months ended June 30, 2025, respectively, compared to the same periods in 2024. The increases were primarily due to higher employee compensation costs and increased marketing expenses as we continue to plan for our future product launches, as well as the recognition of reimbursement for a cost share arrangement that reduced selling, general and administrative expenses for the three and six months ended June 30, 2024, which did not recur during the three and six months ended June 30, 2025.

Previous wording

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

Current wording

Interest income decreased by $1.6 million and $3.6 million for the three and six months ended June 30, 2025, respectively, compared to the same periods in 2024, primarily due to lower marketable debt securities balances.

Previous wording

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

Current wording

Change in fair value of equity investments increased by $4.0 million for the three months ended June 30, 2025, compared to the same period in 2024, primarily due to fluctuations in the value of our investment in Solid Biosciences Inc., or Solid, common stock.

Previous wording

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

Current wording

The non-cash interest expense on liabilities for sales of future royalties decreased by $1.9 million and $3.4 million for the three and six months ended June 30, 2025, respectively, compared to the same periods in 2024, primarily due to a reduction in total royalty obligation balances as a result of increased royalties generated from our collaboration partner, KKC. To the extent the royalty payments are greater or less than our initial estimates or the timing of such payments is materially different than our original estimates, we prospectively adjust the effective interest rate.

Previous wording

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

Current wording

Other income increased by $4.0 million and $6.4 million for the three and six months ended June 30, 2025, respectively, compared to the same periods in 2024. These changes were primarily due to fluctuations in foreign exchange rates.

Previous wording

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

Current wording

The provision for incomes taxes increased by $0.1 million and $0.9 million for the three and six months ended June 30, 2025, respectively, compared to the same periods in 2024. These increases were primarily due to increased foreign activities.

Previous wording

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

Current wording

As of June 30, 2025, we had $539.0 million in available cash, cash equivalents, and marketable debt securities. We believe that our existing capital resources will be sufficient to fund our projected operating requirements for at least the next 12 months. Our cash, cash equivalents, and marketable debt securities are held in a variety of deposit accounts, interest-bearing accounts, corporate bond securities, commercial paper, U.S. government securities, asset-backed securities, and money market funds. Cash in excess of immediate requirements is invested with a view toward liquidity and capital preservation, and we seek to minimize the potential effects of concentration and credit risk.

Previous wording

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

Current wording

In February 2024, we entered into a Sales Agreement with Cowen and Company, LLC, or Cowen, pursuant to which the Company may offer and sell shares of the Company’s common stock having an aggregate offering proceeds up to $350.0 million, from time to time, in ATM offerings through Cowen. As of June 30, 2025, 2,217,138 shares have been sold under this agreement for net proceeds of $79.7 million.

Previous wording

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

Current wording

Cash used in operating activities for the six months ended June 30, 2025 was $274.7 million and primarily reflected a net loss of $266.0 million, partially offset by non-cash items of $69.7 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 $78.4 million, primarily due to a net decrease in accounts payable, accrued and other liabilities primarily due to the payout of the 2024 annual bonuses and decreases in accrued collaboration for payment of a milestone to a collaboration partner of $30.0 million, an increase in prepaid manufacturing expense, as well as an increase in accounts receivable related to timing of orders and collections.

Previous wording

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

Current wording

Cash used in operating activities for the six months ended June 30, 2024 was $267.7 million and primarily reflected a net loss of $302.3 million, partially offset by non-cash items of $74.3 million, net, which consisted primarily of stock-based compensation, amortization of discounts on marketable debt securities, depreciation and amortization, change in fair value of equity investments, 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 $39.7 million, primarily due to an increase in accounts receivable primarily related to an increase in sales of our approved products and timing of when orders were received, combined with a net decrease in accounts payable, accrued and other liabilities primarily due to the payout of the 2023 annual bonus and for the payment of Medicaid obligations.

Previous wording

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

Current wording

Cash provided by investing activities for the six months ended June 30, 2025 was $191.1 million and was primarily related to $210.9 million from net activities in marketable debt securities, partially offset by the payment to a collaboration partner of $15.0 million for the achievement of a milestone under the collaboration agreement recorded as an intangible asset.

Previous wording

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

Current wording

Cash provided by investing activities for the six months ended June 30, 2024 was $155.0 million and was primarily related to $174.8 million from net activities in marketable debt securities, offset by payments to a collaboration partner of $12.5 million for the achievement of milestones under the collaboration agreement recorded as an intangible asset.

Previous wording

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

Current wording

Future minimum lease payments under non-cancellable leases as of June 30, 2025, were approximately $43.7 million, of which $15.0 million is due within one year.

Previous wording

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

Current wording

Manufacturing and service contract obligations primarily relate to manufacturing of inventory for our approved products. As of June 30, 2025, we had obligations of approximately $114.4 million, of which $100.0 million is due within one year.
Rearranged current paragraphs (1)

Current report — wording carried from elsewhere

Bayley-4 cognitive raw score, and the key secondary endpoint will be the Multi-domain Responder Index (MDRI) across the five domains of cognition, receptive communication, behavior, gross motor function, and sleep.
Rearranged previous paragraphs (1)

Previous report — wording carried elsewhere

GTX-102 for the treatment of Angelman Syndrome