ABEONA THERAPEUTICS INC.
For the period ended Sep 30, 2025. Filed Nov 12, 2025.
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, 2025 · Filed Nov 12, 2025
ABEO$4.17+6.6%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2025-06-30; filed 2025-08-14.
0001641172-25-023716
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2025-09-30; filed 2025-11-12.
0001493152-25-021723
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 89 previous paragraphs and 87 current paragraphs.
43 unchanged; 32 reworded; 12 added; 9 removed; 5 previous and 0 current paragraphs rearranged.
Tables are not compared: 9 previous and 7 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 (12)
Current report
ZEVASKYN® is manufactured at our current Good Manufacturing Practices (“cGMP”) manufacturing facility in Cleveland, Ohio, and is made available through ZEVASKYN® Qualified Treatment Centers (“QTCs”).
Current report
During the third quarter, we manufactured a full batch of drug product following patient biopsy collection in August 2025. Although we produced bonafide drug product, it could not be released because a rapid sterility assay, mandated by the FDA as a release assay during the final stage of the Biologics License Application (BLA) review, initially yielded a false positive result for sterility. We immediately implemented a temporary pause on collecting patient biopsies and worked diligently to optimize the release assay to ensure reliable results and avoid any unnecessary future lot rejections. Upon completion of assay optimization and the necessary regulatory submission for its implementation, we resumed biopsy collection in November 2025.
Current report
On October 13, 2025, we announced that ABO-503 for XLRS was selected by the FDA for Rare Disease Endpoint Advancement Pilot (“RDEA”) Program. The RDEA program facilitates the development and timely approval of rare disease therapies by supporting the use of novel efficacy endpoints in clinical trials. As part of the RDEA program, Abeona will have opportunities for enhanced communication and collaboration with the FDA, including frequent advice and regular ad-hoc conversations to accelerate the development and validation of product-specific novel efficacy endpoints for Abeona’s XLRS program.
Current report
On October 20, 2025, we announced the appointment of James A. Gow, MD, MBA, MS, MHCM, as the Senior Vice President, Head of Clinical Development & Medical Affairs, effective immediately. Dr. Gow has over 20 years of industry experience in clinical development and medical affairs and is a recognized expert in gene therapy, especially in ophthalmology.
Current report
On October 30, 2025, we announced that the Centers for Medicare and Medicaid Services (“CMS”) has established a permanent Healthcare Common Procedure Coding System (“HCPCS”) J-code for ZEVASKYN®. The new J-code for ZEVASKYN®, J3389 (Topical administration, prademagene zamikeracel, per treatment) becomes effective on January 1, 2026.
Current report
Cost of sales
Current report
Cost of sales during the three months ended September 30, 2025 primarily includes costs associated with the August 2025 production of a full batch of ZEVASKYN®that could not be released due to technical issues that arose in implementing the rapid sterility lot release assay, mandated by the FDA during BLA review. There was no cost of sales in the same period of 2024, as ZEVASKYN®was approved by the FDA in April 2025.
Current report
Income tax benefit
Current report
Comparison of Nine Months Ended September 30, 2025 and September 30, 2024
Current report
Cost of sales
Current report
Cost of sales during the nine months ended September 30, 2025 primarily includes costs associated with the August 2025 production of a full batch of ZEVASKYN®that could not be released due to technical issues that arose in implementing the rapid sterility lot release assay, mandated by the FDA during BLA review. There was no cost of sales in the same period of 2024, as ZEVASKYN®was approved by the FDA in April 2025.
Current report
Other income, which consists primarily of sublease income, was $0.4 million for the nine months ended September 30, 2025, as compared to $0.5 million in the same period of 2024.
Removed paragraphs (9)
Previous report
In connection with the FDA approval of ZEVASKYN™, Abeona received a Rare Pediatric Disease Priority Review Voucher (“PRV”). ZEVASKYN™ has been granted Orphan Drug and Rare Pediatric Disease designations by the FDA and Orphan Drug Designation by the European Medicines Agency (“EMA”). On May 9, 2025, we entered into a definitive asset purchase agreement to sell our PRV for gross proceeds of $155.0 million. The transaction closed on June 27, 2025 and we received $155.0 million in gross proceeds from the sale of the PRV.
Previous report
We established a current Good Manufacturing Practices (“cGMP”) commercial facility in Cleveland, Ohio for manufacturing ZEVASKYN™ drug product to support our commercial launch. ZEVASKYN™ will be made available beginning in the third quarter of 2025 through ZEVASKYN™ Qualified Treatment Centers (“QTCs”). The QTCs are well-recognized epidermolysis bullosa treatment centers with cell and gene therapy experience, situated across the U.S. to ensure patients nationwide have access to this important treatment. On May 14, 2025, Ann & Robert H. Lurie Children’s Hospital of Chicago (“Lurie Children’s”) was activated as the first QTC for ZEVASKYN™. Lurie Children’s has completed QTC start-up activities enabling it to begin patient identification for scheduling of ZEVASKYN™ treatment. Treatments are expected to begin in the third quarter of 2025.
Previous report
In June 2025, a third-party exercised its option for $0.4 million to license certain of our AAV capsids. This worldwide, non-exclusive license is pursuant to the agreement between Abeona and the third party, announced in July 2024, to evaluate the therapeutic potential of AAV204.
Previous report
Comparison of Six Months Ended June 30, 2025 and June 30, 2024
Previous report
License and other revenues
Previous report
Royalties
Previous report
Excluding these costs, the increase in expenses of $6.2 million was primarily due to:
Previous report
Other income was $0.2 million for the six months ended June 30, 2025, as compared to $0.4 million in the same period of 2024. Other income includes sublease income, which had a minor reduction period over period.
Previous report
Income tax expense
Reworded paragraphs (32)
Previous wording
Abeona is a commercial-stage biopharmaceutical company developing cell and gene therapies for life-threatening diseases. On April 28, 2025, the U.S. Food and Drug Administration (“FDA”) approved ZEVASKYN™ (prademagene zamikeracel) gene-modified cellular sheets, also known as pz-cel, as the first and only autologous cell-based gene therapy for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (“RDEB”), a serious and debilitating genetic skin disease. There is no cure for RDEB, and ZEVASKYN™ is the only FDA-approved product to treat RDEB wounds with a single application.
Current wording
Abeona is a commercial-stage biopharmaceutical company developing cell and gene therapies for life-threatening diseases. On April 28, 2025, the U.S. Food and Drug Administration (“FDA”) approved ZEVASKYN® (prademagene zamikeracel) gene-modified cellular sheets, also known as pz-cel, as the first and only autologous cell-based gene therapy for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (“RDEB”), a serious and debilitating genetic skin disease. There is no cure for RDEB, and ZEVASKYN® is the only FDA-approved product to treat RDEB wounds with a single application. ZEVASKYN® was granted Orphan Drug and Rare Pediatric Disease designations by the FDA.
Previous wording
On July 15, 2025, we announced the activation of the newest QTC for ZEVASKYN™ at Lucile Packard Children’s Hospital Stanford. As a result, RDEB patients will be able to access ZEVASKYN™ at both Lucile Packard Children’s Hospital Stanford and Lurie Children’s Hospital of Chicago.
Current wording
On October 8, 2025, we announced the activation of the newest QTC for ZEVASKYN®at Children’s Hospital Colorado. As a result, RDEB patients will be able to access ZEVASKYN®at Lucile Packard Children’s Hospital Stanford, Lurie Children’s Hospital of Chicago and Children’s Hospital Colorado.
Previous wording
Research and development expenses include, but are not limited to, payroll and personnel expense, preclinical lab supplies, preclinical and development costs, clinical trial costs, preclinical manufacturing and manufacturing facility costs, costs associated with regulatory approvals, preclinical depreciation on lab supplies and manufacturing facilities, and preclinical consultant-related expenses.
Current wording
Research and development expenses include, but are not limited to, payroll and personnel expenses, preclinical lab supplies, preclinical and development costs, clinical trial costs, preclinical manufacturing and manufacturing facility costs, costs associated with regulatory approvals, preclinical depreciation on lab supplies and manufacturing facilities, and preclinical consultant-related expenses.
Previous wording
Total research and development spending for the three months ended June 30, 2025 was $5.9 million, as compared to $9.2 million for the same period of 2024, a decrease of $3.3 million. The reduction in expenses was primarily due to $1.4 million of costs capitalized into inventory and $4.9 million of costs such as engineering runs and other production costs that are no longer considered research and development due to FDA approval of ZEVASKYN™ in April of 2025. Excluding this, total costs would have increased by $3.0 million due to $1.7 million increase in salaries and non-cash stock-based compensation costs due to increased headcount related to scale up of manufacturing capacity in preparation for the planned launch of ZEVASKYN™ and $1.3 million in pre-clinical development work.
Current wording
Total research and development spending for the three months ended September 30, 2025 was $4.2 million, as compared to $8.9 million for the same period of 2024, a decrease of $4.7 million. The reduction in expenses was primarily due to costs capitalized into inventory and engineering runs and other production costs that are no longer considered research and development due to FDA approval of ZEVASKYN®in April of 2025.
Previous wording
Selling, general and administrative expenses primarily consist of payroll and personnel costs, office facility costs, public reporting company related costs, professional fees (e.g., legal expenses), selling and other costs for planned commercial launch and other general operating expenses not otherwise included in research and development expenses.
Current wording
Selling, general and administrative expenses primarily consist of payroll and personnel costs, office facility costs, public reporting company related costs, professional fees (e.g., legal expenses), selling and other costs for commercial launch and other general operating expenses not otherwise included in research and development expenses. We expect our selling, general, and administrative costs to continue to increase as we launch ZEVASKYN®and advance other product candidates toward potential regulatory approval.
Previous wording
Total selling, general and administrative expenses were $17.1 million for the three months ended June 30, 2025, as compared to $8.6 million for the same period of 2024, an increase of $8.5 million. The increase in expenses was primarily due to approximately $4.9 million of costs such as engineering runs and other production costs that are no longer considered research and development due to FDA approval in April of 2025. Excluding these costs, the increase in expenses of $3.7 million was primarily due to:
Current wording
Total selling, general and administrative expenses were $19.3 million for the three months ended September 30, 2025, as compared to $6.4 million for the same period of 2024, an increase of $12.9 million. The increase in expenses was primarily due to increases in commercial costs related to our continued commercialization efforts, increased legal costs associated with commercialization, increases of salaries and stock-based compensation due to new hires, and costs related to engineering runs and other production costs that are no longer considered research and development due to FDA approval in April of 2025.
Previous wording
Interest income was $2.3 million for the six months ended June 30, 2025, as compared to $2.0 million in the same period of 2024. The increase resulted from higher earnings on short-term investments driven by increased average short-term investment balances.
Current wording
Interest income was $1.7 million for the three months ended September 30, 2025, as compared to $1.2 million in the same period of 2024. The increase resulted from higher earnings on short-term investments driven by increased average short-term investment balances.
Previous wording
Interest expense was $1.0 million for the three months ended June 30, 2025 compared to $1.1 million in the same period of 2024. Interest expense is due to the credit facility entered into by the Company in January 2024.
Current wording
Interest expense was $0.9 million for the three months ended September 30, 2025 compared to $1.1 million in the same period of 2024. Interest expense was due to the credit facility entered into by the Company in January 2024 and decreased as a result of the July 2025 Loan Agreement Amendment.
Previous wording
The change in fair value of warrant liabilities was a loss of $5.4 million for the three months ended June 30, 2025. We issued stock purchase warrants that are required to be classified as a liability and valued at fair market value at each reporting period. The loss in the fair value of warrant liabilities was primarily due to the increase in our stock price year over year and a shorter term of the outstanding warrants.
Current wording
The change in fair value of warrant liabilities was a gain of $2.8 million for the three months ended September 30, 2025. We issued stock purchase warrants that are required to be classified as a liability and valued at fair market value at each reporting period. The gain in the fair value of warrant liabilities was primarily due to the decrease in our stock price over the quarter and a shorter term of the outstanding warrants.
Previous wording
The change in fair value of warrant and derivative liabilities was a gain of $24.9 million for the three months ended June 30, 2024. We issued stock purchase warrants that are required to be classified as a liability and valued at fair market value at each reporting period. In addition, the conversion feature in our loan agreement is required to be classified as a liability and valued at fair market value at each reporting period. The gain in the fair value of warrant and derivative liabilities was primarily due to the decrease in our stock price over the year and a reduced term of each of the warrants and derivative liabilities.
Current wording
The change in fair value of warrant and derivative liabilities was a loss of $15.2 million for the three months ended September 30, 2024. We issued stock purchase warrants that are required to be classified as a liability and valued at fair market value at each reporting period. In addition, the conversion feature in our loan agreement is required to be classified as a liability and valued at fair market value at each reporting period. The loss in the fair value of warrant and derivative liabilities was primarily due to the increase in our stock price over the quarter offset by the reduced term of each of the warrants and derivative liabilities.
Previous wording
Other income was $0.1 million for the three months ended June 30, 2025, as compared to $0.2 million in the same period of 2024. Other income includes sublease income, which had a minor reduction period over period.
Current wording
Other income, which consists primarily of sublease income, was $0.1 million for the three months ended September 30, 2025, as compared to $0.1 million in the same period of 2024.
Previous wording
We recorded a current income tax expense of $15.5 million for the six months ended June 30, 2025. We did not record an income tax expense for the six months ended June 30, 2024 as we generated sufficient tax losses, after consideration of discrete items. The current income tax expense for the six months ended June 30, 2025 was driven by pre-tax income from the gain on sale of the PRV, resulting in $14.6 million of federal income tax expense and $0.9 million of state income tax expense. We are currently evaluating the impact of the One Big Beautiful Bill Act on future periods; however, we expect that the favorable changes from the new law will significantly reduce current tax due for 2025. The legislation includes several changes to federal tax law that generally allow for more favorable deductibility of certain business expenses beginning in 2025, including the restoration of immediate expensing of domestic R&D expenditures, reinstatement of 100% bonus depreciation, and more favorable rules for determining the limitation on business interest expense.
Current wording
We recorded a current income tax benefit of $15.2 million for the three months ended September 30, 2025. We did not record an income tax expense for the three months ended September 30, 2024 as we generated sufficient tax losses, after consideration of discrete items. The current income tax benefit for the three months ended September 30, 2025 was by the favorable impact of the One Big Beautiful Bill Act, enacted on July 4, 2025. The legislation restored immediate expensing of domestic R&D expenditures, reinstated 100% bonus depreciation, and provided more favorable rules for determining the limitation on business interest expense, which collectively reduced our taxable income and resulted in a current tax benefit for the three months ended September 30, 2025.
Previous wording
License and other revenues for the three months ended June 30, 2025 was $0.4 million as compared to nil for the same period of 2024. The revenue in 2025 of $0.4 million consists of revenue resulting from a third party exercising its option to license certain of our AAV capsids.
Current wording
License and other revenues for the nine months ended September 30, 2025 was $0.4 million as compared to nil for the same period of 2024. The revenue in 2025 of $0.4 million consists of revenue resulting from a third party exercising its option to license certain of our AAV capsids.
Previous wording
Total royalty expense for the three months ended June 30, 2025 was $0.1 million as compared to nil for the same period of 2024. The increase in expense was due to royalties owed to the University of North Carolina at Chapel Hill resulting from the milestones due from the exercise of an option by a third party to license certain of our AAV capsids.
Current wording
Total royalty expense for the nine months ended September 30, 2025 was $0.1 million as compared to nil for the same period of 2024. The increase in expense was due to royalties owed to the University of North Carolina at Chapel Hill resulting from the milestones due from the exercise of an option by a third party to license certain of our AAV capsids.
Previous wording
Total research and development spending for the six months ended June 30, 2025 was $15.9 million, as compared to $16.4 million for the same period of 2024, a decrease of $0.5 million. The reduction in expenses was primarily due to $1.4 million of costs capitalized into inventory and $4.9 million of costs such as engineering runs and other production costs that are no longer considered research and development due to FDA approval of ZEVASKYN™ in April of 2025. Excluding this, total costs would have increased by $5.8 million due to $3.0 million increase in salaries and non-cash stock-based compensation costs due to increased headcount related to scale up of manufacturing capacity in preparation for the planned launch of ZEVASKYN™ and $2.8 million in pre-clinical development work.
Current wording
Total research and development spending for the nine months ended September 30, 2025 was $20.1 million, as compared to $25.4 million for the same period of 2024, a decrease of $5.3 million. The reduction in expenses was primarily due to costs capitalized into inventory and engineering runs and other production costs that are no longer considered research and development due to FDA approval of ZEVASKYN®in April of 2025.
Previous wording
Total general and administrative expenses were $26.9 million for the six months ended June 30, 2025, as compared to $15.8 million for the same period of 2024, an increase of $11.1 million. The increase in expenses was primarily due to approximately $4.9 million of costs such as engineering runs and other production costs that are no longer considered research and development due to FDA approval of ZEVASKYN™ in April of 2025.
Current wording
Total selling, general and administrative expenses were $46.2 million for the nine months ended September 30, 2025, as compared to $22.2 million for the same period of 2024, an increase of $24.0 million. The increase in expenses was primarily due to increases in commercial costs related to our continued commercialization efforts, increased legal costs associated with commercialization, increases of salaries and stock-based compensation due to new hires, and to costs related to engineering runs and other production costs that are no longer considered research and development due to FDA approval in April of 2025.
Previous wording
Interest income was $1.0 million for the three months ended June 30, 2025, as compared to $1.2 million in the same period of 2024. The decrease resulted from lower earnings on short-term investments driven by decreased average short-term investment balances.
Current wording
Interest income was $4.0 million for the nine months ended September 30, 2025, as compared to $3.2 million in the same period of 2024. The increase resulted from higher earnings on short-term investments driven by increased average short-term investment balances.
Previous wording
Interest expense was $2.0 million for the six months ended June 30, 2025 and 2024. Interest expense is due to the credit facility entered into by the Company in January 2024.
Current wording
Interest expense was $2.9 million for the nine months ended September 30, 2025, as compared to $3.1 million in the same period of 2024. Interest expense was due to the credit facility entered into by the Company in January 2024 and decreased as a result of the July 2025 Loan Agreement Amendment.
Previous wording
The change in fair value of warrant liabilities was a gain of $1.9 million for the six months ended June 30, 2025. We issued stock purchase warrants that are required to be classified as a liability and valued at fair market value at each reporting period. The gain in the fair value of warrant liabilities was primarily due to the shorter term period over period.
Current wording
The change in fair value of warrant liabilities was a gain of $4.6 million for the nine months ended September 30, 2025. We issued stock purchase warrants that are required to be classified as a liability and valued at fair market value at each reporting period. The gain in the fair value of warrant liabilities was primarily due to the decrease in our stock price as of September 30, 2025 compared to December 31, 2024 and to the shorter term period over period.
Previous wording
The change in fair value of warrant and derivative liabilities was a gain of $7.6 million for the six months ended June 30, 2024. In 2024, we issued stock purchase warrants that are required to be classified as a liability and valued at fair market value at each reporting period. In addition, the conversion feature in our loan agreement was required to be classified as a liability through September 30, 2024 and was valued at fair market value at each reporting period during the nine-month period ending September 30, 2024. The change in the fair value of warrant and derivative liabilities was primarily due to the decrease in our stock price year as of June 30, 2024 compared to December 31, 2023.
Current wording
The change in fair value of warrant and derivative liabilities was a loss of $7.5 million for the nine months ended September 30, 2024. In 2024, we issued stock purchase warrants that are required to be classified as a liability and valued at fair market value at each reporting period. In addition, the conversion feature in our loan agreement was required to be classified as a liability through September 30, 2024 and was valued at fair market value at each reporting period during the nine-month period ending September 30, 2024. The change in the fair value of warrant and derivative liabilities was primarily due to the increase in our stock price as of September 30, 2024 compared to December 31, 2023.
Previous wording
In May, 2025, we sold our PRV awarded to us following the FDA approval of ZEVASKYN™. We received gross proceeds of $155.0 million during the six months ended June 30, 2025 and recognized a gain from the PRV sale of $152.4 million, net of transaction costs of $2.6 million, as it did not have a carrying value at the time of sale.
Current wording
In May 2025, we sold our PRV awarded to us following the FDA approval of ZEVASKYN®. We received gross proceeds of $155.0 million during the nine months ended September 30, 2025 and recognized a gain from the PRV sale of $152.4 million, net of transaction costs of $2.6 million, as it did not have a carrying value at the time of sale.
Previous wording
We recorded a current income tax expense of $15.5 million for the three months ended June 30, 2025. We did not record an income tax expense for the three months ended June 30, 2024 as we generated sufficient tax losses, after consideration of discrete items. The current income tax expense for the three months ended June 30, 2025 was driven by pre-tax income from the gain on sale of the PRV, resulting in $14.6 million of federal income tax expense and $0.9 million of state income tax expense. We are currently evaluating the impact of the One Big Beautiful Bill Act on future periods; however, we expect that the favorable changes from the new law will significantly reduce current tax due for 2025. The legislation includes several changes to federal tax law that generally allow for more favorable deductibility of certain business expenses beginning in 2025, including the restoration of immediate expensing of domestic R&D expenditures, reinstatement of 100% bonus depreciation, and more favorable rules for determining the limitation on business interest expense.
Current wording
We recorded a current income tax expense of $0.3 million for the nine months ended September 30, 2025. We did not record an income tax expense for the nine months ended September 30, 2024 as we generated sufficient tax losses, after consideration of discrete items. The current income tax expense for the nine months ended September 30, 2025 was driven by pre-tax income from the gain on sale of the PRV, offset by the favorable impact of the One Big Beautiful Bill Act, enacted on July 4, 2025. The legislation restored immediate expensing of domestic R&D expenditures, reinstated 100% bonus depreciation, and provided more favorable rules for determining the limitation on business interest expense, which collectively reduced the Company’s taxable income and resulting income tax expense for the nine months ended September 30, 2025.
Previous wording
Cash Flows for the Six Months Ended June 30, 2025 and 2024
Current wording
Cash Flows for the Nine Months Ended September 30, 2025 and 2024
Previous wording
Net cash used in operating activities was $37.2 million for the six months ended June 30, 2025, primarily comprised of our net income of $96.8 million and increases in operating assets and liabilities of $12.4 million offset by net non-cash charges of $146.2 million. Non-cash charges consisted primarily of $152.4 million gain on sale of priority review voucher for which the cash proceeds are recorded in investing activities, $1.9 million of gain as a result of the change in fair value of warrant and derivative liabilities, $5.5 million of stock-based compensation and $1.1 million of depreciation and amortization.
Current wording
Net cash used in operating activities was $58.4 million for the nine months ended September 30, 2025, primarily comprised of our net income of $91.6 million, offset by a decrease in operating assets and liabilities of $4.8 million and net non-cash charges of $145.2 million. Non-cash charges consisted primarily of $152.4 million gain on sale of priority review voucher for which the cash proceeds are recorded in investing activities, $4.6 million of gain as a result of the change in fair value of warrant and derivative liabilities, $8.2 million of stock-based compensation and $1.7 million of depreciation and amortization.
Previous wording
Net cash used in operating activities was $27.2 million for the six months ended June 30, 2024, primarily comprised of our net loss of $24.2 million and decreases in operating assets and liabilities of $0.4 million and net non-cash charges of $2.6 million. Non-cash charges consisted primarily of $(7.6) million of the change in fair value of warrant and derivative liabilities, $2.9 million of stock-based compensation and $1.0 million of depreciation and amortization.
Current wording
Net cash used in operating activities was $39.5 million for the nine months ended September 30, 2024, primarily comprised of our net loss of $54.4 million and decreases in operating assets and liabilities of $0.5 million and net non-cash charges of $15.5 million. Non-cash charges consisted primarily of $7.5 million of the change in fair value of warrant and derivative liabilities, $4.7 million of stock-based compensation, $1.1 million of non-cash interest expense and $1.5 million of depreciation and amortization.
Previous wording
Net cash provided by investing activities was $160.1 million for the six months ended June 30, 2025, primarily comprised of net proceeds from sale of priority review voucher of $152.4 million, proceeds from maturities of short-term investments of $80.5 million, offset by purchases of short-term investments of $68.5 million and capital expenditures of $4.3 million.
Current wording
Net cash provided by investing activities was $95.5 million for the nine months ended September 30, 2025, primarily comprised of net proceeds from sale of priority review voucher of $152.4 million, proceeds from maturities of short-term investments of $118.8 million, offset by purchases of short-term investments of $168.9 million and capital expenditures of $6.8 million.
Previous wording
Net cash used in investing activities was $51.9 million for the six months ended June 30, 2024, primarily comprised of proceeds from maturities of short-term investments of $39.4 million, offset by purchases of short-term investments of $89.9 million and capital expenditures of $1.4 million.
Current wording
Net cash used in investing activities was $58.1 million for the nine months ended September 30, 2024, primarily comprised of proceeds from maturities of short-term investments of $90.2 million, offset by purchases of short-term investments of $146.5 million and capital expenditures of $1.8 million.
Previous wording
Net cash provided by financing activities was $17.3 million for the six months ended June 30, 2025, primarily comprised of proceeds of $17.3 million from open market sales of common stock pursuant to the ATM Agreement (as defined below).
Current wording
Net cash provided by financing activities was $22.4 million for the nine months ended September 30, 2025, primarily comprised of proceeds of $17.3 million from open market sales of common stock pursuant to the ATM Agreement (as defined below) and proceeds of $5.2 million from the exercise of stock purchase warrants.
Previous wording
Net cash provided by financing activities was $99.1 million for the six months ended June 30, 2024, primarily comprised of $70.2 million in net proceeds from sales of common stock, $10.0 million from open market sales of common stock pursuant to the ATM Agreement (as defined below) and net proceeds of $19.0 million from our January 2024 Loan Agreement.
Current wording
Net cash provided by financing activities was $98.8 million for the nine months ended September 30, 2024, primarily comprised of $70.2 million in net proceeds from sales of common stock, $10.0 million from open market sales of common stock pursuant to the ATM Agreement (as defined below) and net proceeds of $19.0 million from our January 2024 Loan Agreement.
Previous wording
Our principal source of liquidity is cash, cash equivalents, restricted cash and short-term investments, collectively referred to as our cash resources. As of June 30, 2025, our cash resources were $225.9 million, which include the $155.0 million in gross proceeds from the sale of our priority review voucher. We believe that our current cash and cash equivalents, restricted cash and short-term investments are sufficient to fund operations through at least the next 12 months from the date of this report on Form 10-Q. We may need to secure additional funding to carry out all of our planned research and development and potential commercialization activities. If we are unable to obtain additional financing or generate license or product revenue, the lack of liquidity and sufficient capital resources could have a material adverse effect on our future prospects.
Current wording
Our principal source of liquidity is cash, cash equivalents, restricted cash and short-term investments, collectively referred to as our cash resources. As of September 30, 2025, our cash resources were $207.5 million. We believe that our current cash and cash equivalents, restricted cash and short-term investments are sufficient to fund operations through at least the next 12 months from the date of this report on Form 10-Q. We may need to secure additional funding to carry out all of our planned research and development and potential commercialization activities. If we are unable to obtain additional financing or generate license or product revenue, the lack of liquidity and sufficient capital resources could have a material adverse effect on our future prospects.
Previous wording
We have an open market sale agreement with Jefferies LLC (as amended, the “ATM Agreement”) pursuant to which, we may sell from time to time, through Jefferies LLC, shares of our common stock for an aggregate sales price of up to $75.0 million. Any sales of shares pursuant to this agreement are made under our effective “shelf” registration statement on Form S-3 that is on file with and has been declared effective by the SEC. We sold 3,510,889 shares of our common stock under the ATM Agreement and received $17.3 million of net proceeds during the six months ended June 30, 2025. We sold 1,902,376 shares of our common stock under the ATM Agreement and received $10.0 million of net proceeds during the six months ended June 30, 2024. Under the ATM Agreement and as of June 30, 2025, we have remaining shares of our common stock for an aggregate sales price of up to $51.5 million.
Current wording
We have an open market sale agreement with Jefferies LLC (as amended, the “ATM Agreement”) pursuant to which, we may sell from time to time, through Jefferies LLC, shares of our common stock for an aggregate sales price of up to $75.0 million. Any sales of shares pursuant to this agreement are made under our effective “shelf” registration statement on Form S-3 that is on file with and has been declared effective by the SEC. We sold 3,510,889 shares of our common stock under the ATM Agreement and received $17.3 million of net proceeds during the nine months ended September 30, 2025. We sold 1,902,376 shares of our common stock under the ATM Agreement and received $10.0 million of net proceeds during the nine months ended September 30, 2024. Under the ATM Agreement and as of September 30, 2025, we have remaining shares of our common stock for an aggregate sales price of up to $51.5 million.
Previous wording
Due to uncertainties and certain of the risks described above, under “Risk Factors” and elsewhere in this Quarterly Report on Form 10-Q and in our Annual Report, it is not possible to reliably predict future spending or time to completion by project or product category or the period in which material net cash inflows from significant projects are expected to commence. If we are unable to timely complete a particular project, our research and development efforts could be delayed or reduced, our business could suffer depending on the significance of the project and we might need to raise additional capital to fund operations, as discussed in the risks above.
Current wording
Due to uncertainties and certain of the risks described above, under “Risk Factors” and elsewhere in this Quarterly Report on Form 10-Q and in our Annual Report, it is not possible to reliably predict future spending or time to completion by project or product category or the period in which material net cash inflows from significant projects are expected to commence. If we are unable to timely complete a particular project, our research and development efforts could be delayed or reduced, our business could suffer depending on the significance of the project and we might need to raise additional capital to fund operations, as discussed above.
Rearranged previous paragraphs (5)
Previous report — wording carried elsewhere
In May 2025, we sold our PRV awarded to us following the FDA approval of ZEVASKYN™. We received gross proceeds of $155.0 million during the three months ended June 30, 2025 and recognized a gain the PRV sale of $152.4 million, net of transaction costs of $2.6 million, as it did not have a carrying value at the time of sale.
Previous report — wording carried elsewhere
License and other revenues for the six months ended June 30, 2024 was $0.4 million as compared to nil for the same period of 2024. The revenue in 2025 of $0.4 million consists of revenue resulting from a third party exercising its option to license certain of our AAV capsids.
Previous report — wording carried elsewhere
Total royalty expense for the six months ended June 30, 2025 was $0.1 million as compared to nil for the same period of 2024. The increase in expense was due to royalties owed to the University of North Carolina at Chapel Hill resulting from the milestones due from the exercise of an option by a third party to license certain of our AAV capsids.
Previous report — wording carried elsewhere
Selling, general and administrative expenses primarily consist of payroll and personnel costs, office facility costs, public reporting company related costs, professional fees (e.g., legal expenses), selling and other costs for planned commercial launch and other general operating expenses not otherwise included in research and development expenses.
Previous report — wording carried elsewhere
Gain from sale of priority review voucher, net