ZEVRA THERAPEUTICS, INC.

Previous wording

You should read the following discussion and analysis of our financial condition and results of operations together with our unaudited condensed consolidated financial statements and related notes thereto included elsewhere in this Quarterly Report on Form 10-Q. Some of the information contained in this discussion and analysis or set forth elsewhere in this Quarterly Report on Form 10-Q, including information with respect to our plans and strategy for our business and related financing, includes forward-looking statements that involve risks and uncertainties. As a result of many factors, including those factors set forth in Part II, Item 1A. “Risk Factors” of this Quarterly Report on Form 10-Q and Part I, Item 1A. “Risk Factors” ofour Annual Report on Form 10-K for the fiscal year ended December 31, 2025, filed with the SEC on March 9, 2026, our actual results could differ materially from the results described in or implied by the forward-looking statements contained in the following discussion and analysis.

Current wording

You should read the following discussion and analysis of our financial condition and results of operations together with our unaudited condensed consolidated financial statements and related notes thereto included elsewhere in this Quarterly Report on Form 10-Q. Some of the information contained in this discussion and analysis or set forth elsewhere in this Quarterly Report on Form 10-Q, including information with respect to our plans and strategy for our business and related financing, includes forward-looking statements that involve risks and uncertainties. As a result of many factors, including those factors set forth in Part II, Item 1A. “Risk Factors” of this Quarterly Report on Form 10-Q and Part I, Item 1A. “Risk Factors” ofour Annual Report on Form 10-K for the fiscal year ended December 31, 2025, filed with the SEC on March 9, 2026, as supplemented in Part II, Item 1A. “Risk Factors” of our Quarterly Report on Form 10-Q for the quarter ended March 31, 2026, filed with the SEC on May 6, 2026, our actual results could differ materially from the results described in or implied by the forward-looking statements contained in the following discussion and analysis.

Previous wording

We are a commercial-stage company with a late-stage pipeline committed to redefining what is possible in bringing life-changing therapeutics to people living with rare diseases. We are focused on expanding patient access through geographic expansion opportunities, progressing and increasing our pipeline, and delivering meaningful therapeutics. Our vision is realized through disciplined execution of our strategic plan and our core values — patient centricity, integrity, accountability, innovation, and courage — which guide our efforts to deliver long-term value. The commercialization of our lead product, marketed in the United States for Niemann-Pick disease type C ("NPC"), a rare, progressive neurodegenerative disorder, provides a strong corporate foundation and demonstrates our ability to advance therapies from development to market.

Current wording

We are a commercial-stage company with a late-stage pipeline committed to redefining what is possible in bringing life-changing therapeutics to people living with rare diseases. We are focused on broadening access through geographic expansion opportunities, progressing our pipeline toward key milestones, and delivering meaningful therapeutics. Our vision is realized through disciplined execution of our strategic plan and our core values — patient centricity, integrity, accountability, innovation, and courage — which guide our efforts to deliver long-term value. The commercialization of our lead product, marketed in the United States for Niemann-Pick disease type C ("NPC"), a rare, progressive neurodegenerative disorder, provides a strong corporate foundation and demonstrates our ability to advance therapies from development to market.

Previous wording

On September 20, 2024, the U.S. Food and Drug Administration (“FDA”) approved the New Drug Application (“NDA”) for MIPLYFFA, for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients 2 years of age and older, and MIPLYFFA became commercially available for dispense in the United States in November 2024. In connection with this approval, we received a transferable rare pediatric disease priority review voucher (“PRV”). On April 1, 2025, we consummated the sale of the PRV, resulting in net proceeds of $148.3 million to us. Arimoclomol has also been granted orphan medicinal product designation for the treatment of NPC by the European Commission. We are pursuing regulatory approval of arimoclomol in Europe and filed a Marketing Authorization Application (“MAA”) with the European Medicines Agency (“EMA”) in July 2025; the application is currently under review.

Current wording

On September 20, 2024, the U.S. Food and Drug Administration (“FDA”) approved the New Drug Application (“NDA”) for MIPLYFFA, for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients 2 years of age and older, and MIPLYFFA became commercially available for dispense in the United States in November 2024. In connection with this approval, we received a transferable rare pediatric disease priority review voucher (“PRV”). On April 1, 2025, we consummated the sale of the PRV, resulting in net proceeds of $148.3 million to us. Arimoclomol has also been granted orphan medicinal product designation for the treatment of NPC by the European Commission. We are pursuing regulatory approval of arimoclomol in Europe and filed a Marketing Authorization Application (“MAA”) with the European Medicines Agency (“EMA”) in July 2025. In July 2026, we announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a negative opinion regarding the MAA. Under European regulatory procedures, we have requested a re-examination of the CHMP opinion.

Previous wording

We have had recurring negative net operating cash flows throughout our operating history, and we cannot guarantee or predict when we may begin to consistently generate positive net cash flows from operations, if at all. Net cash provided by (used in) operating activities for the three months ended March 31, 2026, and 2025, was$6.1 million and $(8.2) million, respectively. We expect to continue to incur significant expenses and minimal positive net cash flows from operations or negative net cash flows from operations for the near future, and those expenses and losses may fluctuate significantly from quarter-to-quarter and year-to-year. We anticipate that our expenses will fluctuate substantially as we:

Current wording

While we have recently had several quarters of positive net cash flows from operations, we previously had recurring negative net operating cash flows, and we cannot guarantee that we can continue to generate positive net cash flows from operations. Net cash provided by (used in) operating activities for the six months ended June 30, 2026, and 2025, was$23.2 million and $(11.8) million, respectively. We expect to continue to incur significant expenses and minimal positive net cash flows from operations or negative net cash flows from operations for the near future, and those expenses and losses may fluctuate significantly from quarter-to-quarter and year-to-year. We anticipate that our expenses will fluctuate substantially as we:

Previous wording

Effective therapies to treat NPC are desperately needed, and, for this reason, arimoclomol is currently being made available to NPC patients in France, Germany, and other EU member states, along with select territories outside of Europe under our global EAP.

Current wording

Effective therapies to treat NPC are desperately needed, and, for this reason, arimoclomol is currently being made available to NPC patients in France, Germany, and other EU member states, along with select territories outside of Europe under our global EAP. Arimoclomol has also been granted orphan medicinal product designation for the treatment of NPC by the European Commission. In July 2025, we filed an MAA. In July 2026, we announced that the CHMP of the EMA adopted a negative opinion regarding the MAA. We have requested a re-examination of the CHMP opinion.

Previous wording

As of March 31, 2026, there were a totalof 170 enrollments to receive MIPLYFFA. For MIPLYFFA, an enrollment is a prescription submitted to our specialty pharmacy, initiating the benefits investigation process to determine reimbursement and can lead to a 30-day paid dispense of MIPLYFFA. Our commercial plans focus on raising awareness among people who are living with NPC that are diagnosed and untreated, or undiagnosed.

Current wording

As of June 30, 2026, there were a totalof 184 enrollments to receive MIPLYFFA. For MIPLYFFA, an enrollment is a prescription submitted to our specialty pharmacy, initiating the benefits investigation process to determine reimbursement and can lead to a 30-day paid dispense of MIPLYFFA. Our commercial plans focus on raising awareness among people who are living with NPC that are diagnosed and untreated, or undiagnosed.

Previous wording

UCDs are a group of rare genetic disorders that can cause harmful ammonia to build up in the blood, potentially resulting in brain damage and neurocognitive impairments, if ammonia levels are not controlled. Any increase in ammonia over time is serious. Therefore, it is important to adhere to any dietary protein restrictions and have alternative medication options to help control ammonia levels. Approximately 1 in 100,000 people have UCD, and there are an estimated 800 patients who are actively treated with nitrogen scavenging therapy in the United States. While there are therapies currently approved for the treatment of UCDs, there remain unmet needs for this community of patients. We believe that OLPRUVA offers benefits over other UCD treatments by eliminating issues with palatability, offering improved portability with its single-dose envelopes, and being provided in a dosage personalized to the patient based on weight.

Current wording

UCDs are a group of rare genetic disorders that can cause harmful ammonia to build up in the blood, potentially resulting in brain damage and neurocognitive impairments, if ammonia levels are not controlled. Any increase in ammonia over time is serious. Therefore, it is important to adhere to any dietary protein restrictions and have alternative medication options to help control ammonia levels. Approximately 1 in 100,000 people have UCD, and there are an estimated 800 patients who are actively treated with nitrogen scavenging therapy in the United States. While there are therapies currently approved for the treatment of UCDs, we believe that OLPRUVA offers benefits over other UCD treatments by eliminating issues with palatability, offering improved portability with its single-dose envelopes, and being provided in a dosage personalized to the patient based on weight.

Previous wording

In the fourth quarter of 2023, we began generating revenue from the sale of OLPRUVA in the United States. Zevra has a partnership with Relief Therapeutics SA(“Relief”), which has rights to commercialize OLPRUVA in various European countries, if approved. For the three months ended March 31, 2026, we had $0.3 million in revenue from sales of OLPRUVA. We have made the decision to scale back our sales and marketing efforts for OLPRUVA as we evaluate the path forward and weigh strategic alternatives.

Current wording

In the fourth quarter of 2023, we began generating revenue from the sale of OLPRUVA in the United States. Zevra has a partnership with Relief Therapeutics SA(“Relief”), which has rights to commercialize OLPRUVA in various European countries, if approved. For the three and six months ended June 30, 2026, we had $0.2 million and $0.5 million in revenue, respectively, from sales of OLPRUVA. We have made the decision to scale back our sales and marketing efforts for OLPRUVA as we evaluate the path forward and weigh strategic alternatives.

Previous wording

In July 2022, Acer initiated enrollment in a Phase 3 long-term event-driven clinical trial designed based on the discussions from the May 2021 Type B meeting with the FDA, also known as the DiSCOVER trial. The DiSCOVER trial intends to enroll 150 VEDS patients, with 100 patients receiving celiprolol and 50 patients receiving placebo. Recruitment in the Phase-3 trial was restarted mid-2024, and the trial has 62 enrolled participants as of March 31, 2026. We believe that celiprolol could address significant unmet needs, as there are currently no approved treatments for VEDS in the U.S. We have implemented a broad recruitment drive focusing on collaborating with medical clinics where most patients are being managed. In parallel, we actively engaged the FDA in Type C meeting in the first quarter of 2026 to discuss regulatory options to accelerate the development program and plan to continue the dialogue with regulators going forward.

Current wording

In July 2022, Acer initiated enrollment in a Phase 3 long-term event-driven clinical trial designed based on the discussions from the May 2021 Type B meeting with the FDA, also known as the DiSCOVER trial. The DiSCOVER trial intends to enroll 150 VEDS patients, with 100 patients receiving celiprolol and 50 patients receiving placebo. Recruitment in the Phase-3 trial was restarted mid-2024, and the trial has 66 enrolled participants as of June 30, 2026. We believe that celiprolol could address significant unmet needs, as there are currently no approved treatments for VEDS in the U.S. We have implemented a broad recruitment drive focusing on collaborating with medical clinics where most patients are being managed. In parallel, we actively engaged the FDA in a Type C meeting in the first quarter of 2026 to discuss regulatory options to potentially accelerate the development program and plan to continue the dialogue with regulators going forward with a follow-up meeting in the second half of 2026.

Previous wording

In May 2022, we purchased all the assets and operations of Orphazyme A/S (“Orphazyme”) related to arimoclomol. Prior to this acquisition, Orphazyme had entered into an asset purchase agreement with LadRx Corporation, which was assigned to XOMA (US) LLC, a wholly-owned subsidiary of XOMA Corporation (“XOMA”), in June 2023 (“XOMA License Agreement”). Under the XOMA License Agreement, XOMA is entitled to a mid-single digit percentage royalty with respect to net sales of MIPLYFFA as well as milestone payments based on future potential sales and regulatory milestones, including a $4.0 million regulatory milestone payment upon approval in the E.U.

Current wording

In May 2022, we purchased all the assets and operations of Orphazyme A/S (“Orphazyme”) related to arimoclomol. Prior to this acquisition, Orphazyme had entered into an asset purchase agreement with LadRx Corporation, which was assigned to XOMA (US) LLC, a wholly-owned subsidiary of XOMA Corporation (“XOMA”), in June 2023 (“XOMA License Agreement”). Under the XOMA License Agreement, XOMA is entitled to a mid-single digit percentage royalty with respect to net sales of MIPLYFFA as well as milestone payments based on future potential sales and regulatory milestones, including a $4.0 million regulatory milestone payment upon approval in the E.U. and a $5.0 million sales milestone payment upon annual sales of $100.0 million. XOMA was acquired by Ligand Pharmaceuticals in July 2026.

Previous wording

In connection with our acquisition of Acer, Acer and Relief entered into an exclusive license agreement on August 30, 2023 (the “Relief License Agreement”), which was assumed by Zevra. Pursuant to the Relief License Agreement, Zevra is obligated to pay royalties of 10% of U.S. net sales of OLPRUVA up to a maximum of $45.0 million, plus specified regulatory milestones, for total payments to Relief of up to $56.5 million. On April 10, 2025, Relief sold the rights to this royalty to Soleus Capital Management L.P.

Current wording

In connection with our acquisition of Acer, Acer and Relief entered into a termination agreement on August 28, 2023 (the “Relief Termination Agreement”). Pursuant to the Relief Termination Agreement, we are obligated to pay royalties of 10% of U.S. net sales of OLPRUVA up to a maximum of $45.0 million, plus specified regulatory milestones, for total payments to Relief of up to $56.5 million. On April 10, 2025, Relief sold the rights to this royalty to Soleus Capital Management L.P., and in December 2025, Relief merged with NeuroX Group SA, and the combined company now operates as MindMaze Therapeutics Holding SA.

Previous wording

Pursuant to the Relief License Agreement, Relief holds exclusive development and commercialization rights for OLPRUVA in the EU, Liechtenstein, San Marino, Vatican City, Norway, Iceland, Principality of Monaco, Andorra, Gibraltar, Switzerland, United Kingdom, Albania, Bosnia, Kosovo, Montenegro, Serbia and North Macedonia (“Geographical Europe”). We have the right to receive a royalty of up to 10% of the net sales of OLPRUVA in Geographical Europe.

Current wording

Acer and Relief also entered into an exclusive license agreement on August 28, 2023 (the “Relief License Agreement”). Pursuant to the Relief License Agreement, Relief holds exclusive development and commercialization rights for OLPRUVA in the EU, Liechtenstein, San Marino, Vatican City, Norway, Iceland, Principality of Monaco, Andorra, Gibraltar, Switzerland, United Kingdom, Albania, Bosnia, Kosovo, Montenegro, Serbia and North Macedonia (“Geographical Europe”). We have the right to receive a royalty of up to 10% of the net sales of OLPRUVA in Geographical Europe.

Previous wording

Revenue for the three months ended March 31, 2026, was $36.2 million, compared to revenue of $20.4 million for the three months ended March 31, 2025, an increase of $15.8 million. The increase was primarily attributable to an increase in revenues under the global EAP of $7.9 million and an increase in product sales of MIPLYFFA of $7.5 million.

Current wording

Revenue for the three months ended June 30, 2026, was $39.7 million, compared to revenue of $25.9 million for the three months ended June 30, 2025, an increase of $13.8 million. The increase was primarily attributable to an increase in revenues under the global EAP of $6.4 million and an increase in product sales of MIPLYFFA of $8.7 million.

Previous wording

Cost of product revenue for the three months ended March 31, 2026, was $1.9 million, an increase of $0.6 million compared to cost of product revenue of $1.3 million for the three months ended March 31, 2025. The increase was primarily due to royalty costs related to increased product sales of MIPLYFFA and the write-down of unsaleable inventory.

Current wording

Cost of product revenue for the three months ended June 30, 2026, was $1.5 million, a decrease of $10.9 million compared to cost of product revenue of $12.4 million for the three months ended June 30, 2025. The decrease was primarily due to a decrease in the write-down of unsaleable inventory of $11.7 million, partially offset by an increase in royalty costs related to increased product sales of MIPLYFFA.

Previous wording

Selling, general and administrative expenses increased by approximately $1.2 million, from $19.5 million for the three months ended March 31, 2025, to $20.8 million for the three months ended March 31, 2026. This increase was primarily related to an increase in professional fees, partially offset by a decrease in third party spending.

Current wording

Selling, general and administrative expenses decreased by approximately $4.2 million, from $20.8 million for the three months ended June 30, 2025, to $16.6 million for the three months ended June 30, 2026. This decrease was primarily related to decreases in professional fees and third party spending, partially offset by an increase in personnel related costs.

Previous wording

Other (expense) income decreased from $3.4 million of income for the three months ended March 31, 2025, to $7.3 million of expense for the three months ended March 31, 2026. The decrease in income was primarily attributable to a loss on derivative liability of $7.2 million, a loss on extinguishment of debt of $2.8 million, and a decrease in fair value adjustment related to warrant and CVR liability of $3.9 million, partially offset by an increase in interest and other income, net, of $3.0 million.

Current wording

Other (expense) income decreased from $147.9 million of income for the three months ended June 30, 2025, to $4.0 million of expense for the three months ended June 30, 2026. The decrease in income was primarily attributable to a gain on sale of PRV of $148.3 million in the second quarter of 2025, and a decrease in fair value adjustment related to warrant and CVR liability of $5.7 million, partially offset by a decrease in interest expense of $2.0 million and an increase in interest and other income, net, of $0.2 million,

Previous wording

Income tax expense increased by $5.7 million, from $1.2 million for the three months ended March 31, 2025, to $6.9 million for the three months ended March 31, 2026, due to the tax provision associated with increased income in the current quarter.

Current wording

Income tax expense increased by $1.8 million, from $2.2 million for the three months ended June 30, 2025, to $4.0 million for the three months ended June 30, 2026, due to the tax provision associated with increased income in the current quarter, excluding the gain on sale of PRV for which income tax expense was recognized in the fourth quarter of 2024.

Previous wording

Net income for the three months ended March 31, 2026, was $37.9 million, compared to a net loss of $3.1 million for the three months ended March 31, 2025, an increase to net income of $41.0 million. The increase was primarily attributable to a gain on sale of future royalties, intellectual property, and other assets, net, of $43.3 million under the Commave Settlement Agreement, an increase of $15.8 million in revenue, and an increase in interest and other income, net, of $3.0 million, partially offset by a loss on derivative liability of $7.2 million, loss on extinguishment of debt of $2.8 million, an increase in tax expense of $5.7 million, and a decrease in fair value adjustment related to warrant and CVR liability of $3.9 million.

Current wording

Net income for the six months ended June 30, 2026, was $46.6 million, compared to a net income of $71.6 million for the six months ended June 30, 2025, a decrease to net income of $25.0 million. The decrease was primarily attributable to a gain on sale of PRV of $148.3 million in the second quarter of 2025, loss on derivative liability of $7.2 million, loss on extinguishment of debt of $2.8 million, an increase in tax expense of $7.6 million, and a decrease in fair value adjustment related to warrant and CVR liability of $9.6 million, partially offset by a gain on sale of future royalties, intellectual property, and other assets, net, of $43.3 million under the Commave Settlement Agreement in the first quarter of 2026, a decrease in impairment of intangible assets of $58.7 million, an increase of $29.6 million in revenue, a decrease in inventory obsolescence charges of $11.2 million, a decrease in intangible asset amortization of $2.6 million and an increase in interest and other income, net, of $3.2 million,

Previous wording

Research and development expenses increased by $1.1 million, from $3.3 million for the three months ended March 31, 2025, to $4.4 million for the three months ended March 31, 2026. This increase was primarily driven by an increase in spending for ongoing arimoclomol efforts and the celiprolol Phase 3 study.

Current wording

Research and development expenses increased by $2.2 million, from $6.7 million for the six months ended June 30, 2025, to $8.9 million for the six months ended June 30, 2026. This increase was primarily driven by an increase in spending for ongoing arimoclomol efforts and the celiprolol Phase 3 study.

Previous wording

Through March 31, 2026, we have funded our research and development and operating activities primarily through the issuance of debt and equity and from product sales of MIPLYFFA and OLPRUVA, reimbursements received under the global EAP, royalties or net sales milestone payments generated under the AZSTARYS License Agreement and subsequent Commave Settlement Agreement, our PRV sale consummated on April 1, 2025, and consulting agreements. As of March 31, 2026, we had cash, cash equivalents and investments of $236.8 million.

Current wording

Through June 30, 2026, we have funded our research and development and operating activities primarily through the issuance of debt and equity and from product sales of MIPLYFFA and OLPRUVA, reimbursements received under the global EAP, royalties or net sales milestone payments generated under the AZSTARYS License Agreement and subsequent Commave Settlement Agreement, our PRV sale consummated on April 1, 2025, and consulting agreements. As of June 30, 2026, we had cash, cash equivalents and investments of $260.2 million.

Previous wording

We have had recurring negative net operating cash flows throughout our operating history, and we cannot guarantee or predict when we may begin to consistently generate positive net cash flows from operations, or if at all. We expect that our sources of revenue will be from product sales of approved products, product reimbursements received under the global EAP, and any other future arrangements related to one or more of our products or product candidates.

Current wording

While we have recently had several quarters of positive net cash flows from operations, we previously had recurring negative net operating cash flows, and we cannot guarantee that we can continue to generate positive net cash flows from operations. We expect that our sources of revenue will be from product sales of approved products, product reimbursements received under the global EAP, and any other future arrangements related to one or more of our products or product candidates.

Previous wording

On March 12, 2026, we repaid in full all outstanding obligations under the Credit Agreement. As of the date of repayment, the aggregate principal amount outstanding under the Credit Agreement was approximately $63.1 million (which includes accrued paid-in-kind interest of approximately $3.1 million), plus accrued and unpaid cash interest of $1.4 million. Under the terms of the Credit Agreement, we were also required to pay a final payment premium of $1.8 million, a make-whole amount and prepayment premium of $7.2 million and legal fees of approximately $0.1 million. The Term Loans were secured by a first priority perfected lien on, and security interest in, substantially all current and future assets of ours and certain subsidiaries that were guarantors thereunder. Upon repayment, the Credit Agreement and all related loan documents were terminated, and all liens and security interests granted thereunder were released. We recognized a loss on extinguishment of debt of $2.8 million in the unaudited condensed consolidated statements of operations during the three months ended March 31, 2026.

Current wording

On March 12, 2026, we repaid in full all outstanding obligations under the Credit Agreement. As of the date of repayment, the aggregate principal amount outstanding under the Credit Agreement was approximately $63.1 million (which includes accrued paid-in-kind interest of approximately $3.1 million), plus accrued and unpaid cash interest of $1.4 million. Under the terms of the Credit Agreement, we were also required to pay a final payment premium of $1.8 million, a make-whole amount and prepayment premium of $7.2 million and legal fees of approximately $0.1 million. The Term Loans were secured by a first priority perfected lien on, and security interest in, substantially all current and future assets of ours and certain subsidiaries that were guarantors thereunder. Upon repayment, the Credit Agreement and all related loan documents were terminated, and all liens and security interests granted thereunder were released. We recognized a loss on extinguishment of debt of $2.8 million in the unaudited condensed consolidated statements of operations during the six months ended June 30, 2026.

Previous wording

The following table summarizes our cash flows for thethree months ended March 31, 2026, and 2025 (in thousands):

Current wording

The following table summarizes our cash flows for thesix months ended June 30, 2026, and 2025 (in thousands):

Previous wording

For the three months ended March 31, 2026, net cash provided by operating activities of $6.1 million consisted of net income of $37.9 million, in addition to $41.3 million in adjustments for non-cash items and changes in working capital of $9.6 million. Net income was primarily attributable to income generated under the Commave Settlement Agreement, as well as revenue received from product sales of MIPLYFFA and OLPRUVA, and reimbursements received under the global EAP. This income was partially offset by ongoing operating expenses to support our commercial organization and the resulting gain from the Commave Settlement Agreement, as well as the debt payoff. In addition to the aforementioned transactions, the adjustments for non-cash items primarily consisted of income tax expense of $6.9 million, stock-based compensation expense of $3.1 million, and a gain on foreign currency exchange rates of $1.4 million.

Current wording

For the six months ended June 30, 2026, net cash provided by operating activities of $23.2 million consisted of net income of $46.6 million, in addition to changes in working capital of $8.7 million, partially offset by $32.1 million in adjustments for non-cash items. Net income was primarily attributable to income generated under the Commave Settlement Agreement, as well as revenue received from approved product sales, and reimbursements received under the global EAP. This income was partially offset by ongoing operating expenses to support our commercial organization and the resulting gain from the Commave Settlement Agreement, as well as the debt payoff. In addition to the aforementioned transactions, the adjustments for non-cash items primarily consisted of income tax expense of $7.6 million, stock-based compensation expense of $6.1 million, fair value adjustment related to warrant and CVR liability of $5.4 million and a gain on foreign currency exchange rates of $1.8 million.

Previous wording

For the three months ended March 31, 2025, net cash used in operating activities of $8.2 million consisted of a net loss of $3.1 million and changes in working capital of $6.8 million, partially offset by $1.7 million in adjustments for non-cash items. Net loss was primarily attributable to our spending on research and development programs and operating costs; partially offset by revenue received from product sales of MIPLYFFA and OLPRUVA, royalties generated under the AZSTARYS License Agreement, and reimbursements received under the EAP in France. The changes in working capital consisted of $7.3 million related to a change in accounts payable and accrued expenses, a $0.2 million change in inventories, $0.2 million related to a change in operating lease liabilities, $2.1 million related to a change in accounts and other receivables, partially offset by an increase of $0.6 million related to a change in other liabilities, $0.3 million in prepaids and other assets, $0.2 million related to a change in operating lease right-of-use assets, and $1.9 million related to a change in discount and rebate liabilities. The adjustments for non-cash items primarily consisted of stock-based compensation expense of $3.1 million, interest expense of $0.7 million, and $2.8 million related to depreciation, amortization and other items, partially offset by a change in the fair value of warrant and CVR liability of $4.9 million.

Current wording

For the six months ended June 30, 2025, net cash used in operating activities of $11.8 million consisted of net income of $71.6 million, offset by $69.3 million in adjustments for non-cash items and changes in working capital of $14.1 million. Net income was primarily attributable to the sale of the PRV, as well as revenue received from approved product sales, royalties generated under the AZSTARYS License Agreement, and reimbursements received under the global EAP, partially offset by impairment and obsolescence charges and spend on R&D programs and operating costs. The adjustments for non-cash items primarily consisted of the gain on sale of PRV of $148.3 million, and a change in the fair value of warrant and CVR liability of $4.1 million, partially offset by impairment of intangible assets of $58.7 million, inventory obsolescence of $11.7 million, stock-based compensation expense of $5.6 million, $3.3 million of depreciation and amortization expense, and income tax expense of $3.4 million.

Previous wording

For the three months ended March 31, 2026, net cash provided by investing activities was $79.6 million, which was primarily attributable to sales and maturities of investments of $94.0 million and sale of future royalties, intellectual property, and other assets, net, of $43.9 million, partially offset by $58.3 million in investment purchases.

Current wording

For the six months ended June 30, 2026, net cash provided by investing activities was $111.1 million, which was primarily attributable to sales and maturities of investments of $143.2 million and sale of future royalties, intellectual property, and other assets, net, of $48.9 million, partially offset by $80.8 million in investment purchases.

Previous wording

For the three months ended March 31, 2025, net cash provided by investing activities was $10.5 million, which was primarily attributable to maturities of investments of $18.0 million, partially offset by $7.4 million in purchases of investments.

Current wording

For the six months ended June 30, 2025, net cash provided by investing activities was $22.5 million, which was primarily attributable to proceeds from the sale of the PRV of $150.0 million and maturities of investments of $30.5 million, partially offset by $157.7 million in purchases of investments.

Previous wording

For the three months ended March 31, 2026, net cash used in financing activities was $52.8 million, which was primarily attributable to the repayment of debt of $60.1 million and the payments for employee taxes related to stock awards of $2.6 million, partially offset by proceeds from the issuance of stock for warrants exercised of $9.2 million.

Current wording

For the six months ended June 30, 2026, net cash used in financing activities was $51.2 million, which was primarily attributable to the repayment of debt of $60.1 million and the payments for employee taxes related to stock awards of $1.5 million, partially offset by proceeds from the issuance of stock for warrants exercised of $9.2 million.

Previous wording

For the three months ended March 31, 2025, net cash provided by financing activities was $1.6 million, which was primarily attributable to proceeds from the issuance of stock of $2.0 million partially offset by payments of principal on insurance financing arrangements of $0.4 million.

Current wording

For the six months ended June 30, 2025, net cash provided by financing activities was $3.0 million, which was primarily attributable to proceeds from the issuance of stock of $2.9 million.

Previous wording

•any sales of our approved products;

Current wording

•sales of our approved products;

Previous wording

•any reimbursements received for arimoclomol under the global EAP; and

Current wording

•reimbursements received for arimoclomol under the global EAP; and

Previous wording

•sales of common stock by us under the 2024 ATM Agreement or any other offering of securities made pursuant to the June 2024 Registration Statement.

Current wording

•potential sales of common stock by us under the 2024 ATM Agreement or any other offering of securities made pursuant to the June 2024 Registration Statement.

Previous wording

We have based our estimates of our cash needs and cash runway on assumptions that may prove to be wrong, and we may use our available capital resources sooner than we currently expect. In addition, we cannot guarantee that we will be able to generate sufficient proceeds from sales of approved products, reimbursements received under the global EAP, or other funding transactions to fund our operating expenses. To meet any additional cash requirements, we may seek to sell additional equity or convertible securities that may result in dilution to our stockholders, issue additional debt or seek other third-party funding, including potential strategic transactions, such as licensing or collaboration arrangements. Because of the numerous risks and uncertainties associated with the development and commercialization of product candidates and products, we are unable to estimate the amounts of increased capital outlays and operating expenditures necessary to complete the commercialization and development of our partnered product or product candidates, should they obtain regulatory approval.

Current wording

We have based our estimates of our cash needs and cash runway on assumptions that may prove to be wrong, and we may use our available capital resources sooner than we currently expect. In addition, we cannot guarantee that we will be able to generate sufficient proceeds from sales of approved products, reimbursements received under the global EAP, or other funding transactions to fund our operating expenses. To meet any additional cash requirements, we may seek to sell additional equity or convertible securities that may result in dilution to our stockholders, issue additional debt or seek other third-party funding, including potential strategic transactions, such as licensing or collaboration arrangements. Because of the numerous risks and uncertainties associated with the development and commercialization of product candidates and products, we are unable to estimate the amounts of increased capital outlays and operating expenditures that may be necessary.