ZEVRA THERAPEUTICS, INC.
For the period ended Sep 30, 2026. Filed Nov 3, 2026.
View on SEC.govAbout this filing
A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Sep 30, 2026 · Filed Nov 3, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2026-06-30; filed 2026-08-05.
0001434647-26-000068
ITEM 2. MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONSCurrent report: 10-Q
Period ended 2026-09-30; filed 2026-11-03.
0001434647-26-000087
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 141 previous paragraphs and 144 current paragraphs.
100 unchanged; 33 reworded; 10 added; 6 removed; 2 previous and 1 current paragraphs rearranged.
Tables are not compared: 4 previous and 4 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 (10)
Current report
Net Income (Loss)
Current report
Net income for the three months ended September 30, 2026, was $19.2 million, compared to a net loss of $0.5 million for the three months ended September 30, 2025, an increase to net income of $19.8 million. The increase was primarily attributable to an increase in revenue of $11.7 million, an increase in fair value adjustment related to warrant and CVR liability of $10.6 million, an increase in interest and other income, net, of $0.7 million, and a decrease in interest expense of $2.1 million, partially offset by a decrease in tax benefit of $0.9 million.
Current report
Intangible asset amortization for the three months ended September 30, 2026, of $0.3 million was consistent when compared to intangible asset amortization of $0.3 million for the three months ended September 30, 2025.
Current report
Selling, general and administrative expenses increased by approximately $2.2 million, from $16.9 million for the three months ended September 30, 2025, to $19.1 million for the three months ended September 30, 2026. This increase was primarily due to commercial related costs.
Current report
Other income (expense)
Current report
Income tax (expense) benefit
Current report
Comparison of the nine months ended September 30, 2026 and 2025 (in thousands):
Current report
Net income for the nine months ended September 30, 2025 was $71.1 million, and was comprised of revenue of $72.3 million, a gain on sale of PRV of $148.3 million, interest and other income, net of $5.2 million, partially offset by cost of product revenue of $15.0 million (including inventory obsolescence charges of $11.7 million), intangible asset amortization of $3.5 million, impairment of intangible assets of $58.7 million, research and development of $10.1 million, selling, general and administrative costs of $57.3 million, interest expense of $6.0 million, fair value adjustments of $1.3 million and income tax expense of $2.9 million.
Current report
Other expense for the nine months ended September 30, 2026 was $3.2 million and consisted of a loss on extinguishment of debt of $2.8 million, loss on derivative liability of $7.2 million, interest expense of $1.7 million, and fair value adjustments of $0.7 million, partially offset by interest and other income, net of $9.2 million.
Current report
Other income for the nine months ended September 30, 2025 was $146.3 million and was attributable to a gain on sale of PRV of $148.3 million, interest and other income, net of $5.2 million, partially offset by interest expense of $6.0 million, and fair value adjustments of $1.3 million.
Removed paragraphs (6)
Previous report
Net income for the three months ended June 30, 2026, was $8.8 million, compared to a net income of $74.7 million for the three months ended June 30, 2025, a decrease to net income of $66.0 million. The decrease was primarily attributable to a gain on sale of PRV of $148.3 million in the second quarter of 2025, an increase in interest and other income, net, of $0.2 million, a decrease in fair value adjustment related to warrant and CVR liability of $5.7 million, a decrease in impairment of intangible assets of $58.7 million, and a decrease in the write-down of unsaleable inventory of $11.7 million, partially offset by an increase of $13.8 million in revenue, and a decrease in tax benefit of $1.8 million.
Previous report
Comparison of the six months ended June 30, 2026 and 2025 (in thousands):
Previous report
Net income
Previous report
Other (expense) income
Previous report
Other (expense) income decreased from $151.4 million of income for the six months ended June 30, 2025, to $11.3 million of expense for the six months ended June 30, 2026. The decrease in income was primarily attributable to the gain on sale of PRV of $148.3 million in the second quarter of 2025, 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 $9.6 million, and other assets, partially offset by an increase in interest and other income, net, of $3.2 million and a decrease in interest expense of $2.2 million.
Previous report
Income tax expense
Reworded paragraphs (33)
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 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.
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, MIPLYFFA (arimoclomol), 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
Our strategic plan is focused on transforming Zevra into a leading rare-disease company. We are prioritizing the commercialization and global expansion of our lead product, MIPLYFFA (arimoclomol), while OLPRUVA remains commercially available. We are also advancing the development of our clinical stage asset, celiprolol, and plan to further expand our pipeline through inorganic growth. We intend to become the preferred partner for assets that we believe will allow us to leverage the expertise and infrastructure that we have built to help mitigate risk and enhance our probability of success.
Current wording
Our strategic plan is focused on transforming Zevra into a leading rare-disease company. We are prioritizing the commercialization and global expansion of MIPLYFFA, while OLPRUVA remains commercially available. We are also advancing the development of our clinical stage asset, celiprolol, and plan to further expand our pipeline through inorganic growth. We intend to become the preferred partner for assets that we believe will allow us to leverage the expertise and infrastructure that we have built to help mitigate risk and enhance our probability of success.
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. 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.
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 two 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 EMA adopted a negative opinion regarding the MAA. Under European regulatory procedures, we have requested a re-examination of the CHMP opinion.
Previous 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:
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 nine months ended September 30, 2026, and 2025, was$33.2 million and $(7.1)
Previous wording
NPC is an ultra-rare and progressive neurodegenerative disease characterized by an inability of the body to transport cholesterol and lipids inside of cells. Symptoms of NPC include a progressive impairment of mobility, cognition, speech, and swallowing, often culminating in premature death. The incidence of NPC is estimated to be one in 100,000 to 130,000 live births. We estimate that there are approximately 2,000 individuals with NPC in the United States and Europe combined, of which approximately 900 are in the United States and 1,100 are in Europe, where there is a mature market with an approved treatment available for NPC. Of this estimated population, approximately 300 to 350 people have been diagnosed in the United States. NPC is clinically heterogenous, with significant variability in symptom presentation and rate of progression. Although it has traditionally been considered a pediatric disease due to its genetic origins, nearly half of the people treated with MIPLYFFA are adults. Low diagnostic rates may affect the number of potential patients, and we believe that the availability of treatment options in the United States could increase awareness of the disease and assist in more accurately identifying patients.
Current wording
NPC is an ultra-rare and progressive neurodegenerative disease characterized by an inability of the body to transport cholesterol and lipids inside of cells. Symptoms of NPC include a progressive impairment of mobility, cognition, speech, and swallowing, often culminating in premature death. The incidence of NPC is estimated to be one in 100,000 to 130,000 live births. We estimate that there are approximately 2,000 individuals with NPC in the United States and Europe combined, of which approximately 900 are in the United States and 1,100 are in Europe, where there is a mature market with an approved treatment available for NPC. Of this estimated population, approximately 300 to 350 people have been diagnosed in the United States. NPC is clinically heterogeneous, with significant variability in symptom presentation and rate of progression. Although it has traditionally been considered a pediatric disease due to its genetic origins, nearly half of the people treated with MIPLYFFA are adults. Low diagnostic rates may affect the number of potential patients, and we believe that the availability of treatment options in the United States could increase awareness of the disease and assist in more accurately identifying patients.
Previous wording
In June 2026, Zevra published positive results of an open-label pediatric substudy in the peer reviewed journal Molecular Genetics and Metabolism Reports. This multicenter substudy evaluated arimoclomol, in addition to concomitant miglustat, in five children with NPC aged 12 to <24 months at enrollment over up to 36 months of treatment. Arimoclomol was generally well tolerated, with no new safety signals, and pharmacokinetic results were consistent with older pediatric populations. Developmental assessments showed variable delays, reflecting underlying phenotypic heterogeneity. Although limited by small sample size, the study provides preliminary evidence supporting initiation of MIPLYFFA in this age group.
Current wording
In June 2026, Zevra published positive results of an open-label pediatric substudy in the peer reviewed journal Molecular Genetics and Metabolism Reports. This multicenter substudy evaluated arimoclomol, in addition to concomitant miglustat, in patients with a confirmed diagnosis of NPC1 or NPC2 aged 6 to <24 months at study enrollment over up to 36 months of treatment. In the five patients enrolled, arimoclomol was generally well tolerated, with no new safety signals, and pharmacokinetic results were consistent with older pediatric populations. Developmental assessments showed variable delays, reflecting underlying phenotypic heterogeneity. Although limited by small sample size, the study provides preliminary evidence supporting initiation of MIPLYFFA in this age group.
Previous 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.
Current wording
As of September 30, 2026, there were a totalof 197 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
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.
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 nine months ended September 30, 2026, we had $0.2 million and $0.7 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 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.
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 72 enrolled participants as of September 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 fourth quarter of 2026.
Previous wording
•Evidence of efficacy in Europe and extensive clinical experience from multiple trials. Celiprolol has become the primary treatment for VEDS patients in several European countries. BBEST Clinical Trial data showed 76% reduction in risk of arterial events observed in COLA3A1+ subpopulation, with additional data from a long-term observational study in France.
Current wording
•Evidence of efficacy in Europe and extensive clinical experience from multiple trials. Celiprolol has become the primary treatment for VEDS patients in several European countries. BBEST Clinical Trial data showed 76% reduction in risk of arterial events observed in COL3A1+ subpopulation, with additional data from a long-term observational study in France.
Previous 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.
Current wording
Revenue for the three months ended September 30, 2026, was $37.8 million, compared to revenue of $26.1 million for the three months ended September 30, 2025, an increase of $11.7 million. The increase was primarily attributable to an increase in product sales of MIPLYFFA of $11.6 million.
Previous 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.
Current wording
Cost of product revenue for the three months ended September 30, 2026, was $1.8 million, an increase of $0.6 million compared to cost of product revenue of $1.2 million for the three months ended September 30, 2025. The increase was primarily due to an increase in royalty costs related to increased product sales of MIPLYFFA.
Previous wording
Research and development expenses increased by $1.1 million, from $3.4 million for the three months ended June 30, 2025, to $4.5 million for the three months ended June 30, 2026. This increase was primarily due to ongoing arimoclomol efforts.
Current wording
Research and development expenses increased by $1.5 million, from $3.4 million for the three months ended September 30, 2025, to $4.9 million for the three months ended September 30, 2026. This increase was due primarily to our ongoing arimoclomol efforts and our continued investment in the celiprolol development program.
Previous 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,
Current wording
Other income (expense) increased from $5.1 million of expense for the three months ended September 30, 2025, to $8.1 million of income for the three months ended September 30, 2026. The increase in income was primarily attributable to an increase in fair value adjustment related to warrant and CVR liability of $10.6 million, a decrease in interest expense of $2.1 million, and an increase in interest and other income, net, of $0.7 million,
Previous 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.
Current wording
Income tax (expense) benefit increased by $0.9 million, from $0.4 million benefit for the three months ended September 30, 2025, to $0.5 million expense for the three months ended September 30, 2026, due to the tax provision associated with increased income in the current quarter.
Previous 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,
Current wording
Net income for the nine months ended September 30, 2026, was $65.9 million, and was comprised of revenue of $113.6 million, a gain on sale of future royalties, intellectual property, and other assets, net, of $43.3 million under the Commave Settlement Agreement, interest and other income, net of $9.2 million, partially offset by cost of product revenue of $5.2 million, intangible asset amortization of $0.9 million, research and development expense of $13.8 million, selling, general and administrative costs of $56.5 million, loss on derivative liability of $7.2 million, loss on extinguishment of debt of $2.8 million, interest expense of $1.7 million, fair value adjustments of $0.7 million and income tax expense of $11.5 million.
Previous wording
Revenue for the six months ended June 30, 2026, was $75.9 million, compared to revenue of $46.3 million for the six months ended June 30, 2025, an increase of $29.6 million. The increase was primarily attributable to an increase in revenues under the global EAP of $14.1 million and an increase in product sales of MIPLYFFA of $16.3 million, partially offset by a decrease in royalty revenue of $0.7 million.
Current wording
Revenue for the nine months ended September 30, 2026, was $113.6 million, compared to revenue of $72.3 million for the nine months ended September 30, 2025, an increase of $41.3 million. The increase was primarily attributable to an increase in revenues under the global EAP of $15.4 million and an increase in product sales of MIPLYFFA of $27.9 million, partially offset by a decrease in royalty revenue of $1.9 million.
Previous wording
Cost of product revenue for the six months ended June 30, 2026, was $3.4 million, a decrease of $10.3 million compared to cost of product revenue of $13.7 million for the six months ended June 30, 2025. The decrease was primarily due to a decrease in the write-down of unsaleable inventory, partially offset by an increase in royalty costs related to increased product sales of MIPLYFFA.
Current wording
Cost of product revenue for the nine months ended September 30, 2026, was $5.2 million, a decrease of $9.7 million compared to cost of product revenue of $15.0 million for the nine months ended September 30, 2025. The decrease was primarily due to a decrease in the write-down of unsaleable inventory, partially offset by an increase in royalty costs related to increased product sales of MIPLYFFA.
Previous wording
Intangible asset amortization for the six months ended June 30, 2026, was $0.6 million, a decrease of $2.6 million compared to intangible asset amortization of $3.2 million for the six months ended June 30, 2025. The decrease was a result of definite-lived intangible assets acquired in the acquisition of Acer no longer being amortized as a result of the impairment recorded as of and for the period ended June 30, 2025.
Current wording
Intangible asset amortization for thenine months ended September 30, 2026, was $0.9 million, a decrease of $2.6 million compared to intangible asset amortization of $3.5 million for the nine months ended September 30, 2025. The decrease was a result of definite-lived intangible assets acquired in the acquisition of Acer no longer being amortized as a result of the impairment recorded as of and for the period ended June 30, 2025.
Previous 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.
Current wording
Research and development expenses increased by $3.7 million, from $10.1 million for the nine months ended September 30, 2025, to $13.8 million for the nine months ended September 30, 2026. This increase was primarily driven by an increase in spending for ongoing arimoclomol research and development efforts and the celiprolol Phase 3 study.
Previous wording
Selling, general and administrative expenses decreased by approximately $3.0 million, from $40.3 million for the six months ended June 30, 2025, to $37.3 million for the six months ended June 30, 2026. This decrease was primarily related to a decrease in third party spending and professional fees, partially offset by an increase in personnel related costs.
Current wording
Selling, general and administrative expenses decreased by approximately $0.8 million, from $57.3 million for the nine months ended September 30, 2025, to $56.5 million for the nine months ended September 30, 2026. This decrease was primarily related to a decrease in third party spending and professional fees, partially offset by an increase in personnel related costs.
Previous wording
Income tax expense increased by $7.6 million, from $3.4 million for the six months ended June 30, 2025, to $11.0 million for the six months ended June 30, 2026, due to the tax provision associated with increased income in the current period excluding the gain on sale of PRV for which income tax expense was recognized in the fourth quarter of 2024.
Current wording
Income tax expense increased by $8.5 million, from $2.9 million for the nine months ended September 30, 2025, to $11.5 million for the nine months ended September 30, 2026, due to the tax provision associated with increased income in the current period excluding the gain on sale of PRV for which income tax expense was recognized in the fourth quarter of 2024.
Previous 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.
Current wording
Through September 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, payments received under the Commave Settlement Agreement, our PRV sale consummated on April 1, 2025, and consulting agreements. As of September 30, 2026, we had cash, cash equivalents and investments of $270.9 million.
Previous 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.
Current wording
While we have recently had several quarters of positive net cash flows from operations, 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 six months ended June 30, 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 nine months ended September 30, 2026.
Previous wording
The following table summarizes our cash flows for thesix months ended June 30, 2026, and 2025 (in thousands):
Current wording
The following table summarizes our cash flows for thenine months ended September 30, 2026, and 2025 (in thousands):
Previous 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.
Current wording
For the nine months ended September 30, 2026, net cash provided by operating activities of $33.2 million consisted of net income of $65.9 million, in addition to changes in working capital of $4.3 million, partially offset by $36.9 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 $5.6 million, stock-based compensation expense of $8.7 million, fair value adjustment related to warrant and CVR liability of $0.3 million and a gain on foreign currency exchange rates of $2.7 million.
Previous 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.
Current wording
For the nine months ended September 30, 2025, net cash used in operating activities of $7.1 million consisted of net income of $71.1 million, offset by $61.7 million in adjustments for non-cash items and changes in working capital of $16.5 million. Net income was primarily attributable to the sale of the PRV, as well as revenue received from product sales of MIPLYFFA and OLPRUVA, 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, partially offset by impairment of intangible assets of $58.7 million, inventory obsolescence of $11.7 million, stock-based compensation expense of $8.4 million, $3.7 million of depreciation and amortization expense, and income tax expense of $2.9 million.
Previous 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.
Current wording
For the nine months ended September 30, 2026, net cash provided by investing activities was $76.2 million, which was primarily attributable to sales and maturities of investments of $188.2 million and sale of future royalties, intellectual property, and other assets, net, of $48.9 million, partially offset by $160.4 million in investment purchases.
Previous 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.
Current wording
For the nine months ended September 30, 2025, net cash provided by investing activities was $17.9 million, which was primarily attributable to proceeds from the sale of the PRV of $150.0 million and maturities of investments of $91.0 million, partially offset by $222.8 million in purchases of investments.
Previous 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.
Current wording
For the nine months ended September 30, 2026, net cash used in financing activities was $49.7 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 and proceeds from issuance of common stock for options exercised of $2.3 million.
Previous 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.
Current wording
For the nine months ended September 30, 2025, net cash provided by financing activities was $9.5 million, which was primarily attributable to proceeds from the issuance of stock for warrants exercised of $6.0 million and options exercised or RSUs vested of $3.5 million.
Previous wording
We believe our available cash, cash equivalents and investments, together with our ability to generate operating cash flow and our access to short-term and long-term borrowings, are sufficient to fund our existing and planned capital requirements for at least the next twelve months and the foreseeable future.
Current wording
We believe our available cash, cash equivalents and investments, together with our ability to generate operating cash flow, are sufficient to fund our existing and planned capital requirements for at least the next twelve months and the foreseeable future.
Rearranged current paragraphs (1)
Current report — wording carried from elsewhere
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:
Rearranged previous paragraphs (2)
Previous report — wording carried elsewhere
Intangible asset amortization for the three months ended June 30, 2026, was $0.3 million, a decrease of $1.3 million compared to intangible asset amortization of $1.6 million for the three months ended June 30, 2025. The decrease was a result of definite-lived intangible assets acquired in the acquisition of Acer no longer being amortized as a result of the impairment recorded as of and for the period ended June 30, 2025.
Previous report — wording carried elsewhere
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.