BIOCRYST PHARMACEUTICALS INC

Previous wording

We are a global biotechnology company with a deep commitment to improving the lives of people living with hereditary angioedema (“HAE”) and other rare diseases. We leverage our expertise in structure-guided drug design with the goal of developing first-in-class or best-in-class oral small-molecule and injectable protein therapeutics to target difficult-to-treat rare diseases. In addition to our discovery and development efforts, our business strategy includes the successful commercialization of these drugs, as well as self-funding all of these efforts by achieving and increasing profitability. By focusing primarily on rare disease markets, we believe that we can more effectively control the costs of, and our strategic allocation of financial resources toward, post-approval commercialization.

Current wording

We are a global biotechnology company focused on developing and commercializing medicines for hereditary angioedema (“HAE”) and other rare diseases, driven by our deep commitment to improving the lives of people living with these conditions. We have built a robust commercial infrastructure to support the launch and continued success of ORLADEYO, an oral, once-daily therapy discovered and developed internally for the prevention of HAE attacks. Our business strategy includes leveraging this established commercial platform to successfully commercialize a pipeline of potential first-in-class or best-in-class oral small-molecule and injectable protein therapeutics targeting a range of rare diseases. These programs are being pursued through both internal discovery efforts and strategic business development. By utilizing our existing commercial capabilities and focusing on rare disease markets, we believe that we can more effectively optimize our costs and strategically allocate resources to support long-term, sustainable growth.

Previous wording

ORLADEYO is an oral capsule, once-daily therapy discovered and developed by us for the prevention of HAE attacks. ORLADEYO is approved in the United States and other global markets for the prevention of HAE attacks in adults and pediatric patients 12 years and older. In addition, the ongoing APeX-P clinical trial, which is complete through the primary endpoint, is continuing to assess an oral granule formulation of ORLADEYO in pediatric patients who are 2 to 11 years of age at enrollment.

Current wording

ORLADEYO is an oral capsule, once-daily therapy discovered and developed by us for the prevention of HAE attacks. ORLADEYO is approved in the United States and other global markets for the prevention of HAE attacks in adults and pediatric patients 12 years and older. In addition, the ongoing APeX-P clinical trial, which is complete through the primary endpoint, is continuing to assess an oral granule formulation of ORLADEYO in pediatric patients who are 2 to 11 years of age at enrollment. The Prescription Drug User Fee Act goal date for our new drug application for ORLADEYO granules in children with HAE aged 2 to 11 is December 12, 2025. ORLADEYO would be the first targeted oral prophylactic therapy for children with HAE.

Previous wording

We have built out our U.S. commercial infrastructure to support the launch and continued commercialization of ORLADEYO in the United States and are continuing to build our commercial infrastructure to support launches in other markets. Based on proprietary analyses of HAE prevalence and market research studies with HAE patients, physicians, and payors in the United States and Europe, and over four years of commercialization experience with ORLADEYO, we anticipate that the global commercial market for ORLADEYO has the potential to reach a global peak of $1 billion in annual net ORLADEYO revenues. We expect approximately 80 percent of our revenue at peak to come from the United States. These expectations are subject to numerous risks and uncertainties that may cause our actual results, performance, or achievements to be materially different. There can be no assurance that our commercialization methods and strategies will succeed, or that the market for ORLADEYO will develop in line with our current expectations. See “Risk Factors—Risks Relating to Our Business—Risks Relating to Drug Development and Commercialization—There can be no assurance that our or our partners’ commercialization efforts, methods, and strategies for our products or technologies will succeed, and our future revenue generation is uncertain” in Part II, Item 1A of this report for further discussion of these risks.

Current wording

Based on proprietary analyses of HAE prevalence and market research studies with HAE patients, physicians, and payors in the United States and Europe, and nearly five years of commercialization experience with ORLADEYO, we anticipate that the global commercial market for ORLADEYO has the potential to reach a global peak of $1 billion in annual net ORLADEYO revenues. These expectations are subject to numerous risks and uncertainties that may cause our actual results, performance, or achievements to be materially different. There can be no assurance that our commercialization methods and strategies will succeed, or that the market for ORLADEYO will develop in line with our current expectations. See “Risk Factors—Risks Relating to Our Business—Risks Relating to Drug Development and Commercialization—There can be no assurance that our or our partners’ commercialization efforts, methods, and strategies for our products or technologies will succeed, and our future revenue generation is uncertain” in Part II, Item 1A of this report for further discussion of these risks.

Previous wording

Revenue from sales of ORLADEYO for the three and six months ended June 30, 2025 is discussed under “Results of Operations” in this MD&A. Revenue from sales of ORLADEYO in future periods is subject to uncertainties and will depend on several factors, including our pending transaction with Neopharmed, as defined and discussed below, for the sale of our European ORLADEYO Business (as described further under “Recent Developments—Neopharmed Gentili S.p.A. Stock Purchase Agreement” below), the success of our and our partners’ commercialization efforts in the United States and elsewhere, the number of new patients switching to ORLADEYO, patient retention and demand, the number of physicians prescribing ORLADEYO, the rate of monthly prescriptions, reimbursement from third-party and government payors, the number of patients receiving free product, our pricing strategy, and market trends. We monitor and analyze this data on an ongoing basis as we continue to commercialize ORLADEYO and adjust our forecasts accordingly.

Current wording

Revenue from sales of ORLADEYO for the three and nine months ended September 30, 2025 is discussed under “Results of Operations” in this MD&A. Revenue from sales of ORLADEYO in future periods is subject to uncertainties and will depend on several factors, including, but not limited to, the success of our and our partners’ commercialization efforts in the United States and elsewhere, the number of new patients switching to ORLADEYO, patient retention and demand, the number of physicians prescribing ORLADEYO, the rate of monthly prescriptions, reimbursement from third-party and government payors, the number of patients receiving free product, our pricing strategy, and market trends. We monitor and analyze this data on an ongoing basis as we continue to commercialize ORLADEYO and adjust our forecasts accordingly.

Previous wording

We are developing our investigational plasma kallikrein inhibitor, avoralstat, with Clearside Biomedical, Inc.’s SCS Microinjector®to deliver avoralstat to the back of the eye through the suprachoroidal space to treat patients with diabetic macular edema (“DME”). DME is an important cause of vision loss in diabetes and is due to leakage of fluid from the blood vessels in the retina. While current treatments focus on vascular endothelial growth factor (“VEGF”) inhibition, DME can develop from other mechanisms, such as the kallikrein-bradykinin pathway. This is supported by observations that many DME patients have an incomplete response to intravitreal anti-VEGF therapies that are administered every four to eight weeks. Avoralstat targets the kallikrein-bradykinin system on the retinal vascular endothelial cells and may result in less vascular leakage and less edema. Avoralstat, delivered to the suprachoroidal space, is designed to provide long-lasting exposure to the retinal vessels, which could result in less frequent injections and a reduced burden on patients and the healthcare system.

Current wording

Avoralstat, an investigational plasma kallikrein inhibitor, is designed to treat patients with diabetic macular edema (“DME”) through delivery of avoralstat to the back of the eye through the suprachoroidal space. DME is an important cause of vision loss in diabetes and is due to leakage of fluid from the blood vessels in the retina. While current treatments focus on vascular endothelial growth factor (“VEGF”) inhibition, DME can develop from other mechanisms, such as the kallikrein-bradykinin pathway. This is supported by observations that many DME patients have an incomplete response to intravitreal anti-VEGF therapies that are administered every four to eight weeks. Avoralstat targets the kallikrein-bradykinin system on the retinal vascular endothelial cells and may result in less vascular leakage and less edema. Avoralstat, delivered to the suprachoroidal space, is designed to provide long-lasting exposure to the retinal vessels, which could result in less frequent injections and a reduced burden on patients and the healthcare system.

Previous wording

Our operating expenses are also difficult to predict and depend on several factors, including research and development expenses, drug manufacturing, clinical research activities, the ongoing requirements of our development programs, the costs of commercialization, and direction from regulatory agencies, which are difficult to predict, and the factors discussed in the “Risk Factors” section in Part II, Item 1A of this report. Management may be able to control the timing and level of research and development and selling, general and administrative expenses, but many of these expenditures will occur irrespective of our actions due to contractually committed activities and/or payments.

Current wording

Our operating expenses are also difficult to predict and depend primarily on research and development activities, including drug manufacturing, clinical research activities, and the ongoing requirements of our development programs, as well as the costs of commercialization, direction from regulatory agencies and the factors discussed in the “Risk Factors” section in Part II, Item 1A of this report. Management may be able to control the timing and level of research and development and selling, general and administrative expenses, but many of these expenditures will occur irrespective of our actions due to contractually committed activities and/or payments.

Previous wording

On May 5, 2025, we announced that the FDA has cleared our investigational new drug application, which will enable our clinical trial of BCX17725 to enroll patients in the United States. This phase 1 trial is also open in Australia. In addition, on July 30, 2025, we were notified that the FDA has granted Fast Track designation for BCX17725 for the treatment of Netherton syndrome. On August 4, 2025, we announced that we continue to expect initial data from the program by the end of the year.

Current wording

On July 30, 2025, we were notified that the U.S. Food and Drug Administration (the “FDA”) granted Fast Track designation for BCX17725 for the treatment of Netherton syndrome. On November 3, 2025, we announced that we expect initial data from Netherton syndrome patients participating in our phase 1 trial of BCX17725 by the end of the first quarter of 2026.

Previous wording

On May 5, 2025, we announced that the first clinical trial with suprachoroidal delivery of avoralstat was granted authorization to proceed in Australia. On August 4, 2025, we announced that we continue to expect initial data from this program by the end of the year.

Current wording

On August 4, 2025, we announced that we were enrolling patients in the first clinical trial with suprachoroidal delivery of avoralstat in Australia. On November 3, 2025, we reaffirmed that we expect initial data from the avoralstat program by the end of the year and announced that we plan to seek a strategic partner for development beyond phase 1.

Previous wording

Under the terms of the Stock Purchase Agreement, we have agreed to sell to Neopharmed all of our equity interests in BioCryst Ireland, which, together with its subsidiaries, holds certain assets, rights, and employees related to our European ORLADEYO business (the “European ORLADEYO Business”). Concurrent with the closing of the transactions contemplated by the Purchase Agreement, we and BioCryst Ireland will amend and restate our existing intellectual property license agreement pursuant to which we will continue to grant to BioCryst Ireland certain rights with respect to ORLADEYO in the territory (the “IP License Agreement”). The terms of the IP License Agreement may also extend to the pediatric line extension of ORLADEYO, subject to certain regulatory approvals. Upon closing of the transaction, Neopharmed will pay us$250.0 million in cash, subject to customary purchase price adjustments. In addition, Neopharmed has agreed to pay us up to$14.0 million if certain revenue milestones are achieved prior to December 31, 2032. Neopharmed will also pay a $15.0 million royalty release fee to RPI 2019 Intermediate Finance Trust. The transaction is expected to close in October 2025, and we plan to use the proceeds from the transaction to retire all remaining outstanding term debt under the Pharmakon Loan Agreement.

Current wording

As previously disclosed, on June 27, 2025, we entered into a stock purchase agreement (the “Stock Purchase Agreement”) with BioCryst Ireland Limited (“BioCryst Ireland”), a private limited company incorporated under the laws of Ireland and a wholly owned subsidiary of the Company, and Neopharmed Gentili S.p.A., a corporation organized under the laws of Italy (“Neopharmed”). On October 1, 2025 (the “Closing”), under the terms of the Stock Purchase Agreement, we sold to Neopharmed all of our equity interests in BioCryst Ireland, which, together with its subsidiaries, holds certain assets, rights, and employees related to our European ORLADEYO business (the “European ORLADEYO Business”). At the Closing, we received cash proceeds of$250.0 million, plus customary purchase price adjustments as set forth in the Stock Purchase Agreement. In addition, Neopharmed has agreed to pay us up to$14.0 million if certain revenue milestones are achieved prior to December 31, 2032. In connection with the Closing, Neopharmed also paid a $15.0 million royalty release fee to RPI 2019 Intermediate Finance Trust.

Previous wording

On April 18, 2025, we made a partial prepayment of $75.0 million of the outstanding principal amount under the Pharmakon Loan Agreement. On July 24, 2025, we made an additional partial prepayment of$50.0 million of the outstanding principal amount under the Pharmakon Loan Agreement.

Current wording

As previously disclosed, on July 24, 2025, we made a partial prepayment of$50.0 million of the outstanding principal amount under the Pharmakon Loan Agreement. On October 8, 2025, we used a portion of the proceeds from the sale of the European ORLADEYO Business to pay off in full the outstanding principal balance of $198.7 million and terminate the Pharmakon Loan Agreement.

Previous wording

For the six months ended June 30, 2025, total revenues were $308.9 million compared to $202.1 million for the six months ended June 30, 2024. The increase in total revenues was due to a $93.9 million increase in ORLADEYO net revenue, excluding royalties, primarily due to an increase in direct sales of ORLADEYO due to both an increase in volume, was driven by strong patient demand and an increase in the rate of paid shipments, and an increase in price. The increase in total revenues was also due to an increase in other revenues of $12.9 million, primarily due to an increase in direct sales of peramivir.

Current wording

For the three months ended September 30, 2025, total revenues were $159.4 million compared to $117.1 million for the three months ended September 30, 2024. The increase in total revenues was due to a $42.8 million increase in ORLADEYO net revenue, including royalties, primarily due to an increase in volume of direct sales of ORLADEYO, which was driven by strong patient demand, an increase in price, and an increase in the rate of paid shipments.

Previous wording

Cost of product sales for the six months ended June 30, 2025 and 2024 was $7.4 million and $3.0 million, respectively. The increase in cost of product sales was primarily due to the increase in peramivir and ORLADEYO sales, and an increase in inventory reserves.

Current wording

Cost of product sales for the three months ended September 30, 2025 and 2024 was $2.2 million and $3.2 million, respectively. The decrease in cost of product sales was primarily due to a decrease in inventory reserves recorded during the three months ended September 30, 2025 compared to the three months ended September 30, 2024.

Previous wording

The following table summarizes our research and development expenses for the periods indicated (in thousands):

Current wording

The following table summarizes our research and development expenses, including program specific costs and shared or indirect operating costs recognized as research and development expenses for the periods indicated (in thousands):

Previous wording

Research and development expenses decreased to $80.7 million for the six months ended June 30, 2025 from $84.1 million for the six months ended June 30, 2024, primarily due to a decrease in Factor D program expenses attributed to the discontinuation and close-out of the Factor D programs, a decrease in berotralstat-related regulatory, safety and quality expenses as efforts shifted to selling, general, and administrative related activities, reflecting the program’s commercial progression, and a change in general and administrative expense allocations. These reductions were partially offset by increased spending on preclinical and early clinical work for avoralstat, BCX17725, and early-phase pipeline programs, primarily due to investigational new drug application-enabling activities. Further, there was an increase in stock-based compensation expense as a result of the retirement policy adopted in July 2024 and an increase in restricted stock unit awards granted.

Current wording

•$1.2 million decrease in Factor D Program due to the discontinuation and close-out of the program in 2024; and

Previous wording

Research and development expenses include all direct and indirect expenses relating to research and development and are allocated to specific programs at the point of development of a lead product candidate. Direct expenses are charged directly to the program to which they relate, and indirect expenses are allocated based upon internal direct labor hours dedicated to each respective program. Direct expenses consist of compensation for research and development personnel and costs of outside parties to conduct laboratory studies, develop manufacturing processes and manufacture the product candidates, and conduct and manage clinical trials, as well as other costs related to our clinical and preclinical studies. Additionally, direct expenses consist of those costs necessary to discontinue and close out a development program, including termination fees and other commitments. Indirect expenses consist of lab supplies and services, facility expenses, depreciation of development equipment and other overhead of our research and development efforts. Research and development expenses vary according to the number of programs in clinical development and the stage of development of our clinical programs. Later stage clinical programs tend to cost more than earlier stage programs due to the longer length of time of the clinical trials and the higher number of patients enrolled in these clinical trials.

Current wording

Research and development expenses include all direct and indirect expenses relating to research and development activities. Direct expenses consist of compensation for research and development personnel and costs of outside parties to conduct laboratory studies, develop manufacturing processes and manufacture the product candidates, and conduct and manage clinical trials, as well as other costs related to our clinical and preclinical studies. Additionally, direct expenses consist of those costs necessary to discontinue and close out a development program, including termination fees and other commitments. Indirect expenses consist of lab supplies and services, facility expenses, depreciation of development equipment and other overhead of our research and development efforts. Research and development expenses vary according to the number of programs in clinical development and the stage of development of our clinical programs. Later stage clinical programs tend to cost more than earlier stage programs due to the longer length of time of the clinical trials and the higher number of patients enrolled in these clinical trials.

Previous wording

Selling, general and administrative expenses for the three months ended June 30, 2025 were $87.4 million compared to $61.2 million for the three months ended June 30, 2024. This increase was primarily driven by $5.9 million of transaction costs related to the sale of our European ORLADEYO Business to Neopharmed, an increase in stock-based compensation expense as a result of the retirement policy adopted in July 2024 and an increase in restricted stock unit awards granted. Further, there was an increase in ORLADEYO related regulatory, safety and quality activities as efforts shifted from development to supporting commercial products, reflecting the program’s commercial progression, as well as increases in variable costs driven by the increase in ORLADEYO sales.

Current wording

Selling, general and administrative expenses for the three months ended September 30, 2025 were $83.0 million compared to $65.1 million for the three months ended September 30, 2024. This increase was driven by an increase in ORLADEYO related regulatory, safety and quality activities as efforts shifted from development to supporting commercial products, reflecting the program’s commercial progression, as well as increases in variable costs driven by the increase in ORLADEYO sales and an increase in headcount primarily associated with European growth. This increase was also driven by $3.5 million of transaction-related costs and a $3.4 million increase in stock-based compensation expense as a result of an increase in restricted stock units granted and adjustments for estimated forfeitures.

Previous wording

Interest expense for the three months ended June 30, 2025 was $21.6 million compared to $24.7 million for the three months ended June 30, 2024. Interest expense is primarily comprised of non-cash interest expense due to the amortization of interest associated with the royalty financing obligations and interest expense associated with the borrowings under the Pharmakon Loan Agreement (as defined below), including the amortization of the deferred financing costs, associated with the borrowings under the Pharmakon Loan. The decrease in interest expense was primarily the result of the $75.0 million partial prepayment on the outstanding principal amount under the Pharmakon Term Loan in April 2025, and a decrease in the effective interest rate related to the Pharmakon Loan Agreement.

Current wording

Interest expense for the three months ended September 30, 2025 was $19.7 million compared to $24.8 million for the three months ended September 30, 2024. Interest expense is primarily comprised of non-cash interest expense due to the amortization of interest associated with the royalty financing obligations and interest expense associated with the borrowings under the Pharmakon Loan Agreement (as defined below), including the amortization of the deferred financing costs, associated with the borrowings under the Pharmakon Loan. The decrease in interest expense was primarily the result of the $75.0 million and $50.0 million partial prepayments on the outstanding principal amount under the Pharmakon Term Loan in April 2025 and July 2025, respectively, and a decrease in the effective interest rate related to the Pharmakon Loan Agreement.

Previous wording

For the six months ended June 30, 2025, interest income was $5.5 million compared to $7.6 million for the six months ended June 30, 2024. The decrease in interest income was primarily the result of an overall decrease in our investment portfolio. Net foreign currency losses were less than $0.1 million for the six months ended June 30, 2025 compared to losses of $0.1 million for the six months ended June 30, 2024.

Current wording

For the three months ended September 30, 2025, interest income was $2.2 million compared to $3.6 million for the three months ended September 30, 2024. The decrease in interest income was primarily the result of an overall decrease in our investment portfolio and a decrease in interest rates. Net foreign currency gains were less than $0.1 million for the three months ended September 30, 2025 and $0.1 million for the three months ended September 30, 2024.

Previous wording

In April 2025, the Company made a $75.0 million partial prepayment on the outstanding principal amount under the Pharmakon Term Loan resulting in a one-time loss on extinguishment of debt of $4.2 million for the three months ended June 30, 2025.

Current wording

In July 2025, the Company made a $50.0 million partial prepayment on the outstanding principal amount under the Pharmakon Term Loan resulting in a one-time loss on extinguishment of debt of $2.7 million for the three months ended September 30, 2025.

Previous wording

For the three months ended June 30, 2025, total revenues were $163.4 million compared to $109.3 million for the three months ended June 30, 2024. The increase in total revenues was due to a $48.6 million increase in ORLADEYO net revenue, excluding royalties, primarily due to an increase in direct sales of ORLADEYO due to an increase in volume, which was driven by strong patient demand, and an increase in the rate of paid shipments. The increase in total revenues was also due to an increase in other revenues of $5.5 million, primarily due to an increase in direct sales of peramivir.

Current wording

For the nine months ended September 30, 2025, total revenues were $468.3 million compared to $319.2 million for the nine months ended September 30, 2024. The increase in total revenues was due to a $136.7 million increase in ORLADEYO net revenue, including royalties, primarily due to an increase in volume of direct sales of ORLADEYO, which was driven by strong patient demand, an increase in price, and an increase in the rate of paid shipments. The increase in total revenues was also due to an increase in other revenues of $12.4 million, primarily due to an increase in direct sales of peramivir.

Previous wording

Cost of product sales for the three months ended June 30, 2025 and 2024 was $2.8 million and $1.7 million, respectively. The increase in cost of product sales was primarily due to the increase in peramivir sales and an increase in inventory reserves.

Current wording

Cost of product sales for the nine months ended September 30, 2025 and 2024 was $9.6 million and $6.2 million, respectively. The increase in cost of product sales was primarily due to the increase in peramivir sales.

Previous wording

Research and development expenses include all direct and indirect expenses relating to research and development and are allocated to specific programs at the point of development of a lead product candidate. Direct expenses are charged directly to the program to which they relate, and indirect expenses are allocated based upon internal direct labor hours dedicated to each respective program. Direct expenses consist of compensation for research and development personnel and costs of outside parties to conduct laboratory studies, develop manufacturing processes and manufacture the product candidates, and conduct and manage clinical trials, as well as other costs related to our clinical and preclinical studies. Additionally, direct expenses consist of those costs necessary to discontinue and close out a development program, including termination fees and other commitments. Indirect expenses consist of lab supplies and services, facility expenses, depreciation of development equipment and other overhead of our research and development efforts. Research and development expenses vary according to the number of programs in clinical development and the stage of development of our clinical programs. Later stage clinical programs tend to cost more than earlier stage programs due to the longer length of time of the clinical trials and the higher number of patients enrolled in these clinical trials.

Current wording

Research and development expenses include all direct and indirect expenses relating to research and development activities. Direct expenses consist of compensation for research and development personnel and costs of outside parties to conduct laboratory studies, develop manufacturing processes and manufacture the product candidates, and conduct and manage clinical trials, as well as other costs related to our clinical and preclinical studies. Additionally, direct expenses consist of those costs necessary to discontinue and close out a development program, including termination fees and other commitments. Indirect expenses consist of lab supplies and services, facility expenses, depreciation of development equipment and other overhead of our research and development efforts. Research and development expenses vary according to the number of programs in clinical development and the stage of development of our clinical programs. Later stage clinical programs tend to cost more than earlier stage programs due to the longer length of time of the clinical trials and the higher number of patients enrolled in these clinical trials.

Previous wording

Selling, general and administrative expenses for the six months ended June 30, 2025 were $169.9 million compared to $120.7 million for the six months ended June 30, 2024. The increase was primarily driven by $6.4 million of transaction costs related to the sale of our European ORLADEYO Business, an increase from a change in general and administrative expense allocations, an increase in stock-based compensation expense as a result of the retirement policy adopted in July 2024 and an increase in restricted stock unit awards granted. Further, there was an increase in ORLADEYO related regulatory, safety and quality activities as efforts shifted from development to supporting commercial products, reflecting the program’s commercial progression, as well as increases in variable costs driven by the increase in ORLADEYO sales.

Current wording

Selling, general and administrative expenses for the nine months ended September 30, 2025 were $252.9 million compared to $185.8 million for the nine months ended September 30, 2024. The increase was driven by an increase in ORLADEYO related regulatory, safety and quality activities as efforts shifted from development to supporting commercial products, reflecting the program’s commercial progression, as well as increases in variable costs driven by the increase in ORLADEYO sales, an increase from a change in general and administrative expense allocations, and an increase in headcount primarily associated with European growth. The increase was also driven by $9.9 million of transaction-related costs and a $13.7 million increase in stock-based compensation expense as a result of the retirement policy adopted in July 2024 and an increase in restricted stock unit awards granted.

Previous wording

Interest expense for the six months ended June 30, 2025 was $45.1 million compared to $49.2 million for the six months ended June 30, 2024. Interest expense is primarily comprised of non-cash interest expense due to the amortization of interest associated with the royalty financing obligations and interest expense associated with the borrowings under the Pharmakon Loan Agreement (as defined below), including the amortization of the deferred financing costs, associated with the borrowings under the Pharmakon Loan. The decrease in interest expense was primarily the result of the $75.0 million partial prepayment on the outstanding principal amount under the Pharmakon Term Loan in April 2025, and the decrease in the effective interest rate related to the Pharmakon Loan Agreement.

Current wording

Interest expense for the nine months ended September 30, 2025 was $64.7 million compared to $74.1 million for the nine months ended September 30, 2024. Interest expense is primarily comprised of non-cash interest expense due to the amortization of interest associated with the royalty financing obligations and interest expense associated with the borrowings under the Pharmakon Loan Agreement, including the amortization of the deferred financing costs, associated with the borrowings under the Pharmakon Loan. The decrease in interest expense was primarily the result of the $75.0 million and $50.0 million partial prepayments on the outstanding principal amount under the Pharmakon Term Loan in April 2025 and July 2025, respectively, the decrease in the effective interest rate related to the Pharmakon Loan Agreement, and a decrease in non-cash interest expense associated with our royalty financing obligations as a result of a lower outstanding balance.

Previous wording

For the three months ended June 30, 2025, interest income was $2.5 million compared to $3.6 million for the three months ended June 30, 2024. Net foreign currency losses were less than $0.1 million for the three months ended June 30, 2025 and 2024.

Current wording

For the nine months ended September 30, 2025, interest income was $7.8 million compared to $11.2 million for the nine months ended September 30, 2024. The decrease in interest income was primarily the result of an overall decrease in our investment portfolio and a decrease in interest rates. Net foreign currency losses were less than $0.1 million for the nine months ended September 30, 2025 and 2024.

Previous wording

In April 2025, the Company made a $75.0 million partial prepayment on the outstanding principal amount under the Pharmakon Term Loan resulting in a one-time loss on extinguishment of debt of $4.2 million for the six months ended June 30, 2025.

Current wording

In April 2025 and July 2025, the Company made $75.0 million and $50.0 million partial prepayments on the outstanding principal amount under the Pharmakon Term Loan, respectively, resulting in a one-time loss on extinguishment of debt of $6.9 million for the nine months ended September 30, 2025.

Previous wording

For the three months ended June 30, 2025, income tax expense was $1.4 million compared to $0.2 million for the three months ended June 30, 2024. The increase in income tax expense was primarily driven by the increase in net income for the three months ended June 30, 2025 compared to the net loss incurred in the three months ended June 30, 2024.

Current wording

For the nine months ended September 30, 2025, income tax expense was $1.4 million compared to $1.1 million for the nine months ended September 30, 2024. The increase in income tax expense was primarily driven by the increase in foreign and US taxable income for the nine months ended September 30, 2025 compared to the nine months ended September 30, 2024.

Previous wording

Our operations have principally been funded through public offerings and private placements of equity securities; our credit facilities; revenues from ORLADEYO; royalty financing transactions; and cash from collaborative and other research and development agreements, including U.S. Government contracts. In addition to the above, we have received funding from other sources, including other collaborative and other research and development agreements, government grants, research grants, and interest income on our investments.

Current wording

Our operations have principally been funded through public offerings and private placements of equity securities; our credit facilities; revenues from ORLADEYO; royalty financing transactions; and cash from collaborative and other research and development agreements, including U.S. Government contracts. In addition to the above, we have received funding from other sources, including government grants, research grants, and interest income on our investments.

Previous wording

On April 17, 2023, we entered into a $450.0 million Loan Agreement (the “Pharmakon Loan Agreement”) with BioPharma Credit Investments V (Master) LP and BPCR Limited Partnership, as lenders, and BioPharma Credit PLC, as collateral agent for the lenders. The Pharmakon Loan Agreement provides for an initial term loan in the principal amount of $300.0 million (the “Tranche A Loan”), which was funded on April 17, 2023. We utilized the proceeds from the Tranche A Loan to repay the approximate $241.8 million of outstanding indebtedness under the then-existing credit facility with Athyrium Opportunities III Co-Invest 1 LP (the “Athyrium Credit Agreement”) and to pay transaction costs and fees, and we used the remaining net proceeds of approximately $25.8 million for other general corporate purposes. The Pharmakon Loan Agreement also provided for three additional term loan tranches in principal amounts of $50.0 million each, which we could have requested, at our option, on or prior to September 30, 2024. We chose not to request any of the additional term loan tranches and the options have now expired. The maturity date of the Pharmakon Loan Agreement is April 17, 2028. On April 18, 2025, we made a partial prepayment of $75.0 million of the outstanding principal amount under the Pharmakon Loan Agreement. On July 24, 2025, we made an additional partial prepayment of $50.0 million of the outstanding principal amount under the Pharmakon Loan Agreement. In connection with the closing of the transaction with Neopharmed described above, we plan to use the proceeds from the transaction to retire all remaining outstanding term debt under the Pharmakon Loan Agreement.

Current wording

On April 17, 2023, we entered into a $450.0 million Loan Agreement (the “Pharmakon Loan Agre ement”) with BioPharma Credit Investments V (Master) LP and BPCR Limited Partnership, as lenders, and BioPharma Credit PLC, as collateral agent for the lenders. The Pharmakon Loan Agreement provided for an initial term loan in the principal amount of $300.0 million (the “Tranche A Loan”), which was funded on April 17, 2023. We utilized a portion of the proceeds from the Tranche A Loan to repay the approximate $241.8 million of outstanding indebtedness under the then-existing credit facility with Athyrium Opportunities III Co-Invest 1 LP (the “Athyrium Credit Agreement”) and to pay transaction costs and fees, and we used the remaining net proceeds of approximately $25.8 million for other general corporate purposes.

Previous wording

Our principal sources of liquidity at June 30, 2025 were approximately $285.2 million in cash and cash equivalents and available-for-sale investments, of which $15.1 million was considered held for sale as of June 30, 2025.

Current wording

Our principal sources of liquidity at September 30, 2025 were approximately $267.4 million in cash and cash equivalents and available-for-sale investments, of which $14.8 million was considered held for sale as of September 30, 2025.

Previous wording

During the six months ended June 30, 2025, net cash provided by operating activities of $13.8 million consisted primarily of a net income of $5.1 million and $59.8 million of changes in operating assets and liabilities, primarily due to a decrease in royalty financing obligations and an increase in receivables, offset by $68.4 million of non-cash items, including $26.9 million of non-cash interest expense on royalty financing obligations and $42.7 million of stock-based compensation expense.

Current wording

During the nine months ended September 30, 2025, net cash provided by operating activities of $55.4 million consisted primarily of net income of $18.0 million and $107.7 million of non-cash items. Non-cash items included $61.3 million of stock-based compensation expense, $39.8 million of non-cash interest expense on royalty financing obligations, and a loss on extinguishment of debt of $6.9 million. Net income and non-cash items were partially offset by $61.1 million of changes in operating assets and liabilities, primarily due to a decrease in royalty financing obligations and increases in receivables and accounts payable and accrued expenses, and $9.2 million in payments of Pharmakon PIK interest.

Previous wording

During the six months ended June 30, 2024, net cash used in operating activities of $55.1 million consisted primarily of a net loss of $48.1 million and $66.8 million of changes in operating assets and liabilities, primarily due to a decrease in accounts payable and accrued expenses, a decrease in royalty financing obligations, and an increase in accounts receivable, partially offset by $59.8 million of non-cash items, including $28.5 million in non-cash interest expense on royalty financing obligations and $26.8 million of stock-based compensation expense.

Current wording

During the nine months ended September 30, 2024, net cash used in operating activities of $46.8 million consisted primarily of a net loss of $62.1 million and $76.0 million of changes in operating assets and liabilities, primarily due to a decrease in royalty financing obligations, a decrease in accounts payable and accrued expenses, and an increase in accounts receivable, partially offset by $91.2 million of non-cash items, including $44.1 million of stock-based compensation expense, $42.5 million in non-cash interest expense on royalty financing obligations, and $11.1 million of non-cash interest expense on secured term loan and amortization of debt issuance costs.

Previous wording

During the six months ended June 30, 2025, net cash provided by investing activities of $56.2 million primarily related to sales and maturities of investment securities, partially offset by purchases of investment securities.

Current wording

During the nine months ended September 30, 2025, net cash provided by investing activities of $69.8 million primarily related to sales and maturities of investment securities, partially offset by purchases of investment securities.

Previous wording

During the six months ended June 30, 2024, net cash provided by investing activities of $26.3 million primarily related to maturities of investment securities, partially offset by purchases of investment securities.

Current wording

During the nine months ended September 30, 2024, net cash provided by investing activities of $34.8 million primarily related to maturities of investment securities, partially offset by purchases of investment securities.

Previous wording

During the six months ended June 30, 2025, net cash used in financing activities of $73.2 million primarily consisted of repayment of Pharmakon term loan principal and related prepayment premium and fees, and principal payments on royalty financing obligations, partially offset by net proceeds from common stock issued under stock-based compensation plans.

Current wording

During the nine months ended September 30, 2025, net cash used in financing activities of $132.0 million primarily consisted of repayment of Pharmakon term loan principal and related prepayment premium and fees totaling $119.6 million, and $16.4 million in principal payments on royalty financing obligations, partially offset by net proceeds from common stock issued under stock-based compensation plans.

Previous wording

During the six months ended June 30, 2024, net cash used in financing activities of $3.0 million consisted of withholding taxes paid on stock-based awards and principal payments on finance lease liabilities, partially offset by net proceeds from common stock issued under stock-based compensation plans.

Current wording

During the nine months ended September 30, 2024, net cash used in financing activities of $1.7 million primarily consisted of withholding taxes paid on stock-based awards and principal payments on finance lease liabilities, partially offset by net proceeds from common stock issued under stock-based compensation plans.

Previous wording

We intend to contain costs and cash flow requirements by closely managing our third-party costs and headcount, leasing scientific equipment and facilities, and contracting with other parties to conduct certain research and development projects. We may incur additional expenses, potentially resulting in significant losses, as we continue to pursue our research and development activities, commercialize ORLADEYO, and hire additional personnel. We may incur additional expenses related to the filing, prosecution, maintenance, defense, and enforcement of patent and other intellectual property claims and additional regulatory costs as our clinical programs advance through later stages of development or as regulatory exclusivity for our products expires. The objective of our investment policy is to ensure the safety and preservation of invested funds, as well as to maintain liquidity sufficient to meet cash flow requirements. We place our excess cash with high credit quality financial institutions, commercial companies, and government agencies in order to limit the amount of our credit exposure. We have not realized any significant losses on our investments.

Current wording

We intend to contain costs and cash flow requirements by closely managing our third-party costs and headcount, leasing scientific equipment and facilities, and contracting with other parties to conduct certain research and development projects. We may incur additional expenses, potentially resulting in significant losses, as we continue to pursue our research and development activities, commercialize ORLADEYO, and engage in strategic business development. We may incur additional expenses related to the filing, prosecution, maintenance, defense, and enforcement of patent and other intellectual property claims and additional regulatory costs as our clinical programs advance through later stages of development or as regulatory exclusivity for our products expires. The objective of our investment policy is to ensure the safety and preservation of invested funds, as well as to maintain liquidity sufficient to meet cash flow requirements. We place our excess cash with high credit quality financial institutions, commercial companies, and government agencies in order to limit the amount of our credit exposure. We have not realized any significant losses on our investments.

Previous wording

Our current and planned clinical trials, plus the related development, manufacturing, regulatory approval process requirements, and additional personnel resources and testing required for the continuing development of our product candidates and the commercialization of our products will consume significant capital resources and could increase our expenses.

Current wording

Our current and planned clinical trials, plus the related development, manufacturing, regulatory approval process requirements, and additional resources required for the continuing development of our product candidates and the commercialization of our products will consume significant capital resources and could increase our expenses.

Previous wording

Our expenses, revenues and cash utilization rate could vary significantly depending on many factors, including the development progress of our collaborative agreements for our product candidates, the amount of funding or assistance, if any, we receive from new partnerships with third parties for the development and/or commercialization of our products and product candidates, the amount and timing of funding we receive, if any, from U.S. Government contracts, the progress and results of our current and proposed clinical trials for our most advanced product candidates, the progress made in the manufacturing of our lead product candidates, the success of our commercialization efforts for, and market acceptance of, our products, and the overall progression of our other programs.

Current wording

Our expenses, revenues and cash utilization rate could vary significantly depending on many factors, including the progress and results of our current and proposed clinical trials for our most advanced product candidates; the progress made in the manufacturing of our lead product candidates; the success of our commercialization efforts for, and market acceptance of, our products; the overall progression of our other programs; our business development activities; the amount of funding or assistance, if any, we receive from new partnerships with third parties for the development and/or commercialization of our products and product candidates; the development progress of any collaborative agreements for our product candidates; and the amount and timing of funding we receive, if any, from U.S. Government contracts.

Previous wording

Based on our expectations for revenue and operating expenses, we believe our financial resources will be sufficient to fund our operations for at least the next 12 months. We have no immediate need to access the capital markets, and we did not draw down the additional debt available to us under the Pharmakon Loan Agreement. Our liquidity needs will be largely determined by the success of operations in regard to the successful commercialization of our products and the future progression of our product candidates. From time to time, we evaluate other opportunities to fund future operations, including: (1) out-licensing rights to certain of our products or product candidates, pursuant to which we would receive cash milestone payments; (2) royalty or other monetization transactions; (3) obtaining additional product candidate regulatory approvals, which would generate revenue, milestone payments and cash flow; (4) reducing spending on one or more research and development programs, including by discontinuing development; (5) restructuring operations to change our overhead structure; and/or (6) securing U.S. Government funding of our programs, including obtaining procurement contracts. We may, in the future, issue securities, including common stock, preferred stock, depositary shares, purchase contracts, warrants, debt securities, and units, through private placement transactions or registered public offerings. Our future liquidity needs, and our ability to address those needs, will largely be determined by the success of our products and product candidates; the timing, scope, and magnitude of our research and development and commercial expenses; and key developments and regulatory events and our decisions in the future.

Current wording

Our liquidity needs will be largely determined by the success of operations in regard to the successful commercialization of our products and the future progression of our product candidates. From time to time, we evaluate other opportunities to fund future operations, including: (1) out-licensing rights to certain of our products or product candidates, pursuant to which we would receive cash milestone payments; (2) royalty or other monetization transactions; (3) obtaining additional product candidate regulatory approvals, which would generate revenue, milestone payments and cash flow; (4) reducing spending on one or more research and development programs, including by discontinuing development; (5) restructuring operations to change our overhead structure; and/or (6) securing U.S. Government funding of our programs, including obtaining procurement contracts. We may, in the future, issue securities, including common stock, preferred stock, depositary shares, purchase contracts, warrants, debt securities, and units, through private placement transactions or registered public offerings. Our future liquidity needs, and our ability to address those needs, will largely be determined by the success of our products and product candidates; the timing, scope, and magnitude of our research and development and commercial expenses; and key developments and regulatory events and our decisions in the future.