Core Insights - BioCryst Pharmaceuticals has completed the sale of its European ORLADEYO business to Neopharmed Gentili for $250 million, with potential future milestones of up to $14 million [1][8] - The transaction allows BioCryst to focus on its core U.S. market, significantly improving its operating margin and cash flow generation [2][8] - Neopharmed Gentili will manage the commercialization of ORLADEYO in Europe, retaining the existing European commercial organization built by BioCryst [8] Transaction Details - The sale price of $250 million reflects a multiple of approximately 5.4 times sales over the last twelve months ending June 2025 [8] - BioCryst intends to use the proceeds to retire a $199 million Pharmakon term loan, resulting in a cleaner balance sheet for future strategic activities [8] Strategic Advantages - The divestiture of the European business, which was approximately breakeven on a direct basis, provides an immediate improvement to BioCryst's operating margin [8] - The transaction enhances BioCryst's strategic optionality and positions the company to reach the upper half of its 2025 revenue guidance range of $580 million to $600 million, even without European revenue [2][8]

Core Insights - Investing is viewed as a learning process where failures serve as tuition and successes contribute to lessons learned [1] Group 1 - The author has approximately 5 years of focused research on various stocks, with a primary emphasis on healthcare stocks [1] - The author appreciates feedback from readers, especially those sharing relevant anecdotes and experiences [1] Group 2 - There is a potential for initiating a beneficial long position in BCRX within the next 72 hours [2] - The article expresses the author's own opinions and is not influenced by compensation from any company mentioned [2]

Core Viewpoint - BioCryst Pharmaceuticals is participating in a conference, indicating its active engagement with investors and stakeholders in the biotechnology sector [1][2]. Company Overview - The company is represented by its CFO, Babar Ghias, and President and CCO, Charlie Gayer, who is also referred to as the emerging CEO, highlighting a potential leadership transition [2]. Current State and Outlook - The session aims to provide an introduction to BioCryst, discussing its current state of affairs and outlook for the remainder of the year, suggesting a focus on future growth and strategic direction [2].

Summary of BioCryst Pharmaceuticals (BCRX) 2025 Conference Call Company Overview - **Company**: BioCryst Pharmaceuticals (BCRX) - **Event**: 2025 Conference held on September 4, 2025 - **Key Speakers**: CFO Bob R. Geiss and President & CEO Charlie Guyer Core Points and Arguments Current State and Financial Outlook - BioCryst is experiencing significant growth with its product Orlodea, projected to generate $580 million to $600 million globally in 2025, with a compound annual growth rate (CAGR) of approximately 20% [4] - The company is in the process of selling its European business for $250 million, which will enhance its balance sheet and provide more strategic options [4][13] - The company aims to leverage its existing infrastructure to launch additional products in the rare disease space over the next five years [8] Pipeline Developments - BioCryst has an emerging pipeline, including: - **BCX1775** for Netherton syndrome, with patient data expected later this year [4] - **Avorlstat** for diabetic macular edema (DME), with patient data anticipated at the end of the year [5] - The company is focused on developing treatments for rare diseases, maintaining its commitment to orphan diseases [8][44] Competitive Landscape - The company acknowledges the competitive landscape for Orlodea, particularly with new injectable products entering the market. However, it believes that the unique oral formulation of Orlodea will continue to drive growth [15][16] - BioCryst expects that competition will create more discussions around switching therapies, which could benefit Orlodea [17] Strategic Focus - BioCryst's strategy includes: - Continuing to focus on rare diseases and leveraging existing infrastructure for new product launches [44] - Exploring business development opportunities to acquire late-stage assets in rare diseases, particularly in allergy immunology and rare dermatological conditions [44][47] - The company is open to various deal structures, including staged asset deals and company acquisitions, to expand its portfolio [52] Financial Health and Market Position - The company is transitioning to profitability, with expectations of improved margins following the European business sale [50] - BioCryst's gross-to-net pricing for Orlodea is approximately 15% off the list price, indicating a strong market position [31] Regulatory and Development Path - BioCryst is optimistic about the regulatory path for its Netherton syndrome treatment, anticipating a small pivotal study if strong efficacy is demonstrated [39] - The company is also focused on the pediatric indication for Orlodea, with a PDUFA date set for December 12, which could expand its market significantly [26] Additional Important Insights - The company has built a robust commercial infrastructure that allows for efficient scaling with new products [54] - There is a high willingness among companies to engage in discussions for potential partnerships or acquisitions, creating a favorable environment for BioCryst to pursue strategic opportunities [56] - BioCryst is committed to maintaining a disciplined approach to capital allocation, ensuring that any new investments align with its strategic goals [52][60] This summary encapsulates the key points discussed during the conference call, highlighting BioCryst Pharmaceuticals' current status, strategic outlook, and pipeline developments.

Core Insights - BioCryst Pharmaceuticals announced the departure of Dr. Helen Thackray, the chief research and development officer, effective September 1, 2025, transitioning to an advisory role until the end of the year [1][2] - Dr. Thackray joined BioCryst in 2019 as a board member and became the chief research and development officer in 2021, being a finalist for the CEO succession process [2] - The CEO of BioCryst, Jon Stonehouse, expressed gratitude for Dr. Thackray's contributions, including the launch of a new protein therapeutics platform and advancements in various clinical programs [3] Company Overview - BioCryst Pharmaceuticals is a global biotechnology company focused on improving the lives of individuals with hereditary angioedema and other rare diseases [5] - The company utilizes structure-guided drug design to develop innovative oral small-molecule and protein therapeutics targeting challenging diseases [5] - BioCryst has commercialized ORLADEYO (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of therapies [5]

PART I. FINANCIAL INFORMATION [Item 1. Financial Statements (Unaudited)](index=8&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) Presents unaudited condensed consolidated financial statements, including balance sheets, income, cash flow, and equity, with notes on policies and transactions [Condensed Consolidated Balance Sheets](index=8&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Total assets decreased by **$33.2 million**, liabilities by **$87.6 million**, reducing stockholders' deficit by **$54.3 million**, with new assets held for sale Condensed Consolidated Balance Sheet Highlights (in thousands) | Metric | June 30, 2025 | December 31, 2024 | Change (YoY) | | :-------------------------- | :------------ | :---------------- | :----------- | | Total Assets | $457,188 | $490,420 | $(33,232) | | Total Liabilities | $878,782 | $966,354 | $(87,572) | | Total Stockholders' Deficit | $(421,594) | $(475,934) | $54,340 | | Current assets held for sale | $29,170 | — | $29,170 | | Non-current assets held for sale | $2,825 | — | $2,825 | | Secured term loan | $242,794 | $314,869 | $(72,075) | [Condensed Consolidated Statements of Comprehensive Income (Loss)](index=9&type=section&id=Condensed%20Consolidated%20Statements%20of%20Comprehensive%20Income%20(Loss)) Significant revenue growth and a shift from net loss to net income for Q2 and H1 2025, driven by increased sales and managed expenses Comprehensive Income (Loss) Highlights (in thousands, except per share amounts) | Metric | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--------------------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Revenues | $163,353 | $109,332 | $308,887 | $202,093 | | Total operating expenses | $133,567 | $100,571 | $257,874 | $207,820 | | Income (loss) from operations | $29,786 | $8,761 | $51,013 | $(5,727) | | Net income (loss) | $5,085 | $(12,674) | $5,117 | $(48,053) | | Net income (loss) per common share: basic | $0.02 | $(0.06) | $0.02 | $(0.23) | | Net income (loss) per common share: diluted | $0.02 | $(0.06) | $0.02 | $(0.23) | [Condensed Consolidated Statements of Cash Flows](index=10&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Positive operating cash flow for H1 2025, a significant improvement from prior year, driven by net income despite substantial debt repayment Cash Flow Highlights (Six Months Ended June 30, in thousands) | Metric | 2025 | 2024 | Change (YoY) | | :-------------------------------------------------------------------------------- | :------- | :------- | :----------- | | Net cash provided by (used in) operating activities | $13,785 | $(55,052) | $68,837 | | Net cash provided by investing activities | $56,222 | $26,307 | $29,915 | | Net cash used in financing activities | $(73,157) | $(3,014) | $(70,143) | | Net decrease in cash, and cash equivalents, and restricted cash | $(16,398) | $(32,242) | $15,844 | | Cash, cash equivalents and restricted cash: End of period | $89,925 | $80,205 | $9,720 | [Condensed Consolidated Statements of Stockholders' Deficit](index=12&type=section&id=Condensed%20Consolidated%20Statements%20of%20Stockholders'%20Deficit) Total stockholders' deficit decreased by **$54.3 million** from Dec 2024 to June 2025, driven by net income and stock-based compensation Stockholders' Deficit Highlights (in thousands) | Metric | December 31, 2024 | June 30, 2025 | Change | | :-------------------------- | :---------------- | :------------ | :------- | | Total Stockholders' Deficit | $(475,934) | $(421,594) | $54,340 | | Net income (loss) (6 months) | $(48,053) | $5,117 | $53,170 | | Stock-based compensation expense (6 months) | $26,825 | $42,672 | $15,847 | [Notes to Condensed Consolidated Financial Statements](index=14&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) Provides detailed disclosures on significant accounting policies, financial instruments, and transactions, contextualizing the financial statements [Note 1 — Significant Accounting Policies and Concentrations of Risk](index=14&type=section&id=Note%201%20%E2%80%94%20Significant%20Accounting%20Policies%20and%20Concentrations%20of%20Risk) BioCryst, a global biotech, focuses on rare diseases (HAE, influenza products ORLADEYO, RAPIVAB), expects 12-month funding, operates as one segment, and faces concentration risks - **BioCryst** is a global biotechnology company focused on improving the lives of people with **hereditary angioedema (HAE)** and other rare diseases, utilizing structure-guided drug design to develop oral small-molecule and injectable protein therapeutics[31](index=31&type=chunk) - Marketed products include oral, once-daily **ORLADEYO®** for **HAE** attack prevention and **RAPIVAB®** (peramivir injection) for acute uncomplicated influenza in the United States[32](index=32&type=chunk) - The company believes its financial resources at **June 30, 2025**, will be sufficient to fund operations for at least the **next 12 months**[33](index=33&type=chunk) - The company operates and manages its business as **one reportable and operating segment**[34](index=34&type=chunk) - Revenue from product sales (**ORLADEYO** and **peramivir**) is recognized when the customer obtains control, generally upon delivery, and is recorded at net selling price, including reserves for variable consideration such as government rebates, chargebacks, co-payment assistance, and product returns[43](index=43&type=chunk)[44](index=44&type=chunk)[45](index=45&type=chunk) - Revenue where the specialty pharmacy is considered the customer was approximately **86%** of total net revenues for the three months ended **June 30, 2025**, and **85%** for the six months ended **June 30, 2025**[98](index=98&type=chunk) - The company relies on a **single source manufacturer** for active pharmaceutical ingredient and finished drug product manufacturing and a **single specialty pharmacy** for distribution of approved drug product in the United States, posing concentration risks[100](index=100&type=chunk) [Note 2 — Assets and Liabilities Held for Sale](index=23&type=section&id=Note%202%20%E2%80%94%20Assets%20and%20Liabilities%20Held%20for%20Sale) BioCryst agreed to sell its European ORLADEYO business for **$250 million** upfront, with proceeds to retire debt; assets/liabilities are classified as held for sale - On **June 27, 2025**, BioCryst entered into a definitive agreement to sell its **European ORLADEYO business** (BioCryst Ireland Limited) to Neopharmed Gentili S.p.A[108](index=108&type=chunk) - Neopharmed will pay **$250,000 thousand** in upfront consideration, up to an additional **$14,000 thousand** in contingent milestone payments, and a **$15,000 thousand** royalty release fee[109](index=109&type=chunk) - The transaction is expected to close in the **fourth quarter of 2025**, with proceeds intended to retire all remaining term debt[109](index=109&type=chunk) Assets and Liabilities Held for Sale (June 30, 2025, in thousands) | Category | Amount | | :-------------------------- | :------- | | Current assets held for sale | $29,170 | | Non-current assets held for sale | $2,825 | | **Total assets held for sale** | **$31,995** | | Current liabilities held for sale | $23,708 | | Non-current liabilities held for sale | $454 | | **Total liabilities held for sale** | **$24,162** | [Note 3 — Revenue](index=24&type=section&id=Note%203%20%E2%80%94%20Revenue) Total revenues significantly increased for Q2 and H1 2025, driven by strong U.S. ORLADEYO sales and higher peramivir revenues Revenue Breakdown (in thousands) | Category | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :---------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Total Revenues | $163,353 | $109,332 | $308,887 | $202,093 | | Total ORLADEYO | $156,837 | $108,288 | $291,080 | $197,155 | | ORLADEYO: U.S. | $140,338 | $95,909 | $260,497 | $175,875 | | Other revenues | $6,516 | $1,044 | $17,807 | $4,938 | - Total revenues increased by **$54.0 million (49.4%)** for the three months and **$106.8 million (52.8%)** for the six months ended **June 30, 2025**, compared to the prior year[112](index=112&type=chunk) - **ORLADEYO** revenues increased by **$48.5 million (44.8%)** for the three months and **$93.9 million (47.6%)** for the six months ended **June 30, 2025**, primarily driven by U.S. sales[112](index=112&type=chunk) [Note 4 — Investments](index=25&type=section&id=Note%204%20%E2%80%94%20Investments) Investments, mainly U.S. Government obligations, decreased by **$54.4 million** from Dec 2024 to June 2025, classified as available-for-sale Total Investments (Fair Value, in thousands) | Date | Amount | | :---------------- | :------- | | June 30, 2025 | $182,106 | | December 31, 2024 | $236,460 | | **Change** | **$(54,354)** | Investment Maturity Profile (June 30, 2025, in thousands) | Maturity | Amount | | :-------------------------- | :------- | | Maturing in one year or less | $172,005 | | Maturing after one year through two years | $10,101 | | **Total investments** | **$182,106** | - As of **June 30, 2025**, the company had **six securities** with a total estimated fair value of **$59,117 thousand** in an unrealized loss position, primarily due to interest rate changes, which are considered temporary[116](index=116&type=chunk) [Note 5 — Trade Receivables](index=26&type=section&id=Note%205%20%E2%80%94%20Trade%20Receivables) ORLADEYO trade receivables, net, increased by **$11.3 million** to **$87.6 million**; collaboration receivables and held-for-sale accounts also rose Trade Receivables Highlights (in thousands) | Metric | June 30, 2025 | December 31, 2024 | Change | | :-------------------------- | :------------ | :---------------- | :------- | | ORLADEYO Receivables, net | $87,575 | $76,282 | $11,293 | | Receivables from collaborations | $3,221 | $2,223 | $998 | | Accounts receivable held for sale | $10,403 | — | $10,403 | [Note 6 — Inventory](index=26&type=section&id=Note%206%20%E2%80%94%20Inventory) Total inventory, net, primarily ORLADEYO, decreased by **$4.0 million** to **$27.2 million** at June 30, 2025, including held-for-sale inventory Inventory Breakdown (in thousands) | Category | June 30, 2025 | December 31, 2024 | Change | | :-------------------------- | :------------ | :---------------- | :------- | | Raw materials | $7,252 | $10,006 | $(2,754) | | Work-in-process | $15,943 | $16,152 | $(209) | | Finished goods | $5,965 | $7,765 | $(1,800) | | Total inventory | $29,160 | $33,923 | $(4,763) | | Reserves | $(1,911) | $(2,649) | $738 | | **Total inventory, net** | **$27,249** | **$31,274** | **$(4,025)** | - As of **June 30, 2025**, **$2,043 thousand** of inventory, net, was classified as current assets held for sale, and **$549 thousand** as non-current assets held for sale[123](index=123&type=chunk) [Note 7 — Royalty Financing Obligations](index=26&type=section&id=Note%207%20%E2%80%94%20Royalty%20Financing%20Obligations) Royalty financing obligations decreased by **$30.1 million** to **$483.6 million**; effective interest rates for RPI and OMERS were **21.7%** and **10.1%** Royalty Financing Obligations Activity (in thousands) | Metric | December 31, 2024 | June 30, 2025 | Change | | :------------------------------------------------ | :---------------- | :------------ | :------- | | Balance of Royalty financing obligations | $513,729 | $483,583 | $(30,146) | | Non-cash Interest expense (6 months ended June 30, 2025) | — | $26,916 | $26,916 | | Royalty Revenues Payable (6 months ended June 30, 2025) | — | $(57,062) | $(57,062) | Effective Interest Rates (June 30, 2025) | Agreement | Effective Interest Rate | | :------------------------ | :---------------------- | | 2020 RPI Royalty Agreement | 21.7% | | OMERS Royalty Agreement | 10.1% | - Cash paid for interest on royalty financing obligations was **$36,921 thousand** for the six months ended **June 30, 2025**, compared to **$33,069 thousand** for the same period in **2024**[139](index=139&type=chunk) [Note 8 — Debt](index=29&type=section&id=Note%208%20%E2%80%94%20Debt) Pharmakon Loan borrowings decreased by **$75.0 million** to **$248.7 million** due to prepayment, incurring a **$4.171 million** loss on extinguishment, with variable interest - The Pharmakon Loan Agreement provides for an initial **$300,000 thousand** Tranche A Loan, funded on **April 17, 2023**[141](index=141&type=chunk) Pharmakon Loan Outstanding Borrowings (in thousands) | Date | Amount | | :---------------- | :------- | | June 30, 2025 | $248,704 | | June 30, 2024 | $323,704 | | **Change** | **$(75,000)** | - The Tranche A Loan bears interest at a rate equal to the **three-month SOFR** (no less than **1.75%**) plus **7.00%**, or **SOFR** plus **7.25%** if a Pharmakon PIK Interest Payment was made[143](index=143&type=chunk) - A **$75,000 thousand** partial prepayment was made on **April 18, 2025**, resulting in a one-time loss on extinguishment of debt of **$4,171 thousand**[148](index=148&type=chunk) - Interest expense on the Tranche A Loan for the three and six months ended **June 30, 2025**, was **$7,526 thousand** and **$16,679 thousand**, respectively[149](index=149&type=chunk) [Note 9 — Lease Obligations](index=31&type=section&id=Note%209%20%E2%80%94%20Lease%20Obligations) Operating lease liabilities totaled **$8.881 million** and finance lease liabilities **$3.659 million** as of June 30, 2025, with real estate leases expiring by 2033 Lease Liabilities (June 30, 2025, in thousands) | Lease Type | Total Liabilities | | :---------------- | :---------------- | | Operating leases | $8,881 | | Finance leases | $3,659 | Weighted Average Lease Terms and Discount Rates (June 30, 2025) | Lease Type | Remaining Lease Term | Discount Rate | | :---------------- | :------------------- | :------------ | | Operating leases | 9.9 years | 10.71% | | Finance leases | 2.6 years | 9.42% | [Note 10 — Stockholders' Equity](index=33&type=section&id=Note%2010%20%E2%80%94%20Stockholders'%20Equity) **209,905 thousand** common shares were outstanding as of June 30, 2025, with **71,789 thousand** shares reserved for future issuance under compensation plans - Common stock issued and outstanding as of **June 30, 2025**: **209,905 thousand shares**[26](index=26&type=chunk) Shares Reserved for Future Issuance (in thousands) | Category | June 30, 2025 | | :------------------------------------------ | :------------ | | Shares reserved for exercises of outstanding stock options | 42,093 | | Shares reserved for vesting of restricted stock units | 9,798 | | Shares reserved for future issuance under the Stock Incentive Plan | 13,332 | | Shares reserved for future issuance under the Inducement Equity Incentive Plan | 1,725 | | Shares reserved for future issuance under the Employee Stock Purchase Plan | 4,841 | | **Total shares reserved for future issuance** | **71,789** | [Note 11 — Stock-Based Compensation](index=33&type=section&id=Note%2011%20%E2%80%94%20Stock-Based%20Compensation) Stock-based compensation expense significantly increased to **$42.7 million** for H1 2025, with higher weighted average grant date fair value for stock options Stock-Based Compensation Expense (in thousands) | Plan | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :-------------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Incentive Plan | $19,377 | $10,934 | $38,609 | $22,500 | | Inducement Plan | $1,741 | $2,084 | $3,673 | $4,007 | | ESPP | $186 | $155 | $390 | $318 | | **Total stock-based compensation expense** | **$21,304** | **$13,173** | **$42,672** | **$26,825** | Weighted Average Grant Date Fair Value per Share (Stock Options) | Plan | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :-------------------------- | :------------------------------- | :------------------------------- | | Stock Incentive Plan | $7.12 | $4.56 | | Inducement Equity Incentive Plan | $5.73 | $3.90 | [Note 12 — Collaborative and Other Relationships](index=35&type=section&id=Note%2012%20%E2%80%94%20Collaborative%20and%20Other%20Relationships) BioCryst has collaborations for ORLADEYO (Torii, Pint Pharma) and peramivir (HHS, Shionogi, Green Cross); Torii royalties increased, while HHS will not exercise additional RAPIVAB orders - The Commercialization and License Agreement with **Torii Pharmaceutical Co., Ltd.** for **ORLADEYO** in Japan was amended on **November 30, 2023**, increasing tiered royalty payments to BioCryst from **20-40%** to **20-80%** of annual net sales[168](index=168&type=chunk)[171](index=171&type=chunk) - The **HHS** awarded BioCryst a contract for up to **$69,388 thousand** for **RAPIVAB**, but on **May 15, 2025**, **ASPR** notified the company of its intent not to exercise any additional optional ordering periods[174](index=174&type=chunk) - BioCryst entered into a license agreement with **Clearside Biomedical, Inc.** on **November 3, 2023**, to develop **avoralstat** with Clearside's **SCS Microinjector®** for **diabetic macular edema (DME)**. Clearside received a **$5,000 thousand** upfront license fee and is eligible for up to **$30,000 thousand** in clinical/regulatory milestones and up to **$47,500 thousand** in sales-based milestones[177](index=177&type=chunk)[179](index=179&type=chunk) [Note 13 — Segment Information](index=37&type=section&id=Note%2013%20%E2%80%94%20Segment%20Information) Operates as a single segment, with consolidated cash, cash equivalents, and investments totaling **$285.2 million** as of June 30, 2025 - The company operates as **one reportable and operating segment**, centered around **ORLADEYO** and its pipeline for rare diseases[180](index=180&type=chunk) Consolidated Cash, Cash Equivalents and Investments (in thousands) | Date | Amount | | :---------------- | :------- | | June 30, 2025 | $285,197 | | December 31, 2024 | $341,173 | | **Change** | **$(55,976)** | [Note 14 — Commitments and Contingencies](index=38&type=section&id=Note%2014%20%E2%80%94%20Commitments%20and%20Contingencies) BioCryst filed a patent infringement lawsuit against Annora Pharma on March 10, 2025, challenging its generic ORLADEYO ANDA and seeking injunctive relief - In **January 2025**, **Annora Pharma Private Limited** submitted an **ANDA** to the **FDA** seeking approval for a generic version of **ORLADEYO**, challenging **three patents** expiring in **2039**[184](index=184&type=chunk) - On **March 10, 2025**, BioCryst filed a **patent infringement lawsuit** in the United States District Court for the District of Delaware against **Annora** and related entities, asserting infringement and seeking equitable relief to enjoin infringement and delay **FDA** approval of the **ANDA**[185](index=185&type=chunk) [Note 15 — Net Income (Loss) Per Share](index=39&type=section&id=Note%2015%20%E2%80%94%20Net%20Income%20(Loss)%20Per%20Share) Basic and diluted net income per share for Q2 and H1 2025 was **$0.02**, a significant improvement from net losses of **$(0.06)** and **$(0.23)** in 2024 Net Income (Loss) Per Common Share | Period | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--------------------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Net income (loss) per common share: basic | $0.02 | $(0.06) | $0.02 | $(0.23) | | Net income (loss) per common share: diluted | $0.02 | $(0.06) | $0.02 | $(0.23) | - For periods with a net loss (e.g., three and six months ended **June 30, 2024**), all potentially dilutive securities were excluded from the computation of diluted net loss per share as their effect would be anti-dilutive[188](index=188&type=chunk) [Note 16 — Subsequent Events](index=39&type=section&id=Note%2016%20%E2%80%94%20Subsequent%20Events) On July 24, 2025, a **$50 million** partial prepayment was made on the Pharmakon Term Loan, incurring a **$1.5 million** premium and **$376 thousand** in accrued interest - On **July 24, 2025**, BioCryst made a **$50,000 thousand** partial prepayment on the outstanding principal amount under the **Pharmakon Term Loan**[190](index=190&type=chunk) - In conjunction with the prepayment, the company incurred a **$1,500 thousand** prepayment premium and paid **$376 thousand** of interest accrued[190](index=190&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=40&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses financial condition and results, highlighting products, recent developments, performance comparisons, liquidity, capital, and critical accounting estimates [Overview](index=40&type=section&id=Overview) BioCryst is a global biotech focused on rare diseases, developing first-in-class therapeutics, aiming for commercial success and self-funding through profitability - **BioCryst** is a global biotechnology company focused on improving the lives of people with **hereditary angioedema (HAE)** and other rare diseases[192](index=192&type=chunk) - The company leverages expertise in **structure-guided drug design** to develop first-in-class or best-in-class oral small-molecule and injectable protein therapeutics[192](index=192&type=chunk) - The business strategy includes successful commercialization and self-funding efforts by achieving and increasing profitability, particularly by focusing on **rare disease markets** to control costs[192](index=192&type=chunk) [Products and Product Candidates](index=40&type=section&id=Products%20and%20Product%20Candidates) BioCryst's portfolio includes ORLADEYO (HAE, pediatric NDA pending), pipeline candidates BCX17725 (Netherton), avoralstat (DME), a complement program, and RAPIVAB (influenza) - **ORLADEYO® (berotralstat)** is an oral, once-daily therapy for the prevention of **HAE** attacks, approved in the United States and other global markets for patients **12 years and older**. An **NDA** for an oral granule formulation for pediatric patients aged **2 to 11 years** is under **FDA priority review**[193](index=193&type=chunk)[209](index=209&type=chunk) - The global commercial market for **ORLADEYO** is anticipated to reach a peak of **$1 billion** in annual net revenues, with approximately **80%** expected from the United States[194](index=194&type=chunk) - **BCX17725** is an investigational **KLK5 inhibitor** for **Netherton syndrome**, with an **IND** cleared by the **FDA** and a **Phase 1 trial** open in Australia. It received **Fast Track designation** from the **FDA** on **July 30, 2025**, with initial data expected by year-end[196](index=196&type=chunk)[197](index=197&type=chunk)[216](index=216&type=chunk)[217](index=217&type=chunk) - **Avoralstat**, an investigational plasma kallikrein inhibitor, is being developed with **Clearside Biomedical's SCS Microinjector®** for **diabetic macular edema (DME)**. The first clinical trial was authorized in Australia, with initial data expected by year-end[198](index=198&type=chunk)[218](index=218&type=chunk) - The **complement program** aims to develop first-in-class and/or best-in-class compounds across multiple pathways (classical, lectin, terminal, and alternative) to treat complement-mediated diseases, including **oral C5 and C2 inhibitors**, and a **bifunctional complement inhibitor**[199](index=199&type=chunk)[200](index=200&type=chunk)[201](index=201&type=chunk)[202](index=202&type=chunk) - **RAPIVAB® (peramivir injection)** is approved in the United States for acute uncomplicated influenza in patients **six months and older** and is a key component of the U.S. Government's influenza preparedness efforts[203](index=203&type=chunk) [Revenues and Expenses](index=42&type=section&id=Revenues%20and%20Expenses) Revenues and operating expenses are difficult to predict, influenced by regulatory approvals, commercialization, market acceptance, and R&D costs, making comparisons unreliable - Revenues are difficult to predict and depend on regulatory approval decisions, effectiveness of commercialization efforts, market acceptance of products (especially **ORLADEYO**), and licensing agreements[204](index=204&type=chunk) - Operating expenses are also difficult to predict, influenced by **research and development**, drug manufacturing, clinical research, commercialization costs, and regulatory guidance[205](index=205&type=chunk) - Management can control the timing and level of **R&D** and **SG&A** expenses, but many expenditures are contractually committed[205](index=205&type=chunk) [Recent Developments](index=43&type=section&id=Recent%20Developments) Recent developments include pediatric ORLADEYO NDA, BCX17725 IND/Fast Track, avoralstat trial, Pharmakon prepayments, European ORLADEYO sale, and leadership changes - The percentage of U.S. **HAE** patients preferring oral prophylaxis therapy increased to **70%** in **2025**, up from **50%** in **2023**[207](index=207&type=chunk) - **FDA** accepted the **NDA** for **ORLADEYO** in pediatric **HAE** patients aged **2 to 11 years**, granting **priority review** with a **PDUFA** target action date of **December 12, 2025** (extended from September 12, 2025)[208](index=208&type=chunk)[209](index=209&type=chunk) - **ORLADEYO** received reimbursement approval in the **Netherlands**, marking national reimbursement across all major European countries, and approval in **Colombia** for patients aged **12 years and older**[212](index=212&type=chunk)[213](index=213&type=chunk) - **FDA** cleared the **IND** for **BCX17725** for **Netherton syndrome**, enabling U.S. clinical trials, and granted **Fast Track designation** on **July 30, 2025**. Initial data is expected by year-end[216](index=216&type=chunk)[217](index=217&type=chunk) - The first clinical trial with suprachoroidal delivery of **avoralstat** was authorized in Australia, with initial data expected by year-end[218](index=218&type=chunk) - Partial prepayments of **$75.0 million** (**April 18, 2025**) and **$50.0 million** (**July 24, 2025**) were made on the **Pharmakon Loan Agreement**[219](index=219&type=chunk) - An agreement was signed to sell the **European ORLADEYO Business** to **Neopharmed Gentili S.p.A.** for **$250.0 million** upfront, plus potential milestones, with proceeds planned to retire remaining term debt. The transaction is expected to close in **October 2025**[220](index=220&type=chunk)[221](index=221&type=chunk) - Leadership changes include **Jon Stonehouse's** retirement as **CEO** (effective **Dec 31, 2025**), **Charlie Gayer's** appointment as **President** (**Aug 1, 2025**) and future **CEO** (**Jan 1, 2026**), and **Babar Ghias's** appointment as **CFO** and Head of Corporate Development (**July 7, 2025**)[222](index=222&type=chunk) [Results of Operations (three months ended June 30, 2025 compared to the three months ended June 30, 2024)](index=44&type=section&id=Results%20of%20Operations%20(three%20months%20ended%20June%2030%2C%202025%20compared%20to%20the%20three%20months%20ended%20June%2030%2C%202024)) For Q2 2025, revenues increased by **$54.0 million** to **$163.4 million**, driven by ORLADEYO and peramivir; operating expenses rose, interest expense decreased, resulting in **$5.1 million** net income from a prior year loss - Total revenues increased by **$54.0 million (49.4%)** to **$163.4 million**, primarily due to a **$48.6 million** increase in **ORLADEYO** net revenue and a **$5.5 million** increase in other revenues (**peramivir**)[223](index=223&type=chunk) - Cost of product sales increased to **$2.8 million** from **$1.7 million**, mainly due to higher **peramivir** sales and inventory reserves[224](index=224&type=chunk) - Research and development expenses increased by **$5.8 million (15.4%)** to **$43.4 million**, driven by preclinical/early clinical work for **avoralstat**, **BCX17725**, and early-phase pipeline programs, and increased stock-based compensation. This was partially offset by the discontinuation of **Factor D programs** and a shift of berotralstat-related expenses to **SG&A**[226](index=226&type=chunk) - Selling, general and administrative expenses increased by **$26.2 million (42.8%)** to **$87.4 million**, primarily due to **$5.9 million** in transaction costs for the **European ORLADEYO Business** sale, increased stock-based compensation, and higher **ORLADEYO** commercial support activities[228](index=228&type=chunk) - Interest expense decreased by **$3.1 million** to **$21.6 million**, mainly due to the **$75.0 million** partial prepayment on the **Pharmakon Term Loan** and a decrease in its effective interest rate[229](index=229&type=chunk) - A one-time loss on extinguishment of debt of **$4.2 million** was recognized due to the **Pharmakon Term Loan** prepayment[231](index=231&type=chunk) - Net income was **$5.1 million**, a significant improvement from a net loss of **$12.7 million** in the prior year period[21](index=21&type=chunk) [Results of Operations (six months ended June 30, 2025 compared to the six months ended June 30, 2024)](index=46&type=section&id=Results%20of%20Operations%20(six%20months%20ended%20June%2030%2C%202025%20compared%20to%20the%20six%20months%20ended%20June%2030%2C%202024)) For H1 2025, revenues increased by **$106.8 million** to **$308.9 million**, driven by ORLADEYO and peramivir; R&D decreased, SG&A increased, interest expense decreased, resulting in **$5.1 million** net income from a prior year loss - Total revenues increased by **$106.8 million (52.8%)** to **$308.9 million**, driven by a **$93.9 million** increase in **ORLADEYO** net revenue (volume, paid shipments, price) and a **$12.9 million** increase in other revenues (**peramivir**)[233](index=233&type=chunk) - Cost of product sales increased to **$7.4 million** from **$3.0 million**, primarily due to higher **peramivir** and **ORLADEYO** sales and inventory reserves[234](index=234&type=chunk) - Research and development expenses decreased by **$3.5 million (4.1%)** to **$80.7 million**, mainly due to the discontinuation of **Factor D programs** and a shift of berotralstat-related expenses. This was partially offset by increased spending on **avoralstat**, **BCX17725**, and early-phase pipeline programs, and higher stock-based compensation[235](index=235&type=chunk) - Selling, general and administrative expenses increased by **$49.2 million (40.7%)** to **$169.9 million**, driven by **$6.4 million** in transaction costs for the **European ORLADEYO Business** sale, increased stock-based compensation, and higher **ORLADEYO** commercial support activities[237](index=237&type=chunk) - Interest expense decreased by **$4.1 million** to **$45.1 million**, primarily due to the **$75.0 million** partial prepayment on the **Pharmakon Term Loan** and a decrease in its effective interest rate[238](index=238&type=chunk) - A one-time loss on extinguishment of debt of **$4.2 million** was recognized due to the **Pharmakon Term Loan** prepayment[240](index=240&type=chunk) - Net income was **$5.1 million**, a substantial improvement from a net loss of **$48.1 million** in the prior year period[21](index=21&type=chunk) [Liquidity and Capital Resources](index=47&type=section&id=Liquidity%20and%20Capital%20Resources) Liquidity comes from equity, credit, ORLADEYO revenues, and royalty financing; **$248.7 million** Pharmakon debt outstanding, with European ORLADEYO sale proceeds planned for retirement, sufficient for 12 months - Primary sources of liquidity include public/private equity offerings, credit facilities, **ORLADEYO** revenues, royalty financing transactions, and cash from collaborative and other research and development agreements[242](index=242&type=chunk) - The company entered into a **$450,000 thousand Pharmakon Loan Agreement** in **April 2023**, with an initial **$300,000 thousand** Tranche A Loan. As of **June 30, 2025**, **$248,704 thousand** was outstanding after partial prepayments[243](index=243&type=chunk)[149](index=149&type=chunk) - Proceeds from the pending sale of the **European ORLADEYO Business** to **Neopharmed** are planned to retire all remaining outstanding term debt under the **Pharmakon Loan Agreement**[243](index=243&type=chunk) - As of **June 30, 2025**, principal sources of liquidity were approximately **$285.2 million** in cash, cash equivalents, and available-for-sale investments, including **$15.1 million** classified as held for sale[247](index=247&type=chunk) - The company believes its financial resources will be sufficient to fund operations for at least the **next 12 months**, but future liquidity needs will depend on product success and development progression[258](index=258&type=chunk) [Critical Accounting Estimates](index=50&type=section&id=Critical%20Accounting%20Estimates) Management discusses critical accounting estimates for revenue recognition, inventory valuation, R&D accruals, stock-based compensation, royalty financing interest, and income taxes, all requiring significant judgment - Revenue recognition for product sales involves significant estimates for variable consideration (government rebates, managed care rebates, chargebacks, co-payment assistance, product returns)[269](index=269&type=chunk) - Inventory is valued at the lower of cost or estimated net realizable value, requiring significant judgment for valuation reserves for excess, obsolete, short-dated, or unmarketable inventory[280](index=280&type=chunk)[281](index=281&type=chunk) - **Research and development expenses**, particularly for third-party services, are accrued based on estimates of actual work completed, requiring judgment on expected patient enrollment, site activation, and project duration[286](index=286&type=chunk) - **Stock-based compensation expense** is estimated at the grant date using a **Black-Scholes option-pricing model**, requiring judgment on stock price volatility and expected term, and is reduced for estimated forfeitures[288](index=288&type=chunk) - Interest expense on royalty financing obligations is accrued using the **effective interest rate method**, which requires estimating total future royalty payments over the life of the agreement, with reassessments made each reporting period[289](index=289&type=chunk) - Significant management judgment is required for income taxes, particularly in determining deferred tax assets and liabilities and any valuation allowance against net deferred tax assets, based on estimates of future earnings[291](index=291&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=54&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company faces interest rate risk on investments and variable-rate debt, manages investment risk with short-maturity securities, has foreign currency exposure, and notes inflation has not materially impacted operations - The company is subject to **interest rate risk** on its investment portfolio and borrowings under the **Pharmakon Loan Agreement**, which accrues interest at a variable rate (**three-month SOFR** plus **7.00%** or **7.25%**)[294](index=294&type=chunk)[295](index=295&type=chunk) - Investment policy objectives are to preserve capital, maintain liquidity, and earn a competitive return, achieved by investing in **high credit quality marketable debt securities** with an average maturity of approximately **12 months or less**[296](index=296&type=chunk)[297](index=297&type=chunk) - **Foreign currency risk** arises from operations in various countries, with commercial sales in Europe primarily denominated in **Euros** and **British Pounds**, and royalties from **Torii** in **Japanese Yen**. Other exposures include Swiss Franc, Danish Krone, Swedish Krona, Norwegian Krone, and Canadian Dollar[299](index=299&type=chunk) - Foreign currency transaction gains or losses are not anticipated to be significant at the current level of operations but may increase with international expansion[300](index=300&type=chunk) - **Inflation** could potentially increase operating expenses (cost of product sales, clinical trial costs, selling activities), but has not had a material impact on the business or results of operations during the reported periods[301](index=301&type=chunk) [Item 4. Controls and Procedures](index=55&type=section&id=Item%204.%20Controls%20and%20Procedures) Disclosure controls and procedures were effective as of June 30, 2025, with no material changes in internal control over financial reporting during the quarter - The company's **disclosure controls and procedures** were evaluated and concluded to be effective as of **June 30, 2025**, by the **Chief Executive Officer** and **Chief Financial Officer**[303](index=303&type=chunk) - No changes in **internal control over financial reporting** occurred during the three months ended **June 30, 2025**, that materially affected, or are reasonably likely to materially affect, the company's internal control over financial reporting[304](index=304&type=chunk) PART II. OTHER INFORMATION [Item 1. Legal Proceedings](index=57&type=section&id=Item%201.%20Legal%20Proceedings) BioCryst filed a patent infringement lawsuit against Annora Pharma on March 10, 2025, challenging its generic ORLADEYO ANDA and seeking injunctive relief for patents expiring in 2039 - In **January 2025**, **Annora Pharma Private Limited** submitted an **Abbreviated New Drug Application (ANDA)** to the **FDA** for a generic version of **ORLADEYO**, challenging **three patents** (U.S. Patent Nos. 10,662,160; 11,117,867; and 11,618,733) that expire in **2039**[306](index=306&type=chunk) - On **March 10, 2025**, BioCryst filed a **patent infringement lawsuit** in the United States District Court for the District of Delaware against **Annora** and related entities, asserting infringement and seeking equitable relief to enjoin the defendants and an order to delay the effective date of any **FDA** approval of the **ANDA**[307](index=307&type=chunk) [Item 1A. Risk Factors](index=57&type=section&id=Item%201A.%20Risk%20Factors) This section details various risks including financial, operational, commercialization, competition, legal, intellectual property, and international risks that could adversely affect the company's business - The company may never achieve sustained profitability and may need to raise additional capital in the future, which may not be available on acceptable terms, potentially forcing adjustments to operations or cessation[309](index=309&type=chunk)[311](index=311&type=chunk)[313](index=313&type=chunk) - The pendency or failure to complete the proposed sale of the **European ORLADEYO Business** to **Neopharmed** could disrupt operations, affect relationships, divert management resources, and impact future revenues and stock price[315](index=315&type=chunk)[318](index=318&type=chunk) - Success depends on managing the product candidate pipeline, advancing candidates through complex and uncertain clinical trials, and receiving/maintaining regulatory approvals, with risks including failure to demonstrate efficacy/safety, adverse events, and inadequate trial design[325](index=325&type=chunk)[326](index=326&type=chunk)[327](index=327&type=chunk) - Heavy reliance on **third parties** (development partners, **CROs**, manufacturers, distributors) for many stages of product development and commercialization poses risks of delays, failures, or inability to retain alternative providers[333](index=333&type=chunk)[334](index=334&type=chunk)[362](index=362&type=chunk) - The biotechnology and pharmaceutical industries are **highly competitive**, with many competitors possessing greater resources and experience, potentially leading to reduced demand for **BioCryst's** products or rendering its technologies obsolete[370](index=370&type=chunk)[371](index=371&type=chunk)[375](index=375&type=chunk) - The company is subject to extensive and evolving laws and regulations (e.g., **FDA**, healthcare fraud and abuse, pricing, data privacy), with non-compliance potentially leading to substantial penalties, reputational harm, and increased costs[376](index=376&type=chunk)[378](index=378&type=chunk)[389](index=389&type=chunk)[395](index=395&type=chunk) - Protecting and enforcing **intellectual property rights** is critical but uncertain, with risks of patent infringement, generic competition (e.g., **ORLADEYO ANDA** challenge), costly litigation, and potential loss of trade secrets[398](index=398&type=chunk)[400](index=400&type=chunk)[401](index=401&type=chunk) - Significant indebtedness under the **Pharmakon Loan Agreement** (**$248.7 million** outstanding as of **June 30, 2025**) increases vulnerability to adverse economic conditions, limits financial flexibility, and imposes restrictive covenants that could trigger early repayment or foreclosure on assets[420](index=420&type=chunk)[421](index=421&type=chunk)[422](index=422&type=chunk)[424](index=424&type=chunk) - International expansion exposes the company to business, legal, regulatory, political, operational, financial, and economic risks, including conflicting laws, difficulties in obtaining approvals, foreign currency fluctuations, and governmental pricing controls[425](index=425&type=chunk)[427](index=427&type=chunk)[428](index=428&type=chunk) - Dependence on information technology systems and increasing use of **artificial intelligence (AI)** present risks of cyber incidents, data breaches, operational outages, loss of intellectual property, and challenges in managing **AI** use, potentially leading to legal liabilities and reputational harm[441](index=441&type=chunk)[442](index=442&type=chunk)[446](index=446&type=chunk) - The company's stock price has been and is likely to remain **highly volatile** due to various factors, including clinical trial results, regulatory developments, competition, financial performance, and external economic conditions, which could cause the value of an investment to decline significantly[464](index=464&type=chunk)[465](index=465&type=chunk) [Item 5. Other Information](index=88&type=section&id=Item%205.%20Other%20Information) No Rule 10b5-1 or non-Rule 10b5-1 trading arrangements were adopted or terminated by directors or officers during Q2 2025 - None of the company's directors or officers adopted or terminated a "**Rule 10b5-1 trading arrangement**" or "**non-Rule 10b5-1 trading arrangement**" during the three months ended **June 30, 2025**[480](index=480&type=chunk) [Item 6. Exhibits](index=89&type=section&id=Item%206.%20Exhibits) This section lists various exhibits filed with the 10-Q report, including the Stock Purchase Agreement, corporate charter documents, employment agreements, stock incentive plans, and certifications - Key exhibits include the **Stock Purchase Agreement (2.1)**, **Third Restated Certificate of Incorporation (3.1-3.5)**, **Amended and Restated By-Laws (3.6)**, **Consulting Agreement (10.1)**, **Amendment No. 1 to Consulting Agreement (10.2)**, **Amended and Restated Employment Agreement (10.3)**, **Amendment No. 2 to Employment Agreement (10.4)**, **BioCryst Pharmaceuticals, Inc. Stock Incentive Plan (10.5)**, and various certifications (**31.1, 31.2, 32.1, 32.2**)[481](index=481&type=chunk)[482](index=482&type=chunk) SIGNATURES The report was signed by **Jon P. Stonehouse**, Chief Executive Officer, and **Babar Ghias**, Chief Financial Officer, on **August 5, 2025**[486](index=486&type=chunk)

Financial Data and Key Metrics Changes - Total revenue for Q2 2025 was $163.4 million, with $156.8 million coming from Orlodayo, representing a 45% year-over-year growth in quarterly revenue [22][23] - Non-GAAP operating expenses were $106.4 million, up from $87.4 million in 2024, while non-GAAP operating profit was $57 million and non-GAAP net income was $32.3 million, resulting in a non-GAAP EPS of $0.15 [23][24] - The company generated $45 million in cash during the quarter and paid down $75 million in principal from its term loan in April and an additional $50 million in July, reducing term debt to $199 million [24] Business Line Data and Key Metrics Changes - Orlodayo had its best quarter since approval, with new patient prescriptions up over 10% compared to Q1 2021 and over 15% compared to Q1 2025, leading to revenue exceeding expectations by over $22 million [15][16] - The increase in Orlodayo revenue was attributed to a spike in new patient demand, improved efficiency in paid shipments, lower discontinuation rates, and strong international results [15][16] Market Data and Key Metrics Changes - The U.S. accounted for almost 90% of Orlodayo revenue, with $140.3 million generated in the U.S. alone [22] - There was an uptick in new U.S. prescribers, increasing from 59 in Q1 to 69 in Q2, indicating growing confidence among physicians [18] Company Strategy and Development Direction - The company plans to leverage its commercial capabilities to become a consolidator of rare disease assets, aiming to bring multiple products to market [12][28] - A definitive agreement was signed to sell the European business to Neo Pharma, which is expected to strengthen the company's financial position and allow for debt repayment [11][28] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in reaching the upper half of the revenue guidance of $580 million to $600 million for the full year, even after removing European revenue post-sale [16] - The management highlighted the importance of real-world evidence in building physician confidence and driving long-term growth [18][20] Other Important Information - The company anticipates data from its Netherton Syndrome and DME pipeline programs by the end of the year [10][28] - The transition to new leadership was discussed, with Charlie Gayer set to become the next CEO, emphasizing the company's readiness for the next growth phase [12][13] Q&A Session Summary Question: What contributed to the 45% year-over-year growth in Orlodayo revenue? - The growth was attributed to a mix of volume increase, better paid rates, and improved efficiency in paid shipments [30][31] Question: What are the current discontinuation rates? - The one-year discontinuation rate remains at 60%, with a slight downward trend in overall discontinuation rates as the patient base grows [35][36] Question: How does Orlodayo's persistency compare to other injectable prophylactic regimens? - Orlodayo's one-year persistence rate is statistically similar to other products, with about 60% for all [40][41] Question: How are recent approvals affecting Orlodayo demand? - There has been no negative impact on Orlodayo demand from recent approvals, with new patient prescriptions at an all-time high [45][46] Question: What is the current penetration rate into the addressable market? - Approximately 3,000 patients have tried Orlodayo, with about half still on therapy, indicating significant growth potential [54][55] Question: What is the competitive landscape for the pipeline assets? - The company is optimistic about its pipeline programs, with expectations for accelerated approval pathways due to the unmet needs in the market [108][109]

Financial Data and Key Metrics Changes - Total revenue for Q2 2025 was $163.4 million, with $156.8 million coming from Orlodayo, representing a 45% year-over-year growth in quarterly revenue [20][21] - Non-GAAP operating expenses were $106.4 million, up from $87.4 million in 2024, while non-GAAP operating profit was $57 million, and non-GAAP net income was $32.3 million, resulting in a non-GAAP EPS of $0.15 [21][22] - The company generated $45 million in cash during the quarter and paid down $75 million in principal from its term loan in April and an additional $50 million in July, reducing term debt to $199 million [22] Business Line Data and Key Metrics Changes - Orlodayo had its best quarter since approval, with new patient prescriptions up over 10% compared to Q1 2021 and over 15% compared to Q1 2025, leading to revenue exceeding expectations by over $22 million [14][15] - The gross-to-net improvement was noted to be closer to 15%, with a significant increase in paid shipments contributing to revenue growth [20][98] Market Data and Key Metrics Changes - The U.S. accounted for almost 90% of Orlodayo revenue, with strong demand from Type I and II patients and increasing demand from patients with normal C1 inhibitor [20][18] - The company anticipates continued revenue growth momentum into 2026 and beyond, supported by the expected FDA approval of Orlodayo granules for pediatric use in December [18][19] Company Strategy and Development Direction - The company plans to leverage its commercial capabilities to consolidate rare disease assets and expand its product offerings, aiming for BioCryst 2.0 [10][11] - A definitive agreement was signed to sell the European business, which is expected to strengthen the company's financial position and allow for debt repayment [9][10] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in reaching the upper half of the revenue guidance of $580 million to $600 million for the full year, even after the anticipated sale of the European business [15][19] - The management highlighted the importance of real-world evidence in building physician confidence and driving long-term growth [16][18] Other Important Information - The company is on track to generate approximately $700 million in cash by 2027 and plans to actively deploy this capital into value-creating opportunities [22][25] - The transition of leadership to Charlie Guyer as the new CEO was announced, with a focus on executing the company's growth strategy [11][12] Q&A Session Summary Question: What contributed to the 45% year-over-year growth in Orlodayo revenue? - The growth was attributed to a mix of volume increase, better paid rates, and improved efficiency in shipments [28][30] Question: What are the current discontinuation rates? - The one-year discontinuation rate remains solid at 60%, with a slight downward trend in overall discontinuation rates as the patient base grows [32][33] Question: How does Orlodayo's persistency compare to other injectable prophylactic regimens? - Orlodayo's one-year persistence rate is statistically similar to other products, with about 60% for all three compared therapies [39][40] Question: How are recent approvals affecting Orlodayo demand? - There has been a substantial increase in new patient prescriptions, indicating that physicians are not waiting for new products [43][44] Question: What is the current penetration rate into the addressable market? - Approximately 3,000 patients have tried Orlodayo, with about half still on therapy, indicating significant growth potential [51][52] Question: What is the expected impact of the upcoming pediatric PDUFA delay? - The FDA required more time for review, leading to a new PDUFA date of December 12, but the company remains optimistic about approval [45][46] Question: What is the competitive landscape for the pipeline assets? - The company is optimistic about its position in the market, noting that competitors have not made significant progress recently [76][77] Question: How does the company plan to deploy capital moving forward? - The company aims to consolidate rare disease assets and balance its portfolio with later-stage assets, leveraging its strong cash flow [84][86]

Group 1 - BioCryst Pharmaceuticals reported quarterly earnings of $0.15 per share, exceeding the Zacks Consensus Estimate of $0.03 per share, compared to a loss of $0.06 per share a year ago, representing an earnings surprise of +400.00% [1] - The company posted revenues of $163.35 million for the quarter ended June 2025, surpassing the Zacks Consensus Estimate by 9.74%, and compared to year-ago revenues of $109.33 million [2] - BioCryst has surpassed consensus EPS estimates two times over the last four quarters and topped consensus revenue estimates four times during the same period [2] Group 2 - The stock's immediate price movement will depend on management's commentary during the earnings call, with BioCryst shares gaining about 5.7% since the beginning of the year, compared to the S&P 500's gain of 6.1% [3] - The current consensus EPS estimate for the coming quarter is $0.06 on revenues of $155.49 million, and $0.13 on revenues of $609.2 million for the current fiscal year [7] - The Zacks Industry Rank for Medical - Drugs is currently in the top 32% of over 250 Zacks industries, indicating that the industry outlook can significantly impact stock performance [8]

Financial Performance & Guidance - BioCryst's Q2 2025 was strong, driven by increased ORLADEYO demand[14] - ORLADEYO revenue is expected to meet the previous guidance range of $580-600 million for FY25, even excluding Q4 2025 EU revenue[16] - The company anticipates achieving full-year operating profit in 2026 and positive net income & cash flows in 2026+[58] - Cash, cash equivalents, restricted cash & investments totaled $287 million as of June 30, 2025[63] - The company paid down $50 million of its senior credit facility in July 2025[62] ORLADEYO & Market Dynamics - Market research indicates a growing patient preference for oral administration of HAE long-term prophylaxis (LTP) since 2023[17] - Monte Carlo simulation projects ORLADEYO reaching a steady state of over 2,000 patients in the US by 2028[25] - ORLADEYO revenue advances to royalty-free tier (>$550M)[61] - The blended royalty rate for ORLADEYO is expected to decline to approximately 4% at peak sales once OMERS reaches its cap[67] Pipeline Development - The PDUFA target date for the pediatric NDA for ORLADEYO is December 12, 2025[32] - An IND has been cleared by the FDA for BCX17725, a targeted KLK5 inhibitor for treating Netherton syndrome (NS)[37]