Core Insights - BioCryst Pharmaceuticals is under review by the U.S. FDA for the oral granule formulation of ORLADEYO for pediatric patients aged 2 to <12 years, with a decision expected by December 12, 2025, which would make it the first targeted oral prophylactic therapy for this age group [1][2][4] Clinical Trial Data - The APeX-P clinical trial has shown early and sustained reductions in monthly attack rates for pediatric patients with hereditary angioedema (HAE) treated with ORLADEYO over one year [1][6] - At month 1, 65.5% of patients were attack-free, increasing to 70.4% by month 12, with a median attack rate of 0 for 11 out of 12 months [7] - The trial demonstrated high continuation rates, with 93.1% of patients completing at least 48 weeks of treatment [7] Psychosocial Impact - HAE attacks have a significant negative psychosocial impact on pediatric patients and their caregivers, affecting mental health and quality of life [4][11] - The majority of young patients experience symptoms before age 6, with a median age at diagnosis of 2 years [7][13] - Caregivers report that HAE attacks occur approximately every 3 months and are rated as moderately severe [13] Emergency Department Experiences - Over three-quarters of adolescents and nearly half of caregivers reported at least one emergency department (ED) or hospital visit before age 12, often characterized by treatment delays and heightened stress [23] - HCPs express concern about inadequate care and the long-term mental health impacts of these ED visits on young patients [23] Safety and Tolerability - ORLADEYO was found to be safe and well-tolerated, with no new safety signals identified during the trial [7][8] - The most commonly reported treatment-emergent adverse event was nasopharyngitis [7]

Company Overview - BioCryst Pharmaceuticals, Inc. is a global biotechnology company focused on developing and commercializing medicines for hereditary angioedema (HAE) and other rare diseases [3] - The company has commercialized ORLADEYO® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of potential first-in-class or best-in-class oral small-molecule and injectable protein therapeutics for a range of rare diseases [3] Stock Compensation - The compensation committee of BioCryst's board of directors granted three newly-hired employees restricted stock units (RSUs) covering an aggregate of 30,700 shares of BioCryst common stock as inducements for employment [1] - The RSUs were granted as of November 3, 2025, and will vest in four equal annual installments beginning on the one-year anniversary of the grant date, subject to the new employee's continued service with the company [2] - The RSUs are subject to the terms and conditions of BioCryst's Inducement Equity Incentive Plan and a restricted stock unit agreement covering the grant [2]

Financial Performance - Revenues for Q3 2025 reached $159.4 million, a 36% increase from $117.1 million in Q3 2024[21] - Net income for the nine months ended September 30, 2025, was $18.0 million, compared to a net loss of $62.1 million for the same period in 2024[21] - For the three months ended September 30, 2025, total revenues were $159.4 million, a 36% increase from $117.1 million in the same period of 2024[40] - Product sales, net for the nine months ended September 30, 2025, reached $464.0 million, compared to $313.3 million for the same period in 2024, reflecting a 48% increase[40] - Revenues for the nine months ended September 30, 2025, were $468,282,000, a 46.8% increase from $319,178,000 in the same period of 2024[184] - Segment net income for the three months ended September 30, 2025, was $12,899,000, compared to a net loss of $14,033,000 in the same period of 2024[184] - For the three months ended September 30, 2025, the company reported a net income of $12,899,000 compared to a net loss of $14,033,000 for the same period in 2024, resulting in a basic net income per share of $0.06[188] - For the nine months ended September 30, 2025, the company achieved a net income of $18,016,000, a significant improvement from a net loss of $62,086,000 in the same period of 2024, with a basic net income per share of $0.09[188] Assets and Liabilities - Total current assets decreased to $355.7 million as of September 30, 2025, down from $422.0 million at December 31, 2024, representing a 15.8% decline[20] - Total liabilities decreased to $834.3 million as of September 30, 2025, from $966.4 million at December 31, 2024, a reduction of 13.6%[20] - Cash and cash equivalents at the end of Q3 2025 were $84.1 million, down from $104.7 million at the end of 2024, a decrease of 19.8%[20] - Total stockholders' deficit improved to $(387.9) million as of September 30, 2025, from $(475.9) million at December 31, 2024[20] - As of September 30, 2025, total assets held for sale amounted to $33,231,000, while total liabilities held for sale were $26,859,000[112] - The Company’s inventory as of September 30, 2025, was valued at $30,283,000, down from $33,923,000 as of December 31, 2024[123] - Receivables related to sales of ORLADEYO were $88,034,000 as of September 30, 2025, compared to $76,282,000 at the end of 2024[120] - The Company’s total investments were valued at $168,464,000 as of September 30, 2025, down from $236,460,000 at the end of 2024[119] Expenses - Research and development expenses for Q3 2025 were $44.6 million, slightly up from $41.1 million in Q3 2024[21] - Selling, general and administrative expenses increased to $83.0 million in Q3 2025, compared to $65.1 million in Q3 2024, a rise of 27.5%[21] - Stock-based compensation expense for the three months ended September 30, 2025, was $18.6 million, compared to $21.3 million for the same period in 2024[28] - For the nine months ended September 30, 2025, total stock-based compensation costs amounted to $61,316, compared to $44,074 for the same period in 2024, reflecting an increase of approximately 39%[163] - The Company recorded operating lease expense of $1,596 for the nine months ended September 30, 2025, down from $1,741 in the same period of 2024, indicating a decrease of about 8.3%[156] - The Company incurred a total finance lease expense of $1,907 for the nine months ended September 30, 2025, compared to $1,509 for the same period in 2024, marking an increase of approximately 26.4%[156] Revenue Sources - The primary source of product sales is ORLADEYO, which began shipping in December 2020, along with peramivir sales to licensing partners and the U.S. Department of Health and Human Services[42] - Revenue from ORLADEYO accounted for approximately 88% of total net revenues for the three months ended September 30, 2025[97] - ORLADEYO revenues in the U.S. reached $141,557,000 for the three months ended September 30, 2025, up 37% from $103,112,000 in 2024[113] - The Company recognized collaborative and other revenues of $1.7 million for the three months ended September 30, 2025, compared to $1.0 million in the same period of 2024[40] - The Company estimates rebates to third-party payors based on contracts and government-mandated discounts, which reduces total gross product revenues[45] - The Company recognizes revenue from collaborative agreements when the earnings process is complete, including license fees and royalty payments[50] Strategic Initiatives - The company has built a robust commercial infrastructure to support the launch and continued success of ORLADEYO®, an oral therapy for hereditary angioedema[31] - BioCryst Pharmaceuticals is focused on developing first-in-class or best-in-class therapeutics targeting rare diseases, leveraging its existing commercial capabilities[31] - The company expects its financial resources available at September 30, 2025, to be sufficient to fund operations for at least the next 12 months[33] - The company anticipates that the global commercial market for ORLADEYO could reach a peak of $1 billion in annual net revenues, based on market research and commercialization experience[205] - The company plans to seek a strategic partner for the development of avoralstat beyond phase 1[216] Financing and Debt - The carrying value of royalty financing obligations as of September 30, 2025, was approximately $476,793,000[136] - The effective interest rate for the 2020 RPI Royalty Agreement was 21.6% as of September 30, 2025[136] - The Company entered into a $450,000 Pharmakon Loan Agreement with an effective interest rate of 12.28% for the three months ended September 30, 2025[138][142] - The Company recorded cash paid for interest on royalty financing obligations of $18,319,000 for the three months ended September 30, 2025[136] - As of September 30, 2025, total borrowings under the Pharmakon Loan Agreement, including PIK interest payments, amounted to $198,704, with interest expense for the three and nine months ended September 30, 2025, recorded at $6,110 and $22,789, respectively[150] - The Company made a $75,000 partial prepayment on the Pharmakon Term Loan on April 18, 2025, incurring a total one-time loss of $4,171 due to prepayment premium and unamortized deferred financing costs[147] Market Risks and Challenges - The Company relies on a single specialty pharmacy for distribution of ORLADEYO, which poses a risk to its business operations[99] - The Company’s receivables from ORLADEYO sales are primarily due from one customer, resulting in a concentration of credit risk[102] - Revenue from sales of ORLADEYO for the three and nine months ended September 30, 2025, is subject to uncertainties and depends on factors such as commercialization efforts and patient demand[206] - The effectiveness of commercialization efforts and market acceptance of products, particularly ORLADEYO, are critical for revenue predictions[211] - Operating expenses are primarily driven by research and development activities, which are difficult to predict[212] - Period-to-period comparisons of operating results may not be meaningful due to various uncertainties affecting revenue and expenses[213] Legal and Regulatory Matters - The company is involved in ongoing litigation regarding patent infringement related to ORLADEYO, with a lawsuit filed against Annora Pharma and others[187] - The FDA granted Fast Track designation for BCX17725 for the treatment of Netherton syndrome on July 30, 2025, with initial data expected by the end of Q1 2026[215] - The company has a new drug application for an oral granule formulation of ORLADEYO for pediatric patients aged 2 to 11, with a goal date of December 12, 2025[204]

Financial Data and Key Metrics Changes - Total ORLADEYO revenue was $159.1 million, representing a 37% year-over-year growth, with $141.6 million (89%) coming from the US [19][20] - Non-GAAP operating profit for Q3 2025 was $51.7 million, an increase of 107% year-over-year [20] - Non-GAAP net income for the quarter was $35.6 million, resulting in non-GAAP EPS of $0.17 per share [20] - The company finished the quarter with $269 million in cash, with a pro forma cash balance of approximately $294 million and zero term debt [21] Business Line Data and Key Metrics Changes - ORLADEYO continues to show strong growth, with new prescriptions exceeding the total from the same quarter last year [9][10] - The company raised its ORLADEYO revenue guidance to between $590 million and $600 million for the year [7] - The paid patient rate for ORLADEYO was 82%, consistent with typical second-half patterns [9] Market Data and Key Metrics Changes - The company added 64 new prescribers in the US, exceeding the average of the past eight quarters [9] - The market simulation predicts $1 billion in peak revenue for BioCryst in 2029, despite the sale of the European business [10] Company Strategy and Development Direction - The company plans to spin out or partner the DME program to focus on rare diseases [5] - The acquisition of Astria Therapeutics is expected to strengthen the company's presence in HAE and solidify a double-digit growth trajectory for its portfolio [18] - The company aims to leverage its expertise in HAE with the addition of Nevenobart, anticipating double-digit revenue growth well into the 2030s [10] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in maintaining patient retention rates despite new competition, citing strong control and satisfaction among patients [27][28] - The company expects to remain profitable on a non-GAAP basis and cash flow positive during the development period of Nevenobart [23] Other Important Information - The company successfully closed the sale of its European business, which allowed for the repayment of Pharmakon debt and improved financial positioning [18] - The company is exploring various capital allocation opportunities to generate value for stockholders [21] Q&A Session Summary Question: What contributed to the 37% year-over-year revenue growth? - The growth was driven by steady volume growth and an improvement in paid rates, particularly in the Medicare segment [26][27] Question: What factors are driving new prescriber numbers? - Physicians are becoming more comfortable with ORLADEYO's long-term evidence and real-world effectiveness [33][34] Question: How does the company view the impact of new entrants in the market? - While some patients may switch to injectables, the company does not expect a significant change in new patient prescribing patterns or overall retention rates [42] Question: What is the strategy for pediatric ORLADEYO approval? - The company is already targeting physicians who treat pediatric patients and is prepared for a launch shortly after approval [43] Question: What is the status of the Netherton syndrome program? - Enrollment is slightly delayed, but the company is optimistic about the ongoing studies and the potential for higher dosing [51][52]

ORLADEYO Performance and Outlook - ORLADEYO revenue continues strong growth, with a 37% year-over-year increase in Q3 2025[15] - The FY25 guidance range for ORLADEYO revenue has been raised to $590-600 million[17] - Market research reaffirms a strong outlook for ORLADEYO, with an estimated patient LTP share of 24% in 2034[19] - BioCryst anticipates a sustainable $1 billion peak revenue opportunity for ORLADEYO, with a contribution margin exceeding 80%[14] Astria Acquisition and HAE Portfolio Expansion - BioCryst is set to acquire Astria for approximately $700 million TEV, aiming to strengthen its presence in the HAE market[30] - The acquisition is expected to transform BioCryst's revenue profile, projecting a 10+ year double-digit portfolio CAGR[30] - Post-transaction, BioCryst anticipates maintaining profitability (non-GAAP) and positive cash flow[31] - By 2029, the company expects a cash balance of over $1 billion, providing flexibility for future growth opportunities[32] Pipeline Development - Navenibart presents an opportunity to target 5,000+ patients in the US[34] - BCX17725, a targeted KLK5 inhibitor, is under development for Netherton syndrome, with initial Phase 1 data expected by the end of Q1 2026[59]

Core Insights - BioCryst Pharmaceuticals reported a strong Q3 2025 performance, with ORLADEYO net revenue reaching $159.1 million, a 37% increase year-over-year [1][6][8] - The company raised its FY 2025 revenue guidance for ORLADEYO to between $590 million and $600 million, while lowering non-GAAP operating expense guidance to between $430 million and $440 million [1][17] - BioCryst completed the sale of its European ORLADEYO business, using the proceeds to retire all remaining Pharmakon term debt [1][15] - A definitive agreement was signed to acquire Astria Therapeutics, expected to close in Q1 2026, which will enhance BioCryst's HAE portfolio [1][15] Financial Performance - Total revenues for Q3 2025 were $159.4 million, up 36% from $117.1 million in Q3 2024 [8] - Operating profit for Q3 2025 was $29.6 million, a significant increase of 285% year-over-year, with non-GAAP operating profit at $51.7 million, up 107% [1][11] - Net income for Q3 2025 was $12.9 million, or $0.06 per share, compared to a net loss of $14.0 million, or $0.07 per share, in Q3 2024 [13][27] Market Dynamics - ORLADEYO demand remained strong, with new patient prescriptions slightly exceeding those in Q3 2024 despite new competitors entering the market [4][6] - The U.S. market contributed 89% of global ORLADEYO net revenues in Q3 2025, indicating strong domestic performance [6] Strategic Moves - The sale of the European ORLADEYO business has strengthened the company's financial position and allowed for debt repayment [2][15] - The acquisition of Astria Therapeutics is anticipated to provide opportunities for sustainable growth and profitability in the HAE market [2][15] Research and Development - R&D expenses for Q3 2025 increased to $44.6 million, primarily due to advancements in clinical programs [9] - The company is progressing with clinical trials for BCX17725 and avoralstat, with initial data expected by early 2026 [15]

Core Insights - BioCryst Pharmaceuticals will present four abstracts at the ACAAI Annual Scientific Meeting, focusing on the APeX-P trial results for ORLADEYO in pediatric patients with hereditary angioedema [1][2] Group 1: Presentation Details - The abstracts will include a second interim analysis of the APeX-P trial evaluating long-term prophylaxis with ORLADEYO in children aged 2 to <12 years [1] - Additional findings will address the psychosocial impact of hereditary angioedema on young patients and their caregivers [1] Group 2: Product Information - ORLADEYO (berotralstat) is the first oral therapy specifically designed to prevent hereditary angioedema attacks in patients aged 12 years and older [4] - The drug functions as a plasma kallikrein inhibitor, with a recommended dosage of one capsule per day [4][5] Group 3: Safety and Efficacy - The safety and effectiveness of ORLADEYO for treating acute HAE attacks have not been established, and it should not be used for this purpose [6][10] - Common adverse reactions include abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease [7]

Core Viewpoint - BioCryst Pharmaceuticals will report its third quarter 2025 financial results on November 3, 2025, and will host a conference call to discuss these results and provide a corporate update [1]. Group 1: Financial Reporting - The third quarter 2025 financial results will be announced on November 3, 2025 [1]. - A conference call and webcast will take place at 8:30 a.m. ET on the same day to discuss the financial results [1]. Group 2: Access Information - Domestic callers can access the live call by dialing 1-844-481-2942, while international callers can dial 1-412-317-1866 [2]. - A live webcast and replay of the call will be available on the investors section of the company website [2]. Group 3: Company Overview - BioCryst Pharmaceuticals is a global biotechnology company focused on improving the lives of individuals with hereditary angioedema and other rare diseases [3]. - The company specializes in structure-guided drug design to develop first-in-class or best-in-class oral small-molecule and protein therapeutics [3]. - BioCryst has commercialized ORLADEYO (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of therapies [3].

Core Insights - BioCryst Pharmaceuticals (BCRX) shares increased by 6.1% to $6.81, following a significant trading volume, contrasting with a 17.2% decline over the past month [1][2] Company Developments - The stock surge is attributed to a $700 million acquisition agreement for Astria Therapeutics, which will enhance BCRX's pipeline with navenibart, a long-acting injectable therapy for hereditary angioedema (HAE) [2] - The acquisition is expected to drive double-digit revenue growth, profit accretion, and rapid market adoption, indicating a strategic fit with BCRX's existing commercialization platform [2] Financial Performance Expectations - BioCryst is projected to report quarterly earnings of $0.06 per share, reflecting a year-over-year increase of 185.7%, with revenues anticipated at $161.15 million, a 37.6% rise from the previous year [3] - However, the consensus EPS estimate has been revised down by 4.8% over the last month, which may impact future stock price movements [4] Industry Context - BioCryst is categorized under the Zacks Medical - Drugs industry, where Ironwood Pharmaceuticals (IRWD) also operates, having seen an 8.8% increase in its stock price recently [4] - Ironwood's EPS estimate remains unchanged at $0.09, indicating a significant year-over-year change of 350% [5]

Core Viewpoint - BioCryst Pharmaceuticals is acquiring Astria Therapeutics for an implied $13 per share, resulting in an enterprise value of approximately $700 million, with the transaction expected to close in Q1 2026 [1][2]. Group 1: Acquisition Details - The acquisition will be financed through a combination of BioCryst's available cash and a strategic financing facility of up to $550 million from Blackstone-managed funds [3][4]. - Astria's CEO, Jill Milne, will join BioCryst's board of directors upon completion of the acquisition [1]. Group 2: Strategic Benefits - The acquisition is aimed at expanding BioCryst's portfolio with a late-stage product candidate, navenibart, which is in Phase III clinical development for hereditary angioedema (HAE) [2][5]. - Navenibart is expected to offer advantages over current treatments due to its extended administration schedule of every three to six months [2]. Group 3: Future Prospects - BioCryst's existing commercialization infrastructure and expertise in HAE will enhance the distribution of navenibart [3]. - The company is also acquiring Astria's early-stage atopic dermatitis program, STAR-0310, and is exploring strategic options for this asset [3].