Core Insights - BioCryst Pharmaceuticals is focused on developing oral treatments for rare diseases, particularly with its drug ORLADEYO® targeting hereditary angioedema (HAE) [1] - H.C. Wainwright maintains a "Buy" rating for BioCryst, raising its price target from $30 to $32, reflecting confidence in the company's future performance [2] - The FDA's approval of ORLADEYO in an oral pellet formulation is a significant milestone, especially for children, as it addresses a critical need in managing HAE [3] Company Performance - BioCryst's stock is currently priced at $7.60, with a slight increase of 0.33% today, and has fluctuated between $7.57 and $7.92 [4] - The company's market capitalization is approximately $1.6 billion, with a trading volume of 1,923,583 shares today [4] Market Impact - The expanded approval of ORLADEYO is expected to enhance BioCryst's market presence by targeting a younger demographic, positioning the company to capture a larger share of the HAE market [5]

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The U.S. Food and Drug Administration has expanded the use of BioCryst Pharmaceuticals' drug to include an oral pellet formulation for preventing hereditary angioedema attacks in children aged 2 to un... ...

–ORLADEYO now first and only targeted oral prophylactic therapy for patients with HAE aged 2 and older–-Oral pellet formulation provides child-friendly method of administration- –Showed early and sustained reductions in monthly attack rates in APeX-P– RESEARCH TRIANGLE PARK, N.C., Dec. 12, 2025 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the U.S. Food and Drug Administration (FDA) has approved its New Drug Application (NDA) for the use of an oral pellet formulation ...

–ORLADEYO now first and only targeted oral prophylactic therapy for patients with HAE aged 2 and older–-Oral pellet formulation provides child-friendly method of administration- –Showed early and sustained reductions in monthly attack rates in APeX-P– RESEARCH TRIANGLE PARK, N.C., Dec. 12, 2025 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the U.S. Food and Drug Administration (FDA) has approved its New Drug Application (NDA) for the use of an oral pellet formulation ...

Core Viewpoint - BioCryst Pharmaceuticals has received early termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act for its proposed acquisition of Astria Therapeutics, indicating progress towards the merger [1][2]. Group 1: Merger Details - The termination of the waiting period satisfies one of the conditions for the merger, with BioCryst expecting the merger to close in the first quarter of 2026, pending other customary closing conditions [2]. - BioCryst has filed a registration statement with the SEC, which includes a preliminary proxy statement of Astria and a preliminary prospectus of BioCryst regarding the merger [5][6]. Group 2: Company Overview - BioCryst Pharmaceuticals is a global biotechnology company focused on developing and commercializing medicines for hereditary angioedema and other rare diseases, with a commitment to improving the lives of affected individuals [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 various rare diseases [3].

Core Insights - BioCryst is experiencing significant revenue growth from its product ORLADEYO, with current revenues between $590 million and $600 million, projected to peak at $1 billion [3] - The company has a strong patent protection for ORLADEYO extending to 2040, which supports its growth trajectory [3] - BioCryst has a pipeline that includes two clinical candidates: 17725 for Netherton syndrome and avoralstat for DME [3] - The company is in a favorable financial position, having proposed an acquisition of Astria [4]

Summary of BioCryst Pharmaceuticals Conference Call Company Overview - **Company**: BioCryst Pharmaceuticals (NasdaqGS:BCRX) - **Key Products**: ORLADEYO, Nevenabart - **Revenue**: ORLADEYO revenue projected between $590-$600 million, with a peak potential of $1 billion by 2029 [3][10] Core Points and Arguments ORLADEYO Performance - ORLADEYO is the only oral drug in the market for HAE, showing strong demand despite new competitors [6] - The company expects continued growth in Q4, maintaining revenue guidance of $590-$600 million for the year [6] - The peak sales guidance for ORLADEYO remains at $1 billion, now including pediatric sales and excluding EU sales [10] EU Divestiture - BioCryst divested its EU business for $250 million, which was previously break-even with $50 million in revenue [8] - The divestiture allows for a cleaner balance sheet and supports the acquisition of Astria [9] Astria Acquisition - The acquisition of Astria is expected to close in Q1 2026, enhancing BioCryst's portfolio with Nevenabart, an injectable therapy for HAE [14][40] - Nevenabart is positioned as a differentiated product with a favorable dosing schedule compared to existing therapies [17][28] Market Dynamics - The HAE market is evolving, with 80% of patients on prophylactic therapy, creating a competitive landscape for new entrants [12] - There are approximately 8,000 potential patients who have not yet tried ORLADEYO, indicating significant growth opportunities [11] Pediatric Opportunity - A PDUFA date for pediatric indications is set for December 12, with expectations for pricing to be equivalent to capsules [32] - The pediatric formulation will include four different granule presentations [32] Netherton Syndrome - BioCryst is developing a treatment for Netherton syndrome, with data updates expected in Q1 2026 [34] - The market opportunity is significant, with an identified patient population of 1,600, which is expected to grow as therapies become available [37] Financial Outlook - The company has lowered its expense guidance to $430-$440 million, indicating improved operational efficiency [39] - BioCryst anticipates continued profitability and cash flow positivity, with operating expenses expected to decrease post-Astria acquisition [40] Additional Important Points - The company is focused on maintaining a strong prescriber base and generating real-world evidence to support ORLADEYO's efficacy [13] - BioCryst's strategy includes leveraging existing infrastructure to minimize costs associated with launching Nevenabart [22] - The company is exploring business development opportunities that do not necessarily need to be blockbuster drugs but can still provide significant bottom-line benefits [41] This summary encapsulates the key insights from the BioCryst Pharmaceuticals conference call, highlighting the company's strategic direction, product performance, and market opportunities.

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]