Biocryst Pharmaceuticals Stock Price, News & Analysis

BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) is a global biotechnology company focused on developing and commercializing medicines for hereditary angioedema (HAE) and other rare diseases. According to the company’s public statements, its work is driven by a commitment to improving the lives of people living with these conditions. BioCryst has commercialized ORLADEYO® (berotralstat), described as the first oral, once-daily plasma kallikrein inhibitor for HAE prophylaxis, 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.

Core focus on hereditary angioedema (HAE)

Across multiple press releases and SEC filings, BioCryst highlights HAE as its primary therapeutic focus. HAE is characterized in company communications as a disease that can significantly impair daily living and carries psychosocial and physical burdens, particularly for pediatric patients and their caregivers. BioCryst’s strategy centers on prophylactic therapies that aim to prevent HAE attacks rather than treating them acutely.

ORLADEYO (berotralstat) is described as a plasma kallikrein inhibitor indicated in the United States for prophylaxis to prevent attacks of HAE in adults and pediatric patients 2 years and older. The company emphasizes that ORLADEYO is designed as an oral, once-daily therapy that works by decreasing the activity of plasma kallikrein. Company disclosures also note that ORLADEYO is not approved for treatment of acute HAE attacks and that additional doses or higher-than-prescribed doses are not recommended due to QT interval considerations.

Commercialized product: ORLADEYO (berotralstat)

BioCryst’s commercial portfolio is centered on ORLADEYO. The company describes two main oral formulations:

• Capsule formulation – an oral capsule approved in the U.S. for prophylaxis to prevent HAE attacks in adult and pediatric patients 12 years and older, with approval also noted in more than 45 countries around the world.
• Oral pellets / oral granules formulation – an oral pellet (or granule) formulation approved by the U.S. Food and Drug Administration for prophylactic therapy in pediatric patients with HAE aged 2 to <12 years. This child-focused formulation is described as sprinkle-like in appearance and can be poured directly into the mouth with water or milk, or sprinkled over soft, non-acidic food.

Company communications repeatedly describe ORLADEYO as the first and only targeted oral prophylactic therapy for patients with HAE aged 2 and older. BioCryst also notes that ORLADEYO has been prescribed to thousands of patients in the U.S. and that it has become an important option for families and clinicians seeking a non-injectable prophylactic treatment.

Rare disease pipeline and technology approach

Beyond its marketed product, BioCryst states that it 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. Earlier company descriptions and disclosures indicate that BioCryst’s research and development activities have historically focused on novel small-molecule drugs designed to block key enzymes involved in infectious and inflammatory diseases, using disciplines such as biology, computer modeling, and medicinal chemistry. Its therapeutic areas have included hereditary angioedema, influenza, filoviruses such as Ebola and Marburg, and oncology.

Recent news also references RAPIVAB® (peramivir injection) in the context of total revenue guidance, indicating that BioCryst’s revenue base includes ORLADEYO and RAPIVAB. However, the company’s narrative emphasizes HAE and rare disease therapeutics as the central strategic focus.

Strategic transactions and geographic footprint

BioCryst is incorporated in Delaware and lists its common stock on the Nasdaq Global Select Market under the symbol BCRX. SEC filings show that the company entered into a Stock Purchase Agreement to sell all of its equity interests in BioCryst Ireland Limited, which, together with its subsidiaries, held certain assets and rights related to the company’s European ORLADEYO business. A subsequent Form 8-K reports the completion of this transaction and related agreements, including an amended and restated IP license agreement, a supply agreement under which BioCryst will be the exclusive supplier of ORLADEYO products to BioCryst Ireland for use in the defined territory, a global brand and support agreement, a transition services agreement, and a trademark license agreement.

These arrangements indicate that BioCryst has restructured its European ORLADEYO operations while maintaining a role as supplier and licensor. The company also notes that it has filed applications for the pediatric oral pellets formulation with regulatory agencies such as the European Medicines Agency and the Japan Pharmaceutical and Medical Devices Agency, and that additional regulatory filings are planned in other global territories, including Canada.

Planned acquisition of Astria Therapeutics

BioCryst has disclosed an Agreement and Plan of Merger under which a wholly owned BioCryst subsidiary will merge with Astria Therapeutics, Inc., with Astria surviving as a wholly owned subsidiary of BioCryst. The merger consideration includes a mix of cash and BioCryst common stock, subject to specified conditions and limitations. Company press releases and filings state that the acquisition is intended to expand BioCryst’s impact for HAE patients and to bring in programs such as navenibart (STAR-0215), an investigational monoclonal antibody inhibitor of plasma kallikrein for HAE, and STAR-0310, an investigational OX40 antagonist for atopic dermatitis and potentially other indications.

The merger remains subject to customary closing conditions, including Astria stockholder approval, regulatory clearances, and other conditions detailed in the merger agreement and related SEC filings. BioCryst has also disclosed that early termination of the Hart-Scott-Rodino waiting period has been obtained, satisfying one of the conditions for consummation of the merger.

Financial and operating outlook (structural, not numeric)

BioCryst regularly provides non-GAAP financial measures and guidance in its public communications. The company explains that these non-GAAP measures adjust for items such as stock-based compensation, restructuring, and transaction-related costs, and that management believes these measures help investors understand underlying performance and compare results across periods. Recent guidance has focused on ORLADEYO net revenue, total revenue including RAPIVAB, and non-GAAP operating expenses, as well as expectations for continued non-GAAP profitability.

In addition, BioCryst has communicated that the sale of its European ORLADEYO business and the planned acquisition of Astria Therapeutics are expected to influence its financial profile, including operating expenses associated with advancing navenibart through Phase 3 clinical development and commercial readiness manufacturing activities.

Corporate governance and leadership updates

Recent Form 8-K filings describe several leadership changes. These include the planned retirement of the company’s long-serving President and Chief Executive Officer and the appointment of a new President and future Chief Executive Officer, as well as the appointment of a new Chief Financial Officer who also serves as Principal Accounting Officer and Head of Corporate Development. The filings outline related compensation arrangements, equity inducement grants, and board of directors changes, such as increasing the size of the board and electing the incoming CEO as a director.

BioCryst has also reported inducement grants of stock options and restricted stock units to newly hired employees under its Inducement Equity Incentive Plan, in accordance with Nasdaq Listing Rule 5635(c)(4). These grants typically vest in equal annual installments over four years, subject to continued service.

Regulatory milestones and clinical data

BioCryst’s SEC filings and press releases highlight key regulatory and clinical milestones for ORLADEYO, particularly in pediatric HAE. An 8-K dated December 12, 2025, reports FDA approval of the New Drug Application for the oral pellet formulation of once-daily ORLADEYO for prophylactic therapy in pediatric patients aged 2 to <12 years. The company has also presented interim data from the APeX-P clinical trial, described as the largest pediatric HAE prophylaxis trial to date in this age group, showing early and sustained reductions in monthly HAE attack rates and high continuation rates over one year of treatment.

In parallel, BioCryst has sponsored psychosocial research on the burden of HAE on children and caregivers, reporting findings that HAE attacks often begin early in life and can negatively affect mental health, school participation, and family routines. These data are used by the company to frame the need for child-friendly, long-term prophylactic options.

Risk disclosures and forward-looking statements

BioCryst’s public communications include extensive forward-looking statement disclaimers. The company identifies risks related to commercialization of ORLADEYO, progression of its development pipeline, regulatory decisions, clinical trial enrollment and outcomes, market acceptance of its products, integration of acquisitions such as Astria, indebtedness associated with transactions, and broader economic and regulatory conditions. Investors are directed to the company’s Annual Reports on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K for detailed risk factor discussions.

FAQs about BioCryst Pharmaceuticals (BCRX)

• What does BioCryst Pharmaceuticals do?
  BioCryst Pharmaceuticals is a global biotechnology company focused on developing and commercializing medicines for hereditary angioedema (HAE) and other rare diseases. Its primary commercial product is ORLADEYO (berotralstat), an oral, once-daily plasma kallikrein inhibitor for prophylaxis to prevent HAE attacks.
• What is ORLADEYO and who is it for?
  ORLADEYO (berotralstat) is a plasma kallikrein inhibitor indicated in the U.S. for prophylaxis to prevent attacks of hereditary angioedema in adults and pediatric patients 2 years and older. It is available as an oral capsule for patients 12 years and older and as an oral pellet formulation for patients aged 2 to <12 years.
• Is ORLADEYO used to treat acute HAE attacks?
  No. Company prescribing information and safety communications state that ORLADEYO is indicated for prophylaxis to prevent HAE attacks and should not be used for the treatment of acute HAE attacks. Additional doses or higher-than-prescribed doses are not recommended because of QT interval considerations.
• How is BioCryst expanding its HAE and rare disease portfolio?
  BioCryst reports that it is advancing a pipeline of potential first-in-class or best-in-class oral small-molecule and injectable protein therapeutics for rare diseases. Through a planned acquisition of Astria Therapeutics, it expects to add programs such as navenibart (an investigational monoclonal antibody inhibitor of plasma kallikrein for HAE) and STAR-0310 (an investigational OX40 antagonist for atopic dermatitis).
• What role does BioCryst play in Europe after selling its European ORLADEYO business?
  Following the sale of its equity interests in BioCryst Ireland, which held certain assets and rights related to the European ORLADEYO business, BioCryst entered into an amended and restated IP license agreement, a supply agreement under which it is the exclusive supplier of ORLADEYO products to BioCryst Ireland for defined uses in the territory, a global brand and support agreement, a transition services agreement, and a trademark license agreement.
• On which exchange does BioCryst trade and under what ticker?
  BioCryst Pharmaceuticals, Inc. lists its common stock on the Nasdaq Global Select Market under the trading symbol BCRX, as disclosed in its SEC filings.
• How does BioCryst describe its use of non-GAAP financial measures?
  The company states that it provides non-GAAP measures, such as non-GAAP operating expenses and ORLADEYO revenue excluding certain items, to help management and investors understand financial performance without specific non-cash items and special events. These measures are presented as supplements to, not substitutes for, GAAP measures.
• What are some key risks BioCryst highlights in its public filings?
  BioCryst cites risks related to commercialization of ORLADEYO, clinical and regulatory outcomes for its pipeline, government pricing and reimbursement decisions, the success of business development activities such as the Astria acquisition, indebtedness associated with transactions, and general economic and market conditions. Detailed risk factors are provided in its Form 10-K, Form 10-Q, and Form 8-K filings.
• How does BioCryst address the burden of HAE on pediatric patients and caregivers?
  In presentations and press releases, BioCryst reports psychosocial research showing that HAE attacks often begin early in childhood and can negatively affect mental health, schooling, and family routines. The company positions its pediatric ORLADEYO formulations as addressing the need for child-friendly, long-term prophylactic options.
• What is the status of BioCryst’s acquisition of Astria Therapeutics?
  BioCryst has entered into a merger agreement under which a BioCryst subsidiary will merge with Astria Therapeutics, with Astria becoming a wholly owned subsidiary. Early termination of the Hart-Scott-Rodino waiting period has been obtained, and the transaction remains subject to Astria stockholder approval and other customary closing conditions described in the merger agreement and related SEC filings.