Full-Time
Develops oral drugs for rare diseases
No salary listed
Remote in USA
Remote
Travel up to 20–30% domestically.
Bachelor's, Master's
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BioCryst Pharmaceuticals develops oral small-molecule drugs aimed at treating rare diseases, with a focus on hereditary angioedema (HAE). The company uses traditional cell biology and medicinal chemistry to design synthetic compounds that target specific protein pathways involved in disease. Its products work as oral therapies that modulate disease-related proteins to reduce symptoms and improve daily functioning, progressing from discovery through clinical trials toward commercialization. BioCryst differentiates itself by concentrating on rare diseases and pursuing oral, non-biologic treatments, leveraging a combined science approach and a patient-focused strategy to bring therapies to market. Its goal is to deliver meaningful, clinically validated treatments that enable patients to engage more fully in everyday activities and improve their quality of life, supported by its drug pipeline and potential licensing opportunities.
Company Size
501-1,000
Company Stage
IPO
Headquarters
Durham, North Carolina
Founded
1986
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BioCryst Pharmaceuticals is leveraging profits from its hereditary angioedema drug Orladeyo to acquire rare disease treatments. The company expects up to $645 million in 2026 sales from Orladeyo, which has generated over $2 billion since its 2020 FDA approval. CEO Charlie Gayer said BioCryst became profitable last year and aims to maintain profitability whilst expanding beyond being a single-product company. The firm is now seeking external acquisitions rather than internal development, targeting assets with peak sales around $300 million. This shift reflects a broader trend where small and midsize biotech companies are stepping into rare disease drug development as large pharmaceutical firms increasingly focus on multibillion-dollar assets. BioCryst's shares have risen approximately 25% since the start of the year.
BioCryst Announces Approval of ORLADEYO for Pediatric HAE Patients. Wednesday, August 26, 2026 BioCryst Pharmaceuticals has announced that Japan's Ministry of Health, Labour and Welfare (MHLW) has approved ORLADEYO(R)(berotralstat) for the prevention of hereditary angioedema (HAE) attacks in children aged 2 to under 12 years. The once-daily oral treatment is approved as a prophylactic therapy for paediatric patients with HAE caused by C1-inhibitor deficiency. The approval provides children in Japan with an oral option for long-term prevention of HAE attacks. HAE is a rare genetic condition that can cause sudden and potentially serious swelling in different parts of the body. Traditional preventive treatment for children has often involved injections, which can add to the treatment burden for patients and caregivers. ORLADEYO is available in a granule formulation for young children. The formulation can be taken directly with water or milk, or mixed with a spoonful of soft, non-acidic food, offering a simpler method of administration. The Japanese approval is supported by interim results from the APeX-P clinical trial, which is evaluating the pharmacokinetics, safety and efficacy of ORLADEYO in children aged 2 to under 12 years with HAE due to C1-inhibitor deficiency. The study is assessing long-term prophylaxis in paediatric patients. Interim findings showed that ORLADEYO was generally well tolerated and had a consistent safety profile across the age group. The treatment also produced early and sustained reductions in monthly HAE attack rates, with no new safety concerns identified. Nasopharyngitis was the most commonly reported treatment-emergent adverse event. BioCryst plans to launch ORLADEYO for children in Japan after the completion of the country's National Health Insurance reimbursement pricing process. OrphanPacific serves as BioCryst's partner in Japan and holds the marketing authorisation for the product. ORLADEYO is a once-daily oral plasma kallikrein inhibitor designed to prevent HAE attacks. It is approved for use in more than 45 countries. The capsule formulation was first approved in Japan in January 2021 for adults and children aged 12 years and older. In December 2025, the US Food and Drug Administration approved the oral pellet formulation of ORLADEYO for the prevention of HAE attacks in children aged 2 to under 12 years. BioCryst has also submitted applications for the paediatric indication to the European Medicines Agency and Health Canada, with further regulatory submissions planned in other markets. The latest approval marks the second regulatory approval for ORLADEYO in Japan and expands access to oral preventive treatment for younger children living with HAE.
BioCryst Pharmaceuticals has received approval from Japan's Ministry of Health, Labor and Welfare for ORLADEYO (berotralstat) as a prophylactic therapy for pediatric patients with hereditary angioedema (HAE) aged 2 to under 12 years. ORLADEYO is the first and only oral prophylactic therapy for HAE patients aged 2 and above. The granule formulation can be taken directly with water or milk, or sprinkled over soft food, reducing the treatment burden compared to traditional injections. The approval is based on interim results from the APeX-P clinical trial, the largest trial of long-term prophylaxis in pediatric HAE patients. Results showed the therapy was generally well tolerated with sustained reductions in monthly attack rates. BioCryst will launch ORLADEYO for children in Japan following completion of the National Health Insurance System reimbursement pricing process. OrphanPacific holds the marketing authorisation in Japan.
Research analysts issue forecasts for BCRX Q4 earnings. August 14, 2026 Key points. * HC Wainwright raised its Q4 2026 EPS forecast for BioCryst Pharmaceuticals to $0.18 from $0.14, maintaining a "Buy" rating and $32 price target. * Analyst sentiment remains favorable, with eight Buy ratings and four Holds; the stock has a "Moderate Buy" consensus rating and an average price target of $20.60. * BioCryst exceeded its latest quarterly expectations, reporting EPS of $0.30 versus $0.15 expected and revenue of $218.25 million, up 33.6% year over year. * Five stocks we like better than BioCryst Pharmaceuticals. BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX - Free Report) - Investment analysts at HC Wainwright boosted their Q4 2026 earnings per share (EPS) estimates for BioCryst Pharmaceuticals in a note issued to investors on Thursday, August 6th. HC Wainwright analyst A. Fein now forecasts that the biotechnology company will earn $0.18 per share for the quarter, up from their previous forecast of $0.14. HC Wainwright currently has a "Buy" rating and a $32.00 target price on the stock. The consensus estimate for BioCryst Pharmaceuticals' current full-year earnings is ($2.44) per share. Other research analysts have also issued reports about the stock. Wall Street Zen raised shares of BioCryst Pharmaceuticals from a "hold" rating to a "strong-buy" rating in a research report on Saturday, August 8th. Needham & Company LLC cut their target price on BioCryst Pharmaceuticals from $18.00 to $16.00 and set a "buy" rating on the stock in a research note on Wednesday, May 6th. Weiss Ratings reaffirmed a "hold (c)" rating on shares of BioCryst Pharmaceuticals in a report on Monday, June 1st. JPMorgan Chase & Co. assumed coverage on BioCryst Pharmaceuticals in a report on Monday, July 13th. They issued a "neutral" rating and a $12.00 target price for the company. Finally, Cantor Fitzgerald increased their price target on BioCryst Pharmaceuticals from $27.00 to $29.00 and gave the company an "overweight" rating in a research report on Tuesday, June 30th. Eight analysts have rated the stock with a Buy rating and four have assigned a Hold rating to the company's stock. Based on data from MarketBeat, the company currently has an average rating of "Moderate Buy" and an average price target of $20.60. BioCryst Pharmaceuticals price performance. BCRX opened at $9.81 on Friday. BioCryst Pharmaceuticals has a 1 year low of $6.00 and a 1 year high of $11.22. The company has a market capitalization of $2.51 billion, a price-to-earnings ratio of -6.54 and a beta of 0.55. The firm's fifty day moving average price is $9.47 and its two-hundred day moving average price is $8.82. BioCryst Pharmaceuticals (NASDAQ:BCRX - Get Free Report) last announced its earnings results on Wednesday, August 5th. The biotechnology company reported $0.30 earnings per share (EPS) for the quarter, beating the consensus estimate of $0.15 by $0.15. The company had revenue of $218.25 million for the quarter, compared to analysts' expectations of $183.28 million. BioCryst Pharmaceuticals had a negative return on equity of 86.89% and a negative net margin of 40.90%.BioCryst Pharmaceuticals's quarterly revenue was up 33.6% on a year-over-year basis. During the same quarter in the previous year, the firm earned $0.15 earnings per share. Institutional inflows and outflows. Hedge funds and other institutional investors have recently added to or reduced their stakes in the stock. Visionary Wealth Advisors purchased a new stake in shares of BioCryst Pharmaceuticals during the 2nd quarter worth approximately $169,000. Sanctuary Advisors LLC bought a new position in BioCryst Pharmaceuticals during the second quarter valued at $587,000. Elevation Point Wealth Partners LLC purchased a new stake in BioCryst Pharmaceuticals in the second quarter worth $126,000. Commerce Bank bought a new stake in shares of BioCryst Pharmaceuticals in the second quarter valued at $383,000. Finally, Northwestern Mutual Wealth Management Co. purchased a new position in shares of BioCryst Pharmaceuticals during the 2nd quarter valued at $103,000. 85.88% of the stock is currently owned by hedge funds and other institutional investors. BioCryst Pharmaceuticals company profile. BioCryst Pharmaceuticals, Inc is a clinical-stage biotechnology company headquartered in Durham, North Carolina, that focuses on the discovery and development of novel, oral small-molecule medicines for rare and serious diseases. Since its founding in 1986, the company has leveraged structure-based drug design to advance a pipeline of targeted therapeutics designed to address underlying disease mechanisms rather than just treat symptoms. The company's first commercial product, Orladeyo (berotralstat), is an oral kallikrein inhibitor approved for the prophylactic treatment of hereditary angioedema (HAE) in both the United States and Europe. This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to [email protected]. Continue following MarketBeat Before you consider BioCryst Pharmaceuticals, you'll want to hear this. MarketBeat keeps track of Wall Street's top-rated and best performing research analysts and the stocks they recommend to their clients on a daily basis. MarketBeat has identified the five stocks that top analysts are quietly whispering to their clients to buy now before the broader market catches on... and BioCryst Pharmaceuticals wasn't on the list. 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AL biotech facility focused on rare diseases to close, lay off 47. Montgomery Advertiser Aug. 6, 2026, 4:10 a.m. CT An Alabama biotech facility has made the decision to close its operations at their location in Hoover, triggering just under 50 layoffs. North Carolina-based BioCryst is a biotech company that focuses on developing and commercializing medicines for hereditary angioedema and other rare diseases. Their biggest hit was Orladeyo, a drug used to treat hereditary angioedema that was the first oral, once-daily treatment for the disorder. They're also working to advance a pipeline of "potential first-in-class or best-in-class oral small-molecule and injectable protein therapeutics for a range of rare diseases," according to the company. BioCryst announced plans to close their Hoover location back in June, though the WARN notice about the matter was filed on Aug. 4, according to the Alabama Department of Workforce site. The WARN act, a federal law passed in 1988, requires companies to notify workers in writing before mass layoffs. Specifically, companies with over 100 employees are required to provide a 60-day notice of significant layoffs or plant closings to allow workers to properly accommodate for the loss of work. The facility in Hoover focused on "internal discovery," which refers to the research and development phases of product creation. Biocryst said in June it will "discontinue" its internal discovery programs as they work to change their pipeline process to instead utilize "external innovation," which often in the pharmaceutical field refers to the practice of sourcing the molecules used in the drugs a company produces from smaller labs or startup biotech companies instead of in house. "While our internal discovery programs have played a foundational role in building the business we are today, we believe that our next phase of growth and value creation will come from a more agile, targeted approach to research," said Charlie Gayer, president and chief executive officer of BioCryst. "By leveraging external capabilities and partnerships, as well as our powerful rare disease commercialization engine, we can expand our opportunity set and bring new rare disease therapies to patients faster and in a more capital-efficient manner." When the facility closes on Oct. 7, 47 employees will be laid off. Gayer said that the company is "deeply grateful to the Birmingham team for their meaningful contributions to BioCryst's science," and that the company is "committed to supporting everyone affected through this transition." The exact parameters of what that support will look like is unclear, as BioCryst did not respond to a request for comment on that matter. Sarah Clifton covers business for the Montgomery Advertiser. You can reach her at [email protected] or follow her on X @sarahgclifton and TikTok @sarahgclifton. To support her work, please subscribe to the Montgomery Advertiser.