BioCryst Pharmaceuticals, Inc. (BioCryst) operates as a global biotechnology company.
The company improves the lives of people living with hereditary angioedema (‘HAE’) and other rare diseases. The company leverages its expertise in structure-guided drug design with the intention of developing first-in-class or best-in-class oral small-molecule and injectable protein therapeutics to target difficult-to-treat rare diseases. Structure-guided drug design is a drug discovery approach by which the c...
BioCryst Pharmaceuticals, Inc. (BioCryst) operates as a global biotechnology company.
The company improves the lives of people living with hereditary angioedema (‘HAE’) and other rare diseases. The company leverages its expertise in structure-guided drug design with the intention of developing first-in-class or best-in-class oral small-molecule and injectable protein therapeutics to target difficult-to-treat rare diseases. Structure-guided drug design is a drug discovery approach by which the company designs synthetic compounds from detailed structural knowledge of the active sites of targets associated with particular diseases. The company uses X-ray crystallography, computer modeling of molecular structures and advanced chemistry techniques to focus on the three-dimensional molecular structure and active site characteristics of the protein targets that control cellular biology.
In addition to these discovery and development efforts, the company’s business strategy includes the successful commercialization of these drugs, as well as self-funding all these efforts by achieving and increasing profitability.
Business Strategy
The company’s business strategy is threefold: to serve patients and to create stockholder value by (i) focusing the company’s discovery and development efforts on potential first-in-class or best-in-class oral small-molecule and injectable protein therapeutics to target difficult-to-treat rare diseases, (ii) successfully commercializing these drugs, and (iii) self-funding all of these efforts by achieving and increasing profitability.
The company selects disease targets and product candidates in which an orally administered small-molecule or an injectable protein therapeutic has the potential to be best-in-class or would be the first to market. The company strives to advance its product candidate portfolio from discovery to commercial markets efficiently by utilizing a small group of talented and highly skilled employees working in conjunction with strategic outsource partners. The company is unique in its approach to treat difficult-to-treat rare diseases with orally administered small molecules and with injectable protein therapeutics, each identified by utilizing crystallography and structure-guided drug design.
The principal elements of the company’s strategy include focusing on high value-added structure-guided drug design technologies; selecting inhibitors that are promising product candidates; selecting inhibitors that are promising product candidates; developing its product candidates efficiently; and commercializing the company’s product candidates globally.
Products and Product Candidates
ORLADEYO (berotralstat)
ORLADEYO is an oral capsule, once-daily therapy discovered and developed by the company for the prevention of HAE attacks. By inhibiting plasma kallikrein, ORLADEYO suppresses bradykinin production. Bradykinin is the mediator of acute swelling attacks in HAE patients.
ORLADEYO is approved by the U.S. Food and Drug Administration (‘FDA’) in December 2020 for prophylaxis to prevent attacks of HAE in adults and pediatric patients 12 years and older, and the company subsequently received regulatory approvals for ORLADEYO in other global markets. In addition, the ongoing APeX-P clinical trial, which is complete through the primary endpoint, is continuing to assess an oral granule formulation of ORLADEYO in pediatric patients who are 2 to 11 years of age. The company’s specialty pharmacy provider for ORLADEYO in the United States began shipping ORLADEYO to patients with a prescription in the United States in December 2020. Through EMPOWER Patient Services, administered by the company’s specialty pharmacy provider, it intends to streamline access to therapy by providing each HAE patient and their healthcare provider with a single point of contact for access to ORLADEYO.
The company has entered into a number of collaborations with commercial partners to help support the global commercialization of ORLADEYO.
On February 19, 2024, the company announced that the Italian Medicines Agency finalized reimbursement and recommended ORLADEYO for the routine prevention of recurrent attacks of HAE in eligible patients 12 years and older.
On February 23, 2024, the company announced new analyses of real-world use of ORLADEYO that showed patients who initiated ORLADEYO experienced rapid, substantial and sustained reductions in attack rates through 18 months of treatment regardless of the severity of their disease, their history of prior prophylaxis or their C1-inhibitor level and function.
On April 17, 2024, the company announced that the Brazilian Health Regulatory Agency granted approval for ORLADEYO for the prophylaxis of HAE attacks in adults and pediatric patients 12 years of age or older.
On May 6, 2024, the company announced that enrollment is complete in the APeX-P trial evaluating ORLADEYO in pediatric HAE patients who are 2 to 11 years of age. Data from the trial will support a regulatory filing in 2025 to expand the ORLADEYO label to enable children as young as two years of age to receive ORLADEYO.
On May 9, 2024, the company announced new real-world evidence on the use of ORLADEYO demonstrating that patients with HAE in the United States experience significant reductions in healthcare resource utilization after beginning treatment with ORLADEYO.
On May 13, 2024, the company announced that the Federal Commission for Protection against Health Risks in Mexico granted approval for ORLADEYO for the prophylaxis of HAE attacks in adults and pediatric patients 12 years of age or older.
On June 2, 2024, the company announced new real-world evidence showing that patients with HAE who have normal C1-inhibitor level and function had a reduction in monthly attack rates after starting ORLADEYO.
On July 9, 2024, the company announced that the General Directorate of Medicines, Supplies and Drugs in Peru granted approval for ORLADEYO for the prophylaxis of HAE attacks in adults and pediatric patients 12 years of age or older.
On August 5, 2024, the company announced that a recent market research study showed that 52 percent of allergist/immunologists are extremely likely to prescribe ORLADEYO to more patients, up from 29 percent in early 2023.
On August 5, 2024, the company also announced that it remains on track to submit a regulatory filing in 2025 to expand the ORLADEYO label to enable children as young as two years of age to receive an oral granule formulation of ORLADEYO.
On September 6, 2024, the company announced the presentation of six posters, including the first interim data from APeX-N, a European multi-center observational study assessing the safety (primary objective), effectiveness and quality of life (secondary objectives) of berotralstat 150 mg in routine clinical use. The company also presented new data highlighting the value of shared decision making between healthcare providers and their HAE patients to provide optimal patient outcomes.
On October 14, 2024, the company announced new real-world evidence on the use of ORLADEYO demonstrating that patients with HAE in the United States experience significant reductions in healthcare resource utilization, including significant reductions in hospitalizations, emergency room visits and use of on-demand therapies, after beginning treatment with ORLADEYO.
On October 24, 2024, the company announced new real-world comparative research on the use of ORLADEYO that found high rates of adherence and persistence for ORLADEYO, similar to the rates observed with two other long-term prophylactic (‘LTP’) therapies for HAE. The company also announced new real-world evidence showing statistically significant and sustained HAE attack rate reductions after initiating ORLADEYO in patients with HAE, regardless of their C1-inhibitor deficiency status, and new findings from an HAE patient survey confirming patient preference for an oral LTP therapy.
On November 4, 2024, the company announced that since launch, approximately half of patients who have started ORLADEYO have switched from another prophylactic therapy. The company begun the observational Phase 4 APeX-T study, designed to generate real-world data to inform physicians on the best individual approaches to support transition to ORLADEYO.
On November 18, 2024, the company announced that the Health Services Executive in Ireland recommended ORLADEYO for the routine prevention of recurrent attacks of HAE in eligible patients 12 years and older.
On February 12, 2025, the company announced that Infarmed in Portugal has recommended ORLADEYO for the routine prevention of recurrent attacks of HAE in eligible patients 12 years and older.
On February 24, 2025, the company announced that a new market tracking survey of 60 HAE treaters showed that 97 percent are considering prescribing ORLADEYO and 59 percent (up from 26 percent 18 months prior) of prescribers indicate they are extremely likely to prescribe for more of their patients. In addition, the company announced that additional real-world studies with ORLADEYO show statistically significant HAE attack rate reductions experienced by patients with C1-inhibitor deficiency and normal C1-inhibitor levels and function. Patient-reported outcomes also showed willingness to change long-term prophylaxis and improved treatment satisfaction across varying levels of attack frequency and severity after ORLADEYO initiation.
On February 24, 2025, the company also announced that it is on track to submit a new drug application in 2025 to the U.S. Food and Drug Administration to expand the ORLADEYO label to children with HAE aged 2 to 11 using an oral granule formulation. Additional regulatory filings are planned in global territories, including Europe, Japan, and Canada. ORLADEYO would be the first targeted oral prophylactic therapy for children with HAE. In addition, the company announced positive results from an interim analysis of the ongoing APeX-P clinical trial evaluating an oral granule formulation of ORLADEYO in pediatric patients with HAE aged 2 to 11.
On each of December 7, 2020, and November 19, 2021, the company entered into a Purchase and Sale Agreement with RPI 2019 Intermediate Finance Trust (‘RPI’), pursuant to which it sold to RPI the right to receive certain royalty payments from the company (the ‘RPI Royalty Purchase Agreements’). On November 19, 2021, the company also entered into a Purchase and Sale Agreement (the ‘OMERS Royalty Purchase Agreement’, and collectively with the RPI Royalty Purchase Agreements, the Royalty Purchase Agreements’) with OCM IP Healthcare Holdings Limited, an affiliate of OMERS Capital Markets (‘OMERS’), pursuant to which it sold to OMERS the right to receive certain royalty payments from the company. The transactions contemplated under the Royalty Purchase Agreements are referred to herein as the ‘Royalty Sales.’
The company has built out its U.S. commercial infrastructure to support the launch and continued commercialization of ORLADEYO in the United States and are continuing to build the company’s commercial infrastructure to support launches in other markets. Based on proprietary analyses of HAE prevalence and market research studies with HAE patients, physicians, and payors in the United States and Europe, and four full years of commercialization experience with ORLADEYO in the United States from 2021 through 2024, the company anticipates that the global commercial market for ORLADEYO has the potential to reach a global peak of $1 billion in annual net ORLADEYO revenues.
BCX17725 (Netherton Syndrome)
BCX17725 is a potent and selective investigational protein therapeutic KLK5 inhibitor designed to provide best-in-class, potentially disease-modifying, treatment for people with Netherton syndrome.
On May 6, 2024, the company announced that it expected to advance BCX17725 into the clinic by the end of 2024, and the company reaffirmed this plan on August 5, 2024.
On October 2, 2024, the company announced the enrollment of the first participant in a Phase 1 trial evaluating BCX17725 for people with Netherton syndrome.
On November 4, 2024, the company announced that it expects initial data from the BCX17725 program in 2025, and the company reaffirmed this on February 24, 2025.
Avoralstat
The company is developing its investigational plasma kallikrein inhibitor, avoralstat, with Clearside Biomedical, Inc.’s SCS Microinjector to deliver avoralstat to the back of the eye through the suprachoroidal space to treat patients with diabetic macular edema (‘DME’). Avoralstat targets the kallikrein-bradykinin system on the retinal vascular endothelial cells and may result in less vascular leakage and less edema. Avoralstat, delivered to the suprachoroidal space, is designed to provide high dose levels to the retinal vessels with long-lasting exposure, which could result in less frequent injections and a reduced burden on patients and the healthcare system.
On May 6, 2024, the company announced its plans to advance avoralstat into a clinical trial of patients with DME in 2025.
On August 5, 2024, and November 4, 2024, the company again noted its expected plan to advance avoralstat into a clinical trial of patients with DME in 2025, and the company reaffirmed this on February 24, 2025.
On February 24, 2025, the company announced that initial clinical data from the avoralstat program is targeted by the end of 2025.
Complement-Mediated Diseases
Oral C5 Inhibitor
The company is developing an oral C5 inhibitor that could be the first targeted oral therapy with competitive efficacy to approved injected and infused anti-C5 therapies, such as eculizumab and ravulizumab. A drug with this profile could enable patients to switch from infused therapy and address their disease earlier in the treatment paradigm.
Oral C2 Inhibitor
The company is developing a classical and lectin pathway complement inhibitor. An oral C2 inhibitor developed by it could be first-in-class and allow patients to switch from infused therapy and address their disease earlier in the treatment paradigm.
Bifunctional Complement Inhibitor
The company is developing a bifunctional complement inhibitor anti-C2 monoclonal antibody that could be a first-in-class combined inhibitor of the classical, lectin and alternative pathways of the complement system to treat complex complement-mediated diseases that are influenced by multiple complement pathways.
BCX10013
BCX10013 is an oral Factor D inhibitor that targets the alternative pathway of complement. On January 5, 2024, the company announced that, if its ongoing proof-of-concept trial produced best-in-class data, the company planned to out-license late-stage development and commercialization of BCX10013 to a partner that can drive the speed and breadth of investment required to accelerate BCX10013 for patients across multiple complement-mediated diseases and maximize the commercial potential of the program.
On August 5, 2024, the company announced that it completed its clinical evaluation of BCX10013. The drug was safe and well tolerated at all doses studied; however, the level of clinical activity observed was less than other therapies on the market, and potential partners have declined to make the additional investment required to evaluate higher doses.
Peramivir Injection (RAPIVAB, RAPIACTA, and PERAMIFLU)
RAPIVAB (peramivir injection) was developed under a contract from the Biomedical Advanced Research and Development Authority within the U.S. Department of Health and Human Services (BARDA/HHS). In January 2010, the company’s partner, Shionogi, received the first approval for peramivir injection and launched it in Japan under the commercial name RAPIACTA. It is approved in Japan for the treatment of adults, children, and infants with uncomplicated seasonal influenza and those patients at high-risk for complications associated with influenza. In August 2010, the company’s partner, Green Cross, received marketing and manufacturing approval from the Korean Food & Drug Administration under the commercial name PERAMIFLU to treat patients with influenza A & B viruses, including pandemic H1N1 and avian influenza.
Peramivir was also approved in the United States in 2014, Taiwan in 2016, Canada in 2017, and Australia in 2018. A Supplemental New Drug Application was approved in the United States in February 2021, extending RAPIVAB’s availability for the treatment of acute uncomplicated influenza to pediatric patients six months and older. Prior to this approval, peramivir had been indicated in the United States for pediatric patients two years and older. In the United States, peramivir is indicated for the treatment of acute uncomplicated influenza in patients who have been symptomatic for no more than two days. Since the 2009 H1N1 pandemic, RAPIVAB has been an important component of the U.S. Government’s influenza preparedness efforts.
On September 30, 2024, the company announced that the U.S. Department of Health and Human Services (‘HHS’) awarded the company a contract for the procurement of up to 95,625 doses over a five-year period of RAPIVAB for the treatment of influenza (the ‘HHS Contract’). The company delivered 2,304 doses of RAPIVAB under the HHS Contract in the fourth quarter of 2024. Peramivir was also approved in the United States in 2014, Taiwan in 2016, Canada in 2017, and Australia in 2018. A Supplemental New Drug Application was approved in the United States in February 2021, extending RAPIVAB’s availability for the treatment of acute uncomplicated influenza to pediatric patients six months and older. Prior to this approval, peramivir had been indicated in the United States for pediatric patients two years and older. In the United States, peramivir is indicated for the treatment of acute uncomplicated influenza in patients who have been symptomatic for no more than two days. Since the 2009 H1N1 pandemic, RAPIVAB has been an important component of the U.S. Government’s influenza preparedness efforts. The company delivered 2,304 doses of RAPIVAB under the HHS Contract in the fourth quarter of 2024.
Collaborations and In-License Relationships
ORLADEYO
Torii Pharmaceutical Co., Ltd. (‘Torii’)
On November 5, 2019, the company entered into a Commercialization and License Agreement with Torii (the ‘Original Torii Agreement’), granting Torii the exclusive right to commercialize ORLADEYO for the prevention of HAE attacks in Japan.
On November 30, 2023, the company entered into an Amended and Restated Commercialization and License Agreement with Torii (as amended, the ‘Torii Agreement’).
Under the Torii Agreement, the company is entitled to receive tiered royalty payments, ranging from 20% to 80% of annual net sales of ORLADEYO in Japan during each calendar year. The company is responsible for all commercial promotion activities to support ORLADEYO sales in Japan, and Torii will be responsible for HAE disease awareness activities in Japan.
Other Collaborations for ORLADEYO
The company entered a number of collaborations with commercial partners to help support the global commercialization of ORLADEYO. In 2021, the company entered into supply and distribution agreements with Neopharm Ltd. (‘Neopharm’) and NewBridge Pharmaceuticals (‘NewBridge’) to support commercialization efforts in Israel and the United Arab Emirates (‘UAE’), respectively. On June 9, 2022, the company entered an exclusive collaboration with Pint Pharma to register and promote ORLADEYO in the pan-Latin America region. On January 23, 2023, the company entered a collaboration with Swixx BioPharma AG (‘Swixx’) to commercialize ORLADEYO in Central and Eastern Europe (‘CEE’). On July 19, 2023, the company entered a collaboration with Er-Kim Pharmaceuticals to commercialize ORLADEYO in Turkey.
Peramivir Injection (RAPIVAB, RAPIACTA, and PERAMIFLU)
Shionogi & Co., Ltd. (‘Shionogi’)
In February 2007, the company entered a license, development and commercialization agreement (as amended, supplemented or otherwise modified, the ‘Shionogi Agreement’), an exclusive license agreement with Shionogi to develop and commercialize peramivir in Japan for the treatment of seasonal and potentially life-threatening human influenza. In October 2008, the company and Shionogi amended the Shionogi Agreement to expand the territory covered by the agreement to include Taiwan.
The company developed peramivir under a license from the University of Alabama Birmingham ('UAB’) and have paid sublicense payments to UAB on the upfront payments and will owe sublicense payments on any future event payments and/or royalties received by the company from Shionogi.
Green Cross Corporation (‘Green Cross’)
In June 2006, the company entered into an agreement with Green Cross to develop and commercialize peramivir in Korea. Under the terms of the agreement, Green Cross is responsible for all development, regulatory, and commercialization costs in Korea and the company is entitled to share in profits resulting from the sale of peramivir in Korea, including the sale of peramivir to the Korean government for stockpiling purposes. Furthermore, Green Cross will pay the company a premium over its cost to supply peramivir for development and any future marketing of peramivir products in Korea. Both parties have the right to terminate the agreement in the event of an uncured material breach. In the event of termination, all rights, data, materials, products, and other information would be transferred to the company.
Additional Collaborations
The company delivered 2,304 doses of RAPIVAB under the HHS Contract in the fourth quarter of 2024.
The company also has non-material license agreements with certain third parties, such as Albert Einstein College of Medicine of Yeshiva University (‘AECOM’), Industrial Research, Ltd. (‘IRL’), and the University of Alabama at Birmingham (‘UAB’), which require that it makes certain payments related to development of the product candidates covered by these agreements. The company licensed a series of potent inhibitors of purine nucleoside phosphorylase (‘PNP’) from AECOM and IRL, as well as an exclusive worldwide license of galidesivir for any antiviral use, and the company has agreements with UAB for influenza neuraminidase and complement inhibitors. There is no material activity between the company and UAB on these agreements, but when the company licenses this technology, such as in the case of the Shionogi and Green Cross agreements, or commercialize products related to these programs, the company owes sublicense fees or royalties on amounts received.
The company entered into a license agreement with Clearside Biomedical, Inc. to develop its investigational plasma kallikrein inhibitor, avoralstat, with Clearside’s SCS Microinjector to deliver avoralstat to the back of the eye through the suprachoroidal space to treat patients with DME.
Patents and Proprietary Information
As of December 31, 2024, the company had been issued approximately 44 U.S. patents that expire between 2027 and 2039 and that relate to its kallikrein inhibitor compounds, neuraminidase inhibitor compounds, broad-spectrum antiviral (‘BSAV’) compounds, PNP inhibitor compounds, and complement-mediated disease program compounds. The company has licensed a number of compounds protected by certain composition of matter patents from AECOM and IRL, totaling one additional U.S. patent that expires in 2029. Additionally, the company has approximately 21 Patent Cooperation Treaty or U.S. patent applications pending related to kallikrein inhibitor compounds, neuraminidase inhibitor compounds, BSAV compounds, PNP inhibitor compounds, KLK5 program compounds, and complement-mediated disease program compounds.
Government Regulation
The company’s business is subject to extensive regulation by the FDA and foreign governments. These regulations include, among other things, regulations for the development, testing, manufacture, safety, efficacy, record-keeping, labeling, storage, approval, advertising, promotion, sale and distribution of pharmaceutical products.
After completing preclinical trials, the company must file an investigational new drug application (‘IND’), including a proposal to begin clinical trials, with the FDA.
The company and all its contract manufacturers are also required to comply with the applicable FDA Good Manufacturing Practice (‘cGMP’) regulations during clinical development and to ensure that the product can be consistently manufactured to meet the specifications submitted in an NDA.
To obtain manufacturing/marketing approval, the company must apply for approval to the Ministry of Health, Labor and Welfare (‘MHLW’) with results of nonclinical and clinical studies to show the quality, efficacy, and safety of a new drug.
The company is subject to various federal and state laws pertaining to healthcare ‘fraud and abuse,’ including both federal and state anti-kickback and false claims laws. In addition, the company is subject to the federal Physician Sunshine Act and certain similar physician payment and drug pricing transparency legislation in various states.
The company is also subject to evolving European privacy laws on electronic marketing and cookies. For example, the European Union is in the process of replacing the e-Privacy Directive (2002/58/EC) with a new set of rules taking the form of a regulation that will be directly implemented in the laws of each EU member state.
The company is also subject to the U.S. Foreign Corrupt Practices Act (‘FCPA’), which prohibits corporations and individuals from engaging in certain activities to obtain or retain business or to influence a person working in an official capacity.
Research and Development
The company’s research and development expenses were $174.6 million for the year ended December 31, 2024.
History
BioCryst Pharmaceuticals, Inc. was founded in 1986. The company was incorporated in Delaware in 1991.
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