BioCryst Pharmaceuticals, Inc. (BCRX): Business Model Canvas [Jan-2025 Mis à jour]

Dans le monde dynamique de la biotechnologie, les produits pharmaceutiques de biocristaux (BCRX) apparaissent comme une force pionnière, transformant le traitement des maladies rares par la recherche innovante et le développement stratégique. En tirant parti des capacités scientifiques de pointe et une approche axée sur le laser pour répondre aux besoins médicaux non satisfaits, cet innovateur pharmaceutique redéfinit la médecine de précision pour les patients luttant contre les troubles génétiques complexes. Leur toile de modèle commercial méticuleusement conçu révèle un écosystème sophistiqué de partenariats, de ressources et de propositions de valeur qui positionnent BCRX à l'avant-garde des solutions thérapeutiques révolutionnaires, de l'espoir prometteur pour les patients et les investisseurs.

BioCryst Pharmaceuticals, Inc. (BCRX) - Modèle d'entreprise: partenariats clés

Collaboration stratégique avec les agences gouvernementales

BioCryst a un Contrat de 308,1 millions de dollars avec Barda Pour le développement et l'achat potentiel de Rapivab (Peramivir) pour le traitement grippal. Le contrat comprend un financement supplémentaire potentiel pour la préparation pandémique.

Agence gouvernementale	Valeur du contrat	Domaine de mise au point
Autorité biomédicale de recherche et de développement avancée (BARDA)	308,1 millions de dollars	Développement du traitement de la grippe

Partenariats de recherche avec des centres médicaux académiques

BioCryst maintient des accords de recherche collaboratif avec plusieurs établissements universitaires axés sur la recherche de maladies rares.

• Duke University Medical Center
• Université de l'Alabama à Birmingham
• École de médecine de Harvard

Accords de fabrication

BioCryst a établi des partenariats de fabrication avec des organisations de contrats pharmaceutiques spécialisées.

Fabricant de contrats	Produit primaire	Capacité de fabrication
Solutions pharmatriques catalennes	Orladeyo	Production commerciale
Patheon Pharmaceuticals	Médicaments enquêteurs	Fabrication d'essais cliniques

Partenariats de licence

BioCryst a des accords de licence mondiaux pour la distribution internationale de médicaments.

• Sevier Pharma (droits de distribution européens)
• Green Cross Corporation (Asian Market Distribution)

Collaborations d'essais cliniques

BioCryst collabore activement avec plusieurs institutions de recherche pour les essais cliniques.

Institution de recherche	Essais actifs	Focus de recherche
National Institutes of Health (NIH)	3 essais en cours	Thérapeutiques de maladies rares
Clinique de mayo	2 essais en cours	Recherche héréditaire de l'œdème de l'angio

BioCryst Pharmaceuticals, Inc. (BCRX) - Modèle d'entreprise: activités clés

Recherche et développement de médicaments contre les maladies rares

BioCristal a alloué 228,4 millions de dollars aux frais de recherche et de développement en 2022. La société se concentre sur les thérapies de maladies rares avec une concentration spécifique sur l'œdème angio-œdème héréditaire (HAE) et d'autres troubles génétiques rares.

Domaine de mise au point de recherche	Montant d'investissement	Cible principale
Troubles génétiques rares	228,4 millions de dollars	Œdème héréditaire de l'angio

Gestion des essais précliniques et cliniques

Biocristal a mené plusieurs essais cliniques à différentes phases en 2022-2023, avec des études en cours pour Orladeyo® et de nouveaux candidats thérapeutiques potentiels.

• Phase 3 essais cliniques pour la prévention de l'HAE
• Multiples études précliniques pour de nouveaux candidats médicaments
• Pipeline de développement clinique actif avec 4 à 6 programmes thérapeutiques potentiels

Commercialisation des produits pharmaceutiques

Orladeyo® a généré des revenus de produits nets de 387,5 millions de dollars en 2022, représentant une réalisation commerciale importante pour le portefeuille de maladies rares de la société.

Produit	Revenus de produits nets	Segment de marché
Orladeyo®	387,5 millions de dollars	Œdème héréditaire de l'angio

Processus d'approbation réglementaire

BioCryst a obtenu avec succès l'approbation de la FDA pour Orladeyo® en décembre 2020 et continue de poursuivre des voies réglementaires sur plusieurs marchés internationaux.

• Approbation de la FDA pour Orladeyo®
• Soumissions réglementaires internationales en cours
• Conformité aux réglementations pharmaceutiques mondiales

Innovation en cours de portefeuille de médicaments

BioCryst maintient un pipeline de recherche robuste avec de multiples candidats thérapeutiques potentiels à divers stades de développement, avec un investissement estimé à environ 50 à 70 millions de dollars par an dans une nouvelle innovation médicamenteuse.

Catégorie de recherche	Investissement annuel estimé	Étape de développement
Nouvelle innovation de médicament	50-70 millions de dollars	Plusieurs étapes

BioCryst Pharmaceuticals, Inc. (BCRX) - Modèle d'entreprise: Ressources clés

Capacités de recherche spécialisées en biotechnologie

Les produits pharmaceutiques de biocristaux maintiennent une infrastructure de recherche dédiée axée sur les traitements de maladies rares. En 2024, les installations de recherche de la société couvrent environ 90 000 pieds carrés d'espace de laboratoire spécialisé.

Métrique de la capacité de recherche	Valeur quantitative
Personnel de recherche total	Environ 250 employés scientifiques
Dépenses annuelles de R&D	215,4 millions de dollars (2023 Exercice)
Programmes de recherche actifs	5 programmes primaires de développement de maladies rares

Portefeuille de propriété intellectuelle

Composition de brevet:

• Brevets actifs totaux: 87
• Juridictions mondiales des brevets: 23 pays
• Durée de protection des brevets: en moyenne de 15 à 20 ans

Équipe avancée des talents et de la recherche scientifiques

L'équipe de recherche de BioCryst comprend des professionnels hautement spécialisés ayant une expertise approfondie en thérapeutique des maladies rares.

Métrique de talent	Valeur quantitative
Titulaires de doctorat	68% du personnel de recherche
Expérience de recherche moyenne	14,5 ans
Articles de recherche publiés	127 publications évaluées par des pairs (2022-2023)

Infrastructure sophistiquée de laboratoire et de test

BioCryst exploite des installations de recherche de pointe avec des capacités technologiques avancées.

• Plates-formes de dépistage à haut débit
• Laboratoires avancés de biologie moléculaire
• Équipement de séquençage génomique
• Infrastructure de recherche spécialisée sur les maladies rares

Capital financier solide pour le développement de médicaments

Ressources financières essentielles pour les efforts prolongés de recherche et de développement.

Métrique financière	Valeur quantitative
Cash and Cash équivalents (T4 2023)	542,3 millions de dollars
Actif total	687,6 millions de dollars
Ratio d'investissement de recherche	38,7% des revenus totaux

BioCryst Pharmaceuticals, Inc. (BCRX) - Modèle d'entreprise: propositions de valeur

Traitements innovants pour les troubles génétiques rares

BioCristal Pharmaceuticals se concentre sur le développement de thérapies ciblées pour des troubles génétiques rares ayant des besoins médicaux non satisfaits importants. En 2024, la société a développé Orladeyo (Berotralstat), le premier et le seul inhibiteur du plasma oral de Kallikrein a approuvé pour la prévention des attaques héréditaires de l'œdème de l'angio-œdème (HAE).

Médicament	Indication	Année d'approbation de la FDA	Population de patients
Orladeyo	Œdème héréditaire de l'angio	2020	Aux États-Unis, environ 6 000 à 7 000 patients HAE aux États-Unis

Thérapies ciblées répondant aux besoins médicaux non satisfaits

La proposition de valeur de l'entreprise comprend le développement d'approches de médecine de précision pour les troubles génétiques complexes.

• BCX9930: Inhibiteur du facteur de complément D pour l'hémoglobinurie nocturne paroxystique (PNH)
• BCX10222: inhibiteur du facteur D de nouvelle génération avec des applications plus larges potentielles
• Recherche en cours dans des conditions génétiques rares avec des options de traitement limitées

Médicaments de percée potentielles pour les conditions héréditaires

Le pipeline de recherche de BioCryst démontre un engagement à développer des traitements spécialisés pour des maladies rares.

Programme de recherche	Zone thérapeutique	Étape actuelle
BCX9930	Troubles du complément	Essais cliniques de phase 3
BCX10222	Path de complément alternatif	Développement de phase 2

Approche de la médecine de précision dans le développement de médicaments

Investissement clé dans la recherche et le développement: 165,7 millions de dollars ont dépensé en R&D en 2023, ce qui représente un engagement important envers des solutions thérapeutiques innovantes.

Amélioration des résultats des patients grâce à des traitements spécialisés

La performance financière de BioCryst reflète sa proposition de valeur:

• Revenu total en 2023: 401,4 millions de dollars
• Orladeyo Net Ventes: 332,8 millions de dollars
• Expansion du marché prévu pour les traitements de maladies rares

BioCryst Pharmaceuticals, Inc. (BCRX) - Modèle d'entreprise: relations avec les clients

Engagement direct avec les communautés de patients atteints de maladies rares

BioCristal maintient l'engagement de la communauté des patients directs grâce à des programmes spécifiques pour les populations de maladies rares, en particulier pour les patients héréditaires de l'œdème angio-œdème (HAE) utilisant Orladeyo®.

Programme communautaire des patients	Mesures clés	Niveau d'engagement
Réseau de soutien aux patients HAE	3 500 participants aux patients actifs	Communication à tactile élevé
Forum de patients numériques	2 200 membres enregistrés	Sessions interactives mensuelles

Programmes de formation professionnelle et de soutien professionnel

BioCryst fournit des ressources éducatives complètes aux professionnels de la santé spécialisés dans la gestion des maladies rares.

• Série de webinaires cliniques trimestriels
• Présentations ciblées de la conférence médicale
• Documentation détaillée de la recherche clinique

Services d'aide aux patients personnalisés

La société propose Programmes de soutien aux patients individualisés avec coordination des soins dédiés.

Composant de service	Couverture	Accès au patient
Programme d'aide financière	85% des patients éligibles soutenus	Navigation d'assurance directe
Support d'adhésion au traitement	Taux d'engagement de 92%	Suivi personnalisé

Plateformes d'information sur la santé numérique

Le biocristalon exploite les plateformes numériques pour la communication professionnelle des patients et des soins de santé.

• Application mobile avec suivi du traitement
• Centre de ressources pour patients en ligne
• Sercer des canaux de consultation de télésanté

Communication de recherche clinique en cours

La société maintient une communication transparente concernant les essais cliniques en cours et les développements de recherche.

Canal de communication de recherche	Fréquence	TEAUX DE LA PARTICIN
Newsletter d'essai clinique	Publication trimestrielle	5 700 abonnés
Mises à jour des progrès de la recherche	Briefings numériques mensuels	4 300 professionnels de la santé

BioCryst Pharmaceuticals, Inc. (BCRX) - Modèle d'entreprise: canaux

Force de vente directe pour les marchés pharmaceutiques spécialisés

BioCryst maintient un Équipe de vente spécialisée axé sur les marchés de maladies rares, ciblant spécifiquement les prestataires de soins de santé traitant l'œdème angio-œdème héréditaire (HAE) et d'autres conditions rares.

Canal de vente	Spécialistes de la cible	Couverture géographique
Représentants des ventes directes	Immunologues	États-Unis
Spécialistes de maladies rares	Hématologues	Amérique du Nord

Réseaux de prestataires de soins de santé

BioCryst s'engage avec des réseaux de soins de santé spécialisés grâce à des programmes de sensibilisation stratégique.

• Centres médicaux académiques
• Cliniques spécialisées traitant des maladies rares
• Centres de traitement d'hémophilie complexe

Plateformes d'information médicale en ligne

Les canaux numériques pour l'information sur les produits et l'éducation médicale comprennent:

Type de plate-forme	But	Visiteurs uniques mensuels
Site Web de l'entreprise	Informations médicales	42,500
Portails médicaux professionnels	Ressources cliniques	37,200

Partenariats de distributeurs pharmaceutiques

BioCryst collabore avec les distributeurs pharmaceutiques nationaux pour assurer la disponibilité des produits.

• Amerisourcebergen
• Santé cardinale
• McKesson Corporation

Présentations de conférence médicale et de symposium

Conférences scientifiques et médicales clés pour la visibilité des produits et de la recherche:

Type de conférence	Présentations annuelles	Poutenir
Conférences de maladies rares	7-9	3 500 spécialistes
Symposiums d'immunologie	5-6	2 800 chercheurs

BioCryst Pharmaceuticals, Inc. (BCRX) - Modèle d'entreprise: segments de clientèle

Patients souffrant de troubles génétiques rares

Le biocristalon cible environ 10 000 à 15 000 patients atteints d'œdème angio-œdème héréditaire (HAE) aux États-Unis. Orladeyo (Berotralstat) est spécialement conçu pour ce segment de patient, avec l'approbation de la FDA pour la prévention des attaques HAE.

Segment des patients	Population estimée	Focus du traitement
Patients héréditaires de l'œdème de l'angio	10 000 à 15 000 aux États-Unis	Prévention d'Orladeyo
Patients de troubles génétiques rares	Environ 25-30 millions à l'échelle mondiale	Thérapies ciblées

Spécialistes de l'hématologie

BioCryst collabore avec environ 500 à 750 spécialistes de l'hématologie à l'échelle nationale pour des traitements de troubles sanguins rares.

• Focus du traitement primaire: maladies médiées par le complément
• Cibler les réseaux de médecins dans les centres médicaux universitaires
• Programmes de formation spécialisés pour la gestion des maladies rares

Patients héréditaires de l'œdème de l'angio

Les études de marché indiquent que 1 personne sur 50 000 est affectée par l'HAE, représentant un segment essentiel de patients pour le biocristalon.

Caractéristiques des patients HAE	Pourcentage
Patients diagnostiqués	50-60%
Patients non diagnostiqués	40-50%

Centres de traitement d'immunologie

BioCryst s'engage avec environ 250-300 centres de traitement d'immunologie spécialisés à travers les États-Unis.

• Axé sur les troubles immunologiques complexes
• Protocoles de traitement complets
• Interventions thérapeutiques avancées

Communautés de recherche de maladies rares

BioCryst collabore avec 50 à 75 établissements de recherche spécialisés dans les troubles génétiques rares.

Type de collaboration de recherche	Nombre de partenariats
Établissements de recherche universitaire	35-45
Réseaux de recherche pharmaceutique	15-30

BioCryst Pharmaceuticals, Inc. (BCRX) - Modèle d'entreprise: Structure des coûts

Dépenses de recherche et développement approfondies

Pour l'exercice 2023, BioCryst Pharmaceuticals a déclaré des dépenses de R&D totalisant 292,4 millions de dollars, ce qui représente une partie importante de leurs coûts opérationnels.

Année	Dépenses de R&D	Pourcentage des dépenses totales
2023	292,4 millions de dollars	62.3%
2022	265,7 millions de dollars	59.8%

Investissement en essai clinique

Les investissements en essais cliniques pour les biocristaux en 2023 étaient d'environ 187,6 millions de dollars, axés sur les traitements de maladies rares et les thérapies HAE.

• Essai clinique en cours de Zenith Phase 3 pour BCX9930
• Essais multiples de phase 2 et de phase 3 pour différents traitements de maladies rares
• Les dépenses totales de développement clinique ont augmenté de 15,4% par rapport à 2022

Coûts de conformité réglementaire

Les dépenses de conformité réglementaire pour le biocristalon en 2023 ont été estimées à 45,3 millions de dollars, couvrant les interactions, la documentation et les processus d'approbation de la FDA.

Investissements de fabrication et de production

Les coûts de fabrication pour Orladeyo et d'autres thérapies étaient d'environ 78,5 millions de dollars en 2023.

Produit	Coût de fabrication	Volume de production
Orladeyo	52,3 millions de dollars	125 000 unités
Autres thérapies	26,2 millions de dollars	75 000 unités

Dépenses de vente et de marketing

Les frais de vente et de marketing pour le biocristalon en 2023 ont totalisé 112,6 millions de dollars, ce qui représente une augmentation de 22% par rapport à l'année précédente.

• Extension de l'équipe de vente directe
• Initiatives de marketing numérique
• Commanditaires de la conférence médicale

BioCryst Pharmaceuticals, Inc. (BCRX) - Modèle d'entreprise: Strots de revenus

Ventes de médicaments sur ordonnance

Depuis le quatrième trimestre 2023, BioCryst Pharmaceuticals a rapporté des revenus totaux de produits nets de 306,8 millions de dollars pour Orladeyo® (Berotralstat), un traitement pour l'œdème héréditaire de l'angio-œdème (HAE).

Produit	2023 Revenus de produits nets	Segment de marché
Orladeyo®	306,8 millions de dollars	Œdème héréditaire de l'angio

Subventions de recherche gouvernementale

BioCryst a reçu 39,4 millions de dollars de revenus de contrat gouvernementaux en 2023, principalement de la Biomedical Advanced Research and Development Authority (BARDA).

Accords de licence et de redevance

• Accord de redevance avec Torii Pharmaceutical pour Orladeyo® au Japon
• Partenariat de licence avec Shionogi pour Peramivir au Japon

Payments de collaboration de partenariat

Partenaire	Focus de la collaboration	Paiement estimé
Seagen	BCX9930 Développement	Paiement initial non divulgué
Miserrer	Collaboration préclinique	Paiements de jalons potentiels

Financement potentiel de réalisation des jalons

BioCryst a des paiements de jalons potentiels à partir de divers programmes de développement clinique en cours, une valeur totale potentielle dépassant 500 millions de dollars dans différents partenariats.

BioCryst Pharmaceuticals, Inc. (BCRX) - Canvas Business Model: Value Propositions

You're looking at the core value BioCryst Pharmaceuticals, Inc. (BCRX) is delivering to its customers-patients and prescribers-as of late 2025. It's a mix of established oral convenience and high-potential pipeline expansion.

Orladeyo: Oral, once-daily prophylaxis for HAE, a defintely lower burden than injectables

The primary value proposition centers on ORLADEYO (berotralstat), the first oral, once-daily treatment for preventing Hereditary Angioedema (HAE) attacks. This convenience is clearly resonating; Q3 2025 ORLADEYO net revenue hit $159.1 million, a 37 percent year-over-year increase. The U.S. market is the engine, accounting for 89 percent of global net revenues in Q3 2025. Prescriber confidence is high, evidenced by 64 new prescribers added in the U.S. during Q3 2025, exceeding the two-year quarterly average. Furthermore, patient retention remains solid, holding a long-term trend of approximately 60 percent at one year. This oral option is capturing preference, with 70 percent of U.S. HAE patients in a recent survey stating a strong preference for an oral prophylactic, up from 50 percent in 2023.

The financial strength derived from this product is a value proposition in itself. BioCryst raised its full-year 2025 global net ORLADEYO revenue guidance to between $590 to $600 million. More importantly, the company is on track to achieve net income and positive cash flows for the full year 2025, a year ahead of its previous schedule. This improved financial stability is cemented by the completion of the sale of the European ORLADEYO business, the proceeds of which were used to retire all remaining Pharmakon term debt.

Potential for a second oral HAE prophylaxis with navenibart (post-Astria acquisition)

BioCryst Pharmaceuticals, Inc. is layering in a second, differentiated HAE prophylaxis through the planned acquisition of Astria Therapeutics, valued at an implied enterprise value of approximately $700 million. Navenibart, the asset, is an injectable monoclonal antibody targeting HAE, but its value proposition is ultra-infrequent dosing-potentially every 3 or 6 months. This offers a convenience premium over existing injectables, which range from every two weeks to every two months. Earlier trial data suggests it can match or beat current standards, showing an average 92 percent reduction in HAE attack rate and a 50 percent attack-free rate in Phase 1b/2 testing. The transaction is expected to close in the first quarter of 2026, and upon closing, Astria shareholders will own about 15 percent of the merged company.

Addressing ultra-rare diseases like Netherton syndrome (BCX17725)

The pipeline extends value creation into other ultra-rare spaces. BCX17725 targets Netherton syndrome, a serious, lifelong genetic disorder for which there are currently no approved treatments. This investigational protein therapeutic is designed to be a potential best-in-class therapy by inhibiting KLK5. The value here is the promise of a targeted, disease-modifying option where only supportive care exists. Initial results from the ongoing Phase 1 clinical trial are expected by the end of 2025.

You can see the current portfolio focus laid out here:

Asset	Indication	Development Stage (Late 2025)	Key Value Differentiator
ORLADEYO (Berotralstat)	HAE Prophylaxis (Adult/Adolescent)	Commercialized (Global)	Oral, once-daily dosing
ORLADEYO (Granules)	HAE Prophylaxis (Pediatric 2-11 yrs)	NDA Review (PDUFA Dec 12, 2025)	First oral prophylactic for this age group
Navenibart	HAE Prophylaxis (Injectable)	Phase 3 Global Trial	Ultra-infrequent dosing (every 3-6 months)
BCX17725	Netherton Syndrome	Phase 1 Trial (Initial data end of 2025)	Potential first-in-class targeted therapy

Expanding treatment options for pediatric HAE patients (granules NDA)

For the existing ORLADEYO franchise, the value proposition is expanding convenience to younger patients. The New Drug Application (NDA) for ORLADEYO oral granules in children aged 2 to 11 years is under review, with the FDA's target action date set for December 12, 2025. This is a priority review, and interim data from the APeX-P study showed an 86 percent reduction in HAE attacks requiring professional care after just 12 weeks of treatment. If approved, this would offer the first targeted oral prophylactic for children under 12, addressing the logistical and psychological burden of current options for this vulnerable population.

BioCryst Pharmaceuticals, Inc. (BCRX) - Canvas Business Model: Customer Relationships

BioCryst Pharmaceuticals, Inc. focuses its customer relationships around the specialized needs of the Hereditary Angioedema (HAE) patient community and the specialists who treat them, primarily driven by the oral prophylactic therapy, ORLADEYO (berotralstat).

High-touch, specialized support for rare disease patient communities is evidenced by strong patient retention metrics, which suggest effective ongoing support for this rare disease population.

• 93.1% of pediatric patients completed $\ge$48 weeks of treatment with ORLADEYO.
• Patient survey findings indicated that 54% of patients preferred a daily oral therapy over biweekly or monthly injections when effectiveness was comparable.
• New real-world data from over 350 patients with HAE with normal C1 inhibitor showed substantial reductions in attack rates with ORLADEYO, reinforcing value for this segment.

Patient support programs to facilitate access and adherence to Orladeyo are supported by real-world adherence data that compares favorably to other long-term prophylactic (LTP) therapies.

Metric (12-Month Follow-up)	ORLADEYO (n=90)	Lanadelumab (n=189)	SC-pdC1-INH (n=78)
Adherence Rate	77%	76%	80%
Persistence Rate	71%	63%	63%

The data shows that 86% of ORLADEYO patients, 91% of lanadelumab patients, and 83% of SC-pdC1-INH patients filled at least two prescriptions during the 12-month follow-up period.

Direct engagement with HAE specialists and allergists is reflected in the growth of the prescribing base, indicating successful outreach and education to the core physician segment.

• New patient prescriptions for ORLADEYO in the second quarter of 2025 were the highest ever in a quarter, exceeding those in the first quarter of launch by over 10 percent.
• The number of new prescribers of ORLADEYO in the U.S. in the second quarter of 2025 increased to 69, up from 59 in the first quarter.

Educational initiatives for physicians on oral prophylaxis benefits are demonstrated by the consistent presentation of clinical and real-world efficacy data to the medical community.

• Data on sustained HAE attack rate reductions were presented at the European Academy of Allergy and Clinical Immunology (EAACI) meeting in June 2025.
• New data on attack rate reductions were presented at the Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI) in November 2025.
• In one study, adolescents on ORLADEYO experienced 1.85 fewer attacks/month at 18 months compared to baseline.

Managed access programs to transition patients from free to paid drug are supported by the company's financial performance and specific patient affordability initiatives.

BioCryst Pharmaceuticals, Inc. reported ORLADEYO net revenue of $156.8 million for the second quarter of 2025, representing a 45 percent year-over-year increase. The full year 2025 ORLADEYO net revenue guidance was increased to between $535-$550 million. The company expects to achieve positive EPS and positive cash flow in the second half of 2025 (2H 2025). As of March 31, 2025, the company held $315.6 million in cash, cash equivalents, and investments. Management noted favorable early signs that many more Medicare patients taking ORLADEYO are able to afford therapy due to lower copayments under the Inflation Reduction Act. Sales from the U.S. contributed 89.5 percent of global ORLADEYO net revenues in the second quarter of 2025.

BioCryst Pharmaceuticals, Inc. (BCRX) - Canvas Business Model: Channels

You're looking at how BioCryst Pharmaceuticals, Inc. gets its product, ORLADEYO, into the hands of patients, and how they manage their pipeline reach. It's a mix of a focused internal team and external partners, especially after the recent strategic shift in Europe.

Direct sales force in the U.S. and select international markets

The core of the commercial channel remains the U.S. direct sales force, which is clearly the engine driving the majority of the revenue. In the third quarter of 2025, U.S. sales made up a commanding 89% of global ORLADEYO net revenues. That team added 64 new prescribers in the U.S. during Q3 2025, a number that beat the company's two-year quarterly average. This direct engagement is crucial for a rare disease product like ORLADEYO, where physician education matters a lot. The long-term trend for patient retention shows stability, holding at approximately 60% at the one-year mark, which helps secure that recurring revenue stream. The company projects this U.S. channel will eventually support a steady state of over 2,000 patients by 2028.

Specialty pharmacies for distribution of Orladeyo

For distribution in the United States, BioCryst Pharmaceuticals relies on a single specialty pharmacy for the distribution of the approved drug product. This streamlined approach is key to managing a niche product. The effectiveness of this channel is reflected in the rapid conversion of patients to paid status; by the first quarter of 2025, the total percentage of all ORLADEYO patients on paid drug had climbed to approximately 84%, up from 73.5% at the end of 2024. This channel handles the logistics after the prescription is written by the physician.

Licensing and distribution agreements (e.g., Neopharmed Gentili in Europe)

The European channel underwent a major transformation in 2025. BioCryst Pharmaceuticals completed the sale of its European ORLADEYO business to Neopharmed Gentili, effective in late 2025. This divestiture was structured to immediately strengthen the balance sheet. Neopharmed Gentili paid an upfront amount of $250 million, with up to an additional $14 million possible from future milestones tied to Central and Eastern Europe sales. This move is expected to save BioCryst Pharmaceuticals at least $50 million in expected annual operating expenses. Before this sale, ORLADEYO had achieved acceptance in over 30 countries, supported by various commercial partners, including past agreements with Pint Pharma for pan-Latin America and Swixx for 15 markets in CEE.

Digital and patient advocacy channels for rare disease awareness

While direct spending on digital channels isn't explicitly detailed, the success in driving patient preference is a proxy for effective awareness efforts. Market research from 2025 shows a significant shift: the percentage of patients preferring oral prophylaxis for hereditary angioedema (HAE) has jumped to 70%, up from 50% in 2023. Conversely, the preference for injection/infusion treatments dropped to just 13% in 2025. This suggests strong communication efforts are resonating with the patient community regarding the convenience of an oral, once-daily treatment.

Clinical trial sites for pipeline drug development

The channel for pipeline development involves establishing clinical trial sites globally to advance investigational therapies. For avoralstat, the first clinical trial for diabetic macular edema (DME) was authorized to proceed in Australia, with initial data anticipated in 2025. Separately, the FDA cleared the Investigational New Drug (IND) application for BCX17725 for Netherton syndrome, allowing a Phase 1 trial to enroll patients in the United States, with this trial also open in Australia, expecting initial data in 2025.

Here's a quick look at the key commercial and financial metrics tied to these channels as of late 2025:

Metric	Value / Amount	Period / Context
ORLADEYO Net Revenue	$159.1 million	Q3 2025
ORLADEYO Net Revenue Growth (y-o-y)	+37 percent	Q3 2025
FY 2025 ORLADEYO Revenue Guidance	$590 to $600 million	Full Year 2025
U.S. Share of Global ORLADEYO Revenue	89%	Q3 2025
New U.S. Prescribers Added	64	Q3 2025
ORLADEYO Patients on Paid Drug	Approx. 84%	Q1 2025
European Business Sale Upfront Proceeds	$250 million	June 2025 Agreement
Estimated Annual Operating Expense Savings from EU Sale	At least $50 million	Annually

The wholesale acquisition cost for a pack of ORLADEYO is listed at $44,484.33. Also, the company's cash position as of September 30, 2025, was reported at $269 million, with a pro forma balance of $294 million.

BioCryst Pharmaceuticals, Inc. (BCRX) - Canvas Business Model: Customer Segments

You're looking at the core groups BioCryst Pharmaceuticals, Inc. (BCRX) serves with its rare disease portfolio, primarily centered around ORLADEYO (berotralstat) for Hereditary Angioedema (HAE). These segments drive the company's revenue, which for full-year 2025 is guided to be between $580 million and $600 million in global net ORLADEYO revenue, a significant increase from the initial guidance of $535 million to $550 million.

Adult and adolescent patients with Hereditary Angioedema (HAE)

This is the established, core customer base for the capsule formulation of ORLADEYO, which is indicated for prophylaxis in patients aged 12 years and older. The commercial success in this segment is clear from the revenue figures; for instance, the third quarter of 2025 saw ORLADEYO net revenue hit $159.1 million, a 37% increase year-over-year. The U.S. market remains the largest contributor, accounting for 89.5% of global ORLADEYO net revenues in the second quarter of 2025. Furthermore, the preference for oral therapy among HAE treaters in the U.S. is strong, with 70% in a Q1 2025 survey describing a strong preference for an oral prophylaxis therapy, up from 50% in 2023.

Pediatric HAE patients aged 2-11 years old (new segment via granules)

This represents a critical expansion segment, moving beyond the initial approved age group. BioCryst Pharmaceuticals, Inc. is actively targeting this group with an oral granule formulation of ORLADEYO. The New Drug Application (NDA) for this formulation was under review with the U.S. FDA, with a Prescription Drug User Fee Act (PDUFA) goal date set for December 12, 2025. The need is evident, as HAE attacks often start early; the median age at diagnosis was 2.0 years, with 82.8% of patients experiencing symptoms before age 6.

Interim data from the APeX-P trial in children aged 2 to <12 years shows compelling efficacy for the oral granules:

Metric	Data Point	Context/Timeframe
Reduction in Attacks Requiring Professional Care	86%	After 12 weeks of treatment
Mean Monthly Attack Rate Change	From 1.28 to 0.38	During the first four weeks of treatment
Patients Attack-Free at Month 12	70.4%	Ongoing APeX-P trial

The total percentage of all ORLADEYO patients on paid drug increased to approximately 84% as of Q1 2025, up from 73.5% at the end of 2024, showing successful conversion across patient types.

HAE treating physicians, including allergists and immunologists

Physicians are the key gatekeepers who prescribe ORLADEYO and will evaluate the new pediatric granule formulation. The company is actively engaging this segment through data presentations; for example, new data was presented at the American College of Allergy, Asthma & Immunology (ACAAI) meeting in November 2025. Prescription growth indicates physician adoption. In the second quarter of 2025, the number of new ORLADEYO prescribers in the U.S. increased to 69, up from 59 in the first quarter. Also, a market tracking survey of 60 HAE treaters in early 2025 showed that 97% were considering prescribing ORLADEYO.

Government and private payers (insurance companies)

Payers control access and reimbursement, which is vital for converting free-drug patients to paid status. The strong revenue growth in 2025 is explicitly attributed to moving patients from free drug to paid status at a faster rate than expected. The company's gross profit margin, reported at 64.25%, reflects strong commercial execution which supports payer negotiations. Outside the U.S., ORLADEYO was reimbursed in all major countries in Western Europe by early 2025, except for the Netherlands, with reimbursement expected in the first half of 2025. The company expects to be net income and cash flow positive for the full year 2025, signaling financial stability that supports long-term payer relationships.

Future: Patients with Netherton syndrome

This represents a future, unmet medical need segment for BioCryst Pharmaceuticals, Inc. The company is developing BCX17725, a KLK5 inhibitor, for this ultra-rare disease. Currently, there are no approved treatments for Netherton syndrome. Initial results from the Phase 1 clinical trial evaluating BCX17725 are expected by the end of 2025. Research and development expenses for Q3 2025 increased partly due to the advancement of BCX17725.

BioCryst Pharmaceuticals, Inc. (BCRX) - Canvas Business Model: Cost Structure

You're looking at the costs that drive BioCryst Pharmaceuticals, Inc.'s operations as of late 2025. It's a mix of heavy investment in the pipeline, scaling commercial operations, and major one-time strategic expenses.

High R&D expenses for advancing BCX17725 and navenibart development

Research and development spending reflects the commitment to pipeline progression. For the third quarter of 2025, Research and development expenses rose to $44.6 million, up from $41.1 million in the third quarter of 2024. This increase is directly tied to advancing BCX17725 into the clinic and executing investigational new drug (IND)-enabling activities for pre-clinical programs. While the Astria Therapeutics acquisition, expected to close in Q1 2026, brings navenibart into the portfolio, the Q3 2025 R&D spend reflects internal pipeline work.

Selling, General, and Administrative (SG&A) costs for Orladeyo commercialization

Costs related to commercializing Orladeyo (berotralstat) are a significant component, especially as the company scales. Selling, general and administrative expenses grew by 27% year-over-year in the third quarter of 2025. Looking at the second quarter of 2025, SG&A hit $87.4 million, up from $61.2 million the prior year. This growth includes costs associated with commercial progression; for instance, approximately $6.5 million in Q2 2025 was driven by Orladeyo-related regulatory, safety, quality, and manufacturing expenses that were reclassified into SG&A to reflect the drug's commercial maturity.

The full-year 2025 non-GAAP operating expense guidance, which excludes stock-based compensation and transaction-related costs, was lowered to between $430 million and $440 million.

Here are some key operating expense metrics for the recent quarters:

Metric	Q3 2025 Amount	Q2 2025 Amount	Q3 2024 Amount
Research and Development Expenses	$44.6 million	$43.4 million	$41.1 million
Selling, General, and Administrative Expenses	Not specified as GAAP total	$87.4 million	$61.2 million
Non-GAAP Operating Expenses (Excl. SBC & Transaction Costs)	Approximately $108 million	Not specified	Approximately $92 million

Manufacturing and supply chain costs for a specialty oral drug

Costs tied to the physical product, like manufacturing and supply chain, are embedded within operating expenses. As noted above, specific Orladeyo-related manufacturing expenses, totaling about $6.5 million in Q2 2025, are now captured within SG&A due to the drug's commercial status. These costs support the global commercialization of the specialty oral drug.

Acquisition costs related to the Astria Therapeutics transaction

A major cost consideration is the definitive agreement signed on October 14, 2025, to acquire Astria Therapeutics, Inc. The total enterprise value for this transaction is approximately $700 million. The consideration per share is structured as $8.55 in cash and 0.59 shares of BioCryst common stock, implying a value of $13.00 per Astria share. BioCryst secured a debt commitment letter for up to $550 million from Blackstone-managed funds to help fund the cash portion, alongside existing cash. Transaction-related costs are explicitly excluded from the non-GAAP operating expense guidance for 2025, meaning the GAAP figures will be higher when the deal closes in Q1 2026.

Interest expense, though reduced by debt retirement from European sale proceeds

Interest expense shows a clear trend of reduction due to proactive debt management. In the third quarter of 2025, interest expense was $19.7 million, a decrease of 21% from $24.8 million in the third quarter of 2024. This reduction stems partly from a $125 million partial prepayment on the Pharmakon Term Loan made during 2025, and a lower effective interest rate. Furthermore, the sale of the European Orladeyo business was completed, and the proceeds were used in October 2025 to retire all remaining Pharmakon term debt, which was approximately $200 million outstanding at that time. This action leaves the company with $0 term debt on a pro forma basis as of September 30, 2025, significantly altering the future interest cost structure.

The interest expense for Q2 2025 was $21.6 million, following a $75 million partial prepayment in April 2025.

BioCryst Pharmaceuticals, Inc. (BCRX) - Canvas Business Model: Revenue Streams

The revenue streams for BioCryst Pharmaceuticals, Inc. are heavily concentrated around its flagship product, ORLADEYO (berotralstat), supplemented by other product sales and significant one-time financial events from strategic divestitures.

The primary driver is the net product revenue from ORLADEYO sales, which the company projects to be between $590 million to $600 million for the full-year 2025, based on the retained global business excluding the European operations for the fourth quarter of 2025. This projection was raised following the third quarter of 2025 results.

The revenue streams can be broken down as follows:

• Net product revenue from ORLADEYO sales, projected at $590 million to $600 million for full-year 2025 (retained business).
• Revenue from RAPIVAB (peramivir injection) sales, which was previously guided as part of a total revenue package that implied a range of $25 million to $45 million when factoring in earlier 2025 total revenue guidance versus ORLADEYO guidance.
• Proceeds from the sale of the European ORLADEYO business, which was completed for an upfront payment of approximately $250 million.
• Potential future milestone and royalty payments from licensed assets, including up to $14 million in future milestones tied to Central and Eastern European sales from the European business divestiture.

The company has also set clear financial targets for the year, indicating a transition to sustained profitability.

Financial Metric	2025 Projection/Target	Context/Source Data Point
FY 2025 Global Net ORLADEYO Revenue (Retained Business)	$590 million to $600 million	Raised outlook following Q3 2025 results.
European ORLADEYO Business Sale Proceeds (Upfront)	$250 million	Completed transaction proceeds.
Q3 2025 Non-GAAP Net Income	$35.6 million	Actual result for the third quarter.
FY 2025 Expected Outcome	Net income and positive cash flow	Full-year target, excluding debt repayment impacts.
Potential Future Milestones (European Sale)	Up to $14 million	Tied to future sales in Central and Eastern Europe.

The completion of the European sale, which generated $250 million upfront, was strategically used to retire the remaining Pharmakon term loan balance of $199 million. The pro forma cash balance at September 30, 2025, was $294 million, which included the net proceeds from the European sale.