BioCryst Pharmaceuticals

Pharma

Durham, North Carolina, United States · Est. 1991 · 500 employees

BioCryst Pharmaceuticals is a clinical-stage biotechnology company focused on discovering and developing novel small molecule and protein therapeutics for rare diseases, particularly those involving the complement system and viral infections.

Pipeline Programs↗

Distinct drugs in development (Preclinical–Phase 3)

Indications↗

Distinct diseases across all programs

Clinical Trials↗

All sponsored trials (all statuses)

Orange Book Patents

Across 2 drugs with upcoming expirations

Pipeline

Pipeline Snapshot

By Phase

2 drugs in development

Phase 32

Phase 11

By Therapeutic Area

top 2

Rare Disease

267%

Ophthalmology

133%

R&D Momentum

27/100

Pipeline Velocity

6/100

Trial Completion Rate

76/100

Pipeline Concentration

56/100

Pipeline Freshness

72/100

Strategic Signals

↗Active in Rare Disease but thin pipeline — potential licensing opportunity

Activity

Key Events

Dec 11, 2025FDAOrladeyo — ORLADEYO

Oct 2, 2025TrialA Study of Avoralstat In Participants With Diabetic Macular Edema

Sep 26, 2024TrialA Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725

Oct 24, 2023TrialDose Escalation of BCX10013 in Participants with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Jan 18, 2023TrialLong-term Safety of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria

Oct 25, 2022TrialBerotralstat Treatment in Children With Hereditary Angioedema

Feb 18, 2022TrialStudy to Evaluate the Safety, Tolerability of BCX9930 in Participants With Either Complement 3 Glomerulopathy (C3G), Immunoglobulin A Nephropathy (IgAN), or Primary Membranous Nephropathy (PMN)

Dec 6, 2021TrialBCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants With Inadequate Response to C5 Inhibitor Therapy

4 more events

Oct 26, 2021TrialBCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants Not Receiving Other Complement Inhibitor Therapy

Jul 23, 2021TrialOpen-label Berotralstat Access to HAE Patients Previously Enrolled in Berotralstat Studies

Dec 18, 2020TrialA Long-Term Safety Study of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Dec 3, 2020FDAOrladeyo — ORLADEYO

Details

Company Info

Company Overview

$2.5B

Mkt Cap

$300.0M

Revenue

—

Cash

CEOJon P. Stonehouse

Founded1991

Employees500

HQDurham, North Carolina, United States

Therapeutic Focus

Rare diseasesComplement system disordersAntiviral infections

Patent Expirations

4 patent groups across drugs · Source: FDA Orange Book

DrugExpires

Rapivab2 patentsFeb 12, 2027 Orladeyo12 patentsMar 9, 2035 Orladeyo10 patentsNov 1, 2039 Orladeyo10 patentsMay 1, 2040

Pipeline

Drug Pipeline

Indication

Preclinical

Phase 1

Phase 2

Phase 3

Approved

Regulatory

Regulatory Timeline

20141 event

FDADec 19

Rapivab — RAPIVAB

Application NDA206426 (NDA)

20201 event

FDADec 3

Orladeyo — ORLADEYO

Application NDA214094 (NDA)

20251 event

FDADec 11

Orladeyo — ORLADEYO

Application NDA219776 (NDA)

Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)