Biocryst Pharmaceuticals Inc.
- Address
- 4505 Emperor Boulevard Suite 200
Sponsor Overview
Specializations
Hereditary Angioedema and HAE Management
Biocryst Pharmaceuticals Inc. focuses on clinical research in hereditary angioedema, with attention to long-term treatment use, ongoing safety evaluation, and symptom control in affected patients.
- Berotralstat safety
- Long-term access in HAE
- Oral therapy for HAE attacks
Its research activity includes both adult and pediatric populations, reflecting interest in treatment continuity across different age groups.
Pediatric Hereditary Angioedema
The sponsor funds studies examining HAE in children, with emphasis on how oral treatment behaves in younger patients and how it may be used in routine care.
- Pediatric pharmacokinetics
- Children aged 2 to under 12 years
- Treatment tolerability
This area includes evaluation of oral administration in pediatric subjects weighing at least 12 kg.
Netherton Syndrome
Biocryst Pharmaceuticals Inc. also supports clinical research in Netherton syndrome, a rare dermatologic and systemic disorder, with studies centered on therapeutic potential and treatment safety.
- BCX17725
- Adult and adolescent participants
- Safety and tolerability
Research in this area includes assessment of treatment exposure and serum concentration behavior in affected participants.
Pharmacokinetics and Immunogenicity
A key part of the sponsor’s clinical portfolio involves measuring pharmacokinetic properties and immunogenicity for oral therapies used in HAE and Netherton syndrome.
- Serum concentration profiling
- Oral drug exposure
- Immune response monitoring
These studies support the characterization of treatment behavior in both adult and pediatric populations.
Sponsored Trials
| Trial Name | Country | Status |
|---|---|---|
| A PHASE 1/1B STUDY TO EVALUATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND IMMUNOGENICITY OF SINGLE AND MULTIPLE ASCENDING DOSES OF BCX17725 IN HEALTHY PARTICIPANTS AND MULTIPLE DOSES OF BCX17725 IN PARTICIPANTS WITH NETHERTON SYNDROME |  France Germany The Netherlands | Recruiting |
| Evaluation of the Safety and Pharmacokinetics of Oral Berotralstat in Pediatric Patients Aged 2 to <12 Years with Hereditary Angioedema |  Austria France Germany Italy Poland Spain | Not Recruiting |
| Open-Label Study on Long-Term Safety of Berotralstat in Hereditary Angioedema Patients from Previous Clinical Trials |  Czechia France Germany Italy Poland Slovakia Spain | Recruiting |
Data & Insights
This sponsor works with 1 molecule, of which 1 is not being studied by other sponsors.
This sponsor has worked with 15 sites across their trial portfolio.
Therapeutic Focus
- 1.Congenital, Hereditary, and Neonatal Diseases and Abnormalities1 trial165 other sponsors
- 2.Hemic and Lymphatic Diseases1 trial249 other sponsors
- 3.Skin and Connective Tissue Diseases1 trial247 other sponsors
Partnered Research Centres
15 centres- Address
- 264 Rue Saint Pierre, 13005 Marseille
- Address
- 26 Avenue Du Docteur Arnold Netter, 75012 Paris
- Address
- Via Nicolo' Giustiniani 2, 35128 Padua
- Address
- Boulevard De La Chantourne, Cs 10217, La Tronche, 38043 La Tronche
- Address
- Dr. Molewaterplein 40, 3015 GD Rotterdam
- Address
- Edvarda Benese 1128/13, Jizni Predmesti, 301 00 Pilsen
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