BCX17725 / BioCryst Pharmaceuticals - LARVOL DELTA

Safety, tolerability, immunogenicity, & pharmacokinetics of bcx17725: Single and multiple ascending dose (SID 2026) - P1 | "BCX17725 demonstrated a favorable safety profile and predictable PK in HV, with dose-proportional systemic exposures resulting in penetration into the dermis and epidermis. These results provide a strong foundation for evaluation of BCX17725 as a targeted, mechanism-based therapy for NS."

Clinical • First-in-human • PK/PD data • Genetic Disorders • Inflammation • KLK5

Preclinical characterization of BCX17725, a potent inhibitor of KLK5/14, for the treatment of Netherton Syndrome (SID 2026) - "BCX17725 was 150-fold less potent in inhibiting trypsin than KLK5/14. These data demonstrate that BCX17725 potently inhibits the activities of KLK5 and KLK14 and suppresses KLK5-mediated cytokine release, supporting its development for the treatment of NS."

Preclinical • Genetic Disorders • Inflammation • Targeted Protein Degradation • C1S • CXCL8 • KLK5 • KLK7

BCX17725-101: A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725 (clinicaltrialsregister.eu) - P1/2 | N=6 | Not yet recruiting | Sponsor: Biocryst Pharmaceuticals Inc.

New P1/2 trial

BCX17725-101: A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725 (clinicaltrials.gov) - P1 | N=78 | Recruiting | Sponsor: BioCryst Pharmaceuticals | Trial completion date: Mar 2026 ➔ Dec 2026 | Trial primary completion date: Mar 2026 ➔ Oct 2026

Trial completion date • Trial primary completion date

BCX17725-101: A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725 (clinicaltrials.gov) - P1 | N=66 | Recruiting | Sponsor: BioCryst Pharmaceuticals | Trial completion date: Jul 2025 ➔ Mar 2026 | Trial primary completion date: Jul 2025 ➔ Mar 2026

Trial completion date • Trial primary completion date

BioCryst Reports Fourth Quarter and Full Year 2024 Financial Results and Upcoming Key Milestones (GlobeNewswire) - "ORLADEYO net revenue in the fourth quarter of 2024 was $124.2 million (+36.6 percent y-o-y). 73.5 percent of U.S. patients were on paid product at the end of the fourth quarter (up from 71.4 percent at the end of 2023). Sales from outside the U.S. contributed 13.9 percent of global ORLADEYO net revenues in the fourth quarter and 11.8 percent for full year 2024...ORLADEYO is now reimbursed in all major countries in Western Europe, except the Netherlands, which is expected in 1H 2025...The company has advanced BCX17725, its KLK5 inhibitor for the treatment of Netherton syndrome, into clinical trials and expects initial data from the program in 2025...In 2025, the company plans to advance avoralstat, a plasma kallikrein inhibitor, into a clinical trial of patients with diabetic macular edema (DME)."

Commercial • New trial • P1 data • Reimbursement • Diabetic Macular Edema • Genetic Disorders • Hereditary Angioedema • Rare Diseases

BCX17725-101: A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725 (clinicaltrials.gov) - P1 | N=72 | Recruiting | Sponsor: BioCryst Pharmaceuticals | Not yet recruiting ➔ Recruiting

Enrollment open

A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725 (clinicaltrials.gov) - P1 | N=72 | Not yet recruiting | Sponsor: BioCryst Pharmaceuticals

New P1 trial