Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
BCX17725 · 1 trial · 1 indication
Incidence of TEAEs as assessed by Common Terminology Criteria for Adverse Events (CTCAE) from screening through end-of-study (EOS) in each study part
IASI measures the severity of erythema (IASI-E) and scaling (IASI-S); the maximum sub-scores for the IASI-E and IASI-S being 24, and the maximum total IASI score being 48. Higher scores indicate worse clinical outcome.
| Arm | Type | Description |
|---|---|---|
| Part 1 - BCX17725 single dose | EXPERIMENTAL | Participants randomized to BCX17725 will receive BCX17725 as a single dose in sequential ascending dose cohorts |
| Part 1 - placebo single dose | EXPERIMENTAL | Participants randomized to placebo will receive placebo as a single dose |
| Part 2 - BCX17725 multiple doses | EXPERIMENTAL | Participants randomized to BCX17725 will receive BCX17725 as multiple doses in sequential ascending dose cohorts |
| Part 2 - placebo multiple doses | EXPERIMENTAL | Participants randomized to placebo will receive placebo as multiple doses |
| Part 3 - BCX17725 multiple doses | EXPERIMENTAL | Participants will receive BCX17725 as multiple doses |
| Part 4 - BCX17725 multiple doses | EXPERIMENTAL | Participants will receive BCX17725 as multiple doses |
| Name | Type | Description |
|---|---|---|
| BCX17725 | DRUG | BCX17725 for injection |
| Placebo | DRUG | Placebo for injection |
Key Inclusion Criteria: * Male or non-pregnant, non-lactating female aged 18 to 55 years, inclusive (Parts 1 and 2), 18 to 65 years, inclusive (Part 3), or 12 to 65 years, inclusive (Part 4) * Confirmed diagnosis of Netherton syndrome (Parts 3 and 4) * IGA score of ≥ 3 (Parts 3 and 4) and IASI scor...
BCX17725 is an investigational small molecule being developed for the treatment of Netherton Syndrome, a rare genetic skin disorder. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities. The drug is being studied to evaluate its safety, tolerability, pharmacokinetics, and immunogenicity in patients with this condition.
BCX17725 targets kallikrein-related peptidase 5 (KLK5), an enzyme involved in skin barrier function. By inhibiting KLK5, the drug aims to address the underlying pathophysiology of Netherton Syndrome. This target is considered relevant to the excessive skin shedding and inflammation characteristic of the disease.
BCX17725 is being developed by BioCryst Pharmaceuticals, Inc., a biopharmaceutical company traded on the Nasdaq under the ticker symbol BCRX. The company is conducting clinical trials to evaluate the drug's potential as a treatment for Netherton Syndrome.
BCX17725 is currently in Phase 1 clinical development. It is an investigational drug, meaning it has not yet received FDA approval or approval from other regulatory agencies. The ongoing Phase 1 trial is designed to assess the drug's safety, tolerability, pharmacokinetics, and immunogenicity in humans.
BCX17725 is being studied in a single Phase 1 clinical trial registered as NCT06539507. This randomized, double-blind, controlled study is recruiting participants with Netherton Syndrome and healthy volunteers across the United States, Australia, France, Germany, and the Netherlands. The trial aims to enroll 78 participants aged 12 years and older.
No alternative names for BCX17725 have been disclosed. The drug is identified solely by its code name BCX17725 in clinical trial registrations and by its developer, BioCryst Pharmaceuticals. It is a distinct investigational compound being evaluated specifically for Netherton Syndrome.