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.
BCX17725 is being developed by BioCryst Pharmaceuticals, Inc., a biopharmaceutical company traded on the Nasdaq under the ticker symbol BCRX. The company is conducting a Phase 1 clinical trial of the drug in patients with Netherton Syndrome.
BCX17725 is in Phase 1 clinical development. It is an investigational drug and has not been approved for any use. The ongoing Phase 1 trial is recruiting participants to evaluate the safety, tolerability, pharmacokinetics, and immunogenicity of the drug.
BCX17725 is being studied in a Phase 1 clinical trial registered as NCT06539507. This randomized, double-blind, controlled study is recruiting 78 participants with Netherton Syndrome across the United States, Australia, France, Germany, and the Netherlands. The trial includes healthy volunteers and is currently active.
The specific molecular target and mechanism of action of BCX17725 have not been disclosed. It is a small molecule being developed for Netherton Syndrome, and its mechanism is under investigation in the ongoing Phase 1 clinical trial.