A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725

BioCryst

Status and phase: Enrolling; Phase 1

Conditions: Netherton Syndrome

Treatments: Drug: Placebo; Drug: BCX17725

Study type: Interventional

Funder types: Industry

Identifiers

NCT06539507

2025-521973-16 (EudraCT Number)

U1111-1303-9510 (Other Identifier)

BCX17725-101

Details and patient eligibility

About

This is a first-in-human, Phase 1/1b, 4-part study that includes the evaluation of safety, tolerability, pharmacokinetics (PK), and immunogenicity of BCX17725 when administered via single and multiple doses in healthy adult participants (Parts 1 and 2), and multiple doses in adult participants with Netherton syndrome (Part 3). In Part 4, the effectiveness, safety, and tolerability of BCX17725 when administered via multiple IV and/or SC doses through 12 weeks will be evaluated in adult and adolescent participants with Netherton syndrome.

Full description

Parts 1 and 2 are randomized, placebo-controlled, single-ascending-dose (SAD) and multiple-ascending-dose (MAD) study parts, respectively, in healthy participants. Part 3 will evaluate multiple dose administrations in participants with Netherton syndrome in an open-label design. Part 4 will evaluate multiple administrations of BCX17725 in participants with Netherton syndrome in an open-label study design over 12 weeks, with an 8-week post-treatment follow-up period.

Enrollment: 78 estimated patients

Sex: All

Ages: 12 to 65 years old

Volunteers: Accepts Healthy Volunteers

Inclusion and exclusion criteria

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 score of ≥ 16 (Part 4)
• BMI between 18 and 30 kg/m^2, inclusive (Parts 1 and 2)
• Estimated glomerular filtration rate (eGFR) of ≥ 90 mL/min/1.73 m^2 (Parts 1 and 2) or ≥ 60 mL/min/1.73 m^2 (Part 3)
• Agree to follow the protocol contraception requirements from screening until 90 days after the last dose of study drug
• In the opinion of the investigator, expected to adequately comply with all required study procedures and restrictions for the duration of the study

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Double Blind

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78 participants in 6 patient groups

Part 1 - BCX17725 single dose

Experimental group

Description:

Participants randomized to BCX17725 will receive BCX17725 as a single dose in sequential ascending dose cohorts

Treatment:

Drug: BCX17725

Part 1 - placebo single dose

Experimental group

Description:

Participants randomized to placebo will receive placebo as a single dose

Treatment:

Drug: Placebo

Part 2 - BCX17725 multiple doses

Experimental group

Description:

Participants randomized to BCX17725 will receive BCX17725 as multiple doses in sequential ascending dose cohorts

Treatment:

Drug: BCX17725

Part 2 - placebo multiple doses

Experimental group

Description:

Participants randomized to placebo will receive placebo as multiple doses

Treatment:

Drug: Placebo

Part 3 - BCX17725 multiple doses

Experimental group

Description:

Participants will receive BCX17725 as multiple doses

Treatment:

Drug: BCX17725

Part 4 - BCX17725 multiple doses

Experimental group

Description:

Participants will receive BCX17725 as multiple doses

Treatment:

Drug: BCX17725