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INBRX-101/rhAAT-Fc

Phase 1

Alpha-1 Antitrypsin Deficiency | Small molecule | Other |Inhibrx Biosciences, Inc.|Last Updated: Sep 13, 2022

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment31
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03815396Phase 1 Study to Assess the Safety, PK and PD of INBRX-101 in Adults With Alpha-1 Antitrypsin DeficiencyPHASE1 COMPLETED 31Jul 19, 2019Aug 18, 2022Sep 13, 202210 United States, New Zealand +1
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Study Endpoints
Primary Endpoints
Frequency of adverse events of INBRX-101
Up to 7 months

Adverse events will be assessed by the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), version 4.03.

Severity of adverse events of INBRX-101
Up to 7 months

Severity of adverse events will be assessed and assigned by the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), version 4.03.

Secondary Endpoints
Area under the serum concentration time curve (AUC) of INBRX-101
Up to 7 months
Maximum observed serum concentration (Cmax) of INBRX-101
Up to 7 months
Trough observed serum concentration (Ctrough) of INBRX-101
Up to 7 months
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Part 1 Single Ascending DoseEXPERIMENTALINBRX-101 will be escalated in subjects with alpha-1 antitrypsin deficiency (AATD).
Part 2 Multiple Ascending DoseEXPERIMENTALINBRX-101 will be escalated in subjects with alpha-1 antitrypsin deficiency (AATD).
Interventions
NameTypeDescription
INBRX-101/rhAAT-FcDRUGINBRX-101 is a recombinant human alpha-1 antitrypsin (AAT) Fc fusion protein (rhAAT-Fc).
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Eligibility Criteria
Age Range18 Years — 80 Years
SexALL
Healthy VolunteersNo
Study Sites10

Inclusion Criteria: * Documented alpha-1 antitrypsin (AAT) serum concentration \<11 μM. * Diagnosis of alpha-1 antitrypsin deficiency (AATD) with any allelic combination with exception of the null/null genotype. * For subjects in Part 2 80 and 120 mg/kg cohorts ONLY: post-bronchodilator FEV1 of at ...

Countries:United StatesNew ZealandUnited Kingdom
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