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VSA001

Phase 1

Healthy Adult Volunteers | Small molecule | Other |Arrowhead Pharmaceuticals, Inc.|Last Updated: Mar 20, 2025

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
RandomizedDouble-BlindPLACEBO_CONTROLLEDBiomarker
Total Trials1
Total Enrollment24
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05757596Study of VSA001 Injection in Chinese Healthy Adult VolunteersPHASE1 COMPLETED 24May 17, 2023Dec 31, 2023Mar 20, 20251 China
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Study Endpoints
Primary Endpoints
Safety and tolerability
85 days

Incidence, frequency, and severity of adverse events (AEs) and serious adverse events (SAEs), and the relationship with VSA-01001. Clinically significant abnormalities in laboratory tests, vital signs, physical examination, 12-lead electrocardiograms (ECG).

Secondary Endpoints
Pharmacokinetics parameter: Cmax
48 hours
Pharmacokinetics parameter: Tmax
48 hours
Pharmacokinetics parameter: AUC0-t
48 hours
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
VSA001 injectionACTIVE_COMPARATORA single dose of active drug (VSA001, low or high dose) will be administered by subcutaneous injection on Day 1.
PlaceboPLACEBO_COMPARATORThe placebo is normal saline (0.9%) administered subcutaneously; volume matched to the corresponding VSA001 dose volume.
Interventions
NameTypeDescription
VSA001 injectionDRUGThe active drug is VSA001 injection. The active pharmaceutical ingredient (API) contained in VSA001 is a synthetic, double-stranded, hepatocyte targeted NAG-conjugated RNAi.
PlaceboDRUG0.9% Saline, volume matched
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Eligibility Criteria
Age Range18 Years — 55 Years
SexALL
Healthy VolunteersYes
Study Sites1

Inclusion Criteria: 1. The subjects voluntarily participate in this study who are able to read, understand, and sign the ICF before participation; and have a full understanding of the content, process and possible adverse reactions of the study and are able to complete the study in accordance with ...

Countries:China
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