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Varegacestat

Phase 1

Healthy | Small molecule | Other |Immunome, Inc.|Last Updated: Jun 8, 2026

Target and mechanism

Molecular targetgamma-secretase
Target class-Stat (Enzyme)
ModalitySmall molecule

Also known as Varegacestat Reference Formulation

Success Probability

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Market & Valuation

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Trial Design

RandomizedCONTROLLED
Total Trials1
Total Enrollment20

FDA Designations

No designations recorded

Clinical trial landscape

Varegacestat · 5 trials · 3 indications

Phase 1 5
NCT07610005A Relative Bioavailability and Bioequivalence Study of Varegacestat in Healthy ParticipantsHealthy
RECRUITING20 Analytics
NCT06841315A Varegacestat Hepatic Impairment StudyHealthy Volunteer
COMPLETED35 Analytics
NCT06845852A Phase 1 Mass Balance Study of VaregacestatHealthy Volunteer
COMPLETED6 Analytics
NCT06855264A Cardiac Liability Study of Varegacestat in Healthy ParticipantsHealthy Volunteer
COMPLETED32 Analytics
NCT06677996A Phase 1 Study to Examine the Effects of Itraconazole After Single Dose Administration of Varegacestat (AL102) to Healthy ParticipantsHealthy Volunteer
COMPLETED22 Analytics
PHASE1RECRUITING
A Relative Bioavailability and Bioequivalence Study of Varegacestat in Healthy Participants
HealthyUnlock trial analytics
PHASE1COMPLETED
A Varegacestat Hepatic Impairment Study
Healthy VolunteerUnlock trial analytics
PHASE1COMPLETED
A Phase 1 Mass Balance Study of Varegacestat
Healthy VolunteerUnlock trial analytics
PHASE1COMPLETED
A Cardiac Liability Study of Varegacestat in Healthy Participants
Healthy VolunteerUnlock trial analytics
PHASE1COMPLETED
A Phase 1 Study to Examine the Effects of Itraconazole After Single Dose Administration of Varegacestat (AL102) to Healthy Participants
Healthy VolunteerUnlock trial analytics

Study Endpoints

Primary Endpoints

Evaluate the rBA of varegacestat by AUC0-t
Approximately 3 weeks
Evaluate the rBA of varegacestat by AUC0-inf
Approximately 3 weeks
Evaluate the rBA of varegacestat by Cmax
Approximately 3 weeks
PK of varegacestat and metabolite from a single oral dose of varegacestat in hepatic impaired adult participants to matched healthy adult participants.
Up to 4 weeks

AUC0-t varegacestat and metabolite

The mass balance of varegacestat following administration of a single oral dose (containing ~100 µCi) [14C]varegacestat in healthy adult male participants.
Up to 15 days
Placebo-corrected change from baseline QTc and varegacestat and its metabolite, AL102 MTB, plasma concentrations
24 hours
Effect of itraconazole on the PK of varegacestat in healthy participants.
Up to 5 weeks

AUC0-t for varegacestat administered with and without itraconazole.

Secondary Endpoints

Assess the safety and tolerability of varegacestat by incidence of treatment emergent adverse events
Approximately 4 weeks
Assess the safety and tolerability of varegacestat by incidence of laboratory abnormalities
Approximately 3 weeks
Assess the safety and tolerability of varegacestat by incidence of changes in vital signs from baseline
Approximately 3 weeks
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Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelCROSSOVER
PurposeOTHER

Treatment Arms

ArmTypeDescription
Reference Formulation, then Test FormulationEXPERIMENTAL -
Test Formulation, then Reference FormulationEXPERIMENTAL -
Treatment ArmEXPERIMENTALSingle oral dose of varegacestat administered on study day 1
Drug: varegacestatEXPERIMENTALSingle oral dose of varegacestat administered on study Day 1
VaregacestatEXPERIMENTAL -
PlaceboPLACEBO_COMPARATOR -
MoxifloxacinACTIVE_COMPARATOR -

Interventions

NameTypeDescription
Varegacestat Reference FormulationDRUGOral administration
Varegacestat Test FormulationDRUGOral administration
varegacestatDRUGexperimental intervention
PlaceboDRUGSingle oral dose of placebo
Moxifloxacin 400 mgDRUGSingle oral dose of moxifloxacin
Itraconazole (200 mg)DRUGIntervention to evaluate DDI with experimental intervention
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Eligibility Criteria

Age Range18 Years to 55 Years
SexALL
Healthy VolunteersYes
Study Sites1

Inclusion Criteria: * Healthy, adult, female of non childbearing potential or male, 18 to 55 years of age, inclusive, at the screening visit * Male participants must follow protocol-specified contraception guidance * Continuous non-smoker who has not used nicotine and tobacco containing products fo...

Countries:United States
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Frequently asked questions about Varegacestat

What is Varegacestat Reference Formulation used for?

Varegacestat Reference Formulation is an investigational small molecule being studied in healthy volunteers. It is being evaluated in Phase 1 clinical trials to assess its bioavailability, bioequivalence, cardiac liability, mass balance, and effects in people with hepatic impairment. It is not approved for any disease and remains in early clinical development.

What does Varegacestat Reference Formulation target?

Varegacestat Reference Formulation targets gamma-secretase, an enzyme involved in protein processing. As a gamma-secretase inhibitor, it is being studied in healthy participants to understand its pharmacokinetic and safety profile. The drug is in Phase 1 development by Immunome, Inc.

Who makes Varegacestat Reference Formulation?

Varegacestat Reference Formulation is being developed by Immunome, Inc., a biopharmaceutical company traded on NASDAQ under the ticker IMNM. The company is conducting Phase 1 clinical trials of this investigational small molecule in healthy volunteers in the United States.

What phase is Varegacestat Reference Formulation in?

Varegacestat Reference Formulation is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA. Four Phase 1 trials have been conducted, with one currently recruiting and three completed, all involving healthy participants.

What clinical trials is Varegacestat Reference Formulation in?

Varegacestat Reference Formulation has four Phase 1 trials. NCT07610005 is a recruiting relative bioavailability and bioequivalence study in 20 healthy participants. NCT06855264 is a completed cardiac liability study in 32 participants. NCT06845852 is a completed mass balance study in 6 males. NCT06841315 is a completed hepatic impairment study in 35 participants.

Is Varegacestat Reference Formulation the same as varegacestat?

Varegacestat Reference Formulation is a specific formulation of the drug varegacestat, developed by Immunome, Inc. It is being tested in healthy volunteers to evaluate its bioavailability, cardiac safety, mass balance, and effects in hepatic impairment. The reference formulation is the version used in these Phase 1 clinical trials.