Last update 13 May 2026

Varegacestat

Overview

Basic Info

Drug Type
Small molecule drug
Synonyms
AL 102 (Ayala ), AL 102 (Ayala), AL102 (Ayala )
+ [3]
Action
inhibitors
Mechanism
γ-secretase inhibitors(Gamma-secretase inhibitors)
Active Indication
Originator Organization
Active Organization
Drug Highest PhaseNDA/BLA
First Approval Date-
RegulationOrphan Drug (United States), Orphan Drug (European Union), Fast Track (United States)
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Structure/Sequence

Molecular FormulaC26H25F7N4O3
InChIKeySRJNRAQUSAVENA-GSHUGGBRSA-N
CAS Registry1584647-27-7

External Link

KEGGWikiATCDrug Bank
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R&D Status

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Aggressive FibromatosisNDA/BLA
United States
29 Apr 2026
Advanced cancerPhase 1
United States
01 Nov 2013
Advanced cancerPhase 1
Australia
01 Nov 2013
Advanced cancerPhase 1
Canada
01 Nov 2013
Advanced Malignant Solid NeoplasmPhase 1
United States
01 Nov 2013
Advanced Malignant Solid NeoplasmPhase 1
Australia
01 Nov 2013
Advanced Malignant Solid NeoplasmPhase 1
Canada
01 Nov 2013
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 3
156
trctbjlwtj(ktwwmksruy) = The trial met its primary endpoint of improving PFS, demonstrating a statistically significant and clinically meaningful improvement vs. placebo, with an 84% reduction in the risk of disease progression or death. dianskhdgk (cpiwcflqiq )
Met
Positive
15 Dec 2025
Placebo
Phase 2
42
fkmufvigdz(mdefnabhyg) = zqfoizoujg xxoichqwdw (qjxrymkixe )
Positive
30 May 2025
Phase 2/3
42
mtuksptfcu(pxbvmwrfyr) = cmavyqgnlr rkmfxqiaco (udqkpyzqbq )
Positive
30 May 2025
mtuksptfcu(pxbvmwrfyr) = tjmgpdhhkr rkmfxqiaco (udqkpyzqbq )
Phase 2
42
dmlqwxvrwx(gqsrlytjql) = wyrnspawro nheciqdoug (zlavfibyja )
Positive
14 Sep 2024
AL102 2 mg intermittent
dmlqwxvrwx(gqsrlytjql) = okhskhsggh nheciqdoug (zlavfibyja )
Phase 3
-
hzajvyykvf(ebfrzhizbq) = The data showed high objective response rates, including 75% of evaluable patients (and 64% of intent-to-treat patients) in the 1.2 mg once daily arm vnykdmprqz (corruodule )
Positive
06 Feb 2024
Phase 2
42
AL102 1.2mg QD
sslaxbtpnv(sumncagpja) = mbnuxbaerr lznasqwtxh (sucojwrjnr )
Positive
31 May 2023
AL102 4mg BIW
sslaxbtpnv(sumncagpja) = rknnxyynyz lznasqwtxh (sucojwrjnr )
Phase 2/3
42
AL 102
wvaenlgheq(evvqkhoeev) = homsduiobo gjrjsfnnck (gybmitwagl )
Positive
17 Nov 2022
Phase 2/3
31
1.2 mg QD
syutmadkvi(rzichpfvdm) = neanjqfuio ggqruwpdzr (ofinvvrwrr )
-
12 Sep 2022
2 mg intermittent BIW
syutmadkvi(rzichpfvdm) = nuaxdlduvb ggqruwpdzr (ofinvvrwrr )
Phase 2/3
42
AL 102
klumnvtxjl(dqomtvchhq) = in substantially all patients who were evaluable at 16 weeks wazjmbuzuy (bhpdotnqbg )
Positive
05 Jul 2022
Phase 1
36
(Arm-A continuous daily schedule)
xqsscuvwyt(inpnhyujgi) = rhmdjahrij jgxmghqasd (nmsrxbkoxe )
Positive
01 Dec 2018
(Arm-B intermittent 2 times weekly schedule)
xqsscuvwyt(inpnhyujgi) = jjudgurrio jgxmghqasd (nmsrxbkoxe )
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Core Patent

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Clinical Trial

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Approval

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Regulation

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