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BXCL701

Phase 2

Metastatic Pancreatic Ductal Adenocarcinoma | Small molecule | Oncology |BioXcel Therapeutics, Inc.|Last Updated: Feb 12, 2026

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
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment22
FDA Designations
FAST_TRACKORPHAN_DRUG
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05558982BXCL701 and Pembrolizumab in Patients With Metastatic Pancreatic Ductal AdenocarcinomaPHASE2 ACTIVE NOT_RECRUITING 22Aug 16, 2023Nov 1, 2027Feb 12, 20263 United States
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Study Endpoints
Primary Endpoints
Progression-free survival at 18 weeks
18 weeks
Secondary Endpoints
Incidence of Adverse events
From start of intervention until 30 days following discontinuation of intervention
Objective response rate
Through study completion, on average 18 weeks
Median Duration of response (DOR)
24 months
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
BXCL701 plus PembrolizumabEXPERIMENTAL -
Interventions
NameTypeDescription
BXCL701DRUGBXCL701 0.3 mg, orally, twice a day on days 1-14 every 21 days
PembrolizumabDRUGPembrolizumab 200 mg intravenous (IV) on day 1 every 21 days.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites3

Inclusion Criteria: * Histologically-confirmed pancreatic ductal adenocarcinoma with metastatic disease (mixed histology is acceptable as long adenocarcinoma is the dominant histological subtype) * Patient must consent to two mandatory biopsies and have tumor amenable to serial core biopsies * Meas...

Countries:United States
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT05558982primaryCompletionDate: changed
LOWMay 24, 2026NCT05558982studyFirstPostDate: changed