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Birabresib

Phase 1

NUT Midline Carcinoma | Small molecule | Oncology |Merck & Company, Inc.|Last Updated: Jan 26, 2021

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
CONTROLLEDBiomarker
Total Trials1
Total Enrollment47
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02259114A Dose-Finding Study of Birabresib (MK-8628), a Small Molecule Inhibitor of the Bromodomain and Extra-Terminal (BET) Proteins, in Adults With Selected Advanced Solid Tumors (MK-8628-003)PHASE1 COMPLETED 47Oct 23, 2014Mar 3, 2017Jan 26, 2021 -
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Study Endpoints
Primary Endpoints
Number of Participants Who Experienced a Dose Limiting Toxicity (DLT) During Cycle 1
Up to Cycle 1 Day 21 (Up to 21 days)

A DLT was defined as any of the following toxicities that were considered by the investigator to be related to MK-8628: Hematologic toxicity: Grade 4 hematologic toxicity or febrile neutropenia, Grade 3 neutropenia with infection, Grade 3 thrombocytopenia with bleeding or lasting \>7 days; Non-hematologic toxicity: Grade 3 or 4 non-hematologic toxicity (regardless of duration) unless it was not optimally managed with supportive care, Grade 3 or 4 laboratory abnormality, with or without symptoms, lasting \>48 hours, Intolerable Grade 2 non-hematologic toxicity resulting in study drug discontinuation or delay \>7 days with or without dose reduction, Designated alanine aminotransferase (ALT) or aspartate aminotransferase (AST) liver test abnormalities; Treatment delay \>2 weeks or dose reduction requirement for initiating Cycle 2.

Secondary Endpoints
Number of Participants Who Experienced at Least One Adverse Event (AE)
Up to approximately 17.5 months (Up to 30 days after last dose of study treatment)
Number of Participants Who Discontinued Study Treatment Due to an AE
Up to approximately 16.5 months
Best Overall Response as Assessed in Solid Tumors by Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST 1.1) or in Castration-resistant Prostate Cancer (CRPC) by Prostate Cancer Clinical Trials Working Group (PCWG2) Response Criteria
Up to approximately 16.5 months
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Continuous Dosing RegimenEXPERIMENTALParticipants receive birabresib capsules once daily in a fasted state in the morning on Days 1-21 of each 21-day cycle. Starting dose for dose escalation is 80 mg.
Days 1-7 Dosing RegimenEXPERIMENTALParticipants receive birabresib capsules once daily in a fasted state in the morning on Days 1-7 of each 21-day cycle. Starting dose for dose escalation is 100 mg.
Interventions
NameTypeDescription
BirabresibDRUGBirabresib 10, 20 and/or 40 mg oral capsules
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo

Inclusion Criteria: 1. Signed informed consent obtained prior to initiation of any study-specific procedures and treatment; 2. Histologically or cytologically confirmed diagnosis of one of the following advanced or metastatic solid tumors for which standard therapy either does not exist or has prov...

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