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Mocetinostat

Phase 2

Urothelial Carcinoma | Small molecule | Oncology |Bristol-Myers Squibb Company|Last Updated: Jul 17, 2026

Target and mechanism

Molecular targetHDAC11, HDAC3, HDAC1, HDAC2, HDAC8
Target classInhibitor
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment17

FDA Designations

No designations recorded

Clinical trial landscape

Mocetinostat · 3 trials · 3 indications

Phase 2 1Phase 1 2
NCT02236195Study of Mocetinostat in Patients With Urothelial Carcinoma Having Inactivating Alterations of Specific GenesUrothelial Carcinoma
COMPLETED17 Analytics
PHASE2COMPLETED
Study of Mocetinostat in Patients With Urothelial Carcinoma Having Inactivating Alterations of Specific Genes
Urothelial CarcinomaUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of patients experiencing tumor size reduction
Up to 4 months

Tumors will be measured using radiographic scans. Tumor size reduction and overall disease response will be categorized according to the Response Evaluation Criteria In Solid Tumors (RECIST 1.1).

To describe any dose-limiting toxicity (DLT)
1 year

Percentage of subjects with dose-limiting toxicities (DLTs) as assessed by NCI CTCAE (Version 4.03)

To determine the maximum tolerated dose (MTD) or highest protocol defined doses (in the absence of exceeding the MTD)
1 year

The MTD is the highest dose associated with first-cycle DLT in \< 33% of subjects

Number of subjects with adverse events, including pericardial events, as a measure of safety
6 months

Secondary Endpoints

Number of patients experiencing adverse events
Up to 12 months
Peak blood plasma concentration of mocetinostat
Up to 48 hours
To determine the Recommended Phase 2 Dose (RP2D) for mocetinostat in combination with vinorelbine
1 year
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
MocetinostatEXPERIMENTALMocetinostat (MGCD0103) oral capsules three times weekly, 70 mg doses in first month, with increase to 90 mg doses if tolerated
Treatment (vinorelbine, mocetinostat)EXPERIMENTALParticipants receive mocetinostat in combination with vinorelbine
Mocetinostat and azacitidineEXPERIMENTALDrug: Mocetinostat (MGCD0103) Mocetinostat (a histone deacetylase \[HDAC\] inhibitor) 70 mg or 90 mg dose, oral capsules 3 times weekly beginning on day 5 for 10 doses in each 28 day cycle Drug: Azacitidine (Vidaza) Azacitidine (a hypomethylating agent \[HMA\]) 75 mg/m2 dose, by intravenous (IV) infusion or subcutaneous (SC) injection beginning on day 1 for 7 doses in each 28 day cycle

Interventions

NameTypeDescription
MocetinostatDRUG -
VinorelbineDRUGGiven IV
AzacitidineDRUGAzacitidine (a hypomethylating agent \[HMA\]) 75 mg/m2 dose, by intravenous (IV) infusion or subcutaneous (SC) injection beginning on day 1 for 7 doses in each 28 day cycle
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites14

Inclusion Criteria: * Diagnosis of urothelial carcinoma * Metastatic or locally advanced disease * Prior chemotherapy that included a platinum agent * Test results showing genetic change in tumor gene for CREBBP and/or EP300 * At least one tumor that can be measured Exclusion Criteria: * Uncontro...

Countries:United States
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Frequently asked questions about Mocetinostat

What is Mocetinostat used for?

Mocetinostat is an investigational small molecule being studied for the treatment of Myelodysplastic Syndrome, Rhabdomyosarcoma, and Urothelial Carcinoma. It is in clinical development for these oncology indications, though it is not yet approved by the FDA.

What does Mocetinostat target?

Mocetinostat is a histone deacetylase (HDAC) inhibitor that targets HDAC1, HDAC2, HDAC3, HDAC8, and HDAC11. By inhibiting these enzymes, it is being studied for its potential effects in treating certain cancers.

Who makes Mocetinostat?

Mocetinostat is being developed by Bristol-Myers Squibb Company, which trades under the ticker symbol BMY on the stock exchange. The company is conducting clinical trials to evaluate the drug's safety and efficacy in various cancer types.

What phase is Mocetinostat in?

Mocetinostat is in Phase 1 and Phase 2 clinical trials. It is an investigational drug still in clinical development and has not received FDA approval. The trials are studying its use in Myelodysplastic Syndrome, Rhabdomyosarcoma, and Urothelial Carcinoma.

What clinical trials is Mocetinostat in?

Mocetinostat has been studied in several clinical trials, including NCT02018926 for Myelodysplastic Syndrome, NCT02236195 for Urothelial Carcinoma, and NCT04299113 for Rhabdomyosarcoma. These trials have been completed or are active but not recruiting.

Is Mocetinostat the same as other HDAC inhibitors?

Mocetinostat is a specific HDAC inhibitor that targets HDAC1, HDAC2, HDAC3, HDAC8, and HDAC11. It is distinct from other HDAC inhibitors due to its unique target profile, though it shares the same class of mechanism.