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Unesbulin

Phase 1

Leiomyosarcoma | Small molecule | Oncology |PTC Therapeutics, Inc.|Last Updated: Mar 15, 2024

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
UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment41
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03761095A Study of Unesbulin (PTC596) in Combination With Dacarbazine in Participants With Advanced Leiomyosarcoma (LMS)PHASE1 COMPLETED 41 — — Mar 13, 2019Feb 5, 2024Mar 15, 20244 United States
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Study Endpoints
Primary Endpoints
MTD and RP2D of Unesbulin in Combination With Dacarbazine
First 2 cycles of treatment (6 weeks)

MTD will be determined using the TITE-CRM for dose-finding. MTD is defined as the dose associated with a target probability of DLT of 0.25.

Number of Participants With Adverse Events
From screening until end of study (up to approximately 1.5 years)
Secondary Endpoints
Objective Response Rate (ORR) (Percentage of Participants With Objective Response) as Determined by the Investigator Using Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST v1.1)
From Baseline until the date of objectively documented progression per RECIST v1.1 or the date of initiation of subsequent therapy or palliative local therapy, whichever occurs first (up to approximately 1.5 years)
Time to Response as Determined by the Investigator Using RECIST v1.1
From Baseline until the date of first occurrence of CR or PR (up to approximately 1.5 years)
Duration of Response (DOR)
Time from the date of first confirmed response to the date of the first documented tumor progression or death due to any cause, whichever occurs first (up to approximately 1.5 years)
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Unesbulin and DacarbazineEXPERIMENTALParticipants will receive unesbulin orally twice weekly in combination with dacarbazine IV once every 21 days. The first participant will receive dacarbazine 1000 mg/m\^2 IV every 21 days in combination with unesbulin 200 mg tablet orally twice weekly. For subsequent participants, the dose level at which treatment is initiated will be selected based on the TITE-CRM using the most up to date dose DLT information from all participants previously treated. Participants will receive unesbulin 300 mg twice weekly in combination with dacarbazine in the expansion cohort. Treatment will continue for each participant until evidence of unacceptable toxicity, disease progression, or treatment discontinuation for another reason.
Interventions
NameTypeDescription
UnesbulinDRUGUnesbulin will be administered as per the dose and schedule specified in the arm.
DacarbazineDRUGDacarbazine will be administered as per the dose and schedule specified in the arm.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites4

Inclusion Criteria: 1. Signed consent of an Institutional Review Board (IRB)-approved informed consent form (ICF) and Health Insurance Portability and Accountability Act (HIPAA) authorization for release of personal health information (if appropriate). 2. Willingness and ability to comply with sche...

Countries:United States
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