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SAR302503

Phase 3

Hematopoietic Neoplasm | Small molecule | Oncology |Bristol-Myers Squibb Company|Last Updated: Mar 5, 2025

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
RandomizedDouble-BlindCONTROLLEDBiomarker
Total Trials4
Total Enrollment497
FDA Designations
No designations recorded
Clinical Trials (4)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01437787Phase III Study of SAR302503 in Intermediate-2 and High Risk Patients With MyelofibrosisPHASE3 COMPLETED 289 — — Dec 1, 2011Jun 1, 2014Mar 5, 2025101 United States, Australia +23
NCT01523171Phase II, Open Label, Single Arm Study of SAR302503 In Myelofibrosis Patients Previously Treated With RuxolitinibPHASE2 COMPLETED 97 — — Apr 1, 2012Apr 1, 2014Mar 5, 202542 United States, Austria +8
NCT01420783Study With SAR302503 in Patients With Polycythemia Vera or Essential ThrombocythemiaPHASE2 COMPLETED 81 — — Oct 1, 2011May 1, 2014Mar 5, 202535 United States, Australia +7
NCT01420770Phase 2 Study of SAR302503 in Patients With MyelofibrosisPHASE2 COMPLETED 30 — — Aug 1, 2011Apr 1, 2014Mar 5, 20254 United States
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Study Endpoints
Primary Endpoints
Response Rate (RR), defined as the proportion of patients who have a ≥35% reduction in volume of spleen size at the end of Cycle 6, and confirmed 4 weeks thereafter
6 months
Response Rate (RR), defined as the proportion of subjects who have a ≥35% reduction from baseline in volume of spleen at the end of Cycle 6 as measured by Magnetic Resonance Imaging (MRI) (or CT scan in subjects with contraindications for MRI)
6 months
Dose Ranging Phase: Proportion of PV patients with absence of phlebotomy and hematocrit below 45% and proportion of ET patients with a platelet count ≤ 400 x 10x9/L for a minimum of 3 months during the first 8 cycles of therapy.
2 years
PV Dose Expansion Phase: Proportion of PV patients with absence of phlebotomy eligibility beginning at Day 1 of Cycle 4 visit and continuing through Day 1 of Cycle 6 visit.
2 years
ET Dose Ranging Phase (only 600 mg dose group): Proportion of ET patients with a platelet count ≤400 x 10x9/L beginning at Day 1 of Cycle 4 visit and continuing through Day 1 of Cycle 6 visit.
2 years
The percent change in spleen volume based on MRI at the end of Cycle 3 relative to baseline
1 year
Secondary Endpoints
Symptom Response Rate (SRR): Proportion of patients with ≥50% reduction from baseline to the end of Cycle 6 in the total symptom score.
6 months
OS (overall survival) of either 400 mg/day or 500 mg/day of IMP as compared to placebo.
approximately 5 years
PFS (progression free survival) of either 400 mg/day or 500 mg/day of IMP as compared to placebo.
approximately 5 years
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelCROSSOVER
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Placebo comparatorPLACEBO_COMPARATORonce daily X 28 days, orally, empty stomach, approximately same time each day
SAR302503 400 mgEXPERIMENTALonce daily X 28 days, orally, empty stomach, approximately same time each day
SAR302503 500 mgEXPERIMENTALonce daily X 28 days, orally, empty stomach, approximately same time each day
SAR302503 100 mgEXPERIMENTALonce daily X 28 days
SAR302503 200 mgEXPERIMENTALonce daily X 28 days
SAR302503 600 mgEXPERIMENTALonce daily X 28 days
SAR02503 300 mg qdEXPERIMENTALdaily X 28 days
SAR302503 400 mg qdEXPERIMENTALdaily X 28 days
SAR302503 500 mg qdEXPERIMENTALdaily X 28 days
Interventions
NameTypeDescription
SAR302503DRUGPharmaceutical form:capsule Route of administration: oral
PlaceboDRUGPharmaceutical form:capsule Route of administration: oral
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites101

Inclusion criteria: * Diagnosis of Primary Myelofibrosis (MF) or Post-Polycythemia Vera MF or Post-Essential Thrombocythemia MF, according to the 2008 World Health Organization and International Working Group of Myelofibrosis Research and Treatment (IWG-MRT) criteria. * MF classified as high-risk o...

Countries:United StatesAustraliaAustriaBelgiumBrazilCanadaFranceGermanyHungaryIrelandIsraelItalyLithuaniaMexicoPolandPortugalRomaniaRussiaSingaporeSouth AfricaSouth KoreaSpainSwedenTaiwanUnited KingdomNetherlands
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