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RAF265

Phase 1

Metastatic Melanoma | Small molecule | Oncology |Novartis AG|Last Updated: Dec 19, 2020

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 Enrollment104
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00304525A Study to Evaluate RAF265, an Oral Drug Administered to Subjects With Locally Advanced or Metastatic MelanomaPHASE1 COMPLETED 104Apr 1, 2006Nov 1, 2013Dec 19, 202011 United States, Switzerland
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Study Endpoints
Primary Endpoints
Maximum tolerated dose
at the end of dose escalation
Dose limiting toxicities
during the PK run-in phase and first cycle (28 day cycle)
Safety profile
throughout the study
Evaluate potential pharmacodynamic effects
throughout the study
Pharmacokinetic profile
throughout the study
Secondary Endpoints
Evaluate whether somatic mutations in BRAF and N-RAS genes are associated with modulation of pharmacodynamic markers and clinical response
throughout the study
Determine the response rate for BRAF mutant patients
Every 2 months
Determine the recommended phase two dose
at the end of dose escalation
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
RAF265 - Arm 1EXPERIMENTALPatients received 10mg RAF265 as a once weekly dose until progressive disease was confirmed.
RAF265 - Arm 2EXPERIMENTALRAF265 is given as a single "PK run-in" dose, a single loading dose on day 1 of cycle 1, followed by once daily maintenance doses.
RAF265 - Arm 3EXPERIMENTALPatients were treated with once weekly dosing of RAF265
RAF265 - Arm 4EXPERIMENTALPatients with locally advanced or metastatic melanoma will utilize a dose close to or at the MTD/RPTD of the liquid formulation that was determined in Arm 2.
RAF265 - Arm 5EXPERIMENTALRAF265 was administered as a continuous dose for 2 weeks followed by a dose holiday of 1 week.
Interventions
NameTypeDescription
RAF265DRUGA liquid nonaqueous oral formulation. Switched to a tablet formulations with was 60% bioavailable, relative to the liquid at 50mg dose. The liquid dose will be multiplied by a factor of 1.67 to achieve a comparable tablet dose. Tablets are available in 10mg and 50mg strengths.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites11

Inclusion Criteria: 1. Confirmed diagnosis of melanoma, locally advanced AJCC Stage IIIB to metastatic Stage IV 2. Measurable disease - at least one lesion measured in at least one dimension as ≥ 20 mm with conventional techniques or ≥ 10 mm with spiral computed tomography (CT) scan 3. ECOG perform...

Countries:United StatesSwitzerland
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Competitive Landscape -Melanoma 127 trials
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Pfizer Inc.PFE4PHASE3Encorafenib, Binimetinib, Pembrolizumab, PF-08046049, PF-07799933
IDEAYA Biosciences, Inc.IDYA4PHASE3Darovasertib, IDE196, Crizotinib, Pembrolizumab, Ipilimumab
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Moderna, Inc.MRNA1PHASE2mRNA-4157, Pembrolizumab
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Agenus Inc.AGEN1PHASE2Botensilimab, Balstilimab
ImmunityBio IncIBRX1PHASE2N-803 + Pembrolizumab
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