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PIM447

Phase 1

Myelofibrosis | Small molecule | Other |Novartis AG|Last Updated: Feb 9, 2022

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 Enrollment15
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02370706Study of the Safety of PIM447 in Combination With Ruxolitinib (INC424) and LEE011 in Patients With MyelofibrosisPHASE1 COMPLETED 15May 21, 2015Nov 9, 2020Feb 9, 20229 Australia, Canada +6
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Study Endpoints
Primary Endpoints
Incidence of dose limiting toxicities during the first cycle of study treatment
Cycle 1 (28 days)

To estimate the maximum tolerated dose and/or recommended dose for expansion for each of the following three treatment arms in patients with myelofibrosis (MF): PIM447 plus ruxolitinib (doublet), LEE011 plus ruxolitinib (doublet), PIM447 plus ruxolitinib and LEE 011 (triple combination).

Secondary Endpoints
Number of participants with adverse events/serious adverse events
Approximately 27 months (end of study)
Proportion of patients achieving ≥ 35% reduction in spleen volume by magnetic resonance imaging (MRI) at Week 24
24 weeks
Changes in ratio of mutant to wild type JAK2 alleles (i.e. allele burden)
Approximately 27 months (end of study)
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Study Design & Arms
AllocationNA
MaskingNONE
ModelPARALLEL
PurposeHEALTH_SERVICES_RESEARCH
Treatment Arms
ArmTypeDescription
Dose Escalation Arm 1EXPERIMENTAL -
Dose Escalation Arm 2EXPERIMENTAL -
Dose Escalation Arm 3EXPERIMENTAL -
Dose Expansion Arm 1EXPERIMENTAL -
Dose Expansion Arm 2EXPERIMENTAL -
Dose Expansion Arm 3EXPERIMENTAL -
Interventions
NameTypeDescription
PIM447DRUGpan-pim inhibitor
RuxolitinibDRUGJAK1/JAK2 inhibitor
LEE011DRUGCDK4/6 inhibitor
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites9

Inclusion Criteria: * Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2. * Patient must be diagnosed with JAK2V617F-positive primary or secondary MF. * Dose-escalation and Expansion parts: Patients with a \< 35% reduction in spleen volume by MRI/CT or \< 50% reduction in spleen si...

Countries:AustraliaCanadaFranceGermanyItalyNetherlandsSingaporeUnited Kingdom
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