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LGH447

Phase 1

AML and High Risk MDS | Small molecule | Other |Novartis AG|Last Updated: Dec 17, 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 Enrollment70
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02078609A Safety and Efficacy Study of LGH447 in Patients With Acute Myeloid Leukemia (AML) or High Risk Myelodysplastic Syndrome (MDS)PHASE1 COMPLETED 70 — — Mar 20, 2014Apr 18, 2019Dec 17, 20209 United States, Australia +5
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Study Endpoints
Primary Endpoints
Incidence rate of dose limiting toxicities (DLTs) of LGH447 monotherapy arm in patients with AML or MDS and of LGH447 + midostaurin in patients with AML
28 days post study treatment

Frequency and characteristics of dose limiting toxicities

Secondary Endpoints
Number of participants with the type, frequency, and severity of adverse events (AEs) as a measure of safety and tolerability of the LGH447 monotherapy arm in patients with AML or MDS and of the LGH447 + midostaurin treatment arm in patients with AML
weekly to bi-weekly up to 1.5 years
PK parameters of the LGH447 monotherapy arm in patients with AML or MDS and of the LGH447 + midostaurin treatment arm in patients with AML
days 1, 2, 15, 16, 29, 30, 44, 57, and approximately monthly through Cycle 3
Changes between pre- and post-treatment levels of pS6RP and p4EBP1 in bone marrow aspirates and p4EBP1 in peripheral blood of the LGH447 monotherapy arm in patients with AML or MDS and of the LGH447 + midostaurin treatment arm in patients with AML
screening, days 1 and 29 up to 1.5 years
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
LGH447 monotherapy armEXPERIMENTALLGH447 monotherapy in patients with AML or MDS
LGH447 + midostaurin combination armEXPERIMENTALLGH447 + midostaurin in patients with AML
Interventions
NameTypeDescription
LGH447DRUGLGH447 in patients with AML or MDS
LGH447 + midostaurinDRUGLGH447 + midostaurin in patients with AML
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites9

Inclusion Criteria -Male or female patients ≥18 years of age who present with one of the following: LGH447 monotherapy arm * Refractory/Relapsed AML following no more than 2 prior therapies, or in previously untreated AML patients who are not candidates for standard therapy. * High and very high ...

Countries:United StatesAustraliaFranceGermanyItalyJapanNetherlands
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