Therapy Detail

Therapy Name PF-03084014
Therapy Description

Nirogacestat (PF-03084014) is a small molecule that selectively binds and inhibits Gamma-secretase inhibiting its proteolytic activity on downstream targets including NOTCH receptor proteins thus blocking pathway activation in cancer (NCI Drug Dictionary).

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Drug Name Trade Name Synonyms Drug Classes Drug Description
PF-03084014 Nirogacestat Gamma secretase inhibitor 12 Nirogacestat (PF-03084014) is a small molecule that selectively binds and inhibits Gamma-secretase inhibiting its proteolytic activity on downstream targets including NOTCH receptor proteins thus blocking pathway activation in cancer (NCI Drug Dictionary).
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
NOTCH1 act mut colorectal cancer sensitive PF-03084014 Preclinical - Cell culture Actionable In a preclinical study, the gamma secretase inhibitor PF-03084014 reduced Notch1 cleavage, decreased activation of Notch targets, and increased apoptosis in colorectal cancer cell lines exhibiting increased Notch activation (PMID: 23868008). 23868008
NOTCH1 S2449fs triple-receptor negative breast cancer sensitive PF-03084014 Preclinical - Pdx Actionable In a preclinical study, PF-03084014 treatment resulted in tumor regression in patient derived xenograft animal models of triple receptor negative breast cancer harboring NOTCH1 S2449fs (PMID: 25564152). 25564152
NOTCH1 mutant triple-receptor negative breast cancer sensitive PF-03084014 Preclinical - Pdx Actionable In a preclinical study, PF-03084014 treatment resulted in tumor regression in patient-derived xenograft models of triple receptor negative breast cancer harboring NOTCH1 mutations (PMID: 25564152). 25564152
Clinical Trial Phase Therapies Title Recruitment Status
NCT01981551 Phase II PF-03084014 Phase II Trial of the Gamma-Secretase Inhibitor PF-03084014 in Adults With Desmoid Tumors/Aggressive Fibromatosis Active, not recruiting
NCT02955446 Expanded access PF-03084014 Compassionate Use Protocol for PF-03084014 in Patients With Advanced Solid Tumor Malignancies No longer available
NCT02299635 Phase II PF-03084014 A Study Evaluating PF-03084014 In Patients With Advanced Breast Cancer With Or Without Notch Alterations Terminated
NCT02109445 Phase II Nab-paclitaxel + Gemcitabine PF-03084014 Study Of PF-03084014 In Combination With Gemcitabine And Nab-Paclitaxel In Patients With Metastatic Pancreatic Adenocarcinoma Not Previously Treated With Anticancer Therapies Terminated
NCT02137564 Phase II PF-03084014 Gamma Secretase Inhibitor PF-03084014 in Treating Patients With AIDS-Associated Kaposi Sarcoma Withdrawn