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Therapy Name Alpelisib
Synonyms
Therapy Description

Piqray (alpelisib) is a selective PIK3CA inhibitor that blocks activation of the PI3K signaling pathway and inhibits tumor growth (PMID: 23726034). Piqray (alpelisib) is FDA approved in combination with fulvestrant for use in postmenopausal women, and men, with advanced or metastatic hormone receptor (HR)-positive, ERBB2 (HER2)-negative, PIK3CA-mutant breast cancer (FDA.gov).

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Drug Name Trade Name Synonyms Drug Classes Drug Description
Alpelisib Piqray BYL719 PIK3CA inhibitor 18 Piqray (alpelisib) is a selective PIK3CA inhibitor that blocks activation of the PI3K signaling pathway and inhibits tumor growth (PMID: 23726034). Piqray (alpelisib) is FDA approved in combination with fulvestrant for use in postmenopausal women, and men, with advanced or metastatic hormone receptor (HR)-positive, ERBB2 (HER2)-negative, PIK3CA-mutant breast cancer (FDA.gov).

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Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
PIK3CA E545K lung squamous cell carcinoma sensitive Alpelisib Preclinical - Cell culture Actionable In a preclinical study, Alpelisib (BYL719) induced apoptosis, inhibited cell proliferation, migration, and invasion of lung squamous cell carcinoma cells over expressing PIK3CA E545K in culture (PMID: 26013318). 26013318
PIK3CA mutant Advanced Solid Tumor sensitive Alpelisib Preclinical - Cell line xenograft Actionable In a preclinical study, PIK3CA mutations were associated with sensitivity to Alpelisib (BYL719) in human tumor cell lines in culture and in xenograft models (PMID: 24608574). 24608574
PIK3CA mutant Advanced Solid Tumor sensitive Alpelisib Phase I Actionable In a Phase I trial, Alpelisib (BYL719) demonstrated safety and efficacy in patients with advanced solid tumor harboring PIK3CA mutations, resulted in a disease control rate of 58.2% (78/134, 1 complete response (CR), 7 partial response (PR), 70 stable disease (SD)), and a clinical benefit rate (CR+PR+SD>24 weeks) of 15.7% (21/134) (PMID: 29401002; NCT01219699). 29401002
PIK3CA E545K breast cancer sensitive Alpelisib Preclinical - Cell culture Actionable In a preclinical study, Alpelisib (BYL719) inhibited survival of transformed breast epithelial cell lines overexpressing PIK3CA E545K in culture (PMID: 26627007). 26627007
PIK3CA H1047L breast cancer sensitive Alpelisib Preclinical - Cell culture Actionable In a preclinical study, Alpelisib (BYL719) inhibited survival of transformed breast epithelial cell lines overexpressing PIK3CA H1047L in culture (PMID: 26627007). 26627007
PIK3CA E726K PIK3CA H1047R breast cancer sensitive Alpelisib Preclinical - Cell culture Actionable In a preclinical study, breast epithelial cells expressing PIK3CA E726 and PIK3CA H1047R in cis demonstrated increased sensitivity to growth inhibition by Piqray (Alpelisib) treatment compared to cells expressing individual PIK3CA mutations in culture, while cells expressing PIK3CA E726 and PIK3CA H1047R in trans did not (PMID: 31699932). 31699932
PIK3CA H1047R Advanced Solid Tumor predicted - sensitive Alpelisib Preclinical - Cell culture Actionable In a preclinical study, Piqray (Alpelisib) decreased Akt phosphorylation and inhibited PI3K signaling, and demonstrated dose-dependent inhibition of survival in human pluripotent stem cells harboring PIK3CA H1047R in culture (PMID: 30948643). 30948643
PIK3CA mutant endometrial cancer sensitive Alpelisib Case Reports/Case Series Actionable In a Phase I trial, Alpelisib (BYL719) treatment resulted in 1 complete response and 1 partial response in patients with endometrial cancer harboring PIK3CA mutations (PMID: 29401002; NCT01219699). 29401002
PIK3CA H450_P458del breast cancer sensitive Alpelisib Preclinical - Cell culture Actionable In a preclinical study, transformed breast epithelial cells expressing PIK3CA H450_P458del were sensitive to treatment with Alpelisib (BYL719) in culture, demonstrating growth inhibition and decreased invasiveness of acini (PMID: 29284706). 29284706
BRAF mut PTEN inact mut melanoma decreased response Alpelisib Preclinical Actionable In a preclincal study, melanoma cell lines harboring BRAF mutations and PTEN inactivating mutations were less sensitive to Alpelisib (BYL719)-induced growth inhibition in culture (PMID: 26577700). 26577700
PIK3CA Q75E head and neck squamous cell carcinoma predicted - sensitive Alpelisib Case Reports/Case Series Actionable In clinical case study, treatment with Piqray (alpelisib) resulted in a partial response with 73% tumor shrinkage in a patient with head and neck squamous cell carcinoma (HNSCC) harboring PIK3CA Q75E, and in a preclinical analysis, resulted in inhibition of proliferation and increased apoptosis in a HNSCC cell line expressing PIK3CA Q75E in culture (PMID: 34779417). 34779417
PIK3CA E545K Advanced Solid Tumor sensitive Alpelisib Preclinical - Pdx Actionable In a preclinical study, Alpelisib (BYL719) inhibited PIK3CA E545K and showed anti-tumor activity in patient-derived xenograft models harboring a PIK3CA E545K mutation (PMID: 24608574). 24608574
PTEN C136Y breast cancer resistant Alpelisib Preclinical - Cell culture Actionable In a preclinical study, breast cancer cells harboring PTEN C136Y demonstrated resistance to Alpelisib (BYL719) in culture (PMID: 27604488). 27604488
PIK3CA H1047R breast cancer sensitive Alpelisib Preclinical Actionable In a preclinical study, Alpelisib (BYL719) inhibited survival of transformed breast epithelial cell lines overexpressing PIK3CA H1047R in culture (PMID: 26627007). 26627007
PIK3CA over exp lung squamous cell carcinoma sensitive Alpelisib Preclinical - Cell line xenograft Actionable In a preclinical study, Alpelisib (BYL719) induced apoptosis, inhibited cell proliferation, migration, and invasion of lung squamous cell carcinoma cells over expressing wild-type PIK3CA in culture, and inhibited tumor growth in cell line xenograft models (PMID: 26013318). 26013318
PIK3CA mutant Her2-receptor negative breast cancer sensitive Alpelisib Phase I Actionable In a Phase I trial, Alpelisib (BYL719) treatment resulted in a disease control rate of 60.9% (14/23, 0 complete response (CR), 1 partial response (PR), 13 stable disease (SD)), and a clinical benefit rate (CR+PR+SD>24 weeks) of 17.4% (4/23) in patients with ER-positive, ERBB2 (HER2)-negative breast cancer harboring PIK3CA mutations (PMID: 29401002; NCT01219699). 29401002
PIK3CA N1068Kfs*5 PTEN Y174H PTEN K263* colorectal cancer predicted - sensitive Alpelisib Preclinical - Patient cell culture Actionable In a preclinical study, Piqray (alpelisib) inhibited cell proliferation in a patient-derived colorectal cancer organoid harboring PIK3CA N1068Kfs*5, PTEN Y174H, and PTEN K263* in culture (PMID: 32376656). 32376656
PIK3CA E542K breast cancer sensitive Alpelisib Preclinical - Cell culture Actionable In a preclinical study, Alpelisib (BYL719) inhibited survival of transformed breast epithelial cell lines overexpressing PIK3CA E542K in culture (PMID: 26627007). 26627007
PIK3CA E545K PIK3CA E726K breast cancer sensitive Alpelisib Preclinical - Cell culture Actionable In a preclinical study, breast epithelial cells expressing PIK3CA E545K and PIK3CA E726K in cis demonstrated increased sensitivity to growth inhibition by Piqray (Alpelisib) treatment compared to cells expressing individual PIK3CA mutations in culture (PMID: 31699932). 31699932
ARID1A dec exp urinary bladder cancer sensitive Alpelisib Preclinical - Cell culture Actionable In a preclinical study, Piqray (alpelisib) inhibited viability of bladder cancer cell lines with ARID1A knockdown in culture (PMID: 35852858). 35852858
PIK3CA E545K cervical cancer predicted - sensitive Alpelisib Case Reports/Case Series Actionable In a Phase I trial, treatment with Piqray (alpelisib) resulted in an objective response rate of 33% (2/6; all partial responses), disease control rate of 100% (6/6), with stable disease in 5 patients, in cervical cancer patients harboring PIK3CA mutations, including PIK3CA E545K (PMID: 35311394). 35311394
PIK3CA E453Q PIK3CA H1047R breast cancer sensitive Alpelisib Preclinical - Cell culture Actionable In a preclinical study, breast epithelial cells expressing PIK3CA E543Q and PIK3CA H1047R in cis demonstrated increased sensitivity to growth inhibition by Piqray (Alpelisib) treatment compared to cells expressing individual PIK3CA mutations in culture (PMID: 31699932). 31699932
PIK3CA mutant head and neck cancer sensitive Alpelisib Phase I Actionable In a Phase I trial, Alpelisib (BYL719) treatment resulted in a disease control rate of 68.4% (13/19, 0 complete response (CR), 0 partial response (PR), 13 stable disease (SD)), and a clinical benefit rate (CR+PR+SD>24 weeks) of 10.5% (2/19) in patients with head and neck cancer harboring PIK3CA mutations (PMID: 29401002; NCT01219699). 29401002
PIK3CA E545K PIK3CA M1043L breast cancer sensitive Alpelisib Preclinical - Cell culture Actionable In a preclinical study, breast epithelial cells expressing PIK3CA E545K and PIK3CA M1043L in cis demonstrated increased sensitivity to growth inhibition by Piqray (Alpelisib) treatment compared to cells expressing individual PIK3CA mutations in culture (PMID: 31699932). 31699932
PIK3CA E542K PIK3CA amp lung squamous cell carcinoma sensitive Alpelisib Preclinical - Pdx Actionable In a preclinical study, Alpelisib (BYL719) treatment resulted in response in a lung squamous cell carcinoma patient-derived xenograft (PDX) model harboring PIK3CA E542K and PIK3CA amplification (PMID: 30093452). 30093452
PIK3CA mutant colorectal cancer sensitive Alpelisib Phase I Actionable In a Phase I trial, Alpelisib (BYL719) treatment resulted in a disease control rate of 34.3% (12/35, 0 complete response (CR), 2 partial response (PR), 10 stable disease (SD)), and a clinical benefit rate (CR+PR+SD>24 weeks) of 8.6% (3/35) in patients with colorectal cancer harboring PIK3CA mutations (PMID: 29401002; NCT01219699). 29401002
PIK3CA M1043V head and neck squamous cell carcinoma predicted - sensitive Alpelisib Preclinical - Pdx & cell culture Actionable In a preclinical study, treatment with Piqray (alpelisib) resulted in inhibition of proliferation and increased apoptosis in a head and neck squamous cell carcinoma (HNSCC) cell line expressing PIK3CA M1043V in culture and led to inhibition of tumor growth and decreased phosphorylated Akt levels in a patient-derived xenograft (PDX) model of HNSCC with PIK3CA M1043V (PMID: 34779417). 34779417
PTEN L152P PTEN neg salivary gland carcinoma resistant Alpelisib Preclinical - Cell culture Actionable In a preclinical study, salivary ductal carcinoma cells harboring PTEN L152P and loss of Pten protein expression were resistant to Piqray (Alpelisib)-induced inhibition of Akt phosphorylation and proliferation in culture (PMID: 30289966). 30289966
PIK3CA L10_P18del PIK3CA E545K gastrointestinal stromal tumor predicted - sensitive Alpelisib Case Reports/Case Series Actionable In a clinical case study, Piqray (alpelisib) treatment resulted in symptom improvement and stable disease maintained for more than 12 months in an adolescent patient with SDH-deficient gastrointestinal stromal tumor (GIST) harboring PIK3CA E545K and PIK3CA L10_P18del (PMID: 35917491). 35917491
PTEN loss breast cancer resistant Alpelisib Preclinical - Cell culture Actionable In a preclinical study, breast cancer cells harboring PTEN loss demonstrated resistance to Alpelisib (BYL719) in culture (PMID: 27604488). 27604488
PIK3CA N1044K colorectal cancer predicted - sensitive Alpelisib Preclinical - Patient cell culture Actionable In a preclinical study, Piqray (alpelisib) inhibited cell proliferation in a patient-derived colorectal cancer organoid harboring PIK3CA N1044K, along with KRAS G12D, in culture (PMID: 32376656). 32376656
PIK3CA wild-type Her2-receptor negative breast cancer no benefit Alpelisib Phase I Actionable In a Phase I trial, Alpelisib (BYL719) treatment resulted in no clinical benefit in 4 patients with ER-positive, ERBB2 (HER2)-negative breast cancer harboring PIK3CA mutations (PMID: 29401002; NCT01219699). 29401002
PIK3CA G1049R breast cancer sensitive Alpelisib Preclinical Actionable In a preclinical study, Alpelisib (BYL719) inhibited survival of transformed breast epithelial cell lines overexpressing PIK3CA G1049R in culture (PMID: 26627007). 26627007
PIK3CA Q546K breast cancer sensitive Alpelisib Preclinical - Cell culture Actionable In a preclinical study, Alpelisib (BYL719) inhibited survival of transformed breast epithelial cell lines overexpressing PIK3CA Q546K in culture (PMID: 26627007). 26627007
PIK3CA mut PTEN loss breast cancer resistant Alpelisib Case Reports/Case Series Actionable In a clinical case study, a breast cancer patient harboring a PIK3CA mutation developed resistance to Alpelisib (BYL719) treatment and progressive disease after initial response, accompanied by a loss of PTEN, and the corrresponding patient-derived xenograft model demonstrated resistance to Alpelisib (BYL719)-induced inhibition of tumor growth (PMID: 25409150). detail... 25409150
PIK3CA N345K breast cancer sensitive Alpelisib Preclinical - Cell culture Actionable In a preclinical study, Alpelisib (BYL719) inhibited survival of transformed breast epithelial cell lines overexpressing PIK3CA N345K in culture (PMID: 26627007). 26627007
PIK3CA H1047R lung squamous cell carcinoma sensitive Alpelisib Preclinical - Cell culture Actionable In a preclinical study, Alpelisib (BYL719) induced apoptosis, inhibited cell proliferation, migration, and invasion of lung squamous cell carcinoma cells over expressing PIK3CA H1047R in culture (PMID: 26013318). 26013318
PIK3CA act mut lung squamous cell carcinoma predicted - sensitive Alpelisib Preclinical - Pdx Actionable In a preclinical study, Alpelisib (BYL719) treatment resulted in responses in multiple lung squamous cell carcinoma patient-derived xenograft (PDX) models harboring PIK3CA E545K or E542K mutations, including models with PIK3CA amplification or PTEN loss (PMID: 30093452). 30093452
PTEN A126G prostate cancer sensitive Alpelisib Preclinical - Cell culture Actionable In a preclinical study, Alpelisib (BYL719) inhibited Akt signaling and cell migration in prostate cancer cells expressing PTEN A126G in culture (PMID: 26504226). 26504226
FGFR1 amp estrogen-receptor positive breast cancer resistant Alpelisib Preclinical - Cell culture Actionable In a preclinical study, an estrogen-receptor positive breast cancer cell line harboring an FGFR1 amplification demonstrated resistance to Alpelisib (BYL719) in culture (PMID: 27126994). 27126994
PIK3CA mutant cervical cancer predicted - sensitive Alpelisib Case Reports/Case Series Actionable In a Phase I trial, Alpelisib (BYL719) treatment resulted in partial response in 3 patients with cervical cancer harboring PIK3CA mutations (PMID: 29401002; NCT01219699). 29401002
PIK3CA N345K head and neck squamous cell carcinoma predicted - sensitive Alpelisib Preclinical - Cell culture Actionable In a preclinical study, treatment with Piqray (alpelisib) resulted in inhibition of proliferation and increased apoptosis in a head and neck squamous cell carcinoma cell line expressing PIK3CA N345K in culture (PMID: 34779417). 34779417
PIK3CA E542K PTEN loss lung squamous cell carcinoma sensitive Alpelisib Preclinical - Pdx Actionable In a preclinical study, Alpelisib (BYL719) treatment resulted in response in a lung squamous cell carcinoma patient-derived xenograft (PDX) model harboring PIK3CA E542K and PTEN loss (PMID: 30093452). 30093452

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Clinical Trial Phase Therapies Title Recruitment Status Covered Countries Other Countries
NCT05238831 Phase II Vemurafenib Vinorelbine Palbociclib Irinotecan Alpelisib Capecitabine Atezolizumab Paclitaxel Entrectinib Cobimetinib Nab-paclitaxel Fulvestrant Carboplatin Olaparib Pertuzumab/trastuzumab/hyaluronidase-zzxf Niraparib Vismodegib Alectinib Letrozole Ado-trastuzumab emtansine Bevacizumab Eribulin Anastrozole SMMART Adaptive Clinical Treatment (ACT) Trial Not yet recruiting USA 0
NCT03138070 Phase I Alpelisib A Window of Opportunity Study to Assess the Modulation of Biomarkers in Head and Neck Squamous Cell Cancer (HNSCC) By Preoperative Treatment With BYL719 Active, not recruiting CAN 0
NCT02537223 Phase I Alpelisib Cisplatin Phase I Study of BYL719 in Combination With Cisplatin and Radiotherapy in Patients With Squamous Cell Head and Neck Cancer Completed CAN 0
NCT03941782 Phase 0 Alpelisib Compassionate Use of BYL 719 Alpelisib Available USA 0
NCT03601507 Phase II Alpelisib Alpelisib in Treating Participants With Transorally Resectable HPV-Associated Stage I-IVA Oropharyngeal Cancer Recruiting USA 0
NCT05101564 Phase II Amcenestrant Alpelisib Infigratinib Tamoxifen Letrozole Amcenestrant + Letrozole Umbrella Trial Testing Integrative Subtype-Targeted Therapeutics in HR+ /HER2-Negative Breast Cancer Not yet recruiting USA 0
NCT01219699 Phase I Alpelisib Alpelisib + Fulvestrant A Study of BYL719 in Adult Patients With Advanced Solid Malignancies, Whose Tumors Have an Alteration of the PIK3CA Gene Completed USA | ESP | DEU 2
NCT04801966 Phase 0 Palbociclib Alpelisib Atezolizumab Vemurafenib Ribociclib Talazoparib Cobimetinib Trametinib Abemaciclib Dabrafenib Nivolumab Binimetinib Pembrolizumab Encorafenib Safety and Oversight of the Individually Tailored Treatment Approach: A Novel Pilot Study (TAILOR) Recruiting 1
NCT02437318 Phase III Alpelisib Fulvestrant Study Assessing the Efficacy and Safety of Alpelisib Plus Fulvestrant in Men and Postmenopausal Women With Advanced Breast Cancer Which Progressed on or After Aromatase Inhibitor Treatment. Active, not recruiting USA | ITA | FRA | ESP | DEU | CAN | BEL | AUT 23


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