Therapy Detail
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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 21 | 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 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 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 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 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 |
| 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 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 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 H1047R | breast cancer | sensitive | Alpelisib | Preclinical - Cell culture | Actionable | In a preclinical study, Piqray (alpelisib) inhibited growth of breast cancer organoids harboring PIK3CA H1047R in culture (PMID: 37272756). | 37272756 |
| 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 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 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 |
| 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 H1047R | endometrial cancer | predicted - sensitive | Alpelisib | Case Reports/Case Series | Actionable | In a clinical case study, Piqray (alpelisib) resulted in a partial response after 4 months in a patient with endometrial endometrioid cancer harboring PIK3CA H1047R, with treatment ongoing at 7 months (PMID: 36713525). | 36713525 |
| 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 |
| 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 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 |
| 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 E545K | cervical cancer | predicted - sensitive | Alpelisib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Piqray (alpelisib) increased apoptosis and decreased colony formation in a cervical cancer cell line harboring PIK3CA E545K in culture and decreased tumor cell proliferation and increased CD8-positive T-cell differentiation and proliferation in a cell line xenograft model (PMID: 37469254). | 37469254 |
| 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 |
| 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 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 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 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 |
| 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 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 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 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 |
| 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 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 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 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 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 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 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 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 |
| 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 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 |
| 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 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 |
| FGFR1 N546K | diffuse midline glioma, H3 K27M-mutant | sensitive | Alpelisib | Preclinical - Cell culture | Actionable | In a preclinical study, Piqray (alpelisib) inhibited viability of mouse glioma cells expressing H3.3 K28M (reported as H3.3 K27M) and FGFR1 N546K (reported as N457K) in culture (PMID: 37011011). | 37011011 |
| 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 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 |
| 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 |
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| Clinical Trial | Phase | Therapies | Title | Recruitment Status | Covered Countries | Other Countries |
|---|---|---|---|---|---|---|
| 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 | Terminated | 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 |
| NCT05101564 | Phase II | Amcenestrant Alpelisib Infigratinib Tamoxifen Letrozole Amcenestrant + Letrozole | Umbrella Trial Testing Integrative Subtype-Targeted Therapeutics in HR+ /HER2-Negative Breast Cancer | 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 | GBR | ESP | DEU | 1 |
| 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 |
| 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. (SOLAR-1) | Completed | USA | ITA | GBR | FRA | ESP | DEU | CAN | BEL | AUT | AUS | 21 |
| 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 |
| 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) | Terminated | AUS | 0 |
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