alpelisib
Trade name: Piqray
Approved
May 24, 2019
Alpelisib (BYL719) is a PI3Kα-selective inhibitor. PI3K-AKT-mTOR pathway is frequently activated in cancer, therefore investigational PI3K inhibitor alpelisib is considered to be effective as an anticancer agent and has been in clinical development by Novartis. Alpelisib have demonstrated activity in preclinical models of solid tumors and had favorable tolerability profiles, with the most common adverse events consistent with “on-target” inhibition of PI3K in early clinical studies. There are ongoing clinical trials of alpelisib in a range of cancer types, including breast cancer, head and neck squamous cell carcinoma, non-small cell lung carcinoma, lymphoma, and glioblastoma multiforme. Combination therapy with other chemo therapeutics may be preferable. — NCATS
Clinical trial activity
96 trials · 65 clinical orgs · 1 marketing orgs
Earliest trial started Oct 5, 2010 (NCT01219699)
Timeline
Indications
Studied for
- Androgens · Phase 1
- Arteriovenous Malformations · Phase 2
- Biomarkers · Phase 1
- Breast Neoplasms · Phase 3
- Carcinoma, Squamous Cell · Phase 2
- Colorectal Neoplasms · Phase 1/Phase 2
- Drugs, Investigational · Phase 1
- Dyslipidemias · Phase 1
- Endometrial Neoplasms · Phase 2
- Esophageal Neoplasms · Phase 1
- Esophageal Squamous Cell Carcinoma · Phase 1
- Genetic Diseases, Inborn · Phase 2
- Growth · Phase 2
- Growth and Development · Phase 2
- Head and Neck Neoplasms · Phase 2
- Human Papillomavirus Viruses · Phase 1
- Hyperinsulinism · Phase 1
- Insulin Resistance · Phase 1
- Kidney Neoplasms · Phase 1
- Leukemia, Myeloid, Acute · Phase 1/Phase 2
- Liver Diseases · Phase 1
- Lung Neoplasms · Phase 2
- Lymphangioma · Phase 2
- Lymphangioma, Cystic · Phase 2
- Lymphatic Diseases · Phase 2/Phase 3
- Megalencephaly · Phase 2
- Melanoma · Phase 1/Phase 2
- Meningioma · Phase 1
- Multiple Myeloma · Phase 1/Phase 2
- Mutation · Phase 2
- Myelodysplastic Syndromes · Phase 1/Phase 2
- Neoplasm Metastasis · Phase 1/Phase 2
- Neoplasms · Phase 2
- Non-alcoholic Fatty Liver Disease · Phase 1
- Obesity · Phase 1
- Oropharyngeal Neoplasms · Phase 2
- Ovarian Neoplasms · Phase 3
- Pancreatic Neoplasms · Phase 2
- Papilloma · Phase 1
- Papillomavirus Infections · Phase 2
- Precision Medicine · Phase 2
- Prediabetic State · Phase 1
- Prostatic Neoplasms · Phase 2
- ras Proteins · Phase 2
- Rectal Neoplasms · Phase 1
- Sarcoma · Phase 2
- Squamous Cell Carcinoma of Head and Neck · Phase 2
- Stomach Neoplasms · Phase 1/Phase 2
- Triple Negative Breast Neoplasms · Phase 3
- Vascular Diseases · Phase 2
- Vascular Malformations · Phase 2
- Venous Cutdown · Phase 2
Mechanism of action
Approval history
- approvedPriority reviewMay 24, 2019
Chemistry & pharmacology
SMILES
CC1=C(SC(NC(=O)N2CCC[C@H]2C(N)=O)=N1)C3=CC(=NC=C3)C(C)(C)C(F)(F)F- Mol. weight
- 441.47 g/mol
- Lipinski Ro5
- Pass
- Rule of 3
- No
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
- Delivery
- Oral
- Availability
- Prescription Only
Oral
Yes
Parenteral
No
Topical
No
Sources
- WikipediaAlpelisib ↗
- NCATS08W5N2C97Q ↗
- ChEMBLCHEMBL2396661 ↗
Also known as
- alpelisib
- alpelisib
- alpelisib
- alpelisib
- byl719
- byl719
- byl719
- byl-719
- byl-719
- nvp-byl719
- nvp-byl719
- nvp-byl719
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT06997588 | Phase 2 | Oct 9, 2025 | Novartis |
| NCT06997887 | N/A | May 9, 2025 | National Taiwan University |
| NCT06545682 | Phase 1/Phase 2 (Phase 2) | Dec 1, 2024 | University of Texas at Houston |
| NCT06739395 | Phase 2 | Nov 1, 2024 | Tianjin Medical University |
| NCT06582004 | N/A | Sep 30, 2024 | National Taiwan University |
| NCT06354088 | Phase 1 | Apr 24, 2024 | Columbia University, University of California, Berkeley |
| NCT05948943 | Phase 2/Phase 3 (Phase 3) | Nov 24, 2023 | Novartis |
| NCT05967286 | Phase 2 | Oct 23, 2023 | National Cancer Institute (NCI) |
| NCT05933395 | Phase 2 | Oct 10, 2023 | Dartmouth University |
| NCT05983159 | Phase 2 | Sep 30, 2023 | Murdoch University, Peter MacCallum Cancer Centre, University of Melbourne |
| NCT05966584 | Phase 2 | Jul 6, 2023 | AstraZeneca, Memorial Sloan-Kettering Cancer Center, Novartis |
| NCT05826964 | Phase 2 | Jun 12, 2023 | University of Miami |
| NCT05733455 | Phase 1 | May 9, 2023 | Columbia University |
| NCT05625087 | Phase 2 | Feb 28, 2023 | Breast Cancer Research Foundation, Institut Gustave Roussy, Novartis, UNICANCER |
| NCT05646862 | Phase 3 | Jan 31, 2023 | Roche |
| NCT05563220 | Phase 1/Phase 2 (Phase 2) | Dec 31, 2022 | Pipeline Therapeutics, Inc. |
| NCT05238831 | Phase 2 | Dec 1, 2022 | Genentech, OHSU Knight Cancer Institute, Oregon Health and Science University |
| NCT05577754 | Phase 2 | Nov 28, 2022 | Novartis, University of Burgundy |
| NCT05472220 | Phase 1 | Nov 1, 2022 | Novartis, University of California, San Francisco |
| NCT05501886 | Phase 3 | Sep 30, 2022 | Celcuity |
Organizations
Research & Development (65)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| Novartis | For profit | 61 | 37 | 5 | 2010 |
| National Cancer Institute (NCI) | Government | 6 | 1 | 2 | 2013 |
| Columbia University | Academic/Hospital | 3 | 3 | 1 | 2014 |
| Cornell University | Academic/Hospital | 3 | 3 | 1 | 2013 |
| Peter MacCallum Cancer Centre | Academic/Hospital | 3 | 2 | 2 | 2015 |
| Seoul National University | Academic/Hospital | 3 | 3 | 1 | 2017 |
| University of Texas at Houston | Academic/Hospital | 3 | 3 | 2 | 2018 |
| Array BioPharma | For profit | 2 | 2 | 1 | 2012 |
| AstraZeneca | For profit | 2 | 0 | 2 | 2012 |
| Memorial Sloan-Kettering Cancer Center | Academic/Hospital | 2 | 2 | 1 | 2021 |
| National Taiwan University | Academic/Hospital | 2 | 2 | 1 | 2024 |
| UNICANCER | Academic/Hospital | 2 | 2 | 1 | 2018 |
| University of California, San Francisco | Academic/Hospital | 2 | 2 | 1 | 2016 |
| University of Kansas | Academic/Hospital | 2 | 2 | 2 | 2015 |
| University of Toronto | Academic/Hospital | 2 | 1 | 2 | 2015 |
| Aix-University of Marseille | Academic/Hospital | 1 | 1 | 1 | 2019 |
| Big Ten Cancer Research Consortium | Academic/Hospital | 1 | 1 | 1 | 2021 |
| Borstkanker Onderzoek Groep | Academic/Hospital | 1 | 1 | 1 | 2022 |
| Breast Cancer Research Foundation | Academic/Hospital | 1 | 0 | 1 | 2023 |
| Brown University | Academic/Hospital | 1 | 1 | 1 | 2016 |