Protein / target

Phosphatidylinositol 4,5-bisphosphate 3-kinase catalytic subunit alpha isoform

PIK3CAP42336Homo sapiensSwiss-Prot
Clinically validated
Therapeutic maturity
4
Approved medicines
30
Clinical trials
Small-molecule tractable
Druggability
Approved Drug

Protein at a glance

Biological role

1-phosphatidylinositol-4,5-bisphosphate 3-kinase activity

Primary system

Cardiovascular system

Strongest disease association

megalencephaly-capillary malformation-polymicrogyria syndrome

Genetic evidence · score 0.96

Therapeutic maturity

Clinically validated target

4 approved medicines against this target

Druggability

Small molecule

Open Targets tractability · Approved Drug

Clinical development

4 approved · 27 in clinical development

30 linked trials

Derived from structured UniProt, Open Targets and literature data on this page.

Protein profile

UniProt 2026_02

Canonical identity and biological annotation from UniProt.

Function

Phosphoinositide-3-kinase (PI3K) phosphorylates phosphatidylinositol (PI) and its phosphorylated derivatives at position 3 of the inositol ring to produce 3-phosphoinositides (PubMed:15135396, PubMed:23936502, PubMed:28676499). Uses ATP and PtdIns(4,5)P2 (phosphatidylinositol 4,5-bisphosphate) to generate phosphatidylinositol 3,4,5-trisphosphate (PIP3) (PubMed:15135396, PubMed:28676499). PIP3 plays a key role by recruiting PH domain-containing proteins to the membrane, including AKT1 and PDPK1, activating signaling cascades involved in cell growth, survival, proliferation, motility and morphology. Participates in cellular signaling in response to various growth factors. Involved in the activation of AKT1 upon stimulation by receptor tyrosine kinases ligands such as EGF, insulin, IGF1, VEGFA and PDGF. Involved in signaling via insulin-receptor substrate (IRS) proteins. Essential in endothelial cell migration during vascular development through VEGFA signaling, possibly by regulating RhoA activity. Required for lymphatic vasculature development, possibly by binding to RAS and by activation by EGF and FGF2, but not by PDGF. Regulates invadopodia formation through the PDPK1-AKT1 pathway. Participates in cardiomyogenesis in embryonic stem cells through a AKT1 pathway. Participates in vasculogenesis in embryonic stem cells through PDK1 and protein kinase C pathway. In addition to its lipid kinase activity, it displays a serine-protein kinase activity that results in the autophosphorylation of the p85alpha regulatory subunit as well as phosphorylation of other proteins such as 4EBP1, H-Ras, the IL-3 beta c receptor and possibly others (PubMed:23936502, PubMed:28676499). Plays a role in the positive regulation of phagocytosis and pinocytosis (By similarity)

Domains and Gene Ontology detail (62)

Domains & features

PI3K-ABDPI3K-RBDC2 PI3K-typePIK helicalPI3K/PI4K catalytic

Gene Ontology

  • Ccytoplasm
  • Ccytosol
  • Cintercalated disc
  • Clamellipodium
  • Cperinuclear region of cytoplasm
  • Cphosphatidylinositol 3-kinase complex
  • Cphosphatidylinositol 3-kinase complex, class IA
  • Cphosphatidylinositol 3-kinase complex, class IB
  • Cplasma membrane
  • F1-phosphatidylinositol-3-kinase activity
  • F1-phosphatidylinositol-4,5-bisphosphate 3-kinase activity
  • F1-phosphatidylinositol-4-phosphate 3-kinase activity

1068 aa · 124 kDa

Biological roles

Reactome v97

What this protein does, drawn together from its UniProt function, Gene Ontology terms and Reactome pathways.

Kinase signallingUniProt · GOImmune signallingGO · ReactomeMuscle contractionGOTranscriptional regulationGOHaemostasisGO
View supporting evidence

Kinase signalling

  • ·Phosphoinositide-3-kinase (PI3K) phosphorylates phosphatidylinositol (PI) and its phosph…
  • ·1-phosphatidylinositol-3-kinase activity
  • ·1-phosphatidylinositol-4,5-bisphosphate 3-kinase activity
  • ·1-phosphatidylinositol-4-phosphate 3-kinase activity

Immune signalling

  • ·T cell receptor signaling pathway
  • ·Interleukin-3, Interleukin-5 and GM-CSF signaling

Muscle contraction

  • ·cardiac muscle contraction
  • ·negative regulation of actin filament depolymerization
  • ·regulation of actin filament organization

Transcriptional regulation

  • ·negative regulation of gene expression

Haemostasis

  • ·platelet activation
View underlying pathways (25)

Concepts derived from UniProt GO Reactome — each badge above shows which sources supported that role.

Interaction neighbourhood

STRING v12.0

Proteins with the strongest functional or physical association. Node size and line weight reflect STRING confidence; hover or select a partner to inspect one association.

PIK3R5PIK3R2IRS1EGFRPIK3R3PIK3R1PIK3CDPIK3CBAKT1KRASPIK3CA

10 strongest partners — larger node and heavier line mean higher confidence

Functional and physical associations from STRING v12.0. Only associations with this protein are drawn — partner-to-partner links are not part of this evidence.

Drugs targeting this protein

1

Whether each drug engages this protein directly or through a complex, and how many other targets are recorded for it. Direct binders with few recorded targets are listed first.

Alpelisib
Narrow target profileApprovedInhibitor

PI3-kinase p110-alpha subunit inhibitor

Appears in clinical studies involving breast cancer, breast neoplasm, neoplasm, breast carcinoma

Direct interaction with this protein · Only this protein recorded as a target

ChEMBL mechanism, action type and target identity. Open Targets clinical status and indications.

Translational evidence

Open Targets 26

Why this target matters therapeutically — disease associations, drugs in development, tractability and safety, from Open Targets.

Strongest genetic associations

Human genetic evidence — the most direct causal link between this target and a disease.

megalencephaly-capillary malformation-polymicrogyria syndrome0.96

Genetic · overall 0.85

breast cancer0.92

Genetic · overall 0.75

PIK3CA-related overgrowth spectrum0.91

Genetic · overall 0.77

Cowden syndrome 50.91

Genetic · overall 0.75

CLOVE syndrome0.88

Genetic · overall 0.80

Highest-confidence therapeutic associations

Diseases where a drug acting on this target has already reached clinical development.

ovarian cancer0.18

Clinical · overall 0.73

Highest overall evidence

Remaining associations by Open Targets' aggregated evidence score.

CLOVES syndrome0.79

Somatic mutation

seborrheic keratosis0.73

Genetic

breast adenocarcinoma0.73

Pathway

CLAPO syndrome0.72

Somatic mutation

Show all associations
megalencephaly-capillary malformation-polymicrogyria syndrome0.85
CLOVE syndrome0.80
CLOVES syndrome0.79
PIK3CA-related overgrowth spectrum0.77
breast cancer0.75
Cowden syndrome 50.75
ovarian cancer0.73
seborrheic keratosis0.73
breast adenocarcinoma0.73
CLAPO syndrome0.72

Open Targets ranks 1,791 associations for this target and we store the top 10 by aggregated score. The long tail is largely literature co-mention and RNA-expression evidence, not evidence that this target drives those diseases.

Known drugs · 32 total

PANULISIBPhase 1
PILARALISIBPhase 2

endometrial carcinoma · breast cancer · lymphoma

IZORLISIBPhase 1 2

colorectal cancer · neoplasm · neoplasm

SF-1126Phase 2

head and neck cancer · head and neck squamous cell carcinoma · hepatocellular carcinoma

COPANLISIBApproval

follicular lymphoma · non-Hodgkin lymphoma · neoplasm

PKI-179Phase 1

colorectal cancer · glioblastoma · endometrial cancer

RECILISIBPhase 1

radiation injury

PICTILISIBPhase 2

breast cancer · non-Hodgkin lymphoma · non-small cell lung carcinoma

DACTOLISIBPhase 3

Respiratory tract infection · breast cancer · endometrial carcinoma

SAMOTOLISIBPhase 2

prostate cancer · non-small cell lung carcinoma · endometrial cancer

Tractability

SM · Approved DrugSM · Structure with LigandSM · High-Quality LigandSM · High-Quality PocketSM · Druggable FamilyAB · GO CC med confPR · LiteraturePR · Database UbiquitinationPR · Half-life DataPR · Small Molecule Binder

Safety liabilities

regulation of catalytic activitydrug toxicityNo significant resultsheart disease

Clinical trials

30

Trials of drugs that target this protein — reached indirectly through those drugs, so a trial listed here studies the drug, not the protein.

Show remaining trials (24)

ClinicalTrials.gov via the drug-target graph.

Forefront confidence

Synthesis

Our own weighting of the evidence behind each disease association. Human genetics and clinical evidence count for most; literature co-mention counts for little, because two entities sharing an abstract is not evidence that one drives the other.

megalencephaly-capillary malformation-polymicrogyria syndromeWell supported
0.99
agreement 0.891.00
Genetic58%Somatic mutation35%Clinical6%Literature2%Genetic literaturedup

Open Targets aggregate 0.85 · 4 independent evidence families · 1 not counted as duplicate

breast cancerWell supported
0.98
agreement 0.871.00
Genetic52%Clinical40%Literature9%Genetic literaturedup

Open Targets aggregate 0.75 · 3 independent evidence families · 1 not counted as duplicate

PIK3CA-related overgrowth spectrumWell supported
0.97
agreement 0.881.00
Genetic55%Somatic mutation37%Clinical8%Literature0%Genetic literaturedup

Open Targets aggregate 0.77 · 4 independent evidence families · 1 not counted as duplicate

CLOVE syndromeWell supported
0.95
agreement 0.841.00
Genetic58%Somatic mutation38%Literature4%Genetic literaturedup

Open Targets aggregate 0.80 · 3 independent evidence families · 1 not counted as duplicate

CLOVES syndromeWell supported
0.95
agreement 0.841.00
Genetic58%Somatic mutation38%Literature4%Genetic literaturedup

Open Targets aggregate 0.79 · 3 independent evidence families · 1 not counted as duplicate

The evidence agreement range shows how closely the independent evidence families agree — it is not a statistical confidence interval, and nothing here is fitted to outcome data. Derived from Open Targets evidence types under Forefront weighting; the underlying per-type scores are shown above so the calculation can be checked.

What's happening now

10

Recent activity around this target, drawn from one canonical event stream. Every item is reached through a drug that targets this protein, so each event is news about that drug rather than about the protein directly.

  1. CHMP positive opinion2026-05-22

    CHMP positive opinion: Vijoice (EMA)

    ema · regulatory · ema · via Alpelisib

  2. Label change2025-07-22

    Label change: ALPELISIB (NDA215039)

    fda · regulatory · fda · via Alpelisib

  3. Indication expanded2024-04-24

    Indication expansion: ALPELISIB (NDA215039)

    fda · regulatory · fda · via Alpelisib

  4. New publication2023-09-25
    Incidence, risk factors, and management of alpelisib-associated hyperglycemia in metastatic breast cancer.

    Cancer · 2023 · 21 citations · Europe PMC · via Alpelisib

  5. Label change2022-11-17

    Label change: ALPELISIB (NDA215039)

    fda · regulatory · fda · via Alpelisib

  6. Regulatory approval2022-04-05

    Approval: ALPELISIB (NDA215039)

    fda · regulatory · fda · via Alpelisib

  7. New publication2020-11-25
    Alpelisib plus fulvestrant for PIK3CA-mutated, hormone receptor-positive, human epidermal growth factor receptor-2-negative advanced breast cancer: final overall survival results from SOLAR-1.

    Annals of oncology : official journal of the European Society for Medical Oncology · 2021 · 447 citations · Europe PMC · via Alpelisib

  8. Regulatory approval2020-07-27

    Approval: Piqray (EMA)

    ema · regulatory · ema · via Alpelisib

  9. New publication2020-05-13
    Frequency and spectrum of PIK3CA somatic mutations in breast cancer.

    Breast cancer research : BCR · 2020 · 267 citations · Europe PMC · via Alpelisib

  10. New publication2019-05-01
    Alpelisib for <i>PIK3CA</i>-Mutated, Hormone Receptor-Positive Advanced Breast Cancer.

    The New England journal of medicine · 2019 · 1,948 citations · Europe PMC · via Alpelisib

Objective event titles are shown unmodified; the event kind and significance line are derived from structured fields. Forefront AttentionEvent stream aggregating Europe PMC Regulatory filings ClinicalTrials.gov.