Alpelisib HCl (BLY-719; trade name: Piqray) is a novel, potent, newly approved, orally
bioavailable phosphatidylinositol 3-kinase (PI3K) inhibitor with
antineoplastic activity. As of May 2019, it has been approved by FDA as
the first PI3K inhibitor to treat postmenopausal women, and men, with
hormone receptor (HR)-positive, human epidermal growth factor receptor 2
(HER2)-negative, PIK3CA-mutated, advanced or metastatic breast cancer
(as detected by an FDA-approved test) following progression on or after
an endocrine-based regimen. PI3K inhibitor BYL719 specifically inhibits
PIK3 in the PI3K/AKT kinase (or protein kinase B) signaling pathway,
thereby inhibiting the activation of the PI3K signaling pathway. This
may result in inhibition of tumor cell growth and survival in
susceptible tumor cell populations. Activation of the PI3K signaling
pathway is frequently associated with tumorigenesis. Dysregulated PI3K
signaling may contribute to tumor resistance to a variety of
antineoplastic agents. Clinical data suggests a disable safety profile
with manageable side effects for BYL719.