Table 5 Efficacy and safety data from phase Ib/II trials of alpelisib in HR+, HER2− aBC/mBC

Juric et al. [30], phase Ib, open-label, single-arm

Alpelisib + Fulv^b (300–350–400 mg/d)^d

Postmenopausal PIK3CA-mutated (60%) or PIK3CA-wt (38%) HR+, LABC/mBC progressing on/after prior ET

87

NS

PIK3CA-mutated, 29%; PIK3CA-wt, 0%

PIK3CA-mutated, 9.1; PIK3CA-wt, 4.7

NA

Diarrhea 60%

Nausea 53%

Hyperglycemia 51%

Hyperglycemia 22%

Maculopapular rash 13%

Rash 8%

10%

Mayer et al. [35], phase Ib, multicenter, open-label

Alpelisib + Let (300–350 + 2.5 mg/d)^c

Postmenopausal HR+, HER2− mBC progressing on/after prior ET

26

Yes

PIK3CA-mutated, 25%; PIK3CA-wt, 10%

NA

NA

Alpelisib 300 mg/d

Diarrhea 80%

Nausea 60%

Hyperglicemia 55%

Rash 45%

Fatigue 45%

Diarrhea 10%

Hyperglicemia 10%

AST/ALT elevation 5%

11%

Rugo et al. [51], phase 2, open-label, non-comparative study

Alpelisib + Fulv^bd (300 mg/d)^c, Alpelisib + Let^d (300 + 2.5 mg/d)

Men and women with PIK3CA-mutated HR+, HER2− aBC whose disease progressed on/after CDK4/6i + ET

21, 18

Yes

20%, 18%

NA

NA

NA

Hyperglycemia 38.1% (Fulv)/27.8% (Let)

Rash 4.8% (Fulv)/27.8% (Let)

5%, 5%