Table 2.

Activity summary for single agents and combinations

LineTumor typeTreatment groupMedian time to event (days)P valueaEFS T/CbMedian RTV end of studycCR at 6 weeksGroup responsed
KT-10WilmsTemozolomide21.80.0841.2>4NoSD
KT-10WilmsTalazoparib>EPe<0.001>5.2>4YesCR
KT-10WilmsCombination Af>EP<0.001>5.20.0YesMCR
KT-10WilmsCombination Bg>EP<0.001>5.20.0YesMCR
SK-NEP-1EwingTemozolomide8.90.3890.8>4NoPD1
SK-NEP-1EwingTalazoparib10.10.9161.0>4NoPD1
SK-NEP-1EwingCombination A32.3<0.0013.1>4NoPR
SK-NEP-1EwingCombination B>EP<0.001>8.60.0YesMCR
TC-71EwingTemozolomide10.70.3681.0>4NoPD1
TC-71EwingTalazoparib12.20.0161.2>4NoPD1
TC-71EwingCombination A>EP<0.001>8.80.0YesMCR
TC-71EwingCombination B>EP<0.001>8.80.0YesMCR
CHLA258EwingTemozolomide10.20.2421.1>4NoPD1
CHLA258EwingTalazoparib11.80.0341.2>4NoPD1
CHLA258EwingCombination A>EP<0.001>9.60.0YesMCR
CHLA258EwingCombination B>EP<0.001>9.6>4YesCR
ES6EwingTemozolomide25.10.0391.3>4NoPD1
ES6EwingTalazoparib20.60.8451.1>4NoPD1
ES6EwingCombination A35.9<0.0011.9>4NoPD2
ES6EwingCombination B31.6<0.0011.7>4NoPD2
ES4EwingTemozolomide15.80.0011.4>4NoPD1
ES4EwingTalazoparib14.30.0321.3>4NoPD1
ES4EwingCombination A81.5<0.0017.1>4YesCR
ES4EwingCombination B77.4<0.0016.8>4YesCR
EW5EwingTemozolomide9.90.5071.1>4NoPD1
EW5EwingTalazoparib8.90.9411.0>4NoPD1
EW5EwingCombination A28.6<0.0013.3>4NoPR
EW5EwingCombination B24.0<0.0012.7>4NoCR
ES-2EwingTemozolomide12.70.5671.1>4NoPD1
ES-2EwingTalazoparib14.00.7121.2>4NoPD1
ES-2EwingCombination A19.20.0021.6>4NoPD2
ES-2EwingCombination B30.3<0.0012.6>4NoCR
EW8EwingTemozolomide9.10.0051.3>4NoPD1
EW8EwingTalazoparib13.4<0.0012.0>4NoPD2
EW8EwingCombination A15.7<0.0012.3>4NoPD2
EW8EwingCombination B12.6<0.0011.9>4NoPD2
ES-3EwingTemozolomide10.30.0071.2>4NoPD1
ES-3EwingTalazoparib10.70.0111.2>4NoPD1
ES-3EwingCombination A26.0<0.0012.9>4NoCR
ES-3EwingCombination B18.3<0.0012.1>4NoCR
ES-7EwingTemozolomide18.30.0031.6>4NoPD2
ES-7EwingTalazoparib15.70.0211.4>4NoPD1
ES-7EwingCombination A>EP<0.001>7.80.0YesMCR
ES-7EwingCombination B>EP<0.001>7.80.0YesMCR
ES-8EwingTemozolomide7.10.1101.2>4NoPD1
ES-8EwingTalazoparib10.7<0.0011.9>4NoPD2
ES-8EwingCombination A38.7<0.0016.7>4NoCR
ES-8EwingCombination B33.4<0.0015.8>4NoCR
Rh28hAlveolar RMSTemozolomide>EP<0.001>3.00.0YesMCR
Rh28hAlveolar RMSTalazoparib13.70.0030.5>4NoPD1
Rh28hAlveolar RMSCombination A>EP<0.001>3.00.0YesMCR
Rh28hAlveolar RMSCombination B>EP<0.001>3.00.0YesMCR
Rh30Alveolar RMSTemozolomide16.80.7991.1>4NoPD1
Rh30Alveolar RMSTalazoparib15.00.6241.0>4NoPD1
Rh30Alveolar RMSCombination A50.0<0.0013.2>4NoCR
Rh30Alveolar RMSCombination B37.3<0.0012.4>4NoSD
Rh41Alveolar RMSTemozolomide11.80.1760.9>4NoPD1
Rh41Alveolar RMSTalazoparib11.10.0900.9>4NoPD1
Rh41Alveolar RMSCombination A11.30.0720.9>4NoPD1
Rh41Alveolar RMSCombination B13.90.0611.1>4NoPD1
GBM2hGlioblastomaTemozolomide35.3<0.0015.4>4NoCR
GBM2hGlioblastomaTalazoparib8.00.2951.2>4NoPD1
GBM2hGlioblastomaCombination A83.2<0.00112.8>4YesCR
GBM2hGlioblastomaCombination B>EP<0.001>14.00.0YesMCR
  • aP value comparing EFS distribution of treated and control groups. Values <0.05 are highlighted by bolded text.

  • bEFS T/C: ratio of EFS (median time to event) for treated and control groups. Values >2 are highlighted by bolded text.

  • cRTV: ratio of median relative tumor volume at end of observation period for treated to control groups.

  • dSee Materials and Methods section for definitions of PD1, PD2, SD, PR, CR, and MCR.

  • eEP, median time to event for treatment group extended beyond the observation period.

  • fCombination A (temozolomide 30 mg/kg for 5 days; talazoparib 0.1 mg/kg twice daily for 5 days).

  • gCombination B (temozolomide 12 mg/kg for 5 days; talazoparib 0.25 mg/kg twice daily for 5 days).

  • hLow MGMT-expressing xenograft lines.