Table 3.
Best overall response across key tumor types
| Patients, n (%) | Overall population (n = 56) | Dose escalation | Expansion LGG (n = 12) | All LGG patients (n = 32) | |
|---|---|---|---|---|---|
| LGG (n = 20) | PN in NF1 (n = 12) | ||||
| ORRa | 3 (5) | 2 (10) | 0 | 1 (8) | 3 (9) |
| CR | 0 | 0 | 0 | 0 | 0 |
| PR | 3 (5) | 2 (10) | 0 | 1 (8) | 3 (9) |
| Minor responseb | NA | NA | NA | 1 (8)a | NA |
| SD | 33 (59) | 10 (50) | 12 (100) | 8 (67) | 18 (56) |
| PD | 13 (23) | 5 (25) | 0 | 2 (17) | 7 (22) |
| Non-CR/Non-PD | 1 (2) | 1 (5) | 0 | 0 | 1 (3) |
| Non-evaluable | 2 (4) | 0 | 0 | 1 (8) | 1 (3) |
| Not available | 4 (7) | 2 (10) | 0 | 0 | 2 (6) |
In the dose-escalation stage, ORR was determined by International Neuroblastoma Response Criteria, Response Evaluation Criteria in Solid Tumors (RECIST) v1.1, or Response Assessment in Neuro-Oncology (RANO), as appropriate. For patients with LGG in the expansion stage, ORR was determined by both RANO and RECIST v1.1
CR complete response, LGG low-grade glioma, NA not applicable, ORR objective response rate, PD progressive disease, PN in NF-1 plexiform neurofibroma in neurofibromatosis type 1, PR partial response, SD stable disease
aConfirmation of a MAPK pathway alteration was received for one patient with a PR whose tumor harbored an NF1 truncating mutation. For the other two patients with a PR, the site confirmed no MAPK pathway alteration was present for one patient, and the site for the other patient did not provide any information or the mutation status was unknown (see ESM, Table S3)
bMinor response per RANO criteria