Table 2.

Best response for patients in the Phase 2 and Oral Solution cohorts.

Response Category	Phase 2 (n = 20)	Oral Solution (n = 12)
Complete Response (CR)	0	0
CR-Minimal Residual Disease (MRD)	0	1
Partial Response (PR)	4	0
Minor Response	2*	2**
Stable Disease	8	3
Progressive Disease	5	6
Not Evaluable	1	0
Response Rate (CR + CR-MRD + PR)	4 / 19 = 21.1%	1 / 12 = 8.3%

^*Minor responses in the Phase 2 consisted of one patient with bone marrow CR in setting of SD by MIBG scan and one patient with bone marrow CR-MRD in setting of SD by MIBG and CT scans.

^**Minor responses in the Oral Solution cohort consisted of one patient with an MIBG CR in setting of SD by CT scan and bone marrow criteria and one patient with a PR by CT scan in setting of SD by MIBG scan and bone marrow criteria.