Table 4.
Relapsed AML Patients Who Had an Objective Response to OXA in OX1222 Study (N = 5).
| Bone Marrow Involvement | Treatment Outcome | ||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Patient ID | Cohort | OXi4503 Doses | Diagnosis | Age/Sex/Race | Previous Therapies (Number: List) | Cellularity | Percent Myeloblasts by Morphology/FCM | Karyotype/Mutations | Best Overall Response | Time to Progression (days) | C1D1 to EOS | Other Therapy post EOS | Time to Death or Last FU | Survival Status at lastFU | Cause of Death |
| 106-004 1 | 1 | 4 | AML | 59/M/C | 5: 7 + 3; HiDAC;5-AC; IP; PBSCT | NR | 25/20 | Inv (3) (q21q26.2), del(5)(q)/PTPN11 (9%VAF) | CRi | 54+ | 54 | DLI | 535 | D | PD |
| 106-006 2 | 2 | 4 | AML | 65/M/C | 1: 5AZA | 20–30 | 9/8 | 46, XY, +8 (FISH)//NR | CRi | 64+ | 64 | Mylotarg 5-AC | 521 | D | PD |
| 103-009 3 | 3 | 4 | tAML | 66/M/C | 1: 5-AC+DAUNO | 20–70 | 15 | del(5)(q), del(7)(q), +8 (FISH)/TP53 | CR | 78+ | 78 | 5AZA | 434 | D | PD |
| 106-008 4 | 4 | 4 | AML | 77/F/C | 2: 7 + 3; 5AZA | 80–90 | 89/65 | 46, XX | PR | NA | 61 | NA | 61 | D | IFI |
| 107-003 5 | 5 | 6 | AML | 68/M/C | 1: 7 + 3 | 60 | 15/9 | Inv (16) (p13q22) (FISH)/CBFB | CR | 228+ | NA | Allo PBSCT | 720 | A | NA |
C1D1: Cycle 1 Day 1; FU: Follow-up; AML: Acute myelogenous leukemia; MDS: Myelodysplastic syndromes; PD: Progressive disease; CRi: Complete remission with incomplete hematologic recovery; PR: Partial remission; SD/RES: Stable disease/Refractory; NE: Not evaluable; CR: Complete remission; A: Alive; D: Dead; EOS: End of study; NA: Not available; SAE: Serious adverse event; HU: Hydroxyurea; DLI: Donor leukocyte infusion; 5-AC: 5-Azacytidine; PBSCT: Peripheral blood stem cell transplantation; SCT: Stem cell transplant; 5AZA: Deoxyazacytidine. IFI: Invasive fungal infection. 1 The BM blast percentage went from 25% to 3% microscopically and 20.1% to 0.2% by FCM after one cycle of OXA with clearance of PTPN11. 2 The BM blast percentage went from 9% to 2% microscopically and from 8.1% to 0.3% by FCM after 2 cycles of OXA. 3 The BM blast percentage went from 15% to 2% microscopically and genomic missense mutations in the TP53 gene (c.428T>G; p.V143G; 9% allele frequency) detected by molecular profiling using the Genoptix platform cleared after 2 cycles of OXA. 4 The BM blast percentage went from 89% to 7% microscopically and from 65% to 2% by FCM after one cycle of OXA. 5 The BM percentage went from 15% to 0% microscopically and from 9% to 1–2% by FCM with disappearance (0 of 300 nuclei) of the FISH-detected split signal (10%/30 of 300 nuclei) pre-therapy) due to inv(16)/CBFB rearrangement after one cycle of OXA.