Celgene International Sàrl, a wholly-owned subsidiary of Celgene Corporation (CELG) today announced that results from an ad hoc analysis of a subset of subjects with higher-risk myelodysplastic syndrome (MDS) from two ongoing phase I/II studies of oral epigenetic agent CC-486 (oral azacitidine) were presented at the American Society of Hematology annual meeting in New Orleans, La.
In this analysis, 23 patients with WHO-defined RAEB-1 or RAEB-2 MDS who received CC-486 300mg once per day (n=20) or 200mg twice per day (n=3) for 14 or 21 days of repeated 28-day cycles. For purposes of this analysis, subject data were analyzed collectively. Hematologic responses were defined by International Working Group (IWG) 2006 criteria. Overall response rate (ORR) was calculated as any response of complete or partial remission (CR or PR) and/or any hematologic improvement (HI). Marrow complete remission (mCR) was not included in ORR.
Fourteen subjects (61%) had a diagnosis of RAEB-1 and 9 (39%) had RAEB-2, median time from diagnosis was 2.1 (0.1 - 33.2) months, and ECOG performance status scores were 0 (n=4, 17%), 1 (n=15, 65%), or 2 (n=4, 17%). Five subjects had received prior MDS treatments (azacitidine injection; erythropoiesis stimulating agent [ESA]; thalidomide; azacitidine injection and decitabine; G-CSF, anti-thymocyte globulin, methylprednisolone, cyclosporine, and ESA). Median number of CC-486 treatment cycles was 3 (1 - 30).
An overall response was achieved by 11/23 subjects (48%) with 4/23 (17%) subjects achieving a complete remission, no patients achieving a partial remission and 7/23 patients (30%) achieving a hematologic improvement. Red-blood cell TI was achieved by 5/12 subjects (42%) and platelet TI was achieved by 2/5 subjects (40%). Two subjects were able to consolidate remission and proceed to allogeneic stem cell transplant and 1 subject progressed to AML on-study.
Four subjects discontinued treatment due to an adverse event. Serious treatment-emergent adverse events (STEAE) were consistent with the known safety profile of sub-cutaneous azacitidine. Of 9 subjects who had an STEAE of febrile neutropenia, pneumonia, and/or septic shock, 3 were severely neutropenic (ANC 9/L) at baseline. Other STEAEs were diarrhea, nausea, and vomiting (n=2 subjects each).
Results of this study of patients with higher-risk MDS warrant further clinical evaluation.
These results are from an experimental study. CC-486 is not approved for the treatment of any disease.
Celgene International Sàrl, located in Boudry, in the Canton of Neuchâtel, Switzerland, is a wholly-owned subsidiary and international headquarters of Celgene Corporation. Celgene Corporation, headquartered in Summit, New Jersey, is an integrated global pharmaceutical company engaged primarily in the discovery, development and commercialization of innovative therapies for the treatment of cancer and inflammatory diseases through gene and protein regulation. For more information, please visit www.celgene.com.
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