Patients with MDS, CMML or AML who were relapsed or refractory to prior HMA therapies and who received CC-486 had a 38% overall response rate
In the analysis, a total of 35 patients had received prior HMA therapy (22 with MDS, 2 with CMML and 11 with AML) and received 120-600 mg of CC-486 x7 days (d) following a single sub-cutaneous azacitidine (AZA) cycle (75 mg/m2/d x7d), or 300 mg QD (once daily) or 200 mg BID (twice daily) of CC-486 x14d or 21d (with no initial SC AZA cycle). Before receiving CC-486, 11 patients (31%) had failed more than 1 prior injectable HMA course. Most patients (57%) had previously received more than 4 HMA therapy cycles. Five patients with AML had received prior HMAs during treatment for MDS. Of 25 patients for whom outcomes with prior HMAs were known, 14 patients relapsed and 11 patients were refractory to the injectable HMA. The median number of CC-486 cycles was five (range 1-60).
For all patients treated with CC-486, the overall response rate (ORR), which included complete remission (CR), partial remission (PR), CR with incomplete hematologic recovery (CRi; AML patients only), hematologic improvement (HI), and transfusion independence (TI), was 38%. Marrow CR (mCR) was assessed in MDS pts with ≥5% bone marrow blasts at baseline. Four of 11 patients (36%) who were refractory to prior HMAs responded, including one AML patient who attained CR with CC-486. Five of 14 patients (35%) who had relapsed during or after prior HMA therapy responded. Of patients who had received at least six cycles of prior HMA therapy, the ORR was 33% (6/18).
The most frequent (≥10%) grade 3-4 hematologic adverse events were anemia (34%) and thrombocytopenia (23%), neutropenia (17%).The most frequent grade 3-4 gastrointestinal adverse events were diarrhea and vomiting (11% each).
"The results of this analysis suggest that prior HMA exposure does not
preclude future response to CC-486," said Dr.
The genome provides instructions for synthesis of proteins and
non-coding RNAs.1 The epigenome provides further instructions
on where to express a protein or RNA as well as how much of each to
synthesize.1-3 Dysregulation of the epigenome in cancer cells
may lead to aberrant gene expression of oncogenes and decreased
expression of tumor suppressor genes.1-3
The safety and efficacy of CC-486 and/or uses under investigation have not been established. There is no guarantee that CC-486 will receive health authority approval or become commercially available in any country for the uses being investigated.
This press release contains forward-looking statements, which are
generally statements that are not historical facts. Forward-looking
statements can be identified by the words "expects," "anticipates,"
"believes," "intends," "estimates," "plans," "will," "outlook" and
similar expressions. Forward-looking statements are based on
management's current plans, estimates, assumptions and projections, and
speak only as of the date they are made. We undertake no obligation to
update any forward-looking statement in light of new information or
future events, except as otherwise required by law. Forward-looking
statements involve inherent risks and uncertainties, most of which are
difficult to predict and are generally beyond our control. Actual
results or outcomes may differ materially from those implied by the
forward-looking statements as a result of the impact of a number of
factors, many of which are discussed in more detail in our Annual Report
on Form 10-K and our other reports filed with the
1. Cao J. Biol Proced Online. 2014;16:11.
2. Tarakhovsky A. Nat Immunol. 2010;11:565-568.
3. Roy DM, Walsh LA, Chan TA. Protein Cell. 2014;5:265-296.
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