Celgene Corporation
Dec 5, 2016
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Combined Analysis of Oral Epigenetic Therapy, CC-486, Demonstrated Hematologic Responses in Patients Who Received Prior Epigenetic Hypo-Methylating Agents (HMA)

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

SUMMIT, N.J.--(BUSINESS WIRE)-- Abstract #905 -- Celgene Corporation (NASDAQ:CELG), today announced results from an analysis of three phase I/II studies evaluating CC-486 in patients with myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML) and acute myeloid leukemia (AML) who had received prior hypomethylating agents (HMA) were presented today at the 58th American Society of Hematology Annual Meeting in San Diego, Calif.

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. Guillermo Garcia-Manero, Chief, Section of Myelodysplastic Syndromes at MD Anderson Cancer Center. "The extended dosing and hypomethylation may also differentiate CC-486 and induce these responses. As we look toward new options for patients with MDS, CMML and AML, this oral epigenetic therapy deserves further evaluation."

About Epigenetics

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 Celgene is developing a diverse array of therapeutics to target all major components of the cancer epigenome. Visit http://www.researchoncology.com/platforms-and-pipeline/centers-of-excellence/epigenetics/ to learn more.

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.

About Celgene

Celgene Corporation, headquartered in Summit, New Jersey, is an integrated global biopharmaceutical company engaged primarily in the discovery, development and commercialization of innovative therapies for the treatment of cancer and inflammatory diseases through next-generation solutions in protein homeostasis, immuno-oncology, epigenetics, immunology and neuro-inflammation. For more information, please visit www.celgene.com. Follow Celgene on Social Media: @CelgenePinterestLinkedInFacebook and YouTube.

Forward-Looking Statements

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 Securities and Exchange Commission.

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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