Phase 2 survival data of sapacitabine in patients with MDS presented at ASH Annual Meeting

Cyclacel Pharmaceuticals, Inc. (Nasdaq:CYCC) (Nasdaq:CYCCP) ("Cyclacel") announced today 1-year survival data from a Phase 2 randomized trial of oral sapacitabine capsules, a novel nucleoside analogue, in older patients with myelodysplastic syndromes (MDS) refractory to hypomethylating agents, such as azacitidine and decitabine. The data were reported at a poster presentation at the 52nd Annual Meeting of the American Society of Hematology (ASH) in Orlando, Florida.

"MDS patients have a poor outcome after treatment failures of hypomethylating agents. Effective therapies are urgently needed to improve the survival of these patients," said Hagop M. Kantarjian, M.D., Chairman and Professor, Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, Texas and study chair of the sapacitabine Phase 2 study. "The survival data of sapacitabine in this study are promising and warrant further clinical development in this patient population."

"We are deeply grateful to our investigators and their patients for helping us to reach this milestone in the development of sapacitabine in MDS," said Judy H. Chiao, M.D., Cyclacel's Vice President, Clinical Development & Regulatory Affairs. "We plan to initiate discussions with the FDA regarding potential registration pathways in MDS patients refractory to hypomethylating agents. We are also looking forward to opening enrollment in "SEAMLESS", our pivotal Phase 3 study of sapacitabine in elderly patients with acute myeloid leukemia (AML). If Phase 3 trials are successful, sapacitabine could emerge as the first oral drug for the treatment of AML and MDS as chronic diseases."

MDS Phase 2 survival data

The study uses a selection design with the objective of identifying a dosing schedule which produces a better 1-year survival rate in the event that all three dosing schedules are active. The study enrolled 61 patients aged 60 or older with MDS refractory to hypomethylating agents randomized across three dosing schedules of sapacitabine: 21 patients in Arm A, a 7-day low dose regimen (200 mg b.i.d.); 20 patients in Arm B, a 7-day high dose regimen (300 mg b.i.d.) and 20 patients in Arm C, a 3-day high dose regimen (400 mg b.i.d.). Approximately 77% of patients were aged 70 years or older and 84% were scored as intermediate-2 or high risk by IPSS, the International Prognostic Scoring System. Baseline blast counts were between 11% and 29% in 51% of the patients.  All patients were previously treated with hypomethylating agents: 43% with azacitidine, 34% with decitabine and 23% were double refractory patients as they were treated with both azacitidine and decitabine (7 on Arm A, 4 on Arm B and 3 on Arm C). Approximately 16% were previously treated with lenalidomide in addition to hypomethylating agents.

The primary endpoint of 1-year survival was achieved in 29% of the patients on Arm A, 30% of the patients on Arm B and 35% of the patients on Arm C. The median overall survival was 217 days on Arm A (range of 15 to 663 days), 232 days on Arm B (range of 37 to over 811 days) and 236 days on Arm C (range of 16 to over 672 days). Overall response rate, a secondary endpoint consisting of the rate of CR, CRp, PR, CRi or hematological improvement, was 24% for patients on Arm A, 35% for patients on Arm B and 15% for patients on Arm C. Two patients achieved a CR both on Arm A. Approximately 20% of all patients received sapacitabine for 4 to 6 cycles and 15% for 7 or more cycles. The mortality rate from all causes within thirty days of randomization was 6.6%.