Executive Summary

Cyclacel will submit a Special Protocol Assessment request for its Phase III trial of sapecitabine in acute myeloid leukemia, as well as for a separate trial in myelodysplastic syndromes, during the first quarter of 2010. The company announced Dec. 15 that at a recent type A meeting with FDA, the agency confirmed that the proposed design for a randomized study would be acceptable for the SPA process - through which sponsors gain insight from FDA about clinical trial design and regulatory expectations. Consulting with FDA on the Phase III program is an even more important step in this space following an advisory committee review of two competitors; during its Sept. 1 review of applications for Genzyme's Clolar and Vion's Onrigin for the niche indications to treat elderly patients with AML, FDA's Oncologic Drugs Advisory Committee determined that randomized trials, though difficult to conduct in the space, are necessary for approval (1"The Pink Sheet," Sept. 7, 2009). Both applications have since received "complete response" letters from FDA requesting randomized, controlled trials. Genzyme has already initiated such a study (2Pharmaceutical Approvals Monthly, October 2009). Vion, however, may not be able to fulfill the request (3"The Pink Sheet" DAILY, Dec. 14, 2009)