DOR BioPharma, Inc. has reached agreement with the FDA on the design of a confirmatory, pivotal Phase 3 clinical trial evaluating its lead product [email protected] for the treatment of acute gastrointestinal Graft-versus-Host Disease (GI GVHD). The agreement was made under the FDA’s Special Protocol Assessment (SPA) procedure. An agreement via the SPA procedure is an agreement with the FDA that a Phase 3 clinical trial’s design (e.g., endpoints, sample size, control group and statistical analyses) is acceptable to support a regulatory submission seeking new drug approval. After the study begins, the FDA can only change a SPA for very limited reasons. Based on data from the prior Phase 3 study of [email protected], the upcoming confirmatory Phase 3 protocol will be a highly powered, double-blind, randomized, placebo-controlled, multi-center trial and will seek to enroll an estimated 166 patients. The primary endpoint is the treatment failure rate at Study Day 80. This endpoint was successfully measured as a secondary endpoint (p-value 0.005) in the previous Phase 3 study as a key measure of durability following a 50-day course of treatment with [email protected] (i.e., 30 days following cessation of treatment). “We are very pleased to gain SPA agreement with the FDA on our confirmatory, pivotal Phase 3 study,” stated Christopher J. Schaber, PhD, President and Chief Executive Officer of DOR. “The depth and strength of our available Phase 3 data have allowed us to design and power this pivotal trial that we believe maximizes [email protected]’s chances for success. With our primary endpoint of the ‘treatment failure rate at Study Day 80,’ we expect to replicate statistical significance in this clinically meaningful endpoint with [email protected] We expect to initiate this trial in the first half of 2009.” Dr. Schaber continued, “With the regulatory clarity received from FDA, we are now confident that we have a more clearly defined development path that has the potential to lead to regulatory approval of [email protected] We are excited to move forward with this trial in an effort to address the significant unmet medical need of GI GVHD.”
