PRINCETON, N.J., June 9 /PRNewswire-FirstCall/ -- DOR BioPharma, Inc. (DOR or the Company) (OTC Bulletin Board: DORB), a late-stage biopharmaceutical company, announced today that it has received Protocol Assistance feedback from the European Medicines Agency (EMEA) on the design of its confirmatory, pivotal, Phase 3 clinical trial evaluating its lead product orBec(R) for the treatment of acute gastrointestinal Graft-versus-Host Disease (GI GVHD).
The EMEA agreed that should the new confirmatory Phase 3 study produce positive results, the data would be sufficient to support a marketing authorization approval in all 27 European Union (EU) member states. In so doing, the EMEA agreed to the primary endpoint and the other principal design features of the new study. The EMEA's response is consistent with feedback previously received from United States Food and Drug Administration (FDA), paving the way for potential approval in the US and EU.
DOR and the FDA reached agreement on the design of the upcoming Phase 3 study via the 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.
Based on data from the prior Phase 3 study of orBec(R), the upcoming confirmatory Phase 3 clinical trial will be a highly powered, double-blind, randomized, placebo-controlled, multicenter 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 orBec(R) (i.e., 30 days following cessation of treatment). The confirmatory Phase 3 clinical trial will be conducted at major transplant centers throughout the US and Canada.
'We are very pleased to obtain a positive response from the EMEA regarding the design of our upcoming 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 a pivotal trial that we believe maximizes orBec(R)'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 orBec(R).'
Dr. Schaber continued, 'This feedback, in conjunction with the SPA agreement, clearly validates the direction of the orBec(R) clinical development path that has the potential to lead to regulatory approval of orBec(R) in both the US and the European Union. We are excited to move forward with this trial in an effort to address the significant unmet medical need of acute GI GVHD. We anticipate patient enrollment to commence in the second half of 2009.'
About orBec(R)
Two prior randomized, double-blinded, placebo-controlled Phase 2 and 3 clinical trials support orBec(R)'s ability to provide clinically meaningful outcomes when compared with the current standard of care, including a lowered exposure to systemic corticosteroids following allogeneic transplantation. Currently, there are no approved products to treat GI GVHD. The first trial was a 60-patient Phase 2 single-center clinical trial conducted at the Fred Hutchinson Cancer Research Center. The second trial was a 129-patient pivotal Phase 3 multi-center clinical trial of orBec(R) conducted at 16 leading bone marrow/stem cell transplantation centers in the US and France.
