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Patient Enrollment Completed for Phase 3 Oral Beclomethasone Dipropionate Trial

MIAMI--July 14, 2004--DOR BioPharma, Inc. announced the completion of patient enrollment in DOR's multi-center, pivotal Phase III clinical trial of orBec (oral beclomethasone dipropionate) for the treatment of acute intestinal Graft-versus-Host Disease (GvHD), a severe, potentially life-threatening complication of bone marrow and stem cell transplant. "With the completion of enrollment for this trial we have achieved a major milestone as we move closer to bringing our lead product, orBec®, to market," said DOR's President and acting Chief Executive Officer, Geoff Green. "The treatment phase of the trial will conclude in September, and we anticipate having unblinded data available in the fourth quarter of this year. If, after data analysis, we have positive safety and efficacy data, we anticipate filing a New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) in the first quarter 2005."

OrBec, if approved by the FDA, would be the first oral formulation of beclomethasone dipropionate (BDP) available in the United States. BDP is a highly potent, topically-active corticosteroid that has a local effect on inflamed tissue. BDP has been marketed in the U.S. and worldwide since the early 1970s as a nasal spray and a metered dose inhaler for the treatment of patients with allergic rhinitis and asthma. DOR's oral formulation of BDP is delivered to the gastrointestinal (GI) tract for topical treatment of inflammation within the mucosal tissue. OrBec® is being developed as a two-pill system with dual release characteristics that initially begins to release BDP in the stomach, and continues to release BDP as it travels down the GI tract for broader coverage in the intestines.

Acute GvHD is a frequent complication of bone marrow transplants. In a bone marrow transplant, the patient's diseased bone marrow is destroyed and healthy marrow from a donor (allogeneic transplants) is infused into the patient's blood-stream. The healthy transplanted marrow of the donor often launches an attack on its new host, known as Graft-versus-Host Disease, resulting in inflammation and tissue damage in the mucosal lining of the gastrointestinal tract, as well as the liver and skin.

Orphan Drug Designation and "Fast Track" Designation
OrBec has previously been granted Orphan Drug Designation and has received "fast track" designation from the FDA. The FDA reserves fast track designation for products intended to treat a serious or life threatening condition, and that have the potential to address an unmet medical need. Although it is not guaranteed, products in the fast track development program may be considered for priority review, which is a six month review period from receipt of a complete NDA, and may be considered for a "rolling" submission, which allows for submission of components of the NDA for initial review and feedback prior to submission of the complete NDA.

About the Phase III Pivotal Trial
One hundred and twenty-nine (129) patients were enrolled in the double-blind, placebo controlled, multi-center clinical trial, which was conducted at 16 bone marrow transplant centers in the United States and France. All patients in the Phase III clinical trial were treated initially with a constant daily dose of prednisone, which is the current standard therapy, in combination with either orBec or placebo for the first 10 days. On day 10, if patients were responding to treatment, the prednisone was rapidly tapered and the orBec or placebo continued. The primary endpoint of the study is a comparison of the two treatment arms of the median time to treatment failure, defined as the need for additional therapies due to uncontrolled signs or symptoms of GvHD. Other endpoints in the study include a comparison of the proportion of treatment failures at specified time points, as well as a comparison of cumulative exposure to systemic steroids.

Oral BDP was previously tested in a randomized, placebo-controlled Phase II study (Gastroenterology, 1998; 115: 28-35). In that study, 60 patients with intestinal GvHD were randomized to receive conventional therapy plus either oral BDP or placebo. Initial responders continued to take BDP or placebo for an additional 20 days, during which the conventional therapy was rapidly tapered. The treatment response at day 30 was 22 of 31 (71%) vs. 12 of 29 (41%) in the placebo group, respectively (P= 0.02).

Source: DOR BioPharma

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