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DOR BioPharma Files new Drug Application with FDA to Market orBec for GI GVHD

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DOR BioPharma, Inc. has announced that it has submitted in the Electronic Common Technical Document format a New Drug Application ("NDA") to the U.S. Food and Drug Administration ("FDA") to market orBec® (oral beclomethasone dipropionate) for the treatment of gastrointestinal Graft-versus-Host disease ("GI GVHD"), the most common life-threatening complication of allogeneic hematopoetic stem cell transplantation.

orBec® is a two pill system containing the potent topically active corticosteroid beclomethasone dipropionate designed to target and treat upper and lower GI GVHD with reduced systemic immunosuppressive side effects.

Systemic immunosuppressive agents, such as prednisone, that are currently the standard treatment for GI GVHD are associated with high rates of mortality due to infection and debility.

orBec® has previously been granted Fast Track designation for the treatment of GI GVHD by the FDA and thus may qualify for priority review.

The data provided in the NDA submission demonstrate that orBec® safely provides a lowered risk of mortality above the current standard of care, and a lowered exposure to systemic corticosteroids following allogeneic transplantation. 

The NDA filing is supported by data from two randomized, double-blinded, placebo controlled clinical trials.

The first was a 129 patient pivotal Phase III clinical trial for orBec® conducted at 16 bone marrow/stem cell transplant centers in the U.S. and France.

The second trial was a 60 patient Phase II clinical trial conducted at the Fred Hutchinson Cancer Institute.

While orBec® did not achieve statistical significance in the primary endpoint of its pivotal trial, namely time to treatment failure through Day 50 (p-value 0.1177), orBec® did achieve statistical significance in other key outcomes such as median time to treatment failure through Day 80 (p-value 0.0226), and most importantly, it demonstrated a statistically significant survival advantage in comparison to placebo.

In the pivotal Phase III trial, analysis of patient survival at the pre-specified endpoint of 200 days post-transplant showed a clinically meaningful and statistically significant 66% reduction (p-value 0.0139) in mortality among patients randomized to orBec®.

The mortality benefit in favor of orBec® was corroborated earlier this year in a retrospective analysis of the Phase II study in which there was a 55% reduction in mortality at 200 days post transplant.

At one year after randomization, there were relatively consistent 51% and 45% reductions in the risk of mortality among patients randomized to orBec® in both the Phase III and Phase II studies, respectively.

In the pivotal Phase III trial, a subgroup analysis also revealed that patients dosed with orBec® who had received stem cells from unrelated donors had a 94% reduction in the risk of day-200 mortality.

These data were previously reported at the Tandem BMT Meeting of the American Society for Bone Marrow Transplantation, Honolulu, HI (Oral beclomethasone dipropionate for gastrointestinal GVHD: a corticosteroid-sparing treatment with improved survival at day +200.

Biology of Blood and Marrow Transplantation 12 (Supplement 1): 1 - 2, 2006.) The pivotal Phase III study results as well as the extended mortality results from both trials have been submitted for publication in a peer-reviewed medical journal.

"Filing a first NDA is a major milestone for any company and therefore this is a very exciting time for us," stated Christopher J. Schaber, Ph.D., President and Chief Executive Officer of DOR.

"We have submitted a high quality data package to the FDA that we believe clearly demonstrates the benefit of orBec® for the treatment of GI GVHD."

"I would like to sincerely thank everyone who has contributed to reaching this major milestone."

"I am very confident in the positive outcomes that our orBec® data have demonstrated, and I am looking forward to productive interactions with the FDA as we move forward through the review process with this potentially first in class product."

"There are over 10,000 allogeneic stem cell transplants per year," added Dr. Schaber.

"It is estimated that allogeneic transplants will increase by as much as 20% annually over the next several years, with cord-blood and reduced intensity conditioning regimens (known as 'mini-transplants') in the elderly fueling much of this growth. Unfortunately, more than half of these patients will go on to develop GI GVHD that requires treatment."

"We believe that orBec®, if approved, will potentially provide transplant physicians with an effective and much needed tool to treat their GI GVHD patients and improve survival."

DOR is preparing a Marketing Authorization Application ("MAA") for orBec® to be filed with the European Medicines Evaluation Authority ("EMEA") in November 2006.

In addition to issued and pending patent applications held by or exclusively licensed by DOR, orBec® also enjoys orphan drug designations in the U.S. and in Europe for the treatment of GI GVHD which provide for 7 and 10 years of post-approval market exclusivity, respectively.

George B. McDonald, MD, Head of the Gastroenterology/Hepatology Section at the Fred Hutchinson Cancer Research Center, inventor of orBec® and a consultant to DOR stated, "It is often difficult for those not involved in hematopoietic cell transplants to understand how devastating the problem of GVHD and its treatment can be for patients and their families."

"Not only are the gastrointestinal symptoms unpleasant and persistent, but the standard treatment using prolonged courses of prednisone leads to debility and to serious infections."

"orBec® treatment controls the symptoms of GI GVHD without having to resort to prolonged prednisone exposure."

"I find the data in support of orBec® to be compelling, and look forward to the time when this important new drug can be made available to patients undergoing hematopoietic cell transplant."