DOR BioPharma Announces Results of orBec(R) End of Review Conference With the FDA
Friday December 7, 7:00 am ET
EWING, NJ--(MARKET WIRE)--Dec 7, 2007 -- DOR BioPharma, Inc. (OTC BB:DORB.OB - News) ("DOR" or the "Company") today reported the results of its recent End of Review Conference with the Food and Drug Administration (FDA) for the orBec® New Drug Application (NDA). The purpose of the meeting was to gain further clarification regarding the deficiencies noted in the October 18, 2007 not approvable letter and the data required to gain approval for orBec® for the treatment of gastrointestinal Graft-versus-Host disease (GI GVHD).
A summary of the important results of the FDA meeting are as follows:
1. A single, confirmatory, Phase 3 clinical trial could provide sufficient
evidence of efficacy provided that it is well designed, well executed
and provides clinically and statistically meaningful findings.
2. DOR anticipates working quickly with the FDA to finalize the design of
the confirmatory trial and will be requesting a Special Protocol
Assessment (SPA) meeting in the near future.
3. The FDA would be agreeable to reviewing a plan for a Treatment IND as
long as it does not interfere with patient accrual in a confirmatory
trial, such as potentially enrolling patients that would not be
eligible for the Phase 3 study.
"Our recent meeting with the FDA was a productive one. We are pleased to have clarification on clinical issues resulting from our recent PDUFA letter," stated Christopher J. Schaber, PhD, President and CEO of DOR BioPharma. "Once we obtain agreement from the FDA on the design of the next clinical trial, we will be able to provide guidance on trial size and timeline and also anticipate being able to begin designing a plan for the Treatment IND. Given the pharmacology and clinically important data orBec® has already demonstrated in Phase 2 and 3 clinical trials, both DOR and its clinical investigators are eager to move forward as quickly as possible."
About orBec®
orBec® is a two-tablet system containing the highly potent, topically active corticosteroid beclomethasone dipropionate, and is designed to specifically target and treat upper and lower GI GVHD with reduced systemic immunosuppressive side effects.
Two prior randomized, double-blinded, placebo-controlled Phase 2 and 3 clinical trials demonstrate that orBec® provides 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® conducted at 16 leading bone marrow/stem cell transplant centers in the US and France. Although orBec® did not achieve statistical significance in the primary endpoint of its pivotal trial, namely median time to treatment failure through Day 50 (p-value 0.1177), orBec® did achieve statistical significance in other key secondary endpoints such as median time to treatment failure through Day 80 (p-value 0.0226), as well as a 66% reduction in mortality among patients randomized to orBec® at 200 days post-transplant with only 5 patient (8%) deaths in the orBec® group compared to 16 patient (24%) deaths in the placebo group (p-value 0.0139). At one year post randomization in the pivotal Phase 3 trial, 18 patients (29%) in the orBec® group and 28 patients (42%) in the placebo group died within one year of randomization (46% reduction in mortality, hazard ratio 0.54, 95% CI: 0.30, 0.99, p=0.04, stratified log-rank test).
In the Phase 2 study, the primary endpoint was the clinically relevant determination of whether GI GVHD patients at Day 30 had a durable treatment response as measured by whether or not they were able to consume at least 70% of their daily caloric intake by mouth, as compared to intravenous parenteral nutrition administered in the hospital. The treatment response at Day 30 was 22 of 31 (71%) vs. 12 of 29 (41%) in the orBec® and placebo groups respectively, achieving a statistically significant p-value of 0.02. Additionally, the treatment response at Day 40 was 16 of 31 (52%) vs. 5 of 29 (17%) in the orBec® and placebo groups respectively, achieving a statistically significant p-value of 0.007.
About GI GVHD
GVHD is a debilitating and painful disease. It is a common disorder among immunocompromised cancer patients after receiving allogeneic stem cell or bone marrow transplants. Unlike organ transplants where the patient's body may reject the organ, in GVHD it is the donor cells that begin to attack the patient's body -- most frequently the gut, liver and skin. Patients with mild-to-moderate GI GVHD typically develop symptoms of anorexia, nausea, vomiting and diarrhea. If left untreated, GI GVHD can progress to ulcerations in the lining of the GI tract, and in its most severe form, can be fatal.
Systemic immunosuppressive agents such as prednisone are the current standard treatments for GI GVHD and are associated with high mortality rates due to infection and debility. Further, these drugs have not been approved for treating GI GVHD in the US or European Union, but rather are used off-label as investigational therapies for this indication. |
