orBecTreatment for Intestinal Graft-versus-Host Disease (iGVHD)
DOR BioPharma to File NDA for orBec
MIAMI, April 20, 2005 - DOR BioPharma, Inc. (AMEX: DOR) ("DOR" or the "Company") announced its intention to file a new drug application (NDA) with the FDA for orBec (oral beclomethasone dipropionate) for the treatment of intestinal Graft-versus-Host Disease (iGVHD), a common serious complication of bone marrow transplantation for cancer. The decision follows recent correspondence and a pre-NDA meeting with the FDA.
"We are very encouraged by our meeting with the FDA," stated Michael Sember, President and Chief Executive Officer of DOR. "We are excited with the results of our pivotal Phase III clinical trial. In addition to the strong positive trends on our primary endpoint of median time to treatment failure at study Day 50 in our pivotal trial, we saw a statistically significant result in median time to treatment failure at study Day 80 (p-value 0.0226). This result, combined with a highly statistically significant outcome and approximately 70% reduction in the prospectively defined secondary endpoint of mortality at 200 days post transplant (p-value 0.006), has given us confidence in the consistency and clinical durability of activity of orBec. We believe that our pivotal Phase III clinical trial of orBec and collectively all the clinical data before it, clearly demonstrate a clinical benefit to patients with no other alternative except high dose systemic corticosteroids. Our immediate priorities are to assemble a high quality data package to support the filing of an NDA as soon as possible but by the fourth quarter of 2005, and to submit the results of the pivotal trial for publication in a prominent peer-reviewed publication. We will also take steps to prepare for submission of a Marketing Authorization Application (MAA) with the European Medicines Evaluation Agency (EMEA)."
orBec has previously been granted Orphan Drug Designation and has received "Fast Track" designation from the FDA.
George B. McDonald, MD, Head of the Gastroenterology/Hepatology Section at the Fred Hutchinson Cancer Research Center in Seattle, inventor of orBec, and a consultant to DOR stated, "Those working in the field of hematopoietic cell transplantation have long recognized that prolonged exposure to prednisone in patients with iGVHD led not only to weakness, debility, and changes in body habitus, but also to viral, bacterial, and fungal infections. The pivotal Phase III study provides a clear rationale for the use of orBec in the treatment of iGVHD; control of iGVHD with an oral topical corticosteroid leads to less prednisone exposure, less systemic immunosuppression, fewer fatal infections, and possibly an enhanced graft-versus-leukemia (GVL) effect. The frequency of leukemic relapse after allogeneic hematopoietic cell transplant is lower in patients who experience GVHD after transplant, a phenomenon called the GVL effect. We hypothesize that avoidance of prolonged prednisone exposure preserves the GVL effect, resulting in fewer relapses of leukemia. orBec appears to be safe. Thus, the benefit-to-risk ratio for the use of orBec in the treatment of iGVHD seems strongly in favor of benefit. orBec addresses the unmet medical need for an effective adjunctive therapy for iGVHD with minimal side effects."
About Intestinal Graft-versus-Host Disease
iGVHD is a life threatening condition that is one of the most common causes for the failure of bone marrow transplant procedures. These procedures are being increasingly utilized to treat leukemia and other cancer patients with the prospect of eliminating residual disease and reducing the likelihood of relapse. orBec represents a first-of-its-kind oral, locally acting therapy tailored to treat the gastrointestinal manifestation of GVHD, the organ system where GVHD is most frequently encountered and highly problematic. orBec is intended to reduce the need for systemic immunosuppressive drugs to treat iGVHD.
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 1970's as a nasal spray and in a metered dose inhaler for the treatment of patients with allergic rhinitis and asthma. orBec 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.
Prior Study Results
orBec was previously tested in a randomized, double blind, placebo-controlled Phase II study (Gastroenterology, 1998; 115: 28-35). In that study, 60 patients with iGVHD were randomized to receive conventional prednisone therapy plus either orBec or placebo. Initial responders continued to take orBec or placebo for an additional 20 days, during which time the conventional therapy was rapidly tapered. The primary endpoint for this study was the clinically relevant determination of whether iGVHD patients at Day 30 were or were not 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.
About DOR BioPharma, Inc.
DOR BioPharma, Inc. is a biopharmaceutical company focused on the development of biomedical countermeasures and therapeutic products for areas of unmet medical need. Through our BioDefense Division, we are developing biomedical countermeasures pursuant to the paradigm established by the recently enacted Project BioShield Act of 2004. Our biodefense products in development are bioengineered vaccines designed to protect against the deadly effects of ricin toxin and botulinum toxin, both of which are considered serious bioterrorism threats. Our ricin toxin vaccine is currently the subject of a Phase I clinical trial in normal volunteers. We have also recently announced the initiation of a new botulinum toxin therapeutic development program based on rational drug design.
Through our BioTherapeutics Division, we are developing oral therapeutic products to treat unmet medical needs. Our lead product, orBec (oral beclomethasone dipropionate), is a potent, locally-acting corticosteroid being developed for the treatment of iGVHD as well as other gastrointestinal disorders characterized by severe inflammation.
For further information, please visit www.dorbiopharma.com.
Posted: April 2005
- DOR BioPharma Announces Results of orBec End of Review Conference with the FDA - December 7, 2007
- DOR BioPharma Receives Not Approvable Letter From FDA for orBec for Treatment of Gastrointestinal Graft-vs.-Host Disease - October 19, 2007
- DOR BioPharma, Inc. Announces 3 Month Extension of FDA PDUFA Action Date for orBec New Drug Application to Review Supplemental Data - July 19, 2007
- DOR BioPharma Files New Drug Application with FDA to Market orBec for GI GVHD - September 22, 2006
- DOR BioPharma Provides Regulatory Update on orBec - June 30, 2006
- DOR BioPharma Plans FDA NDA and European MAA Filing Strategy for orBec - November 15, 2005