DOR BioPharma, Inc. (DORB) Receives FDA Orphan Drug Designation for orBec(R) for the Treatment of Chronic Gastrointestinal GVHD
PRINCETON, N.J., Aug. 4 /PRNewswire-FirstCall/ -- DOR BioPharma, Inc., (OTC Bulletin Board: DORB - News; DOR or the Company), a late-stage biopharmaceutical company, announced today that the Office of Orphan Products Development of the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation to Oral BDP (beclomethasone 17,21-dipropionate, or orBec(®)) for the treatment of gastrointestinal symptoms associated with chronic Graft-versus-Host disease (cGVHD) in patients who have undergone allogeneic hematopoietic cell transplantation.
The US Orphan Drug Act is intended to assist and encourage companies to develop safe and effective therapies for the treatment of rare diseases and disorders. In addition to providing a seven-year term of market exclusivity for orBec(® )upon final FDA approval, orphan drug designation also positions DOR to be able to take advantage of a wide range of financial and regulatory benefits, including government grants for conducting clinical trials, waiver of expensive FDA user fees for the potential submission of a New Drug Application for orBec(®), and certain tax credits.
orBec(® )is a highly potent, topically active corticosteroid that has a local effect on inflamed tissue. BDP, the active component of orBec(®), has been marketed in the United States and worldwide since the early 1970s as the active pharmaceutical ingredient in inhalation products for the treatment of patients with allergic rhinitis and asthma. orBec(®) and oral BDP are currently in development by DOR for the treatment and prevention of GVHD, the prevention of acute radiation enteritis and the treatment of Crohn's disease.
"The FDA's decision to grant orBec(®) Orphan Drug Designation for the treatment of GI symptoms of cGVHD marks another step in the expansion of our orBec(®)/oral BDP pipeline," stated Christopher J. Schaber, PhD, President and CEO of DOR BioPharma. "Based on data we have already generated in the treatment of acute GI GVHD, we believe that orBec(® )has the potential to be of significant benefit to cGVHD patients."
About Chronic GVHD
Graft-versus-Host disease (GVHD) is a major complication of allogeneic hematopoietic cell transplantation. GVHD is an inflammatory disease initiated by T cells in the donor graft that recognize histocompatibility and other tissue antigens of the host, and is mediated by a variety of effector cells and inflammatory cytokines. GVHD presents in both acute and chronic forms. The symptoms of cGVHD typically present at between 100 days and three years post-transplant.
Chronic GVHD has features resembling autoimmune and other immunologic disorders such as scleroderma, Sjogren syndrome, primary biliary cirrhosis, wasting syndrome, bronchiolitis obliterans (BO), immune cytopenias and chronic immunodeficiency. The manifestations of cGVHD may be restricted to a single organ or tissue or may be widespread. Chronic GVHD can lead to debilitating consequences, e.g., joint contractures, loss of sight, end-stage lung disease, or mortality resulting from profound chronic immune suppression leading to recurrent or life-threatening infections.
Treatment of cGVHD is a challenge because it can be refractory to frontline immunosuppression. High-dose systemic corticosteroids are used with some success but carry significant toxicity. The risks of prolonged immunosuppression include local and disseminated infections, Epstein-Barr virus associated lymphoproliferative disease, hypothalamic-pituitary-adrenal (HPA) axis suppression, myopathy, glucose intolerance, neuropsychiatric disease and bone demineralization.
About orBec(® )
Beclomethasone dipropionate is a highly potent, topically active corticosteroid and is the active ingredient in orBec(®), DOR's product candidate for the treatment of acute GI GVHD, a common disorder among immunocompromised cancer patients after receiving allogeneic stem cell or bone marrow transplants. Similar to acute GVHD, there is also an inflammatory condition of the GI tract associated with cGVHD. Furthermore, acute GI GVHD occurs in approximately 60 percent of related donor and 70 percent of unrelated donor allogeneic transplantation patients and is a risk factor for development of cGVHD.
DOR has previously run two randomized, double-blind, placebo-controlled clinical trials in acute GI GVHD with orBec(®). 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 conducted at 16 leading bone marrow/stem cell transplant centers in the United States 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 the proportion of patients free of GVHD at Day 50 (p-value 0.05) and Day 80 (p-value 0.005) and the 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 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, p=0.04).
Based on data from the prior Phase 3 study of orBec®, the upcoming confirmatory Phase 3 protocol will be a highly powered, double-blind, randomized, placebo-controlled, multi-center 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® (i.e., 30 days following cessation of treatment).
About DOR BioPharma, Inc.
DOR BioPharma, Inc. (DOR) is a late-stage biopharmaceutical company developing products to treat life-threatening side effects of cancer treatments and serious gastrointestinal diseases, and vaccines for certain bioterrorism agents. DOR's lead product, orBec® (oral beclomethasone dipropionate or BDP), is a potent, locally acting corticosteroid being developed for the treatment of GI GVHD, a common and potentially life-threatening complication of hematopoietic cell transplantation. DOR expects to begin a confirmatory Phase 3 clinical trial of orBec® for the treatment of acute GI GVHD and a Phase 1/2 clinical trial of DOR201 in radiation enteritis in the second half of 2009. orBec® is also currently the subject of an NIH-supported, Phase 2, randomized, double-blind, placebo-controlled trial in the prevention of acute GVHD. Oral BDP may also have application in treating other gastrointestinal disorders characterized by severe inflammation. Additionally, DOR has a Lipid Polymer Micelle (LPM(TM)) drug delivery technology for the oral delivery of leuprolide for the treatment of prostate cancer and endometriosis.
Through its Biodefense Division, DOR is developing biomedical countermeasures pursuant to the Project BioShield Act of 2004. DOR's biodefense products in development are recombinant subunit vaccines designed to protect against the lethal effects of exposure to ricin toxin, botulinum toxin and anthrax. DOR's ricin toxin vaccine, RiVax(TM), has been shown to be well tolerated and immunogenic in a Phase 1 clinical trial in normal volunteers.
For further information regarding DOR BioPharma, Inc., please visit the Company's website at www.dorbiopharma.com.
This press release contains forward-looking statements that reflect DOR BioPharma, Inc.'s current expectations about its future results, performance, prospects and opportunities. Statements that are not historical facts, such as "anticipates," "believes," "intends," or similar expressions, are forward-looking statements. These statements are subject to a number of risks, uncertainties and other factors that could cause actual events or results in future periods to differ materially from what is expressed in, or implied by, these statements. DOR cannot assure you that it will be able to successfully develop or commercialize products based on its technology, including orBec® and LPM(TM), particularly in light of the significant uncertainty inherent in developing vaccines against bioterror threats, manufacturing and conducting preclinical and clinical trials of vaccines, and obtaining regulatory approvals, that its cash expenditures will not exceed projected levels, that product development and commercialization efforts will not be reduced or discontinued due to difficulties or delays in clinical trials or due to lack of progress or positive results from research and development efforts, that it will be able to successfully obtain any further grants and awards, maintain its existing grants which are subject to performance, enter into any biodefense procurement contracts with the US Government or other countries, that the US Congress may not pass any legislation that would provide additional funding for the Project BioShield program, that it will be able to patent, register or protect its technology from challenge and products from competition or maintain or expand its license agreements with its current licensors, or that its business strategy will be successful. Important factors which may affect the future use of orBec® for gastrointestinal GVHD include the risks that: the FDA's requirement that DOR conduct additional clinical trials to demonstrate the safety and efficacy of orBec® will take a significant amount of time and money to complete and positive results leading to regulatory approval cannot be assumed; DOR is dependent on the expertise, effort, priorities and contractual obligations of third parties in the clinical trials, manufacturing, marketing, sales and distribution of its products; orBec® may not gain market acceptance if it is eventually approved by the FDA; and others may develop technologies or products superior to orBec®. Factors affecting the development and use of LPM(TM) are similar to those affecting orBec®. These and other factors are described from time to time in filings with the Securities and Exchange Commission, including, but not limited to, DOR's most recent reports on Forms 10-Q and 10-K. Unless required by law, DOR assumes no obligation to update or revise any forward-looking statements as a result of new information or future events.