SOURCE: DOR BioPharma, Inc.

January 03, 2008 07:30 ET

DOR BioPharma Receives Orphan Drug Designation for Oral BDP for the Treatment of Pediatric Crohn's Disease

Treatment Could Benefit Approximately 100,000 Pediatric Patients in the US Afflicted With Crohn's Disease

EWING, NJ--(Marketwire - January 3, 2008) - DOR BioPharma, Inc., (OTCBB: DORB) ("DOR" or the "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 dipropionate) for the treatment of pediatric Crohn's disease.

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 Oral BDP 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 Oral BDP, and certain tax credits.

Oral BDP is a highly potent, topically active corticosteroid that has a local effect on inflamed tissue. BDP 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. BDP is also the active ingredient in orBec®, currently in development by DOR for the treatment and prevention of Graft-versus-Host disease (GVHD).

"The FDA's decision to grant Oral BDP Orphan Drug Designation for the treatment of pediatric Crohn's disease signifies an important step for DOR as we begin to expand our pipeline with Oral BDP into other important indications beyond GVHD," stated Christopher J. Schaber, PhD, President and CEO of DOR BioPharma. "We anticipate that pediatric Crohn's disease will be the first of several new Orphan Drug Designations both in the US and in Europe as we work to maximize the global value of our Oral BDP and orBec® franchise. We believe that Oral BDP has the potential to be of significant benefit to pediatric Crohn's disease patients."

About Pediatric Crohn's Disease

Crohn's disease is an ongoing disorder that causes inflammation of the gastrointestinal (GI) tract. Crohn's disease can affect any area of the GI tract, from the mouth to the anus, but it most commonly affects the lower part of the small intestine, called the ileum. The swelling caused by the disease extends deep into the lining of the affected organ. The swelling can induce pain and can make the intestines empty frequently, resulting in diarrhea. Because the symptoms of Crohn's disease are similar to other intestinal disorders, such as irritable bowel syndrome and ulcerative colitis, it can be difficult to diagnose. People of Jewish heritage have an increased risk of developing Crohn's disease.

Crohn's disease can appear at any age, but it is most often diagnosed in adults in their 20s and 30s. However, approximately 30% of people with Crohn's disease develop symptoms before 20 years of age. Pediatric Crohn's disease is a subpopulation of patients 0-19 years of age who suffer from Crohn's disease. In the United States, about 100,000 teens and preteens have Crohn's disease.

Crohn's disease presents special challenges for children and teens. In addition to bothersome and often painful symptoms, the disease can stunt growth, delay puberty, and weaken bones. Crohn's disease symptoms may sometimes prevent a child from participating in enjoyable activities. The emotional and psychological issues of living with a chronic disease can be especially difficult for young people.

About Oral BDP

Beclomethasone dipropionate is a highly potent, topically active corticosteroid and is the active ingredient in orBec®, DOR's product candidate for the treatment of gastrointestinal Graft-versus-Host disease (GI GVHD), a common disorder among immunocompromised cancer patients after receiving allogeneic stem cell or bone marrow transplants. Like pediatric Crohn's disease, GI GVHD is also an inflammatory condition of the GI tract. Patients with mild-to-moderate GI GVHD typically develop symptoms of anorexia, nausea, vomiting and diarrhea.

DOR has previously run two randomized, double-blinded, placebo-controlled clinical trials in 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 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 five 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).

About DOR BioPharma, Inc.

DOR BioPharma, Inc. (DOR) is a 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), is a potent, locally acting corticosteroid being developed for the treatment of gastrointestinal Graft-versus-Host disease (GI GVHD), a common and potentially life-threatening complication of bone marrow transplantation. DOR filed a New Drug Application for orBec® with the FDA for the treatment of acute GI GVHD and received a not approvable letter in which the FDA has requested data from an additional clinical trial to demonstrate the safety and efficacy of orBec®. A Marketing Authorization Application with the European Medicines Evaluation Agency has also been validated and is under review. orBec® is currently the subject of an NIH-supported, Phase 2, randomized, double-blind, placebo-controlled trial in the prevention of acute GVHD. orBec® may also have application in treating other gastrointestinal disorders characterized by severe inflammation. DOR has initiated a development program with its Lipid Polymer Micelle (LPM™) drug delivery technology for the oral delivery of leuprolide for the treatment of prostate cancer and endometriosis, as well as a development program with its oral azathioprine technology for the treatment of oral GVHD.

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 and botulinum toxin. DOR's ricin toxin vaccine, RiVax™, has been shown to be well tolerated and immunogenic in a Phase 1 clinical trial in normal volunteers.

For further information regarding DOR BioPharma, please visit the Company's website located at www.dorbiopharma.com.

This press release contains forward-looking statements, within the meaning of Section 21E of the Securities Exchange Act of 1934, that reflect DOR BioPharma, Inc.'s current expectations about its future results, performance, prospects and opportunities, including statements regarding the potential use of orBec® for the treatment of gastrointestinal GVHD and the prospects for regulatory filings for orBec®. Where possible, DOR has tried to identify these forward-looking statements by using words such as "anticipates," "believes," "intends," or similar expressions. 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®, 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 technologies will prove to be safe and effective, that its cash expenditures will not exceed projected levels, that it will be able to obtain future financing or funds when needed, 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®. 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 Form 10-QSB and Form 10-KSB. DOR assumes no obligation to update or revise any forward-looking statements as a result of new information, future events, and changes in circumstances or for any other reason.