Human Genome Sciences (HGS) and GlaxoSmithKline (GSK) announced positive results from BLISS-76, the second of two large-scale phase III clinical trials of BENLYSTA™ (belimumab) for treating systemic lupus. A full presentation of results from BLISS-52 was recently shared at the 73rd Annual Scientific meeting of the American College of Rheumatology. Both trials succeeded in meeting their primary endpoints, which should make BENLYSTA eligible for approval by the U.S. Food and Drug Administration (FDA).

Both trials demonstrate that treatment with BENLYSTA plus standard of care was superior to that of placebo (inactive agent) plus standard of care. BENLYSTA significantly reduced disease activity. If approved by the FDA, BENLYSTA would be the first drug ever developed and approved specifically for the treatment of lupus.

Sandra C. Raymond, President and Chief Executive Officer of the Lupus Foundation of America (LFA) issued the following statement.

“We are truly excited to receive this groundbreaking news! Individuals with lupus and their families have waited more than 50 years to hear that it is possible to develop therapies that control the disease. We believe that this is a significant first step in developing the full arsenal of therapies and personalized treatment lupus requires.

“Conducting clinical trials in lupus has been extremely difficult due to many factors including the heterogeneity of the disease, the selection of appropriate clinical trial endpoints, and the confounding role of required background medications given to clinical trial participants. Human Genome Sciences and GlaxoSmithKline have proven that these barriers, while formidable, can be overcome.

“For decades the entire lupus research community has worked hard to better understand the causes and consequences of the disease. The fruits of that labor are starting to emerge. However, now is not the time for complacency. We must band together for lupus and continue to capitalize on the decades of research made possible through the efforts of the many dedicated researchers, physicians, people with lupus, and advocates.

“This announcement by HGS and GSK and the Overcoming Barriers to Drug Development in Lupus report, commissioned by the Lupus Foundation of America to outline recommendations on ways to overcome the barriers to lupus research, combine to serve as a call to action for a national coordinated effort to accelerate the pace of discovery, to develop more tolerable and effective treatments, and to ultimately find a cure for this perilous disease.

“We congratulate HGS and GSK on reaching this important milestone in lupus research and in the development of new therapies for lupus. We also extend our appreciation to the researchers and study volunteers who made this achievement possible: the physicians who have passionately committed to researching this disease, and the companies that continue to invest in finding new and necessary treatments for this devastating disease.”

The next step in the process is for HGS and GSK to submit marketing applications in the United States, Europe and other regions during the first half of 2010. The LFA will closely follow this process, and continue to keep its constituents apprised of developments.

Source
Lupus Foundation of America

DOR BioPharma, Inc. (DOR or the Company) (OTC Bulletin Board: DORB), a late-stage biopharmaceutical company, announced that it has received Protocol Assistance feedback from the European Medicines Agency (EMEA) on the design of its confirmatory, pivotal, Phase 3 clinical trial evaluating its lead product orBec(R) for the treatment of acute gastrointestinal Graft-versus-Host Disease (GI GVHD).

The EMEA agreed that should the new confirmatory Phase 3 study produce positive results, the data would be sufficient to support a marketing authorization approval in all 27 European Union (EU) member states. In so doing, the EMEA agreed to the primary endpoint and the other principal design features of the new study. The EMEA’s response is consistent with feedback previously received from United States Food and Drug Administration (FDA), paving the way for potential approval in the US and EU.

DOR and the FDA reached agreement on the design of the upcoming Phase 3 study via the Special Protocol Assessment (SPA) procedure. An agreement via the SPA procedure is an agreement with the FDA that a Phase 3 clinical trial’s design (e.g., endpoints, sample size, control group and statistical analyses) is acceptable to support a regulatory submission seeking new drug approval.

Based on data from the prior Phase 3 study of orBec(R), the upcoming confirmatory Phase 3 clinical trial will be a highly powered, double-blind, randomized, placebo-controlled, multicenter 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(R) (i.e., 30 days following cessation of treatment). The confirmatory Phase 3 clinical trial will be conducted at major transplant centers throughout the US and Canada.

“We are very pleased to obtain a positive response from the EMEA regarding the design of our upcoming confirmatory, pivotal Phase 3 study,” stated Christopher J. Schaber, PhD, President and Chief Executive Officer of DOR. “The depth and strength of our available Phase 3 data have allowed us to design and power a pivotal trial that we believe maximizes orBec(R)’s chances for success. With our primary endpoint of the ‘treatment failure rate at Study Day 80,’ we expect to replicate statistical significance in this clinically meaningful endpoint with orBec(R).”

Dr. Schaber continued, “This feedback, in conjunction with the SPA agreement, clearly validates the direction of the orBec(R) clinical development path that has the potential to lead to regulatory approval of orBec(R) in both the US and the European Union. We are excited to move forward with this trial in an effort to address the significant unmet medical need of acute GI GVHD. We anticipate patient enrollment to commence in the second half of 2009.”

About orBec(R)

Two prior randomized, double-blinded, placebo-controlled Phase 2 and 3 clinical trials support orBec(R)’s ability to provide 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(R) conducted at 16 leading bone marrow/stem cell transplantation centers in the US and France. Although orBec(R) 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(R) 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(R) at 200 days post-transplant with only 5 patient (8%) deaths in the orBec(R) 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(R) 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 (the end of treatment) had a durable GVHD treatment response as measured by whether or not they were able to consume at least 70% of their estimated caloric requirement. The GVHD treatment response at Day 30 was 22 of 31 (71%) vs. 12 of 29 (41%) in the orBec(R) and placebo groups, respectively (p-value 0.02). Additionally, the GVHD treatment response at Day 40 (10 days post cessation of therapy) was 16 of 31 (52%) vs. 5 of 29 (17%) in the orBec(R) and placebo groups, respectively (p-value 0.007).

orBec(R) 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(R) is intended to reduce the need for systemic immunosuppressive drugs to treat GI GVHD. Beclomethasone dipropionate (BDP) is a highly potent, topically active corticosteroid that has a local effect on inflamed tissue. BDP has been marketed in the US and worldwide since the early 1970s as the active pharmaceutical ingredient in a nasal spray and in a metered-dose inhaler for the treatment of patients with allergic rhinitis and asthma. orBec(R) is formulated for oral administration as a single product consisting of two tablets; one tablet is intended to release BDP in the proximal portions of the GI tract, and the other tablet is intended to release BDP in the distal portions of the GI tract.

In addition to issued patents and pending worldwide patent applications held by or exclusively licensed to DOR, orBec(R) also benefits from orphan drug designations in the US and in Europe for the treatment of GI GVHD, which provide for seven and 10 years of post-approval market exclusivity, respectively.

About Acute GI GVHD

Acute GI GVHD is a debilitating and painful disease and constitutes an unmet medical need. It is a common disorder among immunocompromised cancer patients after receiving hematopoietic cell transplantation. 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 (host’s) body — most frequently the gastrointestinal tract, 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.

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(R) (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(R) 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(R) 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.

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(R), 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(R) 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(R) 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(R) may not gain market acceptance if it is eventually approved by the FDA; and others may develop technologies or products superior to orBec(R). 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.

Source: DOR BioPharma, Inc

Mylan Inc. (NASDAQ: MYL) announced that Matrix Laboratories, an Indian company in which Mylan owns a controlling stake, has received tentative approval from the U.S. Food and Drug Administration (FDA) under the President’s Emergency Plan for AIDS Relief (PEPFAR) for its Abbreviated New Drug Application (ANDA) for a fixed-dose combination (FDC) of Efavirenz, Emtricitabine, Tenofovir Disoproxil Fumarate Tablets, 600 mg/200 mg/300 mg. This is the first and only generic version of this product and will be eligible for purchase outside the U.S. in many developing countries.

Mylan President Heather Bresch said: “This critical approval only further strengthens Mylan and Matrix’s efforts to expand access to life-saving, affordable AIDS treatments for people living with HIV/AIDS in developing countries. Matrix’s generic fixed-dose combination will dramatically improve access to this more patient-friendly medication while reducing the cost of treatment. Our commitment to growing Matrix’s ARV (antiretroviral) franchise goes hand-in-hand with our desire to raise the standard of care in developing countries to the levels available in countries like the U.S.”

The Efavirenz, Emtricitabine and Tenofovir Disoproxil Fumarate FDC is the generic version of Gilead Sciences’ Atripla® Tablets, which are indicated for the treatment of HIV-1 infection in adults. It combines three anti-AIDS medicines into a single daily dose for either first- or second-line treatment and is one of the best-selling AIDS products in developed countries, with approximately $1.6 billion in U.S. sales alone for the twelve months ending June 30, according to IMS Health. Until now, a generic version of this product has not been available in any market. Cocktails combining numerous drugs into a once daily dose can dramatically reduce pill burden, an improvement which has been shown to enhance patient compliance with complex treatment regimens.

The FDA’s tentative approval under PEPFAR means that Matrix’s product meets all of the agency’s manufacturing quality, safety and efficacy standards. Although existing patents or exclusivity prevent its marketing in the U.S., the product will be eligible for purchase outside the U.S. in many developing countries.

Matrix’s wide range of ARV products includes active pharmaceutical ingredients (API) and first- and second-line finished doses. The company’s emphasis on producing affordable products has allowed it to drive down the average annual cost per patient of effective therapies. Approximately 30% of HIV/AIDS patients in developing countries depend on at least one Matrix ARV product.

Mylan Inc., which provides products to customers in more than 140 countries and territories, ranks among the leading diversified generic and specialty pharmaceutical companies in the world. The company maintains one of the industry’s broadest - and highest quality - product portfolios, supported by a robust product pipeline; owns a controlling interest in the world’s third largest active pharmaceutical ingredient manufacturer; and operates a specialty business focused on respiratory and allergy therapies.

Source: Mylan Inc

Watson Pharmaceuticals, Inc. (NYSE: WPI), a leading specialty pharmaceutical company, announced that it received a Complete Response Letter from the U.S.