Pipeline

Biotherapeutics

Soligenix's lead product, orBec^®, represents a first-of-its-kind oral, locally acting therapy tailored to treat the GI manifestation of acute GVHD, thereby reducing the need for systemic immunosuppressive drugs to treat GI GVHD. Beclomethasone dipropionate (BDP), the active ingredient in orBec^® is a highly potent, topically active corticosteroid that has a local effect on inflamed tissue. orBec^® is uniquely formulated for oral administration as a single product consisting of two tablets; the first intended to release BDP in the proximal portions of the GI tract, and the second in the distal portions. Soligenix anticipates the market potential for orBec^® for the treatment of acute GI GVHD to be approximately 50% of the more than 10,000 allogeneic hematopoietic cell transplantations (HCT) that occur each year in the US, with similar numbers in Europe. Accordingly, orBec^® benefits from issued patents as well as orphan drug designations in the US and in Europe for the treatment of acute GI GVHD, which provides for 7 and 10 years of post-approval market exclusivity, respectively. orBec^® has also been granted fast track designation by the FDA for the treatment of acute gastrointestinal GVHD. In the US, orBec^® has been granted orphan drug designation for the prevention of acute GVHD and the treatment of gastrointestinal symptoms associated with chronic GVHD.

Previous randomized, double-blind, placebo-controlled clinical trials have demonstrated that orBec^® provides clinically meaningful outcomes compared with the standard of care in patients with acute GI GVHD. The prior Phase 3 study enrolled 129 patients across 16 leading HCT centers. Although orBec^® did not achieve statistical significance in the study's primary endpoint (time-to-treatment failure through Day 50 [p-value 0.1177]), it did achieve statistical significance in key secondary endpoints including:

• the proportion of subjects GVHD-free at Day 50 (p-value 0.05) and Day 80 (p-value 0.005);
• the time-to-treatment failure through Day 80 (p-value 0.0226);
• a 66% reduction in mortality 200 days post-transplantation (p-value 0.0139);
• and a 46% reduction in mortality at 1-year post-randomization (p-value 0.04).

Building upon the current data, Soligenix has initiated a confirmatory, Phase 3 clinical trial of orBec^® for the treatment of acute GI GVHD in 2H 2009. This confirmatory trial design was agreed to by the FDA through its Special Protocol Assessment (SPA) procedure and by the EMEA via the Protocol Assistance procedure. The primary endpoint of the study is the acute GI GVHD Treatment Failure rate at Study Day 80. In the previous Phase 3 study, this endpoint was evaluated and statistically significant with a p-value of 0.005.