Soligenix Announces FDA Protocol Clearance of Pivotal Phase 3 Clinical Trial of SGX203 in Pediatric Crohn's Disease

Soligenix, Inc.

(Soligenix or the Company), a late-stage biopharmaceutical company developing products that address unmet medical needs in the areas of inflammation, oncology and biodefense, announced today that agreement has been reached with the US Food and Drug Administration (FDA) on the design of a pivotal, Phase 3 clinical trial evaluating its product, SGX203 (oral beclomethasone 17,21-dipropionate or BDP), in the treatment of pediatric Crohn's disease. SGX203 has previously been granted both orphan drug and fast track designations from the FDA for the treatment of Crohn's Disease in the pediatric population. SGX203 is a proprietary formulation of BDP that uses a two-tablet immediate and delayed release system to orally deliver high mucosal concentrations of the topical steroid to both the upper and lower bowel walls. This makes SGX203 particularly well suited for Crohn's disease in younger patients that are reported to have gastrointestinal (GI) inflammation in both areas of the bowel as opposed to adults who have mainly lower (colon) involvement. Oral BDP has a limited systemic absorption into the blood stream, decreasing the potential for the development of adverse side-effects frequently seen with systemic steroid therapies, like prednisone. These side effects include bone demineralization and interference with normal growth, which are particularly concerning in children. The upcoming pivotal Phase 3 study will be a double-blind, randomized, controlled, multi-national trial and will seek to enroll approximately 150 subjects 6-17 years of age with endoscopically proven mild to moderate Crohn's disease. The trial will compare the rates of improvement of the signs and symptoms of Crohn's disease after 8 weeks of treatment among subjects randomized to one of three SGX203 dose groups (split 60:30:60 among the lowest, middle and highest dose of SGX203). Subjects will be followed for an additional 6 months after the completion of treatment. Entry criteria for the trial are the presence of Crohn's disease symptoms (abdominal pain and/or diarrhea) associated with laboratory evidence of active disease in the blood. The primary clinical efficacy endpoint of the trial will compare the percentage of subjects in each of the three dose groups having resolved their signs and symptoms after an 8-week course of treatment. An adaptive design will be employed in which an independent Data Monitoring Committee will review the efficacy data after approximately 90 subjects have completed treatment and determine if the trial size requires adjustment based on the actual event rate or halted for futility or overwhelming efficacy. The trial is anticipated to begin in the second half of 2015 with primary data available in the first half of 2017. "We are excited to move forward with this trial in an effort to improve the signs and symptoms of acute attacks of pediatric Crohn's disease while minimizing the deleterious side-effects often seen with systemic steroids," stated Richard Straube, MD, Chief Medical Officer of Soligenix. "Currently, most pediatric patients with mild to moderate active disease are treated with off-label prednisone. The side effects of prednisone, such as bone demineralization and interference in normal growth, are particularly concerning in children. We believe that SGX203 has the potential to deliver adequate doses of BDP to the GI tract with markedly reduced systemic toxicity. This, in turn, may offer physicians and patients an effective, and safer, therapy to control disease symptoms." "We are pleased to have FDA agreement on this Phase 3 protocol after having worked closely with the FDA on its design," stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. "We are excited to move forward with this pivotal trial in an effort to address the significant unmet medical need that currently exists in this orphan disease."