A Phase IIB study to evaluate the efficacy and safety of SD-101, a novel topical therapy, for the treatment of non-healing wounds in patients with Epidermolysis Bullosa (EB), was initiated, according to Scioderm, Inc.

The Phase IIB study (SD-003) is a multi-site, prospective, randomized, placebo-controlled trial evaluating the efficacy and safety of SD-101 to close selected chronic cutaneous wounds and reduction in body surface area (BSA) coverage of lesional areas on the skin in patients with EB (Simplex, Recessive Dystrophic, or Junctional (non-Herlitz)). In addition, improvement on pain and itching will also be assessed. Approximately 48 subjects aged six months and older are planned to enroll in the trial, which is being conducted in seven sites across the US. The study will comprise application of SD-101 cream over the entire body daily for a period of three months.  Patients completing the study will be eligible to continue receiving SD-101 once the study is completed.  Additional information about Study SD-003 can be found at www.clinicaltrials.gov.

SD-101 for the treatment of EB has been granted an orphan drug designation in the US and in December 2013 received a positive opinion by the Committee for Orphan Medicinal Products (COMP) in the EU. In addition, Scioderm was the first biotech to receive "Breakthrough Therapy" designation for SD-101 from the FDA for the treatment of skin effects in patients with EB. SD-101 is a topical cream that has previously demonstrated potential to provide improvement in treating the severe skin effects seen in patients across all EB subtypes. An open-label Phase II study was conducted previously in children with either Simplex, Recessive Dystrophic (RDEB), or Junctional EB.  SD-101 was applied topically over the entire body daily for a period of three months. The primary outcome measurements were assessment of target wound reduction and closure, and reduction in body surface area (BSA) coverage of blisters and lesions. In the clinical trial, SD-101 application resulted in complete closure of 88% of target chronic lesions within one month, in addition to a 57% reduction in BSA coverage of blisters and lesions after 3 months of daily treatment.  SD-101 was well tolerated by the children.

"Our patients with EB have few therapeutic options available and none have demonstrated evidence of accelerated closure of chronic wounds, said the study's Principal Investigator, Amy Paller, MD, Walter J. Hamlin Professor and Chair of the Department of Dermatology, Northwestern University Feinberg School of Medicine.  "We look forward to testing this new potential intervention in the double-blind, randomized trial. If the lives of the EB patients are improved with topical use of SD-101 through faster wound healing, as well as decreased pain and itchiness, this cream would be welcomed by our affected families."

"We are very excited that Scioderm has developed this potential therapy to treat some of the more profound symptoms of EB," said Brett Kopelan, MA, Executive Director of the Dystrophic Epidermolysis Bullosa Research Association of America (debra of America).  "The impact on the quality of life of those with EB would be immeasurable if SD-101 was to be proven safe and effective."