R&D

A study in children with Epidermolysis Bullosa (EB) was recently completed at Children's Memorial Hospital, Northwestern University, Chicago, Illinois, under the direction of Dr. Amy Paller. Dr. Paller is the Walter J. Hamlin Professor and Chair, Department of Dermatology, and Professor, Department of Pediatrics, at the Feinberg School of Medicine at Northwestern University. Eight children (age range 6 months to 9 years old) were enrolled in the study, and included children with one of the three major subtypes of EB: Junctional EB, Recessive Dystrophic EB (RDEB), and Simplex EB (See Focus section for details on EB subtypes). Three of the children were considered to be severe (26-50% of body surface area coverage of lesions and erosions) at baseline, while five were considered to be very severe (greater than 50% of body surface area coverage of lesions and erosions) at baseline. Children were treated daily with 3% SD-101 for a 3 month period. Primary endpoints assessed included target lesion closure assessment and change in body surface area coverage in blisters and erosions. The study is complete and the report has been filed with the FDA. Data available to date suggests that SD-101 has the potential to be highly effective in this disease, and there are two additional higher concentrations of SD-101 that may also be evaluated in further patient trials.

The clinical development pathway for the EB indications was discussed with the FDA, and agreement was reached on the preclinical and healthy volunteer clinical study needed prior to conduct of the pivotal trial(s) in this indication. Now that the IND is open, a meeting will be scheduled with the FDA to finalize the protocol for the pivotal study planned in EB children.