The Promise of SD-101 Topical Allantoin Cream in Treating Patients With Epidermolysis Bullosa

A recent clinical trial found that SD-101, a new proprietary topical allantoin cream, can help treat epidermolysis bullosa (EB). Amy S. Paller, MD, chair, department of dermatology and professor of dermatology and pediatrics (dermatology) at Northwestern University, and director of the Northwestern University Skin Disease Research Center, presented her findings from a phase 2b study at the 25th Annual European Academy of Dermatology and Venereology Congress in Vienna. The study is a breakthrough in EB treatment and research as thus far, treatment options for this rare and painful rare connective tissue disorder are limited.

Paller provided an overview of the phase 2b study, known as SD-003, conducted by her and her colleagues, the main point of which, as she explained, was to determine the best way to conduct the phase 3 study that is currently underway: “Epidermolysis bullosa and the mutual history of lesions has not been studied well, so it’s important to do this type of pretest to determine how to run the most effective study.”

The SD-003 study was given breakthrough status by the FDA, because of its promise in advancing the treatment of EB with SD-101, Paller explained. The efficacy of allantoin, the key active ingredient in SD-101, “is based on many studies with allantoin that have gone back decades that have showed improvement in inflammatory responses, bactericidal effects, loosening protein bridges, and promoting collagen.”

The study involved 48 patients with EB ranging in age from 0.5 to 43.6 years. They were given randomized doses of SD-101 6%, SD-101 3%, or placebo. The study found that SD-101 6% was the most efficacious in wound healing and time-to-wound closure, compared to the 3% formulation and placebo. Further, its effects were seen in the 3 major types of EB: junctual, dystrophic, and simplex.

When asked to comment on any other drugs or treatments for EB, Paller explained, “[There is] lots of interest now in gene therapy approaches. There is a study [conducted by Stanford University] of introducing the normal collagen VII gene into keratinocyte skin cells and grafting sheets, some studies of injecting fibroblasts around the wounds, and studies ongoing of bone marrow/stem cell transplantation. These are not commercially available. Another exciting aspect about the SD-101 Phase 3 studies ongoing is that it is really the way to study the value of a medication—as randomized, double-blind, placebo-controlled. Tough for a rare disease, but moving forward.”

Paller’s tone was ultimately hopeful. With the efficacy of SD-101 validated, she is eager to finish the Phase 3 study, and deliver a new treatment to EB patients.

“Our patients are desperate for new treatments that are safe. We are all keeping our fingers crossed that a significant improvement is seen—and can be available," she said.