Scioderm novel topical therapy for Epidermolysis Bullosa achieves Phase 2 targets

DURHAM, N.C.---With results from its Phase 2B study to be announced soon, Scioderm President and CEO Robert Ryan previewed for DDNews why he hopes the company may soon achieve its mission to bringing relief to patients who suffer from Epidermolysis Bullosa (EB), a rare, genetic connective tissue disorder that typically manifests at birth or early childhood. The Phase 2B study was conducted to evaluate the safety and efficacy of SD-101, a novel topical therapy for the treatment of blisters and lesions in patients with EB.

All forms of EB—which has been designated an orphan disease in both the U.S. and Eruope—share the common symptom of fragile skin that blisters and tears from the slightest friction or trauma. Although it afflicts roughly the same number of individuals as cystic fibrosis—50,000 annually in the U.S. and 80,000 in Europe—it is, as Ryan notes, referred to as “The worst disease you’ve never heard of.”

SD-101 is Scioderm’s investigational therapy that is being evaluated for the treatment of skin blistering and lesions associated with EB, including facilitation of healing of skin lesions and reduction of the incidence and/or severity of new lesions. Based on promising early clinical data in EB patients, SD-101 has recently received Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with inherited 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 2 study in children evaluated SD-101 in target wound reduction and closure, and reduction in body surface area (BSA) coverage of blisters and lesions over a period of three months in children across all subtypes of EB. SD-101 application was well-tolerated and resulted in complete closure of 88 percent of target chronic lesions within one month, and a 57 percent reduction in BSA coverage of blisters and lesions after 3 months of daily treatment.

Ryan was active elsewhere in helping develop SD-101. He then acquired the product and moved it forward. “EB,” he says, “is Scioderm’s 100-percent focus.” SD-101 is a small molecule that Ryan is not free to identify. He does point out that the formulation allows the API to penetrate the skin barrier without getting into the systemic circulation.

The Phase 2b study was a prospective, randomized, placebo-controlled trial. SD-101 cream was applied over the entire body once daily for a period of three months. The primary endpoint was closure of selected chronic cutaneous wounds in patients with various subtypes of EB (Simplex, Recessive Dystrophic, or Junctional (non-Herlitz)).

Additional endpoints include reduction in body surface area (BSA) coverage of lesional areas on the skin and improvement in pain and itching.

The trial, which began January 6, 2014, enrolled a total of forty-eight subjects aged six months and older across 7 sites in the U.S. Patients who completed the study will be eligible to continue receiving SD-101 for daily use (study SD-004). Additional information about studies SD-003 and SD-004 can be found at www.clinicaltrials.gov.

“There is currently no treatment for EB,” Ryan states, “and we look forward to analyzing the results of this study in the third quarter of this year and improving the lives of those with EB.”

Other EB therapies under development include autologous fibroblasts modified to express the correct form of the collagen VII gene (being developed by Fibrocell), and fibroblasts combined with a bovine collagen matrix (Apligraf). These approaches are complicated by the need for genetic modification and the inability to cover large portions of the body and only address a single subtype of EB.

Brett Kopelan, executive director of the Dystrophic EB Research Association (debra) of America said, “I would like to congratulate the Scioderm team on … their Phase 2b study of SD-101 and attaining this milestone in such a timely fashion. Scioderm’s relentless pursuit of innovative therapies offers new hope for caregivers and those living with EB.”

Epidermolysis Bullosa (EB) is a rare genetic connective tissue disorder, with many genetic and symptomatic variations. All forms of EB share the common symptom of fragile skin that blisters and tears from the slightest friction or trauma. This particular manifestation has led to EB patients being known as “butterfly children” due to the analogous nature of the fragility of the skin to the wings of a butterfly. As of today there is no cure or effective treatment. The more severe forms of the disease lead to scarring, disfigurement, disability and early death, usually before the age of 30.

Scioderm is a privately held, clinical-stage pharmaceutical company focused on developing innovative therapies to address diseases with critical unmet medical needs, including orphan products. 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.