DURHAM, N.C.--(BUSINESS WIRE)--Scioderm, Inc., a clinical-stage biopharmaceutical company focused on developing innovative therapies for treating diseases with high need, including rare diseases, today announced it has initiated its Phase 3 registration trial to evaluate the safety and efficacy of Zorblisa™ (SD-101), a proprietary and novel topical therapy for the treatment of blisters and lesions in patients with Epidermolysis Bullosa (EB). EB is a rare genetic connective tissue disorder that typically manifests at birth or early childhood and for which there are no approved treatments. There are many genetic and symptomatic variations of EB, but all forms share the common symptom of fragile skin that blisters and tears from the slightest friction or trauma.
“Scioderm is dedicated to improving the lives of people with EB, a devastating disorder for which there are no effective treatment options available today,” said Robert Ryan, Ph.D., president and chief executive officer. “Initiating this study is an important milestone for the EB community and for our Zorblisa clinical development program.“
The Phase 3 trial, designated SD-005, is a randomized, double-blind, placebo-controlled trial being conducted in multiple sites in the U.S. and Europe, designed to evaluate the safety and efficacy of Zorblisa in 130 patients with various subtypes of EB (Simplex, Recessive Dystrophic and Junctional [non-Herlitz]). Participants will be randomized 1:1 to two treatment groups receiving either Zorblisa or placebo applied over their entire body once daily for three months. The primary efficacy endpoint will be evaluation of closure of a selected target chronic wound. In addition, improvement in itching, pain, full-body wound and lesion coverage will also be assessed. Investigators will also assess safety. An open-label extension trial, designated SD-006, which will evaluate long-term safety, will be offered to patients completing the SD-005.
“EB is ‘the worst disease you’ve never heard of’ because it attacks those that live daily with it physically, emotionally and financially,” said Brett Kopelan, executive director of debra of America, the only national not-for-profit that provides all inclusive care to those living with the disease by funding research and providing free services to the EB community. “Unfortunately, there are no approved treatments, so those with EB rely solely on pain management, daily wound care and preventative bandaging. We are excited to learn more about the potential of Zorblisa in helping to alleviate some of the most persistent and problematic symptoms of EB.”
About ZorblisaTM (SD-101)
Zorblisa is a proprietary topical therapy being developed for the treatment of patients with EB. Scioderm recently completed a successful Phase 2 study SD-003 study in EB patients, which was a double-blind, randomized, placebo-controlled, dose-response trial evaluating the efficacy and safety of different dosage strengths of Zorblisa. The data showed acceleration in wound healing and closure of chronic wounds. Scioderm received Breakthrough Therapy designation for Zorblisa from the FDA, as well as orphan drug designation from the FDA and EMA.
About Epidermolysis Bullosa (EB)
Epidermolysis bullosa (EB) is a rare genetic disease leading to fragile skin susceptible to blistering and tearing. EB is chronic, debilitating and potentially disfiguring and patients with EB have painful blisters affecting a substantial percentage of their bodies that can lead to infection and scarring. There are approximately 20,000-30,000 people living with EB in the United States, and 300,000-400,000 worldwide. There is currently no effective or FDA approved treatment for EB. Current standard of care consists solely of bandaging and bathing the open wounds to prevent infection and trying to manage patients’ pain.
About Scioderm, Inc.
Scioderm is a privately held, clinical-stage biopharmaceutical company focused on developing innovative therapies to address diseases with high need, including rare diseases. The company’s lead therapy, SD-101, is in Phase 3 development for treatment of the skin effects associated with Epidermolysis Bullosa (EB), a rare genetic connective tissue disorder. Scioderm was selected as a 2013 “Fierce Top 15” company by FierceBiotech, and considered one of the top 15 emerging companies in the biotech industry. The company is headquartered in Durham, North Carolina. Additional information about Scioderm can be found at www.sderm.com.
Forward Looking Statement
Except for the historical information contained herein, the matters discussed in this press release are forward-looking statements that involve risks and uncertainties, including: our dependence on third parties for the development, regulatory approval and successful commercialization of our products, the inherent risk of failure in developing product candidates based on new technologies, risks associated with the costs of clinical development efforts, as well as other risks. Actual results may differ materially from those projected. These forward-looking statements represent our judgment as of the date of the release. Scioderm disclaims any intent or obligation to update these forward-looking statements.