CRO Novella Clinical, a Quintiles company, has been selected by Scioderm, is a privately held, clinical-stage pharmaceutical company, to manage a phase IIb trial for the treatment of Epidermolysis Bullosa (EB), a rare genetic connective tissue condition. The trial will evaluate Scioderm’s investigational topical product, SD-101, for the treatment of skin blistering and erosions associated with this disease, including facilitation of healing of skin lesions and reduction of the incidence and/or severity of new lesions.
Currently there is no cure or effective treatment for EB, which affects children beginning at birth and presents with the prominent manifestation of extremely fragile skin that blisters or tears with the slightest friction or trauma. The more severe forms of the disease lead to scarring, disfigurement, disability and early death, usually before the age of 30.
The study is scheduled to enroll the first patient in January 2014, and is expected to enroll 36 patients at seven sites in the U.S. The phase IIb trial will evaluate the safety and efficacy of SD-101, a topical cream which has previously demonstrated potential to provide improvement in treating the severe skin effects seen in patients across the primary inherited EB subtypes. Specifically, the trial will examine the safety and efficacy of SD-101 with a primary endpoint of target lesion closure at one month of treatment.
Dr. Robert Ryan, Scioderm president and CEO, said, “Novella Clinical was a natural fit for this trial due to its therapeutic experience within dermatology and wound care, in addition to the company’s commitment to emerging biotechs. Novella offers the flexibility and transparency critical to Scioderm at this point in our development.”
North Carolina-based Scioderm recently became the first biotech to secure Breakthrough Therapy designation by the FDA for SD-101 for the treatment of skin effects in patients with inherited EB. Breakthrough designation provides for fast track status as well as intensive FDA guidance for the SD-101 development program.