Scioderm Inc. has released data from its recently completed phase 2b clinical trial - and plans for a phase 3 trial - on its investigational therapy Zorblisa (SD-101), a topical treatment for blistering and lesions associated with epidermolysis bullosa (EB). Read to learn more
Scioderm Inc. has released data from its recently completed phase 2b clinical trial - and plans for a phase 3 trial - on its investigational therapy Zorblisa (SD-101), a topical treatment for blistering and lesions associated with epidermolysis bullosa (EB).
The prospective, randomized phase 2b trial began in January 2014 and enrolled 48 subjects ages six months and older. Over the course of the trial, patients were given two dosage strengths of Zorblisa (3 percent and 6 percent), applied over the entire body, once daily for three months. The primary endpoint was closure of a selected chronic cutaneous wound in patients with the most common EB subtypes (simplex, recessive dystrophic and junctional). Secondary endpoints assessed included pain, itching and change in body surface area (BSA) coverage of lesional areas.
According to Scioderm president and C.E.O. Robert Ryan, Ph.D., the 6 percent dosage proved most effective and will be tested further in the upcoming trial.
“The higher dosage was selected for the phase 3 program based on the increased proportion and increased rate of wound closures observed in that treatment group relative to the placebo-treated patients,” he tells Dermatology Times. “In addition, the safety profiles of placebo-treated patients were indistinguishable from the Zorblisa-treated patients.”
Dr. Ryan said the phase 3 program will be similar in design to the two trials in the phase 2b program.
“These studies will enroll patients from the U.S. and Europe, with the most notable change being a substantial increase in the size of the patient population to be studied - about 130 - and, as a consequence, the power of the study.”
In phase 3, two groups will apply either Zorblisa or placebo over their entire body, once a day for three months. Primary efficacy endpoint will be evaluation of closure of a selected target chronic wound. Investigators also will assess the two groups’ local and systemic safety.
Dr. Ryan says Zorblisa’s impact on the market could be significant.
“As many as 30,000 Americans suffer from the disease, a similar prevalence to cystic fibrosis, yet DEBRA the Dystrophic Epidermolysis Bullosa Research Association of America - often refers to EB as ‘the worst disease you’ve never heard of,’” he says. “EB is debilitating and potentially disfiguring. There are currently no FDA-approved treatments for EB, nor are there any effective treatments resulting from products approved for other indications.”
The FDA has granted its breakthrough-therapy and orphan-drug designations to Zorblisa. The drug has also been granted orphan-drug status by the European Medicines Agency.