North Carolina startup Scioderm announced today it will kick off a Phase 3 clinical trial for its lead product, Zorblisa.
Its lead compound helps speed up wound closure in patients with Epidermolysis Bullosa – a very painful skin condition among children that stems from extreme skin fragility, and can lead to terrible blistering and itching. Though considered a rare disease it affects some 30,000 Americans – and there aren’t any approved treatments to date. There are likely even more in Europe – and possibly 300,000 to 500,000 EB patients worldwide, CEO Robert Ryan said.
Though it’s seen Zorblisa show positive effect in early clinical trials, much remains a mystery about the Scioderm’s lead compound. Scioderm won’t disclose the molecular makeup of its proprietary compound, nor does it completely understand how it works in EB patients, CEO Robert Ryan said.
“We don’t know the exact mechanism because there aren’t translatable animal models,” Ryan said. “But what we’ve seen is that it has the ability to heal chronic wounds and diminish itching – a debilitating side effect of the disease.
Because of this, the startup earned Breakthrough Status from the Food and Drug Administration back in 2013, and if the Phase 3 goes as planned, the drug will enter the market next year. The company closed a $20 million Series B round this past December, and closed a $16 million Series A in 2013. That $36 million should be sufficient to take the drug to market, Ryan said.
The upcoming Zorblisa trial will take place in both Europe and the U.S., enrolling 130 patients. The treatment (or a placebo), will be applied in lotion-form all over the patient’s bodies each day for three months. Once complete, an open-label extension trial to evaluate longterm safety will be offered to the participating patients.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
Whether or not there are other applications for Zorblisa, Ryan won’t yet say – Scioderm’s “100 percent focused on Epidermolysis Bullosa.” But
