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that their wounds healed faster with the treatment. Based on that feedback, Ryan says Alwyn decided to develop a formulation for a Phase 1 study in eight patients.
Ryan learned of the allantoin cream while working in regulatory affairs for Wilmington, NC contract research organization PPD, which was working with Alwyn. After completing the Phase 1 study in 2010, Alwyn decided to pursue a different drug program, Ryan says. In 2012, Ryan reached a deal to acquire the allantoin cream and Alwyn’s patents. The cream became the lead drug candidate for the newly-formed Scioderm.
While Alwyn’s formulation showed efficacy, Ryan suspected that more of the active ingredient would yield better results so Scioderm’s topical cream, Zorblisa, has a higher concentration of allantoin than the Alwyn cream. Ryan says the higher concentrations have demonstrated better healing results. In 2012, Scioderm completed a Phase 2 study of eight patients evaluating the drug. It was an open label study, in which both patients and doctors were aware that the patients were being treated with the experimental drug.
EB patients can have wounds that persist for months, even years, Ryan says. But in Scioderm’s Phase 2 study, the cream resulted in complete closure of 88 percent of chronic lesions within one month. Body surface area coverage of lesions and erosions were reduced by 57 percent after three months of daily treatment.
A randomized and placebo-controlled Phase 2b trial completed earlier this year studied 48 patients, with similar results. “We had them healing in a month, in some cases even earlier than that,” Ryan says. “Wounds that would not heal are healing and are healing at a much faster rate.”
Based on an early evaluation of the results and the fact that there are no current treatments for EB, the FDA last year designated Scioderm’s topical cream a “breakthrough therapy.” That provides the company more active communication with regulators during clinical trials. It also means that the FDA could approve Scioderm’s cream without a Phase 3 trial, based on the results from the Phase 2 studies. If that happens, Scioderm would need to conduct a trial after the drug reaches the market in order to confirm the results. But European regulators will require a Phase 3 trial for approval so Ryan says Scioderm plans on late-stage studies, regardless of what the FDA decides. Scioderm has not yet met with the agency to discuss its latest clinical trial results. Scioderm’s next steps will depend on the outcome of those discussions.
But the FDA has also designated the Scioderm cream an “orphan drug,” reserved for drugs that treat rare diseases affecting fewer than 200,000 people. Orphan drug status waives regulatory filing fees and, if the FDA approves the drug, grants marketing exclusivity and tax breaks. Kopelan says those incentives make rare disease drug development attractive to companies that would otherwise be wary of developing and selling a product for a tiny patient population.
Scioderm was able to raise $16 million in a Series A round led last year by Morgenthaler Ventures, with participation from Technology Partners. The funding is enough to carry Scioderm through Phase 3 testing, so it won’t need the deeper pockets of a large pharmaceutical company to finance late-stage studies. But Ryan says he has already spoken with large pharmaceutical companies interested in the orphan drug. Ryan says Scioderm could license the allantoin cream to pharmaceutical partners, who would sell the drug in specific geographic regions around the world. Or Scioderm could become part of a larger company in an acquisition.
Ryan says Scioderm could also opt to commercialize the drug alone, financing the endeavor … Next Page »