Orphan Drug From Scioderm Aims to Treat Rare Skin Disease

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with an initial public stock offering. Because EB is a rare disease addressing a small patient group, Scioderm would not need a large sales force, so it’s feasible for the small company to go to market alone. Based on his discussions with insurance companies, Ryan believes payers will reimburse for the treatment because EB patients currently have no approved treatment options.

Scioderm, however, isn’t the only company working on EB treatments. There’s growing interest in the pharmaceutical industry, and among academic researchers, because of the orphan drug status for EB treatments and because of the major unmet medical need, says Kopelan.

In 2007, the University of Minnesota used bone marrow transplants to implant cells that produce the collagen that EB patients lack. In that study, funded in part by the National Institutes of Health, researchers observed more collagen in the patients’ skin. They also did not detect antibodies to the donor cells, according to study results published in the New England Journal of Medicine.

In a different approach, Fibrocell Science (NASDAQ: FCSC) is working with patients’ own cells. The idea is to genetically modify patients’ collagen-producing cells, inserting the gene that produces collagen with the goal of producing healthy skin. Fibrocell has partnered with synthetic biology company Intrexon (NYSE: XON) to develop the treatment, which is being prepared for clinical trials.

Another tack, taken by Shire (NASDAQ: SHPG), is trying to grow skin over the wounds of EB patients using a diabetic foot ulcer treatment called Dermagraft. Shire acquired Dermagraft in 2011 from San Diego firm Advanced Biohealing for $750 million. Placed over a wound, Dermagraft is a mesh material that contains skin cells that produce connective tissue. As the body absorbs the mesh, the cells in Dermagraft help new skin to form. Shire took Dermagraft as far as Phase 3 clinical trials in EB but terminated the study last year, then sold Dermagraft to Organogenesis as part of a companywide reorganization that halted Shire’s regenerative medicine ambitions. Organogenesis markets Dermagraft as a diabetic foot ulcer treatment, but has said nothing about developing it for EB. Meanwhile, Shire’s EB efforts are now focused on a protein replacement therapy. Shire acquired the pre-clinical technology, which involves a genetically-engineered form of collagen, from Lotus Tissue Repair.

The business case for developing all of these EB treatments, says Kopelan, is further bolstered by opportunities to apply successful treatments to other skin disorders. Ryan, for instance, acknowledges potential broader applications of Scioderm’s allantoin cream, but says that right now, the company is focused on EB.

In addition, there will probably be markets for several EB treatments, Kopelan believes. He envisions that EB will be treated with a combination of products, perhaps a gene or cell therapy and skin grafts, along with topical agents to address persistent wounds. New treatments, including Scioderm’s allantoin cream, could make EB a chronic condition managed by modern medicine, he says.

“You’re not curing the disease, but you’re addressing some of the most prominent symptoms out there and that’s incredibly important,” he says.

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