Durham-based Scioderm will be acquired by Amicus Therapeutics of Cranbury, N.J., for $229 million, and the deal could grow to nearly $1 billion if Scioderm’s lead drug compound achieves certain clinical, regulatory and sales milestones.

Scioderm has begun phase three clinical trials of Zorblisa, its topical therapy for the treatment of blisters and lesions in patients with epidermolysis bullosa (EB), a rare genetic disease of the connective tissue.

“We believe we are well-positioned to rapidly complete the clinical development of Zorblisa and to make Zorblisa commercially available for all EB patients as quickly as possible,” said John F. Crowley, chairman and chief executive officer of Amicus and board member of Scioderm.

Amicus estimates that EB, which affects an estimated 30,000 to 40,000 people in the United States and 400,000 people worldwide, may be a potential $1 billion-plus global market opportunity.

“This combination of Amicus and Scioderm is a major win for EB patients,” said Robert Ryan, Ph.D., co-founder, president and chief executive officer of Scioderm, who will join the senior leadership team at Amicus. “With the added resources and expertise that Amicus provides for the Zorblisa program, we are more confident than ever in our potential for success and our ability to deliver significant benefits to patients and families living with the devastating effects of EB.”

Zorblisa is a potential first-to-market therapy for EB. In 2013 it received “breakthrough therapy” designation from the U.S. Food and Drug Administration, a status given to drugs that demonstrate early clinical evidence of significant improvement over available therapy. The designation is intended to accelerate the development and review of medicines for serious or life-threatening conditions.

In phase two clinical trials Zorblisa was the first treatment to show significant benefit in wound closure across all major subtypes of the disease.

Therapy derived from natural substance

Zorblisa is a topical cream containing a stabilized and concentrated form of allantoin, a natural substance found in some plants, bacteria and mammals. Allantoin, a common ingredient in toothpaste, mouthwash, shampoo, lipstick, acne creams and sun-care products, works by softening keratin, a family of fibrous structural proteins that make up the outer layer of human skin.

Scioderm announced in July it had agreed with the FDA to submit a rolling New Drug Application (NDA) for Zorblisa. That will accelerate the regulatory process by enabling review of portions of the NDA by the FDA prior to submission of the complete application.

EB is a debilitating, disfiguring and sometimes fatal disease characterized by fragile skin that blisters and tears from minor friction or trauma. Young EB patients are often known as “butterfly children” because their skin is delicate like the wings of a butterfly.

The current standard of care is palliative treatments that cost $10,000 to $15,000 per month and mainly consist of bandaging, treating the open wounds to prevent infection and trying to manage patients’ pain.

“Amicus is a champion of the rare disease community that, together with Scioderm, understands our sense of urgency to see a treatment approved for EB,” said Brett Kopelan, executive director of the Dystrophic Epidermolysis Bullosa Research Association of America. “The EB community will be well-served by the experience and broad, global capabilities that Amicus adds to Scioderm.”

The Transaction

Amicus will acquire Scioderm in a cash and stock transaction approved by the boards of both companies. At closing, expected in the third quarter of this year, Amicus will pay Scioderm shareholders $125 million in cash and $104 million through the issuance of 7 million newly issued Amicus shares.

Amicus has agreed to pay up to an additional $361 million to Scioderm shareholders in cash or stock upon achievement of certain clinical and regulatory milestones and $257 million upon achievement of certain sales milestones.

If Zorblisa is approved, EB qualifies as a rare pediatric disease and a Priority Review Voucher is obtained and subsequently sold, Amicus will also pay Scioderm shareholders the lesser of $100 million or 50 percent of the sale’s proceeds.

Scioderm, a privately held biopharmaceutical company founded in 2013, raised $36 million in Series A and B venture capital financing. It has nine employees.

Amicus, publicly traded on the Nasdaq stock market under the symbol FOLD, is developing therapies for rare and orphan diseases with a focus on lysosomal storage disorders. Its lead programs in development include the small molecule pharmacological chaperone migalastat for Fabry disease, as well as next-generation enzyme replacement therapies for Fabry disease, Pompe disease and MPS I.

(C) N.C. Biotechnology Center