Scioderm

Scioderm, Inc. operated as a clinical-stage biopharmaceutical company with a focus on developing treatments for diseases with significant unmet needs, particularly rare or "orphan" diseases. The company was co-founded in April 2013 by Robert Coull and Dr. Robert Ryan, who also served as President and CEO. Dr. Ryan brought over three decades of experience in the pharmaceutical and biotech industries, with senior roles at companies like Roche, Pfizer, and Bristol-Myers Squibb. His extensive background in drug development, spanning preclinical research to late-stage clinical trials across various therapeutic areas, was instrumental to Scioderm's direction. The founders acquired the company's primary asset from another firm where its development had stalled due to a lack of funding.

The company's business model was centered on advancing its lead product candidate, a topical cream known as Zorblisa™ (SD-101), through the clinical trial and regulatory approval process to commercialization. Its target market was patients suffering from Epidermolysis Bullosa (EB), a group of rare and debilitating genetic connective tissue disorders that cause extremely fragile skin, leading to painful blisters and chronic wounds. Scioderm's lead product, SD-101, was a proprietary topical cream containing allantoin, designed for whole-body application to promote wound healing and reduce blistering associated with all major EB subtypes. In a Phase 2 trial, SD-101 demonstrated a significant acceleration in wound closure compared to a placebo. The promising results led to the FDA granting SD-101 a Breakthrough Therapy designation, a first for a biotech company at the time, as well as Orphan Drug status in both the U.S. and Europe.

Scioderm's progress attracted substantial investor interest, securing an initial $16 million in Series A financing in 2013 from Morgenthaler Ventures and Technology Partners, with subsequent funding led by Redmile Group. This financial backing enabled the company to advance SD-101 into a pivotal Phase 3 registration trial in March 2015. In September 2015, Scioderm was acquired by Amicus Therapeutics (Nasdaq: FOLD) in a deal valued at an initial $229 million with the potential for over $600 million in additional milestone payments. The acquisition aimed to leverage Amicus's infrastructure to bring SD-101 to a global market estimated to be worth over $1 billion. However, in 2017, the Phase 3 trial failed to meet its primary endpoint, showing no statistically significant difference from the placebo, and Amicus subsequently discontinued the program.

Keywords: Scioderm, Epidermolysis Bullosa, SD-101, Zorblisa, Robert Ryan, Amicus Therapeutics, rare disease, orphan disease, clinical trial, biopharmaceutical, dermatology, topical therapy, wound healing, connective tissue disorder, Breakthrough Therapy, Morgenthaler Ventures, Redmile Group, genetic disease, skin disorder, allantoin