Amicus Therapeutics, Inc., a biotechnology company, has successfully completed its previously announced acquisition of 100 per cent of the capital stock of Scioderm, Inc., a privately-held biopharmaceutical company focused on developing innovative therapies for treating diseases with high unmet need.

"The successful closing of the Scioderm acquisition is another important step forward toward fulfilling our patient-centric vision to build one of the world's leading rare disease biotechnology companies," said John F. Crowley, chairman and chief executive officer of Amicus.

"We now have one of the industry's leading pipelines of advanced therapies for devastating rare and orphan diseases. Within the next 12 months, we have the potential to have a marketed product for Fabry disease in the EU, marketing applications ready to submit for our investigational product for Epidermolysis Bullosa (EB) in the US and EU, and a Pompe programme entering phase 3 development. Today we believe that Amicus is stronger than ever and has the potential to create significant near- and long-term value for patients as well as our shareholders."

The acquisition of Scioderm strengthens Amicus' pipeline significantly with the addition of a novel, late-stage, proprietary topical cream and potential first-to-market therapy for EB (SD-101). This investigational product was granted FDA breakthrough therapy designation in 2013 based on results from phase 2 studies for the treatment of lesions in patients suffering with EB. SD-101 is currently being investigated in a phase 3 study to support global regulatory submissions and was the first-ever treatment in EB clinical studies to show improvements in wound closure across all major EB subtypes.

Amicus estimates that EB represents a potential $1 billion+ global market opportunity, based on third party market research. The current standard of care is palliative treatments, which mainly consist of bandaging, treating the open wounds to prevent infection and trying to manage patients' pain. An estimated 30,000 to 40,000+ people are currently diagnosed with EB in major markets.

Leerink Partners LLC acted as financial advisor to Amicus. Skadden, Arps, Slate, Meagher & Flom LLP acted as legal counsel to Amicus. J.P. Morgan acted as financial advisor to Scioderm. Cooley LLP acted as legal advisor to Scioderm.

With the closing of the Scioderm acquisition and the forecasted spending on SD-101 development, Amicus expects to end 2015 with between $200 million and $225 million of cash on hand. Pro-forma cash post-closing is expected to fund the current operating plan (including SD-101) into 2017.

Epidermolysis Bullosa (EB) is a chronic, rare genetic connective tissue disorder with no approved treatment options. EB is debilitating, disfiguring, and potentially fatal. There are many genetic and symptomatic variations of EB that all share the prevalent manifestation of fragile skin that blisters and tears from minor friction or trauma. Patients with the more severe forms of EB have generalized blistering and lesions affecting a substantial percentage of their bodies that can lead to infection and scarring, and, in severe cases, death. Internal organs and bodily systems can also be severely affected by the secondary complications and illnesses. There is currently no FDA approved treatment for EB. Current standard of care consists of bandaging and bathing the open wounds to prevent infection and trying to manage patients' pain. EB affects all races, ethnicities and genders equally.

A phase 3 multi-center, randomized, double-blind, placebo-controlled study (SD-005) in the US and Europe is currently underway and expected to support registration globally. The study is currently enrolling individuals who are 1 month and older with a diagnosis of Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB who have at least 1 target wound present for 21 days or more. Half the patients receive SD-101 cream (also known as SD-101) and the other half receive placebo cream, applied topically once daily to the entire body for 90 days. The primary outcome measure is complete target wound closure within 2 months. Secondary outcome measures include 1) median time to complete target wound closure; 2) change in lesional skin at month 2; 3) change in itching at day 7; and 4) change in pain at day 7. Patients who complete the 90-day primary treatment period will be eligible to receive SD-101 in an open-label extension study (SD-006).