Amicus Therapeutics, a biotechnology company at the forefront of therapies for rare and orphan diseases, and Scioderm, Inc., a privately-held biopharmaceutical company focused on developing innovative therapies for treating diseases with high unmet need, have signed a definitive agreement under which Amicus will acquire 100 per cent of the capital stock of Scioderm, Inc.
"This acquisition is a major step forward toward our strategic vision and is transformative for the Epidermolysis bullosa (EB), community as well as the shareholders of Amicus and Scioderm," said John F. Crowley, chairman and chief executive officer of Amicus and board member of Scioderm.
"EB is a disorder that is utterly devastating and painful as it causes extremely fragile skin that blisters and tears from minor friction or trauma. In many children it leads to severe complications and a very early death. Amicus is committed now to advancing the tremendous mission of Scioderm's co-founder and CEO Dr. Robert Ryan, who we are proud to welcome to our senior leadership team at Amicus. 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. When combined with migalastat for Fabry disease and ATB200 for Pompe disease, this acquisition solidly positions Amicus as a leading global rare disease company dedicated to bringing substantial value to patients and shareholders."
Scioderm's lead product candidate Zorblisa is a novel, late-stage, proprietary topical cream and potential first-to-market therapy for EB. Zorblisa has established positive proof-of-concept in phase 2 studies for the treatment of lesions in patients suffering with EB, and is currently being investigated in a phase 3 study to support global regulatory approvals. Zorblisa was one of the first products to receive FDA breakthrough therapy designation in 2013, and was the first-ever treatment in EB clinical studies to show significant benefit in wound closure across all major EB subtypes.
Amicus estimates that EB may represent a potential $1 billion+ global market opportunity based on third party market research. The current standard of care is palliative treatments which 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. An estimated 30,000 to 40,000+ people are currently diagnosed with EB in major markets.
"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 (DebRA).
"The EB community will be well-served by the experience and broad, global capabilities that Amicus adds to Scioderm."
"Both Amicus and Scioderm are wholly and passionately focused on patients with rare diseases, and share a common vision and similar values," said Robert Ryan, Ph.D., president and chief executive officer of Scioderm.
"John Crowley has been a dedicated board member providing valuable counsel to Scioderm over the past several years, during which time he has been deeply involved with the EB community. This combination of Amicus and Scioderm is a major win for EB patients. With the added resources and expertise that Amicus provides for the Zorblisa programme, 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."
Amicus will acquire Scioderm in a cash and stock transaction. At closing, Amicus will pay Scioderm shareholders $229 million, of which $125 million will be paid in cash and $104 million will be paid 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 to Scioderm shareholders in cash or stock upon achievement of certain sales milestones. If Zorblisa is approved, EB qualifies as a rare pediatric disease and a Priority Review Voucher will be requested. If the Priority Review voucher is obtained and subsequently sold, Amicus will pay Scioderm shareholders the lesser of $100 million or 50 per cent of the proceeds of such sale. The transaction is subject to customary conditions, including the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act. The boards of both companies have approved the transaction and the companies currently anticipate that the transaction will be completed in the third quarter of 2015.
Amicus intends to finance the acquisition through cash on hand and has a $50 million debt commitment from Redmile Group. Leerink Partners LLC is acting as financial advisor to Amicus. Skadden, Arps, Slate, Meagher & Flom LLP is acting as legal counsel to Amicus. J.P. Morgan is acting as financial advisor to Scioderm. Cooley LLP is acting as legal advisor to Scioderm.
Based on the closing of the Scioderm acquisition, the anticipated debt financing and the forecasted spending on Zorblisa development, Amicus expects to end 2015 with $200 million to $225 million of cash on hand. Pro-forma cash post-closing is expected to fund the current operating plan (including Zorblisa) 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 racial, ethnic 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 Zorblisa 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 Zorblisa in an open-label extension study (SD-006).