Amicus Therapeutics, a biopharmaceutical company focused on developing treatments for rare and orphan diseases, has agreed to acquire Scioderm, Inc. a privately held biopharmaceutical company, also focused on developing rare diseases, in a cash and stock transaction valued up to $947 million. 

Scioderm’s lead product candidate is Zorblisa a late-stage, proprietary topical cream and potential first-to-market therapy for treating epidermolysis bullosa (EB), a chronic, rare genetic connective tissue disorder that results in fragile skin that blisters and tears from minor friction or trauma. Zorblisa has established positive proof-of-concept in Phase II studies for treating lesions in patients suffering with EB and is currently being investigated in a Phase III study to support global regulatory approvals. Zorblisa received US Food and Drug Administration breakthrough therapy designation in 2013.

Under the deal, 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% of the proceeds of such sale.

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, the company estimates 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, according to information from Amicus.

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.

Source: Amicus Therapeutics