Fibrocell inks deal with Castle Creek worth up to $135 million to jointly develop FCX-007 gene therapy

Fibrocell Science on Monday announced that it signed a deal worth as much as $135 million to jointly develop and commercialise its investigational gene therapy FCX-007 with Castle Creek Pharmaceuticals for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). John Maslowski, chief executive at Fibrocell, commented "with [Castle Creek] providing funding for the development of FCX-007, Fibrocell can allocate additional resources to advance clinical development of FCX-013 for the treatment of moderate-to-severe localised scleroderma." 

Under the agreed terms, Castle Creek gains an exclusive licence to commercialise FCX-007 in the US. Meanwhile, Fibrocell will receive an upfront payment of $7.5 million, as well as payments of $2.5 million for the first patient enrolled in a late-stage study and $30 million upon regulatory approval and commercial readiness. Fibrocell is also eligible for up to $75 million in various milestones, plus a 30-percent share of gross profits from FCX-007 sales. 

Castle Creek will hold responsibility for all development and manufacturing expenses up to $20 million prior to regulatory submission of FCX-007 in the US. If development spending exceeds $20 million, Castle Creek will be responsible for 70 percent of the excess costs, with Fibrocell covering the remaining. Castle Creek will also control all commercialisation activities for the gene therapy. 

Meanwhile, Fibrocell will be in charge of clinical development, regulatory interactions and manufacturing of the product under a future supply agreement with Castle Creek. The company, which has obtained a rare pediatric disease designation for FCX-007 by the FDA in RDEB, said it will also retain sole ownership of a priority review voucher, which may be granted upon market approval of the therapy.

According to Fibrocell, FCX-007 is a genetically modified autologous fibroblast encoding the gene for COL7, and "offers the potential to address the underlying cause of the disease by providing high levels of COL7 directly to the affected areas while avoiding systemic distribution." Following recent positive feedback from the FDA, Fibrocell said it plans to initiate the Phase III DEFI-RDEB study, with a targeted enrolment of 15 to 20 patients, in the second quarter. 

FCX-007 has also been granted fast track designation from the FDA for the treatment of RDEB. Fibrocell is already partnering with Intrexon on the development of genetically-modified fibroblasts, including FCX-007, to treat chronic inflammatory and degenerative diseases of the joint. As part of that deal, Intrexon will be due 50 percent of all upfront, milestone and profit share agreements received by Fibrocell from Castle Creek. Meanwhile, FCX-013 incorporates Intrexon's proprietary RheoSwitch Therapeutic System to control protein expression at the site of the localised scleroderma lesions, Fibrocell said.

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