April 18, 2016 1:00pm
A “butterfly” company – because their depth is just as fragile … SELL into any strength
FCSC received allowance from the FDA to initiate a P1/2 clinical trial for FCX-007 in adults
FCX-007 is an orphan gene-therapy product candidate for the treatment of recessive dystrophic epidermolysis bullosa
The Bottom Line: Fibrocell expects to initiate the open label, P1/2clinical trial in Q2/16. Welcome to “riding the tail” on Intrexon (XON). A first positive in a while but, a real data read-out in which year or century? The primary objective of this trial will be to evaluate the safety of FCX-007 in RDEB subjects. Additionally, the trial will evaluate type VII collagen expression and the presence of anchoring fibrils resulting from FCX-007, as well evidence of wound healing.
FCSC closed Friday at $2.69 and is trading UP +$0.09 – watch for the wings to fall off this “butterfly”
Recessive dystrophic epidermolysis bullosa (RDEB) is the most severe form of dystrophic epidermolysis bullosa (DEB), a congenital, progressive, devastatingly painful and debilitating genetic disorder that often leads to death. RDEB is caused by a mutation of the COL7A1 gene, the gene which encodes for type VII collagen, a protein that forms anchoring fibrils. Anchoring fibrils hold together the layers of skin, and without them, skin layers separate causing severe blistering, open wounds and scarring in response to any kind of friction, including normal daily activities like rubbing or scratching. Children who inherit the condition are often called "butterfly children" because their skin is as fragile as a butterfly's wings. We estimate there are approximately 1,100 – 2,500 RDEB patients in the U.S. Currently, treatments for RDEB address only the sequelae, including daily bandaging, hydrogel dressings, antibiotics, feeding tubes and surgeries.