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.