Fibrocell Science, Inc a gene therapy company focused on transformational autologous cell-based therapies for skin and connective tissue diseases, today reported interim results in its Phase 1/2 clinical trial of FCX-007 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB).
Three adult non-collagenous (NC)1+ patients have been dosed with a single intradermal injection session of FCX-007 in the margins of and across targeted wounds, as well as in separate intact skin sites. Five wounds were treated on the three subjects, ranging in size from 4.4cm2 to 13.1cm2.
Data from these patients show FCX-007 was well-tolerated through 12 weeks post-administration. There were no serious adverse events and no product related adverse events reported.
Wounds were evaluated during a monitoring period prior to dosing and they were observed to be open for up to eight months. Compared to the baseline measurement collected at Day 0 before the single intradermal injection session of FCX-007, at four weeks post-administration 100% (5/5) of wounds were > 75% healed. At 12 weeks post-administration, 80% (4/5) of wounds were > 70% healed.
Various pharmacology signals for vector DNA, type VII collagen (COL7) mRNA, or COL7 protein expression were detected throughout the data set in each patient for one or more assays up to 12 weeks post-administration (qPCR, electron microscopy or immunofluorescence). Anchoring fibrils have not been detected to date, whereas expressed COL7 mRNA and COL7 protein have been confirmed in multiple patient samples including one that detected linear expression of COL7 at the basement membrane zone.
“I am pleased with the interim data collected for the first three patients in the trial,” said Alfred Lane, MD, Chief Medical Advisor of Fibrocell and Professor of Dermatology and Pediatrics (Emeritus) at the Stanford University School of Medicine. “Safety is paramount in any new gene therapy candidate, and the detection of linear COL7 expression at the basement membrane zone is encouraging as it indicates the potential of FCX-007 to produce COL7 in the proper location of the skin structure. We believe increases in dosing and expression will enhance the consistency of effect as we continue clinical analysis and expand the treated patient population.”
The Data Safety Monitoring Board for the trial reviewed the interim data and concluded that safety and potential benefit were established, and allowed continuation of enrollment and dosing. With data from the first three patients meeting the primary trial objective of safety, the Company plans to increase expression and dosing FCX-007.
“FCX-007 to date has shown encouraging safety and positive early trends in pharmacology and wound healing with only a single injection session of cells,” stated John Maslowski, President and Chief Executive Officer of Fibrocell. “These interim results are a positive step towards our ultimate goal of bringing relief to patients and families suffering from this debilitating disease. In the coming months, we look forward to performing additional dosing and enhancing COL7 expression, and working with the FDA to further advance the program.”
