Fibrocell Science, Inc., a gene therapy company focused on transformational autologous cell-based therapies for skin and connective tissue diseases, reported progress in interim results from its Phase 1/2 clinical trial of FCX-007 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB).
Details were anounced at the 7th International Investigative Dermatology meeting held last weekend in Orlando.
Four adult patients (n=7 wounds) aged 20 to 37 have been dosed with FCX-007 in the margins of and across targeted wounds, as well as in separate intact skin sites. Three patients received a single intradermal injection session at baseline. One patient received a second injection session in the remaining unhealed areas of wounds at 25 weeks post-administration, as allowed by the clinical trial protocol.
Safety data from these patients show FCX-007 was well-tolerated up to 52 weeks post-administration. There were no serious adverse events and no product related adverse events reported. No type VII collagen (COL7) autoantibody response was noted.
Various COL7 expression signals were detected throughout the data set using either immunofluorescence (IF) or immunoelectron microscopy (IEM) up to 52 weeks post-administration. Anchoring fibril structures have also been observed using IEM.
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 administration of FCX-007, the percentage of dosed wounds healing > 50% when compared to baseline were observed as follows:
– 100% (7/7) at 4 weeks post-administration
– 86% (6/7) at 12 weeks
– 67% (2/3) at 25/32 weeks
– 100% (1/1) at 52 weeks
A similar trend was also observed for treated wounds healing > 75% when compared to baseline. Untreated wounds of similar size to the treated wounds were selected and monitored as controls on each patient. The percentage of untreated control wounds healing > 50% when compared to baseline were observed as follows:
– 14% (1/7) at 4 weeks post-administration
– 17% (1/6) at 12 weeks
– 0% (0/2) at 25/32 weeks
– 0% (0/1) at 52 weeks
“The Phase 1 portion of the trial of FCX-007 continues to be encouraging and reinforces the potential for treating RDEB patients,” said Alfred Lane, M.D., chief medical advisor of Fibrocell and professor of dermatology and pediatrics (Emeritus) at the Stanford University School of Medicine. “As we move into Phase 2 of the trial, I am looking forward to incorporating these learnings into the trial and determining the impact on patient outcomes.”
There is one patient enrolled in the Phase 2 portion of the trial, with three additional screening visits scheduled prior to the end of June 2018. Enrollment of six patients is expected to be completed in the third quarter of 2018.
Fibrocell is developing FCX-007 in collaboration with Precigen, Inc., a wholly owned subsidiary of Intrexon Corporation, a synthetic biology company.
(Source: Fibrocell Science, Inc.)
Filed Under: Drug Discovery