Phase 1/2 Clinical Trial Results of FCX-007 Prove Efficacy

This morning, Fibrocell Science, Inc released interim results from its phase 1/2 clinical trial of FCX-007 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB).

M. Peter Marinkovich, MD, the trial’s Study Director at the Stanford University site and Associate Professor of Dermatology at the Stanford University School of Medicine presented the results at the 7th International Investigative Dermatology meeting on May 19, 2018.

“The phase 1 portion of the trial of FCX-007 continues to be encouraging and reinforces the potential for treating RDEB patients,” stated Alfred Lane, MD, Chief Medical Advisor of Fibrocell and Professor of Dermatology and Pediatrics (Emeritus) at the Stanford University School of Medicine in a recent statement. “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.”

FCX-007 is clinical-stage, gene therapy product candidate. It is a genetically-modified autologous fibroblast that encodes the gene for COL7 and delivers the treatment to the affected areas while evading systemic distribution.

DEB is a debilitating, genetic condition that causes the skin to be very fragile and blister easily. The slightest of injuries, such as bumping or rubbing, can cause the skin to break and blister. DEB is caused by a mutation in the gene called COL7A1. When mutated, COL7A1 doesn’t produce the required protein collagen VII to create the anchoring fibers that hold the skin together.

In the margins of and across targeted wounds, as well as in separate intact skin sites, 4 adult patients (n=7 wounds) aged 20 to 37 have been administered FCX-007. A single intradermal injection session at baseline was received by 3 patients while a second injection session was received by 1 patient at 25 weeks post-administration (as allowed by the clinical trial protocol) in the remaining unhealed wound areas.

FCX-007 was shown to be well tolerated and safe in patients up to 52 weeks post-administration with neither serious adverse events nor product related adverse events being reported. No type VII collagen (COL7) autoantibody response was noted as well.

Up to 52 weeks post-administration, various COL7 expression signals were detected throughout the data set by using either immunofluorescence (IF) or immunoelectron microscopy (IEM). By using IEM, anchoring fibril structures were also noted.

During a monitoring period prior to dosing, wounds were assessed and observed to be open for up to 8 months. The percentage of dosed wounds healing > 50% when compared to the baseline (collected at Day 0 before the administration of FCX-007) were measured. At 4 weeks post-administration, 100% (7/7) of dosed wounds healed > 50% compared to the baseline; 12 weeks, 86% (6/7) of dosed wounds healed > 50% compared to the baseline; at 25/32 weeks, 67% (2/3) of dosed wounds healed > 50% compared to the baseline; and at 52 weeks, 100% (1/1) of dosed wounds healed > 50% compared to the baseline.

When compared to baseline, a similar pattern was also shown for treated wounds healing > 75%. For controls for each patient, untreated wounds of similar size to the treated wounds were selected and monitored. The percentage of untreated control wounds healing > 50% when compared to the baseline were measured. At 4 weeks post-administration, 14% (1/7) of treated wounds healed > 75% compared to baseline; at 12 weeks, 17% (⅙) of treated wounds healed > 75% compared to baseline; at 25/32 weeks, 0% (0/2) of treated wounds healed > 75% compared to baseline; and at 52 weeks, 0% (0/1) of treated wounds healed > 75% compared to baseline.

Currently, 1 patient is enrolled in the phase 2 portion of the trial; 3 additional screening visits are scheduled for prior to the end of June 2018. An enrollment of 6 patients is anticipated during the third quarter of 2018.

“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, MD, 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.”

Previously, Orphan Drug, Rare Pediatric Disease and Fast Track Designations by the US Food and Drug Administration (FDA) FCX-007 were granted to FCX-007.

For more breaking news from the rare disease community, subscribe to Rare Disease Report’s e-newsletter.