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FIBROCELL SCIENCE, INC. filed this Form 10-Q on 08/09/2018
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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 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

Based on safety, pharmacology and wound healing data from the Phase 1 portion of the trial, we have incorporated learnings on dose and administration in our clinical trial protocol, including an increase in the overall cells administered and a reduction of the interval between injections.
We plan to enroll six patients ages seven and older in the Phase 2 portion of the clinical trial. With the allowance from the FDA, we have now included enrollment of pediatric patients. One adult RDEB patient has been enrolled as the first patient in Phase 2 and dosing of this patient occurred in June 2018. Three pediatric patients have recently been enrolled in Phase 2 for a total of four patients. We expect to complete enrollment of Phase 2 patients by the end of the third quarter of 2018. We plan to report another interim data readout and trial update in the first quarter of 2019.

We plan to use data from the Phase 1/2 clinical trial to also support a petition for Regenerative Medicine Advanced Therapy or Breakthrough Therapy Designation for FCX-007.

We have designated our existing, current good manufacturing practices (cGMP) cell therapy manufacturing facility in Exton, PA as the production site for FCX-007 in our IND application. The FCX-007 drug product dosed in the fourth quarter of 2017 was produced and distributed from our Exton, PA facility. The facility will be used for the remaining clinical and future commercial manufacture of FCX-007, as we have sufficient cGMP vector supply on hand and capacity to serve the U.S. market for RDEB. The approximately 13,000 square foot facility previously supported commercial autologous fibroblast manufacturing, with multiple FDA inspections conducted at the site. The facility includes cleanroom cell therapy manufacturing, quality control testing, cryogenic storage, shipping/receiving and warehousing space.

FCX-013 for Moderate to Severe Localized Scleroderma

Localized scleroderma is a chronic autoimmune skin disorder that manifests as excess production of extracellular matrix, specifically collagen, resulting in thickening of the skin and connective tissue. Localized scleroderma encompasses several subtypes which are classified based on the depth and pattern of the lesion(s). The moderate to severe forms of the disorder include any subtype that affects function or produces symptoms of discomfort, tightness and pain. Current treatments for localized scleroderma include systemic or topical corticosteroids which target inflammation, UVA light therapy and physical therapy. There are few treatment options that address excessive collagen accumulation in the skin and connective tissue. We estimate that there are approximately 90,000 patients in the U.S. considered to have moderate to severe localized scleroderma.

Our second gene therapy product candidate, FCX-013, is in development for the treatment of moderate to severe localized scleroderma. FCX-013 is an autologous fibroblast genetically-modified using lentivirus and encoded for matrix metalloproteinase 1 (MMP-1), the protein responsible for breaking down collagen. FCX-013 incorporates Precigen’s