EB Groups Give Fibrocell $900,000 to Advance Gene Therapy in Clinical Trials

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by Alice Melao |

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RDEB funding support

The EB Research Partnership (EBRP) and Epidermolysis Bullosa Medical Research Foundation (EBMRF) granted $900,000 to Fibrocell, a gene therapy company, to support the clinical development of FCX-007 to possibly treat recessive dystrophic epidermolysis bullosa (RDEB).

“We are grateful for this investment from EBRP and EBMRF that will be used to support the continued clinical development of FCX-007,” John Maslowski, president and CEO of Fibrocell, said in a press release.

“We believe this funding further recognizes the significant potential of FCX-007 to make a difference for RDEB patients by treating the underlying cause of the chronic, debilitating and painful wounds and blisters of the disease.”

FCX-007, a gene therapy, is being developed by Fibrocell in collaboration with Precigen (a subsidiary of Intrexon) to correct the cause of RDEB — a defect in the Col7A1 gene that leads to the production of faulty type VII collagen protein.

Collagen is the main protein that binds skin layers and ensures proper skin structure and resistance. The lack of, or presence of defective, type VII collagen protein makes the skin more fragile and prone to frequent blistering. (Recessive dystophic EB is the most severe form of this disease, with widespread blistering.)

FCX-007 treats the patients’ own skin cells, called fibroblasts. These cells are collected and modified in the lab to produce the correct form of COL7 protein. They are then injected locally back to patient’s wounds to provide high levels of COL7 directly at the affected areas.

Fibrocell is exploring the therapeutic potential of FCX-007 in a Phase 1/2 clinical trial (NCT02810951) that is recruiting patients with RDEB at two U.S. sites.

Preliminary results of the trial demonstrated that FCX-007 was safe and well-tolerated by four adult patients (ages 20-37) with RDEB up to 52 weeks after administration. The analysis, conducted at four weeks and 12 weeks after the FCX-007 injection, showed that 100% (seven out of seven) and 86% (six out of seven) of the wounds, respectively, healed by more than half, relative to the beginning of the trial.

The ongoing Phase 2 portion of the trial is assessing FCX-007 potential in six RDEB patients ages 7 and older.

The decision to support the ongoing clinical testing of FCX-007 was made by EBRP’s scientific advisory board, which gives grants to products and therapies that are believed to hold promise for the treatment or cure of epidermolysis bullosa.

“The EB Medical Research Foundation is pleased to support Fibrocell’s clinical development of FCX-007, a potentially life-changing, gene therapy that addresses the underlying cause of RDEB,” said Paul Joseph, chief financial officer of EBMRF. “Our organization is dedicated to finding a cure for EB and, we believe, Fibrocell’s innovative approach aligns with our goal and distinguishes FCX-007 as a promising therapeutic solution for patients suffering from this devastating disease.”

The U.S. Food and Drug Administration (FDA) granted FCX-007 with orphan drug, rare pediatric disease and fast track designations last year, intended to expedite the development and regulatory review of this investigational therapy.

Fibrocell recently completed a regulatory meeting with the FDA to discuss the design of a Phase 3 clinical trial for FCX-007. The company has plans to submit the design protocol by the end of the year, and launch the trial in the first half of 2019.

“EB Research Partnership is proud to support the trailblazers of industry, research and academia who are tirelessly working to develop potentially transformative therapies for EB patients,” added Michael Hund, executive director of EBRP. “We are delighted to have Fibrocell as a partner who shares our team’s dedication and vision to one day deliver a cure for EB.”