Fibrocell Science Highlights Recent Pipeline Accomplishments

Fibrocell Science, Inc. recently highlighted several recent accomplishments related to the continued advancement of the company’s pipeline.

“Recently, Fibrocell has made significant progress across our pipeline of personalized biologics, positioning the company to achieve value-creating milestones in 2016,” said David Pernock, Chairman, and Chief Executive Officer. “We are encouraged by this progress that brings us closer to delivering potentially transformative therapies for patients suffering from debilitating diseases of the skin and connective tissue.”

Fibrocell completed patient dosing in its Phase II clinical trial of azficel-T for the treatment of vocal fold scarring resulting in chronic or severe dysphonia. The ongoing study is a double-blind, randomized, placebo-controlled trial designed to test the safety and efficacy of azficel-T in subjects with chronic dysphonia caused by idiopathic vocal fold scarring or atrophy. As efficacy endpoints will be assessed four months after final treatment, Fibrocell continues to expect to report these primary endpoint results in the second quarter of 2016.

Fibrocell has also initiated the toxicology study previously requested by the US FDA related to the company’s Investigational New Drug (IND) application for FCX-007, its gene-therapy product candidate for the treatment of recessive dystrophic epidermolysis bullosa. In this study, FCX-007 was injected in non-grafted SCID (severe combined immunodeficiency) mice. Fibrocell expects to amend its IND to include data from this toxicology study in the first quarter of 2016 and, subject to FDA approval, initiate a Phase I/II clinical trial in the second quarter of 2016.

Furthermore, Fibrocell completed a proof-of-concept study for FCX-013 – the company’s gene-therapy product candidate for the treatment of linear scleroderma – to determine its potential to reduce dermal thickness in fibrotic tissue. FCX-013 was evaluated in a bleomycin-induced scleroderma model, utilizing SCID mice. Data from the study demonstrated that FCX-013 reduced the dermal thickness of fibrotic tissue to levels similar to non-bleomycin (saline) treated skin and further reduced the thickness of the sub-dermal muscle layer. Fibrocell will now advance FCX-013 into preclinical dose-ranging studies for product optimization and now expects to submit an IND application to the FDA in 2017. Both FCX-007 and FCX-013 are being developed in collaboration with Intrexon Corporation, a leader in synthetic biology.

Fibrocell is an autologous cell and gene therapy company translating personalized biologics into medical breakthroughs. Fibrocell’s most advanced product candidate, azficel-T, uses its proprietary autologous fibroblast technology and is in a Phase II clinical trial for the treatment of vocal fold scarring resulting in chronic or severe dysphonia. In collaboration with Intrexon Corporation, Fibrocell is also developing gene therapies for skin and joint diseases using genetically modified autologous fibroblasts. Fibrocell is in preclinical development of FCX-007, its orphan gene-therapy product candidate for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). Fibrocell is also in preclinical development of FCX-013, its gene-therapy product candidate for the treatment of linear scleroderma. In addition, Fibrocell and Intrexon will be commencing preclinical development of a gene-therapy for the treatment of chronic inflammatory and degenerative diseases of the joint, including arthritis and related conditions. For more information, visit www.fibrocell.com.