Fibrocell announces FDA-allowance of FCX-013 application for severe, localized scleroderma

Fibrocell Science, Inc., a gene therapy company focused on transformational autologous, cell-based therapies for skin and connective tissue diseases, today announced that the United States Food and Drug Administration (FDA) has granted allowance of its Investigational New Drug (IND) application for FCX-013 – one of the company’s gene therapy candidates – to begin clinical trials for the treatment of moderate to severe, localized scleroderma. 


“We are pleased the FDA has granted allowance of our IND for FCX-013 to begin clinical trials for the treatment of moderate to severe, localized scleroderma, offering patients the potential for relief from this chronic, painful, and debilitating disorder,” said John Maslowski, president and chief executive officer of Fibrocell. “With no FDA-approved therapies available, we look forward to advancing FCX-013 into the clinic.”


Localized scleroderma is a chronic, autoimmune skin disorder characterized by excessive collagen deposition resulting in thickening of the dermis and underlying tissue. Moderate to severe forms of localized scleroderma can result in significant morbidity, including pain, restricted motion, disfigurement, and developmental issues. FCX-013 is an autologous fibroblast genetically modified to express matrix metalloproteinase 1 (MMP-1) – a protein responsible for breaking down collagen. FCX-013 incorporates Intrexon Corporation’s proprietary RheoSwitch Therapeutic System®, a biologic switch activated by an orally administered compound (veledimex) to control protein expression.


Fibrocell expects to initiate enrollment for an open label, single arm, phase ½, clinical trial in the third quarter of 2018. The primary objective of the trial is to evaluate the safety of FCX-013. Secondary analyses consist of several fibrosis assessments including histology, skin scores, ultrasound, and additional measurements of targeted sclerotic lesions and control sites at various time points up to 16 weeks post-administration of FCX-013. Ten patients with any subtype of localized scleroderma are targeted for enrollment (approximately five patients per phase). The phase one portion will enroll adult patients. Dosing for the first three adult patients will be staggered prior to dosing the rest of the trial’s population. 


Fibrocell intends to include pediatric patients in the phase two portion of the trial after submission and approval of safety and activity data from the adult phase one patients to the FDA and the Data Safety Monitoring Board for the trial.


“The impact of localized scleroderma on patients, particularly children, can be devastating –affecting growth and mobility of their affected limbs,” said Alfred Lane, MD, chief medical advisor of Fibrocell and professor of dermatology and pediatrics (emeritus) at the Stanford University School of Medicine. “There are no approved therapies for localized scleroderma. Current treatments are aimed at impacting inflammation, but few options exist to treat the excessive collagen deposition in the skin and soft tissue, which may produce pain and limitation in motion and growth. With FCX-013, the goal is to bring relief to patients by targeting the abnormal collagen metabolism to improve skin function.”


The FDA has granted Orphan Drug designation to FCX-013 for the treatment of localized scleroderma. In addition, FCX-013 has been granted Rare Pediatric Disease designation for the treatment of moderate to severe, localized scleroderma. FCX-013 is being developed in collaboration with Intrexon Corporation, a leader in synthetic biology.

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