First Patient Treated in Trial Testing FCX-013 Gene Therapy for Localized Scleroderma

A Phase 1/2 clinical trial of FCX-013, Castle Creek Biosciences‘ experimental gene therapy for moderate to severe localized scleroderma, has dosed the first participant.

The study (NCT03740724), which is expected to enroll up to 10 adults with localized scleroderma, or morphea, is still recruiting at its testing center in Philadelphia. To be eligible, patients must have failed to respond to standard therapy and have shown clinically inactive disease over the three months prior to the study. More information on the study site and contacts may be found here.

“Dosing the first patient is an important milestone in the clinical development program for FCX-013, which we believe has the potential to be the first therapy to treat excessive collagen deposition at the site of localized scleroderma lesions in the skin and soft tissue,” John Maslowski, CEO of Castle Creek, said in a press release.

“Our hope is to relieve the … impact of localized scleroderma in patients who currently have limited treatment options,” Maslowski added.

Localized scleroderma results from the excessive production and accumulation of collagen, which causes hard and thick patches of skin. Its symptoms are typically treated with steroids to address the overactive immune system and UV light therapy to reduce inflammation and soften the skin. Physical therapy, under the supervision of a physiotherapist, is used to improve muscle and joint function, and manage pain.

FCX-013 uses genetically modified fibroblasts — connective tissue cells with a key role in wound healing — collected from the patient to deliver the enzyme matrix metalloproteinase 1 (MMP-1), which breaks down collagen. The investigational gene therapy is administered by intradermal (under-the-skin) injection at the site of the localized scleroderma lesions and is taken in conjunction with the oral medication Veledimex, which promotes the production of MMP-1.

The Phase 1/2 trial will assess the safety and efficacy of FCX-013. Researchers will assess the side effects of the potential gene therapy and its ability to reduce fibrosis (hardening or scarring of tissue) throughout the 26-week study period.

“With our proprietary fibroblast technology, we have an opportunity to develop and evaluate new personalized therapies that are designed for durability and formulated to be compatible with each patient’s unique biology,” said Mary Spellman, MD, Castle Creek’s chief medical officer.

FCX-013 was granted fast track, rare pediatric disease, and orphan drug designations by the U.S. Food and Drug Administration, all of which convey financial and regulatory incentives for the clinical development of the treatment for localized scleroderma. The therapy was originally developed by Fibrocell Science, which was acquired by Castle Creek in 2019.