$75M Funding to Advance Gene Therapy for RDEB Entering Phase 3 Trial

Castle Creek Biosciences has received a $75 million investment to advance its gene therapy program, including FCX-007, a treatment candidate for people with recessive dystrophic epidermolysis bullosa (RDEB) that now in a pivotal clinical trial.

“We are proud to have the strategic support of world-class investors whose impact enables our efforts to transform the lives of patients and the future of medicine,” John Maslowski, Castle Creek’s CEO, said in a press release.

Part of the  money will go to supporting a Phase 3 clinical trial (NCT04213261), which is evaluating whether adding FCX-007 to standard of care improves wound healing, compared to standard of care alone, in up to 20 children, adolescents, and adults with RDEB.

Enrollment is ongoing at Dell Children’s Medical Group, in Austin, with two other U.S. sites yet to open. More information on contacts and locations here.

Castle Creek also plans to seek regulatory approval of FCX-007 in the U.S. in 2021.

RDEB is caused by mutations in the gene coding for type VII collagen (COL7), a protein that helps give connective tissues their structure and strength. The lack of COL7 in RDEB patients makes the skin to tear and blister more easily.

FCX-007 is a cell-based therapy in which an individual’s own dermal fibroblasts (a type of skin cell) are collected and genetically modified so they can produce functional COL7. Then, the cells are delivered directly to the same patient’s blisters and wounds, aiming to improve healing and hold skin layers together.

Interim results from an ongoing Phase 1/2 clinical trial (NCT02810951) showed that all treated wounds closed completely in four participants with RDEB within one year of treatment.

In addition to FCX-007, Castle Creek is developing CCP-020, an ointment treatment for EB simplex (EBS) that contains the anti-inflammatory compound diacerein. Results of a Phase 2 study showed a 60% reduction in skin blistering in EBS patients given the CCP-020 cream for four weeks, compared to a 15% reduction in a control group. None of the reported side effects were deemed associated with the treatment.

“We are steadfast in our commitment to the epidermolysis bullosa community and will continue to keep patients, caregivers and clinicians informed on the progress of our current programs, including FCX-007 and diacerein topical ointment, while we expand the scope of our gene therapy platform,” Maslowski said.

This investment was led by Paragon Biosciences, of which Castle Creek Biosciences is a portfolio company. Funds were received from Fidelity Management & Research Company, Valor Equity Partners, and Horizon Technology Finance Corporation.

Besides helping advance Castle Creek’s gene therapy programs (boosted by its recent acquisition of Fibrocell Science), the money will be used to expand the company’s good practice manufacturing capabilities.

“As investors, we are excited by the progress that the team has made and are committed to growing the Castle Creek Biosciences platform to address multiple rare genetic diseases,” said Jeffery Aronin, chairman and CEO at Paragon Biosciences.