International randomized, double-blind, parallel group Phase 2/3 clinical trial to evaluate safety and efficacy of potentially disease-modifying therapy
PARSIPPANY, NJ – June 5, 2017 – Castle Creek Pharmaceuticals (CCP), a global company dedicated to delivering transformative therapies to patients with orphan dermatologic and other underserved conditions, today announced the first patient has enrolled in the company’s Phase 2/3 DELIVERS clinical trial evaluating the safety and efficacy of diacerein 1% ointment (CCP-020) for the treatment of epidermolysis bullosa simplex (EBS).
“Currently there are no treatment options that target the underlying cause of EBS, the most common form of a family of rare conditions leading to extremely fragile skin,” said Amy Paller, MD, director of the Northwestern University Skin Disease Research Center and lead investigator on the DELIVERS study. “The topical formulation of diacerein 1% that we are investigating is potentially disease-modifying, which would represent an important advance in treatment for patients.”
EBS is believed to be caused by mutations in genes that support development of the tough, fibrous proteins that glue layers of the skin together. People living with EBS produce abnormal proteins or no proteins at all, resulting in mild to severe blistering, skin erosion and peeling of the epidermis layers in response to minor injury or friction. Damaged skin associated with EBS can lead to widespread health problems including limited mobility, trauma to internal organs, and dehydration. The National Institutes of Health (NIH) estimates EBS affects one in 30,000 to 50,000 people.
“Generalized EBS can be quite debilitating from infancy into adulthood because many patients experience open, blistering wounds that need frequent bandage changes and present a risk of infection,” said Brett Kopelan, executive director of the nonprofit organization debra of America. “There is no treatment or cure. Pain management, wound care, and protective bandaging are the only treatment options. Supportive care, often by multiple providers including physicians, nurses, nutritionists, and psychological counselors, is all these patients have available right now. We are excited that Castle Creek is addressing EBS, which is the most common type of epidermolysis bullosa but is often the most underserved in terms of research into a treatment. We look forward to working hand-in-hand to educate the population about this trial.”
CCP-020 is engineered to block an important inflammatory signaling pathway associated with EBS, which could strengthen epidermal tissue and support healing. In a previously completed Phase 2 clinical trial that included 17 patients with EBS, patients treated with CCP-020 showed an average 60 percent reduction in blistering after four weeks.
“At Castle Creek we are focused on addressing complex health problems that often affect smaller, underserved and highly specialized patient communities,” said Greg Licholai, MD, president and chief scientific officer at Castle Creek Pharmaceuticals. “As we continue the development of CCP-020, we have an opportunity to fulfill our mission with a new treatment option for people living with EBS and the potential to significantly improve standard of care and patient health and quality of life.”
The randomized, double-blind, parallel group DELIVERS study is designed to enroll about 80 participants who have a clinical and laboratory confirmed diagnosis of EBS at research sites in the U.S., Europe, Israel and Australia. Researchers will compare the safety and efficacy of CCP-020 to a control ointment when applied once-daily for eight weeks. Participants will report outcomes including pruritus, pain, and mobility week-to-week using an electronic diary.
“Many EBS patients are children who face an uphill battle knowing they might be in for a lifetime of frequent bandage changes and painful sores and blistering,” said John Browning, MD, chief of dermatology at the Children’s Hospital of San Antonio and DELIVERS study investigator. “As a focal point for a network of pediatric services in our community, we are pleased to participate in this study and look forward to advancing research that could lead to a new treatment option and a more positive outlook for these children in the years ahead.”
About Castle Creek Pharmaceuticals
Castle Creek Pharmaceuticals (CCP) is a privately held biopharmaceutical company with a robust and diversified pipeline of late-stage products. The company business strategy is based on a demonstrated ability to identify and advance therapies backed by strong science with the potential to represent significant advances in the treatment of debilitating dermatologic and head and neck conditions with high unmet needs. CCP is working with TWi Biotechnology, Inc. based in Taipei, Taiwan for manufacture and development of CCP-020. For more information, please visit www.castlecreekpharma.com.