New multi-center Phase 1 study expected to enroll up to 20 people living with rare genetic condition that leads to extremely fragile skin
PARSIPPANY, NJ – June 25, 2018 – Castle Creek Pharmaceuticals (CCP), a global company dedicated to delivering transformative therapies to patients with orphan dermatologic and other underserved conditions, today announced that the first patient has been enrolled at UNC-Chapel Hill Dermatology & Skin Cancer Center in a new Phase 1 study evaluating the pharmacokinetics (PK) and safety of diacerein 1% ointment (CCP-020) under maximum use conditions in patients with epidermolysis bullosa (EB).
In addition to the PK study, the company is currently evaluating CCP-020 in the ongoing DELIVERS study for the treatment of patients with epidermolysis bullosa simplex (EBS), a subtype of EB. CCP-020 is an investigational drug believed to block an inflammatory signaling pathway associated with EBS resulting in strengthening epidermal tissue and healing of skin blisters.
“We remain committed to supporting people who are impacted by EB through research that can lead to insights on the disease and innovative treatment approaches,” said Amir Tavakkol, PhD, executive vice president and chief development officer at Castle Creek Pharmaceuticals. “By collecting pharmacokinetic data and profiling drug absorption and safety and tolerability, this Phase 1 study will play an integral role in the continued development of CCP-020.”
The open-label multi-center, global Phase 1 study is expected to enroll 16-20 people living with EB age 6 months and older in Europe and age 4 years and older within the U.S. Patients who enroll in the study will be treated with CCP-020 for 10 days and will be asked to provide blood samples while receiving CCP-020. Patients who participate in the PK study may be eligible to enroll in a separate open-label extension study, where they will receive CCP-020 for two additional treatment cycles.
“There has been no treatment available and most of the EB community is excited to participate in anything that will give researchers knowledge of the disease as a whole,” said Dr. Aída Lugo-Somolinos, principal investigator at UNC-Chapel Hill. “People with localized EB lesions on the hands or feet, or generalized lesions on arms, legs, and torso are encouraged to enroll in this study as long as the lesions cover more than 2% of the total body surface area.” Dr Lugo-Somolinos added, “Not only evaluating efficacy is important but also we have a responsibility to find a product that is safe to use in open skin. To evaluate safety, a shorter but very important PK study is enrolling patients right now.”
For more information about the Phase 1 study, including a list of participating sites and their contact information, visit https://clinicaltrials.gov/ct2/show/NCT03472287.
EB is a rare genetic condition that leads to extremely fragile skin resulting in mild to severe blistering, skin erosion and peeling of the epidermis layers in response to minor injury. There are currently no approved treatment options for any form of EB.
Diacerein is a slow-acting, small molecular weight compound of the class anthraquinone with potent anti-inflammatory properties. Diacerein and its active metabolite rhein have been shown to inhibit the in vitro and in vivo production and activity of IL-1β and other pro-inflammatory cytokines. CCP-020 is a formulation of diacerein, 1% ointment developed for topical application. Diacerein in the formulation is hydrolyzed to rhein in the epidermis and dermis following administration and is believed to block an inflammatory signaling pathway associated with EBS resulting in strengthening epidermal tissue and healing of skin blisters.
In a previously completed Phase 2 clinical trial that included 17 patients with EBS, 60% of patients treated with a diacerein cream formulation experienced at least a 40% reduction in blistering after four weeks of treatment, compared to 18% on vehicle.[i],[ii] In this study, adverse events (AE) occurred in 6 patients on diacerein versus 11 patients on vehicle. The most notable AEs were increase in blistering (1), pruritus (1) and skin infection (1). However, none of the listed events were considered treatment related or involved the treatment area.
About Castle Creek Pharmaceuticals
Castle Creek Pharmaceuticals (CCP), part of the Paragon Biosciences portfolio of innovative biotech companies, is a privately held biopharmaceutical company developing innovative therapies for patients with rare, serious or debilitating dermatologic conditions. The company has a robust and diversified pipeline of late-stage products that have the potential to transform lives. For more information, please visit www.castlecreekpharma.com.
[i] Wally et. al., Diacerein for the treatment of epidermolysis bullosa – a phase II randomized, placebo controlled, double-blind multi-center clinical trial, 25th EADV congress, Vienna, Austria 2016.
[ii] Total (%) patients with >40% reduction of blister numbers after two 4-week intervention with diacerein or its vehicle for all 17 patients during a 2-year cross-over trial.