Castle Creek Pharma Secures $71.8M Financing to Advance Orphan-Designated CCP-020 for Epidermolysis Bullosa Simplex

On October 22, 2018, VCBeat (WeChat ID: VCbeat) learned that biopharmaceutical company Castle Creek Pharma (hereinafter referred to as “CCP”) had completed a new round of financing amounting to $71.8 million. This round was participated in by Fidelity Management and Research Company and Valor Equity Partners, coming two years after its previous $48 million venture capital raise. To date, CCP has secured approximately $120 million in total funding.

CCP plans to use the funds to continue collaborating with TWi Biotechnology, a rare-disease biopharmaceutical company based in Taipei, Taiwan, on the production and development of CCP-020 for the treatment of EBS (epidermolysis bullosa simplex).

Epidermolysis bullosa simplex (EBS) is the most common form of epidermolysis bullosa (EB), a rare genetic disorder characterized by extreme skin fragility, leading to mild-to-severe blistering, skin erosion, and epidermal detachment upon minor trauma. CCP-020 is a topical 1% diacerein ointment. Its active ingredient, diacerein, is a slow-acting, low-molecular-weight anthraquinone compound with potent anti-inflammatory properties. Diacerein is hydrolyzed in the epidermis and dermis into rhein, which inhibits the production and activity of interleukin-1β (IL-1β) and other pro-inflammatory cytokines both in vitro and in vivo. This mechanism blocks inflammation signaling pathways associated with EBS, thereby promoting the healing of epidermal tissue and skin blisters. Currently, however, no treatment regimen for any type of EB, including EBS, has been approved by the U.S. Food and Drug Administration (FDA).

On August 9, 2018, the U.S. Food and Drug Administration (FDA) designated CCP-020 for inclusion in the Fast Track development program for the treatment of epidermolysis bullosa simplex (EBS). This designation indicates that CCP-020 is eligible for priority review and allows for frequent interactions with the FDA. Previously, the FDA had granted CCP-020 orphan drug designation for the treatment of epidermolysis bullosa (EB) and qualified it for the Rare Pediatric Disease designation.

Currently, CCP-020 is undergoing the global Phase I DELIVERS study, which aims to evaluate its safety and efficacy. In a previously completed Phase II clinical trial involving 17 patients with epidermolysis bullosa simplex (EBS), 60% of patients treated with CCP-020 experienced a reduction of more than 40% in skin blistering symptoms after four weeks of treatment, compared to only 18% in the vehicle control group. In this study, six adverse events (AEs) were reported in the CCP-020 group, whereas 11 were reported in the vehicle control group. The main adverse events included exacerbation of blistering (one case), pruritus (one case), and skin infection (one case). However, researchers considered these adverse events unrelated to the treatment and not associated with the treatment sites.

Children with EB (stock image)

Castle Creek Pharma, founded in 2015 and headquartered in Parsippany, Illinois, is a portfolio company of Paragon Biosciences, a global healthcare company dedicated to accelerating biomedical research (which has been acquired by Emalex Biosciences). CCP is committed to developing innovative therapies for patients with rare, severe, or debilitating dermatological conditions.