TWi Biotechnology Announces Collaboration with EB House Austria to Explore AC-203 for the Treatment of Dystrophic Epidermolysis Bullosa

TWi Biotechnology, Inc. (“TWiB”) today announced a collaboration with the EB House Austria, a globally recognized center of expertise in epidermolysis bullosa (EB), to evaluate the feasibility of AC-203 (diacerein 1% ointment) for the treatment of dystrophic epidermolysis bullosa (DEB), a rare and severe genetic skin disorder.

EB is a group of inherited disorders characterized by extreme skin fragility. DEB is a major subtype of EB, most commonly occurring in its recessive form (RDEB), and is caused by mutations in the COL7A1 gene, which result in a deficiency of type VII collagen. Patients suffer from recurrent blistering, chronic wounds, pain, increased risk of infection, and a potential progression to cutaneous squamous cell carcinoma. While a limited number of therapies have recently been approved, including gene therapy (e.g., Vyjuvek) and topical wound treatments (e.g., Filsuvez), access to these therapies remains limited, and significant unmet medical needs persist.

AC-203 is a topical formulation of diacerein designed to modulate inflammatory pathways implicated in EB. Preclinical and translational studies suggest that diacerein inhibits IL-1β–mediated inflammatory signaling and may reduce downstream MMP-9 expression, which is associated with chronic wound formation and tissue degradation. By targeting the IL-1β–MMP-9 axis, AC-203 has the potential to promote wound healing and improve clinical outcomes in patients with DEB.

Under this collaboration, the EB House Austria will administer AC-203 to selected DEB patients through a Named Patient Program (NPP). The EB House Austria will be responsible for patient selection, treatment, and clinical follow-up in accordance with local regulatory and ethical requirements, while TWiB will provide the investigational product.

Prof. Johann Bauer, medical director of the EB House Austria, stated: “We are excited to expand the treatment range of diacerein. This program will close important gaps in the management of DEB patients.”

Notably, the EB House Austria has previously treated two RDEB patients with topical diacerein and observed meaningful clinical improvements: particularly enhanced wound healing and an increase in the quality of life as reported by the patients’ caregivers. These findings, together with published mechanistic data, support further exploration of AC-203 in DEB (https://onlinelibrary.wiley.com/doi/10.1111/1346-8138.17621).

“This collaboration represents an important step in expanding the potential of AC-203 beyond EBS,” said Dr. Chen-En Tsai, President and Chief Medical Officer of TWiB. “We look forward to working closely with EB House Austria to generate valuable clinical insights that may help inform future development in DEB.”

The program is expected to be initiated in April 2026. As an NPP, patient enrollment and treatment will depend on clinical practice and availability. Observations from this program are expected to support the further evaluation of the development potential of AC-203 in DEB.