TWi Biotechnology Announces Another Major Milestone for AC-203 FDA Provides Development Direction for DEB, Opening Path for Same Drug to Serve More "Bullous" Patients

TAIPEI, Oct. 2, 2026 — TWi Biotechnology Inc. (“TWi Biotechnology” or the “Company”; 6610.TWO) today announced that it recently received Type C meeting minutes from the U.S. Food and Drug Administration (FDA) regarding its investigational new drug AC-203 for the treatment of "Dystrophic Epidermolysis Bullosa (DEB)." The FDA has provided positive feedback on AC-203's development for DEB and offered extensive recommendations for future clinical trial design, primary endpoints, and assessment methods, giving clear direction for the next stage of AC-203's development. In other words, the scope of AC-203's development may expand beyond its current indication of "Epidermolysis Bullosa Simplex (EBS)" to include DEB. Furthermore, the FDA indicated that most of the preclinical data and CMC data that TWi has already accumulated for AC-203 in treating EBS can be leveraged to support future DEB clinical trials.

What Is " Epidermolysis Bullosa "?

EBS and DEB are different types of Epidermolysis Bullosa (EB). EB is a rare group of congenital skin disorders. Due to genetic mutations, patients have more fragile skin structure than average; even minor friction or bumps can cause blisters, skin breaks, and wounds. EB shows no significant ethnic or geographic variation worldwide, with approximately 500,000 patients globally—about 70% having EBS and 25% having DEB. Compared with EBS, DEB wounds heal much more slowly and may be accompanied by persistent pain; severe DEB patients can even develop squamous cell carcinoma of the skin over time, leading to high mortality rates. Consequently, DEB remains an area with highly unmet medical needs. Currently, several treatment options for DEB are FDA-approved, but they involve localized gene or cell therapies, which not only face limited accessibility but also entail annual costs ranging from hundreds of thousands to over a million dollars.. Thus, the market continues to have significant unmet needs.

AC-203 is a topical small-molecule drug. If future DEB clinical trials demonstrate its safety and efficacy, it could offer a convenient, effective, and cost-advantaged treatment option for DEB patients.

AC-203 EBS Global Phase II/III Clinical Trials Enter Critical Stage

TWi Biotechnology's ongoing global phase II/III randomized double-blind clinical trial for AC-203 in EBS has reached the critical pre-unblinding phase. The trial completed enrollment of 81 patients worldwide in April 2026 and is expected to announce topline results in Q4 2026. "Unblinding" refers to the rigorous statistical analysis performed after trial completion to confirm whether AC-203-treated patients show a significant therapeutic advantage over the placebo control group. As the EBS global clinical trial approaches unblinding stage, TWi Biotechnology has simultaneously started commercialization preparations, continuing discussions with multiple international pharmaceutical companies for global licensing. The company aims to accelerate AC-203's regulatory filings and global launch through strategic international partners.

From Single Indication to Multiple Patient Populations

The FDA's guidance on AC-203's development for DEB represents an opportunity to further expand AC-203's indications. According to TWi Biotechnology 's estimates, extending AC-203 from EBS to DEB will significantly enlarge the potential patient population, offering new treatment options to more epidermolysis bullosa patients while enhancing AC-203's market value.

Dr. Chen-En Tsai, President of TWi Biotechnology, stated: "We are pleased that the FDA has provided clear development guidance for AC-203 in treating DEB. We are currently collaborating with EB House Austria, a rare skin disease research institution, on a designated patient use program for specific DEB patients, and progress is quite smooth. Following the FDA's recommendations, we will continue to advance AC-203's subsequent clinical development, aiming to provide convenient and effective treatments for more epidermolysis bullosa patients."