TAIPEI, Taiwan – [2/5/2026] – TWi Biotechnology, Inc. (Stock Code: 6610) today announced that the interim analysis has been completed for its global Phase 2/3 clinical trial, EBShield Study. This trial evaluates the efficacy and safety of AC-203 in patients with epidermolysis bullosa simplex (EBS), a rare genetic skin disorder.
Upon reviewing the interim analysis data, the Independent Data and Safety Monitoring Board (DSMB) confirmed that the overall safety profile of the trial remains acceptable. Consequently, the DSMB recommended continuing the study as planned, with an enrollment target of 80 subjects.
The EBShield Study is a 40-week trial consisting of two parts. Part A is a randomized, double-blind, vehicle -controlled treatment phase with an 8-week dosing period followed by an 8-week off-treatment follow-up. Part B is an open-label extension phase in which all subjects receive AC-203 for 24 weeks, designed to accumulate long-term safety and efficacy data. The study is designed to enroll in either 80 or 100 subjects. Per protocol, an interim analysis will be conducted after the first 60 subjects have completed 8 consecutive weeks of treatment in Part A. Based on safety and efficacy data, the DSMB will determine the final sample size to enhance the probability of achieving statistical significance.
The DSMB meeting was held on the evening of February 4, 2026, to review the interim data from the EBShield Study and concluded that there were no safety concerns. The DSMB recommended that the trial proceed per the original clinical protocol until enrollment of 80 subjects is completed. The study is projected to complete patient enrollment and primary efficacy endpoint assessments by the end of 2026. Subsequent regulatory consultations with U.S. and European authorities will be conducted, with timelines subject to the progress of the ongoing trial.
Epidermolysis bullosa simplex (EBS) is a rare inherited skin disorder characterized by extreme skin fragility, where minor mechanical friction may result in blistering and skin injury. Patients often require continuous wound care, dressing changes, and bandaging to maintain skin integrity and prevent infection, placing a significant burden on both patients and caregivers. According to published studies, the average annual cost of care per patient with such rare diseases may reach up to USD 100,000, representing a substantial economic burden on families and society.
Currently, there are no approved therapies worldwide for the treatment of EBS. AC-203 is the most advanced clinical-stage drug candidate for this indication and has received orphan drug designation (ODD) in the United States, the European Union, and Taiwan, as well as rare pediatric disease designation (RPDD) and Fast Track designation from the U.S. Food and Drug Administration (FDA), underscoring its potential clinical and regulatory advantages in addressing a significant unmet medical need.
In addition, AC-203 is eligible for application for a Rare Pediatric Disease Priority Review Voucher (RPD-PRV). The RPD-PRV program was originally set to expire on September 30, 2026. With strong advocacy from multiple rare disease organizations, both chambers of the U.S. Congress have recently passed the “Give Kids a Chance” Act, which has been signed into law by President Trump. The Act extends the RPD-PRV program through September 30, 2029.
“AC-203 is a topical formulation containing diacerein as its active ingredient, with anti-inflammatory properties designed to reduce blister formation and skin damage associated with EBS,” said Chen-En Tsai, President and Chief Medical Officer of TWi Biotechnology. Data from earlier clinical studies demonstrated that AC-203 may help reduce blistering and improve patients' quality of life. The EBShield Study is currently enrolling patients across 36 clinical sites in 18 countries worldwide and is expected to become the first-in-class approved therapy for patients with EBS globally.
From the business development perspective, TWi Biotechnology has licensed AC-203 in Greater China, Japan, and South Korea, and has entered into term sheets or memoranda of understanding with partners in Russia, the Middle East, and North Africa. The Company is also actively engaged in discussions with multiple international pharmaceutical companies regarding potential commercialization partnerships in the United States and Europe. With the completion of the interim analysis for the EBShield Study, the Company plans to accelerate global partnering efforts, especially in United States and Europe.
In addition, the Company's other investigational drug, NORA520, being developed for the treatment of postpartum depression, has completed a Phase 2 clinical trial. Topline results indicated that mean plasma concentrations in the treatment group were lower than expected, with several patients exhibiting levels below the limit of quantification. The Company is currently conducting a root cause analysis to determine if procedural or operational issues occurred during the trial. Following the exclusion of affected subjects, the ad-hoc analysis is ongoing to refine subsequent clinical trial design and enhance clinical trial operational oversight.
Looking ahead, TWi Biotechnology will continue advancing the global Phase 2/3 clinical trial and worldwide commercialization of AC-203, while consulting with the U.S. FDA on the further clinical development steps for NORA520. In parallel, the Company is planning a Phase 2a clinical trial for another topical investigational drug, AC-1101, for the treatment of alopecia areata. By continuous strengthening its diverse development pipeline and proactively mitigating operational risks, TWi Biotechnology remains committed to enhancing long-term corporate value.
