Events．2025.12.27
Beyond Pain, Empowering Voices: Advancing Innovation for Rare Disease Care
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Beyond Pain, Empowering Voices: Advancing Innovation for Rare Disease Care
TWi Biotechnology, Inc. (TWSE: 6610) is sharing information related to the patient advocacy event “Beyond Pain, I Want to Say…”, which highlights the resilience and determination of individuals living with Epidermolysis Bullosa (EB), a rare and severe genetic skin disorder.
The event underscores the importance of patient empowerment, self-advocacy, and public awareness, reflecting the critical role that patient voices play in addressing significant unmet medical needs in rare diseases. Through personal narratives and public engagement, EB patients demonstrate how resilience and advocacy contribute to a more holistic understanding of disease management and quality of life.
Addressing Unmet Needs in EB Through Inflammation-Modulating Research
Epidermolysis Bullosa (EB) is characterized by extreme skin fragility, recurrent blistering, and chronic wounds resulting from genetic mutations affecting skin integrity. Inflammatory responses are widely recognized as a contributing factor to disease severity and progression, making inflammation modulation an important area of ongoing scientific research.
TWi Biotechnology’s lead investigational product, AC-203, is a topical formulation containing Diacerein, a small-molecule compound with more than two decades of clinical use in osteoarthritis. Diacerein acts by inhibiting intracellular pro-inflammatory mediators, thereby reducing downstream inflammatory signaling.
Based on translational research and previously reported clinical observations, Diacerein may have the potential to reduce blister formation and support wound healing in certain EB patient populations. Building on findings reported by European clinicians, TWi Biotechnology further optimized the formulation to address dermatological characteristics commonly observed in EB patients, resulting in the proprietary AC-203 ointment with enhanced moisturizing properties.
AC-203 ointment (1%) has received:
- Orphan Drug Designation from the U.S. Food and Drug Administration (FDA)
- Rare Disease Drug Designation from the Taiwan Food and Drug Administration (TFDA) for the treatment of Epidermolysis Bullosa Simplex (EBS)
These regulatory recognitions reflect the significant unmet medical need and support the continued clinical development of AC-203.
Pipeline Progress and Global Development Strategy
Founded in 2010 by internationally recognized pharmaceutical scientist and serial entrepreneur Dr. Chin-Ming Chen, TWi Biotechnology focuses on innovative drug development through a drug repositioning strategy, leveraging clinically validated compounds to potentially accelerate development timelines while managing risk.
The company’s research and development efforts are centered on rare diseases and inflammatory dermatological conditions, with key pipeline milestones including:
- AC-203 for Bullous Pemphigoid (BP): Phase II clinical trial completed
- AC-203 for Epidermolysis Bullosa Simplex (EBS): Ongoing Phase II/III clinical trial
Rooted in Taiwan and aligned with international regulatory standards, TWi Biotechnology continues to advance its pipeline with a long-term global development perspective.
Commitment to Long-Term Value Creation
TWi Biotechnology is committed to addressing high-unmet medical needs through patient-focused research and scientifically grounded innovation. By advancing therapies for rare diseases and maintaining transparent communication with stakeholders, the company aims to create sustainable long-term value for patients, healthcare systems, and shareholders.
For additional company information, please visit:
https://www.twibiotech.com/