Accro Bioscience announced that AC-101 tablets have been included in the Rare Diseases Innovative Drug Development Encouragement Pilot Program of China's National Medical Products Administration (NMPA), with Blau Syndrome (BS) as the proposed indication. The relevant public notice period ended on September 11, 2026.

Previously, in June 2026, the US Food and Drug Administration (FDA) granted AC-101 Rare Pediatric Disease (RPD) designation for the treatment of BS.

AC-101 is a novel, selective RIPK2 inhibitor derived from Accro Bioscience's proprietary drug discovery platform targeting regulatory cell death and inflammation. It is being developed for the treatment of moderate-to-severe Ulcerative Colitis (UC).

Receptor Interacting Protein Kinase 2 (RIPK2) is a key mediator in the NOD signaling pathway, and dysregulation of NOD/RIPK2 signaling is associated with multiple inflammatory and autoimmune diseases, including Inflammatory Bowel Disease (IBD).

AC-101 has completed phase 1 studies in healthy volunteers in Australia and China, demonstrating favorable safety and PK/PD profile. A phase 1b/2a proof-of-concept study in Chinese patients with moderate-to-severe UC has been completed. The efficacy and safety results from this study will be presented at UEG Week 2026.

BS is a rare hereditary autoinflammatory disease caused by pathogenic variants in the NOD2 gene. It usually begins in early childhood and can involve multiple systems, including the skin, joints and eyes. Its classic clinical presentation is the triad of granulomatous dermatitis, arthritis and uveitis.

Joint involvement may lead to joint deformity and functional impairment over time. Ocular involvement can cause uveitis; if not controlled promptly, it may lead to severe visual impairment or even blindness.