INSILICO (03696) and Hengtai Biotech have jointly developed the blood-brain barrier penetrating NLRP3 inhibitor ISM8969/HT-001, which has received approval for clinical trials from the NMPA.

INSILICO (03696) announced that the innovative oral NLRP3 inhibitor ISM8969/HT-001, co-developed with Shenzhen Hengtai Biotechnology Co., Ltd. (Hengtai Bio), has received approval from the National Medical Products Administration (NMPA) of China for new drug clinical trials for the treatment of Parkinson's disease. This approval marks the official commencement of parallel clinical evaluations in both China and Australia, following the announcement of the first human dosing in a Phase I study in Australia in June 2026. The purpose of this clinical trial is to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) characteristics of ISM8969/HT-001 in a healthy participant population, as well as its preliminary efficacy in patients with Parkinson's disease, laying the foundation for subsequent clinical development. In preclinical evaluation studies, ISM8969/HT-001 demonstrated balanced drug-like properties, including good in vitro activity and safety, excellent in vivo PK/PD characteristics, and efficacy against inflammation in multiple mouse disease models, which encompass acute inflammatory diseases and chronic conditions. Compared to other NLRP3 inhibitors that progress rapidly in clinical trials but are limited to peripheral effects, ISM8969/HT-001 can penetrate the blood-brain barrier and enter the central nervous system, suggesting its potential for treating neuroinflammation-related diseases.