ShanghaiJune 17, 2026 /PRNewswire/ — Insilico Medicine (HKEX: 3696), a clinical-stage generative artificial intelligence (AI)-driven biotechnology company, today announced that ISM8969, co-developed with Hygtia Therapeutics, has achieved its first clinical milestone with the first patient dosed in a Phase I clinical study. ISM8969 is an oral, brain-penetrant small molecule NLRP3 inflammasome inhibitor with potential best-in-class potential, intended for the treatment of chronic neuroinflammation and central nervous system (CNS) diseases, including Parkinson’s disease.
AI-driven NLRP3 inhibitor ISM8969 completes first subject dosing in Phase I clinical trial
As the first study in the clinical development of ISM8969, this single-center, Phase I, randomized, double-blind, placebo-controlled study includes single ascending dose and multiple ascending dose cohorts. It aims to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of oral ISM8969 in healthy subjects and obese adult subjects with cardiovascular risk. The Phase I study is being conducted in Australia and is expected to enroll 80 healthy subjects and 20 obese adult subjects with cardiovascular risk.
Additionally, the study will collect cerebrospinal fluid samples from subjects to assess ISM8969’s ability to penetrate the central nervous system and determine its pharmacokinetic and pharmacodynamic profile in the target tissue. Data obtained from this study is expected to provide key evidence for dose selection in subsequent clinical development and support the evaluation of its potential therapeutic relevance.
Dr. Feng Ren, Co-CEO and Chief Scientific Officer of Insilico Medicine, said: “Advancing ISM8969 from an AI-generated concept molecule to first-in-human dosing represents a significant milestone and the first clinical achievement in our collaboration with Hygtia Therapeutics. Developing a potent NLRP3 inhibitor that can safely cross the blood-brain barrier has long been an industry challenge. Leveraging Chemistry42, we optimized the molecule to demonstrate promising efficacy and favorable permeability in preclinical studies. We look forward to evaluating its translational potential in this Phase I study and continuing to work closely with Hygtia Therapeutics to advance innovative neuroscience therapies for patients worldwide.”
The NLRP3 inflammasome is a key component of the body’s innate immune response. However, its excessive or chronic activation leads to overproduction of pro-inflammatory cytokines, driving persistent neuroinflammation and cellular damage, which contributes to the progression of debilitating neurodegenerative diseases. By inhibiting NLRP3, ISM8969 aims to modulate this pathological inflammation, thereby helping maintain neuronal survival and function in patients with neurodegenerative diseases.
In preclinical evaluation studies, ISM8969 demonstrated a balanced drug-like profile, including favorable in vitro activity and safety, excellent in vivo PK/PD characteristics, and significant anti-inflammatory efficacy in multiple mouse disease models, covering both acute inflammatory diseases and chronic conditions. Compared to other NLRP3 inhibitors that are progressing faster clinically but are peripherally restricted, ISM8969 can penetrate the blood-brain barrier and enter the central nervous system, making it potentially suitable for treating diseases associated with neuroinflammation. Based on these preclinical data, Insilico Medicine selected ISM8969 as a preclinical candidate compound for this program in December 2024.
To accelerate the global development of this candidate drug, Insilico Medicine entered into a global exclusive strategic co-development collaboration with Hygtia Therapeutics, a portfolio company of Shenzhen Pengfu Capital and Fosun Pharma, under Fosun Health Capital. Under the agreement, both parties hold a 50% share of the global rights to the program, with Insilico Medicine responsible for advancing the IND filing and Phase I clinical trial. As the program progresses, Insilico Medicine is eligible to receive up to $66 million in upfront and milestone payments.
As an AI-driven biotechnology company, Insilico Medicine is redefining preclinical drug development efficiency through its advanced AI and automation platforms, setting new industry standards. Compared to traditional early drug discovery, which typically takes 2.5 to 4 years, Insilico Medicine advances projects from initiation to preclinical candidate compound nomination in an average of 12 to 18 months, with only 60 to 200 molecules synthesized and tested per project. Since 2021, the company has nominated 31 preclinical candidate compounds, 13 of which have received IND approvals.
While continuously expanding the practical application of its technology in drug R&D and life science research, Insilico Medicine is also enhancing the performance of its AI platform. Leveraging the rich experience and data accumulated from its training platform, the company has distilled thousands of benchmarks and integrated them into MMAI Gym. As a “trainer” and “benchmarking platform” for scientific AI, MMAI Gym helps institutions train models with domain-specific reasoning capabilities and rigorously evaluate their performance on real-world tasks, thereby advancing the journey toward “pharmaceutical superintelligence.” To date, Human Longevity and Liquid AI have partnered with Insilico Medicine as MMAI Gym collaborators.
About Insilico Medicine
Insilico Medicine is a global pioneer biotechnology company dedicated to integrating artificial intelligence and automation technologies to accelerate drug discovery and drive innovation in life sciences, empowering people to live longer, healthier lives. On December 30, 2025, the company was listed on the Main Board of the Hong Kong Stock Exchange under the stock code: 03696.HK.
Using its proprietary Pharma.AI platform and advanced automated biology laboratory, Insilico Medicine is providing innovative drug solutions for unmet disease areas such as fibrosis, oncology, immunology, pain, obesity, and metabolic disorders. Additionally, Insilico Medicine continues to expand the application of Pharma.AI into diverse fields, including advanced materials, agriculture, nutritional products, and veterinary medicine. For more information, please visit www.insilico.com
About Hygtia Therapeutics
Hygtia Therapeutics is an innovative biotechnology company focused on autoimmune and related areas, dedicated to introducing and developing innovative pipelines with “best-in-class” potential globally, and providing breakthrough treatment options for patients through efficient translational and clinical development capabilities.