Insilico Medicine (“Insilico”, HKEX: 3696), a clinical-stage biotechnology company powered by generative artificial intelligence (AI) , today announced that ISM8969/HT-001, an innovative oral NLRP3 inhibitor co-developed with Hygtia Therapeutics, has received Investigational New Drug (IND) clearance from the National Medical Products Administration (NMPA) of China for the treatment of Parkinson's disease. Following the announcement of first-in-human dosing in Australia under the Phase I study in June 2026, this approval marks the official launch of parallel clinical evaluations in China and Australia. The clinical trial aims to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of ISM8969/HT-001 in healthy subjects and preliminary efficacy of ISM8969/HT-001 in patients with Parkinson's disease, establishing a foundation for its subsequent clinical development.
Previously, in January 2026, Insilico Medicine announced exclusive global strategic co-development collaboration with Hygtia Therapeutics, an incubatee of Shenzhen Pengfu Fund of Fosun Health Capital and Fosun Pharma, to jointly advance the global development of ISM8969/HT-001. Under the agreement, both parties hold a 50% stake in the global rights and interests of the program, and Insilico remains eligible to receive up to $66 million in upfront and milestone payments as the program progresses.
"Supported by our partner, the ISM8969/HT-001 program has maintained the speed momentum from early discovery to clinical development. Following IND clearance from the US FDA and the completion of first-in-human dosing in Australia of Phase I study, receiving IND approval letter from China's NMPA represents another major milestone", says Dr. Carol Satler, Senior Vice President of Non-Oncology Clinical Development at Insilico Medicin e, “With strong preclinical data, we look forward to validating its unique blood-brain barrier permeability and anti-inflammatory efficacy through rigorous clinical studies, accelerating the delivery of this novel therapy with both peripheral and central therapeutic potential to patients worldwide in urgent need of novel treatments."
ISM8969/HT-001 is a potentially best-in-class, orally available, brain-penetrant small-molecule inhibitor of the NLRP3 inflammasome, with its molecular structure was discovered and optimized using Chemistry42, Insilico’s proprietary generative chemistry engine, and was officially nominated as a Preclinical Candidate (PCC) in December 2024. By inhibiting NLRP3, ISM8969/HT-001 aims to modulate pathological inflammation, supporting neuronal survival and function among patients with neurodegenerative disease.
In preclinical evaluation studies, ISM8969/HT-001 exhibited a balanced druggability profile, with promising in vitro activity and safety, favorable in vivo PK/PD profile, as well as efficacy against inflammation in multiple mouse disease models, including acute inflammatory disease and chronic disease models. Compared with other clinically advanced peripherally restricted NLRP3 inhibitors, ISM8969/HT-001 is BBB-penetrant and can access the CNS, enabling potential treatment of neuroinflammation-related diseases.
Since 2026, Insilico Medicine has witnessed a clinical validation wave in its self-developed portfolio, achieving 8 major clinical milestones. Notably, Rentosertib (ISM001-055), its most advanced candidate and an innovative small-molecule TNIK inhibitor, has entered a global multicenter Phase III clinical trial. Additionally, ISM5411, a gut-restricted PHD inhibitor developed for ulcerative colitis (UC) and other inflammatory bowel diseases, completed first patient dosing in the BETHESDA Phase IIa study; ISM8969/HT-001, an oral BBB-permeable NLRP3 inhibitor, secured IND approvals from both the US FDA and China NMPA, and successfully completed first-in-human dosing in Australia; MEN2501 (ISM9682), a KIF18A inhibitor out-licensed to Menarini, completed first patient dosing and triggered a milestone payment; and ISM6331, a pan-TEAD inhibitor targeting Hippo pathway-altered solid tumors, was granted Fast Track Designation by the US FDA.
As an AI-native biotechnology company, Insilico is redefining the efficiency of preclinical drug development through its advanced AI and automation platform, setting a new standard for the industry. While traditional early-stage drug discovery typically takes 2.5 to 4 years, Insilico has consistently reached preclinical candidate (PCC) nomination in an average of just 12 to 18 months, with only 60 to 200 molecules synthesized and tested per program. Since 2021, the company has nominated 31 PCCs, 13 of which have received IND approval or clearance.
While expanding the practical applications of its technology in drug discovery and life science research, Insilico is also continuously enhancing the performance of its AI platform. Drawing on extensive experience and datasets from its training platform, the company has distilled thousands of benchmarks and integrated them into MMAI Gym. Serving as both a “trainer and benchmark” for scientific AI, MMAI Gym enables organizations to train models for domain-specific reasoning while rigorously evaluating their performance on real-world tasks, advancing the path toward pharma superintelligence. To date, Human Longevity and Liquid AI have collaborated with Insilico, joining as partners of MMAI Gym.
About ISM8969/HT-001
ISM8969/HT-001 is an orally available, brain penetrant NLRP3 inhibitor discovered using Insilico’s generative AI platform Chemistry42, which has demonstrated strong efficacy, favorable safety, and robust blood-brain barrier (BBB) penetration in preclinical studies.
Since its nomination as a Preclinical Candidate (PCC) in December 2024, ISM8969/HT-001 has rapidly advanced through clinical development: receiving US FDA IND clearance and announcing a co-development partnership with Hygtia Therapeutics in January 2026; completing first-in-human dosing in Australia of Phase I study in June 2026 as its first clinical milestone; and receiving IND approval letter from China's NMPA in July 2026.
About Insilico Medicine
Insilico Medicine is a pioneering global biotechnology company dedicated to integrating artificial intelligence and automation technologies to accelerate drug discovery, drive innovation in the life sciences, and extend health longevity to people on the planet. The company was listed on the Main Board of the Hong Kong Stock Exchange on December 30, 2025, HKEX:3696.
By integrating AI and automation technologies and deep in-house drug discovery capabilities, Insilico is delivering innovative drug solutions for unmet needs including fibrosis, oncology, immunology, pain, and obesity and metabolic disorders. Additionally, Insilico extends the reach of Pharma.AI across diverse industries, such as 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 fields. The company is committed to introducing and developing innovative pipelines with "best-in-class" potential globally. Through efficient translational and clinical development capabilities, Hygtia aims to provide patients with breakthrough treatment options.