Insilico Medicine, a clinical-stage biotech powered by generative artificial intelligence (AI), has announced that its experimental drug, ISM8969, has received US FDA nod for the treatment of Parkinson’s Disease. 

 

ISM8969 is an orally available NLRP3 inhibitor designed to tackle inflammation and neurodegenerative disorders.

 

The upcoming Phase I trial will test the safety, tolerability, and pharmacokinetics of ISM8969 in healthy volunteers and determine the optimal dosing for future studies.

 

“NLRP3 has emerged as a key contributor to chronic neuroinflammation and disease progression in neurodegenerative disorders. A novel NLRP3 inhibitor, ISM8969 with the desired brain penetrant property made possible via our AI-powered design process, offers the potential to advance Parkinson's Disease treatment to the next generation,” said Carol Satler, Senior VP for Clinical Development, Non-Oncology at Insilico Medicine. 

 

“We are excited to get the greenlight from FDA to advance this novel therapeutic to human clinical trials, and we hope to induce a genuine paradigm shift with AI breakthrough in novel drug discovery.”

 

Excessive NLRP3 activation drives overproduction of inflammatory molecules, causing tissue damage and chronic inflammation. ISM8969 aims to block this process, potentially preserving neuronal function in patients with neurodegenerative diseases. The drug was nominated as a potential best-in-class preclinical candidate in December 2024.

 

ISM8969 was discovered and optimized using Insilico’s Chemistry42 AI platform, which streamlines drug design and development. Notably, ISM8969 can cross the blood-brain barrier, giving it a strategic advantage in treating central nervous system disorders, including Parkinson’s Disease. Preclinical studies in inflammatory and chronic disease mouse models have shown promising efficacy.

 

To accelerate development, Insilico has partnered with Hygtia Therapeutics under a co-development agreement. Both companies will share 50% of global rights and interests in ISM8969, with Insilico eligible for up to $66 million in upfront and milestone payments.

 

Insilico’s broader AI-driven portfolio, Pharma.AI, has already attracted partnerships with major pharmaceutical players, including Sanofi, Lilly, Exelixis, and Menarini, with license deals totaling up to $2.1 billion. 

 

The company has shortened the typical early-stage drug discovery timeline from an industry average of 4.5 years to just 12–18 months per program, synthesizing and testing only 60–200 molecules per candidate.