News Release

Insilico Medicine nominates preclinical candidate of inhalation solution for ISM001-055, the first generative AI IPF drug

Business Announcement

InSilico Medicine

Inhalation Solution for First Generative AI Drug for IPF

image: Insilico Medicine merges nebulizer device with AI-driven drug discovery. view more 

Credit: Insilico Medicine

●  Insilico Medicine merges nebulizer device with AI-driven drug discovery;

●  Potential advantages for the inhalation solution include a smaller effective dose, larger safety window and reduced side effects; 

●  As demonstrated in preclinical studies, the inhalation solution of ISM001-055 is well tolerated with good anti-fibrotic and anti-inflammatory efficacy, favorable safety and pharmacokinetic (PK) profiles, and no local or systemic toxicity observed.


Insilico Medicine ("Insilico"), a generative artificial intelligence (AI)-driven clinical-stage drug discovery company, today nominates the preclinical candidate of an inhalation solution for ISM001-055, the first anti-fibrotic small molecule inhibitor developed leveraging its proprietary AI drug discovery platform Pharma.AI for the treatment of Idiopathic Pulmonary Fibrosis (IPF). With promising preclinical results, Insilico is proceeding with IND filing as the first AI drug discovery company to venture into nebulized formulations.

“I am thrilled to see the announcement of inhalation solution for ISM001-055, which is a remarkable milestone for a novel drug candidate, as 99% of programs fail at the preclinical stage,” said Alex Zhavoronkov, Ph.D., founder and CEO of Insilico Medicine. “With the support of Pharma.AI driven by generative artificial intelligence, we are making drug discovery more efficient and more accurate, as demonstrated by our three programs in the clinical stage.” 

Administration by inhalation is a complex drug delivery technology that requires combining formulation and devices, presenting higher technical barriers. Inhalation solutions deliver treatment as vapor or aerosol through special devices into the respiratory tract (i.e. the target organ), which is considered one of the most efficient routes of drug delivery as it may provide advantages including rapid action onset, high bioavailability, lowered effective dose. and reduced side effects with a noninvasive and targeted route of administration.

In preclinical studies, the inhalation solution of ISM001-055 was able to achieve high lung exposure with low systemic exposure and demonstrated antifibrotic and anti-inflammatory efficacy in animal models. Moreover, it showed good pharmacokinetic (PK) and safety profiles, as well as good stability and solubility.

“At Insilico, innovation never stops, and that means multidimensional validation of our AI platform capabilities for various disease areas, indications and formulations,” said Feng Ren, Ph.D., Co-CEO and CSO of Insilico Medicine. “We hope to proceed the inhalation formulation of ISM001-055 to clinic as soon as possible to address unmet clinical needs.”

Powered by Pharma.AI, the Company’s proprietary AI drug discovery platform across biology, chemistry and clinical development, Insilico has developed a comprehensive portfolio comprising 30+ pipelines and covering 29 targets, with 3 currently in the clinical stage. In addition to this inhaled solution which intersects pharmacology, particle dynamics, and nebulizer design, Insilico continues to explore the integration of advanced technologies into its proprietary Pharma.AI platform, including quantum computing, multimodal transformer technology, and large language models


About INS018_055

INS018_055 is a potentially first-in-class small molecule inhibitor with a novel target discovered by Insilico’s target identification engine, PandaOmics, and a novel molecular structure designed by its generative chemistry engine, Chemistry42. In February 2021, Insilico nominated INS018_055 as a preclinical candidate for the treatment of IPF, a chronic lung disease that results in a progressive and irreversible decline in lung function, and initiated a first-in-human study in November 2021. The FDA granted Orphan Drug Designation to INS018_055 for the treatment of Idiopathic Pulmonary Fibrosis in February 2023. INS018_055 is currently being assessed in international multicenter Phase II trials, with the first patients dosed in June 2023.


About Insilico Medicine

Insilico Medicine, a clinical-stage biotech company powered by generative AI, is connecting biology, chemistry, and clinical trials analysis using next-generation AI systems. The company has developed AI platforms that utilize deep generative models, reinforcement learning, transformers, and other modern machine learning techniques for novel target discovery and the generation of novel molecular structures with desired properties. Insilico Medicine is developing breakthrough solutions to discover and develop innovative drugs for cancer, fibrosis, immunity, central nervous system diseases, infectious diseases, autoimmune diseases, and aging-related diseases. 

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