Insilico Medicine Nominates Preclinical Candidate for ISM001-055, First Generative AI IPF Drug

24 July 2023 | Monday | News

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 by 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.
Image Source : Public Domain

Image Source : Public Domain


  • 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.

"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, which is proved by the 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 higher 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.

"Here 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+ pipeline product candidates 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 computingmultimodal transformer technology, and large language models.


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