Q&A: How Insilico Medicine’s AI identified a new IPF drug target in record time

Alex Zhavoronkov, Ph.D.

Idiopathic Pulmonary Fibrosis (IPF), a devastating lung disease affecting millions with increasing incidence, may have a new treatment hope thanks to a novel inhibitor of TNIK, a kinase newly implicated in fibrosis, identified using generative AI drug discovery platforms in just 18 months.

Researchers at Insilico Medicine, along with international collaborators, harnessed the power of AI platforms PandaOmics and Chemistry42 to make a breakthrough discovery. Nature Biotechnology recently published the group’s findings, showcase the potential of AI to accelerate drug discovery. The scientists identified TNIK as a promising therapeutic target for fibrotic diseases and developed INS018_055, a potent TNIK inhibitor. Preclinical models demonstrated INS018_055’s superior anti-fibrotic and anti-inflammatory effects compared to current treatments.

Beyond fibrotic diseas…
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Insilico’s AI-discovered INS018_055 graduates to phase 2

Roughly a year ago, Insilico Medicine announced that it had dosed the first patient in a phase 1 study of INS018_055, an AI-discovered, first-in-class small molecule inhibitor. Now, the company has progressed to the next stage, launching a phase 2 study for the drug candidate.

Insilico, a founding member of NVIDIA Inception, developed its AI drug-discovery platform on NVIDIA GPUs. Inception is a complimentary program that provides startups with technical training and AI platform support.

The discovery of INS018_055 was the result of combining multiple technologies. Insilico’s proprietary Pharma.AI platform incorporates AI models trained on massive amounts of data. Insilico’s target identification platform, PandaOmics, discovered a novel target. Then Insilico’s generative chemistry platform, Chemistry42, designed the molecule’s structure. In addition, these AI systems rely on machine learning techniques like deep generative models, reinforcement lear…

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Why Aria Pharmaceuticals is upbeat about two novel IPF treatment candidates 

Small molecule drug developer Aria Pharmaceuticals (Palo Alto, Calif.) recently revealed that two investigational treatments for idiopathic pulmonary fibrosis (IPF), TXR-1002 and TXR-1007, demonstrated promising results in preclinical research.

There are currently only two FDA-approved treatments for IPF on the market — nintedanib (Ofev) from Boehringer Ingelheim and pirfenidone (Esbriet) from Roche. While those introductions marked a turning point in treating IPF, managing the disease remains challenging for many patients.

Both TXR-1002 and TXR-1007 rely on a mechanism of action than that of nintedanib or pirfenidone. And Aria reports that preclinical testing indicated that the drugs were well tolerated. “Certainly, you can’t tell if a rat is suffering from nausea or something like that, but at a gross level, the animals did well through the course of the study,” said Mark Eller, senior vice president, R&D at Aria Pharmaceuticals. “We’re optimistic based o…

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Why pulmonary fibrosis deserves more attention

Idiopathic pulmonary fibrosis x-ray courtesy of Wikimedia Commons.

This September is the inaugural Pulmonary Fibrosis Awareness Month, which the Pulmonary Fibrosis Foundation and allies have launched to educate the public about the disease family. Involving scarring of the lungs, pulmonary fibrosis gradually robs the breath from patients it affects. 

Some 200,000 Americans live with idiopathic pulmonary fibrosis.  

At present, two drugs are available to treat the condition in the U.S., but pulmonary fibrosis remains an active focus area for drug developers.

Given that September is Pulmonary Fibrosis Awareness Month, the anniversary of the September 11 attacks also can serve as a reminder of the impact of interstitial lung disease. “Lots of lung disease has occurred in the first responders of the World Trade Center,” said Dr. Joyce Lee, a senior medical advisor for the Pulmonary Fibrosis Foundat…

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The promise of mitochondria-based therapeutics for NASH

Liver with signs of nonalcoholic fatty liver disease. Image courtesy of NIH.

Although nonalcoholic steatohepatitis (NASH) is one of the most common types of chronic liver disease in the U.S., it is a disease that is unfamiliar to many Americans. Despite this, its prevalence is increasing, and by some estimates, 27 million Americans will be living with NASH by 2030.

NASH is a progressive condition that starts with excess fat buildup in the liver, leading to inflammation and liver damage. While the cause of NASH is unknown, it is associated with a broader set of metabolic disorders. Important risk factors include elevated triglyceride or cholesterol levels, type 2 diabetes, high blood pressure and obesity, particularly with body fat concentrated around the waist. NASH is also more prevalent in certain ethnic groups, including Asian and Hispanic populations. While there are generally no symptoms until late in t…

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The pulmonary fibrosis treatment landscape: An interview with an expert

Photo by Anna Shvets from Pexels

In 2014, FDA approved two medications for idiopathic pulmonary fibrosis (IPF) — nintedanib from Boehringer Ingelheim and pirfenidone from Roche (OTCMKTS:RHHBY). “That was a huge success for our community but certainly, it’s the beginning of what we need to be doing for our patients,” said Dr. Joyce Lee, a senior medical advisor for the Pulmonary Fibrosis Foundation, a nonprofit dedicated to supporting research and medical advances related to the treatment of the condition.

Idiopathic pulmonary fibrosis, which is associated with shortness of breath and a significant symptom burden, has no known cause or cure.

[Related: 6 notable pulmonary fibrosis research efforts]

But there is reason for hope, according to Lee, who is also a professor of medicine specializing in pulmonary sciences and critical care at the University of Colorado. “There is a lot of investigation …

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6 notable pulmonary fibrosis research efforts 

Image courtesy of Pixabay

Idiopathic pulmonary fibrosis (IPF) is frequently a debilitating disease associated with significant morbidity and mortality. Although a rare disease, its incidence has increased in recent decades, and it leads to more deaths than some cancers. 

The treatment landscape for the condition changed in 2014 when FDA approved the first drugs indicated for IPF, nintedanib from Boehringer Ingelheim and pirfenidone from Roche (OTCMKTS:RHHBY). 

[Related: The pulmonary fibrosis treatment landscape: An expert interview]

Research on pulmonary fibrosis has increased in recent years. 

Here’s a summary of notable IPF research: 

Boehringer Ingelheim has an international study investigating a phosphodiesterase 4b (PDE4b) inhibitor in patients with idiopathic pulmonary fibrosis. Researchers have found that the drug has anti-fibrotic effects in animal models and lung fibrosis …
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