Insilico Medicine's AI-driven drug RentoStib gets its official generic name

Insilico Medicine's AI-driven drug RentoStib gets its official generic name

Insilico Medicine (“Insilico”), a generative artificial intelligence (AI) biotechnology company, recently announced that its breakthrough discopathic pulmonary fibrosis (IPF) drug, Rentosertib (formerly known as ISM001-055), has been given an official generic name (formerly known as ISM001-055), Council Name (among others). RentoSertib is the first investigative agent where both the biological target and therapeutic compound were discovered using generative AI.

Idiopathic pulmonary fibrosis (IPF) is a chronic, debilitating lung disease characterized by a progressive and irreversible decline in lung function. IPF affects approximately 5 million people worldwide and carries a poor prognosis with a median survival of 3 to 4 years. Current treatments, including antifibrotic drugs, can slow, halt, or reverse disease progression but fail to reverse it, leaving a significant unmet need for more effective, disease-modifying therapies.

Insilico took a pioneering approach to anti-IPF research by leveraging its proprietary Pharma.AI platform to discover innovative therapeutics with the potential to halt or even reverse disease progression. The process began with Pandaomics, the platform's biology engine, which analyzed large omics and clinical datasets to identify TNIK (TRAF2 and NCK interacting kinase) as a promising novel target for IPF. Building on this discovery, researchers used Chemistry42, the platform's generative chemistry engine, to rapidly design and optimize new small molecule compounds targeting TNIK, leading to the nomination of RentoSertib as a preclinical candidate.

This integrated AI-driven workflow dramatically accelerated the development timeline, moving from initial target identification to a preclinical candidate in just 18 months, as detailed in InsilicoNatural biotechnologyThe paper published in March 2024. Notably, the name RentoSertib comes in part from Feng Ren, PhD, Insilico's co-CEO and chief scientific officer, who was also the first author of the landmark paper.

This is an important moment for the pharmaceutical industry and AI – RentoSertib is the first drug whose target and design were discovered by modern generative AI, and now it has achieved an official name on its way to patients. The name RentoSertib makes particular sense to us because it not only reflects the contributions of Dr. Ren, but also to advance the essential interplay between human scientific expertise and artificial intelligence in advancing this innovative program. We hope that the success of RentoSertib in IPF paves the way for faster and more cost-effective discoveries of life-saving treatments for many other diseases using AI. “

Alex Zhavoronkov, Founder and CEO of Insilico Medicine

“I am honored to have witnessed and observed RentoSertib from target discovery to clinical development,” said Feng Ren, PhD, co-CEO and chief scientific officer of Insilico Medicine, “an official generic name is typically assigned when a drug enters mid-stage development in mid-stage development. The recognized candidates. Studies that replace the laboratory code.

Currently, RentoSertib has progressed through several clinical trials with encouraging results. In two Phase I trials in New Zealand and China, RentoSertib was administered orally to healthy volunteers in Phase I trials, producing consistent results. The studies demonstrated favorable safety, tolerability and pharmacokinetics (PK) of RentoSertib and provided robust evidence to support Phase II clinical trials.

Building on this foundation, Insilico conducted a Phase IIA clinical trial in IPF patients to evaluate the efficacy of RentoSertib. In this 12-week Phase IIA study, RentoSertib met its primary endpoint of safety and tolerability at all dose levels. Positive results were also reported for the secondary efficacy endpoint, with dose-dependent improvement in vital capacity (FVC) observed. Key findings from the Phase IIA trial include:

• Dose-dependent improvements in lung function:Patients who received RentoSertib showed greater improvement in lung capacity as measured by FVC. At the highest doses of 60 mg QD, patients experienced an average improvement in FVC of 98.4 mL from baseline versus an average decrease in FVC change from baseline of -62.3 mL for patients in the placebo group.

• Additional improved clinical outcomes:A similar dose-related trend was observed in other measures - for example, high-dose patients showed a small improvement in percent FVC (a normalization of lung capacity for age and size), while the placebo group decreased. Patients taking RentoSertib also reported improvements in quality of life measures such as cough and general respiratory symptoms at the highest dose.

• Strong safety and tolerability:RentoSertib continued to demonstrate a favorable safety profile in patients and the drug was well tolerated in all dosage groups. Most drug-related side effects were mild to moderate. No serious adverse events associated with RentoSertib were reported and safety results were consistent with those seen in the previous Phase I trials.

With positive Phase IIA results and an official USAN name, Insilico plans to engage with global regulators and initiate larger pivotal trials to further evaluate the efficacy of RentoSertib in IPF. Insilico is dedicated to advancing RentoSertib with the goal of making it the first AI-discovered therapy to reach patients, providing a critically needed new option for those affected by IPF and demonstrating the transformative potential of generative AI in accelerating medical breakthroughs.

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