Insilico Medicine, a pioneer in AI-powered drug discovery, has achieved FDA clearance for ISM5939, an oral small molecule inhibitor designed to target ENPP1 for the treatment of solid tumors. This milestone marks the tenth AI-generated molecule developed by Insilico to receive clearance for clinical trials, showcasing the potential of AI in revolutionizing cancer therapeutics.
ENPP1, an ecto-nucleotide pyrophosphatase, plays a crucial role in purinergic signaling, impacting immune, cardiovascular, neurological, and hematological functions. Elevated levels of ENPP1 are often associated with cancer metastasis and poor prognosis. By inhibiting ENPP1, ISM5939 enhances the anti-tumor response of the host immune system, leveraging the cGAS-STING pathway to regulate extracellular cGAMP levels.
Preclinical data revealed its strong anti-tumor efficacy in vivo, a favorable safety profile, and promising pharmacokinetic characteristics. Dr. Sujata Rao, Insilico Medicine’s Chief Medical Officer, expressed optimism about ISM5939’s clinical potential, citing its safety and compatibility for combination therapies as critical advantages for broadening cancer treatment options.
The development of ISM5939 was powered by Chemistry42, Insilico’s proprietary generative AI platform affiliated with Pharma.AI. Within three months, the platform enabled the identification and optimization of a novel molecular structure through AI-driven techniques and structural modifications.
Insilico Medicine’s leadership in generative AI dates back to 2016, when the concept was first detailed in a peer-reviewed journal. Since then, the company has integrated ongoing technical advancements into its Pharma.AI platform, which now spans biology, chemistry, medicine development, and scientific research.
In 2024, Insilico highlighted its AI-driven achievements in a Nature Biotechnology publication detailing the development journey of ISM001_055, another drug targeting an AI-discovered molecule. This molecule demonstrated favorable safety, tolerability, and efficacy during Phase II clinical trials, reaffirming the potential of AI in drug discovery.
Insilico Medicine continues to innovate in AI-powered therapeutics, making strides toward effective and accessible treatments for cancer patients.
Reference:
1. https://insilico.com/
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