AI Drug ISM5939 Granted FDA Clearance
Insilico Medicine, a pioneering company in generative artificial intelligence (AI) drug discovery, has recently announced the receipt of Investigational New Drug (IND) clearance from the FDA for ISM5939. This drug, which acts as a small molecule inhibitor targeting ENPP1, is poised for use in the treatment of solid tumors, marking a significant advancement in the field of cancer immunotherapy.
Mechanism of Action
ENPP1, an ecto-nucleotide pyrophosphatase, plays a pivotal role in various biological functions, including the immune response and purinergic signaling. Notably, high levels of ENPP1 have been linked to metastasis and poor prognosis across several cancer types. By inhibiting ENPP1, ISM5939 aims to bolster the body’s immune response against tumors, enhancing the effectiveness of cancer treatments through the activation of the cGAS-STING pathway.
Preclinical Success
Before receiving FDA clearance, ISM5939 was identified as a preclinical candidate in May 2023. Preclinical studies demonstrated robust anti-tumor efficacy, favorable safety profiles, and positive pharmacokinetic data, underscoring the drug’s potential for safe and effective cancer treatment.
Expert Insights
“I am looking forward to the forthcoming clinical signal of ISM5939,” commented Sujata Rao, M.D., Chief Medical Officer of Insilico Medicine. “With its favorable safety window and high potential for combination therapy, it is poised to significantly broaden the spectrum of treatment options for cancer patients.”
The Role of AI in Drug Discovery
Insilico’s drug discovery success is underpinned by its proprietary generative chemistry engine, Chemistry42, which facilitated the rapid identification and optimization of ISM5939’s novel molecular structure. The company has made impressive strides, achieving IND approvals for 10 AI-enabled drug candidates to date.
Feng Ren, Ph.D., Co-CEO and Chief Scientific Officer of Insilico Medicine highlighted, “The AI-driven drug discovery industry requires real-world proof-of-concept cases. At Insilico, we have showcased the potential of our proprietary AI platform through consistent milestones in our ongoing pipeline.” This reflects a significant shift in how AI can expedite and enhance the drug development process.
Vision for the Future
Since introducing generative AI for drug design in 2016, Insilico has continually integrated advancements into its Pharma.AI platform, which spans various domains including biology and chemistry. The publication of a recent study in Nature Biotechnology underscoring the transition from AI algorithms to clinical trials highlights the growing legitimacy and potential of AI in drug development.
Looking ahead, Insilico’s focus remains on leveraging AI to innovate cancer treatments while expanding its portfolio, which now includes over 30 assets and 20 preclinical candidates.