In a notable development in the field of artificial intelligence-aided drug discovery, the U.S. Food and Drug Administration (FDA) has approved an investigational new drug (IND) application for a Phase I/II clinical trial. This trial concerns REC-1245, a promising new chemical entity aimed at treating biomarker-enriched solid tumours and lymphoma, developed by Recursion Pharmaceuticals, an innovator in AI-driven drug discovery based in Salt Lake City.
Founded in 2013, Recursion has made significant strides in reducing the time and costs traditionally associated with drug discovery. Under the leadership of CEO Chris Gibson, the firm has leveraged its end-to-end AI platform to design REC-1245, a drug candidate that moved from identifying a novel target to preclinical testing in just under 18 months—a pace nearly twice as fast as what is customary in the pharmaceutical industry.
REC-1245 operates by selectively degrading RBM39, a newly identified target by Recursion’s AI technology. This target is akin in biological function to CDK12, a notoriously difficult-to-drug target that plays a role in cancer development through its control over cell cycle checkpoints and DNA damage responses. While efforts to inhibit CDK12 often also affect CDK13, resulting in unwanted toxicity, targeting RBM39 has shown to mitigate these issues, focusing solely on impeding CDK12-like functions without impacting CDK13.
The approval for clinical trials marks a significant milestone in Recursion’s trajectory and offers optimism for thousands of potential beneficiaries. The trial for REC-1245 is anticipated to commence in the fourth quarter of 2024, with an estimated patient population exceeding 100,000 across the U.S. and EU. This new avenue for tackling solid tumours holds potential for patients who have limited existing treatment options.
Najat Khan, PhD, the company’s Chief Research and Development Officer and Chief Commercial Officer, highlighted the therapeutic promise of REC-1245. Khan, who joined Recursion earlier this year following a pivotal role at Johnson & Johnson, emphasised the capability of their AI platform in drug target discovery. Recursion’s technology, the Recursion Operating System (OS), utilises an extensive dataset from phenomic studies to model complex biological systems. By simulating disease conditions in human cell lines, they develop compounds to correct these states, identifying effective novel drug targets through machine learning algorithms.
Recursion’s achievements are underscored by recent expansions, including the acquisition of companies like Cyclica and Valence, which enhanced their computational capabilities. Additionally, a strategic merger with U.K.-based Exscientia stands to integrate chemically driven, precision-targeting capabilities, forecasted to generate around ten clinical readouts in the coming year and a half. Partnerships with pharmaceutical giants such as Roche-Genentech, Bayer, Sanofi, and Merck KGaA are similarly positioned to streamline this innovative drug discovery model.
The journey through clinical trials in the drug industry is fraught with challenges, as only a fraction reach successful market entry due to inherent biological complexities. However, Gibson remains optimistic, clarifying that the company’s intent in leveraging computational power is central to untangling these complex parameters, a task unmanageable through human effort alone.
While the field of AI-driven drug discovery is still emerging with isolated successes such as REC-1245, the momentum suggests a transformative shift in how drugs might be discovered and developed. The forthcoming pipeline at Recursion, along with contributions from industry collaborations, hints at a future where AI could become a cornerstone in creating effective, affordable medical treatments.
Source: Noah Wire Services