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Leapfrog Bio Publishes Breakthrough Validation of Precision PGx Platform™ in Nature PJ Precision Oncology

NextEdge Admin

05 Sep 2024

2 min 18 sec

Leapfrog Bio, a clinical-stage precision oncology company, has announced a breakthrough publication in Nature PJ Precision Oncology showcasing its Precision PGx Platform™, a novel pharmacogenomic approach designed to accelerate oncology drug development. The platform targets undruggable cancer-driving mutations by uncovering vulnerabilities in loss-of-function (LoF) mutations and matching them with drugs that exploit these weaknesses to eliminate cancer cells.

Using driver-focused CRISPR screens in isogenically controlled cell lines, coupled with next-generation sequencing (NGS), the platform identifies genes that "drop out" when mutated and treated with specific drugs. This approach was validated through proof-of-concept studies using PARP inhibitors, which are currently the only drugs targeting LoF mutation-driven cancers. The findings were further corroborated by 100% validation in mouse xenografts, highlighting the platform’s ability to predict patient responses based on tumor genetics.

CSO Tomas Babak emphasized the significance of pharmacogenomic interactions, noting that these capture more complex drug effects than traditional synthetic lethality screens. Leapfrog Bio’s platform addresses a critical gap, as two-thirds of cancers result from LoF mutations, where no functional protein exists to target.

CEO Greg Vontz added that over 2,400 cancer therapeutics have failed to achieve FDA approval due to lack of efficacy, despite being safe. After screening a small portion of these molecules, Leapfrog has identified several promising drug-genotype combinations and filed intellectual property applications for them, aiming to bring new hope to patients with currently untreatable cancers.