Phase I Trial for Novel Cancer Drug: VVD-130850

A Phase I clinical trial has been initiated ( in Feb 2024) to evaluate an investigational small-molecule inhibitor (VVD-130850) that targets the STAT3 transcription factor in advanced solid and hematologic tumors.

This oral agent will be tested both as a standalone therapy and in combination with checkpoint inhibitors. The drug’s mechanism involves binding to a novel allosteric pocket on the STAT3 protein, directly inhibiting DNA binding and the expression of STAT3 target genes—an approach aimed at modulating STAT3 activity without altering its overall level.

STAT3 hyperactivation is known to drive tumor progression, metastasis, immune evasion, and other oncogenic processes in various malignancies, including prostate cancer. Because of its fundamental role in cancer cell signaling, STAT3 has been regarded as a high-priority therapeutic target.

This new agent emerged from a chemoproteomics platform designed to identify previously unknown functional pockets on validated proteins and then develop highly selective compounds against those targets.

As part of the trial, investigators will assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary anti-tumor activity of VVD-130850. The launch of this study highlights the promise of using chemoproteomics to uncover novel ways to inhibit critical signaling pathways such as STAT3 in a range of tumors, including prostate cancer.

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