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Vividion’s WRN inhibitor shows first clinical proof of synthetic lethality in MSI-high cancers

Vividion’s WRN inhibitor shows first clinical proof of synthetic lethality in MSI-high cancers

First peer-reviewed clinical data for San Diego-based Vividion Therapeutics, Inc.'s WRN inhibitor VVD-214 showed antitumor activity and prolonged disease control in heavily pretreated MSI-high/dMMR solid tumors, providing early clinical support for WRN inhibition as a synthetic-lethal strategy, according to a Nature Medicine publication. Vividion is a wholly owned subsidiary of Bayer AG.

VVD-214 (RO7589831) is an investigational oral selective covalent WRN helicase inhibitor designed to cause DNA damage accumulation and synthetic lethality in MSI-high/dMMR cancers. The molecule originated from a 2020 Vividion-Roche collaboration, while Vividion and Bayer secured exclusive worldwide rights in June 2025.

The single-arm Phase I trial enrolled 88 previously treated patients with advanced MSI-high and/or dMMR solid tumors. Among 66 efficacy-evaluable patients with MSI-high tumors, who had received a median of three prior lines of therapy and included 95.5% previously treated with immune checkpoint inhibitors, VVD-214 produced a disease control rate (DCR) of 74.2%, including seven confirmed partial responses (10.6%). Median progression-free survival was 6.7 months and median overall survival was 17.6 months.

In the colorectal cancer subset, DCR was 80.5%, including three confirmed partial responses (7.3%). Median progression-free survival was 7.3 months, estimated 12-month overall survival was 78.3%, and median overall survival was not yet estimable. All three CRC responders remained in response at data cutoff.

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Established immunotherapy approaches in MSI-high/dMMR colorectal cancer include anti-PD-1 monoclonal antibody pembrolizumab (Keytruda), studied in KEYNOTE-177, and PD-1 plus CTLA-4 blockade with nivolumab plus ipilimumab, studied in CheckMate 142. These therapies restore antitumor immunity, whereas WRN inhibition exploits a DNA-repair dependency created by microsatellite instability.

Novartis is also clinically testing the reversible WRN inhibitor HRO761 (NCT05838768) in MSI-high/dMMR solid tumors. Early Phase I data cited in the VVD-214 paper showed a 10.5% objective response rate and 84.2% disease control rate in colorectal cancer, broadly consistent with the activity seen with VVD-214, although the compounds use different WRN-binding mechanisms.


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