TOPLINE:

Rigosertib, a polo-like kinase 1 (PLK1) inhibitor, demonstrated antitumor efficacy in patients with recessive dystrophic epidermolysis bullosa (RDEB) and advanced squamous cell carcinoma (SCC), with an objective response achieved in four of five patients.

METHODOLOGY:

Researchers conducted an open-label, single-arm phase 2 study in Austria and the United States of five patients with advanced, treatment-refractory RDEB-SCC who had failed prior standard of care and were then treated with oral or intravenous (IV) rigosertib.

Participants (median age, 23 years) received either oral rigosertib (560 mg twice daily on days 1-21 of each 28-day cycle for 13 cycles) or IV rigosertib (1800 mg/24 h as a 72-h continuous infusion on days 1-3 of each 14-day cycle for eight cycles, then on da

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