CAMBRIDGE, Mass. & MONTREAL - Repare Therapeutics Inc. (NASDAQ:RPTX), a clinical-stage precision oncology company, announced today that the first patient has been dosed in their Phase 1 LIONS clinical trial. The trial is evaluating RP-1664, a potential first-in-class oral inhibitor targeting PLK4 in patients with TRIM37-high solid tumors.
RP-1664 has shown promising results in preclinical studies, including deep tumor growth inhibition and regressions in various solid tumor models. Following safety assessments in the LIONS trial, plans are in place to proceed to a Phase 1/2 trial focusing on high-risk, recurrent pediatric neuroblastoma—a cancer with a high incidence of TRIM37 alterations and limited treatment options.
The LIONS trial (NCT06232408) is a multicenter, open-label study that aims to enroll approximately 80 patients. It will investigate the safety, pharmacokinetics, and preliminary efficacy of RP-1664, with primary endpoints including safety, tolerability, and early antitumor activity.
RP-1664 operates by exploiting a synthetic lethal relationship with TRIM37 amplification or overexpression in solid tumors. Elevated TRIM37 is a common characteristic in a range of solid tumors and is present in around 80% of high-grade neuroblastomas. RP-1664 is currently the only selective PLK4 inhibitor in clinical trials.
Repare Therapeutics leverages its proprietary SNIPRx® platform, a genome-wide CRISPR-based screening approach, to identify synthetic lethal gene pairs and develop targeted cancer therapies. The company's pipeline includes several other candidates in various stages of clinical and preclinical development.
The information in this article is based on a press release statement from Repare Therapeutics Inc.
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