Ryvu Therapeutics has initiated a Phase II clinical trial of RVU120, dosing the first patient with relapsed/refractory acute myeloid leukaemia (r/r AML) and high-risk myelodysplastic syndromes (HR-MDS).

The trial, known as RIVER-52, is designed to evaluate the tolerability, safety, anti-tumour activity, pharmacokinetics, and pharmacodynamics of RVU120 as a single agent in adults without any other treatment options.

It is a two-part, multicentre, open-label study and aims to enrol nearly 140 patients.

The first part will focus on anti-tumour activity in people with specific genetic subtypes of AML, including those with NPM1 mutations and in subjects with HR-MDS.

Following the results from Part 1, Part 2 of the trial will continue to evaluate the tolerability, safety, and anti-tumour activity of RVU120 in a larger patient group, targeting subtypes that have shown the highest sensitivity.

The selected dose for the evaluation is 250mg given every other day, based on positive signs of clinical activity noted in a Phase Ib study.

Initially, the trial will commence at study sites in Poland and Italy, with plans to extend to up to 80 sites across the European Union (EU) and non-EU countries.

This study is a component of the RVU120 Development Plan unveiled in October last year and is in line with the company’s financial projections until the first quarter of 2026.

In the first half of 2024, Ryvu intends to launch four Phase II studies of RVU120 across various conditions, including r/r AML, HR-MDS, low-risk MDS (LR-MDS), and myelofibrosis, with a target to enrol more than 100 subjects by year-end.

Ryvu Therapeutics chief medical officer Hendrik Nogai said: “We are pleased to announce the initiation of another RVU120 Phase II study, in line with the development plans presented last year.

“Based on the demonstrated safety profile and observed signs of activity from the Phase Ib study in patients with r/r AML or HR-MDS, we continue to be at the forefront of developing first-in-class CDK8/19 inhibitors.”

Earlier this month, the company dosed the first subject in the Phase II RIVER-81 trial of RVU120 and venetoclax for r/r AML.