US-based Syros Pharmaceuticals has initiated its Phase II clinical trial of SY-1425 by dosing the first patient suffering from relapsed or refractory acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS).

SY-1425 has been developed as a selective retinoic acid receptor alpha (RARα) agonist.

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The company has used a gene control platform to detect subsets of AML and MDS patients whose tumours expressed a highly specialised regulatory region of non-coding DNA, known as a super-enhancer associated with RARA gene, which codes for the RARα transcription factor.

The super-enhancer causes an over-production of RARα transcription factor, resulting in the locking of cells in an immature, undifferentiated and proliferative state.

"Treatment with SY-1425 represents a promising therapeutic strategy for subsets of AML and MDS patients with a novel biomarker that we discovered using our gene control platform."

The multi-centre, open label Phase II trial is intended to test the safety and efficacy of SY-1425 in treating 40 relapsed or refractory AML or high-risk MDS patients selected using the company's biomarker strategy.

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The trial is primarily focused on determining the overall response rate resulted by SY-1425, as well as the pharmacodynamic biomarkers, duration of response, safety and tolerability, and survival resulted from the RARα antagonist.

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Syros chief medical officer David Roth said: “There has been little improvement in the treatment of AML and MDS for the past 20 years, and survival rates for these patients lag behind many other blood cancers.

"Treatment with SY-1425 represents a promising therapeutic strategy for subsets of AML and MDS patients with a novel biomarker that we discovered using our gene control platform.

"Our pioneering approach is designed to advance a new wave of medicines to control the expression of disease-driving genes in genomically defined subsets of patients to provide them with a profound and durable clinical benefit."