Step Pharma announces publication in Haematologica of key data supporting CTPS1 inhibition as a therapeutic target in blood cancer

On July 24, 2024 Step Pharma, the world leader in CTPS1 inhibition for the targeted treatment of cancer, reported the publication in the August edition of Haematologica of preclinical data from the University of Nantes (CRCI2NA) further supporting the therapeutic activity of Step’s highly selective CTPS1 inhibitors in the treatment of blood cancers (Press release, Step Pharma, JUL 24, 2024, View Source [SID1234645044]).

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The therapeutic activity was observed for mantle cell lymphoma (MCL), including difficult-to-treat in vitro and in vivo models. Furthermore, Step Pharma’s CTPS1 inhibitors show significant synergy when combined with venetoclax, a selective BCL2 inhibitor that is commonly used to treat certain types of lymphoma and leukaemia.

CTPS1, an enzyme crucial in pyrimidine synthesis, plays a significant role in cancer cell proliferation. Step Pharma’s compounds selectively inhibit the de novo pyrimidine synthesis pathway by targeting CTPS1, providing a novel approach to cancer treatment. The Company’s lead asset, dencatistat (STP938), a first-in-class, highly selective, orally bioavailable CTPS1 inhibitor, is currently in phase 1 clinical development for T cell and B cell lymphoma (NCT05463263) with study sites open in France, the UK, and the USA.

MCL accounts for approximately 5% of B cell lymphoma. Despite recent advancements in the treatment of MCL, there is still an unmet clinical need for those who have not responded to Bruton’s Tyrosine Kinase (BTK) inhibitor therapy.

Andrew Parker, Chief Executive Officer of Step Pharma, commented

"The publication in Haematologica of these preclinical data provides additional evidence of the importance of inhibiting CTPS1 for the treatment of blood malignancies, including T and B cell lymphoma. The findings further support our approach as we continue to progress our phase 1 trial to develop targeted and efficient treatment options for individuals with blood cancer."

David Chiron, CNRS researcher at CRCI2NA, Nantes University, added

"These data represent a significant advancement in our understanding of blood cancer biology, particularly regarding the role of CTPS1. Our research on inhibiting CTPS1 emphasises the potential of this approach to fill an important gap in current treatment methods. These findings support the targeted inhibition of CTPS1 as a promising therapeutic strategy and pave the way for further research to gain a deeper understanding of its role and broader applications in different types of blood cancers."