Step Pharma is developing a novel class of oral nucleotide synthesis inhibitors specifically targeting cytidine triphosphate synthase 1 (CTPS1) to induce selective modulation of T and B cell populations.
The discovery of CTPS1 as a potential target for immunomodulation was based on studies of humans with a CTPS1 deficiency (Martin et al., Nature. 2014 Jun 12;510(7504):288-92) providing genetic evidence that CTPS1 inhibition could be used to treat lymphoproliferative cancers and auto-immune diseases.
Cytidine nucleotide triphosphate (CTP) is a precursor required for DNA synthesis, a crucial step in cell division. De novo synthesis of CTP depends on two enzymes, the CTP synthases 1 and 2 (CTPS1 and CTPS2).
CTPS2 is expressed in most tissues whereas CTPS1 expression is low in all tissues including resting T and B cells, but rapidly and strongly up-regulated specifically in T and B cells following activation.
Importantly, CTPS1-deficient patients only exhibit specific immunodeficiencies without other clinical problems and CTPS2 is not able to complement for the lack of CTPS1 in T and B cells. CTPS1 therefore represents a therapeutic target for a new class of highly selective immunomodulatory and oncology drugs.
The anti-proliferative activity of CTPS1 inhibition has been demonstrated in multiple cancer cell lines and the pharmacokinetic/pharmacodynamic relationship has been established using several animal models of disease.
Step Pharma is now preparing to take it's lead compound, STP938, forward into IND/CTA enabling studies with the aim to enter clinical development in 2021.
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Frequently asked questions
When was Step Pharma founded?
Step Pharma was founded in 2014.
What industry is Step Pharma in?
Step Pharma operates in Biotechnology. Find similar Biotechnology investors on gritt.io.
Where is Step Pharma based?
Step Pharma is headquartered in Archamps, France.
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