Step Pharma will use the proceeds of its first fundraising round to support the development of immunomodulators for autoimmune diseases towards the clinic.

Step Pharma develops inhibitors targeting an enzyme called cytidine nucleotide triphosphate (CTPS1) for the treatment of a range of autoimmune diseases. The company is working on several candidates, the most advanced of which is undergoing lead optimization. The €14.5M fundraising round was supported by Pontifax Venture Capital, which joined existing investors including Kurma Partners Bpifrance and Inserm Transfert Initiative. The funds will help to advance the company’s candidates into clinical studies.

Step Pharma is based in Paris and was founded in 2014 by Kurma Partners, the Imagine Institute and Sygnature Discovery. The biotech targets autoimmune diseases using immunomodulators to bring the immune system back under control, stopping it from attacking the body’s own cells and tissues. The company started targeting CTPS1 on the back of research carried out by Peter Arkwright of the University of Manchester and the teams of Sylvain Latour and Alain Fischer at the Imagine Institute in Paris.

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Step Pharma’s CTPS1 inhibitors modulate B and T cells specifically, which are important components of the immune response. In an autoimmune disease, these cells can become overactive, so we need a way to bring them back under control. Cytidine nucleotide triphosphate (CTP) is an essential precursor for DNA synthesis, a crucial step in cell division, which is required for an effective immune response. CTPS1 synthesizes CTP, so the proliferation of B and T cells in response to an antigen is defective in T and B lymphocytes lacking the enzyme.

The effect of a mutation in the CTPS1 gene on an individual’s immune response.

Importantly, CTPS1-deficient patients only suffer from a particular group of immunodeficiencies, which suggests a key role of CTPS1 in selected immune cell populations. This means CTPS1 could be a therapeutic target for a new class of highly selective immunomodulators.

The global autoimmune disease treatment market could be worth over $45B (€38B) by 2022, so Step Pharma will be keen to secure its place in the field. There are a lot of companies taking various approaches to the market, including Boehringer Ingelheim and its Humira biosimilar and Topas Therapeutics, which is using the natural ability of the liver to induce immune tolerance.

Current treatments for autoimmune diseases like physical therapy, non-steroidal anti-inflammatory drugs (NSAIDs) and immunosuppressives cannot cure the disease, but look to control the immune response. Nor can Step Pharma’s inhibitors, but they may help patients without the nasty side effects that are associated with immunosuppressives and long-term NSAID use. If the company successfully treats autoimmune diseases, it may look to branch out into cancer.


Images – Stock Photo / shutterstock.com; Step Pharma

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