Step Pharma announced the closing of a Series A financing totalling €14.5 million. Pontifax joined existing investors led by Kurma Partners, and including Bpifrance (Fonds Biothérapies Innovantes et Maladies Rares), Inserm Transfert Initiative, Idinvest, Sygnature Discovery and the Imagine Institute in an €8.6 million second closing of the round.
The additional proceeds will be used to advance the company’s novel class of oral immunomodulators targeting cytidine triphosphate synthase 1 (CTPS1) to clinical studies. CTPS1 is a novel target discovered at the Imagine Institute (Paris, France) in rare genetic disease patients exhibiting deficiency in this enzyme involved in CTP nucleotide production.
“There is a long, established history of using nucleotide synthesis inhibitors to treat autoimmune diseases, such as rheumatoid arthritis, multiple sclerosis or psoriasis, but their clinical use is limited due to lack of tissue selectivity. Targeting CTPS1 should allow selective modulation of specific immune cell populations, such as T and B cells, without affecting other tissues, which should translate to better efficacy and an improved therapeutic index,” said Geoffroy de Ribains, CEO and co-founder of Step Pharma. “Our funding to date has enabled us to build a proprietary assay platform, discover first series of CTPS1 compounds and show initial proof of concept in vivo. With the continued support of our investors and new investor Pontifax, we will be able to advance our CTPS1 program into clinical testing.”
Cytidine nucleotide triphosphate (CTP) is a precursor required for several key processes involved in cell division. It originates from two sources: a salvage pathway and a de novo synthesis pathway that depends on several enzymes, including CTPS1. Importantly, patients deficient in CTPS1 only exhibit certain immunodeficiencies without other clinical problems, strongly suggesting a predominant and key role of CTPS1 in selected immune cell populations. CTPS1 therefore represents a therapeutic target for a new class of highly selective immunomodulatory drugs.
Step Pharma is focused on the development of a novel class of oral nucleotide synthesis inhibitors targeting CTPS1 for the improved treatment of a range of autoimmune diseases. The company has identified several series of CTPS1 inhibitors, with the most advanced series currently undergoing lead optimization. Company was founded in June 2014 by Kurma Partners, the Imagine Institute, and Sygnature Discovery based on the scientific discoveries of Dr Sylvain Latour’s laboratory and Prof. Alain Fischer. Step Pharma is based in Paris, France.