Press Release
St. Genis-Pouilly, France, 22 May 2025
Step Pharma (“the Company”), the global leader in CTPS1 inhibition for targeted cancer treatment, today announced that the US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to its lead candidate, dencatistat, for the treatment of T cell lymphoma, encompassing all subtypes including peripheral T cell lymphoma and cutaneous T cell lymphoma.
The designation highlights the significant unmet medical need in T cell lymphoma, a rare and aggressive group of blood cancers. It also recognises the potential of dencatistat as a novel, selective therapeutic option. Orphan drug designation provides incentives designed to support the development of drugs for rare diseases, including market exclusivity upon approval, tax credits for clinical trial costs, and waiver of certain FDA fees.
Andrew Parker, Chief Executive Officer of Step Pharma, commented:
“Receiving orphan drug designation from the FDA, which may confer seven years of market exclusivity for dencatistat, marks an important milestone in our mission to deliver highly selective cancer therapies for patients with difficult-to-treat cancers. T cell lymphoma remains a challenging disease with limited treatment options, and we believe CTPS1 inhibition represents a new therapeutic approach with the potential to meaningfully improve outcomes.”
Dencatistat is being evaluated in a phase 1/2 clinical trial for adult patients with relapsed/refractory T or B cell lymphoma. The Company is also evaluating dencatistat in a phase 1 trial in solid tumour patients, with expansion cohorts in CTPS2-null ovarian and lung cancer planned. Additionally, a phase 1b trial in essential thrombocythaemia is expected to commence in the first half of 2025, further supporting dencatistat’s potential as a targeted therapy across multiple cancer types.
Contacts
Step Pharma
Andrew Parker, Chief Executive Officer
contact@step-ph.com
Media Relations
Consilium Strategic Communications
Amber Fennell, Namrata Taak, Davide Salvi
T. +44 (0) 20 3709 5700
steppharma@icrhealthcare.com
About dencatistat
Dencatistat (STP938) is a first-in-class, highly selective, orally bioavailable inhibitor of CTP synthase 1 (CTPS1), a key component of the pyrimidine synthesis pathway. CTPS1 inhibition blocks the proliferation of cancer cells and results in cell death. All cancers appear to be addicted to CTPS1 for DNA synthesis. Dencatistat entered clinical development in October 2022 for the treatment of T cell and B cell lymphoma and in September 2024 for the treatment of solid tumours.
Dencatistat has the potential to become the backbone of treatment regimens for a broad range of haematological and solid tumours, as well as being a potent monotherapy for hard-to-treat blood cancers.
About Step Pharma
Step Pharma’s goal is to bring about a step change in how cancer is treated with targeted therapies that kill cancer cells and leave healthy cells unharmed. The Company is the world leader in CTPS1 inhibition, a new approach with the potential to yield highly selective, safe and effective treatments for both blood cancers and solid tumours.
Step Pharma was founded in 2014 by Kurma Partners, the Imagine Institute and Sygnature Discovery, based on the scientific discoveries of Prof. Alain Fischer and Dr Sylvain Latour. Step Pharma is based in Saint-Genis-Pouilly, France, and is supported by a strong syndicate of investors led by Kurma Partners and including Bpifrance (Fonds Biothérapies Innovantes et Maladies Rares and InnoBio2 Fund), Pontifax, Hadean Ventures, Sunstone Life Science Ventures, Inserm Transfert Initiative, Idinvest, Sygnature Discovery and the Imagine Institute. More information on the company can be found at www.step-ph.com.
