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Step Pharma

Step Pharma
In Press Release

Step Pharma announces the appointment of Neil Gallagher as Non-Executive Director

Press Release

St. Genis-Pouilly, France, 1 July 2026

Neil Gallagher, MD, PhD, joins with over 25 years clinical development experience, having contributed to multiple commercial therapeutics across haematology and oncology

His appointment will strengthen Step Pharma’s board as it advances dencatistat across three clinical programmes for essential thrombocythaemia (ET), lymphoma and solid tumours.

Step Pharma (“the Company”), the global leader in CTPS1 inhibition for targeted cancer treatment, today announces the appointment of Neil J. Gallagher, MD, PhD, as Non-Executive Director effective immediately. He will support the Company as it continues to advance its first-in-class CTPS1 inhibitor, dencatistat, as a treatment for essential thrombocythaemia, lymphoma and solid tumours.

Neil has worked across haematology and oncology for over 25 years, having held various senior executive positions at several global pharmaceutical companies including Amgen, AstraZeneca, Novartis and AbbVie, where as Chief Medical Officer he led the development organisation through multiple regulatory approvals in immunology, oncology, neuroscience and eye care.

He currently serves as a Non-Executive Director at several public and private oncology companies, including Foghorn Therapeutics (NASDAQ: FHTX) and iOnctura, and as a Board Advisor to Nouscom. Over the course of his executive career, Dr Gallagher has built an exceptional track record of bringing innovative oncology therapies to patients, including venetoclax during his time as CMO at AbbVie and overseeing the FDA approvals of revumenib and axatilimab as President, Head of R&D at Syndax Pharmaceuticals.

Neil received his MD from Trinity College Dublin, Ireland, alongside a PhD in Cancer Biology from the University of Birmingham, UK. He is also a Member of the Royal College of Obstetricians and Gynaecologists (MRCOG).

 

Andrew Parker, Chief Executive Officer, Step Pharma, commented:

“Neil’s deep expertise in successfully leading complex development programmes through to regulatory approval across haematology and oncology will be invaluable as we continue to advance our first-in-class CTPS1 inhibitor, dencatistat, for essential thrombocythaemia, lymphoma and solid tumours. His appointment comes at a pivotal moment for the Company, and we are delighted to welcome him to the Step Pharma board.”

Neil Gallagher, newly appointed member of the board, Step Pharma, added:

“I am delighted to join the Step Pharma board at such an exciting point in the Company’s evolution. The advancement of dencatistat into three parallel programmes reflects the strength of Step Pharma’s innovative ‘pipeline in a product’ strategy. This is a truly efficient approach to clinical development that has the potential to deliver new treatment options across multiple indications where unmet need remains high. I look forward to applying my experience in haematology and oncology drug development to help make this a reality for patients.”

Contacts

Step Pharma
Andrew Parker, Chief Executive Officer
contact@step-ph.com

Media Relations
ICR Healthcare
Amber Fennell, Namrata Taak, Phillip Marriage
T. +44 (0) 20 3709 5700
steppharma@icrhealthcare.com

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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 including  Kurma Partners, V-Bio Ventures, 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.

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Step Pharma
In Press Release

Step Pharma advances dencatistat into phase 2 clinical trial for essential thrombocythaemia

Press Release

St. Genis-Pouilly, France, 17 June 2026

Step Pharma (“the Company”), the global leader in CTPS1 inhibition for targeted cancer treatment, today announces that it has advanced dencatistat, the Company’s first-in-class, highly selective, oral CTPS1 inhibitor, into a phase 2 clinical trial for essential thrombocythaemia (ET), a rare clonal blood disorder characterised by overproduction of platelets and risk of thrombosis.

The progress into phase 2 follows positive data from the Company’s phase 1b trial which was initiated in September 2025 and has enrolled participants across all ET mutation subtypes, including JAK2, CALR and MPL-mutated disease, as well as triple-negative ET. This diverse population has provided a clinically relevant dataset in ET with early benefits seen across all participants. Building on these initial encouraging results, the phase 2 trial will evaluate the safety, tolerability and efficacy of dencatistat in up to 50 individuals with high risk ET across sites in the US, EU and UK.

Andrew Parker, Chief Executive Officer, Step Pharma, commented:

“Treatment approaches for essential thrombocythaemia have remained largely unchanged for more than a decade so the advancement of dencatistat into a phase 2 trial is a key milestone. We believe dencatistat’s novel mechanism of action and encouraging safety profile positions it as a promising potential new treatment option for individuals who are resistant to or intolerant of existing therapies.”

The Company expects to progress towards phase 3 trial initiation in ET during 2028.

The ET programme originated from Step Pharma’s trial of dencatistat in lymphoma which revealed that continual treatment results in a reversible, dose-related reduction in platelet levels. This observation, which has been successfully managed through intermittent dosing schedules in both lymphoma and solid tumour, combined with dencatistat’s favourable safety profile, established a compelling basis to expand into ET.

Further details of the phase 2 clinical trial for ET, named the VECTRA trial, can be found on ClinicalTrials.gov under the identifier NCT06786234.

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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 including Kurma Partners, V-Bio Ventures, 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.

About the EIC Accelerator Programme

Part of the EU’s Horizon Europe Research and Innovation Programme, the EIC Accelerator offers support to start-ups and SMEs that have innovative, game-changing products, services or business models that could create new markets or disrupt existing ones in Europe and worldwide.

In addition, EIC-selected companies also receive coaching, mentoring, access to investors and corporates, among other opportunities.

For more information please contact:

Step Pharma
Andrew Parker, Chief Executive Officer
contact@step-ph.com

Media Relations
ICR Healthcare
Amber Fennell, Namrata Taak, Phillip Marriage
Tel. +44 (0) 20 3709 5700
steppharma@icrhealthcare.com

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Step Pharma
In Blog

Philip Beer: Why essential thrombocythaemia treatment is overdue a revolution

Philip Beer, Chief Scientific Officer at Step Pharma, reflects on the myleproliferative neoplasms field, and specifically the treatment of essential thrombocythaemia, 16 years after completing his PhD in the subject:

Plus ça change, plus c’est la même chose. These famous words (meaning ‘the more things change, the more they stay the same’) from the French critic Jean-Baptiste Alphonse Karr posed a relevant question as I returned to the field of myeloproliferative neoplasms after over a decade away. Step Pharma’s phase 1b study in essential thrombocythaemia (ET), a rare clonal blood disorder where platelet numbers are increased, opened to recruitment at the end of 2025, years after I completed my PhD with Tony Green in Cambridge studying ET and related myeloproliferative neoplasms (MPN).

My time in Tony’s lab saw a seismic shift in our understanding of MPN biology, with the discovery of activating mutations in the JAK2 gene followed closely by similar mutations in the thrombopoietic receptor (MPL) acting as key drivers for these diseases. The identification of signalling via JAK2 as the key nodal point driving MPN biology led to the rapid development of therapeutic JAK inhibitors, setting a new benchmark for bench to bedside translation. JAK inhibitors have gone on to improve outcomes, including symptom control and survival, for individuals living with myelofibrosis.

But what of ET? Advances in biology have delivered much change, including streamlined diagnosis and better prognostic tools; however, much has also stayed the same, including the treatment of ET which is essentially unchanged over the last 16 years. Unmet need remains for the significant number of individuals failing hydroxycarbamide (hydroxyurea). Whilst new therapies have entered clinical development for ET, none have yet demonstrated convincing proof of concept as a well-tolerated, easy to administer, efficacious therapy. In this regard, Step’s ET study is timely, bringing a novel mechanism of action that has already been extensively derisked in ongoing clinical trials in cancer indications. The ET study, which is recruiting individuals who are resistant to or intolerant of existing therapies, originated from Step’s study of lead asset dencatistat in lymphoma. This trial revealed a dose-dependent reduction in platelet count with continual dosing which we now understand to be mechanism-based (and which can be mitigated by intermittent dosing for individuals with lymphoma or solid tumours). This effect on platelets, along with the strong safety profile demonstrated from our studies in cancer indications, provided a clear rationale to expand into ET.

Along with updating myself on science and clinical development in the MPN space, a particular highlight of our work in ET has been re-engaging with the people who are striving to make a difference in our understanding and treatment of this condition. Many field-leading clinicians and scientists from my PhD days are still active, along with a new cohort of experts, including several contemporaries, who have made their mark on the field.

The next year will be an exciting time for Step Pharma on several fronts, including the possibility of fomenting real change in the treatment of ET.

Step Pharma
In Press Release

Step Pharma secures €2.5 million EIC grant to accelerate development of dencatistat for CTPS2 null gynaecological tumours

Press Release

St. Genis-Pouilly, France, 19 February 2026

Blended finance award also includes up to €10 million in equity investment from the EIC

Step Pharma’s lead product, dencatistat, expected to begin evaluation in phase 1b expansion cohorts for CTPS2 null ovarian and endometrial cancers later in 2026

Step Pharma (“the Company”), the global leader in CTPS1 inhibition for targeted cancer treatment, today announces that it has been awarded a blended finance grant from the European Innovation Council (EIC) under the EIC Accelerator programme. The funding consists of a €2.5 million grant and potentially up to €10 million in equity investment to support the advancement of Step Pharma’s first-in-class, highly selective, orally bioavailable CTPS1 inhibitor, dencatistat, in CTPS2 null ovarian and endometrial cancers.

All cancers are dependent on a ready supply of CTP for proliferation, the production of which is catalysed by two isoenzymes, CTPS1 and CTPS2. Loss of CTPS2 in cancer cells creates a synthetic lethal dependency on CTPS1; selective CTPS1 inhibition kills CTPS2 null cancer cells whilst sparing normal tissues. Step Pharma’s highly selective CTPS1 inhibitor, dencatistat, has the potential to deliver efficacy without toxicity.

Dencatistat is currently being evaluated in a phase 1a dose escalation study in patients with solid tumours. This will be followed by safety expansion cohorts in patients with CTPS2 null cancers, including ovarian and endometrial where the prevalence of CTPS2 loss is 15-20%. These expansion cohorts are expected to start enrolling in 2026, with an additional cohort in CTPS2 null lung cancer also planned.

Andrew Parker, Chief Executive Officer, Step Pharma, commented:

“Being selected for an EIC Accelerator award is an important validation of our precision oncology approach targeting the synthetic lethal dependency on CTPS1 in CTPS2 null solid tumours. Building on strong momentum across our three clinical programmes, this award will enable us to advance dencatistat into expansion cohorts in ovarian and endometrial cancer, gynaecological indications where initiatives such as the EIC Accelerator can play a key role in improving outcomes. We look forward to working with the EIC as we continue to drive a step change in the way cancer is treated.”

The EIC Accelerator award follows a period of significant clinical and corporate progress for Step Pharma, including the completion of a €38 million Series C financing in October 2025 led by V-Bio Ventures, the appointment of Dr Karen Smith as Chief Medical Officer, and the initiation of the Company’s third clinical programme for dencatistat in essential thrombocythaemia (ET). Dencatistat is also being evaluated in a phase 1/2 clinical trial for adult patients with relapsed/refractory T or B cell lymphoma, and received US FDA orphan drug designation for the treatment of T cell lymphoma in May 2025.

The EIC Accelerator supports breakthrough innovations with the potential to create new markets or disrupt existing ones, through combined grant and equity investments. Step Pharma’s blended finance award will fund pivotal activities including:

  • Expansion of dencatistat phase 1b trial into CTPS2 null ovarian and endometrial cancer cohorts; and
  • Preparation for phase 2 studies in gynaecological cancers including drug manufacture and pivotal toxicology studies.

The support also includes access to EIC Business Acceleration Services, providing strategic guidance, networking opportunities, and investor connections.

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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 including Kurma Partners, V-Bio Ventures, 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.

About the EIC Accelerator Programme

Part of the EU’s Horizon Europe Research and Innovation Programme, the EIC Accelerator offers support to start-ups and SMEs that have innovative, game-changing products, services or business models that could create new markets or disrupt existing ones in Europe and worldwide.

In addition, EIC-selected companies also receive coaching, mentoring, access to investors and corporates, among other opportunities.

For more information please contact:

Step Pharma
Andrew Parker, Chief Executive Officer
contact@step-ph.com

Media Relations
ICR Healthcare
Amber Fennell, Namrata Taak, Phillip Marriage
Tel. +44 (0) 20 3709 5700
steppharma@icrhealthcare.com

+ LEARN MORE
Step Pharma
In Press Release

Step Pharma announces the appointment of Dr Karen Smith as Chief Medical Officer

Press Release

St. Genis-Pouilly, France, 28 October 2025

Karen Smith, MD, PhD, MBA, LLM, joins with over 25 years of global pharma and biotech experience, having contributed to more than 20 regulatory approvals

Dr Smith’s appointment will accelerate Step Pharma’s ‘pipeline in a product’ strategy, leveraging her clinical and regulatory expertise to advance dencatistat, a
first-in-class CTPS1 inhibitor across multiple cancer and blood disorder indications

Appointment follows Step Pharma’s recently announced €38 million Series C financing

Step Pharma (“the Company”), the global leader in CTPS1 inhibition for targeted cancer treatment, today announces the appointment of Karen L. Smith, MD, PhD, MBA, LLM as its Chief Medical Officer (CMO) effective immediately.

Dr Smith brings over 25 years of healthcare industry experience, having held various senior executive positions across global pharmaceutical and biotechnology companies including Bristol Myers Squibb, AstraZeneca and Merck. She brings exceptional expertise in clinical development, regulatory affairs and medical leadership. She has successfully overseen more than 100 clinical trials and 20 regulatory approvals across multiple therapeutic areas including oncology, cardiology, dermatology, rare diseases and neuroscience, leading to global launches of small molecules, biologics and devices.

Dr Smith is currently a board member of several public and private biotech companies including Skye Bioscience (NASDAQ: SKYE), and Context Therapeutics (NASDAQ: CNTX).  In her previous board roles, Dr Smith helped deliver significant value through seven corporate exits for pre-clinical, clinical-stage, and commercial businesses totalling almost US $20 billion, including most recently, Capstan Tx (acquired by AbbVie in 2025 for $2.1 billion) and Mariana Oncology (acquired by Novartis in 2024 for $1.75 billion).

She received her MD from the University of Warwick, UK, a PhD in oncology from UCLA/University of Western Australia, as well as an MBA from the University of New England, Australia, and an LLM from the University of Salford, UK.

Andrew Parker, Chief Executive Officer, Step Pharma, commented:

“Karen’s passion for innovative medicine and her experience in leading clinical programmes will help to accelerate our mission to transform cancer and blood disorder treatment through our unique approach to CTPS1 inhibition. Her appointment as Chief Medical Officer, combined with our recently completed Series C financing, leaves us strongly positioned to execute our clinical development plans for dencatistat across multiple indications and ultimately deliver improved outcomes for patients.”

Dr Karen Smith, newly appointed Chief Medical Officer, Step Pharma, added:

“I am honoured to join Step Pharma at this point in the Company’s evolution, and excited by the opportunity to advance dencatistat, a first-in-class CTPS1 inhibitor with promising therapeutic potential across multiple indications. Step Pharma’s innovative ‘pipeline in a product’ strategy represents an efficient approach to clinical development that could provide new treatment options to a huge number of patients, and I look forward to applying my clinical development and regulatory experience to help bring this transformative therapy to patients.”

Contacts

Step Pharma
Andrew Parker, Chief Executive Officer
contact@step-ph.com

Media Relations
ICR Healthcare
Amber Fennell, Namrata Taak, Phillip Marriage
T. +44 (0) 20 3709 5700
steppharma@icrhealthcare.com

+ DOWNLOAD PDF

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 including  Kurma Partners, V-Bio Ventures, 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.

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Step Pharma
In Press Release

Step Pharma to spotlight work in CTPS2-null solid tumours at 2025 EORTC-NCI-AACR International Conference on Molecular Targets and Cancer Therapeutics

Press Release

St. Genis-Pouilly, France, 21 October 2025

Session: AACR Project GENIE: Powering Drug Discovery Through an International Consortium’

Presentation title: Using GENIE data to drive oncology target identification and drug development.

Thursday 23 October 2025, 17:55 EDT

Step Pharma (“the Company”), the global leader in CTPS1 inhibition for targeted cancer treatment, today announces that its Chief Scientific Officer, Philip Beer, will be presenting data on the identification of a CTPS1-dependent synthetic lethality in solid tumours at the 2025 EORTC-NCI-AACR International Conference on Molecular Targets and Cancer Therapeutics, being held from 22-26 October in Boston, MA.

The presentation will focus on the use of the AACR Project GENIE data, a publicly accessible cancer registry of real-world clinico-genomic data, to identify loss of CTPS2 as a novel biomarker to select solid tumours with a synthetic lethal dependency on CTPS1. Deletion of CTPS2 was subsequently identified as a frequent event in many cancer types, with the highest prevalence found in ovarian cancer. As tumours derived from epithelial tissues can utilise both CTPS1 and CTPS2 in the final step of the CTP synthesis cascade, loss of CTPS2 in solid tumours exposes a dependence which could be targeted with CTPS1 inhibition to prevent tumour cell proliferation.

As a result of these findings, dencatistat, an orally available and highly potent inhibitor of CTPS1, which is currently being evaluated in a phase 1a dose escalation study in patients with solid tumours, will be evaluated in safety expansion cohorts in patients with CTPS2 null cancers, with the first being ovarian cancer (NCT06297525). Selecting patients whose tumours have deleted CTPS2 represents a precision oncology approach that is expected to maximise the therapeutic potential of dencatistat.

Philip Beer, Chief Scientific Officer of Step Pharma, said:

“Our work analysing clinico-genomic data led us to identify patients with CTPS2 null solid tumours as a promising therapeutic application for dencatistat. About 15-20% of ovarian cancers harbour this deletion, and so through inhibition of CTPS1 with dencatistat, we can selectively target the de novo pyrimidine synthesis pathway in ovarian cancer and other solid tumours. I look forward to presenting at the EORTC-NCI-AACR international conference and to initiating the first expansion cohort in the near future.”

Session: AACR Project GENIE: Powering Drug Discovery Through an International Consortium’

Presentation title: Using GENIE data to drive oncology target identification and drug development.

Date: Thursday 23 October 2025

Time: 17:55 EDT

Contacts

Step Pharma
Andrew Parker, Chief Executive Officer
contact@step-ph.com

Media Relations
ICR Healthcare
Amber Fennell, Namrata Taak, Phillip Marriage
T. +44 (0) 20 3709 5700
steppharma@icrhealthcare.com

+ DOWNLOAD PDF

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 including Kurma Partners, V-Bio Ventures, 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.

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Step Pharma
In Press Release

Step Pharma announces completion of €38 million Series C financing

Press Release

St. Genis-Pouilly, France, 15 October 2025

Financing will support the Company’s ongoing clinical development of its lead asset, dencatistat, across multiple indications

Round led by new investor V-Bio Ventures, with participation from existing investors Pontifax, Bpifrance, Kurma Partners, Hadean Ventures, Sunstone Life Science Ventures and Inserm Transfert Initiative

Financing follows recent progress of dencatistat into its third clinical programme, in essential thrombocythaemia, following lymphoma and solid tumours

Ward Capoen, Partner, V-Bio Ventures joins Step Pharma’s Board of Directors

Step Pharma (“the Company”), the global leader in CTPS1 inhibition for targeted cancer treatment, today announces the completion of a €38 million Series C financing round.

The Series C round was led by new investor V-Bio Ventures, a Ghent-based life sciences venture capital fund, with participation from existing investors Pontifax, Bpifrance, Kurma Partners, Hadean Ventures, Sunstone Life Science Ventures and Inserm Transfert Initiative.

The financing will enable Step Pharma to continue advancing its ‘pipeline in a product’ strategy for dencatistat, a first-in-class, highly selective, orally bioavailable CTPS1 inhibitor currently in development for the treatment of cancers and blood disorders. All cancers appear to be highly reliant on CTPS1, a key component of the pyrimidine synthesis pathway, for DNA synthesis and cell proliferation.

Dencatistat is being evaluated in a phase 1/2 clinical trial for adult patients with relapsed/refractory T or B cell lymphoma, and received orphan drug designation for the treatment of T cell lymphoma in May 2025. The Company is also evaluating dencatistat in a phase 1 trial in solid tumour patients, with expansion cohorts in CTPS2-null ovarian, endometrial and lung cancer planned.

Clinical trials of dencatistat in lymphoma demonstrated that continual administration results in a dose dependent and reversible lowering of the platelet count. This led to the initiation of Step Pharma’s third clinical programme for dencatistat, in essential thrombocythaemia, a rare clonal blood disorder in which the bone marrow produces too many platelets.

Andrew Parker, Chief Executive Officer, Step Pharma, commented:

“This Series C financing will allow us to build upon the significant clinical progress we have made with dencatistat, with three clinical programmes underpinning our ‘pipeline in a product’ strategy. These funds will enable us to expand our clinical dataset, complete all ongoing phase 1 studies, and further derisk dencatistat as we enter phase 2 and explore its potential to improve patient outcomes through CTPS1 inhibition. I’d like to thank V-Bio Ventures for joining our investor syndicate and leading this round, and our existing investors for their continued participation and support.”

Ward Capoen, Partner, V-Bio Ventures, said:

“We are delighted to support Step Pharma in its next phase of growth through this Series C financing. The Company’s approach to inhibiting CTPS1 to selectively block the proliferation of cancer cells is highly promising, and the team has followed the data into essential thrombocythaemia after observing dencatistat’s effect on platelet levels. This multi-pronged clinical plan has further derisked dencatistat and positions Step Pharma to provide new therapeutic options for patients.”

Contacts

Step Pharma
Andrew Parker, Chief Executive Officer
contact@step-ph.com

Media Relations
ICR Healthcare
Amber Fennell, Namrata Taak, Phillip Marriage
T. +44 (0) 20 3709 5700
steppharma@icrhealthcare.com

+ DOWNLOAD PDF

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 including Kurma Partners, V-Bio Ventures, 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.

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Step Pharma
In Press Release

Step Pharma announces first participant dosed in a phase 1b clinical trial of dencatistat for essential thrombocythaemia

Press Release

St. Genis-Pouilly, France, 2 September 2025

Step Pharma (“the Company”), the global leader in CTPS1 inhibition for targeted cancer treatment, today announced that the first participant has been dosed with its lead asset dencatistat in a phase 1b clinical trial for essential thrombocythaemia (ET).

ET is a rare clonal blood disorder in which the bone marrow produces too many platelets, increasing the risk of blood clots that can lead to serious thrombotic complications such as stroke or heart attack. Current treatments aim to lower platelet counts in order to reduce the risk of complications, but they can be associated with side effects or may not be suitable for all individuals, highlighting the need for more targeted therapies with improved safety profiles.

Clinical trials of dencatistat in lymphoma have demonstrated that continual administration of dencatistat results in a dose dependent and reversible lowering of the platelet count. This finding (which has been mitigated by intermittent dosing for patients with lymphoma or solid tumours), along with the excellent tolerability profile of the drug, provides a strong rationale for investigating dencatistat as a treatment for ET.

Andrew Parker, Chief Executive Officer of Step Pharma, commented: “Initiating the clinical evaluation of dencatistat in essential thrombocythaemia represents a significant milestone in our ‘pipeline in a product’ strategy. We believe we can harness selective CTPS1 inhibition to provide a safer, targeted approach to the treatment of ET. This expansion into a new indication, alongside our ongoing trials in lymphoma and solid tumours, further demonstrates the   potential versatility of dencatistat as a targeted therapy. We look forward to collaborating with clinical investigators and those living with ET to advance this important programme.”

The phase 1b open-label trial will evaluate the safety, tolerability, and preliminary activity of dencatistat in approximately 20 adult participants with high-risk ET who are resistant to, or intolerant of, hydroxycarbamide (hydroxyurea) therapy. The trial is being conducted at 12 sites across France and the UK.

Further details of the phase 1b trial for ET can be found on clinicaltrials.gov under the identifier NCT06786234.

Contacts

Step Pharma
Andrew Parker, Chief Executive Officer
contact@step-ph.com

Media Relations
ICR Healthcare
Amber Fennell, Namrata Taak, Phillip Marriage
T. +44 (0) 20 3709 5700
steppharma@icrhealthcare.com

+ DOWNLOAD PDF

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.

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Step Pharma
In Press Release

Step Pharma receives US FDA orphan drug designation for dencatistat in T-Cell lymphoma

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

+ DOWNLOAD PDF

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.

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Step Pharma
In Press Release

Step Pharma announces first patient dosed in a phase 1 trial of dencatistat for patients with solid tumours

Press Release

St. Genis-Pouilly, France, 3 September 2024

Step Pharma, the world leader in CTPS1 inhibition for the targeted treatment of cancer, today announces 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.

Step Pharma (“the Company”), the global leader in CTPS1 inhibition for targeted cancer treatment, today announced that the first patient has been dosed at Next Oncology, San Antonio, TX USA, with its lead asset dencatistat (STP938) in a clinical trial for patients with solid tumours.

This open label trial is evaluating the safety, tolerability and pharmacokinetics of dencatistat, a first-in-class, highly selective, orally bioavailable CTPS1 inhibitor. The trial consists of two parts: a phase 1a dose escalation recruiting patients with solid tumours, followed by a phase 1b expansion cohort specifically for patients with CTPS2 null ovarian cancer, planned to start in Q1 2025.

Approximately 325,000 women worldwide are diagnosed with ovarian cancer each year. 15-20% of ovarian cancers harbour a deletion of the gene encoding CTPS2. Selecting patients whose tumours have deleted CTPS2 represents a precision oncology approach that is expected to maximise the therapeutic potential of dencatistat. The trial is recruiting patients with advanced cancer who have no other treatment options available.

Andrew Parker, Chief Executive Officer of Step Pharma, commented:

“The start of our first clinical trial of dencatistat for solid tumours marks a significant milestone in our ‘pipeline in a product’ strategy. Dencatistat, our first-in-class CTPS1 inhibitor, is now expanding from blood cancers to solid tumours, demonstrating its versatility. With a substantial number of ovarian cancer patients potentially harbouring CTPS2 deletions, and similar deletions observed in other cancer types, we are excited about the potential of dencatistat as a tumour-agnostic targeted treatment.”

Furthermore, the Company announces that the Clinical Trial Application for the phase 1 trial of dencatistat in patients with solid tumours has been approved by the UK Medicines and Healthcare products Regulatory Agency (MHRA). This approval, along with the previously cleared Investigational New Drug (IND) application to the US Food and Drug Administration (FDA), announced in June 2024, enables the Company to progress dencatistat into clinical trials in both the UK and the US.

The start of this solid tumour trial follows the ongoing phase 1/2 trial of dencatistat for adult patients with relapsed/refractory T or B cell lymphoma, which commenced in October 2022.

Step Pharma is pioneering a novel class of oral drugs that specifically inhibit nucleotide synthesis and the enzyme CTPS1 in particular, which was originally identified as an essential gene for lymphocyte proliferation. By targeting CTPS1, Step Pharma has unlocked the ability to selectively target the de novo pyrimidine synthesis pathway in cancer cells. This groundbreaking approach is predicted to enable the highly selective treatment of both blood cancers and solid tumours.

Further details of the planned Phase 1 trial can be found on clinicaltrials.gov under the identifier NCT06297525.

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

+ DOWNLOAD PDF

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.

+ LEARN MORE