- First presentation of clinical data from ET programme demonstrating a consistent and clinically meaningful platelet control with favourable tolerability profile
- VECTRA trial will enrol up to 50 participants with high-risk ET across sites in the US, EU and UK
Saint-Genis-Pouilly, France, 5 November 2026 – Step Pharma (“the Company”), the global leader in CTPS1 inhibition for targeted cancer treatment, today announces that an oral presentation disclosing the first preliminary data from its ongoing, open-label phase 2 VECTRA trial of dencatistat for high risk essential thrombocythaemia (ET) will be delivered at the 68th American Society of Hematology (ASH) Annual Meeting, taking place in New Orleans, Louisiana from 12–15 December 2026.
ET is a rare blood cancer characterised by the overproduction of platelets and increased risk of thrombosis that can lead to serious complications such as stroke or heart attack. The VECTRA trial is evaluating the safety, tolerability and efficacy of dencatistat, the Company’s first-in-class, highly selective, oral CTPS1 inhibitor, in up to 50 participants with high-risk ET across sites in the US, EU and UK. The trial’s primary endpoints are efficacy, safety, and tolerability. Secondary endpoints include durability of haematologic response and rates of thrombosis, haemorrhage, and disease transformation.
As of 21 July 2026, 10 out of 12 (83%) evaluable participants achieved platelet normalisation, and all achieved a platelet count of <600 ×10⁹/L, with a median maximal platelet reduction of 76% (22–86%) and no thromboembolic events occurring. A favourable tolerability profile was observed with nine participants (64%) having treatment related adverse events, predominantly anaemia and nausea. Further data will be presented at the meeting.
Oral Presentation Details:
Presentation title: A phase 2 study of dencatistat, an oral CTP synthase 1 inhibitor, for the treatment of high risk essential thrombocythemia; preliminary data from the VECTRA study.
Session: 634: Myeloproliferative Syndromes: Clinical and Epidemiological: Molecular Insights and New Approaches in MPNs
Date and time: Monday 14 December 2026, 4:00 PM to 4:15 PM CT
Presenter: Michael Loschi MD PhD
Location: Hall B, Ernest N. Morial Convention Center (MCCNO), New Orleans, Louisiana
Further details of 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.
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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