On November 4, 2026 Step Pharma ("the Company"), the global leader in CTPS1 inhibition for targeted cancer treatment, reported 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) (Free ASH Whitepaper) Annual Meeting, taking place in New Orleans, Louisiana from 12–15 December 2026.
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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.
(Press release, Step Pharma, NOV 4, 2026, View Source [SID1234674249])