Step Pharma Advances First-in-Class CTPS1 Inhibitor Dencatistat into Phase 2 Trial for Essential Thrombocythaemia
核心洞察
Step Pharma (搜索) has advanced its first-in-class oral CTPS1 (搜索) inhibitor dencatistat into a phase 2 clinical trial for essential thrombocythaemia (搜索), a rare clonal blood disorder with limited treatment options.
The phase 2 VECTRA trial will evaluate safety, tolerability, and efficacy in up to 50 high-risk ET patients across sites in the US, EU, and UK, following positive phase 1b data.
The company also announced the appointment of Dr. Neil Gallagher, a veteran oncology drug developer, as Non-Executive Director to support dencatistat's advancement across three clinical programmes.
Step Pharma (搜索), the global leader in CTPS1 (搜索) inhibition for targeted cancer treatment, has advanced its first-in-class oral CTPS1 inhibitor dencatistat into a phase 2 clinical trial for essential thrombocythaemia (搜索) (ET), the company announced on 17 June 2026. The milestone comes alongside the appointment of Neil J. Gallagher, MD, PhD, as Non-Executive Director, bringing over 25 years of haematology and oncology clinical development experience to the company's board.
The phase 2 trial, named the VECTRA trial, will evaluate the safety, tolerability, and efficacy of dencatistat in up to 50 individuals with high-risk ET across sites in the United States, European Union, and United Kingdom. The trial is registered on ClinicalTrials.gov under identifier NCT06786234.
Positive Phase 1b Data Pave the Way
The progression into phase 2 follows encouraging data from the company's phase 1b trial, which was initiated in September 2025. That study enrolled participants across all ET mutation subtypes, including JAK2, CALR, and MPL-mutated disease, as well as triple-negative ET. According to the company, this diverse population provided a clinically relevant dataset with early benefits seen across all participants.
"Treatment approaches for essential thrombocythaemia (搜索) have remained largely unchanged for more than a decade so the advancement of dencatistat into a phase 2 trial is a key milestone," said Andrew Parker, Chief Executive Officer of Step Pharma (搜索). "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."
A Pipeline in a Product Strategy
Dencatistat's journey into ET originated from an unexpected observation during Step Pharma (搜索)'s lymphoma trial, where researchers noted that continual treatment resulted in a reversible, dose-related reduction in platelet levels. This finding, successfully managed through intermittent dosing schedules in both lymphoma and solid tumour studies, combined with dencatistat's favourable safety profile, established a compelling basis to expand into ET.
The company is now advancing dencatistat across three parallel clinical programmes: ET, lymphoma, and solid tumours. Step Pharma (搜索) expects to progress toward phase 3 trial initiation in ET during 2028.
Board Strengthened with Veteran Oncology Drug Developer
In a separate announcement on 1 July 2026, Step Pharma (搜索) confirmed the appointment of Neil Gallagher as Non-Executive Director, effective immediately. Dr. Gallagher brings an extensive track record in oncology drug development, having held senior executive positions at Amgen, AstraZeneca, Novartis, and AbbVie (搜索), where as Chief Medical Officer he led the development organisation through multiple regulatory approvals in immunology, oncology, neuroscience, and eye care.
"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 (搜索) inhibitor, dencatistat," said Parker.
Dr. Gallagher's contributions to oncology include venetoclax during his tenure as CMO at AbbVie (搜索) and overseeing the FDA approvals of revumenib and axatilimab as President, Head of R&D at Syndax Pharmaceuticals. He currently serves as a Non-Executive Director at Foghorn Therapeutics and iOnctura, and as a Board Advisor to Nouscom.
"The advancement of dencatistat into three parallel programmes reflects the strength of Step Pharma (搜索)'s innovative 'pipeline in a product' strategy," said Dr. Gallagher. "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."
About CTPS1 (搜索) Inhibition
Step Pharma (搜索), founded in 2014 by Kurma Partners, the Imagine Institute, and Sygnature Discovery, is based in Saint-Genis-Pouilly, France. The company's approach targets CTPS1 (搜索), an enzyme critical for nucleotide synthesis in rapidly dividing cancer cells, with the goal of delivering highly selective, safe, and effective treatments for both blood cancers and solid tumours while leaving healthy cells unharmed.