Step Pharma has announced the dosing of the first participant in its Phase 1b clinical trial evaluating dencatistat for essential thrombocythaemia (ET). This trial marks a significant advancement in the development of targeted therapies for this rare blood disorder, characterized by excessive platelet production that can lead to serious complications such as thrombosis.
The current treatment landscape for ET includes therapies aimed at reducing platelet counts, but these often come with limitations and side effects. Dencatistat, a selective inhibitor of CTP synthase 1 (CTPS1), has shown promise in prior trials for lymphoma, demonstrating a reversible reduction in platelet counts with a favorable safety profile. This positions dencatistat as a potentially safer alternative for patients who are resistant to or intolerant of existing treatments.
As Step Pharma embarks on this trial, the focus will be on assessing the safety, tolerability, and preliminary efficacy of dencatistat in approximately 20 adult participants. The trial’s execution across multiple sites in France and the UK underscores the company’s commitment to advancing innovative therapies for underserved patient populations. The outcomes of this trial could significantly influence the treatment paradigm for ET, offering hope for improved patient care.
