Silence Therapeutics’ siRNA Therapeutic Divesiran Achieves 88% Response Rate in Phase II Trial for Rare Blood Cancer Polycythemia Vera

August 23, 2026

BioXconomy UK

Overview

Silence Therapeutics announced positive interim Phase II results from its SANRECO trial for divesiran, an siRNA therapeutic targeting polycythemia vera (PV), a rare blood cancer. The trial successfully met its primary endpoint, demonstrating an 88% response rate in the divesiran arm compared to placebo. Divesiran functions by silencing the TMPRSS6 gene, showing promising efficacy in long-term disease management and addressing the underlying pathology of PV.

In Depth

Key Findings

Silence Therapeutics has reported highly encouraging interim results from its Phase II SANRECO clinical trial for divesiran, an investigational siRNA (small interfering RNA) therapeutic designed to treat polycythemia vera (PV), a rare chronic blood cancer. The trial successfully met its primary endpoint, with an impressive 88% response rate observed in patients receiving divesiran, significantly outperforming the placebo arm. This outcome underscores the substantial potential of divesiran as a novel and effective treatment option for PV.

Technical/Clinical Details

Divesiran is specifically engineered to silence the gene encoding for transmembrane protease, serine 6 (TMPRSS6). TMPRSS6 plays a critical role in regulating iron availability and erythropoiesis, the process of red blood cell production. By inhibiting the expression of this gene, divesiran effectively reduces the overproduction of red blood cells, which is the hallmark pathology of PV. The SANRECO trial’s interim data not only highlights the high response rate but also indicates a favorable safety profile, with no serious adverse events reported that were attributable to the drug. This targeted genetic approach promises to provide more effective disease control, potentially reducing the need for phlebotomy and improving quality of life for patients by mitigating symptoms such as splenomegaly and reducing thrombotic events.

Background & Context

Polycythemia vera is a myeloproliferative neoplasm characterized by an overproduction of red blood cells in the bone marrow, leading to increased blood viscosity, which elevates the risk of blood clots, strokes, and heart attacks. Current treatments, including phlebotomy and cytoreductive agents like hydroxyurea, are often associated with side effects and may not fully address the underlying mechanisms of the disease or prevent long-term complications. The emergence of siRNA therapeutics, which precisely interfere with gene expression, represents a paradigm shift in treating diseases at their genetic root. Silence Therapeutics is a leader in this field, leveraging its expertise to develop targeted therapies that can offer significant advantages over conventional treatments for unmet medical needs like PV.

Strategic Significance & Outlook

The successful Phase II results for divesiran are a crucial step forward for patients suffering from PV and for Silence Therapeutics. These promising findings are expected to accelerate the drug’s progression into Phase III clinical trials, bringing it closer to market availability. If approved, divesiran could revolutionize PV treatment, offering a highly effective, well-tolerated, and potentially disease-modifying therapy. Furthermore, this success strengthens the credibility of siRNA technology, potentially paving the way for the development of similar gene-silencing therapies for other rare hematological cancers and genetic disorders, positioning Silence Therapeutics at the forefront of this innovative therapeutic modality.

Source: https://www.bioxconomy.com/modalities/silence-therapeutics-progresses-rare-blood-cancer-sirna-with-positive-trial-results

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