Key Findings
Silence Therapeutics’ GalNAc-siRNA therapeutic, divesiran, developed for polycythemia vera (PV), successfully met its primary endpoint in the Phase 2 SANRECO study, demonstrating a remarkable 88% response rate compared to a mere 19% in the placebo group. Building on these promising results, the company is accelerating its transition to Phase 3 clinical trials.
Technical and Clinical Details
Divesiran is a liver-targeted GalNAc-conjugated small interfering RNA (siRNA) designed to specifically silence the expression of a gene critically involved in the pathophysiology of polycythemia vera. The Phase 2 SANRECO trial evaluated the safety and efficacy of divesiran in patients with PV. The results highlighted that 88% of patients in the divesiran arm achieved a response, a significantly higher figure compared to 19% in the placebo group. This response indicates a substantial reduction in the need for phlebotomy (therapeutic blood removal) and effective control of hematocrit levels (the proportion of red blood cells in the blood). This therapy holds the potential to alleviate the burden of disease management for patients and enhance their quality of life. A robust efficacy and favorable safety profile were confirmed throughout the trial, with the drug reported to be well-tolerated. Silence Therapeutics is now planning a Phase 3 trial involving approximately 250 patients, with a projected quarterly subcutaneous dosing regimen, and is engaging in ongoing discussions with regulatory authorities.
Background & Context
Polycythemia vera is a rare myeloproliferative neoplasm characterized by the overproduction of red blood cells due to abnormalities in bone marrow hematopoietic stem cells. This condition significantly increases the risk of thrombosis and severely impairs patients’ quality of life. Existing treatments include phlebotomy and cytoreductive agents like hydroxyurea, but these often come with side effects or issues of treatment resistance. siRNA technology, which allows for intervention at the genetic level, offers a transformative therapeutic approach for diseases involving genetic aberrations, such as PV. Silence Therapeutics is at the forefront of siRNA-based drug development, and the success of this Phase 2 trial underscores the potential for siRNA to address unmet medical needs in rare hematological disorders.
Strategic Significance & Outlook
The exceptional results from the divesiran Phase 2 trial have the potential to usher in a paradigm shift in PV treatment. If the Phase 3 trial proves successful, divesiran could become a convenient new treatment option that reduces phlebotomy frequency and maintains more stable hematocrit levels. The anticipated quarterly dosing interval could also improve patient adherence, profoundly impacting long-term disease management. This advancement is expected to open doors for the application of siRNA technology to other hematological and genetic diseases, drawing significant interest from researchers, clinicians, and investors alike.
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