Silence Therapeutics’ Divesiran Moves Into Phase 2 for High‑Burden Polycythaemia Vera

Silence Therapeutics plc has announced the completion of enrolment in its global, randomized, double‑blind, placebo‑controlled Phase 2 trial (named SANRECO) of Divesiran for Polycythaemia Vera (PV), a blood‑cancer condition marked by excess red‑blood‑cell production. In the study, 48 phlebotomy‑dependent PV patients were enrolled, and the primary endpoint is the proportion of patients who maintain haematocrit (HCT) below 45% without needing phlebotomies between weeks 18 and 36 while receiving Divesiran versus placebo. The trial includes two dosing intervals — every 6 weeks (Q6W) and every 12 weeks (Q12W) — reflecting encouraging Phase 1 data in which Divesiran maintained HCT target levels with less frequent dosing. Divesiran is a siRNA (short interfering RNA) therapy built on Silence Therapeutics’ proprietary mRNAi GOLD™ platform, designed to silence the TMPRSS6 protein in liver cells. TMPRSS6 normally suppresses hepcidin (the body’s master regulator of iron metabolism). By silencing TMPRSS6, the therapy boosts hepcidin production, redirects iron away from the bone marrow and reduces red‑blood‑cell production — a novel mechanism in PV. PV represents a significant unmet medical need. Elevated haematocrit levels over 45% are associated with a four‑fold increased rate of cardiovascular or thrombotic events and a wide range of burdensome symptoms including fatigue, cognitive disturbance and itching. Current standard care involves repeated phlebotomies and cytoreductive agents, but no therapies are specifically approved to target red‑blood‑cell production and haematocrit in PV. According to Silence’s statement, topline Phase 2 results are expected in the third quarter of 2026. CEO Craig Tooman emphasised that the rapid completion of enrolment underscores the strong interest in Divesiran and the urgent need for better therapies in PV. He described the therapy as a potential first‑in‑class siRNA treatment for this indication. #SilenceTherapeutics #Divesiran #PolycythaemiaVera #RNAiTherapy #SlimScan #GrowthStocks #CANSLIM