ASH 2024 | Initial results of the SANRECO trial evaluating divesiran in patients with polycythemia vera

The SANRECO trial was a first trial in polycythemia vera using something called small interfering RNA. And it’s basically used to knock down the negative regulator of hepcidin production. So the point is to increase endogenous hepcidin production. And by increasing hepcidin, we restrict the amount of iron that is available for erythropoiesis in the bone marrow, which results in decreased red blood cell production. Previous study with the drug called rusfertide has shown that by increasing hepcidin, in that case using the hepcidin mimetic rusfertide, you can control hematocrit in patients with PV and they no longer need phlebotomies or at least need much less phlebotomies and therefore control their disease, potentially also improve their symptoms. So this is a different approach to endogenously increase hepcidin. 

And these were results from a Phase I studies, so early results. And it showed that this approach seemed to work. These patients were enrolled in different dosing cohorts, but for all dosing cohorts, the number of phlebotomies that the patient needed went down significantly, and especially for those patients that had hematocrits that were well controlled at baseline, they really didn’t need any more phlebotomies. Importantly for Phase I study, the drug was very well tolerated. The most significant side effect was injection site reaction that was seen most frequently in patients, but it was self-limited, patients really did not need any treatment to treat the injection site reaction. Most of the adverse events were Grade 1.

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