The approval of rusfertide—now to be branded Mimrylo—for polycythemia vera marks a key win for Takeda, which now has crossed the FDA finish line for two of three critical product launches slated for the coming year.

Original title: “Takeda, Protagonist score broad FDA nod in rare blood cancer, setting up $2B opportunity”

Earlier this month, biotechnology ... RNA (siRNA ) therapies, announced positive initial results from its Phase II clinical trial, SANRECO (NCT05499013). The trial was evaluating the efficacy and safety of divesiran, a siRNA drug for the treatment of the rare blood cancer ... Earlier this month, biotechnology company Silence Therapeutics, a specialist in small interfering RNA (siRNA ) therapies, announced positive initial results from its Phase II clinical trial, SANRECO (NCT05499013). The trial was evaluating the efficacy and safety of divesiran, a siRNA drug for the treatment of the rare blood cancer polycythemia vera (PV). Silence Therapeutics recently announced positive Phase II trial results for its siRNA drug. However, Silence appears to have its sights set on the next phase of clinical trials. “Because of how siRNA works, the effect of divesiran is long-lasting; we are planning to move forward with quarterly dosing into Phase III,” disclosed Rambaran. “Based on what we’ve seen so far, divesiran has the potential to become an attractive, best in class option for a broad range of PV patients who are currently relying on frequent phlebotomies to control their disease.” · Related:mAb combo therapy misses the mark in Phase III clinical trial for advanced colorectal cancer PV is a rare slow-growing form of blood cancer, which can increase the viscosity of the blood by producing too many red blood cells. patients with PV are at risk of life-threatening blood clots if blood is not routinely removed via phlebotomy. “Data from large clinical studies have shown that keeping hematocrit [the percentage of red blood cells] under 45% significantly reduces the risk of major thrombotic events,” elaborated Rambaran.

Original title: “Silence Therapeutics progresses blood cancer siRNA therapy”

The terms of the agreement with Royalty Pharma suggest the market potential of Zealand Pharma’s polycythemia vera drug candidate may be undervalued, according to Jefferies analysts.

Original title: “Zealand sells milestone, royalty rights to rare blood cancer asset for up to $100M”

Some analysts believe Silence’s polycythemia vera drug could be superior to a closely watched Takeda medicine. Elsewhere, the FDA spurned a radiopharmaceutical and AbCellera shares climbed to levels not seen since 2023.

Original title: “Silence cashes in on ‘differentiated’ data; UK reaffirms support for childhood vaccines”

The phase 2 SANRECO trial met its primary end point, with 88% of this polycythemia vera population achieving a response with divesiran vs 19% with placebo. The phase 2 SANRECO trial met its primary end point, with 88% of this polycythemia vera population achieving a response with divesiran vs 19% with placebo. ... Divesiran, a first-in-class small interfering RNA (siRNA) therapy, met the primary end point of the phase 2 SANRECO trial (NCT05499013) in patients with polycythemia vera (PV), according to a news release from Silence Therapeutics.¹ Silence Therapeutics announces positive topline results from phase 2 SANRECO trial of divesiran in polycythemia vera, supporting its potential best-in-class profile. News release. Silence Therapeutics plc. August 10, 2026. Accessed August 11, 2026. https://tinyurl.com/yz5ypmz2 · Study to assess SLN124 in patients with polycythemia vera (SLN). ClinicalTrials.gov. The company plans to initiate a phase 3 trial evaluating divesiran vs placebo in the first half of 2027. “Across the SANRECO phase 1/2 program, divesiran has been well tolerated and has consistently delivered durable hematocrit control in patients [dependent on phlebotomy] with PV, regardless of risk level or disease severity,” said Marina Kremyanskaya, MD, PhD, associate professor of medicine, hematology and medical oncology, at the Icahn School of Medicine at Mount Sinai, in the press release.¹ “These compelling results highlight divesiran’s potential to transform PV management with convenient, infrequent dosing that reliably controls hematocrit and addresses longstanding unmet needs for patients.” The trial assessed divesiran at 6 mg/kg administered subcutaneously every 6 weeks or every 12 weeks in patients with uncontrolled hematocrit who remained phlebotomy dependent despite standard-of-care treatment, which could include hydroxyurea, interferon, and/or ruxolitinib (Jakafi).2 All patients have completed the placebo-controlled portion of the trial and are now in the 3-year double-blind and open-label extension periods.

Original title: “Divesiran Shows Clinical Responses in Phase 2 Polycythemia Vera Trial | CancerNetwork”

The mid-stage win of Silence Therapeutics’ siRNA drug in polycythemia vera validates the therapeutic approach of similar assets in development—but also sets a high standard for therapies from Takeda, Agios and Disc Medicine.

Original title: “Silence causes uproar in rare blood cancer space as data sets high bar for competitors”

progress in the study of polycythemia vera in children

Original title: “[Progress in the study of polycythemia vera in children].”

araf mutated langerhans cell histiocytosis developed on the background of araf jak2 mutated polycythaemia vera evidence of branching evolution from the same progenitor cell a case report and literature review

Original title: “ARAF mutated langerhans cell histiocytosis developed on the background of ARAF/JAK2 mutated polycythaemia vera: evidence of branching evolution from the same progenitor cell. A case report and literature review.”

burden of thromboembolism disease progression and phlebotomy among polycythemia vera patients in the united states a systematic literature review

Original title: “Burden of thromboembolism, disease progression, and phlebotomy among polycythemia vera patients in the United States: a systematic literature review.”

neutrophil to lymphocyte ratio half reduction as a surrogate of molecular response in polycythemia vera treated with ropeginterferon alfa 2b

Original title: “Neutrophil-to-lymphocyte ratio half reduction as a surrogate of molecular response in polycythemia vera treated with ropeginterferon alfa-2b.”

ruxolitinib treatment in patients with polycythemia vera reduces jak2 variant allele frequency and improves symptom burden and hematocrit control

Original title: “Ruxolitinib treatment in patients with polycythemia vera reduces JAK2 variant allele frequency and improves symptom burden and hematocrit control.”

Prithviraj Bose, MD, reviews the case of a 57-year-old man diagnosed with polycythemia vera. Thank you for joining us for this Targeted Oncology™ Virtual Tumor Board® focused on myeloproliferative neoplasms [MPNs]. In today’s presentation, my colleagues and I will review 3 clinical cases. We will discuss an individualized approach to treatment for each patient and review key clinical trial data that impacts our decisions. Ruben Mesa, MD: You raised some great points. It was recently Rare Disease Day. I tell my patients with PV that “rare” is in the eyes of the beholder. If you have a disease, it’s not rare. You’ve got it, so it’s clearly a problem for you. But as far as rare diseases go, I tell them that it’s somewhere in the middle. It’s not rare, but not common. This is clearly not the common cold, breast cancer, or prostate cancer, but there are also many incredibly obscure diseases people can have. There are roughly 150,000 patients in the United States. I definitely agree with you that there are probably a lot of more subtle presentations—perhaps not as extreme—because people are iron deficient. Prithviraj Bose, MD: Sure, Ruben. Thank you. The case I’m presenting is a case of polycythemia vera [PV]. We’ll walk you through the initial presentation and the disease course of this patient, with a focus on hydroxyurea resistance that eventually develops.

Original title: “Case 1: Hydroxyurea-Resistant Polycythemia Vera (P. Vera) | Targeted Oncology - Immunotherapy, Biomarkers, and Cancer Pathways”

Conditions: Polycythemia Vera; ET (Essential Thrombocythemia); Polycythemia Vera (PV); Essential Thrombocythemia (ET); Primary Myelofibrosis (MF); Primary Myelofibrosis (PMF); Myelofibrosis; Myelofibrosis (MF); Myelofibrosis, Primary; Myelofibrosis, Post ET; Myelofibrosis, Post PV; Myelofibrosis (PMF); Myelofibrosis，MF; Myelofibrosis; Primary Myelofibrosis; Post-polycythemia Vera Myelofibrosis; Post-essential Thrombocythemia Myelofibrosis; Myelofibrosis Due to and Following Polycythemia Vera; My

Original title: “MPN PROGRESSion Registry: Observational Study Tracking Symptoms, Treatments, and Disease Progression in People With Myeloproliferative Neoplasms (MPNs)”