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”

ITM Isotope Technologies plans on resubmitting its advanced cancer radiopharma therapy to the FDA after addressing the agency’s concerns.

Original title: “FDA rejects ITM Isotope’s radiopharma therapy over manufacturing issues”

Sionna’s medicine didn’t have a meaningful additive impact when combined with Vertex’s top-selling Trikafta, surprising analysts and collapsing company shares.

Original title: “Sionna cystic fibrosis drug fails key trial, removing threat to Vertex”

ITM Isotope Technologies Munich said the agency cited manufacturing issues in its letter rejecting the radiopharmaceutical treatment. The FDA has rejected a radiopharmaceutical therapy because of manufacturing issues, halting what would have been a competitor for Novartis. Radiopharmaceuticals are an emerging type of treatment designed to shoot radioactive isotopes directly at tumor cells. ... ITM’s therapy was designed to treat gastroenteropancreatic neuroendocrine tumors, or GEP-NETs, a rare type of cancer that grows in the pancreas, stomach, small intestine, and other parts of the gastrointestinal system.

Original title: “In surprise decision, FDA rejects radiopharma therapy expected to rival Novartis treatment”

Definium Therapeutics’ closely watched anxiety readout could further validate its single-dose LSD candidate, while Compass Pathways advances a psilocybin program through a rolling FDA filing.

Original title: “All eyes on psychedelic drugmakers as Definium, Compass near major catalysts”

Patients treated with Tenax Therapeutics’ heart failure drug TNX-103 did not show significantly better functional performance—though Leerink analysts found biomarker improvements and subgroup analyses in elderly and more severe patients “very encouraging.”

Original title: “Tenax shares nosedive as heart failure drug fails in Phase 3”

Acalabrutinib combinations approved for CLL and MCL

Original title: “NICE backs new blood cancer regimens”

First patient enrolled in Agni-01 expansion

Original title: “Obsidian begins registration cohort for amsoki-cel”