Lower blood pressure target after stroke cuts cardiovascular events; FDA grants priority review to Huntington gene therapy; sevasemten fails in Becker

• Research Nov 7, 2026
  Systolic target under 120 mm Hg after stroke cuts cardiovascular events versus under 140 in 4,368-patient OPTIMAL-STROKE trial
  In the open-label OPTIMAL-STROKE trial at 30 sites in Brazil, 4,368 patients with high blood pressure and a prior ischemic stroke or transient ischemic attack were randomized to a systolic target under 120 or under 140 mm Hg. Over a median 3.9 years, the primary composite of cardiovascular death, nonfatal myocardial infarction, nonfatal stroke or hospitalization for unstable angina or heart failure occurred in 10.9% versus 13.5% (hazard ratio 0.79; 95% CI 0.67 to 0.94; P = .007). Renal outcomes were more common with intensive treatment (3.9% versus 2.2%). The trial was presented at the American Heart Association Scientific Sessions.
  Sources: New England Journal of Medicine, Healio
• FDA Nov 2, 2026
  FDA accepts uniQure's Huntington disease gene therapy application for priority review, with May 1, 2027 action date
  uniQure said the Food and Drug Administration (FDA) accepted its biologics license application for ifezuntirgene inilparvovec (AMT-130) under the accelerated approval pathway and granted priority review, with a target action date of May 1, 2027. The application compares phase 1/2 data with a propensity score-matched external control from the Enroll-HD natural history database; 51 patients have been dosed. The FDA is tentatively planning an advisory committee meeting, according to the company. The company said the therapy could become the first disease-modifying treatment for Huntington disease.
  Sources: uniQure, NeurologyLive
• Research Nov 3, 2026
  Sevasemten misses primary and secondary end points in 18-month GRAND CANYON trial in Becker muscular dystrophy, Servier says
  Servier said its 18-month, placebo-controlled GRAND CANYON trial of sevasemten in Becker muscular dystrophy showed no statistically significant benefit on the North Star Ambulatory Assessment, the primary end point, and that secondary end points also were not met. The company said the drug showed a continued favorable safety and tolerability profile and that it is analyzing the full dataset, with results to be presented later. The results are topline and not peer reviewed.
  Sources: Servier, NeurologyLive

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