Allogeneic human induced pluripotent stem cell-derived cardiomyocytes injected into the myocardium during CABG met the safety primary endpoint in the HEAL-CHF trial (n = 20): no sustained ventricular tachycardia in the 1- to 6-month window and no tumorigenicity at 12 months. All 10 cell-treated patients developed accelerated idioventricular rhythm within 4 weeks, and 2 had ventricular tachycardia over 140 bpm that resolved after cardioversion. Cell therapy improved 6-min walk distance, myocardial perfusion and relative wall thickening beyond CABG alone, but not ejection fraction, ventricular volumes, scar size, NYHA class or quality-of-life scores.
A prespecified secondary analysis of the phase 3 VESALIUS-CV trial found that Repatha (evolocumab) added to standard statins or other LDL-C-lowering therapy reduced the risk of all-cause death by a significant 20% in more than 12,000 high-risk adults with no history of myocardial infarction or stroke. Amgen reports this makes Repatha the first and only PCSK9 inhibitor shown in a phase 3 trial to reduce all-cause death, including cardiovascular death, in high-risk primary prevention patients.
Pulmonary artery denervation (PADN) added to guideline-directed medical therapy (GDMT) reduced the risk of clinical worsening by 51% (HR 0.49, 95% CI 0.30-0.82; P=0.006) in patients with pulmonary hypertension due to left heart disease (PH-LHD). In the 264-patient, 25-center randomized PADN-HF-PH trial, presented at ESC 2026 and published in the New England Journal of Medicine, the combination also cut the annualized recurrence of clinical worsening events by 35.5% and lowered the risk of exercise-capacity decline by 63%, with a net 24.8-meter gain in six-minute walk distance. It is the first device randomized trial to show a positive primary endpoint in PH-LHD.
The anti-ANGPTL3 fully human monoclonal antibody SHR-1918 (ruisiruiximab) from Hengrui Medicine cut LDL-C by 64.2% relative to placebo in a nationwide multicenter phase 3 trial in patients with homozygous familial hypercholesterolemia. Led by Professor Daoquan Peng of the Second Xiangya Hospital of Central South University, the study was selected for the LBA session and presented orally at the ESC congress in Munich, held August 28 to 31, 2026.
Novartis reported that two phase 3 trials of Rhapsido in relapsing multiple sclerosis met their goals, with no liver safety signal in either. The BTK inhibitor class has been dogged by liver safety concerns, and Rhapsido's clean readout suggests that barrier may not apply here. The company positions the result as a class first.
Adding atezolizumab to neoadjuvant chemotherapy did not improve event-free survival in the phase 3 NSABP B-59/GeparDouze trial of patients with early-stage triple-negative breast cancer, missing its primary endpoint. Translational analyses identified tumor microenvironment-dependent determinants of response, indicating basal-like immune-activated tumors may respond to atezolizumab plus chemotherapy.
Why it matters: Negative phase 3 result for atezolizumab in early triple-negative breast cancer, with biomarker data indicating which tumors may still benefit.
Eplontersen, an antisense oligonucleotide that reduces hepatic transthyretin production, did not reduce cardiovascular death or recurrent cardiovascular events in transthyretin amyloid cardiomyopathy. In the phase 3 CARDIO-TTRansform trial, 1432 patients received subcutaneous eplontersen 45 mg or placebo every 4 weeks for 140 weeks; the primary composite occurred at a rate ratio of 0.89 (95% CI, 0.73 to 1.09; P = 0.28). The finding leaves no demonstrated outcome benefit for eplontersen on top of standard treatment in ATTR-CM.
Rezpegaldesleukin, an interleukin-2 receptor agonist that expands regulatory T cells, met its primary endpoint in adults with moderate-to-severe atopic dermatitis naive to biological treatment. At week 16, mean percentage change in EASI was -61% for 24 μg/kg every 2 weeks, -58% for 18 μg/kg every 2 weeks and -53% for 24 μg/kg every 4 weeks, versus -31% for placebo, with treatment differences of -30, -27 and -22 percentage points respectively (p<0·0001, p<0·0001, p=0·0002). Injection-site reactions occurred in 70% of rezpegaldesleukin-treated patients but were nearly all mild to moderate and resolved, with no deaths reported.
Belzutifan plus lenvatinib extended progression-free survival over cabozantinib in previously treated advanced clear-cell renal cell carcinoma. In the phase 3 LITESPARK-011 trial, 747 patients with disease progression after anti-PD-1 or anti-PD-L1 therapy were randomly assigned to belzutifan-lenvatinib or cabozantinib. Progression-free survival by masked independent central review was significantly longer with belzutifan-lenvatinib, while overall survival did not differ significantly, and the authors suggest belzutifan-lenvatinib may become a new standard of care in this setting.
In an open-label, dose-escalation phase 1b clinical trial, autologous peptide-coupled red blood cells (pcRBCs) loaded with seven immunodominant myelin peptides were safe and well tolerated. Single-cell RNA sequencing three months after a single administration showed expansion of antigen-specific CD4+ memory regulatory T cells, fewer proinflammatory cells, and reduced neurofilament light chain levels, indicating less brain tissue inflammation.
Why it matters: A phase 1 trial reporting both safety and single-cell transcriptomic mechanistic readouts can serve as a mechanistic control for later dose and indication expansion.
Oral anticoagulation with a factor Xa inhibitor after transcatheter aortic valve implant reduced leaflet thrombosis compared with acetylsalicylic acid, in a randomized trial of 360 patients aged 65 to 80 with severe aortic valve stenosis. HALT on 12-month 4-dimensional CT occurred in 16.2% of the NOAC group vs 28.6% of the ASA group (risk ratio 0.55; 95% CI 0.37-0.82). The composite safety endpoint of VARC-3 bleeding, thromboembolic events, and all-cause death met noninferiority (7.5% vs 10.6%).
Switching virologically suppressed adults with HIV-1 to a once-weekly, single-tablet regimen of islatravir (2 mg)-lenacapavir (300 mg) was non-inferior to continuing daily oral standard of care in the phase 3 ISLEND-2 trial. At week 48, HIV-1 RNA viral load of 50 copies per mL or higher occurred in 1 of 314 participants on islatravir-lenacapavir versus 4 of 312 on standard of care, meeting the non-inferiority margin of 4%. Treatment-related adverse events occurred in 18% of the islatravir-lenacapavir group versus fewer than 1% of the standard-of-care group, though discontinuations due to adverse events were rare in both groups.
Adding individualized low-voltage zone ablation to pulmonary vein isolation substantially improved rhythm outcomes in persistent atrial fibrillation patients with low-voltage zones of 3.0 cm2 or greater. In the IDEAL-AF trial, 209 patients at 5 Swedish centers were randomized after pulmonary vein isolation to adjunctive low-voltage zone ablation or no further ablation. Arrhythmia-free survival off antiarrhythmic drugs at 12 months was 67.6% vs 37.4% (odds ratio 3.5; P < .001), with greater quality-of-life gains and no excess serious adverse events.
A single dose of the RSVpreF vaccine remained associated with fewer RSV-related RTD hospitalizations among adults 60 years or older in a second RSV season, with an incidence of 0.11 versus 0.25 events per 1000 person-years and vaccine effectiveness of 56.2% (95% CI, -12.4% to 84.8%), down from 83.3% in season one. Across both seasons of the 130,991-participant intention-to-treat analysis, vaccine effectiveness was 70.6% (95% CI, 39.1%-87.0%). The results extend RSVpreF protection beyond one season while indicating waning estimates.
A secondary analysis of the CARDIOTTRansform phase 3 randomized controlled trial found that eplontersen, a transthyretin-targeting antisense oligonucleotide, benefited patients with transthyretin amyloid cardiomyopathy who were not on transthyretin stabilizers at baseline, but showed no such effect in those already taking stabilizers. The result separates responders from non-responders by background therapy. Findings were presented at the 2026 ESC Congress.
Adding milvexian, an oral factor XIa inhibitor, to standard antiplatelet therapy within 7 days after an acute coronary syndrome did not reduce recurrent ischemic events. In 14,194 patients followed for a median of 12.2 months, the composite of cardiovascular death, myocardial infarction, or ischemic stroke occurred in 5.4% with milvexian versus 5.1% with placebo (hazard ratio, 1.05; 95% CI, 0.91 to 1.21). The trial was stopped for futility at a planned interim analysis; major bleeding rates were 0.3% in both groups.
Evolocumab started in the catheterization laboratory before PCI for high-risk acute MI did not change clinical outcomes at 1 year. All-cause death or unplanned cardiovascular hospitalization occurred in 14.6% of 1087 patients given evolocumab plus standard care vs 15.4% of 1074 given standard care alone (adjusted odds ratio 0.94; P = .59). It did, however, achieve the LDL-C target (<55 mg/dL and at least 50% reduction) in 82% vs 40% of patients at 12 months, with median LDL-C of 16 vs 56 mg/dL at 6 weeks.
Dual antithrombotic therapy combining direct oral anticoagulants with potent P2Y12 inhibitors in patients with atrial fibrillation and acute coronary syndrome was associated with higher bleeding rates than direct oral anticoagulants with clopidogrel and aspirin, with no evidence of reduced ischemic events. The randomized controlled trial results were presented at the 2026 European Society of Cardiology Congress.
First-line sunvozertinib extended median progression-free survival to 10.3 months versus 7.5 months with carboplatin-pemetrexed in advanced nonsquamous NSCLC with EGFR exon 20 insertion mutations (hazard ratio 0.65; P<0.001), in a phase 3 trial of 324 patients. Objective response was 58.9% versus 31.1%, with median duration of response of 11.2 versus 7.1 months. The findings support moving sunvozertinib, already accelerated-approved in later lines, into first-line use.
Eplontersen, co-developed by AstraZeneca and Ionis, missed the primary endpoint in the Phase III CARDIO-TTRansform trial in ATTR-CM. Detailed data show that among patients already on a TTR stabilizer at baseline, adding eplontersen led to worse outcomes than the stabilizer alone. The miss, announced in July, is a major setback for a planned future core revenue driver for both companies.
