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Clinical readouts

Phase III and pivotal trial results — positive and negative, treated alike.

Latest picks

321–340 of 383
Aug 21Fri
  1. BMJ66

    Head positioning after endovascular therapy for acute stroke due to large vessel occlusion (HeadSOAR): multicentre randomised controlled trial.

    Elevating the head to 30-40° for 72 hours after endovascular thrombectomy did not significantly improve 90 day functional outcomes versus a flat position of 0-10° in patients with anterior circulation large vessel occlusion stroke and successful reperfusion. Among 1368 adults at 67 Chinese stroke centres, the adjusted generalised odds ratio for lower disability on the modified Rankin scale was 1.12 (95% CI 0.97 to 1.29, P=0.14), and 90 day mortality was 18.3% versus 20.3% (P=0.18). Head position after thrombectomy appears to have little effect on recovery, though a modest clinically meaningful benefit remains possible.

    Why it matters: It tests a common, zero-cost post-thrombectomy care decision in a large multicentre randomised trial.

  2. The Lancet78

    Oral step-down, optimal drug, and total duration of antibiotic treatment in African children hospitalised with severe community-acquired pneumonia (PediCAP): a factorial randomised controlled trial.

    In sub-Saharan African children hospitalised with severe community-acquired pneumonia, stepping down to oral amoxicillin after initial intravenous antibiotics was non-inferior to WHO-recommended 5-day intravenous-only treatment for the primary outcome of day-28 readmission or death, and co-amoxiclav showed no superiority over amoxicillin. All total durations from 4 to 8 days were non-inferior to the 8-day schedule. The trial randomly allocated 1101 children across 13 hospitals in five countries.

    Why it matters: It supports replacing prolonged intravenous therapy with early oral step-down and shorter total durations in severe childhood pneumonia.

  3. The Lancet69

    High-dose chemotherapy followed by autologous stem-cell transplantation versus non-myeloablative consolidation in primary CNS lymphoma (MATRix/IELSG43): a randomised phase 3 trial.

    Thiotepa-based HCT-ASCT consolidation after MATRix induction significantly improved 3-year progression-free survival in newly diagnosed primary CNS lymphoma compared with non-myeloablative R-DeVIC: 78% versus 51% in the 229 analysed patients. HCT-ASCT caused more adverse events per patient and five fatal consolidation-related events versus two with R-DeVIC. The findings establish HCT-ASCT as the preferred consolidation strategy in fit patients.

    Why it matters: First randomised phase 3 comparison showing HCT-ASCT superiority over non-myeloablative consolidation in primary CNS lymphoma.

  4. WeChat68

    intismeran autogene plus Keytruda hits Phase III primary endpoint; Moderna market value surges, BioNTech exits

    Merck and Moderna announced that the mRNA cancer vaccine intismeran autogene (V940) combined with Keytruda met the primary endpoint in the Phase III INTerpath-001 study in melanoma. Moderna closed the prior trading day with a market value of about $25 billion, up 176.97% in one day and approaching the $70 billion mark, while BioNTech has withdrawn from this direction.

    Why it matters: INTerpath-001 hit its Phase III primary endpoint and Moderna's market value rose nearly 177% in a day, contrasting with BioNTech's retreat in the same field.

  5. WeChat76

    Merck and Moderna mRNA Cancer Vaccine Phase III Trial Meets Primary Endpoint, Reducing Melanoma Recurrence and Metastasis Risk

    Merck and Moderna's individualized mRNA neoantigen therapy intismeran autogene, combined with KEYTRUDA as adjuvant therapy for patients with fully resected stage IIB-IV melanoma, met the primary endpoint of recurrence-free survival (RFS) and the key secondary endpoint of distant metastasis-free survival (DMFS) in a Phase III trial.

    Why it matters: The source reports adjuvant RFS and DMFS results that can serve as a benchmark for mRNA neoantigen therapy in solid-tumor adjuvant settings.

  6. WeChat77

    Personalized Neoantigen mRNA Cancer Vaccine Plus Keytruda Meets Pivotal RFS Primary Endpoint, Filing Planned

    A pivotal trial of a personalized neoantigen mRNA cancer vaccine combined with Keytruda met its primary endpoint of recurrence-free survival (RFS) and the key secondary endpoint of distant metastasis-free survival (DMFS) at a prespecified interim analysis. The combination showed statistically significant and clinically meaningful improvement over Keytruda monotherapy as adjuvant therapy in resected melanoma.

    Why it matters: The interim analysis provides RFS and DMFS data readers can compare directly with existing Keytruda monotherapy adjuvant data in resected melanoma.

  7. WeChat76

    Moderna Personalized mRNA Cancer Vaccine Succeeds in Phase III INTerpath-001 Randomized Trial

    Moderna's personalized mRNA cancer vaccine met its endpoint in the phase III randomized controlled INTerpath-001 trial. The study enrolled 1,137 patients with stage IIB-IV cutaneous melanoma after complete surgical resection and no prior systemic therapy. It is the first personalized neoantigen therapy to succeed in a phase III randomized controlled trial.

    Why it matters: The source gives the 1,137-patient stage IIB-IV post-surgery enrollment range and safety readout for comparison with prior phase II efficacy signals.

  8. WeChat80

    Intismeran Autogene Plus KEYTRUDA Meets RFS Primary Endpoint in Melanoma Adjuvant Trial

    Intismeran autogene, a personalized neoantigen mRNA therapy jointly developed by Merck and Moderna, met the primary endpoint of recurrence-free survival (RFS) and the key secondary endpoint of distant metastasis-free survival (DMFS) when combined with KEYTRUDA. The results come from INTerpath-001, an international multicenter trial in adjuvant treatment of completely resected stage IIB-IV melanoma.

    Why it matters: The brief gives INTerpath-001 topline and primary endpoint data for judging the added benefit of the mRNA neoantigen therapy over KEYTRUDA alone.

  9. WeChat71

    Moderna and Merck intismeran plus pembrolizumab posts positive Phase III topline in melanoma adjuvant therapy

    Moderna and Merck jointly announced that the Phase III INTerpath-001 trial of the personalized mRNA neoantigen therapy intismeran (V940/mRNA-4157) combined with pembrolizumab as adjuvant therapy for melanoma met positive topline results. Each intismeran course encodes up to 34 neoantigens, tailored to each patient's tumor mutation profile; Moderna shares surged 177% on the news.

    Why it matters: Reports the positive INTerpath-001 topline readout and Moderna's 177% share surge as a market reference for personalized mRNA neoantigen therapy in melanoma adjuvant treatment.

  10. WeChat72

    Merck and Moderna Say intismeran autogene Plus KEYTRUDA Phase III Hits RFS and DMFS Endpoints

    Merck and Moderna announced that the Phase III INTerpath-001 trial met both its recurrence-free survival (RFS) and distant metastasis-free survival (DMFS) endpoints. The trial evaluated the personalized cancer vaccine intismeran autogene (V940 or mRNA-4157) combined with KEYTRUDA as adjuvant therapy in patients with fully resected stage IIB–IV melanoma. Moderna shares closed up 176.97% after the announcement.

    Why it matters: The trial met both RFS and DMFS endpoints in resected melanoma adjuvant therapy, providing a benchmark for personalized vaccine plus PD-1 regimens.

  11. WeChat82

    Moderna Cancer Therapy Meets Recurrence-Free Survival Endpoint in Phase III INTerpath-001 Melanoma Trial

    Moderna's cancer therapy met its primary endpoint of recurrence-free survival (RFS) in the Phase III INTerpath-001 trial, which enrolled 1137 patients with high-risk melanoma. It reduced the risk of recurrence or death by 49% and the risk of distant metastasis or death by 59%. On the news, Moderna's stock rose 177% in a single day and Merck's rose nearly 13%.

    Why it matters: The brief gives dual endpoint data of a 49% reduction in recurrence or death risk and a 59% reduction in distant metastasis risk, plus a 1137-patient sample, directly quantifying efficacy.

  12. WeChat77

    Moderna and Merck's Personalized mRNA Cancer Vaccine intismeran autogene with Keytruda Succeeds in INTerpath-001 Trial

    On August 19, 2026, Moderna and Merck jointly announced that the INTerpath-001 trial of the personalized mRNA cancer vaccine intismeran autogene (V940/mRNA-4157) combined with Keytruda (pembrolizumab) succeeded. The original article reports Moderna's stock surged 177% that day, calls it an "extraordinary milestone," and says the company's next three years hinge on converting this result into sustainable cash flow.

    Why it matters: Reports the key INTerpath-001 success so readers can assess the combination's relevance to mRNA cancer vaccines.

Aug 20Thu
  1. 顶刊论文(社媒热度)73

    Sirolimus-Coated Balloon Angioplasty for Infrainguinal Artery Disease.

    Sirolimus-coated balloon angioplasty cut major adverse limb events at 1 year in patients with infrainguinal artery disease: the primary composite of unplanned major amputation or revascularization for critical limb ischemia occurred in 8.8% versus 15.0% with uncoated balloons (risk difference -4.9 percentage points; P = 0.009 for superiority). All-cause death was similar, 11.8% versus 12.8%.

    Why it matters: First large trial (1252 patients) showing sirolimus-coated balloons are superior, not just noninferior, for infrainguinal artery disease.

  2. BioCentury73

    Moderna, Merck Phase III win opens door to personalized neoantigen vaccines

    An interim analysis of the Phase III INTerpath-001 trial, run by Moderna and Merck, met its primary endpoint. Adjuvant treatment with intismeran autogene plus Keytruda improved recurrence-free survival (RFS) in patients with completely resected stage IIB-IV melanoma and met the key secondary endpoint of distant metastasis-free survival (DMFS). The results provide key Phase III evidence for personalized neoantigen vaccines in melanoma adjuvant therapy.

    Why it matters: The positive Phase III data validate personalized neoantigen vaccines in melanoma adjuvant therapy and inform extending the mRNA platform to oncology.

  3. WeChat74

    AstraZeneca Discontinues Phase III eVOLVE-Lung02 of PD-1/CTLA-4 Bispecific Volrustomig in First-Line NSCLC

    AstraZeneca has discontinued the Phase III eVOLVE-Lung02 study of its PD-1/CTLA-4 bispecific antibody Volrustomig as first-line therapy for non-small cell lung cancer. After a pre-specified interim analysis, the independent data monitoring committee concluded the drug was almost certainly unable to beat the standard regimen of pembrolizumab plus chemotherapy in the PD-L1 negative population. The global, multicenter pivotal trial enrolled 895 patients and failed at its first readout.

    Why it matters: Provides a Phase III interim failure for a PD-1/CTLA-4 bispecific in first-line NSCLC, with the comparator regimen, as a reference for the same-target pipeline.

  4. WeChat79

    Amylyx first-in-class GLP-1 inhibitor avexitide hits primary endpoint in Phase 3 LUCIDITY

    On August 18 local time, Amylyx Pharmaceuticals announced that avexitide met the FDA-agreed primary endpoint in the Phase 3 LUCIDITY study in post-bariatric hypoglycemia (PBH). The composite incidence of Grade 2 and Grade 3 hypoglycemia events fell 55% versus placebo (p=0.000003). The source describes avexitide as a first-in-class GLP-1 inhibitor.

    Why it matters: The 55% event reduction and p=0.000003 let readers compare avexitide with other drugs in development for the same indication.

  5. WeChat68

    DURAVYU Misses Non-Inferiority Primary Endpoint in Phase III LUGANO Wet AMD Study

    On August 17 local time, EyePoint announced topline results from LUGANO, the first pivotal Phase III study of DURAVYU in wet age-related macular degeneration (wAMD). DURAVYU failed to meet the primary endpoint of non-inferiority to aflibercept in best corrected visual acuity (BCVA). The Phase III result also dragged down two companies based far apart, one described in reports as an oncology giant with a market value of about 20 billion.

    Why it matters: The topline result missed the non-inferiority primary endpoint, letting readers assess the competitive landscape for long-acting VEGF agents in wet AMD.

  6. WeChat74

    Amylyx avexitide Phase 3 trial hits primary endpoint, cutting severe hypoglycemia risk by 55%

    Amylyx Pharmaceuticals announced positive topline results from the Phase 3 LUCIDITY trial. The GLP-1 receptor antagonist avexitide reduced the risk of severe hypoglycemic episodes by 55% in patients with post-bariatric hypoglycemia (PBH), meeting the primary endpoint; all secondary endpoints were also met.

    Why it matters: avexitide, an antagonist acting opposite to GLP-1 agonists like semaglutide and tirzepatide, showed a 55% risk reduction, enabling direct comparison with other post-bariatric complication data.

Aug 19Wed
  1. JAMA64

    Daily Zinc Supplementation for Infection Prevention in Children With Sickle Cell Anemia: The ZIPS-2 Randomized Clinical Trial.

    Daily zinc sulfate at 20 mg reduced all-cause infections in Ugandan children aged 1.00 to 4.99 years with sickle cell anemia. Over 6 months, the zinc group had 305.7 infections per 100 person-years versus 480.7 with placebo, an adjusted incidence rate ratio of 0.62 (95% CI, 0.45-0.86), with no adverse events requiring discontinuation. The finding adds a simple, safe preventive option for a high-morbidity outcome in this population.

    Why it matters: A placebo-controlled trial showing a common supplement cuts infections in young children with sickle cell anemia.

  2. STAT74

    STAT+: Moderna and Merck say mRNA cancer vaccine succeeded in late-stage melanoma trial

    Moderna and Merck announced that the personalized mRNA neoantigen vaccine intismeran combined with Keytruda met its primary endpoint in a Phase 3 adjuvant melanoma trial, delaying recurrence and distant metastasis. The release did not provide the HR, confidence intervals, or detailed endpoint results; those await a full readout and comparison with existing adjuvant immunotherapy regimens.

    Why it matters: Topline result for a personalized mRNA vaccine plus Keytruda in adjuvant melanoma, but HR, confidence intervals, and stratification details were not released, so it serves only as a preliminary reference for Phase 3 neoantigen vaccine readouts.

CBACBA Frontier Insights (CFI)Chinese Biopharmaceutical Association (CBA)www.cba-usa.org