The Menarini Group and Karyopharm Therapeutics report that selinexor plus ruxolitinib improves spleen response over standard monotherapy in the Phase 3 SENTRY trial in myelofibrosis, Agios Pharmaceuticals reinforces the anti-hemolytic profile of mitapivat with new transfusion data from the Phase 3 RISE UP trial in sickle cell disease, AstraZeneca reports a prespecified subgroup survival benefit for anselamimab in the Phase 3 CARES programme in light chain amyloidosis, Merck secures FDA approval for belzutifan plus pembrolizumab as an adjuvant treatment in kidney cancer, and AbbVie presents nine-year fixed-duration data for venetoclax in the Phase 3 CLL14 trial in chronic lymphocytic leukaemia, the most significant data from across the pipeline.
This week the most consequential data was dominated by a single venue, the European Hematology Association Congress in Stockholm, making it one of the most concentrated weeks for blood cancer and rare blood disorder research so far this year. The unifying theme was a maturing evidence base for combination and fixed-duration strategies in diseases where patients have long faced either lifelong therapy or a bleak prognosis. Several readouts also illustrated the nuance of modern trial design, with the headline benefits resting on individual co-primary endpoints or prespecified subgroups rather than clean sweeps, even as a major regulatory decision in solid tumour oncology landed in parallel. Here, Life Science Daily News brings you the most significant clinical trial results 19 June 2026.
Selinexor Combination Improves Spleen Response in Phase 3 SENTRY Myelofibrosis Trial
The Menarini Group and Karyopharm Therapeutics announced on 14 June that the pivotal Phase 3 SENTRY trial of selinexor plus ruxolitinib in frontline myelofibrosis met the first of its two co-primary endpoints. Among the 353 patients randomised to the combination or to ruxolitinib alone, 49.8 per cent of those receiving the selinexor combination achieved a 35 per cent or greater spleen volume reduction at week 24, compared with 28.0 per cent on monotherapy. The combination did not, however, meet the second co-primary endpoint: the improvement in absolute total symptom score at week 24 was 9.9 points with the combination versus 10.9 points with ruxolitinib alone, a difference that was not statistically significant. The data were selected for a late-breaking oral presentation at the congress.
A pre-specified secondary analysis pointed to a striking, though still immature, survival signal, with a greater than 50 per cent reduction in the risk of death for patients on the selinexor combination, corresponding to a hazard ratio of 0.43. Spleen benefit was consistent across prespecified subgroups, including patients receiving lower ruxolitinib doses, and a marker of potential disease modification, variant allele frequency reduction, was observed in 32 per cent of combination patients versus 23.9 per cent of those on monotherapy at week 24. Safety was consistent with the established profiles of both agents, with no new signals reported. Myelofibrosis is a rare bone marrow cancer with a median survival of around six years from diagnosis, and because spleen reduction remains a central goal of treatment, a regimen that deepens that response while hinting at longer survival represents a potentially meaningful step forward.
Mitapivat Reinforces Anti-Hemolytic Profile with New Transfusion Data in Phase 3 RISE UP Sickle Cell Trial
Agios Pharmaceuticals presented detailed results from the 52-week double-blind period of the global Phase 3 RISE UP trial of mitapivat, an oral pyruvate kinase activator, during the plenary session at the congress on 13 June. The trial randomised 207 patients aged 16 and over with sickle cell disease, two to one, to mitapivat or placebo. As previously reported, the trial met the haemoglobin-response co-primary endpoint, with 40.6 per cent of mitapivat patients responding versus 2.9 per cent on placebo, a statistically significant difference. The second co-primary endpoint, the annualised rate of sickle cell pain crises, showed a reduction but did not reach statistical significance.
The genuinely new analyses presented at the congress centred on transfusion burden, where mitapivat was associated with a 41.1 per cent relative reduction in the proportion of patients requiring transfusions, at 23.9 per cent versus 40.6 per cent on placebo, and a 55.9 per cent relative reduction in red blood cell units transfused per patient. Patients who achieved a haemoglobin response also showed clinically meaningful improvements across pain, fatigue, sleep and physical function compared with non-responders. The therapy was well tolerated, with no treatment-related deaths. Agios submitted a supplemental New Drug Application to the FDA in May 2026 seeking accelerated approval, and the company describes RISE UP as the first pivotal trial to validate pyruvate kinase activation as a treatment approach in sickle cell disease.
Anselamimab Shows Prespecified Subgroup Survival Benefit in Phase 3 CARES Amyloidosis Programme
Detailed results from the Phase 3 CARES programme of anselamimab, an intravenous amyloid fibril depleter antibody developed by AstraZeneca’s Alexion rare disease unit, were published in the Journal of Clinical Oncology, with the data also presented at the ASCO Annual Meeting in May. The programme comprised two parallel placebo-controlled trials enrolling 406 patients with advanced disease across 19 countries, making it the largest prospective study conducted to date in cardiac light chain amyloidosis. The primary endpoint, a hierarchical combination of time to all-cause mortality and frequency of cardiovascular hospitalisations, was not met in the overall population.
In a prespecified subgroup of patients, however, anselamimab produced a clinically meaningful improvement in both survival and cardiovascular hospitalisations when added to standard plasma cell directed therapy. The treatment was generally well tolerated. Anselamimab is the first investigational fibril depleter to show clinical benefit in this patient subgroup in light chain amyloidosis, a devastating disease in which misfolded light chains accumulate as amyloid deposits and damage the heart and kidneys, and the subgroup findings have formed the basis of a European marketing application. Taken together, the findings underscore both the promise of clearing existing amyloid deposits and the difficulty of demonstrating benefit across an entire advanced-stage population.
Belzutifan Plus Pembrolizumab Wins FDA Approval in Adjuvant Kidney Cancer
The FDA approved belzutifan on 12 June, marketed by Merck as Welireg, in combination with pembrolizumab (Keytruda) or its subcutaneous formulation (Keytruda Qlex), for the adjuvant treatment of adults with renal cell carcinoma with a clear cell component, at intermediate-high or high risk of recurrence following nephrectomy, or following nephrectomy and resection of metastatic lesions. The approval, announced by Merck, marks the first approved combination of a PD-1 inhibitor and a HIF-2 alpha inhibitor, and the first global Phase 3 study to improve disease-free survival over pembrolizumab alone in this setting.
The approval rests on the Phase 3 LITESPARK-022 trial, which randomised 1,841 patients one to one to the combination or to placebo plus pembrolizumab. The combination reduced the risk of disease recurrence, metastasis or death by 28 per cent, with a hazard ratio of 0.72 and a p-value of 0.0003, and the estimated 24-month disease-free survival rate was 81 per cent for the combination versus 74 per cent for pembrolizumab alone. Belzutifan is a first-in-class oral HIF-2 alpha inhibitor and carries a boxed warning for embryo-fetal toxicity alongside precautions for anaemia and hypoxia. Grade 3 or higher treatment-emergent adverse events occurred in 52.1 per cent of combination patients compared with 30.2 per cent on pembrolizumab alone, a tolerability trade-off that clinicians will weigh against the recurrence benefit.
Venetoclax Delivers Nine-Year Fixed-Duration Data in Phase 3 CLL14 Leukaemia Trial
AbbVie presented the final nine-year analysis of the Phase 3 CLL14 trial at the congress on 12 June, reporting durable long-term outcomes for a fixed-duration regimen of venetoclax plus obinutuzumab in previously untreated chronic lymphocytic leukaemia. The trial, run with the German CLL Study Group, randomised 432 patients with coexisting medical conditions to either 12 months of venetoclax with six cycles of obinutuzumab or to chlorambucil plus obinutuzumab.
After a median follow-up of 9.2 years, the venetoclax combination maintained a clear advantage, with a median progression-free survival of 6.4 years versus 3.2 years for the chlorambucil-based regimen, a hazard ratio of 0.50. The median time to next treatment reached 7.6 years, meaning patients gained many years off therapy after just one year of treatment. The analysis reinforces the case for time-limited, chemotherapy-free first-line treatment in older or less fit patients, offering an alternative to the continuous therapy that has dominated much of the field and adding long-term reassurance on both efficacy and safety.
Looking Ahead
This week’s clinical trial results 19 June 2026 underline how a single congress can reshape the outlook across several blood disorders at once. The myelofibrosis and sickle cell readouts show combination and mechanism-based approaches advancing even where not every endpoint was met, while the amyloidosis programme highlights both the promise of fibril clearance and the challenge of proving benefit across a whole advanced-stage population. The long-term leukaemia data strengthen the case for fixed-duration treatment, and the kidney cancer approval extends precision combinations further into the adjuvant setting. Life Science Daily News will continue to bring you accurate, timely coverage of the clinical trial results 19 June 2026 that matter most across the global life sciences pipeline.
