Metrics & Benchmarks

In this video interview, C.K. Wang, MD, chief medical officer, COTA, discusses a recent study he led on the validity of using real-world data for reporting mortality.

Early findings from Johnson & Johnson’s Phase IIb SunRISe-1 trial show the promise of TAR-200 in patients with Bacillus Calmette-Guérin–unresponsive, high-risk non–muscle-invasive bladder cancer, highlighting its potential as a non-surgical treatment alternative.

Interim KEYNOTE-689 trial data show that perioperative Keytruda significantly lowers the risk of disease progression or recurrence in resectable, locally advanced head and neck squamous cell carcinoma, marking the first major clinical advance for this patient population in more than two decades.

Lorundrostat, a selective aldosterone synthase inhibitor, significantly reduced systolic blood pressure in patients with uncontrolled and treatment-resistant hypertension in the Phase II Advance-HTN trial, indicating its potential as a safer, more effective alternative to current therapies.

Brensocatib significantly reduced the rate of pulmonary exacerbations and slowed lung function decline in patients with non-cystic fibrosis bronchiectasis in the Phase III ASPEN trial.

In the Phase III SELECT-GCA trial, Rinvoq (upadacitinib) 15 mg with a 26-week glucocorticoid taper significantly improved sustained remission rates and reduced disease flares in patients with giant-cell arteritis, offering a promising glucocorticoid-sparing treatment option with a favorable safety profile.

Tolebrutinib did not show superiority over Aubagio (teriflunomide) in reducing relapse rates or MRI-detected inflammatory activity in relapsing multiple sclerosis, but early signals suggest it may have potential in slowing disability progression, warranting further long-term and comparative studies.

Full results from the Phase III ASCENT-04/KEYNOTE-D19 study demonstrated statistically significant and clinically meaningful survival benefits in patients with previously untreated, PD-L1-positive metastatic triple-negative breast cancer.

In the Phase III DESTINY-Breast09 trial, Enhertu (trastuzumab deruxtecan) and Perjeta (pertuzumab) significantly improved progression-free survival compared to the current standard of care in the first-line treatment of HER2-positive metastatic breast cancer, suggesting a potential new standard of care.

In Phase III MINT trial, Uplizna (inebilizumab) demonstrated significant short-term efficacy and a manageable safety profile in adult patients with generalized myasthenia gravis, offering a promising B-cell–targeting treatment option pending long-term data.

Beqvez (fidanacogene elaparvovec), an FDA-approved one-time gene therapy for hemophilia B, demonstrated sustained factor IX expression, low bleeding rates, and a favorable safety profile over long-term follow-up.

Adding Farxiga (dapagliflozin) to standard care significantly reduced the risk of death or worsening heart failure in older patients with severe aortic stenosis following transcatheter aortic-valve implantation, with consistent benefits across patient subgroups and a favorable safety profile.

Tolebrutinib, a brain-penetrant BTK inhibitor, may fill a significant unmet need by delaying disability progression in patients with non-relapsing secondary progressive multiple sclerosis.

In the Phase III EXPECTS trial, patients experiencing mostly mild posterior circulation ischemic stroke who were ineligible for thrombectomy showed significantly better functional recovery at 90 days when treated with alteplase between 4.5 and 24 hours after stroke onset, without an increased risk of symptomatic intracranial hemorrhage.

Long-term extension data show Tremfya (guselkumab) significantly reduced symptoms and inhibited structural joint damage progression in patients with active psoriatic arthritis.

Phase III SOUL trial shows daily oral semaglutide lowers the risk of major adverse cardiovascular events in patients with type 2 diabetes and atherosclerotic cardiovascular disease or chronic kidney disease, with benefits consistent with injectable semaglutide and other GLP-1 receptor agonists.

The Phase III API-CAT trial found that extended anticoagulant therapy with reduced-dose Eliquis (apixaban) was noninferior to the full-dose regimen in preventing recurrent venous thromboembolism in patients with cancer.

Oral PCSK9 inhibitor, AZD0780, demonstrated a significant 50.7% reduction in LDL cholesterol levels in the Phase IIb PURSUIT trial when added to standard statin therapy.

Eli Lilly’s lepodisiran, an investigational siRNA therapy, achieved significant and durable reductions in lipoprotein(a) levels, a major genetic risk factor for cardiovascular disease.

Solriamfetol achieved the primary and key secondary endpoint of the Phase III FOCUS trial by significantly lowering attention-deficit hyperactivity disorder symptoms and disease severity in adults compared to placebo, with a favorable safety and tolerability profile.

FDA grants Priority Review status to Sanofi's tolebrutinib for the treatment of non-relapsing secondary progressive multiple sclerosis based on positive findings from multiple Phase III trials.

Phase III VERITAC-2 trial results show vepdegestrant significantly improved progression-free survivalcompared to fulvestrant in patients with ESR1-mutant (ESR1m) advanced or metastatic breast cancer, but did not achieve statistical significance in the overall intent-to-treat population.

Phase III IMvoke010 trial found that maintenance treatment with Tecentriq (atezolizumab) did not significantly improve survival compared with placebo in patients with locally advanced squamous cell carcinoma of the head and neck following multimodal definitive treatment.

Phase III DIPPER trial shows that adjuvant camrelizumab administered after induction-concurrent chemoradiotherapy significantly improved event-free survival in patients with locoregionally advanced nasopharyngeal carcinoma, along with a favorable safety profile.

FDA grants Fast Track designation to Johnson & Johnson’s investigational therapy nipocalimab for the treatment of moderate-to-severe Sjögren’s disease, which could be the first FcRn blocker ever approved for the autoimmune condition.