Metrics & Benchmarks

Results from the long-term extension of the Phase III TRAILBLAZER-ALZ 2 trial (NCT04437511) showed sustained benefits with early Kisunla treatment, including reduced risk of disease progression and high rates of amyloid clearance in early Alzheimer disease.

Results from the Phase III Clarity AD open-label extension trial (NCT03887455) suggest weekly subcutaneous maintenance dosing with Leqembi may offer a more convenient and well-tolerated alternative for patients continuing long-term treatment.

Results from the Phase III BRUIN CLL-314 trial (NCT05254743) showed that Jaypirca (pirtobrutinib) achieved superiority in overall response rates compared to Imbruvica (ibrutinib) in patients with chronic lymphocytic leukemia or small lymphocytic lymphoma.

Results from the Phase III VIKTORIA-1 trial (NCT05501886) demonstrated that gedatolisib administered with fulvestrant, with or without Ibrance (palbociclib), showed statistically significant and clinically meaningful improvements in progression-free survival in patients with HR-positive, HER2-negative, PIK3CA wild-type advanced breast cancer.

Results from the Phase III PREVAIL trial (NCT05556096) of gefurulimab demonstrated a statistically significant and clinically meaningful improvement in Myasthenia Gravis Activities of Daily Living at 26 weeks compared to placebo.

Results from the Phase III ABTECT-1 (NCT05507203) and ABTECT-2 (NCT05507216) trials showed that Abivax’s obefazimod demonstrated significant clinical remission in patients with ulcerative colitis.

The Phase III INDEPENDENCE trial (NCT04717414) failed to meet its primary endpoint of achieving transfusion independence in patients with myelofibrosis-associated anemia treated with Reblozyl (luspatercept-aamt).

Results from the Phase III PURPOSE 1 (NCT04994509) and PURPOSE 2 (NCT04925752) trials highlight Yeztugo’s (lenacapavir) safety and efficacy in pregnant women, young adults, and others historically excluded from HIV prevention studies.

Results from a Phase Ib trial (NCT03666000) show that azer-cel demonstrated a 75% overall response rate and a 55% complete response in patients with relapsed/refractory diffuse large b-cell lymphoma.

Results from a retrospective study found that Amtagvi (lifileucel) achieved a 48.8% objective response rate in patients with advanced melanoma who had previously received immune checkpoint inhibitors.

Results from the Phase III CAHtalyst show that patients treated with Crenessity for classic congenital adrenal hyperplasia achieved and maintained lower, more physiologic glucocorticoid doses while keeping key adrenal biomarkers at or below baseline levels.

Results from the Phase III EMBARK trial show that in combination with leuprolide, Xtandi (enzalutamide) demonstrated a statistically significant and clinically meaningful overall survival benefit in men with non-metastatic hormone-sensitive prostate cancer and high-risk biochemical recurrence.

Results from the Phase III KEYNOTE-689 trial show that adding perioperative Keytruda (pembrolizumab) to standard-of-care radiotherapy improves event-free survival in patients with resectable, locally advanced head and neck squamous cell carcinoma, potentially setting a new benchmark for treatment in this setting.

The Phase I/IIa CLARICO trial of OpCT-001 marks the first clinical trial of an investigational induced pluripotent stem cell-derived therapy for primary photoreceptor diseases.

Once-weekly cagrilintide–semaglutide combination therapy produced unprecedented weight reductions in adults with overweight or obesity, with 60% achieving ≥20% loss and strong cardiometabolic benefits, according to Phase IIIa trial results published in The New England Journal of Medicine.

The global Phase III PROMINENT trial has begun dosing patients to evaluate felzartamab in treating primary membranous nephropathy, a serious autoimmune kidney disorder with no FDA-approved therapies.

Updated results from the Phase II RedirecTT-1 trial show that the dual bispecific antibody regimen of Talvey and Tecvayli delivers deep, lasting responses in patients with triple-class exposed relapsed or refractory multiple myeloma and true extramedullary disease.

Although the SYMMETRY Phase IIb trial of the FGF21 analogue efruxifermin did not meet its primary endpoint at 36 weeks, extended 96-week data suggest potential benefits in fibrosis reduction and liver health in patients with compensated cirrhosis due to MASH, supporting continued evaluation in Phase III studies.

Detailed Phase IIIb STEP UP trial results presented at the 2025 ADA Scientific Sessions found that an investigational 7.2 mg dose of Wegovy (semaglutide) led to a mean weight loss of 21% in adults with obesity and no diabetes, showing the potential to expand dosing options in obesity management.

In a Phase II trial, Amgen’s investigational therapy MariTide (maridebart cafraglutide) delivered significant, sustained weight loss of up to 20% in adults with obesity, with or without type 2 diabetes, alongside notable improvements in glycemic control, supporting its advancement to Phase III studies as a potential once-monthly treatment option.

New Phase III data published in The New England Journal of Medicine show elinzanetant, a nonhormonal dual NK-1/NK-3 receptor antagonist, significantly reduced vasomotor symptoms, improved sleep, and enhanced quality of life for women undergoing endocrine therapy for HR-positive breast cancer.

Primary results from the Phase II TRANSCEND FL trial show Breyanzi (lisocabtagene maraleucel) achieved a 95.5% response rate and durable efficacy in relapsed or refractory marginal zone lymphoma, supporting its potential as a transformative CAR T-cell therapy.

New data from the POETYK PsA-Phase III trial, presented at the 2025 EULAR Congress, demonstrate that Bristol Myers Squibb’s Sotyktu (deucravacitinib) significantly improves joint and skin symptoms in adults with active psoriatic arthritis, maintaining efficacy through 52 weeks of treatment.

Data from the Phase IIIb APEX study, presented at EULAR 2025, show that guselkumab (Tremfya) improved joint and skin symptoms and inhibited structural joint damage in patients with active psoriatic arthritis, reinforcing its role as a leading IL-23 inhibitor for long-term disease control.

Phase III DeLLphi-304 results show T-cell engager Imdelltra (tarlatamab) delivers superior overall survival, improved progression-free survival, and better symptom control compared to chemotherapy in patients with small-cell lung cancer following platinum-based treatment.

Phase II CONFIDENCE study data reveal that simultaneous initiation of Kerendia and Jardiance significantly improves UACR and delays chronic kidney disease progression in patients with type 2 diabetes.

New data from the IRAKLIA and IZALCO trials, presented at the 2025 ASCO Annual Meeting, demonstrate that subcutaneous Sarclisa delivered via an on-body delivery system offers non-inferior efficacy to IV infusion in patients with relapsed or refractory multiple myeloma, while significantly reducing infusion-related reactions and improving patient satisfaction.

Results from the Phase Ib/II CARTITUDE-1 trial demonstrated that a single infusion of Carvykti delivered durable, treatment-free remissions for at least five years in a third of patients with relapsed or refractory multiple myeloma.

Interim results from the global MATTERHORN study mark the first time an immunotherapy has demonstrated statistically significant event-free survival in a Phase III trial for resectable gastric and gastroesophageal junction cancers, signaling a potential shift in standard perioperative care.

Phase III data presented at ASCO 2025 and published in The New England Journal of Medicine show that Pfizer’s Braftovi (encorafenib), combined with Erbitux (cetuximab) and mFOLFOX6, significantly improves overall survival and progression-free survival in treatment-naïve patients with BRAF V600E-mutant metastatic colorectal cancer—potentially establishing a new first-line standard-of-care for this high-risk population.