Don Tracy, Associate Editor

The FDA-approved therapy is now included in National Comprehensive Cancer Network clinical guidelines as a treatment option for both pediatric and adult patients with aggressive H3 K27M-mutant diffuse gliomas, offering a new standard of care for recurrent disease.

Results from the two-year AVONELLE-X extension study (NCT04777201) and the Phase IIIb/IV SALWEEN trial showed that Roche’s Vabysmo demonstrated durable vision improvements, extended dosing potential, and reliable safety treating wet age-related macular degeneration and polypoidal choroidal vasculopathy.

The designation supports expedited development of SAR402663, Sanofi’s one-time gene therapy designed to reduce treatment burden and slow vision loss in patients with wet age-related macular degeneration.

Results from a Phase II trial (NCT06449209) showed that BioNTech’s and Bristol Myers Squibb’s pumitamig in combination with standard chemotherapy demonstrated encouraging anti-tumor activity in patients with extensive-stage small cell lung cancer.

Results from the Phase III ASCEND trial (NCT05882877) showed rocatinlimab maintained long-term efficacy with a favorable safety profile, offering potential for extended dosing intervals in adults with moderate to severe atopic dermatitis.

Results from the Phase III FLAUR2 trial (NCT04035486) showed that Tagrisso combined with pemetrexed and platinum-based chemotherapy achieved a median overall survival of 47.5 months in patients with locally advanced or metastatic EGFR-mutated non-small cell lung cancer.

Results from the Phase III HARMONi trial (NCT06396065) showed that ivonescimab demonstrated a median overall survival rate of 16.8 months in patients with EGFR-mutated, locally advanced or metastatic non-squamous non-small cell lung cancer.

Results from a Phase III trial (NCT07069309) demonstrated that the LP.8.1-adapted Comirnaty vaccine generated a robust fourfold increase in neutralizing antibodies of COVID-19.

Phase IIa trial (NCT06413537) results show SPG601 improves neurophysiological and cognitive measures in patients with Fragile X syndrome.

Results from the Phase II EPCORE NHL-6 trial (NCT05451810) showed that Epkinly demonstrated an overall response rate of 64.3% in patients with relapsed or refractory diffuse large B-cell lymphoma.

Designation supports the potential of olomorasib plus Keytruda to address unmet needs in the first-line treatment of patients with KRAS G12C-mutant non-small lung cancer with high PD-L1 expression.

Results from the Phase IV V-DIFFERENCE trial (NCT05192941) found that 85% of patients on Leqvio reached guideline-recommended LDL-C targets versus 31% on placebo at 90 days.

Results from the Phase III CORALreef Lipids trial (CT05952856) showed that patients treated with enlicitide decanoate for hypercholesterolemia demonstrated statistically significant and clinically meaningful reductions in LDL cholesterol.

Results from the Phase III BaxHTN trial (NCT06034743) showed baxdrostat achieved statistically significant and clinically meaningful reductions in systolic blood pressure in patients with hard-to-control hypertension.

The ZENITH outcomes trial will enroll 11,000 patients worldwide to evaluate twice-yearly zilebesiran, aiming to improve long-term blood pressure control and reduce cardiovascular risk in uncontrolled hypertension.

The Phase III VICTOR trial (NCT05093933) evaluated Verquvo in patients with stable, well-treated chronic heart failure and reduced ejection fraction without a recent worsening heart failure event.

Breakthrough Therapy Designation was based on results from the Phase I/II RAINFOL-01 trial, which showed encouraging clinical activity in patients treated with Rina-S (rinatabart sesutecan) for advanced endometrial cancer.

Ampreloxetine is being developed to address the root cause of blood pressure instability in multiple system atrophy, aiming to improve patient outcomes and independence.

Results from the first part of a Phase I/II trial (NCT06569823) showed that Z-1018 generated immune responses comparable to Shingrix, with a 100% antibody response rate and nearly 90% CD4⁺ T-cell response in patients with shingles.

The Phase II EvolvE trial (NCT05774184) confirmed barzolvolimab’s mechanism of action but showed no measurable improvement in dysphagia symptoms or esophageal inflammation in patients with eosinophilic esophagitis.

With approval to proceed, the Phase II trial (NCT06092034) of RP-A501 for Danon disease restarts under an optimized dosing and immunomodulatory strategy.

Results from the Phase II OptiTROP-Lung01 trial (NCT05351788) showed that Sichuan Kelun-Biotech’s sac-TMT combined with tagitanlimab demonstrated promising anti-tumor activity and manageable safety as a first-line treatment for advanced or metastatic non-small cell lung cancer.

Breakthrough Therapy Designation was based on results from the Phase II IDeate-Lung01 trial (NCT05280470), which demonstrated clinically meaningful benefits in patients with extensive-stage small cell lung cancer treated with ifinatamab deruxtecan.

Ron Lanton, partner, Lanton Law, explains how shifts in policy and government guidance could reduce public participation and complicate the design and recruitment of future vaccine trials.

The trial’s first patient has been treated to assess the BREATHE Airway Scaffold, which was developed to enhance breathing and daily functioning in patients with severe emphysema.

Ron Lanton, partner, Lanton Law, explains why evolving federal guidance and new technologies are pushing clinical operations teams to strengthen oversight and safeguard the credibility of clinical research.

Results from the Phase II COLLECTiVE202 trial (NCT05370885) demonstrated a statistically significant difference between VE202 and placebo in producing endoscopic or clinical responses in patients with mild-to-moderate ulcerative colitis.

Jon Walsh, founder, chief scientific officer, Unlearn, explains how AI and digital twins are helping clinical trials become more efficient, patient-centric, and capable of supporting innovative study designs over the next decade.

Jon Walsh, founder, chief scientific officer, Unlearn, explains how AI and digital twin technologies are being applied to improve data transparency, ensure reproducibility, and strengthen the reliability of clinical trial outcomes.

Results from a Phase II trial (NCT06640972) showed that patients treated with RDX-002 experienced significant reductions in postprandial triglycerides, less weight regain after GLP-1 therapy, and improvements in key cardiometabolic markers.