Curated news and analysis on clinical trials, drug approvals, and medical research.
The FDA said uniQure’s Phase I/II data for Huntington’s disease gene therapy AMT-130 are not adequate for a marketing application. The agency recommended a new randomised, double-blind, sham surgery-controlled study.
A publication in NEJM AI outlined seven human factors-related risks for AI-enabled medical devices and called for clearer workflows, training, fallback options and post-market monitoring. The analysis said current approval requirements only partially address these risks, while separate evidence cited persistent accuracy and verification problems in medical AI use.
TMEM87A was identified as a mediator of ferroptosis resistance through buffering Golgi pH. In murine tumor models, TMEM87A ablation suppressed tumor progression and potentiated PD1 blockade therapy.
Karmanos and the Detroit Public Library have opened registration for the 2026 Community Conversations on Cancer series. Three sessions will address colon cancer, implicit bias in care, and multiple myeloma.
Wafik S. El-Deiry highlighted p53 research and the August 2025 approval of dordaviprone/modeyso, the first treatment for diffuse gliomas with H3K27M mutations. Separate posts also described glioblastoma precision oncology discussions and a lifetime achievement award in Mumbai.
GRI Bio discussed positive Phase 2a idiopathic pulmonary fibrosis data for GRI-0621, highlighting biomarker results indicating improvements in fibrosis and lung repair.
Merck reported Phase 2 CADENCE results showing WINREVAIR met the primary endpoint in adults with CpcPH-HFpEF. The company said the data support advancement into a registrational Phase 3 study.
Rua Gold filed a fast-track application for its Auld Creek project in New Zealand’s Reefton Goldfield. A decision is expected within three months, with studies and permitting work advancing in parallel.
Rua Gold submitted a referral application for fast-track permitting for its Auld Creek underground mine and processing plant near Reefton. A decision is expected by mid-July.
Evidence on ketogenic diets remains limited for psychiatric disorders and diabetes, while a systematic review in overweight and obese women with PCOS found significant short-term improvements. Experts said more controlled, long-term studies are needed.
Eli Lilly and Company has agreed to acquire Orna Therapeutics for up to $2.4 billion in cash. The deal includes ORN-252, a clinical-trial-ready CD19-targeting in vivo CAR-T therapy for B cell-driven autoimmune diseases.
BridgeBio Oncology Therapeutics said Bbo-11818 received FDA Fast Track designation for adult patients with advanced KRAS-mutated pancreatic ductal adenocarcinoma. The status may accelerate development and review and allow more frequent FDA communication during clinical trials.
Two osteosarcoma studies identified prognostic immune biomarkers, including serum sB7-H3 and an immune cell infiltration score. Both linked immune features with survival, metastasis and treatment response.
MicroOrganoSpheres predicted treatment success in 83% of 21 advanced colorectal cancer cases, reaching 100% accuracy with primary-tumor samples. The automated test can be performed within days.
A securities class action alleges Corcept misled investors about the regulatory prospects for relacorilant. The suit cites an FDA Complete Response Letter, a 50.4% stock drop, and an April 21, 2026 lead plaintiff deadline.
VYLOY (zolbetuximab-clzb) remains the only approved CLDN18.2-targeted therapy for first-line HER2-negative gastric or gastroesophageal junction adenocarcinoma with CLDN18.2-positive tumors. The pipeline is focused on next-generation antibodies, ADCs, CAR-T, NK-cell and bispecific approaches aiming to build on zolbetuximab benchmark data.
Xenon Pharmaceuticals reported positive Phase III X-TOLE2 results for azetukalner in focal onset seizures. The company plans an FDA new drug application in Q3 2026.
Compass Pathways reported positive late-stage COMP360 psilocybin data in treatment-resistant depression and plans FDA talks ahead of a filing between October and December. The update lifted shares across several psychedelics developers.
A cost-effectiveness analysis found home-based anal cancer screening increased participation versus clinic-based screening among sexual and gender minority individuals. The approach remained investigational because the FDA has not approved self-sampling for anal cancer screening.
The Trump administration has agreed to release federal funding for the $16 billion Gateway rail tunnel and Second Avenue Subway Phase 2, ending months-long suspensions that halted work and caused layoffs. Gateway funding had been suspended for over four months, leaving $205 million owed and forcing 1,000 layoffs, while Second Avenue Subway funds were withheld for seven months pending compliance reviews.
