Cytokinetics' aficamten meets both primary endpoints in the Phase 3 ACACIA-HCM trial in non-obstructive hypertrophic cardiomyopathy, Takeda and Protagonist's Mimrylo receives FDA approval as the first hepcidin mimetic for polycythaemia vera, Gilead's Bixlenvo is...
Autoimmune CAR-T safety is under review after Novartis halted eight rap-cel studies and Bristol Myers Squibb paused zola-cel enrolment.
A single screen failure tells us something about a patient. A pattern of screen failures may tell us something about the trial. Clinical research is very good at studying the patients who make it into a trial. We analyze their baseline characteristics, laboratory...
BioNTech and Genentech have stopped a Phase 2 trial of autogene cevumeran in colorectal cancer. An independent data safety monitoring board recommended the termination. The companies confirmed the decision on 28 August 2026. It arrives nine days after a landmark...
Revolution Medicines’ Rasonque receives FDA approval as what the FDA calls the first in class targeted therapy for metastatic pancreatic cancer, Priovant’s Lisraya is cleared as the first oral therapy indicated for dermatomyositis in adults, Johnson & Johnson’s...
Merck and Moderna have reported what they describe as the first positive Phase 3 result for an individualised cancer therapy. Intismeran autogene, given with pembrolizumab, met its primary endpoint in resected melanoma. The companies announced the outcome on 19 August...
There’s a lot of discussion right now around AI in healthcare, but most of it feels disconnected from how clinical research actually works day-to-day. A lot of the conversation is centered around automation, replacing people, chatbots, and efficiency. Some of that...
Merck's sacituzumab tirumotecan meets both primary endpoints in the pivotal Phase 3 TroFuse-005 trial for advanced endometrial cancer, Relay Therapeutics reports the first clinical evidence of PI3Kα mutant-selective inhibition in PIK3CA-driven vascular anomalies in...
Each year on May 20, International Clinical Trials Day recognizes James Lind’s 1747 scurvy trial and the patients, clinicians, coordinators, sponsors and researchers who make clinical progress possible. It is also a moment to look honestly at where clinical research...
The FDA's approval of Veppanu (vepdegestrant) marks the first regulatory clearance for a PROTAC in any disease area, and opens a new chapter in the treatment of ESR1-mutated advanced breast cancer. On 1 May 2026, the United States Food and Drug Administration approved...
Autoantibody testing is a central part of autoimmune disease diagnosis, including Sjögren’s disease (SjD). The American College of Rheumatology and European Alliance of Associations of Rheumatology (ACR/EULAR) classification criteria for SjD incorporate ocular and...
AstraZeneca's eneboparatide meets the Phase 3 CALYPSO primary endpoint in chronic hypoparathyroidism with full data presented at the European Congress of Endocrinology, Alkermes' LUMRYZ meets its primary endpoint in the Phase 3 REVITALYZ trial for idiopathic...
Alzheimer’s disease (AD) represents a significant medical concern for all of us as we grow older, as the NIH has published that the average risk of dementia over 55 years old is now 42%. AD is presently affecting 6.9 million Americans aged 65 or older and is expected...
Cytokinetics’ aficamten meets dual primary endpoints in the pivotal Phase 3 ACACIA-HCM trial for non-obstructive hypertrophic cardiomyopathy, Axsome Therapeutics secures FDA approval of AUVELITY as the first non-antipsychotic treatment for Alzheimer’s disease...
Children have long been described as therapeutic orphans. It is a term first used by the American paediatrician Harry Shirkey of the Children’s Hospital in Birmingham, Alabama, at a 1963 conference and formally published in 1968. Despite more than five decades of...
For decades, clinical trials have failed to adequately represent the patients who will ultimately use the medicines and treatments being tested. Gaps in enrolment across race, ethnicity, sex, age, and socioeconomic background have created an incomplete evidence base,...
Etavopivat delivers Phase 3 success in sickle cell disease, satralizumab becomes the first positive Phase 3 result in MOGAD, AstraZeneca completes its triple-positive COPD biologic programme, and a busy week for paediatric medicine brings new approvals and data in...
Speech is rapidly emerging as an important, non-invasive, digital clinical marker in neurodegenerative disease research, according to leading biotech SynaptixBio. The company has facilitated a study run by Australia-based speech analytics company Redenlab, in...