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Regulatory approval – first-in-class for ultra-rare Alexander disease
The FDA approved Ionis Pharmaceuticals’ antisense therapy Zanvastro (zilganersen) for pediatric and adult patients with Alexander disease, establishing the first disease-modifying treatment for...
Cardiovascular late-stage failure – pelacarsen
Novartis disclosed that pelacarsen failed to meet endpoints in a pivotal Phase 3 outcome trial, a major setback for the company’s Lp(a)-lowering strategy in preventing cardiovascular events. In...
Oncology – Phase 3 readouts reshape treatment risk-benefit
AbbVie reported a Phase 3 win for its T-cell engager etentamig in multiple myeloma, meeting the trial’s main goal. Company results indicated a 60% risk reduction for disease progression or death...
Oncology partnering – GSK licenses KRAS–EGFR antibody-targeted conjugate from Hutchmed
GSK agreed to license Hutchmed’s HMPL-A830, a KRAS–EGFR antibody-targeted therapy conjugate, in a deal valued at up to $1.295 billion. GSK will pay $110 million upfront for rights to develop and...
Trial failure reshapes rare neurodevelopment pipeline – Angelman syndrome
Ultragenyx said its Phase 3 Angelman syndrome trial, Aspire, failed both the primary endpoint and a key secondary endpoint for apazunersen (GTX-102). The company indicated it will evaluate next...
Cell and gene therapy safety – CAR-T trial pauses
Novartis halted CAR-T therapy trials after three patient deaths associated with severe autoimmune reactions following dosing with zola-cel for autoimmune conditions, according to the report....
Funding – NeuShen Series B to advance brain-health drugs
NeuShen Therapeutics raised more than $80 million in a Series B to advance three clinical-stage brain-health programs, spanning development from neurology to cognitive and neurological functional...
BTK inhibitors – remibrutinib enters multiple sclerosis regulatory race
Novartis’ remibrutinib delivered positive top-line Phase 3 results in relapsing multiple sclerosis, marking the second oral BTK inhibitor to post favorable Phase 3 readouts. The company’s data...
Antimicrobial strategy – phage therapy in a human micro-bladder model
Researchers at University College London used a human 3D micro-bladder model to test phage therapy against uropathogenic E. coli associated with recurrent urinary tract infections. The study...
Regulatory approvals – rare pediatric neurology
The U.S. FDA has approved Ionis Pharmaceuticals’ antisense therapy Zanvastro (zilganersen) for Alexander disease in both pediatric and adult patients, establishing the first and only...
Phase 3 – multiple myeloma bispecific win
AbbVie reported that its dual-acting multiple myeloma bispecific, etentamig, met the main goal in a Phase 3 trial, improving progression-free outcomes versus standard regimens in patients who had...
Phase 3 – rare neuro disorder setback
Ultragenyx said its Phase 3 Angelman syndrome trial failed, withdrawing optimism built on earlier-stage data for apazunersen (GTX-102). The company reported the Aspire study did not meet the...
Oncology partnerships and licensing – GSK adds KRAS/EGFR conjugate
GSK agreed to license Hutchmed’s HMPL-A830, an antibody-targeted therapy conjugate combining an EGFR-directed antibody with a small-molecule KRAS inhibitor payload. The deal is valued up to $1.295...
Regulatory supply chain – estrogen patch market pressure
A policy shift at the FDA has reshaped demand dynamics for estradiol transdermal patches, according to reporting tied to an apparent rethink of menopausal hormone therapy benefits. While...
Financing – NeuShen raises to advance brain-health programs
NeuShen Therapeutics, a Shanghai-and-Boston biotech led by a veteran transpacific drug developer, raised more than $80 million to advance three clinical-stage brain-health drugs. The funding,...
Financing – Superluminal obesity drug program
Superluminal Medicines closed an oversubscribed $60 million Series B to advance its obesity program in the clinic. The company is developing next-generation small-molecule GPCR-targeted therapies...
Cell & gene therapy – CAR-T trial holds after fatalities
Novartis halted CAR-T trials after three patient deaths linked to a severe autoimmune reaction following dosing with zola-cel (rapcabtagene autoleucel). The company said it is investigating...
Late-stage neuro-psychiatry preclinical translation – psilocybin to prevent CIPN
Researchers at The University of Texas MD Anderson Cancer Center reported preclinical evidence that psilocybin can prevent chemotherapy-induced peripheral neuropathy (CIPN) in mouse models. In...
Clinical trial evidence – pulmonary infection origin changes outcomes
A retrospective study from China suggests pleural infection outcomes differ meaningfully based on whether cases are hospital-acquired or community-acquired. The findings challenge the assumption...
Regulatory approval: rare neurodevelopmental disease
Ionis Pharmaceuticals has won FDA approval for Zanvastro, the first disease-modifying therapy for Alexander disease. The agency approved the antisense therapy for children and adults after a...