Also, CRO Syneos Health appointed an executive to its newly created DE&I position, while Alterity started a new Phase II study in multiple system atrophy, Antabio dosed the first patients in its Phase I study of an antibiotic for hospital infections, and Structure Therapeutics started a Phase IIa trial of its GLP-1 agonist in obesity and diabetes, with plans for a Phase IIb trial in 2024.

The drug showed efficacy in hemophilia A and B with no sign of clots, an issue that historically has dogged anticoagulation agents, but it remains unclear whether that advantage will hold.

AstraZeneca plans to unveil multiple major data packages, while Servier reveals much-anticipated results in glioma, and ImmunoGen and Novartis will present key pivotal data in women’s cancers.

The ARCHER study did not show improvement in lesion growth, but still showed an improvement in best corrected visual acuity, potentially opening a pathway toward a pivotal trial.

The US FDA gave the drug a broad label in adults with indolent SM, without restrictions for disease severity or prior treatment, and Blueprint sees a potential $1.5bn market opportunity.

The company estimates there are about 9,000 reimbursable patients with dystrophic epidermolysis bullosa worldwide and anticipates a third-quarter launch following FDA approval.