Tempus AI’s FDA-cleared ECG-PH software turns an ordinary 12-lead electrocardiogram into a machine-learning risk signal for pulmonary hypertension. The clinical-performance data show why the technology could be useful for ruling out disease and prioritising follow-up, while also explaining why it cannot replace echocardiography or right-heart catheterisation.
PTC Therapeutics secured Sangamo Therapeutics’ ST-920 Fabry disease programme through a bankruptcy auction for $111 million upfront plus up to $100 million in regulatory milestones. The unusual transaction gives PTC a near-registration gene therapy at a fraction of the cost of many biotechnology acquisitions, but regulatory, manufacturing and commercial risks remain concentrated.
Curium’s agreement to acquire Lantheus Holdings combines a major United States radiodiagnostics franchise with Curium’s global isotope, manufacturing and nuclear-medicine infrastructure. The strategic logic goes well beyond one imaging agent and points toward increasing value being placed on end-to-end radiopharmaceutical capabilities.
ISEMBYLD is not replacing the therapies that transformed spinal muscular atrophy treatment. Scholar Rock is asking physicians and payers to add a muscle-targeted medicine on top of them, creating a commercial proposition that could expand the market while also making reimbursement and treatment logistics central to adoption.
Novartis’ acquisition of Avidity Biosciences brought three late-stage antibody oligonucleotide conjugate programmes into its neuromuscular pipeline. The failure of del-desiran’s Phase 3 HARBOR study now forces investors to separate a damaging lead-asset setback from the value that may remain elsewhere in the platform.
IDEAYA and Servier begin a 450-patient Phase 3 trial testing whether darovasertib plus crizotinib can prevent uveal melanoma recurrence.
Tyra Biosciences’ dabogratinib showed encouraging single-lesion responses in SURF302, but mixed broader data sent shares sharply lower.
BioMarin’s VOXZOGO met its Phase 3 hypochondroplasia endpoint with a 2.33 cm/year growth velocity benefit as an FDA filing moves forward.
LUMA Vision expands FDA clearance for VERAFEYE, integrating cardiac imaging, 3D anatomy and catheter tracking ahead of its planned 2027 launch.
Acarix heart failure algorithms showed strong sensitivity and fewer false positives than NT-proBNP, but larger external validation is still required.