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Morning. Today, we discuss the implications of a child’s tragic death after receiving a brain-focused gene therapy, hear Siddhartha Mukherjee opine on why the next frontier in cancer treatment is metabolic, and more.
The need-to-know this morning
- Kyverna Therapeutics said its patient-specific CAR-T therapy improved walking ability in patients with stiff person syndrome, an autoimmune disease. The results achieved the primary goal of the pivotal clinical trial and will support a marketing submission to the FDA next year.
- Argenx discontinued a Phase 3 study of Vyvgart Hytrulo in thyroid eye disease after independent data monitors conducted an interim analysis and concluded the drug would not achieve the study’s primary goal.
- A drug developed by Immunome reduced the risk of disease worsening or death by 84% compared to placebo in patients with advanced desmoid tumors. The results achieved the primary goal of the Phase 3 study. Immunome plans to seek FDA approval next year.
- Milestone Pharmaceuticals won FDA approval for a new nasal-spray medicine to treat episodes of paroxysmal supraventricular tachycardia, a a common type of abnormal heart rhythm.
Brain-targeted gene therapy’s sobering reckoning
A long-anticipated breakthrough in gene therapy for brain diseases has been jolted by tragedy after the first patient dosed with a new, brain-penetrating virus — developed by Capsida Biotherapeutics — died days later from cerebral edema. This outcome wasn’t predicted by the extensive animal testing that led up to the study, STAT’s Jason Mast writes.
Continue to STAT+ to read the full story…
STAT Pharma: The science and business of new drug development
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