Capsida Biotherapeutics, Sobi, GSK: Readout Newsletter
- This week in biotech: a serious adverse event halts progress in brain-focused gene therapy, promising results emerge for autoimmune conditions and rare cancers, and a new treatment gains...
- A promising field of gene therapy for brain diseases has faced a important setback following the death of the first patient treated with a novel, brain-penetrating viral vector...
- The death underscores the inherent risks of pioneering therapies, especially those targeting the central nervous system.
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Biotech Roundup: Gene Therapy Setback, Clinical Trial Updates & More
Table of Contents
This week in biotech: a serious adverse event halts progress in brain-focused gene therapy, promising results emerge for autoimmune conditions and rare cancers, and a new treatment gains FDA approval for heart rhythm disorders.
The Need-to-Know This Morning
- Kyverna Therapeutics announced positive topline data from a pivotal clinical trial of its patient-specific CAR-T therapy for stiff person syndrome, an autoimmune disease. The therapy demonstrated improved walking ability in patients, achieving the primary endpoint and supporting a planned marketing submission to the FDA next year.
- Argenx discontinued a Phase 3 study of Vyvgart Hytrulo in thyroid eye disease following an interim analysis indicating the drug was unlikely to meet its primary goal.
- Immunome reported that its drug,varegacestat,reduced the risk of disease worsening or death by 84% compared to placebo in patients with advanced desmoid tumors. The company plans to seek FDA approval in the coming year.
- Milestone Pharmaceuticals received FDA approval for Cardamyst (etripamil), a novel nasal spray for the acute treatment of paroxysmal supraventricular tachycardia (PSVT), a common type of abnormal heart rhythm.
Brain-Targeted Gene Therapy’s Sobering Reckoning
A promising field of gene therapy for brain diseases has faced a important setback following the death of the first patient treated with a novel, brain-penetrating viral vector developed by Capsida Biotherapeutics. The patient died days after receiving the therapy from cerebral edema – brain swelling. This adverse event was not predicted during extensive preclinical animal studies, raising critical questions about the translation of safety data from animals to humans, as reported by STAT’s Jason Mast.