Neuroscience
A report from analysts at Jefferies suggested that new screenings for metachromatic leukodystrophy and Duchenne muscular dystrophy could bump sales of the gene therapy Libmeldy by more than $100 million.
The Massachusetts biotech will also be eligible to milestone payments, though the exact amount has yet to be disclosed.
The star of the agreement targets a specific type of tau protein, helping to prevent toxic accumulation in the brain while also preserving the function of healthy tau.
Kyverna plans to submit mivocabtagene autoleucel to the FDA for approval in the first half of 2026. If approved, it would be the first CAR T therapy for an autoimmune disease.
Sanofi’s multiple sclerosis hopeful tolebrutinib faced dual setbacks on Monday, with a late-stage failure in one form of the disease and yet another regulatory setback in another.
In the midst of regulatory and political upheaval, biopharma’s R&D engine kept running, churning out highs and lows in equal parts. Here are some of this year’s most glorious clinical trial victories.
Coming up in the back half of December, the FDA will issue a verdict on Vanda Pharmaceuticals’ gastroparesis drug tradipitant, which it rejected last September, triggering a very public dispute with the company.
Every year in biopharma brings its share of grueling defeats, and 2025 was no different, especially for companies targeting neurological diseases. Some failures split up partners, and one particularly egregious case even led to the demise of an entire company.
In a first for the Parkinson’s field, AC Immune’s immunotherapy has stabilized key biomarkers that suggest an effect on the disease’s course.
Pfizer deals again in obesity space as Wave and Structure drop splashy weight loss results; what CDER Director Richard Pazdur’s sudden retirement means for biopharma; neuro diseases take center stage at CTAD; and more.
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