French pharma Trophos has reported positive top-line data from its pivotal Phase II/III study of olesoxime in spinal muscular atrophy (SMA), a product the company has resolutely backed despite past setbacks.
French pharma Trophos has reported positive top-line data from its pivotal Phase II/III study of olesoxime in spinal muscular atrophy (SMA), a product the company has resolutely backed despite past setbacks.
Olesoxime, a cholesterol-like compound that targets and preserves mitochondrial integrity and function in stressed cells, was shown to prevent loss...
A new muscle-building front is opening in the fight against obesity, with Lilly among the leaders. But it plans to remain tight-lipped on upcoming Phase II data for its myostatin inhibitor in combination with Novo’s Wegovy for fear of playing into the hands of its rival.
The Mereo-partnered drug is in pivotal development for osteogenesis imperfecta. Ultragenyx also recently reported positive Phase III data for a rare disease gene therapy.
The company sees a $5bn opportunity to treat children with achondroplasia and the less severe growth disorder variant, hypochondroplasia.
Phase I/II data for its ADC candidates in myotonic dystrophy type 1 and Duchenne muscular dystrophy indicate potential for biomarker-based accelerated approval in both diseases, Dyne asserts.
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Pipelines in bioconjugates continue to develop, from ADCs to XDCs, with radionuclide drug conjugates (RDCs) and antibody-oligonucleotide conjugates (AOCs) leading the expansion.
Interim results from the Phase III REMARK study presented at WCLC showed the Chinese company’s second-generation ALK/ROS1 inhibitor met its primary endpoint as a first-line therapy in ALK-positive advanced NSCLC.
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