Researchers reported preclinical efficacy data for CDX-6114, an oral phenylalanine ammonia lyase in a large-animal phenylketonuria model. Presented at the Society for the Study of Inborn Errors of Metabolism (SSIEM), the update adds nonclinical support for CDX-6114’s potential to reduce phenylalanine exposure using an enzyme-based mechanism. The data focus on pharmacologic activity and efficacy in a model expected to better reflect real-world physiology than smaller species. For PKU programs, sustained phenylalanine control is a key differentiator, given the long-term dietary and treatment burden for patients. The large-animal findings move the program further along the translational path, helping de-risk next steps in clinical development.
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