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Biotech firm PTC Therapeutics said Wednesday that its drug for phenylketonuria, or PKU, met its key goal in a Phase 3 study.

In PKU, patients’ bodies are unable to break down the amino acid phenylalanine, resulting in intellectual disability, memory problems, and other neurological issues. In the 156-volunteer trial, the drug, sepiapterin, reduced levels of phenylalanine by 63% compared to a placebo, PTC said in a press release. The company did not release detailed results, but said that no severe adverse events occurred in the study.


Brian Abrahams, an analyst at RBC Capital Markets, wrote that the results appear to meet high expectations that investors had for the drug. He’d previously said that a 40% to 50% reduction in phenylalanine, or “Phe,” levels would be clinically important.

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