PTC Therapeutics, Inc. (PTCT), Thursday announced new data of the Phase 3 APHENITY trial and subsequent open-label extension study, evaluating the potential meaningful benefits of sepiapterin treatment in phenylketonuria or PKU patients.
Sepiapterin has a dual mechanism of action to increase activity of the phenylalanine hydroxylase enzyme in patients with PKU, a rare, inherited metabolic disease.
The new data revealed that over 97 percent of trial participants demonstrated the ability to liberalize their diet while on sepiapterin treatment, with a mean increase in protein intake of 126 percent.
Meanwhile, 66 percent of candidates participating in the Phe tolerance sub study reached or exceeded the age-adjusted recommended daily allowance of protein intake for an individual without PKU, while maintaining control of blood Phe levels.
Additionally, genetic variant analysis of subjects participating in the APHENITY study demonstrates that over 70 percent had a Genotype-Phenotype Value consistent with classical PKU.
The biopharmaceutical company noted that the new data provide further evidence of the meaningful benefit sepiapterin can provide for children and adults with PKU, including those with the most severe form of the disease.
In the pre-market hours, PTC's stock is trading at $57.24, up 0.46 percent on the Nasdaq.
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