PTC’s Phenylketonuria Treatment Meets Phase III Endpoint

May 17, 2023

PTC Therapeutics announced positive results from a phase III clinical trial for its drug sepiapterin in the treatment of phenylketonuria (PKU), a rare disease causing excessive accumulation of the amino acid phenylalanine. The drug met the primary endpoint of reducing blood levels of phenylalanine in both adult and pediatric PKU patients in the trial.

According to Paul Schloesser, “PTC conducted the trial in two parts. The first part was a run-in phase, where screened patients were on sepiapterin for 14 days. After those two weeks, the second phase kicked in — the study took participants who showed at least a 15% reduction in phenylalanine levels in the blood and randomized them to receive either the drug or placebo. The primary analysis population of 98 patients only showed patients who showed a 30% reduction from baseline in part 1 of the trial, the company said.”

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