2023-05-17 07:32:24 ET
PTC Therapeutics' ( NASDAQ: PTCT ) drug sepiapterin met the main goal of a phase 3 trial to treat adults and children with phenylketonuria (PKU).
PKU is an inherited metabolic disorder that causes an amino acid called phenylalanine to build up in the body. It is caused by a change in the phenylalanine hydroxylase (PAH) gene, which helps create an enzyme needed to break down phenylalanine. The disorder can cause intellectual disability, seizures, delayed development, memory loss, and behavioral issues.
The late-stage study, dubbed APHENITY, enrolled 156 children and adults with PKU. The trial consisted of two parts and only those patients who showed a reduction in phenylalanine levels of 15% or more from baseline in Part 1 were randomized to receive either sepiapterin or placebo in Part 2. The main goal was reduction in blood phenylalanine levels.
The placebo-controlled portion of the trial included 98 patients in the primary analysis population.
The company said the average percentage Phe reduction for those on sepiapterin was 63%. Meanwhile, in a subset of classical patients with PKU, the mean percent Phe reduction was 69%.
Minimal reductions in Phe levels were seen for those on placebo thus showing a highly statistically significant benefit from sepiapterin treatment, the company added.
"The Phe reductions observed in the placebo-controlled portion of the study are consistent with, and, in some cases, exceed the magnitude of Phe reductions recorded in the open label portion of the study. We look forward to meeting with regulatory authorities to discuss the path to approval," said PTC CEO Matthew Klein.
PTC noted that sepiapterin was generally well tolerated with no serious adverse events.
According to the company sepiapterin (formerly PTC923) is an oral formulation of synthetic sepiapterin, a precursor to intracellular tetrahydrobiopterin.
PTCT +3.04% to $55.85 premarket May 11
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PTC Therapeutics' metabolic disorder drug sepiapterin meets main goal in phase 3 trial