Why PTC Therapeutics Shares Are Gaining Today

PTC Therapeutics Inc PTCT says its APHENITY Phase 3 trial of sepiapterin in adult and pediatric patients with phenylketonuria (PKU) has achieved the primary endpoint.

PKU is a birth defect that causes an amino acid called phenylalanine to build up in the body.

The placebo-controlled portion of the study included 98 patients in the primary analysis population. 

The mean percent Phe reduction in sepiapterin-treated patients was 63%. 

The mean percent Phe reduction was 69% in the subset of classical PKU patients. Minimal reductions in Phe levels were observed in the placebo-treated patients resulting in a highly statistically significant sepiapterin treatment benefit (p<0.0001). 

Sepiapterin was generally well tolerated with no serious adverse events.

"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," said Matthew Klein, Chief Executive Officer.

Price Action: PTCT shares are up 4.02% at $56.38 on the last check Wednesday.