APHENITY trial met primary endpoint with sepiapterin in PKU patients
23.05.2023 16:39
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced that the primary endpoint was met in the APHENITY pivotal Phase 3 clinical trial of sepiapterin in adult and pediatric patients with phenylketonuria (PKU).
“The positive results from the APHENITY trial bring us one step closer to providing a therapy that could provide meaningful benefit to PKU patients,” said Dr. med. Matthew B. Klein, Chief Executive Officer of PTC Therapeutics. “The Phe reductions observed in the placebo-controlled portion of the study are consistent with, and in some cases even exceed, the magnitude of the Phe reductions seen in the open-label portion of the study. We look forward to meeting with regulatory authorities in the US (FDA) and Europe (EMA) to discuss the path to approval.”
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 %. In the subgroup of classic PKU patients, the mean percent Phe reduction was 69 %. A minimal decrease in Phe levels was observed in placebo-treated patients, resulting in a highly statistically significant benefit of sepiapterin treatment (p0.0001). Sepiapterin was generally well tolerated with no serious adverse events.
About APHENITY
APHENITY was a global, double-blind, placebo-controlled, pivotal study that enrolled 156 children and adults with PKU. Participants were randomized to receive either Sepiapterin or placebo for six weeks, with the primary endpoint being reductions in blood phenylalanine levels. The study consisted of two parts. Part 1 was a run-in phase in which all subjects studied received Sepiapterin for two weeks. Only subjects who demonstrated 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 of the clinical trial. The primary analysis population will consist of those who had a reduction in phenylalanine levels of more than 30 % from baseline in Part 1 of the study. The primary outcome is the reduction in blood phenylalanine levels compared to baseline at weeks 5 and 6 in patients in part 2 of the clinical trial. All patients are eligible to participate in a long-term, open-label clinical trial to further evaluate the long-term safety and durable effects of Sepiapterin.
About PTC Therapeutics, Inc.
PTC is a science-based, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that benefit patients with rare diseases. PTC's ability to develop new therapies and commercialize products globally provides the foundation for investing in a robust and diverse pipeline of transformative medicines. PTC's mission is to provide access to best-in-class therapies for patients who have few or no treatment options. PTC's strategy is to leverage its strong scientific and clinical expertise and global commercial infrastructure to bring therapies to patients. PTC believes this allows it to maximize value for all of its stakeholders.