Rare Daily Staff

Censa Pharmaceuticals reported positive results from its phase 2 trial for CNSA-001 sepiapterin, its experimental medicine for the treatment of phenylketonuria.

CNSA-001 met its primary and secondary endpoints, achieving a statistically-significant and clinically-meaningful reduction in blood phenylalanine levels compared to both baseline and an active control group taking BioMarin Pharmaceuticals’ Kuvan.

Phenylketonuria (PKU) is a rare, inherited metabolic disorder that is caused by mutations in the phenylalanine hydroxylase (PAH) gene. Patients with mutations in the PAH gene inefficiently metabolize phenylalanine (Phe) leading to potentially toxic levels of Phe in the blood that can cause severe intellectual disabilities. Newborn screening is available for this disorder.

“There is a significant unmet need for an oral therapy that is effective in both mild and severe PKU patients,” said Jonathan Reis, CEO of Censa Pharmaceuticals. “Our preliminary data suggests that CNSA-001 better controls blood Phe levels and has the potential to improve neurocognitive symptoms more effectively than current standard of care therapies.”

The phase 2 trial of CNSA-001 was a randomized, multicenter, three-period crossover, open-label, active-controlled study of CNSA-001 in 24 patients with PKU. Reduction in blood Phe in patients treated with CNSA-001 was consistent across all baseline levels of blood Phe. CNSA-001 was well tolerated with no serious adverse events or study discontinuations. The primary endpoint was change in blood Phe concentration and the secondary endpoints were safety and tolerability.

 “The phase 2 study data demonstrate that CNSA-001 could become a first line pharmacological therapy for responsive patients with PKU,” said Drago Bratkovic, unit head of the Metabolic Clinic, Women’s and Children’s Hospital and University of Adelaide, Australia, and principal investigator of the study.

Data from the phase 2 study of CNSA-001 will be presented at The American College of Medical Genetics and Genomics (ACMG) Meeting in March 2020.

Photo: Jonathan Reis, CEO of Censa Pharmaceuticals

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