Censa Pharmaceuticals Announces CNSA-001 Met Primary and Secondary Endpoints in Phase 2 Trial in Patients with PKU

WELLESLEY, Mass., Dec. 3, 2019 /PRNewswire/ -- Censa Pharmaceuticals, Inc. ('Censa") today announced positive results from its Phase 2 trial for CNSA-001 (sepiapterin), a potential new medicine for the treatment of phenylketonuria (PKU). CNSA-001 met its primary and secondary endpoints, achieving a statistically-significant and clinically-meaningful reduction in blood phenylalanine (Phe) levels compared to both baseline and an active control group (Kuvan®, sapropterin dihydrochloride).

Phenylketonuria 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 Phe leading to potentially toxic levels of Phe in the blood and serious health problems as a result.

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.

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

Dr. Drago Bratkovic, Unit Head Metabolic Clinic, Women's and Children's Hospital and University of Adelaide, Australia, and Principal Investigator on the CNSA-001 Phase 2 trial said: "The Phase 2 study data demonstrate that CNSA-001 could become a first line pharmacological therapy for responsive patients with PKU."  

Data from the Phase 2 study of CNSA-001 will be presented at The American College of Medical Genetics and Genomics (ACMG) Meeting on March 18th, 2020.

Previous preclinical and healthy volunteer studies showed that an oral dose of CNSA-001 increased target tissue and plasma tetrahydrobiopterin more effectively than equivalent oral doses of sapropterin dihydrochloride (Smith N et al., Mol Genet Metab 2019;126:406-12). Oral CNSA-001 administration also increased neurotransmitter production and corrected abnormally low levels of neurotransmitter metabolites in an individual human subject. (Smith N et al., Mol Genet Metab R 2019;21:100500).

About Censa Pharmaceuticals and CNSA-001 (sepiapterin)

Censa Pharmaceuticals, Inc. is a late-stage pharmaceutical company developing CNSA-001, an oral first-line therapy for phenylketonuria (PKU) patients across all ages and disease severities. CNSA-001 has demonstrated excellent tolerability and efficacy with clear superiority over sapropterin in a Phase 2 study. CNSA-001 is also being studied in other diseases associated with defects in the tetrahydrobiopterin biochemical pathways.

For more information, please visit www.censapharma.com and follow us on Twitter @CensaPharma

KUVAN® is a registered trademark of BioMarin Pharmaceutical Inc.

SOURCE Censa Pharmaceuticals, Inc.

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