Dysregulation of metabolic pathways due to various gene mutations can lead to either shortages of necessary end products, or buildup of toxic constituents, either of which may lead to debilitating diseases.
CNSA-001 is an oral formulation of synthetic sepiapterin, a precursor to intracellular tetrahydrobiopterin, a critical enzymatic cofactor involved in the metabolism and synthesis of numerous metabolic products.
CNSA-001 is being developed as a possible treatment for orphan metabolic diseases associated with defects in the tetrahydrobiopterin biochemical pathways including phenylketonuria and primary BH4 deficiency.
Developing Medicines to Improve the Lives of Patients with Debilitating Diseases
Censa Pharmaceuticals Announces CNSA- 001 Met Primary and Secondary Endpoints in Phase 2 Trial in Patients with PKU
Positive results from Phase 2 trial demonstrate a statistically-significant and clinically-meaningful reduction in Phenylalanine Wellesley (MA) – December 3, 2019 – 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 […]
WELLESLEY, MA – November 1st, 2018, Censa Pharmaceuticals Inc., a biopharmaceutical company focused on the development of CNSA-001 for orphan diseases, announced that it has successfully completed a Phase I study and has initiated enrollment into several Phase II studies. The Phase I study showed CNSA-001’s favorable safety profile and promising pharmacokinetic & pharmacodynamic characteristics. […]