Date: 2018-01-05

Type of information: Development agreement

Compound: CNSA-001

Company: Retrotophin (USA - CA) Censa Pharmaceuticals (USA - MA)

Therapeutic area: Rare diseases - Genetic diseases - Metabolic diseases

Type agreement: development

Action mechanism:

  • CNSA-001 is an orally bioavailable proprietary form of sepiapterin, a natural precursor of tetrahydrobiopterin (BH4) that is converted by an endogenous enzymatic pathway to BH4. Preclinical research has suggested CNSA-001 may provide improved bioavailability, plasma stability and tissue exposure, leading to higher intracellular BH4 levels and subsequent greater phenylalanine (Phe) reduction when compared to the current standard of care in PKU. In pre-clinical models, sepiapterin has also shown an ability to cross the blood-brain barrier which, if supported by clinical data, may lead to broader utility in additional indications such as primary BH4 deficiency (PBD) and Segawa syndrome.

Disease: phenylketonuria


  • • On January 5, 2018, Retrophin and Censa Pharmaceuticals announced a joint development agreement to evaluate CNSA-001 for the treatment of phenylketonuria (PKU). CNSA-001 is an orally bioavailable form of a natural precursor of tetrahydrobiopterin (BH4) with the potential to provide improved phenylalanine (Phe) reduction in patients with PKU when compared to BH4. CNSA-001 is currently being evaluated in a single ascending dose (SAD) study and a Phase 2 proof of concept study in PKU is expected to commence in mid-2018.

Financial terms:

  • Under the terms of the agreement, Retrophin is making a $10 million upfront payment to Censa and will provide funding for development of CNSA-001 in PKU. Censa will run the development program, which will be conducted under the oversight of a joint steering committee. As part of the agreement, Retrophin will pay certain milestone payments and obtains the exclusive option to acquire Censa upon conclusion of a specified option period, pending clinical proof of concept of CNSA-001 in PKU.

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Is general: Yes