ASCENDIS PHARMA

Ascendis Pharma is applying its innovative platform technology to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by their core values of patients, science and passion, they use their TransCon^® technology to fulfill our mission of developing new and potentially best-in-class therapies that address unmet medical needs.¹

transcon cnp – DESIGNED FOR CONTINUOUS CNP EXPOSURE TO REBALANCE BONE GROWTH IN SKELETAL DISORDERS

C-type natriuretic peptide (CNP) is a naturally occurring peptide that regulates bone growth. It has shown promise as a therapeutic target for treating growth disorders and skeletal disorders such as dwarfism.²

COMMITTED TO UNDERSTANDING ACHONDROPLASIA
Ascendis is conducting the global ACHieve Study as one way to inform their development of TransCon CNP and learn about the experiences of children living with this rare condition. No study medication is given in ACHieve.²

CNP MAY REBALANCE BONE GROWTH
CNP has been shown to counteract the growth-inhibiting effects of the FGFR3 mutation and stimulate growth. Increasing CNP levels to modulate excessive FGFR3 signaling and rebalance the pathways that regulate growth may reduce the complications and comorbidities of ACH.²

Yet CNP has a very short half-life. When administered as a therapeutic, it does not remain in the body long enough to exert an effect without assistance. This has prevented it from being effectively developed as a treatment. We believe prolonged exposure of CNP maximizes its potential as a therapeutic to address the medical complications related to ACH.²

TransCon^® technology offers a potential solution to achieve this goal.²

transcon cnp

TransCon technology has extended exposure of CNP from 2.5 minutes to 120 hours – unlocking its therapeutic potential to pursue a new frontier of growth biology

TransCon CNP is an investigational long-acting prodrug of CNP in development for the treatment of ACH in children. Designed to provide continuous CNP exposure, the goal of TransCon CNP is to optimize efficacy with a safe and convenient once-weekly dose. TransCon CNP has orphan designation for the treatment of ACH in both the U.S and Europe.

PROGRAM STATUS
Ascendis is conducting the ACcomplisH Trial, a global phase 2 clinical trial designed to evaluate the safety and efficacy of TransCon CNP in children with ACH from ages two to ten. In addition to height, they are evaluating various endpoints, including body proportionality and patient-reported outcome (PRO) measures using a tool developed with extensive input from those affected by ACH. These measures provide patient-related perspectives on the impact of a disease or treatment.

THE NEW FRONTIER OF GROWTH
Ascendis is committed to leveraging the CNP pathway not only for achondroplasia but for other growth disorders. With the possibility of a sustained release profile and continuous exposure enabled by their TransCon technology, they are opening up new frontiers to investigate CNP in their future research.

Image: https://ascendispharma.us/pipeline/endocrinology/transcon-cnp/#tranconcnp

The ACHieve Study is a natural history study designed to gain insight into the experience of children with ACH and achieve a greater global knowledge of ACH.