Adebola Giwa, MD
- Associate Medical Director, Clinical Development
- 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 our core values of patients, science and passion, we use our TransCon™ technology to fulfill our mission of developing new and potentially best-in-class therapies that address unmet medical needs.
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.
Achondroplasia (ACH) is the most common form of dwarfism, caused by a genetic mutation in the fibroblast growth factor receptor 3 (FGFR3). This leads to an imbalance between the stimulatory and inhibitory signaling pathways involved in regulating bone growth.
People living with ACH may experience serious complications and comorbidities due to inhibited skeletal development. Complications may include sleep apnea and respiratory problems, chronic back and leg pain from lower spine impingement, and sudden infant death from compression of the brain stem. Chronic ear infections due to eustachian tube problems can lead to hearing loss and speech delay. Children with ACH may also experience social and emotional challenges.
There is currently no approved medical therapy for ACH for those who may choose a therapeutic option to manage its complications.
Committed to understanding achondroplasia
We are conducting the global ACHieve Study as one way to inform our development of TransCon CNP and learn about the experiences of children living with this rare condition. No study medication is given in ACHieve.
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.
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.
TransCon™ technology offers a potential solution to achieve this goal.
TransCon CNP is an investigational 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.
We are 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, we 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
We are 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 our TransCon technology, we are opening up new frontiers to investigate CNP in our future research.
TransCon CNP for Achondroplasia
The latest information on the development of TransCon CNP for patients with achondroplasia (ACH). TransCon CNP is an investigational long-acting prodrug of CNP in development for the treatment of ACH in children.