TransCon™: A powerful technology platform central to our innovative approach
At the core of Ascendis Pharma is a powerful, flexible technology platform that uses advanced knowledge of chemistry to overcome challenges of developing new therapeutics. When applied using known biology, this technology platform helps us address unmet patient needs in unprecedented ways.
TransCon = transient conjugation
TransCon refers to “transient conjugation,” or our unique ability to temporarily (transiently) link an inert carrier to a parent drug with known biology.
Depending on the carrier used, TransCon prodrugs can be designed to act systemically (throughout the body) or locally (e.g. intratumorally), focused on meeting a specific therapeutic goal. TransCon is widely applicable to proteins, peptides or small molecules in multiple therapeutic areas1-3.
- Karpf, D.B., Pihl, S., Mourya, S., Mortensen, E., Kovoor, E., Markova, D. and Leff, J.A. (2020). A Randomized Double‐Blind Placebo‐Controlled First‐In‐Human Phase 1 Trial of TransCon PTH in Healthy Adults. Journal of Bone and Mineral Research, 35(8), pp.1430–1440. doi:https://doi.org/10.1002/jbmr.4016.
- Khan, A.A., Rejnmark, L., Rubin, M., Schwarz, P., Vokes, T., Clarke, B., Ahmed, I., Hofbauer, L., Marcocci, C., Pagotto, U., Palermo, A., Eriksen, E., Brod, M., Markova, D., Smith, A., Pihl, S., Mourya, S., Karpf, D.B. and Shu, A.D. (2021). PaTH Forward: a Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial of TransCon PTH in Adult Hypoparathyroidism. The Journal of Clinical Endocrinology & Metabolism. doi:https://doi.org/10.1210/clinem/dgab577.
- Holten‐Andersen, L., Pihl, S., Rasmussen, C.E., Zettler, J., Maitro, G., Baron, J., Heinig, S., Hoffmann, E., Wegge, T., Krusch, M., Faltinger, F., Killian, S., Sprogoe, K., Karpf, D.B., Breinholt, V.M. and Cleemann, F. (2019). Design and Preclinical Development of TransCon PTH, an Investigational Sustained‐Release PTH Replacement Therapy for Hypoparathyroidism. Journal of Bone and Mineral Research, 34(11), pp.2075–2086. doi:https://doi.org/10.1002/jbmr.3824.
A platform to design therapies with best-in-class potential
TransCon is an innovative technology platform for drug development. It combines known biology with the benefits of prodrug and sustained-release technologies to potentially optimize therapeutic effect. This could mean enhancing efficacy, improving tolerability and convenience, or reducing caregiver burden.
TransCon Prodrug: 3 components
TransCon molecules have three components: an unmodified parent drug with known biology, an inert carrier that protects it, and a linker that temporarily binds the two. When bound, the carrier inactivates and shields the parent drug from clearance. When injected, physiologic conditions in the body initiate the release of the active, unmodified parent drug in a predictable manner at a predetermined rate. Because the parent drug is unmodified, its original mode of action is expected to be maintained.
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