Beyond Achondroplasia

Growing together with Clara

TransCon CNP for achondroplasia starts Phase 1 clinical trial


Ascendis Pharma announced on the 8th May it has dosed the first volunteers in a first-in-human phase 1 trial of TransCon CNP. (2)

Image 1: The TransCon technology. Credits: Ascendis Pharma Company Presentation. 8th May 2018 (1)

Ascendis TransCon technology includes the TransCon CNP that is a long-acting prodrug of a C-type natriuretic peptide (CNP) in development as a therapeutic option for achondroplasia and potentially for other fibroblast growth factor receptor (FGFR)-related skeletal disorders.

Phase 1 is taking place in Australia with healthy volunteers and is a double-blind, randomized and placebo-controlled phase 1 trial will evaluate single ascending doses of TransCon CNP in healthy adult subjects to assess 1. safety, 2. tolerability and 3. pharmacokinetics.

Understanding clinical trials

What is a phase 1?

Phase 1 is the first stage and usually involves small groups of healthy people, or sometimes patients. Phase 1 trials are mainly aimed at finding out how safe a drug is. (3)

Image 2: Medicines development process. Credits: EUPATI (5)

What is a double-blind trial?

In a blind trial, the people taking part are not told which group they are in. This is because if they knew which treatment they
were getting, it might influence how they felt or how they reported their symptoms. Some trials are ‘double blind’, which
means that the people taking part and the doctors treating them do not know who is getting the new treatment. (3)

What is a randomized trial?

When people are put in the trials treatment groups at random, usually by using a computer programme. This is done so that each group has a similar mix of people of different ages, sexes, and states of health.(3)

What is a placebo-controlled trial?

Controlled trials are designed to compare different treatments. Most controlled trials compare a new treatment with the standard
or usual treatment by setting up two groups of people. One group, known as the trial group or intervention group, are given the new
treatment. The other group known as the control group is given the standard treatment and in situations where there is no standard
treatment (as in achondroplasia), the control group may not be given any treatment at all or may be given a ‘placebo’ (a dummy drug). A placebo is designed to look very similar to the treatment being tested. So, in a drug trial the placebo looks exactly like the real drug, but does not do anything. By comparing people’s responses to the placebo and to the treatment being tested, researchers can tell whether the treatment is having any real benefit.(3)

What is pharmacokinetics?

The word is derived from the Greek words pharmakon (drug) and kinetikos (movement), is the study of the disposition of a drug after its delivery to an organism—in short, a study of “what the body does to a drug“. is used to describe the absorption, distribution, metabolism, and excretion of a compound. (4)

Figures 3-5: The TransCon technology. Credits: Ascendis Pharma Company Presentation. 8th May 2018 (1)

Expected timelines for the TransCon CNP clinical trial


Figure 6: adapted from Ascendis Pharma company presentation (1)

Jonathan Leff, M.D., Ascendis Pharma’s Chief Medical Officer. “TransCon CNP has been designed to provide continuous CNP exposure to optimize efficacy without cardiovascular risk in a convenient once-weekly dose. Data from this trial will help validate our target product profile, once again translating our promising preclinical results into clinical data.” (2)

TransCon CNP is the third product candidate in Ascendis Pharma’s rare disease endocrinology pipeline to advance into clinic, developed using its innovative TransCon technology platform. The company anticipates top-line data from the phase 1 trial to be available in the fourth quarter of 2018. (2)



  1. Ascendis Pharma Company Presentation
  2. Ascendis Pharma- Investors and News
  3. NHS”Understanding clinical trial booklet
  4. ScienceDirect “Pharmacokinetics
  5. EUPATI- Medicines development process


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