Ascendis Pharma starts Phase 1 Trial of TransCon CNP for achondroplasia

On the 8th May 2018, Ascendis Pharma announced the dosing of the first subjects 

for phase 1 trial of TransCon CNP.

Ascendis Pharma A/S is a biopharmaceutical company that developed the TransCon technology, a long-acting prodrug of 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.

 

In this phase 1, participants are healthy adults and is a double-blind, randomized, and placebo-controlled. The aim is to evaluate single ascending doses of TransCon CNP to assess safety, tolerability, and pharmacokinetics.

 

Jonathan Leff, M.D., Ascendis Pharma’s Chief Medical Officer said that “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.

 

The key announcement in this press release is that the company anticipates top-line data from the phase 1 trial to be available in the fourth quarter of 2018.

 

Notes:

Placebo-controlled: is when in a clinical trial some volunteers in this trial will receive TransCon CNP while others will receive saline, without knowing which of both are they receiving.

Pharmacokinetics - study of the time course of drug absorption, distribution, metabolism, and excretion

 

Read the press release here.