Phase 1 data for TransCon CNP

Ascendis Pharma presented on the 28th November 2018, preliminary results of Phase 1 for TransCon CNP. Ascendis stated that TransCon CNP was effective in delivered continuous exposure of CNP at target levels for seven days with a single subcutaneous administration (one administration per week) and that the TransCon CNP phase 1 results reinforced the preclinical data and support target product profile. 1

The TransCon technology

 

Image 1- Ascendis Pharma A/S Dec 2018

 

Image 2 - In this scheme, the company explains the advantages of the TransCon CNP technology, that can prevent the CNP to be destroyed by natural body existent enzymes.  Credits: Ascendis Pharma A/S Dec 2018 

Phase 1 highlights

  • This is a double-blind, randomized, placebo-controlled trial, with 45 healthy adult subjects enrolled.
  • Five different oses of TransCon CNP were tested in a sequential mode, beginning with the lowest dose: 3.0, 10, 25, 75 and 150 microgram/kg of TransCon CNP.
  • Up to 10 participants in each dose cohort were randomized to receive TransCon CNP or placebo in a 4:1 ratio. This means that the proportion of participants was of 4 participants receiving TransCon CNP and 1 participant receiving placebo.
  • After each cohort (the same as study group) completed dosing, a Data Safety Monitoring Board (DSMB) reviewed the blinded data to approve escalation to the next higher dose.
  • The primary endpoint (the key goal to be achieved in the study) was the frequency of adverse events after administration of TransCon CNP.
  • Secondary endpoints included additional safety parameters, tolerability, and pharmacokinetics. 2

Goals achieved in this phase

Data from phase 1 revealed that TransCon CNP was generally well tolerated across all cohorts, with unchanged mean resting blood pressure and heart rate compared to predose. Also, the injections were well tolerated. 2

Next stage of development

Ascendis plans to initiate a phase 2 program of TransCon CNP in children with achondroplasia in mid-2019. The clinical centers where the study will take place are still to be announced. 2

 

Resources

  1. Ascendis Investors & News
  2. Ascendis Pharma A/S December 2018