TransConCNP ACcomplisH Trial News and Results

Ascendis Pharma, the Denmark based emerging pharmaceutical company, shared the results on the Phase 2, Multicenter, Double-blind, Randomized, Placebo-controlled, Dose Escalation Trial Evaluating the Safety, Efficacy, and Pharmacokinetics of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 12 months in Prepubertal Children with Achondroplasia.

Important Information:

The company states that in this Phase 2 Trial, TransCon CNP demonstrated the potential to meet patient and caregiver needs for a safe, effective, tolerable, and convenient treatment. The primary endpoint, annualized height velocity (AHV) at Week 52, demonstrated superiority of TransCon CNP at 100 µg/kg/week compared to placebo (p=0.0218).

Furthermore, TransCon CNP was generally well tolerated with low frequency of injection site reactions, which allowed that all 57 randomized children stayed with the treatment beyond two years.

The data found showed important and consistent results across age groups and dose levels, supporting continued development with the selected dose of 100 µg/kg/week.

Read the official document here: