Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
TransCon CNP · 2 trials · 1 indication
Incidence of Treatment-Emergent Adverse Events
Number of standard deviations
The primary efficacy analysis compared the difference in the primary efficacy endpoint between the TransCon CNP treatment group and the pooled placebo group using an ANCOVA model with the annualized height velocity (AHV) at Week 52 as the response variable, treatment (dose groups and placebo) and sex as factors, baseline age and baseline height SDS as the covariates, and based on the Full Analysis Set.
| Arm | Type | Description |
|---|---|---|
| TransCon CNP 100 mcg | EXPERIMENTAL | TransCon CNP 100 mcg delivered once weekly by subcutaneous injection |
| TransCon CNP 6 mcg | EXPERIMENTAL | TransCon CNP 6 mcg CNP/kg delivered once weekly by subcutaneous injection. |
| TransCon CNP 20 mcg | EXPERIMENTAL | TransCon CNP 20 mcg CNP/kg delivered once weekly by subcutaneous injection. |
| TransCon CNP 50 mcg | EXPERIMENTAL | TransCon CNP 50 mcg CNP/kg delivered once weekly by subcutaneous injection. |
| Placebo | PLACEBO_COMPARATOR | Placebo mimicking 6, 20, 50, or 100 mcg CNP/kg delivered once weekly by subcutaneous injection. |
| Open-Label Extension Period: TransCon CNP | EXPERIMENTAL | Participants who completed the 52-week blinded treatment period continued into the 104-week open-label extension period and received treatment with TransCon CNP (navepegritide) doses escalated up to a maximum of 100 mcg/kg delivered once weekly by subcutaneous injection. |
| Name | Type | Description |
|---|---|---|
| TransCon CNP | DRUG | TransCon CNP drug product is a lyophilized powder in a single-use vial. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle. |
| Placebo for TransCon CNP | DRUG | Weekly subcutaneously injection of placebo. |
Inclusion Criteria: * Written, signed informed consent of the parent(s) or legal guardian(s) of the participant, and as required by the institutional review board/human research ethics committee/independent ethics committee (IRB/HREC/IEC). For participants who are below the age of consent, a writte...
TransCon CNP is an investigational small molecule being developed for achondroplasia, a rare genetic condition affecting bone growth. It is administered as a weekly subcutaneous injection and is currently in Phase 2 clinical development. TransCon CNP is designed to address the underlying growth deficiency in children and adolescents with achondroplasia.
TransCon CNP targets CNP, which stands for C-type natriuretic peptide. By targeting CNP, TransCon CNP aims to modulate the signaling pathway involved in bone growth. This mechanism is being studied as a potential treatment for achondroplasia, with the goal of improving growth outcomes in affected children and adolescents.
TransCon CNP is being developed by Ascendis Pharma A/S, a biopharmaceutical company listed on the NASDAQ under the ticker symbol ASND. Ascendis Pharma is conducting clinical trials to evaluate the safety and efficacy of TransCon CNP for the treatment of achondroplasia in pediatric patients.
TransCon CNP is currently in Phase 2 clinical development. It is an investigational drug and has not been approved by regulatory authorities. The ongoing Phase 2 trials are designed to assess the long-term safety, tolerability, and efficacy of weekly subcutaneous doses of TransCon CNP in children and adolescents with achondroplasia.
TransCon CNP is being evaluated in two Phase 2 clinical trials. The first, NCT05929807, is an ongoing study enrolling by invitation with 140 participants, investigating long-term safety and efficacy in children and adolescents with achondroplasia. The second, NCT04085523, is a completed dose escalation trial with 57 participants in prepubertal children with achondroplasia.
TransCon CNP is not the same as CNP. CNP, or C-type natriuretic peptide, is the molecular target of TransCon CNP. TransCon CNP is a prodrug designed to provide sustained exposure to CNP, which is being studied for its potential to improve growth in children with achondroplasia.