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 designed for once-weekly subcutaneous administration in children and adolescents with achondroplasia. The drug is currently in Phase 2 clinical development and has not yet been approved by regulatory authorities.
TransCon CNP is being developed by Ascendis Pharma A/S, a biopharmaceutical company traded on the NASDAQ under the ticker ASND. Ascendis Pharma is conducting clinical trials of TransCon CNP in multiple countries, including the United States, Australia, and several European nations, for the treatment of achondroplasia.
TransCon CNP is in Phase 2 clinical development for achondroplasia. It is an investigational drug, meaning it has not been approved by the FDA or other regulatory agencies. Two Phase 2 trials have been conducted or are ongoing, with one completed and one currently enrolling participants by invitation.
TransCon CNP has been studied in two Phase 2 clinical trials. The first, NCT04085523, was a completed dose escalation trial in prepubertal children with achondroplasia. The second, NCT05929807, is an ongoing long-term safety and efficacy trial in children and adolescents with achondroplasia, enrolling 140 participants across multiple countries.
TransCon CNP is designed to deliver C-type natriuretic peptide (CNP) for the treatment of achondroplasia. CNP is a naturally occurring peptide that regulates endochondral bone growth. The drug is formulated to provide sustained exposure to CNP with once-weekly dosing, which may help address the underlying growth deficiency in achondroplasia.
TransCon CNP is a specific investigational product developed by Ascendis Pharma. It is designed as a long-acting form of CNP for weekly administration. While other CNP-based therapies may exist, TransCon CNP is distinguished by its TransCon technology platform, which is intended to optimize the pharmacokinetic profile of the peptide.