Ascendis Pharma has reported week 26 interim analysis outcomes from the ongoing Phase II COACH Trial assessing combo treatment using TransCon CNP (navepegritide) and TransCon hGH (lonapegsomatropin) in children with a rare genetic condition known as achondroplasia.
Findings indicate that TransCon hGH enhances the effects of TransCon CNP, leading to improved growth and body proportionality in children post-26 weeks.
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The safety and tolerability profile of the combo aligns with that of the individual monotherapies.
TransCon CNP and TransCon hGH are administered one time a week.
The proof-of-concept (PoC), prospective, open-label COACH Trial is testing the combination treatment of TransCon CNP at 100 µg/kg/week and TransCon hGH at 0.30mg/kg/week in those aged two to 11 years.
It includes two cohorts: one naive to TransCon CNP treatment and another that received the treatment previously.
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By GlobalDataIn the trial, TransCon CNP-naive subjects showed a significant increase in mean annualised growth velocity (AGV), with a notable improvement in ACH height Z-score. Similarly, those previously treated with TransCon CNP also exhibited increased AGV and height Z-score.
Those who received the combo’s treatment showed an expedited improvement in body proportionality at week 26.
Additionally, bone age progression matched chronological age.
The company noted that the interim analysis will be followed by week 52 data, anticipated in the fourth quarter (Q4) of this year. It plans to begin a Phase III trial during this quarter.
Currently under priority review by the US Food and Drug Administration (FDA) for achondroplasia, TransCon CNP is a prodrug of C-type natriuretic peptide (CNP) that offers continuous exposure of active CNP to receptors on the tissues throughout the body.
The prodrug of somatropin, TransCon hGH, was approved and is being marketed under the name Skytrofa to treat paediatric growth hormone deficiency.
Ascendis Pharma Endocrine & Rare Disease Medical Sciences executive vice-president and chief medical officer Aimee Shu said: “These results highlight the unique portfolio of once-weekly TransCon CNP and once-weekly TransCon hGH, with complementary modes of action, to improve the treatment landscape for growth disorders and physical functioning.”
In 2023, the company announced positive outcomes from a Phase III trial of TransCon hGH in adults with growth hormone deficiency.
