Ascendis Pharma reported that its COACH Phase 2 study of once‑weekly TransCon CNP (navepegritide) combined with TransCon hGH (lonapegsomatropin) achieved its primary goal at the 52‑week mark, showing durable linear growth in children with achondroplasia without compromising safety or tolerability.
The 21‑patient trial, which enrolled children aged 2 to 12, demonstrated an annualized growth velocity that exceeded the 97th percentile for average‑stature peers. All participants completed the full 52‑week course and remain on therapy, and the combination also produced measurable gains in body proportionality and arm span, indicating benefits beyond height alone.
Safety data mirrored the profiles of the individual monotherapies; only mild treatment‑emergent adverse events were reported, and no serious safety signals emerged. The favorable safety and tolerability profile supports the hypothesis that TransCon CNP primes growth plates, amplifying the effect of TransCon hGH and positioning the dual‑agent regimen as a potential new standard of care for achondroplasia.
The results strengthen Ascendis’s rare‑disease pipeline and reinforce its regulatory strategy. TransCon CNP is currently under FDA Priority Review with a decision date of February 28, 2026, and the company is preparing a pivotal Phase 3 trial that will compare TransCon CNP monotherapy with the combination regimen. A successful Phase 3 program could unlock a blockbuster product and broaden the company’s reach into other growth disorders.
Analysts have remained positive, citing the robust clinical data and the company’s clear path to regulatory approval. The COACH trial’s outcomes are expected to enhance investor confidence in Ascendis’s rare‑disease platform and its long‑term growth prospects.
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