Ascendis Pharma A/S (ASND) announced positive topline data from the Week 52 COACH Phase 2 trial evaluating once-weekly TransCon CNP in combination with TransCon hGH in pediatric patients with achondroplasia. The study demonstrated statistically significant increases in annualized growth velocity (AGV) and improvements in body proportionality, while maintaining a favorable safety profile.
Achondroplasia, the most common form of dwarfism, is caused by mutations in the FGFR3 gene and leads to impaired bone growth. Children affected by the condition often face complications beyond short stature, including disproportionate body features and potential health risks.
Trial Design and Outcomes:
The trial enrolled children with achondroplasia, including both treatment-naïve patients and those previously exposed to TransCon CNP.
The primary endpoint was annualized growth velocity at Week 52. Results showed that treatment-naïve patients achieved an annualized growth velocity of 8.8 cm per year, a gain of 3.9 cm from baseline, while TransCon CNP-experienced patients achieved 8.42 cm per year, a gain of 3.28 cm from baseline.
Improvements in height Z-scores further confirmed efficacy beyond monotherapy. The combination therapy was generally well tolerated, with most adverse events mild in nature. Importantly, no symptomatic hypotension or fractures were reported, and all patients completed the 52-week treatment period.
Clinical Significance:
These findings suggest that combining TransCon CNP with intermittent TransCon hGH boosts may deliver superior growth outcomes compared to monotherapy. Improvements in body proportionally and arm span highlighted the therapy's potential to address both stature and quality-of-life aspects of achondroplasia. The durability of the results underscores the promise of this combination approach for long-term management of the condition.
Next Steps:
Ascendis is preparing a pivotal Phase 3 trial to compare TransCon CNP monotherapy with the combination regimen. The company also plans to integrate the combination therapy into its existing U.S. regulatory filing strategy for TransCon CNP.
Looking ahead, Ascendis aims to simplify treatment into a single-injection presentation, enhancing convenience for patients and caregivers.
Upcoming Milestones:
The company expects to initiate the Phase 3 program of TransCon CNP combination therapy, continue regulatory review of TransCon CNP monotherapy in the U.S. and EU, and expand its TransCon technology into other endocrine and rare disease indications.
The company ended the third quarter with a cash, cash equivalents, and marketable securities balance of approximately €539 million.
ASND has traded between $118.03 and $229.94 over the past year. The stock closed yesterday's trading at $208.75, down 2.12%.
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