Clinical Read2026-08-28

ION775

Ionis Pharmaceuticals Inc.IONS
Indication
Hypertriglyceridemia
Stage
phase1
Event
Phase 1
Details

August 25, 2026: Ascendis Pharma A/S (NASDAQ: ASND) announced an ISDS 2026 oral presentation of Week 104 data from the pivotal ApproaCH trial open-label extension of once-weekly TransCon CNP (navepegritide) in children with achondroplasia. The presentation, “Improved Growth and Physical Functioning in Children with Achondroplasia Treated with Navepegritide in the ApproaCH Trial Open-Label Extension,” is scheduled for August 28, 2026, in Toronto.

The company said long-term trial data showed durable improvements in height, lower-extremity alignment, body proportionality, spinal canal dimensions, muscle function, and physical functioning, with a safety and tolerability profile similar to placebo and a low rate of injection-site reactions.

Ascendis will also present a poster at ISDS 2026 on August 26-29 regarding a number-needed-to-harm analysis for injection-site reactions.

Regulatory status: TransCon CNP was approved by the FDA in February 2026 as YUVIWEL to increase linear growth in pediatric patients aged 2 years and older with achondroplasia and open epiphyses. Ascendis said its European Marketing Authorisation Application for YUVIWEL is under EMA review, with a regulatory decision anticipated in Q4 2026.