Ascendis Pharma A/S (NASDAQ: ASND) presented Thursday September 10, 2026 at the closing day of the ESPE 2026 congress in Marseille the trial-design abstract for HighLiGHts, a Phase 3 study of lonapegsomatropin (TransCon human growth hormone, a sustained-release growth hormone prodrug branded SKYTROFA in the U.S. and marketed for pediatric growth hormone deficiency since 2021) in children with short stature or growth failure due to Turner syndrome, SHOX deficiency, small-for-gestational-age (SGA), or idiopathic short stature. The four-indication combined trial design targets patients whose short stature is not attributable to growth hormone deficiency per se, expanding beyond the current SKYTROFA U.S. label for pediatric GHD. The presentation was one of several Ascendis ESPE items following the Tuesday September 8 podium delivery of first Week 52 sentinel-cohort data from the Phase 3 reACHin trial of navepegritide (TransCon CNP) in infants with achondroplasia. Ascendis reported €55 million in Q2 2026 SKYTROFA revenue alongside €252 million from YORVIPATH and €8 million from YUVIWEL, for a €315 million combined product-revenue quarter.
Ascendis Pharma A/S (NASDAQ: ASND) presented first sentinel-cohort data from the Phase 3 reACHin trial of navepegritide (TransCon CNP, a sustained-release C-type natriuretic peptide prodrug branded YUVIWEL in the U.S. for achondroplasia in children ≥2 years) in infants aged 0 to under 2 years at the ESPE 2026 congress in Marseille on Tuesday September 8, 2026 (abstract FC4.6, 3:00-4:00 p.m. CEST podium session; Genevieve Baujat MD, Necker Hospital, presenting). The reACHin study completed target enrollment and extends navepegritide's tested age range down to infancy, the population most vulnerable to achondroplasia complications from cervicomedullary compression, foramen magnum stenosis, and delayed motor milestones. Additional ESPE presentations include the HighLiGHts Phase 3 trial design for lonapegsomatropin (TransCon hGH) across Turner syndrome, SHOX deficiency, small-for-gestational-age, and idiopathic short stature; two poster presentations on adolescent hypoparathyroidism patient-reported outcomes; and a systematic literature review on pediatric growth hormone deficiency prevalence. Ascendis' TransCon franchise generated €315 million in Q2 2026 product revenue (+105% year-over-year) with YUVIWEL contributing €8 million in its first U.S. commercial quarter.
Ascendis Pharma A/S (NASDAQ: ASND) announced Monday September 7, 2026 that the first sentinel-cohort data from the Phase 3 reACHin trial of navepegritide (TransCon CNP, a sustained-release CNP peptide prodrug) in infants with achondroplasia aged 0 to under 2 years will be presented as a podium talk at the European Society for Paediatric Endocrinology (ESPE) 2026 congress in Marseille, France (September 8-10). The reACHin presentation (FC4.6) is scheduled for Tuesday September 8, 3:00-4:00 p.m. CEST by Genevieve Baujat MD. Additional presentations include the HighLiGHts Phase 3 trial design for lonapegsomatropin (TransCon hGH) across Turner syndrome, SHOX deficiency, small-for-gestational-age, and idiopathic short stature; hypoparathyroidism patient-reported-outcome data from adolescents; and a systematic literature review on pediatric growth hormone deficiency prevalence. Ascendis submitted the TransCon CNP marketing authorization application to the EMA in October 2025 with an EU decision expected Q4 2026. Navepegritide is engineered to provide continuous CNP receptor stimulation without the peak-and-trough kinetics that constrained earlier vosoritide (Voxzogo) dosing.