Peptide News Digest

#Lonapegsomatropin

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Clinical Trials · View digest

Ascendis Pharma Presents HighLiGHts Phase 3 Trial Design for Lonapegsomatropin (TransCon hGH) Across Four Pediatric Short-Stature Indications at ESPE 2026 Closing Day

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.

Clinical Trials · View digest

Ascendis Pharma Presents First Sentinel-Cohort Podium Data for Navepegritide (TransCon CNP) in Infants With Achondroplasia at ESPE 2026 Tuesday September 8

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.

Industry · View digest

Ascendis Pharma to Present First reACHin Sentinel-Cohort Data for Navepegritide (TransCon CNP) in Achondroplasia Infants at ESPE 2026 Tuesday September 8

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.

Industry · View digest

Ascendis Pharma (NASDAQ: ASND) Reported Thursday August 13 Q2 2026 Financial Results With Product Revenue of €315 Million (+105% Year-Over-Year), Anchored by YORVIPATH (Palopegteriparatide, a TransCon Parathyroid Hormone Peptide Analog for Chronic Hypoparathyroidism) at €252 Million on Consistent New Patient Demand in the US and Continued Ex-US Global Launch Expansion With Full Reimbursement, SKYTROFA (Lonapegsomatropin, a TransCon Growth Hormone Peptide) at €55 Million, and YUVIWEL (TransCon C-Type Natriuretic Peptide for Achondroplasia) at €8 Million With More Than 220 US Patient Enrollments Through July 31, 2026; The YUVIWEL Launch Positions Ascendis Directly Against BioMarin's Voxzogo (Vosoritide) in the Same Achondroplasia Pediatric Indication and Opens a New Front in the CNP Peptide Analog Commercial Category

Ascendis Pharma (NASDAQ: ASND) reported Thursday August 13, 2026 Q2 2026 financial results with product revenue of €315 million (+105% year-over-year). YORVIPATH (palopegteriparatide, a TransCon parathyroid hormone peptide analog for chronic hypoparathyroidism) delivered €252 million on consistent new patient demand in the US and continued ex-US global launch expansion with full reimbursement in additional markets. SKYTROFA (lonapegsomatropin, a TransCon growth hormone peptide for pediatric growth hormone deficiency) delivered €55 million. YUVIWEL (TransCon C-type natriuretic peptide for achondroplasia) delivered €8 million with more than 220 US patient enrollments through July 31, 2026. Total Q2 revenue reached €339 million (+100% YoY) including €24 million in collaboration and milestone income. The YUVIWEL launch positions Ascendis directly against BioMarin's Voxzogo (vosoritide) in the same achondroplasia pediatric indication, opening a new front in the CNP peptide analog commercial category. YORVIPATH's €252 million quarterly revenue trajectory suggests a $1+ billion annual run-rate for the peptide franchise by end of 2026, a notable rare-disease peptide launch magnitude. Ascendis's TransCon platform uses a transient linker chemistry to release parent drug over an extended half-life, allowing weekly dosing of peptides that natively have short in vivo lifespans.