Ascendis Pharma Stock Price, News & Analysis

Ascendis Pharma (Nasdaq: ASND) has received FDA approval for SKYTROFA® (lonapegsomatropin-tcgd) for treating adult growth hormone deficiency (GHD). This marks a significant expansion from its 2021 pediatric GHD approval. SKYTROFA is administered once-weekly, offering a less burdensome alternative to traditional daily somatropin injections.

The approval was based on the foresiGHt Phase 3 trial, which demonstrated efficacy and safety compared to placebo and daily somatropin. This milestone aligns with Ascendis's Vision 2030 goal to become the leading endocrinology rare disease company, with plans to initiate additional trials for various conditions in Q4 2025.

Ascendis Pharma (Nasdaq: ASND) announced promising 3-year Phase 3 data for TransCon PTH (palopegteriparatide) in treating adults with hypoparathyroidism. The PaTHway Trial demonstrated significant sustained efficacy with 88% of patients achieving normal calcium levels and 96% becoming independent from conventional therapy at Week 156.

Key outcomes include improved kidney function with mean eGFR increase of 8.76 mL/min/1.73 m2 across all participants, and 13.98 mL/min/1.73 m2 in patients with lower baseline eGFR. The trial showed high retention with 89% completion rate, while demonstrating sustained improvements in patient symptoms and quality of life. The treatment was generally well-tolerated with no new safety concerns identified.

Ascendis Pharma (Nasdaq: ASND) announced two key presentations at ENDO 2025, scheduled for July 12-15, 2025, in San Francisco. The presentations will showcase important clinical data from their Endocrinology Rare Disease portfolio.

Dr. Aliya Khan will present Week 156 data from the Phase 3 PaTHway Trial of TransCon® PTH in adults with hypoparathyroidism, demonstrating long-term safety, efficacy, and improved renal function. Additionally, Dr. Carlos Bacino will present Week 52 safety and tolerability data from the pivotal ApproaCH Trial of TransCon® CNP in children with achondroplasia.

Ascendis Pharma announced positive Week 26 interim results from the Phase 2 COACH Trial evaluating combination treatment of TransCon CNP and TransCon hGH in children with achondroplasia. The treatment-naïve cohort achieved mean annualized growth velocity of 9.14 cm/year with a +0.53 improvement in ACH height Z-score, while the TransCon CNP-treated cohort showed 8.25 cm/year growth with +0.44 Z-score improvement. The combination therapy demonstrated accelerated body proportionality improvements and was generally well-tolerated with mild adverse events. TransCon CNP is currently under FDA priority review as a monotherapy, while TransCon hGH (SKYTROFA) is already approved for pediatric growth hormone deficiency. The company plans to initiate a Phase 3 trial in Q4 2025, with Week 52 data expected in the same quarter.

Ascendis Pharma announced that the FDA has accepted their New Drug Application (NDA) for TransCon CNP (navepegritide) with Priority Review designation for treating children with achondroplasia. The FDA set a PDUFA date of November 30, 2025, and indicated no plans for an advisory committee meeting. TransCon CNP, a once-weekly investigational prodrug, demonstrated significant improvements in annualized growth velocity compared to placebo in clinical trials. The treatment showed multiple benefits beyond linear growth, including improvements in lower limb alignment, spinal canal dimensions, and muscle strength, while maintaining a safety profile similar to placebo. The Priority Review status indicates the FDA's recognition of TransCon CNP's potential to significantly improve treatment for this serious condition.

Specialised Therapeutics (ST) has announced that SKYTROFA® (lonapegsomatropin) has received approval from Australia's Therapeutic Goods Administration (TGA) for treating pediatric growth hormone deficiency (GHD). This once-weekly injectable therapy is approved for children and adolescents aged 3-18 years with insufficient endogenous growth hormone secretion. GHD affects approximately 2,000 Australian children, occurring at a rate of 2-3 per 10,000 people. SKYTROFA utilizes Ascendis Pharma's proprietary TransConTM technology platform, which enables sustained release of active, unmodified somatropin over one week. The approval follows successful Phase 3 clinical trials (heiGHt, fliGHt, and enliGHten) involving over 300 pediatric patients. The therapy is distributed by ST under an exclusive agreement with Ascendis Pharma A/S covering several Asia-Pacific countries. Currently, SKYTROFA is not listed on the Pharmaceutical Benefits Scheme (PBS).

Ascendis Pharma (ASND) announced positive Week 52 data from its pivotal ApproaCH Trial of TransCon CNP (navepegritide) in children with achondroplasia. The double-blind placebo-controlled trial, involving 84 children aged 2-11 years, demonstrated superior annualized growth velocity compared to placebo. The drug showed a favorable safety profile with no treatment-related serious adverse events, symptomatic hypotension, fractures, or bone age acceleration.

Key improvements were observed in bone morphometry, including better lower limb alignment, proportional growth, and increased spinal canal dimensions. The trial featured a 2:1 randomization ratio (TransCon CNP:placebo) for 52 weeks, followed by an open-label extension period. The results support the company's goal to provide benefits beyond linear growth and potentially reduce future complications associated with achondroplasia.

Ascendis Pharma (NASDAQ: ASND) released promising 4-year data from its Phase 2 PaTH Forward Trial of TransCon PTH (palopegteriparatide) for adults with hypoparathyroidism. The study demonstrated sustained efficacy with 95% patient retention (56 of 59 patients) at Week 214.

Key findings include: - 98% of patients maintained normal albumin-adjusted serum calcium levels - 93% remained independent from conventional therapy - 67.8% showed clinically meaningful improvement in kidney function (eGFR) - Bone turnover markers stabilized above baseline - Bone mineral density stayed within normal range

The treatment was well-tolerated with mostly mild to moderate adverse events, no serious treatment-related adverse events, and no treatment-related discontinuations.

Ascendis Pharma (NASDAQ: ASND) will present new clinical data at the joint ESPE & ESE 2025 congress in Copenhagen from May 10-13, 2025. The presentations will showcase results from three key programs: 1. TransCon PTH (palopegteriparatide): 4-year efficacy and safety data from Phase 2 PaTH Forward Trial in chronic hypoparathyroidism 2. TransCon CNP (navepegritide): Week 52 growth and bone morphometry data from pivotal ApproaCH Trial in children with achondroplasia 3. TransCon hGH (lonapegsomatropin): Phase 3 foresiGHt Trial results in adults with growth hormone deficiency The company will participate in multiple sessions including oral presentations, symposiums, and poster presentations focusing on these rare endocrine diseases.