Ascendis Pharma (ASND) Stock News

Ascendis Pharma (Nasdaq: ASND) has announced its participation in the upcoming 2025 Wells Fargo Healthcare Conference. Company executives will engage in a fireside chat scheduled for September 3, 2025, at 8:00 a.m. Eastern Time in Boston, Massachusetts.

Investors can access the presentation through a live webcast on the company's investor relations website. A replay will be available for 30 days following the event.

Ascendis Pharma (NASDAQ:ASND) reported strong Q2 2025 financial results, with total revenue of €158.0 million, a significant increase from €36.0 million in Q2 2024. YORVIPATH® generated €103.0 million in revenue with approximately 3,100 unique patient enrollments, while SKYTROFA® contributed €50.7 million.

Key developments include FDA's priority review of TransCon® CNP (navepegritide) for children with achondroplasia with a PDUFA date of November 30, 2025, and SKYTROFA's U.S. approval for adult growth hormone deficiency treatment. The company reported a net loss of €38.9 million (€0.64 per share) and maintained a strong cash position of €494 million as of June 30, 2025.

Ascendis Pharma (Nasdaq: ASND) has scheduled its second quarter 2025 financial results announcement and business update for Thursday, August 7, 2025, after U.S. market close. The company will host a conference call and webcast at 4:30 p.m. ET on the same day.

Investors can access the live webcast through the company's investor relations website, and a replay will be available for 30 days following the event. Participants interested in the teleconference can register in advance through the provided link.

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).