Ascendis Pharma Stock Price, News & Analysis

Ascendis (Nasdaq: ASND) submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency for TransCon CNP (navepegritide) as a once‑weekly treatment for children with achondroplasia.

The MAA is supported by data from three randomized, double‑blind, placebo‑controlled trials and up to three years of open‑label extension data, including the pivotal ApproaCH Trial. Ascendis reported TransCon CNP was generally well tolerated with no discontinuations related to the study drug. Ascendis said it will engage with the EMA during review. In the U.S., TransCon CNP is under FDA priority review with a PDUFA target date of November 30, 2025.

Ascendis Pharma (NASDAQ:ASND) will present three oral presentations featuring its latest clinical data at the ASBMR 2025 conference in Seattle from September 5-8, 2025. The presentations will showcase new analyses for treatments in achondroplasia and hypoparathyroidism.

Key presentations include:

• New analysis correlating leg bowing improvements with physical functioning in children treated with TransCon CNP
• First presentation of clinical trial data combining TransCon CNP and TransCon hGH therapy in children with achondroplasia
• Clinical data showing 4-year sustained skeletal improvements in adults with hypoparathyroidism treated with TransCon PTH

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.