Ascendis Pharma Stock Price, News & Analysis

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.

Ascendis Pharma (ASND) reported Q1 2025 financial results with total revenue of €101.0 million, up from €95.9 million in Q1 2024. The company's two main products showed strong performance: YORVIPATH® generated €44.7 million in revenue with over 1,750 prescriptions, while SKYTROFA® contributed €51.3 million. The company reported a net loss of €94.6 million (€1.58 per share), improved from €131.0 million loss in Q1 2024. Cash position stands at €518 million as of March 31, 2025. Key upcoming milestones include TransCon CNP's FDA review, COACH combination trial data in Q2 2025, and SKYTROFA's PDUFA date of July 27, 2025, for adult growth hormone deficiency treatment.

Ascendis Pharma (Nasdaq: ASND) has scheduled its first quarter 2025 financial results announcement and business update for Thursday, May 1, 2025, after U.S. market close. The company will host a conference call and webcast at 4:30 p.m. Eastern Time on the same day.

Interested participants can access the live webcast or register for the teleconference through the company's website. A replay will be available in the Investors & News section at investors.ascendispharma.com for 30 days following the event.

Ascendis Pharma (ASND) has submitted a New Drug Application (NDA) to the FDA for TransCon CNP (navepegritide) to treat children with achondroplasia. The submission is supported by data from three randomized, double-blind, placebo-controlled clinical trials with up to three years of open-label extension data.

The treatment, administered once weekly, is designed as a prodrug of C-type natriuretic peptide (CNP) that provides continuous exposure to receptors throughout the body. Clinical data showed multiple benefits including:

• Increased growth velocity
• Reduced health-related burden
• Stronger muscle function
• Straightening of abnormal leg bowing in most treated children

The company plans to submit a Marketing Authorisation Application (MAA) to the European Medicines Agency during Q3 2025.

Ascendis Pharma A/S (Nasdaq: ASND) has announced its participation in the TD Cowen 45th Annual Health Care Conference. Company executives will engage in a fireside chat scheduled for Tuesday, March 4, 2025, at 11:10 a.m. Eastern Time / 8:10 a.m. Pacific Time in Boston, Massachusetts.

The event will be accessible through a live webcast on the Investors & News section of the Ascendis Pharma website at investors.ascendispharma.com. For those unable to attend live, a replay of the webcast will be available on the same website for 30 days following the event.