Ascendis Pharma A/S

Ascendis Pharma announces U.S. commercial availability of YORVIPATH, the first FDA-approved treatment for hypoparathyroidism in adults. YORVIPATH, a once-daily prodrug of parathyroid hormone, aims to provide continuous exposure to active PTH. Ascendis commits to ensuring affordable access through a dedicated support program.

Ascendis Pharma's YORVIPATH® (palopegteriparatide) is now available in the U.S. for treating hypoparathyroidism in adults, offering continuous exposure to active PTH. The company has established a dedicated team to support patient access and treatment.

Ascendis Pharma announced positive Week 26 results from its Phase 2 New InsiGHTS trial, showing once-weekly TransCon hGH's safety, tolerability, and efficacy comparable to daily somatropin in Turner syndrome children. TransCon hGH demonstrated similar growth improvement and was generally safe and well-tolerated, with no discontinuations related to the study drug.

Ascendis Pharma announced positive Week 26 results from the New InsiGHTS trial, showing once-weekly TransCon hGH provided comparable safety and growth benefits to daily somatropin in Turner syndrome children. TransCon hGH was generally safe and well-tolerated, with no discontinuations related to the study drug.

Ascendis Pharma announced FDA acceptance of its sBLA for TransCon hGH to treat adult growth hormone deficiency (GHD), with a PDUFA date set for July 27, 2025. The application is supported by the foresiGHt Phase 3 trial, which demonstrated TransCon hGH's superiority in reducing trunk fat and increasing lean body mass, with a safe and well-tolerated profile.

Ascendis Pharma faces challenges with Skytrofa sales but prepares to launch Yorvipath and TransCon CNP, aiming to offset setbacks and drive growth.

Ascendis Pharma grants Novo Nordisk exclusive rights to TransCon tech for metabolic diseases, including obesity and type 2 diabetes. Novo Nordisk can expand into other therapeutic areas. Lead project is a once-monthly GLP-1 receptor agonist. Ascendis to receive up to $285m in upfront, development, and regulatory milestone payments, plus sales-based milestones and royalties. Ascendis leads early-stage development, with Novo Nordisk funding and handling later stages. Transaction pending regulatory approval, expected to close by end of 2024.

3-year results from Ascendis Pharma's Phase 2 PaTH Forward Trial show long-term treatment with TransCon™ PTH (palopegteriparatide) normalized bone remodeling in hypoparathyroidism patients, promoting skeletal health parameters similar to parathyroid sufficiency.

Ascendis Pharma A/S (ASND) is a biotech company using TransCon technology to develop prodrug therapies, focusing on North America, China, and Europe. Key products include SKYTROFA and YORVIPATH. In 2023, revenue grew to €266.7M, driven by SKYTROFA and an upfront payment. Ascendis aims to launch TransCon PTH in the U.S. in 2024 and achieve operating cash flow breakeven. Vision 2030 includes blockbuster status for three Endocrinology Rare Disease products.

Ascendis Pharma's Phase 1/2 IL-Believe Trial showed 29% (4/14) efficacy-evaluable platinum-resistant ovarian cancer (PROC) patients responded to TransCon IL-2 β/γ plus chemotherapy, suggesting clinical activity in heavily pre-treated patients. The treatment was generally well-tolerated, with most adverse events being grade 1 or 2. Results will be presented at ESMO 2024 in Barcelona.