COPENHAGEN - Ascendis Pharma A/S (NASDAQ:ASND) announced positive long-term results from its ongoing Phase 2 PaTH Forward Trial, showing that TransCon PTH (palopegteriparatide), marketed as YORVIPATH®, normalized bone remodeling in adults with hypoparathyroidism over a three-year period. The trial, which involved 57 adults, demonstrated that continuous treatment with the drug could lead to skeletal health parameters consistent with parathyroid sufficiency.
Hypoparathyroidism is characterized by insufficient levels of parathyroid hormone (PTH), leading to complications such as neuromuscular irritability, renal issues, and cognitive impairment. TransCon PTH is designed to release PTH over a 24-hour period with a once-daily administration, aiming to restore the balance of calcium and phosphate in the body.
The results were presented by Dr. Mishaela Rubin, Associate Professor of Medicine at Columbia University, during the American Society for Bone & Mineral Research annual meeting in Toronto, which concluded today. Dr. Rubin, an expert in bone abnormalities linked to hypoparathyroidism, emphasized the significance of these findings for patients who typically suffer from low rates of bone remodeling and poor bone quality.
Ascendis Pharma, utilizing its TransCon technology platform, is focused on developing therapies that could potentially be best-in-class for various diseases. The company is headquartered in Copenhagen with additional operations in Europe and the United States.
While the press release included forward-looking statements regarding Ascendis' ambitions to become a leading biopharma company and its ability to create best-in-class therapies using its TransCon technologies, it is important to note that these statements are based on current plans and expectations, which are subject to risks and uncertainties that may cause actual results to differ.
The information provided in this news article is based on a press release statement from Ascendis Pharma. The company may face various challenges, including manufacturing dependencies, unexpected clinical trial results, and market acceptance of its products. Ascendis' future developments are subject to factors such as regulatory requirements, funding needs, and the complex nature of biopharmaceutical research and development.
In other recent news, Ascendis Pharma has reported several significant developments. The company has sought FDA approval for TransCon hGH, a growth hormone treatment currently used for pediatric patients, to be expanded for adult use. This submission is supported by positive results from the foresiGHt Phase 3 trial. Ascendis Pharma has also initiated a public offering of American Depositary Shares valued at $300 million, managed by J.P. Morgan, Morgan Stanley, Evercore ISI, and Goldman Sachs & Co. LLC.
In terms of financials, Ascendis Pharma has secured a $150 million funding agreement with Royalty Pharma and anticipates SKYTROFA revenue to be between EUR220 million and EUR240 million for the full year of 2024. Ascendis Pharma has also seen positive analyst activity from firms such as BofA Securities, Oppenheimer, Evercore ISI, and Jefferies, all adjusting their price targets upward. This follows encouraging results from Ascendis Pharma's Phase 3 trial for achondroplasia treatment, TransCon CNP, which demonstrated a statistically significant annual growth velocity.
Several analysts have highlighted the once-weekly injection schedule of TransCon CNP as a significant advantage over competing treatments, potentially driving patient preference and uptake. These recent developments highlight Ascendis Pharma's ongoing efforts to develop and commercialize novel treatments.
InvestingPro Insights
Ascendis Pharma's positive clinical results for TransCon PTH align with the company's strong revenue growth, as reflected in InvestingPro data. The firm's revenue grew by an impressive 166.54% over the last twelve months, indicating significant market traction for its innovative therapies. This growth is particularly noteworthy given the company's focus on developing potentially best-in-class treatments for diseases like hypoparathyroidism.
However, investors should be aware that Ascendis is currently operating at a loss, with an adjusted operating income of -$378.6 million in the last twelve months. This is not uncommon for biopharmaceutical companies investing heavily in research and development. An InvestingPro Tip highlights that analysts do not anticipate the company to be profitable this year, which is consistent with the ongoing investment in clinical trials and product development.
Despite the current lack of profitability, the market seems optimistic about Ascendis' potential, as evidenced by its high revenue valuation multiple. This valuation may be supported by the promising clinical results and the company's innovative TransCon technology platform. Another InvestingPro Tip notes that Ascendis has seen a high return over the last year, with a one-year price total return of 54.44%, suggesting investor confidence in the company's long-term prospects.
For those interested in a deeper analysis, InvestingPro offers 6 additional tips that could provide further insights into Ascendis Pharma's financial health and market position.
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