COPENHAGEN - Ascendis Pharma A/S (NASDAQ:ASND) has submitted a supplemental Biologics License Application (sBLA) to the U.S. Food & Drug Administration for TransCon hGH, currently marketed for pediatric use, to expand its treatment to adults with growth hormone deficiency (GHD). This move aligns with the company's strategy to evolve SKYTROFA into a major product by widening its label.
The application is underpinned by the results from the foresiGHt Phase 3 trial, which assessed the efficacy and safety of weekly TransCon hGH against a placebo and daily hGH in a group of 259 adults with GHD. The study demonstrated that patients treated with TransCon hGH experienced a significant reduction in trunk fat and an increase in total body lean mass compared to the placebo group at week 38, with a safety profile comparable to daily hGH.
Jan Mikkelsen, President and CEO of Ascendis Pharma, highlighted the potential of TransCon hGH to fulfill an unmet medical need, given that most adults with GHD are not currently receiving treatment. He emphasized the importance of growth hormone in maintaining health across all ages and expressed confidence in the weekly treatment's ability to offer a new option for adults with GHD.
Ascendis Pharma, headquartered in Copenhagen with additional facilities in Europe and the U.S., is known for its TransCon technology platform which seeks to develop new therapies that could potentially be best-in-class.
The company cautions that this press release contains forward-looking statements that involve risks and uncertainties, and actual results could differ materially from those projected. These forward-looking statements should not be relied upon as guarantees of future performance.
The information in this article is based on a press release statement from Ascendis Pharma.
In other recent news, Ascendis Pharma has seen a flurry of positive analyst activity, with 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. Analysts have highlighted the once-weekly injection schedule of TransCon CNP as a significant advantage over competing treatments, potentially driving patient preference and uptake.
Ascendis Pharma has also launched 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. The biopharmaceutical company has reported promising results from its Phase 1/2 IL-Believe Trial with TransCon IL-2 β/γ in patients with platinum-resistant ovarian cancer, and secured a $150 million funding agreement with Royalty Pharma.
The company has received U.S. approval for its product YORVIPATH for adult hyperparathyroidism and ended the quarter with EUR259 million in cash and equivalents. Ascendis Pharma projects SKYTROFA revenue to be between EUR220 million and EUR240 million for the full year of 2024. These recent developments highlight Ascendis Pharma's ongoing efforts to develop and commercialize novel treatments.
InvestingPro Insights
As Ascendis Pharma A/S (NASDAQ:ASND) moves to expand its TransCon hGH treatment to adults with growth hormone deficiency, investors should consider some key financial metrics and insights from InvestingPro.
The company's market capitalization stands at $8.63 billion, reflecting significant investor interest in its growth potential. Ascendis Pharma has demonstrated impressive revenue growth, with a 166.54% increase in the last twelve months as of Q2 2024. This aligns with the company's strategy to evolve SKYTROFA into a major product by widening its label.
However, it's important to note that Ascendis is not currently profitable. An InvestingPro Tip reveals that analysts do not anticipate the company will be profitable this year. This is not uncommon for biotech companies investing heavily in research and development, as Ascendis is doing with its TransCon technology platform.
Despite the lack of profitability, the company has shown a high return over the last year, with a one-year price total return of 54.44%. This suggests that investors are optimistic about Ascendis Pharma's future prospects, including the potential approval of TransCon hGH for adult use.
InvestingPro Tips also indicate that Ascendis operates with a moderate level of debt, which could provide financial flexibility as it seeks to expand its product offerings. However, short-term obligations exceed liquid assets, which may require careful financial management in the near term.
For investors seeking more comprehensive analysis, InvestingPro offers additional tips and insights. In fact, there are 8 more InvestingPro Tips available for Ascendis Pharma, providing a deeper understanding of the company's financial position and market performance.
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