Goldman Sachs has updated its outlook on Ascendis Pharma (NASDAQ: NASDAQ:ASND), increasing the price target to $200 from the previous $180, while reaffirming a Buy rating for the stock.
The adjustment follows the announcement of positive topline results from Ascendis Pharma's Phase 3 ApproaCH study of TransCon CNP in achondroplasia, a bone growth disorder. The study successfully met its primary and secondary endpoints.
Ascendis Pharma shared that TransCon CNP was generally well-tolerated in the study, with no participants discontinuing treatment and no instances of hypotension reported.
The company's findings suggest that TransCon CNP has a competitive edge in terms of efficacy, safety, and convenience when compared to existing treatments such as Voxogo (vosoritide) by BioMarin Pharmaceutical (NASDAQ:BMRN) and infigratinib by BridgeBio Pharma (NASDAQ:BBIO).
The once-weekly injection schedule of TransCon CNP is seen as more favorable compared to the daily administration required for the other treatments. These positive topline results have led to an increase in Goldman Sachs' forecast for TransCon CNP, though the firm awaits further detailed data and regulatory updates.
Looking ahead, Ascendis Pharma is preparing to take the next steps in the regulatory process. The company has outlined plans to submit a New Drug Application (NDA) for TransCon CNP in the United States in the first quarter of 2025, followed by a Marketing Authorization Application (MAA) in the European Union in the third quarter of 2025.
In other recent news, Ascendis Pharma reported positive topline results from the phase 3 ApproaCH trial for its TransCon CNP treatment in patients with achondroplasia. The study achieved its primary endpoint, showing a significant difference in annual growth velocity compared to placebo after 52 weeks.
BofA Securities and Stifel maintained Buy ratings for Ascendis Pharma, with price targets of $175.00 and $200.00, respectively. Citi also reaffirmed its Buy rating, maintaining a price target of $178.00.
In addition, Ascendis Pharma revealed promising signs from its ongoing Phase 1/2 IL-Believe Trial, with TransCon IL-2 β/γ demonstrating clinical activity in patients with platinum-resistant ovarian cancer.
The company also secured a new funding agreement with Royalty Pharma worth $150 million and received U.S. approval for its product YORVIPATH for adult hypoparathyroidism. Ascendis Pharma's R&D costs decreased by 21% year-over-year, while SG&A expenses increased due to higher employee costs.
The company ended the quarter with EUR259 million in cash and equivalents, with forecasts for SKYTROFA revenue set at EUR220 million to EUR240 million for the full year of 2024.
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