COPENHAGEN - Ascendis Pharma A/S (NASDAQ:ASND) today revealed positive topline results from its ApproaCH Trial, evaluating the efficacy of TransCon CNP in children with achondroplasia. The pivotal study showed that the investigational drug led to a statistically significant increase in annualized growth velocity (AGV) compared to placebo.
The phase 3 trial included 84 children between the ages of 2-11, randomized in a 2:1 ratio to receive either TransCon CNP or placebo. The primary endpoint at Week 52 indicated that those treated with TransCon CNP had an LS mean AGV of 5.89 cm/year, surpassing the 4.41 cm/year observed in the placebo group, with an LS mean difference of 1.49 cm/year (p
Secondary endpoints, including changes in ACH Height Z-score and CDC Height Z-score, also favored TransCon CNP treatment. The drug was generally well-tolerated with a low incidence of mild injection site reactions.
TransCon CNP is a once-weekly administered prodrug designed to release active CNP continuously, potentially offering a more convenient treatment option for achondroplasia with reduced injection frequency. Dr. Ravi Savarirayan, an investigator in the trial, expressed optimism about TransCon CNP as a potential new treatment that could lessen caregiver burden and improve patient compliance.
Ascendis plans to submit a New Drug Application to the U.S. Food & Drug Administration for TransCon CNP in the first quarter of 2025, followed by a Marketing Authorisation Application to the European Medicines Agency in the third quarter of the same year.
Jan Mikkelsen, President and CEO of Ascendis, highlighted the potential of TransCon CNP to provide catch-up growth for children with achondroplasia, noting the company's ambition to lead in the treatment of skeletal dysplasias and growth disorders.
The company will host an investor conference call today to discuss the trial results. This announcement is based on a press release statement from Ascendis Pharma.
In other recent news, Ascendis Pharma has shown 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 reported a decrease in SKYTROFA revenue due to adjustments and higher sales deductions, but secured a new funding agreement with Royalty Pharma worth $150 million. Ascendis Pharma's product YORVIPATH for adult hypoparathyroidism received U.S. approval.
Analyst firms including Citi, TD Cowen, and Oppenheimer have maintained or upgraded their ratings on Ascendis Pharma. Citi kept its Buy rating with a target of $178, while TD Cowen maintained its Buy rating with a target of $157. Oppenheimer upgraded the company from Perform to Outperform.
These recent developments come as Ascendis Pharma anticipates data from its ApproaCH trial for TransCon CNP, a treatment for achondroplasia. The company's R&D costs decreased by 21% year-over-year, while SG&A expenses increased due to higher employee costs. Ascendis Pharma 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.
InvestingPro Insights
As Ascendis Pharma A/S (NASDAQ:ASND) announces promising trial results for TransCon CNP, investors may be weighing the implications for the company's financial health and market position. According to InvestingPro, analysts have adjusted their earnings expectations downward for Ascendis Pharma in the upcoming period. This revision could suggest a cautious outlook on the company's near-term profitability, particularly as InvestingPro Tips indicate that analysts do not expect the company to be profitable this year.
InvestingPro Data metrics shed light on the company's current financial standing. Ascendis Pharma operates with a moderate level of debt, which is an important consideration for investors as it impacts financial flexibility and risk. Moreover, the company's short-term obligations surpass its liquid assets, which may raise concerns about liquidity and its ability to meet immediate financial commitments. Despite these challenges, Ascendis Pharma has demonstrated a high return over the last decade, reflecting its potential for long-term growth.
Investors should also note that Ascendis Pharma is trading at a high revenue valuation multiple, which indicates a premium market valuation relative to its revenue. This could be a reflection of investor confidence in the company's growth prospects, particularly in light of the recent positive trial outcomes. However, it's important to consider whether this valuation is sustainable in the absence of near-term profitability.
For those interested in a deeper analysis, InvestingPro offers additional insights on Ascendis Pharma, including further metrics and tips that can help investors make informed decisions. As of now, there are six more InvestingPro Tips available for Ascendis Pharma at InvestingPro.
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