Data from Phase 2 ACcomplisH Trial of TransCon CNP in Children with Achondroplasia Presented at ICCHBH 2024
Ascendis Pharma's Phase 2 ACcomplisH Trial data on TransCon CNP for children with achondroplasia was presented at ICCBH 2024. The trial showed significant improvements in well-being and physical functioning compared to placebo at the 100μg/kg/week dose, suggesting benefits beyond linear growth. Conducted in 57 children aged 2-10, the trial's results highlighted TransCon CNP's potential to enhance health-related quality of life. Statistically significant improvements were noted in daily living functioning and emotional well-being, with no serious adverse events reported.
- Significant improvements in well-being and physical functioning compared to placebo.
- Potential additional direct treatment effects of TransCon CNP beyond linear growth.
- Statistically significant improvements in Daily Living Functioning (p=0.047) and Emotional Well-Being (p=0.045).
- No serious adverse events related to the study drug.
- Most treatment-emergent adverse events were Grade 1-2, with a low frequency of injection site reactions.
- None.
Insights
The Phase 2 ACcomplisH Trial results for TransCon CNP in children with achondroplasia show promising improvements beyond just linear growth. The significant enhancements in well-being and physical functioning suggest that TransCon CNP might offer comprehensive benefits for patients. This could position the drug as a multifaceted treatment option, addressing both physical stature and quality of life aspects.
The trial's methodological rigor, being a multicenter, randomized, double-blind and placebo-controlled study, strengthens the credibility of these findings. The observed significant improvements in daily living functioning and emotional well-being were statistically validated, with p-values of
An important point to highlight is the drug's safety profile. The absence of serious adverse events and the prevalence of only mild side effects (mostly Grade 1-2) suggest good tolerability, which is important for long-term pediatric treatments. Additionally, the consistency in growth results across the trial reaffirms the primary efficacy of the pivotal dose.
For a retail investor, these results could signify potential long-term benefits and increased market adoption, given the unmet need in achondroplasia treatments. However, it's essential to remain cautious until further phases confirm these findings and the drug potentially achieves regulatory approval.
The data from this trial opens up new avenues for Ascendis Pharma in the market of achondroplasia treatments. The significant improvements in quality of life and physical functioning point to a potential first-mover advantage in a niche market with few treatment options available. This could lead to increased market share and revenue potential once the drug progresses through further clinical stages and gains regulatory approval.
The participation in an international conference like ICCBH also enhances the company's visibility and credibility within the medical community. Such exposure can facilitate partnerships and collaborative opportunities, possibly driving further innovations and advancements.
From an investor's perspective, the positive trial outcomes and the favorable safety profile could enhance investor confidence, potentially leading to an upward trend in stock prices. However, it is important to monitor upcoming phases of trials and regulatory milestones to gauge the drug's commercial viability fully.
• Data demonstrate significant improvements in well-being and physical functioning compared to placebo in patients treated for 1 year with TransCon CNP at the pivotal 100μg/kg/week dose
• Improvements could not be explained by changes in linear growth only, supporting a potential additional direct treatment effect of TransCon CNP beyond linear growth
COPENHAGEN, Denmark, June 24, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced the oral presentation of data from its Phase 2 ACcomplisH Trial of TransCon CNP (navepegritide) in children aged 2 to 10 years old with achondroplasia by Ravi Savarirayan, M.D., Murdoch Children’s Research Center (Australia), during ICCBH 2024, the International Conference on Children’s Bone Health being held in Salzburg, Austria.
The data showed that patients treated for one year at the pivotal 100μg/kg/week dose demonstrated significant improvements in well-being and physical functioning compared to placebo. These improvements could not be explained by changes in linear growth only, supporting a potential additional direct treatment effect of TransCon CNP beyond linear growth.
“Once-weekly TransCon CNP has demonstrated a positive impact on linear growth with a favorable safety profile, as well as benefits beyond growth,” said Dr. Savarirayan. “As the first investigational pharmaceutical treatment for achondroplasia to demonstrate statistically significant improvements compared to placebo in health-related quality of life measures, TransCon CNP at the pivotal dose has potential to meet the need for a treatment addressing the health and quality-of-life complications of this condition.”
TransCon CNP is an investigational prodrug of C-type natriuretic peptide (CNP) administered once weekly, designed to provide sustained release of active CNP. ACcomplisH is a Phase 2, multicenter, randomized, double-blind, placebo-controlled, dose-escalation trial of once-weekly TransCon CNP versus placebo in 57 children with achondroplasia (aged 2-10 years old). Patients were randomized 3:1 to receive TransCon CNP across 4 dose-escalation cohorts or placebo for 52 weeks, after which participants could receive TransCon CNP in an ongoing open-label extension at the 100μg/kg/week dose. Health-related quality of life assessments compared 52-week results from patients whose initial TransCon CNP dose was 100μg/kg/week (the pivotal trial dose) with results from the pooled placebo group in the randomized period. Of the 57 participants, 56 continue with open-label treatment.
Statistically significant improvements were observed for various exploratory endpoints including Daily Living Functioning (p=0.047; n=16; 13 [treated;placebo]) and Emotional Well-Being (p=0.045; n=13;9) domains of the Achondroplasia Child Experience Measure-Impact (ACEM) assessment, and the SF-10 Physical Summary among participants 5 years of age or older (p=0.002; n=9;5). Growth across the full trial population (n=57) on TransCon CNP at the 100μg/kg/week dose for 52 weeks was consistent with results from this dose cohort during the randomized period.
No new safety signals were observed, and no serious adverse events related to the study drug were reported. Most treatment-emergent adverse events (TEAEs) were Grade 1-2, with a low frequency of injection site reactions.
About Ascendis Pharma A/S
Ascendis Pharma is applying its innovative TransCon technology platform to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of Patients, Science, and Passion, Ascendis uses its TransCon technologies to create new and potentially best-in-class therapies. Ascendis is headquartered in Copenhagen, Denmark and has additional facilities in Europe and the United States. Please visit ascendispharma.com to learn more.
Forward-Looking Statements
This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding Ascendis’ future operations, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to (i) improvements in well-being and physical functioning supporting a potential additional direct treatment effect of TransCon CNP, (ii) TransCon CNP’s potential to meet the need for a treatment addressing the health and quality-of-life complications of achondroplasia, (iii) Ascendis’ ability to apply its TransCon technology platform to build a leading, fully integrated biopharma company, and (iv) Ascendis’ use of its TransCon technologies to create new and potentially best-in-class therapies. Ascendis may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations, and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements that Ascendis makes, including the following: dependence on third party manufacturers, distributors and service providers for Ascendis’ products and product candidates; unforeseen safety or efficacy results in Ascendis’ development programs or on-market products; unforeseen expenses related to commercialization of any approved Ascendis products; unforeseen expenses related to Ascendis’ development programs; unforeseen selling, general and administrative expenses, other research and development expenses and Ascendis’ business generally; delays in the development of its programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; Ascendis’ ability to obtain additional funding, if needed, to support its business activities; the impact of international economic, political, legal, compliance, social and business factors. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Ascendis’ business in general, see Ascendis’ Annual Report on Form 20-F filed with the U.S. Securities and Exchange Commission (SEC) on February 7, 2024, and Ascendis’ other future reports filed with, or submitted to, the SEC. Forward-looking statements do not reflect the potential impact of any future licensing, collaborations, acquisitions, mergers, dispositions, joint ventures, or investments that Ascendis may enter into or make. Ascendis does not assume any obligation to update any forward-looking statements, except as required by law.
Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo, and TransCon are trademarks owned by the Ascendis Pharma group. © June 2024 Ascendis Pharma A/S.
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| Tim Lee Ascendis Pharma +1 (650) 374-6343 tle@ascendispharma.com ir@ascendispharma.com Patti Bank ICR Westwicke +1 (415) 513-1284 patti.bank@westwicke.com | Melinda Baker Ascendis Pharma +1 (650) 709-8875 media@ascendispharma.com |
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