Once-Weekly TransCon™ CNP Achieved Primary Efficacy Objective, with Superiority at 100 µg/kg/week, in Ascendis Pharma’s Phase 2 ACcomplisH Trial of Children with Achondroplasia Aged 2 to 10 Years

-   Data demonstrated that once-weekly TransCon CNP has the potential to meet patient and caregiver needs for a safe, effective, tolerable and convenient treatment

-   The primary endpoint, annualized height velocity (AHV) at Week 52, demonstrated superiority of TransCon CNP at 100 µg/kg/week compared to placebo (p=0.0218)

-   TransCon CNP was generally well tolerated with low frequency of injection site reactions; all 57 randomized children continued, with the longest treatment duration beyond two years

-   Data showed robust and consistent results in prespecified analyses across age groups and dose levels, supporting continued development at the selected dose of 100 µg/kg/week

COPENHAGEN, Denmark I November 14, 2022 I Ascendis Pharma A/S (Nasdaq: ASND) today announced positive topline results from the ACcomplisH Trial, its Phase 2 randomized, double-blind, placebo-controlled, dose-escalation trial evaluating the safety and efficacy of once-weekly TransCon CNP compared to placebo in children with achondroplasia aged 2 to 10 years old. TransCon CNP is an investigational long-acting prodrug of C-type natriuretic peptide (CNP), designed to provide continuous exposure of CNP at safe, therapeutic levels via a single, weekly subcutaneous dose.

The ACcomplisH Trial evaluated 57 children with achondroplasia aged 2 to 10 years old, randomized in a 3:1 ratio to receive either sequential ascending doses of once-weekly TransCon CNP or placebo for 52 weeks. All 57 randomized children completed the blinded portion of ACcomplisH and are currently continuing in the open label extension (OLE) at the 100 µg/kg/week dose.

The trial met its primary objectives, demonstrating that TransCon CNP at 100 µg/kg/week was superior to placebo on the primary efficacy endpoint of AHV at 52 weeks. A slide presentation with these data can be found on the Investor Relations & News section of the Ascendis Pharma website: https://investors.ascendispharma.com.

Key data include:

TransCon CNP Dose Group (n) AHV (cm/year)
LS Mean [95% CI]
p-value
(TransCon CNP vs. Pooled Placebo)
6 µg/kg/week (n=10) 4.09
[3.34, 4.84]
0.6004
20 µg/kg/week (n=11) 4.52,
[3.82, 5.22]
0.7022
50 µg/kg/week (n=10) 5.16
[4.43, 5.90]
0.0849
100 µg/kg/week (n=11) 5.42
[4.74, 6.11]
0.0218
Pooled Placebo (n=15) 4.35
[3.75, 4.94]
NA

Additional highlights:

  • TransCon CNP demonstrated a consistent dose-response in AHV across the four dose groups.
  • Mean improvements in AHV for TransCon CNP-treated patients were consistent across age groups <5 years and >5 years, with dose response established.
  • TransCon CNP at 100 µg/kg/week demonstrated superiority in change in ACH-specific height SDS compared to placebo.
  • TransCon CNP was generally safe and well tolerated, with no discontinuations.
  • No serious AEs (SAEs) related to treatment were reported; two unrelated SAEs were reported.
  • Injections were generally well tolerated with low frequency of injection site reactions (ISRs):
    • 11 mild ISRs (in 8 patients) out of >2,000 injections.
  • Patients treated ≥6 months at 100 µg/kg/week in the blinded or OLE period demonstrated a consistent and sustained response, with mean AHV of 5.39 cm/year (n=40).

“Development of a drug with potential to be both an accepted and a preferred treatment option for children with achondroplasia and their caregivers has a high bar related to safety, efficacy, tolerability and convenience,” said Aimee Shu, M.D., Vice President, Clinical Development, Endocrine & Rare Diseases at Ascendis Pharma. “We are excited to see once-weekly TransCon CNP demonstrated statistically superior growth compared to placebo at the 100 µg/kg/week dose level, with safety and tolerability results supporting further development in children with achondroplasia.”

About Ascendis Pharma A/S
Ascendis Pharma is applying its innovative TransCon platform to build a leading, fully integrated, global biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of patients, science and passion, the company uses its TransCon technologies to create new and potentially best-in-class therapies. Ascendis is headquartered in Copenhagen, Denmark, and has additional facilities in Heidelberg and Berlin, Germany; Palo Alto and Redwood City, California; and Princeton, New Jersey. Please visit www.ascendispharma.com to learn more.

SOURCE: Ascendis Pharma

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