Date: 2011-09-13
Type of information:
phase: 2
Announcement: results
Company: Ascendis Pharma (USA-Denmark)
Product: ACP-001
Action mechanism: ACP-001 is a human growth-hormone prodrug that releases efficacious levels of unmodified human growth hormone in a controlled manner, allowing the drug to be administered once weekly.
Disease: growth hormone deficiency
Therapeutic area: Hormonal diseases - Endocrine diseases
Country: several Western European countries
Trial
details: The study was a randomized, four-week, multiple-dose study comparing three dose levels of once-weekly ACP-001 to daily growth hormone.
Conducted in a number of Western European countries, the Phase 2 trial was designed to investigate safety, tolerability, pharmacokinetics and pharmacodynamics in 37 adult patients with growth hormone deficiency.
Latest
news: Ascendis Pharma has announced positive results of a Phase 2 clinical study of ACP-001 in growth hormone deficient adults. The Phase 2 clinical study met all primary and secondary endpoints, the primary efficacy endpoint being the change in IGF-I levels from baseline compared to daily growth hormone.
ACP-001 administered once-weekly was as effective in stimulating IGF-I as seven daily injections of the equivalent weekly dose of growth hormone. ACP-001 stimulated a dose-proportional increase in IGF-I levels across the three dose levels. IGF-I exposure was maintained throughout the week, thereby achieving a low peak-to-trough ratio.
The Phase 2 trial also confirmed the positive safety profile of ACP-001 previously observed in the Phase 1 clinical trial, and safety comparable with daily growth hormone. No treatment-emergent anti-hGH antibody formation was observed during this multiple-dose trial.
ACP-001 was administered as a subcutaneous injection through a very fine needle (30G), and no difference in injection-site reactions were noted between ACP-001 and daily growth hormone.
