Dr. Klaus Paulini reports
AETERNA ZENTARIS ANNOUNCES COMPLETION OF PATIENT RECRUITMENT IN DOSE-FINDING PEDIATRIC STUDY OF MACIMORELIN
Aeterna Zentaris Inc.
has successfully completed patient recruitment for the first pediatric study of
as a growth hormone stimulation test for the evaluation of growth hormone deficiency (GHD) in children. This study, AEZS-130-P01, is the first of two studies as agreed with the European Medicines Agency (EMA) in the company's pediatric investigation plan (PIP) for macimorelin. Macimorelin, a ghrelin agonist, is an orally active small molecule that stimulates the secretion of growth hormone from the pituitary gland into the circulatory system.
The goal of study P01 is to establish a dose that can both be safely administered to pediatric patients and cause a clear rise in growth hormone concentration in subjects ultimately diagnosed as not having GHD. The recommended dose derived from study P01 will be evaluated in the pivotal second study, AEZS-130-P02, on diagnostic efficacy and safety. Study P01 is an international, multicentre study which is being conducted in Hungary, Poland, Ukraine, Serbia and Russia.
Dr. Nicola Ammer, chief medical officer of Aeterna, commented: "We are pleased to have completed patient recruitment in this important study for the pediatric development of macimorelin. In all dosing cohorts of study P01, macimorelin administration was completed safely at the end of December, 2019."
Study P01 is an open-label, group comparison, dose escalation trial designed to investigate the safety, tolerability and pharmacokinetic/pharmacodynamic (PK/PD) of macimorelin acetate after ascending single oral doses of macimorelin at 0.25, 0.5 and 1.0 milligram per kilogram body weight in pediatric patients from two to less than 18 years of age with suspected GHD. The company enrolled a total of 24 pediatric patients across the three cohorts of the study.
"The completion of patient recruitment in study P01 represents a significant milestone and an important step in the pediatric development of macimorelin in this important patient population. We are looking forward to the successful completion of study P01 and expect to report final results in the second quarter of 2020," said
Dr. Klaus Paulini, chief executive officer of Aeterna.
Per study protocol, all enrolled patients will complete four study visits after successful completion of the screening period. At visit 1 and visit 3, a provocative GH stimulation test will be conducted according to the study sites' local practices. At visit 2, the macimorelin test will be performed: Following the oral administration of the macimorelin solution, blood samples will be taken at predefined times for PK/PD assessment. Visit 4 is a safety follow-up visit at study end.
For more information about study P01, please visit
the European Union clinical trials register
and reference EudraCT No. 2018-001988-23.
Macimorelin, a ghrelin agonist, is an orally active small molecule that stimulates the secretion of growth hormone from the pituitary gland. Stimulated growth hormone levels are measured in blood samples after oral administration of macimorelin for the assessment of GHD.
In December, 2017, the United States Food and Drug Administration (FDA) granted Aeterna Zentaris marketing approval for macimorelin to be used in the diagnosis of patients with adult growth hormone deficiency (AGHD). Macrilen has been granted orphan drug designation by the FDA for diagnosis of AGHD. In January, 2019, the European Commission granted marketing authorization for macimorelin to Aeterna Zentaris for diagnosis of growth hormone deficiency in adults. In March, 2017, the pediatric committee of the EMA agreed to the company's PIP for macimorelin, a prerequisite for filing a marketing authorization application for any new medicinal product in Europe.
About Aeterna Zentaris Inc.
Aeterna Zentaris is a specialty biopharmaceutical company engaged in commercializing novel pharmaceutical therapies, principally through outlicensing arrangements. Aeterna Zentaris is the licensor and party to a licence and assignment agreement with Novo Nordisk A/S to carry out joint development, manufacturing, registration, regulatory and supply chain for the commercialization of Macrilen (macimorelin), which is to be used in the diagnosis of patients with adult growth hormone deficiency in the United States and Canada. The clinical development of Macrilen (macimorelin) for pediatric use is continuing.
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