Aeterna Zentaris Announces Completion of Patient Recruitment in Dose-Finding Pediatric Study of Macimorelin

– Study P01 is the first of two studies as agreed with the EMA in the Company’s Pediatric Investigation Plan for macimorelin as a growth hormone deficiency diagnostic –

– Study P01 final study results expected in Q2 2020 –

CHARLESTON, S.C., Jan. 28, 2020 (GLOBE NEWSWIRE) -- Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZS) (“Aeterna” or the “Company”), a specialty biopharmaceutical company engaged in commercializing novel pharmaceutical therapies, today announced the successful completion of patient recruitment for the first pediatric study of macimorelin as a growth hormone stimulation test for the evaluation of growth hormone deficiency (“GHD”) in children. This study, AEZS-130-P01 (“Study 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, multicenter 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 mg per kg body weight in pediatric patients from 2 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.