Aeterna Zentaris Announced Presentation of Positive Results from First Pediatric Study of Macimorelin at the 22nd European Congress of Endocrinology
- PK and PD profiles of macimorelin for pediatric use were within the expected range and comparable to results observed in adults -
- Data from this study support the choice of 1.0 mg/kg dose of macimorelin for validity testing in a Phase 3 trial, expected to commence in Q1 2021 -
CHARLESTON, S.C., Sept. 08, 2020 (GLOBE NEWSWIRE) -- Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZS) (the “Company”), a specialty biopharmaceutical company commercializing and developing therapeutics and diagnostic tests, today announced the presentation of results from its first pediatric study of macimorelin as a growth hormone stimulation test for the evaluation of childhood-onset growth hormone deficiency (“CGHD”) at the 22nd European Congress of Endocrinology (e-ECE 2020), being held September 5-9, 2020.
The data were presented during the event in an ePoster titled, Pharmacokinetics and pharmacodynamics of macimorelin acetate (AEZS-130) in paediatric patients with suspected growth hormone deficiency (GHD), accommodated by an audio commentary. The full poster is now available on the Company’s website.
“We are grateful to have the opportunity to present at the e-ECE 2020 congress. We are incredibly encouraged by the results from this study, which demonstrated macimorelin’s safety and tolerability, and a PK and PD profile in a range expected from the adult development program,” commented Dr. Nicola Ammer, Chief Medical Officer of Aeterna Zentaris. “As we advance into our pivotal Study P02, which we expect to initiate in the first quarter of 2021, we are confident that we have selected with 1.0 mg/kg macimorelin the correct dosing regimen based on insights gained from the Study P01.”
The AEZS-130-P01 study (“Study P01”), was an open-label, group comparison, dose escalation trial and the first of two studies as agreed with the European Medicines Agency (“EMA”) in the Company’s Pediatric Investigation Plan (“PIP”) for macimorelin. Study P01 was designed to investigate the pharmacokinetics (“PK”), pharmacodynamics (“PD”), safety, and tolerability of macimorelin after single oral dosing of 0.25, 0.5, and 1.0 mg/kg in pediatric patients with suspected growth hormone deficiency (“GHD”).
The Company completed the study and announced positive results in April 2020. The completed study included 24 subjects aged 4 to 15 years. In the subjects who completed the study in accordance with the protocol, macimorelin demonstrated an excellent safety and tolerability profile. There were 88 adverse events (“AE”) reported in 23 subjects, none of which was assessed by the investigator as related to macimorelin. The majority of AEs (approximately 70%) were expected side effects related to the hypoglycemia introduced by the Insulin Tolerance Test. No significant changes in ECG parameters and safety laboratory values were noted in any of the three dosing cohorts.