| Ticker | Status | Jurisdiction | Filing Date | CP Start | CP End | CP Loss | Deadline |
|---|
| Ticker | Case Name | Status | CP Start | CP End | Deadline | Settlement Amt |
|---|
| Ticker | Name | Date | Analyst Firm | Up/Down | Target ($) | Rating Change | Rating Current |
|---|
– Safety and efficacy study for macimorelin initiated as agreed with U.S. FDA and the European Medicines Agency ("EMA") in the Company's Pediatric Investigation Plan ("PIP")
– Patient enrollment on track to start this quarter
– Planned completion of pivotal Phase 3 study expected in Q3 2022
CHARLESTON, S.C., May 13, 2021 (GLOBE NEWSWIRE) -- Aeterna Zentaris Inc. (NASDAQ:AEZS) (TSX:AEZS) (“Aeterna” or the “Company”), a specialty biopharmaceutical company developing and commercializing a diversified portfolio of pharmaceutical and diagnostic products, today announced the commencement of its pivotal Phase 3 safety and efficacy study AEZS-130-P02 (“the DETECT-trial”) evaluating macimorelin for the diagnosis of childhood-onset growth hormone deficiency (“CGHD”).
The investigational new drug application (“IND”), “Multicenter, open-label trial to investigate the efficacy and safety of a single oral dose of 1.0 mg/kg macimorelin acetate as growth hormone stimulation test (“GHST”) in pediatric patients with suspected growth hormone deficiency (“GHD”),” for the study is active and the first clinical site in the U.S. is open for patient recruitment.
Children and adolescents from two to less than 18 years of age with suspected growth hormone deficiency are planned to be included. The study is expected to enroll approximately 100 participants worldwide, with at least 40 participants in pre-pubertal and 40 participants in pubertal status. A macimorelin GHST will be performed twice to ensure the repeatability of the data. Two standard GHSTs will be used as controls: arginine (i.v.) and clonidine (p.o.). The study design is expected to be suitable to support a claim for potential stand-alone testing with macimorelin, if successful.
“The initiation of the pivotal Phase 3 DETECT-trial is a key milestone for the Company,” commented Dr. Klaus Paulini, Chief Executive Officer of Aeterna. “While we continue to make progress on multiple fronts across our development pipeline, the launch of this pivotal trial for the diagnosis of CGHD remains an integral piece of our product portfolio and an area of key focus. As sponsors of the trial, we look forward to getting the remainder of the clinical sites active and patient enrollment underway as quickly and efficiently as possible.”
The Company announced positive results in April 2020 from AEZS-130-P01 ("Study P01"), the first of two studies as agreed with FDA and EMA. The goal of Study P01 was to establish a dose that could both be safely administered to pediatric participants and causes a clear rise in growth hormone concentration in participants ultimately diagnosed as not having GHD. Results from the study demonstrated that the pharmacokinetic and pharmacodynamic profile of macimorelin proved to be in the expected range and in general comparable to data in adults.
Under new terms and conditions of the license agreement revised in November 2020, Aeterna is closely coordinating the activities related to the development of macimorelin in CGHD through a joint steering committee with Novo Nordisk, Aeterna’s licensee for the U.S. and Canada.
For more information about Study P01 and the DETECT-trial, please visit EU Clinical Trials Register and reference EudraCT #2018-001988-23 and clinicaltrials.gov identifier NCT04786873.
