Aeterna Zentaris’ confirmatory Phase 3 study for Macrilen in AGHD meets EMA’s study-design expectations

Aeterna Zentaris has received written scientific advice from the European Medicines Agency (EMA) regarding the further development plan, including the study design, for the new confirmatory Phase 3 clinical study of Macrilen™ (macimorelin) for use in evaluating adult growth hormone deficiency (AGHD).

As a result of the advice, the Company believes that the confirmatory Phase 3 study now meets with the EMA’s study-design expectations. 

Dr Richard Sachse, Aeterna Zentaris’ Chief Scientific Officer and Chief Medical Officer, stated, “We are very pleased with the EMA’s advice as we continue to believe that our trial will confirm the efficacy and safety of this product. In principle, the EMA agreed with our proposed protocol design in the evaluation of the product. In addition, it proposed that we consider additional aspects regarding the demonstration of reproducibility of a diagnosis made using Macrilen, which will further enhance the profile of our compound. We believe we will be able to provide the information that EMA requires in a timely and cost effective manner.”

David A. Dodd, Chairman and Chief Executive Officer of the Company, commented on the future development of Macrilen, “We are committed to moving forward swiftly with the development of MacrilenTM in AGHD. We believe that completion of the confirmatory Phase 3 study and the QT study will take 15 to18 months and will require a combined expenditure of between $5 million and $6 million. We have the resources necessary to bring this product to market and intend to do so as rapidly as possible, pending regulatory approvals.” 

A Phase 3 QT study to evaluate the effecy of Macrilen on myocardial repolarisatoin is planned for 2016

AGHD affects approximately 75,000 adults across the US, Canada and Europe. Growth hormone not only plays an important role in growth from childhood to adulthood, but also helps promote a hormonally-balanced health status.

The confirmatory efficacy Phase 3 clinical study with Macrilen for the evaluation of AGHD will be conducted as a two-way crossover study with the insulin tolerance test as the benchmark comparator. The study population will consist of patients with a medical history documenting risk factors for AGHD and will include a spectrum of patients from those with a low risk of having AGHD to those with a high risk of having the condition. The Company will submit a proposed final protocol to the FDA for their review prior to commencing the confirmatory efficacy Phase 3 study. A dedicated thorough Phase 3 QT study to evaluate the effect of Macrilen on myocardial repolarisation is expected to be initiated in early 2016.