Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZ) (the "Company") today announced plans to conduct a new, confirmatory Phase 3 clinical study to demonstrate the efficacy of Macrilen™ (macimorelin), a novel orally-active ghrelin agonist for use in evaluating adult growth hormone deficiency ("AGHD"), as well as a dedicated thorough QT study to evaluate the effect of Macrilen™ on myocardial repolarization. This decision follows a positive and helpful meeting with the U.S. Food and Drug Administration ("FDA") regarding its New Drug Application for Macrilen™. The Company requested the meeting to gain clarity on the approval deficiencies described in the Complete Response Letter ("CRL") the Company received on November 6, 2014.
Following receipt of the CRL, the Company convened a panel of US and EU endocrinology experts to advise it regarding the options for Macrilen™. The panel advised the Company to continue to seek approval for the compound because of their confidence in its efficacy and because there currently is no FDA-approved diagnostic test for AGHD. Dr. Richard Sachse, Chief Scientific Officer and Chief Medical Officer of the Company stated, "I am very grateful for the advice of our advisory committee and for their confidence in Macrilen™. Their recommendations were very helpful in formulating our proposal to the FDA. Also, as we've continued to evaluate the available information on Macrilen™, we continue to believe further trials will confirm the efficacy and safety of this product. Our intent is to successfully develop this product, providing a much needed improved and FDA-approved method for endocrinologists to evaluate and diagnose AGHD."
During an end-of-review meeting with the FDA on March 6, 2015, the Company and the FDA agreed on the general design of the confirmatory study as well as evaluation criteria. The study will be conducted as a two-way crossover 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 approval prior to commencing the confirmatory study.
David A. Dodd, Chairman and Chief Executive Officer of the Company commented on the future development of MacrilenTM: "We are committed to moving forward rapidly with the development and trials of MacrilenTM. We believe that completion of the confirmatory Phase 3 study and the QT study will take about 18 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. I want to thank my team for their extraordinary efforts in dealing with the Complete Response Letter and in quickly finding a path forward for MacrilenTM."
The Company's goal is to conduct a Phase 3 study that will satisfy the registration requirements of the European Medicines Agency ("EMA") as well as the FDA. The Company expects to receive comments from the EMA regarding the study design during a Scientific Advice Meeting in early May.