All amounts are in US dollars
QUÉBEC CITY, April 13, 2015 /PRNewswire/ - 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.
About MacrilenTM (macimorelin)
Macimorelin, a ghrelin agonist, is a novel orally-active small molecule that stimulates the secretion of growth hormone. Macimorelin, under the trade name MacrilenTM, has been granted orphan drug designation by the FDA for diagnosis of AGHD. The Company owns the worldwide rights to this novel patented compound.
About Adult Growth Hormone Deficiency (AGHD)
AGHD affects approximately 75,000 adults across the U.S., 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. AGHD mostly results from damage to the pituitary gland. It is usually characterized by a reduction in bone mineral density, lean body mass, exercise capacity, and overall quality of life.
About Aeterna Zentaris
Aeterna Zentaris is a specialty biopharmaceutical company engaged in developing and commercializing novel treatments in oncology, endocrinology and women’s health. For more information, visit www.aezsinc.com.
Forward-Looking Statements
This press release contains forward-looking statements made pursuant to the safe harbor provisions of the US Securities Litigation Reform Act of 1995. Forward-looking statements involve known and unknown risks and uncertainties that could cause the Company’s actual results to differ materially from those in the forward-looking statements. Such risks and uncertainties include, among others, the availability of funds and resources to pursue R&D projects and clinical trials, the successful and timely completion of clinical studies, the risk that safety and efficacy data from any of our Phase 3 trials may not coincide with the data analyses from previously reported Phase 1 and/or Phase 2 clinical trials, the ability of the Company to efficiently commercialize one or more of its products or product candidates, the ability of the Company to take advantage of business opportunities in the pharmaceutical industry, uncertainties related to the regulatory process, the ability to protect our intellectual property, the potential of liability arising from shareholder lawsuits and general changes in economic conditions. Investors should consult the Company’s quarterly and annual filings with the Canadian and US securities commissions for additional information on risks and uncertainties relating to forward-looking statements. Investors are cautioned not to place undue reliance on these forward-looking statements. The Company does not undertake to update these forward-looking statements. We disclaim any obligation to update any such factors or to publicly announce the result of any revisions to any of the forward-looking statements contained herein to reflect future results, events or developments, unless required to do so by a governmental authority or by applicable law.
SOURCE Aeterna Zentaris Inc.
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