Aeterna Zentaris Inc. (NASDAQ: AEZS)(TSX: AEZS) (the “Company”) announced today that the Pediatric Committee (“PDCO”) of the European Medicines Agency (“EMA”) agreed to the Company’s Pediatric Investigation Plan (“PIP”) for Macrilen™ and agreed that the Company may defer conducting the PIP until after it files a Marketing Authorization Application (“MAA”) seeking marketing authorization for the use of Macrilen™ for the evaluation of adult growth hormone deficiency.
As part of the regulatory process for the registration of new medicines in Europe, pharmaceutical companies are required to provide a PIP outlining their strategy for investigation of the new medicinal product in the pediatric population. An accepted PIP is a prerequisite for filing an MAA for any new medicinal product in Europe. However, the Company will be able to file an MAA substantially earlier than if it was required to complete the PIP before filing, because the PDCO permitted the Company to defer conducting the studies defined in the PIP.
The Company’s PIP provides for a dose-escalation safety and PK/PD study followed by a diagnostic efficacy and safety study. The goal of the first study is to establish a dose that can safely be administered to pediatric patients and that causes a clear rise in growth hormone concentration in subjects ultimately diagnosed as not having growth hormone deficiency (“GHD”). The recommended dose derived from this study will be evaluated in the second study for diagnostic efficacy and safety. Both studies will be conducted in patients at least three years old who are undergoing the study sites’ regular diagnostic evaluation for the presence of GHD.
Dr. Richard Sachse, M.D., Ph.D., Senior Vice President, Chief Medical Officer and Chief Scientific Officer of the Company, said: “PDCO’s agreement that we may defer conducting our PIP, allowing us to go forward with an MAA in adults prior to initiating of the pediatric development program, is an important step forward in our efforts to bring Macrilen™ to market. Likewise, acceptance of our PIP is an important regulatory milestone for the product. At this time, we are looking forward to our upcoming FDA meeting, which has been scheduled for late March 2017, as a pre-requisite for a potential NDA submission based on our most recently announced completion of the pivotal Phase 3 study. Similarly, we are preparing for meetings with European agencies to discuss whether the conducted development program suffices for an MAA.”
About MacrilenTM (macimorelin)
Macimorelin, a ghrelin receptor agonist, is an orally-active small molecule that stimulates the secretion of growth hormone. Macimorelin has been granted orphan drug designation by the FDA for diagnosis of AGHD. The Company owns the worldwide rights to this patented compound and has significant patent protection left. The Company’s U.S. composition of matter patent expires in 2022 and its U.S. utility patent runs through 2027. The Company proposes, subject to FDA approval, to market macimorelin in the United States under the tradename Macrilen™.
About AGHD
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. 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 as well as an increase of cardiovascular risks.
About Aeterna Zentaris Inc.
Aeterna Zentaris is a specialty biopharmaceutical company engaged in developing and commercializing novel treatments in oncology, endocrinology and women’s health. We are engaged in drug development activities and in the promotion of products for others. We recently completed Phase 3 studies of two internally developed compounds. The focus of our business development efforts is the acquisition of licenses to products that are relevant to our therapeutic areas of focus. We also intend to license out certain commercial rights of internally developed products to licensees in non-US territories where such out-licensing would enable us to ensure development, registration and launch of our product candidates. Our goal is to become a growth-oriented specialty biopharmaceutical company by pursuing successful development and commercialization of our product portfolio, achieving successful commercial presence and growth, while consistently delivering value to our shareholders, employees and the medical providers and patients who will benefit from our products. For more information, visit www.aezsinc.com.
Forward-Looking Statements
This press release contains forward-looking statements made pursuant to the safe harbor provision of the U.S. Securities Litigation Reform Act of 1995, which reflect our current expectations regarding future events. Forward-looking statements may include, but are not limited to statements preceded by, followed by, or that include the words “expects,” “believes,” “intends,” “anticipates,” and similar terms that relate to future events, performance, or our results. Forward-looking statements involve known risks and uncertainties, many of which are discussed under the caption “Key Information – Risk Factors” in our most recent Annual Report on Form 20-F filed with the relevant Canadian securities regulatory authorities in lieu of an annual information form and with the U.S. Securities and Exchange Commission (“SEC”). Such statements include, but are not limited to, statements about the progress of our research, development and clinical trials and the timing of, and prospects for, regulatory approval and commercialization of our product candidates, the timing of expected results of our studies, anticipated results of these studies, statements about the status of our efforts to establish a commercial operation and to obtain the right to promote or sell products that we did not develop and estimates regarding our capital requirements and our needs for, and our ability to obtain, additional financing. Known and unknown risks and uncertainties could cause our actual results to differ materially from those in forward-looking statements. Such risks and uncertainties include, among others, the availability of funds and resources to pursue our research and development 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 rejection or non-acceptance of any new drug application by one or more regulatory authorities and, more generally, uncertainties related to the regulatory process (including whether or not the regulatory authorities will accept the Company’s conclusions regarding Macrilen™ following its comprehensive review of the Phase 3 study data described elsewhere in this press release), the ability of the Company to efficiently commercialize one or more of its products or product candidates, the degree of market acceptance once our products are approved for commercialization, our ability to take advantage of business opportunities in the pharmaceutical industry, our ability to protect our intellectual property, and 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 securities commissions and the SEC for additional information on risks and uncertainties. Given these uncertainties and risk factors, readers are cautioned not to place undue reliance on these forward-looking statements. We disclaim any obligation to update any such factors or to publicly announce 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 applicable law.
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Contacts:
Philip A. Theodore, 843-900-3223
Senior
Vice President
ir@aezsinc.com
