CHARLESTON, S.C.--(BUSINESS WIRE)--Aeterna Zentaris Inc. (NASDAQ: AEZS)(TSX: AEZ) (the “Company”) today announced that Jose M. Garcia, M.D., Ph.D., an Associate Professor of Medicine at the Puget Sound Veterans Administration Hospital and the University of Washington, will present an abstract entitled “Validation of Macimorelin as a Diagnostic Test for Adult Growth Hormone Deficiency (AGHD): A Phase 3 Study in Comparison with the Insulin Tolerance Test (ITT)” from 1:00 pm through 3:00 pm E.T. on Sunday, April 1, 2017 at the 99th Annual Meeting of the Endocrine Society in Orlando, Florida.
In the abstract, Dr. Garcia and the co-authors of the abstract conclude, based on their review of the data from the confirmatory Phase 3 study of Macrilen™, that growth hormone stimulation with macimorelin is a simple, well-tolerated, reproducible and safe diagnostic test for AGHD, with comparable accuracy to that of the ITT and that macimorelin results in a more potent growth hormone stimulatory release compared to the ITT. Dr. Garcia will present his conclusion to the US Food and Drug Administration (“FDA”) at the end of March 2017 at a meeting arranged by the Company. The Company will seek the FDA’s concurrence with the Company’s opinion that Macrilen™ demonstrated performance supportive of achieving registration.
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 under the tradename Macrilen™.
About the Study
The confirmatory Phase 3 clinical study of Macrilen™, entitled Confirmatory validation of oral macimorelin as a growth hormone (GH) stimulation test (ST) for the diagnosis of adult growth hormone deficiency (AGHD) in comparison with the insulin tolerance test (ITT), was designed as a two-way crossover study with the ITT as the benchmark comparator and involved some 26 sites in the United States and Europe. The trial involved 157 subjects, of whom 140 completed two evaluable tests for AGHD using both Macrilen™ and the ITT. Thirty-four of the patients were evaluated using Macrilen™ a second time to measure the repeatability of the result obtained using Macrilen™ as the evaluation method. The study population consisted of 115 patients who were suspected of having AGHD as a result of the presence of one or more symptoms or risk factors. This segment of the population included a range of patients from those considered at low risk of having AGHD to those considered at high risk. The study population also included 25 healthy subjects, who had no known risk of having AGHD. Under the study protocol, the evaluation of AGHD with Macrilen™ will be considered successful, if the lower bound of the two-sided 95% confidence interval (or lower bound of the one-sided 97.5% confidence interval) for the primary efficacy variables is 75% or higher for “percent negative agreement”, and 70% or higher for the “percent positive agreement”. Dr. Jose M. Garcia, MD, PhD, an Associate Professor of Medicine at the Puget Sound VA Hospital and the University of Washington in Seattle, was the principal investigator of the confirmatory Phase 3 clinical trial. More details about the trial are available at the following link: https://www.clinicaltrials.gov/ct2/show/NCT02558829?term=macimorelin&rank=1.
We announced the top-line results from the confirmatory Phase 3 study on January 4, 2017. In the announcement, we stated that, while the estimated percent negative agreement met the success criteria, the estimated percent positive agreement did not reach the criteria for a successful outcome. We believe that the estimated percent negative agreement is considered as the more relevant endpoint, and that, therefore, the study demonstrated that the Macrilen™ test provides medical benefit.
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.
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.