QUEBEC CITY, Oct. 18, 2012 /CNW Telbec/ - Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZ) (the "Company") today announced that Phase 3 results for its ghrelin agonist, AEZS-130, show that it has promise as a safe and simple oral diagnostic test for adult growth hormone deficiency (AGHD), with accuracy comparable to available testing procedures. Results were presented earlier today by George R. Merriam, MD, Director of the Clinical Study Unit at the VA Puget Sound Health Care System, and Professor of Medicine at the University of Washington, Seattle and Tacoma, WA, at the 6th International Congress of the Growth Hormone Research (GRS) and Insulin-like Growth Factor (IGF) Society, currently being held in Munich, Germany.
Juergen Engel, PhD, President and CEO at Aeterna Zentaris, commented, "The data presented earlier today by Dr. Merriam, extend those presented on this same study last June at ENDO by Dr. Jose M. Garcia, MD, PhD, of the Baylor College of Medicine and the Michael E. DeBakey VA Medical Center. Both confirm AEZS-130's potential as possibly the first approved oral diagnostic test for AGHD, with Dr. Merriam's data set showing the impact of Body Mass Index on cut-off values. We are currently focusing our efforts on submitting a New Drug Application for AEZS-130 in this indication during the first quarter of 2013."
This multicenter open-label study was originally designed as a cross-over trial of AEZS-130 vs growth hormone-releasing hormone (GHRH)+L-Arginine (ARG) in AGHD patients and in controls matched for Body Mass Index (BMI), estrogen status, gender and age. After 43 AGHD patients and 10 controls had been tested, GHRH became unavailable. The study was completed by testing 10 more AGHD patients and 38 controls with AEZS-130 alone.
Of the 53 AGHD subjects enrolled, 52 received AEZS-130, and 50 who had confirmed AGHD prior to study entry were included in this analysis, along with 48 controls. Two AGHD subjects could not be matched due to the combination of young age, high BMI and estrogen use. The objective of this clinical trial was to determine the efficacy and safety of AEZS-130 in the diagnosis of AGHD.
Mean peak growth hormone (GH) levels in AGHD patients and controls following AEZS-130 administration were 2.36ng/mL (range 0.03-33) and 17.71ng/mL (range 10.5-94), respectively. The receiver operating characteristic (ROC) plot analysis yielded an optimal GH cut-point of 2.7ng/mL, with 82% sensitivity, 92% specificity and a 13% misclassification rate. Obesity (BMI>30) was present in 58% of cases and controls, and peak GH levels were inversely associated with BMI in controls.
Adverse events (AE) were seen in 37% of AGHD patients and in 21% of controls following AEZS-130. In contrast, 61% of AGHD subjects and 30% of controls experienced AEs with L-ARG+GHRH. The most common AEs after AEZS-130 were unpleasant taste (19.2%) and diarrhea (3.8%) for the AGHD patients and unpleasant taste (4.2%) and diarrhea (4.2%) for the matched controls. AEs were generally mild or moderate in severity.
Of the 50 subjects studied with both stimulation tests, 70% expressed a preference for AEZS-130 over L-ARG+GHRH.
Summary and conclusions on the effect of BMI on optimal cut-off
Responses in normal subjects classified as obese, with BMI's above 30, were significantly lower than in leaner subjects. Since GH deficiency can lead to increased body fat, many of the patients also met criteria for obesity, and therefore, a lower peak GH cutoff is more accurate in separating obese normals from obese patients. Based upon these study results, a cut-off of 2.7 μg/L was optimal for subjects with a BMI ≥ 30 and a cutoff of 6.8 μg/L for subjects with a BMI of <30. Age had a much weaker effect on test performance and gender made no difference. Thus GH stimulation with oral AEZS-130 may provide a simple, rapid, safe, and well-tolerated diagnostic test for AGHD, with accuracy comparable to that of the GHRH-ARG test.
The abstract used for the presentation, "Use of an orally-active ghrelin mimetic, macimorelin (AEZS-130), as a safe, simple test for Adult Growth Hormone Deficiency (AGHD): Effect of BMI on optimal cut-off ", can be viewed by clicking on the following link.
AEZS-130, a ghrelin agonist, is a novel orally-active small molecule that stimulates the secretion of growth hormone. The Company has completed a Phase 3 trial for use as an oral diagnostic test for AGHD. AEZS-130 has been granted orphan drug designation by the FDA for use in this indication. Aeterna Zentaris owns the worldwide rights to AEZS-130.
About Aeterna Zentaris
Aeterna Zentaris is an oncology and endocrinology drug development company currently investigating treatments for various unmet medical needs. The Company's pipeline encompasses compounds at all stages of development, from drug discovery through to marketed products. For more information please visit www.aezsinc.com.
This press release contains forward-looking statements made pursuant to the safe harbour provisions of the U.S. 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. For example, because the market price of the Company's Common Shares will also be based on the Company's financial and operational results, its financial position, including its capital availabilities and liquidity resources, the development of its product pipeline, market conditions, the market perception of its business and other factors, which are unrelated to the number of shares outstanding, there can be no assurance that the market price of the Common Shares will in fact increase following the Consolidation or will not decrease in the future, or that the minimum closing bid price of the Common Shares will meet NASDAQ's minimum bid price requirement. Further, there can be no assurance that the Consolidation alone will guarantee the continued listing of the Common Shares on The NASDAQ Global Market or that the Common Shares will not be delisted due to a failure to meet other NASDAQ continued listing requirements. Other such risks and uncertainties include, among others, the availability of funds and resources to pursue R&D projects, 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 take advantage of business opportunities in the pharmaceutical industry, uncertainties related to the regulatory process and general changes in economic conditions. Investors should consult the Company's quarterly and annual filings with the Canadian and U.S. securities commissions for additional information on risks and uncertainties relating to forward-looking statements. Investors are cautioned not to rely 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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