QUÉBEC CITY, June 26, 2012 /PRNewswire/ - Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZ) (the “Company”) today announced that final Phase 3 results for its oral ghrelin agonist, AEZS-130, show that the drug is safe and effective in diagnosing adult growth hormone deficiency (AGHD). Jose M. Garcia, MD, PhD, of the Baylor College of Medicine and the Michael E. DeBakey VA Medical Center, disclosed these data during an oral presentation yesterday at the 94th ENDO Annual Meeting and Expo currently being held in Houston, Texas.
The Study
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 body mass index (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.
Results
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.
Data are available on preference of the two tests for 50 subjects. Of these, 70% expressed a preference for AEZS-130 over L-ARG+GHRH.
Conclusion
Data show AEZS-130 to be safe and effective in diagnosing AGHD.
The abstract titled, “AEZS-130 (Macimorelin) - Stimulated GH Test: Validation of a Novel Test for the Diagnosis of Adult Growth Hormone Deficiency (AGHD)”, J. M. Garcia, R. Swerdloff, C. Wang, M. Kyle, M. Kipnes, B. Biller, D. Cook, K. Yuen, V. Bonert, A. Dobs, M. Molitch, G. Merriam, is available through this link.
About AEZS-130
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. AEZS-130 is also in a Phase 2A trial as a treatment for cancer induced cachexia. 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.
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