Trial conducted through a Cooperative Research and Development Agreement
QUÉBEC CITY, March 8, 2012 /CNW Telbec/ - Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZ) (the "Company") today announced that the Michael E. DeBakey Veterans Affairs Medical Center, in Houston, Texas, has initiated a Phase 2A trial assessing the safety and efficacy of repeated doses of the Company's ghrelin agonist, AEZS-130 (macimorelin), in patients with cancer cachexia. The study is conducted under a Cooperative Research and Development Agreement (CRADA) between Aeterna Zentaris and the Michael E. DeBakey Veterans Affairs Medical Center which is funding the study. Cachexia, characterized by diminished appetite and food intake in cancer patients, is defined as an involuntary weight loss of at least 5% of the pre-illness body weight over the previous 6 months. Jose M. Garcia, MD, PhD, Assistant Professor, Division of Diabetes Endocrinology and Metabolism, Departments of Medicine, Molecular and Cell Biology, the Dan L. Duncan Cancer Center and the Huffington Center on Aging at Baylor College of Medicine and Michael E. DeBakey Veterans Affairs Medical Center, will act as the Principal Investigator of this trial.
Juergen Engel, PhD, President and CEO of Aeterna Zentaris stated, "Reaching agreement on this CRADA with the Michael E. DeBakey VA Medical Center was an important step which paved the way for the initiation of this trial. Furthermore, the study with AEZS-130 in cancer cachexia opens another important pathway for the development of this drug in the clinic."
Jose M. Garcia, MD, PhD, added, "I am pleased that we reached agreement on the conduct of this research cooperatively and that we can now initiate this study. The results generated by the proposed experiments will set the basis for future trials in this and other wasting conditions that also are very prevalent including chronic obstructive pulmonary disease (COPD), renal failure or chronic heart failure (CHF)-induced cachexia."
This is a double-blind, randomized, placebo-controlled Phase 2A trial to test the effects of different doses of the ghrelin agonist, AEZS-130, in 18 to 26 patients with cancer-cachexia. AEZS-130 will be provided by Aeterna Zentaris. The study will involve 3 sequential groups receiving differing doses of AEZS-130. Each dose group will have 6 patients who will receive AEZS-130 and 2-4 patients who will receive placebo. After analysis of safety and efficacy at each dose level vs. placebo, a decision will be taken either to decrease or increase the dose. If there are major safety issues, the study will be stopped. For this study, adequate efficacy will be defined as a ≥0.8 kg of body weight gain or a ≥50 ng/mL increase in plasma IGF-1 levels. The primary objective of the study is to evaluate the safety and efficacy of repeated oral administration of AEZS-130 at different doses daily for 1 week in view of developing a treatment for cachexia. The following parameters will be recorded to assess efficacy during the study: change of body weight, change of IGF-1 plasma levels, and change of quality of life score (Anderson Symptom Assessment Scale, FACIT-F). Other secondary objectives will include food intake, and changes in the following: appetite, muscle strength, energy expenditure, reward from food and functional brain connectivity.
About Cancer Cachexia
Over 1,500,000 new diagnoses of cancer are made in the U.S. every year. In most cases, this condition can have a significant impact on the individuals' survival and quality of life. Cachexia, a complex metabolic syndrome associated with underlying illness, is common in patients with cancer. These patients have diminished appetite, food intake and insulin sensitivity which contribute to a decrease in functional performance, takes a heavy toll on patients' quality of life and is associated with poor survival.
Despite the significant burden that cachexia and other cancer-related symptoms such as anorexia and poor functional status represent to cancer patients, these symptoms often remain undiagnosed and untreated. Moreover, there are currently no approved treatments for cancer-related cachexia and off-label treatments used for this condition such as appetite stimulants (e.g., megestrol acetate) are largely ineffective and are associated with potentially serious side effects (i.e., adrenal insufficiency, hypogonadism, deep venous thrombosis, etc.).
AEZS-130, a ghrelin agonist, is a novel orally-active small molecule that stimulates the secretion of growth hormone by binding the ghrelin receptor (GHSR-1a). It can be used in both oncology and endocrinology indications. In oncology, an IND has been granted for a Phase 2A trial with AEZS-130 in cancer induced cachexia, a disease which leads to significant weight loss and diminished functional performance. Since ghrelin agonists such as AEZS-130 have been shown to stimulate food intake and increase body weight in rats and mice, AEZS-130 could lead to better quality of life for patients with cancer-induced cachexia. Ghrelin agonists have been in clinical trials for over a decade and have demonstrated good safety and efficacy profiles.
In endocrinology, the Company has completed a Phase 3 trial for use as a simple oral diagnostic test for adult growth hormone deficiency (AGHD) and expects to file an NDA in 2012. AEZS-130 works by stimulating a patient's growth hormone secretion, which normally only occurs during sleep, after which a healthcare provider will measure how well the body responds to that stimulation based on the patient's growth hormone levels over a period of time. Low growth hormone levels, despite giving an effective stimulating agent, confirm a diagnosis of AGHD. AEZS-130 has been granted orphan-drug designation by the FDA for use as a diagnostic test for growth hormone deficiency. Aeterna Zentaris owns the worldwide rights to AEZS-130 which, if approved, would become the first orally-administered diagnostic test for AGHD.
About Aeterna Zentaris Inc.
Aeterna Zentaris is a late-stage oncology drug development company currently investigating potential treatments for various cancers including colorectal, multiple myeloma, endometrial, ovarian, prostate and bladder cancer. The Company's innovative approach of "personalized medicine" means tailoring treatments to a patient's specific condition and to unmet medical needs. Aeterna Zentaris' deep pipeline is drawn from its proprietary discovery unit providing the Company with constant and long-term access to state-of-the-art therapeutic options. 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. 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.
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