Company will file New Drug Application
QUBEC CITY, Aug. 30, 2011 /PRNewswire/ - Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZ) (the "Company") today announced favorable top-line results of its completed Phase 3 study with AEZS-130 as the first oral diagnostic test for Adult Growth Hormone Deficiency (AGHD). The results show that AEZS-130 reached its primary endpoint demonstrating >90% area-under-the-curve (AUC) of the Receiver Operating Characteristic (ROC) curve, which determines the level of specificity and sensitivity of the product. The Company is currently proceeding with further detailed analyses of the data and preparing for a pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA) in the upcoming months, which would be followed by the filing of a NDA for the registration of AEZS-130 in the United States.
The parameters of the study, as defined below under Study Design, were achieved as agreed to with FDA under our Special Protocol Assessment (SPA). Importantly, the primary efficacy parameters show that the study achieved both specificity and sensitivity at a level of 90% or greater. In addition, 8 of the 10 newly enrolled AGHD patients were correctly classified by a pre-specified peak GH threshold level. The use of AEZS-130 was shown to be safe and well tolerated overall throughout the completion of this trial.
"We are pleased with the results obtained and we therefore expect to meet with the FDA and work out the content of a submission for an NDA. We believe that AEZS-130 could become the first approved oral test for the diagnosis of AGHD, providing patients with a potentially safer, accurate and more convenient alternative to the current injectable tests", stated Juergen Engel, Ph.D., President and CEO at Aeterna Zentaris.
Study History
The study titled, "A Multi-Center Study Investigating a New, Oral Growth Hormone Secretagogue (AEZS-130, formerly ARD-07) as a Growth Hormone (GH) Stimulation Test in Terms of Safety and Efficacy", was originally initiated to compare the performance of AEZS-130 against the then-available diagnostic growth hormone-releasing hormone (GHRH) Geref Diagnostic® + Arginine (ARG) standard test. Geref Diagnostic® was subsequently withdrawn from the market, worldwide, in 2008; the trial's sponsor, Ardana Biosciences (Ardana), discontinued the study for financial reasons before it was completed. In 2009, Aeterna Zentaris entered into an agreement with administrators of Ardana and regained the rights to AEZS-130, and with the FDA, established the best way forward to complete this Phase 3 study and continue to utilize the data already obtained, in light of the loss of the original comparator. A Special Protocol Assessment (SPA) granted by the FDA, resulted in a modification of the original study, without altering the basic study design so that the completed portion of the study and the new part of the study would provide one, complete, Phase 3 study.
Study Design
The first part of the study conducted by Ardana was a two-way crossover study involving 42 patients with confirmed AGHD or multiple pituitary hormone deficiencies and a low insulin-like growth factor-I. A control group of 10 subjects without AGHD were matched to patients for age, gender, body mass index and (for females) estrogen status.
Each patient received two dosing regimens in random order, while fasting, at least 1 week apart. One regimen consisted of a 1 µg/kg (max. 100 µg) dose of GHRH (Geref Diagnostic®, Serono) with 30 g of ARG (Ar-Gine®, Pfizer) administered intravenously over 30 minutes; the other regimen was a dose of 0.5 mg/kg body weight of AEZS-130 given in an oral solution of 0.5 mg/ml.
As a result of the SPA reached with the FDA in order to complete the trial, the second part of the study contained the following revisions/additions to the first protocol:
- An additional 30 normal control subjects were to be enrolled to match the AGHD patients from the original cohort;
- Further, an additional 20 subjects were to be enrolled: 10 AGHD patients and 10 matched normal control subjects;
- The above brought the database to ~100 subjects;
- All subjects received a dose of 0.5 mg/kg body weight of AEZS-130;
- As a secondary endpoint, the protocol required that at least 8 of the 10 newly enrolled AGHD patients be correctly classified by a pre-specified peak GH threshold level.
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 a simple oral diagnostic test for adult growth hormone deficiency (AGHD). 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 AGHD
AGHD affects 35,000 adult Americans, with 6,000 new adult patients diagnosed each year. Growth hormone not only plays an important role in growth from childhood to adulthood, but helps promote good health throughout life. AGHD is usually characterized by low energy levels, decreased strength and exercise tolerance, increased weight or difficulty losing weight, emotional changes, anxiety and impaired sleep. Available diagnostic tests for AGHD are complex and can produce significant side effects.
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.
Forward-Looking Statements
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 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.