Aeterna Zentaris Inc. announced completion of its Phase 3 study with AEZS-130 as the first oral diagnostic test for Adult Growth Hormone Deficiency (AGHD). The Company is currently proceeding with 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.
“We wish to thank all those involved in this project for their utmost dedication. Preliminary results are encouraging and we therefore expect to submit a NDA to the FDA. 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.
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 Geref Diagnostic was subsequently withdrawn from the market, worldwide, in 2008; the trial’s sponsor, Ardana Biosciences (Ardana), discontinued the study for financial reasons before it was completed.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.
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 enrolled to match the AGHD patients from the original cohort;
• Further, an additional 20 subjects were 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.
Date: August 3, 2011
Source: Aeterna Zentaris Inc.
Filed Under: Drug Discovery