Ambrilia Reports Positive Phase III Preliminary Results Showing the Safety and Efficacy of its Prolonged-Release Formulation of OctreotideÂ
Posted : Tue, 22 Jan 2008
MONTREAL, QUEBEC -- 01/22/08 -- Ambrilia Biopharma Inc. (TSX: AMB) reported preliminary Phase III results today for its proprietary prolonged-release formulation of Octreotide ("C2L") in acromegaly patients. The primary objective of this study comparing C2L to SandostatinÂ®LAR was met with both C2L and SandostatinÂ® LAR, inducing a highly statistically significant decrease of Insulin-like Growth Factor 1 ("IGF-1") (p less than0.005 for both arms) and Growth Hormone ("GH") (p less than0.0001 for both arms) plasma levels. It was not possible to demonstrate non-inferiority, nor inferiority, of C2L over SandostatinÂ®LAR. The Company believes that it would have been difficult to reach this endpoint due to the small patient population and large initial differences in both IGF-1 and GH values. There were no dropouts during the study. No differences were observed between the two products in adverse events, which were mild and transient
"These preliminary data confirm that C2L is very effective at reducing GH, IGF-1 and clinical symptoms in acromegaly patients, with minimal side effects", said Professor Stafford Lightman, Professor of Medicine, University of Bristol and Henry-Wellcome Center for Integrative Neuroscience & Endocrinology (LINE), U.K., and principal investigator of the study.
Dr. Philippe Calais, President and Chief Executive Officer of Ambrilia, said "I am encouraged by the preliminary results of this study. Accordingly, Ambrilia continues to pursue its planned clinical development program for C2L and as previously announced, expects regulatory filings by its licensing partners to start during the second half of 2008".
TRIAL DESIGN AND RESULTS
This open label, multicenter, randomized, non-inferiority study compared the safety and efficacy of C2L to SandostatinÂ®LAR in acromegalic patients. Sixty-five (65) patients with active acromegaly were randomized to receive either C2L 30 mg every six weeks (34 patients), or SandostatinÂ®LAR 30 mg ("Sandostatin") every four weeks (31 patients) for a period of 12 weeks (84 days). All patients completed the study. The primary objectives of the study were the reduction in IGF-1 and GH plasma levels, from baseline levels to the level on day 84.
C2L and Sandostatin both induced a statistically significant decrease of IGF-1 (p less than0.005 for both arms) and GH plasma levels (p less than0.0001 for both arms), at all time points throughout the study and at day 84. The mean percentage of GH decrease was similar in both groups throughout the study. IGF-1 response was also similar, though more pronounced in the Sandostatin group on day 84. The European authorities require statistical validation of IGF-1 levels whereas the U.S. Food and Drug Administration requires descriptive statistics of GH levels.
The clinical efficacy of both products as measured by changes in signs and symptoms of acromegaly, acromegaly symptom scores and patient health status was also assessed as part of the study's secondary objectives. Improvement was marked and similar in both groups for all criteria.
The adverse events reported were minor and not unexpected, and their incidence and severity were similar in both groups. No patient withdrew from the trial due to the occurrence of adverse events, or for any other reason, despite the expected higher plateau observed in the pharmacokinetic profile of C2L.
In light of these results, the Company moves forward with its planned clinical development of C2L. As some patients (14 of the 31 in the Sandostatin group, and 16 of the 34 in the C2L group), began to experience increasing GH plasma levels at the end of the treatment period, Ambrilia is evaluating the introduction of dose interval adjustments for those patients in the ongoing long term safety study designed as a continuation of the initial study, in which all 65 patients are currently receiving C2L. Furthermore, these dose interval adjustments will also be available for appropriate patients, in a separate study, which is currently being conducted in the U.S. and Europe. This study is designed to evaluate the safety and efficacy of 20 mg and 10 mg C2L doses in the same indication.
"It is quite common for physicians in charge of acromegaly patients to adjust the doses, or the intervals between doses, according to the severity of their condition and to the results obtained with treatment", added Professor Lightman.
ABOUT OCTREOTIDE, SANDOSTATINÂ®LAR AND ACROMEGALY
Ambrilia's product is a proprietary prolonged-release formulation of Octreotide. The original product is commercialized as SandostatinÂ®LAR (octreotide acetate for injectable suspension, a registered trademark of Novartis Pharmaceuticals Corporation). Octreotide is used for the treatment of a rare disease called acromegaly caused by a tumor of the pituitary gland, and for certain rare digestive tumors.
Acromegaly is a serious chronic condition related to a permanent hypersecretion of Growth Hormone (GH) by the pituitary gland, generally of tumoral origin. This causes an excessive production of Insulin-like Growth Factor 1 (IGF-1), a hormone secreted from the liver and other tissues. Excessive production of IGF-1 and GH results in uncontrolled growth of various organs, and debilitating symptoms. Control of the GH and IGF-1 levels by Octreotide normalizes such excessive growth and symptoms. Appropriate treatment has been shown to improve quality of life and more importantly, to increase life expectancy, in patients with acromegaly. It is a life long treatment, with few, mild side effects.
CONFERENCE CALL AND WEBCAST DETAILS
Ambrilia will be hosting a conference call and webcast today, January 22 at 9:00 am ET, to discuss the Phase III preliminary results for its prolonged-release formulation of Octreotide, C2L, in acromegaly patients.
Dr. Philippe Calais, President and Chief Executive Officer, and Dr. Bonabes de Rouge, Senior Vice-President and Chief Scientific Officer, will be joined by the principal investigator of the study, Professor Stafford Lightman, Professor of Medicine, University of Bristol and Henry-Wellcome Center for Integrative Neuroscience & Endocrinology (LINE), U.K.
Interested parties may access the conference call by way of telephone or webcast. The numbers to access the conference call are (416) 644-3415 (international) and 1(800) 733-7560 (toll free). The webcast will be available on the Company's website at www.ambrilia.com, Investors' section, Conference calls and webcasts, and will be archived for 365 days.
A replay of the call will be available on the Company's website at www.ambrilia.com, Investors' section, Conference calls and webcasts, from Tuesday, January 22, 11:00 am ET to Tuesday, January 29, 11:59 pm ET, and the numbers to access the replay are (416) 640-1917 (international) and 1(877) 289-8525 (toll free) with access code 21260411.
Ambrilia's forward-looking statements
This press release contains forward-looking statements that reflect the Company's current expectation regarding future events. These forward-looking statements involve risks and uncertainties. Actual events could differ materially from those projected herein and depend on a number of factors including, but not limited to, changing market conditions, successful and timely completion of clinical studies, uncertainties related to the regulatory approval process, establishment of corporate alliances and other risks detailed from time to time in the Company's filings. We refer you to the Risk Factors section of the Company's Management's Discussion & Analysis of Financial Condition and Results of Operations which contain a more exhaustive analysis of the risks and uncertainties that are generally connected to the business of the Company. Such statements are also based on various assumptions, including the successful and timely completion of clinical studies on Ambrilia's products demonstrating efficacy and safety for human use, their successful commercialization within the forecasted timelines and the attainment of the forecasted milestone payments and other revenues. While Ambrilia anticipates that subsequent events and developments may cause Ambrilia's views to change, Ambrilia specifically disclaims any obligation to update these forward looking statements, unless obligated to do so by applicable securities laws.
ABOUT AMBRILIA BIOPHARMA
Ambrilia Biopharma Inc. (TSX: AMB) is a biopharmaceutical company dedicated to the discovery and development of novel treatments for viral diseases and cancer. Ambrilia's product portfolio includes an HIV protease inhibitor program (with lead compound PPL-100), an HIV integrase inhibitor program, two new formulations of existing peptides (Octreotide and Goserelin), other tumor targeted peptides such as PCK3145 and the Tumor and tumor Vasculature Targeting (TVT) technology platform, as well as other anti-viral programs. Exclusive worldwide rights to PPL-100 and its related compounds have been granted to Merck & Co., Inc. in return for milestone payments and royalties. Ambrilia's head office, research and development and manufacturing facilities are located in Montreal with a regional office in France. For more information, please visit the Company's web site: www.ambrilia.com