Aeterna Zentaris Concludes Successful Meeting of Clinical Investigators for Confirmatory Phase 3 Trial of Macrilen(TM)
Frankfurt, Germany / CRWEPRESSRELEASE / January 19, 2016 – Aeterna Zentaris Inc. (NASDAQ: AEZS; TSX: AEZ) (the “Company”) announced today that it concluded a successful meeting of the clinical investigators for the confirmatory Phase 3 trial of Macrilen™ (macimorelin), a novel orally-active ghrelin agonist for use in evaluating adult growth hormone deficiency (“AGHD”). As a result, the Company is confident that it will complete the confirmatory trial by year-end 2016.
Dr. Richard Sachse, the Company's Chief Scientific Officer stated, “On January 16, we held an Investigators' Meeting for our multi-center confirmatory Phase 3 clinical trial of Macrilen™. The purpose of the meeting was to review Macrilen™ with the investigators, as well as the clinical trial protocol. The 77 investigators and site personnel who attended the meeting included Jose Manuel Garcia, MD, PhD, Associate Professor of Medicine at University of Washington School of Medicine, Seattle, who is serving as coordinating investigator. Dr. Garcia expressed his commitment to the development of Macrilen™ because of the medical need for such a convenient test in the absence of an FDA-approved diagnostic test for AGHD.”
Patients who are believed to have AGHD are now very often evaluated by means of the insulin tolerance test (“ITT”). The ITT is the historical gold standard for the evaluation of AGHD because of its high sensitivity and specificity. However, the ITT is inconvenient to the patients and physicians and contra-indicated in certain patients, such as patients with coronary heart disease or seizure disorder, because it requires the patient to experience hypoglycemia to obtain a result. Some physicians will not induce full hypoglycemia, intentionally compromising accuracy to increase safety and comfort for the patient. Furthermore, administration of the ITT is expensive because the patient must be constantly monitored by a physician for the 2-4 hour duration of the test and the test must be administered in a setting where emergency equipment is available and where the patient may be quickly hospitalized. The ITT is not used for patients with co-morbidities, such as CV, seizure disorder or a history of brain cancer or for patients who are elderly and frail, due to safety concerns.
The Company believes that Macrilen™, if it is approved, is likely to be rapidly adopted by physicians as the preferred means of evaluating AGHD for the following reasons:
- it is safer than the ITT because it does not require the patient to become hypoglycemic;
- Macrilen™ is administered orally, while the ITT requires an intravenous infusion of insulin;
- the evaluation of AGHD using Macrilen™ is much less time consuming and labor intensive than the ITT and, therefore, it is less expensive to conduct; and
- the evaluation can be conducted in the physician's office rather than in a hospital setting.
About the Study
The confirmatory Phase 3 clinical study of Macrilen™, entitled Confirmatory validation of oral macimorelin as a growth hormone (GH) stimulation test (ST) for the diagnosis of adult growth hormone deficiency (AGHD) in comparison with the insulin tolerance test (ITT), is designed as a two-way crossover study with the insulin tolerance test as the benchmark comparator and will involve some 30 sites in the United States and Europe. The study population will consist of approximately 110 subjects (at least 55 ITT-positive and 55 ITT-negative) with a medical history documenting risk factors for AGHD, and will include a spectrum of subjects from those with a low risk of having AGHD to those with a high risk of having the condition. The primary endpoint is validation of a single oral dose of macimorelin for the diagnosis of AGHD, using the ITT as a comparator. Based on meetings with the US Food and Drug Administration (“FDA”) as well as the European Medicines Agency (“EMA”) and subsequent written scientific advice, the Company believes that the study meets the FDA's and the EMA's study-design expectations allowing US and European approval, if successful. As of the date of this release, five patients at three separate centers have been enrolled in the study. For more details on the trial, please consult this link: https://www.clinicaltrial.gov/ct2/show/NCT02558829?term=macimorelin&rank=1.
Based on the current rate of enrollment, the Company expects the confirmatory Phase 3 clinical study of Macrilen™ to be concluded by the end of 2016. The Company further expects to be able to submit a New Drug Application for Macrilen™ to the FDA by mid-year 2017 and, if the study is successful in meeting its primary endpoint, to obtain approval of the drug by year-end 2017.
About Macrilen™ (macimorelin)
Macimorelin, a ghrelin agonist, is a novel orally-active small molecule that stimulates the secretion of growth hormone. Macimorelin, under the trade name Macrilen™, has been granted orphan drug designation by the FDA for diagnosis of AGHD. The Company owns the worldwide rights to this novel patented compound.
AGHD affects approximately 75,000 adults across the US, Canada and Europe. Growth hormone not only plays an important role in growth from childhood to adulthood, but also helps promote a hormonally-balanced health status. AGHD mostly results from damage to the pituitary gland. It is usually characterized by a reduction in bone mineral density, lean body mass, exercise capacity, and overall quality of life.
About Aeterna Zentaris Inc.
Aeterna Zentaris is a specialty biopharmaceutical company engaged in developing and commercializing novel treatments in oncology, endocrinology and women's health. We are engaged in drug development activities and in the promotion of products for others. We are now conducting Phase 3 studies of two internally developed compounds. The focus of our business development efforts is the acquisition of licenses to products that are relevant to our therapeutic areas of focus. We also intend to license out certain commercial rights of internally developed products to licensees in territories where such out-licensing would enable us to ensure development, registration and launch of our product candidates. Our goal is to become a growth-oriented specialty biopharmaceutical company by pursuing successful development and commercialization of our product portfolio, achieving successful commercial presence and growth, while consistently delivering value to our shareholders, employees and the medical providers and patients who will benefit from our products. For more information, visit www.aezsinc.com.
This press release contains forward-looking statements made pursuant to the safe harbor provisions of the US Securities Litigation Reform Act of 1995. Forward-looking statements may include, but are not limited to statements preceded by, followed by, or that include the words “expects,” “believes,” “intends,” “anticipates,” and similar terms that relate to future events, performance, or our results. 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 and clinical trials, 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 efficiently commercialize one or more of its products or product candidates, the ability of the Company to take advantage of business opportunities in the pharmaceutical industry, uncertainties related to the regulatory process, the ability to protect our intellectual property, the potential of liability arising from shareholder lawsuits and general changes in economic conditions. Investors should consult the Company's quarterly and annual filings with the Canadian and US securities commissions for additional information on risks and uncertainties relating to forward-looking statements. Investors are cautioned not to place undue reliance 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.
Philip A. Theodore
Senior Vice President
Source: Aeterna Zentaris