MacrilenTreatment for Adult Human Growth Hormone Deficiency
Aeterna Zentaris Completes Patient Recruitment for Confirmatory Phase 3 Trial of Macrilen
CHARLESTON, S.C.--(BUSINESS WIRE) October 26, 2016 --Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZ) (the “Company”) today announced it has successfully completed patient recruitment for the confirmatory Phase 3 clinical trial of Macrilen (macimorelin) as a growth hormone stimulation test for the evaluation of growth hormone deficiency in adults (“AGHD”). The Company also confirmed that it expects to file a New Drug Application for Macrilen with the United States Food and Drug Administration (the “FDA”) during the first half of 2017, if the results of the trial warrant doing so. Macrilen is the Company’s proposed tradename for macimorelin. The proposed tradename is subject to approval by the FDA.
Dr. Richard Sachse, the Company’s Chief Scientific Officer, stated, “We are committed 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 suspected of having AGHD are now very often evaluated by means of the insulin tolerance test (“ITT”). While the ITT is the historical gold-standard for the evaluation of AGHD, the procedure is inconvenient for patients and physicians and contraindicated 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. In addition, it is not an FDA-approved procedure. Furthermore, administration of the ITT is expensive because the patient must be constantly monitored by a physician for the 2- to 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, if necessary. With the completion of recruitment for our confirmatory Phase 3 clinical trial of Macrilen™ for the evaluation of AGHD, we are another step closer to being able to provide this important product to clinicians and patients.”
The Company believes that, in the US alone, approximately 40,000 confirmatory tests for AGHD will be conducted each year after the introduction of Macrilen™, if it is approved by the FDA, which represents the target market for Macrilen™ at the time of its anticipated commercialization. Furthermore, 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 and thus avoids the symptoms and potential complications of hypoglycemia;
- Macrilen™ is administered orally, while the ITT requires an intravenous infusion of insulin; the evaluation of AGHD using Macrilen™ is significantly 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.
As a result, the Company believes that Macrilen™, if it is approved, may be used for 40% to 50% of AGHD evaluations in the U.S. during the first year after its introduction and that the percentage could grow to as high as 85% within the first three years of commercialization.
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), was designed as a two-way crossover study with the ITT as the benchmark comparator and involves some 30 sites in the United States and Europe. The study population consists of more than 110 subjects (at least 55 ITT-positive and 55 ITT-negative) with a medical history documenting risk factors for AGHD, and includes 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 objective 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 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.
About Macrilen (macimorelin)
Macimorelin, a ghrelin agonist, is an orally-active small molecule that stimulates the secretion of growth hormone. Macimorelin has been granted orphan drug designation by the FDA for diagnosis of AGHD. The Company owns the worldwide rights to this patented compound and has significant patent protection left. The Company’s U.S. composition of matter patent expires in 2022 and its U.S. utility patent runs through 2027. The Company proposes, subject to FDA approval, to market macimorelin under the tradename Macrilen™.
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 as well as an increase of cardiovascular risks.
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 rejection or non-acceptance of any new drug application by one or more regulatory authorities and, more generally, uncertainties related to the regulatory process, the ability of the Company to efficiently commercialize one or more of its products or product candidates, the degree of market acceptance once our products are approved for commercialization, the ability of the Company to take advantage of business opportunities in the pharmaceutical industry, 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, except if required to do so.
Source: Aeterna Zentaris Inc.
Posted: October 2016
- Aeterna Zentaris Announces Plans to Pursue FDA Registration of Macrilen - February 13, 2017
- Aeterna Zentaris Announces Plans to Initiate Phase 3 Study of Macrilen in AGHD - April 13, 2015
- FDA Issues Complete Response Letter for Macrilen (macimorelin) NDA in Adult Growth Hormone Deficiency - November 6, 2014
- Aeterna Zentaris NDA for Macimorelin Acetate in AGHD Accepted for Filing by the FDA - January 6, 2014
- Aeterna Zentaris Submits New Drug Application to FDA for Macimorelin Acetate (AEZS-130) for Evaluation of AGHD - November 5, 2013