Expects to File NDA in H1 of 2017, if Warranted by Trial Results
CHARLESTON, SC, USA I October 26, 2016 I 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. For more details on the trial, please consult this link:
https://www.clinicaltrials.gov/ct2/show/NCT02558829?term=macimorelin&rank=1.
About MacrilenTM (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™.
About AGHD
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.
SOURCE: Aeterna Zentaris
Post Views: 97
Expects to File NDA in H1 of 2017, if Warranted by Trial Results
CHARLESTON, SC, USA I October 26, 2016 I 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. For more details on the trial, please consult this link:
https://www.clinicaltrials.gov/ct2/show/NCT02558829?term=macimorelin&rank=1.
About MacrilenTM (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™.
About AGHD
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.
SOURCE: Aeterna Zentaris
Post Views: 97
