Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZS) (“Aeterna” or the
“Company”), a specialty biopharmaceutical company engaged in
commercializing novel pharmaceutical therapies, today announced the
successful completion of patient recruitment for the first
pediatric study of macimorelin as a growth hormone stimulation test
for the evaluation of growth hormone deficiency (“GHD”) in
children. This study, AEZS-130-P01 (“Study P01”), is the first of
two studies as agreed with the European Medicines Agency (“EMA”) in
the Company’s Pediatric Investigation Plan (“PIP”) for macimorelin.
Macimorelin, a ghrelin agonist, is an orally active small
molecule that stimulates the secretion of growth hormone from the
pituitary gland into the circulatory system.
The goal of Study P01 is to establish a dose
that can both be safely administered to pediatric patients and
cause a clear rise in growth hormone concentration in subjects
ultimately diagnosed as not having GHD. The recommended dose
derived from Study P01 will be evaluated in the pivotal second
study AEZS-130-P02 on diagnostic efficacy and safety. Study P01 is
an international, multicenter study which is being conducted in
Hungary, Poland, Ukraine, Serbia, and Russia.
Dr. Nicola Ammer, Chief Medical Officer of
Aeterna, commented, “We are pleased to have completed patient
recruitment in this important study for the pediatric development
of macimorelin. In all dosing cohorts of Study P01, macimorelin
administration was completed safely at the end of December
2019.”
Study P01 is an open label, group comparison,
dose escalation trial designed to investigate the safety,
tolerability, and pharmacokinetic/pharmacodynamic (“PK/PD”) of
macimorelin acetate after ascending single oral doses of
macimorelin at 0.25, 0.5, and 1.0 mg per kg body weight in
pediatric patients from 2 to less than 18 years of age with
suspected GHD. The Company enrolled a total of 24 pediatric
patients across the three cohorts of the study.
“The completion of patient recruitment in Study
P01 represents a significant milestone and an important step in the
pediatric development of macimorelin in this important patient
population. We are looking forward to the successful completion of
Study P01 and expect to report final results in the second quarter
of 2020,” said Dr. Klaus Paulini, Chief Executive Officer of
Aeterna.
Per study protocol, all enrolled patients will
complete four study visits after successful completion of the
screening period. At Visit 1 and Visit 3, a provocative GH
stimulation test will be conducted according to the study sites’
local practices. At Visit 2, the macimorelin test will be
performed: following the oral administration of the macimorelin
solution, blood samples will be taken at predefined times for PK/PD
assessment. Visit 4 is a safety follow-up visit at study end.
For more information about Study P01, please
visit EU Clinical Trials Register and reference EudraCT
#2018-001988-23.
About Macimorelin
Macimorelin, a ghrelin agonist, is an
orally-active small molecule that stimulates the secretion of
growth hormone from the pituitary gland. Stimulated growth hormone
levels are measured in blood samples after oral administration of
Macimorelin for the assessment of GHD.
In December 2017, the United States Food and
Drug Administration (“FDA”) granted Aeterna Zentaris marketing
approval for macimorelin to be used in the diagnosis of patients
with adult growth hormone deficiency (“AGHD”). Macrilen™ has been
granted Orphan Drug designation by the FDA for diagnosis of AGHD.
In January 2019, the European Commission granted marketing
authorization for macimorelin to Aeterna Zentaris for diagnosis of
growth hormone deficiency in adults. In March 2017, the Pediatric
Committee of the EMA agreed to the Company’s PIP for macimorelin, a
prerequisite for filing a marketing authorization application for
any new medicinal product in Europe.
About Aeterna Zentaris Inc.
Aeterna Zentaris Inc. is a specialty
biopharmaceutical company engaged in commercializing novel
pharmaceutical therapies, principally through out-licensing
arrangements. Aeterna Zentaris is the licensor and party to a
license and assignment agreement with Novo Nordisk A/S to carry out
joint development, manufacturing, registration, regulatory, and
supply chain for the commercialization of Macrilen™ (macimorelin),
which is to be used in the diagnosis of patients with adult growth
hormone deficiency in the United States and Canada. The clinical
development of Macrilen™ (macimorelin) for pediatric use is
ongoing. In addition, we are actively pursuing business development
opportunities for macimorelin in the rest of the world and to
monetize the value of our non-strategic assets. For more
information, please visit the Company’s website at
www.zentaris.com.
Forward Looking Statements
This press release contains certain
forward-looking statements within the meaning of Section 27A of the
Securities Act of 1933 and Section 21E of the Securities Exchange
Act of 1934 and Private Securities Litigation Reform Act, as
amended, including those relating to the Company's product
development, clinical and regulatory timelines, market opportunity,
competitive position, possible or assumed future results of
operations, business strategies, potential growth opportunities and
other statement that are predictive in nature. These
forward-looking statements are based on current expectations,
estimates, forecasts and projections about the industry and markets
in which we operate and management's current beliefs and
assumptions.
These statements may be identified by the use of
forward-looking expressions, including, but not limited to,
"expect," "anticipate," "intend," "plan," "believe," "estimate,"
"potential, "predict," "project," "should," "would" and similar
expressions and the negatives of those terms. These statements
relate to future events or our financial performance and involve
known and unknown risks, uncertainties, and other factors which may
cause actual results, performance or achievements to be materially
different from any future results, performance or achievements
expressed or implied by the forward-looking statements. Such
factors include those set forth in the Company's filings with the
Securities and Exchange Commission. Prospective investors are
cautioned not to place undue reliance on such forward-looking
statements, which speak only as of the date of this press release.
The Company undertakes no obligation to publicly update any
forward-looking statement, whether as a result of new information,
future events or otherwise.
Investor Contact:
Jenene Thomas JTC Team T (US): +1 (833) 475-8247 E:
aezs@jtcir.com
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