Aeterna Zentaris Commences Pivotal Phase 3 DETECT-Trial for the Diagnosis of Childhood-Onset Growth Hormone Deficiency
May 13 2021 - 8:05AM
Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZS) (“Aeterna” or the
“Company”), a specialty biopharmaceutical company developing and
commercializing a diversified portfolio of pharmaceutical and
diagnostic products, today announced the commencement of its
pivotal Phase 3 safety and efficacy study AEZS-130-P02 (“the
DETECT-trial”) evaluating macimorelin for the diagnosis of
childhood-onset growth hormone deficiency (“CGHD”).
The investigational new drug application
(“IND”), “Multicenter, open-label trial to investigate the efficacy
and safety of a single oral dose of 1.0 mg/kg macimorelin acetate
as growth hormone stimulation test (“GHST”) in pediatric patients
with suspected growth hormone deficiency (“GHD”),” for the study is
active and the first clinical site in the U.S. is open for patient
recruitment.
Children and adolescents from two to less than
18 years of age with suspected growth hormone deficiency are
planned to be included. The study is expected to enroll
approximately 100 participants worldwide, with at least 40
participants in pre-pubertal and 40 participants in pubertal
status. A macimorelin GHST will be performed twice to ensure the
repeatability of the data. Two standard GHSTs will be used as
controls: arginine (i.v.) and clonidine (p.o.). The study design is
expected to be suitable to support a claim for potential
stand-alone testing with macimorelin, if successful.
“The initiation of the pivotal Phase 3
DETECT-trial is a key milestone for the Company,” commented Dr.
Klaus Paulini, Chief Executive Officer of Aeterna. “While we
continue to make progress on multiple fronts across our development
pipeline, the launch of this pivotal trial for the diagnosis of
CGHD remains an integral piece of our product portfolio and an area
of key focus. As sponsors of the trial, we look forward to getting
the remainder of the clinical sites active and patient enrollment
underway as quickly and efficiently as possible.”
The Company announced positive results in April
2020 from AEZS-130-P01 ("Study P01"), the first of two studies as
agreed with FDA and EMA. The goal of Study P01 was to establish a
dose that could both be safely administered to pediatric
participants and causes a clear rise in growth hormone
concentration in participants ultimately diagnosed as not having
GHD. Results from the study demonstrated that the pharmacokinetic
and pharmacodynamic profile of macimorelin proved to be in the
expected range and in general comparable to data in adults.
Under new terms and conditions of the license
agreement revised in November 2020, Aeterna is closely coordinating
the activities related to the development of macimorelin in CGHD
through a joint steering committee with Novo Nordisk, Aeterna’s
licensee for the U.S. and Canada.
For more information about Study P01 and the
DETECT-trial, please visit EU Clinical Trials Register and
reference EudraCT #2018-001988-23 and clinicaltrials.gov identifier
NCT04786873.
About Aeterna Zentaris Inc.
Aeterna Zentaris is a specialty
biopharmaceutical company developing and commercializing a
diversified portfolio of pharmaceutical and diagnostic products
focused on areas of significant unmet medical need. The Company’s
lead product, macimorelin (Macrilen™), is the first and only U.S.
FDA and European Commission approved oral test indicated for the
diagnosis of adult growth hormone deficiency (AGHD). The Company is
leveraging the clinical success and compelling safety profile of
macimorelin to develop it for the diagnosis of childhood-onset
growth hormone deficiency (CGHD) in collaboration with Novo
Nordisk.
Aeterna Zentaris is dedicated to the development
of therapeutic assets and has recently taken steps to establish a
growing preclinical pipeline to potentially address unmet medical
needs across a number of indications, including neuromyelitis
optica spectrum disorder (NMOSD), hypoparathyroidism and an
undisclosed neurodegenerative disease. Additionally, the Company is
developing an oral prophylactic bacterial vaccine against
SARS-CoV-2, the virus that causes COVID-19.
For more information, please visit
www.zentaris.com and connect with the Company on Twitter, LinkedIn
and Facebook.
Forward-Looking Statements
This press release contains statements that may
constitute forward-looking statements within the meaning of U.S.
and Canadian securities legislation and regulations, and such
statements are made pursuant to the safe-harbor provision of the
U.S. Securities Litigation Reform Act of 1995. Forward-looking
statements are frequently, but not always, identified by words such
as “expects,” “anticipates,” “believes,” “intends,” “potential,”
“possible,” and similar expressions. Such statements, based as they
are on current expectations of management, inherently involve
numerous risks, uncertainties, and assumptions, known and unknown,
many of which are beyond our control. Forward-looking statements in
this press release include, but are not limited to, those relating
to: Aeterna’s expectation with respect to Study P02 (including the
ability to initiate the remainder of the clinical sites, to enroll
subjects in the USA or elsewhere in Study P02, and expectations
that Study P02 are suitable to support a claim (regulatory
approval) for potential stand-alone testing with macimorelin), and
Aeterna’s intentions with respect to growth opportunities and its
business focus, including its development pipeline.
Forward-looking statements involve known and
unknown risks and uncertainties, and other factors which may cause
the actual results, performance or achievements stated herein to be
materially different from any future results, performance or
achievements expressed or implied by the forward-looking
information. Such risks and uncertainties include, among others,
our reliance on the success of the pediatric clinical trial in the
European Union and U.S. for Macrilen™ (macimorelin); we may be
unable to enroll the expected number of subjects in Study P02 and
the result of Study P02 may not support receipt of regulatory
approval in CGHD, we may be delayed or unsuccessful in obtaining
pricing and reimbursement approvals in Europe and the UK to market
macimorelin; our other products under development may not be
successful or may not support advancing the product to human
clinical trials; our ability to raise capital and obtain financing
to continue our currently planned operations; our now heavy
dependence on the success of Macrilen™ (macimorelin) and related
out-licensing arrangements and the continued availability of funds
and resources to successfully commercialize the product, including
our heavy reliance on the success of the License Agreement with
Novo Nordisk; the global instability due to the global pandemic of
COVID-19, and its unknown potential effect on our planned
operations; our ability to enter into out-licensing, development,
manufacturing, marketing and distribution agreements with other
pharmaceutical companies and keep such agreements in effect.
Investors should consult our quarterly and annual filings with the
Canadian and U.S. securities commissions for additional information
on risks and uncertainties, including those risks discussed in our
Annual Report on Form 40-F and annual information form, under the
caption "Risk Factors". Given the uncertainties and risk factors,
readers are cautioned not to place undue reliance on these
forward-looking statements. We disclaim any obligation to update
any such factors or to publicly announce 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 applicable law.
No securities regulatory authority has either
approved or disapproved of the contents of this news release. The
Toronto Stock Exchange accepts no responsibility for the adequacy
or accuracy of this release.
Investor Contact:
Jenene ThomasJTC TeamT (US): +1 (833) 475-8247E:
aezs@jtcir.com
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