Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZS) (“Aeterna” or the
“Company”), a specialty biopharmaceutical company commercializing
and developing therapeutics and diagnostic tests, today announced
positive results for the first pediatric study of macimorelin as a
growth hormone stimulation test for the evaluation of child-onset
growth hormone deficiency (“CGHD”).
This study, AEZS-130-P01 (“Study P01”), was the
first of two studies as agreed with the European Medicines Agency
(“EMA”) in the Company’s Pediatric Investigation Plan (“PIP”) for
macimorelin. The goal of Study P01 was to establish a dose that
could 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 completion of Study P01 is a noteworthy
accomplishment in our pediatric development program for
macimorelin. We are pleased with the final results of Study P01
which demonstrated positive safety and tolerability data for use of
macimorelin in child-onset growth hormone deficiency. Furthermore,
the PK/PD data observed are in the range as expected from the adult
studies, which bolsters our confidence in the potential of
macimorelin to address the significant market expansion
opportunity. With the results from Study P01 in hand, we have the
necessary data as a promising basis for test validation Study P02,”
said Dr. Klaus Paulini, Chief Executive Officer of Aeterna
Zentaris.
Study P01 was an international, multicenter
study conducted in Hungary, Poland, Ukraine, Serbia, Belarus and
Russia. In three cohorts comprising 8 subjects each, macimorelin
doses of 0.25, 0.5 and 1.0 mg/kg body weight were investigated. In
accordance with the study protocol, all enrolled patients completed
four study visits after successful completion of the screening
period. At Visit 1 and Visit 3, a provocative GH stimulation test
was conducted according to the study sites’ local practices. At
Visit 2, the macimorelin test was performed: following the oral
administration of the macimorelin solution, blood samples were
taken at predefined times for PK/PD assessment. Visit 4 was a
safety follow-up visit at study end.
The completed study included 24 subjects aged 4
to 15 years. In the subjects who completed the study in accordance
with the protocol, macimorelin demonstrated an excellent safety and
tolerability profile. There were 88 adverse events (“AE”) reported
in 23 subjects, none of which was assessed by the investigator
as related to macimorelin. The majority of AEs (approximately 70%)
were expected side effects related to the hypoglycemia introduced
by the Insulin Tolerance Test. No significant changes in ECG
parameters and safety laboratory values were noted in any of the
three dosing cohorts.
The pharmacokinetic and pharmacodynamic profile
of macimorelin proved to be in the expected range and in general
comparable to data in adults.
“In Cohort 3, all eight subjects presented a
prominent exposure to macimorelin in their PK data as well as
growth hormone (GH) values above a threshold of 10ng/mL, which is
the upper cut-off point in children for the pediatric GHSTs,”
commented, Dr. Nicola Ammer, Chief Medical Officer of Aeterna
Zentaris. “Based on these data, and in line with faster metabolism
in children, a macimorelin dose of 1.0 mg/kg was identified as the
dose to be evaluated in the second study AEZS-130-P02 (“Study P02”)
on diagnostic efficacy and safety of macimorelin as GHD diagnostic
in children.”
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 commercializing and developing
therapeutics and diagnostic tests. The Company’s lead product,
Macrilen™ (macimorelin), is the first and only U.S. FDA and
European Commission approved oral test indicated for the diagnosis
of adult growth hormone deficiency (AGHD). Macrilen™ is currently
marketed in the United States through a license agreement with Novo
Nordisk and Aeterna Zentaris receives double-digit royalties on
sales. Aeterna Zentaris owns all rights to macimorelin outside of
the U.S. and Canada.
Aeterna Zentaris is also leveraging the clinical
success and compelling safety profile of macimorelin to develop it
for the diagnosis of child-onset growth hormone deficiency (CGHD),
an area of significant unmet need.
The Company is actively pursuing business
development opportunities for the commercialization of macimorelin
in Europe and the rest of the world, in addition to other
non-strategic assets to monetize their value. 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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