Aeterna Zentaris Receives Agreement from FDA on Special Protocol Assessment for Solorel(R) (AEZS-130, Macimorelin) to Complete Registration Study for Diagnosis of Adult Growth Hormone Deficiency
TSX: AEZ) (the “Company”) today announced that it has reached agreement
with the Food and Drug Administration (FDA) on a Special Protocol
Assessment (SPA) for Solorel((R)) (AEZS-130, macimorelin) which will enable the Company to complete the
ongoing registration study required to gain approval as a diagnostic
test for Adult Growth Hormone Deficiency (AGHD).
“We are pleased with the agreement with the FDA and now look forward to
the completion of the Phase 3 trial with Solorel((R)) and the NDA filing in 2011 for use as a diagnostic test in AGHD”,
line with our innovative approach, Solorel((R) )could become the first approved oral test for the diagnosis of AGHD,
providing patients with a potentially safer, accurate and more
convenient alternative to the current injectable tests.”
The study was initiated to compare the performance of Solorel((R)) against the then-available diagnostic growth hormone releasing hormone
(GHRH) Geref Diagnostic((R) )+ Arginine (ARG) standard test. GHRH Geref Diagnostic((R)) was subsequently withdrawn from the market, worldwide, in 2008. At the
time of withdrawal, the trial included 42 patients with AGHD and ten
control subjects. In 2009, Aeterna Zentaris regained the rights to
Solorel((R)) and had been working with the FDA to establish the best way forward to
complete this registration study and continue to utilize the data
obtained thus far, in light of the loss of the original comparator.
The protocol is titled,“A Multi-Center Study Investigating a New, Oral Growth Hormone
Secretagogue (AEZS-130, formerly ARD-07) as a Growth Hormone (GH)
Stimulation Test in Terms of Safety and Efficacy.” (Clinicaltrials.gov Identifier: NCT00448747)
The SPA agreement has resulted in a modification of the original study,
but does not alter the basic study design so that the completed portion
of the study will work with the new part of the study to provide one,
complete, Phase 3 study.
The completed part of the study was a two-way crossover study, and
included 42 patients with confirmed AGHD or multiple pituitary hormone
deficiencies and a low insulin-like growth factor-I. A control group of
10 subjects without AGHD were matched to patients for age, gender, body
mass index and (for females) estrogen status.
Each patient received two dosing regimens in random order, while
fasting, at least 1 week apart. One regimen consisted of a 1 g/kg
(max. 100 g) dose of GHRH (Geref Diagnostic((R)), Serono) with 30 g of ARG (Ar-Gine((R)), Pfizer) administered intravenously over 30 minutes; the other regimen
was a dose of 0.5 mg/kg body weight of Solorel((R) )given in an oral solution of 0.5 mg/ml.
SPA Agreed Study Amendment
Completion of the study will be accomplished with the following
revisions/additions to the current protocol:
An additional 30 normal controls subjects will be enrolled to match the
AGHD patients from the original cohort;
Further, an additional 20 subjects will be enrolled – 10 AGHD patients
and 10 matched normal control subjects;
- The above will bring the database to approximately 100 patients;
All subjects will be receiving a dose of 0.5 mg/kg body weight of
As a secondary endpoint, the protocol will require that at least 8 of
the 10 newly enrolled AGHD patients be correctly classified by a
pre-specified peak Growth Hormone (GH) threshold level.
Solorel((R)), a ghrelin agonist, is a novel orally active small molecule that
stimulates the secretion of growth hormone (GH).The orally-administered
product is currently in a Phase 3 trial for use as a simple diagnostic
test for adult growth hormone deficiency (AGHD). Solorel((R)) works by stimulating a patient’s GH secretion after which a healthcare
provider will measure how well the body responds to that stimulation
based on the patient’s GH levels over a period of time. Low GH levels,
despite giving an effective stimulating agent, confirm a diagnosis of
AGHD. Solorel((R)) has been granted orphan-drug designation by the FDA for use as a
diagnostic test for growth hormone deficiency. Aeterna Zentaris owns
the worldwide rights to Solorel((R)) which, if approved, would become the first orally available diagnostic
test for AGHD.
AGHD affects 35,000 adult Americans, with 6,000 new adult patients
diagnosed each year. Growth hormone (GH) not only plays an important
role in growth from childhood to adulthood, but helps promote good
health throughout life. AGHD is usually characterized by low energy
levels, decreased strength and exercise tolerance, increased weight or
difficulty losing weight, emotional changes, anxiety and impaired
sleep. Available diagnostic tests for AGHD are complex and can produce
significant side effects.
About Aeterna Zentaris Inc.
Aeterna Zentaris is a late-stage oncology drug development company
currently investigating potential treatments for various cancers
including colorectal, ovarian, endometrial cancer and multiple myeloma.
The Company’s innovative approach of “personalized medicine” means
tailoring treatments to a patient’s specific condition and to unmet
medical needs. Aeterna Zentaris’ deep pipeline is drawn from its
proprietary discovery unit providing the Company with constant and
long-term access to state-of-the-art therapeutic options. For more
information please visit www.aezsinc.com.
This press release contains forward-looking statements made pursuant to
the safe harbour provisions of the U.S. Securities Litigation Reform
Act of 1995. 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, the successful and timely
completion of clinical studies, the ability of the Company to take
advantage of business opportunities in the pharmaceutical industry,
uncertainties related to the regulatory process and general changes in
economic conditions. Investors should consult the Company’s quarterly
and annual filings with the Canadian and U.S. securities commissions
for additional information on risks and uncertainties relating to
forward-looking statements. Investors are cautioned not to rely 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, unless required to do
so by a governmental authority or by applicable law.
SOURCE AETERNA ZENTARIS INC.