TOORAK, Australia, March
21, 2014 /PRNewswire/ -- Antisense Therapeutics Limited
("ANP") is pleased to report that 24 acromegalic patients have been
successfully enrolled and randomized to one of the two treatment
regimens of dosing in the Phase II trial of ATL1103 for the
growth disorder, acromegaly. This satisfies the necessary patient
numbers proposed for the trial.
Following from the positive results achieved from the interim
analysis of the serum Insulin-like Growth Factor-I (sIGF-I) data
from the 8 patients who had completed the full 13 weeks of
dosing in the trial, a further 8 (16 in total) have now
completed dosing. Notably to date no patients dosed with ATL1103
have withdrawn from the study nor have there been any reports of
serious adverse events identified as treatment related.
With patient enrollment now complete, ANP anticipates reporting
the results of the statistical analysis of the sIGF-I data from all
patients by mid 2014. Reducing sIGF-I levels is the primary marker
of ATL1103 activity in this trial as acromegaly patients have
elevated sIGF-I levels compared to the normal population, and
reduction of sIGF-I to within a normal range in a significant
proportion of patients is the goal in Phase III registration trials
for acromegaly treatments.
Mark Diamond, Managing Director
and CEO of Antisense Therapeutics said, "Completion of patient
recruitment into a clinical trial is a major milestone and
especially in the case of trials involving patients with a rare or
orphan status disease such as acromegaly and so we are very pleased
to report this significant achievement. We now look forward to
completing the dosing in all patients and to the reporting of the
results."
ATL1103 Phase II trial is a randomised, open-label,
parallel group study of the safety, tolerability, pharmacokinetics
and efficacy of two subcutaneous dosing regimens of ATL1103 in 24
adult patients with acromegaly dosed with ATL1103 for 13 weeks (3
months) with two months of follow up. Two ATL1103 dosing regimens
are being tested (a) 200 mg 3 times in the first week then once
weekly thereafter (200 mg/week) or (b) 200 mg 3 times in the first
week then twice weekly thereafter (400 mg/week). The primary
endpoints or main purposes of the trial as listed on the trial
protocol are (i) to evaluate the safety and tolerability of ATL1103
in patients with acromegaly, and (ii) to evaluate the single dose
and multiple dose pharmacokinetic profiles of ATL1103 via the
subcutaneous route in patients with acromegaly. A secondary, but
important endpoint that is also on the trial protocol is the
evaluation of ATL1103's effect on serum insulin like growth factor
I (IGF-I) levels in patients. The secondary endpoint is the average
percentage reduction in serum IGF-I levels at the end of treatment
compared to baseline levels for each of the two dosing regimens
used in the Phase II study.
ATL1103 is a second generation antisense drug designed to
block growth hormone receptor (GHr) expression thereby reducing
levels of the hormone insulin-like growth factor-I (IGF-I) in the
blood and is a potential treatment for diseases associated with
excessive growth hormone and IGF-I action. These diseases include
acromegaly, an abnormal growth disorder of organs, face, hands and
feet, diabetic retinopathy, a common disease of the eye and a major
cause of blindness, diabetic nephropathy, a common disease of the
kidney and major cause of kidney failure, and some forms of cancer.
Acromegalic patients are known to have significantly higher blood
IGF-I levels than healthy individuals. Reduction of these levels to
normal is accepted by clinical authorities as the primary marker of
an effective drug treatment for the disease. GHr is a clinically
validated target in the treatment of acromegaly. In the case of
diabetic retinopathy, published clinical studies have shown that
treatments producing a reduction in IGF-I levels retarded the
progression of the disease and improve vision in patients.
Scientific papers have been published on the suppression of blood
IGF-I levels in mice (Tachas et al., 2006, J Endocrinol 189,
147-54) and inhibition of retinopathy in a mouse retinopathy model
(Wilkinson-Berka et al., 2007, Molecular Vision 13, 1529- 38;)
using an antisense drug to the GHr. ANP have also reported that
ATL1103 suppressed circulating levels of IGF-I in primates. In a
Phase I study in normal volunteers, ATL1103 was assessed as being
safe and well tolerated, while also demonstrating a preliminary
indication of drug activity including suppression of IGF-I and the
target GHR (growth hormone binding protein) levels. ATL1103
commercialisation is covered by patents to at least 2024, with the
potential for extensions up to 2029 in some countries and 2030 in
the US.
Acromegaly is a serious chronic life threatening disease
triggered by excess secretion of growth hormone (GH) by benign
pituitary tumours. Oversupply of GH over stimulates liver, fat and
kidney cells, through their GH receptors, to produce excess levels
of (IGF-I) in the blood manifesting in abnormal growth of the face,
hands and feet, and enlargement of body organs including liver,
kidney and heart. The primary treatments for acromegaly are to
surgically remove the pituitary gland and/or drug therapy to
normalize GH and serum IGF-I levels. In North America and Europe there are approximately 85,000
acromegaly patients with about half requiring drug therapy. Cost of
drug therapy ranges from approximatelyA$30,000/annum to over
A$60,000/annum depending on the
treatment.
Antisense Therapeutics Limited (ASX: ANP) is an
Australian publicly listed biopharmaceutical drug discovery and
development company. Its mission is to create, develop and
commercialise second generation antisense pharmaceuticals for large
unmet markets. ANP has 5 products in its development pipeline that
it has in-licensed from Isis Pharmaceuticals Inc., world leaders in
antisense drug development and commercialisation - ATL1102
(injection) which has successfully completed a Phase II efficacy
and safety trial, significantly reducing the number of brain
lesions in patients with multiple sclerosis and is also in clinical
development as a potential stem cell mobilisation agent, ATL1103 a
second-generation antisense drug designed to block GHr production
and thereby lower blood IGF-I levels and is in clinical development
as a potential treatment for growth and other GH-IGF-I disorders,
ATL1102 (inhaled) which is at the pre-clinical research stage as a
potential treatment for asthma and ATL1101 a second-generation
antisense drug at the pre-clinical stage being investigated as a
potential treatment for cancer.
Contact Information:
Website: www.antisense.com.au
Managing Director: Mark Diamond +61
(3) 9827 8999
USA Investor/Media: Joshua Drumm +(1) 212 375 2664;
jdrumm@tiberend.com
Australia Investor/Media: Simon
Watkin +61 (0)413 153 272; simon@marketconnect.com.au
SOURCE Antisense Therapeutics Limited