QLT initiates Phase Ib study of QLT091001 in Leber Congenital Amaurosis
15 Diciembre 2009 - 6:00AM
PR Newswire (US)
VANCOUVER, Dec. 15 /PRNewswire-FirstCall/ -- QLT Inc. (NASDAQ:
QLTI; TSX: QLT) ("QLT" or the "Company") today announced that it
has initiated enrollment in a Phase Ib trial of QLT091001 in
pediatric patients with Leber Congenital Amaurosis (LCA), an
inherited progressive retinal degenerative disease that leads to
retinal dysfunction and visual impairment beginning at birth.
QLT091001 is an orally administered synthetic retinoid replacement
for 11-cis-retinal, which is a key biochemical component of the
visual retinoid cycle. The Phase Ib trial is an open-label, single
center trial to evaluate the safety profile and effects on retinal
function in 8 pediatric patients (aged 5 to 14 years) with LCA due
to inherited deficiency of RPE65 or LRAT. Patients will receive
daily doses of QLT091001 for 7 days at the Montreal Children's
Hospital in Montreal, Canada under the trial's Principal
Investigator, Dr. Robert Koenekoop. The trial will monitor for
changes in several visual function parameters including the
best-corrected visual acuity over the duration of the study. "Given
the encouraging nonclinical testing in animal models and the safety
results from the Phase Ia healthy adult volunteer trial, we are
very excited to move QLT091001 into Phase Ib testing," said Bob
Butchofsky, President and Chief Executive Officer of QLT. "Children
with LCA have few treatment options and we are hopeful that QLT
will be able to advance this compound in clinical trials. We look
forward to reporting data from this trial in the next few months."
About Synthetic Retinoid Drugs Genetic diseases in the eye such as
LCA and Retinitis Pigmentosa (RP) arise from gene mutations of
enzymes or proteins required in the biochemistry of vision.
QLT091001 is a replacement for 11-cis-retinal, which is an
essential component of the retinoid-rhodopsin cycle and visual
function. Two different gene mutations (Retinal pigment epithelium
protein 65 (RPE65) and lecithin-retinol acyltransferase (LRAT)
result in an inadequate production of 11-cis-retinal and occur in
approximately 10% of patients with LCA and to a lesser extent in
RP. The basis for using synthetic retinoids as replacement therapy
for conditions where genetic defects result in deficiency of
11-cis-retinal is founded on experiments in mouse genetic models.
These experiments used mice that have mutations in either the Rpe65
or Lrat genes, the same as those associated with LCA in humans.
Both mouse models have clinical features of the human disease. The
biological activity of the synthetic retinoid was monitored by
measuring the level of pigment-related compounds in the eye.
Retinal function was also assessed by detecting electroretinograms
(ERGs) and electrical nerve signals from the retina. Oral
administration of QLT091001 showed evidence of having corrected the
biochemical defect in the retinoid cycle in light-sensing cells
(rods) and appeared to restore ERG responses to light in both
models of LCA. About Leber's Congenital Amaurosis LCA is an
inherited degenerative retinal disease characterized by
abnormalities such as roving eye movements and sensitivity to
light, and manifesting in severe vision loss from birth. Eye
examinations of infants with LCA reveal normal appearing retinas.
However, low level of retinal activity, measured by
electroretinography, indicates very little visual function. About
QLT QLT Inc. is a pharmaceutical company dedicated to the
development and commercialization of innovative therapies for the
eye. We are focused on our commercial product Visudyne for the
treatment of wet-AMD, and the development of drugs to be delivered
in our proprietary punctal plug devices. For more information,
visit our website at http://www.qltinc.com/. In April of 2006, QLT
entered into an exclusive worldwide co-development and licensing
agreement with Retinagenix, LLC to develop active synthetic
retinoid products for the treatment of degenerative retinal
diseases. Under the terms of the agreement, QLT is responsible for
developing and commercializing the products for use in ocular and
all other human diseases. Retinagenix has participated in research
in support of the co-development collaboration and is eligible to
receive payments upon achievement of certain development, approval
and sales milestones as well as a royalty on net sales. QLT Plug
Delivery, Inc. is a wholly-owned subsidiary of QLT Inc. Visudyne is
a registered trademark of Novartis AG. QLT Inc. is listed on The
NASDAQ Stock Market under the trading symbol "QLTI" and on The
Toronto Stock Exchange under the trading symbol "QLT." Certain
statements contained in this press release, which are not
historical facts, are "forward-looking statements," of QLT within
the meaning of the Private Securities Litigation Reform Act of 1995
and constitute "forward-looking information" within the meaning of
applicable Canadian securities laws. Such statements include, but
are not limited to: our beliefs regarding the potential benefits,
targets, market opportunity and commercial success of our synthetic
retinoid drug; our expectations regarding our clinical development
plans and strategy for the technology and timelines associated with
these; and statements which contain language such as "expects,"
"will," "plans," "estimates," "intends," "believes" and similar
expressions that do not relate to historical matters.
Forward-looking statements are predictions only which involve known
and unknown risks, uncertainties and other factors that may cause
our actual results to be materially different from the results
expressed or implied by such statements. Many such risks,
uncertainties and other factors are taken into account as part of
our assumptions underlying these forward-looking statements and
include, among others, the following: risks and uncertainties
associated with the timing, expense and outcome of research and
development programs and commercialization of products (including
the difficulty of predicting the timing and outcome of the
synthetic retinoid drug development efforts, clinical testing and
regulatory approvals or actions); uncertainties regarding the
impact of competitive products and pricing; risks and uncertainties
associated with the safety and effectiveness of our technology;
risks and uncertainties related to the scope, validity, and
enforceability of our intellectual property rights and the impact
of patents and other intellectual property of third parties; and
other factors as described in detail in QLT's Annual Report on Form
10-K, Quarterly Reports on Form 10-Q and other filings with the
U.S. Securities and Exchange Commission and Canadian securities
regulatory authorities. Forward-looking statements are based on the
current expectations of QLT and QLT does not assume any obligation
to update such information to reflect later events or developments
except as required by law. DATASOURCE: QLT Inc. CONTACT: QLT Inc.
Media Contact: Vancouver, Canada, Karen Peterson, Telephone: (604)
707-7000 or 1-800-663-5486, Fax: (604) 707-7001, ; The Trout Group
Investor Relations Contact: New York, USA, Christine Yang,
Telephone: (646) 378-2929, or Marcy Nanus, Telephone: (646)
378-2927,
Copyright