---------- Forwarded message ----------
From: Prasad Gurav <pbgu...@yahoo.com>
Date: Sat, 1 May 2010 23:29:42 -0700 (PDT)
Subject: (RPP-India) Fw: Pressrelease by LPT on LCA trial
To: rpp-in...@googlegroups.com, rfic...@googlegroups.com
One more hope for LCA patients! Read On and Enjoy your Sunday.
-Prasad
QLT Announces Positive Interim Results From Phase 1b Study of its Oral
Synthetic Retinoid Compound in Leber Congenital Amaurosis
VANCOUVER, British Columbia, Apr 20, 2010 (GlobeNewswire via COMTEX)
-- QLT Inc. /quotes/comstock/15*!qlti/quotes/nls/qlti (QLTI 6.32,
-0.14, -2.17%) /quotes/comstock/11t!e:qlt (CA:QLT 6.42, -0.13, -1.98%)
("QLT" or the "Company") today announced interim results from the
first 3 subjects enrolled in a Phase 1b clinical proof-of-concept
study of QLT091001 in the treatment of Leber congenital amaurosis
(LCA), an inherited progressive retinal degenerative disease that
leads to retinal dysfunction and significant visual impairment
beginning at birth. QLT091001 is an orally administered synthetic
retinoid replacement for 11-cis-retinal, which is a key biochemical
component of visual function.
The Phase 1b trial is a short-term, open-label, single-center study to
evaluate the safety profile and effects on retinal function in 8
pediatric subjects (aged 5 to 14 years) diagnosed with LCA due to
inherited deficiency of retinal pigment epithelium protein 65 (RPE65)
or lecithin:retinol acyltransferase (LRAT). Based on the positive
results from the first 2 pediatric patients, a protocol exception was
granted to also treat an adult patient. Subjects receive daily oral
doses of QLT091001 for 7 days at the Montreal Children's Hospital at
the McGill University Health Centre, Montreal, Canada under the
supervision of the trial's principal investigator, Robert K.
Koenekoop, M.D., Ph.D. Patients were monitored to ensure overall
safety. Efficacy assessments included several visual function
parameters including best-corrected visual acuity and visual field
testing.
Interim Results
Three subjects aged 10, 12, and 38 years, all of whom have a genetic
mutation in LRAT, have been enrolled and treated to date. After 7 days
of treatment with QLT091001, all of the subjects experienced
clinically relevant improvements in one or more visual function
parameters, including best-corrected visual acuity, Goldmann visual
field, and/or retinal sensitivity as measured by full-field
sensitivity threshold testing. Subjects have also reported meaningful
improvements in their visual performance related to tasks of daily
living. The onset of visual changes was rapid and there was
progressive improvement beyond the 7 days of treatment, with some
effects persisting for up to 4 months after treatment was completed.
Improvements were most pronounced in the youngest subject, but
clinically relevant changes were also noted in the one adult subject
treated to date. The study treatment has been well-tolerated, with
mild to moderate adverse events observed
including transient headache and photophobia, and an increase in
triglyceride levels. The study is ongoing and will enroll additional
subjects, including those who have LCA due to mutations in RPE65.
Because of the prolonged treatment effects, the study will also
continue to gather longer-term follow-up data on these subjects.
Completion of the current trial is expected before year end.
The results from the first 3 subjects will be introduced and discussed
by Dr. Koenekoop in a previously scheduled mini-symposium entitled,
"An Overview of Retinal Dystrophies: from Gene Discoveries to New
Therapies" at the Association for Research in Vision and Ophthalmology
(ARVO) Annual Meeting in Fort Lauderdale, Florida on May 3, 2010 at
3:45 p.m. (Eastern Time).
"These preliminary results are very exciting, are better than
expected, and provide a measure of hope that a treatment might be
developed for this devastating disease. We are intent on continuing
the trial and undertaking further research into the safety and
efficacy of this compound," said Dr. Koenekoop, Director of the McGill
Ocular Genetics Laboratory and Chief of Pediatric Ophthalmology at
Montreal Children's Hospital. "We look forward to sharing these data
with the ophthalmology community beginning at ARVO in early May."
"We are very excited about the positive outcomes for these patients
and are eager to complete patient enrollment," said Bob Butchofsky,
President and Chief Executive Officer of QLT.
While these early results are promising, the safety and efficacy of
QLT091001 remains to be fully evaluated through additional preclinical
and clinical testing. QLT091001 cannot be made available to patients
with LCA outside of regulated clinical trials, such as the current
study.
About Leber Congenital Amaurosis (LCA)
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, a low level
of retinal activity, measured by electroretinography, indicates very
little visual function. Approximately 1 child out of every 81,000
births will inherit the disease. Mutations in the genes for 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 retinitis pigmentosa (RP), another inherited
retinal dystrophy.
About Synthetic Retinoid Drugs
Genetic diseases in the eye such as LCA and 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.
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,
including those developed in the laboratory of Dr. Krzysztof
Palczewski. 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 QLT
QLT Inc. is a biotechnology company dedicated to the development and
commercialization of innovative therapies for the eye. We are focused
on our commercial product Visudyne(R) for the treatment of wet-AMD,
developing drugs to be delivered in our proprietary punctal plug
delivery system, as well as developing our synthetic retinoid program
for the treatment of certain inherited retinal diseases. For more
information, visit our website at 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.
Visudyne(R) 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."
The QLT Inc. logo is available at
http://www.globenewswire.com/newsroom/prs/?pkgid=6933
Certain statements in this press release constitute "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. Forward looking statements include, but are not limited to: our
statements related to our development plans and enrollment for our
QLT091001 Phase Ib clinical trial and any other development plans for
QLT091001; our statement relating to the expected timing for
completion of the Phase Ib clinical trial; our statements relating to
the prospects for the development of a treatment for LCA; and
statements which contain language such as: "assuming," "may," "will,"
"prospects," "future," "projects," "believes," "expects" and
"outlook." Forward-looking statements are predictions only which
involve known and unknown risks, uncertainties and other factors that
may cause actual results to be materially
different from those expressed in 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: the Company's future operating results
are uncertain and likely to fluctuate; uncertainties relating to the
timing and results of the clinical development and commercialization
of our products and technologies (including, but not limited to,
Visudyne, our punctal plug technology and our synthetic retinoid
program); outcomes for our clinical trials of our programs (including,
but not limited to, our punctal plug technology and our synthetic
retinoid program) may not be favorable or may be less favorable than
interim results and/or previous trials; there may be varying
interpretations of data produced by one or more of our clinical
trials; uncertainties relating to the associated costs of our
programs; the timing, expense
and uncertainty associated with the regulatory approval process for
products; uncertainties regarding the impact of competitive products
and pricing; risks and uncertainties associated with the safety and
effectiveness of our technology and products, including our synthetic
retinoid product; 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 general economic conditions and other factors 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.
This news release was distributed by GlobeNewswire, www.globenewswire.com
SOURCE: QLT Inc.
CONTACT: QLT Inc.
Media Contact:
Karen Peterson
604-707-7000
1-800-663-5486
Fax: 604-707-7001
kpeter...@qltinc.com
Vancouver, Canada
The Trout Group
Investor Relations Contact:
New York, U.S.A.
Christine Yang
646-378-2929
cy...@troutgroup.com
Boston, Massachusetts, USA
Tricia Swanson
617-583-1306
tswan...@troutgroup.com
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