VANCOUVER, Oct. 9 /CNW/ - QLT Inc. (NASDAQ: QLTI; TSX: QLT) announced
today that it has initiated a Phase I safety study in healthy adults of
QLT091001, an orally administered synthetic retinoid replacement therapy for
11-cis-retinal, which is a key biochemical component of the visual retinoid
cycle. The drug is being developed for the potential treatment of Leber's
Congenital Amourosis (LCA), an inherited progressive retinal degenerative
disease that leads to retinal dysfunction and visual impairment beginning at
The Phase I study is an open-label, single center, ascending dose trial
that will determine the safety and tolerance of multiple administrations of
the synthetic retinoid drug in approximately 18 healthy adult volunteers.
Participants will be enrolled in up to 6 cohorts of increasing doses.
"We believe our retinoid synthetic drug program is a very interesting
opportunity as there are no currently available treatments for patients with
LCA, which affects one in one-hundred thousand newborns world-wide," said Bob
Butchofsky, President and Chief Executive Officer of QLT. "This program also
supports the Company's new ocular focus and we look forward to reporting the
Phase I data in the first half of 2009. We hope that our findings will support
further clinical studies in this orphan indication."
About Synthetic Retinoid Drugs
Genetic diseases in the eye such as Leber's Congenital Amaurosis (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), 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
Leber 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 (ERG), indicates
very little visual function.
QLT Inc. is a global biopharmaceutical company dedicated to the
discovery, development and commercialization of innovative therapies. Our
research and development efforts are focused on pharmaceutical products in the
fields of ophthalmology and dermatology. In addition, we utilize three unique
technology platforms, photodynamic therapy, Atrigel(R) and punctal plugs with
drugs, to create products such as Visudyne(R) and Eligard(R) and future
product opportunities. For more information, visit our web site at
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.
Pre-clinical studies have demonstrated that orally administered synthetic
retinoid drugs cause long-lasting restoration of retinal function. Under the
terms of the agreement, QLT is responsible to develop and commercialize 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 single digit royalty on net sales.
QLT Plug Delivery, Inc. is a wholly-owned subsidiary of QLT Inc.
Atrigel is a registered trademark of QLT USA, Inc.
Visudyne is a registered trademark of Novartis AG.
Eligard is a registered trademark of Sanofi-aventis.
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.
List of references:
Batten ML, Imanishi Y, Tu DC, et al. Pharmacological and rAAV gene
therapy rescue of visual functions in a blind mouse model of Leber
congenital amaurosis. PLoS Medicine. 2005;2(11):1177-1189.
Van Hooser JP, Liang Y, Maeda T, et al. Recovery of visual functions in a
mouse model of Leber congenital amaurosis. J Biol Chem.
Van Hooser JP, Aleman TS, He Y-G, et al. Rapid restoration of visual
pigment and function with oral retinoid in a mouse model of childhood
blindness. Proc Natl Acad Sci USA. 2000;97(15):8623-8628.
Travis GH, Golczak M, Moise AR, and Palczewski K. Diseases caused by
defects in the Visual Cycle: Retinoids as Potential Therapeutic Agents.
Annu. Rev. Pharmocol. Toxicol. 2007;47: 469-512
For further information:
For further information: 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 Strickler Telephone: (646) 378-2927