QLT Inc. (NASDAQ:QLTI); (TSX:QLT) (“QLT” or the “Company”)
announced today completion of enrollment in its retrospective,
uncontrolled, multicenter Natural History Study in subjects with
Inherited Retinal Disease (IRD) phenotypically diagnosed with
Retinitis Pigmentosa (RP) or Leber Congenital Amaurosis (LCA) due
to underlying Retinal Pigment Epithelial 65 protein (RPE65) or
Lecithin:Retinol Acyltransferase (LRAT) gene mutations. The
objective of the retrospective, open label study is to gather data
on the natural progression of the disease in subjects over time,
including from childhood through to adulthood. The study
included both subjects who had previously received treatment in
QLT-sponsored clinical trials with QLT’s synthetic oral retinoid
product, QLT091001, as well as subjects who had not received any
prior therapeutic treatment. For subjects who had received
prior treatment with QLT091001, the study included a retrospective
review of subjects’ medical records prior to and after treatment
with QLT091001.
“We are very pleased to have completed on
schedule the collection of the data from what we believe to be the
first retrospective natural history clinical study conducted
specifically in RP and LCA patients due to underlying RPE65 or LRAT
gene mutations,” said Geoffrey F. Cox Ph.D., QLT’s Interim Chief
Executive Officer. “We sincerely thank our participating
physicians, all of whom are noted leaders in the field of Inherited
Retinal Disease, along with their corresponding sites for their
contributions to this study. Although the deterioration over
time of both visual field and visual acuity and various other
visual functions in these patients, has, for a long time, been the
assumption of treating physicians of these patients, we believe
this study will provide important comparative data for the future
treatment and care of these patients. This study is also
expected to provide important data to support the ongoing
development program for QLT091001, including our planned future
submissions for regulatory approval in Europe and the U.S.
Following our planned discussions with regulatory
authorities, we look forward to publicly reporting the final
results from the Natural History Study.”
Historically, it has been generally accepted
that subjects born with IRD due to RPE65 or LRAT
gene mutations would be expected to demonstrate an overall decline
in visual field and visual acuity, but at varying rates and over
varying periods of time (years). In addition, these patients
may also demonstrate increasing retinal atrophy as the disease
progresses. The intention of this study was to collect data
from these patients retrospectively, but under a formal protocol,
thereby providing an opportunity to better observe and understand
the long term natural disease progression of untreated patients,
and to provide comparative data to further assess the extent to
which treatment with QLT091001 may prolong or improve visual
function relative to the underlying natural disease progression in
these subjects. The study was a multi-site study intended to
enroll up to 60 subjects from established IRD patient populations
at 10 clinical study sites in Europe, the U.S. and Canada. In
total, 59 patients were enrolled in the study, 25 of whom had
participated in previous clinical studies with QLT091001, and 34 of
whom were treatment naive. Data was collected from patient
records for a variety of visual outcome measures, with particular
reference to changes in visual field and visual acuity for
individual subjects over multiple years, and in some cases,
decades, often starting in childhood.
The Company has completed its preliminary
analysis and believes that the data suggests that these patients,
without other interventions, demonstrate a continuing decline in
visual field and eventually visual acuity over time.
The Company intends to present the results for
discussion with selected national European Agencies and later with
the U.S. FDA. As a follow-up to previous meetings held with
European regulatory authorities in the first quarter of 2015 to
discuss the potential for conditional approval of QLT091001 on the
basis of existing clinical data, a meeting with selected national
European Agencies is scheduled to take place during the second
quarter of 2016. The purpose of the meeting is to present the
results of the Natural History Study and discuss whether the
results may support a potential filing of a marketing authorization
application (MAA) to the EMA for conditional approval in the second
half of this year.
In addition, the Company is working towards
initiating a pivotal, Phase III multi-center, placebo-controlled,
double-masked clinical study for this indication potentially in the
third quarter of 2016. The objective of this study will be to
further evaluate the efficacy (with particular reference to changes
in visual field and visual acuity) and safety of QLT091001,
with a goal of supporting future application for full
approval of QLT091001 for this indication to the EMA and the U.S.
FDA. The pivotal trial is expected to enroll 48 patients at
approximately 12 sites in the EU, U.S. and Canada.
About Synthetic Retinoid
Drugs
Inherited retinal diseases in the eye such as
Leber 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, and is under
investigation for the treatment of Inherited Retinal Disease (IRD)
in subjects phenotypically diagnosed with Retinitis Pigmentosa (RP)
or Leber Congenital Amaurosis (LCA) due to underlying gene
mutations in RPE65 or LRAT. QLT091001 has received orphan drug
designations for the treatment of LCA and RP (all mutations) by the
European Medicines Agency, and for the treatment of RP (all
mutations) and LCA (due to inherited mutations in the LRAT and
RPE65 genes) by the U.S. Food and Drug Administration (FDA).
The drug has also been granted two Fast Track designations by the
FDA for the treatment of the LRAT and RPE65 genetic mutations in
both LCA and autosomal recessive RP.
About Leber Congenital Amaurosis
(LCA) Due to RPE65 and LRAT Mutations
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. Both rod and cone photoreceptors are affected in LCA. 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.
According to current epidemiological estimates, LCA affects
approximately one in 81,000 newborns worldwide, of which
approximately 10% carry the inherited deficiencies of either RPE65
or LRAT.
About Retinitis Pigmentosa (RP) Due to RPE65 and LRAT
Mutations
RP is a set of hereditary retinal diseases demonstrating
clinical features similar to LCA. RP is also characterized by
degeneration of rod and cone photoreceptors, but it presents with a
more variable loss of vision in late childhood to adulthood.
Deficits in dark adaptation and peripheral vision are particular
hallmarks of RP. RP is currently estimated to affect at least
300,000 individuals worldwide, of which approximately 20%-30% are
autosomal recessive (arRP). It is currently estimated that less
than 3% of autosomal recessive RP patients carry the inherited
deficiencies of either RPE65 or LRAT.
About QLT
QLT is a biotechnology company dedicated to the
development and commercialization of innovative ocular products
that address the unmet medical needs of patients and clinicians
worldwide. We are focused on developing our synthetic retinoid
program for the treatment of certain inherited retinal
diseases.
QLT’s head office is based in Vancouver, Canada
and the Company is publicly traded on NASDAQ Stock Market (symbol:
QLTI) and the Toronto Stock Exchange (symbol: QLT). For more
information about the Company’s products and developments, please
visit our web site at www.qltinc.com.
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
concerning the potential efficacy, safety and market potential for
QLT091001; statements concerning the potential results and utility
of the Natural History Study, and the expected timing to complete
the final data analysis and potential discussions with the
regulatory authorities; statements concerning our ability to and
anticipated timing to make submissions to the regulatory
authorities concerning QLT091001, including potentially for
conditional approval with the EMA and potentially for full approval
with the EMA and FDA; statements concerning our plans to and
expected timing to commence a pivotal trial in QLT091001; and
statements which contain language such as: “assuming,” “prospects,”
“goal,” “future” “projects,” “potential,” “could,” “believes,”
“expects”; “hopes” 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 risks,
uncertainties and other factors: the effect that QLT’s
announcements and actions will have on the market price of our
securities; QLT’s development plans, timing and results of the
clinical development of our synthetic retinoid program are
uncertain; the risk that it may take longer and cost more than
anticipated to complete data analyses, complete preparatory work,
meet with regulators and commence trials (including a pivotal
trial) for numerous reasons both within and outside of our control;
assumptions related to the associated costs of our synthetic
retinoid program may prove incorrect; the risk that outcomes for
our clinical trials may not be favorable or may be less favorable
than interim/preliminary results disclosed and/or previous trials;
the possibility that interpretations of data produced by one or
more of our clinical trials will vary, including by the regulatory
authorities; the timing, expense and uncertainty associated
with the regulatory approval process for products to advance
through development stages; risks and uncertainties associated with
the safety and effectiveness of our synthetic retinoid program;
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; the
Company’s future operating results, which are uncertain and likely
to fluctuate; currency fluctuations; 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.
QLT Inc. Contacts:
Investor Relations
Andrea Rabney or David Pitts
Argot Partners
212-600-1902
andrea@argotpartners.com
david@argotpartners.com
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