QLT Inc. (Nasdaq:QLTI) (TSX:QLT) ("QLT" or the "Company") today
announced positive preliminary results from its international,
multi-center, Phase 1b clinical trial of repeated treatments of
oral QLT091001 in subjects with Leber Congenital Amaurosis (LCA) or
Retinitis Pigmentosa (RP) due to inherited genetic mutations in
retinal pigment epithelium (RPE65) or lecithin:retinol
acyltransferase (LRAT).
Preliminary results of this Phase 1b study showed clinically
meaningful improvements in visual fields (VF) and visual acuity
(VA). To date, 19 of 27 subjects (70%) had an increase in VF
retinal area from baseline of ≥ 20% in at least 1 eye at 2
consecutive visits within 6 months from the start of any QLT091001
treatment course. In addition, 70% of subjects had an increase
in VA from baseline of ≥ 5 letters in at least 1 eye at 2
consecutive visits within 6 months from the start of any treatment
course. The percentage of VF and VA responders identified by
disease and mutation is summarized in the Table below. Dosing
in the study is now completed and subject follow-up is
ongoing. The final clinical data, including duration of
response and other evaluations, are anticipated for release in the
third quarter of 2014.
"We are very pleased to see additional positive data from
our ultra orphan program in LCA and RP," said Jason M. Aryeh,
Chairman of the Board. "Such results are highly encouraging to
us and our investigator colleagues to
move QLT091001 forward into a pivotal study. We hope that
QLT's Synthetic Retinoid will become a first-in-class oral therapy
for these important areas of unmet medical need. The QLT team
would like to thank all the patients who participated in this study
and appreciate the desire of many of them to continue therapy with
QLT091001."
This multicenter, open-label Phase 1b proof-of-concept trial was
an extension study in which LCA or RP subjects with RPE65 or LRAT
mutations who had been previously treated with a single course of
QLT091001 in the Company's previously completed Phase Ib study,
received up to three 7-day courses of QLT091001 to assess visual
outcomes and safety following retreatment. VF was assessed using
Goldmann Visual Field (GVF) and VA was assessed using
best-corrected visual acuity (BCVA) at baseline and days 7, 14, 30
and 60 after each treatment course, then bimonthly until the next
treatment course. Safety assessments included complete ophthalmic
and physical examinations, electroretinograms (ERGs), optical
coherence tomography (OCT) retinal architecture,
electrocardiograms, laboratory testing and reported adverse events.
Subjects received treatment with QLT091001 at doses of 10, 40 or
60 mg/m2, with the majority of subjects receiving 40 mg/m2.
Retreatment was initially determined based on established
retreatment criteria or at Investigator discretion. This was later
amended to allow retreatment to occur as early as 30 days but no
later than 60 days after a previous treatment course to maintain
dosing within a fixed interval. For these reasons, the time between
each treatment course in this trial varied between subjects for
each course and also varied between courses for each subject (1-13
months). There was also a wide range in time (7-32 months) per
subject that elapsed between the single course treatment in the
previously completed Phase Ib trial and treatment in this trial.
All adverse events reported in this trial were consistent with
the retinoid class of medications and were transient and/or
reversible. Only one serious adverse drug reaction (intracranial
hypertension (ICH), a known class effect of retinoids), was
reported in the study and it was resolved.
Table: Preliminary Results for Visual Field and Visual
Acuity Responders
|
|
|
|
|
N |
Visual Field
Respondersa Number (%) of Subjects |
Visual Acuity
Respondersb Number (%) of Subjects |
|
|
|
|
All Subjects |
27 |
19 (70%) |
19 (70%) |
|
|
|
|
All LCA |
13 |
7 (54%) |
10 (77%) |
All RP |
14 |
12 (86%) |
9 (64%) |
|
|
|
|
All RPE65 |
15 |
11 (73%) |
8 (53%) |
All LRAT |
12 |
8 (67%) |
11 (92%) |
a: ≥ 20% change in retinal area from baseline at 2 consecutive
visits in at least 1 eye within 6 months of any treatment
course.
b: ≥ 5 letter increase from baseline at 2 consecutive visits in
at least 1 eye within 6 months of any treatment course.
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. 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 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, and is under investigation for the treatment of LCA and
RP. QLT091001 has received orphan drug designations for the
treatment of LCA (due to inherited mutations in LRAT or RPE65
genes) and RP (all mutations) by the FDA, and for the treatment of
LCA and RP (all mutations) by the EMA. The drug has also been
granted two Fast Track designations by the FDA for the treatment of
LCA and RP due to inherited mutations in the LRAT and RPE65
genes. Recently, we submitted orphan drug designation
applications to the FDA and EMA for QLT091001 for the treatment of
inherited retinal disease caused by LRAT or RPE65 mutations, an
indication which includes both LCA and RP patients. The clinical
characteristics and progression of disease in LCA and RP overlap as
do some of their genetic causes. At least 7 of the known LCA
disease genes, including LRAT and RPE65, have also been linked to
the clinical appearance of RP. Despite disease heterogeneity
and terminology, there is an overlap in the genetic mechanisms
underlying some forms of LCA and RP such as those caused by LRAT
and RPE65 mutations where 11-cis-retinal production is either
severely or completely compromised. RP is the most common
inherited retinal disease, and is generally the diagnosis given to
patients who begin to lose vision after the first decade of life,
whereas the diagnosis of LCA is given to patients who have central
vision loss soon after birth. There is no universally accepted
diagnostic term for patients with characteristics in between;
clinicians have considered such cases as either LCA or severe
RP.
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 inherited retinal diseases.
QLT is based in Vancouver, Canada and the Company is publicly
traded on NASDAQ (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 our timing to
complete analysis and release final data on the trial; our
statements concerning the timing and our ability to launch a
pivotal trial of 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 risks,
uncertainties and other factors following: the effect that QLT's
announcements and actions will have on the market price of our
securities; our development plans, timing and results of the
clinical development of our synthetic retinoid program; assumptions
related to continued enrollment trends, efforts and success, and
the associated costs of our synthetic retinoid program; outcomes
for our clinical trials may not be favorable or may be less
favorable than interim/preliminary results disclosed and/or
previous trials; varying interpretations of data produced by one or
more of our clinical trials; 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.
CONTACT: QLT Inc. Contacts:
Investor & Media Relations
Andrea Rabney or David Pitts
Argot Partners
212-600-1902
andrea@argotpartners.com
david@argotpartners.com
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