QLT Inc. (Nasdaq:QLTI) (TSX:QLT) ("QLT" or the "Company") today
announced the publication of data from its Phase 1b
proof-of-concept trial of QLT091001 in subjects with Leber
Congenital Amaurosis (LCA) due to inherited genetic mutations in
retinal pigment epithelium (RPE65) or lecithin:retinol
acyltransferase (LRAT) in The Lancet. The peer-reviewed journal
article is available first in the online edition of The Lancet at
www.thelancet.com. In the study, treatment for 7 days with oral
QLT091001 demonstrated a restoration of clinically meaningful
visual function in 11 of the 14 LCA patients, as measured by
improvement in Goldmann Visual Fields (GVF) or visual acuity.
Self-reported or parent-reported improvements in activities of
daily living supported these findings.
"Currently, children and adults with LCA due to these genetic
mutations progressively lose vision, eventually becoming totally
blind as there are no proven therapies that either slow or reverse
their condition," stated David A. Saperstein, M.D., a retina
physician who serves as Chief Medical Advisor to QLT and is a
co-author of the Lancet paper. "The results of this trial are quite
promising. If borne out in further clinical trials, QLT091001 would
have the potential to become the first oral medication to improve
vision and quality of life in patients with these forms of
LCA."
The trial enrolled 14 patients, ages 6–38, with Leber Congenital
Amaurosis due to mutations in RPE65 or LRAT. Patients received 7
days of oral QLT091001 (12 subjects received 40 mg/m2 per day and 2
subjects received 10 mg/m2 per day). Patients were assessed at
baseline and days 7, 9, 14, and 30, and every 2 months thereafter,
for up to 3 years, for safety outcomes and visual function
endpoints, which included GVF, visual acuity, and a functional MRI
assessment.
In the study, 10 of 14 patients had an improvement of GVF, with
a mean increase in retinal area of 28–683%. Six patients had an
improvement in visual acuity, with a mean increase of 2–30 letters.
After two years, three patients had a sustained GVF response and
four had a sustained visual acuity response. Four patients had
functional MRI scans, which correlated with visual response or
absence of response to treatment. Ten of the responders had
self-reported improvements in activities of daily living.
No serious adverse events occurred, although transient
headaches, photophobia, reduction in serum HDL concentrations, and
mild, reversible increases in serum triglycerides and aspartate
aminotransferase concentrations were observed.
"Publication of this data in a preeminent journal such as The
Lancet further highlights the importance of our findings and the
potential of QLT091001 to rapidly improve visual function in
patients with LCA," stated Jason M. Aryeh, Chairman of the Board.
"Together with our Phase 1b retreatment data reported earlier this
year, these results underscore QLT091001's value as a potential
treatment for inherited retinal diseases due to RPE65 and LRAT
mutations."
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.
The FDA has also formally acknowledged that the orphan drug
designations granted by the FDA on QLT091001 also cover QLT091001
for the treatment of Inherited Retinal Disease caused by LRAT or
RPE65 mutations, including severe early childhood onset retinal
dystrophy, which disease/condition we believe subsumes both LCA and
RP. 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 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.
On June 25, 2014, QLT entered into an Agreement and Plan of
Merger (the "Merger Agreement") among QLT, Auxilium
Pharmaceuticals, Inc., a Delaware corporation ("Auxilium"), QLT
Holding Corp., a Delaware corporation and a wholly owned subsidiary
of QLT ("HoldCo"), and QLT Acquisition Corp., a Delaware
corporation and a wholly owned subsidiary of HoldCo ("AcquireCo").
The Merger Agreement provides for a business combination whereby
AcquireCo will be merged with and into Auxilium (the "Merger"). As
a result of the Merger, the separate corporate existence of
AcquireCo will cease and Auxilium will continue as the surviving
corporation. On the date of the closing of the Merger, Auxilium
will become an indirect wholly owned subsidiary of QLT (the
"Combined Company").
No Offer or Solicitation
This communication is not intended to and does not constitute an
offer to sell or the solicitation of an offer to subscribe for or
buy or an invitation to purchase or subscribe for any securities or
the solicitation of any vote or approval in any jurisdiction
pursuant to the acquisition or otherwise, nor shall there be any
sale, issuance or transfer of securities in any jurisdiction in
contravention of applicable law. No offer of securities shall be
made except by means of a prospectus meeting the requirements of
Section 10 of the Securities Act of 1933, as amended.
Additional Information
In connection with the proposed Merger, QLT plans to file with
the Securities and Exchange Commission (the "SEC") a registration
statement on Form S-4 that will include the joint proxy
statement/circular of Auxilium and QLT and also constitutes a
prospectus of QLT. Auxilium and QLT plan to mail the joint proxy
statement/circular to their respective stockholders. INVESTORS ARE
URGED TO READ THE JOINT PROXY STATEMENT/CIRCULAR WHEN IT BECOMES
AVAILABLE BECAUSE IT WILL CONTAIN IMPORTANT INFORMATION. You will
be able to obtain the joint proxy statement/circular, as well as
other filings containing information about Auxilium and QLT, free
of charge, at the website maintained by the SEC at www.sec.gov and,
in QLT's case, also on the System for Electronic Document Analysis
Retrieval ("SEDAR") website maintained by the Canadian Securities
Administrators ("CSA") at www.sedar.com. QLT stockholders may also
obtain these documents, free of charge, from QLT's website at
www.qltinc.com under the heading "Investors" and then under the
heading "Proxy Circulars" or upon request directly to QLT to the
attention of "QLT Investor Relations," 887 Great Northern Way,
Suite 250, Vancouver, British Columbia, Canada, V5T 4T5.
Auxilium stockholders may also obtain these documents, free of
charge, from Auxilium's website (www.Auxilium.com) under the
heading "Investors—SEC Filings" or by directing a request to made
to Auxilium's Secretary at Auxilium Pharmaceuticals, Inc., 640 Lee
Road, Chesterbrook, PA 19087.
Participants in the Solicitation
The respective directors and executive officers of QLT and
Auxilium and other persons may be deemed to be participants in the
solicitation of proxies in respect of the transactions contemplated
by the joint proxy statement/circular. Information regarding QLT
directors and executive officers is available in its Annual Report
on Form 10-K/A filed with the SEC and CSA by QLT on
April 30, 2014, and information regarding Auxilium's directors
and executive officers is available in its definitive proxy
statement filed with the SEC by Auxilium on April 10, 2014.
These documents can be obtained free of charge from the sources
indicated above. Other information regarding the interests of the
participants in the proxy solicitation will be included in the
joint proxy statement/circular and other relevant materials to be
filed with the SEC and the CSA when they become available.
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 statement that the data suggests that
QLT091001 may improve the lives of patients with LCA due to LRAT or
RPE65 mutations; our statements concerning the potential efficacy,
safety and market potential for QLT091001; statements relating to
the expected timing of the completion of the Merger, the expected
benefits of the proposed Merger, whether the combined company's
profitability will significantly increase and the competitive
ability and position of the combined company; 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; the failure to receive, on a
timely basis or otherwise, the required approvals by Auxilium and
QLT stockholders and government or regulatory agencies (including
the terms of such approvals); the risk that a condition to closing
of the Merger may not be satisfied; the possibility that the
anticipated benefits and synergies from the proposed Merger cannot
be fully realized or may take longer to realize than expected; the
ability of Auxilium and QLT to obtain consents of lenders or to
obtain refinancing in connection with the transaction, and, if the
transaction is consummated, the adequacy of the capital resources
of the Combined Company; the possibility that costs or difficulties
related to the integration of Auxilium and QLT operations will be
greater than expected; the ability of the Combined Company to
retain and hire key personnel and maintain relationships with
customers, suppliers or other business partners; the impact of
legislative, regulatory, competitive and technological changes,
including changes in tax laws or interpretations that could
increase the Combined Company's or Auxilium's consolidated tax
liabilities, including, if the transaction is consummated, changes
in tax laws that would result in the Combined Company being treated
as a domestic corporation for United States federal tax purposes;
the risk that the credit ratings of the combined company may be
different from what the companies expect; QLT's development plans,
timing and results of the clinical development of our synthetic
retinoid program; the risk that our assumptions related to
continued enrollment trends, efforts and success, and the
associated costs of our synthetic retinoid program will 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; 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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