Public Device & Biopharma Ophthalmology Company Showcase - QLT at OIS@AAO 2016.
Presenter:
David Saperstein, MD, Chief Medical Advisor
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Public Device & Biopharma Ophthalmology Company Showcase - QLT
1. New Products in Sight
Potential for Vision Improvement
in Inherited Retinal Diseases
LCA and RP due to RPE65 and LRAT Mutations
Using Oral Zuretinol Acetate (QLT 091001)
2. Forward-looking Statement
Certain statements in this presentation 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. Such statements include, but are not limited to: statements concerning our clinical development programs and future plans
for QLT091001 (Zuretinol Acetate), including regulatory and clinical plans and pathway and associated costs; any potential
submission for conditional approval with the European Medicines Agency; the results of our Natural History Study; our
advancement towards a pivotal trial of Zuretinol Acetate; the expected timing to make regulatory submissions and to commence
and receive data from clinical trials, including our assumptions related to initiation of new studies, current and future study
enrollment and timing to treat patients; statements concerning the potential benefits and success of our development programs; and
statements which contain language such as: “plan,” “potential,” “future,” “project,” “will,” “may,” “believe,” “intend,” “estimate,”
“expect,” “anticipate,” “target” and similar expressions. Forward-looking statements are based on estimates and assumptions made
by QLT in light of its experience and its perception of historical trends, current conditions and expected future developments, as
well as other factors that QLT believes are appropriate in the circumstances, including but not limited to: general economic
conditions, competition, clinical trial designs, progression of enrollment and development and interpretation of data. 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 our synthetic retinoid program) and
the associated costs of these programs; outcomes of our discussions with regulators and our studies for our synthetic retinoid
program may not be favorable or, in the case of studies, 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 studies, including our Natural History Study; the timing, expense
and uncertainty associated with the regulatory approval process for products; 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; currency fluctuations; and general economic
conditions. These factors and others relating to QLT are discussed in greater detail in the “Risk Factors” section of QLT’s Annual
Report on Form 10-K, Quarterly Reports on Form 10-Q, as well as in its other filings with the U.S. Securities and Exchange
Commission and Canadian securities regulatory authorities. Given these uncertainties, assumptions and risk factors, investors are
cautioned not to place undue reliance on such forward-looking statements. QLT has no intention and assumes no obligation to
update such information to reflect later events or developments, except as required by law. The views of Dr. Saperstein expressed
during this presentation are his personal views and do not necessarily represent the views of QLT. QLT expressly disclaims any
liability with respect to the views, opinions and beliefs expressed by Dr. Saperstein.
2
3. The Normal Visual Cycle
all trans retinol
LRATRPE 65
11-cis-retinal
rhodopsin
3
opsin
Vit A from
serum
Vision
Photoreceptor
Retinal Pigment
Epithelium
4. Mutations in RPE65 or LRAT Lead to Vision
Loss
all trans retinol
LRATRPE 65
11-cis-retinal
rhodopsin
4
opsin
Vit A from
serum
Vision
5. Oral Zuretinol Acetate Replaces 11-cis-retinal
and Restores Vision
all trans retinol
LRATRPE 65
9-cis-retinal
rhodopsin
5
opsin
Vit A from
serum
Oral
Zuretinol
Acetate
Vision
6. Vision Rescue in RPE65 Mutant Dog with
Intravitreal Zuretinol Acetate
Narfstrom, ARVO 2009
7. IRD01 - Phase 1b Clinical Trial
LCA and Early onset RP due to LRAT
and RPE65 mutations
Diagnosed shortly after birth or in
childhood
Open label, Multicentered (7)
Single 7 day oral dosing period
Several Exploratory endpoints and
safety evaluations
Patients followed until the effect
subsided
Dr. Koenekoop, Montreal
Dr. Fishman, Chicago Dr. Jacobson, Philadelphia
Dr. Scholl, Baltimore Dr. Moore, London
Dr. Zrenner, Tübingen Dr. van den Born,
Rotterdam
LRAT Subject Results
71% (10/14) of subjects were GVF
responders*
43% (6/14) of subjects were VA
responders**
RP Subject Results
44% (8/18) of subjects were GVF
responders*
67% (12/18) of subjects were VA
responders**
Combined Results
81% (26/32) of subjects responded
to GVF and/or VA*,**
* ≥ 20% improvement in mean log retinal area in at least one eye on 2 consecutive visits within 2 months
** ≥ 5-letter improvement on 2 consecutive visits within 2 months
8. Screening OS Screening OD
Day 14 OD
1 month post dosing OS 1 month post dosing OD
4 months post dosing OS 4 months post dosing
OD
Day 7 OS Day 7 OD
Day 14 OS
A
B
C
E
Visual Fields in 10 Year Old Patient Treated
with Oral Zuretinol Acetate
8
D
F
11 months post dosing OS 11 months post dosing
OD
9. RET IRD 02 – Zuretinol Acetate
Retreatment Study
Open-label, multi-center, Phase 1b
trial in the IRD 01 trial
Up to 3 - 7 day treatment courses
13 LCA subjects and 14 RP (27/32
IRD 01) enrolled
PATIENT REPORTED OUTCOMES
Walk without use of cane (2 subjects)
Maneuvering in the Dark
Navigating in public places
Increased peripheral vision
Read writing on a blackboard
Play Video games
Attention to Appearance
RESULTS
70% GVF Responders *
70% VA Responders**
9
* ≥ 20% improvement in mean log retinal area in at least one eye on 2 consecutive visits within 2 months
** ≥ 5-letter improvement on 2 consecutive visits within 2 months
***Subject with most marked pattern of VF treatment response observed; not representative of all treated patients.
***
10. Adverse Events
Most Common
Dose dependent
Transient/ Reversible
Headache
Photophobia
Increased serum
cholesterol & triglycerides
Increased ALT and AST
2 Serious Adverse Events
1) Hypersensitivity
Reaction
2) Elevated Intracranial
Pressure
Reversible with treatment
10
Safety - Consistent with Retinoid Class
144 people treated to date
13. New Products in Sight
Why Bother?
(at least for RPE65 mutations)
13
14. Zuretinol Acetate Development
Potential for more tools in your doctor’s tool box
Oral Delivery – no surgery necessary
Drug delivered to enter retina of both eyes
Data suggests reversible effects upon cessation
of drug
May be merit for study of potential combination
with gene therapy approaches
14
15. Acknowledgements
15
RET IRD 01 / 02
Montreal Children’s Hospital, McGill
University Health Centre, Montreal,
Canada – Dr. Robert K. Koenekoop
Chicago Lighthouse, Chicago, IL –
Dr. Gerald A. Fishman
Scheie Eye Institute, University of
Pennsylvania, Philadelphia, PA –
Dr. Samuel G. Jacobson
Wilmer Eye Institute, Johns Hopkins
University, Baltimore, MD –
Dr. Hendrik Scholl
Moorfields Eye Hospital, London, UK –
Dr. Anthony T. Moore
Institute for Ophthalmic Research,
Tubingen, Germany –
Dr. Eberhart Zrenner
Rotterdam Eye Hospital, Rotterdam,
Netherlands –
Dr. L. Ingeborgh van den Born
RET RP 01
Montreal Children’s Hospital, McGill
University Health Centre, Montreal, Canada
– Dr, Robert K. Koenekoop
Royal Victoria Eye and Ear Hospital,
Dublin, Ireland – Dr. Paul Kenna
RET NAT 01
9 international sites (US, Canada, Europe)
QLT Inc.
Zuretinol Acetate Program Sponsor
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