Public Device & Biopharma Ophthalmology Company Showcase - QLT

Public Device & Biopharma Ophthalmology Company Showcase - QLT

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  New Products inSight Potential for Vision Improvement in Inherited Retinal Diseases LCA and RP due to RPE65 and LRAT Mutations Using Oral Zuretinol Acetate (QLT 091001)
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  Forward-looking Statement Certain statementsin 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
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  The Normal VisualCycle all trans retinol LRATRPE 65 11-cis-retinal rhodopsin 3 opsin Vit A from serum Vision Photoreceptor Retinal Pigment Epithelium
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  Mutations in RPE65or LRAT Lead to Vision Loss all trans retinol LRATRPE 65 11-cis-retinal rhodopsin 4 opsin Vit A from serum Vision
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  Oral Zuretinol AcetateReplaces 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
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  Vision Rescue inRPE65 Mutant Dog with Intravitreal Zuretinol Acetate Narfstrom, ARVO 2009
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  IRD01 - Phase1b 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
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  Screening OS ScreeningOD 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
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  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. ***
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  Adverse Events Most Common Dosedependent 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
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  New Products inSight So What’s Next…...?
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  RET IRD 04– Zuretinol Acetate Phase III Trial  Placebo Controlled  Double Masked  Autosomal Recessive Inherited Retinal Disease (RP/LCA)  RPE65 or LRAT Mutations  Multicentered (US, Canada, Brazil, Europe)  Trial initiation planned for H2 2016 12
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  New Products inSight Why Bother? (at least for RPE65 mutations) 13
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  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
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  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  Editorial and presentation assistance
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  New Products inSight Thank You
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