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Leader in Gene Therapy for the Eye!
Thomas W. Chalberg, Ph.D. | Founder & CEO!
Forward Looking Statements!This presentation may contain forward-looking statements that are based on our current expectations, estimates and projections about our industry as well as management's beliefs and assumptions. Words such as "anticipates," "expects," "intends," "plans," "believes," "seeks,” "estimates," "may," "will," and variations of these words or similar expressions are intended to identify forward-looking statements. These statements include projections about the growth of the market for aesthetic procedures, and speak only as of the date hereof. Such statements are based upon the information available to us now and are subject to change. We will not necessarily inform you of such changes.!
These statements are not guarantees of future performance and are subject to certain risks, uncertainties and assumptions that are difficult to predict. Therefore actual results could differ materially and adversely from those expressed in any forward-looking statements as a result of various factors. Factors which could cause actual results to differ materially from those in the forward-looking statements include, among others, a downturn or leveling off of demand for aesthetic procedures, the availability of competitive alternatives to our products, domestic or international economic uncertainty, and other events beyond our control.!
Ocular BioFactory™ is a trademark of Avalanche Biotechnologies, Inc.!
CONFIDENTIAL! 2!
Company Highlights !
• Potential one-time transformative treatments for sight-threatening major and orphan diseases in ophthalmology!
• Ocular BioFactory™: integrated gene therapy discovery, development and manufacturing platform!
• AVA-101: Clinical proof-of-concept for wet AMD!
• Product engine with pipeline and deal potential including Regeneron collaboration!
• Industry-leading team in gene therapy and ophthalmology!
CONFIDENTIAL! 3!
Avalanche’s Ocular BioFactoryTM Enables Long-Term Protein Delivery to the Eye!
CONFIDENTIAL! 4!
The Ocular BioFactoryTM secretes therapeutic protein for years !following a single administration!
Therapeutic DNA! Adeno-associated Vector!(AAV)!
Long-term Therapeutic Protein Expression!
Novel Vectors Penetrate Retina, Overcoming Delivery Limitations of Naturally Occurring AAVs!
CONFIDENTIAL! 5!
Deliver Payload Specific to Treat Different Diseases!AAVs Engineered for Specific Cell Types!
Glaucoma!!
Retinal Degenerations (Optogenetics)!
Diabetic Retinopathy!!
Macular Telangiectasia!
XLRS!!
Forms of Retinitis Pigmentosa!
Wet AMD!!Geographic Atrophy / Dry AMD!!Forms of Retinitis Pigmentosa!
Discovery & ManufacturingExpertise in Gene Therapy! Development & Commercialization Expertise
in Ophthalmology!Ocular BioFactoryTM
Platform!
Layers of !the Retina!
Ganglion Cell Layer!
Inner Nuclear Layer!
Outer Nuclear Layer!
Pigmented Epithelium!
Inner Plexiform Layer!
Outer Plexiform Layer!
Outer Segments!
Inner Segments!
Nerve Fiber Layer!
Choroid!
AVA-101: Product Overview!
CONFIDENTIAL! 6!
Potential for One- Time Transformative Treatment!
Promising Clinical Data!
Significant Market Opportunity!
Progress!
• One-time, subretinal injection offers “functional cure” of wet AMD !
• AAV2 vector containing gene encoding sFlt-1, a naturally occurring VEGF inhibitor, administered directly to retina cells!
• Well tolerated with no drug-related adverse events!
• Subjects gained/maintained vision with no or minimal need for additional treatment over one year!
• Wet AMD is a leading cause of vision loss that affects three million people worldwide with over $6B sales !
• Compliance with existing treatments is challenging and longer-lasting treatment is a major unmet need!
• Phase 2a trial fully enrolled in Australia; data expected mid-2015!
• Phase 2b in the U.S. planned for 2H-2015!
Poor Compliance Leads to Progressive Vision Loss!
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-‐-‐-‐-‐-‐-‐-‐ Mean combined results of MARINA and ANCHOR studies (Genentech) during which paAents received anA-‐VEGF injecAons every four weeks. -‐-‐-‐-‐-‐-‐-‐-‐-‐ Mean results of the studies indicated during which paAents received anA-‐VEGF injecAons less frequently than every 4-‐8 weeks as described above. All trials were conducted by third parAes.
More VEGF suppression is better (multiple large, Phase 3 studies)!
CONFIDENTIAL!
AVA-101 Upregulates a Naturally-Occurring VEGF Inhibitor Implicated in AMD!
CONFIDENTIAL! † Wiesmann et al. Cell, 1997; 91: 695.! 8!
Very high affinity (10 pM)binding neutralizes VEGF-A†!
sFLT-1: Naturally occurring endogenous VEGF inhibitor!
Well-characterized binding to clinically validated target! Synthetic fusion constructs D2 with IgG1 Fc!
N!
2!S-S!
Binding domain (D2)!
1!S-S!
2!S-S!
3!S-S!
4! 5!S-S!
6!S-S!
Majority of AVA-101 Treated Patients Did Not Require Any Rescue Treatment in Year 1!
CONFIDENTIAL!
• Treated subjects needed only 0.33 injections out of 12 opportunities!• Statistically significant versus control group and historical controls (p < 0.001)!
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Central Retinal Thickness Improved and Maintained Through Month 12!
Spectralis HD-OCT: !• Non-invasive imaging allows measurement of central retinal thickness!• Surrogate endpoint to measure disease activity!
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Best-Corrected Visual Acuity!• Treated subjects gained visual acuity from baseline!• Low dose AVA-101 demonstrated most significant change from baseline !
CONFIDENTIAL ! 11!
Case Study: R1002 - 24 Previous Injections!
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Avalanche Product Pipeline!
• Active R&D programs to expand pipeline!
CONFIDENTIAL! 13!
Product Candidate ! Indication!
Stage of Development!
Near-Term Milestones ! Worldwide Commercial Rights!Research! Preclinical! Phase 1/2!
AVA-101! Wet AMD!
Top-line phase 2a data expected mid-2015 !IND filing 2H 2015!
Avalanche!
AVA-101! DME, RVO! IND-enabling studies planned for 2014-2015! Avalanche!
AVA-201! Wet AMD!(prevention)! Preclinical studies in 2014-2015! Avalanche!
AVA-311! XLRS!
Regeneron;!Avalanche receives milestones and royalties and has an option to share development costs and profits!
Regeneron Collaboration (May 2014)!• Collaboration Highlights:!
– Covers up to eight distinct therapeutic targets!– Avalanche option to share up to 35% on profits/
development costs for 2 targets!– Regeneron has a time-limited right of first negotiation
for certain rights to AVA-101 !
• Financial Highlights:!– $8M upfront payment !– Up to $640M in milestones, plus royalties!– Reimbursement of research costs!
CONFIDENTIAL!
Ocular BioFactoryTM &Gene Therapy Expertise!
Proprietary Molecules & Ophthalmology Expertise!
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Management Team!
Thomas W. Chalberg, Ph.D.!Founder & CEO!
Linda C. Bain!Chief Financial Officer!
Samuel B. Barone, M.D.!Chief Medical Officer!
Hans P. Hull!VP Legal & Corporate Development!
Mehdi Gasmi, Ph.D.!VP Pharmaceutical Development!
Roman G. Rubio, M.D.!SVP & Head of Translational Medicine!
Building a World-Class Ophthalmology Team!
Top-tier Board of Directors, Clinical and Scientific Advisory Board!CONFIDENTIAL! 15!
Management Team!
Company Highlights !
• Potential one-time transformative treatments for sight-threatening major and orphan diseases in ophthalmology!
• Ocular BioFactory™: integrated gene therapy discovery, development and manufacturing platform!
• AVA-101: Clinical proof-of-concept for wet AMD!
• Product engine with pipeline and deal potential including Regeneron collaboration!
• Industry-leading team in gene therapy and ophthalmology!
CONFIDENTIAL! 16!
www.avalanchebiotech.com!
