Visionary Science for Life Changing Cures OIS 2017

Transcription

1 Visionary Science for Life Changing Cures OIS

2 Forward Looking Statements Today s presentation includes forward-looking statements intended to qualify for the Safe Harbor from liability established by the Private Securities Litigation Reform Act of These forward-looking statements, including statements regarding our planned pre-clinical and clinical studies, timing or ability to close partnerships, regulatory approval process and demand for our product candidates, are subject to risks, uncertainties and other factors that could cause actual results to differ materially from those suggested by our forward-looking statements. These factors include, but are not limited to, the following: We have incurred significant losses since inception and anticipate that we will continue to incur significant losses for the foreseeable future. Our ability to generate revenue from product sales is highly uncertain. We may need to raise additional funding in the future, which may not be available on acceptable terms, or at all. No gene therapy products have been approved in the United States, and we may not be able to obtain regulatory approvals for our product candidates. We have encountered and may continue to encounter substantial delays in our clinical trials or fail to demonstrate safety and efficacy to the satisfaction of applicable regulatory authorities. We rely on third parties to conduct, supervise and monitor our clinical trials and to conduct certain aspects of our product manufacturing and protocol development. The insurance coverage and reimbursement status of our product candidates is uncertain. Negative public opinion and increased regulatory scrutiny of gene therapy and genetic research may adversely affect public perception of our product candidates and prospects for our business. If we are unable to obtain and maintain adequate patent protection for our technology and products our competitors could develop and commercialize technology and products similar or identical to ours. 2

3 Company Highlights AGTC is developing genetic therapies for patients with inherited diseases. Treatments are precisely designed to meet the needs of each specific genetic disorder. Clear Vision Become the leader in ophthalmology gene therapy Deep Expertise Extensive IP Portfolio Broad Pipeline In vector selection, design, manufacturing and delivery >100 patents/applications covering genes, capsids, process, delivery Multiple opportunities to provide long-term value to patients Key Partnership Broad collaboration with Biogen for multiple indications 3

4 4 Lead Product Pipeline Multiple Shots on Goal

5 The Eye The Retina - a highly organized tissue Iris Pupil Lens Cornea Retinal pigment epithelial cell Vitreous humour Rod cell Cone cell Retina Mueller cell Horizontal cell Rod diseases Bipolar cell Rod diseases Amacrine cell Fovea Optic nerve Cone diseases Ganglion cell Optogenetics Light enters retina 5

6 X-linked Retinoschisis (XLRS) Disease Missing structural protein results in poor vision not correctable with eyeglasses ~35,000 patients in US and EU No current treatments Retinal layers: XLRS eye Retinal layers: normal eye Impact Poor vision (20/100) detected by school age Difficulty reading, driving, and recognizing faces 30% chance of retinal detachment or vitreous hemorrhage at any age Positioned for Success Robust animal models showing potential to improve vision Understanding of human disease phenotype from natural history Accepted clinical endpoints Strong IP position 6

7 XLRS Phase 1 / 2 Key Elements Dose Ranging Low,Med,High 12 Patients Expansion Group at High Dose 9-15 Patients Generally Safe & Well Tolerated Enrollment Complete Q1 Efficacy Read 6 months After Last Patient Dosed Completed Ongoing Established Delivery Intravitreal Injection Primary endpoint: Safety Secondary Endpoints: Visual Acuity; Visual Fields; ERG; OCT and QoL Survey 7

8 Achromatopsia (ACHM) Disease 70% of ACHM is caused by mutations in the A3 and B3 genes. AGTC is currently working on these two genetics mutations that result in severely impaired vision and day blindness Missing cone photoreceptor protein results in poor vision not correctable with eyeglasses ~28,000 patients in US and EU No current treatments Normal Bright light ACHM Impact Extremely poor vision, legally blind Extreme light sensitivity (day blind) Complete loss of color discrimination Positioned for Success Robust animal models showing potential to improve vision Understanding of human disease phenotype from natural history Accepted clinical endpoints Strong IP position Dim light 8

9 ACHM Phase 1 / 2 Key Elements Dose Ranging Low,Med,High 9-11 Patients Expansion Group at High Dose 9-15 Patients Generally Safe & Well Tolerated Enrollment Complete Efficacy Read 6 months after Last Patient Dosed Ongoing: Conducting ACHMB3 and ACHMA3 trials Delivery - Sub-Retinal Injection Primary endpoint - Safety Secondary Endpoints - Visual Acuity, Photophobia, Color Vision, ERG 9

10 X-linked Retinitis Pigmentosa (XLRP) Disease Missing protein results in degeneration of rods and cones ~20,000 patients in US and EU No current treatments Photoreceptor Preservation in Dog Model of XLRP Untreated area Treated area Constriction of Visual Field Photoreceptor Nuclei blue Primate Rod/Cone Targeting Impact Early night blindness, progressive constriction of visual fields Legally blind by age 45 Positioned for Success Robust animal models showing potential to improve vision Primate photoreceptor targeting Understanding of human disease phenotype from natural history Accepted clinical endpoints Strong IP position 10

11 XLRP Study Design Key Elements Dose Ranging Low,Med,High Patients Expansion Group at High Dose 6 Patients Generally Safe & Well Tolerated Enrollment Complete Efficacy Read 6 months after last patient dosed Enrollment in Near Future Delivery - Sub-Retinal Injection Primary Endpoint - Safety Secondary Endpoints - Visual Function (perimetry); Visual Acuity; OCT; QoL Survey 11

12 Bionic Sight: Optogenetics Program The Bionic Sight system bypasses damaged tissue of the input side of the retina and provides direct stimulation to the output cells in order to send visual information to the brain. Normal Photoreceptors Ganglion cells Standard Prosthetic Comp Electronics case Light Image code sent to the brain Light Image code sent to the brain Blind Bionic Sight Light Light No code transmitted Image code sent to the brain Encoder (Transducer) Photoresponsive ganglion cells* 12

13 Experienced Team Sue Washer Chief Executive Officer Matt Feinsod, M.D. Chief Medical Officer Jeff Chulay, M.D. Executive Director of Clinical Strategy Rabia Ozden, M.D. VP Clinical Development Stephen Potter Chief Business Officer Bill Sullivan Chief Financial Officer Mark Shearman, Ph.D. Chief Scientific Officer Andrew Ashe J.D. General Counsel 13

14 Manufacturing Expertise and Capacity Manufacturing strength Suspension system scalable to large volumes Process adaptable to any serotype Three CMO partners Substantial IP position Expertise Successfully transferred to multiple parties 7 successful cgmp batches completed Repeatable consistent yields and characterization Completely integrated process & analytics Analytics advancing to late stage readiness 14

15 Financial Summary Strong Balance Sheet $129.6 Million Cash & Investments as of 9/30/2017 Sufficient Cash to: Complete enrollment and analysis XLRS, ACHMB3, ACHMA3 Phase 1/2 Clinical Trials Initiate and analyze XLRP Phase 1/2 Clinical Trial Move pre-clinical optogenetic program forward In collaboration with Bionic Sight Fund company for at least two years 15

16 Company Highlights AGTC is developing genetic therapies for patients with inherited diseases. Treatments are precisely designed to meet the needs of each specific genetic disorder. Clear Vision Become the leader in ophthalmology gene therapy Deep Expertise Extensive IP Portfolio Broad Pipeline In vector selection, design, manufacturing and delivery >100 patents/applications covering genes, capsids, process, delivery Multiple opportunities to provide long-term value to patients Key Partnership Broad collaboration with Biogen for multiple indications 16

17 Visionary Science for Life Changing Cures