8:00 am Registration
8:45 am Chair’s Opening Remarks
Developing Clinically Validated Novel Candidate Endpoints for Dry AMD, iAMD & GA
9:00 am Targeting Lipids in Early & Intermediate AMD by the Oil Spill Strategies
9:30 am Spotlight on The Prima Bionic Vision System: Improving Visual Acuity in Patients with Advanced Dry AMD
Synopsis
- Take a look at the work Pixium Vision, a bioelectronics company specialized in neuromodulation are doing to create a world of bionic vision for those who have lost their sight
- Understand PRIMA a brain machine interface with a retinal implant which helps patient suffering from dry AMD regain artificial visual perceptions
- Understand how entering in the pivotal study, and demonstrating promising clinical data, shows how the Prima device allows simultaneous Perception of Prosthetic and Natural Vision
- Explore patients demonstrating Landolt acuity of 1.17 ± 0.13 pixels and with electronic magnification of up to a factor of 8, demonstrate acuity in the range of 20/63 − 20/98
10:00 am Defining Visual Impairment and Clinically Relevant Endpoints in Intermediate AMD: The MACUSTAR Project
Synopsis
- Identify hurdles for clinical development in intermediate AMD
- Learn about MACUSTAR, an IMI (Innovative Medicines Initiative) project aiming to characterize visual functional deficits, structural changes and longitudinal progression in early and intermediate AMD
- Discuss the industry perspective on the MACUSTAR project and its potential to support regulatory evaluations and patient access in intermediate AMD
10:30 am Virtual Speed Networking
Synopsis
Grab a coffee from the comfort of your own kitchen and jump into this 1:1 networking session to make new connections, exchange virtual business cards and maybe even see a friendly face or two!
Preclinical & Translational Development
De-risking Future Drug Candidates: Eliminating Obstacles with Preclinical Animal & In-Vitro Models
11:00 am Overcome Challenges in Developing In-Vivo Models for Dry-AMD
Synopsis
- Explore how the development of dry-AMD therapy is hampered by a lack of translatable animal models
- Since AMD involves the macula, discuss how one major challenge of relying on animal models is lacking in this feature
- Examine how relevant aspects of human disease can be recapitulated in an animal model and may provide a path forward
11:30 am Discuss a Novel Animal Model of GA that is Translatable to Human Disease
Synopsis
- Hear a brief background of the model as delivered using custom optics
- Explore structural, functional, and behavioral/psychophysics data as compared with early-stage AMD patients
- Examine the advantages of this approach to evaluate potential therapeutics to treat AMD/GA
Reduce Toxic By-Product Accumulation: Reduce DHA peroxidation
12:00 pm Breaking the Vicious Circle of AMD Progression with Oxidation-Resistant DHA
Synopsis
- Explore how retinal OSE proliferation can turn the idling alternative complement pathway into a pathologic response as demonstrated by the impaired ability of the Factor H AMD risk variant CFH 402H to neutralize retinal OSEs
- Examine a custom-designed allylic deuteriumreinforced analogue of DHA (D-DHA) which represents a novel approach to preventing retinal LPO and its downstream effects on AMD progression
- Discuss the specific advantages of D-DHA for treating dry AMD (oral delivery, preclinical safety, target engagement and pleiotropic mechanism of action)
Clinical Development
Photoreceptor & RPE Loss: Stem Cell-Based Therapies Learnings & Updates from Clinical Case Studies
11:00 am A Further Spotlight on OpRegen – A RPE Transplant for Dry AMD with GA
Synopsis
- Reiterative how a transplant of RPE cells can be both robust and endured by patients with dry-AMD
- Understand further the benefits of OpRegen RPE cells in specific patients with dry-AMD, as well as primary indication of RPE-photoreceptor complex regeneration (GA reduction)
- Evaluate how RPE cell suspensions can be reasonably scaled to marketable quantities
11:30 am Spotlight on Phase 1/2a Clinical Trial of a Bio-Engineered Implant for GA
Synopsis
- Analyse the design of implant as a tissue replacement therapy
- Understand the delivery of the implant to the subretinal space
- Take a look at phase 1/2a clinical trial design and results
- Examine the next steps in clinical development
Visual Cycle Modulation
12:00 pm Targeting the Visual Cycle Through Vitamin A Modification
Synopsis
- Dry AMD treatment with minerals, antioxidants, and other natural compounds.
- Current challenges and therapeutic approaches for the treatment of
- Geographic Atrophy: drug delivery, animal models, study designs
- Visual cycle: to slow or not to slow? Use of ALK-001 as vitamin A replacement therapy for the treatment of dryAMD or Geographic Atrophy
- Leonide Saad, President & Chief
12:30 pm Networking Lunch
Examine Novel Approaches: Inflammasome Inhibition & HtrA1 Inhibitor
1:30 pm On the Horizon – HtrA1 Inhibition in the Treatment of GA
Synopsis
- Strong human genetic evidence associates ocular HtrA1 overexpression with geographic atrophy and all neovascular forms of AMD
- Growing understanding of how HtrA1 substrates and products play important roles in the pathogenesis of AMD
- Compelling preclinical and clinical evidence for role of HtrA1 in AMD
- HtrA1 pathway is non-overlapping with VEGF and is independent of complement risk factors
2:00 pm Spotlight on GR39821- Phase 1 Single & Multiple-Dose Safety Study of Anti-High Temperature Requirement A1 (HtrA1) in GA
Synopsis
- Therapeutic Hypothesis for Targeting HtrA1
- Review of GR39821 Phase 1 open-label, multicenter, single-and multiple-dose study of anti-HtrA1 in patients with GA secondary to AMD
- Review ocular specific enzyme activity-based antiHtra1 PD assay
2:30 pm How do Inflammasomes, Key Molecular Regulators, Play a Vital Role in Dry AMD?
Synopsis
- Examine inflammasome activators in dry-AMD
- Explore inflammasome activation inhibitors in pre-clinical models
- Evaluate novel, recent clinical data
- Discuss the therapeutic strategy for inflammasome inhibition in dry-AMD
Examining the Growing Need for Validated Biomarkers & Endpoints for Dry-AMD Clinical Research & Trials
1:30 pm Optimize Novel Imaging Biomarkers & Endpoints for Dry AMD Trials
Synopsis
- Review current FAF and OCT biomarkers for AMD progression
- Discuss endpoints for assessing efficacy in current GA trials
- Explore novel OCT-A biomarkers for use in clinical research and clinical trials
2:00 pm Enhance Novel Clinical Trial Endpoints for Trials in GA
Synopsis
- Examine Structural versus Functional endpoints in GA Trials
- Review current endpoints for registration
- Explore the role of microperimetry as an emerging endpoint
- Analyze novel endpoints for clinical trials in GA
- Discuss the use of artificial intelligence in GA endpoints of the future
2:30 pm Opportunities for Personalized Medicine – Automated Risk Classification for Progression in AMD & Utilizing Machine Learning Enhanced OCT Feature Extraction
Synopsis
- Explore OCT-based biomarkers linked to AMD progression to subfoveal geographic atrophy
- Discuss the potential automated solutions for image interrogation and characterization for quantification of these biomarkers
- Examine the potential risk classification system using these imaging biomarkers for clinical trial enrichment, treatment decision-making, and prognostication
3:00 pm Afternoon Networking Break
3:30 pm Panel Discussion & Presentation: The Future Landscape of Dry-AMD Therapies
Synopsis
- Further analysis of the treatment landscape for Dry-AMD/GA – What’s on the horizon?
- Examine patient treatment journey with the emerging new treatment landscape
- Examine how clinicians/developers can support and deal with treatment burden
- Explore clinical trial development and the continued impact of the pandemic on on-going clinical trials