8:00 am Preconference Virtual Networking

8:55 am Chair’s Opening Remarks

9:00 am Setting the Scene: An analysis of the Challenges Facing Dry AMD Therapeutics Developers and patients

  • Jeffrey S. Heier CO-President & Medical Director , Ophthalmic Consultants of Boston

Panel Discussion: Defining the Therapeutic Journey for Dry AMD Patients

9:15 am Topics to be discussed:

Synopsis

• Understanding the continuum of care for preventing, slowing, or reversing dry AMD at various stages of
the disease – where do pharmacological, cell therapies, gene therapy approaches, light therapies and
vision prosthesis play a role in the patient’s therapeutic journey?
• Discussing the potential for sequential or combination regimens depending on stage of disease
• Providing a potential therapeutic journey framework for dry AMD patient populations from early stages,
through to advanced cases with GA secondary to dry AMD or wet AMD

10:00 am Preclinical Models for Drug Discovery in Dry AMD

  • Simon Kaja Chief Scientific Officer & Vice-President, Americas, Experimentica

Synopsis

  • How to choose a preclinical model for proof-of-concept and pharmacologic efficacy studies in dry AMD?
  • What imaging modalities are available to monitor dry AMD pathology in preclinical models?
  • What can structure-function relationships in preclinical models for dry AMD tell us?

Applying Cell Therapy Approaches to Replace RPE Cells and Restore Vision

10:30 am Virtual Speed Networking & Break

11:00 am OpRegen, an RPE Transplant for the Treatment of Dry AMD

11:20 am Development of a Composite RPE Cell-Ultrathin Membrane Implant for the Treatment of the Dry Form of Age-Related Macular Degeneration

Synopsis

• Development of a bioengineered implant as a tissue replacement
therapy for geographic atrophy
• Selection and development of materials and manufacturing method for
the implant
• Delivery and clinical evaluation of the implant

11:40 am Adult RPE Stem Cell Transplantation as Therapy for dry AMD

  • Jeffrey Stern co-Chief Executive Officer , Neural Stem Cell Institute & Luxa Biotechnology

Synopsis

• Adult RPE stem cells (RPESC) can be expanded extensively
• Poised to produce native RPE with stable phenotype
• Progenitor stage RPE progeny enhance engraftment and vision rescue

12:00 pm Live Presenter Q&A

  • Brian M. Culley Chief Executive Officer , Lineage Cell Therapeutics, Inc
  • Jeffrey Stern co-Chief Executive Officer , Neural Stem Cell Institute & Luxa Biotechnology
  • Jane S. Lebkowski President , Regenerative Patch Technologies

Targeting the Complement & Inflammasome Systems in Dry AMD Effectively and Safely

12:15 pm Virtual Networking Break

1:00 pm Neuroprotection in Geographic Atrophy

  • Victor Chong Global Head of Medicine, Retinal Health , Boehringer Ingelheim International GmbH

Synopsis

• There are different types of GA
• Pseudoreticular drusen patient has extensive photoreceptor loss before
RPE loss
• Complement activation might be less important in Oriental races
• Potential keep photoreceptors from dying is the most important goal

1:20 pm Analysis of Complement in of Donor/Post-Mortem Eyes with and Without Age-Related Macular Degeneration (Amd), Implications for Future Intermediate Amd or Geographic Atrophy (Ga) Clinical Trials

  • Stephen Poor Director & Head of Retinal Pharmacology Unit & Imaging Technologies Team , Novartis

Synopsis

• The majority of complement components in both healthy and AMD
donor eyes are in the Retinal pigment epithelium-choroid ( ≥ 10 fold
more than in the retina)
• Complement gene expression is upregulated in atrophic AMD retina,
Iba1 +ve microglia cells are the primary cellular source of complement
• Systemic complement inhibition should be considered for future
intermediate AMD or GA clinical trials

1:40 pm Live Presenter Q&A

  • Victor Chong Global Head of Medicine, Retinal Health , Boehringer Ingelheim International GmbH
  • Stephen Poor Director & Head of Retinal Pharmacology Unit & Imaging Technologies Team , Novartis

1:50 pm Advancing the Targeted C3 Therapy, Pegcetacoplan, for the Treatment of Geographic Atrophy

Synopsis

• Pegcetacoplan targets the complement cascade at C3 to control and
slow the irreversible lesion growth in geographic atrophy (GA)
• Phase 2 study results showed that monthly treatment with
pegcetacoplan resulted in a 29% (p=0.008) reduction in the rate of
GA lesion growth compared to sham injections at 12 months with an
acceptable safety and tolerability profile
• Pegcetacoplan is being studied in two large, randomized global Phase 3
clinical studies

2:10 pm Zimura, Novel Complement C5 Inhibitor, Significantly Reduces the Mean Rate of Geographic Atrophy Growth in a Phase 3 Clinical Trial

Synopsis

• Robust independent imaging and prespecified statistical analysis plan
• Primary efficacy endpoint was achieved for both Zimura 2 mg and Zimura
4 mg dose, leading to a ~27% reduction in GA growth over 12 months
• Reduction in GA growth observed already at 6 months
• Increase in Absolute Difference up to 18 Months
• Sham arm performed as expected
• Both Zimura 2 mg and 4 mg were well tolerated over 18 months
• No Zimura related AE
• No endophthalmitis
• Lower rate of CNV

2:30 pm Re-imagining complement-based therapies for AMD

Synopsis

• Complement inhibition as a means of halting the progression of
geographic atrophy
• Possible Reasons for Failures and Success of Complement Based
Therapies
• Interaction of two key AMD risk factors: complement factor H and
lipoproteins

2:50 pm Live Presenter Q&A

3:05 pm Virtual Networking Break

3:40 pm Elamipretide, a Mitochondrial-Targeted Drug, in the Treatment of Dry AMD

  • Reenie McCarthy Director, President & Chief Executive Officer Stealth BioTherapeutics Inc

Synopsis

  • Elamipretide preclinical data showed reversal of AMD pathophysiology & improvement of vision
  • A phase 1 open label safety study of elamipretide in patients with dry AMD showed improvements in multiple measures of vision
  • A phase 2, randomized, double-masked, placebo-controlled clinical study of elamipreide in patients with dry AMD is on-going.

Assessing Biomarkers, Functional Outcomes & Clinically Relevant Endpoints in Dry AMD

4:00 pm Patient Selection and Monitoring for Efficacy in Dry AMD trials

  • SriniVas Sadda President and Chief Scientific Officer, Doheny Eye Institute; Professor of Ophthalmology, Doheny Eye Institute, UCLA

Synopsis

  • Use of multimodal imaging to identify patients at high-risk for
    progression
  • New biomarkers of AMD progression
  • Design of precision endpoints, tailored to specific therapeutics

4:20 pm Current Efforts to Evaluate Functional Visual Impairment and Clinically Relevant Endpoint Candidates for Intermediate Dry AMD

  • Ulrich Luhmann Biomarker Experimental Medicine Leader, Early Clinical Development , Roche

Synopsis

• Defining functional impairment in early and intermediate AMD in a
longitudinal prospective natural history studies
• An industry perspective on the MACUSTAR initiative – an European
Innovative Medicine Initiative (IMI2) to evaluate and validate clinical
endpoints for intermediate AMD

4:40 pm Establishing Correlations Between Functional outcomes, Imaging- and Blood-Based Biomarkers in Nonexudative AMD: Lessons Learned and Regulatory Implications

  • Eleonora Lad Associate Professor of Ophthalmology, Director of Grading Duke Reading Center, Duke University

Synopsis

  • Structure-function correlations in national history studies of early and intermediate AMD and learnings from other retinal degenerative diseases
  • Blood-based biomarkers of disease progression (genetics, flow cytometry of monocytes, other)
  • Deep learning algorithms to predict progression to geographic atrophy and visual loss on retinal imaging

5:00 pm Imaging in Dry Age-related Macular Degeneration

  • Jacque Duncan Professor, Clinical Ophthalmology, University of California, San Francisco

Synopsis

• New imaging modalities can provide information about retinal structure
and function in patients with dry AMD
• Structural measures correlate with measures of visual function
• Imaging retinal structure and function can be used to monitor disease
progression and response to therapy for patients with dry AMD

5:20 pm Clinical Implementation of an Ultrathin Stem-Cell Derived RPE Monolayer in Phase 1/2a Clinical Trial for Treatment of Advanced Dry AMD with Geographic Atrophy

  • Amir Kashani Associate Professor of Ophthalmology (Clinical Scholar) , University of Southern California

Synopsis

• Review of clinical study design
• Review of methods for surgical delivery of ultrathin implant in subretinal
space and within area of GA
• Review of surgical results and preliminary study results

5:40 pm Live Presenter Q&A

  • Jacque Duncan Professor, Clinical Ophthalmology, University of California, San Francisco
  • Amir Kashani Associate Professor of Ophthalmology (Clinical Scholar) , University of Southern California
  • Ulrich Luhmann Biomarker Experimental Medicine Leader, Early Clinical Development , Roche
  • Eleonora Lad Associate Professor of Ophthalmology, Director of Grading Duke Reading Center, Duke University
  • Reenie McCarthy Director, President & Chief Executive Officer Stealth BioTherapeutics Inc

6:00 pm End of Conference

6:05 pm Chair’s closing remarks