8:00 am Registration

8:45 am Chair’s Opening Remarks

  • Chad Jackson Director, Preclinical Translational Research Program, Foundation Fighting Blindness

An Overview of the Current Treatment Landscape: What Do We Already Know & What is on the Horizon?

9:00 am Opening Keynote: An Analysis of the Current Landscape of Dry-AMD Therapeutic Development – How to Overcome Current Regulatory & Clinical Hurdles to Moving Promising Agents to the Clinic

  • Peter Kaiser Professor of Ophthalmology, Cleveland Clinic Lerner College of Medicine

Synopsis

  • Summary of the major therapeutic strategies currently under development
  • What are the key barriers to progress?
  • What does the future of Dry AMD Therapeutic development look like?

Therapeutic Opportunity & Latest Clinical Results from Targeting Inflammatory Pathways in Dry AMD & GA

9:30 am Spotlight on Zimura – A C5 Inhibitor for GA

  • Dhaval Desai Senior Vice-President & Chief Development Officer, IVERIC Bio

Synopsis

  • Examine Zimura, a high-affinity, high-specificity pegylated RNA aptamer that binds human C5
  • Learn how prespecified primary efficacy endpoint was achieved for reduction in geographic atrophy (GA) growth rate vs. sham @ 12 months
  • Explore how Zimura was well tolerated over 18 months

10:00 am The Role of Complement in Dry-AMD

Synopsis

  • Review the role of complement, specifically C3, in the pathology of dry-AMD
  • Advance the targeted C3 therapy for the treatment of geographic Atrophy
  • An update from the phase 3 clinical studies of pegcetacoplan in dry-AMD

10:30 am Panel Discussion: Emerging Treatments for Dry AMD & GA – Therapeutic Avenues, Clinical Trials & Future Directions

Synopsis

  • Examine the current treatment options and emerging targeted therapies
  • If targeted therapies show limited efficacy as single agents, can the combination of several targeted
    therapies be of benefit to Dry-AMD patients?
  • How can the industry design novel therapeutic strategies for the treatment of dry-AMD?

11:15 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 | Potential of Complement Therapeutics – What is Next in Human Clinical Drug Candidates?

Novel Areas of Research & Development: Advancing Early Stage & Translational Studies

11:45 am Spotlight on CB 2782-PEG – A Complement Factor C3- Inactivating Protease for Dry AMD

Synopsis

  • Discuss the pre-clinical development and evaluation of CB 2782-PEG
  • Examine Molecular design of CB 2782-PEG
  • Evaluate the advantage of protease therapeutics to regulate biological cascades and processes

12:15 pm Optimize Macrophage Modulation for Late Nonexudative Dry AMD

Synopsis

  • Examine how macrophages play a critical role in the response to tissue stress and are highly “plastic” in their behaviour
  • Explore the dampening of the pro-inflammatory M1 response reduces down-stream mediators including cytokines, complement and the inflammasome
  • Analyze transcriptional modulation with TMi-018 dose-dependently reduces GA-like onset & expansion in pre-clinical testing
  • Showcasing how as a small molecule, TMi-018 is demonstrated suitable for extended delivery

Clinical Development | Clinical Promise of Next-Generation Complement Therapeutics

Targeting Inflammation Through the Complement System: Learnings & Updates from Key Clinical Case Studies

11:45 am Spotlight on ANX007 – Inhibition of C1q for GA

  • Donald Fong Vice President, Ophthalmology Clinical Development, Annexon Biosciences

Synopsis

  • Discuss rationale for chronic classical complement activation as a driver of GA
  • Assess potential advantages of selective, local inhibition of C1q for regulating complement activity in GA
  • Review Phase 1 safety and target engagement with ANX007
  • Provide update on ARCHER, the ongoing Phase 2 study of ANX007 in GA

12:15 pm Spotlight on IONIS-FB-LRx – Systemic Administration of a RNA-Targeting Therapeutic for GA

  • Michael McCaleb Vice President, Clinical Development, & Head of Ophthalmology Disease Franchise, Ionis Pharmaceuticals

Synopsis

  • Discuss the novel approach for addressing systemic etiologies of GA
  • Examine the clinical utility of Antisense (ASO) therapeutics for common and rare diseases
  • Analyses of complement regulation in Phase 1 studies with the factor B ASO
  • Update on ongoing Ph 2 GA trial, the GOLDEN Study

12:45 pm Networking Lunch

1:45 pm Leverage CFH Gene Therapy for Dry AMD

  • Sue Washer President & CEO, Applied Genetic Technologies Corporation (AGTC)

Synopsis

  • Examine the relevance of complement factor H in dry macular degeneration
  • Discuss vector construct optimization
  • Showcasing new pre-clinical data for the treatment of dry-AMD

Suprachoroidal Delivery of Gene Therapy for Dry-AMD

2:15 pm Enhanced Surgical & Office Based Delivery of Gene Therapy

  • Allen Ho Wills Eye Hospital Attending Surgeon, Director of Retina Research and President, The Retina Society

Synopsis

  • Examine standard PPV and retinotomy and how new surgical approaches and hardware may improve consistency and precision
  • Explore suprachoroidal injection with an approved suprachoroidal injection system
  • Evaluate how a suprachoroidal to subretinal microcatheter delivery system procedure avoids retinotomy

2:45 pm Testing a Novel Therapeutic Approach in Two Rat Models with GA Features

  • Malia Edwards Assistant Professor, Wilmer Eye Institute, Johns Hopkins Medicine

Synopsis

  • Describe two different rat models for dry AMD, an acute subretinal NaIO3 model, and our 48/80 model driven by mast cell degranulation
  • Describe Stuart Therapeutics novel treatment compounds and why these models are an ideal  modality to test efficacy and mode of action
  • Investigate how intravitreal injection of PolyColTM peptides affects disease pathology in rat GA model (s)

3:15 pm Spotlight on OCU410- Novel Modifier Gene AMD

Synopsis

  • Discover how modifier gene therapy platform introduces a functional gene to modify the expression of many genes, gene-networks, and regulate basic biological processes in the retina
  • Explore AAV delivery platform for retinal delivery of the RORA gene.
  • Examine various genes associated with AMD are regulated by RORA
  • Learn how RORA protein plays an important role in lipid metabolism and inflammation

1:45 pm Spotlight on GEM103 – Restoring Physiologic Complement Activity with Complement Factor H (CFH) for GA

Synopsis

  • Examine how Complement Factor H (CFH) dysfunction is evident in the pathogenesis of dry AMD
  • Explore GEM103, a novel recombinant human CFH in clinical development for the treatment of dry AMD and
    GA
  • Examine phase 2a data showing intravitreal GEM103 dosing results in sustained supraphysiological CFH in aqueous humor with functionality confirmed through reduction in complement activation biomarkers
  • Explore how intravitreal GEM103 shows strong safety profile, well tolerated with minimal inflammation/CNV

Gene Therapy for Dry-AMD

2:15 pm Optimize the Potential for CFI Based Gene Therapy in GA

Synopsis

  • Examine the scientific rationale: Why CFI?
  • Evaluate why gene therapy can work in Geographic Atrophy
  • Explore interim clinical biomarker data
  • Discover future milestones for CFI based gene therapy in GA

Neuroprotection: Repair Mitochondrial Dysfunction/Oxidative Stress

2:45 pm Enhance Mitochondrial Health & Visual Acuity in Dry AMD

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

Synopsis

  • Examine the rationale for mitochondrial targeted therapeutic for dry AMD and visual acuity endpoints in dry AMD
  • Investigate how phase 1 data demonstrates improved visual acuity with 6 mos. elamipretide therapy
  • Analyze Quantitative Compartmental OCT Analysis may predict response from baseline parameters of mitochondrial RPE health
  • Evelyne phase 2 trial design and baseline characteristics

3:15 pm Progress on the Development of Photobiomodulation (PBM) for the Treatment of Ocular Damage & Disease

Synopsis

  • Examine mitochondrial Mechanisms of Action of PBM
  • Explore multiwavelength Valeda Light Delivery System
  • Understand Valeda Clinical Trial Update for Dry AMD

3:45 pm Virtual Networking & Scientific Poster Sessions

Synopsis

  • Explore innovative research and success stories in mitochondria targeted therapies, directly from your peers
  • Visit the Live Demo Area & Virtual Exhibition Hall

Neuroprotection: Repair Mitochondrial Dysfunction/Oxidative Stress

4:15 pm Explore Oxidative Stress Regulating Therapy with Risuteganib in Dry-AMD

Synopsis

  • A Look a pre-clinical studies with risuteganib, a synthetic RGD-class oligopeptide oxidative stress regulator, suggested to protect human RPE cells against oxidative stress-associated cellular dysfunction
  • Learn how collectively, preclinical studies have indicated the retinal cytoprotective, anti-inflammatory, mitochondrial stabilization and antiangiogenic properties of risuteganib in in vitro and in vivo models
  • Examine how in a Phase 2 clinical trial evaluating risuteganib compared to sham, risuteganib met its primary endpoint of proportion of patients gaining >8 ETDRS letters in patients with less advanced dry AMD (48% vs 7%, p=0.013), and had a good safety profile
  • Understand how a larger Phase 2b/3 study is planned to confirm the Phase 2
    study’s findings

4:45 pm Panel Discussion: With Unprecedented Phase III Progress & Expected Regulatory Feedback on Submissions, What Direction is Provided for the Dry AMD/GA Field?

Synopsis

  • How can drug developers cut to the real barriers, and debate what is truly required to break through to
    successful treatments for dry-AMD?
  • Discuss how slow disease progression and a less than ideal endpoint measurement method lead to
    clinical trials that are necessarily large, lengthy and expensive
  • Examine how innovations in clinical trials with novel endpoints, non-traditional study designs and the
    use of surrogate diseases, developers can reduce the patient sample size and consequently lower the
    budget of clinical trials

5:30 pm Chair’s Closing Remarks

  • Chad Jackson Director, Preclinical Translational Research Program, Foundation Fighting Blindness

5:45 pm End of Day 1