8:00 am Registration
8:45 am Chair’s Opening Remarks
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
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
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
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
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
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
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
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
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