7:00 am Registration & Morning Coffee

7:40 am Chair’s Opening Remarks

Discovering the Next Generation of Biomarkers for Effective Translation of Preclinical Dry AMD Models to the Clinic

7:50 am Translation to the Clinic: Role of Preclinical Models & Biomarkers to Optimize Dry AMD Clinical Trials

  • Daniel Chao Senior Director & Clinical Lead, Translational Medicine, Janssen


  • Translatability and considerations of preclinical models for dry AMD
  • Lessons learned from the clinic – how can we maximally de-risk preclinical assets prior to initiation of clinical trials?
  • Role of molecular and imaging biomarkers for early POC clinical trials

8:20 am Reviewing the Key Lessons Learned from Phase 2 GA Trials

  • Stephen Poor Director of Translational Biomarkers at NIBR, Novartis


  • Review of lessons learned from our IVT complement Factor P inhibitor GA Phase 2 trial
  • Data on how geographic atrophy is measured – the importance of patient level data vs summed data
  • Fundus autofluorescence measure vs OCT measurements – functional outcomes including chart-based tests vs microperimetry

8:50 am Identification of DKK3 as an Ocular Pharmacodynamic Biomarker for Anti-HtrA1 in a Clinical Trial for Geographic Atrophy Secondary to Agerelated Macular Degeneration

  • Hao Chen Principal Clinical Scientist, Genentech


  • Identification of HtrA1 substrates enabled the development of a clinical PD biomarker for evaluating anti-HtrA1 Fab activity
  • Enzyme activity–based PD assay (reduction in cleaved-DKK3 fragments) suggests potential for 8-week target inhibition at higher doses
  • Analysis of cleaved DKK3 in patient aqueous humor provided evidence of anti-HtrA1 activity and information on duration of activity in phase 1

9:20 am Dry AMD Therapeutics, iPSC to the Rescue


  • Patient-derived RPE cells and unique phenotypic assays
  • Linking genetics to RPE cells response to AMD stress
  • High-throughput platform to screen new neuroprotective agents with no a
    priori on targets

9:30 am How Vision is Impaired from Aging to Intermediate AMD: Insights from ALSTAR2 Baseline

  • Cynthia Owsley Director of Clinical Research Unit & Professor, University of Alabama at Birmingham


  • Structure/function endpoints for AMD should be motivated by the pathobiology of the disease. RMDA has a strong history in outer retina neurophysiology and psychophysics
  • Using z-scores to standardize vision measures – greatest difference between visual function in normal and intermediate AMD eyes
  • Delayed rod-mediated dark adaptation in normal eyes doubles the risk for incident AMD

10:00 am A Complement Alternative Pathway Inhibitor Delivered by Gene Therapy in Mouse Models of Age-Related Macular Degeneration

  • Baerbel Rohrer Endowed Chair, Gene & Pharmaceutical Treatment of Retinal Degenerative Disease, Medical University of South Carolina


  • Discover how the eye generates its own complement
    microenvironment, which is composed of RPE/choroid-derived
    proteins and those delivered by the liver
    (circulation), and set up in part by differential permeability
    of Bruch’s Membrane to complement proteins
  • Complement activation appears to occur outside of the
    Blood Retina Barrier (basal side of RPE, BrM and CC)
  • As the goal is to identify a treatment for dry AMD that
    can be used early in disease, to prevent conversion
    from early to intermediate AMD (i.e., in the presence
    of a presumably intact BRB), how do we deliver the
    therapeutics accordingly?

10:30 am Data Solutions for Imaging Biomarker Discovery and Clinical Endpoint Validation

  • Eric Buckland Chief Executive Officer, Translational Imaging Innovations


  • Meaningful prognostic biomarkers and clinical outcome measures are essential
    to the development of treatments for degenerative retinal disease
  • Biomarkers and endpoints are not born, they are discovered and validated
  • Clinical standards of care are often insufficient, and must be supported with
    emerging imaging modalities and analyses
  • TII integrates clinical and research data, data flow automations, and advanced
    analytics to accelerate the discovery and validation of biomarkers and endpoints

11:00 am Round-Table Discussion: Discussing Regulatory Challenges Across Different Stages of Dry AMD Therapeutics Development


Some of the biggest challenges when developing drug for dry AMD is understanding the regulatory landscape for all stages of drug development. This session facilitates discussions on a range of regulatory checkpoints including:

  • Pre-IND
  • Endpoint Designation
  • Recruiting & Phase I
  • Phase II & Phase III

11:30 am Morning Break

Reviewing Therapeutic Modalities for Dry AMD & Geographic Atrophy to Fully Gauge the Treatment Landscape

12:00 pm Phase 2 Trial of ALK-001 in Stargardt Disease and Treatment Indications for Dry AMD/GA

  • Leonide Saad Chief Executive Officer, Alkeus Pharmaceuticals


  • ALK-001 is a first in class inhibitor of vitamin A dimerization
  • Results of a randomized, placebo-controlled, clinical trial indicates that
  • ALK-001 has disease modifying ability on the progression of Stargardt Disease
  • ALK-001 is also being tested in Geographic Atrophy associated with dry-AMD in a fully enrolled Phase 3 study.

12:30 pm IONIS-FB-LRx, an Investigational Antisense Oligonucleotide to Treat AMD Associated Geographic Atrophy

  • Maria Khan Clinical Development Lead for FB-LRX, Ionis Pharmaceuticals


  • Developing a non-invasive therapeutic option with novel mechanism of action
    targeting complement Factor B (FB)
  • Preclinical and clinical data demonstrating reduction in systemic FB and
    excellent safety profile
  • Phase 2 clinical trial (GOLDEN) baseline study characteristics

1:00 pm A Modifier Gene Therapy Approach for Treatment of Dry-AMD


  • Dry-AMD disease pathophysiology and risk factors
  • Modifier gene therapy, an innovative therapeutic approach for treatment of complex multifactorial diseases
  • In vitro and In vivo preclinical studies demonstrating potential of OCU410 gene therapy for dry-AMD

1:30 pm Lunch Break

2:30 pm Small molecule-driven endogenous regeneration of RPE cells: a novel approach to treating Geographic Atrophy


  • The therapeutic concept: inducing endogenous regeneration
  • In vitro GA models for testing the regenerative effect of potential therapeutics
  • In vivo POC: lessons learned from different animal models

3:00 pm Exploring a Novel Approach Using a Glyco-Immune Therapy Targeting Both Complement and Macrophages


  • A discussion of how macrophages/microglia are involved in the pathobiology of AMD
  • What are Siglecs and self-associated molecular patterns and how do they modulate immune responses?
  • A novel glyco-engineered nanoparticle that inhibits over-activation of complement and macrophages

3:30 pm ReCLAIM-2: Phase 2 Trial of Subcutaneous Elamipretide in Patients with Noncentral Geographic Atrophy

  • Brian Hotchkiss Executive Vice President of Ophthalmology, Stealth Biotherapeutics


  • Elamipretide is a first in class mitochondrial restorative compound
  • Review results of phase 2 trial demonstrating positive secondary endpoints and possible path to phase 3 enrichment
  • Cover ellipsoid zone endpoint and how it relates to mitochondrial health

4:00 pm Afternoon Break

4:30 pm Targeting the Sigma-2 Receptor (S2R) for Dry AMD with an Oral Small Molecule Approach: Preclinical & Clinical Biomarker Support

  • Mary Hamby Vice President of Research, Cognition Therapeutics


  • Background on target and rationale for dry AMD
  • Preclinical data support a unique mechanism of action to restore RPE homeostatic function
  • Proteomic biomarker evidence that the S2R modulator CT1812 can alter dry AMD relevant protein and pathways in an aged population

Understanding the Patient Perception in the Clinic & Appropriate Delivery Modalities to Better Understand Clinical Processes

5:00 pm Hearing Patient Reception of Long Term & Invasive Treatments for Dry AMD to Understand Patient Experience


  • Viewing patient testimonies to understand key desires and thoughts of the recipients
  • What is the patient reception of current clinical trials?
  • How can the industry meet the needs of their patients?

5:30 pm Routine Suprachoroidal Drug Administration: the Role in Intermediate AMD/GA Therapies


  • Choroidal administration of therapies in intermediate AMD/GA
  • Novel principle of targeted routine drug suprachoroidal administration
  • Case studies: Use of the suprachoroidal Oxulumis® illuminated microcatheter in back-of-the-eye disorders

6:00 pm Chair’s Closing Remarks

End of Conference Day Two