7:50 am Chairs Opening Remarks

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

8:00 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

8:30 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

9:00 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

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

9:30 am Baseline Characteristics in the Phase 2 GOLDEN Study of IONIS-FB-LRx, an Investigational Antisense Oligonucleotide to Treat AMD-Associated Geographic Atrophy

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


  • Assess GOLDEN baseline study characteristics
  • Infer GA growth rates – dependence on size, location and focality
  • Informing future trials

10:00 am Morning Break

11:00 am In Vitro & In Vivo Preclinical Studies of a Modifier Gene Therapy Candidate for Treating 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

11:30 am A Novel Approach Toward Treating GA: Small Molecular for Endogenous Regeneration of RPE Cells


  • The therapeutic concept: inducing endogenous regeneration
  • In vitro GA models for testing the regenerative effect of potential therapeutics
  • In vivo POC: using clinically accepted endpoints in animal studies to determine MoA and efficacy

12:00 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

12:30 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

1:00 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

1:30 pm Networking Lunch Break

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

2:30 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?

3:15 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

3:45 pm Panel Discussion – Reviewing Efficacy of Administration Routes for Different Therapeutic Modalities Targeting Dry AMD & Geographic Atrophy


  • Is subretinal a viable route for the delivery of therapeutics for dry AMD?
  • Surgical complications associated with intravitreal and subretinal deliveries
  • Moving towards outpatient delivery for better treatment turnover

4:15 pm Roundtables: 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

End of Conference Day Two