8:30 am Check-In & Coffee + Light Breakfast
9:25 am Chair’s Opening Remarks
DELIVERING TRANSFORMATIVE DRUGS TO THE RETINAL VASCULAR SYSTEM
9:30 am An Overview on Pathways & Outcomes of Wet AMD & Diabetic Retinopathy
Synopsis
- Exploring molecular pathways contributing to diabetic retinopathy and wet AMD to understand variations in treatment efficacy
- Identifying key biomarkers that guide the selection of effective therapies and support combination approaches
- Addressing unmet needs by informing drug development with insights on patients who are unresponsive to current treatments
10:00 am Driving forward Phase 3 DIAMOND trials of OCS-01 eye drops, a topical OPTIREACH formulation of high-concentration dexamethasone for DME
Synopsis
- Highlighting the OPTIREACH formulation technology and associated benefits
- Discussing remaining unmet medical needs for DME patient management
- Exploring the clinical evidence to date on the efficacy and safety of OCS-01 eye drops for DME
10:30 am Morning Break & Refreshments
11:00 am Using an Oral Anti-Inflammatory Drug to Improve Treatment Options for DME & DR Patients Non-Responsive to Anti-VEGF Therapy
Synopsis
- Addressing the unmet need in DR and DME patients receiving anti-VEGF treatment by targeting non-responders with Xiflam, a once-daily oral small molecule that inhibits the pathologically open Connexin43 (Cx43) hemichannel
- Demonstrating positive preclinical results, Xiflam shows promise in modulating the dysregulated inflammasome pathway, reducing autoinflammation in DME and DR
- Exploring the benefits of oral administration over injections, particularly for early-stage disease patients who prefer non-invasive treatment options
11:30 am Drug Delivery Aspects & Device Impacts on Retinal Vascular Treatments
Synopsis
- Optimizing dosing strategies, particularly for gene therapy, to support both physician and patient compliance and improve treatment outcomes
- Refining device aspects to streamline administration and enhance the overall experience for both patients and healthcare providers
- Addressing CMC challenges to ensure consistent, reliable treatment options that benefit both physicians and patients through improved compliance and satisfaction
12:00 pm Lunch & Networking
GENE THERAPY BREAKTHROUGHS: PIONEERING NEW PATHWAYS IN WET AMD WITH ONE-TIME TREATMENT OPTIONS
1:00 pm Transforming Wet AMD Treatment with Ixo-vec, an Intravitreal Gene Therapy
Synopsis
- Explore the innovative approach of Ixo-vec, which uses the AAV.7m8 capsid to deliver a gene encoding aflibercept, enabling continuous production of the VEGF antagonist after a single intravitreal injection
- Review the LUNA Phae 2 trial results, demonstrating the safety and efficacy of Ixo-vec in reducing the need for frequent anti-VEGF injections and maintaining visual and anatomical outcomes in patients with wet AMD
- Discuss the potential impact of Ixo-vec on patient quality of life, highlighting the reduced treatment burden and the promise of long-term vision preservation with a single treatment
1:30 pm In-Vitro & In-Vivo Studies to Support the World’s First CRISPR/RNATargeting Therapy, HG202, for Patients with Neovascular Age-Related Macular Degeneration
Synopsis
- Highlight the significant unmet medical need for effective treatments for neovascular age-related macular degeneration
- Explain the molecular composition, mechanism of action, and preclinical studies of HG202 using animal models
- Outline the clinical trial design, preliminary safety data, and case studies demonstrating the clinical efficacy of HG202
2:00 pm Afternoon Refreshments
2:15 pm Panel Discussion: Comparing Gene Therapy Approaches for Wet AMD: Delivery Methods, Targets, & Therapeutic Potential
Synopsis
- How are intravitreal and subretinal delivery methods impacting treatment precision, effectiveness, and patient experience?
- What are the implications of targeting VEGF suppression versus complement modulation on efficacy and safety?
- How can we optimize long-term outcomes by addressing durability, immune response, and safety across different gene therapy approaches?
SUCCEEDING IN A COMPETITIVE ANTI-VEGF MARKET
3:45 pm Fireside Chat: Comparative Market Analysis of Wet AMD Treatments: Trends & Opportunities
Synopsis
- Evaluate the current landscape of anti-VEGF therapies across Wet AMD, highlighting differences in treatment efficacy, patient demographics, and injection frequencies
- Analyze the economic impact and market penetration of newer treatments like faricimab and biosimilars, considering their uptake and performance in real-world settings compared to traditional therapies
- Identify emerging trends and unmet needs in each treatment area, focusing on the potential for innovative therapies to address gaps in efficacy, durability, and patient compliance
Moderated by: Daniil Gataulin, Senior Research Analyst, Chardan Capital Markets