Preserving Sight: A Deep Dive into ONL Therapeutics' Innovative Approach to Combat Retinal Diseases

David Esposito, CEO, ONL Therapeutics

In the rapidly evolving biotechnology landscape, pioneering companies like ONL Therapeutics are leading the charge with groundbreaking research and innovative therapeutics. With a firm commitment to preserving sight for patients with retinal diseases, ONL Therapeutics is making significant strides in the field. David Esposito, CEO of ONL Therapeutics, is at the helm of these efforts. In this interview, Esposito provides a glimpse into the company's mission, discusses the challenges and strategies associated with clinical trials, and shares how ONL Therapeutics is tackling the arduous task of securing funding in a highly competitive industry. He also delves into the intricacies of their lead compound, ONL 1204, and its potential to transform treatment paradigms for retinal diseases.

Moe Alsumidaie: ONL Therapeutics is making waves in the biotech world. Could you tell us more about the company and its mission?

David Esposito: At ONL Therapeutics, we're committed to preserving sight for patients diagnosed with retinal diseases. Our core mission is to develop innovative therapeutics that block the activation of specific receptors triggered by the stress of disease in conditions such as glaucoma, age-related macular degeneration, and retinal detachment. Activating these receptors leads to the death of retinal cells, so our work focuses on blocking this process to protect these cells.

Alsumidaie: David, could you provide us with a detailed explanation of your leading compound, ONL 1204, and its role in mitigating the progression of geographic atrophy, a late stage of dry age-related macular degeneration?

Esposito: ONL 1204 is a small, peptide comprising 12 specific amino acids. It is administered through an intravitreal injection, which means it's delivered directly into the eye, right to where it's needed. In geographic atrophy, this condition is considered the late stage of dry age-related macular degeneration. It's characterized by the formation of lesions on the retina, which unfortunately leads to further growth of those lesions and the death of functioning retinal cells. As these lesions expand and cover the center of the visual field, the patient gradually moves toward blindness. Herein lies the potential of ONL 1204. The uniqueness of ONL 1204 lies in its target—the Fas receptor. When activated due to stress of disease, this receptor can set off a chain of events, leading to the death of retinal cells. By precisely blocking the activation of the Fas receptor, ONL 1204 acts as a shield for these vulnerable retinal cells, protecting them from this damaging cascade. Furthermore, it has the added benefit of damping down the inflammatory response often accompanying these disease states.

Alsumidaie: What challenges do you anticipate during the clinical trials of ONL 1204, and how do you plan to tackle them?

Esposito: Every clinical trial has its unique challenges. With ONL 1204, we're testing it across three different indications: retinal detachment, dry age-related macular degeneration (AMD), and open-angle glaucoma, each with unique hurdles. For retinal detachment, an acute condition, patients need immediate treatment. This makes patient recruitment challenging. To address this, we have designed our Phase II study to align with real-world scenarios, making the patient entry criteria more flexible. We have also invested time in training clinical staff specifically to handle the patient consent process during these acute situations. In the case of dry AMD, many patients have remained in the care of optometrists and general ophthalmologists for years because retina specialists did not have any approved therapies to treat the disease which made finding and recruiting these patients difficult. We have been utilizing web-based outreaches, patient advocacy groups, and social media to reach these patients, even though it can be complex due to the age demographic of these patients. Patient education is critical for open-angle glaucoma as our drug is administered via IVT injection, a procedure not commonly associated with glaucoma treatment. We have been investing in educating patients, clinical study staff, and clinicians about the benefits and process of this treatment.

Alsumidaie: How do you ensure you choose the best sites for your trials?

Esposito: Site selection plays a crucial role in the success of clinical trials. Our approach revolves around working closely with our clinical research organization partners to thoroughly assess potential sites based on their track record, patient population, and logistical considerations. We also consider the expertise and commitment of the investigators and their teams. Once the sites are selected, we provide clear, concise training to ensure all parties agree regarding protocol procedures and study objectives. We believe in fostering strong relationships with our clinical trial sites, and to this end, we maintain regular communication with them. This involves sending monthly newsletters with critical updates, conducting virtual meetings, and providing timely reminders. We also appreciate the hard work done by our site partners and we provide some tokens of appreciation to acknowledge their efforts throughout the study. It is important that we strike a balance in communications with the sites as support is essential but we try not to become overwhelming. We respect that our site partners have multiple studies to manage and ensure our interactions add value to their work.

Alsumidaie: Securing funding is a common challenge in the biotech sector. How have you addressed this?

Esposito: Raising capital is challenging, particularly in the competitive biotech sector. Our approach has been to establish a strategic plan that includes specific, time-bound milestones that demonstrate the potential of our work and add value to our proposition. We've gained investors' confidence by consistently delivering positive results on these milestones. We've shown our investors the scientific value of our work and the potential market value of our therapeutics. We've also adopted a global mindset when it comes to potential investors. While we look for domestic investment, we've also cast our net wider to include investors from Europe and Asia. This diversifies our investor base and opens up new potential sources of funding. By maintaining transparency and demonstrating our commitment to our mission, we've navigated the complexities of securing financing in the biotech sector.

Alsumidaie: Is there anything else you'd like to share with our readers?

Esposito: I want to emphasize that while our work is challenging, it's gratifying. We're working tirelessly to bring innovative treatments to the market that can significantly improve people's lives. We're genuinely grateful for the support we've received and excited about what the future holds for ONL Therapeutics and the patients and healthcare professionals we aim to serve.

Moe Alsumidaie, MBA, MSF, is a thought leader and expert in the application of business analytics toward clinical trials, and regular contributor to Applied Clinical Trials.