On May 15, 2024, we had the pleasure of hosting a webinar with Dr. George Magrath, the CEO of Ocuphire Pharma, who joined the company in November 2023. The event was moderated by Echo Hindle-Yang, Founder andCEO of MSQ Ventures. Dr. Magrath brought his extensive experience in medical and business administration to Ocuphire, aiming to lead the company through its next phase of innovation and growth. 

Dr. Magrath shared insights into his professional journey, starting from his medical training and practice in ophthalmology to his roles in biotech and capital markets. His career has been driven by a commitment to making a transformative impact in ophthalmology, leading him to Ocuphire Pharma where he saw significant potential in their innovative work. 

Ocuphire’s Strategy and Vision 

Under Dr. Magrath’s leadership, Ocuphire Pharma has focused on building a lean but highly skilled team, essential for executing their ambitious diabetic retinopathy program. The company's vision is to shift the paradigm in treating diabetic retinopathy, the leading cause of blindness in America, by addressing the condition early, before severe complications arise. 

Partnerships and Financial Stability 

Ocuphire's strategic partnerships have been pivotal in their development. Dr. Magrath highlighted the collaboration with Viatris for the commercial launch of RyzumV, a product designed to reverse eye dilation after exams, which has significantly strengthened Ocuphire’s financial position. This partnership exemplifies the importance of leveraging external expertise to accelerate product dissemination and market reach. 

Innovative Pipeline  

Dr. Magrath provided an overview of Ocuphire’s pipeline, including their leading asset, APX3330, an oral therapy for diabetic retinopathy. APX3330 targets the REF-1 transcription factor, aiming to restore normal levels of VEGF, thereby preventing disease progression rather than just treating acute symptoms. This approach could transform how early-stage diabetic eye disease is managed. 

He also touched upon APX2009 and APX2014, other promising oral therapies in their pipeline. These drugs, with different affinities and properties, target geographic atrophy through IL-6 mediated pathways and could offer significant improvements over current treatments by preventing complications like neovascularization. d for surgery. 

Presentation Highlights  

Team Expertise 

• Nirav Javari: Extensive experience in capital markets and as a CFO in biotech firms. 

• Ash Jayagopal: Chief Scientific and Development Officer with expertise in diabetic retinopathy from his time at Ro and Kodiak. 

• Joseph Schachle: Chief Operating Officer with a career in building pharmaceutical and biotech companies. 

• Kim Morris: Long-time collaborator with expertise in medical affairs and communications.  

Paradigm Shift in Diabetic Eye Disease Treatment 

Diabetic eye disease is a leading cause of blindness in America, affecting 10 million patients. 

• Current treatments only begin after vision loss. The goal is to offer early intervention to slow progression and prevent vision loss, similar to how statins prevent heart attacks. 

• The innovative approach involves an oral medication, APX 3330, targeting pathways of diabetic retinopathy without the need for invasive eye injections. 

Clinical Data and Trials 

• Zeta 1 Phase II Trial: Demonstrated that fewer patients on APX 3330 experienced disease progression compared to placebo. Significant reductions in progression to vision-threatening complications and vision loss were observed. 

• Zeta 2 Study: Designed to confirm these results over a year-long study with 300 patients, focusing on those at high risk for progression. This study aims to align with FDA feedback and ensure rigorous testing of the drug’s efficacy. 

Market Potential and Competitive Landscape  

• The target market includes 8 million patients with early-stage diabetic retinopathy who currently lack non-invasive treatment options. 

• APX 3330 stands out by targeting both angiogenesis and inflammation, unlike current treatments that only address one aspect. 

Mechanism of Action and Safety Profile 

• APX 3330 works by modulating Ref-1, a transcription factor affecting angiogenesis, oxidative stress, and inflammation. 

• The drug maintains VEGF at a homeostatic level, reducing systemic side effects and allowing for oral administration. 

• The safety profile is favorable, with manageable side effects like itching and rash. 

Dr. Magrath highlighted the innovative approach to treating diabetic eye disease, emphasizing the importance of early intervention and non-invasive treatments. The promising clinical data, coupled with a strong team and strategic partnerships, positions the company well for future success in improving patient outcomes and generating revenue.