Adverum Biotechnologies(ADVM) - 2020 Q1 - Earnings Call Transcript

Financial Data and Key Metrics Changes - The company ended Q1 2020 with $297 million in cash, cash equivalents, and marketable securities, which is expected to finance operations until 2022 [11] - The financial results for the first quarter of 2020 were reported alongside the announcement of the new INFINITY trial for ADVM-022 [5] Business Line Data and Key Metrics Changes - The ongoing OPTIC Phase I trial for ADVM-022 has shown promising data, demonstrating long-term durability beyond one year with zero rescue injections in patients treated in Cohort 1 [9][14] - The INFINITY Phase 2 trial for diabetic macular edema (DME) is now open for patient enrollment, with the FDA having approved the IND for diabetic retinopathy [10][15] Market Data and Key Metrics Changes - Approximately 30 million people in the U.S. are impacted by diabetes, with about 1.5 to 2 million patients likely suffering from DME, representing a significant market opportunity [15][42] - The current standard-of-care therapy for DME requires frequent anti-VEGF intravitreal injections, which presents a challenge for patient compliance [16] Company Strategy and Development Direction - The company aims to position itself as a leader in gene therapy, focusing on developing a pipeline of novel gene therapies beyond ADVM-022 [10] - The INFINITY trial is designed to demonstrate superior control of disease activity following a single injection of ADVM-022 compared to aflibercept [17] Management's Comments on Operating Environment and Future Outlook - Management acknowledged the impact of COVID-19 on operations but emphasized the commitment to advancing clinical trials and maintaining patient safety [7][12] - The company remains focused on developing transformative one-time treatments for patients with wet AMD and DME [20] Other Important Information - The collaboration with Regeneron has been terminated, allowing the company to explore new indications and targets [77] - The company has implemented remote study monitoring and precautionary measures at study sites due to COVID-19 [7][12] Q&A Session Summary Question: Can you clarify the DME trial design and the criteria for rescue injections? - Management confirmed that the criteria for rescue injections in the INFINITY trial are stringent, with expectations that more than half of aflibercept patients would require rescue by week 16 [24][34] Question: What are the implications of the trial design for future registrational studies? - Management indicated that the trial design is robust and aims to show superiority over current standard care, with potential for creative approaches in future studies [29][66] Question: How does the company plan to address the dosing for DME patients? - The same two doses used in wet AMD trials will be applied, with flexibility to adapt based on interim analysis results [66] Question: What is the expected timeline for data from the INFINITY trial? - Management stated that data will be shared once patients are enrolled and treated, with further guidance on timelines to follow [69] Question: Can you discuss the inflammation kinetics observed in the OPTIC trial? - Inflammation is expected to peak early post-injection, with steroid eye drops effectively managing it [72][74] Question: What are the next steps following the OPTIC trial? - The company plans to analyze data from Cohort 4 before determining the design of future studies [48][68]