Adverum Biotechnologies(ADVM) - 2019 Q2 - Earnings Call Transcript

Financial Data and Key Metrics Changes - The company is focused on developing ADVM-022 for treating VEGF-driven retinal diseases, particularly wet AMD and diabetic retinopathy, which are significant causes of vision loss [6][9] - The company has achieved significant clinical and regulatory milestones with ADVM-022, with plans to present 24-week data from the Phase I OPTIC trial at an upcoming meeting [8][21] Business Line Data and Key Metrics Changes - ADVM-022 is being developed as a single intravitreal injection approach, which could provide a more durable treatment option compared to current therapies that require frequent injections [6][13] - The company has discontinued the gene therapy program targeting A1AT deficiency and has moved the program for Hereditary Angioedema back into early development [9][10] Market Data and Key Metrics Changes - Approximately 1.2 million individuals in the US are affected by wet AMD, and the prevalence of diabetic retinopathy is also increasing, indicating a growing market for ADVM-022 [7][19] - The company plans to submit an IND for ADVM-022 in diabetic retinopathy in the first half of 2020, highlighting the potential expansion of its market reach [20] Company Strategy and Development Direction - The company aims to scale up its manufacturing process to support later-stage development and commercial use, with a new corporate headquarters to enhance in-house capabilities [10] - The focus remains on developing ADVM-022 for AMD and diabetic retinopathy, while exploring opportunities in other VEGF-driven diseases [9][20] Management's Comments on Operating Environment and Future Outlook - Management believes that gene therapy can be transformative for patients and is committed to advancing their development pipeline [6][11] - The company is optimistic about the upcoming data presentations and the potential impact of ADVM-022 on treatment burdens for patients [21][65] Other Important Information - Recent corporate updates include the retirement of board member Mitch Finer and the transition of President and Chief Scientific Officer Mehdi Gasmi to the Board of Directors [10] - The company is expanding its talent pool with recent senior management additions to support its mission [11] Q&A Session All Questions and Answers Question: Can you provide clarity on the types of patients being enrolled in the OPTIC trial? - The protocol stipulates that patients must have had at least two injections in the previous four months, ensuring they require frequent anti-VEGF injections [23][24] Question: Can you elaborate on the criteria for rescue injections in the trial? - Rescue injections are based on OCT and vision criteria, with stringent protocols to ensure patients only receive them when necessary [27][29] Question: What is the rationale behind using prophylactic oral steroid dosing in the study? - The use of tapering doses of prednisone is to manage expected immune responses following gene therapy [30][31] Question: How do you view the acceptable level of inflammation for less frequently administered intravitreal gene therapy? - A balance is expected, where some immune response is acceptable if it leads to a significant reduction in the need for rescue injections [33][34] Question: What was the reasoning for the decision to lower the dose in the second cohort? - The decision was based on observing robust preliminary anatomical responses and not due to safety concerns [36][38] Question: Can you clarify the relationship between anatomical responses and the need for additional anti-VEGF injections? - Robust anatomical responses are assessed through OCT, and the goal is to reduce the number of rescue injections required [63][64]