Adverum Biotechnologies(ADVM) - 2019 Q4 - Earnings Call Transcript

Financial Data and Key Metrics Changes - The company reported a strong financial position with over $300 million in cash, following a public follow-on offering that raised approximately $141 million, expected to fund operations into 2022 [10][11] - Specific financial metrics for Q4 2019 were not detailed in the provided content, but the overall financial health appears robust [10] Business Line Data and Key Metrics Changes - The lead gene therapy candidate, ADVM-022, is advancing with significant clinical progress, particularly in the OPTIC trial for wet AMD, with promising data from Cohorts 1 and 2 showing robust efficacy and a favorable safety profile [8][14][15] - The company plans to explore a second indication for diabetic retinopathy (DR) with an IND submission expected in the first half of 2020 and a Phase 1/2 trial beginning in the second half of the year [8][17] Market Data and Key Metrics Changes - The diabetic retinopathy market is highlighted as a large and underserved area, with an estimated 8 million people affected in the U.S., but only 2 million diagnosed and 1 million treated [17][18] - The prevalence of diabetic retinopathy is expected to increase alongside the growing prevalence of diabetes, indicating a significant unmet need for effective therapies [18] Company Strategy and Development Direction - The company is focused on delivering transformative therapies for patients with serious ocular and rare diseases, positioning itself as a leader in gene therapy [7][9] - The strategy includes advancing the clinical development of ADVM-022 and expanding the pipeline of novel gene therapies using their industry-leading AAV platform [9] Management's Comments on Operating Environment and Future Outlook - Management acknowledged the fluid situation regarding the coronavirus and its potential impact on operations, but reported no specific impacts on the OPTIC trial to date [6][12] - The management expressed excitement about the potential of ADVM-022 as a one-time treatment for wet AMD and DR, emphasizing the commitment to advancing these programs [11][18] Other Important Information - The company has moved to a new headquarters in Redwood City to support growth plans and expand core capabilities [9] - Angela Thedinga was appointed as Chief Technology Officer to lead the manufacturing strategy as the company advances toward later-stage clinical trials [10] Q&A Session Summary Question: Enrollment status for Cohort 4 and potential read-throughs for diabetic retinopathy - Management confirmed active screening for Cohort 4 with strong interest from investigators, indicating rapid enrollment is expected [21][22] - Regarding diabetic retinopathy, management discussed potential read-throughs from AMD data, emphasizing safety and efficacy considerations [23][24] Question: Safety signals and implications for intravitreal therapy - Management highlighted the differences in inflammation types between ADVM-022 and other therapies, noting that ADVM-022 has shown manageable low-grade inflammation [30][33] Question: Status of steroid tapering in cohorts - Management provided insights into the tapering of steroids used in previous cohorts, indicating that inflammation has been low-grade and manageable [48][51]