Financial Data and Key Metrics Changes - The company reported a significant reduction in non-GAAP operating expenses by 50% year-on-year in 2024, focusing on key priorities to enhance efficiency [16][11] - An equity offering was announced to extend the company's cash runway, which is expected to last until late in the third quarter of 2025 [15][11] Business Line Data and Key Metrics Changes - The company signed a third capsid license agreement with Eli Lilly, receiving an upfront fee of 1.4 billion across five disease targets [5][6] - The Fabry program achieved key de-risking milestones, with all patients in the Phase 1/2 STAR study completing at least 52 weeks of follow-up, indicating a positive mean eGFR slope [9][10] Market Data and Key Metrics Changes - The company is actively engaging in discussions with multiple potential partners for its Fabry disease program, indicating strong interest in its technology [12][32] - The FDA's recent interactions have been positive, with no significant changes in their approach to the company's programs, including Fabry disease [32][33] Company Strategy and Development Direction - The long-term vision is to focus on neurology as a core area, aiming to deliver transformational therapies while maximizing shareholder value [13][15] - The company is committed to securing a commercial partnership for the Fabry program to support its neurology mission and ensure sustainable funding [11][18] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the FDA's engagement and the clarity provided regarding the chemistry, manufacturing, and controls pathway for the BLA submission [10][32] - The company remains focused on advancing its neurology pipeline and securing necessary funding to support ongoing clinical trials [11][18] Other Important Information - The company plans to present data at the ASGCT Annual Meeting, showcasing advancements in its neurology pipeline and the effectiveness of its capsid delivery technology [8][18] - The company is exploring additional funding opportunities through collaborations related to its technology platforms [12] Q&A Session Summary Question: Can you provide more color on what exactly you plan to show in the top line eGFR data? - Management confirmed that they will share the updated mean eGFR slope and additional information at a later date [21][23] Question: How many potential partners are you currently in conversations with regarding STABBI? - Management indicated that there are multiple potential partners but did not disclose specific details [26] Question: Has the pace of conversations with potential partners changed due to the macro landscape? - Management reported that interactions with the FDA have been positive and have not impacted their program [33] Question: Do you still plan to file based on 52-week eGFR data? - Management confirmed that they are pursuing the agreement with the FDA to use 52-week eGFR data for the entire patient population [37] Question: Are all patients who were on ERT still off ERT? - Management confirmed that all 18 patients who started on ERT are still off ERT [46] Question: Is there any threshold for the eGFR slope that should be looked at? - Management stated they are not commenting on specific statistical significance at this time but confirmed the mean eGFR slope remains positive [48][49]
Sangamo Therapeutics(SGMO) - 2025 Q1 - Earnings Call Transcript