Sangamo Therapeutics(SGMO)2024-11-13 02:25Financial Data and Key Metrics Changes - Sangamo Therapeutics has transitioned from a Phase I/II company to a pre-BLA company due to significant regulatory developments in its Fabry disease program [8] - The company has received 220 million in regulatory and commercial milestones over the next 2 years [10][26] - The company submitted its first IND application for a neurology indication, ST-503 for intractable pain, expected to advance into the clinic in mid-2025 [13][33] Market Data and Key Metrics Changes - The FDA has confirmed that eGFR slope data at 52 weeks can serve as the primary basis for approval under the accelerated approval pathway for the Fabry program [15][18] - The ongoing Phase I/II STAR study has enrolled and dosed 33 patients, representing a broad range of Fabry patients [20] Company Strategy and Development Direction - Sangamo is focused on advancing its core neurology pipeline and has engaged in ongoing business development discussions to expedite treatment delivery [10][11] - The company aims to submit BLA applications for up to 2 separate gene therapy programs in 2025, which could provide a long-term financial foundation [11][38] - Sangamo is committed to transforming into a neurology genomic medicine company, with plans to advance its therapies to patients in need [39] Management's Comments on Operating Environment and Future Outlook - Management expressed pride in the progress made in 2024 and emphasized the commitment to translating groundbreaking science into medicines for serious neurological diseases [7] - The company anticipates sharing a regulatory update in early 2025 and is preparing for continued discussions with the European Medicines Agency [22] - Management highlighted the positive feedback from patients regarding the Fabry treatment, indicating a strong unmet medical need [70] Other Important Information - The company has begun executing BLA readiness activities for the Fabry program and is preparing for a potential submission in the second half of 2025 [19][22] - Sangamo's technology aims to address the challenges of delivering treatments to the central nervous system, which has historically been difficult [27] Q&A Session Summary Question: FDA comments on eGFR slope and partnership strategy for Fabry program - Management clarified that the FDA agreed to use eGFR slope at 52 weeks as the primary basis for approval and that no confirmatory study is required [41][45] - Regarding partnerships, management indicated ongoing interest from potential partners and emphasized the importance of securing a deal that benefits patients [46][47] Question: Next data update from the Fabry program - Management expects to have data available in the second quarter of 2025, with the last patient visit occurring in April 2025 [48][49] Question: Propensity match control data and its importance - Management stated that the FDA will consider the totality of the data, including eGFR and other clinical endpoints, in their evaluation [53][55] Question: Evolution of FDA conversations and confidence in Pfizer's commitment - Management noted that the FDA's approach has evolved to accommodate the unique challenges of rare diseases, and expressed confidence in Pfizer's commitment to the hemophilia A program [60][64] Question: Inclusion of female Fabry patients in pivotal data set - Management confirmed that discussions with the FDA have focused on all Fabry patients, and they will consider all relevant data in the submission [66][67]