Precigen(US:PGEN)2025-03-19 23:47Financial Data and Key Metrics Changes - The company reported a net loss of $126.2 million for 2024, compared to a net loss of $95.9 million in 2023, representing an increase in loss per share from $0.39 to $0.47 [36][41] - Cash burn for 2024 totaled $76.8 million, which includes cash used in operations and capital expenditures [36][41] - The company raised $79 million through a preferred stock issuance at the end of 2024, which included warrants for common stock [38][40] Business Line Data and Key Metrics Changes - The lead asset, PRGN-2012, has shown a 51% complete response rate in pivotal clinical trials, with a median durability of response at 24 months [9][10] - The confirmatory trial for PRGN-2012 has been initiated, with 35 patients to be enrolled, and is designed in alignment with FDA requirements [13][70] Market Data and Key Metrics Changes - The estimated number of adult patients with RRP in the US is now believed to be up to 27,000, higher than previous estimates of 20,000, indicating a greater unmet need [22][61] - The company anticipates commercial revenues from PRGN-2012 to begin in the second half of 2025 [23][41] Company Strategy and Development Direction - The company is transitioning from R&D to commercialization, focusing on the launch of PRGN-2012 as the first and only medical treatment for RRP [24][35] - The commercialization strategy includes a partnership with EVERSANA to support the US launch, emphasizing a patient-centric approach [20][22] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing interactions with the FDA regarding the BLA submission and the priority review process [46][48] - There is an expectation of pent-up demand for PRGN-2012, as patients have had limited treatment options historically [56][57] Other Important Information - The company has made significant investments in GMP manufacturing capabilities to support the commercial launch of PRGN-2012 [16][17] - The UltraCAR-T platform is advancing, with ongoing discussions with the FDA regarding pivotal Phase 2 studies [32][34] Q&A Session Summary Question: Update on FDA interactions and commercial launch plans - Management confirmed close interactions with the FDA regarding the BLA submission and characterized the ongoing review positively [46][48] Question: Timing of additional FDA meetings and inspections - Management indicated that pre-approval inspections are anticipated but could not comment on specific timing [54][78] Question: Anticipated patient demand and treatment access - Management acknowledged expected pent-up demand for PRGN-2012 and plans to deploy efforts to ensure timely access for patients [56][57] Question: Status of the confirmatory trial for PRGN-2012 - Management confirmed that the confirmatory trial has been initiated and is expected to take 12 months to complete [70] Question: Pricing and payer access expectations - Management discussed ongoing discussions with payers, indicating a focus on value-based pricing and the importance of the drug's clinical value [96][100]