Treatment with 60 mg of BEAM-302 Led to Mean Steady-state Total AAT Level of 16.1 µM and All Patients Consistently and Durably Above the 11 µM Protective AAT Threshold with up to 12 Months of Follow-up Corrected M-AAT Comprised 94% of Total AAT with a Concomitant 84% Reduction in Mutant Z-AAT Following BEAM-302 60 mg Treatment Post-treatment Inducibility of AAT Observed During Respiratory Infection with a Patient Reaching ~30 µM Total AAT, Retaining 95% M-AAT Composition Well-tolerated Safety Profile Observ ...

Financial Data and Key Metrics Changes - Total revenue for Q4 2025 was $200 million, and for the full year 2025, it was $1 billion, which included a one-time $275 million upfront payment from the Sobi royalty repurchase agreement [20] - Syfovre net product revenue for Q4 was $155 million and $587 million for the full year 2025, with total injections growing approximately 17% year-over-year despite a modest decline in revenue due to elevated use of free goods [11][20] - Empaveli reported U.S. net product revenue of $35 million for Q4 and $102 million for the full year 2025, with a market penetration of over 5% after its first full quarter [22][9] Business Line Data and Key Metrics Changes - Syfovre continues to lead the geographic atrophy (GA) market, with strong physician and patient demand, and is expected to remain a stable revenue stream through 2026 [7][11] - Empaveli's launch trajectory is progressing well, with strong early market receptivity and a growing patient pipeline, particularly in C3G and primary IC-MPGN [8][12] - The company is advancing pivotal trials in nephrology for focal segmental glomerulosclerosis and delayed graft function, expanding the Empaveli franchise [10][18] Market Data and Key Metrics Changes - The company expects to strengthen Syfovre's leadership in geographic atrophy and drive growth with Empaveli across rare kidney diseases [7] - Empaveli's strong efficacy and safety profile are driving adoption, with expectations that it could ultimately be used by up to 50% of the estimated 5,000 U.S. patient population for C3G and primary IC-MPGN [9][15] Company Strategy and Development Direction - The company is focused on three strategic pillars: strengthening Syfovre's leadership, driving growth with Empaveli, and advancing an innovative pipeline [7] - The introduction of a best-in-class prefilled syringe and an AI-enabled approach for Syfovre is aimed at improving treatment accessibility and workflow efficiency [8][10] - The company aims to self-fund its pipeline and drive long-term value through disciplined financial execution [10][23] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the long-term value creation potential of the company, emphasizing a strong balance sheet and disciplined execution [5][25] - The management highlighted the importance of patient access and the evolving dynamics of free goods utilization as third-party programs resume activity [21][57] - The company anticipates modest inventory reduction in Q1 2026 alongside typical seasonal dynamics, with a focus on maintaining a stable net price through the year [21][64] Other Important Information - The company ended 2025 with $466 million in cash and cash equivalents, providing substantial flexibility to fund operations [23] - A $25 million milestone payment was triggered by Sobi's European Commission approval for Aspaveli in C3G and primary IC-MPGN [23] Q&A Session Summary Question: Empaveli launch and growth potential - Management noted that Empaveli is on a clear path to blockbuster status, with a focus on broadening accounts and improving patient identification [29][30] Question: Syfovre five-year data significance - The five-year GALE data shows significant benefits in delaying progression of geographic atrophy, which may lead to earlier treatment for patients [34][36] Question: Empaveli penetration timeline - Management expects a steady ramp in penetration, with a focus on pediatric and post-transplant patients as key growth areas [39][40] Question: Prefilled syringe impact - The prefilled syringe is expected to enhance practice efficiency and support broader adoption of Syfovre [50][52] Question: Free goods dynamics - Management emphasized the commitment to patient access through free goods, which may impact new patient onboarding [97][98]

Financial Data and Key Metrics Changes - The company ended 2025 with $1.25 billion in cash equivalents and marketable securities, with an anticipated minimum draw of $200 million from a strategic financing agreement, extending the runway into mid-2029 [40][41] - A strategic financing agreement with Sixth Street provides up to $500 million in long-term, non-dilutive capital to support the anticipated launch of risto-cel, enhancing financial flexibility [39][40] Business Line Data and Key Metrics Changes - The company is expanding its portfolio with a new development program for phenylketonuria (PKU) called BEAM-304, targeting the two most common mutations found in nearly half of PKU patients [9][19] - The risto-cel program is positioned as a potentially best-in-class autologous cell therapy for sickle cell disease, with significant demand observed in the market [39][73] Market Data and Key Metrics Changes - Approximately 20,000 individuals in the U.S. are living with PKU, representing a significant unmet need for new treatment options [10][19] - The market for risto-cel is characterized by a significant demand, with no refusals for payment reported despite high treatment costs [73] Company Strategy and Development Direction - The company aims to provide lifelong cures through base editing, focusing on one-time durable genetic medicines with predictable outcomes [5][6] - The modularity of the base editing platform allows for scalable development across multiple genetic diseases, with a focus on addressing additional mutations over time [8][20] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of base editing to deliver transformative therapies, emphasizing the importance of predictability in outcomes for patients and the healthcare ecosystem [7][42] - The company is well-positioned to realize the power of predictability across its growing portfolio, with plans to file the IND for BEAM-304 in 2026 [42][43] Other Important Information - The company has established a foundation of financial strength for sustainable growth, with a focus on efficient investments and building commercial capabilities ahead of the risto-cel launch [40][41] - The regulatory landscape is evolving, with the FDA showing openness to innovative trial designs that could accelerate the development of therapies for ultra-rare diseases [60][69] Q&A Session Summary Question: Regulatory path for multiple mutations and capital allocation - Management indicated that research efforts for additional mutations are already underway, with timelines expected to be fast due to the platform approach [48] - The strategic financing allows for flexibility in reallocating capital to additional liver-targeted indications [50] Question: Intellectual property and delivery mechanisms for BEAM-304 - The company has access to necessary IP, with pioneering work from Dr. Musunuru's lab contributing to the development of therapies for specific mutations [54] - BEAM-304 will utilize broadly similar lipid nanoparticle approaches as previous programs, ensuring consistency in delivery mechanisms [54] Question: FDA guidance on individualized therapy and mutation targeting - The FDA's draft guidance on individualized therapy is geared towards ultra-rare diseases, with ambiguity regarding the classification of PKU mutations [60][61] - The company plans to develop mutation-specific guide RNAs for each mutation within the same clinical program [68] Question: Responses of R408W carriers to current therapies and recruitment age range - R408W patients typically do not respond to existing therapies due to low enzyme activity, highlighting the need for new treatment options [84] - The FDA is showing collaboration in potentially allowing trials to include younger patient populations as data is gathered [83]

Financial Data and Key Metrics Changes - The company ended 2025 with $1.25 billion in cash equivalents and marketable securities, with an anticipated minimum draw of $200 million from a strategic financing agreement, extending the runway into mid-2029 [37][38] - A strategic financing agreement with Sixth Street provides up to $500 million in long-term, non-dilutive capital to support the anticipated launch of risto-cel, enhancing financial flexibility [36][37] Business Line Data and Key Metrics Changes - The company is expanding its portfolio with a new development program for phenylketonuria (PKU) called BEAM-304, targeting the two most common mutations found in nearly half of PKU patients [9][17] - The BEAM-304 program aims to leverage proprietary base editing technology and LNP delivery systems to correct disease-causing mutations in PKU patients [18][19] Market Data and Key Metrics Changes - Approximately 20,000 individuals in the U.S. are living with PKU, with significant unmet needs despite existing therapies [9][17] - The company is focusing on addressing a large population of PKU patients through innovative development approaches and regulatory pathways [9][18] Company Strategy and Development Direction - The company aims to provide lifelong cures for serious diseases through base editing, emphasizing predictable and reproducible outcomes [4][5] - The strategy includes leveraging modular technology to address multiple mutations and expand into other metabolic disorders over time [6][23] - The company is committed to developing a one-time treatment for as many PKU patients as possible, with plans to file the IND for BEAM-304 in 2026 [22][23] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the scalability of the base editing platform and its potential to deliver transformative therapies to patients [9][40] - The company is well-positioned to realize the power of predictability across its growing portfolio, with a focus on accelerating paths to approval for its lead programs [40][41] Other Important Information - The company has established a foundation of financial strength for sustainable growth, with a focus on building commercial capabilities ahead of the anticipated risto-cel launch [37][39] - The regulatory environment is evolving, with the FDA showing openness to innovative trial designs that could accelerate the development of gene editing therapies [60][68] Q&A Session Summary Question: Regulatory path for multiple mutations and capital allocation - Management indicated that research efforts for additional mutations are already underway, with expectations for faster timelines due to the platform approach [45][46] - The strategic financing allows for reallocation of capital to additional liver-targeted indications [48] Question: Intellectual property and delivery mechanisms for BEAM-304 - The company has access to necessary IP and will utilize similar LNP approaches as in previous programs [51][52] Question: FDA's draft guidance on individualized therapy - The FDA's guidance is geared towards ultra-rare diseases, with ambiguity regarding the classification of PKU mutations [60][61] Question: Responses of R408W carriers to current therapies - R408W patients typically do not respond to existing therapies due to low PAH enzyme activity, highlighting the need for new treatment options [86][87]

Core Insights - Beam Therapeutics is expanding its pipeline with a new program, BEAM-304, aimed at treating phenylketonuria (PKU), a rare metabolic disorder affecting approximately 20,000 individuals in the U.S. [1][3] - The company anticipates filing an Investigational New Drug (IND) application for BEAM-304 in 2026 and expects to report updated Phase 1/2 data for BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD) by Q1 2026 [1][5] - A strategic financing agreement with Sixth Street provides up to $500 million in long-term, non-dilutive capital to support the anticipated launch of risto-cel for Sickle Cell Disease (SCD), with a U.S. Biologics License Application (BLA) submission expected by year-end 2026 [1][11] Financial Performance - As of December 31, 2025, Beam's cash, cash equivalents, and marketable securities totaled $1.25 billion, an increase from $850.7 million in 2024 [18][20] - Research and Development (R&D) expenses for Q4 2025 were $99.3 million, compared to $101.4 million in Q4 2024, while full-year R&D expenses rose to $409.6 million from $367.6 million [18][21] - The company reported a net income of $244.3 million for Q4 2025, translating to $2.37 per basic share, compared to a net loss of $90.4 million in Q4 2024 [18][22] Pipeline Developments - BEAM-304 utilizes Beam's proprietary base editing technology and lipid nanoparticle (LNP) delivery to correct mutations in the PAH gene responsible for PKU, aiming to normalize phenylalanine levels and improve dietary freedom for patients [3][4] - The initial clinical development of BEAM-304 will focus on the R408W mutation, with plans to address additional mutations in future expansions [6][4] - Beam's lead program, BEAM-302, is designed to treat AATD and is on track for pivotal development, with an expected BLA submission based on AAT biomarkers evaluated over 12 months [11][14] Strategic Partnerships - In December 2025, Pfizer opted for an exclusive worldwide license for a liver-targeted development candidate from Beam, which will involve shared responsibilities for development and commercialization [11][14] - The strategic financing agreement with Sixth Street includes a $100 million initial funding, with additional funds contingent on achieving specific milestones [11][14] Future Outlook - Beam expects its cash runway to extend into mid-2029, supporting the launch of risto-cel, the pivotal development of BEAM-302, and the clinical proof of concept for BEAM-304 [14][18] - The company is well-positioned to execute its clinical, regulatory, and commercial objectives, aiming to deliver transformative genetic medicines to patients [2][11]

CAMBRIDGE, Mass., Feb. 23, 2026 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the company will announce its fourth quarter and full year 2025 financial results and a new liver-targeted genetic disease program on an investor webcast on Tuesday, February 24, 2026, at 8:00 a.m. ET.The live webcast will be available in the investor section of the company's website at www.beamtx.com and will be ...

2 Transforming innovation into durable value creation Apellis: The Leading Complement Company 44th Annual J.P. Morgan Healthcare Conference Cedric Francois, M.D., Ph.D. Co-Founder, Chief Executive Officer and President January 12, 2026 ARCHER Living with C3G Forward-looking statements Statements in this presentation about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of ...

Core Insights - Beam Therapeutics has reached alignment with the U.S. FDA on a potential accelerated approval pathway for BEAM-302, a treatment for Alpha-1 Antitrypsin Deficiency (AATD), based on biomarker endpoints [1][4] - The company expects to submit a Biologics Licensing Application (BLA) for risto-cel (formerly BEAM-101) as early as the end of 2026 [1][13] - Beam's cash position is estimated at $1.25 billion as of December 31, 2025, providing an operating runway into 2029 to support the anticipated launch of risto-cel and the execution of the BEAM-302 pivotal development plan [10][11] Liver-targeted Genetic Disease Franchise - Beam is developing a platform for precision gene editing therapies targeting liver diseases, utilizing lipid-nanoparticle delivery systems [3] - BEAM-302 aims to be a first-in-class therapy for AATD, demonstrating clinical proof of concept in an ongoing Phase 1/2 trial with over 25 patients treated [4] - The company plans to enroll approximately 50 additional patients for the expansion of the Phase 1/2 study to support the BLA submission [4] Hematology Franchise - Risto-cel is an investigational autologous cell therapy for sickle cell disease (SCD), showing a differentiated treatment profile in the ongoing BEACON Phase 1/2 clinical trial [8] - The therapy has demonstrated a deeper resolution of SCD markers and reduced hospital time, with a robust manufacturing process [8] - Beam is also focusing on next-generation programs for SCD, including in vivo delivery strategies and the development of the ESCAPE platform for non-genotoxic treatments [9] Financial Position and Future Outlook - The estimated cash and marketable securities position of $1.25 billion will support Beam's operating expenses and capital requirements through 2029 [10][11] - The company is well-positioned to advance its pipeline of genetic therapies, with a focus on regulatory execution and clinical data generation [2][11] - Beam plans to announce a new program for its liver-targeted genetic disease franchise in the first half of 2026 [6]

Company Overview - Beam Therapeutics Inc. is a biotechnology company focused on developing precision genetic medicines through base editing [3] - The company aims to establish a leading, fully integrated platform for precision genetic medicines, incorporating gene editing, delivery, and internal manufacturing capabilities [3] - Beam's proprietary base editing technology allows for precise, predictable, and efficient single base changes in targeted genomic sequences without causing double-stranded breaks in DNA [3] Upcoming Event - John Evans, the CEO of Beam Therapeutics, will present at the 44th Annual J.P. Morgan Healthcare Conference on January 13, 2026, at 5:15 p.m. PT in San Francisco [1] - The live webcast of the presentation will be available on the company's investor section of the website and archived for 60 days [2]

Core Insights - Beam Therapeutics has announced updated safety and efficacy data for ristoglogene autogetemcel (risto-cel), a genetically modified cell therapy for sickle cell disease (SCD), showing significant clinical benefits and a durable response in patients [1][2] Efficacy and Safety Data - The BEACON Phase 1/2 trial included 31 patients with severe SCD, demonstrating a mean Hemoglobin F (HbF) induction of over 60% and a reduction in Hemoglobin S (HbS) to below 40% [1][3] - Patients experienced resolution of anemia, with total hemoglobin levels increasing rapidly and normalization of hemolysis markers [10] - No severe vaso-occlusive crises (VOCs) were reported post-engraftment, indicating a favorable safety profile [3][10] Treatment Process and Outcomes - Patients required a median of one stem cell collection cycle, with a median of three total collection days for manufacturing risto-cel, highlighting the efficiency of the process [3][5] - Rapid engraftment was observed, with a median time to neutrophil engraftment of 17.5 days and platelet engraftment at 19 days [3][10] - The treatment demonstrated durable, high editing efficiency, with mean peripheral blood editing rates of 67.4% at Month 6 and 72.8% at Month 12 [3][4] Company Overview - Beam Therapeutics focuses on precision genetic medicines through base editing technology, aiming to provide life-long cures for serious diseases [8] - The company is advancing a diversified portfolio of base editing programs, with risto-cel being a key investigational therapy for SCD [6][8]