Apellis Pharmaceuticals (NasdaqGS:APLS) Q4 2025 Earnings call February 24, 2026 08:30 AM ET Company ParticipantsAnupam Rama - Biotechnology Equity ResearchCaroline Baumal - CMOCedric Francois - Co-Founder, CEO, and PresidentDavid Acheson - EVP of CommercialDouglas Tsao - Managing Director of Equity ResearchEllie Merrill - Biotechnology Equity Research AssociateEva Stroynowski - Investor Relations and Corporate Affairs Biotechnology ExecutiveJayad Momin - Biopharma Equity Research AssociateJonathan Miller - ...

Beam Therapeutics (NasdaqGS:BEAM) Q4 2025 Earnings call February 24, 2026 08:00 AM ET Company ParticipantsAditya Lonial - Senior Managing DirectorAlec Stranahan - VP of Equity ResearchAmy Simon - Chief Medical OfficerGiuseppe Ciaramella - PresidentGopi Shanker - Chief Scientific OfficerHolly Manning - Vice President of Investor Relations and External CommunicationsJohn Evans - CEOKiran Musunuru - Professor of MedicineSravan Emany - CFOYannan Liu - Equity Research AssociateConference Call ParticipantsBrian C ...

Beam Therapeutics (NasdaqGS:BEAM) Q4 2025 Earnings call February 24, 2026 08:00 AM ET Speaker14Good morning, and welcome to Beam Therapeutics conference call. At this time, all participants are in a listen-only mode. There will be a question and answer session at the end. Please be advised that this call is being recorded at Beam's request. I would now like to turn the call over to Holly Manning, Vice President of Investor Relations and External Communications. Please go ahead.Speaker9Thank you, operator. G ...

New Program Designed as Platform-based Approach for Direct Correction of Mutations Causing PKU; Investigational New Drug (IND) Filing for BEAM‑304 Anticipated in 2026 Updated Phase 1/2 Data and Next Steps for Pivotal Development for BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD) on Track for Q1 2026 Strategic Financing Agreement with Sixth Street Provides up to $500 Million in Long-term, Non-dilutive Capital to Fund Anticipated Launch of Risto-cel in Sickle Cell Disease (SCD); U.S. Biologics License Appl ...

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]

Core Insights - Beam Therapeutics Inc. is participating in the 2025 Jefferies London Healthcare Conference on November 19, 2025, at 3:30 p.m. GMT [1] - The live webcast of the event will be available on the company's investor website and archived for 60 days [2] Company Overview - Beam Therapeutics is a biotechnology company focused on precision genetic medicines through base editing [3] - The company has developed an integrated platform that includes gene editing, delivery, and internal manufacturing capabilities [3] - Beam's proprietary base editing technology allows for precise and efficient single base changes in targeted genomic sequences without causing double-stranded breaks in DNA [3] - The company aims to advance a diversified portfolio of base editing programs to provide life-long cures for patients with serious diseases [3]