Beam Therapeutics Inc. · NASDAQ:BEAM
Beam Therapeutics — FY 2025 Earnings Presentation
39 slides from Beam Therapeutics's earnings presentation, Feb 24, 2026
Slide 1: Liver-targeted Genetic Disease Franchise Update and FY 2025 Earnings
Liver-targeted Genetic Disease Franchise Update and FY 2025 Earnings FEBRUARY 24, 2026 NASDAQ: BEAM
Slide 2: Beam event participants
Beam event participants TOPIC PARTICIPANT Holly Manning Introduction Vice President, Investor Relations & External Communications Beam Overview & Introduction to New John Evans Liver-targeted Genetic Disease Program Chief Executive Officer Amy Simon, M.D. PKU Disease Overview and Standard of Care Ch
Slide 3: Cautionary note regarding forward-looking statements
Cautionary note regarding forward-looking statements This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding: the therapeutic applications and potential of our techn
Slide 4: Our vision is to provide lifelong cures for patients suffering from serious diseases
Our vision is to provide lifelong cures for patients suffering from serious diseases GENE EDITING FOR POTENTIAL FOR PLATFORM FOR rare and common one-time, curative rapidly programmable diseases therapies precision medicines 4
Slide 5: Beam was founded on a simple concept with profound implications
Beam was founded on a simple concept with profound implications BASE EDITING TECHNOLOGY CONSISTENT gene sequence outcomes DURABLE correction for one-time cures Predictable, Reproducible Outcomes for LESS GENOTOXICITY than Patients traditional gene editing 5
Slide 6: Predictability as a driver of progress
Predictability as a driver of progress: The potential to ripple through the broader healthcare ecosystem Streamlined R&D Cycles • Reduced development risk Regulatory Acceleration • Efficient regulatory pathways Physician Confidence • Predictable safety and durability Predictable, • Clarity in treatm
Slide 7: The power of predictability: Beam is building a reliable model for advancing genetic medicine
The power of predictability: Beam is building a reliable model for advancing genetic medicine Easily ADAPTABLE to new targets Multiple growing, high-value franchises REUSABLE delivery technology PRECISION GENETIC CONSISTENT preclinical MEDICINES Active pipeline and clinical outcomes PLATFORM expansi
Slide 8: Rapidly advancing and growing a portfolio of liver-targeted in vivo programs
Rapidly advancing and growing a portfolio of liver-targeted in vivo programs for genetic diseases Potential Strategic Industry- Platform best-in-class pipeline leading LNP synergies well and first-in- expansion capabilities positioned for class AATD into PKU novel regulatory program pathways LEAD PR
Slide 9: PKU represents strategic expansion of the portfolio and ideal application of Beam's platform
PKU represents strategic expansion of the portfolio and ideal application of Beam’s genetic medicines platform Relevant Technology Large Addressable Existing and Novel and Expertise Market Regulatory Pathways • PKU often caused by point • Approximately 20,000 people • Bar for full phenylalanine muta
Slide 10: PKU: Disease Overview & Current Standard of Care
PKU: Disease Overview & Current Standard of Care Amy Simon, M.D., Chief Medical Officer 10
Slide 11: PKU is a genetic metabolic disease in which failure to metabolize phenylalanine leads to neurotoxicity
PKU is a genetic metabolic disease in which failure to metabolize phenylalanine leads to neurotoxicity PKU is caused by mutations in PAH gene, resulting in loss of PAH Classic activity, failure to metabolize Phe PKU and elevated Phe levels >1200 U.S. PKU patients identified with Moderate Neurotoxici
Slide 12: Significant impact on health and quality of life for people with PKU
Significant impact on health and quality of life for people with PKU, and most patients are not within recommended Phe levels • In children, very elevated Phe levels can lead to impaired brain development, intellectual Blood Phe Concentrations Increase with Age disability and seizures • In adults, w
Slide 13: PKU unmet need exists due to severe diet limitations and burden of available treatments
PKU unmet need exists due to severe diet limitations and burden of available treatments Most classical PKU patients can handle • Available therapies impose significant burden on only 5-10 g of protein/day from diet patients given the lifetime need for diet restrictions, poor taste of medical foods a
Slide 14: Target product profile for PKU gene therapy
Target product profile for PKU gene therapy driven by established regulatory precedents, the literature and clinician feedback • Blood Phe has been an accepted surrogate endpoint for Significant and sustained reduction in Phe levels (<360μM) patients with PKU for full approval in U.S. and EU Target
Slide 15: Base Editing as a Potential One-time Treatment Option for PKU
Base Editing as a Potential One-time Treatment Option for PKU Gopi Shanker, Ph.D., Chief Scientific Officer 15
Slide 16: Beam has potential to address vast majority of patients with PKU
Beam has potential to address vast majority of patients with PKU, leveraging an efficient development approach ~20,000 PKU Individuals in U.S. Additional • Two identified development mutations candidates have potential to R408W, 33% address nearly half of PKU patients Mutation J • Ongoing research e
Slide 17: BEAM-304 program uses base editing to directly correct multiple mutations causing PKU
BEAM-304 program uses base editing to directly correct multiple mutations causing PKU BEAM BASE EDITOR CRISPR Protein Deaminase Guide RNA (mutation -specific) PAH Mutation Corrected PAH gene 17
Slide 18: Beam has established leading LNP capabilities for in vivo treatment of liver-targeted genetic diseases
Beam has established leading LNP capabilities for in vivo treatment of liver-targeted genetic diseases Advantages of LNP delivery Beam's LNP Leadership Position ✓ Outpatient dosing via simple • Significant investment and expertise IV infusion in LNP optimization and process ✓ Titratable and redosabl
Slide 19: Collaboration with the Musunuru lab at UPenn to bring base editing to patients with PKU
Collaboration with the Musunuru lab at UPenn to bring base editing to patients with PKU • Musunuru lab established preclinical proof-of- concept for base editing in PKU • Beam leveraged expertise and platform capability to rapidly advance program to IND in <2 years 19
Slide 20: Preclinical data demonstrate the potential of BEAM-304 to correct underlying PKU mutations
Preclinical data demonstrate the potential of BEAM-304 to correct underlying PKU mutations and rapidly normalize plasma Phe R408W MUTATION MUTATION B Control Control 0.3 mg/kg 0.3 mg/kg 2500 2500 plasma Phe (μmol/L) plasma Phe (μmol/L) 2000 2000 1500 1500 1000 1000 500 Therapeutic 500 Therapeutic th
Slide 21: Low-level editing sufficient to normalize plasma Phe in mouse models
Low-level editing sufficient to normalize plasma Phe in mouse models at clinically relevant doses Therapeutic threshold Source: Beam internal data 21
Slide 22: BEAM-304 Phase 1/2 trial in PKU patients designed to achieve early clinical proof of concept
BEAM-304 Phase 1/2 trial in PKU patients designed to achieve early clinical proof of concept PHASE 1/2 Open-label, single-ascending dose trial • Beam will initially focus DOSE DOSE development of BEAM- EXPLORATION EXPANSION 304 in U.S. • Potential to rapidly KEY ENDPOINTS expand to other mutations •
Slide 23: Initial learnings from BEAM-304 in R408W expected to provide predictable path for future mutations
Initial learnings from BEAM-304 in R408W expected to provide predictable path for accelerated development for future mutations R408W Other mutations Base editor mRNA Internally developed LNP Potential to Manufacturing process accelerate clinical trial, Regulatory pathway BLA filing, launch and Clini
Slide 24: Emerging Approaches to Developing Customized Genetic Medicines
Emerging Approaches to Developing Customized Genetic Medicines Kiran Musunuru, M.D., Ph.D., University of Pennsylvania 24
Slide 25: Poor metabolic control in PKU patients
Poor metabolic control in PKU patients 2000 Blood phenylalanine (µmol/L) 1500 Rebecca Ahrens-Nicklas Children’s Hospital of Philadelphia (CHOP) 1000 500 Guidelines maximum phenylalanine level 0 H 1 H 2 H 3 H 4 H 5 H 6 H 7 H 8 H t9 H 10 H 11 H 12 H 13 H 14 H 15 H 16 H 17 H 18 H 19 H 20 H 21 H 22 H 23
Slide 26: LNP base editing treatment of humanized PKU mice (P281L)
LNP base editing treatment of “humanized” PKU mice (P281L) LNPs with adenine LNPs base editor with ABE8.8 mRNA mRNA + P281L-specific + PAH1 guide RNA gRNA - short term 2500 Blood Phe levels 2000 (micromol/L) 1500 PKU mice treated with LNPs 1000 PKU mice untreated non-PKU mice untreated 500 0 0 1 2 3
Slide 27: Pathogenic PAH variants in PKU patients
Pathogenic PAH variants in PKU patients What about the 1,000+ other cataloged variants? And all the variants not yet cataloged … Hillert et al. Am J Hum Genet 2020;107:234-50
Slide 28: Newborn patients with grievous inborn errors of metabolism
Newborn patients with grievous inborn errors of metabolism Urea cycle disorders Hyperammonemia episodes, starting immediately after birth, cause progressive brain injury and early death Liver transplantation—if available—has to be deferred until late infancy and has substantial morbidity and mortali
Slide 29: Interactions with the FDA
Interactions with the FDA PKU Pre-IND: Major question: Could many different variant-specific versions of an LNP- editor drug product targeting a single gene be included in a single IND? Phenylketonuria (PKU) INTERACT: Answers: • A single IND application is appropriate. Major question: Could a single
Slide 30: Umbrella clinical trial for PKU – pre-specified variants
Umbrella clinical trial for PKU – pre-specified variants Cross- reference Umbrella clinical trial Single IND To add PKU variant N: IND amendment with in vitro data for each variant- specific guide RNA Ahrens-Nicklas & Musunuru. Am J Hum Genet 2025;112:2826-9
Slide 31: Umbrella clinical trial for PKU – rapid addition of new variants
Umbrella clinical trial for PKU – rapid addition of new variants Cross- reference Umbrella clinical trial Single IND To add PKU variant N: To add PKU IND amendment withvariant in vitroN:data IND amendment for each variant- with in vitro specific data guide for each RNAvariant- specific guide RNA Ahr
Slide 32: Master protocol clinical trial for urea cycle disorders
Master protocol clinical trial for 7 urea cycle disorders Cross-referencing Primary IND with master protocol of primary IND for clinical trial for infantile-onset urea cycle disorders OTC Deficiency Gene-specific INDs Arginase Deficiency To add variant N: ORNT1 amendment to Deficiency gene-specific
Slide 33: Financial Update
Financial Update Sravan Emany, Chief Financial Officer 33
Slide 34: Beam announces $500 million strategic financing facility with Sixth Street
Beam announces $500 million strategic financing facility with Sixth Street to support risto-cel launch Balance sheet strengthened $100 million funded at close with long-term, non-dilutive capital with the potential to draw up to • Flexible repayment mechanics an additional $400 million during the se
Slide 35: Establishing a foundation of financial strength for sustainable growth
Establishing a foundation of financial strength for sustainable growth Clear Path to Value and Financial Strength Focused Investment Sustainable Growth • With $1.25 billion in cash as of • Optimized spend and • Wholly owned pipeline Dec. 31, 2025* and the expense management anticipated $200 million
Slide 36: Wrap up
Wrap up 36
Slide 37: PKU illustrates the power and potential of Beam's genetic medicines platform
PKU illustrates the power and potential of Beam’s genetic medicines platform Base editing addresses the Rapid adaptation for additional underlying genetic cause of mutations, enabled by novel disease regulatory pathways Precision medicine with potential Large initial addressable market for early POC
Slide 38: Beam is well positioned to realize the power of predictability in 2026
Beam is well positioned to realize the power of predictability in 2026 through key anticipated milestones Pursue Path to Approval Advance and Expand Maintain for Lead Programs Pipeline Financial Strength • Report updated Phase • File IND for BEAM-304 • $1.25 billion in cash as 1/2 data for BEAM-302
Slide 39: Q&A
Q&A 39