Question-and-Answer SessionGreat. So I was wondering if you could start off by providing an overview of the company and any upcoming catalysts that investors should keep in mind?John EvansCEO & Director Sure. So Beam Therapeutics is a next-generation gene editing company. We use an important form of CRISPR called base editing, in which we use the same precision targeting ability of CRISPR to edit any spot in the genome, but we've modified the system so that it doesn't make double-stranded breaks where you l ...

PresentationPatrick TrucchioMD of Equity Research & Senior Healthcare Analyst Great. Good morning, everyone, and thank you for joining H. C. Wainwright's 27th Annual Global Investment Conference, September 8 to September 10, 2025. My name is Patrick Trucchio. I'm the senior healthcare at H. C. Wainwright. It's my pleasure to introduce our next company, and next speaker. It's my pleasure to welcome Beam Therapeutics, a biotechnology company, leveraging its fully integrated precision genetic medicine platform ...

Beam Therapeutics Conference Call Summary Company Overview - **Company**: Beam Therapeutics - **Industry**: Biotechnology - **Focus**: Precision genetic medicine, specifically utilizing base editing technology for gene therapy Key Points and Arguments Base Editing Technology - Beam Therapeutics is pioneering a next-generation CRISPR gene editing technology known as base editing, which allows for precise single-letter changes in DNA without causing double-stranded breaks [2][5] - This technology enables the correction of single-letter mutations that can lead to genetic diseases, such as sickle cell disease and alpha-1 antitrypsin deficiency [6][10] Clinical Programs - **BEAM-101**: Focused on sickle cell disease, aiming to upregulate fetal hemoglobin through precise edits in the promoter region of genes [10][11] - Clinical proof of concept has been demonstrated, with 17 evaluable patients showing a trait-like HBF/HBS ratio, indicating a significant therapeutic effect [11] - The program has shown resolution of anemia and normalization of EPO levels, suggesting long-term functional benefits [13] - No vaso-occlusive crises (VOCs) have been reported in treated patients, indicating a robust transformation of their condition [14] - **Regulatory Strategy for BEAM-101**: Plans to follow a similar registration path as other approved therapies, with a single trial (Beacon trial) involving 50 patients, aiming for a BLA filing by late 2026 [16][18] - **BEAM-302**: Targets alpha-1 antitrypsin deficiency by correcting a single point mutation in the liver, which is crucial for producing a functional protein [40] - The dual-action therapy aims to stop the production of a toxic protein while simultaneously increasing the levels of a normal protein [41] - Initial data shows promising results, with patients achieving levels indicative of carriers, suggesting a potential curative effect [42] Future Programs - **Escape Technology**: Aims to treat a broader patient population with sickle cell disease by eliminating the need for chemotherapy in the treatment process [22][23] - This involves creating an ex vivo version that uses an antibody to remove old stem cells while ensuring the new edited cells remain unaffected [23] - The potential for in vivo applications is also being explored, which could further expand treatment options [24] Cost and Market Considerations - Current gene therapies for sickle cell disease are priced between $2 million to $3 million, justified by the significant lifetime costs associated with managing the disease [29][30] - The company anticipates that costs will decrease over time as production scales up, particularly for in vivo therapies [33][34] - The pricing model is supported by a broad alignment among government and payers, emphasizing the long-term savings from curing patients rather than ongoing treatment costs [31] Investor Outlook - Key upcoming milestones include updates on BEAM-101 at the ASH conference and continued operational progress on BEAM-302 [50] - The company is optimistic about the potential to impact a large number of patients with its innovative therapies [50] Additional Important Content - The company emphasizes the repeatability and predictability of its base editing platform, which could lower the risk and investment needed for future drug development [9] - The potential for BEAM-101 to compete with existing sickle cell treatments is highlighted, with improvements in manufacturing and patient outcomes being key differentiators [19][20] - The company is focused on expanding its pipeline to address various liver diseases using similar base editing approaches [49]

Beam Therapeutics (BEAM) FY 2025 Conference September 04, 2025 01:30 PM ET Speaker0Thanks, everyone, for being here. My name is Yann Anzou. I'm one of the biotech analysts here at Wells Fargo. It is my great pleasure to introduce our next fireside chat. We are privileged to have the Beam Therapeutics team with us. Here on stage are John Evans, CEO, and Pino Ciaramella, President of the company. Thank you, Pino, and thank you, John, for being here.Speaker2Thanks for having us.Speaker0Great. I was wondering i ...

Question-and-Answer SessionAbsolutely. So why don't we just jump right in then. Tell us a little bit about Beam. Tell us a little bit about some of your programs that you have going on, and then we can just dive deeper into each of them from there.John EvansCEO & Director Wonderful. So Beam is working on next-generation gene editing using CRISPR tools. Specifically, our lead technology is a technology called base editing, which allows us to precisely target the genome similar to other CRISPR companies. But ...

Summary of Beam Therapeutics Conference Call Company Overview - Beam Therapeutics is focused on next-generation gene editing using CRISPR tools, specifically base editing, which allows precise genome targeting without double-stranded breaks [4][5] Key Programs - **Hematology Franchise**: - Lead program BIM-101 targets severe sickle cell disease, aiming to provide a best-in-class option for patients [5] - Future versions aim to deliver functional cures with less invasive methods, avoiding chemotherapy [5] - **Liver Franchise**: - Lead program BEAM-302 targets alpha-one antitrypsin deficiency, showing strong evidence of correcting the causative mutation [6][8] - Additional programs include MUM-301 for glycogen storage disease and other liver-targeted genetic medicine programs [6] Financial Position - The company is well-financed with $1.2 billion in cash, sufficient to support its pipeline through significant milestones until 2028 [6] Clinical Data and Efficacy - **BEAM-302**: - Initial Phase I trial data showed a dose response with total alpha-one levels reaching approximately 12.4 micromolar, with 90% being the corrected form (M) and only 10% being the toxic form (Z) [12][13] - The Z protein was reduced by about 80%, indicating a significant therapeutic correction [13] - The treatment is designed to be a one-time durable correction, potentially providing lifelong benefits [16] Safety and Tolerability - The LNP delivery system and base editing have shown good safety profiles, with no significant adverse effects reported [15][20] - Ongoing studies will further explore dosing and safety in patients with liver involvement [31] Future Development Plans - The company plans to explore higher single doses and multi-dose cohorts to optimize efficacy while maintaining safety [18][20] - An update on data and future steps is expected in early 2026 [21][80] Competitive Landscape - Beam Therapeutics positions BEAM-302 as a potentially best-in-class option compared to augmentation therapies and RNA editing approaches, emphasizing its one-time, permanent solution [56][57] - The company acknowledges the presence of competitors but believes its approach offers significant advantages in terms of efficacy and durability [60] Market Considerations - The market for sickle cell disease treatments is complex, with reimbursement processes still being established [84][86] - Beam Therapeutics is optimistic about its market entry, drawing parallels to the CAR T-cell therapy market's evolution [88] Patient Demand and Community Response - There is strong demand from patients for a one-time curative approach, especially given the severity of diseases like alpha-one antitrypsin deficiency [69][70] - The company is actively working to identify and educate more patients about genetic testing and treatment options [72] Regulatory Pathway - Beam Therapeutics is considering accelerated approval pathways due to the clear therapeutic signal from its data [77][79] - The company is preparing for potential regulatory discussions to expedite patient access to its therapies [79] Conclusion - Beam Therapeutics is making significant progress in gene editing therapies, with promising clinical data and a strong financial position to support its innovative programs aimed at treating severe genetic diseases [99]

Group 1 - Beam Therapeutics Inc. is a biotechnology company focused on developing precision genetic medicines through base editing technology [2] - The company has a fully integrated platform that includes gene editing, delivery, and internal manufacturing capabilities [2] - 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 [2] Group 2 - Management will participate in several upcoming investor conferences, including Citi's 2025 Biopharma Back to School Conference and the Wells Fargo Healthcare Conference [3] - The live webcasts of these events will be available on the company's website and archived for 60 days [1]

Core Insights - Beam Therapeutics has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for BEAM-101, a genetically modified cell therapy aimed at treating sickle cell disease (SCD) [1][2] - The RMAT designation follows an earlier orphan drug designation and is expected to facilitate collaboration with the FDA as the company progresses towards a Biologics License Application (BLA) filing [2][4] - The BEACON Phase 1/2 trial has dosed 30 patients, with promising clinical data indicating robust increases in fetal hemoglobin and reductions in sickle hemoglobin [3][5] Company Overview - Beam Therapeutics is focused on developing precision genetic medicines using its proprietary base editing technology, which allows for precise genetic modifications without double-stranded breaks in DNA [7][8] - The company aims to create a fully integrated platform for gene editing, delivery, and manufacturing to provide long-term cures for serious diseases [7][8] Product Details - BEAM-101 is an investigational therapy that utilizes autologous CD34+ hematopoietic stem and progenitor cells, which are base-edited to enhance fetal hemoglobin production [5] - The therapy is designed to mimic naturally occurring variants that lead to increased fetal hemoglobin, potentially alleviating symptoms of SCD [5][6] Clinical Trial Insights - Updated clinical data from the BEACON trial presented at the EHA 2025 Congress showed durable increases in fetal hemoglobin and no vaso-occlusive crises reported post-engraftment [3][4] - The trial is ongoing, with additional data expected by the end of 2025, further supporting the potential of BEAM-101 as a transformative treatment for SCD [2][4]

Financial Performance - Beam Therapeutics reported a loss of $1.00 per share in Q2 2025, which was better than the Zacks Consensus Estimate of a loss of $1.04 and improved from a loss of $1.11 per share in the same quarter last year [1][6] - Total revenues for the second quarter were $8.5 million, down from $11.8 million year-over-year, and missed the consensus estimate of $14 million [1][6] - Research and development expenses increased by approximately 17% to $101.8 million compared to the previous year, while general and administrative expenses decreased by around 9.1% to $26.9 million [3] Cash Position - As of June 30, 2025, Beam Therapeutics had cash, cash equivalents, and marketable securities totaling $1.2 billion, unchanged from March 31, 2025, and is expected to fund operations into 2028 [3] Pipeline Developments - The company is advancing its ex-vivo genome-editing candidate BEAM-101 in a phase I/II BEACON study for sickle cell disease, with enrollment completed for both adult and adolescent cohorts [7] - BEAM-302 is being developed for alpha-1 antitrypsin deficiency (AATD) and is currently in a phase I/II dose-escalation study, with positive initial safety and efficacy data reported [8][9] - The phase I/II study for BEAM-302 consists of two parts, with ongoing dosing and enrollment in Part B for AATD patients with mild-to-moderate liver disease [10][11] Market Performance - Shares of Beam Therapeutics have declined by 24.6% year-to-date, contrasting with a 1.9% rise in the industry [2]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE TRANSITION PERIOD FROM TO Commission File Number 001-39208 Beam Therapeutics Inc. (Exact name of Registrant as specified in its Charter) FORM 10-Q (Mark One) Delaware 81-5238376 (State or other jurisdict ...