Beam Therapeutics (NasdaqGS:BEAM) Update / briefing March 25, 2026 08:00 AM ET Speaker11Good morning, and welcome to Beam Therapeutics conference call. At this time, all participants are in 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.Speaker5Thank you, operator. Good morning, everyone, and ...

BEAM-302 Topline Data Update March 25, 2026 NASDAQ: BEAM 2 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 technology, including with respect to AATD; our plans, and anticipated timing, to advance our programs, including risto-cel, BEAM-103, BEAM-301, BEAM-304 and ...

Core Viewpoint - Beam Therapeutics Inc. is recognized as a leading gene therapy stock, with recent adjustments in price targets and strategic financing aimed at supporting its growth and commercialization efforts [1][2][3]. Financial Performance - Beam Therapeutics reported its fiscal Q4 and full-year 2025 results, highlighting a significant increase in cash and marketable securities from $850.7 million in 2024 to $1.25 billion in 2025 [3]. Strategic Developments - The company is targeting a Biologics License Application (BLA) submission for sickle cell disease within the year and has entered a non-dilutive debt agreement with Sixth Street to support this initiative [1][3]. - Beam Therapeutics is expanding its liver-targeted genetic disease franchise with a new program, BEAM-304, aimed at treating phenylketonuria (PKU), which affects approximately 20,000 individuals in the U.S. [2]. Research and Development Focus - The company is advancing several key programs, including BEAM-101, Engineered Stem Cell Antibody Paired Evasion (ESCAPE), BEAM-302, BEAM-301, and BEAM-201, with a focus on sickle cell disease and alpha-1 antitrypsin deficiency [4]. - Plans for 2026 include reporting updated Phase 1/2 data and preparing for a potential BLA submission for risto-cel by the end of the year [2].

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 financing facility, extending the runway into mid-2029 [40][41] - A strategic financing agreement was announced, providing up to $500 million in long-term, non-dilutive capital to support the commercialization of risto-cel, an investigational autologous cell therapy for sickle cell disease [39] Business Line Data and Key Metrics Changes - The company is expanding its portfolio with a new development program for phenylketonuria (PKU) called BEAM-304, which targets multiple disease-causing mutations [9][10] - The PKU program aims to demonstrate the scalability of the company's platform and deliver transformative therapeutic options to patients [10][17] Market Data and Key Metrics Changes - Approximately 20,000 individuals in the U.S. are affected by PKU, with a significant unmet need for new treatment options [19][12] - The company is leveraging its proprietary lipid nanoparticle (LNP) delivery system to target hepatocytes in the liver for the treatment of PKU [20][22] Company Strategy and Development Direction - The company aims to provide lifelong cures for patients suffering from serious diseases through its base editing technology, which allows for precise genetic modifications [5][6] - The strategic focus includes accelerating paths to approval for its lead programs and expanding its pipeline to address additional mutations and metabolic disorders [42][41] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the predictability of outcomes from their base editing platform, which is expected to streamline R&D and improve confidence among stakeholders [7][42] - The company is well-positioned to realize the potential of its growing portfolio, with plans to file the IND for BEAM-304 in 2026 and report initial data for BEAM-301 in GSD Ia [43][40] 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 anticipated risto-cel launch [41][39] - The regulatory landscape is evolving, with the FDA showing openness to innovative trial designs that could facilitate the development of therapies for ultra-rare diseases [64][70] 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 fast timelines due to the platform approach [48] - The strategic financing enhances the company's ability to redirect capital towards the growth of its pipeline [50] Question: Intellectual property and delivery mechanisms for BEAM-304 - The company confirmed access to necessary IP and will utilize similar LNP approaches as in previous programs for BEAM-304 [55] Question: FDA's draft guidance on individualized therapy - Management discussed the ambiguity regarding what qualifies as ultra-rare diseases under the new framework, emphasizing the need for case-by-case discussions with the FDA [62][66] Question: Responses of R408W carriers to current therapies - Management noted that R408W patients typically do not respond to existing therapies due to the lack of residual enzyme activity, highlighting the need for new treatment options [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][11] - 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][21] - 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][23] - General and Administrative (G&A) expenses for Q4 2025 were $32.3 million, up from $28.7 million in Q4 2024, with full-year G&A expenses totaling $113.8 million compared to $111.5 million in 2024 [18][23] Clinical Development - BEAM-304 utilizes Beam's proprietary base editing technology and lipid nanoparticle (LNP) delivery to correct mutations in the phenylalanine hydroxylase (PAH) gene, aiming to reduce toxic phenylalanine levels and normalize diet for PKU patients [3][4] - The initial clinical development of BEAM-304 will focus on patients with the R408W mutation, with plans to expand to additional mutations [6][11] - The company is also advancing BEAM-302 for AATD and BEAM-301 for Glycogen Storage Disease type Ia (GSDIa), with initial clinical data expected in 2026 [8][9][11] Strategic Partnerships - Beam has entered a strategic financing agreement with Sixth Street, which includes a $500 million senior secured credit facility to support the launch of risto-cel [11] - In December 2025, Pfizer opted for an exclusive worldwide license for a liver-targeted development candidate from Beam, which will involve milestone payments and a potential co-development agreement [11][18]

Company Overview - Beam Therapeutics aims to provide lifelong cures for patients with serious diseases through gene editing for both rare and common disorders, potentially leading to one-time curative therapies with lasting effects [3]. Recent Developments - In late 2024, Beam Therapeutics revealed significant data on base editing for severe sickle cell disease, demonstrating dramatic results in patient outcomes [4]. - In March, the company published its first data on base editing for alpha-1 antitrypsin deficiency using the BEAM-302 approach, marking a shift to in vivo editing [4]. Technology and Vision - The company emphasizes the power of predictability in its technology, which is designed to create a wide range of medicines over time, reflecting a remarkable year for base editing advancements [2][3].

Beam Therapeutics Conference Call Summary Company Overview - **Company**: Beam Therapeutics (NasdaqGS:BEAM) - **Industry**: Biotechnology, specifically gene editing for rare and common disorders Key Points and Arguments Vision and Technology - Beam Therapeutics aims to provide lifelong cures for patients with serious diseases through gene editing, focusing on one-time curative therapies with lasting effects [2][4] - The company utilizes base editing technology, which allows precise single-letter changes in genes without creating double-stranded breaks, leading to predictable and durable outcomes [4][5] Recent Achievements - In 2024, Beam presented significant data on base editing for severe sickle cell disease, demonstrating dramatic results [2][3] - The first data for base editing in alpha-1 antitrypsin deficiency (Beam 302) was published, showcasing the ability to rewrite a broken gene back to normal [3][12] Clinical Programs - **Beam 302**: Aimed at correcting the single-letter misspelling in the SERPINA1 gene responsible for alpha-1 antitrypsin deficiency, with the goal of restoring normal protein levels and addressing both lung and liver issues [15][16] - **Ristacel**: Targeting sickle cell disease, with a focus on achieving deeper resolution of the disease and improving patient outcomes [22][23] Regulatory Progress - Beam has received RMAT designation from the FDA, allowing for accelerated approval pathways for its therapies [18][19] - The company anticipates submitting a Biologics License Application (BLA) for Ristacel by the end of 2026 [29] Financial Position - Beam reported having $1.25 billion in cash at the end of the year, providing a runway into 2029 for the development and commercialization of its therapies [10][11] Market Potential - The company believes that its therapies will reduce lifetime healthcare costs and improve patient outcomes, making them attractive to payers [7][8] - There is significant patient interest and a growing number of treatment centers prepared to administer Beam's therapies [25][27] Future Directions - Beam plans to expand its pipeline with additional liver-targeted genetic disease programs and continue advancing its hematology franchise [33][34] - The company is also focusing on in vivo delivery methods to enhance scalability and reach more patients [30][31] Additional Important Information - Beam's base editing technology is adaptable and scalable, with a strong emphasis on manufacturing efficiency [9][43] - The company has established partnerships that have generated over $900 million in non-dilutive funding, enhancing its research capabilities [32] - Beam is committed to addressing unmet medical needs in rare diseases, with a focus on both therapeutic impact and commercial viability [56] This summary encapsulates the key points discussed during the conference call, highlighting Beam Therapeutics' innovative approach to gene editing, recent achievements, regulatory progress, financial strength, and future plans in the biotechnology sector.

Financial Position and Runway - Beam had $1.25 billion in cash as of December 31, 2025, inclusive of $255.1 million proceeds from the Orbital acquisition[16] - The company's operating runway is expected into 2029, funding through risto-cel launch and execution of BEAM-302 pivotal development plan[16,60] Program Advancements and Regulatory Alignment - Beam achieved first human proof of concept for in vivo gene correction with BEAM-302 in 2025[17] - Alignment was reached with the FDA on a potential accelerated approval pathway for BEAM-302 in AATD, with primary endpoint expected to be based on AAT biomarkers evaluated over 12 months[27] - The company plans to submit the risto-cel BLA package as early as YE 2026[45,60] Clinical Data and Trial Updates - BEAM-302 Phase 1/2 trial has dosed >25 patients across cohorts to date, with updated clinical data expected by the end of first quarter 2026[25,60] - Initial clinical data for BEAM-301 is planned to be reported by YE 2026[31,60] - BEACON results for risto-cel in SCD show HbF levels >60% and HbS levels <40%, comparable to sickle trait, with resolution of anemia and normalized or improved markers of hemolysis and oxygen delivery[37] - At Day 28, BEAM-302 showed mean total AAT in circulation at 12.4µM, mean % change in Z-AAT at -78%, and corrected M-AAT reached >90% of total circulating AAT[24] Strategic Initiatives - Beam completed a $500 million financing and runway extension into 2029[17] - The company intends to pursue an accelerated approval pathway for BEAM-302 based on FDA feedback to date[26]

Summary of Prime Medicine FY Conference Call Company Overview - **Company**: Prime Medicine (NasdaqGM:PRME) - **Focus**: Gene editing technology, specifically prime editing Key Points on Prime Editing Technology - **Definition**: Prime editing is described as the most versatile and safest method for genome editing, allowing for precise and permanent corrections in the DNA sequence [6][14] - **Comparison with Other Technologies**: - **CRISPR**: Effective for knocking out genes but has limitations such as off-target effects and potential immunogenicity [6][7][9] - **Base Editing**: Allows for single-letter changes in DNA but is limited to specific types of mutations [9][10][14] - **Prime Editing Advantages**: Capable of correcting larger mutations and multiple mutations simultaneously, with lower off-target effects [14][15] Industry Context - **Competitive Landscape**: Other companies, such as Tessera and Regeneron, are also developing prime editing technologies, indicating a competitive market [34] - **Market Need**: There is a significant unmet need for therapies targeting conditions like Wilson disease and alpha-1 antitrypsin deficiency, which are suitable for prime editing [32][33] Corporate Strategy and Pipeline - **Recent Changes**: The company underwent restructuring to focus on high-value areas, reducing the number of programs from 18 to prioritize those with higher probabilities of success [31][32] - **Key Programs**: - **Wilson Disease**: Targeting specific mutations with no existing gene editing therapies [32] - **Alpha-1 Antitrypsin Deficiency**: A competitive area where prime editing is believed to provide superior outcomes [33] - **Cystic Fibrosis and Ex Vivo CAR T Programs**: Continued focus on these areas, while deprioritizing ocular and neurological programs [38][40] Development and Clinical Trials - **IND Timeline**: The company aims to submit an Investigational New Drug (IND) application for Wilson disease in the first half of 2026, with proof of concept data expected by 2027 [48][49] - **Trial Design**: The Wilson disease trial will be a dose escalation study focusing on safety and efficacy measures, targeting patients with specific mutations [52][54] - **Biomarkers**: Various biomarkers will be used to assess treatment efficacy, including copper PET studies and ceruloplasmin levels [54] Off-Target Editing and Safety - **Off-Target Analysis**: Prime Medicine has conducted extensive off-target analysis for its programs, reporting no evidence of off-target effects in their first program for chronic granulomatous disease [16][31] - **Assay Development**: The company has developed its own assays for measuring off-target effects, which may differ from those used by competitors [17][30] Future Outlook - **Partnerships**: While the company is open to partnerships, it sees significant value in independently advancing its key programs [41][42] - **Long-Term Vision**: Prime Medicine aims to expand its portfolio beyond single products and diseases, leveraging its technology for a broader range of genetic conditions [41] Conclusion - Prime Medicine is positioned as a leader in the gene editing space with its prime editing technology, focusing on high-value therapeutic areas with significant unmet needs. The company is preparing for upcoming clinical trials and aims to establish itself as a key player in the competitive landscape of gene editing therapies.

Summary of Prime Medicine Conference Call Company Overview - **Company**: Prime Medicine (NasdaqGM:PRME) - **Technology**: Prime Editing, described as the most versatile and safest gene editing technology available, differentiating itself from CRISPR and base editing technologies [5][10][12] Key Points and Arguments Technology Differentiation - **CRISPR vs. Prime Editing**: CRISPR involves double-stranded breaks leading to potential off-target effects and chromosomal rearrangements, while Prime Editing allows for precise edits without unwanted off-target effects [8][10] - **Base Editing**: Base editing allows for single base pair changes but can result in bystander edits, which may not yield wild-type proteins [9][32] - **Prime Editing Advantages**: Capable of large multi-kilobase insertions and precise edits, returning to wild-type protein without unwanted effects [10][11] Universal LNP Technology - **LNP Differentiation**: Prime Medicine uses a unique ionizable lipid for LNP formulation, showing favorable preclinical profiles in terms of safety and potential dosing [12][13] Wilson's Disease Program - **Market Opportunity**: Targeting transversion mutations in Wilson's disease, a condition with high unmet medical need and limited competition [15][22] - **Clinical Development**: Plans to file IND/CTA in the first half of next year, with clinical data expected in 2027 [14][15] - **Biomarkers**: Utilizing copper PET scans and other biomarkers to assess treatment efficacy and potential to take patients off standard care [16][20] Alpha-1 Antitrypsin Deficiency (AATD) Program - **Clinical Timeline**: Expected to enter the clinic in mid-next year with data readout in 2027 [32] - **Comparison with Base Editing**: Prime Editing aims to provide wild-type protein correction, potentially leading to better patient outcomes compared to base editing [32][33] Cystic Fibrosis Program - **Collaboration with Cystic Fibrosis Foundation**: Utilizing both AAV and LNP technologies to address unmet needs in cystic fibrosis patients who do not respond to current therapies [42][43] - **Preclinical Data Timeline**: Anticipated preclinical data in 2026 [42] Other Important Insights - **Regulatory Strategy**: Discussions with the FDA regarding a plausible mechanism pathway to expedite approvals for multiple mutations under one IND [29][30] - **Business Development**: Potential for additional partnerships and collaborations to fund and advance various programs [45][46] - **Key Catalysts**: Focus on regulatory filings for Wilson's and AATD, patient enrollment, preclinical data for cystic fibrosis, and business development deals over the next 6-12 months [52][53] This summary encapsulates the critical aspects of Prime Medicine's conference call, highlighting the company's innovative gene editing technology, ongoing clinical programs, and strategic plans for future growth.