Beam Therapeutics Inc.: Q4 FY2025 earnings call

What management said about Q4 FY2025, from the documents filed with the SEC on 24 February 2026: press release. Guidance figures: 1 raised, 3 new, 5 kept. The quarter’s numbers.

Key takeaways

Quarter net income reflected a gain: Net income attributable to common stockholders was $244.3 million, or $2.33 per diluted share, in the quarter. The statement of operations shows a $255.1 million gain on sale of an equity method investment. Source
Collaboration revenue rose sharply: License and collaboration revenue was $114.1 million in the quarter, against $30.1 million a year earlier, and $139.7 million for the year, against $63.5 million. Source
BEAM-304 targets PKU mutations: BEAM-304 uses base editing and LNP delivery to correct PAH gene mutations behind PKU. Initial clinical work targets the two most prevalent variants, found in nearly half of US patients. Source
BEAM-302 accelerated approval path discussed: Accelerated approval would rest on AAT biomarkers over 12 months, a pathway the company describes as potential. About 50 more patients are planned in an expansion. Source
Risto-cel BLA as early as 2026: Management expects a risto-cel BLA as early as year-end 2026. Manufacturing of all clinical doses in the BEACON Phase 1/2 trial is complete. Source
Cash rose to $1.25 billion: Cash, cash equivalents and marketable securities were $1.25 billion at December 31, 2025, up from $850.7 million a year earlier. Source
Sixth Street facility terms: The $500 million senior secured facility has $100 million funded at close and $300 million tied to risto-cel milestones. A further $100 million is at Beam's option, with principal due by early 2033. Source

“In 2025, we established base editing as a best-in-class technology for genetic medicine, with positive proof-of-concept data and regulatory and clinical development paths to approval across multiple high-value programs,”
John Evans, chief executive officer of Beam Therapeutics

Guidance

Area	Period	Figure	Against last quarter
Cash runway Source	Into mid-2029	into mid-2029	Raised from into 2029
Sixth Street drawdown Source	Under the facility	minimum of $200 million	New
Sixth Street principal repayment Source	Seven-year term	by early 2033	New
BEAM-302 updated data and pivotal plan Source	End of first quarter 2026	by the end of the first quarter of 2026	Kept
BEAM-302 expansion enrollment Source	Expansion of Phase 1/2 study	approximately 50 additional patients	Kept
Risto-cel BLA submission Source	Year-end 2026	as early as year-end 2026	Kept
BEAM-304 IND filing Source	2026	in 2026	New
BEAM-301 initial clinical data Source	2026	in 2026	Kept
BEAM-103 Phase 1 dosing Source	First half of 2026	complete dosing in the first half of 2026	Kept

What changed from last quarter

Guidance raised: Cash runway was extended to mid-2029, from into 2029 in the prior release, and now includes the $200 million minimum drawdown. Source
New: A strategic financing agreement with Sixth Street was announced with these results, providing non-dilutive capital for risto-cel. Source
New: Pfizer opted in to an exclusive worldwide license for a liver-targeted development candidate, at the end of their research collaboration in December 2025. Source
New: The BEAM-304 PKU program was announced in this release. The January release had said the next liver-targeted program would be announced in the first half of 2026. Source
New guidance: An IND filing for BEAM-304 in 2026 is new guidance in this release. Source
Guidance kept: The timing for updated BEAM-302 Phase 1/2 data and pivotal next steps is unchanged at the end of the first quarter of 2026. Source
Ended: The January plan to disclose the next liver-targeted program in the first half of 2026 is completed by the BEAM-304 announcement.

Results and net income

Quarter net income reflected a gain: Net income attributable to common stockholders was $244.3 million, or $2.33 per diluted share, in the quarter. The statement of operations shows a $255.1 million gain on sale of an equity method investment. Source
Collaboration revenue rose sharply: License and collaboration revenue was $114.1 million in the quarter, against $30.1 million a year earlier, and $139.7 million for the year, against $63.5 million. Source
Full-year net loss narrowed: The net loss for 2025 was $80.0 million, or $0.81 per share, compared with $376.7 million, or $4.58 per share, in 2024. Source
Operating loss narrowed for the year: Loss from operations was $383.7 million for 2025, against $415.6 million for 2024. The fourth-quarter operating loss was $17.4 million, against $100.0 million a year earlier. Source

Operating costs

R&D spending rose for the year: R&D expenses were $409.6 million for 2025, up from $367.6 million in 2024. Fourth-quarter R&D was $99.3 million, against $101.4 million a year earlier. Source
G&A rose in quarter and year: G&A expenses were $32.3 million in the fourth quarter, against $28.7 million a year earlier, and $113.8 million for 2025, against $111.5 million for 2024. Source
Total operating expenses for the year: Total operating expenses were $523.4 million for 2025, against $479.1 million in 2024, and $131.6 million in the fourth quarter, against $130.1 million. Source

“The announcement of BEAM-304 for PKU marks an important expansion of our pipeline and exemplifies the power and scalability of our platform.”
John Evans, chief executive officer of Beam Therapeutics

Liver-targeted franchise and PKU

BEAM-304 targets PKU mutations: BEAM-304 uses base editing and LNP delivery to correct PAH gene mutations behind PKU. Initial clinical work targets the two most prevalent variants, found in nearly half of US patients. Source
Preclinical Phe reduction in mice: Preclinical data showed BEAM-304 normalized plasma Phe levels in mouse models at clinically relevant doses, with robust on-target editing in the liver. Source
BEAM-302 accelerated approval path discussed: Accelerated approval would rest on AAT biomarkers over 12 months, a pathway the company describes as potential. About 50 more patients are planned in an expansion. Source
BEAM-301 dosing progressed in GSDIa: BEAM-301 for GSDIa has completed dosing in its first cohort, and enrollment has begun in the second cohort. Initial clinical data are expected in 2026. Source

Hematology and risto-cel

Risto-cel BLA as early as 2026: Management expects a risto-cel BLA as early as year-end 2026. Manufacturing of all clinical doses in the BEACON Phase 1/2 trial is complete. Source
Risto-cel data profile described: Updated BEACON data presented at ASH were described as showing deep resolution of SCD markers, reduced hospitalization and rapid engraftment, with a predictable manufacturing process. Source
BEAM-103 healthy volunteer dosing: The Phase 1 healthy volunteer trial of BEAM-103, an anti-CD117 antibody that enables ESCAPE, is expected to complete dosing in the first half of 2026. Source

“Supported by a balance sheet that was further strengthened through the non-dilutive financing with Sixth Street and anticipated runway into mid-2029, we believe Beam is well positioned to deliver on our clinical, regulatory, and commercial objectives”
John Evans, chief executive officer of Beam Therapeutics

Cash, balance sheet and financing

Cash rose to $1.25 billion: Cash, cash equivalents and marketable securities were $1.25 billion at December 31, 2025, up from $850.7 million a year earlier. Source
Orbital sale cash included: The cash total includes $255.1 million of closing consideration from Bristol-Myers Squibb's acquisition of Orbital Therapeutics. Up to $26.3 million more may be received from escrows.
Sixth Street facility terms: The $500 million senior secured facility has $100 million funded at close and $300 million tied to risto-cel milestones. A further $100 million is at Beam's option, with principal due by early 2033. Source
Year-end balance sheet totals: Total assets were $1.48 billion and total liabilities $242.8 million at December 31, 2025, against stockholders' equity of $1.24 billion. Source

Partnerships and development approach

Pfizer took liver candidate license: At the end of a four-year research collaboration, Pfizer took an exclusive worldwide license to a liver-targeted candidate using Beam's LNP. Beam is eligible for milestone payments. Source
Option to co-develop with Pfizer: After Phase 1/2, Beam may opt in for an option fee and share net profits and costs with Pfizer in a 35%/65% split, Beam to Pfizer. Source
Multiple base editors in one trial: Management said BEAM-304 advances multiple base editors within a single clinical program, aiming to address different PKU patient populations. Source

Coming up, as management said on 24 February 2026

BEAM-302 data and pivotal plan: Updated Phase 1/2 BEAM-302 data and next steps for pivotal development are due by the end of the first quarter of 2026. Source
BEAM-304 IND filing: An IND application for BEAM-304 is planned for 2026, following completion of pre-IND activities. Source
BEAM-301 initial clinical data: Initial clinical data for BEAM-301 in glycogen storage disease type Ia are expected in 2026. Source
Risto-cel BLA submission: A BLA for risto-cel in sickle cell disease could be submitted as early as year-end 2026. Source

Figures management reported

Figure	Reported
R&D expenses Source	$409.6 million full year 2025; $99.3 million fourth quarter 2025
Pipeline milestones Source	BEAM-302 updated Phase 1/2 data by end of Q1 2026; risto-cel BLA as early as year-end 2026; BEAM-304 IND in 2026

Risks management discussed

Regulatory: The FDA accelerated approval pathway for BEAM-302 is described as potential, and it rests on biomarker data over 12 months and roughly 50 more patients. Source
Financial: Part of the Sixth Street facility depends on risto-cel milestones, and the $100 million option requires mutual agreement with Sixth Street. Source

Earlier plans management reported on

On track: The release headline states the BEAM-302 update is on track for the first quarter of 2026. Source
Not stated: The January release said the next liver-targeted program would be disclosed in the first half of 2026. BEAM-304 is announced in this release.
Not stated: The company continues to expect a risto-cel BLA as early as year-end 2026, the same timing given in the January release. Source

Other quarters

Summarised with AI from the documents the company filed with the SEC. Every point is checked against the filing before it is shown: management’s own words word for word, figures against the line or table row they come from. A point that fails the check is left out. Source opens the filing at that line.

As filed with the SEC

Open the filing on EDGAR

Beam Therapeutics Reports Fourth Quarter and Year-End 2025 Financial Results and Announces New Liver-Targeted Genetic Disease Program in Phenylketonuria (PKU)

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 Application (BLA) Submission Expected as Early as Year-End 2026

Expected Cash Runway Now into Mid-2029 Through Execution of Key Clinical, Regulatory and Commercial Milestones

Beam to Host Investor Webcast Today, February 24, 2026, at 8:00 a.m. ET

Cambridge, Mass., February 24, 2026 - Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today reported financial results for the fourth quarter and year ended December 31, 2025, and reiterated 2026 milestones. In addition, the company announced the expansion of its liver-targeted genetic disease franchise with a new program, BEAM-304, for the treatment of phenylketonuria (PKU), a disease with significant unmet need that affects approximately 20,000 individuals in the U.S.

“In 2025, we established base editing as a best-in-class technology for genetic medicine, with positive proof-of-concept data and regulatory and clinical development paths to approval across multiple high-value programs,” said John Evans, chief executive officer of Beam Therapeutics. “The announcement of BEAM-304 for PKU marks an important expansion of our pipeline and exemplifies the power and scalability of our platform. By combining our clinically validated base editing technology with our internally discovered and optimized lipid nanoparticle (LNP) delivery, we have the potential to bring forward one-time, durable treatments for the vast majority of PKU patients. Moreover, with BEAM-304 we are pursuing an innovative and efficient development approach designed to advance multiple base editors within a single clinical program to address different PKU patient populations - one of the first such programs to reach the clinic.”

“As we look ahead to 2026, our focus is on execution across our most advanced programs, including reporting updated Phase 1/2 data and further defining the pivotal path forward for BEAM-302 and preparing for a potential BLA submission for risto-cel as early as year-end. Supported by a balance sheet that was further strengthened through the non-dilutive financing with Sixth Street and anticipated runway into mid-2029, we believe Beam is well positioned to deliver on our clinical, regulatory, and commercial objectives and to bring transformative genetic medicines to patients.”

New Liver-targeted Genetic Disease Program: BEAM-304 for the Treatment of PKU

Beam’s newest liver-targeted genetic disease program, BEAM-304, leverages Beam’s proprietary and clinically validated base editing technology and lipid nanoparticle (LNP) delivery capabilities to directly and durably correct mutations in the phenylalanine hydroxylase (PAH) gene that cause PKU. PKU is a rare, inherited metabolic disorder that results in toxic accumulation of phenylalanine (Phe), leading to serious neurologic and neurocognitive impairments and lifelong dietary management. By correcting mutations in the PAH gene, BEAM-304 aims to reduce toxic Phe to within recommended guidelines while enabling normalization of diet and freedom from medical food.

Beam is advancing BEAM-304 using an innovative development approach in which multiple mutation-specific base editors are developed efficiently within a single clinical program. With this approach, Beam’s platform has the potential to create transformative, one-time therapies for the vast majority of patients with PKU. Initial clinical development will focus on base editors addressing the two most prevalent variants found in nearly half of patients with PKU in the U.S., with ongoing research effort to address additional pathogenic mutations. Preclinical data with both base editors demonstrate that BEAM-304 normalized plasma Phe levels in mouse models at clinically relevant doses with robust on-target editing in the liver.

Beam expects to file an investigational new drug (IND) application with the U.S. Food and Drug Administration (FDA) for BEAM-304 in 2026 following completion of pre-IND activities. The planned Phase 1/2 trial will initially evaluate safety, tolerability, and reduction of blood Phe levels in PKU patients with the R408W mutation, followed thereafter by a base editor for a second mutation, with a goal of establishing clinical proof of concept for base editing in PKU and laying the foundation for future expansion to patients with additional PAH mutations.

Recent Highlights and 2026 Anticipated Milestones

Corporate

Today, Beam announced that it has entered into a strategic financing agreement with Sixth Street for significant, long-term, non-dilutive capital to fund the potential launch of risto-cel in sickle cell disease (SCD). The $500 million senior secured credit facility includes: $100 million funded at close; an additional $300 million available following the achievement of certain clinical, regulatory, and commercial milestones for risto-cel; and an additional $100 million available at Beam’s option, subject to mutual agreement between Sixth Street and Beam, during the seven-year term of the agreement, with principal repayment due by early 2033. Beam is expecting to draw a minimum of $200 million of capital under the overall facility.

In December 2025, at the completion of the four-year research collaboration agreement between Pfizer and Beam focused on in vivo base editing programs, Pfizer opted in to an exclusive, worldwide license for a liver-targeted development candidate. The development candidate employs Beam’s proprietary, liver-targeting LNP to deliver base editing reagents. In connection with the opt-in, Pfizer will take an exclusive, worldwide license to the development candidate, after which it will be responsible for all development activities, as well as potential regulatory approvals, manufacturing, and commercialization. Beam will be eligible for development, regulatory and commercial milestone payments and will have a right to opt in, at the end of Phase 1/2 clinical trials, upon the payment of an option exercise fee, to a global co-development and co-commercialization agreement pursuant to which Beam and Pfizer would share net profits as well as development and commercialization (including manufacturing) costs in a 35%/65% ratio (Beam/Pfizer).

Liver-targeted Genetic Disease Franchise

BEAM-302: Beam’s lead genetic disease program is designed to be a best-in-class and first-in-class liver-targeting therapy for alpha-1 antitrypsin deficiency (AATD) that addresses the underlying pathophysiology of both liver and lung disease.

In January, Beam shared that it has reached alignment with the FDA on a potential accelerated approval pathway for BEAM-302 based on AAT biomarkers evaluated over 12 months. To support a future biologics license application (BLA) submission, the company anticipates enrolling approximately 50 additional patients to be treated with the selected optimal biological dose of BEAM-302 in an expansion of the ongoing Phase 1/2 study.

Beam expects to report updated data from the Phase 1/2 trial and next steps for pivotal development by the end of the first quarter of 2026.

BEAM-301: BEAM-301 aims to correct the most common disease-causing mutation, R83C, in patients with glycogen storage disease type Ia (GSDIa).

BEAM-301 is currently being evaluated in an open-label Phase 1/2 dose-exploration trial in patients with GSDIa. Dosing is complete in the first cohort and enrollment has been initiated in the second cohort.

Beam expects to report initial clinical data in 2026.

Hematology Franchise

Risto-cel: Ristoglogene autogetemcel (risto-cel, formerly known as BEAM-101) is an investigational autologous cell therapy with a potential best-in-class profile for the treatment of SCD.

Updated data from the ongoing BEACON Phase 1/2 trial presented at the 67th American Society of Hematology (ASH) Annual Meeting continue to demonstrate risto-cel’s differentiated profile, including deep resolution of SCD markers, reduced hospitalization, rapid engraftment, and a predictable manufacturing process that may improve patient experience and treatment center capacity and reduce the length of the transplant process.

Manufacturing of all clinical doses in the BEACON Phase 1/2 trial is complete.

Beam expects to submit a BLA for risto-cel as early as year-end 2026.

Next-generation Programs in Sickle Cell Disease and Hematology:

The ongoing Phase 1 healthy volunteer clinical trial of BEAM-103, an anti-CD117 monoclonal antibody that enables ESCAPE, is expected to complete dosing in the first half of 2026.

Fourth Quarter and Full-year 2025 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $1.25 billion as of December 31, 2025, compared to $850.7 million as of December 31, 2024.

Research & Development (R&D) Expenses: R&D expenses were $99.3 million for the fourth quarter of 2025 and $409.6 million for the full year ended December 31, 2025, compared to $101.4 million for the fourth quarter of 2024 and $367.6 million for the full year ended December 31, 2024.

General & Administrative (G&A) Expenses: G&A expenses were $32.3 million for the fourth quarter of 2025 and $113.8 million for the full year ended December 31, 2025, compared to $28.7 million for the fourth quarter of 2024 and $111.5 million for the full year ended December 31, 2024.

Net Income (Loss): Net income attributable to common stockholders was $244.3 million, or $2.37 per basic share and $2.33 per diluted share, for the fourth quarter of 2025 and net loss attributable to common stockholders was $80.0 million, or $0.81 per share, for the year ended December 31, 2025, compared to net losses of $90.4 million, or $1.09 per share, for the fourth quarter of 2024 and $376.7 million, or $4.58 per share, for the full year ended December 31, 2024.

Cash Runway

Beam expects that its cash, cash equivalents and marketable securities as of December 31, 2025, combined with the anticipated $200 million minimum drawdown from the Sixth Street facility, will enable the company to cover its anticipated operating expenses and capital expenditure requirements into mid-2029, funding the company through the anticipated launch of risto-cel in SCD, execution of the BEAM-302 pivotal development plan in AATD, and clinical proof of concept for BEAM-304 in PKU.

Investor Webcast Information Beam will host a conference call and webcast today, February 24, 2026, at 8:00 a.m. ET to review the PKU program, Sixth Street facility, and fourth quarter and year-end 2025 financial results. A live webcast of the presentation will be available under "Events" in the Investors section of the company's website at www.beamtx.com, and a replay will be available shortly after the event.

About Beam Therapeutics

Beam Therapeutics (Nasdaq: BEAM) is a biotechnology company committed to establishing the leading, fully integrated platform for precision genetic medicines. To achieve this vision, Beam has assembled a platform with integrated gene editing, delivery and internal manufacturing capabilities. Beam’s suite of gene editing technologies is anchored by base editing, a proprietary technology that is designed to enable precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This has the potential to enable a wide range of potential therapeutic editing strategies that Beam is using to advance a diversified portfolio of base editing programs. Beam is a values-driven organization committed to its people, cutting-edge science, and a vision of providing lifelong cures to patients suffering from serious diseases.

Cautionary Note Regarding Forward-Looking Statements

Forward-looking statements notice

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Investors are cautioned not to place undue reliance on these forward-looking statements, including, but not limited to, statements related to: the therapeutic applications and potential of our technology, including with respect to SCD, AATD, PKU, ESCAPE and GSDIa; our plans, and anticipated timing, to advance our programs and present data from ongoing clinical trials; the clinical trial designs and expectations for risto-cel, BEAM-103, BEAM-301, BEAM-302 and BEAM-304; our anticipated regulatory interactions and filings; the sufficiency of our capital resources to fund operating expenses and capital expenditure requirements and the period in which such resources are expected to be available; and our ability to develop lifelong, curative, precision genetic medicines for patients through base editing. Each forward-looking statement is subject to important risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including, without limitation, risks and uncertainties related to: our ability to develop, obtain regulatory approval for, and commercialize our product candidates, which may take longer or cost more than planned; our ability to raise additional funding, which may not be available; our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; the uncertainty that our product candidates will receive regulatory approval necessary to initiate or continue human clinical trials; that preclinical testing of our product candidates and preliminary or interim data from preclinical studies and clinical trials may not be predictive of the results or success of ongoing or later clinical trials; that initiation and enrollment of, and anticipated timing to advance, our clinical trials may take longer than expected; that our product candidates, including the delivery modalities we rely on to administer them, may cause serious adverse events; that our product candidates may experience manufacturing or supply interruptions or failures; risks related to competitive products; and the other risks and uncertainties identified under the headings “Risk Factors Summary” and “Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2024, our Quarterly Report on Form 10-Q for the quarter ended September 30, 2025, and in any subsequent filings with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by applicable law.

	December 31, 2025	December 31, 2024
Condensed Consolidated Balance Sheet Data (unaudited)
(in thousands)
Cash, cash equivalents, and marketable securities	$1,245,210	$850,740
Total assets	1,481,177	1,103,824
Total liabilities	242,819	370,279
Total stockholders’ equity	1,238,358	733,545

	Three Months Ended December 31,		Year Ended December 31,
	2025	2024	2025	2024
Condensed Consolidated Statement of Operations (unaudited)
(in thousands, except share and per share data)
License and collaboration revenue	$114,109	$30,067	$139,743	$63,518
Operating expenses:
Research and development	99,275	101,444	409,618	367,561
General and administrative	32,279	28,660	113,818	111,525
Total operating expenses	131,554	130,104	523,436	479,086
Loss from operations	(17,445)	(100,037)	(383,693)	(415,568)
Other income (expense):
Change in fair value of derivative liabilities	50	(128)	700	2,272
Change in fair value of non-controlling equity investments	(3,329)	(1,090)	3,942	(14,093)
Change in fair value of contingent consideration liabilities	(765)	(27)	180	1,592
Gain on sale of equity method investment	255,146	-	255,146	-
Interest and other income (expense), net	10,640	10,928	43,733	49,094
Total other income (expense)	261,742	9,683	303,701	38,865
Net loss before income taxes	$244,297	$(90,354)	$(79,992)	$(376,703)
Provision for income taxes	-	-	-	(39)
Net loss	$244,297	$(90,354)	$(79,992)	$(376,742)
Unrealized gain (loss) on marketable securities	321	(1,080)	432	75
Comprehensive loss	$244,618	$(91,434)	$(79,560)	$(376,667)
Net income (loss) per common share - basic	$2.37	$(1.09)	$(0.81)	$(4.58)
Basic weighted-average common shares outstanding	102,876,980	82,824,151	98,905,577	82,313,008
Net income (loss) per common share - diluted	$2.33	$(1.09)	$(0.81)	$(4.58)
Diluted weighted-average common shares outstanding	104,927,041	82,824,151	98,905,577	82,313,008

Contacts
Holly Manning
Beam Therapeutics hmanning@beamtx.com
Josie Butler
1AB josie@1abmedia.com