Beam Therapeutics Inc.
For the period ended Jun 30, 2025. Filed Aug 5, 2025.
View on SEC.govAbout this filing
A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Jun 30, 2025 · Filed Aug 5, 2025
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2025-03-31; filed 2025-05-06.
0000950170-25-063966
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2025-06-30; filed 2025-08-05.
0000950170-25-102393
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 182 previous paragraphs and 202 current paragraphs.
132 unchanged; 35 reworded; 25 added; 8 removed; 7 previous and 10 current paragraphs rearranged.
Tables are not compared: 5 previous and 7 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (25)
Current report
We expect to provide updated data from the BEACON trial by the end of 2025.
Current report
To finalize dose selection and prepare BEAM-302 for registrational development, we have expanded dose exploration in Part A of the Phase 1/2 trial and initiated enrollment in Part B. In Part A, we are enrolling a total of six patients each in the 60 mg and 75 mg cohorts and initiated screening for a multi-dose cohort of two 60 mg doses administered eight weeks apart. Continued dose escalation may also be evaluated based on ongoing safety and efficacy findings. In Part B, patients will initially receive 30 mg of BEAM-302, followed by additional dose escalation cohorts.
Current report
As of August 1, 2025, a total of 17 patients have been dosed in Part A, with follow-up ranging from three days to 14 months. All AEs were mild to moderate, with no serious AEs and no dose-limiting toxicities reported. All liver transaminase elevations continued to be Grade 1 or below and resolved to normal without intervention. All infusion-related reactions were mild to moderate. Treatment with BEAM-302 continued to demonstrate restoration of AAT physiology by inducing production of corrected and functional M-AAT, reducing circulating mutant Z-AAT, and correcting the disease-causing mutation in a dose-dependent manner as measured by the percent change in total circulating AAT from baseline. Changes in total and functional AAT levels and the ratio of circulating M-AAT to Z-AAT continued to be durable for all patients.
Current report
We expect to finalize dose selection for registrational development based on the totality of data from the BEAM-302 trial. Clinical data from Part A and Part B are expected to be shared in early 2026, along with an updated clinical development plan for BEAM-302 in patients with AATD.
Current report
The increase of $14.7 million was primarily due to the following:
Current report
An increase of $0.4 million in facility and IT related expenses, including depreciation, related to our leased facilities.
Current report
The increase was partially offset by the following:
Current report
A decrease of $4.0 million in stock-based compensation driven by the decline in our stock price and additional stock awards granted to employees in 2024; and
Current report
General and administrative expenses were $26.9 million and $29.6 million for the three months ended June 30, 2025 and 2024, respectively. The decrease of $2.8 million was primarily due to the following:
Current report
A decrease of $3.3 million in stock-based compensation driven by the decline in our stock price and additional stock awards granted to employees in 2024;
Current report
A decrease of $1.0 million in legal expenses.
Current report
Interest and other income (expense), net was $12.3 million and $14.2 million for the three months ended June 30, 2025 and 2024, respectively. The change was primarily due to one-time credits received during the three months ended June 30, 2024.
Current report
Comparison of the six months ended June 30, 2025 and 2024
Current report
License and collaboration revenue
Current report
Research and development expenses were $200.6 million and $171.9 million for the six months ended June 30, 2025 and 2024, respectively. The following table summarizes our research and development expenses for the six months ended June 30, 2025 and 2024 (in thousands):
Current report
The increase of $28.7 million was primarily due to the following:
Current report
An increase of $9.3 million of employee related expenses due to the increase in research and development employees from 364 as of June 30, 2024 to 401 as of June 30, 2025; and
Current report
An increase of $1.4 million in facility and IT related expenses, including depreciation, related to our leased facilities.
Current report
The increase was partially offset by the following:
Current report
A decrease of $5.9 million in stock-based compensation driven by the decline in our stock price and additional stock awards granted to employees in 2024; and
Current report
General and administrative expenses were $54.8 million and $56.4 million for the six months ended June 30, 2025 and 2024, respectively. The decrease of $1.6 million was primarily due to the following:
Current report
A decrease of $4.0 million in stock-based compensation driven by the decline in our stock price and additional stock awards granted to employees in 2024;
Current report
A decrease of $0.5 million in legal expenses;
Current report
A decrease of $0.5 million in other expenses; and
Current report
During each of the six months ended June 30, 2025 and 2024, we recorded less than $0.1 million of other expense and $1.6 million of other income, respectively, related to the change in fair value of the technology and product contingent consideration liabilities.
Removed paragraphs (8)
Previous report
Research and development expenses
Previous report
General and administrative expenses
Previous report
Research and development expenses
Previous report
The increase of $14.0 million was primarily due to the following:
Previous report
The decrease was partially offset by the following:
Previous report
A decrease of $1.9 million in stock-based compensation driven by the decline in our stock price and additional stock awards granted to employees in 2024.
Previous report
General and administrative expenses
Previous report
General and administrative expenses were $27.9 million and $26.7 million for the three months ended March 31, 2025 and 2024, respectively. The increase of $1.2 million was primarily due to the following:
Reworded paragraphs (35)
Previous wording
We are conducting a Phase 1/2 clinical trial designed to assess the safety and efficacy of BEAM-101 for the treatment of sickle cell disease, which we refer to as our BEACON trial. The BEACON trial includes up to 45 patients ages 18 to 35 with severe sickle cell disease who have received prior treatment with at least one disease-modifying agent with inadequate response or intolerance. Following mobilization, conditioning and treatment with BEAM-101, patients are assessed for safety and tolerability, with safety endpoints including neutrophil and platelet engraftment. Patients are also assessed for efficacy, with efficacy endpoints including the change from baseline in severe vaso-occlusive events, transfusion requirements, HbF levels, and quality of life assessments. The trial’s Data Monitoring Committee and the U.S. Food & Drug Administration have cleared the trial to enroll adolescents from 12 to 17 years old. The adult enrollment target for BEACON has been achieved, and the first adolescent patients have cleared screening and enrolled in the trial. We expect to dose 30 patients in the BEACON trial by mid-2025. We also plan to present updated data from the trial at the European Hematology Association 2025 Congress in June 2025.
Current wording
We are conducting a Phase 1/2 clinical trial designed to assess the safety and efficacy of BEAM-101 for the treatment of sickle cell disease, which we refer to as our BEACON trial. The BEACON trial includes up to 45 patients ages 18 to 35 with severe sickle cell disease who have received prior treatment with at least one disease-modifying agent with inadequate response or intolerance. Following mobilization, conditioning and treatment with BEAM-101, patients are assessed for safety and tolerability, with safety endpoints including neutrophil and platelet engraftment. Patients are also assessed for efficacy, with efficacy endpoints including the change from baseline in severe vaso-occlusive events, transfusion requirements, HbF levels, and quality of life assessments. The adult and adolescent enrollment targets for BEACON have been achieved, and 30 patients in the BEACON trial have been dosed. In addition, the first adolescent patient has been dosed. The U.S. Food and Drug Administration, or the FDA, has granted orphan drug designation to BEAM-101.
Previous wording
In December 2024, we presented initial data from the BEACON trial at the 2024 American Society of Hematology Annual Meeting and Exposition, or ASH. The presentation contained preliminary data as of October 28, 2024 from seven patients in the trial, with follow up ranging from one to 11 months. The presentation data included the following:
Current wording
In June 2025, we presented updated data from the BEACON trial at the European Hematology Association 2025 Congress. The presentation contained preliminary data as of February 2025, from 17 patients in the trial, with follow up ranging from 0.2 to 15.1 months. The presentation data included the following:
Previous wording
All patients achieved endogenous HbF levels exceeding 60% and reduction in corresponding sickle HbS below 40% that was durable through the data cutoff date. A pancellular distribution of HbF was also observed after the elimination of transfused blood. Total hemoglobin levels increased rapidly with resolution of anemia in all patients after elimination of the transfused blood.
Current wording
All patients achieved endogenous HbF levels exceeding 60% and reduction in corresponding sickle HbS below 40% that was durable through the data cutoff date. A pancellular distribution of HbF, with HbF levels per cell above the sickling threshold, was maintained through follow-up. Total hemoglobin levels increased rapidly with resolution of anemia in all patients after elimination of the transfused blood.
Previous wording
All patients achieved the minimum target cell dose in either 1 or 2 cycles of mobilization (average: 1.4). The mean time to neutrophil engraftment was 17.1 days (range: 15–21), with a low mean duration of neutropenia (6.3 days). The mean time to platelet engraftment was 19.1 days (range: 11–34).
Current wording
Patients achieved the minimum target cell dose in a median of one cycle of mobilization (range: 1-3). The median time to neutrophil engraftment was 16.5 days (range: 12-30), with a median duration of severe neutropenia of 7.0 days (range: 4-17). The median time to platelet engraftment was 19.5 days (range: 11-34).
Previous wording
Key markers of hemolysis, including indirect bilirubin, haptoglobin, lactate dehydrogenase and reticulocytes, normalized or improved in all patients following BEAM-101 treatment.
Current wording
Key markers of hemolysis, including indirect bilirubin, haptoglobin, lactate dehydrogenase and reticulocytes, normalized or improved in all patients following BEAM-101 treatment. Erythropoietin levels also decreased to normal or near normal, indicating significant improvement in oxygen delivery to tissues.
Previous wording
The initial safety profile of BEAM-101 was consistent with busulfan conditioning and autologous HSCT. The most common treatment-emergent adverse events were consistent with busulfan conditioning, including febrile neutropenia, stomatitis and anemia. One patient died four months after BEAM-101 infusion due to respiratory failure that was determined by the investigator to be likely related to busulfan conditioning and deemed unrelated to BEAM-101. No vaso-occlusive crises were reported post-engraftment.
Current wording
The initial safety profile of BEAM-101 was consistent with busulfan conditioning, autologous HSCT and underlying sickle cell disease. The most common treatment-emergent adverse events were consistent with busulfan conditioning, including febrile neutropenia, stomatitis and anemia. As previously reported, one patient died four months after BEAM-101 infusion due to respiratory failure that was determined by the investigator to be likely related to busulfan conditioning and deemed unrelated to BEAM-101. No vaso-occlusive crises were reported post-engraftment.
Previous wording
We plan to continue the dose-escalation portion of Part A of the ongoing Phase 1/2 clinical trial, including enrolling and dosing a fourth dose cohort (75 mg).We expect to report further data at a medical conference in the second half of 2025. In addition, we plan to dose the first patient in Part B, which will include AATD patients with mild to moderate liver disease, in the second half of 2025. In March 2025, the FDA cleared the investigational new drug application, or IND, for BEAM-302 for the treatment of AATD, enabling us to begin activating sites in the United States.
Current wording
In March 2025, the FDA cleared the investigational new drug application, or IND, for BEAM-302 for the treatment of AATD, enabling us to begin activating sites in the United States. In May 2025, the FDA granted regenerative medicine advanced therapy and orphan drug designations to BEAM-302.
Previous wording
In April 2019, we entered into a collaboration and license agreement, or the Verve Agreement, with Verve Therapeutics, Inc., or Verve, a company focused on gene editing for cardiovascular disease treatments, and in July 2022, we and Verve amended the Verve Agreement. Under the terms of the Verve Agreement, as amended, we granted Verve exclusive worldwide licenses under certain of our editing technologies for human therapeutic applications against a total of three liver-mediated, cardiovascular disease targets, including use of our base editing technology for each of these targets and use of certain of our gene editing technology for two of such targets. In exchange, we received shares of Verve common stock. In October 2023, we entered into a transfer and delegation agreement, or the Lilly Agreement, with Eli Lilly and Company, or Lilly, pursuant to which Lilly acquired certain assets and other rights under the Verve Agreement, including our opt-in rights to co-develop and co-commercialize each of Verve’s base editing programs for cardiovascular disease, which consist of programs targeting PCSK9, ANGPTL3 and an undisclosed liver-mediated, cardiovascular target. In addition, Lilly acquired the right to receive any future milestone or royalty payments payable to us under the Verve Agreement. Under the terms of the Lilly Agreement, we received a $200.0 million payment and are eligible to receive up to $350.0 million in potential future development-stage payments upon the completion of certain clinical, regulatory and alliance events, of which $25.0 million has been received through March 31, 2025. There were no milestone payments received during the three months ended March 31, 2025.
Current wording
In April 2019, we entered into a collaboration and license agreement, or the Verve Agreement, with Verve Therapeutics, Inc., or Verve, a company focused on gene editing for cardiovascular disease treatments, and in July 2022, we and Verve amended the Verve Agreement. Under the terms of the Verve Agreement, as amended, we granted Verve exclusive worldwide licenses under certain of our editing technologies for human therapeutic applications against a total of three liver-mediated, cardiovascular disease targets, including use of our base editing technology for each of these targets and use of certain of our gene editing technology for two of such targets. In exchange, we received shares of Verve common stock. In October 2023, we entered into a transfer and delegation agreement, or the Lilly Agreement, with Eli Lilly and Company, or Lilly, pursuant to which Lilly acquired certain assets and other rights under the Verve Agreement, including our opt-in rights to co-develop and co-commercialize each of Verve’s base editing programs for cardiovascular disease, which consist of programs targeting PCSK9, ANGPTL3 and an undisclosed liver-mediated, cardiovascular target. In addition, Lilly acquired the right to receive any future milestone or royalty payments payable to us under the Verve Agreement. Under the terms of the Lilly Agreement, we received a $200.0 million payment and are eligible to receive up to $350.0 million in potential future development-stage payments upon the completion of certain clinical, regulatory and alliance events, of which $25.0 million has been received through June 30, 2025. There were no milestone payments received during the six months ended June 30, 2025.
Previous wording
In September 2022, we entered into a license and research collaboration agreement, or the Orbital Agreement, with Orbital, pursuant to which each of us granted the other licenses to certain technology controlled during the three years after entry into the Orbital Agreement that are necessary or reasonably useful for the non-viral delivery or the design or manufacture of RNA for the prevention, treatment or diagnosis of human disease. Our license to Orbital is for all fields other than our exclusive field and also excludes the targets and substantially all of the indications that are the subject of our existing programs. Our exclusive field consists of all products and biologics that function in the process of gene editing or conditioning for use in cell transplantation, or that act in combination with any such products or biologics. Orbital’s license to us is for all fields other than Orbital’s exclusive field. Orbital’s exclusive field consists of products and biologics that function as vaccines and also of therapeutic proteins, other than therapeutic proteins (i) that use gene editing, (ii) for use in conditioning, (iii) for use in regenerative medicine, (iv) for use as a CAR immune therapy, including
Current wording
In September 2022, we entered into a license and research collaboration agreement, or the Orbital Agreement, with Orbital, pursuant to which each of us granted the other licenses to certain technology controlled during the three years after entry into the Orbital Agreement that are necessary or reasonably useful for the non-viral delivery or the design or manufacture of RNA for the prevention, treatment or diagnosis of human disease. Our license to Orbital is for all fields other than our exclusive field and also excludes the targets and substantially all of the indications that are the subject of our existing programs. Our exclusive field consists of all products and biologics that function in the process of gene editing or conditioning for use in cell transplantation, or that act in combination with any such products or biologics. Orbital’s license to us is for all fields other than Orbital’s exclusive field. Orbital’s exclusive field consists of products and biologics that function as vaccines and also of therapeutic proteins, other than therapeutic proteins (i) that use gene editing, (ii) for use in conditioning, (iii) for use in regenerative medicine, (iv) for use as a CAR immune therapy, including CAR-T, CAR-NK and CAR-macrophage compositions, (v) for use as a T-cell receptor therapy or (vi) that modulate certain immune responses. The licenses are exclusive in each party’s exclusive field for three years and non-exclusive in those fields thereafter. We and Orbital agreed that for a period of three years after entry into the Orbital Agreement, subject to limited exceptions, we would not research, develop and commercialize, or grant licenses to research, develop and commercialize, products or biologics within the other party’s exclusive field.
Previous wording
We are an early-stage company, and the majority of our programs are at a preclinical or clinical stage of development. To date, we have not generated any revenue from product sales and do not expect to generate revenue from the sale of products for the foreseeable future. Our revenue to date has been primarily derived from license and collaboration agreements with partners. Since inception we have incurred significant operating losses. Our net losses for the three months ended March 31, 2025 and 2024 were $109.3 million and $98.7 million, respectively. As of March 31, 2025, we had an accumulated deficit of $1.7 billion. We expect to continue to incur significant expenses and increasing operating losses in connection with ongoing development activities related to our internal programs and collaborations as we continue our preclinical and clinical development of product candidates; advance additional product candidates toward clinical development; operate our cGMP facility in North Carolina; further develop our base editing platform; continue to make investments in delivery technology for our base editors; conduct research activities as we seek to discover and develop additional product candidates; maintain, expand, enforce, defend and protect our intellectual property portfolio; and continue to hire research and development, clinical, technical operations and commercial personnel. In addition, we expect to continue to incur the costs associated with operating as a public company.
Current wording
We are an early-stage company, and the majority of our programs are at a preclinical or clinical stage of development. To date, we have not generated any revenue from product sales and do not expect to generate revenue from the sale of products for the foreseeable future. Our revenue to date has been primarily derived from license and collaboration agreements with partners. Since inception we have incurred significant operating losses. Our net losses for the six months ended June 30, 2025 and 2024 were $211.6 million and $189.7 million, respectively. As of June 30, 2025, we had an accumulated deficit of $1.8 billion. We expect to continue to incur significant expenses and increasing operating losses in connection with ongoing development activities related to our internal programs and collaborations as we continue our preclinical and clinical development of product candidates; advance additional product candidates toward clinical development; operate our cGMP facility in North Carolina; further develop our base editing platform; continue to make investments in delivery technology for our base editors; conduct research activities as we seek to discover and develop additional product candidates; maintain, expand, enforce, defend and protect our intellectual property portfolio; and continue to hire research and development, clinical, technical operations and commercial personnel. In addition, we expect to continue to incur the costs associated with operating as a public company.
Previous wording
We have not generated any revenue to date from product sales and do not expect to do so in the near future. During the three months ended March 31, 2025 and 2024, we recognized $7.5 million and $7.4 million of license and collaboration revenue, respectively.
Current wording
We have not generated any revenue to date from product sales and do not expect to do so in the near future. During the six months ended June 30, 2025 and 2024, we recognized $15.9 million and $19.2 million of license and collaboration revenue, respectively.
Previous wording
License and collaboration revenue was $7.5 million and $7.4 million for the three months ended March 31, 2025 and 2024, respectively. The increase in revenue of $0.1 million is due to a slightly increased level of research activities on our license and collaboration programs. License and collaboration revenue represents revenue recorded under each of the Pfizer, Apellis and Orbital Agreements.
Current wording
License and collaboration revenue was $8.5 million and $11.8 million for the three months ended June 30, 2025 and 2024, respectively. The decrease in revenue of $3.3 million is due to a decreased level of research activities on our license and collaboration programs. License and collaboration revenue represents revenue recorded under each of the Pfizer, Apellis and Orbital Agreements.
Previous wording
Research and development expenses were $98.8 million and $84.8 million for the three months ended March 31, 2025 and 2024, respectively. The following table summarizes our research and development expenses for the three months ended March 31, 2025 and 2024 (in thousands):
Current wording
Research and development expenses were $101.8 million and $87.0 million for the three months ended June 30, 2025 and 2024, respectively. The following table summarizes our research and development expenses for the three months ended June 30, 2025 and 2024 (in thousands):
Previous wording
An increase of $9.6 million in external research and development expenses driven by $9.4 million in outsourced services, primarily due to manufacturing and clinical activities for BEAM-101 and BEAM-302 and an increase in lab supply expenses of $0.2 million due to increased research activities when compared to the prior year;
Current wording
An increase of $15.7 million in external research and development expenses driven by $15.1 million in outsourced services, primarily due to manufacturing and clinical activities and an increase in lab supply expenses of $0.6 million due to increased research activities when compared to the prior year;
Previous wording
An increase of $4.7 million of employee related costs due to the increase in research and development employees from 346 as of March 31, 2024 to 393 as of March 31, 2025;
Current wording
An increase of $4.6 million of employee related expenses due to the increase in research and development employees from 364 as of June 30, 2024 to 401 as of June 30, 2025; and
Previous wording
An increase in other expenses of $0.6 million driven by milestone payments during the three months ended March 31, 2025.
Current wording
A decrease in other expenses of $1.9 million mostly driven by milestone payments during the three months ended June 30, 2024.
Previous wording
The increase in general and administrative expenses was partially offset by the following:
Current wording
The decrease in general and administrative expenses was partially offset by the following:
Previous wording
An increase of $2.0 million in employee related costs due to the growth in general and administrative employees from 93 as of March 31, 2024 to 109 as of March 31, 2025; and
Current wording
An increase of $1.6 million in employee related costs due to the growth in general and administrative employees from 97 as of June 30, 2024 to 108 as of June 30, 2025.
Previous wording
During the three months ended March 31, 2025 and 2024, we recorded $2.3 million of other income and $2.9 million of expense, respectively, primarily related to the change in fair value of success payment liabilities due to changes in the price of our common stock over the related periods as well as changes in our settlement liability. A portion of the success payment obligations were paid in June 2021; the remaining success payment obligations are still outstanding as of March 31, 2025 and will continue to be revalued at each reporting period.
Current wording
During the three months ended June 30, 2025 and 2024, we recorded $1.1 million and $5.5 million of other income, respectively, primarily related to the change in fair value of success payment liabilities due to changes in the price of our common stock over the related periods as well as changes in our settlement liability. A portion of the success payment obligations was paid in June 2021; the remaining success payment obligations are still outstanding as of June 30, 2025 and will continue to be revalued at each reporting period.
Previous wording
During the three months ended March 31, 2025 and 2024, we recorded $2.1 million and $3.4 million of other expense, respectively, as a result of changes in the fair value of our investment in Verve and Prime common stock.
Current wording
During the three months ended June 30, 2025 and 2024, we recorded $4.4 million of other income and $7.6 million of other expense, respectively, as a result of changes in the fair value of our investment in Verve and Prime common stock.
Previous wording
During each of the three months ended March 31, 2025 and 2024, we recorded less than $0.1 million of other expense, related to the change in fair value of the technology and product contingent consideration liabilities.
Current wording
During the three months ended June 30, 2025 and 2024, we recorded less than $0.1 million of other expense and $1.8 million of other income, respectively, related to the change in fair value of the technology and product contingent consideration liabilities.
Previous wording
Interest and other income (expense), net was $9.9 million and $11.8 million for the three months ended March 31, 2025 and 2024, respectively. The change was primarily due to a lower cash balance as of March 31, 2025 compared to March 31, 2024.
Current wording
Interest and other income (expense), net was $22.2 million and $26.0 million for the six months ended June 30, 2025 and 2024, respectively. The change was primarily due to lower interest rates as of June 30, 2025 compared to June 30, 2024 and one-time credits received during the six months ended June 30, 2024.
Previous wording
In March 2025, we closed an underwritten public offering of 16,151,686 shares of common stock at a public offering price of $28.48 per share and pre-funded warrants to purchase 1,404,988 shares of common stock at a purchase price of $28.47 per pre-funded warrant for aggregate net proceeds of $471.2 million, after deducting underwriting discounts, commissions and approximately $0.8 million related to legal, accounting and other fees in connection with the offering.
Current wording
In March 2025, we closed an underwritten public offering of 16,151,686 shares of common stock at a public offering price of $28.48 per share and pre-funded warrants to purchase 1,404,988 shares of common stock at a purchase price of $28.47 per pre-funded warrant for aggregate net proceeds of $470.5 million, after deducting underwriting discounts, commissions and approximately $0.8 million related to legal, accounting and other fees in connection with the offering.
Previous wording
We have entered into the Sales Agreement with Jefferies pursuant to which we are entitled to offer and sell, from time to time at prevailing market prices, shares of our common stock having aggregate gross proceeds of up to $1.1 billion. We agreed to pay Jefferies a commission of up to 3.0% of the aggregate gross sale proceeds of any shares sold by Jefferies under the Sales Agreement. As of March 31, 2025, we have sold 13,769,001 shares of common stock under the Sales Agreement at an average price of $62.75 per share for aggregate gross proceeds of $864.0 million, before deducting commissions and offering expenses payable by us. There were no shares sold under the Sales Agreement during the three months ended March 31, 2025.
Current wording
We have entered into the Sales Agreement with Jefferies pursuant to which we are entitled to offer and sell, from time to time at prevailing market prices, shares of our common stock having aggregate gross proceeds of up to $1.1 billion. We agreed to pay Jefferies a commission of up to 3.0% of the aggregate gross sale proceeds of any shares sold by Jefferies under the Sales Agreement. As of June 30, 2025, we have sold 13,769,001 shares of common stock under the Sales Agreement at an average price of $62.75 per share for aggregate gross proceeds of $864.0 million, before deducting commissions and offering expenses payable by us. There were no shares sold under the Sales Agreement during the six months ended June 30, 2025.
Previous wording
Net cash used in operating activities for the three months ended March 31, 2025 was $103.9 million, including our net loss of $109.3 million, decreases in accrued expenses and other liabilities of $18.5 million, deferred revenue of $7.5 million, operating lease liabilities totaling $3.3 million and a decrease in other long-term liabilities of $0.1 million. In addition, noncash items, including the amortization of investment premiums of $3.9 million and a net decrease in the fair value of derivative liabilities of $2.3 million also contributed to net cash used in operating activities.
Current wording
Net cash used in operating activities for the six months ended June 30, 2025 was $180.3 million, including our net loss of $211.6 million, decreases in accrued expenses and other liabilities of $8.6 million, deferred revenue of $15.9 million, operating lease liabilities totaling $6.6 million and a decrease in other long-term liabilities of $0.1 million. In addition, noncash items, including the amortization of investment premiums of $8.5 million, a net decrease in the fair value of derivative liabilities of $3.4 million and an increase in the fair value of non-controlling equity investments of $2.3 million also contributed to net cash used in operating activities.
Previous wording
These uses of cash were partially offset by an increase in accounts payable of $3.8 million and an increase of less than $0.1 million in the fair value of our contingent consideration liabilities. In addition, we recorded noncash items consisting of stock-based compensation expense of $26.7 million, depreciation and amortization expense of $5.5 million, a decrease in the fair value of non-controlling equity investments of $2.1 million and a decrease in operating lease right-of-use, or ROU, assets of $2.6 million.
Current wording
These uses of cash were partially offset by an increase in accounts payable of $6.4 million, a decrease of $2.8 million in prepaid expenses and other current assets and an increase of less than $0.1 million in the fair value of our contingent consideration liabilities. In addition, we recorded noncash items consisting of stock-based compensation expense of $51.0 million, depreciation and amortization expense of $11.1 million and a decrease in operating lease right-of-use, or ROU, assets of $5.3 million.
Previous wording
Net cash used in operating activities for the three months ended March 31, 2024 was $99.7 million, consisting of our net loss of $98.7 million, decreases of accrued expenses and other liabilities of $24.3 million, a decrease in deferred revenue of $6.9 million, a decrease in operating lease liabilities totaling $3.1 million and increases of prepaid expenses and other current assets of $5.5 million. In addition, a noncash item, the amortization of investment premiums of $6.6 million, also contributed to net cash used in operating activities.
Current wording
Net cash used in operating activities for the six months ended June 30, 2024 was $182.8 million, including our net loss of $189.7 million, decreases in accrued expenses and other liabilities of $43.8 million, deferred revenue of $18.2 million, and operating lease liabilities totaling $6.2 million and an increase of prepaid expenses and other current assets of $0.7 million. In addition, noncash items, including the amortization of investment premiums of $12.5 million, a decrease in the fair value of derivative liabilities of $2.6 million and a decrease of $1.6 million in the fair value of our contingent consideration liabilities also contributed to net cash used in operating activities.
Previous wording
These uses of cash were partially offset by an increase in accounts payable of $1.3 million and an increase of other long-term liabilities of $0.6 million. In addition, we recorded noncash items consisting of stock-based compensation expense of $29.3 million, depreciation and amortization expense of $5.4 million, decreases in the fair value of non-controlling equity investments of $3.4 million, increases in the fair value of derivative liabilities of $2.9 million, changes in operating lease ROU assets of $2.4 million, and a $0.1 million increase in fair value of our contingent consideration liabilities.
Current wording
These uses of cash were partially offset by an increase in accounts payable of $3.1 million and other long-term liabilities of $0.5 million, respectively. In addition, we recorded noncash items consisting of stock-based compensation expense of $60.9 million, depreciation and amortization expense of $11.0 million, a decrease in the fair value of non-controlling equity investments of $10.9 million and a decrease in operating lease right-of-use, or ROU, assets of $4.8 million.
Previous wording
For the three months ended March 31, 2025, cash used in investing activities consisted of net purchases of marketable securities of $122.0 million and purchases of property and equipment of $3.1 million.
Current wording
For the six months ended June 30, 2025, cash used in investing activities consisted of net purchases of marketable securities of $289.3 million and purchases of property and equipment of $6.2 million.
Previous wording
For the three months ended March 31, 2024, cash used in investing activities consisted of net purchases of marketable securities of $51.0 million and purchases of property and equipment of $2.4 million.
Current wording
For the six months ended June 30, 2024, cash provided by investing activities consisted of net maturities of marketable securities of $38.4 million, partially offset by purchases of property and equipment of $4.4 million.
Previous wording
Net cash provided by financing activities for the three months ended March 31, 2025 consisted of $471.2 million of proceeds from the March 2025 issuance of common stock and pre-funded warrants, $1.5 million of proceeds from the issuance of common stock under our Employee Stock Purchase Plan, or ESPP, and $0.7 million of proceeds from the exercise of stock options.
Current wording
Net cash provided by financing activities for the six months ended June 30, 2025 consisted of $470.5 million of proceeds from the March 2025 issuance of common stock and pre-funded warrants, $1.5 million of proceeds from the issuance of common stock under our Employee Stock Purchase Plan, or ESPP, and $2.5 million of proceeds from the exercise of stock options.
Previous wording
Net cash provided by financing activities for the three months ended March 31, 2024 consisted of $1.7 million of proceeds from the exercise of stock options and $1.4 million of proceeds from the issuance of common stock under our Employee Stock Purchase Plan, offset in part by repayments of equipment financing liabilities of $0.2 million.
Current wording
Net cash provided by financing activities for the six months ended June 30, 2024 consisted of $1.9 million of proceeds from the exercise of stock options and $1.4 million of proceeds from the issuance of common stock under our ESPP, offset in part by repayments of equipment financing liabilities of $0.3 million.
Previous wording
the payment of success liabilities to Harvard and Broad Institute pursuant to the respective terms of the Harvard License Agreement and the Broad Institute License Agreement, should we choose to pay in cash;
Current wording
the payment of success liabilities to Harvard and Broad Institute pursuant to the respective terms of the Harvard License Agreement and the Broad License Agreement, should we choose to pay in cash;
Previous wording
We lease certain assets under noncancelable operating and finance leases. The leases relate primarily to office space and laboratory space. As of March 31, 2025, aggregate future minimum commitments under these office and laboratory leases are $224.4 million, of which $21.0 million will be payable in 2025. These minimum lease payments exclude our share of the facility operating expenses, real-estate taxes and other costs that are reimbursable to the landlord under the leases.
Current wording
We lease certain assets under noncancelable operating and finance leases. The leases relate primarily to office space and laboratory space. As of June 30, 2025, aggregate future minimum commitments under these office and laboratory leases are $219.7 million, of which $16.4 million will be payable in 2025. These minimum lease payments exclude our share of the facility operating expenses, real-estate taxes and other costs that are reimbursable to the landlord under the leases.
Previous wording
During the three months ended March 31, 2025, there were no material changes to our contractual obligations and commitments described under Management’s Discussion and Analysis of Financial Condition and Results of Operations in the 2024 Form 10-K.
Current wording
During the six months ended June 30, 2025, there were no material changes to our contractual obligations and commitments described under Management’s Discussion and Analysis of Financial Condition and Results of Operations in the 2024 Form 10-K.
Rearranged current paragraphs (10)
Current report — wording carried from elsewhere
A decrease of $0.2 million in other expenses; and
Current report — wording carried from elsewhere
The following table summarizes our results of operations (in thousands):
Current report — wording carried from elsewhere
License and collaboration revenue was $15.9 million and $19.2 million for the six months ended June 30, 2025 and 2024, respectively. The decrease in revenue of $3.3 million is due to a slightly decreased level of research activities on our license and collaboration programs. License and collaboration revenue represents revenue recorded under each of the Pfizer, Apellis and Orbital Agreements.
Current report — wording carried from elsewhere
An increase of $25.3 million in external research and development expenses driven by $24.5 million in outsourced services, primarily due to manufacturing and clinical activities and an increase in lab supply expenses of $0.8 million due to increased research activities when compared to the prior year;
Current report — wording carried from elsewhere
A decrease in other expenses of $1.4 million driven by milestone payments during the six months ended June 30, 2024.
Current report — wording carried from elsewhere
The decrease in general and administrative expenses was partially offset by the following:
Current report — wording carried from elsewhere
An increase of $3.6 million in employee related costs due to the growth in general and administrative employees from 97 as of June 30, 2024 to 108 as of June 30, 2025.
Current report — wording carried from elsewhere
During the six months ended June 30, 2025 and 2024, we recorded $3.4 million and $2.6 million of other income, respectively, primarily related to the change in fair value of success payment liabilities due to changes in the price of our common stock over the related periods as well as changes in our settlement liability. A portion of the success payment obligations was paid in June 2021; the remaining success payment obligations are still outstanding as of June 30, 2025 and will continue to be revalued at each reporting period.
Current report — wording carried from elsewhere
During the six months ended June 30, 2025 and 2024, we recorded $2.3 million other income and $10.9 million of other expense, respectively, as a result of changes in the fair value of our investment in Verve and Prime common stock.
Current report — wording carried from elsewhere
Interest and other income (expense), net
Rearranged previous paragraphs (7)
Previous report — wording carried elsewhere
CAR-T, CAR-NK and CAR-macrophage compositions, (v) for use as a T-cell receptor therapy or (vi) that modulate certain immune responses. The licenses are exclusive in each party’s exclusive field for three years and non-exclusive in those fields thereafter. We and Orbital agreed that for a period of three years after entry into the Orbital Agreement, subject to limited exceptions, we would not research, develop and commercialize, or grant licenses to research, develop and commercialize, products or biologics within the other party’s exclusive field.
Previous report — wording carried elsewhere
An increase of $1.1 million in facility and IT allocated costs, including depreciation, related to our leased facilities; and
Previous report — wording carried elsewhere
An increase of $0.1 million in other expenses.
Previous report — wording carried elsewhere
A decrease of $0.7 million in stock-based compensation driven by the decline in our stock price and additional stock awards granted to employees in 2024; and
Previous report — wording carried elsewhere
Change in fair value of derivative liabilities
Previous report — wording carried elsewhere
Change in fair value of non-controlling equity investments
Previous report — wording carried elsewhere
Change in fair value of contingent consideration liabilities