Beam Therapeutics Inc.

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 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.

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. The U.S. Food and Drug Administration, or the FDA, has granted orphan drug designation and regenerative medicine advanced therapy designation to BEAM-101.

Previous 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:

Current wording

In November 2025, we disclosed updated data from the BEACON trial in an abstract for a presentation at the American Society of Hematology 2025 Annual Meeting, or ASH. The abstract contained preliminary data as of June 16, 2025, from 26 patients in the trial, with follow up ranging from 1.0 to 18.7 months. The presentation data included the following:

Previous 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).

Current wording

Patients achieved the minimum target cell dose in a median of one cycle of mobilization (range: 1-4). The median time to neutrophil engraftment (n=26) was 18.0 days (range: 12-30), with a median duration of neutropenia of 7.0 days (range: 4-17). The median time to platelet engraftment (n=24) was 19.0 days (range: 11-50).

Previous 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.

Current wording

Key markers of hemolysis and sickling parameters normalized or improved in all patients following BEAM-101 treatment. Erythropoietin levels trended towards normal in all patients, including those with elevated total Hb.

Previous 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.

Current wording

The initial safety profile of BEAM-101 continues to be consistent with busulfan conditioning, autologous HSCT and underlying sickle cell disease. No patients reported Grade 3 or higher adverse events or serious adverse events related to treatment with BEAM-101.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 expect to provide updated data from the BEACON trial by the end of 2025.

Current wording

We expect to provide updated data from the BEACON trial at ASH.

Previous wording

We have nominated a development candidate for our ESCAPE technology comprised of two investigational drug products: BEAM-103, an anti-CD117 monoclonal antibody, and BEAM-104, a cell therapy that includes the same therapeutic edit as BEAM-101 (editing the HBG1/2 genes to elevate fetal hemoglobin), plus an additional edit to CD117 designed to block binding of BEAM-103, allowing the edited cells to evade suppression by the antibody. We intend to advance BEAM-103 and BEAM-104 for development in sickle cell disease and beta-thalassemia, potentially building on the same regulatory, manufacturing, clinical and commercial foundations being established with BEAM-101. We expect to initiate a Phase 1 healthy volunteer clinical trial of BEAM-103 by the end of 2025.

Current wording

We have nominated a development candidate for our ESCAPE technology comprised of two investigational drug products: BEAM-103, an anti-CD117 monoclonal antibody, and BEAM-104, a cell therapy that includes the same therapeutic edit as BEAM-101 (editing the HBG1/2 genes to elevate fetal hemoglobin), plus an additional edit to CD117 designed to block binding of BEAM-103, allowing the edited cells to evade suppression by the antibody. We intend to advance BEAM-103 and BEAM-104 for development in sickle cell disease and beta-thalassemia, potentially building on the same regulatory, manufacturing, clinical and commercial foundations being established with BEAM-101. The first subject has been dosed in a Phase 1 healthy volunteer clinical trial of BEAM-103.

Previous wording

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 wording

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 dosing in Part B. In Part A, we are enrolling a total of six patients each in the 60 mg and 75 mg cohorts and have dosed the first patient in 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. The first patient has been dosed in the first cohort in Part B of the trial.

Previous wording

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 wording

As of August 1, 2025, a total of 17 patients had 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.

Previous wording

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 wording

We expect to finalize dose selection for registrational development based on the totality of data from the BEAM-302 trial. Clinical data from the dose-escalation portions of 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.

Previous wording

In June 2021, we entered into a research collaboration agreement, or the Apellis Agreement, with Apellis Pharmaceuticals, Inc., or Apellis, focused on the use of our base editing technology to discover new treatments for complement system-driven diseases. Under the terms of the Apellis Agreement, we will conduct preclinical research on six base editing programs that target specific genes within the complement system in various organs, including the eye, liver, and brain. Apellis has an exclusive option to license any or all of the six programs and will assume responsibility for subsequent development. We may elect to enter into a 50-50 U.S. co-development and co-commercialization agreement with Apellis with respect to one program licensed under the collaboration.

Current wording

In June 2021, we entered into a research collaboration agreement, or the Apellis Agreement, with Apellis Pharmaceuticals, Inc., or Apellis, focused on the use of our base editing technology to discover new treatments for complement system-driven diseases. Under the terms of the Apellis Agreement, we will conduct preclinical research on six base editing programs that target specific genes within the complement system in various organs, including the eye, liver, and brain. Apellis has an exclusive option to license any or all of the six programs and will assume responsibility for subsequent development. As of September 30, 2025, Apellis notified us of its decision to opt in to one of the six base editing programs. As a result of Apellis' decision to opt in to the program, we received a cash opt in fee of $3.8 million during the three months ended September 30, 2025. We may elect to enter into a 50-50 U.S. co-development and co-commercialization agreement with Apellis with respect to the program licensed under the collaboration.

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 June 30, 2025. There were no milestone payments received during the six months ended June 30, 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 September 30, 2025. There were no milestone payments received during the nine months ended September 30, 2025. In July 2025, Lilly announced that it had completed its acquisition of Verve.

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 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.

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 have granted the other non-exclusive 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 the Beam field, as described below, and also excludes the targets and substantially all of the indications that are the subject of our existing programs. The Beam 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 field, which 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. On October 10, 2025, Bristol Myers Squibb announced that it and Orbital had entered into a definitive merger agreement pursuant to which Bristol Myers Squibb will acquire Orbital for $1.5 billion in cash. As of the announcement, we held 75 million shares of Orbital common stock, which represented a fully-diluted ownership stake of approximately 17%. The acquisition is subject to the satisfaction of closing conditions, and the sale price is subject to certain price adjustments and holdbacks. As a result, we are unable to predict the timing and final amount of proceeds we may receive from the transaction, if any, with certainty.

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 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.

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 nine months ended September 30, 2025 and 2024 were $324.3 million and $286.4 million, respectively. As of September 30, 2025, we had an accumulated deficit of $1.9 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

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. In June 2021, we entered into a research collaboration agreement, or the Apellis Agreement, with Apellis Pharmaceuticals, Inc., or Apellis, focused on the use of our base editing technology to discover new treatments for complement system-driven diseases. In October 2021, we entered into an option and license agreement, or the Sana Agreement, with Sana Biotechnology, Inc., or Sana, pursuant to which we granted Sana non-exclusive research and development and commercial rights to our CRISPR Cas12b technology to perform nuclease editing for certainex vivo engineered cell therapy programs. In December 2021, we entered into a four-year research collaboration agreement, or the Pfizer Agreement, with Pfizer Inc., or Pfizer, focused onin vivo base editing programs for three targets for rare genetic diseases of the liver, muscle and central nervous system. In September 2022, we entered into a License and Research Collaboration Agreement, or the Orbital Agreement, with Orbital Therapeutics, Inc., or Orbital, a newly formed entity focused on advancing non-viral delivery and RNA technologies. 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 Verve’s base editing programs for cardiovascular disease.

Current wording

In April 2019, we entered into the Verve Agreement with Verve, a company focused on gene editing for cardiovascular disease treatments. In June 2021, we entered into the Apellis Agreement with Apellis, focused on the use of our base editing technology to discover new treatments for complement system-driven diseases. In October 2021, we entered into the Sana Agreement with Sana pursuant to which we granted Sana non-exclusive research and development and commercial rights to our CRISPR Cas12b technology to perform nuclease editing for certainex vivo engineered cell therapy programs. In December 2021, we entered into the Pfizer Agreement with Pfizer, focused onin vivo base editing programs for three targets for rare genetic diseases of the liver, muscle and central nervous system. In September 2022, we entered into the Orbital Agreement with Orbital, a company focused on advancing non-viral delivery and RNA technologies. In October 2023, we entered into the Lilly Agreement with 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 Verve’s base editing programs for cardiovascular disease.

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 six months ended June 30, 2025 and 2024, we recognized $15.9 million and $19.2 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 nine months ended September 30, 2025 and 2024, we recognized $25.6 million and $33.5 million of license and collaboration revenue, respectively.

Previous wording

We anticipate that our general and administrative expenses will increase in the future to support our increased research and development activities. We also expect to continue to incur costs associated with being a public company and maintaining controls over financial reporting, including costs of accounting, audit, legal, regulatory and tax-related services associated with maintaining compliance with Nasdaq and SEC requirements, director and officer insurance costs, and investor and public relations costs.

Current wording

We anticipate that our general and administrative expenses will increase in the future to support our increased research and development and commercial readiness activities. We also expect to continue to incur costs associated with being a public company and maintaining controls over financial reporting, including costs of accounting, audit, legal, regulatory and tax-related services associated with maintaining compliance with Nasdaq and SEC requirements, director and officer insurance costs, and investor and public relations costs.

Previous wording

Change in fair value of contingent consideration liabilities consists of remeasurement of the fair value of the technology and product contingent consideration liabilities related to acquisitions.

Current wording

Change in fair value of contingent consideration liabilities consists of remeasurement of the fair value of the milestone payments associated with our contingent consideration liabilities from acquisitions.

Previous 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.

Current wording

License and collaboration revenue was $9.7 million and $14.3 million for the three months ended September 30, 2025 and 2024, respectively. The decrease in revenue of $4.6 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 $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):

Current wording

Research and development expenses were $109.8 million and $94.3 million for the three months ended September 30, 2025 and 2024, respectively. The following table summarizes our research and development expenses for the three months ended September 30, 2025 and 2024 (in thousands):

Previous wording

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 wording

An increase of $3.7 million of employee related expenses due to the increase in research and development employees from 374 as of September 30, 2024 to 397 as of September 30, 2025;

Previous wording

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 wording

An increase of $0.1 million in external research and development expenses driven by an increase of $1.5 million in outsourced services, primarily due to manufacturing and clinical activities, offset by a decrease in lab supply expenses of $1.4 million due to a decrease in research activities when compared to the prior year.

Previous wording

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 wording

An increase of $2.2 million in employee related costs due to the growth in general and administrative employees from 98 as of September 30, 2024 to 113 as of September 30, 2025; and

Previous wording

The decrease in general and administrative expenses was partially offset by the following:

Current wording

The increase in general and administrative expenses was partially offset by the following:

Previous wording

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 wording

During the three months ended September 30, 2025 and 2024, we recorded $2.8 million and $0.2 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 was paid in June 2021; the remaining success payment obligations are still outstanding as of September 30, 2025 and will continue to be revalued at each reporting period.

Previous 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.

Current wording

During the three months ended September 30, 2025 and 2024, we recorded $4.9 million of other income and $2.1 million of other expense, respectively, as a result of changes in the fair value of our investment in Prime common stock and the sale of our investment in Verve's common stock.

Previous 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.

Current wording

During the three months ended September 30, 2025 and 2024, we recorded $1.0 million of other income and less than $0.1 million of other expense, respectively, related to the change in fair value of the milestone payments related to our contingent consideration liabilities.

Previous wording

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 wording

Interest and other income (expense), net was $10.9 million and $12.1 million for the three months ended September 30, 2025 and 2024, respectively. The change was primarily due to a decline in interest rates during the three months ended September 30, 2025 compared to the three months ended September 30, 2024.

Previous wording

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 wording

License and collaboration revenue was $25.6 million and $33.5 million for the nine months ended September 30, 2025 and 2024, respectively. The decrease in revenue of $7.8 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 $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 wording

Research and development expenses were $310.3 million and $266.1 million for the nine months ended September 30, 2025 and 2024, respectively. The following table summarizes our research and development expenses for the nine months ended September 30, 2025 and 2024 (in thousands):

Previous 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;

Current wording

An increase of $25.4 million in external research and development expenses driven by $26.0 million in outsourced services, primarily due to manufacturing and clinical activities, partially offset by a decrease in lab supply expenses of $0.6 million due to decreased research activities when compared to the prior year;

Previous 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

Current wording

An increase of $13.0 million of employee related expenses due to the increase in research and development employees from 374 as of September 30, 2024 to 397 as of September 30, 2025; and

Previous wording

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 wording

General and administrative expenses were $81.5 million and $82.9 million for the nine months ended September 30, 2025 and 2024, respectively. The decrease of $1.3 million was primarily due to the following:

Previous wording

A decrease of $1.0 million in legal expenses.

Current wording

A decrease of $0.4 million in legal expenses; and

Previous 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.

Current wording

An increase of $5.7 million in employee related costs due to the growth in general and administrative employees from 98 as of September 30, 2024 to 113 as of September 30, 2025.

Previous 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.

Current wording

During the nine months ended September 30, 2025 and 2024, we recorded $0.7 million and $2.4 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 September 30, 2025 and will continue to be revalued at each reporting period.

Previous wording

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 wording

During the nine months ended September 30, 2025 and 2024, we recorded $7.3 million of other income and $13.0 million of other expense, respectively, as a result of changes in the fair value of our investment in Prime's common stock and the sale of our investment in Verve's common stock.

Previous wording

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.

Current wording

During each of the nine months ended September 30, 2025 and 2024, we recorded $0.9 million and $1.6 million of other income, respectively, related to the change in fair value of the milestone payments related to our contingent consideration liabilities.

Previous 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.

Current wording

Interest and other income (expense), net was $33.1 million and $38.2 million for the nine months ended September 30, 2025 and 2024, respectively. The change was primarily due to lower interest rates as of September 30, 2025 compared to September 30, 2024 and one-time credits received during the nine months ended September 30, 2024.

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 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.

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 September 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 nine months ended September 30, 2025.