Beam Therapeutics Inc

Previous wording

In May 2024, we announced that the three patients in the sentinel cohort of the BEACON trial have each been dosed and successfully achieved engraftment. Following engraftment of the third patient, we initiated the expansion phase of the trial. We believe that dosing of the first patient in the expansion cohort is imminent. We expect to report data on multiple patients treated with BEAM-101 in the second half of 2024.

Current wording

In May 2024, we announced that the three patients in the sentinel cohort of the BEACON trial have each been dosed and successfully achieved engraftment. Following engraftment of the third patient, we have dosed three patients in the expansion phase of the trial. To date more than twenty patients have cleared screening and enrolled in the trial. We expect to report data on all patients from the sentinel cohort as well as multiple patients from the expansion cohort in the fourth quarter of 2024.

Previous wording

With the high efficiency and precision of our base editors, we aim to utilize our adenine base editors, or ABEs, to precisely correct the E342K point mutation and restore the production of functional AAT protein. In 2020, we demonstrated the ability of base editors to directly correct the mutation causing AATD, providing bothin vitro andin vivo preclinical proof-of-concept for base editing to correct this disease. In March 2024, we announced the clearance of our clinical trial authorisation application, or CTA, for BEAM-302 for the treatment of AATD by the United Kingdom Medicines and Healthcare Products Regulatory Agency. Following CTA clearance, we began enrolling patients in a Phase 1/2 open label, dose escalation study of BEAM-302 at trial sites located in the United Kingdom. The trial will evaluate the safety, pharmacodynamics, pharmacokinetics and efficacy of BEAM-302 initially in patients with AATD-associated lung disease. The study design includes a dose exploration portion followed by a dose expansion portion to identify the optimal dose to take forward in a pivotal study. We expect to initiate the Phase 1/2 trial of BEAM-302 in the first half of 2024.

Current wording

We are conducting a Phase 1/2 open label, dose escalation study of BEAM-302 at trial sites located in the United Kingdom. The trial will evaluate the safety, pharmacodynamics, pharmacokinetics and efficacy of BEAM-302 initially in patients with AATD-associated lung disease. The study design includes a dose exploration portion followed by a dose expansion portion to identify the optimal dose to take forward in a pivotal study. In June 2024, we announced that the first patient had been dosed in the dose exploration portion of the trial. We anticipate continuing to enroll the Phase 1/2 clinical trial and expect to report initial clinical data in 2025.

Previous wording

In October 2023, we presented new preclinical data demonstrating the ability of BEAM-301 to directly correct the R83C mutation. These data showed that a single dose of BEAM-301 restored clinically meaningful endpoints inin vivo rodent disease models out to at least one year. In the first half of 2024, we plan to submit an investigational new drug, or IND, application to evaluate BEAM-301 for the treatment of GSD1a at a select number of trial sites in the United States.

Current wording

In October 2023, we presented new preclinical data demonstrating the ability of BEAM-301 to directly correct the R83C mutation. These data showed that a single dose of BEAM-301 restored clinically meaningful endpoints inin vivo rodent disease models out to at least one year. In July 2024, we received clearance from the U.S. Food and Drug Administration, or the FDA, for our IND for BEAM-301. We are initiating site activation activities for a Phase 1/2 clinical trial for BEAM-301 in patients with GSD1a with patient dosing expected to commence in early 2025.

Previous wording

BEAM-201 is a development candidate comprised of T cells derived from healthy donors that are simultaneously edited at TRAC, CD7, CD52 and PDCD1 and then transduced with a lentivirus encoding for an anti-CD7 chimeric antigen receptor, or CAR, that is designed to create allogeneic CD7 targeting CAR-T cells, resistant to both fratricide and immunosuppression. We have dosed the first patient in a first-in-human Phase 1/2 clinical trial designed to evaluate the safety and efficacy of BEAM-201 in patients with relapsed/refractory T-cell acute lymphoblastic leukemia/T-cell lymphoblastic lymphoma, or T-ALL/T-LL. Key safety endpoints for the trial include treatment-emergent and treatment-related adverse events, and key efficacy endpoints include proportion of patients with complete or partial responses, proportion eligible for HSC transplant and proportion achieving minimal residual disease negative status. We are continuing enrollment in the Phase 1/2 clinical trial and expect to report an initial clinical dataset for BEAM-201 in the second half of 2024 and to seek potential partnership for this and other potentialex vivo CAR-T programs, including our ongoing research into creating next-generation allogeneic cell therapies with multiplex base editing.

Current wording

BEAM-201 is a development candidate comprised of T cells derived from healthy donors that are simultaneously edited at TRAC, CD7, CD52 and PDCD1 and then transduced with a lentivirus encoding for an anti-CD7 chimeric antigen receptor, or CAR, that is designed to create allogeneic CD7 targeting CAR-T cells, resistant to both fratricide and immunosuppression. We have dosed multiple patients in a first-in-human Phase 1/2 clinical trial designed to evaluate the safety and efficacy of BEAM-201 in patients with relapsed/refractory T-cell acute lymphoblastic leukemia/T-cell lymphoblastic lymphoma. Key safety endpoints for the trial include treatment-emergent and treatment-related adverse events, and key efficacy endpoints include proportion of patients with complete or partial responses, proportion eligible for HSC transplant and proportion achieving minimal residual disease negative status. We expect to report an initial clinical dataset for BEAM-201 in the fourth quarter of 2024.

Previous wording

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. Under the terms of the Sana Agreement, licensed products include certain specified allogeneic T cell and stem cell-derived products directed at specified genetic targets, with certain limited rights for Sana to add and substitute such products and targets. The Sana Agreement excludes the grant of any Beam-controlled rights to perform base editing. In January 2023, Sana announced that the U.S. Food and Drug Administration, or FDA cleared its IND application to initiate a first-in-human study of SC291, its CD19-targeted allogeneic CAR-T cell therapy, in patients with various B-cell malignancies. In November 2023, Sana announced that the FDA cleared its IND application to initiate a first-in-human trial of SC291, in patients with various B-cell mediated autoimmune diseases. In January 2024, Sana announced that the FDA cleared its IND application to initiate a first-in-human trial of SC262, its CD22-directed allogeneic CAR-T cell therapy, in patients with relapsed or refractory B-cell malignancies. In connection with each of the foregoing events, Sana made immaterial milestone payments to us under the Sana Agreement.

Current wording

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. Under the terms of the Sana Agreement, licensed products include certain specified allogeneic T cell and stem cell-derived products directed at specified genetic targets, with certain limited rights for Sana to add and substitute such products and targets. The Sana Agreement excludes the grant of any Beam-controlled rights to perform base editing. In January 2023, Sana announced that the FDA cleared its IND application to initiate a first-in-human study of SC291, its CD19-targeted allogeneic CAR-T cell therapy, in patients with various B-cell malignancies. In November 2023, Sana announced that the FDA cleared its IND application to initiate a first-in-human trial of SC291, in patients with various B-cell mediated autoimmune diseases. In January 2024, Sana announced that the FDA cleared its IND application to initiate a first-in-human trial of SC262, its CD22-directed allogeneic CAR-T cell therapy, in patients with relapsed or refractory B-cell malignancies. In connection with each of the foregoing events, Sana made immaterial milestone payments to us under the Sana Agreement.

Previous wording

We were founded in January 2017 and began operations in July 2017. Since our inception, we have devoted substantially all of our resources to building our base editing platform and advancing development of our portfolio of programs, establishing and protecting our intellectual property, conducting research and development activities, organizing and staffing our company, conducting clinical trials, business planning, raising capital and providing general and administrative support for these operations. To date, we have financed our operations primarily through the sales of our redeemable convertible preferred stock, proceeds from offerings of our common stock and payments received under collaboration and license agreements.

Current wording

We were founded in January 2017 and began operations in July 2017. Since our inception, we have devoted substantially all of our resources to building our base editing platform and advancing development of our portfolio of programs, establishing and protecting our intellectual property, conducting research and development activities, organizing and staffing our company, conducting clinical trials, maintaining and expanding internal manufacturing capabilities, business planning, raising capital and providing general and administrative support for these operations. To date, we have financed our operations primarily through the sales of our redeemable convertible preferred stock, proceeds from offerings of our common stock and payments received under collaboration and license agreements.

Previous wording

We are an early-stage company, and the majority of our programs are at a preclinical or early 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, 2024 and 2023 were $98.7 million and $96.5 million, respectively. As of March 31, 2024, we had an accumulated deficit of $1.3 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 early 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, 2024 and 2023 were $189.7 million and $179.2 million, respectively. As of June 30, 2024, we had an accumulated deficit of $1.4 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, 2024 and 2023, we recognized $7.4 million and $24.2 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 six months ended June 30, 2024 and 2023, we recognized $19.2 million and $44.3 million of license and collaboration revenue, respectively.

Previous wording

License and collaboration revenue was $7.4 million and $24.2 million for the three months ended March 31, 2024 and 2023, respectively. The decline in revenue of $16.8 million is due to the timing of research activities on our license and collaboration programs. License and collaboration revenue represents revenue recorded under each of the Pfizer, Apellis, Verve, and Orbital Agreements.

Current wording

License and collaboration revenue was $11.8 million and $20.1 million for the three months ended June 30, 2024 and 2023, respectively. The decline in revenue of $8.3 million is due to the lower level of research activities on our license and collaboration programs. License and collaboration revenue represents revenue recorded under each of the Pfizer, Apellis, Verve, and Orbital Agreements.

Previous wording

Research and development expenses were $84.8 million and $99.6 million for the three months ended March 31, 2024 and 2023, respectively. The following table summarizes our research and development expenses for the three months ended March 31, 2024 and 2023 (in thousands):

Current wording

Research and development expenses were $87.0 million and $97.6 million for the three months ended June 30, 2024 and 2023, respectively. The following table summarizes our research and development expenses for the three months ended June 30, 2024 and 2023 (in thousands):

Previous wording

An increase of $1.0 million of facility and IT allocated costs, including depreciation. This increase was due to the expense allocated to research and development related to our leased facilities; and

Current wording

An increase of $1.9 million of facility and IT allocated costs, including depreciation, due to the expense allocated to research and development related to our leased facilities; and

Previous wording

General and administrative expenses were $26.7 million and $23.5 million for the three months ended March 31, 2024 and 2023, respectively. The increase of $3.2 million was primarily due to the following:

Current wording

General and administrative expenses were $29.6 million and $24.7 million for the three months ended June 30, 2024 and 2023, respectively. The increase of $5.0 million was primarily due to the following:

Previous wording

An increase of $0.4 million in legal costs.

Current wording

An increase of $1.2 million in legal costs.

Previous wording

During the three months ended March 31, 2024 and 2023, we recorded $2.9 million of expense and $5.6 million of other income, respectively, 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. A portion of the success payment obligations were paid in June 2021; the remaining success payment obligations are still outstanding as of March 31, 2024 and will continue to be revalued at each reporting period.

Current wording

During the three months ended June 30, 2024 and 2023, we recorded $5.5 million of other income and $0.9 million of other expense, respectively, 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. A portion of the success payment obligations were paid in June 2021; the remaining success payment obligations are still outstanding as of June 30, 2024 and will continue to be revalued at each reporting period.

Previous wording

During the three months ended March 31, 2024 and 2023, we recorded $0.1 million and $0.3 million of other expense, respectively, related to the change in fair value of the Guide technology and product contingent consideration liabilities.

Current wording

During the three months ended June 30, 2024 and 2023, we recorded $1.8 million and $2.2 million of other income, respectively, related to the change in fair value of the Guide technology and product contingent consideration liabilities.

Previous wording

Interest and other income (expense), net was $11.8 million and $10.0 million of net income for the three months ended March 31, 2024 and 2023, respectively. The change was primarily due to increases in interest income driven by increased market rates.

Current wording

Interest and other income (expense), net was $14.2 million and $12.0 million of net income for the three months ended June 30, 2024 and 2023, respectively. The change was primarily due to increases in interest income driven by increased market rates.

Previous wording

A $16.2 million decrease in external research and development expenses driven by a $10.8 million decrease in outsourced services, driven primarily by the strategic reprioritization of pipeline programs and fewer IND-enabling activities, partially offset by increased clinical activities for BEAM-101 and BEAM-302. Also contributing to the decrease in expenses is a decline of $5.4 million in lab supplies primarily driven by a decrease in research and development activities as well as a shift of programs from research into development;

Current wording

A decrease of $28.9 million in external research and development expenses driven by a $19.3 million decrease in outsourced services driven primarily by the strategic reprioritization of pipeline programs, timing of manufacturing spend and fewer IND-enabling activities, partially offset by increased clinical activities for BEAM-101 and BEAM-302. Also contributing to the decrease in expenses is a decline of $9.6 million in lab supply expenses, primarily due to a decrease in research and development activities as well as a shift of programs from research into development;

Previous wording

A decrease of $2.8 million of employee related expenses due to a reduction in the number of research and development employees from 429 at March 31, 2023 to 346 at March 31, 2024;

Current wording

A decrease of $7.3 million of employee related expenses due to a reduction in the number of research and development employees from 444 as of June 30, 2023 to 364 as of June 30, 2024;

Previous wording

During the three months ended March 31, 2024 and 2023, we recorded $3.4 million and $12.8 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 six months ended June 30, 2024 and 2023, we recorded $10.9 million and $6.6 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

In October 2023, we entered into the Lilly Agreement, 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. We received a $200.0 million upfront 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. In connection with the Lilly Agreement, we and Lilly entered into a Stock Purchase Agreement providing for the sale and issuance of 2,004,811 shares of our common stock to Lilly for an aggregate purchase price of $50.0 million. We received the consideration under the Stock Purchase Agreement in October 2023 and the upfront payment in November 2023. We have accrued approximately $42.3 million of contingent obligations that may be due associated with payments received under the Lilly Agreement for which discussions are continuing related to the potential applicability to such payments of the terms of license agreements.

Current wording

In October 2023, we entered into the Lilly Agreement, 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. We received a $200.0 million upfront 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. In connection with the Lilly Agreement, we and Lilly entered into a Stock Purchase Agreement providing for the sale and issuance of 2,004,811 shares of our common stock to Lilly for an aggregate purchase price of $50.0 million. We received the consideration under the Stock Purchase Agreement in October 2023 and the upfront payment in November 2023. We have accrued approximately $21.6 million of contingent obligations that may be due associated with payments received under the Lilly Agreement for which discussions are continuing related to the potential applicability to such payments of the terms of license agreements.

Previous wording

As of March 31, 2024, we had $1.1 billion in cash, cash equivalents, and marketable securities.

Current wording

As of June 30, 2024, we had $1.0 billion in cash, cash equivalents, and marketable securities.

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 right-of-use, or 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

Net cash used in operating activities for the three months ended March 31, 2023 was $109.8 million, consisting of our net loss of $96.5 million, a decrease in deferred revenue of $23.7 million, decreases in accrued expenses and other liabilities of $9.9 million, increases in prepaid expenses and other current assets of $8.4 million, and a decrease in operating lease liabilities totaling $2.6 million. In addition, we recorded noncash items consisting of amortization of investment premiums of $6.9 million and a decrease in the fair value of derivative liabilities of $5.6 million.

Current wording

Net cash used in operating activities for the six months ended June 30, 2023 was $194.5 million, consisting of our net loss of $179.2 million, a decrease in deferred revenue of $42.8 million, decreases in accrued expenses and other liabilities of $10.2 million, increases in prepaid expenses and other current assets of $6.5 million and a decrease in operating lease liabilities totaling $4.1 million. In addition, we recorded noncash items consisting of amortization of investment premiums of $14.5 million and decreases in the fair value of derivative liabilities and contingent consideration liabilities of $4.7 million and $1.9 million, respectively.

Previous wording

These uses of cash were partially offset by noncash items consisting of stock-based compensation expense of $23.9 million, decreases in the fair value of non-controlling equity investments of $12.8 million, depreciation and amortization expense of $4.6 million, changes in operating lease ROU assets of $2.4 million, and an increase in the fair value of contingent consideration liabilities of $0.3 million.

Current wording

These uses of cash were partially offset by an increase in other long-term liabilities of $1.0 million and noncash items consisting of stock-based compensation expense of $50.2 million, decreases in the fair value of non-controlling equity investments of $6.6 million, depreciation and amortization expense of $9.5 million and changes in operating lease ROU assets of $4.8 million.

Previous wording

For the three months ended March 31, 2023, cash provided by investing activities consisted of the net maturities of marketable securities of $31.5 million, offset in part by purchases of property and equipment of $6.0 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

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, 2023, cash used in investing activities consisted of purchases of property and equipment of $22.2 million, partially offset by the net maturities of marketable securities of $6.2 million.

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 Employee Stock Purchase Plan, or ESPP, offset in part by repayments of equipment financing liabilities of $0.3 million.

Previous wording

Net cash provided by financing activities for the three months ended March 31, 2023 consisted of net proceeds from equity offerings of $94.0 million, $3.4 million of proceeds from the exercise of stock options and $1.7 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 $0.6 million and payment of equity offering costs of $0.1 million.

Current wording

Net cash provided by financing activities for the six months ended June 30, 2023 consisted of net proceeds from equity offerings of $201.6 million, $4.1 million of proceeds from the exercise of stock options and $1.7 million of proceeds from the issuance of common stock under our ESPP, offset in part by repayments of equipment financing liabilities of $1.2 million and payment of equity offering costs of $0.2 million.

Previous wording

We lease certain assets under noncancelable operating and finance leases. The leases relate primarily to office space and laboratory space in addition to equipment. As of March 31, 2024, aggregate future minimum commitments under these office and laboratory leases and equipment leases are $243.8 million, of which $18.1 million will be payable in 2024. 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 in addition to equipment. As of June 30, 2024, aggregate future minimum commitments under these office and laboratory leases and equipment leases are $237.9 million, of which $12.1 million will be payable in 2024. 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, 2024, 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 2023 Form 10-K.

Current wording

During the six months ended June 30, 2024, 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 2023 Form 10-K.