Editas Medicine Inc
For the period ended Sep 30, 2024. Filed Nov 4, 2024.
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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.
Sep 30, 2024 · Filed Nov 4, 2024
EDIT$2.57+0.8%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2024-06-30; filed 2024-08-07.
0001650664-24-000097
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2024-09-30; filed 2024-11-04.
0001650664-24-000121
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 136 previous paragraphs and 135 current paragraphs.
78 unchanged; 44 reworded; 10 added; 13 removed; 1 previous and 3 current paragraphs rearranged.
Tables are not compared: 7 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 (10)
Current report
In October 2024, we announced that we achievedin vivo preclinical proof of concept of HBG1/2 editing in hematopoietic stem and progenitor cells (“HSPC”) and fetal hemoglobin (“HbF”) induction in humanized mice engrafted with human hematopoietic stem cells and lacking their own hematopoietic cells. Utilizing a hematopoietic stem cell targeting strategy and a novel, Editas-proprietary targeted lipid nanoparticle (“tLNP”) for extrahepatic tissue delivery, we enabled an editing level of 29% of HSPCs after a single dose. Editing with our proprietary tLNP formulation resulted in the functional outcome of HbF induction, indicated by the presence of HbF expressing human red blood cells (on average 20%) that populate in the host by one month. We expect to provide an update on ourin vivo progress and pipeline development in the first quarter of 2025.
Current report
•approximately $2.0 million in increased employee related expenses related to increased headcount to support business operations due to the progression of our reni-cel program;
Current report
•approximately $0.4 million in increased professional service expenses.
Current report
These increases were partially offset by approximately $0.4 million in decreased facility and other expenses.
Current report
Comparison of the Nine Months ended September 30, 2024 and 2023
Current report
The increase in research and development expenses for the nine months ended September 30, 2024 compared to the nine months ended September 30, 2023 was primarily attributable to:
Current report
•approximately $2.1 million in increased other expenses related to increased consulting fees to support clinical and quality initiatives in connection with our reni-cel program.
Current report
General and administrative expenses increased by $0.4 million to $55.6 million for the nine months ended September 30, 2024 compared to $55.2 million for the nine months ended September 30, 2023. The following table summarizes our general and administrative expenses for the nine months ended September 30, 2024 and 2023, together with the changes in those items in dollars (in thousands) and the respective percentages of change:
Current report
The increase in general and administrative expenses for the nine months ended September 30, 2024 compared to the nine months ended September 30, 2023 was primarily attributable to:
Current report
•the outcome of our process to partner or out-license reni-cel to focus our resources on in vivo pipeline development.
Removed paragraphs (13)
Previous report
In the RUBY clinical trial, all 18 patients were free of vaso-occlusive events since reni-cel infusion with follow-up ranging from 2.4 to 22.8 months. Patients demonstrated early normalization of total hemoglobin (“Hb”) with a mean within the normal range at more than 14g/dL and rapid and sustained improvements in fetal hemoglobin (“HbF”) well above levels of over 40%. Across patients with at least six months follow-up, at month six, the mean total Hb was 14.3 g/dL for nine patients with a mean HbF of 48.5% for 10 patients. The mean percentage of F-cells increased early and were sustained at over 90% from month four through subsequent follow-ups for all 12 patients with at least four months follow-up. Mean corpuscular fetal hemoglobin of HbF-containing red cells (F-cells) was sustained above the anti-sickling threshold of 10 pg/F-cell by month three after reni-cel infusion for all 14 patients with at least three months follow-up. All patients in the RUBY trial showed sustained high levels of editing in the HBG1 and HBG2 promoter regions. Markers of hemolysis had been normalized or improved in patients treated with reni-cel. Reni-cel was well-tolerated and demonstrated a safety profile consistent with myeloablative conditioning with busulfan, the regimen that is necessary for current gene editing therapies for SCD and TDT, and autologous hematopoietic stem cell transplant by all 18 evaluated RUBY trial patients. Patients in the RUBY trial underwent a median of two apheresis and mobilization cycles. After reni-cel infusion, all 18 patients demonstrated successful neutrophil and platelet engraftment. Neutrophil engraftment occurred at a median of 23 days, and platelet engraftment occurred at a median of 24 days. No serious adverse events related to reni-cel treatment in the RUBY trial have been reported.
Previous report
In the EdiTHAL clinical trial, patients demonstrated early and robust total Hb and HbF increases, with total Hb rising above the transfusion independence threshold of 9.0 g/dL. For the six patients with at least six months follow-up, the mean total Hb and HbF concentrations at month six were 12.1 g/dL and 10.9 g/dL, respectively, which were sustained at or above the transfusion threshold from six months through subsequent follow-ups. All seven patients were transfusion free for a range of 4.1 to 12.8 months after receiving the last red blood cell transfusion at 0.5-2.2 months post-reni-cel infusion. All patients in the EdiTHAL trial showed sustained high levels of editing in the HBG1 and HBG2 promoter regions. Reni-cel was well-tolerated and demonstrated a safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant by all seven evaluated EdiTHAL trial patients. All patients in the EdiTHAL trial underwent one apheresis and mobilization cycle. After reni-cel infusion, all seven patients demonstrated successful neutrophil and platelet engraftment. Neutrophil engraftment occurred at a median of 23 days, and platelet engraftment occurred at a median of 38 days. No serious adverse events related to reni-cel treatment in the EdiTHAL trial have been reported.
Previous report
•approximately $0.3 million in decreased other expenses; and
Previous report
•approximately $0.2 million in increased stock-based compensation expenses; and
Previous report
•approximately $0.2 million in increased employee related expenses.
Previous report
Comparison of the Six Months ended June 30, 2024 and 2023
Previous report
The increase in research and development expenses for the six months ended June 30, 2024 compared to the six months ended June 30, 2023 was primarily attributable to:
Previous report
•approximately $0.4 million in increased stock-based compensation expense; and
Previous report
General and administrative expenses decreased by $2.7 million to $37.5 million for the six months ended June 30, 2024 compared to $40.2 million for the six months ended June 30, 2023. The following table summarizes our general and administrative expenses for the six months ended June 30, 2024 and 2023, together with the changes in those items in dollars (in thousands) and the respective percentages of change:
Previous report
The decrease in general and administrative expenses for the six months ended June 30, 2024 compared to the six months ended June 30, 2023 was primarily attributable to:
Previous report
•approximately $0.1 million in decreased intellectual property and patent related fees due to reduced legal activity related to reductions in intellectual property defense.
Previous report
These decreases were partially offset by:
Previous report
•approximately $0.8 million in increased facility and other expense primarily related to increased facility operation costs and information technology costs.
Reworded paragraphs (44)
Previous wording
Our lead program, reni-cel, is an experimentalex vivo gene-edited medicine to treat sickle cell disease (“SCD”), a severe inherited blood disease that causes premature death, and transfusion-dependent beta thalassemia (“TDT”), the most severe form of beta-thalassemia, another inherited blood disorder characterized by severe anemia. We are investigating reni-cel in a single Phase 1/2/3 clinical trial, referred to as the RUBY trial, for the treatment of severe SCD. In 2022, we dosed the first patient in the RUBY trial, and after completing sequential dosing of the first two patients, we commenced concurrent patient dosing in the first quarter of 2023. We previously completed enrollment of the adult cohort and continue to dose adult patients. In the second quarter of 2024, we completed enrollment of the adolescent cohort and are concurrently manufacturing drug product and scheduling dosing for the initial adolescent cohort patients. Based on our discussions to date with the U.S. Food and Drug Administration (the “FDA”), we expect any future biologics license application package for reni-cel for the treatment of severe SCD to be similar in number of patients and duration of follow-up to the package submitted for a previously FDA-approved gene editing medicine for severe SCD.
Current wording
Our lead program, reni-cel, is an experimentalex vivo gene-edited medicine to treat sickle cell disease (“SCD”), a severe inherited blood disease that causes premature death, and transfusion-dependent beta thalassemia (“TDT”), the most severe form of beta-thalassemia, another inherited blood disorder characterized by severe anemia. We are investigating reni-cel in a single Phase 1/2/3 clinical trial, referred to as the RUBY trial, for the treatment of severe SCD. In 2022, we dosed the first patient in the RUBY trial, and after completing sequential dosing of the first two patients, we commenced concurrent patient dosing in the first quarter of 2023. We continue to dose adult patients and have dosed 28 patients as of the date of this Quarterly Report on Form 10-Q. We previously completed enrollment of the adult and adolescent cohorts and continue to manufacture drug product and schedule dosing for the initial adolescent cohort patients. Based on our discussions to date with the U.S. Food and Drug Administration (the “FDA”), we expect any future biologics license application package for reni-cel for the treatment of severe SCD to be similar in number of patients and duration of follow-up to the package submitted for a previously FDA-approved gene editing medicine for severe SCD.
Previous wording
In June 2024, we presented new safety and efficacy data as of a May 8, 2024 data cutoff date in 18 patients treated with reni-cel in the RUBY clinical trial and in seven patients treated with reni-cel in the EdiTHAL clinical trial. We remain on track to present additional clinical data from both the RUBY and EdiTHAL trials by year-end 2024.
Current wording
In June 2024, we presented new safety and efficacy data as of a May 8, 2024 data cutoff date in 18 patients treated with reni-cel in the RUBY clinical trial and in seven patients treated with reni-cel in the EdiTHAL clinical trial. We are on track to present additional safety and efficacy clinical data, including hematology parameters, vaso-occlusive events, and patient reported outcomes, from 28 patients in the RUBY trial in December 2024 at the American Society of Hematology Annual Meeting and Exposition, as well as additional clinical data from the EdiTHAL trial by year-end 2024. In October 2024, we announced that we have initiated a global process to partner or out-license reni-cel to focus our resources onin vivo pipeline development.
Previous wording
We are also pursuing the development of next generationin vivo administered gene editing medicines, in which the medicine is injected or infused into the patient to edit the cells inside their body. We are initially focused on editing HSCs through targeted delivery of our AsCas12a enzyme to our clinically validated HBG1 and HBG2 promotor site. Our internal development efforts leverage our indel CRISPR technology we use to upregulate the expression of the gamma globin gene, a functional homolog of the beta globin gene, through direct editing of the HBG1/2 promotor site in ourex vivo reni-cel program. Using this approach, we are pursuing anin vivo strategy aimed at driving functional upregulation of gene expression to address loss of function or deleterious mutations in genetic diseases in rare and orphan disease patient populations, from which we intend to expand to diseases with larger patient populations. We are evaluating lipid nanoparticles for delivery of gene editing cargo into multiple tissue types with multiple companies, and are also evaluating additional, next generation delivery technologies. We are on track to establishin vivo preclinical proof-of-concept for an undisclosed indication by year-end 2024.
Current wording
We are also pursuing the development of next generationin vivo administered gene editing medicines, in which the medicine is injected or infused into the patient to edit the cells inside their body. We are initially focused on editing HSCs through targeted delivery of our AsCas12a enzyme to our clinically validated HBG1 and HBG2 promotor site. Our internal development efforts leverage our indel CRISPR technology we use to upregulate the expression of the gamma globin gene, a functional homolog of the beta globin gene, through direct editing of the HBG1/2 promotor site in ourex vivo reni-cel program. Using this approach, we are pursuing anin vivo strategy aimed at driving functional upregulation of gene expression to address loss of function or deleterious mutations in genetic diseases in rare and orphan disease patient populations, from which we intend to expand to diseases with larger patient populations. We are utilizing lipid nanoparticles for delivery of gene editing cargo into multiple tissue types, while also evaluating additional, next generation delivery technology.
Previous wording
In December 2023, we and Vertex Pharmaceuticals Incorporated (“Vertex”) entered into a license agreement, under which Vertex obtained a non-exclusive license for our Cas9 gene editing technology forex vivo gene editing medicines targeting the BCL11A gene in the fields of SCD and TDT, including Vertex’s CASGEVYTM (exagamglogene autotemcel). We received a $50.0 million upfront cash payment in the fourth quarter of 2023 and the 2024 annual license fee of $10.0 million in the first quarter of 2024. We are eligible to receive an additional $50.0 million contingent upfront payment. We are also eligible to receive further annual license fees, ranging from $10.0 million to $40.0 million annually, inclusive of certain sales-based annual license fee increases, through 2034. We are required to pay The Broad Institute, Inc. (“Broad”) and the President and Fellows of Harvard College (“Harvard”) a mid-double-digit percentage of amounts received from Vertex under the license agreement as it relates to Cas9 technology licensed by us from Broad and Harvard.
Current wording
In December 2023, we and Vertex Pharmaceuticals Incorporated (“Vertex”) entered into a license agreement, under which Vertex obtained a non-exclusive license for our Cas9 gene editing technology for ex vivo gene editing medicines targeting the BCL11A gene in the fields of SCD and TDT, including Vertex’s CASGEVYTM (exagamglogene autotemcel). We received a $50.0 million upfront cash payment in the fourth quarter of 2023 and the 2024 annual license fee of $10.0 million in the first quarter of 2024. The license agreement further provides for the payment by Vertex of a potential additional $50.0 million contingent upfront payment and further annual license fees, ranging from $10.0 million to $40.0 million annually, inclusive of certain sales-based annual license fee increases, through 2034. We are required to pay The Broad Institute, Inc. (“Broad”) and the President and Fellows of Harvard College (“Harvard”) a mid-double-digit percentage of amounts payable to us from Vertex under the license agreement as it relates to Cas9 technology licensed by us from Broad and Harvard. In October 2024, we entered into an agreement (the “DRI Agreement”) with a wholly owned subsidiary of DRI Healthcare Trust (“DRI”) providing for an upfront cash payment by DRI to us of $57.0 million in exchange for the acquisition by DRI of up to 100% of certain of the annual license fees owed to us under the Vertex license agreement, which fees range from $5.0 million to $40.0 million per year (inclusive of certain sales-based annual license fees that may become due), and a mid-double-digit percentage of the $50.0 million contingent upfront payment, in each case after subtracting amounts owed by us to Broad and Harvard. We have retained rights to our portions of certain other sales-based annual license fees and the contingent upfront payment that may become due under the license agreement with Vertex, and the amounts that correspond to our licensor obligations.
Previous wording
Since inception, we have incurred significant operating losses. Our net losses were $129.6 million and $89.3 million for the six months ended June 30, 2024 and 2023, respectively. As of June 30, 2024, we had an accumulated deficit of $1.4 billion. We expect to continue to incur significant expenses and operating losses for the foreseeable future. Our net losses may fluctuate significantly from quarter to quarter and from year to year. We anticipate that our expenses will increase substantially as we continue our current research programs and our preclinical development activities; progress the clinical development of reni-cel; seek to identify additional research programs and additional product candidates; initiate preclinical testing and clinical trials for other product candidates we identify and develop; maintain, expand, and protect our intellectual property portfolio, including reimbursing our licensors for such expenses related to the intellectual property that we in-license from such licensors; further develop our gene editing platform; hire additional clinical, quality control, and scientific personnel; and incur additional costs associated with operating as a public company. We do not expect to be profitable for the year ending December 31, 2024 or the foreseeable future.
Current wording
Since inception, we have incurred significant operating losses. Our net losses were $191.7 million and $134.3 million for the nine months ended September 30, 2024 and 2023, respectively. As of September 30, 2024, we had an accumulated deficit of $1.4 billion. We expect to continue to incur significant expenses and operating losses for the foreseeable future. Our net losses may fluctuate significantly from quarter to quarter and from year to year. We anticipate that our expenses will increase substantially as we continue our current research programs and our preclinical development activities; progress the clinical development of reni-cel; seek to identify additional research programs and additional product candidates; initiate preclinical testing and clinical trials for other product candidates we identify and develop; maintain, expand, and protect our intellectual property portfolio, including reimbursing our licensors for such expenses related to the intellectual property that we in-license from such licensors; further develop our gene editing platform; hire additional clinical, quality control, and scientific personnel; and incur additional costs associated with operating as a public company. We do not expect to be profitable for the year ending December 31, 2024 or the foreseeable future.
Previous wording
Pursuant to the license agreement with Vertex, we received a $50.0 million upfront cash payment in the fourth quarter of 2023 upon execution of the agreement and the 2024 annual license fee of $10.0 million in the first quarter of 2024. We are eligible to receive an additional $50.0 million contingent upfront payment. We are also eligible to receive further annual license fees, ranging from $10.0 million to $40.0 million annually, inclusive of certain sales-based annual license fee increases, through 2034.
Current wording
Pursuant to the license agreement with Vertex, we received a $50.0 million upfront cash payment in the fourth quarter of 2023 upon execution of the agreement and the 2024 annual license fee of $10.0 million in the first quarter of 2024. The license agreement further provides for the payment by Vertex of a potential additional $50.0 million contingent upfront payment and further annual license fees, ranging from $10.0 million to $40.0 million annually, inclusive of certain sales-based annual license fee increases, through 2034.
Previous wording
For the six months ended June 30, 2024 and June 30, 2023, other income, net consisted primarily of interest income and the amortization of premiums or discounts on marketable securities.
Current wording
For the nine months ended September 30, 2024 and September 30, 2023, other income, net consisted primarily of interest income and the amortization of premiums or discounts on marketable securities.
Previous wording
Collaboration and other research and development revenues were $0.5 million for the three months ended June 30, 2024 compared to $2.9 million for the same period in 2023. The decrease from the three months ended June 30, 2023 is primarily attributable to reduced drug supply activity with collaborators.
Current wording
Collaboration and other research and development revenues were $0.1 million for the three months ended September 30, 2024 compared to $5.3 million for the same period in 2023. The decrease from the three months ended September 30, 2023 is primarily attributable to the receipt of the upfront payment for the non-exclusive license to Vor Bio in the third quarter of 2023.
Previous wording
Research and development expenses increased by $24.4 million to $54.2 million for the three months ended June 30, 2024 compared to $29.8 million for the same period in 2023. The following table summarizes our research and development expenses for the three months ended June 30, 2024 and 2023, together with the changes in those items in dollars (in thousands) and the respective percentages of change:
Current wording
Research and development expenses increased by $7.1 million to $47.6 million for the three months ended September 30, 2024 compared to $40.5 million for the same period in 2023. The following table summarizes our research and development expenses for the three months ended September 30, 2024 and 2023, together with the changes in those items in dollars (in thousands) and the respective percentages of change:
Previous wording
•approximately $3.3 million in increased employee-related expenses related to increased headcount to support manufacturing, quality, and clinical work related to our reni-cel program; and
Current wording
•approximately $3.3 million in increased employee-related expenses related to increased headcount to support manufacturing, quality, and clinical work related to our reni-cel program;
Previous wording
•approximately $19.9 million in increased external research and development expenses primarily related to clinical and manufacturing costs related to the accelerated progression of our reni-cel program as well as costs attributable toin vivo research and discovery;
Current wording
•approximately $2.5 million in increased external research and development expenses primarily related to clinical and manufacturing costs related to the accelerated progression of our reni-cel program as well as costs attributable toin vivo research and discovery;
Previous wording
•approximately $2.1 million in increased facility expenses primarily related to increased rent expense incurred in connection with a lease commencement for manufacturing space in the second quarter of 2024.
Current wording
•approximately $2.2 million in increased facility expenses primarily related to increased rent expense incurred in connection with a lease commencement for manufacturing space in the second quarter of 2024; and
Previous wording
•approximately $0.3 million in increased other expenses.
Current wording
•approximately $1.8 million in increased other expenses related to external manufacturing cost savings realized in the third quarter of 2023.
Previous wording
•approximately $0.2 million in decreased sublicense and license fees.
Current wording
•approximately $2.1 million in decreased sublicense and license fees related to reduced licensing activity in the third quarter of 2024 compared to 2023; and
Previous wording
•approximately $0.4 million in decreased stock-based compensation expense due primarily to a reduction in the market price of our common stock, resulting in a lower valuation of equity awards granted;
Current wording
•approximately $0.5 million in decreased stock-based compensation expense.
Previous wording
General and administrative expenses increased by $1.0 million to $18.2 million for the three months ended June 30, 2024 compared to $17.2 million for the three months ended June 30, 2023. The following table summarizes our general and administrative expenses for the three months ended June 30, 2024 and 2023, together with the changes in those items in dollars (in thousands) and the respective percentages of change:
Current wording
General and administrative expenses increased by $3.1 million to $18.1 million for the three months ended September 30, 2024 compared to $15.0 million for the three months ended September 30, 2023. The following table summarizes our general and administrative expenses for the three months ended September 30, 2024 and 2023, together with the changes in those items in dollars (in thousands) and the respective percentages of change:
Previous wording
•approximately $1.0 million in increased intellectual property and patent related fees due to increased legal activity;
Current wording
•approximately $0.5 million in increased intellectual property and patent related fees; and
Previous wording
For the three months ended June 30, 2024, and June 30, 2023 other income, net was $4.3 million and $3.8 million, respectively, which primarily relates to interest income and accretion of discounts and premiums associated with marketable securities. The increase is attributable to favorable market rates.
Current wording
For the three months ended September 30, 2024, and September 30, 2023 other income, net was $3.5 million and $5.1 million, respectively, which primarily relates to interest income and accretion of discounts and premiums associated with marketable securities. The decrease is attributable to a reduction in our investments for the three months ended September 30, 2024 compared to the three months ended September 30, 2023.
Previous wording
The following table summarizes our results of operations for the six months ended June 30, 2024 and 2023, together with the changes in those items in dollars (in thousands) and the respective percentages of change:
Current wording
The following table summarizes our results of operations for the nine months ended September 30, 2024 and 2023, together with the changes in those items in dollars (in thousands) and the respective percentages of change:
Previous wording
Collaboration and other research and development revenues decreased by $11.1 million to $1.6 million for the six months ended June 30, 2024 compared to $12.7 million for six months ended June 30, 2023. The decrease was primarily related to the sale of our wholly owned oncology assets and licenses in January 2023.
Current wording
Collaboration and other research and development revenues decreased by $16.4 million to $1.7 million for the nine months ended September 30, 2024 compared to $18.1 million for nine months ended September 30, 2023. The decrease was primarily related to the sale of our wholly owned oncology assets and licenses in January 2023 and the receipt of the upfront payment for the non-exclusive license to Vor Bio in the third quarter of 2023, for which there was no equivalent revenue in 2024 due to the decrease of collaboration license activity.
Previous wording
Research and development expenses increased by $35.4 million to $103.0 million for the six months ended June 30, 2024 compared to $67.6 million for the six months ended June 30, 2023. The following table summarizes our research development expenses for the six months ended June 30, 2024 and 2023, together with the changes in those items in dollars (in thousands) and the respective percentages of change:
Current wording
Research and development expenses increased by $42.5 million to $150.6 million for the nine months ended September 30, 2024 compared to $108.1 million for the nine months ended September 30, 2023. The following table summarizes our research development expenses for the nine months ended September 30, 2024 and 2023, together with the changes in those items in dollars (in thousands) and the respective percentages of change:
Previous wording
•approximately $25.1 million in increased external research and development expenses primarily related to clinical and manufacturing costs related to the accelerated progression of our reni-cel program as well as costs attributable toin vivo research and discovery;
Current wording
•approximately $27.6 million in increased external research and development expenses primarily related to clinical and manufacturing costs related to the accelerated progression of our reni-cel program as well as costs attributable toin vivo research and discovery;
Previous wording
•approximately $3.6 million in increased employee related expenses related to increased headcount to support manufacturing, quality, and clinical work related to our reni-cel program;
Current wording
•approximately $6.9 million in increased employee related expenses related to increased headcount to support manufacturing, quality, and clinical work related to our reni-cel program;
Previous wording
•approximately $1.6 million in increased facility expenses primarily related to increased rent expense incurred in connection with a lease commencement for manufacturing space;
Current wording
•approximately $3.8 million in increased facility expenses primarily related to increased rent expense incurred in connection with a lease commencement for manufacturing space;
Previous wording
•approximately $4.4 million in increased sublicense and license fees paid in connection with licensing activity;
Current wording
•approximately $2.4 million in increased sublicense and license fees paid in connection with licensing activity in 2024; and
Previous wording
•approximately $2.5 million in increased stock-based compensation expenses due primarily to the achievement of certain performance-based vesting of restricted stock units in 2024;
Current wording
•approximately $3.0 million in increased stock-based compensation expenses due primarily to the achievement of certain performance-based vesting of restricted stock units in 2024;
Previous wording
•approximately $0.9 million in increased employee related expenses related to increased headcount to support business operations; and
Current wording
•approximately $2.9 million in increased employee related expenses related to increased headcount to support business operations and progression of reni-cel program;
Previous wording
•approximately $0.3 million in increased facility and other expenses primarily related to increased facility operation costs and information technology costs;
Current wording
•approximately $0.3 million in increased facility and other expense; and
Previous wording
These increases were partially offset by approximately $0.6 million in decreased professional service expenses related to expenses to support strategic initiatives and business development activities in 2023.
Current wording
These increases were partially offset by approximately $6.3 million in decreased professional service expenses primarily related to one-time expenses to support strategic initiatives and business development activities in the nine months ended September 30, 2023.
Previous wording
For the six months ended June 30, 2024 and June 30, 2023, other income, net was $9.3 million and $5.7 million, respectively, which was primarily attributable to interest income, partially offset by accretion of discounts associated with other marketable securities. The increase for the six months ended June 30, 2024 is attributable to increased invested balances and favorable market rates.
Current wording
For the nine months ended September 30, 2024 and September 30, 2023, other income, net was $12.9 million and $10.9 million, respectively, which was primarily attributable to interest income, partially offset by accretion of discounts associated with other marketable securities. The increase for the nine months ended September 30, 2024 is attributable to increased invested balances throughout the nine months ended September 30, 2024 compared to the nine months ended September 30, 2023 and favorable market rates.
Previous wording
As of June 30, 2024, we have raised an aggregate of $1.0 billion in net proceeds through the sale of shares of our common stock in public offerings and at-the-market offerings. We also have funded our business from our research collaboration with BMS, our former strategic alliance with Allergan, and payments received under the license agreement with Vertex. As of June 30, 2024, we had cash, cash equivalents and marketable securities of $318.3 million.
Current wording
As of September 30, 2024, we have raised an aggregate of $1.0 billion in net proceeds through the sale of shares of our common stock in public offerings and at-the-market offerings. We also have funded our business from our research collaboration with BMS, our former strategic alliance with Allergan, and payments received under the license agreement with Vertex. As of September 30, 2024, we had cash, cash equivalents and marketable securities of $265.1 million. Subsequently, in October 2024, we received the $57.0 million payment under the DRI Agreement.
Previous wording
In addition to our existing cash, cash equivalents and marketable securities, we are eligible to earn milestone and other payments under our collaboration agreement with BMS and our other collaboration and license agreements. Our ability to earn applicable milestone and other payments and the timing of earning these amounts are dependent upon the timing and outcome of development, regulatory and commercial activities and, as such, are uncertain at this time. As of June 30, 2024, our right to contingent payments under our collaboration agreement with BMS and our license agreement with Vor Bio, as well as our contingent upfront payment and annual license fees with Vertex, are our only significant committed potential external source of funds.
Current wording
In addition to our existing cash, cash equivalents and marketable securities, we are eligible to earn milestone and other payments under our collaboration agreement with BMS and our other collaboration and license agreements. Our ability to earn applicable milestone and other payments and the timing of earning these amounts are dependent upon the timing and outcome of development, regulatory and commercial activities and, as such, are uncertain at this time. As of September 30, 2024, our right to contingent payments under our collaboration agreement with BMS and our license agreement with Vor Bio, as well as the retained portions of the contingent upfront payment and other amounts under our license agreement with Vertex, are our only significant committed potential external source of funds.
Previous wording
The following table provides information regarding our cash flows for the six months ended June 30, 2024 and 2023 (in thousands):
Current wording
The following table provides information regarding our cash flows for the nine months ended September 30, 2024 and 2023 (in thousands):
Previous wording
Net cash used in operating activities was approximately $106.9 million for the six months ended June 30, 2024, which primarily consisted of operating expenses that related to increasing our research efforts, the focused progression of clinical and manufacturing activities in support of the reni-cel program, sublicense and license payments, and supporting business operations.
Current wording
Net cash used in operating activities was approximately $159.5 million for the nine months ended September 30, 2024, which primarily consisted of operating expenses that related to increasing our research efforts, the focused progression of clinical and manufacturing activities in support of the reni-cel program, sublicense and license payments, and supporting business operations.
Previous wording
Net cash used in operating activities was approximately $74.5 million for the six months ended June 30, 2023, which primarily consisted of progressing our reni-cel program and supporting business operations.
Current wording
Net cash used in operating activities was approximately $109.9 million for the nine months ended September 30, 2023, which primarily consisted of operating expenses that related to increasing our research efforts, the focused progression of clinical and manufacturing activities in support of reni-cel, and supporting business operations.
Previous wording
Net cash provided by investing activities was approximately $47.2 million for the six months ended June 30, 2024, primarily related to the proceeds from the maturities of marketable securities of $138.8 million, and partially offset by the purchases of marketable securities of $86.2 million and purchases of property and equipment of $5.4 million.
Current wording
Net cash provided by investing activities was approximately $131.2 million for the nine months ended September 30, 2024, primarily related to the proceeds from the maturities of marketable securities of $225.7 million, and partially offset by the purchases of marketable securities of $86.2 million and purchases of property and equipment of $8.3 million.
Previous wording
Net cash provided by investing activities was approximately $36.2 million for the six months ended June 30, 2023, primarily related to proceeds from maturities of marketable securities of $107.6 million, partially offset by purchases of marketable securities of $68.2 million and purchases of property and equipment of $3.2 million.
Current wording
Net cash used in investing activities was approximately $66.6 million for the nine months ended September 30, 2023, primarily related to the purchases of marketable securities of $219.8 million, partially offset by the proceeds from the maturities of marketable securities of $156.6 million.
Previous wording
Net cash provided by financing activities was approximately $0.5 million for the six months ended June 30, 2024, related to the proceeds received from issuance of common stock under the employee stock purchase plan of $0.3 million and exercises of options for our common stock of $0.2 million.
Current wording
Net cash provided by financing activities was approximately $0.5 million for the nine months ended September 30, 2024, related to the proceeds received from issuance of common stock under the employee stock purchase plan of $0.3 million and exercises of options for our common stock of $0.2 million.
Previous wording
Net cash provided by financing activities was approximately $117.5 million for the six months ended June 30, 2023, primarily related to proceeds from offering of common stock of $117.1 million after deducting underwriting discounts and commissions and other offering costs, and proceeds received from issuance of common stock under the employee stock purchase plan of $0.4 million
Current wording
Net cash provided by financing activities was approximately $117.5 million for the nine months ended September 30, 2023, primarily related to proceeds from offering of common stock of $117.1 million after deducting underwriting discounts and commissions and other offering costs, and proceeds received from issuance of common stock under the employee stock purchase plan of $0.4 million
Previous wording
We expect that our existing cash, cash equivalents and marketable securities on June 30, 2024, together with the near-term annual license fees and the contingent upfront payment payable under our license agreement with Vertex, will enable us to fund our operating expenses and capital expenditure requirements into 2026. Our forecast of the period of time through which our existing cash and cash equivalents and investments will be adequate to support our operations is a forward-looking statement and involves significant risks and uncertainties. We have based this forecast on assumptions that may prove to be wrong, and actual results could vary materially from our expectations, which may adversely affect our capital resources and liquidity. We could utilize our available capital resources sooner than we currently expect. The amount and timing of future funding requirements, both near- and long-term, will depend on many factors, including, but not limited to:
Current wording
We expect that our existing cash, cash equivalents and marketable securities on September 30, 2024, together with the upfront cash payment from DRI in October 2024, and the retained portions of the payments payable under our license agreement with Vertex, will enable us to fund our operating expenses and capital expenditure requirements into thesecond quarter of 2026. Our forecast of the period of time through which our existing cash and cash equivalents and investments will be adequate to support our operations is a forward-looking statement and involves significant risks and uncertainties. We have based this forecast on assumptions that may prove to be wrong, and actual results could vary materially from our expectations, which may adversely affect our capital resources and liquidity. We could utilize our available capital resources sooner than we currently expect. The amount and timing of future funding requirements, both near- and long-term, will depend on many factors, including, but not limited to:
Showing the first 40 of 44 pairs. Open the source sections for the rest.
Rearranged current paragraphs (3)
Current report — wording carried from elsewhere
•approximately $0.6 million in increased stock-based compensation expenses related to increased headcount;
Current report — wording carried from elsewhere
These increases were partially offset by $0.1 million in decreased stock-based compensation expense.
Current report — wording carried from elsewhere
•approximately $0.4 million in increased intellectual property and patent related fees; and
Rearranged previous paragraphs (1)
Previous report — wording carried elsewhere
•approximately $6.7 million in decreased professional service expenses primarily related to one-time expenses to support strategic initiatives and business development activities in 2023; and