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Lexeo Therapeutics, Inc.

For the period ended Mar 31, 2025. Filed May 12, 2025.

View on SEC.gov

About this filing

A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.

Mar 31, 2025 · Filed May 12, 2025

LXEO$2.89-0.5%Stored close · as of Nov 6, 2026

What changed in Management’s Discussion and Analysis?

Read the current filing

Previous report: 10-Q

Period ended 2024-09-30; filed 2024-11-13.

0000950170-24-125943

Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations

Current report: 10-Q

Period ended 2025-03-31; filed 2025-05-12.

0000950170-25-068536

Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations

The 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 104 previous paragraphs and 94 current paragraphs.

58 unchanged; 26 reworded; 10 added; 18 removed; 2 previous and 0 current paragraphs rearranged.

Tables are not compared: 5 previous and 3 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

We are a clinical stage genetic medicine company dedicated to reshaping heart health by applying pioneering science to fundamentally change how cardiovascular diseases are treated. We are advancing a portfolio of therapeutic candidates that take aim at the underlying genetic causes of conditions, including FA cardiomyopathy, PKP2 arrhythmogenic cardiomyopathy, and other devastating diseases with high unmet need.

Current report

Our second most advanced cardiovascular product candidate, LX2020 for the treatment of ACM caused by mutations in the PKP2 gene, referred to as PKP2-ACM, is currently being evaluated in HEROIC-PKP2, an ongoing Phase 1/2 clinical trial. To date, six participants have been enrolled in this trial: three in cohort 1 and three in cohort 2. We have obtained post-treatment cardiac biopsies from two participants in cohort 1; the third cohort 1 participant elected to not undergo the post-treatment biopsy procedure. In these two participants with post-treatment samples, we have observed an increase in PKP2 protein expression in the heart quantified using western blot assay showing a 71% and 115% increase in PKP2 protein expression versus pre-treatment baseline. In addition, the first participant to reach six-months post-treatment experienced a 67% reduction in PVCs from baseline and normalization of QRS duration. Across all participants dosed, LX2020 has been generally well-tolerated with no treatment-related serious adverse events to date. We expect to provide an interim data readout focused on clinical efficacy biomarkers in the second half of 2025.

Current report

In April 2025, we identified approximately $20 million in capital to redeploy towards our LX2006 and LX2020 programs. This capital was redeployed from preclinical and non-cardiac pipeline activities and included a limited reduction in force impacting approximately 15% of employees. The updated capital structure is expected to enable us to execute against key milestones for our clinical-stage pipeline, accelerate work to initiate a registrational study for our LX2006 program by early 2026, and maintain operational runway into 2027.

Current report

We expect that our general and administrative expenses will decrease in the near to medium term future relative to those incurred during the three months ended March 31, 2025 on a quarterly and annualized basis primarily due to lower anticipated third-party legal fees and related costs, as well as lower anticipated administrative personnel and related costs due to the limited reduction in force undertaken in April 2025, while supporting the continued research and development of our programs and the growth of our business.

Current report

Amortization of premium on investments consists of amortization of premium on our investments in U.S. Treasury securities.

Current report

We recognized interest income of $1.2 million and $1.7 million for the three months ended March 31, 2025 and March 31, 2024, respectively, primarily related to interest earned on our investments in U.S. government money market funds and our investments in U.S Treasury securities, with the decrease primarily due to lower interest rates, as well as lower invested balances.

Current report

On March 24, 2025 we filed a prospectus supplement to the prospectus which forms a part of our effective shelf registration statement on Form S-3, dated December 13, 2024, which registers the offering, issuance, and sale of up to $75.0 million of common stock pursuant to our ATM Program. As of March 31, 2025, we did not sell any shares of common stock pursuant to the ATM Program.
Removed paragraphs (18)

Previous report

We are a clinical stage genetic medicine company dedicated to transforming healthcare by applying pioneering science to fundamentally change how disease is treated. By taking aim at the underlying genetic cause of the devastating diseases we target, we seek to create substantial positive impact and reduce the overwhelming burdens placed on people receiving treatment, their caregivers, and healthcare systems. Our current pipeline consists of candidates targeting patient populations that place significant burden on society and are most amenable to our genetic medicine approach.

Previous report

Our lead Alzheimer’s disease product candidate, LX1001, for the treatment of APOE4 homozygous patients with Alzheimer’s disease, is in an ongoing Phase 1/2 trial. In October 2024, we reported interim data across all four dose cohorts at the Clinical Trials on Alzheimer's Disease conference in Madrid, Spain. LX1001 was generally well-tolerated across all dose cohorts with no reports of amyloid-related imaging abnormalities. Four serious adverse events were reported, with three deemed unrelated to treatment and one event of mild-moderate sensorineural hearing loss assessed as possibly related to treatment with repeat audiometry pending. A dose and time-dependent increase in neuroprotective APOE2 expression was observed in all participants with ongoing durability at 12 months, and reductions in cerebrospinal fluid tau biomarkers and tau positron emission tomography were seen in a majority of patients.

Previous report

We are targeting diseases that have seen limited penetration of precision medicine, which we define as medications that treat the underlying molecular mechanism of a disease, and where we believe there is significant opportunity for gene therapy to play a role as a key therapeutic option. We believe the specific indications we are initially targeting, FA cardiomyopathy, PKP2-ACM and APOE4-associated Alzheimer’s disease, are highly amenable to gene therapy, where administration of a single dose has the potential to either restore loss-of-function or minimize gain-of-function mutations by treating the underlying genetic cause of the disease. Although few precision medicines are currently approved for the treatment of cardiovascular conditions or Alzheimer’s disease, recent approvals by the FDA suggest a willingness to approve new precision medicines based on biomarkers and functional endpoints. Together with improved diagnostics and increased testing, these developments may offer one of the most substantial opportunities for the uptake of precision medicines in the global pharmaceutical marketplace.

Previous report

By specifically tailoring our technological approach to each targeted disease, we believe we can optimize our programs to achieve the highest likelihood of having therapeutic impact. We target genetically defined indications in specific sub-groups of patients that offer the potential to demonstrate therapeutic impact through improvement in functional endpoints or biomarkers, have high unmet need and large market opportunities, have established proof-of-concept in relevant preclinical models, and have organized patient advocacy groups and identifiable patient populations. In addition to targeting cardiovascular diseases and APOE4-associated Alzheimer’s disease that we believe can be addressed by our current approach utilizing AAVrh10, we have ongoing discovery efforts to identify next-generation vector technologies with the best potential therapeutic profile. Finally, we continuously seek to bolster our pipeline through relationships with academic institutions, providing us access to cutting edge genetic medicines research which will include not only AAV gene therapy but also other potential therapeutic payload types and non-viral delivery systems. In August 2023, we announced a strategic investment from Sarepta Therapeutics, Inc. to explore collaboration opportunities within our preclinical cardiovascular pipeline.

Previous report

We expect that our general and administrative expenses will increase significantly in the near-to-medium term as we incur additional expenses associated with operating as a public company, including increased expenses for insurance premiums and audit, legal, regulatory, and tax-related services associated with maintaining compliance with the rules and regulations of the Securities and Exchange Commission, or SEC, and standards applicable to companies listed on a national securities exchange, investor relations activities and other administrative and professional services. We also plan to increase our general and administrative headcount to support the continued research and development of our programs and the growth of our business.

Previous report

Fair value adjustment to convertible SAFE Note consists of losses on the estimated fair value of the convertible SAFE Note.

Previous report

The net increase of $5.1 million in general and administrative expenses for the three months ended September 30, 2024 compared to the three months ended September 30, 2023 was primarily due to increases in (i) employee compensation and stock-based compensation expenses of $2.5 million primarily due to increased headcount and equity awards granted to existing employees since September 30, 2023, (ii) third-party legal fees and associated costs of $1.8 million, and (iii) third-party audit, tax, and other professional service provider fees, as well as insurance expenses, of $0.4 million primarily due to our becoming a publicly traded company in November 2023.

Previous report

We recognized interest income of $2.1 million and $0.5 million for the three months ended September 30, 2024 and September 30, 2023, respectively, primarily related to interest earned on our investment in a U.S. government money market fund with an increased average invested balance in 2024 primarily due to the net proceeds received from our IPO and the subsequent partial exercise of the underwriters' option to purchase additional shares in November 2023, as well the net proceeds received from our Private Placement offering in March 2024.

Previous report

Comparison of the nine months ended September 30, 2024 and 2023

Previous report

The net increase of $10.8 million in total research and development expenses for the nine months ended September 30, 2024 compared to the nine months ended September 30, 2023 was primarily due to increases in (i) employee compensation and stock-based compensation expenses of $7.1 million primarily due to increased headcount and equity awards granted to existing employees since September 30, 2023, (ii) CMC expenses of $2.9 million, (iii) net milestone expense of $2.6 million, including a $6.0 million development milestone for our LX2020 program that was achieved during the third quarter of 2024 and subsequently paid to the selling shareholders of Stelios, which was partially offset by a $3.5 million development milestone for our LX2006 program that was achieved and paid to Adverum in the first quarter of 2023, (iv) clinical trial costs of $1.9 million excluding an adjustment reducing estimated accrued clinical trial expenses by $2.2 million for our LX1001 program recorded in the second quarter of 2024, (v) quality assurance expenses of $0.7 million, and (vi) license fees of $0.7 million primarily related to the Third Cornell License Agreement. These increases were partially offset by decreases in (i) non-clinical and preclinical expenses of $3.3 million primarily related to our early stage cardiovascular and central nervous system disease programs, and (ii) estimated accrued clinical trial expenses of $2.2 million resulting from an adjustment recorded in the second quarter of 2024 for our LX1001 program.

Previous report

We recognized interest income of $6.1 million and $1.8 million for the nine months ended September 30, 2024 and September 30, 2023, respectively, primarily related to interest earned on our investment in a U.S. government money market fund with an increased average invested balance in 2024 primarily due to the net proceeds received from our IPO and the subsequent partial exercise of the underwriters' option to purchase additional shares in November 2023, as well the net proceeds received from our Private Placement offering in March 2024.

Previous report

Critical accounting policies and significant judgments and estimates
Reworded paragraphs (26)

Previous wording

Our most advanced cardiovascular product candidate, LX2006 for the treatment of patients with Friedreich ataxia, or FA, cardiomyopathy, is currently being evaluated in SUNRISE-FA, an ongoing Phase 1/2 clinical trial. We have observed an increase in frataxin protein expression in the hearts of four patients that have undergone cardiac biopsies across cohort 1 (n=1) and cohort 2 (n=3). In July 2024, we announced interim clinical data of LX2006 from the ongoing Lexeo-sponsored SUNRISE-FA trial and Cornell investigator-initiated trial, providing baseline characteristics from 11 participants that had been treated and follow-up data from 8 participants that had reached at least 6-months of follow-up, showing improvements in key cardiac biomarkers including left ventricular mass index, lateral wall thickness, and high-sensitivity troponin I. LX2006 has been generally well-tolerated across both SUNRISE-FA and Weill Cornell investigator-initiated trials to date. One possibly treatment-related Grade 2 event of asymptomatic myocarditis was observed one year after dosing; participant with multiple comorbidities and history of flu-like symptoms prior to diagnosis which may have been a contributing factor. A biopsy was performed six weeks after diagnosis and results were negative for myocarditis and participant remains asymptomatic. In November 2024, we announced that we reached alignment with the U.S. Food and Drug Administration, or FDA, on key elements of a registrational development plan for LX2006, including accelerated approval pathway with left ventricular mass index and frataxin protein expression as co-primary registrational endpoints. LX2006 has received Rare Pediatric Disease designation, Orphan Drug designation, Fast Track designation and Regenerative Medicine Advanced Therapy, or RMAT, designation from the FDA, and Orphan Medicinal Product designation from the European Commission. Our second most advanced cardiovascular product candidate, LX2020 for the treatment of arrhythmogenic cardiomyopathy, or ACM, caused by mutations in the PKP2 gene, referred to as PKP2-ACM, is currently being evaluated in HEROIC-PKP2, an ongoing Phase 1/2 clinical trial. We have completed enrollment of cohort 1 and expect to provide an interim data readout from that cohort in the later part of the first quarter or early part of the second quarter of 2025. LX2020 received Fast Track and Orphan Drug designations from the FDA in December 2023.

Current wording

Our most advanced cardiovascular product candidate, LX2006 for the treatment of FA cardiomyopathy, is currently being evaluated in SUNRISE-FA, our ongoing Phase 1/2 clinical trial and in a Cornell investigator-initiated trial. In July 2024, we provided an interim clinical update, which included baseline characteristics from the 11 treated participants across the two studies and data from the eight participants who had reached at least six-months of follow-up as of that time. These data showed improvements in key cardiac biomarkers including left ventricular mass index, lateral wall thickness, and high-sensitivity troponin I. Additionally, in November 2024 we reported that we observed an increase in frataxin protein expression in the hearts of four patients that had undergone cardiac biopsies (cohort 1 (n=1), cohort 2 (n=3)) as measured by liquid chromatography-mass spectrometry, or LCMS, and immunohistochemistry. LX2006 has been generally well-tolerated across both trials to date. One year after dosing, one participant with multiple comorbidities and a history of flu-like symptoms presented with Grade 2 asymptomatic myocarditis. Six weeks later, a biopsy was negative for myocarditis and the participant remains asymptomatic. In November 2024 we announced alignment with the FDA on key elements of a registrational development plan for LX2006, including an accelerated approval pathway with left ventricular mass index and frataxin protein expression as co-primary registrational endpoints. In February 2025, we reached further alignment with the FDA that the frataxin protein expression co-primary endpoint would be evaluated for any increase from baseline in frataxin positive area as measured by immunohistochemistry as opposed to any specific numerical threshold. In April 2025, we provided an interim clinical update, which included baseline characteristics from the 16 treated participants across the two studies, frataxin protein expression from four additional patients that had undergone cardiac biopsies (cohort 3 (n=4)), and data from the 12 participants who had reached at least six-months of follow-up as of that time. These data showed an increase in frataxin expression versus baseline in all patients that had undergone cardiac biopsies (cohort 1 (n=1), cohort 2 (n=3), cohort 3 (n=4)) as measured by LCMS, upon which we reached further alignment with the FDA to measure protein expression. These data also showed dose responsive increases in frataxin expression relative to dose cohorts, improvements in key cardiac biomarkers including left ventricular mass index, lateral wall thickness, and high-sensitivity troponin I, as well as improvements in functional measures including the Modified Friedreich's Ataxia Rating Scale, or mFARS, and Kansas City Cardiomyopathy Questionnaire, or KCCQ-12.

Previous wording

Each of our gene therapy candidates utilizes the vector construct, dose and route of administration that we believe will result in the most favorable biodistribution and safety profile for our product candidate for each disease. Our most advanced cardiovascular and APOE4-associated Alzheimer’s disease programs use the AAVrh10 vector due to its high transduction efficiency in both myocardial cells and neurons, potential for lower toxicity given the opportunity to utilize lower doses compared to other well-established AAV serotypes, and low pre-existing immunity.

Current wording

Each of our gene therapy candidates utilizes the vector construct, dose and route of administration that we believe will result in the most favorable biodistribution and safety profile for our product candidate for each disease. Our most advanced programs use the AAVrh10 vector due to its high transduction efficiency in myocardial cells, potential for lower toxicity given the opportunity to utilize lower doses compared to other well-established AAV serotypes, and low pre-existing immunity.

Previous wording

To date, we have funded our operations primarily through proceeds from the sale of shares of our convertible preferred stock and common stock, including our Private Placement, IPO and the subsequent partial exercise of the underwriters’ 30-day option to purchase additional shares of common stock. As of September 30, 2024, we had $157.0 million of cash and cash equivalents, and we had raised aggregate net proceeds of $100.3 million from our IPO and the subsequent partial exercise of the underwriters’ 30-day option to purchase additional shares of common stock, as well as totals of $88.7 million, $185.0 million and $3.9 million of net proceeds from the Private Placement, the sales of our convertible equity securities, and the convertible SAFE Note, respectively (see Note 8 to the unaudited consolidated financial statements appearing elsewhere in this Quarterly Report for more information). We have incurred significant operating losses since the commencement of our operations. Our ability to generate product revenue sufficient to achieve profitability will depend heavily on the successful development and eventual commercialization of one or more of our current gene therapy candidates or any future gene therapy candidates. Our net losses for the nine months ended September 30, 2024 and the year ended December 31, 2023 were $72.4 million and $66.4 million, respectively, and our accumulated deficit was $254.2 million at September 30, 2024. We expect to continue to incur significant losses for the foreseeable future as we advance our current and future product candidates through preclinical and clinical development, continue to build our operations and operate as a public company.

Current wording

To date, we have funded our operations primarily through proceeds from the sale of shares of our convertible preferred stock and common stock, including our Private Placement, IPO and the subsequent partial exercise of the underwriters’ 30-day option to purchase additional shares of common stock. As of March 31, 2025, we had $106.9 million of cash, cash equivalents, and investments. We have incurred significant operating losses since the commencement of our operations. Our ability to generate product revenue sufficient to achieve profitability will depend heavily on the successful development and eventual commercialization of one or more of our current gene therapy candidates or any future gene therapy candidates. Our net losses for the three months ended March 31, 2025 and the year ended December 31, 2024 were $32.7 million and $98.3 million, respectively, and our accumulated deficit was $312.8 million at March 31, 2025. We expect to continue to incur significant losses for the foreseeable future as we advance our current and future product candidates through preclinical and clinical development, continue to build our operations and operate as a public company.

Previous wording

continue our ongoing and planned clinical trials as well as research and development of our FA cardiomyopathy (LX2006), APOE-associated Alzheimer’s disease (LX1001), and arrhythmogenic cardiomyopathy caused by mutations in the PKP2 gene, or PKP2-ACM (LX2020) programs and other product candidates;

Current wording

continue our ongoing and planned clinical trials as well as research and development of our FA cardiomyopathy (LX2006) and arrhythmogenic cardiomyopathy caused by mutations in the PKP2 gene, or PKP2-ACM (LX2020) programs and other product candidates;

Previous wording

initiate preclinical studies and clinical trials for any additional product candidates that we may pursue in thefuture;

Current wording

initiate preclinical studies and clinical trials for any additional product candidates that we may pursue in the future;

Previous wording

To date, we have not generated any revenue from product sales. If our development efforts for LX2006, LX1001, and LX2020 or any future product candidates, are successful and result in regulatory approval, or if we enter into collaboration or license agreements with third parties, we may generate revenue in the future from product sales, royalties or payments from such collaboration or license agreements, or a combination of product sales and payments from such agreements.

Current wording

To date, we have not generated any revenue from product sales. If our development efforts for LX2006 and LX2020, or any future product candidates, are successful and result in regulatory approval, or if we enter into collaboration or license agreements with third parties, we may generate revenue in the future from product sales, royalties or payments from such collaboration or license agreements, or a combination of product sales and payments from such agreements.

Previous wording

the cost of developing and scaling our manufacturing process and manufacturing product candidates for use in our research, preclinical studies andclinical trials, including under agreements with third parties, such as consultants, contractors and contract manufacturing organizations, or CMOs;

Current wording

the cost of developing and scaling our manufacturing process and manufacturing product candidates for use in our research, preclinical studies and clinical trials, including under agreements with third parties, such as consultants, contractors and contract manufacturing organizations, or CMOs;

Previous wording

We expense research and development costs as incurred. Nonrefundable advance payments for goods and services that will be used in future research and development activities are expensed when the activity has been performed or when the goods have been received rather than when the payment is made. When third-party service providers’ billing terms do not coincide with our period-end, we are required to make estimates of our obligations to those third parties incurred in a given accounting period and record accruals at the end of the period. We base these estimates on our knowledge of the research and development programs, services performed for the period, past history for related activities and the expected duration of the third-party service contract, where applicable. If timelines or contracts are modified based upon changes in the scope of work to be performed, we modify our estimates of accrued expenses accordingly on a prospective basis; therefore, actual results could differ from our estimates. Upfront payments under license agreements are expensed upon receipt of the license, and annual maintenance fees under license agreements are expensed in the period in which they are incurred. Milestone payments under license agreements are accrued, with a corresponding expense being recognized, in the period in which the milestone is determined to be probable of achievement and the related amount is reasonably estimable.

Current wording

We expense research and development costs as expenses when incurred. Nonrefundable advance payments for goods and services that will be used in future research and development activities are expensed when the activity has been performed or when the goods have been received rather than when the payment is made. When third-party service providers’ billing terms do not coincide with our period-end, we are required to make estimates of our obligations to those third parties incurred in a given accounting period and record accruals at the end of the period. We base these estimates on our knowledge of the research and development programs, services performed for the period, past history for related activities and the expected duration of the third-party service contract, where applicable. If timelines or contracts are modified based upon changes in the scope of work to be performed, we modify our estimates of accrued expenses accordingly on a prospective basis; therefore, actual results could differ from our estimates. Upfront payments under license agreements are expensed upon receipt of the license, and annual maintenance fees under license agreements are expensed in the period in which they are incurred. Milestone payments under license agreements are accrued, with a corresponding expense being recognized, in the period in which the milestone is determined to be probable of achievement and the related amount is reasonably estimable.

Previous wording

Research and development activities are central to our business model and account for a significant portion of our operating expenses. Product candidates in later stages of clinical development generally have higher development costs than those in earlier stages of clinical development, primarily due to the increased size and duration of later-stage clinical trials. As a result, we expect our research and development expenses to increase substantially in absolute dollars for the foreseeable future as we further advance LX2006, LX2020, LX1001, and any other future product candidates that we may develop, into and through preclinical studies and clinical trials and pursue regulatory approvals. We cannot determine with certainty the timing of initiation, the duration or the completion costs of current or future preclinical studies and clinical trials of our product candidates due to the inherently unpredictable nature of preclinical and clinical development. Clinical and preclinical development timelines, the probability of success and development costs can differ materially from expectations. We anticipate that we will make determinations as to which product candidates to pursue and how much funding to direct to each product candidate on an ongoing basis in response to the results of ongoing and future research, preclinical studies and clinical trials, regulatory developments and our assessments as to each product candidate’s commercial potential. In addition, we cannot forecast whether any of our current or future product candidates will be subject to future collaborations, when such arrangements will be secured, if at all, and to what degree such arrangements would affect our development plans and capital requirements. We are also unable to predict when, if ever, we will generate revenue from our product candidates to offset our expenses.

Current wording

Research and development activities are central to our business model and account for a significant portion of our operating expenses. Product candidates in later stages of clinical development generally have higher development costs than those in earlier stages of clinical development, primarily due to the increased size and duration of later-stage clinical trials. As a result, we expect our research and development expenses to increase for the foreseeable future as we further advance LX2006 and LX2020, and any other future product candidates that we may develop, into and through preclinical studies and clinical trials and pursue regulatory approvals. We cannot determine with certainty the timing of initiation, the duration or the completion costs of current or future preclinical studies and clinical trials of our product candidates due to the inherently unpredictable nature of preclinical and clinical development. Clinical and preclinical development timelines, the probability of success and development costs can differ materially from expectations. We anticipate that we will make determinations as to which product candidates to pursue and how much funding to direct to each product candidate on an ongoing basis in response to the results of ongoing and future research, preclinical studies and clinical trials, regulatory developments and our assessments as to each product candidate’s commercial potential. In addition, we cannot forecast whether any of our current or future product candidates will be subject to future collaborations, when such arrangements will be secured, if at all, and to what degree such arrangements would affect our development plans and capital requirements. We are also unable to predict when, if ever, we will generate revenue from our product candidates to offset our expenses.

Previous wording

Interest income is primarily related to interest earned from our investment in a U.S. government money market fund, as well as interest earned on interest-bearing demand deposit cash accounts.

Current wording

Interest income is primarily related to interest earned from our investments in U.S. government money market funds and interest earned from our investments in U.S. Treasury securities, as well as interest earned on interest-bearing demand deposit cash accounts and a cash collateral account.

Previous wording

Provision for income taxes consists of U.S. federal and state income taxes in which we conduct business. Since our inception, we have not recorded any income tax benefits for the net losses we have incurred in each year or for our research and development tax credits, as we believe, based upon the weight of available evidence, that it is more likely than not that all of our net operating loss, or NOL, carryforwards and tax credits will not be realized. Accordingly, we have recorded a full valuation allowance against our net deferred tax assets at September 30, 2024, December 31, 2023, September 30, 2023 and December 31, 2022. As of September 30, 2024, December 31, 2023, September 30, 2023 and December 31, 2022, we had no unrecognized tax benefits.

Current wording

Provision for income taxes consists of U.S. federal and state income taxes in the jurisdictions where we conduct business. Since our inception, we have not recorded any income tax benefits for the net losses we have incurred in each year or for our research and development tax credits, as we believe, based upon the weight of available evidence, that it is more likely than not that all of our net operating loss, or NOL, carryforwards and tax credits will not be realized. Accordingly, we have recorded a full valuation allowance against our net deferred tax assets at March 31, 2025, December 31, 2024 and March 31, 2024. As of March 31, 2025, December 31, 2024 and March 31, 2024, we had no unrecognized tax benefits.

Previous wording

The net increase of $6.2 million in total research and development expenses for the three months ended September 30, 2024 compared to the three months ended September 30, 2023 was primarily due to increases in (i) milestone expenses of $6.1 million, consisting primarily of a $6.0 million development milestone for our LX2020 program that was achieved during the third quarter of 2024 and subsequently paid to the selling shareholders of Stelios, (ii) employee compensation and stock-based compensation expenses of $2.4 million primarily due to increased headcount and equity awards granted to existing employees since September 30, 2023, (iii) quality assurance expenses of $0.3 million, (iv) preclinical expenses of $0.2 million, and (v) regulatory and program and portfolio management expenses of $0.2 million. These increases were partially offset by decreases in (i) clinical trial expenses of $1.5 million, (ii) chemistry, manufacturing and controls, or CMC, expenses of $1.4 million, and (iii) non-clinical expenses of $0.2 million.

Current wording

The net increase of $1.4 million in total research and development expenses for the three months ended March 31, 2025 compared to the three months ended March 31, 2024 was primarily due to increases in (i) employee compensation and stock-based compensation expenses of $1.2 million primarily due to increased headcount and equity awards granted since March 31, 2024, (ii) clinical trial costs of $0.3 million, and (iii) non-clinical and preclinical expenses of $0.2 million primarily related to our cardiovascular disease programs. These increases were partially offset by decreases in (i) chemistry, manufacturing, and control, or CMC, expenses of $0.2 million, and (ii) quality assurance expenses of $0.2 million.

Previous wording

The net increase of $14.0 million in general and administrative expenses for the nine months ended September 30, 2024 compared to the nine months ended September 30, 2023 was primarily due to increases in (i) third-party legal fees and associated costs of $6.0 million, (ii) employee compensation and stock-based compensation expenses of $5.5 million primarily due to increased headcount and equity awards granted to existing employees since September 30, 2023, (iii) third-party audit, tax, investor relations, public relations and other professional service provider fees, as well as insurance expenses, of $1.4 million primarily due to our becoming a publicly traded company in November 2023, (iv) travel, software and office supplies expenses of $0.4 million, and (v) capital tax expense of $0.2 million.

Current wording

The net increase of $9.1 million in general and administrative expenses for the three months ended March 31, 2025 compared to the three months ended March 31, 2024 was primarily due to increases in (i) third-party legal fees and associated costs of $7.5 million, (ii) employee compensation and stock-based compensation expenses of $1.4 million primarily due to increased headcount and equity awards granted since March 31, 2024, and (iii) software, office supplies, and dues and subscription expenses of $0.4 million. These increases were partially offset by a decrease in third-party audit, accounting, investor relations, public relations and other professional service provider fees, as well as insurance expenses, of $0.2 million.

Previous wording

Since our inception, we have not generated any revenue from product sales and have incurred significant operating losses and negative cash flows from our operations. We expect to incur significant expenses and operating losses for the foreseeable future as we advance the clinical development of our product candidates. Since our inception through September 30, 2024, we funded our operations primarily with total net proceeds from sales of our common stock, convertible SAFE Note and convertible equity securities of $377.9 million. As of September 30, 2024 and December 31, 2023, we had cash and cash equivalents of $157.0 million and $121.5 million, respectively.

Current wording

Since our inception, we have not generated any revenue from product sales and have incurred significant operating losses and negative cash flows from our operations. We expect to incur significant expenses and operating losses for the foreseeable future as we advance the clinical development of our product candidates. Since our inception through March 31, 2025, we funded our operations primarily with total net proceeds from sales of our common stock, convertible SAFE Note and convertible equity securities of $377.9 million. As of March 31, 2025 and December 31, 2024, we had cash, cash equivalents, and investments of $106.9 million and $128.5 million, respectively.

Previous wording

Based on our current operating plans, we expect the net proceeds from the IPO and subsequent partial exercise of the underwriters' option to purchase additional shares in November 2023 and Private Placement offering in March 2024, together with our existing cash and cash equivalents, will be sufficient to fund our planned operating expenses and capital expenditure requirements into 2027. Our total future capital requirements will depend on many factors and is subject to the risks and uncertainties set forth in the section titled “Risk Factors.”

Current wording

We believe that our cash, cash equivalents, and investments balances will be sufficient to fund our planned operating expenses and capital expenditure requirements into 2027. Our total future capital requirements will depend on many factors and are subject to the risks and uncertainties set forth in “Item 1A Risk Factors.”

Previous wording

The following table summarizes our sources and uses of cash for the nine months ended September 30, 2024 and September 30, 2023 (in thousands):

Current wording

The following table summarizes our sources and uses of cash for the three months ended March 31, 2025 and March 31, 2024 (in thousands):

Previous wording

During the nine months ended September 30, 2024, net cash used in operating activities consisted primarily of our net loss of $72.4 million, which was partially offset by $9.1 million of net cash provided by changes in operating assets and liabilities, $9.0 million of stock-based compensation expense, and $1.5 million of depreciation and amortization of operating and finance right-of-use assets, as well as property and equipment.

Current wording

During the three months ended March 31, 2025, net cash used in operating activities consisted primarily of our net loss of $32.7 million, which was partially offset by $6.7 million of net cash provided by changes in operating assets and liabilities, $3.7 million of stock-based compensation expense and $0.5 million of depreciation and amortization of operating and finance right-of-use assets.

Previous wording

During the nine months ended September 30, 2023, net cash used in operating activities consisted primarily of our net loss of $52.2 million, which was partially offset by $2.8 million of net cash provided by changes in operating assets and liabilities, $2.2 million of stock-based compensation expense, and $1.4 million of depreciation and amortization of operating and finance right-of-use assets, as well as property and equipment.

Current wording

During the three months ended March 31, 2024, net cash used in operating activities consisted primarily of our net loss of $21.7 million, which was partially offset by $4.0 million of net cash provided by changes in operating assets and liabilities, $2.3 million of stock-based compensation expense and $0.5 million of depreciation and amortization of operating and finance right-of-use assets.

Previous wording

During the nine months ended September 30, 2024, net cash used in investing activities was $0.5 million and consisted primarily of purchases of lab equipment.

Current wording

During the three months ended March 31, 2025, net cash provided by investing activities was $15.6 million and consisted of proceeds from maturities of investments.

Previous wording

During the nine months ended September 30, 2023, net cash used in investing activities of $0.1 million consisted primarily of purchases of lab equipment and internal use software.

Current wording

During the three months ended March 31, 2024, net cash used in investing activities was $0.2 million and consisted of purchases of lab equipment and internal use software.

Previous wording

During the nine months ended September 30, 2024, net cash provided by financing activities consisted primarily of the net proceeds received from the Private Placement offering of $88.7 million, as well as $0.5 million of proceeds received from the exercise of stock options, which were partially offset by $0.3 million of principal payments made on equipment finance leases.

Current wording

During the three months ended March 31, 2025, net cash provided by financing activities consisted of proceeds received from the common stock issuances under the 2023 ESPP for $0.2 million, which was partially offset by $0.2 million of principal payments made on equipment finance leases.

Previous wording

During the nine months ended September 30, 2023, net cash provided by financing activities consisted primarily of $4.0 million of proceeds from the issuance of the convertible SAFE Note, which was partially offset by $0.3 million of principal payments made on equipment finance leases.

Current wording

During the three months ended March 31, 2024, net cash provided by financing activities consisted primarily of the net proceeds received from the Private Placement offering of $88.8 million, which was partially offset by $0.1 million of principal payments made on equipment finance leases.

Previous wording

We expect our expenses and capital requirements to increase significantly in connection with our ongoing activities, particularly as we advance our lead product candidates and other development programs, in addition to the costs associated with operating as a public company. Accordingly, and beyond the net proceeds raised in the IPO and the subsequent partial exercise of the underwriters' option to purchase additional shares of common stock, as well as the Private Placement, we will continue to require substantial additional funding to support our continuing operations.

Current wording

We expect our expenses and capital requirements to increase significantly in connection with our ongoing activities, particularly as we advance our lead product candidates and other development programs. Accordingly, we will continue to require substantial additional funding to support our continuing operations.

Previous wording

our ability to maintain our relationships with Weill Cornell Medicine, Adverum, UCSD, and any other key licensors or collaborators;

Current wording

our ability to maintain our relationships with Cornell University, Adverum, The Regents of UCSD, and any other key licensors or collaborators;

Previous wording

We may be unable to raise additional funds or enter into potential collaborations, strategic partnerships or marketing, distribution, licensing or other similar agreements or arrangements on favorable terms, or at all. To the extent that we raise additional capital through the sale of equity or convertible debt securities, your ownership interest will be diluted. If we fail to raise capital or enter into such agreements or arrangements as, and when, needed, we may have to significantly delay, scale back or discontinue the development or commercialization of our product candidates or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.

Current wording

We may be unable to raise additional funds or enter into potential collaborations, strategic partnerships or marketing, distribution, licensing or other similar agreements or arrangements on favorable terms, or at all. To the extent that we raise additional capital through the sale of equity or convertible debt securities, your stockholder ownership interest will be diluted. If we fail to raise capital or enter into such agreements or arrangements as, and when, needed, we may have to significantly delay, scale back or discontinue the development or commercialization of our product candidates or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.

Previous wording

Other than as disclosed in Note 2 to the condensed financial statements included in this Quarterly Report on Form 10-Q, there have been no significant changes to our critical accounting estimates from those described in our audited consolidated financial statements as of and for the year ended December 31, 2023 included in our Annual Report on Form 10-K, filed with the SEC on March 11, 2024.

Current wording

Other than as disclosed in Note 2 to the condensed financial statements included in this Quarterly Report on Form 10-Q, there have been no significant changes to our critical accounting estimates from those described in our audited financial statements as of and for the year ended December 31, 2024 included in our Annual Report on Form 10-K, filed with the SEC on March 24, 2025.
Rearranged previous paragraphs (2)

Previous report — wording carried elsewhere

The following table summarizes our results of operations for the nine months ended September 30, 2024 and 2023 (in thousands):

Previous report — wording carried elsewhere

The following table summarizes our research and development expenses incurred for the nine months ended September 30, 2024 and 2023 (in thousands):