Lexeo Therapeutics Conference Call Summary Company Overview - **Company**: Lexeo Therapeutics (NasdaqGM:LXEO) - **Focus**: Development of LX2006 for the treatment of Friedreich ataxia cardiomyopathy Key Industry Insights - **Regulatory Update**: Discussions with the FDA regarding a potential accelerated approval pathway for LX2006 are ongoing, with the FDA open to a BLA submission that pools clinical data from ongoing Phase 1-2 studies and an upcoming pivotal study [2][4] - **Clinical Data**: New interim clinical data from two ongoing Phase 1-2 studies show promising results in safety and efficacy for LX2006 [2][4] Core Points and Arguments - **FDA Feedback**: The FDA has agreed to evaluate the co-primary endpoint of left ventricular mass index (LVMI) at an earlier time point than 12 months, potentially shortening the pivotal study duration [4][11] - **Clinical Improvements**: - A 23% mean improvement in LVMI observed at 12 months, with an 18% mean improvement at six months, exceeding the FDA's threshold of 10% for the pivotal study [5][21] - Clinically meaningful improvements in the modified Friedreich ataxia rating scale (MFARS) were also reported [5][27] - **Safety Profile**: LX2006 has been generally well tolerated across all dose cohorts, with no significant adverse events reported [5][18] Additional Important Information - **Patient Population**: Friedreich ataxia affects approximately 5,000 people in the U.S. and 15,000 globally, with cardiac complications being the leading cause of death [6][7] - **Mechanism of Action**: LX2006 aims to treat the root cause of the disease by restoring frataxin, a protein that is deficient in individuals with Friedreich ataxia [8][9] - **Manufacturing Changes**: Lexeo has transitioned to an optimized SF9 baculovirus manufacturing platform, which is expected to produce high-yield, high-quality vector with a low empty capsid ratio [10][11] - **Future Plans**: The pivotal study is expected to be initiated in the first half of 2026, pending finalization of the protocol with the FDA [13][44] Summary of Clinical Data - **Participant Data**: - 17 participants treated to date, with 6 having abnormal LVMI at baseline [14][15] - Improvements in cardiac biomarkers observed, with reductions in LVMI and troponin levels [20][21][23] - **Functional Improvement**: Evidence of functional improvement in MFARS scores, indicating better daily living activities for participants [27][29] Conclusion - Lexeo Therapeutics is making significant progress in the development of LX2006 for Friedreich ataxia cardiomyopathy, with promising clinical data and a favorable safety profile. The company is actively engaging with the FDA to expedite the approval process, aiming to address the urgent unmet need for effective treatments in this patient population [35][77]

Lexeo Therapeutics Announces Progress in FDA Discussions for Accelerated Approval Pathway and Positive Interim Clinical Data for LX2006 in FA Cardiomyopathy October 7, 2025 1 Forward Looking Statements This presentation contains "forward-looking statements" within the meaning of the federal securities laws, including, but not limited to, statements regarding Lexeo's expectations and plans regarding its current product candidates and programs, including statements regarding the structure of and timelines for ...

Core Insights - The U.S. FDA is open to pooling data from ongoing Phase I/II studies of LX2006 with pivotal data to support a Biologics License Application (BLA) for Accelerated Approval [1][4] - Interim clinical data show significant improvements in cardiac and neurologic measures for participants with Friedreich ataxia [1][6] - The company plans to initiate a pivotal study for LX2006 in the first half of 2026, pending finalization of the trial protocol [5] FDA Engagement - The FDA has indicated a willingness to accept a BLA submission that includes pooled clinical data from ongoing Phase I/II studies and new data from the planned pivotal study [4] - Lexeo Therapeutics is required to submit enhanced manufacturing comparability data and meet additional nonclinical requirements before starting the pivotal study [4] Clinical Data Highlights - Participants with abnormal left ventricular mass index (LVMI) achieved an 18% mean reduction at 6 months and a 23% mean reduction at 12 months, surpassing the FDA's target threshold of 10% [1][6] - The modified Friedreich Ataxia Rating Scale (mFARS) showed clinically meaningful improvements, indicating slowed disease progression [1][6] - Among participants with abnormal baseline LVMI, all achieved LVMI measurements within the normal range, and five out of six participants showed over 10% improvement by 12 months [7] Safety and Efficacy - LX2006 has demonstrated encouraging safety and efficacy, with no Grade 3+ serious adverse events reported to date [13] - The treatment has been well tolerated, with minimal transient liver function test elevations and no signs of frataxin over-expression in cardiac tissue [13] Company Overview - Lexeo Therapeutics is focused on developing gene therapies for cardiovascular diseases, with LX2006 targeting Friedreich ataxia cardiomyopathy, which affects approximately 5,000 individuals in the U.S. [11][12] - The company has received multiple designations from the FDA, including Breakthrough Therapy and Fast Track, to expedite the development of LX2006 [5][11]

Lexeo Therapeutics Conference Call Summary Company Overview - **Company**: Lexeo Therapeutics (NasdaqGM:LXEO) - **Industry**: Clinical stage genetic medicines - **Focus**: Treatment of rare diseases with high unmet medical needs, specifically Friedreich's ataxia and arrhythmogenic cardiomyopathy [2][3] Key Programs 1. **Friedreich's Ataxia (FA)** - **Therapy**: Gene therapy using the ABRH10 vector to deliver the frataxin gene to the heart and skeletal muscle - **Current Status**: Rapidly moving into a pivotal study in 2026 - **Clinical Data**: Achieved a 23% reduction in left ventricular mass index (LVMI) in patients with elevated LVMI, exceeding the FDA's required 10% reduction [9][10] - **FDA Engagement**: Alignment on co-primary endpoints of LVMI reduction and frataxin expression, with 100% of patients showing frataxin expression post-treatment [10][11] 2. **Arrhythmogenic Cardiomyopathy (PKP2)** - **Focus**: Targeting the PKP2 mutation, the most common genetic cause of arrhythmogenic cardiomyopathy - **Current Status**: Eight patients dosed, with a phase one study readout expected by the end of 2025 [3][30] - **Patient Experience**: Patients experience significant anxiety and fear due to symptoms like skipped heartbeats and potential shocks from defibrillators [30][31] Clinical Data and Endpoints - **Friedreich's Ataxia**: - Significant changes in LV mass observed, with a focus on achieving statistical power in the pivotal trial [8][9] - Safety profile is strong, with no significant elevations in liver enzymes or adverse events reported [24][25] - **Arrhythmogenic Cardiomyopathy**: - Focus on multiple clinical endpoints including premature ventricular contractions (PVCs) and right ventricular function [34][36] - Aim to demonstrate improvement across multiple domains to show therapeutic benefit [39] Safety and Regulatory Considerations - **Safety Profile**: Lexeo emphasizes a compelling safety profile due to lower dosing compared to other gene therapies, with no drug-related serious adverse events reported [25][41] - **Regulatory Engagement**: Ongoing discussions with the FDA to finalize the size of the pivotal study, expected to be a 2026 event [11][45] Market Potential and Commercial Strategy - **Target Market**: Initial focus on high LVMI patients, with potential expansion to earlier-stage patients as treatment evolves [26][27] - **Cash Runway**: Recently completed a capital raise, providing a runway into 2028, well-positioned for upcoming milestones [47] Conclusion - Lexeo Therapeutics is advancing its gene therapy programs with a strong focus on safety and efficacy, aiming to address significant unmet needs in rare cardiovascular diseases. The company is well-capitalized and strategically positioned for future clinical trials and market entry.

Lexeo Therapeutics Conference Call Summary Company Overview - **Company**: Lexeo Therapeutics (NasdaqGM:LXEO) - **Industry**: Gene Therapy, specifically focusing on cardiac genetic medicines - **Foundation**: Originated from Weill Cornell University, led by Ron Kristol, a pioneer in gene therapy [2][3] Core Programs and Focus Areas - **Friedreich's Ataxia Program**: - Most advanced program targeting the cardiac component of Friedreich's Ataxia, with promising data suggesting potential benefits for the neurological aspects as well [2][3][9] - Data readout expected in the first half of 2025, with a pivotal study planned for 2026 [3][21] - **Arrhythmogenic Cardiomyopathy (ACM)**: - Second advanced program targeting a rare disease affecting approximately 60,000 patients in the U.S. [3][28] - The program is in early clinical stages, with data expected by the end of the year [32][33] Key Data and Clinical Results - **Friedreich's Ataxia**: - Achieved a 24% reduction in left ventricular (LV) mass, exceeding the FDA's primary endpoint threshold of 10% [11][12] - 100% of patients showed increased frataxin expression post-therapy [12] - Notable reductions in troponin levels, with some patients experiencing up to a 90% reduction [13] - Improvement observed in the mFARS scale, exceeding effects seen in pivotal studies of existing therapies [16][17] - **Safety Profile**: - Utilization of AAVRH10 vector, which has superior cardiac tropism and a favorable safety profile, avoiding common adverse events associated with gene therapy [4][5][20] - No treatment-related serious adverse events (SAEs) reported at the current dosing levels [19][20][45] Market Opportunity - **Friedreich's Ataxia**: - Approximately 5,000 patients in the U.S., with 40% having abnormal LV mass, indicating a significant market for therapy [26][27] - 70% of Friedreich's Ataxia patients will experience mortality from cardiomyopathy, highlighting the urgent need for effective treatments [26] - **Arrhythmogenic Cardiomyopathy**: - Represents a high unmet need with significant mortality and morbidity, making it a leading target for gene therapy [28][29] Financial Overview - **Cash Position**: - Closed Q2 with $153 million in cash and marketable securities, providing runway into 2028 [47] - Recent elevated G&A expenses due to resolved litigation, expected to normalize post-Q3 [47] Regulatory and Developmental Path - **FDA Engagement**: - Ongoing discussions with the FDA regarding pivotal study design and breakthrough therapy designation received in July [23][21] - Focus on aligning statistical analysis plans and operational steps for the upcoming pivotal study [21][46] Conclusion - Lexeo Therapeutics is positioned as a promising player in the gene therapy space, particularly in cardiac diseases, with a strong focus on safety and efficacy through innovative approaches. The company is on track for significant clinical milestones and has a robust financial foundation to support its development efforts [49]

Summary of Lexeo Therapeutics Conference Call Company Overview - **Company**: Lexeo Therapeutics - **Industry**: Gene Therapy, specifically focused on cardiovascular diseases - **Key Programs**: - Friedreich's ataxia (FA) targeting cardiac pathology - PKP2-mediated arrhythmogenic cardiomyopathy Core Points and Arguments - **Friedreich's Ataxia Program**: - 70% of FA patients die from cardiac disease, making it a critical focus for treatment [2][18] - The program is advancing into a pivotal study next year, with data readout expected by the end of the year [3][2] - Achieved a 100% protein expression rate across patients treated at a low dose of 1E12 vector genomes/kg, significantly lower than doses used in other therapies [6][9] - Observed a 25% average reduction in left ventricular mass index, exceeding the FDA's requirement of a 10% reduction for approval [30][33] - Notable improvements in cardiac biomarkers, including a 60% reduction in troponin levels [31][27] - **PKP2-mediated Arrhythmogenic Cardiomyopathy**: - Affects approximately 60,000 patients in the U.S., making it a significant target for gene therapy [2] - Currently in a phase 1/2 study with eight patients dosed, aiming for data readout towards the end of the year [2][64] - Primary endpoint includes reduction in premature ventricular contractions (PVCs), a quantifiable measure of the disease [64] - **Safety Profile**: - Utilization of AAVRH10 vector has shown a compelling safety profile with no treatment-related serious adverse events (SAEs) reported in the FA program [9][15] - The company has maintained a low empty to full capsid ratio, enhancing safety [13][5] - The approach to gene therapy emphasizes selecting the right vector and dosing to minimize safety risks [5][10] - **Regulatory Progress**: - Received breakthrough designation from the FDA, indicating alignment and interest in accelerating the therapy's development [45][44] - The pivotal trial will focus on both cardiac surrogate endpoints and functional endpoints like the MFARS scale for full approval [53][48] Additional Important Information - **Clinical Benefits**: - The therapy is showing benefits beyond cardiac symptoms, with improvements in neurologic scales associated with FA [19][22] - The mechanism of action suggests potential for skeletal muscle transduction, contributing to overall patient improvement [22][19] - **Future Milestones**: - Expecting to start the pivotal study for FA in early 2026, with data anticipated in mid-2027 [59][61] - Ongoing updates will include safety data and efficacy results from both the FA and PKP2 programs [71][70] - **Financial Outlook**: - The company has a cash runway into 2028, supporting ongoing clinical trials and operational needs [70][69] This summary encapsulates the key points discussed during the conference call, highlighting Lexeo Therapeutics' strategic focus on gene therapy for cardiovascular diseases, its promising clinical data, and regulatory progress.

[Special Note Regarding Forward Looking Statements](index=3&type=section&id=Special%20Note%20Regarding%20Forward%20Looking%20Statements) This section highlights that the report contains forward-looking statements involving risks and uncertainties regarding future financial and operational results - The report contains forward-looking statements that involve risks and uncertainties, covering future results of operations, financial position, business strategy, clinical trials, R&D costs, and regulatory strategy[9](index=9&type=chunk) - These forward-looking statements are based on current expectations and projections and are not guarantees of future performance; actual results could differ materially due to risks and uncertainties[10](index=10&type=chunk) [PART I - FINANCIAL INFORMATION](index=5&type=section&id=PART%20I%20-%20FINANCIAL%20INFORMATION) This section details Lexeo Therapeutics' unaudited condensed financial statements and management's analysis of financial condition and operations - The unaudited condensed financial statements are prepared in conformity with generally accepted accounting principles (GAAP) for interim financial information and should be read in conjunction with the Company's audited financial statements for the year ended December 31, 2024[23](index=23&type=chunk) - Operating results for the three and six months ended June 30, 2025, are not necessarily indicative of the results that may be expected for the full year ending December 31, 2025, or any other future period[23](index=23&type=chunk) [Item 1. Financial Statements.](index=5&type=section&id=Item%201.%20Financial%20Statements.) This section presents Lexeo Therapeutics' unaudited condensed financial statements, prepared in conformity with GAAP for interim reporting [Condensed Balance Sheets (unaudited)](index=6&type=section&id=Condensed%20Balance%20Sheets%20(unaudited)) This section provides Lexeo Therapeutics' unaudited condensed balance sheets as of June 30, 2025, and December 31, 2024 Condensed Balance Sheets (in thousands) | Metric | June 30, 2025 (in thousands) | December 31, 2024 (in thousands) | | :----------------------------------- | :----------------------------- | :------------------------------- | | Total assets | $176,068 | $146,942 | | Total liabilities | $37,850 | $30,100 | | Total stockholders' equity | $138,218 | $116,842 | | Cash and cash equivalents | $35,480 | $35,014 | | Current portion of investments in U.S. Treasury securities | $97,413 | $86,504 | | Non-current portion of investments in U.S. Treasury securities | $19,613 | $7,012 | | Accounts payable | $20,025 | $6,417 | | Accrued expenses and other current liabilities | $8,912 | $13,759 | [Condensed Statements of Operations and Comprehensive Loss (unaudited)](index=7&type=section&id=Condensed%20Statements%20of%20Operations%20and%20Comprehensive%20Loss%20(unaudited)) This section presents Lexeo Therapeutics' unaudited condensed statements of operations and comprehensive loss for specified periods Condensed Statements of Operations and Comprehensive Loss (in thousands) | Metric | Three Months Ended June 30, 2025 (in thousands) | Three Months Ended June 30, 2024 (in thousands) | Six Months Ended June 30, 2025 (in thousands) | Six Months Ended June 30, 2024 (in thousands) | | :----------------------------------- | :-------------------------------------------- | :-------------------------------------------- | :------------------------------------------ | :------------------------------------------ | | Research and development | $14,721 | $16,560 | $31,892 | $32,302 | | General and administrative | $15,967 | $6,990 | $32,601 | $14,539 | | Total operating expenses | $30,688 | $23,550 | $64,493 | $46,841 | | Operating loss | $(30,688) | $(23,550) | $(64,493) | $(46,841) | | Gain on long-term investment | $3,390 | $- | $3,390 | $- | | Interest income | $1,268 | $2,348 | $2,461 | $3,999 | | Net loss | $(26,103) | $(21,238) | $(58,759) | $(42,920) | | Net loss per common share, basic and diluted | $(0.60) | $(0.64) | $(1.53) | $(1.41) | [Condensed Statements of Stockholders' Equity (unaudited)](index=8&type=section&id=Condensed%20Statements%20of%20Stockholders'%20Equity%20(unaudited)) This section details Lexeo Therapeutics' unaudited condensed statements of stockholders' equity for the periods presented Condensed Statements of Stockholders' Equity (in thousands) | Metric | December 31, 2024 (in thousands) | June 30, 2025 (in thousands) | | :----------------------------------- | :------------------------------- | :----------------------------- | | Total Stockholders' Equity | $116,842 | $138,218 | | Additional Paid-in Capital | $397,132 | $477,175 | | Accumulated Deficit | $(280,173) | $(338,932) | - Issuance of common stock, pre-funded warrants, and warrants from the May 2025 Private Placement contributed **$73.08 million** to additional paid-in capital[18](index=18&type=chunk) - Stock-based compensation expense for the six months ended June 30, 2025, was **$6.77 million**[18](index=18&type=chunk) [Condensed Statements of Cash Flows (unaudited)](index=9&type=section&id=Condensed%20Statements%20of%20Cash%20Flows%20(unaudited)) This section presents Lexeo Therapeutics' unaudited condensed statements of cash flows for the six months ended June 30, 2025 and 2024 Condensed Statements of Cash Flows (in thousands) | Cash Flow Activity | Six Months Ended June 30, 2025 (in thousands) | Six Months Ended June 30, 2024 (in thousands) | | :----------------------------------- | :-------------------------------------------- | :-------------------------------------------- | | Net cash used in operating activities | $(48,935) | $(35,019) | | Net cash used in investing activities | $(23,574) | $(411) | | Net cash provided by financing activities | $72,971 | $88,945 | | Net change in cash, cash equivalents and restricted cash | $462 | $53,515 | - Net cash used in operating activities increased by **$13.9 million** for the six months ended June 30, 2025, primarily due to a higher net loss and an unrealized gain on long-term investment[19](index=19&type=chunk)[131](index=131&type=chunk) - Net cash used in investing activities significantly increased to **$23.6 million** in 2025, primarily due to **$55.4 million** in purchases of U.S. Treasury securities, partially offset by **$31.9 million** from maturities[19](index=19&type=chunk)[133](index=133&type=chunk) - Net cash provided by financing activities in 2025 was primarily driven by **$73.1 million** in net proceeds from the May 2025 Private Placement offering[19](index=19&type=chunk)[134](index=134&type=chunk) [Notes to Condensed Financial Statements (unaudited)](index=10&type=section&id=Notes%20to%20Condensed%20Financial%20Statements%20(unaudited)) This section provides detailed notes accompanying Lexeo Therapeutics' unaudited condensed financial statements [1. Description of Business and Basis of Presentation](index=10&type=section&id=1.%20Description%20of%20Business%20and%20Basis%20of%20Presentation) Lexeo Therapeutics is a clinical-stage genetic medicine company focused on AAV-engineered gene therapies, with financial statements prepared under GAAP - Lexeo Therapeutics is a clinical-stage genetic medicine company focused on hereditary and acquired diseases, utilizing adeno-associated viruses (AAV) to transfer genes[20](index=20&type=chunk) - The company has incurred net losses and negative cash flows from operations since inception, with an accumulated deficit of **$338.9 million** as of June 30, 2025[25](index=25&type=chunk) - Current cash, cash equivalents, and investments in U.S. Treasury securities (**$152.5 million** as of June 30, 2025) are estimated to fund operations for at least **12 months** from the financial statements' issuance date[25](index=25&type=chunk) [2. Summary of Significant Accounting Policies](index=12&type=section&id=2.%20Summary%20of%20Significant%20Accounting%20Policies) This section outlines the company's significant accounting policies, noting no material changes from its annual financial statements - There have been no significant changes in the Company's accounting policies from those disclosed in its Annual Financial Statements[28](index=28&type=chunk) - Management makes significant estimates and assumptions, particularly for accruals of research and development costs and the fair value of stock option awards[29](index=29&type=chunk) - The two-class method is used for net loss per common share, with anti-dilutive securities (e.g., stock options, RSUs, warrants) excluded from diluted net loss per share computation during periods of net loss[30](index=30&type=chunk)[32](index=32&type=chunk) [3. Fair Value Measurements](index=13&type=section&id=3.%20Fair%20Value%20Measurements) The company measures

[Executive Summary & Highlights](index=1&type=section&id=Executive%20Summary%20%26%20Highlights) [Key Operational and Financial Highlights](index=1&type=section&id=Key%20Operational%20and%20Financial%20Highlights) Lexeo Therapeutics reported significant operational progress in Q2 2025, including Breakthrough Therapy designation for LX2006, selection for the FDA's CDRP program, continued dosing in the LX2020 trial, and a new strategic partnership for non-viral RNA therapeutics. Financially, the company secured an $80 million equity financing, extending its operational runway into 2028, and appointed Louis Tamayo as Chief Financial Officer - **Breakthrough Therapy designation** granted for LX2006 based on interim Phase I/II data demonstrating clinically meaningful improvements in cardiac and neurologic measures of Friedreich ataxia[1](index=1&type=chunk)[3](index=3&type=chunk) - LX2006 selected for FDA Chemistry, Manufacturing, and Controls Development and Readiness Pilot (**CDRP program**) to facilitate CMC registrational readiness and support faster patient access[1](index=1&type=chunk)[4](index=4&type=chunk) - **Eight participants** dosed in Phase I/II clinical trial (HEROIC-PKP2) of LX2020 for PKP2-ACM; interim clinical data update on track for second half of 2025[1](index=1&type=chunk)[4](index=4&type=chunk) - **Strategic partnership** announced with Perceptive Xontogeny Venture Funds and venBio Partners to advance non-viral, RNA-based therapeutics for genetic cardiac diseases[1](index=1&type=chunk)[6](index=6&type=chunk) - **$80 million equity financing** closed to support development of clinical stage pipeline[1](index=1&type=chunk)[6](index=6&type=chunk) - Cash, cash equivalents and investments in marketable securities of **$152.5 million** expected to provide operational runway into **2028**[1](index=1&type=chunk)[6](index=6&type=chunk) - **Louis Tamayo** appointed **Chief Financial Officer**[1](index=1&type=chunk)[6](index=6&type=chunk) [Business and Program Updates](index=1&type=section&id=Business%20and%20Program%20Updates) [LX2006 in Friedreich Ataxia (FA)](index=1&type=section&id=LX2006%20in%20Friedreich%20Ataxia%20(FA)) LX2006 received FDA Breakthrough Therapy designation and was selected for the CDRP program, accelerating its path to a registrational study expected to begin in early 2026 with potential efficacy data in 2027. The CLARITY-FA natural history study is enrolling to serve as an external control, and LX2006 continues to demonstrate a generally well-tolerated safety profile - In July 2025, LX2006 received **FDA Breakthrough Therapy designation** based on interim clinical data demonstrating clinically meaningful improvements in cardiac and neurologic measures of FA[4](index=4&type=chunk) - LX2006 selected for FDA Chemistry, Manufacturing, and Controls (CMC) Development Readiness Pilot (**CDRP program**) to accelerate CMC registrational readiness[4](index=4&type=chunk) - Lexeo expects final alignment with FDA on the LX2006 registrational study in **late Q3 to early Q4 2025**, with initiation planned for **early 2026** and potential efficacy readout in **2027**[4](index=4&type=chunk) - The **CLARITY-FA natural history study** is currently enrolling and is expected to serve as a concurrent external control arm for the planned registrational study[4](index=4&type=chunk) - LX2006 continues to be **generally well tolerated** with no clinically significant complement activation and no new treatment-related serious adverse events[4](index=4&type=chunk) [LX2020 in PKP2-ACM](index=1&type=section&id=LX2020%20in%20PKP2-ACM) Eight participants have been dosed in the HEROIC-PKP2 Phase I/II clinical trial for LX2020, across three cohorts, with the high-dose Cohort 3 still enrolling. The therapy has shown a generally well-tolerated safety profile, and interim clinical data is anticipated in the second half of 2025 - **Eight participants** have been dosed to date in the HEROIC-PKP2 Phase I/II clinical trial for LX2020, including **three in Cohort 1** (low dose), **three in Cohort 2** (high dose), and **two in dose-expansion Cohort 3** (high dose)[4](index=4&type=chunk) - **Cohort 3** is still enrolling, and up to **two additional participants** may be dosed in this cohort[4](index=4&type=chunk) - LX2020 has been **generally well tolerated** with no clinically significant complement activation and no treatment-related serious adverse events to date across all dose cohorts[4](index=4&type=chunk) - Lexeo expects to share an interim clinical data update in the **second half of 2025**[4](index=4&type=chunk) [Strategic Developments & Corporate Updates](index=2&type=section&id=Strategic%20Developments%20%26%20Corporate%20Updates) [Equity Financing](index=2&type=section&id=Equity%20Financing) Lexeo closed an $80 million equity financing in May 2025, which is expected to fund operations into 2028, covering a potential efficacy readout for the LX2006 registrational study in 2027 - Closed an **$80 million equity financing** in **May 2025** to further advance development of its clinical stage genetic medicine candidates[6](index=6&type=chunk) - Lexeo anticipates that current cash, cash equivalents and marketable securities will be sufficient to fund operating and capital expenditures into **2028**, through a potential efficacy readout for the registrational study of LX2006 in **2027**[6](index=6&type=chunk) [Research Collaboration](index=2&type=section&id=Research%20Collaboration) In June 2025, Lexeo announced a strategic partnership with Perceptive Xontogeny Venture Funds and venBio Partners to develop non-viral RNA-based therapies for genetic cardiac diseases. The partners will contribute up to $40 million in financing to a new entity, with Lexeo contributing expertise and intellectual property for an equity position, milestone payments, royalties, and opt-in rights - **Strategic partnership** announced in **June 2025** with Perceptive Xontogeny Venture Funds and venBio Partners to develop therapies for genetic cardiac diseases utilizing a novel non-viral RNA platform[6](index=6&type=chunk) - PXV Funds and venBio will contribute up to **$40 million** in private equity financing to a new entity[6](index=6&type=chunk) - Lexeo is contributing expertise and know-how in cardiac genetic medicines, preclinical intellectual property and technology to the partnership, with a **double-digit percentage equity position** in the new entity at transaction close alongside entitlement to future milestone payments, royalties, and opt-in rights[6](index=6&type=chunk) [New Leadership Appointment](index=2&type=section&id=New%20Leadership%20Appointment) Louis Tamayo was appointed Chief Financial Officer, succeeding Kyle Rasbach. Mr. Tamayo brings extensive commercial finance experience from Siemens Healthineers AG, Becton, Dickinson and Company, and Pfizer, and will support Lexeo's late-stage clinical and commercialization plans - **Louis Tamayo** has been appointed **Chief Financial Officer**, succeeding Kyle Rasbach[6](index=6&type=chunk) - Mr. Tamayo will support Lexeo's **late-stage clinical and commercialization plans** as LX2006 development accelerates and LX2020 development continues, alongside strategic planning, portfolio management, capital allocation, and other financial operations[6](index=6&type=chunk) - Mr. Tamayo brings extensive commercial finance experience, having previously served as Senior Vice President at Siemens Healthineers AG, and held roles at Becton, Dickinson and Company and Pfizer[6](index=6&type=chunk) [Recent Data Presentations](index=2&type=section&id=Recent%20Data%20Presentations) Lexeo presented new data at ASGCT on AAV manufacturing optimization via its Sf9-baculovirus process, demonstrating high purity, potency, improved scalability, and reduced cost. The company also presented data at the Global Cell and Gene Therapy Summit reviewing the favorable complement profile of AAVrh10, with no clinically significant complement activation observed in three gene therapy studies - Lexeo presented new data at the 28th American Society of Gene & Cell Therapy (ASGCT) Annual Meeting on AAV manufacturing optimization via the Company's Sf9-baculovirus process, showing **high purity and potency** of yields with **improved scalability** and **reduced cost**[6](index=6&type=chunk) - Lexeo also presented data at the Global Cell and Gene Therapy Summit reviewing the **favorable complement profile** of AAVrh10 based on clinical monitoring experience across three gene therapy studies in FA and PKP2, with **no clinically significant events** related to complement activation observed[6](index=6&type=chunk) [Second Quarter 2025 Financial Results](index=2&type=section&id=Second%20Quarter%202025%20Financial%20Results) [Financial Summary](index=2&type=section&id=Financial%20Summary) For the three months ended June 30, 2025, Lexeo reported a net loss of $26.1 million, or $0.60 per share, compared to a net loss of $21.2 million, or $0.64 per share, in the prior year period. Research and development expenses decreased slightly, while general and administrative expenses significantly increased. The company's cash position as of June 30, 2025, was $152.5 million, projected to fund operations into 2028 Key Financial Metrics (Three Months Ended June 30) | Financial Metric (in thousands) | 2025 | 2024 | Change (YoY) | | :-------------------------------------------- | :--- | :--- | :----------- | | Research and Development Expenses | $14,721 | $16,560 | -$1,839 | | General and Administrative Expenses | $15,967 | $6,990 | +$8,977 | | Total Operating Expenses | $30,688 | $23,550 | +$7,138 | | Operating Loss | $(30,688) | $(23,550) | -$(7,138) | | Net Loss | $(26,103) | $(21,238) | -$(4,865) | | Net Loss per Common Share (basic and diluted) | $(0.60) | $(0.64) | +$0.04 | - Net loss increased to **$26.1 million** in Q2 2025 from **$21.2 million** in Q2 2024[6](index=6&type=chunk)[9](index=9&type=chunk) - General and Administrative expenses significantly increased to **$16.0 million** in Q2 2025 from **$7.0 million** in Q2 2024[6](index=6&type=chunk)[9](index=9&type=chunk) - Cash, cash equivalents, and investments in marketable securities were **$152.5 million** as of June 30, 2025, which Lexeo believes will be sufficient to fund operations into **2028**[6](index=6&type=chunk)[10](index=10&type=chunk) [Condensed Statements of Operations](index=4&type=section&id=Condensed%20Statements%20of%20Operations) The condensed statements of operations show a net loss of $26.1 million for Q2 2025 and $58.8 million for the six months ended June 30, 2025. Total operating expenses increased significantly year-over-year for both the quarter and year-to-date periods, primarily driven by general and administrative costs Condensed Statements of Operations (Unaudited, in thousands) | Metric | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :-------------------------------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Research and development | $14,721 | $16,560 | $31,892 | $32,302 | | General and administrative | $15,967 | $6,990 | $32,601 | $14,539 | | Total operating expenses | $30,688 | $23,550 | $64,493 | $46,841 | | Operating loss | $(30,688) | $(23,550) | $(64,493) | $(46,841) | | Net loss | $(26,103) | $(21,238) | $(58,759) | $(42,920) | | Net loss per common share, basic and diluted | $(0.60) | $(0.64) | $(1.53) | $(1.41) | | Weighted average number of shares outstanding | 43,573,628 | 33,001,946 | 38,372,704 | 30,490,892 | - Total operating expenses for the three months ended June 30, 2025, were **$30.7 million**, an increase from **$23.6 million** in the prior year period[9](index=9&type=chunk) - Total operating expenses for the six months ended June 30, 2025, were **$64.5 million**, up from **$46.8 million** in the prior year period[9](index=9&type=chunk) [Condensed Balance Sheet Data](index=4&type=section&id=Condensed%20Balance%20Sheet%20Data) As of June 30, 2025, Lexeo's cash, cash equivalents, and investments in U.S. Treasury securities increased to $152.5 million from $128.5 million at December 31, 2024. Total assets also increased, while total liabilities saw a moderate rise Condensed Balance Sheet Data (Unaudited, in thousands) | Metric | June 30, 2025 | December 31, 2024 | Change | | :------------------------------------------------ | :------------ | :---------------- | :----- | | Cash, cash equivalents, and investments in U.S. Treasury securities | $152,506 | $128,530 | +$23,976 | | Total assets | $176,068 | $146,942 | +$29,126 | | Total liabilities | $37,850 | $30,100 | +$7,750 | | Total stockholders' equity | $138,218 | $116,842 | +$21,376 | - Cash, cash equivalents, and investments in U.S. Treasury securities increased by **$23.98 million** from December 31, 2024, to June 30, 2025[10](index=10&type=chunk) [About Lexeo Therapeutics](index=2&type=section&id=About%20Lexeo%20Therapeutics) [Company Overview](index=2&type=section&id=Company%20Overview) Lexeo Therapeutics is a New York City-based clinical stage genetic medicine company focused on developing novel treatments for cardiovascular diseases by targeting underlying genetic causes. Its pipeline includes LX2006 for Friedreich ataxia cardiomyopathy and LX2020 for PKP2 arrhythmogenic cardiomyopathy - Lexeo Therapeutics is a New York City-based, **clinical stage genetic medicine company** dedicated to reshaping heart health by applying pioneering science to fundamentally change how cardiovascular diseases are treated[5](index=5&type=chunk) - The Company is advancing a portfolio of therapeutic candidates that take aim at the underlying genetic causes of conditions, including **LX2006** in **Friedreich ataxia cardiomyopathy (FA)** and **LX2020** in **plakophilin-2 (PKP2) arrhythmogenic cardiomyopathy**[5](index=5&type=chunk) [Legal & Contact Information](index=3&type=section&id=Legal%20%26%20Contact%20Information) [Cautionary Note Regarding Forward-Looking Statements](index=3&type=section&id=Cautionary%20Note%20Regarding%20Forward-Looking%20Statements) The report contains forward-looking statements subject to various risks and uncertainties, including those related to clinical trials, regulatory approvals, and financial resources. Lexeo advises against undue reliance on these statements and disclaims any obligation to update them, except as required by law, under the protection of the Private Securities Litigation Reform Act of 1995 - Certain statements in this press release may constitute **'forward-looking statements'** regarding Lexeo's product candidates, clinical trials, regulatory developments, and financial condition[7](index=7&type=chunk) - These forward-looking statements are subject to various risks and uncertainties, including those set forth in Lexeo's SEC filings, and actual results could be materially different[7](index=7&type=chunk) - Lexeo claims the protection of the Safe Harbor contained in the **Private Securities Litigation Reform Act of 1995** for forward-looking statements and expressly disclaims any obligation to update or alter any statements, except as required by law[7](index=7&type=chunk) [Media and Investor Contacts](index=3&type=section&id=Media%20and%20Investor%20Contacts) Contact information is provided for media inquiries (Media@lexeotx.com) and investor relations (Carlo Tanzi, Ph.D., ctanzi@kendallir.com) - Media inquiries can be directed to **Media@lexeotx.com**[8](index=8&type=chunk) - Investor inquiries can be directed to **Carlo Tanzi, Ph.D.**, at **ctanzi@kendallir.com**[8](index=8&type=chunk)

Core Insights - Lexeo Therapeutics has received FDA Breakthrough Therapy designation for LX2006, indicating significant potential in treating Friedreich ataxia based on interim clinical data [6][3] - The company has secured an $80 million equity financing to support the development of its clinical stage pipeline, with a cash position of $152.5 million expected to fund operations into 2028 [7][10] - A strategic partnership has been established with Perceptive Xontogeny Venture Funds and venBio Partners to advance non-viral, RNA-based therapeutics for genetic cardiac diseases [10][3] Business and Program Updates - LX2006 is being developed for Friedreich ataxia, with plans to initiate a registrational study in early 2026 and potential efficacy readout in 2027 [6][4] - The LX2020 program for PKP2-ACM has dosed eight participants, with interim clinical data updates expected in the second half of 2025 [6][5] Financial Performance - For Q2 2025, Lexeo reported a net loss of $26.1 million, or $0.60 per share, compared to a net loss of $21.2 million, or $0.64 per share, in Q2 2024 [12][13] - Research and Development expenses were $14.7 million for Q2 2025, down from $16.6 million in Q2 2024, while General and Administrative expenses increased to $16.0 million from $7.0 million [12][13] Leadership and Strategic Developments - Louis Tamayo has been appointed as Chief Financial Officer, bringing extensive experience in commercial finance to support Lexeo's clinical and commercialization plans [10][3] - The company presented new data at the 28th American Society of Gene & Cell Therapy Annual Meeting, highlighting advancements in AAV manufacturing optimization [10]

Core Insights - The FDA has granted Breakthrough Therapy designation to LX2006, indicating its potential to significantly improve treatment for Friedreich ataxia (FA) based on interim clinical data [1][2] - LX2006 has also been selected for the FDA Chemistry, Manufacturing, and Controls Development and Readiness Pilot program, aimed at facilitating faster patient access to therapies [1][3] Company Overview - Lexeo Therapeutics is a clinical stage genetic medicine company focused on innovative treatments for cardiovascular diseases, including LX2006 for FA and LX2020 for plakophilin-2 arrhythmogenic cardiomyopathy [4] Clinical Data and Trials - Interim clinical data shows that treatment with LX2006 resulted in clinically significant improvements in cardiac biomarkers and functional measures, with increased frataxin expression observed in all participants with cardiac biopsies at three months post-treatment [2] - A total of 17 participants have been treated across two trials: the SUNRISE-FA Phase 1/2 clinical trial and the Weill Cornell Medicine investigator-initiated Phase 1A trial [2] - The company is currently enrolling participants for a prospective natural history study, CLARITY-FA, which will serve as a control arm for the registrational study expected to begin by early 2026 [2]