Rimiplagene Parvec

Finalized SUNRISE-FA 2 open-label, pivotal trial protocol and SAP for LX2006 in support of accelerated approval pathway; topline data expected in second half of 2027 Initiated SUNRISE-FA 2 with first patient enrolled into pivotal study in June; site activation efforts ongoing CLARITY-FA enrollment continues to advance, supporting identification of eligible participants for SUNRISE-FA 2 FDA granted RMAT designation for LX2020 based on recent interim clinical data in PKP2-ACM ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); Cash, cash equivalents and investments of $234.2 million expected to provide operational runway into 2028 NEW YORK, Aug. 12, 2026 (GLOBE NEWSWIRE) -- Lexeo Therapeutics, Inc. (Nasdaq: LXEO), a clinical stage genetic medicine company dedicated to pioneering novel treatments for cardiovascular diseases, today provided business updates across its portfolio and reported financial results for the second quarter 2026. “We closed the second quarter with significant momentum, highlighted by the initiation of SUNRISE-FA 2, the pivotal study for LX2006 in Friedreich ataxia cardiomyopathy,” said R. Nolan Townsend, Chief Executive Officer of Lexeo Therapeutics. “With SUNRISE-FA 2 now underway, we are focused on execution and advancing what we believe could be the first disease-modifying gene therapy for FA cardiomyopathy. Beyond LX2006, we continue to advance LX2020 in PKP2 arrhythmogenic cardiomyopathy and are pleased to have recently received RMAT designation from the FDA, an important milestone that reinforces the strength of the emerging clinical evidence and supports the potential expedited development of the program. With multiple value-driving catalysts ahead and a strong balance sheet to support our key clinical and regulatory milestones, we believe Lexeo is well positioned to deliver on our mission of transforming the treatment of genetically defined diseases.” Program Updates and Recent Progress LX2006 in Friedreich Ataxia (FA) In June 2026, Lexeo finalized the SUNRISE-FA 2 pivotal trial protocol and statistical analysis plan (SAP) for LX2006 under the accelerated approval pathway. SUNRISE-FA 2 : The study was initiated in June with activation of the first trial site and enrollment of the first patient. Site activation efforts are ongoing to support continued enrollment in the pivotal study. CLARITY-FA (Natural History Study) : Enrollment continues to advance across multiple sites in the US and globally. Patients enrolled in CLARITY-FA are eligible to participate in SUNRISE-FA 2, supporting continued enrollment into the pivotal study once trial sites are active. In June 2026, Lexeo announced publication of LX2006 data in the Journal of the American Medical Association (JAMA) Cardiology demonstrating LX2006 is generally well tolerated, with signs of efficacy in Phase I/II studies. In April 2026, Lexeo presented data at ASGCT highlighting LX2006’s continued positive clinical profile, along with preclinical and CMC data supporting potential sequential dosing strategies and the Company’s scalable manufacturing platform. Presentations and publications from ASGCT 2026 can be found here . Anticipated milestones for the program include: Continued FDA engagement on confirmatory evidence strategy; update expected once finalized Topline data readout expected in second half of 2027 Potential BLA submission under accelerated approval pathway in first half of 2028 LX2020 in PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM) In August 2026, Lexeo received Regenerative Medicine Advanced Therapy (RMAT) designation for LX2020 from the FDA based on recent interim data from its ongoing Phase I/II clinical trial in PKP2-ACM. RMAT designation provides opportunities for increased FDA interaction and may help expedite the development of LX2020. LX2020 remains generally well tolerated across ten participants dosed with no clinically significant complement activation to date. Anticipated milestones for the program include: 12-month data update for all high dose participants in Q4 2026 Regulatory engagement with the FDA expected in 2026 Pre-Clinical Assets In May 2026, Lexeo presented preclinical data for LX2022 at ASGCT demonstrating proof-of-concept efficacy for TNNI3 gene replacement in a newly developed porcine model of hypertrophic cardiomyopathy. Presentations and publications from ASGCT 2026 can be found here . Second Quarter 2026 Financial Results Cash Position: As of June 30, 2026, cash, cash equivalents, and investments in marketable securities were $234.2 million, which Lexeo believes will be sufficient to fund operations into 2028. Research & Development Expenses: Research and Development expenses were $19.0 million for the three months ended June 30, 2026, compared to $14.7 million for the three months ended June 30, 2025. General & Administrative Expenses: General and Administrative expenses were $9.0 million for the three months ended June 30, 2026, compared to $16.0 million for the three months ended June 30, 2025. Net Loss: Net loss was $25.9 million or $0.30 per share (basic and diluted) for the three months ended June 30, 2026, compared to $26.1 million or $0.60 per share (basic and diluted) for the three months ended June 30, 2025. About Lexeo Therapeutics 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. The Company is advancing a portfolio of therapeutic candidates that take aim at the underlying genetic causes of conditions, including LX2006 in Friedreich ataxia (FA), LX2020 in plakophilin-2 (PKP2) arrhythmogenic cardiomyopathy, and others in devastating diseases with high unmet need. Cautionary Note Regarding Forward-Looking Statements Certain statements in this press release may constitute “forward-looking statements” within the meaning of the federal securities laws, including, but not limited to, Lexeo’s expectations and plans regarding its current product candidates and programs, the anticipated benefits of its current product candidates, the timing for receipt and announcement of data from its clinical trials, the timing and likelihood of potential regulatory developments, trial design changes and approval, and expectations regarding the time period over which Lexeo’s capital resources will be sufficient to fund its anticipated operations and estimates regarding Lexeo’s financial condition. Words such as “may,” “might,” “will,” “objective,” “intend,” “should,” “could,” “can,” “would,” “expect,” “believe,” “design,” “estimate,” “predict,” “potential,” “develop,” “plan” or the negative of these terms, and similar expressions, or statements regarding intent, belief, or current expectations, are forward-looking statements. While Lexeo believes these forward-looking statements are reasonable, undue reliance should not be placed on any such forward-looking statements. These forward-looking statements are based upon current information available to the company as well as certain estimates and assumptions and are subject to various risks and uncertainties (including, without limitation, those set forth in Lexeo’s filings with the U.S. Securities and Exchange Commission (SEC)), many of which are beyond the company’s control and subject to change. Actual results could be materially different from those indicated by such forward-looking statements as a result of many factors, including but not limited to: risks and uncertainties related to global macroeconomic conditions and related volatility; the outcome of ongoing discussions with the U.S. Food and Drug Administration (FDA) regarding the design of our pivotal trial for accelerated approval pathway and the design of our confirmatory study for obtaining full approval; expectations regarding the initiation, progress, and expected results of Lexeo’s preclinical studies, clinical trials and research and development programs; the unpredictable relationship between preclinical study results and clinical study results; delays in submission of regulatory filings or failure to receive regulatory approval; liquidity and capital resources; and other risks and uncertainties identified in Lexeo’s Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2026, filed with the SEC on May 11, 2026, and subsequent future filings Lexeo may make with the SEC. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Lexeo claims the protection of the Safe Harbor contained in the Private Securities Litigation Reform Act of 1995 for forward-looking statements. Lexeo expressly disclaims any obligation to update or alter any statements whether as a result of new information, future events or otherwise, except as required by law. Media Response: Media@lexeotx.com Investor Response: Ashley Kaplowitz akaplowitz@lexeotx.com Lexeo Therapeutics, Inc. Selected Financial Information (Unaudited, in thousands, except share and per share amounts) Statements of Operations Three Months Ended June 30, Six Months Ended, 2026 2025 2026 2025 Operating expenses Research and development $ 19,027 $ 14,721 $ 34,730 $ 31,892 General and administrative 8,993 15,967 15,623 32,601 Total operating expenses 28,020 30,688 50,353 64,493 Operating loss (28,020 ) (30,688 ) (50,353 ) (64,493 ) Other income and expense Gain on long-term investment - 3,390 - 3,390 Other expense, net (6 ) (14 ) (7 ) (18 ) Interest expense (14 ) (25 ) (31 ) (53 ) Interest income 2,194 1,268 4,307 2,461 (Amortization of premium) accretion of discount on investments in U.S. Treasury securities, net (21 ) (34 ) 21 (46 ) Total other income and expense 2,153 4,585 4,290 5,734 Loss from operations before income taxes (25,867 ) (26,103 ) (46,063 ) (58,759 ) Income taxes - - - - Net loss $ (25,867 ) $ (26,103 ) $ (46,063 ) $ (58,759 ) Net loss per common share, basic and diluted $ (0.30 ) $ (0.60 ) $ (0.55 ) $ (1.53 ) Weighted average number of shares outstanding used in computation of net loss per common share, basic and diluted 85,626,751 43,573,628 83,417,555 38,372,704 Balance Sheet Data June 30, December 31, 2026 2025 Cash, cash equivalents, and investments in U.S. Treasury securities $ 234,171 $ 246,568 Total assets 254,898 268,688 Total liabilities 19,837 22,019 Total stockholders' equity 235,061 246,669

RMAT designation granted based on recent interim clinical data from ongoing Phase I/II clinical trial evaluating LX2020 for PKP2-ACM LX2020 now holds RMAT, Orphan Drug and Fast Track designations by the FDA NEW YORK, Aug. 05, 2026 (GLOBE NEWSWIRE) -- Lexeo Therapeutics, Inc. (Nasdaq: LXEO), a clinical stage genetic medicine company dedicated to pioneering novel treatments for cardiovascular diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to LX2020, the Company’s investigational AAV-based gene therapy candidate for the treatment of PKP2-associated arrhythmogenic cardiomyopathy (PKP2-ACM). RMAT designation for LX2020 is based on recent interim clinical data from the ongoing HEROIC-PKP2 Phase I/II clinical trial in PKP2-ACM. “Receiving RMAT designation for LX2020 is an important regulatory milestone that underscores the strength of the emerging clinical evidence from our HEROIC-PKP2 trial and the potential of LX2020 to address the underlying genetic cause of PKP2-ACM,” said Narinder Bhalla, M.D., Chief Medical Officer of Lexeo Therapeutics. “With no approved disease-modifying treatments currently available for patients living with this serious, progressive cardiovascular disease, this designation provides a valuable opportunity for early and ongoing engagement with the FDA as we work to advance LX2020 as a potential one-time gene therapy designed to address this significant unmet need. We look forward to sharing additional clinical and regulatory updates before the end of the year.” ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); RMAT designation is an FDA expedited program intended to facilitate the development and review of regenerative medicine therapies for serious conditions where preliminary clinical evidence indicates the potential to address unmet medical needs. Along with Orphan Drug and Fast Track designations, RMAT designation provides enhanced opportunities for interaction with the FDA, including early and ongoing guidance regarding clinical development, manufacturing and potential regulatory pathways, and it may provide eligibility for accelerated approval, priority review and rolling review. About LX2020 LX2020 is an AAV-based gene therapy candidate for the treatment of plakophilin-2-associated arrhythmogenic cardiomyopathy (PKP2-ACM). Mutations in the PKP2 gene are the most common genetic cause of ACM, responsible for approximately 50% of cases and estimated to affect approximately 60,000 people in the United States. PKP2 deficiency in ACM can lead to myocardial cell death, fibrosis, heart dysfunction, rhythm abnormalities, and sudden cardiac death. LX2020 is designed to systemically deliver a functional, full-length PKP2 gene within an adeno-associated viral capsid, AAVrh10, to cardiomyocytes to restore the desmosomal complex and cell-to-cell adhesion. LX2020 is being evaluated in the single-arm, open-label, multi-center HEROIC-PKP2 Phase I/II clinical trial ( NCT06109181 ). LX2020 has been granted RMAT, Orphan Drug and Fast Track designations by the FDA. About Lexeo Therapeutics 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. The Company is advancing a portfolio of therapeutic candidates that take aim at the underlying genetic causes of conditions, including LX2006 in Friedreich ataxia (FA), LX2020 in plakophilin-2 (PKP2) arrhythmogenic cardiomyopathy, and others in devastating diseases with high unmet need. Cautionary Note Regarding Forward-Looking Statements Certain statements in this press release may constitute “forward-looking statements” within the meaning of the federal securities laws, including, but not limited to, Lexeo’s expectations and plans regarding its current product candidates and programs, the anticipated benefits of its current product candidates, the timing for receipt and announcement of data from our clinical trials, and the timing and likelihood of potential regulatory developments and approval. Words such as “may,” “might,” “will,” “objective,” “intend,” “should,” “could,” “can,” “would,” “expect,” “believe,” “design,” “estimate,” “predict,” “potential,” “develop,” “plan” or the negative of these terms, and similar expressions, or statements regarding intent, belief, or current expectations, are forward-looking statements. While Lexeo believes these forward-looking statements are reasonable, undue reliance should not be placed on any such forward-looking statements. These forward-looking statements are based upon current information available to the company as well as certain estimates and assumptions and are subject to various risks and uncertainties (including, without limitation, those set forth in Lexeo’s filings with the U.S. Securities and Exchange Commission (SEC)), many of which are beyond the company’s control and subject to change. Actual results could be materially different from those indicated by such forward-looking statements as a result of many factors, including but not limited to: expectations regarding the initiation, progress, and expected results of Lexeo’s preclinical studies, clinical trials and research and development programs; the unpredictable relationship between preclinical study results and clinical study results; delays in submission of regulatory filings or failure to receive regulatory approval; liquidity and capital resources; and other risks and uncertainties identified in Lexeo’s Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2026, filed with the SEC on May 11, 2026, and subsequent future filings Lexeo may make with the SEC. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Lexeo claims the protection of the Safe Harbor contained in the Private Securities Litigation Reform Act of 1995 for forward-looking statements. Lexeo expressly disclaims any obligation to update or alter any statements whether as a result of new information, future events or otherwise, except as required by law. Media Response: Media@lexeotx.com Investor Response: Ashley Kaplowitz akaplowitz@lexeotx.com