LXEO

SUNRISE-FA 2 open-label, pivotal trial protocol and SAP for LX2006 submitted to FDA in Q1 2026; Awaiting final FDA feedback Multiple presentations highlighting progress across cardiac genetic medicine pipeline and optimized, Sf9-baculovirus AAV manufacturing platform to be presented at ASGCT 2026 Appointed Laura Sepp-Lorenzino, Ph.D. to Board of Directors Cash, cash equivalents and investments of $227.6 million expected to provide operational runway into 2028 NEW YORK, May 11, 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 first quarter 2026. “We continued to make steady progress across our key priorities in the first quarter of 2026. As we work with the FDA to finalize the SUNRISE‑FA 2 pivotal study protocol, we are advancing site readiness and patient identification activities to ensure we are prepared to initiate the study promptly once the protocol is complete,” said R. Nolan Townsend, Chief Executive Officer of Lexeo Therapeutics. “We will also have a meaningful scientific presence at ASGCT this week, where we will share updates from our LX2006 program as well as new preclinical data for LX2022. We remain committed to advancing our pipeline toward meaningful therapies for patients and look forward to providing further updates as our programs continue to progress.” Program Updates and Recent Progress LX2006 in Friedreich Ataxia (FA) In May 2026, Lexeo will share multiple presentations on LX2006 at the 29th American Society of Gene and Cell Therapies (ASGCT) Annual Meeting: Phase I/II interim clinical data of LX2006 continue to show sustained or deepening improvements across both cardiac and neurologic measures of FA, including statistically significant improvement in mean mFARS scores for LX2006-treated participants compared to a propensity-matched control cohort from the UNIFAI natural history study (n=17; p=0.003). LX2006 remains generally well tolerated with no Grade 3+ SAEs to date. Nonhuman primate research with LX2006 conducted by researchers at Weill Cornell Medicine demonstrates the potential of sequential dosing strategies to treat FA. Eight weeks following systemic intravenous administration of LX2006, nonhuman primates were...

SUNRISE-FA 2 open-label, pivotal trial protocol and SAP for LX2006 submitted to FDA in Q1 2026 following Type B meeting; study initiation on track for 1H 2026 First CMC Development and Readiness Program (CDRP) meeting for LX2006 in Q1 2026; aligned with FDA on flexible approach to process validation such as reduced PPQ manufacturing batches Late-breaking oral presentations at the American College of Cardiology (ACC) Annual Meeting demonstrate continued, encouraging efficacy and safety data across LX2006 and LX2020 programs Established research collaboration with Johnson & Johnson to explore targeted cardiac delivery of AAV gene therapy using novel, localized routes of administration Strengthened leadership team with addition of new Chief Medical Officer and senior appointments bolstering cardiac expertise Cash, cash equivalents and investments of $246.6 million expected to provide operational runway into 2028 NEW YORK, March 30, 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 fourth quarter and full year 2025. “Over the past year we have made meaningful progress across our cardiac pipeline, including significant advancements for both LX2006 and LX2020,” said R. Nolan Townsend, Chief Executive Officer of Lexeo Therapeutics. “Most recently, we were pleased to be selected for two Late-Breaker presentations at the American College of Cardiology and appreciate the opportunity to engage the cardiology community around the future of precision medicine in cardiovascular care. Looking ahead, we remain focused on disciplined execution as we continue to advance our pipeline toward key milestones. With additional resources from our October financing, the expansion of our cardiac leadership team, and multiple announced preclinical research collaborations, we believe Lexeo is well positioned to drive significant long-term value for patients and shareholders.” Program Updates and Recent Progress LX2006 in Friedreich Ataxia (FA) In October 2025, Lexeo announced progress in FDA discussions and reported positive interim clinical data from both ongoing Phase I/II studies of LX2006 demonstrating an encouraging safety profile and evidence of meaningful cardiac...

FDA open to pooling data from ongoing Phase I/II studies of LX2006 with data from pivotal trial, and to earlier co-primary endpoint assessment, to support a Biologics License Application FDA approved comparability report between LX2006 HEK and Sf9 manufacturing processes in November 2025, endorsing use of Sf9 final commercial manufacturing process to begin dosing patients in upcoming pivotal study LX2006 interim clinical data show clinically meaningful improvements across cardiac and neurologic measures of Friedreich ataxia, including left ventricular mass index and the modified Friedreich Ataxia Rating Scale Completed enrollment of LX2020 HEROIC-PKP2 Phase I/II trial with ten participants dosed; interim data from low-dose cohort reported and additional clinical data from high-dose cohorts on track for January 2026 $154 million equity financing to support LX2006 registrational activities and further development of cardiac pipeline; in addition to Q3-25 end cash, cash equivalents and investments, expected to fund operations into 2028 NEW YORK, Nov. 05, 2025 (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 third quarter 2025 financial results. “We continue to build significant momentum across our cardiac pipeline, and the recent financing strengthens our ability to execute on essential manufacturing and commercial activities for LX2006 as we look towards registrational readiness,” said R. Nolan Townsend, Chief Executive Officer of Lexeo Therapeutics. “Interim clinical data for LX2006 demonstrate meaningful benefit across measures of cardiac health and neurologic function, including improvements in the modified Friedreich Ataxia Rating Scale (mFARS), and we believe this therapy could represent a transformational step forward in the standard of care for FA. Furthermore, with enrollment complete in the HEROIC-PKP2 Phase I/II trial, we look forward to sharing new clinical data for LX2020 in January.” Business and Program Updates LX2006 in Friedreich Ataxia (FA): Regulatory Progress: In response to questions regarding the possibility of a faster path to a Biologics License Application (BLA), the FDA has indicated openness to a BLA submission for accelerated approval that include...

Breakthrough Therapy designation granted for LX2006 based on interim data from Phase I/II trials demonstrating clinically meaningful improvements in cardiac and neurologic measures of Friedreich ataxia LX2006 selected for FDA Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) program, created to facilitate CMC registrational readiness and support faster patient access 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 Strategic partnership announced with Perceptive Xontogeny Venture Funds and venBio Partners to advance non-viral, RNA-based therapeutics for genetic cardiac diseases $80 million equity financing to support development of clinical stage pipeline; cash, cash equivalents and investments in marketable securities of $152.5 million expected to provide operational runway into 2028 Louis Tamayo appointed Chief Financial Officer NEW YORK, Aug. 14, 2025 (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 second quarter 2025 financial results. “Over the last several months, Lexeo has made significant progress advancing our clinical stage programs, diversifying our pipeline through a strategic partnership that we believe enables us to stay on the cusp of leading-edge cardiovascular science, and further strengthening our balance sheet,” said R. Nolan Townsend, Chief Executive Officer of Lexeo Therapeutics. “FDA Breakthrough Therapy designation for LX2006 underscores the potential of this gene therapy candidate, and we are moving as quickly as possible in close partnership with patient advocates, clinicians, and the FA community to initiate a registrational study early next year. We are also continuing to advance our LX2020 program for arrhythmogenic cardiomyopathy with eight participants dosed to date and data updates expected in the second half of this year.” Business and Program Updates LX2006 in Friedreich Ataxia (FA): Regulatory Updates: In July 2025, Lexeo received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) for LX2006 based on interim clinical data demonstrating clinically meaningful improvements in car...

Announced positive interim data for LX2006 from Phase 1/2 studies in Friederich ataxia (FA) cardiomyopathy; frataxin expression and LVMI improvement exceeded co-primary target thresholds for planned registrational study LX2006 registrational study expected to begin by early 2026; commencing enrollment in prospective natural history study, CLARITY-FA, in Q2 2025 to serve as concurrent external control Phase 1/2 clinical trial of LX2020 (HEROIC-PKP2) currently enrolling patients in Cohort 3; interim clinical data update on track for second half of 2025 Redeployed $20 million to focus on clinical-stage programs; cash, cash equivalents and investments of $106.9 million expected to provide operational runway into 2027 NEW YORK, May 12, 2025 (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 first quarter 2025 financial results. “Based on the highly encouraging clinical data shared to date, we believe LX2006 could be transformational and establish a new standard of care in FA cardiomyopathy,” said R. Nolan Townsend, Chief Executive Officer of Lexeo Therapeutics. “We look forward to beginning enrollment in the CLARITY-FA natural history study imminently and moving as quickly as possible to initiate a registrational study for LX2006 by early 2026. We were also proud to share the promising, early data for LX2020 for the treatment of PKP2-associated arrhythmogenic cardiomyopathy and we look forward to sharing additional clinical updates later in 2025 for this program.” Business and Program Updates LX2006 for the Treatment of FA Cardiomyopathy: In April 2025, Lexeo announced positive interim data of LX2006 across both the Lexeo-sponsored SUNRISE-FA Phase 1/2 clinical trial (NCT05445323) and the Weill Cornell Medicine investigator-initiated Phase 1A trial (NCT05302271). Efficacy: Clinically meaningful improvements were observed across cardiac biomarkers and functional measures in the majority of participants across both studies. Participants with abnormal left ventricular mass index (LVMI) at baseline achieved 25% mean reduction in LVMI by 12 months or sooner, exceeding the 10% target reduction in LVMI by 12 months aligned with the U.S. Food and Drug Administration (FDA) for the planne...

Additional alignment with FDA on LX2006 planned pivotal study including protein expression co-primary endpoint: based on improvements in LVMI across participants with abnormal LVMI at baseline, frataxin expression to be evaluated for any increase from baseline rather than numerical threshold Interim update from cohort 1 of LX2020 HEROIC-PKP2 Phase 1/2 trial: observed 71% and 115% increases in PKP2 protein expression in first two post-treatment biopsies; first participant evaluated 6-months after dosing experienced 67% reduction in premature ventricular contractions (PVCs) Completed enrollment of cohort 2 of LX2020 HEROIC-PKP2 Phase 1/2 trial; interim clinical data update expected in second half of 2025 LX2020 has been generally well tolerated with no treatment-related serious adverse events to date Cash, cash equivalents and investments of $128.5 million expected to provide operational runway into 2027 NEW YORK, March 24, 2025 (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 fourth quarter and full year 2024 financial results. “We are pleased to share further regulatory clarity for LX2006 for the treatment of Friedreich ataxia (FA) cardiomyopathy, and we appreciate the continued partnership from the FDA on an accelerated approval pathway to support adults and children living with this devastating condition,” said R. Nolan Townsend, Chief Executive Officer of Lexeo Therapeutics. “We are also encouraged by the favorable safety profile and early data observed in participants dosed with LX2020 to date. We look forward to sharing additional clinical updates later in 2025 now that the second cohort of the LX2020 HEROIC-PKP2 Phase 1/2 trial in arrhythmogenic cardiomyopathy is fully enrolled.” Business and Program Updates LX2006 for the Treatment of FA Cardiomyopathy: Regulatory Update: Further alignment on elements of the accelerated development pathway following a Type B RMAT meeting with the U.S. Food and Drug Administration (FDA): Frataxin expression to be evaluated for any increase from baseline rather than numerical threshold, based on improvements to date in LVMI across participants with abnormal LVMI at baseline Inclusion of pediatric cohorts, both adolescents and childre...