Lexeo Therapeutics Announces Progress in FDA Discussions for Accelerated Approval Pathway and Positive Interim Clinical Data for LX2006 in Friedreich Ataxia Cardiomyopathy
Lexeo Therapeutics (Nasdaq: LXEO) reported FDA openness to pooling ongoing Phase I/II data with pivotal study data to support an Accelerated Approval BLA for LX2006 in Friedreich ataxia cardiomyopathy and said the FDA may allow the LVMI co-primary endpoint to be assessed earlier than 12 months. Interim clinical data (n=16 with >6 months follow-up) showed mean LVMI reductions of 18% at 6 months and 23% at 12 months (6 participants with abnormal baseline LVMI), dose-dependent improvements in mid/high cohorts, a 2.0-point mean mFARS improvement, and broad reductions in high-sensitivity troponin I. Safety was generally acceptable with no Grade 3+ SAEs and one previously disclosed Grade 2 myocarditis. Lexeo plans to start the pivotal study in H1 2026 pending final protocol and comparability work.
Lexeo Therapeutics (Nasdaq: LXEO) ha riferito che la FDA è disponibile a combinare i dati in corso di fase I/II con i dati dello studio pivot per supportare una BLA di Approvazione Accelerata per LX2006 nella cardiomiopatia da atassia di Friedreich e ha detto che la FDA potrebbe consentire di valutare l'endpoint co-primario LVMI prima di 12 mesi. I dati clinici interinali (n=16 con >6 mesi di follow-up) hanno mostrato una riduzione media di LVMI dell'18% a 6 mesi e del 23% a 12 mesi (6 partecipanti con LVMI basale anomalo), miglioramenti dose-dipendenti nei gruppi medio-alti, un miglioramento medio di 2,0 punti mFARS, e riduzioni ampie della troponina I ad alta sensibilità. La sicurezza è stata in generale accettabile con nessun SAE di grado 3+ e una miocardite di grado 2 precedentemente dichiarata. Lexeo prevede di avviare lo studio principale nel primo semestre del 2026, in attesa del protocollo finale e del lavoro di comparabilità.
Lexeo Therapeutics (Nasdaq: LXEO) informó que la FDA está abierta a fusionar datos en curso de Fase I/II con datos del estudio pivotal para respaldar una BLA de Aprobación Acelerada para LX2006 en la cardiomiopatía por Friedreich y dijo que la FDA podría permitir que el endpoint co-principal LVMI se evalúe antes de 12 meses. Los datos clínicos interinos (n=16 con >6 meses de seguimiento) mostraron reducciones medias de LVMI del 18% a los 6 meses y del 23% a los 12 meses (6 participantes con LVMI basal anómalo), mejoras dependientes de la dosis en cohortes medias/altas, una mejora media de 2,0 puntos en mFARS, y reducciones amplias en la troponina I de alta sensibilidad. La seguridad fue generalmente aceptable sin SAE grado 3+ y una miocarditis grado 2 previamente divulgada. Lexeo planea iniciar el estudio pivotal en la H1 de 2026 sujetos a protocolo final y trabajo de comparabilidad.
Lexeo Therapeutics (Nasdaq: LXEO)는 FDA가 LX2006를 위한 Accelerated Approval BLA를 지원하기 위해 현재 진행 중인 1상/2상 데이터를 핵심 연구 데이터와 결합하는 데 열려 있다고 보고했고, FDA가 LVMI를 공동 주요 종점으로 12개월 이전에 평가를 허용할 수 있다고 밝혔다. 중간 임상 데이터(n=16, 추적기간 >6개월)는 6개월에 LVMI가 평균 18% 감소, 12개월에 23% 감소(기저 LVMI 비정상 6명), 중간/상위 코호트에서 용량 의존적 개선, 평균 2.0점의 mFARS 개선, 고감도 트로포닌 I의 광범위한 감소를 보였다. 안전성은 전반적으로 수용 가능했고 3등급 이상의 SAE는 없었으며 미리 공개된 2등급 심근염이 한 사례 있었다. Lexeo는 최종 프로토콜 및 비교 가능성 작업이 완료되는 것을 전제로 2026년 상반기에 결정 연구를 시작할 계획이다.
Lexeo Therapeutics (Nasdaq: LXEO) a indiqué que la FDA est ouverte à regrouper les données en cours de Phase I/II avec les données de l’étude pivot pour soutenir une demande d’Approbation Accélérée (BLA) pour LX2006 dans la cardiopathie Friedreich et a déclaré que la FDA pourrait permettre d’évaluer l’endpoint co-principal LVMI plus tôt que 12 mois. Les données cliniques intermédiaires (n=16 avec >6 mois de suivi) ont montré des réductions moyennes du LVMI de 18% à 6 mois et de 23% à 12 mois (6 participants avec LVMI de base anormal), des améliorations dépendantes de la dose dans les cohortes médianes/élevées, une amélioration moyenne de 2,0 points du mFARS et des réductions importantes de la troponine I haute sensibilité. La sécurité était globalement acceptable sans SAE de grade 3+ et une myocardite de grade 2 préalablement divulguée. Lexeo prévoit de lancer l’étude pivot au cours du premier semestre 2026, sous réserve du protocole final et des travaux de comparabilité.
Lexeo Therapeutics (Nasdaq: LXEO) berichtete, dass die FDA offen dafür sei, aktuelle Phase-I/II-Daten mit Daten der pivotalen Studie zusammenzuführen, um eine Accelerated-Approval-BLA für LX2006 bei der Friedreich-Ataxie-Kardiomyopathie zu unterstützen, und erklärte, dass die FDA möglicherweise zulassen könnte, dass den LVMI-Co-Primäreindeutungen vor Ablauf von 12 Monaten bewertet werden. Vorläufige klinische Daten (n=16, >6 Monate Follow-up) zeigten mittlere LVMI-Reduktionen von 18% nach 6 Monaten und 23% nach 12 Monaten (6 Teilnehmer mit abnormalem LVMI-Baseline), dosisabhängige Verbesserungen in mittel-hohen Kohorten, eine mittlere Verbesserung von 2,0 Punkten im mFARS und breite Reduktionen des hochsensitiven Troponin I. Die Sicherheit war im Allgemeinen akzeptabel, es gab keine Grade-3+ SAEs und eine zuvor veröffentlichte Grad-2-Myokarditis. Lexeo plant, die pivotal Studie im ersten Halbjahr 2026 zu starten, vorbehaltlich des endgültigen Protokolls und der Vergleichsarbeiten.
Lexeo Therapeutics (Nasdaq: LXEO) قالت إن إدارة الغذاء والدواء الأمريكية منفتحة على دمج البيانات الحالية للمرحلتين I/II مع بيانات الدراسة المحورية لدعم BLA للموافقة المعزَّزة لـ LX2006 في اعتلالات قلب Friedreich، وقالت إن FDA قد تسمح بتقييم نقطة النهاية المشتركة LVMI قبل مرور 12 شهراً. أظهرت البيانات السريرية المؤقتة (عدد المشاركين = 16 مع متابعة >6 أشهر) انخفاضات متوسطة في LVMI بنسبة 18% عند 6 أشهر و23% عند 12 شهراً (6 مشاركين لديهم LVMI ابتدائي غير طبيعي)، وتحسنات تعتمد على الجرعة في مجاميع متوسطة/عالية، وتحسن بمقدار نقطة واحدة بمعدل 2.0 في mFARS، وانخفاضات واسعة في Troponin I عالي الحساسية. السلامة كانت مقبولة بشكل عام بدون حوادث خطيرة من الدرجة 3+، ووجود ميوكارديتس من الدرجة 2 كما disclose سابقاً. تخطط Lexeo لبدء الدراسة المحورية في النصف الأول من عام 2026 رهناً بالبروتوكول النهائي وعملية المقارنة.
Lexeo Therapeutics（纳斯达克股票代码：LXEO）表示，FDA愿意将正在进行的I/II期数据与关键研究数据汇集在一起，以支持LX2006在 Friedreich 共济运动性心肌病中的加速批准生物制品许可申请（BLA），并表示FDA可能允许在12个月前评估LVMI的共同主要终点。中期临床数据（n=16，随访>6个月）显示在6个月时LVMI平均下降18%，在12个月时下降23%（有6名基线LVMI异常的参与者），中等及高剂量队列存在剂量依赖性改善，mFARS平均改善2.0点，高敏肌钙蛋白I普遍下降。总体安全性可接受，未出现3级及以上不良事件，一例此前披露的2级心肌炎。Lexeo计划在2026年上半年启动关键研究，前提是最终方案和可比性工作完成。
- FDA receptive to pooling Phase I/II data for Accelerated Approval
- LVMI mean −18% at 6 months and −23% at 12 months (abnormal baseline n=6)
- Dose-dependent LVMI gains: mid/high cohorts −28% at 6 months and −33% at 12 months (n=3)
- mFARS mean improvement of 2.0 points across participants with >6 months follow-up
- 14 of 16 participants showed >25% reduction in high-sensitivity troponin I
- Must submit enhanced manufacturing comparability and additional nonclinical data before pivotal study
- One possibly treatment-related Grade 2 asymptomatic myocarditis event observed one year after dosing
- Interim dataset is small (n=16 with >6 months follow-up), limiting statistical certainty
Insights
FDA openness to pooling data plus interim efficacy exceeding agreed thresholds materially advances LX2006's accelerated approval pathway.
Lexeo obtained FDA willingness to accept pooled data from ongoing Phase I/II studies with a planned pivotal study and noted the Agency will consider the co-primary LVMI endpoint earlier than
The regulatory path now depends on three explicit, article-stated items: submission of enhanced manufacturing comparability data for the Sf9-baculovirus process versus the earlier HEK293 supply, meeting an additional nonclinical requirement, and final pivotal protocol agreement. Lexeo plans to start the pivotal study in
Watch for completion and acceptance of the comparability package, resolution of the nonclinical requirement, final pivotal protocol that confirms pooling methods and timing of the LVMI co-primary, and the actual initiation of the pivotal study in
U.S. Food and Drug Administration (FDA) 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
Interim clinical data show sustained or deepening improvements in the majority of participants across both cardiac and neurologic measures of Friedreich ataxia
Participants with abnormal left ventricular mass index (LVMI) at baseline achieved
Clinically meaningful improvement observed in the modified Friedreich Ataxia Rating Scale (mFARS), indicative of slowed disease progression and improved function
Company to host webcast today at 8:00 AM ET
NEW YORK, Oct. 07, 2025 (GLOBE NEWSWIRE) -- Lexeo Therapeutics, Inc. (Nasdaq: LXEO), a clinical stage genetic medicine company dedicated to pioneering novel treatments for cardiovascular diseases, today announced updates to key components of an Accelerated Approval pathway for LX2006 in Friedreich ataxia (FA) cardiomyopathy, alongside new interim clinical data from ongoing Phase I/II studies.
“We are encouraged by our recent dialogue with the FDA on LX2006, and we appreciate the Agency’s collaborative spirit as we work to deliver a potentially life-changing therapy to the FA community as efficiently as possible,” said R. Nolan Townsend, Chief Executive Officer of Lexeo Therapeutics. “Given the highly compelling data to date that demonstrate clinically meaningful improvements across both cardiac and neurologic measures of FA, we are now pursuing a development strategy that could enable a smaller pivotal study, given the potential to pool data with the ongoing Phase I/II trials, as well as potentially assessing the co-primary endpoint of LVMI earlier than 12 months. This approach could accelerate our overall timeline toward a BLA submission for LX2006 under the Accelerated Approval pathway.”
FDA Feedback to Date
In response to questions from the Company regarding the possibility of a faster path to a BLA, the FDA has indicated openness to a BLA submission for accelerated approval that includes clinical data from the ongoing Phase I/II studies of LX2006 pooled with new clinical data to be generated in the planned pivotal study. To enable pooling of these data to support licensure, Lexeo will submit enhanced manufacturing comparability data and meet an additional nonclinical requirement prior to the initiation of the planned pivotal study, given the Company’s intention to leverage its optimized, high-yield Sf9-baculovirus manufacturing platform for future clinical and commercial drug supply, compared to the adherent HEK293 process used for Phase I/II clinical supply. The FDA also previously agreed to evaluate the co-primary endpoint of LVMI at a time point earlier than 12 months. Lexeo continues to engage with the FDA on the pivotal protocol and comparability. In discussions to date, there have been no changes to the previously disclosed alignment with the FDA on key parameters related to the LX2006 planned registrational study.
Collectively, Lexeo believes this regulatory feedback has the potential to reduce the size and length of the planned pivotal study, possibly accelerating the overall timeline to BLA submission. Lexeo plans to initiate the LX2006 pivotal study as soon as possible in the first half of 2026, pending finalization of the trial protocol. FDA has previously granted Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug and Fast Track designations to LX2006, and admitted LX2006 into the CMC Development and Readiness Pilot (CDRP) program.
LX2006 Interim Clinical Update (n=16 participants with >6-months of follow-up)
Updated interim clinical data from both ongoing Phase I/II studies of LX2006 continue to show encouraging safety and efficacy, exceeding the thresholds previously agreed with FDA for co-primary endpoints LVMI and frataxin expression.
Left ventricular mass index (LVMI):
- Among participants with abnormal baseline LVMI (key inclusion criteria for pivotal study; n=6):
- 6 of 6 participants achieved LVMI measurements within the normal range as of latest visit
- 5 of 6 participants achieved >
10% improvement by 12 months 18% mean improvement in LVMI at 6 months and23% mean improvement at 12 months, exceeding10% FDA-aligned threshold for pivotal study28% mean improvement in LVMI at 6 months and33% mean improvement at 12 months in mid- and high-dose cohorts (n=3), suggesting dose-dependent improvement
- Among participants with normal baseline LVMI (n=10), the majority demonstrated LVMI improvement or stabilization over time
Secondary cardiac biomarkers:
- 14 of 16 participants achieved >
25% reduction in high-sensitivity troponin I from baseline at latest visit - 14 of 16 participants achieved reduction or stabilization in lateral wall thickness (LWT) from baseline at latest visit
Modified Friedreich Ataxia Rating Scale (mFARS):
- 2.0-point mean improvement from baseline at latest visit across all participants with >6-months of follow-up (n=16)
- 11 of 16 participants achieved reduction or stabilization in mFARS from baseline at latest visit
Previously reported data from Lexeo’s ongoing SUNRISE-FA trial (n=8) showed that all study participants achieved increases in frataxin protein expression from baseline at 3 months, with dose-dependent increases observed across cohorts.
LX2006 Interim Safety Update (n=17 participants)
- Treatment with LX2006 has been generally well tolerated with no Grade 3+ SAEs to date
- No clinically significant complement activation
- Minimal, transient liver function test (LFT) elevations
- No signs of frataxin over-expression observed in cardiac tissue
- No participants discontinued from either study
- One previously disclosed, possibly treatment-related Grade 2 event of asymptomatic myocarditis observed one year after dosing
Corporate Webcast Details
Lexeo Therapeutics will host a webcast at 8:00 AM ET today, October 7, 2025. Analysts and investors can participate by accessing the webcast live on the News & Events page in the Investors section of Lexeo’s website, www.lexeotx.com. The webcast will be archived on the company’s website following completion of the call, and presentation materials will be available on the Presentations page of the website.
About LX2006
LX2006 is an AAV-based gene therapy candidate for the treatment of FA cardiomyopathy, the leading cause of death in individuals with FA which affects approximately 5,000 people in the United States. LX2006 is designed to systemically deliver a functional frataxin gene to promote the expression of the frataxin protein and restore mitochondrial function in myocardial cells. LX2006 is being evaluated in two clinical trials, the Lexeo-sponsored SUNRISE-FA Phase 1/2 clinical trial (NCT05445323) and the Weill Cornell Medicine investigator-initiated Phase 1A trial (NCT05302271). LX2006 has been granted Breakthrough Therapy, Regenerative Medicine Advanced Therapy, Orphan Drug, Rare Pediatric Disease and Fast Track designations by the FDA, admitted into the FDA CMC Development and Readiness Pilot (CDRP) program, and granted orphan medicinal product designation by the European Commission.
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) cardiomyopathy, 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 structure of and timelines for completion of any current or additional clinical trials required by the FDA, the timing for receipt and announcement of data from any such clinical trials, and the timing and likelihood of potential regulatory developments, trial design changes 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: risks and uncertainties related to global macroeconomic conditions and related volatility; 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 June 30, 2025, filed with the SEC on August 14, 2025, 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:
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FAQ
What did Lexeo (LXEO) announce about FDA support for an Accelerated Approval pathway on October 7, 2025?
What were the LX2006 interim LVMI results reported by Lexeo (LXEO) on October 7, 2025?
How did LX2006 affect neurologic function in the interim Lexeo (LXEO) data?
What safety findings did Lexeo (LXEO) report for LX2006 in the October 7, 2025 update?
When does Lexeo plan to start the LX2006 pivotal study and what conditions apply?
How might the FDA feedback affect LXEO's pivotal study size and timeline?
