Lexeo Therapeutics Announces FDA Breakthrough Therapy Designation for LX2006 in Friedreich Ataxia
Lexeo Therapeutics (Nasdaq: LXEO) has achieved a significant milestone as its drug candidate LX2006 received FDA Breakthrough Therapy designation for treating Friedreich ataxia (FA). The designation was based on promising interim clinical data from Phase I/II trials showing meaningful improvements in cardiac biomarkers and functional measures.
The drug has demonstrated clinically significant improvements in cardiac and neurologic functional measures, with increased frataxin expression observed in all cardiac biopsy participants at three months post-treatment. To date, 17 participants have been treated across two trials. The company plans to initiate a registrational study by early 2026.
Additionally, LX2006 has been selected for the FDA's Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) program, aimed at facilitating faster patient access to promising therapies.
Lexeo Therapeutics (Nasdaq: LXEO) ha raggiunto un traguardo importante: il suo candidato farmaco LX2006 ha ottenuto la designazione di Terapia Innovativa dalla FDA per il trattamento dell'atassia di Friedreich (FA). Questa designazione si basa su dati clinici intermedi promettenti provenienti dagli studi di Fase I/II, che hanno mostrato miglioramenti significativi nei biomarcatori cardiaci e nelle misure funzionali.
Il farmaco ha dimostrato miglioramenti clinicamente rilevanti nelle funzioni cardiache e neurologiche, con un aumento dell'espressione di frataxina osservato in tutti i partecipanti sottoposti a biopsia cardiaca a tre mesi dal trattamento. Finora, 17 partecipanti sono stati trattati in due studi. L'azienda prevede di avviare uno studio registrativo entro i primi mesi del 2026.
Inoltre, LX2006 è stato selezionato per il programma pilota della FDA Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP), volto a facilitare un accesso più rapido dei pazienti a terapie promettenti.
Lexeo Therapeutics (Nasdaq: LXEO) ha alcanzado un hito significativo: su candidato a medicamento LX2006 recibió la designación de Terapia Innovadora por la FDA para el tratamiento de la ataxia de Friedreich (FA). Esta designación se basó en datos clínicos interinos prometedores de los ensayos de Fase I/II que mostraron mejoras importantes en biomarcadores cardíacos y medidas funcionales.
El medicamento ha demostrado mejoras clínicamente significativas en funciones cardíacas y neurológicas, con un aumento en la expresión de frataxina observado en todos los participantes con biopsia cardíaca a los tres meses post-tratamiento. Hasta la fecha, 17 participantes han sido tratados en dos ensayos. La compañía planea iniciar un estudio registracional para principios de 2026.
Además, LX2006 ha sido seleccionado para el programa piloto de la FDA Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP), que busca facilitar un acceso más rápido de los pacientes a terapias prometedoras.
Lexeo Therapeutics (나스닥: LXEO)가 중요한 이정표를 달성했습니다. 후보 약물 LX2006이 프리드라이히 운동실조증(FA) 치료를 위해 FDA의 혁신 치료제 지정(Breakthrough Therapy designation)을 받았습니다. 이 지정은 1/2상 임상 시험의 유망한 중간 데이터에 근거하며, 심장 바이오마커 및 기능적 지표에서 의미 있는 개선을 보여주었습니다.
이 약물은 심장 및 신경 기능 지표에서 임상적으로 중요한 개선을 입증했으며, 치료 후 3개월 시점에 모든 심장 생검 참여자에서 프라탁신 발현 증가가 관찰되었습니다. 현재까지 17명의 참여자가 두 건의 시험에서 치료를 받았습니다. 회사는 2026년 초에 등록 연구를 시작할 계획입니다.
또한, LX2006은 FDA의 Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) 프로그램에 선정되어 유망한 치료제에 대한 환자의 신속한 접근을 지원합니다.
Lexeo Therapeutics (Nasdaq : LXEO) a franchi une étape importante : son candidat-médicament LX2006 a obtenu la désignation de thérapie révolutionnaire (Breakthrough Therapy) par la FDA pour le traitement de l’ataxie de Friedreich (FA). Cette désignation repose sur des données cliniques intermédiaires prometteuses issues des essais de phase I/II montrant des améliorations significatives des biomarqueurs cardiaques et des mesures fonctionnelles.
Le médicament a démontré des améliorations cliniquement significatives des fonctions cardiaques et neurologiques, avec une augmentation de l’expression de la frataxine observée chez tous les participants ayant subi une biopsie cardiaque trois mois après le traitement. À ce jour, 17 participants ont été traités dans le cadre de deux essais. La société prévoit de lancer une étude d’enregistrement d’ici début 2026.
Par ailleurs, LX2006 a été sélectionné pour le programme pilote de la FDA Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP), visant à faciliter un accès plus rapide des patients à des thérapies prometteuses.
Lexeo Therapeutics (Nasdaq: LXEO) hat einen bedeutenden Meilenstein erreicht: Ihr Wirkstoffkandidat LX2006 erhielt die FDA Breakthrough Therapy-Zulassung zur Behandlung der Friedreich-Ataxie (FA). Diese Zulassung basiert auf vielversprechenden Zwischenresultaten aus Phase I/II-Studien, die signifikante Verbesserungen bei kardialen Biomarkern und funktionellen Messwerten zeigten.
Das Medikament zeigte klinisch bedeutsame Verbesserungen der kardialen und neurologischen Funktionen, wobei bei allen Teilnehmern mit kardialer Biopsie drei Monate nach der Behandlung eine erhöhte Frataxin-Expression festgestellt wurde. Bis heute wurden 17 Teilnehmer in zwei Studien behandelt. Das Unternehmen plant, Anfang 2026 eine Zulassungsstudie zu starten.
Darüber hinaus wurde LX2006 für das FDA-Programm Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) ausgewählt, das darauf abzielt, den Patienten einen schnelleren Zugang zu vielversprechenden Therapien zu ermöglichen.
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Insights
Lexeo's FDA Breakthrough designation for LX2006 significantly accelerates its path to market for treating fatal Friedreich Ataxia cardiomyopathy.
The FDA's Breakthrough Therapy designation for Lexeo's LX2006 represents a major regulatory milestone that could substantially accelerate the development timeline for this gene therapy targeting Friedreich Ataxia (FA). This rare genetic disorder currently has no approved treatments for its cardiac manifestations, which are the leading cause of death among patients.
The designation was granted based on compelling interim clinical data showing clinically significant improvements in cardiac biomarkers and both cardiac and neurologic functional measures. Importantly, increased frataxin expression—the protein deficient in FA patients—was observed in all participants with cardiac biopsies at three months post-treatment, providing a direct biological marker of efficacy.
Equally significant is Lexeo's inclusion in the FDA's Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) program. This specialized program is designed to streamline manufacturing and quality control aspects specifically for therapies with expedited clinical timelines, addressing a critical bottleneck that often delays breakthrough treatments from reaching patients.
The company is taking a methodical approach by conducting the CLARITY-FA natural history study as a concurrent external control arm, which will strengthen the statistical power of their upcoming registrational trial planned for early 2026. This demonstrates strategic regulatory planning, as gene therapy trials often face challenges in designing appropriate control groups.
With four special FDA designations now secured (Breakthrough Therapy, RMAT, Orphan Drug, and Fast Track), Lexeo has maximized regulatory advantages available for expediting LX2006's development, potentially shortening the path to market by years while maintaining rigorous efficacy and safety standards.
Breakthrough Therapy designation based on interim clinical data from Phase I/II trials showing clinically meaningful improvements in cardiac biomarkers and functional measures
LX2006 also selected for FDA Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) program, created to facilitate CMC registrational readiness and support faster patient access
NEW YORK, July 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 that the U.S. Food and Drug administration (FDA) has granted Breakthrough Therapy designation to LX2006 based on clinical evidence generated on both cardiac and neurologic measures of Friedreich ataxia (FA). LX2006 has also been selected to participate in the FDA Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) program, intended to enable earlier patient access to therapies with expedited clinical development timelines.
“Receiving Breakthrough Therapy designation is a significant milestone, highlighting the potential of LX2006 and the strength of clinical evidence generated to date,” said Dr. Sandi See Tai, Chief Development Officer of Lexeo Therapeutics. “We are highly encouraged by the impact of LX2006 on key measures of cardiac health, especially given the lack of treatments for FA cardiomyopathy today, which is the leading cause of death in FA. We are also optimistic about the improvements we have observed in functional measures of FA more broadly, and we look forward to a continued partnership with the FDA through the Breakthrough Therapy designation and the CDRP program as we work to bring this potential treatment to patients as quickly as possible.”
The FDA decision was based on interim clinical data demonstrating that treatment with LX2006 was associated with clinically significant improvements in cardiac biomarkers and in cardiac and neurologic functional measures, with increased frataxin expression observed in all participants with cardiac biopsies at three months post treatment. To date, 17 participants have been treated across two trials: the Lexeo-sponsored SUNRISE-FA Phase 1/2 clinical trial (NCT05445323) and the Weill Cornell Medicine investigator-initiated Phase 1A trial (NCT05302271). Lexeo is currently enrolling a prospective natural history study, CLARITY-FA, which will serve as a concurrent external control arm for the registrational study. The Company expects to initiate the registrational study by early 2026 and is actively working with FDA to finalize the statistical analysis plan (SAP).
Breakthrough Therapy designation is intended to accelerate the development and review of investigational therapies that aim to treat serious or life-threatening diseases and where preliminary clinical evidence indicates that the therapy may demonstrate substantial improvement over available alternatives. This designation is in addition to Regenerative Medicine Advanced Therapy (RMAT) designation, Orphan Drug designation and Fast Track designation, all previously granted to LX2006 by the FDA. The CDRP program was created by the FDA to facilitate expedited CMC development of investigational therapies with expedited clinical development timeframes. This program increases communication between FDA and sponsors on CMC development specifically, with the goal of enabling earlier patient access to promising therapies in areas of high unmet need.
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 and the timing for receipt and announcement of data from its 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: 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 Annual Report on Form 10-K for the annual period ended December 31, 2024, filed with the SEC on March 24, 2025, Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2025, filed with the SEC on May 12, 2025, as amended, 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:
Carlo Tanzi, Ph.D.
ctanzi@kendallir.com
FAQ
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