Lexeo Therapeutics Reports First Quarter 2025 Financial Results and Operational Highlights
- LX2006 showed 115% increase in cardiac frataxin expression in high-dose cohort
- 25% mean reduction in LVMI achieved, exceeding 10% target threshold
- LX2020 demonstrated 71-115% increases in PKP2 protein expression
- Strong cash position of $106.9 million providing runway into 2027
- $20 million redeployed to focus on clinical-stage programs
- 15% workforce reduction implemented
- Increased net loss to $32.7 million in Q1 2025 from $21.7 million in Q1 2024
- General and Administrative expenses more than doubled to $16.6 million from $7.5 million YoY
Insights
Lexeo reports promising clinical data for gene therapies in heart diseases with strong efficacy signals, extending cash runway into 2027 through strategic redeployment.
Lexeo Therapeutics has reported highly encouraging interim results for its lead gene therapy candidates targeting rare cardiac diseases. For LX2006 in Friedreich ataxia cardiomyopathy, patients showed a 25% mean reduction in left ventricular mass index (LVMI), significantly exceeding the 10% target threshold agreed with the FDA for the planned registrational study. The therapy also demonstrated impressive 115% average increase in cardiac frataxin expression in the high-dose cohort.
Their second candidate, LX2020 for PKP2-associated arrhythmogenic cardiomyopathy, showed protein expression increases of 71% and 115% in two patients, with one patient experiencing a substantial 67% reduction in premature ventricular contractions at 6 months. Both therapies have been generally well-tolerated with no treatment-related serious adverse events reported.
On the financial front, Lexeo has strategically redeployed
The company has detailed clear regulatory milestones, including the imminent launch of the CLARITY-FA natural history study as an external control arm, with plans to initiate the LX2006 registrational study by early 2026 and potential efficacy readout in 2027. For LX2020, enrollment in cohort 3 is ongoing with an interim data update expected in H2 2025.
This strategic focus on two promising clinical-stage cardiac gene therapies positions Lexeo well in the rare disease space, particularly as both programs target diseases with significant unmet needs and have shown early signals of transformative potential.
Lexeo's gene therapies show remarkable early efficacy in cardiac genetic disorders with impressive protein expression and functional improvements.
The interim clinical data from Lexeo's gene therapy programs represent potentially transformative advances for patients with genetic cardiac disorders. For LX2006 in Friedreich ataxia cardiomyopathy, the 25% mean reduction in LVMI is particularly significant as left ventricular hypertrophy is a hallmark of FA cardiomyopathy and a key predictor of adverse outcomes. This substantial reduction exceeds the minimal threshold needed for clinical significance and suggests a genuine disease-modifying effect.
Most impressive is the 115% average increase in cardiac frataxin expression in the high-dose cohort. Frataxin deficiency is the root cause of Friedreich ataxia, and restoring expression to these levels addresses the fundamental molecular defect. The fact that protein increases translated to structural improvements (reduced LVMI) provides strong evidence of therapeutic effect and disease modification potential.
For the PKP2-ACM program (LX2020), the protein expression data showing 71-115% increases is similarly encouraging. The 67% reduction in PVCs observed in one patient at 6 months suggests the therapy may ameliorate the electrical instability characteristic of this condition. PKP2 deficiency compromises desmosomal integrity in cardiomyocytes, leading to arrhythmias and structural heart disease that can cause sudden cardiac death - making these early signals particularly meaningful.
The clean safety profile for both therapies is noteworthy given the challenges often associated with cardiac gene therapy, including inflammatory responses and potential arrhythmogenicity. The apparent absence of treatment-related serious adverse events supports a favorable risk-benefit profile.
The regulatory strategy appears sound, with Lexeo's concurrent natural history study (CLARITY-FA) providing a pragmatic approach to generate external control data. The FDA alignment on co-primary endpoints demonstrates regulatory engagement and increases confidence in the development pathway toward potential approval.
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
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 the10% target reduction in LVMI by 12 months aligned with the U.S. Food and Drug Administration (FDA) for the planned registrational study. - Protein Expression: Increases in cardiac frataxin expression were observed in all SUNRISE-FA participants at 3-months post treatment, with an average increase of
115% over baseline observed in the high-dose cohort. - Safety: LX2006 continues to be generally well tolerated with no new treatment-related serious adverse events to report.
- Regulatory Plans: Lexeo expects final alignment with FDA on the LX2006 planned pivotal study protocol and statistical analysis plan in 2025. Lexeo previously aligned with FDA on co-primary endpoints for the study and measurement thresholds: greater than
10% reduction in LVMI as measured by cardiac MRI, and any increase from baseline cardiac frataxin expression as measured by liquid chromatography mass spectrometry (LCMS). - Next Steps: In Q2 2025, Lexeo expects to begin enrollment in a prospective natural history study serving as a concurrent external control arm for the registrational study. The Company expects to initiate a registrational study by early 2026 with a potential efficacy readout in 2027.
- 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
- LX2020 for the Treatment of PKP2-ACM: In March 2025, Lexeo shared positive interim data of LX2020 from the low-dose cohort in the HEROIC-PKP2 Phase 1/2 clinical trial.
- Cohort 1 Interim Update: At 3-months post-treatment, cardiac biopsies from two participants in cohort 1 showed
71% and115% increases, respectively, in PKP2 protein expression from baseline; the third cohort 1 participant elected not to undergo a post-treatment biopsy. The first participant evaluated 6-months post treatment experienced a67% reduction in premature ventricular contractions (PVCs) from baseline. - Safety: LX2020 has been generally well tolerated with no treatment-related serious adverse events to date across both dose cohorts.
- Next Steps: Currently enrolling cohort 3 of LX2020 HEROIC-PKP2 (n=4), with an interim clinical data update expected in the second half of 2025.
- Cohort 1 Interim Update: At 3-months post-treatment, cardiac biopsies from two participants in cohort 1 showed
- Capital Redeployment and Cash Runway: In April 2025, Lexeo identified approximately
$20 million in capital to redeploy towards the Company’s lead cardiac programs, LX2006 and LX2020. This capital was redeployed from preclinical and non-cardiac pipeline activities and included a limited reduction in force impacting approximately15% of employees. The updated capital structure is expected to enable the Company to execute against key milestones for its clinical-stage pipeline, accelerate work to initiate a registrational study for LX2006 by early 2026, and maintain operational runway into 2027.
First Quarter Financial Results
- Cash Position: As of March 31, 2025, cash, cash equivalents, and investments were
$106.9 million , which Lexeo believes will be sufficient to fund operations into 2027. - Research and Development Expenses: Research and Development expenses were
$17.2 million for the three months ended March 31, 2025, compared to$15.7 million for the three months ended March 31, 2024. - General and Administrative Expenses: General and Administrative expenses were
$16.6 million for the three months ended March 31, 2025, compared to$7.5 million for the three months ended March 31, 2024. - Net Loss: Net loss was
$32.7 million or$0.99 per share (basic and diluted) for the three months ended March 31, 2025, compared to$21.7 million or$0.77 per share (basic and diluted) for the three months ended March 31, 2024.
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 for the treatment of Friedreich ataxia (FA) cardiomyopathy, LX2020 for the treatment of plakophilin-2 (PKP2) arrhythmogenic cardiomyopathy, and others for 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, the timing and likelihood of potential regulatory developments and approval, 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; 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, 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
| Lexeo Therapeutics, Inc. Selected Financial Information (in thousands, except share and per share amounts) | |||||||
| Statements of Operations | |||||||
| Three Months Ended March 31, | |||||||
| 2025 | 2024 | ||||||
| (unaudited) | (unaudited) | ||||||
| Operating expenses | |||||||
| Research and development | $ | 17,171 | $ | 15,742 | |||
| General and administrative | 16,634 | 7,549 | |||||
| Total operating expenses | 33,805 | 23,291 | |||||
| Operating loss | (33,805 | ) | (23,291 | ) | |||
| Other income and expense | |||||||
| Other income (expense), net | (4 | ) | (5 | ) | |||
| Interest expense | (28 | ) | (37 | ) | |||
| Interest income | 1,193 | 1,651 | |||||
| Amortization of premium on investments | (12 | ) | - | ||||
| Total other income and expense | 1,149 | 1,609 | |||||
| Loss from operations before income taxes | (32,656 | ) | (21,682 | ) | |||
| Income taxes | - | - | |||||
| Net loss | $ | (32,656 | ) | $ | (21,682 | ) | |
| Net loss per common share, basic and diluted | $ | (0.99 | ) | $ | (0.77 | ) | |
| Weighted average number of shares outstanding used in computation of net loss per common share, basic and diluted | 33,113,991 | 27,979,838 | |||||
| Balance Sheet Data | |||||||
| March 31, | December 31, | ||||||
| 2025 | 2024 | ||||||
| (unaudited) | |||||||
| Cash, cash equivalents, and investments | $ | 106,866 | $ | 128,530 | |||
| Total assets | 125,690 | 146,942 | |||||
| Total liabilities | 37,575 | 30,100 | |||||
| Total stockholders' equity | 88,115 | 116,842 | |||||
FAQ
What were the key findings from LXEO's LX2006 trial in Friederich ataxia cardiomyopathy?
What is Lexeo Therapeutics' (LXEO) cash runway as of Q1 2025?
How did LXEO's Q1 2025 financial results compare to Q1 2024?
What were the interim results of LXEO's LX2020 trial for PKP2-ACM?
When does Lexeo Therapeutics (LXEO) plan to start the LX2006 registrational study?
