Lexeo Therapeutics, Inc. (Nasdaq: LXEO), a clinical stage genetic
medicine company dedicated to pioneering treatments for genetically
defined cardiovascular diseases and APOE4-associated Alzheimer’s
disease, today announced positive interim data of LX2006 for the
treatment of Friedreich ataxia (FA) cardiomyopathy. Across both the
Lexeo SUNRISE-FA Phase 1/2 clinical trial (NCT05445323) and the
Weill Cornell Medicine investigator-initiated Phase 1A trial
(NCT05302271), LX2006 was well tolerated with no treatment-related
serious adverse events, and clinically meaningful improvements in
cardiac biomarkers were observed with increasing improvement over
time.
“We are very encouraged by these data and the
potential of LX2006 to treat FA cardiomyopathy, a devastating and
fatal condition with no currently approved therapies,” said Dr.
Eric Adler, Chief Medical Officer and Head of Research at Lexeo
Therapeutics. “Based on the favorable safety profile and clinical
benefits observed to date, we are excited to explore expedited
clinical development of LX2006, including potential for accelerated
approval of this possibly life-saving treatment.”
“The interim data shared today demonstrate
clinically meaningful improvements across multiple cardiac
biomarkers of hypertrophy, a hallmark of FA cardiomyopathy,” said
Dr. Sandi See Tai, Chief Development Officer at Lexeo. “Together
with the increases in frataxin protein expression observed in
SUNRISE-FA cardiac biopsies to date, these results further
highlight the potential of LX2006 to positively impact outcomes for
people with FA cardiomyopathy. I would like to thank the
participants, caregivers, and investigators participating in these
trials who have helped to achieve this important milestone.”
FA cardiomyopathy is a devastating, rare, and
progressive disorder caused by loss of function mutations in the
frataxin gene. Thus far in participants in the SUNRISE-FA trial
with cardiac biopsies, low levels of frataxin have been found in
the heart at baseline, estimated to be 2% or less of normal. In
terms of clinical presentation, FA cardiomyopathy is typically
characterized by left ventricular hypertrophy ultimately
progressing to heart failure, and cardiac dysfunction is the cause
of death in up to 80% of individuals with FA. A new natural history
subset analysis conducted by Lexeo showed elevated left ventricular
mass index (LVMI) in adults with FA cardiomyopathy, and LVMI
remained stable or increased with age without spontaneous
improvement. Elevated LVMI is an indicator of left ventricular
hypertrophy and correlated with mortality in multiple
cardiovascular conditions including FA cardiomyopathy.
Interim Safety Results
- LX2006 was well tolerated with no treatment-related serious
adverse events to date in either study
- No signs of complement activation or other immunogenicity
observed
- No cardiac or hepatic safety signals observed
- All adverse events were transient and resolved
- No participants discontinued from either study
Interim Clinical Results (from 8
participants with > 6-months
of follow-up)
- Left ventricular mass index (LVMI): Of participants with
elevated LVMI at baseline, 75% achieved >10% reduction at 12
months (n=4). Of all participants, 50% achieved >10% reduction
in LVMI at 12 months (n=6).
- Among the participants with elevated LVMI at baseline, mean
reduction in LVMI was 11.4% at 12 months (n=4) and 18.3% at 18
months (n=2).
- Left ventricular (LV) lateral wall thickness: wall thickening,
an early indicator of left ventricular hypertrophy, was reduced by
13.6% on average in all participants at 12 months (n=6).
- High-sensitivity Troponin I (hsTnI): troponin I, a biomarker of
myocardial injury, was reduced by 53.3% on average in all
participants at 12 months (n=5).
- Frataxin protein expression evaluated via myocardial biopsy:
observed increased frataxin levels compared to baseline following
treatment with LX2006 in all participants evaluated to date
utilizing two distinct measurements:
- LCMS: frataxin increase observed in 3 of 3 evaluable
participants.
- IHC: frataxin increase observed in 2 of 2 evaluable
participants.
Dosing Update and Next Steps
- As of July 15, 2024, 13 participants dosed to date across two
trials:
- Cohort 1 (1.8x1011vg/kg): n=6
- Cohort 2 (5.6x1011 vg/kg): n=6
- Cohort 3 (1.2x1012 vg/kg): n=1
- SUNRISE-FA independent Data and Safety Monitoring Board
endorsed proceeding to Cohort 3 dose level (1.2x1012vg/kg). This
cohort has started enrollment with 1 participant dosed to date and
will include at least 3 participants.
- The Weill Cornell investigator-initiated trial is currently
enrolling in Cohort 2.
- Lexeo expects to share further details of these interim
results, including an additional cardiac biopsy from Cohort 2, at a
scientific conference in Fall 2024.
Corporate Webcast DetailsLexeo
Therapeutics will host a webcast at 8:00 AM ET today, July 15,
2024. Analysts and investors can participate by accessing the
webcast live here or 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 the completion of the
call.
About the Clinical Studies
SUNRISE-FA is a Lexeo-sponsored, multicenter, 52-week, open-label
trial evaluating the safety and preliminary efficacy of LX2006 in
people who have FA cardiomyopathy, with three ascending-dose
cohorts. Investigators at Weill Cornell Medicine are conducting a
Phase 1A study of AAVrh.10hFXN, known as LX2006 at Lexeo, in a
single-site, 52-week, open-label trial evaluating the safety and
preliminary efficacy in people who have FA cardiomyopathy, in two
ascending-dose cohorts with five participants per cohort.
About LX2006LX2006 is an AAV-based
gene therapy candidate delivered intravenously for the treatment of
FA cardiomyopathy, the most common cause of mortality in
individuals with FA affecting approximately 5,000 people in the
United States. LX2006 is designed to target the cardiac
manifestations of FA by delivering a functional frataxin gene to
promote the expression of the frataxin protein and restore
mitochondrial function in myocardial cells. In preclinical studies,
LX2006 reversed the cardiac abnormalities in FA disease models and
showed improvement in cardiac function and survival while
demonstrating a favorable safety profile. The FDA has granted Rare
Pediatric Disease designation, Fast Track designation, and Orphan
Drug designation to LX2006 for the treatment of FA
cardiomyopathy.
About Lexeo
Therapeutics Lexeo Therapeutics is a New York
City-based, clinical stage genetic medicine company dedicated to
transforming healthcare by applying pioneering science to
fundamentally change how genetically defined cardiovascular
diseases and APOE4-associated Alzheimer’s disease are treated.
Using a stepwise development approach, Lexeo is leveraging early
proof-of-concept functional and biomarker data to advance a
pipeline of cardiovascular and APOE4-associated Alzheimer’s disease
programs.
Cautionary Note Regarding Forward-Looking
StatementsCertain statements in this press release may
constitute “forward-looking statements” within the meaning of the
federal securities laws, including, but not limited to, our
expectations and plans regarding our current product candidates and
programs, including statements regarding the potential benefits of
LX2006 for the treatment of Friedreich ataxia cardiomyopathy and
the timing for receipt and announcement of data from its clinical
trials, and the timing and likelihood of potential regulatory
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 March 31, 2023, filed with
the SEC on May 9, 2024, 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:Janine Bogris (201)
245-6838 janine.bogris@inizioevoke.com
Investor Response:Stephen
Jasper(858) 525-2047stephen@gilmartinir.com
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